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

Phase 2

Completed

• Conditions: Variegate Porphyria
• Interventions: Drug: Afamelanotide 16 MG

• Registration Number: NCT05854784
• Lead Sponsor: Clinuvel Pharmaceuticals Limited

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2023-03-28
• Study First Submitted: 2023-05-03
• Study First Submitted QC: 2023-05-03
• Study First Posted: 2023-05-11
• Primary Completion: 2023-12-28
• Study Completion: 2023-12-28
• Results First Submitted: 2024-12-20
• Status Verified: 2025-04
• Results First Submitted QC: 2025-04-23
• Last Update Submitted: 2025-04-23
• Results First Posted: 2025-04-27
• Last Update Posted: 2025-04-27

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 6

Inclusion Criteria

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

Exclusion Criteria

• 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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Afamelanotide	Afamelanotide 16 MG	-

Primary Outcome Measures

Name	Time	Method
The Change in Disease Severity in Patients With VP as Measured by CGIC.	From baseline to Day 168	The higher the score, the less severe the disease. Scale ranges from +3 to -3.

Secondary Outcome Measures

Name	Time	Method
The Change in the Quality of Life in Patients With VP as Measured by VP QoL.	Assessed at baseline and Day 168 to reflect retrospective changes over the previous 12 months.	"Quality of life measured by the number of responses of "much better than a year ago" to the question "How would you judge the condition of your skin of your face and hands today, in comparison to the situation last year (12 months ago)?"
The Change in Disease Severity in Patients With VP as Measured by 11-point VAS IGA.	Median change from baseline to Day 168.	A lower score indicates a reduced severity of the disease.Scale ranges from 0 to 10.
The Change in Disease Severity in Patients With VP as Measured by 5-point IGA.	Median change from baseline to Day 168	A lower score indicates a reduced severity of the disease. Scale ranges from 0 to 4.
The Change in Disease Severity in Patients With VP as Measured by PGIC.	Median change from baseline to Day 168	The higher the score, the less severe the disease. Score ranges from +3 to -3.
The Change in Disease Severity in Patients With VP as Measured by PGA Using VAS.	Median change from baseline to Day 168	A lower score indicates a reduced severity of the disease. Scale ranges from 0 to 10.
The Change in Number of New Skin Lesions Formed.	Median change from baseline to Day 168.	A lower number indicates a reduced severity of the disease.
The Change in the Quality of Life in Patients With VP as Measured by WPAI:GH.	Median of change from baseline to Day 168	Higher score indicates greater impairment and less productivity. Score is percentage impairment.
The Change in the Quality of Life in Patients With VP as Measured by VP-derived QOLEB.	From baseline to Day 168	Higher scores represent worse health-related quality of life. Scale ranges from 0 to 45.
The Change in Outdoor Light Exposure Over Time (Daily Diary)	From baseline to Day 168.	Daily diaries recording outdoor light exposure.

Trial Locations

Locations (1)

CLINUVEL investigational site; 🇳🇱 Rotterdam, Netherlands