A Phase 2 Multicenter, Randomized, Double-blind, Placebo-controlled Study to Assess the Safety and Efficacy of Ifetroban in Patients With Diffuse Cutaneous Systemic Sclerosis (SSc) or SSc-associated Pulmonary Arterial Hypertension (SSc-PAH)
Overview
- Phase
- Phase 2
- Intervention
- Oral Placebo
- Conditions
- Scleroderma, Diffuse
- Sponsor
- Cumberland Pharmaceuticals
- Enrollment
- 34
- Locations
- 24
- Primary Endpoint
- Incidence of adverse events (AEs) and Serious AEs (SAEs)
- Status
- Active, not recruiting
- Last Updated
- 17 days ago
Overview
Brief Summary
The purpose of this phase 2 multicenter, randomized, double-blind, placebo-controlled, study is to assess the safety and efficacy of ifetroban in patients with diffuse cutaneous systemic SSc (dcSSc) or SSc-associated pulmonary arterial hypertension (SSc-PAH).
Detailed Description
This study is a randomized, placebo-controlled, double-blind phase 2 trial of patients with dcSSc or SSc-PAH. Twenty participants with SSc-PAH and 14 participants with dcSSc will be randomized to receive either oral ifetroban daily or matching placebo. Study participants will be treated for 12 months, followed by a 30-day follow-up period. The study will test whether ifetroban is safe and statistically superior to placebo in reducing the effects of their disease at month 12 and explore the ability of ifetroban to prevent or reverse progression in patients with early disease duration and reverse established disease in patients with longer disease duration.
Investigators
Eligibility Criteria
Inclusion Criteria
- •Diffuse Cutaneous Criterion:
- •1\. Systematic Sclerosis (SSc), as defined using the 2013 American College of Rheumatology/ European Union League Against Rheumatism Classification Criteria and dcSSc within 7 years following initial diagnosis as defined by the onset of the first non-Raynaud symptom.
- •SSc-PAH Criteria:
- •Adults fulfilling the 2013 American College of Rheumatology/ European Union League Against Rheumatism Classification Criteria with confirmed SSc-PAH (limited or dcSSc) confirmed via previous cardiac catheterization
- •Stable oral therapy for PAH for at least 30 days (monotherapy or combination)
- •New York Heart Association (NYHA) Class I-III Heart Failure
Exclusion Criteria
- •Have a diagnosis of systemic sclerosis sine scleroderma;
- •Be less than 18 years of age or greater than or equal to 80 years of age;
- •Be pregnant, nursing, or planning to become pregnant;
- •Current or planned treatment with prostanoid therapy;
- •Current or planned treatment with pirfenidone;
- •Use of rituximab in the last 3 months;
- •Use of mycophenolic acid (Myfortic, CellCept) at a stable dose for less than 3 months;
- •Current or planned corticosteroid therapy greater than 15mg per day of prednisone or prednisone equivalent;
- •Significant lung disease, defined as FVC \< 50% predicted or DLCO \<40% predicted;
- •Significant kidney disease, defined as Glomerular Filtration Rate (GFR) \< 60 ml/min;
Arms & Interventions
Patients with dcSSc
Patients with dcSSc will be randomized to receive either oral ifetroban or oral placebo daily for 365 days
Intervention: Oral Placebo
Patients with SSc-PAH
Patients with SSc-PAH will be randomized to receive either oral ifetroban or oral placebo daily for 365 days
Intervention: Oral Ifetroban
Patients with SSc-PAH
Patients with SSc-PAH will be randomized to receive either oral ifetroban or oral placebo daily for 365 days
Intervention: Oral Placebo
Patients with dcSSc
Patients with dcSSc will be randomized to receive either oral ifetroban or oral placebo daily for 365 days
Intervention: Oral Ifetroban
Outcomes
Primary Outcomes
Incidence of adverse events (AEs) and Serious AEs (SAEs)
Time Frame: 56 weeks
Safety is measured using AEs, including clinical significant changes in vital signs, laboratory test abnormalities and clinical tolerability of ifetroban.
Secondary Outcomes
- Change from baseline in the modified Rodnan skin score (mRSS)(Baseline, 12, 26, 39, and 52 weeks)
- Change from baseline in forced vital capacity (FVC)(Baseline, 12, 26, and 52 weeks)
- Change from baseline in diffusion capacity for carbon monoxide (DLCO)(Baseline, 12, 26, and 52 weeks)