Multicentre Phase III Erythropoietic Protoporphyria Study

What Was Tested

In this phase 3 trial, researchers set out to test Melanotan I.

The study was conducted with two parallel study arms with crossover between treatments every 60 days.

It had two arms (groups): Group A (experimental) and Group B (placebo comparator).

Group A was administered active implants on Days 0, 120, 240 and placebo implants on Days 60, 180, 300.

Group B was administered placebo implants on Days 0, 120, 240 and active implants on Days 60, 180, 300.

The registry classes the interventions as drug.

More on the Plan

It is a synthetically produced analogue of human alpha melanocyte stimulating hormone (alpha-MSH).

The study will involve the use of an implant, which comes in the form of a small rod to be administered under the skin.

The implant may contain the study drug afamelanotide or a placebo (inactive medication).

Who Took Part

Enrollment: 100 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 a diagnosis of EPP (confirmed by elevated free protoporphyrin in peripheral erythrocytes) of sufficient severity that they have requested treatment to alleviate their symptoms. Aged 18-70 years. Written informed consent prior to the performance of any study-specific procedure.

The full record lists 3 inclusion and 16 exclusion criteria; among the exclusions: any allergy to afamelanotide or the polymer contained in the implant or to lignocaine or other local anaesthetic used during the administration of study medication; personal history of melanoma or dysplastic nevus syndrome; current bowen's disease, basal cell carcinoma, squamous cell carcinoma, or other malignant or premalignant skin lesions.

How the Study Was Run

It was run as a randomized, crossover and quadruple-blind study.

The participants were allocated by chance, so the Melanotan I group and the comparison group should start out alike.

In the crossover set-up each of the participants took both Melanotan I and the comparison in turn, acting as their own control.

As a double-blind Melanotan I study, the assignments were hidden from both participants and investigators until the end.

Those kept unaware of assignments: participant, care provider, investigator and outcomes assessor.

It started in 2007-05 with completion listed for 2009-12-09.

Its stated primary purpose is treatment.

What Was Measured

It also tracked 4 secondary outcomes, for example Skin Melanin Density (Study Completers Population), Change in Quality of Life Using SF36 Questionnaire (Physical Component Score) for Study Completers Population and Change in Quality of Life Using SF36 Questionnaire (Mental Component Score) for Study Completers Population.

What This Means in Plain Language

Put simply, this is a phase 3, randomized trial of Melanotan I in 100 people.

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.