A Study to Investigate the Improvement of Photosensitivity in Terms of Skin Lesions Associated Wi… (NCT07024316) | Clinical Trial Compass
RecruitingPhase 1/2
A Study to Investigate the Improvement of Photosensitivity in Terms of Skin Lesions Associated With CEP Following Administration of Oral ATL-001 (Ciclopirox Oral Solution) in Participants Aged >18 Years of Age With CEP
United States6 participantsStarted 2026-01-29
Plain-language summary
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
Who can participate
Age range18 Years
SexALL
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Inclusion criteria
✓. 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
Exclusion criteria
✕. 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)
✕
What they're measuring
1
To evaluate the efficacy of ATL-001 in patients with CEP in terms of change in lesions associated with photosensitivity.
Timeframe: From Baseline to 12 weeks intervals until the end of study (48 weeks)
. 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