Evaluation of Safety, Tolerability and Preliminary Efficacy of EHP-101 in Diffuse Cutaneous Systemic Sclerosis

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

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).

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.

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;
  • Total bilirubin ≥1.5 x upper limit of normal.