A Study to Evaluate the Safety and Efficacy of Afamelanotide in Patients With Xeroderma Pigmentosum C and V
Study Details
Study Description
Brief Summary
The CUV152 study will evaluate the safety of afamelanotide in XP-C and XP-V 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) in patients with XP-C. [From baseline to day 76.]
MED is the lowest dose of UV light that causes reddening of the skin.
- Change in MED in patients with XP-V. [From baseline to day 76.]
MED is the lowest dose of UV light that causes reddening of the skin.
Secondary Outcome Measures
- Changes in UV-induced DNA damage, as assessed by quantification of UV photoproduct levels in patients with XP-C. [From baseline to day 76.]
Analysis of UV photoproducts from skin samples.
- Changes in UV-induced DNA damage repair capacity, as assessed by quantification of DNA repair mechanisms in patients with XP-C. [From baseline to day 76.]
Analysis of DNA repair mechanisms from skin samples.
- Changes in UV-induced DNA damage, as assessed by quantification of UV photoproduct levels in patients with XP-V. [From baseline to day 76.]
Analysis of UV photoproducts from skin samples.
- Changes in UV-induced DNA damage repair capacity, as assessed by quantification of DNA repair mechanisms in patients with XP-V. [From baseline to day 76.]
Analysis of DNA repair mechanisms from skin samples.
- Change in skin disease severity in patients with XP-C (A). [From baseline to day 76.]
The higher the score, the more severe the disease.
- Change in skin disease severity in patients with XP-V (A) [From baseline to day 76.]
The higher the score, the more severe the disease.
- Change in skin disease severity in patients with XP-C (B). [From baseline to day 76.]
The higher the score, the more severe the disease.
- Change in skin disease severity in patients with XP-V (B). [From baseline to day 76.]
The higher the score, the more severe the disease.
- Change in skin disease severity in patients with XP-C (C). [From baseline to day 76.]
The higher the score, the more severe the disease.
- Change in skin disease severity in patients with XP-V (C). [From baseline to day 76.]
The higher the score, the more severe the disease.
- Change in quality of life assessed by a disease specific tool (A) in patients with XP-C. [From baseline to day 76.]
Higher scores represent worse health-related quality of life.
- Change in quality of life assessed by a disease specific tool (A) in patients with XP-V. [From baseline to day 76.]
Higher scores represent worse health-related quality of life.
- Change in quality of life assessed by a validated global quality of life tool (B) in patients with XP-C. [From baseline to day 76.]
Score calculated in impairment percentages, with higher numbers indicating greater impaired quality of life.
- Change in quality of life assessed by a validated global quality of life tool (B) in patients with XP-V. [From baseline to day 76.]
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 or XP-V;
-
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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Existing melanoma;
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Female who is pregnant or lactating;
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Females of child-bearing potential (pre-menopausal, not surgically sterile) not using adequate contraceptive measures;
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Sexually active man with a partner of child-bearing potential who is not using adequate contraceptive measures;
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Use of any other prior and concomitant therapy which may interfere with the objective of the study;
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Participation in a clinical trial for an investigational agent.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | CLINUVEL Investigational site | Clinuvel Investigational Site | Belgium | ||
| 2 | CLINUVEL Investigational site | Clinuvel Investigational Site | Spain |
Sponsors and Collaborators
- Clinuvel Europe Limited
Investigators
None specified.Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- CUV152