Study to Evaluate the Safety and Efficacy of Afamelanotide in Patients with Variegate Porphyria (VP)-related skin disease

Phase 1

• Conditions: Variegate Porphyria (VP)-related skin disease; Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]

• Registration Number: EUCTR2018-004164-60-NL
• Lead Sponsor: CLINUVEL (UK) LTD

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2022-02-21
• Last Update Posted: 30 May 2022

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 6

Inclusion Criteria

Male or female patients with confirmed diagnosis of VP.
Patients with VP-related skin symptoms assessed with a score equal or above 7 on the 11-point VAS IGA.
Aged 18-70 years.

Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 6
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 1

Exclusion Criteria

Known allergy to afamelanotide or the polymer or to lignocaine/lidocaine or other local anaesthetic.
Had two or more acute attacks of hepatic porphyria lasting more than two days, within 12 months prior to the Screening period.
History of certain malignant and premalignant skin lesions.
Individual or family history of melanoma.
Severe hepatic disease.
Renal impairment (eGFR (MDRD) < 30 ml/min*1.73m2).
Female who is pregnant or lactating.
Females of child-bearing potential not using adequate contraceptive measures.
Sexually active man with a partner of child-bearing potential who is not using adequate contraceptive measures.
Not suitable for trial participation in the opinion of the Investigator.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: determine whether afamelanotide implants can reduce the severity of the skin disease in patients with VP;Secondary Objective: Evaluate the safety and tolerability of afamelanotide in patients with VP. Evaluate the impact of afamelanotide on the quality of life of patients with VP.;Primary end point(s): E.5.1Primary End Point (repeat as necessary) The change in severity from baseline to Days 28, 56, 84, 112, 140 and 168 and then from Day 168 to Day 196, as measured by the 11-point VAS IGA scale.;Timepoint(s) of evaluation of this end point: E.5.1Primary End Point (repeat as necessary) The change in severity from baseline to Days 28, 56, 84, 112, 140 and 168 and then from Day 168 to Day 196, as measured by the 11-point VAS IGA scale.