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Tadalafil for Sarcoidosis Associated Pulmonary Hypertension (SAPH)

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ClinicalTrials.gov Identifier: NCT01324999
Recruitment Status : Completed
First Posted : March 29, 2011
Results First Posted : July 24, 2017
Last Update Posted : July 24, 2017
Sponsor:
Collaborators:
Eli Lilly and Company
United Therapeutics
University of Cincinnati
Information provided by (Responsible Party):
H. James Ford, MD, University of North Carolina, Chapel Hill

Study Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Pulmonary Hypertension
Intervention Drug: Tadalafil
Enrollment 12
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Tadalafil
Hide Arm/Group Description 40 mg daily
Period Title: Overall Study
Started 12
Completed 7
Not Completed 5
Reason Not Completed
Adverse Event             3
Withdrawal by Subject             2
Arm/Group Title Tadalafil
Hide Arm/Group Description 40 mg daily
Overall Number of Baseline Participants 12
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 12 participants
54.5  (9.48)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 12 participants
Female
8
  66.7%
Male
4
  33.3%
Race/Ethnicity, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 12 participants
Black or African American
9
  75.0%
White
3
  25.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 12 participants
12
FEV1  
Mean (Standard Deviation)
Unit of measure:  Percent predicted
Number Analyzed 12 participants
58.0  (14.7)
FVC  
Mean (Standard Deviation)
Unit of measure:  Percent predicted
Number Analyzed 12 participants
65.9  (13.4)
DLCO  
Mean (Standard Deviation)
Unit of measure:  Percent predicted
Number Analyzed 12 participants
60.3  (25.0)
Right Atrial Pressure  
Mean (Standard Deviation)
Unit of measure:  Mm Hg
Number Analyzed 12 participants
6.2  (4.0)
Mean Pulmonary Artery Pressure  
Mean (Standard Deviation)
Unit of measure:  Mm Hg
Number Analyzed 12 participants
34.8  (7.1)
Pulmonary Artery Wedge Pressure  
Mean (Standard Deviation)
Unit of measure:  Mm Hg
Number Analyzed 12 participants
10.3  (3.6)
Cardiac Index  
Mean (Standard Deviation)
Unit of measure:  L/min/m²
Number Analyzed 12 participants
2.4  (0.6)
Pulmonary Vascular Resistance  
Mean (Standard Deviation)
Unit of measure:  Wood Units
Number Analyzed 12 participants
6.2  (3.6)
1.Primary Outcome
Title 6 Minute Walk Distance
Hide Description [Not Specified]
Time Frame Baseline, Week 8, Week 16, Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
Only 6 of the 7 study completers performed 6 minute walk test through week 24
Arm/Group Title Tadalafil
Hide Arm/Group Description:
40 mg daily
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: meters
Baseline 419.1  (170.1)
Week 8 418.1  (143.5)
Week 16 436.5  (178.1)
Week 24 377.1  (140.9)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Tadalafil
Comments Comparing baseline to week 24.
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.68
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
2.Secondary Outcome
Title Resting Oxygen Saturation
Hide Description Percent oxygen saturation of hemoglobin as measured by pulse oximetry in the resting state.
Time Frame Baseline, Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
only 6 of the 7 study completers performed the 6 minute walk test through week 24.
Arm/Group Title Tadalafil
Hide Arm/Group Description:
40 mg daily
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: percent
Baseline 96.5  (1.8)
Week 24 95.2  (2.6)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Tadalafil
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.34
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
3.Secondary Outcome
Title Oxygen Desaturation During 6 Minute Walk Test
Hide Description Change (decrease) in oxygen saturation from start of 6 minute walk to nadir during the 6 minute walk test
Time Frame Baseline, Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
Only 6 of the 7 study completers performed the 6 minute walk test through week 24
Arm/Group Title Tadalafil
Hide Arm/Group Description:
40 mg daily
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: oxygen saturation percentage points
Baseline 9  (6)
Week 24 9  (9)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Tadalafil
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.99
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
4.Secondary Outcome
Title Maximum Borg Dyspnea Score During 6 Minute Walk Test
Hide Description The modified Borg scale consists of a vertical scale labelled 0 to10 with corresponding verbal expressions of progressively increasing sensation (shortness of breath) intensity. 0 represents no dyspnea, and 10 is the highest level of perceived dyspnea.
Time Frame Baseline, Week 8, Week 16, Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
Only 6 of the 7 study completers performed 6 minute walk test through the end of the study.
Arm/Group Title Tadalafil
Hide Arm/Group Description:
40 mg daily
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: units on a scale
Baseline 4.8  (1.2)
Week 8 5.0  (2.4)
Week 16 5.3  (2.1)
Week 24 6.3  (2.3)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Tadalafil
Comments comparing baseline to week 24
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.19
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
5.Secondary Outcome
Title Brain Natriuretic Peptide Level
Hide Description [Not Specified]
Time Frame Baseline, Week 8, Week 16, Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Tadalafil
Hide Arm/Group Description:
40 mg daily
Overall Number of Participants Analyzed 7
Mean (Standard Deviation)
Unit of Measure: pg/mL
Baseline 58.8  (89.6)
Week 8 32.3  (46.6)
Week 16 37.5  (51.4)
Week 24 42.6  (49.7)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Tadalafil
Comments Comparing Baseline to week 24.
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.68
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
6.Secondary Outcome
Title Short Form-36 Global Score
Hide Description SF- 36 investigates the standard of quality of life through a general health assessment and not specific to a particular disease, age or treatment group. It is a 36-item questionnaire measuring 8 domains (physical functioning, role physical, bodily pain, general health, vitality, social functioning, role emotional, and mental health). Each domain score ranges from 0 (worst) to 100 (best), with higher scores reflecting better health-related functional status. The global score is determined by taking an average of the individual domain scores. The global score range is from 0 (worst) to 100 (best).
Time Frame Baseline, Week 8, Week 16, Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Tadalafil
Hide Arm/Group Description:
40 mg daily
Overall Number of Participants Analyzed 7
Mean (Standard Deviation)
Unit of Measure: units on a scale
Baseline 38.5  (5.3)
Week 8 37.7  (6.7)
Week 16 39.2  (6.5)
Week 24 38.9  (8.5)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Tadalafil
Comments comparing baseline to week 24
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.92
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
7.Secondary Outcome
Title St. George's Respiratory Questionnaire (SGRQ) Score
Hide Description The SGRQ ranges from 0 (no impairment of quality of life) to 100 (highest impairment of quality of life)
Time Frame Baseline, Week 8, Week 16, Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Tadalafil
Hide Arm/Group Description:
40 mg daily
Overall Number of Participants Analyzed 7
Mean (Standard Deviation)
Unit of Measure: units on a scale
Baseline 64.5  (15.5)
Week 8 65.1  (18.9)
Week 16 64.3  (15.2)
Week 24 71.1  (15.2)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Tadalafil
Comments comparison of baseline to week 24
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.44
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
8.Secondary Outcome
Title Number of Participants With Change in WHO Functional Class (WHO FC)
Hide Description The WHO functional classification ranges from I (patient's disease does not affect daily activities) to IV (patient's disease causes severe impairment). Higher scores indicate more severe impairment.
Time Frame Baseline, Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Tadalafil
Hide Arm/Group Description:
40 mg daily
Overall Number of Participants Analyzed 7
Measure Type: Number
Unit of Measure: participants
Number of participants that improved to WHO FC I 1
Number of participants that improved to WHO FC II 0
Number of participants that improved to WHO FC III 0
Time Frame 24 weeks
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Tadalafil
Hide Arm/Group Description 40 mg daily
All-Cause Mortality
Tadalafil
Affected / at Risk (%)
Total   0/12 (0.00%)    
Hide Serious Adverse Events
Tadalafil
Affected / at Risk (%) # Events
Total   0/12 (0.00%)    
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Tadalafil
Affected / at Risk (%) # Events
Total   5/12 (41.67%)    
Musculoskeletal and connective tissue disorders   
Joint pain *  1/12 (8.33%)  1
Nervous system disorders   
Headache *  2/12 (16.67%)  2
Respiratory, thoracic and mediastinal disorders   
Influenza/pneumonia *  1/12 (8.33%)  1
Skin and subcutaneous tissue disorders   
Herpes zoster *  1/12 (8.33%)  1
*
Indicates events were collected by non-systematic assessment
Since this was a proof of concept study evaluating safety and tolerability of the intervention in this population, it was not powered to achieve statistical significance in changes observed from baseline to week 24 in any of the endpoints.
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: H. James Ford, MD
Organization: University of North Carolina at Chapel Hill
Phone: (919) 966-2531
EMail: hjford@med.unc.edu
Layout table for additonal information
Responsible Party: H. James Ford, MD, University of North Carolina, Chapel Hill
ClinicalTrials.gov Identifier: NCT01324999    
Other Study ID Numbers: 09-2326
First Submitted: March 24, 2011
First Posted: March 29, 2011
Results First Submitted: March 21, 2017
Results First Posted: July 24, 2017
Last Update Posted: July 24, 2017