Congenital Erythropoietic Porphyria (CEP)Phase 1/2ATL001-PII-CEPNCT07024316
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
A Phase 1/2 study testing ATL-001 (Ciclopirox oral solution) in people with Congenital Erythropoietic Porphyria (CEP), run by Atlas Molecular Pharma at 1 site in United States.
Trial 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 Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Congenital Erythropoietic Porphyria (CEP)
- What is being tested?
- ATL-001 (Ciclopirox oral solution)
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 1 siteUnited States
- Study dates
- 29 Jan 2026to 1 Jun 2028
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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) 4. 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 5. 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 6. Able to understand the study aims, procedures, and requirements, and provide written informed consent 7. Able to comply with all study procedures 8. Able and committed to complete study patient diaries and questionnaires
Who cannot join
- 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
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- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
The Cleveland Clinics
Cleveland, Ohio, 44195
Clinical Research Project Manager, 216-780-9515, Laruscg@ccf.org
Angelika Erwin, M.D.
About this study
Official title: 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)
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
- Being tested
- ATL-001 (Ciclopirox oral solution)
- Medical subject headings
- Porphyria, Erythropoietic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ATL-001 (Ciclopirox oral solution)Drug
Daily administration of oral ATL-001
| Study group | Role | What they receive |
|---|---|---|
| ATL-001 | Experimental | Not described Receives: Drug: ATL-001 (Ciclopirox oral solution) |
What is being measured
The primary outcome is the main question the study is designed to answer.
To 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
Measured: From Baseline to 12 weeks intervals until the end of study (48 weeks)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Jun 2025
- Study start29 Jan 2026
- Primary completion1 May 2028 (estimated)
- Study completion1 Jun 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Congenital Erythropoietic Porphyria (CEP)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- N-of-1, Open-Label, Prospective Study. One group treated with the investigational drug
- Masking
- None (open label)
- Enrolment
- 6 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Atlas Molecular Pharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07024316
- Sponsor's protocol ID
- ATL001-PII-CEP
- Other IDs
- 1R01FD008540-01
- Acronym
- ATL001-PII-CEP
- Status last verified
- Mar 2026
- First submitted
- 3 Jun 2025
- Last updated
- 24 Mar 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 24 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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)
- ClinicalTrials.gov ID
- NCT07024316
- Acronym / sponsor ID
- ATL001-PII-CEP / ATL001-PII-CEP
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 6 participants (estimated)
- Lead sponsor
- Atlas Molecular PharmaIndustry (pharma, biotech, devices)
- Study start
- 29 Jan 2026 (actual)
- Primary completion
- 1 May 2028 (estimated)
- Study completion
- 1 Jun 2028 (estimated)
- First posted
- 17 Jun 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- N-of-1, Open-Label, Prospective Study. One group treated with the investigational drug
| Arm | Type | Receives |
|---|---|---|
| ATL-001 | Experimental | Drug: ATL-001 (Ciclopirox oral solution) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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) 4. 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 5. 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 6. Able to understand the study aims, procedures, and requirements, and provide written informed consent 7. Able to comply with all study procedures 8. Able and committed to complete study patient diaries and questionnaires
Key exclusion criteria (12 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ATL-001 (Ciclopirox oral solution)Daily administration of oral ATL-001 | Drug | ATL-001 |
Primary outcome
To evaluate the efficacy of ATL-001 in patients with CEP in terms of change in lesions associated with photosensitivity.
Time frame: 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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Mar 2026.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing ATL-001 (Ciclopirox oral solution) in people with Congenital Erythropoietic Porphyria (CEP), run by Atlas Molecular Pharma at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 24 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Cristina Garrido (+34 688 849 117, cgarrido@atlasmolecularpharma.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To evaluate the efficacy of ATL-001 in patients with CEP in terms of change in lesions associated with photosensitivity. (time frame: From Baseline to 12 weeks intervals until the end of study (48 weeks)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Mar 2026.