A Study to Evaluate the Safety and Efficacy of Afamelanotide in Patients With Xeroderma Pigmentosum C and V

Overview

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.

Full Title of Study: “A Proof of Concept, Phase IIa, Open Label Study to Evaluate the Safety and Efficacy of Subcutaneous Implants of Afamelanotide in Patients With Xeroderma Pigmentosum C and V (XPC and XPV)”

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: N/A
    • Intervention Model: Single Group Assignment
    • Primary Purpose: Treatment
    • Masking: None (Open Label)
  • Study Primary Completion Date: June 2024

Interventions

  • Drug: Afamelanotide
    • Patients will receive afamelanotide every two weeks for twelve weeks and undergo a follow up visit 6 months later.

Arms, Groups and Cohorts

  • Experimental: Afamelanotide

Clinical Trial Outcome Measures

Primary Measures

  • Change in minimal erythema dose (MED) in patients with XP-C.
    • Time Frame: 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.
    • Time Frame: From baseline to day 76.
    • MED is the lowest dose of UV light that causes reddening of the skin.

Secondary Measures

  • Changes in UV-induced DNA damage, as assessed by quantification of UV photoproduct levels in patients with XP-C.
    • Time Frame: 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.
    • Time Frame: 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.
    • Time Frame: 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.
    • Time Frame: From baseline to day 76.
    • Analysis of DNA repair mechanisms from skin samples.
  • Change in skin disease severity in patients with XP-C (A).
    • Time Frame: 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)
    • Time Frame: 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).
    • Time Frame: 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).
    • Time Frame: 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).
    • Time Frame: 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).
    • Time Frame: 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.
    • Time Frame: 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.
    • Time Frame: 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.
    • Time Frame: 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.
    • Time Frame: From baseline to day 76.
    • Score calculated in impairment percentages, with higher numbers indicating greater impaired quality of life.

Participating in This Clinical Trial

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.

Gender Eligibility: All

Minimum Age: 18 Years

Maximum Age: 75 Years

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • Clinuvel Europe Limited
  • Provider of Information About this Clinical Study
    • Sponsor
  • Overall Contact(s)
    • Head of Clinical Operations, +441372860765, mail@clinuvel.com

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