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

What Was Tested

In this phase 2 trial, researchers set out to test Melanotan I.

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.

It had 1 arm (groups): Afamelanotide (experimental).

The registry classes the interventions as drug.

Who Took Part

The registry lists 6 actual participants took part.

It was open to people of any sex aged from 18 years up to 70 years.

Healthy volunteers were not accepted; participants needed the condition being studied.

Examples of who could join: Male or female patients with confirmed diagnosis of VP. Patients with VP-related skin symptoms. Aged 18-70 years.

The full record lists 3 inclusion and 11 exclusion criteria; among the exclusions: allergy to afamelanotide or polymer or to local anaesthetic to be used if applicable; individual history of malignant or premalignant skin lesions; individual or family history of melanoma.

How the Study Was Run

The set-up was single-group and open-label.

Being open-label, the Melanotan I study did not mask who received what.

Everyone received the same intervention, so there was no separate comparison group inside the trial.

It started in 2023-03-28 with completion listed for 2023-12-28.

It ran at 1 site in Netherlands.

Its stated primary purpose is treatment.

What Was Measured

Primary outcome: The Change in Disease Severity in Patients With VP as Measured by CGIC (time frame: From baseline to Day 168).

It also tracked 9 secondary outcomes, for example The Change in Disease Severity in Patients With VP as Measured by 11-point VAS IGA, The Change in Disease Severity in Patients With VP as Measured by 5-point IGA, The Change in Disease Severity in Patients With VP as Measured by PGIC, The Change in Disease Severity in Patients With VP as Measured by PGA Using VAS, The Change in Number of New Skin Lesions Formed and The Change in the Quality of Life in Patients With VP as Measured by WPAI:GH.

What This Means in Plain Language

Put simply, this is a phase 2 trial of Melanotan I in 6 people.

Its size is small, so on its own it can only give an early look.

The trial is marked completed; results are posted on the registry, and this page does not restate them.

More context: the Melanotan I compound page, all human trials.