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 Pharma
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
Eligibility
- Age range
- 18+ years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: 1. Aged 18 years or older at the time of signing the informed consent form (ICF) 2. 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 3. 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…
Interventions
- DrugATL-001 (Ciclopirox oral solution)
Daily administration of oral ATL-001
Location
- The Cleveland ClinicsCleveland, Ohio