inborn error of heme biosynthesis porphyria variegata variegate porphyria
Conditions
Interventions
Afamelanotide 16 mg every 28±2 days (up to six doses)
Sponsors
Clinuvel (UK) LTD
Eligibility
Age
18 Years to 64 Years
Inclusion criteria
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
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)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The change in severity of skin disease 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. | — |
Secondary
| Measure | Time frame |
|---|---|
| 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 5-point IGA; The Patient*s Global Assessment using a VAS. - The change in the number of new skin lesions formed during the preceding 28 days from baseline to Days 28, 56, 84, 112, 140 and 168 then from Day 168 to Day 196 as counted by the Investigator. - The change in Quality of Life from baseline to Day 28, 56, 84, 112, 140 and 168 then from Day 168 to Day 196, as measured by the three instruments: WPAI:GH, VP-derived QOLEB, VP-QoL. - Change in outdoors light exposure over time (Daily Diary). Trauma events will be tabulated. | — |
Countries
Netherlands
Outcome results
None listed