A Randomized, Double-Blind, Placebo-Controlled Trial of Topical C-82 in Systemic Sclerosis - A Phase I/II Biomarker and Safety Trial
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 17
- 主要终点
- frequency and character of adverse events and abnormal clinical tests
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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 ≥
- •a MRSS of ≥
- •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.
排除标准
- •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.
- •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.
研究组 & 干预措施
placebo
C-82 Topical Gel, Placebo
干预措施: C-82 Topical Gel, Placebo (Drug)
Active
C-82 Topical Gel, 1%
干预措施: C-82 Topical Gel, 1% (Drug)
结局指标
主要结局
frequency and character of adverse events and abnormal clinical tests
时间窗: 28 days
change in gene biomarker expression of THBS1 and COMP in skin biopsies over time compared to placebo
时间窗: 28 days
次要结局
未报告次要终点