Phase 1b Clinical Trial Using Topical Sirolimus for the Treatment of Pachyonychia Congenita
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 15
- 试验地点
- 1
- 主要终点
- Evaluation of Systemic Absorption Through Measurement of Serum Sirolimus Trough Levels
研究概览
简要总结
A study to evaluate safety and efficacy of topical sirolimus to treat plantar keratoderma in adults with PC. Subjects may receive either placebo or treatment with at least 1 foot receiving topical sirolimus at some time. For certain phases of the study treatment assignment to the right and left foot will be randomized in a double blind fashion. Blood levels will test systemic absorption of sirolimus. Other safety and efficacy measures will be taken through the 39-week study duration. Funding Source - FDA OOPD
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 16 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subjects must:
- •Be capable of understanding the purpose and risks of the study and sign a written Informed Consent Form (ICF)
- •Be male or female ≥ 16 years of age at the time of the screening visit
- •Have a confirmed diagnosis of PC by genotyping (e.g., familial) and clinically correlated painful keratoderma.
- •Have roughly symmetrical calluses of similar severity on the plantar surface of both feet
- •Women of childbearing potential must have a negative serum pregnancy test
- •Subjects, whether male or female, with reproductive potential and who are sexually active must agree to use double-barrier contraception methods
排除标准
- •A Subject with any of the following criteria is not eligible for inclusion in this study:
- •Use of other investigational drugs within 30 days of the screening visit and/or has not recovered from any side effects of prior investigational drugs or procedure in the affected area (e.g., a biopsy)
- •Significant condition in the dermatologic treatment area such as trauma, or nonhealing chronic wound
- •Pregnant or nursing (lactating) female, or a positive serum pregnancy test
- •Active infection either systemic or local near the site of treatment requiring chronic or prolonged use of antimicrobial agents
- •Known immunodeficiency including: Hepatitis A; Hepatitis B; Hepatitis C; Human Immunodeficiency Virus (HIV)
- •Prior and Current Treatment
- •Unable to be discontinued from drugs known to either be inducers or inhibitors of CYP3A4/5 enzymes
- •Unable to be discontinued from drugs known to be P-glycoprotein inhibitors
研究组 & 干预措施
Split-body 1% sirolimus cream (TD201 1%)
This is a split-body design. Subjects will self-administer 1% topical sirolimus cream or placebo cream (no drug, vehicle control) on the plantar surface of each foot. At least one foot will be treated with topical sirolimus at some time during the study. Application will be one time daily for a total of 26 weeks. There will be an additional follow-up visit 3 months after the last application of study drug. The total duration of the study is 39 weeks.
干预措施: 1% sirolimus cream (TD201 1%) (Drug)
结局指标
主要结局
Evaluation of Systemic Absorption Through Measurement of Serum Sirolimus Trough Levels
时间窗: Two weeks and every 1-2 months for 24 weeks or within 2 weeks after the last dose of study drug
The primary outcome measure for this Phase 1b safety study is evaluation of system absorption through measurement of serum sirolimus trough levels. The limit of detection of the assay was 2.0 ng/mL.
次要结局
- Weekly Assessments Recorded in the PC Quality of Life Index(Weekly for 39 weeks)
- Daily Assessments Recording in the PC Measurement Diary(Weekly for 39 weeks)