Systemic Scleroderma
Conditions
Keywords
systemic sclerosis, scleroderma
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.
Detailed description
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 study medication daily for 4 weeks to either the right or left forearm. All subjects will apply placebo (i.e., a topical gel without C-82) to the contralateral forearm. Both physician and subject will be blinded to treatment arm assignment. Skin biopsies will be taken from the mid-forearm of both arms at baseline and after four weeks of study drug to test for biomarker gene expression; instructions and supplies will be provided to each site. Safety assessments will extend to 4 weeks after the final dose of study drug/placebo.
Interventions
active
placebo
Sponsors
Prism Pharma Co., Ltd.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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 |
Countries
United States
Outcome results
None listed