Multiple Sclerosis Clinical Trial
Dimethyl Fumarate (DMF) in Systemic Sclerosis-Associated Pulmonary Arterial Hypertension
Summary
A double-blinded, placebo-controlled study of Dimethyl fumarate (DMF) in 34 Systemic Sclerosis-Pulmonary Hypertension (SSc-PAH) patients. The study will determine safety and the primary outcome variability for DMF in treating SSc-PAH; the primary outcome of clinical efficacy in this pilot trial will be improvement in 6-minute walk distance (6MWD).
Full Description
A double-blinded, placebo-controlled study of Dimethyl fumarate (DMF) in 34 Systemic Sclerosis-Pulmonary Hypertension (SSc-PAH) patients. The study medication will be added to stable background PAH medication(s). Subjects will be dosed for 24 weeks, will undergo examination every 8 weeks, and will be finally evaluated 12 weeks after completion of treatment. Dosage will begin at once daily oral doses of 120mg for the first 7 days and follow the up-titration schedule to a maintenance dose of 240mg twice a day (or highest tolerated dose of a minimum of 120mg twice a day by the start of Week 8) for the remainder of the study. Participation will be for a total of 40 weeks, including a 4-week screening period, 24 weeks of drug, and a safety follow-up 12 weeks after the last dose. The study will determine the safety and the primary outcome variability for DMF in treating SSc-PAH; the primary outcome of clinical efficacy in this pilot trial will be improvement in 6-minute walk distance (6MWD).
Eligibility Criteria
Inclusion Criteria:
Signed inform consent prior to any study-mandated procedures
Adult patients 18-80 years of age
World Health Organization Group 1 PAH associated with scleroderma (SSc-PAH)
WHO functional Class II-III
6MWD 150 to 450 meters
Right heart catheterization demonstrating mPAP≥ 25 mmHg and PCWP or left ventricular end diastolic pressure ≤15mm Hg and pulmonary vascular resistance ≥240 dynes/cm-5 (3 Wood units) within 12 weeks prior to study entry.
ACR defined systemic sclerosis
Exclusion Criteria:
Pulmonary hypertension associated with
PAH of any etiology other than scleroderma
PH of any etiology other than WHO Group I PAH
Pulmonary venous hypertension defined as PCWP or LVEDP >15 mHg
Untreated sleep apnea with AHI >20 or SaO2 Nadir <87%
Chronic thromboembolic disease
Sarcoidosis
Participation in a clinical investigational study within the previous 30 days
Moderate to severe hepatic impairment (e.g., Child-Pugh Class B or C)
Renal failure defined as:
estimated creatinine clearance <30 m/min
serum creatinine>2.5 mg/dl
Serum aspartate aminotransferase (AST) and or alanine aminotransferase (ALT) > 1.5 times the upper limit of normal
Systolic blood pressure < 90mmHg
Recently started (< 8 weeks prior to randomization) or planned cardiopulmonary rehabilitation program based on exercise
Pregnant or lactating women
Need for HAART therapy
Planned treatment or treatment with another investigational drug within 1 month prior to start
Moderate to severe interstitial lung disease, defined by FVC < 80% or evidence on HRCT of fibrosis or ground glass changes involving more than 30% of lung parenchyma
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There are 4 Locations for this study
Denver Colorado, 80206, United States
Baltimore Maryland, 21205, United States
Boston Massachusetts, 02118, United States
Pittsburgh Pennsylvania, 15213, United States
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