A 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.

Phase 2

Recruiting

Conditions: inborn error of heme biosynthesis
porphyria variegata
variegate porphyria
10027424

Registration Number: NL-OMON54663

Lead Sponsor: Clinuvel (UK) LTD

Brief Summary: Trial is onging in other countries

Detailed Description: Not available

Recruitment & Eligibility

Status: Recruiting

Sex: Not specified

Target Recruitment: 3

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.

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

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>The change in severity of skin disease from baseline to Days 28, 56, 84, 112,<br /><br>140 and 168 and then from Day 168 to Day 196, as measured by the 11-point VAS<br /><br>IGA scale.</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>The change in severity from baseline to Days 28, 56, 84, 112, 140 and 168 and<br /><br>then from Day 168 to Day 196, as measured by: The 5-point IGA; The Patient*s<br /><br>Global Assessment using a VAS. - The change in the number of new skin lesions<br /><br>formed during the preceding 28 days from baseline to Days 28, 56, 84, 112, 140<br /><br>and 168 then from Day 168 to Day 196 as counted by the Investigator. - The<br /><br>change in Quality of Life from baseline to Day 28, 56, 84, 112, 140 and 168<br /><br>then from Day 168 to Day 196, as measured by the three instruments: WPAI:GH,<br /><br>VP-derived QOLEB, VP-QoL. - Change in outdoors light exposure over time (Daily<br /><br>Diary). Trauma events will be tabulated.</p><br>