A Study to Evaluate the Safety and Efficacy of Afamelanotide in Patients With Xeroderma Pigmentosum (XP)

NCT05159752 | Unknown Phase 2 FDA Drug

• 1 other identifier: CUV156
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

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.

Conditions

Xeroderma Pigmentosum

Outcome Measures

Primary Outcomes (1)

• Change in minimal erythema dose (MED).

  MED is the lowest dose of UV light that causes reddening of the skin.

  From Baseline to Day 76.

Secondary Outcomes (7)

• Change in UV-induced DNA damage and repair capacity.

  From Baseline to Day 76.

• Change in skin disease severity (A).

  From Baseline to Day 238.

• Change in skin disease severity (B).

  From Baseline to Day 238.

• Change in skin disease severity (C).

  From Baseline to Day 238.

• Change in dermal melanin density.

  From Baseline to Day 238.

Study Arms (1)

Afamelanotide

EXPERIMENTAL

Drug: Afamelanotide

Interventions

Afamelanotide DRUG

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

Afamelanotide

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female patient with a molecular-genetically confirmed diagnosis of XP-C;
• Aged 18-75 years.

You may not qualify if:

• 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;
• Female who is pregnant (confirmed by positive urine beta-Human chorionic gonadotropin pregnancy test) or lactating;
• 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;
• 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;
• 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;
• Participation in a clinical trial for an investigational agent within 30 days prior to the Screening visit.

Sponsors & Collaborators

• Clinuvel Europe Limited: lead

Study Sites (1)

CLINUVEL investigative site

Regensburg, Germany

RECRUITING

MeSH Terms

Conditions

Xeroderma Pigmentosum

Interventions

afamelanotide

Condition Hierarchy (Ancestors)

Precancerous Conditions; Neoplasms; Skin Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases, Genetic; Genetic Diseases, Inborn; Photosensitivity Disorders; Skin Diseases; Skin and Connective Tissue Diseases; Pigmentation Disorders; DNA Repair-Deficiency Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases

Central Study Contacts

Head of Clinical Operations

CONTACT

+441372860765; mail@clinuvel.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 9, 2021
• First Posted: December 16, 2021
• Study Start: October 19, 2021
• Primary Completion: March 1, 2024
• Study Completion: October 1, 2024
• Last Updated: June 18, 2023

Record last verified: 2023-06

Locations

DE (1)