Study to Evaluate the Safety and Efficacy of Afamelanotide in Patients With Variegate Porphyria (VP)

NCT05854784 | Completed Phase 2 Results FDA Drug

• 1 other identifier: CUV040
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

Brief Summary

The primary objective is to evaluate the impact of afamelanotide on the severity of skin disease in patients with Variegate Porphyria (VP). The secondary objectives are to evaluate the safety and tolerability of afamelanotide in patients with VP and evaluate the impact of afamelanotide on the quality of life of patients with VP.

Conditions

Variegate Porphyria

Outcome Measures

Primary Outcomes (1)

• The Change in Disease Severity in Patients With VP as Measured by CGIC.

  The higher the score, the less severe the disease. Scale ranges from +3 to -3.

  From baseline to Day 168

Secondary Outcomes (9)

• The Change in Disease Severity in Patients With VP as Measured by 11-point VAS IGA.

  Median change from baseline to Day 168.

• The Change in Disease Severity in Patients With VP as Measured by 5-point IGA.

  Median change from baseline to Day 168

• The Change in Disease Severity in Patients With VP as Measured by PGIC.

  Median change from baseline to Day 168

• The Change in Disease Severity in Patients With VP as Measured by PGA Using VAS.

  Median change from baseline to Day 168

• The Change in Number of New Skin Lesions Formed.

  Median change from baseline to Day 168.

Study Arms (1)

Afamelanotide

EXPERIMENTAL

Drug: Afamelanotide 16 MG

Interventions

Afamelanotide 16 MG DRUG

Eligible patients will receive one dose of afamelanotide 16mg every 28 days (up to six doses), as a controlled-release formulation.

Afamelanotide

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female patients with confirmed diagnosis of VP.
• Patients with VP-related skin symptoms.
• Aged 18-70 years.

You may not qualify if:

• Allergy to afamelanotide or polymer or to local anaesthetic to be used if applicable.
• Had two or more acute attacks of hepatic porphyria, as defined by the occurrence or acute porphyric symptoms within 12 months prior to the Screening period.
• Individual history of malignant or premalignant skin lesions.
• Individual or family history of melanoma.
• Presence of severe hepatic disease.
• Renal impairment.
• Female who is pregnant or lactating.
• Females of child-bearing potential not using adequate contraceptive or a life-style excluding pregnancy.
• Sexually active man with a partner of child-bearing potential not using adequate contraceptive measures.
• Use of any other prior and concomitant therapy which may interfere with the objective of the study, within 30 days prior to the Screening period.
• Participation in a clinical trial within 30 days prior to the Screening period.

Sponsors & Collaborators

• Clinuvel Pharmaceuticals Limited: lead

Study Sites (1)

CLINUVEL investigational site

Rotterdam, Netherlands

Location

MeSH Terms

Conditions

Porphyria, Variegate

Interventions

afamelanotide

Condition Hierarchy (Ancestors)

Porphyrias, Hepatic; Liver Diseases; Digestive System Diseases; Skin Diseases, Genetic; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases; Skin and Connective Tissue Diseases; Porphyrias; Metabolic Diseases; Nutritional and Metabolic Diseases

Results Point of Contact

• Title: Director, Global Clinical Affairs
• Organization: CLINUVEL UK LTD

mail@clinuvel.com

+4401372860765

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

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: May 3, 2023
• First Posted: May 11, 2023
• Study Start: March 28, 2023
• Primary Completion: December 28, 2023
• Study Completion: December 28, 2023
• Last Updated: April 27, 2025
• Results First Posted: April 27, 2025

Record last verified: 2025-04

Locations

NL (1)