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

NCT04166552 | Suspended Phase 2 DMC FDA Drug

• 1 other identifier: EHP-101-SS01
• Study Type: interventional
• Target: 36
• Locations: 4 countries
• Sites: 23

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).

Conditions

Diffuse Cutaneous Systemic Sclerosis

Keywords

Scleroderma; Systemic sclerosis; EHP-101 Oral Solution; dcSSc; diffuse; pathological processes; connective tissue disease; skin disease

Outcome Measures

Primary Outcomes (1)

• Incidence and severity of Treatment Emergent Adverse Events

  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.

  Day 113

Secondary Outcomes (6)

• 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 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

Other Outcomes (6)

• Change from baseline of EHP-101 compared to placebo in Patient-Reported Outcomes Measurement Information system-29 Short

  Day 85

• Change from baseline of EHP-101 compared to placebo in 5 dimensions Itch Score

  Day 85

• Change from baseline of EHP-101 compared to placebo in University of California, Los Angeles Scleroderma Clinical Trial Consortium Gastrointestinal Tract Questionnaire

  Day 85

Study Arms (4)

EHP-101 low dose once a day

EXPERIMENTAL

Drug: Patients will be randomized to receive EHP-101 or Placebo

EHP-101 low dose twice a day

EXPERIMENTAL

Drug: Patients will be randomized to receive EHP-101 or Placebo

EHP-101 high dose once a day

EXPERIMENTAL

Drug: Patients will be randomized to receive EHP-101 or Placebo

EHP-101 high dose twice a day

EXPERIMENTAL

Drug: Patients will be randomized to receive EHP-101 or Placebo

Interventions

Patients will be randomized to receive EHP-101 or Placebo DRUG

EHP-101 or placebo will be taken once a day

EHP-101 high dose once a day; EHP-101 low dose once a day

Eligibility Criteria

• Age: 18 Years - 74 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Emerald Health Pharmaceuticals: lead

Study Sites (23)

Arizona Arthritis & Rheumatology Associates, P.C.

Phoenix, Arizona, 85032, United States

Location

Care Access Research - Huntington

Huntington Beach, California, 92648, United States

Location

Pacific Arthritis Care Center

Los Angeles, California, 90045, United States

Location

UCLA Division of Rheumatology

Los Angeles, California, 90095, United States

Location

Inland Rheumatology Clinical Trials

Upland, California, 91786, United States

Location

Yale University

New Haven, Connecticut, 06511, United States

Location

Central Florida Pulmonary Group

Altamonte Springs, Florida, 32701, United States

Location

Life Clinical Trials

Margate, Florida, 33063, United States

Location

Novel Clinical Research Center

Miami, Florida, 33186, United States

Location

Northwestern University

Chicago, Illinois, 60208, United States

Location

University of Minnesota

Minneapolis, Minnesota, 55455, United States

Location

Rutgers, The State University of New Jersey

New Brunswick, New Jersey, 08901, United States

Location

Cleveland Clinic

Cleveland, Ohio, 44195, United States

Location

Royal Adelaide Hospital

Adelaide, 5000, Australia

Location

Royal Prince Alfred Hospital

Camperdown, 2050, Australia

Location

Footscray Hospital

Footscray, 3011, Australia

Location

Griffith University

Gold Coast, 9726, Australia

Location

Fiona Stanley Hospital

Murdoch, 6150, Australia

Location

Westmead Hospital

Sydney, 2145, Australia

Location

Wellington Hospital

Wellington, 6021, New Zealand

Location

Centro Reumatologico

Caguas, 00725, Puerto Rico

Location

Mindful Medical Research

San Juan, 00918, Puerto Rico

Location

University of Puerto Rico, Medical Sciences Campus

San Juan, 00935, Puerto Rico

Location

MeSH Terms

Conditions

Scleroderma, Diffuse; Scleroderma, Systemic; Pathologic Processes; Connective Tissue Diseases; Skin Diseases

Condition Hierarchy (Ancestors)

Skin and Connective Tissue Diseases; Pathological Conditions, Signs and Symptoms

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Masking Details: Double (intracohort)
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: 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

Study Record Dates

• First Submitted: November 6, 2019
• First Posted: November 18, 2019
• Study Start: June 11, 2020
• Primary Completion: July 1, 2024
• Study Completion: October 1, 2024
• Last Updated: September 21, 2023

Record last verified: 2022-09

Data Sharing

• IPD Sharing: Will not share

Locations

AU (6); NZ (1); PR (3); US (13)