Clinical trial NCT04166552: Evaluation of Safety, Tolerability and Preliminary Efficacy of EHP-101 in Diffuse Cutaneous Systemic Sclerosis
Official title: 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
Study type: INTERVENTIONAL
Overall status: SUSPENDED
Phase: PHASE2
Conditions: Diffuse Cutaneous Systemic Sclerosis
Keywords: Scleroderma
Systemic sclerosis
EHP-101 Oral Solution
dcSSc
diffuse
pathological processes
connective tissue disease
skin disease
Lead sponsor: Emerald Health Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2019-11-18
Enrollment: 36
Sex: ALL
Minimum age (years): 18
Maximum age (years): 74
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: This is a dose escalation double-blind intracohort trial starting with Cohorts 1 and 2 commencing in parallel and then Cohorts 3 and 4 commencing in parallel
Primary purpose: TREATMENT

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.

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

Primary outcomes:
measure: Incidence and severity of Treatment Emergent Adverse Events
timeFrame: Day 113
Secondary outcomes:
measure: Treatment effect of EHP-101 compared to placebo as measured by the American College of Rheumatology composite response index in diffuse cutaneous Systemic Sclerosis
timeFrame: Day 85 and Day 113
measure: Treatment effect of EHP-101 compared to placebo in modified Rodnan Skin Score
timeFrame: Day 85 and Day 113
measure: Treatment effect of EHP-101 compared to placebo in forced vital capacity percent predicted
timeFrame: Day 85 and Day 113
measure: Treatment effect of EHP-101 compared to placebo in physician global assessment score
timeFrame: Day 85 and Day 113
measure: Treatment effect of EHP-101 compared to placebo in patient global assessments score
timeFrame: Day 85 and Day 113
measure: Treatment effect of EHP-101 compared to placebo in Scleroderma Health Assessment Questionnaire - Disability Index
timeFrame: Day 85 and Day 113
Other outcomes:
measure: Change from baseline of EHP-101 compared to placebo in Patient-Reported Outcomes Measurement Information system-29 Short
timeFrame: Day 85
measure: Change from baseline of EHP-101 compared to placebo in 5 dimensions Itch Score
timeFrame: Day 85
measure: Change from baseline of EHP-101 compared to placebo in University of California, Los Angeles Scleroderma Clinical Trial Consortium Gastrointestinal Tract Questionnaire
timeFrame: Day 85
measure: Change from baseline of EHP-101 compared to placebo in Functional Assessment of Chronic Illness Therapy: Fatigue
timeFrame: Day 85
measure: Change from baseline of EHP-101 compared to placebo in the European Quality of Life 5 Domain Questionnaire
timeFrame: Day 85
measure: Quantitation of drug plasma levels at different timepoints to determine trough
timeFrame: Day 113
Locations:
city: Phoenix
country: United States
facility: Arizona Arthritis & Rheumatology Associates, P.C.
state: Arizona
city: Huntington Beach
country: United States
facility: Care Access Research - Huntington
state: California
city: Los Angeles
country: United States
facility: Pacific Arthritis Care Center
state: California
city: Los Angeles
country: United States
facility: UCLA Division of Rheumatology
state: California
city: Upland
country: United States
facility: Inland Rheumatology Clinical Trials
state: California
city: New Haven
country: United States
facility: Yale University
state: Connecticut
city: Altamonte Springs
country: United States
facility: Central Florida Pulmonary Group
state: Florida
city: Margate
country: United States
facility: Life Clinical Trials
state: Florida
city: Miami
country: United States
facility: Novel Clinical Research Center
state: Florida
city: Chicago
country: United States
facility: Northwestern University
state: Illinois
city: Minneapolis
country: United States
facility: University of Minnesota
state: Minnesota
city: New Brunswick
country: United States
facility: Rutgers, The State University of New Jersey
state: New Jersey
city: Cleveland
country: United States
facility: Cleveland Clinic
state: Ohio
city: Adelaide
country: Australia
facility: Royal Adelaide Hospital
city: Camperdown
country: Australia
facility: Royal Prince Alfred Hospital
city: Footscray
country: Australia
facility: Footscray Hospital
city: Gold Coast
country: Australia
facility: Griffith University
city: Murdoch
country: Australia
facility: Fiona Stanley Hospital
city: Sydney
country: Australia
facility: Westmead Hospital
city: Wellington
country: New Zealand
facility: Wellington Hospital
city: Caguas
country: Puerto Rico
facility: Centro Reumatologico
city: San Juan
country: Puerto Rico
facility: Mindful Medical Research
city: San Juan
country: Puerto Rico
facility: University of Puerto Rico, Medical Sciences Campus
Study officials:
References: