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

Clinuvel

Status and phase

Completed

Phase 2

Conditions

Variegate Porphyria

Treatments

Drug: Afamelanotide 16 MG

Study type

Interventional

Funder types

Industry

Identifiers

NCT05854784

CUV040

Details and patient eligibility

About

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.

Enrollment

6 patients

Sex

All

Ages

18 to 70 years old

Volunteers

No Healthy Volunteers

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.