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Evaluation of Pazopanib on Bleeding in Subjects With Hereditary Haemorrhagic Telangiectasia

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ClinicalTrials.gov Identifier: NCT02204371
Recruitment Status : Terminated (The study was terminated as the asset was sold to another company who decided not to pursue this indication with this drug.)
First Posted : July 30, 2014
Results First Posted : July 6, 2017
Last Update Posted : July 6, 2017
Sponsor:
Information provided by (Responsible Party):
GlaxoSmithKline

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Telangiectasia, Hereditary Hemorrhagic
Interventions Drug: Pazopanib
Drug: Placebo
Enrollment 7
Recruitment Details This study was planned as a two part study (Part A and Part B). As efficacy based dose escalation stopping criteria was met, there was only one dose cohort (50 milligrams [mg] once daily cohort) in Part A. The study was stopped prior to Part B being conducted.
Pre-assignment Details Run-in period started at the screening visit or up to 2 weeks after the screening visit. Participants (par.) were stabilized on a anemia management plan for 4-8 weeks prior to the first dose. Unstable par. were allowed to continue in the run-in period for additional 4 weeks. 2 par. did not met the eligibility criteria.
Arm/Group Title GW786034 50 mg Once Daily
Hide Arm/Group Description Participants with hereditary hemorrhagic telangiectasia (HHT) received GW786034 50 mg tablet orally once daily at least one hour before or two hours after a meal in the morning for up to 12 weeks in the open label non-randomized phase.
Period Title: Overall Study
Started 7
Completed 6
Not Completed 1
Reason Not Completed
Adverse Event             1
Arm/Group Title GW786034 50 mg Once Daily
Hide Arm/Group Description Participants with hereditary hemorrhagic telangiectasia (HHT) received GW786034 50 mg tablet orally once daily at least one hour before or two hours after a meal in the morning for up to 12 weeks in the open label non-randomized phase.
Overall Number of Baseline Participants 7
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 7 participants
57.3  (9.14)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 7 participants
Female
4
  57.1%
Male
3
  42.9%
Race/Ethnicity, Customized  
Measure Type: Number
Unit of measure:  Participants
White - White/Caucasian/European Heritage Number Analyzed 7 participants
7
1.Primary Outcome
Title Change From Baseline in Epistaxis Severity Score at the Indicated Time Points
Hide Description The Epistaxis (nose bleeding) severity score (ESS) is a 6-item par-reported outcome measure designed to be a uniform epistaxis severity scoring system to assess the effectiveness of specific treatments on HHT-related epistaxis. Four questions document epistaxis frequency, duration, intensity and need for treatment, whereas two additional questions detail the presence of anemia and if a par has required a blood transfusion as a consequence of their epistaxis. Questions are variably weighted and results are tabulated on a 0-10 scale (0=no disease, 10 = severe disease). The minimum important difference is 0.71. Baseline is the Day1 pre-dose assessment value. Change from Baseline is calculated as the Post dose value at the indicated visit minus the Baseline value. Par were evaluated at Baseline, Treatment period (Weeks 6 and 12) and Follow-up period (Weeks 16, 20, 24 and 28). Only those par available at the indicated timepoints were analysed (specified by n=X in the category titles).
Time Frame Baseline, Week 6, Week 12, Week 16, Week 20, Week 24 and Week 28
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacodynamic Population: All par. who received at least one dose of study treatment (Safety Population) and who also provided data from at least one pharmacodynamic assessment (hemoglobin, ferritin, epistaxis daily diary).
Arm/Group Title GW786034 50 mg Once Daily
Hide Arm/Group Description:
Participants with hereditary hemorrhagic telangiectasia (HHT) received GW786034 50 mg tablet orally once daily at least one hour before or two hours after a meal in the morning for up to 12 weeks in the open label non-randomized phase.
Overall Number of Participants Analyzed 7
Mean (Standard Deviation)
Unit of Measure: Scores on a scale
Week 6, n=7 -0.59  (0.802)
Week 12, n=6 -1.16  (1.023)
Follow-up Week 16, n=7 -0.60  (1.299)
Follow-up Week 20, n=6 -1.41  (1.674)
Follow-up Week 24, n=5 -1.27  (1.519)
Follow-up Week 28, n=5 -0.08  (1.920)
2.Primary Outcome
Title Change From Baseline in the Average of the Last 3 Hemoglobin Measures in the Dosing Period (Week 9, Week 10.5 and Week 12)
Hide Description For post-Baseline hemoglobin assessments, average of the last 3 measurements of the dosing period (Weeks 9, 10.5 and 12) was computed. Only pre-transfusion hemoglobin values have been included in the analyses. Baseline hemoglobin value is the average of the last two measurements during the run-in period. . Average of the last two measurements during the run-in period was calculated as sum of the last 2 measured values of hemoglobin divided by 2. Change from Baseline was calculated as the average of the last 3 measured values of hemoglobin minus the Baseline value. Average of the last 3 measurements was calculated as sum of the last 3 measured values of hemoglobin divided by 3. If measurements were missing at one or two of the 3 visits at Weeks 9, 10.5 and 12, then the average was based on the available measurements.
Time Frame Baseline, Week 9, Week 10.5 and Week 12
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacodynamic Population. Only those par. available at the indicated time points were analyzed.
Arm/Group Title GW786034 50 mg Once Daily
Hide Arm/Group Description:
Participants with hereditary hemorrhagic telangiectasia (HHT) received GW786034 50 mg tablet orally once daily at least one hour before or two hours after a meal in the morning for up to 12 weeks in the open label non-randomized phase.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: Grams per liter (g/L)
7.58  (17.543)
3.Primary Outcome
Title Change From Baseline in Hemoglobin at the Indicated Time Points
Hide Description Only pre-transfusion hemoglobin values have been included in the analyses. All hemoglobin values that fall within 5 days of packed red blood cells (PRBC) transfusion are considered as post-transfusion values. Baseline hemoglobin value is defined as the average of the last two measurements during the run-in period. Average of the last two measurements during the run-in period was calculated as sum of the last 2 measured values of hemoglobin divided by 2. Change from Baseline was calculated as the Post dose value at the indicated visit minus the Baseline value. Par. were evaluated at Treatment period (Weeks 1.5, 3, 4.5, 6, 7.5, 9, 10.5 and 12) and Follow-up period (Weeks 16, 20, 24 and 28).
Time Frame Baseline, Week 1.5, Week 3, Week 4.5, Week 6, Week 7.5, Week 9, Week 10.5, Week 12, Week 16, Week 20, Week 24 and Week 28
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacodynamic Population. Only those par. available at the indicated time points were analyzed (specified by n=X in the category titles).
Arm/Group Title GW786034 50 mg Once Daily
Hide Arm/Group Description:
Participants with hereditary hemorrhagic telangiectasia (HHT) received GW786034 50 mg tablet orally once daily at least one hour before or two hours after a meal in the morning for up to 12 weeks in the open label non-randomized phase.
Overall Number of Participants Analyzed 7
Mean (Standard Deviation)
Unit of Measure: g/L
Week 1.5, n=7 7.50  (11.113)
Week 3, n=6 11.92  (13.492)
Week 4.5, n=7 7.50  (22.042)
Week 6, n=7 6.48  (19.967)
Week 7.5, n=6 8.83  (12.770)
Week 9, n=6 6.67  (14.865)
Week 10.5, n=6 7.50  (15.485)
Week 12, n=6 8.58  (25.150)
Follow-Up Week 16, n=7 7.00  (22.739)
Follow-Up Week 20, n=6 7.56  (19.212)
Follow-Up Week 24, n=5 4.93  (11.086)
Follow-Up Week 28, n=5 -3.80  (7.926)
4.Primary Outcome
Title Duration of Epistaxis Over the Last 2 Weeks of the Dosing Period and by Time Over the Entire Dosing and Follow-up Period by 2 Week Interval (From Daily Diaries)
Hide Description Duration of epistaxis based on daily diaries has been reported over Baseline, On-Therapy (OT) to Follow-up (F). Individual participant data from the daily diaries has been reported.
Time Frame Over last 2 weeks of the run-in phase and then 2 week intervals throughout treatment period and follow-up period (from daily diaries)
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacodynamic Population
Arm/Group Title GW786034 50 mg Once Daily
Hide Arm/Group Description:
Participants with hereditary hemorrhagic telangiectasia (HHT) received GW786034 50 mg tablet orally once daily at least one hour before or two hours after a meal in the morning for up to 12 weeks in the open label non-randomized phase.
Overall Number of Participants Analyzed 7
Measure Type: Number
Unit of Measure: Minutes
Participant 1, Baseline 26
Participant 1, OT Day1-13 28
Participant 1, OT Day 14-27 32
Participant 1, OT Day 28-41 32
Participant 1, OT Day 42-55 34
Participant 1, OT Day 56-69 26
Participant 1, OT Day 70 - 83 30
Participant 1, F Day 1-14 22
Participant 1, F Day 15 - 28 28
Participant 1, F Day 29 - 42 34
Participant 1, F Day 43 - 56 43
Participant 1, F Day 57 - 70 33
Participant 1, F Day 71 - 84 27
Participant 1, F Day 85 - 98 46
Participant 1, F Day 99 - 112 66
Participant 2, Baseline 846
Participant 2, OT Day1-5 301
Participant 2, OT Day 6 - 19 966
Participant 2, OT Day 20 - 33 1288
Participant 2, OT Day 34 - 47 980
Participant 2, OT Day 48 - 61 589
Participant 2, OT Day 62 - 75 594
Participant 2, OT Day 76 - 89 386
Participant 2, F Day 1 - 14 240
Participant 2, F Day 15 - 28 191
Participant 2, F Day 29 - 42 426
Participant 2, F Day 43 - 56 261
Participant 2, F Day 57 - 70 236
Participant 2, F Day 71 - 84 115
Participant 2, F Day 85 - 98 467
Participant 2, F Day 99 14
Participant 3, Baseline 130
Participant 3, OT Day1 - 14 210
Participant 3, OT Day 15 - 28 132
Participant 3, OT Day 29 - 42 118
Participant 3, OT Day 43 - 56 84
Participant 3, OT Day57 - 70 65
Participant 3, OT Day71 - 84 68
Participant , F Day 1 - 14 127
Participant 3, F Day 15 - 28 8
Participant 3, F Day 29 - 42 48
Participant 3, F Day 43 - 56 63
Participant 3, F Day 57 - 70 70
Participant 3, F Day 71 - 84 36
Participant 3, F Day 85 - 98 51
Participant 3, F Day 99 - 112 46
Participant 3, F Day 113 - 115 5
Participant 4, Baseline 269
Participant 4, OT Day1 30
Participant 4, OT Day 2 - 15 312
Participant 4, OT Day 16 - 29 125
Participant 4, OT Day 30 - 43 122
Participant 4, OT Day 44 - 57 93
Participant 4, OT Day 58 - 71 40
Participant 4, OT Day 72 - 85 40
Participant 4, F Day 1-14 55
Participant 4, F Day 15 - 28 84
Participant 4, F Day 29 - 42 232
Participant 4, F Day 43 - 56 95
Participant 4, F Day 57 - 70 140
Participant 4, F Day 71 - 84 79
Participant 4, F Day 85 - 98 104
Participant 4, F Day 99 - 112 165
Participant 4, F Day 113 - 120 127
Participant 5, Baseline 361
Participant 5, OT Day 1 - 2 63
Participant 5 OT Day 3 - 16 378
Participant 5, OT Day 17 - 30 404
Participant 5, OT Day 31 - 44 396
Participant 5, OT Day 45 - 58 211
Participant 5, OT Day 59 - 72 278
Participant 5, OT Day 73 - 86 202
Participant 5, F Day 1-14 224
Participant 5, F Day 15 - 28 130
Participant 5, F Day 29 - 42 163
Participant 5, F Day 43 - 56 151
Participant 5, F Day 57 - 70 207
Participant 5, F Day 71 - 84 170
Participant 5, F Day 85 - 98 176
Participant 5, F Day 99 - 112 253
Participant 5, F Day 113 - 118 94
Participant 6, Baseline 26
Participant 6, OT Day 1 0
Participant 6, OT Day 2 - 15 12
Participant 6, OT Day 16 - 29 28
Participant 6, OT Day 30 - 43 19
Participant 6, F Day 1-14 19
Participant 6, F Day 15 - 28 15
Participant 6, F Day 29 - 42 8
Participant 6, F Day 43 - 56 26
Participant 7, Baseline 213
Participant 7, OT Day 1 - 14 190
Participant 7, OT Day 15 - 28 58
Participant 7, OT Day 29 - 42 110
Participant 7, OT Day 43 - 56 15
Participant 7, OT Day 57 - 70 36
Participant 7, OT Day 71 - 84 53
Participant 7, F Day 1-14 40
Participant 7, F Day 15 - 28 114
Participant 7, F Day 29 - 42 68
Participant 7, F Day 43 - 54 87
5.Primary Outcome
Title Frequency of Epistaxis Over the Last 2 Weeks of the Dosing Period and by Time Over the Entire Dosing and Follow-up Period by 2 Week Interval (From Daily Diaries)
Hide Description Frequency of epistaxis based on daily diaries has been reported over Baseline, On-Therapy (OT) to Follow-up (F). Individual participant data from the daily diaries has been reported.
Time Frame Over last 2 weeks of the run-in phase and then 2 week intervals throughout treatment period and follow-up period (from daily diaries)
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacodynamic Population
Arm/Group Title GW786034 50 mg Once Daily
Hide Arm/Group Description:
Participants with hereditary hemorrhagic telangiectasia (HHT) received GW786034 50 mg tablet orally once daily at least one hour before or two hours after a meal in the morning for up to 12 weeks in the open label non-randomized phase.
Overall Number of Participants Analyzed 7
Measure Type: Number
Unit of Measure: Number of nosebleeds
Participant 1, Baseline 21
Participant 1, OT Day1-13 23
Participant 1, OT Day 14-27 23
Participant 1, OT Day 28-41 22
Participant 1, OT Day 42-55 23
Participant 1, OT Day 56-69 20
Participant 1, OT Day 70 - 83 25
Participant 1, F Day 1-14 23
Participant 1, F Day 15 - 28 24
Participant 1, F Day 29 - 42 28
Participant 1, F Day 43 - 56 30
Participant 1, F Day 57 - 70 21
Participant 1, F Day 71 - 84 23
Participant 1, F Day 85 - 98 31
Participant 1, F Day 99 - 112 34
Participant 2, Baseline 42
Participant 2, OT Day1-5 17
Participant 2, OT Day 6 - 19 42
Participant 2, OT Day 20 - 33 55
Participant 2, OT Day 34 - 47 44
Participant 2, OT Day 48 - 61 32
Participant 2, OT Day 62 - 75 42
Participant 2, OT Day 76 - 89 37
Participant 2, F Day 1 - 14 32
Participant 2, F Day 15 - 28 21
Participant 2, F Day 29 - 42 28
Participant 2, F Day 43 - 56 28
Participant 2, F Day 57 - 70 33
Participant 2, F Day 71 - 84 28
Participant 2, F Day 85 - 98 34
Participant 2, F Day 99 3
Participant 3, Baseline 11
Participant 3, OT Day1 - 14 17
Participant 3, OT Day 15 - 28 16
Participant 3, OT Day 29 - 42 13
Participant 3, OT Day 43 - 56 11
Participant 3, OT Day57 - 70 8
Participant 3, OT Day71 - 84 13
Participant , F Day 1 - 14 12
Participant 3, F Day 15 - 28 3
Participant 3, F Day 29 - 42 8
Participant 3, F Day 43 - 56 11
Participant 3, F Day 57 - 70 11
Participant 3, F Day 71 - 84 5
Participant 3, F Day 85 - 98 5
Participant 3, F Day 99 - 112 9
Participant 3, F Day 113 - 115 2
Participant 4, Baseline 27
Participant 4, OT Day1 1
Participant 4, OT Day 2 - 15 28
Participant 4, OT Day 16 - 29 18
Participant 4, OT Day 30 - 43 19
Participant 4, OT Day 44 - 57 17
Participant 4, OT Day 58 - 71 7
Participant 4, OT Day 72 - 85 6
Participant 4, F Day 1-14 6
Participant 4, F Day 15 - 28 9
Participant 4, F Day 29 - 42 18
Participant 4, F Day 43 - 56 12
Participant 4, F Day 57 - 70 19
Participant 4, F Day 71 - 84 11
Participant 4, F Day 85 - 98 12
Participant 4, F Day 99 - 112 14
Participant 4, F Day 113 - 120 10
Participant 5, Baseline 35
Participant 5, OT Day 1 - 2 6
Participant 5 OT Day 3 - 16 33
Participant 5, OT Day 17 - 30 37
Participant 5, OT Day 31 - 44 34
Participant 5, OT Day 45 - 58 28
Participant 5, OT Day 59 - 72 34
Participant 5, OT Day 73 - 86 30
Participant 5, F Day 1-14 30
Participant 5, F Day 15 - 28 19
Participant 5, F Day 29 - 42 22
Participant 5, F Day 43 - 56 23
Participant 5, F Day 57 - 70 28
Participant 5, F Day 71 - 84 25
Participant 5, F Day 85 - 98 27
Participant 5, F Day 99 - 112 31
Participant 5, F Day 113 - 118 9
Participant 6, Baseline 18
Participant 6, OT Day 1 0
Participant 6, OT Day 2 - 15 13
Participant 6, OT Day 16 - 29 28
Participant 6, OT Day 30 - 43 19
Participant 6, F Day 1-14 17
Participant 6, F Day 15 - 28 13
Participant 6, F Day 29 - 42 9
Participant 6, F Day 43 - 56 17
Participant 7, Baseline 13
Participant 7, OT Day 1 - 14 9
Participant 7, OT Day 15 - 28 5
Participant 7, OT Day 29 - 42 11
Participant 7, OT Day 43 - 56 5
Participant 7, OT Day 57 - 70 5
Participant 7, OT Day 71 - 84 6
Participant 7, F Day 1-14 7
Participant 7, F Day 15 - 28 12
Participant 7, F Day 29 - 42 7
Participant 7, F Day 43 - 54 6
6.Primary Outcome
Title Intensity of Epistaxis Over the Last 2 Weeks of the Dosing Period and by Time Over the Entire Dosing and Follow-up Period by 2 Week Interval (From Daily Diaries)
Hide Description Intensity of epistaxis based on daily diaries has been reported as total gushing and total non gushing from Baseline, On-Therapy (OT) to Follow-up (F). Individual participant data from the daily diaries has been reported.
Time Frame Over last 2 weeks of the run-in phase and then 2 week intervals throughout treatment period and follow-up period (from daily diaries)
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacodynamic Population
Arm/Group Title GW786034 50 mg Once Daily
Hide Arm/Group Description:
Participants with hereditary hemorrhagic telangiectasia (HHT) received GW786034 50 mg tablet orally once daily at least one hour before or two hours after a meal in the morning for up to 12 weeks in the open label non-randomized phase.
Overall Number of Participants Analyzed 7
Measure Type: Number
Unit of Measure: Number of gushing/non-gushing nosebleeds
Participant 1, Baseline, Total Gushing 2
Participant 1, Baseline, Total Non-Gushing 19
Participant 1, OT Day1-13, Total Gushing 0
Participant 1, OT Day1-13, Total Non-Gushing 23
Participant 1, OT Day 14-27, Total Gushing 3
Participant 1, OT Day 14-27, Total Non-Gushing 20
Participant 1, OT Day 28-41, Total Gushing 4
Participant 1, OT Day 28-41, Total Non-Gushing 18
Participant 1, OT Day 42-55, Total Gushing 3
Participant 1, OT Day 42-55, Total Non-Gushing 20
Participant 1, OT Day 56-69, Total Gushing 2
Participant 1, OT Day 56-69, Total Non-Gushing 18
Participant 1, OT Day 70 - 83, Total Gushing 2
Participant 1, OT Day 70 - 83, Total Non-Gushing 23
Participant 1, F Day 1-14, Total Gushing 0
Participant 1, F Day 1-14, Total Non-Gushing 23
Participant 1, F Day 15 - 28, Total Gushing 2
Participant 1, F Day 15 - 28, Total Non-Gushing 22
Participant 1, F Day 29 - 42 , Total Gushing 0
Participant 1, F Day 29 - 42 , Total Non-Gushing 28
Participant 1, F Day 43 - 56, Total Gushing 5
Participant 1, F Day 43 - 56, Total Non-Gushing 25
Participant 1, F Day 57 - 70 , Total Gushing 4
Participant 1, F Day 57 - 70 , Total Non-Gushing 17
Participant 1, F Day 71 - 84 , Total Gushing 0
Participant 1, F Day 71 - 84 , Total Non-Gushing 23
Participant 1, F Day 85 - 98 , Total Gushing 4
Participant 1, F Day 85 - 98 , Total Non-Gushing 27
Participant 1, F Day 99 - 112 , Total Gushing 6
Participant 1, F Day 99 - 112 , Total Non-Gushing 28
Participant 2, Baseline, Total Gushing 25
Participant 2, Baseline, Total Non-Gushing 17
Participant 2, OT Day1-5, Total Gushing 5
Participant 2, OT Day1-5, Total Non-Gushing 12
Participant 2, OT Day 6 - 19 , Total Gushing 24
Participant 2, OT Day 6 - 19 , Total Non-Gushing 18
Participant 2, OT Day 20 - 33 , Total Gushing 17
Participant 2, OT Day 20 - 33 , Total Non-Gushing 38
Participant 2, OT Day 34 - 47, Total Gushing 10
Participant 2, OT Day 34 - 47 , Total Non-Gushing 34
Participant 2, OT Day 48 - 61 , Total Gushing 10
Participant 2, OT Day 48 - 61 , Total Non-Gushing 22
Participant 2, OT Day 62 - 75 , Total Gushing 20
Participant 2, OT Day 62 - 75 , Total Non-Gushing 22
Participant 2, OT Day 76 - 89 , Total Gushing 17
Participant 2, OT Day 76 - 89 , Total Non-Gushing 20
Participant 2, F Day 1 - 14 , Total Gushing 14
Participant 2, F Day 1 - 14 , Total Non-Gushing 18
Participant 2, F Day 15 - 28 , Total Gushing 14
Participant 2, F Day 15 - 28 , Total Non-Gushing 7
Participant 2, F Day 29 - 42 , Total Gushing 18
Participant 2, F Day 29 - 42 , Total Non-Gushing 10
Participant 2, F Day 43 - 56 , Total Gushing 17
Participant 2, F Day 43 - 56 , Total Non-Gushing 11
Participant 2, F Day 57 - 70 , Total Gushing 14
Participant 2, F Day 57 - 70 , Total Non-Gushing 19
Participant 2, F Day 71 - 84 , Total Gushing 9
Participant 2, F Day 71 - 84 , Total Non-Gushing 19
Participant 2, F Day 85 - 98 , Total Gushing 13
Participant 2, F Day 85 - 98 , Total Non-Gushing 21
Participant 2, F Day 99 , Total Gushing 1
Participant 2, F Day 99, Total Non-Gushing 2
Participant 3, Baseline , Total Gushing 10
Participant 3, Baseline, Total Non-Gushing 1
Participant 3, OT Day1 - 14 , Total Gushing 8
Participant 3, OT Day1 - 14 , Total Non-Gushing 9
Participant 3, OT Day 15 - 28 , Total Gushing 7
Participant 3, OT Day 15 - 28 , Total Non-Gushing 9
Participant 3, OT Day 29 - 42 , Total Gushing 12
Participant 3, OT Day 29 - 42 , Total Non-Gushing 1
Participant 3, OT Day 43 - 56 , Total Gushing 6
Participant 3, OT Day 43 - 56 , Total Non-Gushing 5
Participant 3, OT Day 57 - 70 , Total Gushing 4
Participant 3, OT Day 57 - 70 , Total Non-Gushing 4
Participant 3, OT Day 71 - 84 , Total Gushing 7
Participant 3, OT Day 71 - 84 , Total Non-Gushing 6
Participant , F Day 1 - 14 , Total Gushing 9
Participant , F Day 1 - 14 , Total Non-Gushing 3
Participant 3, F Day 15 - 28 , Total Gushing 0
Participant 3, F Day 15 - 28 , Total Non-Gushing 3
Participant 3, F Day 29 - 42, Total Gushing 4
Participant 3, F Day 29 - 42 , Total Non-Gushing 4
Participant 3, F Day 43 - 56 , Total Gushing 7
Participant 3, F Day 43 - 56 , Total Non-Gushing 4
Participant 3, F Day 57 - 70 , Total Gushing 6
Participant 3, F Day 57 - 70 , Total Non-Gushing 5
Participant 3, F Day 71 - 84 , Total Gushing 4
Participant 3, F Day 71 - 84 , Total Non-Gushing 1
Participant 3, F Day 85 - 98 , Total Gushing 4
Participant 3, F Day 85 - 98 , Total Non-Gushing 1
Participant 3, F Day 99 - 112 , Total Gushing 6
Participant 3, F Day 99 - 112 , Total Non-Gushing 3
Participant 3, F Day 113 - 115 , Total Gushing 0
Participant 3, F Day 113 - 115 , Total Non-Gushing 2
Participant 4, Baseline, Total Gushing 3
Participant 4, Baseline, Total Non-Gushing 24
Participant 4, OT Day1, Total Gushing 1
Participant 4, OT Day1, Total Non-Gushing 0
Participant 4, OT Day 2 - 15 , Total Gushing 9
Participant 4, OT Day 2 - 15 , Total Non-Gushing 19
Participant 4, OT Day 16 - 29 , Total Gushing 2
Participant 4, OT Day 16 - 29 , Total Non-Gushing 16
Participant 4, OT Day 30 - 43 , Total Gushing 2
Participant 4, OT Day 30 - 43 , Total Non-Gushing 17
Participant 4, OT Day 44 - 57 , Total Gushing 2
Participant 4, OT Day 44 - 57 , Total Non-Gushing 15
Participant 4, OT Day 58 - 71 , Total Gushing 1
Participant 4, OT Day 58 - 71 , Total Non-Gushing 6
Participant 4, OT Day 72 - 85 , Total Gushing 0
Participant 4, OT Day 72 - 85 , Total Non-Gushing 6
Participant 4, F Day 1-14, Total Gushing 0
Participant 4, F Day 1-14, Total Non-Gushing 6
Participant 4, F Day 15 - 28 , Total Gushing 0
Participant 4, F Day 15 - 28 , Total Non-Gushing 9
Participant 4, F Day 29 - 42 , Total Gushing 6
Participant 4, F Day 29 - 42 , Total Non-Gushing 12
Participant 4, F Day 43 - 56 , Total Gushing 2
Participant 4, F Day 43 - 56 , Total Non-Gushing 10
Participant 4, F Day 57 - 70 , Total Gushing 3
Participant 4, F Day 57 - 70 , Total Non-Gushing 16
Participant 4, F Day 71 - 84 , Total Gushing 0
Participant 4, F Day 71 - 84 , Total Non-Gushing 11
Participant 4, F Day 85 - 98 , Total Gushing 3
Participant 4, F Day 85 - 98 , Total Non-Gushing 9
Participant 4, F Day 99 - 112 , Total Gushing 3
Participant 4, F Day 99 - 112 , Total Non-Gushing 11
Participant 4, F Day 113 - 120 , Total Gushing 2
Participant 4, F Day 113 - 120 , Total Non-Gushing 8
Participant 5, Baseline, Total Gushing 13
Participant 5, Baseline, Total Non-Gushing 22
Participant 5, OT Day 1 - 2 , Total Gushing 2
Participant 5, OT Day 1 - 2 , Total Non-Gushing 4
Participant 5 OT Day 3 - 16 , Total Gushing 11
Participant 5 OT Day 3 - 16 , Total Non-Gushing 22
Participant 5, OT Day 17 - 30 , Total Gushing 15
Participant 5, OT Day 17 - 30 , Total Non-Gushing 22
Participant 5, OT Day 31 - 44 , Total Gushing 11
Participant 5, OT Day 31 - 44 , Total Non-Gushing 23
Participant 5, OT Day 45 - 58 , Total Gushing 4
Participant 5, OT Day 45 - 58 , Total Non-Gushing 24
Participant 5, OT Day 59 - 72 , Total Gushing 6
Participant 5, OT Day 59 - 72 , Total Non-Gushing 28
Participant 5, OT Day 73 - 86, Total Gushing 4
Participant 5, OT Day 73 - 86 , Total Non-Gushing 26
Participant 5, F Day 1-14, Total Gushing 3
Participant 5, F Day 1-14, Total Non-Gushing 27
Participant 5, F Day 15 - 28 , Total Gushing 4
Participant 5, F Day 15 - 28 , Total Non-Gushing 15
Participant 5, F Day 29 - 42, Total Gushing 12
Participant 5, F Day 29 - 42 , Total Non-Gushing 10
Participant 5, F Day 43 - 56 , Total Gushing 6
Participant 5, F Day 43 - 56 , Total Non-Gushing 17
Participant 5, F Day 57 - 70 , Total Gushing 13
Participant 5, F Day 57 - 70 , Total Non-Gushing 15
Participant 5, F Day 71 - 84 , Total Gushing 9
Participant 5, F Day 71 - 84 , Total Non-Gushing 16
Participant 5, F Day 85 - 98 , Total Gushing 11
Participant 5, F Day 85 - 98 , Total Non-Gushing 16
Participant 5, F Day 99 - 112 , Total Gushing 13
Participant 5, F Day 99 - 112 , Total Non-Gushing 18
Participant 5, F Day 113 - 118 , Total Gushing 5
Participant 5, F Day 113 - 118 , Total Non-Gushing 4
Participant 6, Baseline, Total Gushing 0
Participant 6, Baseline, Total Non-Gushing 18
Participant 6, OT Day 1, Total Gushing 0
Participant 6, OT Day 1 , Total Non-Gushing 0
Participant 6, OT Day 2 - 15 , Total Gushing 0
Participant 6, OT Day 2 - 15 , Total Non-Gushing 13
Participant 6, OT Day 16 - 29 , Total Gushing 0
Participant 6, OT Day 16 - 29 , Total Non-Gushing 28
Participant 6, OT Day 30 - 43 , Total Gushing 0
Participant 6, OT Day 30 - 43 , Total Non-Gushing 19
Participant 6, F Day 1-14, Total Gushing 0
Participant 6, F Day 1-14, Total Non-Gushing 17
Participant 6, F Day 15 - 28 , Total Gushing 0
Participant 6, F Day 15 - 28 , Total Non-Gushing 13
Participant 6, F Day 29 - 42 , Total Gushing 0
Participant 6, F Day 29 - 42 , Total Non-Gushing 9
Participant 6, F Day 43 - 56 , Total Gushing 0
Participant 6, F Day 43 - 56 , Total Non-Gushing 17
Participant 7, Baseline, Total Gushing 3
Participant 7, Baseline, Total Non-Gushing 10
Participant 7, OT Day 1 - 14 , Total Gushing 3
Participant 7, OT Day 1 - 14 , Total Non-Gushing 6
Participant 7, OT Day 15 - 28 , Total Gushing 0
Participant 7, OT Day 15 - 28 , Total Non-Gushing 5
Participant 7, OT Day 29 - 42 , Total Gushing 2
Participant 7, OT Day 29 - 42 , Total Non-Gushing 9
Participant 7, OT Day 43 - 56 , Total Gushing 2
Participant 7, OT Day 43 - 56 , Total Non-Gushing 3
Participant 7, OT Day 57 - 70 , Total Gushing 1
Participant 7, OT Day 57 - 70 , Total Non-Gushing 4
Participant 7, OT Day 71 - 84 , Total Gushing 3
Participant 7, OT Day 71 - 84 , Total Non-Gushing 3
Participant 7, F Day 1-14, Total Gushing 2
Participant 7, F Day 1-14, Total Non-Gushing 5
Participant 7, F Day 15 - 28 , Total Gushing 6
Participant 7, F Day 15 - 28 , Total Non-Gushing 6
Participant 7, F Day 29 - 42 , Total Gushing 3
Participant 7, F Day 29 - 42 , Total Non-Gushing 4
Participant 7, F Day 43 - 54 , Total Gushing 3
Participant 7, F Day 43 - 54 , Total Non-Gushing 3
7.Primary Outcome
Title Total Iron Intake Over the Last 4 Weeks of the Dosing Period
Hide Description Total iron intake at Baseline is defined as the sum total of iron intake (oral + intravenous infusion) during the last 4 weeks of run-in period (i.e., Day -28 to Day -1). Total iron intake over the last 4 weeks of run-in and during last 4 weeks of dosing period was listed. Individual participant data has been reported.
Time Frame Last 4 weeks of run-in and during last 4 weeks of dosing period
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacodynamic Population
Arm/Group Title GW786034 50 mg Once Daily
Hide Arm/Group Description:
Participants with hereditary hemorrhagic telangiectasia (HHT) received GW786034 50 mg tablet orally once daily at least one hour before or two hours after a meal in the morning for up to 12 weeks in the open label non-randomized phase.
Overall Number of Participants Analyzed 7
Measure Type: Number
Unit of Measure: Milligram
Participant 1, Baseline 300
Participant 1, Dosing period, Day 1 - 27 300
Participant 1, Dosing period, Day 28 - 55 300
Participant 1, Dosing period, Day 56 - 83 600
Participant 2, Baseline 1000
Participant 2, Dosing period, Day 1 - 5 0
Participant 2, Dosing period, Day 6 - 33 2000
Participant 2, Dosing period, Day 34 - 61 1000
Participant 2, Dosing period, Day 62 - 89 1000
Participant 3, Baseline 3000
Participant 3, Dosing period, Day 1 - 28 1000
Participant 3, Dosing period, Day 29 - 56 1000
Participant 3, Dosing period, Day 57 - 84 2000
Participant 4, Baseline 800
Participant 4, Dosing period, Day 1 0
Participant 4, Dosing period, Day 2 - 29 800
Participant 4, Dosing period, Day 30 - 57 400
Participant 4, Dosing period, Day 58 - 85 300
Participant 5, Baseline 750
Participant 5, Dosing period, Day 1 - 2 0
Participant 5, Dosing period, Day 3 - 30 0
Participant 5, Dosing period, Day 31 - 58 1500
Participant 5, Dosing period, Day 59 - 86 0
Participant 6, Baseline 4150
Participant 6, Dosing period, Day 1 - 15 2460
Participant 6, Dosing period, Day 16 - 43 3640
Participant 7, Baseline 510
Participant 7, Dosing period, Day 1 - 28 510
Participant 7, Dosing period, Day 29 - 56 0
Participant 7, Dosing period, Day 57 - 84 1020
8.Primary Outcome
Title Total Iron Intake Over the Entire Dosing and Follow-up Period by 4 Week Interval
Hide Description Total iron intake at Baseline is defined as the sum total of iron intake (oral + intravenous infusion) during the last 4 weeks of run-in period (i.e., Day -28 to Day -1). Total iron intake over the entire dosing and follow-up period was listed by 4 week interval. Individual participant data has been reported.
Time Frame Last 4 weeks of run-in and during last 4 weeks of dosing period
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacodynamic Population
Arm/Group Title GW786034 50 mg Once Daily
Hide Arm/Group Description:
Participants with hereditary hemorrhagic telangiectasia (HHT) received GW786034 50 mg tablet orally once daily at least one hour before or two hours after a meal in the morning for up to 12 weeks in the open label non-randomized phase.
Overall Number of Participants Analyzed 7
Measure Type: Number
Unit of Measure: Milligram
Participant 1, Baseline 300
Participant 1, Dosing period, Day 1 - 27 300
Participant 1, Dosing period, Day 28 - 55 300
Participant 1, Dosing period, Day 56 - 83 600
Participant 1, Follow up, Day 1 - 28 0
Participant 1, Follow up, Day 29 - 56 300
Participant 1, Follow up, Day 57 - 84 600
Participant 1, Follow up, Day 85 - 112 0
Participant 2, Baseline 1000
Participant 2, Dosing period, Day 1 - 5 0
Participant 2, Dosing period, Day 6 - 33 2000
Participant 2, Dosing period, Day 34 - 61 1000
Participant 2, Dosing period, Day 62 - 89 1000
Participant 2, Follow up, Day 1 - 28 1000
Participant 2, Follow up, Day 29 - 56 500
Participant 2, Follow up, Day 57 - 84 1500
Participant 2, Follow up, Day 85 - 106 500
Participant 3, Baseline 3000
Dosing period, Day 1 - 28 1000
Participant 3, Dosing period, Day 29 - 56 1000
Participant 3, Dosing period, Day 57 - 84 2000
Participant 3, Follow up, Day 1 - 28 3000
Participant 3, Follow up, Day 29 - 56 1000
Participant 3, Follow up, Day 57 - 84 2000
Participant 3, Follow up, Day 85 - 112 2000
Participant 3, Follow up, Day 113 1000
Participant 4, Baseline 800
Participant 4, Dosing period, Day 1 0
Participant 4, Dosing period, Day 2 - 29 800
Participant 4, Dosing period, Day 30 - 57 400
Participant 4, Dosing period, Day 58 - 85 300
Participant 4, Follow up, Day 1 - 28 100
Participant 4, Follow up, Day 29 - 56 200
Participant 4, Follow up, Day 57 - 84 200
Participant 4, Follow up, Day 85 - 112 200
Participant 4, Follow up, Day 113 0
Participant 5, Baseline 750
Participant 5, Dosing period, Day 1 - 2 0
Participant 5, Dosing period, Day 3 - 30 0
Participant 5, Dosing period, Day 31 - 58 1500
Participant 5, Dosing period, Day 59 - 86 0
Participant 5, Follow up, Day 1 - 28 0
Participant 5, Follow up, Day 29 - 56 750
Participant 5, Follow up, Day 57 - 84 0
Participant 5, Follow up, Day 85 - 112 750
Participant 5, Follow up, Day 113 0
Participant 6, Baseline 4150
Participant 6, Dosing period, Day 1 - 15 2460
Participant 6, Dosing period, Day 16 - 43 3640
Participant 6, Follow up, Day 1 - 28 3640
Participant 6, Follow up, Day 29 - 56 4660
Participant 7, Baseline 510
Participant 7, Dosing period, Day 1 - 28 510
Participant 7, Dosing period, Day 29 - 56 0
Dosing period, Day 57 - 84 1020
Participant 7, Follow up, Day 1 - 28 0
Participant 7, Follow up, Day 29 - 55 1020
9.Primary Outcome
Title Total Units of Packed Red Blood Cells (PRBCs) Transfused During the Entire Dosing and Follow-up Period by 4 Week Interval
Hide Description Baseline PRBC transfused is defined as the number of units of PRBC transfused during the last 4 weeks of run-in period (i.e., Day -28 to Day -1). Total units of PRBCs transfused during the entire dosing and follow-up period was listed by 4 week interval. Individual participant data been reported.
Time Frame Over the last 4 weeks of run-in and at 4 week intervals during dosing and follow-up
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacodynamic Population
Arm/Group Title GW786034 50 mg Once Daily
Hide Arm/Group Description:
Participants with hereditary hemorrhagic telangiectasia (HHT) received GW786034 50 mg tablet orally once daily at least one hour before or two hours after a meal in the morning for up to 12 weeks in the open label non-randomized phase.
Overall Number of Participants Analyzed 7
Measure Type: Number
Unit of Measure: Number of units
Participant 1, Baseline 0
Participant 1, Dosing period, Day 1 - 27 0
Participant 1, Dosing period, Day 28 - 55 0
Participant 1, Dosing period, Day 56 - 83 0
Participant 1, Follow up, Day 1 - 28 0
Participant 1, Follow up, Day 29 - 56 0
Participant 1, Follow up, Day 57 - 84 0
Participant 1, Follow up, Day 85 - 112 0
Participant 2, Baseline 0
Participant 2, Dosing period, Day 1 - 5 0
Participant 2, Dosing period, Day 6 - 33 0
Participant 2, Dosing period, Day 34 - 61 0
Participant 2, Dosing period, Day 62 - 89 0
Participant 2, Follow up, Day 1 - 28 0
Participant 2, Follow up, Day 29 - 56 0
Participant 2, Follow up, Day 57 - 84 0
Participant 2, Follow up, Day 85 - 106 0
Participant 3, Baseline 2
Participant 3, Dosing period, Day 1 - 28 2
Participant 3, Dosing period, Day 29 - 56 0
Participant 3, Dosing period, Day 57 - 84 0
Participant 3, Follow up, Day 1 - 28 0
Participant 3, Follow up, Day 29 - 56 0
Participant 3, Follow up, Day 57 - 84 0
Participant 3, Follow up, Day 85 - 112 2
Participant 3, Follow up, Day 113 0
Participant 4, Baseline 0
Participant 4, Dosing period, Day 1 0
Participant 4, Dosing period, Day 2 - 29 0
Participant 4, Dosing period, Day 30 - 57 0
Participant 4, Dosing period, Day 58 - 85 0
Participant 4, Follow up, Day 1 - 28 0
Participant 4, Follow up, Day 29 - 56 0
Participant 4, Follow up, Day 57 - 84 0
Participant 4, Follow up, Day 85 - 112 0
Participant 4, Follow up, Day 113 0
Participant 5, Baseline 0
Participant 5, Dosing period, Day 1 - 2 0
Participant 5, Dosing period, Day 3 - 30 0
Participant 5, Dosing period, Day 31 - 58 0
Participant 5, Dosing period, Day 59 - 86 0
Participant 5, Follow up, Day 1 - 28 0
Participant 5, Follow up, Day 29 - 56 0
Participant 5, Follow up, Day 57 - 84 0
Participant 5, Follow up, Day 85 - 112 2
Participant 5, Follow up, Day 113 0
Participant 6, Baseline 0
Participant 6, Dosing period, Day 1 - 15 0
Participant 6, Dosing period, Day 16 - 43 0
Participant 6, Follow up, Day 1 - 28 0
Participant 6, Follow up, Day 29 - 56 0
Participant 7, Baseline 0
Participant 7, Dosing period, Day 1 - 28 0
Participant 7, Dosing period, Day 29 - 56 0
Participant 7, Dosing period, Day 57 - 84 0
Participant 7, Follow up, Day 1 - 28 0
Participant 7, Follow up, Day 29 - 55 0
10.Secondary Outcome
Title Change From Baseline in the Average of the Last 3 Ferritin Measures in the Dosing Period (Week 9, Week 10.5 and Week 12)
Hide Description For post-Baseline ferritin assessments, average of the last 3 measurements of the dosing period (Weeks 9, 10.5 and 12) was computed. Only pre-infusion ferritin values have been included in the analyses. Baseline ferritin value is the average of the last two measurements during the run-in period. Average of the last two measurements during the run-in period was calculated as sum of the last 2 measured values of ferritin divided by 2. Change from Baseline was calculated as the average of the last 3 measured values of ferritin minus the Baseline value. Average of the last 3 measurements was calculated as sum of the last 3 measured values of ferritin divided by 3. If measurements were missing at one or two of the 3 visits at Weeks 9, 10.5 and 12, then the average was based on the available measurements.
Time Frame Baseline, Week 9, Week 10.5 and Week 12
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacodynamic Population. Only those par. available at the indicated time points were analyzed.
Arm/Group Title GW786034 50 mg Once Daily
Hide Arm/Group Description:
Participants with hereditary hemorrhagic telangiectasia (HHT) received GW786034 50 mg tablet orally once daily at least one hour before or two hours after a meal in the morning for up to 12 weeks in the open label non-randomized phase.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: micrograms per liter (µg per L)
-73.17  (115.016)
11.Secondary Outcome
Title Change From Baseline in Ferritin at the Indicated Time Points
Hide Description Only pre-infusion ferritin values have been included in the analyses. All ferritin measurements that fall within 5 days of iron infusion date are considered post-infusion. Baseline ferritin value is defined as the average of the last two measurements during the run-in period. Average of the last two measurements during the run-in period was calculated as sum of the last 2 measured values of ferritin divided by 2. Change from Baseline is calculated as the difference between the Post dose value at indicated visit minus Baseline value. Par. were evaluated at baseline, treatment period (Weeks 1.5, 3, 4.5, 6, 7.5, 9, 10.5 and 12) and follow-up period (16, 20, 24 and 28).
Time Frame Baseline, Week 1.5, Week 3, Week 4.5, Week 6, Week 7.5, Week 9, Week 10.5, Week 12, Week 16, Week 20, Week 24 and Week 28
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacodynamic Population. Only those par. available at the indicated time points were analyzed (specified by n=X in the category titles).
Arm/Group Title GW786034 50 mg Once Daily
Hide Arm/Group Description:
Participants with hereditary hemorrhagic telangiectasia (HHT) received GW786034 50 mg tablet orally once daily at least one hour before or two hours after a meal in the morning for up to 12 weeks in the open label non-randomized phase.
Overall Number of Participants Analyzed 7
Mean (Standard Deviation)
Unit of Measure: µg per L
Week 1.5, n=4 -73.75  (223.827)
Week 3, n=6 -59.33  (182.538)
Week 4.5, n=5 -118.30  (184.946)
Week 6, n=7 7.07  (62.727)
Week 7.5, n=2 -31.75  (67.529)
Week 9, n=6 -48.83  (86.762)
Week 10.5, n=5 -82.90  (127.769)
Week 12, n=3 -111.67  (231.107)
Follow-Up Week 16, n=7 -85.36  (154.078)
Follow-Up Week 20, n=6 -14.56  (119.056)
Follow-Up Week 24, n=4 -70.25  (237.869)
Follow-Up Week 28, n=5 1.90  (82.750)
12.Secondary Outcome
Title Overall Health-related (HR) Quality of Life (QOL) Score Measured Using SF-36v2 at Day 1, Week 6 and Week 12
Hide Description SF-36v2 is a generic HR QOL instrument with 36 items covering 8 subscales (SS) clustering into 2 global scores, the physical component summary score (PCS: physical functioning (PF), role physical (RP), bodily pain (BP), and general health (GH)) and mental component summary score (MCS: vitality (VT), social functioning (SF), role emotional (RE) and mental health (MH)). All scores are normalized so that mean score for a representative US population = 50, with a standard deviation = 10. Information was used to observe a direction in overall QOL. Ranges are shown below. Higher scores represent better QOL and minimum important differences are PF, 3; RP, 3; BP, 3; GH, 2; VT, 2; SF, 3; RE, 4; and MH, 3 PCS, 2; MCS, 3. Response Consistency Index (RCI) measures the consistency of responses to individual survey responses. Lower the score the more consistent the individual responses. SF-6D Health Utility Index (HUI) Score = 0 (worst measured health state) to 1 (best measured health state).
Time Frame Day (D) 1, Week (W) 6 and Week 12
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacodynamic Population. Only those par. available at the indicated time points were analyzed (specified by n=X in the category titles).
Arm/Group Title GW786034 50 mg Once Daily
Hide Arm/Group Description:
Participants with hereditary hemorrhagic telangiectasia (HHT) received GW786034 50 mg tablet orally once daily at least one hour before or two hours after a meal in the morning for up to 12 weeks in the open label non-randomized phase.
Overall Number of Participants Analyzed 7
Mean (Standard Deviation)
Unit of Measure: Scores on a scale
BP (0-100 score), D1, n=7 58.29  (20.966)
BP (0-100 score), W6, n=7 70.86  (29.396)
BP (0-100 score), W12, n=6 53.67  (19.232)
GH (0-100 score), D1, n=7 50.14  (28.510)
GH (0-100 score), W6, n=7 51.86  (26.448)
GH (0-100 score), W12, n=6 49.67  (17.351)
MH (0-100 score), D1, n=7 61.43  (22.678)
MH (0-100 score), W6, n=7 70.00  (21.602)
MH (0-100 score), W12, n=6 71.67  (12.111)
PF (0-100 score), D1, n=7 52.86  (26.590)
PF (0-100 score), W6, n=7 55.72  (24.397)
PF (0-100 score), W12, n=6 68.33  (12.906)
RE (0-100 score), D1, n=7 63.10  (31.861)
RE (0-100 score), W6, n=7 83.33  (23.570)
RE (0-100 score), W12, n=6 75.00  (16.667)
RP (0-100 score), D1, n=7 58.04  (30.129)
RP (0-100 score), W6, n=7 66.96  (23.583)
RP (0-100 score), W12, n=6 66.67  (25.820)
SF (0-100 score), D1, n=7 66.07  (25.733)
SF (0-100 score), W6, n=7 83.93  (17.252)
SF (0-100 score), W12, n=6 62.50  (20.917)
VT (0-100 score), D1, n=7 42.86  (18.196)
VT (0-100 score), W6, n=7 53.57  (20.684)
VT (0-100 score), W12, n=6 47.92  (11.637)
BP(norm-based score), D1, n=7 (21.68-62.0 score) 44.40  (8.278)
BP(norm-based score), W6, n=7 (21.68-62.0 score) 49.37  (11.605)
BP(norm-based score), W12, n=6 (21.68-62.0 score) 42.58  (7.592)
GH(norm-based score), D1, n=7 (18.95-66.5 score) 43.41  (12.577)
GH(norm-based score), W6, n=7 (18.95-66.5 score) 44.17  (11.666)
GH(norm-based score), W12, n=6 (18.95-66.5 score) 43.20  (7.655)
MH(norm-based score), D1, n=7 (11.63-63.95 score) 43.56  (11.237)
MH(norm-based score), W6, n=7 (11.63-63.95 score) 47.81  (10.704)
MH(norm-based score), W12, n=6 (11.63-63.95 score) 48.63  (6.002)
PF(norm-based score), D1, n=7 (19.26-57.54 score) 39.41  (10.256)
PF(norm-based score), W6, n=7 (19.26-57.54 score) 40.52  (9.410)
PF(norm-based score),W12, n=6 (19.26-57.54 score) 45.38  (4.979)
RE(norm-based score), D1, n=7 (14.39-56.17 score) 38.74  (14.593)
RE(norm-based score), W6, n=7 (14.39-56.17 score) 48.01  (10.795)
RE(norm-based score), W12, n=6 (14.39-56.17 score) 44.19  (7.635)
RP(norm-based score), D1, n=7 (21.23-57.16 score) 42.34  (10.614)
RP(norm-based score), W6, n=7 (21.23-57.16 score) 45.48  (8.308)
RP(norm-based score), W12, n=6 (21.23-57.16 score) 45.38  (9.096)
SF(norm-based score), D1, n=7 (17.23-57.34 score) 50.38  (6.823)
SF(norm-based score), W6, n=7 (17.23-57.34 score) 43.32  (10.174)
SF(norm-based score), W12,n=6 (17.23-57.34 score) 41.91  (8.270)
VT(norm-based score), D1, n=7 (22.89-70.4 score) 44.26  (7.923)
VT(norm-based score), W6, n=7 (22.89-70.4 score) 48.93  (9.007)
VT(norm-based score), W12, n=6 (22.89-70.4 score) 46.47  (5.069)
PCS, D1, n=7 (5.02-79.78 score) 42.87  (7.767)
PCS, W6, n=7 (5.02-79.78 score) 43.57  (9.507)
PCS, W12, n=6 (5.02-79.78 score) 43.74  (6.743)
MCS, D1, n=7 (-3.33-80.09 score) 42.87  (12.198)
MCS, W6, n=7 (-3.33-80.09 score) 50.81  (9.652)
MCS, W12, n=6 (-3.33-80.09 score) 46.27  (6.933)
RCI, D1, n=7 (0-15 score) 0.00  (0.000)
RCI, W6, n=7 (0-15 score) 0.00  (0.000)
RCI, W12, n=6 (0-15 score) 0.00  (0.000)
SF-6D HUI Score, D1, n=7 (0-1 score) 0.64  (0.114)
SF-6D HUI Score, W6, n=7 (0-1 score) 0.70  (0.114)
SF-6D HUI Score, W12, n=6 (0-1 score) 0.64  (0.051)
MH Enhanced Score, D1, n=7 (0-63 score) 11.57  (7.955)
MH Enhanced Score, W6, n=7 (0-63 score) 8.62  (7.459)
MH Enhanced Score, W12, n=6 (0-63 score) 7.83  (3.939)
13.Secondary Outcome
Title Number of Participants With the Indicated Clinical Chemistry Values of Potential Clinical Concern
Hide Description The following laboratory parameters were analyzed: hemoglobin, hematocrit, red blood cell count, platelet count, white blood cell count, total neutrophils, lymphocytes; alanine amino transferase (ALT), alkaline phosphatase (ALP), aspartate amino transferase (AST), gamma glutamyl transferase (GGT), total bilirubin, albumin, total protein, blood urea nitrogen, creatinine, uric acid, sodium, potassium, chloride, calcium, total carbondioxide, glucose, magnesium, and ferritin. Only those parameters for which at least one value of clinical concern are reported in the table. Values above upper limit of normal and below lower limit to normal have been presented as high and low respectively.
Time Frame Up to Week 16
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Population: The Safety Population comprises of all participants who received at least one dose of study treatment. This population is based on the treatment the participant actually received.
Arm/Group Title GW786034 50 mg Once Daily
Hide Arm/Group Description:
Participants with hereditary hemorrhagic telangiectasia (HHT) received GW786034 50 mg tablet orally once daily at least one hour before or two hours after a meal in the morning for up to 12 weeks in the open label non-randomized phase.
Overall Number of Participants Analyzed 7
Measure Type: Number
Unit of Measure: Participants
ALP, High 1
ALP, Low 0
ALT, High 1
ALT, Low 0
AST, High 1
AST, Low 0
Calcium, High 0
Calcium, Low 1
Ferritin, High 0
Ferrtin, Low 4
GGT, High 3
GGT, Low 0
Glucose, High 0
Glucose, Low 3
Hemoglobin, High 0
Hemoglobin, Low 4
Hematocrit, High 0
Hematocrit, Low 2
Lymphocytes, High 0
Lymphocytes, Low 1
Platelet count, High 1
Platelet count, Low 0
Total Protein, High 0
Total Protein, Low 1
WBC, High 0
WBC, Low 3
14.Secondary Outcome
Title Number of Participants With Vital Signs Data Meeting Criteria of Potential Clinical Concern
Hide Description The following laboratory parameters were analyzed in supine position after 10 minutes rest: Diastolic blood pressure (DBP), Systolic blood pressure (SBP) and Heart rate (HR). Values above upper limit of normal and below lower limit to normal have been presented as high and low respectively.
Time Frame Up to Week 16
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Population
Arm/Group Title GW786034 50 mg Once Daily
Hide Arm/Group Description:
Participants with hereditary hemorrhagic telangiectasia (HHT) received GW786034 50 mg tablet orally once daily at least one hour before or two hours after a meal in the morning for up to 12 weeks in the open label non-randomized phase.
Overall Number of Participants Analyzed 7
Measure Type: Number
Unit of Measure: Participants
DBP, Anytime OT, High,n=7 Number Analyzed 7 participants
0
DBP, Anytime OT, Low, n=7 Number Analyzed 7 participants
1
DBP, Day 1, High, n=7 Number Analyzed 7 participants
0
DBP, Day 1, Low, n=7 Number Analyzed 7 participants
0
DBP, Week 3, High, n=7 Number Analyzed 7 participants
0
DBP, Week 3, Low, n=7 Number Analyzed 7 participants
1
DBP, Week 6, High, n=7 Number Analyzed 7 participants
0
DBP, Week 6, Low, n=7 Number Analyzed 7 participants
0
DBP, Week 9, High, n=6 Number Analyzed 6 participants
0
DBP, Week 9, Low, n=6 Number Analyzed 6 participants
0
DBP, Week 12, High, n=6 Number Analyzed 6 participants
0
DBP, Week 12, Low, n=6 Number Analyzed 6 participants
0
HR, Anytime OT, High, n=7 Number Analyzed 7 participants
0
HR, Anytime OT, Low, n=7 Number Analyzed 7 participants
0
HR, Day 1, High, n=7 Number Analyzed 7 participants
0
HR, Day 1, Low, n=7 Number Analyzed 7 participants
0
HR, Week 3, High, n=7 Number Analyzed 7 participants
0
HR, Week 3, Low, n=7 Number Analyzed 7 participants
0
HR, Week 6, High, n=7 Number Analyzed 7 participants
0
HR, Week 6, Low, n=7 Number Analyzed 7 participants
0
HR, Week 9, High, n=6 Number Analyzed 6 participants
0
HR, Week 9, Low, n=6 Number Analyzed 6 participants
0
HR, Week 12, High, n=6 Number Analyzed 6 participants
0
HR, Week 12, Low, n=6 Number Analyzed 6 participants
0
SBP, Anytime OT, High, n=7 Number Analyzed 7 participants
0
SBP, Anytime OT, Low, n=7 Number Analyzed 7 participants
1
SBP, Day 1, High, n=7 Number Analyzed 7 participants
0
SBP, Day 1, Low, n=7 Number Analyzed 7 participants
1
SBP, Week 3, High, n=7 Number Analyzed 7 participants
0
SBP, Week 3, Low, n=7 Number Analyzed 7 participants
0
SBP, Week 6, High, n=7 Number Analyzed 7 participants
0
SBP, Week 6, Low, n=7 Number Analyzed 7 participants
0
SBP, Week 9, High, n=6 Number Analyzed 6 participants
0
SBP, Week 9, Low, n=6 Number Analyzed 6 participants
0
SBP, Week 12, High, n=6 Number Analyzed 6 participants
0
SBP, Week 12, Low, n=6 Number Analyzed 6 participants
0
15.Secondary Outcome
Title Number of Participants With Electrocardiogram (ECG) Data Meeting Criteria of Potential Clinical Concern
Hide Description The following ECG parameters were analyzed: PR, QRS, QT, corrected QT [QTc] intervals. Criteria for clinical concern:. QT where value is > 450, QT[QTc] where value is > 450, PR where value is < 110 or > 220, QRS where value is < 75 or >110.
Time Frame Up to Week 16
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Safety population
Arm/Group Title GW786034 50 mg Once Daily
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Participants with hereditary hemorrhagic telangiectasia (HHT) received GW786034 50 mg tablet orally once daily at least one hour before or two hours after a meal in the morning for up to 12 weeks in the open label non-randomized phase.
Overall Number of Participants Analyzed 7
Measure Type: Number
Unit of Measure: Participants
1
16.Secondary Outcome
Title Number of Participants With Urinalysis Data Meeting Criteria of Potential Clinical Concern
Hide Description Protein – values of clinical concern if change from “trace” at baseline to 3+ any time on-therapy or from 0 at baseline to 2+ any time on-therapy.
Time Frame Up to Week 16
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Safety Population
Arm/Group Title GW786034 50 mg Once Daily
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Participants with hereditary hemorrhagic telangiectasia (HHT) received GW786034 50 mg tablet orally once daily at least one hour before or two hours after a meal in the morning for up to 12 weeks in the open label non-randomized phase.
Overall Number of Participants Analyzed 7
Measure Type: Number
Unit of Measure: Participants
0
17.Secondary Outcome
Title Number of Participants With Any Adverse Events (AE) or Serious Adverse Event (SAE)
Hide Description An AE is defined as any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of a medicinal product.
Time Frame From start of investigational product (IP) through the Study Phase (12 weeks post-dose) (assessed up to 28 weeks)
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Safety Population
Arm/Group Title GW786034 50 mg Once Daily
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Participants with hereditary hemorrhagic telangiectasia (HHT) received GW786034 50 mg tablet orally once daily at least one hour before or two hours after a meal in the morning for up to 12 weeks in the open label non-randomized phase.
Overall Number of Participants Analyzed 7
Measure Type: Number
Unit of Measure: Participants
Any AE 7
Serious AE 0
18.Secondary Outcome
Title Plasma Concentration of GW786034 at the Indicated Time Points
Hide Description Predose (trough) blood samples were collected at weeks 3, 6, 9, and 12. Blood samples for pharmacokinetic (PK) profile were collected at pre-dose, 1, 2, 3, 4, 6 and 8 hours post dose. Area under the curve (0-tau), Concentration tau (Ctau), and maximum concentration (Cmax) following repeat administration was to be studied if data permitted.
Time Frame Weeks 3, 6, 9 and 12
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Pharmacokinetic Population: The Pharmacokinetic Population includes participants who had a pharmacokinetic sample obtained and analyzed. Only those par. available at the indicated time points were analyzed (specified by n=X in the category titles).
Arm/Group Title GW786034 50 mg Once Daily
Hide Arm/Group Description:
Participants with hereditary hemorrhagic telangiectasia (HHT) received GW786034 50 mg tablet orally once daily at least one hour before or two hours after a meal in the morning for up to 12 weeks in the open label non-randomized phase.
Overall Number of Participants Analyzed 7
Mean (Standard Deviation)
Unit of Measure: µg/mL
Week 3, n=7 6.3  (4.45)
Week 6, n=7 6.5  (5.33)
Week 9, n=6 6.2  (4.98)
Week 12, n=6 6.8  (6.85)
19.Secondary Outcome
Title Graphical Exploration of PK/Pharmacodynamic (PD) Relationships Between Pazopanib Exposure and Selected PD
Hide Description Graphical exploration of PK/PD relationships between pazopanib exposure and selected parameters was to be explored if data permitted. Due to the small sample size and the fact that only one dose was studied these analyses were not performed.
Time Frame Weeks 3, 6, 9 and 12
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Pharmacokinetic/Pharmacodynamic Population
Arm/Group Title GW786034 50 mg Once Daily
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Participants with hereditary hemorrhagic telangiectasia (HHT) received GW786034 50 mg tablet orally once daily at least one hour before or two hours after a meal in the morning for up to 12 weeks in the open label non-randomized phase.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
20.Secondary Outcome
Title PK/PD Modeling Analysis to Characterize the Relationship Between Pazopanib Trough Concentrations and Epistaxis Frequency and Duration/Severity
Hide Description A repeated categorical event per time interval PK/PD modeling analysis was planned (data permitting) to characterize the relationship between pazopanib trough concentrations and epistaxis frequency and duration/severity. Due to the small sample size and the fact that only one dose was studied these analyses were not performed.
Time Frame Weeks 3, 6, 9 and 12
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Pharmacokinetic/Pharmacodynamic Population
Arm/Group Title GW786034 50 mg Once Daily
Hide Arm/Group Description:
Participants with hereditary hemorrhagic telangiectasia (HHT) received GW786034 50 mg tablet orally once daily at least one hour before or two hours after a meal in the morning for up to 12 weeks in the open label non-randomized phase.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
Time Frame On-treatment serious adverse events (SAEs) and non-serious adverse events (AEs) were collected from start of IP through the Study Phase (12 weeks post-dose) to follow-up (assessed up to 28 weeks).
Adverse Event Reporting Description On-treatment SAEs and non-serious AEs are reported for the Safety Population, which comprises of all randomized participants who received at least one dose of study treatment and is based on the actual study treatment received.
 
Arm/Group Title GW786034 50 mg Once Daily
Hide Arm/Group Description Participants with hereditary hemorrhagic telangiectasia (HHT) received GW786034 50 mg tablet orally once daily at least one hour before or two hours after a meal in the morning for up to 12 weeks in the open label non-randomized phase.
All-Cause Mortality
GW786034 50 mg Once Daily
Affected / at Risk (%)
Total   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
GW786034 50 mg Once Daily
Affected / at Risk (%)
Total   0/7 (0.00%) 
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
GW786034 50 mg Once Daily
Affected / at Risk (%)
Total   7/7 (100.00%) 
Gastrointestinal disorders   
Abdominal distension  1  1/7 (14.29%) 
Nausea  1  2/7 (28.57%) 
Infections and infestations   
Bronchitis  1  2/7 (28.57%) 
Nasopharyngitis  1  1/7 (14.29%) 
Otitis media  1  1/7 (14.29%) 
Pharyngitis streptococcal  1  1/7 (14.29%) 
Injury, poisoning and procedural complications   
Muscle strain  1  1/7 (14.29%) 
Sunburn  1  1/7 (14.29%) 
Investigations   
Alanine aminotransferase increased  1  1/7 (14.29%) 
Gamma-glutamyltransferase increased  1  1/7 (14.29%) 
Weight decreased  1  1/7 (14.29%) 
Musculoskeletal and connective tissue disorders   
Arthralgia  1  1/7 (14.29%) 
Muscle spasms  1  1/7 (14.29%) 
Musculoskeletal stiffness  1  1/7 (14.29%) 
Pain in extremity  1  1/7 (14.29%) 
Rheumatoid arthritis  1  1/7 (14.29%) 
Nervous system disorders   
Dizziness  1  1/7 (14.29%) 
Headache  1  1/7 (14.29%) 
Psychiatric disorders   
Confusional state  1  1/7 (14.29%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
GSK agreements may vary with individual investigators, but will not prohibit any investigator from publishing. GSK supports the publication of results from all centers of a multi-center trial but requests that reports based on single-site data not precede the primary publication of the entire clinical trial.
Results Point of Contact
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Name/Title: GSK Response Center
Organization: GlaxoSmithKline
Phone: 866-435-7343
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Responsible Party: GlaxoSmithKline
ClinicalTrials.gov Identifier: NCT02204371     History of Changes
Other Study ID Numbers: 201128
First Submitted: July 28, 2014
First Posted: July 30, 2014
Results First Submitted: April 20, 2017
Results First Posted: July 6, 2017
Last Update Posted: July 6, 2017