A Study to Evaluate the Safety and Efficacy of Afamelanotide in Patients With Xeroderma Pigmentosum (XP)
Study Details
Study Description
Brief Summary
The CUV156 study will evaluate the safety of afamelanotide in XP-C patients, as well as the drug's ability to assist reparative processes following ultraviolet (UV) provoked DNA damage of the skin. It will assess whether SCENESSE® increases the amount of UV light needed to cause DNA damage of skin cells, as well as the extent of skin repair before and after treatment.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
| Phase 2 |
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: Afamelanotide
|
Drug: Afamelanotide
Patients will receive afamelanotide every two weeks for twelve weeks and undergo a follow up visit 6 months later.
|
Outcome Measures
Primary Outcome Measures
- Change in minimal erythema dose (MED). [From Baseline to Day 76.]
MED is the lowest dose of UV light that causes reddening of the skin.
Secondary Outcome Measures
- Change in UV-induced DNA damage and repair capacity. [From Baseline to Day 76.]
Analysis of UV photoproducts and DNA repair mechanisms.
- Change in skin disease severity (A). [From Baseline to Day 238.]
The higher the score, the more severe the disease.
- Change in skin disease severity (B). [From Baseline to Day 238.]
The higher the score, the more severe the disease.
- Change in skin disease severity (C). [From Baseline to Day 238.]
The higher the score, the more severe the disease.
- Change in dermal melanin density. [From Baseline to Day 238.]
Non-invasive quantitative skin reflectance measurement.
- Change in quality of life assessed by a disease specific tool (A) [From Baseline to Day 238.]
Higher scores represent worse health-related quality of life.
- Change in quality of life assessed by a validated global quality of life tool (B) [From Baseline to Day 238.]
Score calculated in impairment percentages, with higher numbers indicating greater impaired quality of life.
Eligibility Criteria
Criteria
Inclusion Criteria:
-
Male or female patient with a molecular-genetically confirmed diagnosis of XP-C;
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Aged 18-75 years.
Exclusion Criteria:
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Known allergy to afamelanotide or the polymer contained in the implant;
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Presence of severe hepatic disease or hepatic impairment;
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Renal impairment;
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Any other medical condition which may interfere with the study protocol;
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Female who is pregnant (confirmed by positive urine beta-Human chorionic gonadotropin pregnancy test) or lactating;
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Females of child-bearing potential (pre-menopausal, not surgically sterile) not using highly effective contraceptive measures with a failure rate of less than 1% per year when used consistently and correctly (i.e. oral contraceptives, intrauterine device) or a life-style excluding pregnancy, for up to three months after the last implant administration;
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Sexually active man with a partner of child-bearing potential (pre-menopausal, not surgically sterile) who is not using highly effective contraceptive measures, as described above;
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Use of any other prior and concomitant therapy which may interfere with the objective of the study, within 30 days prior to the Screening visit;
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Participation in a clinical trial for an investigational agent within 30 days prior to the Screening visit.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | CLINUVEL investigative site | Regensburg | Germany |
Sponsors and Collaborators
- Clinuvel Europe Limited
Investigators
None specified.Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- CUV156