Systemic Sclerosis Clinical Trial
Official 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.
Verified date | March 2022 |
Source | University of Pittsburgh |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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).
Status | Terminated |
Enrollment | 6 |
Est. completion date | February 10, 2020 |
Est. primary completion date | February 10, 2020 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 18 Years to 80 Years |
Eligibility | 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 |
Country | Name | City | State |
---|---|---|---|
United States | John Hopkins | Baltimore | Maryland |
United States | Boston University | Boston | Massachusetts |
United States | National Jewish | Denver | Colorado |
United States | University of Pittsburgh | Pittsburgh | Pennsylvania |
Lead Sponsor | Collaborator |
---|---|
Robert Lafyatis | Biogen |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | 6 Minute Walk Distance (6MWD) | The primary outcome of clinical efficacy in this study is improvement in 6-minute walk distance (6MWD). Data depict the mean change (%) at end-of-study-treatment (Week 24) from baseline in both treatment groups, utilizing the Last Observation Carried Forward of withdrawn subjects. | Baseline to Week 24 | |
Secondary | Clinical Worsening | The change in time to clinical worsening in DMF compared to placebo treated patients. | Baseline to Week 24 | |
Secondary | Borg Dyspnea Index (BDI) | The change in Borg Dyspnea Index (BDI) at 24 weeks from baseline in DMF compared to placebo treated patients | Baseline to Week 24 | |
Secondary | Serum Markers of Oxidative Stress | The change from baseline of serum markers of oxidative stress at 24 weeks, comparing DMF to placebo treated patients. | Baseline to Week 24 | |
Secondary | Proteomic Biomarkers | The change from baseline in proteomic biomarkers, including BNP, at 24 weeks, comparing DMF to placebo treated patients. | Baseline to Week 24 |
Status | Clinical Trial | Phase | |
---|---|---|---|
Completed |
NCT03274076 -
Evaluation of Tofacitinib in Early Diffuse Cutaneous Systemic Sclerosis (dcSSc)
|
Phase 1/Phase 2 | |
Completed |
NCT04300426 -
Safety and Efficacy of Anaerobic Cultivated Human Intestinal Microbiome Transplantation in Systemic Sclerosis (ReSScue)
|
Phase 2 | |
Recruiting |
NCT06058091 -
RY_SW01 Cell Injection Therapy in Systemic Sclerosis
|
Phase 1/Phase 2 | |
Recruiting |
NCT04356755 -
Subcutaneous Injections of ASC to Heal Digital Ulcers in Patients With Scleroderma.
|
Phase 2 | |
Suspended |
NCT06210945 -
Study to Evaluate the Safety, Tolerability, and Activity of CM-101 in Patients With Systemic Sclerosis
|
Phase 2 | |
Not yet recruiting |
NCT05947682 -
Manufacturing of Allogeneic Adipose Tissue-derived Mesenchymal Stromal Cells for Treatment of Severe Systemic Sclerosis
|
N/A | |
Not yet recruiting |
NCT05177380 -
Efficacy of a Personalized Rehabilitation Program of Facial Involvement in Systemic Sclerosis
|
N/A | |
Not yet recruiting |
NCT04303208 -
Sirtuin 3 and Sirtuin 7 in Systemic Sclerosis
|
N/A | |
Recruiting |
NCT02551042 -
CSL Behring Sclero XIII
|
Phase 2 | |
Terminated |
NCT02243111 -
Detecting Pulmonary Arterial Hypertension (PAH) in Patients With Systemic Sclerosis (SSc) by Ultrasound
|
N/A | |
Terminated |
NCT02246348 -
Evaluating Lung Doppler Signals in Patients With Systemic Sclerosis (SSc)
|
N/A | |
Completed |
NCT01933334 -
Safety and Tolerability of Pirfenidone in Patients With Systemic SclerosisâRelated Interstitial Lung Disease (SSc-ILD) (LOTUSS)
|
Phase 2 | |
Completed |
NCT01468792 -
Hemodynamic Changes in Connective Tissue Disease
|
N/A | |
Terminated |
NCT00848107 -
Open-Label Study of Oral Treprostinil in Digital Ulcers
|
Phase 2 | |
Completed |
NCT00984932 -
Effect of Rosuvastatin on Systemic Sclerosis-related Pulmonary Hypertension
|
Phase 3 | |
Completed |
NCT00074568 -
Scleroderma Registry
|
||
Not yet recruiting |
NCT06412614 -
Evaluation of Patients With Systemic Sclerosis Without Specific or Associated Autoantibodies
|
||
Terminated |
NCT00622687 -
Effect of Different Iloprost Doses on Symptoms in Systemic Sclerosis
|
Phase 2 | |
Recruiting |
NCT04464434 -
Upfront Autologous HSCT Versus Immunosuppression in Early Diffuse Cutaneous Systemic Sclerosis
|
Phase 4 | |
Recruiting |
NCT04246528 -
SPIN Self-Management Feasibility Trial With Progression to Full-scale Trial (SPIN-SELF)
|
N/A |