ATL-001 for Congenital Erythropoietic Porphyria
(ATL001-PII-CEP Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests an oral solution called ATL-001 (ciclopirox) to determine if it benefits individuals with Congenital Erythropoietic Porphyria (CEP). Researchers aim to see if it can improve skin issues, fatigue, and other symptoms while monitoring blood health. The trial is open to those diagnosed with CEP who are willing to track their symptoms and adhere to the study guidelines. As a Phase 1, Phase 2 trial, this research focuses on understanding how the treatment works in people and measuring its effectiveness in an initial, smaller group.
Will I have to stop taking my current medications?
The trial does not clearly state if you need to stop taking your current medications. However, if you are taking afamelanotide or dersimelagon, you must stop them at least 2 months before starting the trial.
Is there any evidence suggesting that ATL-001 (Ciclopirox Oral Solution) is likely to be safe for humans?
Research shows that ciclopirox, the main ingredient in ATL-001, is usually safe when used on the skin for treating fungal infections. Currently, it is being tested as a liquid medicine taken by mouth for a rare blood condition called congenital erythropoietic porphyria (CEP).
Early studies have assessed the safety of oral administration. When adults took it, some reported minor side effects, similar to those from topical use, such as mild skin irritation. Since ciclopirox is already approved for other uses, much is known about its safety. However, using it orally for CEP is new, so further research is needed to ensure its safety in this form.
In summary, while ciclopirox has been safely used on the skin, its use as a liquid medicine for CEP remains under study. Trial participants will contribute to understanding its effectiveness and safety for treating CEP.12345Why do researchers think this study treatment might be promising?
Unlike the standard treatments for Congenital Erythropoietic Porphyria, which often involve blood transfusions or bone marrow transplants, ATL-001 (Ciclopirox Oral Solution) offers a potentially groundbreaking approach. Researchers are excited because ATL-001 works by targeting the underlying biochemical processes of the disease rather than just managing symptoms. This oral solution form also provides a non-invasive and more convenient alternative to current therapies, which can be burdensome for patients. If successful, ATL-001 could significantly improve quality of life for those affected by this rare condition.
What evidence suggests that ATL-001 might be an effective treatment for CEP?
Research shows that ATL-001, a liquid medicine taken by mouth, may help treat Congenital Erythropoietic Porphyria (CEP). Studies have found that this treatment can improve enzyme activity in lab tests, potentially reducing symptoms like skin sores and tiredness in people with CEP. Early results suggest that ATL-001 can ease many symptoms linked to CEP. The drug is designed for better absorption by the body, which could enhance its effectiveness. While more research is needed, these early findings offer hope for those living with CEP.16789
Are You a Good Fit for This Trial?
Adults over 18 with Congenital Erythropoietic Porphyria (CEP) can join this trial. They must not be pregnant or breastfeeding, agree to use effective contraception, and have a negative pregnancy test. Participants should understand the study and consent to it, follow procedures, complete diaries/questionnaires, and meet specific medical criteria for CEP diagnosis.Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Run-in
Participants undergo a 6-month run-in period to establish baseline measurements
Treatment
Participants receive oral ATL-001 (ciclopirox) to evaluate improvement in skin lesions and other symptoms
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- ATL-001 (Ciclopirox Oral Solution)
Trial Overview
The trial is testing ATL-001 (Ciclopirox oral solution) to see if it improves skin lesions, fatigue, and other symptoms in patients with CEP. It also monitors blood parameters. The study's effectiveness will be measured throughout its duration.
How Is the Trial Designed?
1
Treatment groups
Experimental Treatment
Find a Clinic Near You
Who Is Running the Clinical Trial?
Atlas Molecular Pharma
Lead Sponsor
Citations
NCT07024316 | A Study to Investigate the Improvement of ...
To evaluate the efficacy of ATL-001 in patients with CEP in terms of change in lesions associated with photosensitivity.
Improving the Pharmacological Properties of Ciclopirox for ...
ATLAS molecular Pharma is developing the drug ciclopirox for its use in congenital erythropoietic porphyria. Footnotes. Publisher's Note ...
Statistical Analysis Plan
ATL-001 is a new oral formulation of ciclopirox olamine, including alginate and tocophersolan, currently under development for the treatment of ...
Repurposing ciclopirox as a pharmacological chaperone in ...
The drug restored enzymatic activity in vitro and ex vivo and was able to alleviate most clinical symptoms of congenital erythropoietic porphyria.
A formulation for an effective oral administration of ...
The results of this study showed that there is a dose-dependency in the C max and AUC for increasing doses of ATL-001, as both AUC and C max for ...
Ciclopirox | C12H17NO2 | CID 2749 - PubChem - NIH
It is used a a topical treatment of fungal skin and nail infections. Treatment of congenital erythropoietic porphyria. After oral administration of 10 mg of ...
Ciclopirox: Uses, Interactions, Mechanism of Action
Ciclopirox is a broad-spectrum topical antifungal agent used to treat mild to moderate onychomycosis of fingernails and toenails. Congenital Erythropoietic ...
Improving the Pharmacological Properties of Ciclopirox for ...
ATLAS molecular Pharma is developing the drug ciclopirox for its use in congenital erythropoietic porphyria. Abbreviations. ADME, absorption ...
CICLOPIROX – Application in Therapy and Current Clinical ...
oral solution (ATL-001) CEP causes severe photosensitivity, with patients developing painful skin lesions. Common side effects of topical ciclopirox may ...
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