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New treatment option for Congenital Erythropoietic Porphyria (CEP)
Official title 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
ClinicalTrials.gov ID: NCT07024316
What this study is testing
What is ATL-001 (Ciclopirox oral solution)?
ATL-001 (Ciclopirox oral solution) is an investigational medicine, given as an once-daily pill taken by mouth, being studied as a potential treatment for congenital erythropoietic porphyria (cep).
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older
You may be able to join if
- 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...
- a) Female participants must not be pregnant or lactating at screening or admission 3\. b) Female participants must be of nonchildbearing potential or...
You likely can't join if
- 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...
See the full eligibility 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...
- 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...
- 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...
- 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.
The study team makes the final eligibility decision.
Where it's taking place
- Cleveland, Ohio, United States
Compensation & support
Compensation mentioned.
ClinicalTrials.gov doesn't provide a reliable structured field for payment or travel support - confirm details with the study team.
Questions & answers
Do participants get paid in this trial?
This study's listing includes signals that participants may be compensated or receive a stipend. Amounts vary and are set by the study team - confirm the details with them.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Cleveland, Ohio, United States. Enter your location above to see the nearest site and check your eligibility.
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BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.