NCT07024316招募中1 期
A Phase I-II, N-of-1, Open-Label, Prospective Study to Evaluate the Efficacy and Safety of ATL-001 in Adult Patients With Congenital Erythropoietic Porphyria (CEP)
Atlas Molecular Pharma1 个研究点 分布在 1 个国家目标入组 6 人开始时间: 2026年1月29日最近更新:
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 发起方
- 入组人数
- 6
- 试验地点
- 1
- 主要终点
- To evaluate the efficacy of ATL-001 in patients with CEP in terms of change in lesions associated with photosensitivity.
研究概览
简要总结
This is a study to investigate the effect of oral ATL-001 (ciclopirox) in CEP (Congenital Erythropoietic Porphyria) patients. During the study, it will be measured the improvement of skin lesions, fatigue and other clinical symptoms as well as blood parameters. Funding source - FDA OOPD
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
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
- •Aged 18 years or older at the time of signing the informed consent form (ICF)
- •Diagnosis of CEP, based on medical history and/or biochemical porphyrin analysis. Sponsor will review the CEP diagnostic criteria (medical history or biochemical porphyrin analysis) of each potential patient, written Sponsor approval before starting the run-in period will be required
- •a) Female participants must not be pregnant or lactating at screening or admission
- •3. b) Female participants must be of nonchildbearing potential or agree to use 2 adequate forms of highly effective method(s) of contraception throughout the entire duration of study participation and for at least 4 weeks after the last dose of any study intervention and have negative pregnancy test results at screening (serum) and admission (urine)
- •Male participants with female partners of childbearing potential must be surgically sterile (successful vasectomy at least 4 weeks prior to Day 1) or agree to use adequate forms of highly effective contraception (together with their female partner) for the duration of the study and for at least 4 weeks after the last dose of study intervention; must also agree to refrain from sperm donation during the study and for at least 4 weeks after the last dose of study intervention
- •Negative pregnancy test (females of childbearing potential) at Screening (Month -6), at Baseline (Day 1) and at every visit site until EoS prior to dosing
- •Able to understand the study aims, procedures, and requirements, and provide written informed consent
- •Able to comply with all study procedures
- •Able and committed to complete study patient diaries and questionnaires
排除标准
- •Other than CEP, an inherited or acquired red cell disease associated with anemia
- •Any other photodermatosis such as solar urticaria
- •A history or known allergic reaction to any investigational product excipients or the investigational drug
- •Major surgery within 8 weeks before Screening, incomplete recovery from any previous surgery or planned major surgery during the study
- •History of alcohol dependence or excessive alcohol consumption, as assessed by the Investigator
- •Human immunodeficiency virus (HIV), active Hepatitis B, or C. A positive hepatitis result should be discussed between the Investigator and Sponsor prior to enrolment
- •Score of Personal Health Questionnaire Depression Scale (PHQ-8) ≥10 at screening or any response of "yes" on the Columbia-Suicide Severity Rating Scale (C-SSRS)
- •Uncontrolled medical condition or concomitant medication that would confound the ability to interpret clinical, clinical laboratory, or participant diary data, including a major psychiatric condition that has had an exacerbation or required hospitalization in the last 6 months
- •Administration of afamelanotide and dersimelagon within the last 2 months prior to start of the run-in period
- •Total bilirubin >2× ULN (unless documented Gilbert syndrome) at Screening
- •If female, pregnant or breastfeeding. Final_V2.1 38 of 106 11 Apr 2025
- •Participation in any other clinical protocol or investigational trial that involves administration of experimental therapy and/or therapeutic devices within 30 days of Screening.
研究组 & 干预措施
ATL-001
Experimental
干预措施: ATL-001 (Ciclopirox oral solution) (Drug)
结局指标
主要结局
To evaluate the efficacy of ATL-001 in patients with CEP in terms of change in lesions associated with photosensitivity.
时间窗: From Baseline to 12 weeks intervals until the end of study (48 weeks)
Change between 6-month run-in period and 12-weekly treatment intervals in number and severity of sunlight-induced events measured with the Dermatological Sunlight Questionnaire (DSQ) on a daily basis: * Number of lesions * Average time to recovery of lesions * Number of other symptoms and clinical signs
次要结局
未报告次要终点
研究者
研究点 (1)
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