Dimethyl Fumarate (DMF) in Systemic Sclerosis-Associated Pulmonary Arterial Hypertension

• Register: ClinicalTrials.gov
• Last refreshed on: 28 March 2022
• Main ID: NCT02981082
• Date of registration: 30/11/2016
• Prospective Registration: Yes
• Primary sponsor: Robert Lafyatis
• Public title: Dimethyl Fumarate (DMF) in Systemic Sclerosis-Associated Pulmonary Arterial Hypertension
• Scientific title: A Double-blinded, Placebo-controlled Pilot Study of Dimethyl Fumarate (DMF) in Pulmonary Arterial Hypertension (PAH) Associated With Systemic Sclerosis (SSc-PAH): The Effect of DMF on Clinical Disease and Biomarkers of Oxidative Stress.
• Date of first enrolment: December 2016
• Target sample size: 6
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT02981082
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 1

Countries of recruitment

United States

Contacts

• Name: Robert A Lafyatis, MD
• Affiliation: University of Pittsburgh

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Signed inform consent prior to any study-mandated procedures

2. Adult patients 18-80 years of age

3. World Health Organization Group 1 PAH associated with scleroderma (SSc-PAH)

4. WHO functional Class II-III

5. 6MWD 150 to 450 meters

6. 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.

7. ACR defined systemic sclerosis

Exclusion Criteria:

1. 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

2. Participation in a clinical investigational study within the previous 30 days

3. Moderate to severe hepatic impairment (e.g., Child-Pugh Class B or C)

4. Renal failure defined as:

- estimated creatinine clearance <30 m/min

- serum creatinine>2.5 mg/dl

5. Serum aspartate aminotransferase (AST) and or alanine aminotransferase (ALT) > 1.5
times the upper limit of normal

6. Systolic blood pressure < 90mmHg

7. Recently started (< 8 weeks prior to randomization) or planned cardiopulmonary
rehabilitation program based on exercise

8. Pregnant or lactating women

9. Need for HAART therapy

10. Planned treatment or treatment with another investigational drug within 1 month prior
to start

11. 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



Age minimum: 18 Years
Age maximum: 80 Years
Gender: All

Health Condition(s) or Problem(s) studied

Systemic Sclerosis

Pulmonary; Hypertension

Intervention(s)

Drug: Dimethyl Fumarate (DMF)

Drug: Placebo Oral Tablet

Primary Outcome(s)

6 Minute Walk Distance (6MWD) [Time Frame: Baseline to Week 24]

Secondary Outcome(s)

Clinical Worsening [Time Frame: Baseline to Week 24]

Proteomic Biomarkers [Time Frame: Baseline to Week 24]

Borg Dyspnea Index (BDI) [Time Frame: Baseline to Week 24]

Serum Markers of Oxidative Stress [Time Frame: Baseline to Week 24]

Secondary ID(s)

PRO16070614

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Biogen

Results

• Results available: Yes
• Date Posted: 17/03/2022
• URL: https://clinicaltrials.gov/ct2/show/results/NCT02981082