A Phase IIa, Double-Blind, Randomised, Intracohort Placebo-Controlled, Multicentre Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of EHP-101 in Patients With Diffuse Cutaneous Systemic Sclerosis
Overview
- Phase
- Phase 2
- Intervention
- Patients will be randomized to receive EHP-101 or Placebo
- Conditions
- Diffuse Cutaneous Systemic Sclerosis
- Sponsor
- Emerald Health Pharmaceuticals
- Enrollment
- 36
- Locations
- 23
- Primary Endpoint
- Incidence and severity of Treatment Emergent Adverse Events
- Status
- Suspended
- Last Updated
- 2 years ago
Overview
Brief Summary
The purpose of this trial is to evaluate the safety, tolerability, pharmacokinetics, and preliminary efficacy of EHP-101 in adult subjects with diffuse cutaneous Systemic Sclerosis (dcSSc).
Detailed Description
An interventional, double-blind, randomized, intracohort placebo-controlled design will be used to test safety, tolerability, pharmacokinetics, and preliminary efficacy of EHP-101 in 36 patients ≥ 18 and ≤ 74 years of age with documented dcSSc. There is a screening period of 28 days, 84 days treatment period, and 28 days follow-up.
Investigators
Eligibility Criteria
Inclusion Criteria
- •Patients male and female ≥18 years and ≤74 years at the time of consent;
- •American College of Rheumatology/ European League Against Rheumatism 2013 Criteria for SSc; dcSSc (skin thickening on upper arms, upper legs, or trunk);
- •Documented SSc for up to 6 years from the first non-Raynaud's phenomenon with a total mRSS of ≥15;
- •No new or increased doses of immunosuppressants medications within 3 months prior to Screening;
- •Effective method of contraception for participants and their partners.
Exclusion Criteria
- •Severe or unstable Systemic Sclerosis (SSc) or SSc with end-stage organ failure;
- •Patient with FVC \<60%;
- •History of clinically significant medical condition or concurrent medical therapies that would exclude the patient, preclude participation in the clinical trial, influence response to study product, or interfere with study assessments;
- •History of gastrointestinal dysmotility requiring total parenteral nutrition or hospitalization within 6 months before Visit 1;
- •Any one of the following values for laboratory tests at screening:
- •Haemoglobin \<9 g/dL;
- •Neutrophils \<1.0 x 10\^9/L;
- •Platelets \<75 x 10\^9/L;
- •Estimated creatinine clearance \<50 mL/min according to the Cockcroft-Gault equation;
- •Serum transaminases \>2.0 x upper normal limit;
Arms & Interventions
EHP-101 low dose once a day
Intervention: Patients will be randomized to receive EHP-101 or Placebo
EHP-101 low dose twice a day
Intervention: Patients will be randomized to receive EHP-101 or Placebo
EHP-101 high dose once a day
Intervention: Patients will be randomized to receive EHP-101 or Placebo
EHP-101 high dose twice a day
Intervention: Patients will be randomized to receive EHP-101 or Placebo
Outcomes
Primary Outcomes
Incidence and severity of Treatment Emergent Adverse Events
Time Frame: Day 113
This safety outcome combines the measure of the number of subjects experiencing adverse events (AEs), the nature and severity of those AEs and their relationship to the study treatments.
Secondary Outcomes
- Treatment effect of EHP-101 compared to placebo as measured by the American College of Rheumatology composite response index in diffuse cutaneous Systemic Sclerosis(Day 85 and Day 113)
- Treatment effect of EHP-101 compared to placebo in modified Rodnan Skin Score(Day 85 and Day 113)
- Treatment effect of EHP-101 compared to placebo in Scleroderma Health Assessment Questionnaire - Disability Index(Day 85 and Day 113)
- Treatment effect of EHP-101 compared to placebo in forced vital capacity percent predicted(Day 85 and Day 113)
- Treatment effect of EHP-101 compared to placebo in physician global assessment score(Day 85 and Day 113)
- Treatment effect of EHP-101 compared to placebo in patient global assessments score(Day 85 and Day 113)