Placebo Controlled Trial of Bosentan in Scleroderma Patients
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ClinicalTrials.gov Identifier: NCT00377455 |
Recruitment Status :
Terminated
(Study was terminated due to inadequate enrolment)
First Posted : September 18, 2006
Results First Posted : July 20, 2011
Last Update Posted : May 24, 2018
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Systemic Scleroderma Pulmonary Hypertension | Drug: Bosentan Drug: Placebo | Phase 2 |
Pulmonary hypertension (PAH) is a common and usually fatal form of lung disease in systemic sclerosis (SSc). Multiple drugs have been approved for the treatment of New York Heart Association (NYHA)Class III/IV PAH in scleroderma. Bosentan is an endothelin-1 antagonist which showed significant improvement in distance walked during 12 week clinical trials in PAH patients (7). Therapy for asymptomatic systemic sclerosis patients diagnosed incidentally with PAH (World Health Organization (WHO) Functional Class I) remains controversial. We hypothesize that asymptomatic or minimally symptomatic patients with systemic sclerosis and normal resting pulmonary artery pressures who demonstrate an abnormal rise in pulmonary artery systolic pressure with stress Doppler echocardiography testing represent a subset of patients who already have pulmonary vascular disease and who are at risk for the development of severe PAH. We further hypothesize that early identification and treatment of such patients may retard the progression of that disease.
Hypotheses:
- Stress echocardiography identifies early pulmonary vascular disease by detecting exercise-induced pulmonary hypertension in patients with systemic sclerosis.
- Treatment of exercise-induced PAH with Bosentan will lead to improved exercise endurance in patients with systemic sclerosis.
Subjects will be recruited from those patients who have had an abnormal exercise test as part of an earlier study, Exercise Echocardiograms in Scleroderma (IRB# 03-363).
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 5 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Masking: | Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) |
Primary Purpose: | Treatment |
Official Title: | Placebo Controlled Trial of Bosentan vs Placebo in NYHA Class I/II Scleroderma Patients With Exercise Induced Pulmonary Hypertension |
Study Start Date : | September 2006 |
Actual Primary Completion Date : | September 2009 |
Actual Study Completion Date : | March 2010 |

Arm | Intervention/treatment |
---|---|
Experimental: Bosentan |
Drug: Bosentan
62.5 mg by mouth (PO) twice daily (Bid) for 1 month, followed by 125 mg PO Bid thereafter, for a total of 16 weeks |
Placebo Comparator: Placebo |
Drug: Placebo
62.5 mg PO Bid for 1 month, followed by 125 mg PO Bid thereafter, for a total of 16 weeks |
- Total Exercise Time on the Exercise Echocardiogram Using the Standard Bruce Stress Protocol. [ Time Frame: This will be determined after 16 weeks on the study medication. ]The total exercise time measured using the exercise echocardiogram is evaluated with the standard Bruce Stress Protocol, and this will be determined after 16 weeks on the study medication.
- 6-minute Walk Distance [ Time Frame: 16 weeks ]The distance walked during a 6-minute walk test.
- Brain Natriuretic Peptide (BNP) Level [ Time Frame: 16 weeks ]Serum BNP level to evaluate Brain Natriuretic Peptide (BNP) level
- Endothelin-1(ET-1) Level [ Time Frame: 16 weeks ]From saved serum to determine Endothelian-1 (ET-1) levels in patients
- Quality of Life (QOL) [ Time Frame: 16 weeks ]QOL is measured using the Short Form 36 Health Survey (SF-36, which measures health on eight dimensions: general health perception, physical and social functioning, role limitations by physical or emotional problems, mental health, vitality, and bodily pain. For each dimension items are coded, summed, and transformed on to a scale from 0 (worst health) to 100 (best health).

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Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- SSc patients > 18 with NYHA functional Class I/II symptoms, informed consent, and who are willing to participate in the Pulmonary Hypertension Assessment and Recognition of Outcomes in Scleroderma (PHAROS) long term study (Georgetown IRB 04-227)
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Right heart catheterization with
- Normal Mean Pulmonary Arterial Pressure (PAP) at rest
- Mean PAP > 30 with exercise
- Wedge Pressure < 18
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Entry criteria for participating in the exercise echocardiogram study (Georegtown IRB 03-363)
- Diffusing Capacity (DLCO) <60 with a Forced Vital Capacity (FVC) >60%, or
- FVC/DLCO > 1.6, or
- a resting Pulmonary Arterial Systolic Pressure (PASP)> 40mmHg
Exclusion Criteria:
- Established resting pulmonary hypertension
- Congestive heart failure
- Diastolic dysfunction
- Pregnancy
- Inability to adequately walk/exercise
- Severe liver disease

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00377455
United States, Connecticut | |
University of Connecticut | |
Farmington, Connecticut, United States, 06030 | |
United States, District of Columbia | |
Georgetown University Medical Center | |
Washington, District of Columbia, United States, 20007 |
Principal Investigator: | Virginia D Steen, MD | Georgetown University |
Responsible Party: | Virginia Steen, MD, Professor of Medicine, Georgetown University |
ClinicalTrials.gov Identifier: | NCT00377455 |
Other Study ID Numbers: |
IRB 06-043 Bosentan ( Other Identifier: Georgetown University ) |
First Posted: | September 18, 2006 Key Record Dates |
Results First Posted: | July 20, 2011 |
Last Update Posted: | May 24, 2018 |
Last Verified: | April 2018 |
scleroderma systemic sclerosis bosentan pulmonary hypertension |
PHAROS PHROS exercise echocardiogram |
Hypertension, Pulmonary Hypertension Scleroderma, Systemic Scleroderma, Diffuse Scleroderma, Localized Vascular Diseases Cardiovascular Diseases Lung Diseases |
Respiratory Tract Diseases Connective Tissue Diseases Skin Diseases Bosentan Antihypertensive Agents Endothelin Receptor Antagonists Molecular Mechanisms of Pharmacological Action |