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
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
6
Daily administration of oral ATL-001
The Cleveland Clinics
Cleveland, Ohio, United States
RECRUITINGTo evaluate the efficacy of ATL-001 in patients with CEP in terms of change in lesions associated with photosensitivity.
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
Time frame: From Baseline to 12 weeks intervals until the end of study (48 weeks)
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