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

Variegate Porphyria Clinical Trial

Official title:

Proof of Concept, Phase IIa, Open Label Study to Evaluate the Safety and Efficacy of Afamelanotide in Patients With Variegate Porphyria (VP)-Related Skin Disease.

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT05854784
• Other study ID #: CUV040
• Status: Completed
• Phase: Phase 2
• Start date: March 28, 2023
• Est. completion date: December 28, 2023

Study information

• Verified date: January 2024
• Source: Clinuvel Pharmaceuticals Limited
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 6
• Est. completion date: December 28, 2023
• Est. primary completion date: December 28, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 70 Years

Eligibility

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.

Related Conditions & MeSH terms

• Porphyria, Erythropoietic
• Porphyrias
• Variegate Porphyria

Intervention

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

Locations

Country	Name	City	State
Netherlands	CLINUVEL investigational site	Rotterdam

Sponsors (1)

Lead Sponsor	Collaborator
Clinuvel (UK) Ltd.

Country where clinical trial is conducted

Netherlands, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	The change in disease severity in patients with VP as measured by a scoring system (A).	The higher the score, the less severe the disease	From baseline to Day 168
Secondary	The change in disease severity in patients with VP as measured by a scoring system (B).	The higher the score, the more severe the disease	From baseline to Day 168
Secondary	The change in disease severity in patients with VP as measured by a scoring system (C)	The higher the score, the more severe the disease	From baseline to Day 168
Secondary	The change in disease severity in patients with VP as measured by a scoring system (D)	The higher the score, the less severe the disease	From baseline to Day 168
Secondary	The change in disease severity in patients with VP as measured by a scoring system (E)	The higher the score, the more severe the disease	From baseline to Day 168
Secondary	The change in number of new skin lesions formed	The higher the number of new lesions, the more severe the disease	From baseline to Day 168
Secondary	The change in the Quality of Life in patients with VP as measured by a validated global quality of life tool (A)	Score calculated in impairment percentages, with higher numbers indicating greater impaired quality of life	From baseline to Day 168
Secondary	The change in the Quality of Life in patients with VP as measured by a disease specific tool (B)	Higher scores represent worse health-related quality of life	From baseline to Day 168
Secondary	The change in the Quality of Life in patients with VP as measured by a disease specific questionnaire (C)	Higher scores indicate a better quality of life	From baseline to Day 168
Secondary	The change in outdoor light exposure over time (Daily Diary)	Daily diaries recording outdoor light exposure and trauma events	From baseline to Day 168