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Trial record 93 of 380 for:    FERRIC CATION

Safety and Efficacy Study of Oral Ferric Iron To Treat Iron Deficiency Anaemia in Quiescent Ulcerative Colitis (AEGIS-1) (AEGIS-1)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01340872
Recruitment Status : Completed
First Posted : April 25, 2011
Results First Posted : August 3, 2018
Last Update Posted : February 1, 2019
Sponsor:
Information provided by (Responsible Party):
Shield Therapeutics

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Double (Participant, Investigator);   Primary Purpose: Treatment
Conditions Iron Deficiency Anaemia
Inflammatory Bowel Disease
Ulcerative Colitis
Interventions Drug: ST10-021
Drug: Placebo Comparator
Enrollment 128
Recruitment Details Potential subjects were selected from the general population attending each centre in UK, DE, AT or HU for routine care of their IBD and anaemia. Individuals interested in participating were invited for the Screening visit in order to assess eligibility. Written informed consent was obtained prior to conducting any study specific assessments.
Pre-assignment Details Subjects were males or females aged ≥18 years with a confirmed diagnosis of UC, required to be in remission have a mild-to-moderate UC (defined by SCCAI score <4 at entry); mild-to-moderate IDA (Hb concentration ≥9.5 g/dL and <12.0 g/dL for females and ≥9.5 g/dL and <13.0 g/dL for males; serum ferritin levels <30 μg/L at Screening.
Arm/Group Title ST10 Placebo ST10 - Open-label Continuation From Active Arm in Double-blind Placebo Switch to Open-label Extension ST10 Treatment
Hide Arm/Group Description 30mg ST10 capsules BD - double-blind phase Matching placebo for ST10 capsules - double-blind phase Open-label extension of ST10 active treatment arm from double-blind phase Open-label extension ST10 treatment arm for placebo subjects completing double-blind phase
Period Title: Double-blind Phase
Started 64 64 0 [1] 0 [1]
Completed 55 53 0 [1] 0 [1]
Not Completed 9 11 0 0
Reason Not Completed
Adverse Event             6             4             0             0
Withdrawal by Subject             3             5             0             0
Lost to Follow-up             0             1             0             0
Protocol Violation             0             1             0             0
[1]
Not Applicable to double-blind phase
Period Title: Open-label Phase
Started 0 [1] 0 [1] 50 [2] 47 [2]
Completed 0 [1] 0 [1] 37 36
Not Completed 0 0 13 11
Reason Not Completed
Physician Decision             0             0             2             1
Pregnancy             0             0             1             0
Adverse Event             0             0             8             4
Withdrawal by Subject             0             0             1             6
Worsening of IBD             0             0             1             0
[1]
Not Applicable to open-label phase
[2]
Only subjects in DE, UK and HU could enter the open-label extension (OLE); OLE rejected by EC in AT
Arm/Group Title ST10 Placebo Total
Hide Arm/Group Description ST10: 30 mg Ferric Maltol capsules taken orally twice a day during a 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase. Matching Placebo capsules taken orally twice a day during a 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase. Total of all reporting groups
Overall Number of Baseline Participants 64 64 128
Hide Baseline Analysis Population Description
Potential subjects were screened for eligibility to participate based on their demographics, medical/surgical history, physical examination, concomitant medications, vital signs, clinical laboratory tests (including pregnancy test for females of child-bearing potential) and current IBD disease status (based on SCCAI clinical score). FAS - Safety
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 64 participants 64 participants 128 participants
<=18 years
0
   0.0%
0
   0.0%
0
   0.0%
Between 18 and 65 years
62
  96.9%
63
  98.4%
125
  97.7%
>=65 years
2
   3.1%
1
   1.6%
3
   2.3%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 64 participants 64 participants 128 participants
40.1  (13.52) 38.5  (12.3) 39.2  (12.9)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 64 participants 64 participants 128 participants
Female
40
  62.5%
43
  67.2%
83
  64.8%
Male
24
  37.5%
21
  32.8%
45
  35.2%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 64 participants 64 participants 128 participants
United Kingdom 15 10 25
Hungary 11 15 26
Germany 35 32 67
Austria 4 6 10
Duration of ulcerative colitis (years)   [1] 
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 64 participants 64 participants 128 participants
9.0  (8.28) 10.99  (11.43) 9.99  (9.8)
[1]
Measure Description: Duration of ulcerative colitis (years); for participants with UC
Time since last IBD flare-up (months)   [1] 
Mean (Standard Deviation)
Unit of measure:  Months
Number Analyzed 64 participants 64 participants 128 participants
24.8  (59.16) 21.51  (38.46) 23.2  (48.4)
[1]
Measure Description: Time since last IBD flare-up (months) recorded at baseline
Time since last OFP dose (months)   [1] 
Mean (Standard Deviation)
Unit of measure:  Months
Number Analyzed 64 participants 64 participants 128 participants
36.17  (40.1) 33.34  (44.04) 34.76  (41.7)
[1]
Measure Description: Time since last prior oral ferrous product (OFP) dose (months)
Haemoglobin concentration at baseline   [1] 
Mean (Standard Deviation)
Unit of measure:  g/dL
Number Analyzed 64 participants 64 participants 128 participants
11  (1.03) 11.1  (0.85) 11.05  (0.93)
[1]
Measure Description: Haemoglobin concentration at baseline of the 12 week double-blind phase
Serum Ferritin concentration at baseline   [1] 
Mean (Standard Deviation)
Unit of measure:  μg/L
Number Analyzed 64 participants 64 participants 128 participants
8.6  (6.8) 8.2  (6.5) 8.4  (6.6)
[1]
Measure Description: Serum Ferritin concentration at baseline of the 12 week double-blind phase
TSAT% at baseline   [1] 
Mean (Standard Deviation)
Unit of measure:  Percentage
Number Analyzed 64 participants 64 participants 128 participants
10.6  (11.7) 9.5  (7.5) 10.05  (9.6)
[1]
Measure Description: TSAT% (transferrin saturation %) at baseline of the 12 week double-blind phase
Irritable Bowel Disease Questionnaire (IBDQ) score at baseline   [1] 
Mean (Standard Deviation)
Unit of measure:  Score on a scale
Number Analyzed 64 participants 64 participants 128 participants
175.0  (30.92) 171.0  (33.56) 173.02  (32.23)
[1]
Measure Description:

Irritable Bowel Disease Questionnaire (IBDQ) score at baseline (randomisation) of the 12 week double-blind phase.

The IBDQ was developed as an activity index for determining the effect of Ulcerative Colitis symptoms on perceived quality of life. It is a 32-item questionnaire with four dimensions: bowel function, emotional status, systemic symptoms and social function. Total IBDQ score ranges from 32 to 224, with higher scores indicating better quality of life. The score of patients in remission usually is between 170 and 190.

Simple Clinical Colitis Activity Index (SCCAI) score at baseline   [1] 
Mean (Full Range)
Unit of measure:  Score on a scale
Number Analyzed 64 participants 64 participants 128 participants
1.8
(0 to 3)
1.4
(0 to 3)
1.6
(0 to 3)
[1]
Measure Description:

Simple Clinical Colitis Activity Index (SCCAI) score at baseline (randomisation) of the 12-week double-blind phase (in participants with UC).

The SCCAI is a diagnostic and research questionnaire used to assess the severity of symptoms in people who suffer from UC. The calculated score ranges from 0 to 19, where active disease is a score of 5 or higher.

The score is determined by asking the person with UC questions regarding:

  • bowel frequency at day/night
  • urgency of defecation
  • blood in stool
  • general health
  • extracolonic manifestations
1.Primary Outcome
Title Change in Haemoglobin (Hb) Concentration From Baseline to Week 12 (Full Analysis Set, FAS)
Hide Description Primary efficacy endpoint, defined as the change in Hb concentration from Baseline to Week 12. Baseline was defined as the pre-dose Hb concentration measured at the Randomisation Visit (Week 0). Missing Randomisation Hb values were replaced by Screening Hb values, if the randomisation was within the protocol-specified window. Hb concentration (g/dL) was analysed by a central laboratory from blood samples collected at every clinic visit: Screening, Randomisation (Week 0), Weeks 4, 8, 12, 14, 16, 20, 24, 36, 48, 64, Weeks 14 to 64 were open-label. The baseline, absolute concentration and change from baseline in Hb at all post-randomisation visits were listed and summarised by week using descriptive statistics. An analysis of covariance (ANCOVA) was used to analyse the primary endpoint; this included treatment, gender and disease as factors and baseline Hb as a covariate.
Time Frame Baseline to Week 12 - double-blind phase
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS)
Arm/Group Title ST10 Placebo
Hide Arm/Group Description:
ST10: 30 mg Ferric Maltol capsules taken orally twice a day during 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase.
Matching Placebo capsules taken orally twice a day during 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase.
Overall Number of Participants Analyzed 58 53
Mean (Standard Deviation)
Unit of Measure: g/dL
2.26  (1.184) 0.01  (0.764)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection ST10, Placebo
Comments ANCOVA for primary endpoint, Change in Hb concentration from Baseline to Week 12 in double-blind phase, FAS
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value < 0.0001
Comments [Not Specified]
Method ANCOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value 2.18
Confidence Interval (1-Sided) 97.5%
1.81
Parameter Dispersion
Type: Standard Error of the Mean
Value: 0.19
Estimation Comments [Not Specified]
2.Secondary Outcome
Title Proportion of Subjects That Achieved ≥1 g/dL Change From Baseline in Hb Concentration at Week 12 (Full Analysis Set, FAS)
Hide Description Logistic regression analysis of proportion of subjects that achieved ≥1 g/dL change from baseline in Hb concentration at Week 12 in the double-blind phase
Time Frame Subjects that achieved ≥1 g/dL change from baseline in Hb concentration at Week 12 - double-blind phase
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS)
Arm/Group Title ST10 Placebo
Hide Arm/Group Description:
ST10: 30 mg Ferric Maltol capsules taken orally twice a day during 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase.
Matching Placebo capsules taken orally twice a day during 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase.
Overall Number of Participants Analyzed 64 64
Measure Type: Count of Participants
Unit of Measure: Participants
Yes
50
  78.1%
7
  10.9%
No
14
  21.9%
57
  89.1%
3.Secondary Outcome
Title Proportion of Subjects That Achieved ≥2 g/dL Change From Baseline in Hb Concentration at Week 12 (Full Analysis Set, FAS)
Hide Description Logistic regression analysis of proportion of subjects that achieved ≥2 g/dL change from baseline in Hb concentration at Week 12 in the double-blind phase
Time Frame Baseline to Week 12 - double-blind phase
Hide Outcome Measure Data
Hide Analysis Population Description
FAS
Arm/Group Title ST10 Placebo
Hide Arm/Group Description:
ST10: 30 mg Ferric Maltol capsules taken orally twice a day during 12-week double-blind phase.
Matching Placebo capsules taken orally twice a day during 12-week double-blind phase.
Overall Number of Participants Analyzed 64 64
Measure Type: Count of Participants
Unit of Measure: Participants
Yes
36
  56.3%
0
   0.0%
No
28
  43.8%
64
 100.0%
4.Secondary Outcome
Title Proportion of Subjects That Achieved Hb Concentration Within Normal Range at Week 12 (Full Analysis Set, FAS)
Hide Description Logistic regression analysis of proportion of subjects that achieved Hb concentration within normal range at Week 12 - end of double-blind phase
Time Frame Baseline to Week 12 - double-blind phase
Hide Outcome Measure Data
Hide Analysis Population Description
FAS
Arm/Group Title ST10 Placebo
Hide Arm/Group Description:
ST10: 30 mg Ferric Maltol capsules taken orally twice a day during 12-week double-blind phase.
Matching Placebo capsules taken orally twice a day during 12-week double-blind phase.
Overall Number of Participants Analyzed 64 64
Measure Type: Count of Participants
Unit of Measure: Participants
Yes
42
  65.6%
8
  12.5%
No
22
  34.4%
56
  87.5%
5.Secondary Outcome
Title Change in Hb Concentration From Baseline to Week 4 (Full Analysis Set, FAS)
Hide Description ANCOVA analysis of the change in Hb concentration from Baseline to Week 4 of the double-blind phase - Full Analysis Set, multiple imputation
Time Frame Baseline to Week 4 - double-blind phase
Hide Outcome Measure Data
Hide Analysis Population Description
FAS
Arm/Group Title ST10 Placebo
Hide Arm/Group Description:
ST10: 30 mg Ferric Maltol capsules taken orally twice a day during 12-week double-blind phase.
Matching Placebo capsules taken orally twice a day during 12-week double-blind phase.
Overall Number of Participants Analyzed 59 61
Mean (Standard Deviation)
Unit of Measure: g/dL
1.08  (0.676) 0  (0.67)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection ST10, Placebo
Comments ANCOVA analysis of the change in Hb concentration from Baseline to Week 4 of the double-blind phase - Full Analysis Set, multiple imputation
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value < 0.0001
Comments [Not Specified]
Method ANCOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value 1.04
Confidence Interval (1-Sided) 97.5%
0.82
Parameter Dispersion
Type: Standard Error of the Mean
Value: 0.11
Estimation Comments [Not Specified]
6.Secondary Outcome
Title Change in Hb Concentration From Baseline to Week 8 (Full Analysis Set, FAS)
Hide Description ANCOVA analysis of Change in Hb concentration from Baseline to Week 8 of double-blind phase - FAS, multiple imputation
Time Frame Baseline to Week 8 - double-blind phase
Hide Outcome Measure Data
Hide Analysis Population Description
FAS
Arm/Group Title ST10 Placebo
Hide Arm/Group Description:
ST10: 30 mg Ferric Maltol capsules taken orally twice a day during 12-week double-blind phase.
Matching Placebo capsules taken orally twice a day during 12-week double-blind phase.
Overall Number of Participants Analyzed 59 56
Mean (Standard Deviation)
Unit of Measure: g/dL
1.79  (1.037) 0.04  (0.722)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection ST10, Placebo
Comments ANCOVA analysis of Change in Hb concentration from Baseline to Week 8 of double-blind phase - FAS, multiple imputation
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value < 0.0001
Comments [Not Specified]
Method ANCOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value 1.73
Confidence Interval (1-Sided) 97.5%
1.43
Parameter Dispersion
Type: Standard Error of the Mean
Value: 0.15
Estimation Comments [Not Specified]
7.Secondary Outcome
Title Change in Haemoglobin Concentration From Baseline to Week 16 (Full Analysis Set, FAS)
Hide Description Change in Haemoglobin Concentration from Baseline to Week 16 (FAS), after 12-week double-blind phase and first 4 weeks of open-label ST10 treatment.
Time Frame Baseline to Week 16 - open-label phase
Hide Outcome Measure Data
Hide Analysis Population Description
FAS
Arm/Group Title ST10 - Open-label Continuation From Active Arm in Double-blind Placebo Switch to Open-label Extension ST10 Treatment
Hide Arm/Group Description:
Open-label extension of ST10 active treatment arm from double-blind phase
Open-label extension ST10 treatment arm for placebo subjects completing double-blind phase
Overall Number of Participants Analyzed 46 45
Mean (Standard Deviation)
Unit of Measure: g/dL
2.34  (1.281) 1.04  (1.023)
8.Secondary Outcome
Title Change in Haemoglobin Concentration From Baseline to Week 20 (Full Analysis Set, FAS)
Hide Description Change in Haemoglobin Concentration from Baseline to Week 20 (FAS), after 12-week double-blind phase and then 8 weeks of open-label ST10 treatment
Time Frame Baseline to Week 20 - open-label phase
Hide Outcome Measure Data
Hide Analysis Population Description
FAS
Arm/Group Title ST10 - Open-label Continuation From Active Arm in Double-blind Placebo Switch to Open-label Extension ST10 Treatment
Hide Arm/Group Description:
Open-label extension of ST10 active treatment arm from double-blind phase
Open-label extension ST10 treatment arm for placebo subjects completing double-blind phase
Overall Number of Participants Analyzed 43 43
Mean (Standard Deviation)
Unit of Measure: g/dL
2.45  (1.213) 1.46  (1.056)
9.Secondary Outcome
Title Change in Haemoglobin Concentration From Baseline to Week 24 (Full Analysis Set, FAS)
Hide Description Change in Haemoglobin Concentration from Baseline to Week 24 (FAS), after 12-week double-blind phase and then 12 weeks of open-label ST10 treatment
Time Frame Baseline to Week 24 - open-label phase
Hide Outcome Measure Data
Hide Analysis Population Description
FAS
Arm/Group Title ST10 - Open-label Continuation From Active Arm in Double-blind Placebo Switch to Open-label Extension ST10 Treatment
Hide Arm/Group Description:
Open-label extension of ST10 active treatment arm from double-blind phase
Open-label extension ST10 treatment arm for placebo subjects completing double-blind phase
Overall Number of Participants Analyzed 43 41
Mean (Standard Deviation)
Unit of Measure: g/dL
2.68  (1.127) 1.87  (1.195)
10.Secondary Outcome
Title Change in Haemoglobin Concentration From Baseline to Week 36 (Full Analysis Set, FAS)
Hide Description Change in Haemoglobin Concentration from Baseline to Week 36 (FAS), after 12-week double-blind phase and then 24 weeks of open-label ST10 treatment
Time Frame Baseline to Week 36 - open-label phase
Hide Outcome Measure Data
Hide Analysis Population Description
FAS
Arm/Group Title ST10 - Open-label Continuation From Active Arm in Double-blind Placebo Switch to Open-label Extension ST10 Treatment
Hide Arm/Group Description:
Open-label extension of ST10 active treatment arm from double-blind phase
Open-label extension ST10 treatment arm for placebo subjects completing double-blind phase
Overall Number of Participants Analyzed 41 36
Mean (Standard Deviation)
Unit of Measure: g/dL
2.85  (1.227) 2.17  (1.048)
11.Secondary Outcome
Title Change in Haemoglobin Concentration From Baseline to Week 48 (Full Analysis Set, FAS)
Hide Description Change in Haemoglobin Concentration from Baseline to Week 48 (FAS), after 12-week double-blind phase and then 36 weeks of open-label ST10 treatment
Time Frame Baseline to Week 48 - open-label phase
Hide Outcome Measure Data
Hide Analysis Population Description
FAS
Arm/Group Title ST10 - Open-label Continuation From Active Arm in Double-blind Placebo Switch to Open-label Extension ST10 Treatment
Hide Arm/Group Description:
Open-label extension of ST10 active treatment arm from double-blind phase
Open-label extension ST10 treatment arm for placebo subjects completing double-blind phase
Overall Number of Participants Analyzed 40 37
Mean (Standard Deviation)
Unit of Measure: g/dL
3.09  (1.339) 2.0  (1.191)
12.Secondary Outcome
Title Change in Haemoglobin Concentration From Baseline to Week 64 (Full Analysis Set, FAS)
Hide Description Change in Haemoglobin Concentration from Baseline to Week 64 (FAS), after 12-week double-blind phase and then 52 weeks of open-label ST10 treatment
Time Frame Baseline to Week 64 - open-label phase
Hide Outcome Measure Data
Hide Analysis Population Description
FAS
Arm/Group Title ST10 - Open-label Continuation From Active Arm in Double-blind Placebo Switch to Open-label Extension ST10 Treatment
Hide Arm/Group Description:
Open-label extension of ST10 active treatment arm from double-blind phase
Open-label extension ST10 treatment arm for placebo subjects completing double-blind phase
Overall Number of Participants Analyzed 35 36
Mean (Standard Deviation)
Unit of Measure: g/dL
3.07  (1.457) 2.19  (1.605)
13.Secondary Outcome
Title Change in Haemoglobin Concentration From Baseline to Week 64 EOS (Full Analysis Set, FAS)
Hide Description Change in Haemoglobin Concentration from Baseline to Week 64 EOS (FAS) - Week 64 was re-categorised as Week 64 EOS for those subjects who withdrew from the study early and the ‘Week 64’ visit was outside the visit window of 64 weeks ± 2 days
Time Frame Baseline to Week 64 EOS - open-label phase
Hide Outcome Measure Data
Hide Analysis Population Description
FAS
Arm/Group Title ST10 - Open-label Continuation From Active Arm in Double-blind Placebo Switch to Open-label Extension ST10 Treatment
Hide Arm/Group Description:
Open-label extension of ST10 active treatment arm from double-blind phase
Open-label extension ST10 treatment arm for placebo subjects completing double-blind phase
Overall Number of Participants Analyzed 10 17
Mean (Standard Deviation)
Unit of Measure: g/dL
1.32  (1.713) 0.52  (1.417)
14.Secondary Outcome
Title Proportion of Subjects That Achieved Haemoglobin Concentration Within Normal Range at Week 16 (Full Analysis Set, FAS)
Hide Description Proportion of subjects that achieved Haemoglobin Concentration within normal range at Week 16 (Full Analysis Set), after 12-week double-blind phase and first 4 weeks of open-label ST10 treatment
Time Frame Baseline to Week 16 - open-label phase
Hide Outcome Measure Data
Hide Analysis Population Description
FAS
Arm/Group Title ST10 - Open-label Continuation From Active Arm in Double-blind Placebo Switch to Open-label Extension ST10 Treatment
Hide Arm/Group Description:
Open-label extension of ST10 active treatment arm from double-blind phase
Open-label extension ST10 treatment arm for placebo subjects completing double-blind phase
Overall Number of Participants Analyzed 46 45
Measure Type: Count of Participants
Unit of Measure: Participants
Yes
36
  78.3%
17
  37.8%
No
10
  21.7%
28
  62.2%
15.Secondary Outcome
Title Proportion of Subjects That Achieved Haemoglobin Concentration Within Normal Range at Week 36 (Full Analysis Set, FAS)
Hide Description Proportion of subjects that achieved Haemoglobin Concentration within normal range at Week 36 (Full Analysis Set), after 12-week double-blind phase and 24 weeks of open-label ST10 treatment
Time Frame Baseline to Week 36 - open-label phase
Hide Outcome Measure Data
Hide Analysis Population Description
FAS
Arm/Group Title ST10 - Open-label Continuation From Active Arm in Double-blind Placebo Switch to Open-label Extension ST10 Treatment
Hide Arm/Group Description:
Open-label extension of ST10 active treatment arm from double-blind phase
Open-label extension ST10 treatment arm for placebo subjects completing double-blind phase
Overall Number of Participants Analyzed 41 36
Measure Type: Count of Participants
Unit of Measure: Participants
Yes
35
  85.4%
29
  80.6%
No
6
  14.6%
7
  19.4%
16.Secondary Outcome
Title Proportion of Subjects That Achieved Haemoglobin Concentration Within Normal Range at Week 64 (Full Analysis Set, FAS)
Hide Description Proportion of subjects that achieved Haemoglobin Concentration within normal range at Week 64 (Full Analysis Set), after 12-week double-blind phase and 52 weeks of open-label ST10 treatment
Time Frame Baseline to Week 64 - open-label phase
Hide Outcome Measure Data
Hide Analysis Population Description
FAS
Arm/Group Title ST10 - Open-label Continuation From Active Arm in Double-blind Placebo Switch to Open-label Extension ST10 Treatment
Hide Arm/Group Description:
Open-label extension of ST10 active treatment arm from double-blind phase
Open-label extension ST10 treatment arm for placebo subjects completing double-blind phase
Overall Number of Participants Analyzed 36 36
Measure Type: Count of Participants
Unit of Measure: Participants
Yes
31
  86.1%
30
  83.3%
No
5
  13.9%
6
  16.7%
17.Other Pre-specified Outcome
Title Change in Haemoglobin Concentration From Baseline to Week 12 (Per Protocol Analysis Set, PPAS)
Hide Description ANCOVA sensitivity analysis of the Primary efficacy endpoint analysis on the PPAS - Change in Haemoglobin Concentration from Baseline to Week 12
Time Frame Baseline to Week 12 - double-blind phase
Hide Outcome Measure Data
Hide Analysis Population Description
Per-Protocol Analysis Set - sensitivity analysis of primary endpoint
Arm/Group Title ST10 Placebo
Hide Arm/Group Description:
ST10: 30 mg Ferric Maltol capsules taken orally twice a day during 12-week double-blind phase.
Matching Placebo capsules taken orally twice a day during 12-week double-blind phase.
Overall Number of Participants Analyzed 55 57
Least Squares Mean (Standard Error)
Unit of Measure: g/dL
2.23  (0.13) 0.05  (0.13)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection ST10, Placebo
Comments ANCOVA sensitivity analysis of the Primary efficacy endpoint analysis on the PPAS
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value < 0.0001
Comments [Not Specified]
Method ANCOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value 2.18
Confidence Interval (1-Sided) 97.5%
1.87
Parameter Dispersion
Type: Standard Error of the Mean
Value: 0.17
Estimation Comments [Not Specified]
18.Other Pre-specified Outcome
Title Change in Haemoglobin Concentration From Baseline to Week 12 (Full Analysis Set [FAS] LOCF)
Hide Description ANCOVA sensitivity analysis of the Primary efficacy endpoint analysis on the FAS LOCF - Change in Haemoglobin Concentration from Baseline to Week 12
Time Frame Baseline to Week 12 - double-blind phase
Hide Outcome Measure Data
Hide Analysis Population Description
FAS LOCF - sensitivity analysis of primary endpoint
Arm/Group Title ST10 Placebo
Hide Arm/Group Description:
ST10: 30 mg Ferric Maltol capsules taken orally twice a day during 12-week double-blind phase.
Matching Placebo capsules taken orally twice a day during 12-week double-blind phase.
Overall Number of Participants Analyzed 64 64
Least Squares Mean (Standard Error)
Unit of Measure: g/dL
2.11  (0.12) -0.03  (0.12)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection ST10, Placebo
Comments ANCOVA sensitivity analysis of the Primary efficacy endpoint analysis on the FAS LOCF - Change in Haemoglobin Concentration from Baseline to Week 12
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value < 0.0001
Comments [Not Specified]
Method ANCOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value 2.15
Confidence Interval (1-Sided) 97.5%
1.82
Parameter Dispersion
Type: Standard Error of the Mean
Value: 0.16
Estimation Comments [Not Specified]
19.Other Pre-specified Outcome
Title Change in Serum Ferritin Concentration From Baseline to Week 12 (Full Analysis Set, FAS)
Hide Description Change in serum Ferritin concentration from Baseline to Week 12 (Full Analysis Set), after 12-week double-blind phase
Time Frame Baseline to Week 12 - double-blind phase
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FAS
Arm/Group Title ST10 Placebo
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ST10: 30 mg Ferric Maltol capsules taken orally twice a day during 12-week double-blind phase.
Matching Placebo capsules taken orally twice a day during 12-week double-blind phase.
Overall Number of Participants Analyzed 59 53
Mean (Standard Deviation)
Unit of Measure: μg/dL
17.3  (28.3) 1.2  (7.85)
20.Other Pre-specified Outcome
Title Change in Serum Ferritin Concentration From Baseline to Week 64 (Full Analysis Set, FAS)
Hide Description Change in serum Ferritin concentration from Baseline to Week 64 (FAS), after 12-week double-blind phase and 52 weeks open-label ST10 treatment
Time Frame Baseline to Week 64 - open-label phase
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Hide Analysis Population Description
FAS
Arm/Group Title ST10 - Open-label Continuation From Active Arm in Double-blind Placebo Switch to Open-label Extension ST10 Treatment
Hide Arm/Group Description:
Open-label extension of ST10 active treatment arm from double-blind phase
Open-label extension ST10 treatment arm for placebo subjects completing double-blind phase
Overall Number of Participants Analyzed 36 36
Mean (Standard Deviation)
Unit of Measure: μg/dL
60.4  (93.35) 36.6  (46.8)
21.Other Pre-specified Outcome
Title Change in Serum TSAT% From Baseline to Week 12 (Full Analysis Set, FAS)
Hide Description Change in serum TSAT% from Baseline to Week 12 (FAS), after 12-week double-blind phase
Time Frame Baseline to Week 12 - double-blind phase
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Hide Analysis Population Description
FAS
Arm/Group Title ST10 Placebo
Hide Arm/Group Description:
ST10: 30 mg Ferric Maltol capsules taken orally twice a day during 12-week double-blind phase.
Matching Placebo capsules taken orally twice a day during 12-week double-blind phase.
Overall Number of Participants Analyzed 59 52
Mean (Standard Deviation)
Unit of Measure: % serum TSAT
18.0  (20.17) -0.4  (7.82)
22.Other Pre-specified Outcome
Title Change in Serum TSAT% From Baseline to Week 64 (Full Analysis Set, FAS)
Hide Description Change in serum TSAT% from Baseline to Week 64 (Full Analysis Set), after 12-week double-blind phase and 52 weeks open-label ST10 treatment
Time Frame Baseline to Week 64 - open-label phase
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Hide Analysis Population Description
FAS
Arm/Group Title ST10 - Open-label Continuation From Active Arm in Double-blind Placebo Switch to Open-label Extension ST10 Treatment
Hide Arm/Group Description:
Open-label extension of ST10 active treatment arm from double-blind phase
Open-label extension ST10 treatment arm for placebo subjects completing double-blind phase
Overall Number of Participants Analyzed 36 36
Mean (Standard Deviation)
Unit of Measure: % serum TSAT
18.8  (12.46) 17.7  (16.2)
23.Other Pre-specified Outcome
Title Irritable Bowel Disease Questionnaire (IBDQ) Score at Week 12 (Full Analysis Set, FAS)
Hide Description

Irritable Bowel Disease Questionnaire (IBDQ) score at Week 12 (FAS), end of double-blind phase.

The IBDQ was developed as an activity index for determining the effect of Ulcerative Colitis symptoms on perceived quality of life. It is a 32-item questionnaire with four dimensions: bowel function, emotional status, systemic symptoms and social function. Total IBDQ score ranges from 32 to 224, with higher scores indicating better quality of life. The score of patients in remission usually is between 170 and 190.

Time Frame Week 12 - double-blind phase
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Hide Analysis Population Description
FAS
Arm/Group Title ST10 Placebo
Hide Arm/Group Description:
ST10: 30 mg Ferric Maltol capsules taken orally twice a day during 12-week double-blind phase.
Matching Placebo capsules taken orally twice a day during 12-week double-blind phase.
Overall Number of Participants Analyzed 60 60
Mean (Standard Deviation)
Unit of Measure: score on a scale
178.3  (32.36) 176.3  (31.5)
24.Other Pre-specified Outcome
Title Irritable Bowel Disease Questionnaire (IBDQ) Score at Week 64 (Full Analysis Set, FAS)
Hide Description

Irritable Bowel Disease Questionnaire (IBDQ) score at Week 64 (FAS), after 12-week double-blind phase and 52 weeks of open-label ST10 treatment.

The IBDQ was developed as an activity index for determining the effect of Ulcerative Colitis symptoms on perceived quality of life. It is a 32-item questionnaire with four dimensions: bowel function, emotional status, systemic symptoms and social function. Total IBDQ score ranges from 32 to 224, with higher scores indicating better quality of life. The score of patients in remission usually is between 170 and 190.

Time Frame Week 64 - open-label phase
Hide Outcome Measure Data
Hide Analysis Population Description
FAS
Arm/Group Title ST10 - Open-label Continuation From Active Arm in Double-blind Placebo Switch to Open-label Extension ST10 Treatment
Hide Arm/Group Description:
Open-label extension of ST10 active treatment arm from double-blind phase
Open-label extension ST10 treatment arm for placebo subjects completing double-blind phase
Overall Number of Participants Analyzed 37 36
Mean (Standard Deviation)
Unit of Measure: score on a scale
180.7  (30.14) 177.2  (36.97)
25.Other Pre-specified Outcome
Title Change From Baseline in Simple Clinical Colitis Activity Index (SCCAI) Score at Week 12 (Full Analysis Set, FAS)
Hide Description

Change from baseline in Simple Clinical Colitis Activity Index (SCCAI) score at Week 12 (FAS), end of double-blind phase (in subjects with UC).

The SCCAI is a diagnostic and research questionnaire used to assess the severity of symptoms in people who suffer from UC. The calculated score ranges from 0 to 19, where active disease is a score of 5 or higher.

The score is determined by asking the person with UC questions regarding:

  • bowel frequency at day/night
  • urgency of defecation
  • blood in stool
  • general health
  • extracolonic manifestations
Time Frame Baseline to Week 12 - double-blind phase
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Hide Analysis Population Description
FAS - participants with UC only
Arm/Group Title ST10 Placebo
Hide Arm/Group Description:
ST10: 30 mg Ferric Maltol capsules taken orally twice a day during 12-week double-blind phase.
Matching Placebo capsules taken orally twice a day during 12-week double-blind phase.
Overall Number of Participants Analyzed 28 29
Median (Full Range)
Unit of Measure: score on a scale
0
(-2 to 5)
0
(-1 to 5)
26.Other Pre-specified Outcome
Title Change From Baseline in Simple Clinical Colitis Activity Index (SCCAI) Score at Week 64 (Full Analysis Set)
Hide Description

Change from baseline in Simple Clinical Colitis Activity Index (SCCAI) score at Week 64 (FAS), after 12-week double-blind phase and 52 weeks open-label ST10 treatment (in participants with UC only).

The SCCAI is a diagnostic and research questionnaire used to assess the severity of symptoms in people who suffer from UC. The calculated score ranges from 0 to 19, where active disease is a score of 5 or higher.

The score is determined by asking the person with UC questions regarding:

  • bowel frequency at day/night
  • urgency of defecation
  • blood in stool
  • general health
  • extracolonic manifestations
Time Frame Baseline to Week 64 - open-label phase
Hide Outcome Measure Data
Hide Analysis Population Description
FAS
Arm/Group Title ST10 - Open-label Continuation From Active Arm in Double-blind Placebo Switch to Open-label Extension ST10 Treatment
Hide Arm/Group Description:
Open-label extension of ST10 active treatment arm from double-blind phase
Open-label extension ST10 treatment arm for placebo subjects completing double-blind phase
Overall Number of Participants Analyzed 19 19
Median (Full Range)
Unit of Measure: score on a scale
0
(-3 to 2)
0
(-2 to 5)
Time Frame Adverse events (AEs) were solicited after informed consent and until 4 weeks after the last dose of study drug (at study completion or early termination).
Adverse Event Reporting Description Subjects were expected to volunteer information about AEs they experienced. The Investigator/designee also questioned the subject at each visit about AEs and recorded these as well as all other AEs apparent. AEs/SAEs were monitored until they were resolved or determined to be due to a subject’s on-going condition or inter-current illness.
 
Arm/Group Title ST10 - Safety Set, Double-blind Phase Placebo - Safety Set, Double-blind Phase ST10 Continuation - Safety Set, Open-label Phase Placebo Switch to ST10 Treatment-Safety Set, Open-label Phase
Hide Arm/Group Description Adverse events reported in the double-blind phase active treatment arm with ST10 (Ferric Maltol). ST10 30 mg capsules taken orally twice a day during a 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase. Adverse events reported in the double-blind phase placebo treatment arm. Matching placebo capsules for ST10 taken orally twice a day during a 12-week double-blind phase. For study participants in the UK, DE and HU only, the 12-week double-blind phase was followed by a 52-week open-label ST10 extension treatment phase. Adverse events reported in the open-label extension phase for continuation of active treatment with ST10 (Ferric Maltol) from the double-blind phase active treatment arm. Adverse events reported in the open-label extension phase from those subjects continuing treatment from the double-blind placebo arm; subjects commenced ST10 open-label treatment after completion of the double-blind phase at the Week 12 visit.
All-Cause Mortality
ST10 - Safety Set, Double-blind Phase Placebo - Safety Set, Double-blind Phase ST10 Continuation - Safety Set, Open-label Phase Placebo Switch to ST10 Treatment-Safety Set, Open-label Phase
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/64 (0.00%)      0/64 (0.00%)      0/50 (0.00%)      0/47 (0.00%)    
Show Serious Adverse Events Hide Serious Adverse Events
ST10 - Safety Set, Double-blind Phase Placebo - Safety Set, Double-blind Phase ST10 Continuation - Safety Set, Open-label Phase Placebo Switch to ST10 Treatment-Safety Set, Open-label Phase
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   1/64 (1.56%)      2/64 (3.13%)      8/50 (16.00%)      2/47 (4.26%)    
Cardiac disorders         
Angina pectoris  1  0/64 (0.00%)  0 0/64 (0.00%)  0 0/50 (0.00%)  0 1/47 (2.13%)  1
Gastrointestinal disorders         
Abdominal Pain  1  1/64 (1.56%)  1 0/64 (0.00%)  0 2/50 (4.00%)  2 0/47 (0.00%)  0
Diarrhoea  1  1/64 (1.56%)  1 0/64 (0.00%)  0 0/50 (0.00%)  0 0/47 (0.00%)  0
Crohn's disease  1  1/64 (1.56%)  1 1/64 (1.56%)  1 0/50 (0.00%)  0 0/47 (0.00%)  0
Rectal abscess  1  0/64 (0.00%)  0 1/64 (1.56%)  1 0/50 (0.00%)  0 0/47 (0.00%)  0
Colitis ulcerative  1  0/64 (0.00%)  0 0/64 (0.00%)  0 1/50 (2.00%)  1 0/47 (0.00%)  0
Rectal haemorrhage  1  0/64 (0.00%)  0 0/64 (0.00%)  0 1/50 (2.00%)  1 0/47 (0.00%)  0
General disorders         
Hernia  1  0/64 (0.00%)  0 0/64 (0.00%)  0 1/50 (2.00%)  1 0/47 (0.00%)  0
Infections and infestations         
Herpes zoster  1  0/64 (0.00%)  0 0/64 (0.00%)  0 1/50 (2.00%)  1 0/47 (0.00%)  0
Peritonitis  1  0/64 (0.00%)  0 0/64 (0.00%)  0 1/50 (2.00%)  1 0/47 (0.00%)  0
Pneumonia  1  0/64 (0.00%)  0 0/64 (0.00%)  0 0/50 (0.00%)  0 1/47 (2.13%)  1
Respiratory, thoracic and mediastinal disorders         
Pulmonary embolism  1  0/64 (0.00%)  0 0/64 (0.00%)  0 0/50 (0.00%)  0 1/47 (2.13%)  1
Surgical and medical procedures         
Cholesteatoma removal  1  0/64 (0.00%)  0 0/64 (0.00%)  0 1/50 (2.00%)  1 0/47 (0.00%)  0
1
Term from vocabulary, MedDRA (16.0)
Indicates events were collected by systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 3%
ST10 - Safety Set, Double-blind Phase Placebo - Safety Set, Double-blind Phase ST10 Continuation - Safety Set, Open-label Phase Placebo Switch to ST10 Treatment-Safety Set, Open-label Phase
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   39/64 (60.94%)      46/64 (71.88%)      40/50 (80.00%)      35/47 (74.47%)    
Gastrointestinal disorders         
Rectal haemorrhage  1  3/64 (4.69%)  3 1/64 (1.56%)  1 0/50 (0.00%)  0 1/47 (2.13%)  1
Abdominal discomfort  1  3/64 (4.69%)  3 0/64 (0.00%)  0 1/50 (2.00%)  1 0/47 (0.00%)  0
Abdominal distension  1  2/64 (3.13%)  2 0/64 (0.00%)  0 0/50 (0.00%)  0 2/47 (4.26%)  2
Abdominal pain  1  7/64 (10.94%)  8 5/64 (7.81%)  6 2/50 (4.00%)  2 7/47 (14.89%)  8
Abdominal pain upper  1  1/64 (1.56%)  1 3/64 (4.69%)  3 1/50 (2.00%)  1 4/47 (8.51%)  4
Abdominal pain lower  1  0/64 (0.00%)  0 0/64 (0.00%)  0 0/50 (0.00%)  0 2/47 (4.26%)  2
Constipation  1  5/64 (7.81%)  5 1/64 (1.56%)  1 2/50 (4.00%)  2 1/47 (2.13%)  1
Colitis ulcerative  1  0/64 (0.00%)  0 0/64 (0.00%)  0 4/50 (8.00%)  4 6/47 (12.77%)  7
Crohn's disease  1  0/64 (0.00%)  0 4/64 (6.25%)  4 2/50 (4.00%)  2 5/47 (10.64%)  6
Diarrhoea  1  5/64 (7.81%)  6 4/64 (6.25%)  5 4/50 (8.00%)  4 6/47 (12.77%)  6
Flatulence  1  4/64 (6.25%)  4 0/64 (0.00%)  0 3/50 (6.00%)  3 3/47 (6.38%)  3
Gastrooesophageal reflux disease  1  2/64 (3.13%)  2 0/64 (0.00%)  0 1/50 (2.00%)  1 1/47 (2.13%)  1
Haematochezia  1  1/64 (1.56%)  1 1/64 (1.56%)  1 1/50 (2.00%)  1 2/47 (4.26%)  2
Nausea  1  0/64 (0.00%)  0 1/64 (1.56%)  1 2/50 (4.00%)  2 3/47 (6.38%)  3
Vomiting  1  1/64 (1.56%)  1 2/64 (3.13%)  2 1/50 (2.00%)  1 2/47 (4.26%)  2
Sinusitis  1  0/64 (0.00%)  0 1/64 (1.56%)  1 2/50 (4.00%)  2 1/47 (2.13%)  1
General disorders         
Fatigue  1  2/64 (3.13%)  2 3/64 (4.69%)  3 0/50 (0.00%)  0 0/47 (0.00%)  0
Pyrexia  1  1/64 (1.56%)  1 2/64 (3.13%)  2 1/50 (2.00%)  1 1/47 (2.13%)  1
Immune system disorders         
Seasonal allergy  1  0/64 (0.00%)  0 2/64 (3.13%)  2 2/50 (4.00%)  2 2/47 (4.26%)  2
Infections and infestations         
Gastroenteritis  1  0/64 (0.00%)  0 1/64 (1.56%)  1 2/50 (4.00%)  2 0/47 (0.00%)  0
Influenza  1  2/64 (3.13%)  2 0/64 (0.00%)  0 2/50 (4.00%)  2 0/47 (0.00%)  0
Nasopharyngitis  1  4/64 (6.25%)  4 8/64 (12.50%)  8 12/50 (24.00%)  14 8/47 (17.02%)  8
Upper respiratory tract infection  1  1/64 (1.56%)  1 2/64 (3.13%)  2 0/50 (0.00%)  0 0/47 (0.00%)  0
Musculoskeletal and connective tissue disorders         
Arthralgia  1  3/64 (4.69%)  3 0/64 (0.00%)  0 4/50 (8.00%)  4 3/47 (6.38%)  3
Back pain  1  0/64 (0.00%)  0 0/64 (0.00%)  0 1/50 (2.00%)  1 3/47 (6.38%)  3
Musculoskeletal stiffness  1  0/64 (0.00%)  0 0/64 (0.00%)  0 2/50 (4.00%)  2 0/47 (0.00%)  0
Nervous system disorders         
Headache  1  2/64 (3.13%)  2 4/64 (6.25%)  4 1/50 (2.00%)  1 2/47 (4.26%)  3
Respiratory, thoracic and mediastinal disorders         
Oropharyngeal pain  1  0/64 (0.00%)  0 3/64 (4.69%)  3 0/50 (0.00%)  0 2/47 (4.26%)  2
Cough  1  0/64 (0.00%)  0 0/64 (0.00%)  0 3/50 (6.00%)  3 0/47 (0.00%)  0
Skin and subcutaneous tissue disorders         
Pruritus  1  0/64 (0.00%)  0 2/64 (3.13%)  2 0/50 (0.00%)  0 0/47 (0.00%)  0
Rash  1  0/64 (0.00%)  0 1/64 (1.56%)  1 1/50 (2.00%)  1 2/47 (4.26%)  2
1
Term from vocabulary, MedDRA (16.0)
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT 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.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Mark Sampson
Organization: Shield Therapeutics
Phone: +44 (0) 207 186 8500
EMail: msampson@shieldtx.com
Layout table for additonal information
Responsible Party: Shield Therapeutics
ClinicalTrials.gov Identifier: NCT01340872     History of Changes
Other Study ID Numbers: ST10-01-301
2010-023588-16 ( EudraCT Number )
First Submitted: April 19, 2011
First Posted: April 25, 2011
Results First Submitted: June 9, 2017
Results First Posted: August 3, 2018
Last Update Posted: February 1, 2019