Trial of Topical C-82 in Systemic Sclerosis - A Phase I/II Biomarker and Safety Trial

NCT02349009 | Completed Phase 1 DMC

• 1 other identifier: PRI-C82T-3101
• Study Type: interventional
• Target: 17
• Locations: 1 country
• Sites: 3

Brief Summary

1:1 active treatment: placebo, blinded trial, evaluating the effect of a 4-week treatment period with topical C-82 on skin expression of two gene biomarker surrogates (THBS1 and COMP) for the modified Rodnan skin score (MRSS). Study subjects will be randomized to apply the active study medication daily for 4 weeks to either the right or left forearm and placebo to the contralateral forearm.

Conditions

Systemic Scleroderma

Keywords

systemic sclerosis; scleroderma

Outcome Measures

Primary Outcomes (2)

• frequency and character of adverse events and abnormal clinical tests

  28 days

• change in gene biomarker expression of THBS1 and COMP in skin biopsies over time compared to placebo

  28 days

Study Arms (2)

placebo

PLACEBO COMPARATOR

C-82 Topical Gel, Placebo

Drug: C-82 Topical Gel, Placebo

Active

ACTIVE COMPARATOR

C-82 Topical Gel, 1%

Drug: C-82 Topical Gel, 1%

Interventions

C-82 Topical Gel, 1% DRUG

active

Active

C-82 Topical Gel, Placebo DRUG

placebo

placebo

Eligibility Criteria

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

You may qualify if:

• meet the American College of Rheumatology criteria for systemic sclerosis with diffuse cutaneous involvement (clinical skin involvement proximal to forearms and or knees, not including the face).
• Disease duration of \<36 months since the onset of the first SSc manifestation other than Raynaud's phenomenon, or patients with progressive disease based on new or worsening skin disease based on physician assessment.
• local skin score over the bilateral forearm of ≥ 2.
• a MRSS of ≥ 12.
• on stable dose of any immunosuppressive other than cyclophosphamide or high dose steroids (excluded treatments) for at least one month and through the course of study treatment.
• Subjects of child-producing potential must agree to use effective contraception while and for at least 3 months after the last treatment.

You may not qualify if:

• Receiving treatment as part of an interventional clinical trial within 4 weeks of screening or 5 half-lives of the investigational drug (whichever is longer).
• Ongoing use of high dose steroids (\> 10mg/day prednisone or equivalent) or unstable steroid dose in the past 4 weeks.
• Use of topical creams or gels on the forearm area within the past month and through the course of study treatment.
• UV light therapy for 4 weeks before or during the study period.
• Treatment with cyclophosphamide within the past month and through the course of study treatment.
• Known active bacterial, viral fungal mycobacterial, or other infection
• history of malignancy within the past 2 years.
• Moderate to severe hepatic impairment, .
• Scleroderma renal crisis within 4 months or creatinine greater than 2.0.
• Pregnancy.
• Nursing mothers are to be excluded.
• Gastrointestinal involvement requiring total parenteral nutrition or hospitalization within the past 3 months for pseudo-obstruction
• Moderately severe pulmonary disease with FVC \< 40%, or DLCO \< 30% predicted or evidence of progressive lung disease as manifest by a decrease in FVC or 10% or more over the previous year.
• Moderately severe cardiac disease with clinically significant heart failure, or unstable angina.
• AST or ALT \> 2.5 x Upper Limit of Normal.

Sponsors & Collaborators

• Prism Pharma Co., Ltd.: lead

Study Sites (3)

Unknown Facility

Chicago, Illinois, United States

Location

Unknown Facility

Boston, Massachusetts, United States

Location

Unknown Facility

New York, New York, United States

Location

MeSH Terms

Conditions

Scleroderma, Systemic; Scleroderma, Diffuse

Condition Hierarchy (Ancestors)

Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases

Study Officials

• Robert Lafyatis, MD

  Boston University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 18, 2015
• First Posted: January 28, 2015
• Study Start: June 1, 2015
• Primary Completion: July 1, 2016
• Study Completion: September 1, 2016
• Last Updated: August 17, 2017

Record last verified: 2016-09

Locations

US (3)