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

• Status: Completed
• Trial end date: 2016-09-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Prism Pharma Co., Ltd.

Criteria

Inclusion Criteria:

• 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.

Exclusion Criteria:

• 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.

• Total bilirubin > 1.5 x upper limit of normal (ULN). Patients with Gilbert's Disease may be included if their total bilirubin is ≤ 3.0 mg/dL.

• significant medical or psychosocial problems that warrant exclusion.