Xeroderma Pigmentosum
Conditions
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.
Interventions
DRUGAfamelanotide
Patients will receive afamelanotide every two weeks for twelve weeks and undergo a follow up visit 6 months later.
Sponsors
Clinuvel Europe Limited
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 75 Years
Healthy volunteers
No
Inclusion criteria
* Male or female patient with a molecular-genetically confirmed diagnosis of XP-C or XP-V; * Aged 18-75 years.
Exclusion criteria
* Known allergy to afamelanotide or the polymer contained in the implant; * Presence of severe hepatic disease or hepatic impairment; * Renal impairment; * Any other medical condition which may interfere with the study protocol; * Existing melanoma; * Female who is pregnant or lactating; * Females of child-bearing potential (pre-menopausal, not surgically sterile) not using adequate contraceptive measures; * Sexually active man with a partner of child-bearing potential who is not using adequate contraceptive measures; * Use of any other prior and concomitant therapy which may interfere with the objective of the study; * Participation in a clinical trial for an investigational agent.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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. |
| 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. |
| 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. |
| 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. |
| 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. |
Countries
Belgium, Spain
Contacts
Primary ContactHead of Clinical Operations
Outcome results
None listed