Oral Ifetroban to Treat Diffuse Cutaneous Systemic Sclerosis (SSc) or SSc-associated Pulmonary Arterial Hypertension

Purpose

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).

Conditions

  • Scleroderma, Diffuse
  • Scleroderma, Systemic
  • Scleroderma, Limited
  • Sclerosis, Progressive Systemic
  • Skin Diseases
  • Connective Tissue Diseases
  • Pathologic Processes
  • Autoimmune Diseases

Eligibility

Eligible Ages
Between 18 Years and 80 Years
Eligible Genders
All
Accepts Healthy Volunteers
No

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: 1. 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 2. Stable oral therapy for PAH for at least 30 days (monotherapy or combination) 3. New York Heart Association (NYHA) Class I-III Heart Failure

Exclusion Criteria

  1. Have a diagnosis of systemic sclerosis sine scleroderma; 2. Be less than 18 years of age or greater than or equal to 80 years of age; 3. Be pregnant, nursing, or planning to become pregnant; 4. Current or planned treatment with prostanoid therapy; 5. Current or planned treatment with pirfenidone; 6. Use of rituximab in the last 3 months; 7. Use of mycophenolic acid (Myfortic, CellCept) at a stable dose for less than 3 months; 8. Current or planned corticosteroid therapy greater than 15mg per day of prednisone or prednisone equivalent; 9. Significant lung disease, defined as FVC < 50% predicted or DLCO <40% predicted; 10. Significant kidney disease, defined as Glomerular Filtration Rate (GFR) < 60 ml/min; 11. Have moderate or severe hepatic impairment; 12. Contraindication to MRI (e.g., implanted magnetic material, claustrophobia); 13. Known hypersensitivity to gadolinium; 14. Any cause of pulmonary hypertension other than World Health Organization (WHO) Group I associated with SSc; 15. Use of aspirin > 81 mg per day in the last two weeks; 16. Use of warfarin, heparin or other anticoagulants in the last 30 days; 17. Recent (within 6 weeks) myocardial infarction or persistent atrial arrhythmias; 18. Have a history of allergy or hypersensitivity to ifetroban; 19. Have taken investigational drugs within 30 days before study treatment administration; 20. Inability to understand the requirements of the study, inability to understand spoken English and abide by the study restrictions and to return for the required treatments and assessments; 21. Be otherwise unsuitable for the study, in the opinion of the investigator.

Study Design

Phase
Phase 2
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
Patients with dcSSc 		Patients with dcSSc will be randomized to receive either oral ifetroban or oral placebo daily for 365 days
  • Drug: Oral Ifetroban
    Subjects will be treated with oral ifetroban or placebo daily for 365 days
    Other names:
    • Ifetroban
  • Drug: Oral Placebo
    Subjects will be treated with oral ifetroban or placebo daily for 365 days
    Other names:
    • Ifetroban
Experimental
Patients with SSc-PAH 		Patients with SSc-PAH will be randomized to receive either oral ifetroban or oral placebo daily for 365 days
  • Drug: Oral Ifetroban
    Subjects will be treated with oral ifetroban or placebo daily for 365 days
    Other names:
    • Ifetroban
  • Drug: Oral Placebo
    Subjects will be treated with oral ifetroban or placebo daily for 365 days
    Other names:
    • Ifetroban

Recruiting Locations

More Details

Status
Recruiting
Sponsor
Cumberland Pharmaceuticals

Study Contact

Ingrid Anderson, PhD, CCRP
615-255-0068
ianderson@cumberlandpharma.com

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.