Phase III Confirmatory Study in Erythropoietic Protoporphyria (EPP)

What Was Tested

This registered phase 3 trial tested Melanotan I.

It is a synthetically produced analogue of human alpha melanocyte stimulating hormone (alpha-MSH) and is not yet available on the market.

It had two arms (groups): Afamelanotide (experimental) and Placebo (placebo comparator).

The registry classes the interventions as drug.

More on the Plan

THEORETICAL FRAMEWORK: EPP is a genetic photosensitivity disorder where the mainstays of management are covering up from sunlight, systemic beta carotene and the use of controlled courses of UVR treatment.

The study will be performed in compliance with Good Clinical Practice (GCP) including the archiving of essential documents.

METHODOLOGY: The target population consists of male and female participants.

Up to 70 patients with diagnosed EPP (from past case history) and fulfilling the necessary inclusion/exclusion criteria will be enrolled.

Who Took Part

74 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 subjects 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 (inclusive). Written informed consent prior to the performance of any study-specific procedures.

The full record lists 3 inclusion and 13 exclusion criteria; among the exclusions: 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; any other photodermatosis such as ple, dle or solar urticaria.

How the Study Was Run

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

Because people in this Melanotan I study were split into groups at random, differences between the groups are less likely to reflect who happened to join each one.

The Melanotan I group and the comparison group ran side by side rather than switching.

Neither the participants nor the staff measuring them knew who was on Melanotan I, so expectations could not easily colour the readings.

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

It started in 2009-09 with completion listed for 2011-05.

It ran at 8 sites in Netherlands (2), United Kingdom (2), Finland (1), France (1), Germany (1) and Ireland (1).

Its stated primary purpose is prevention.

What Was Measured

The main thing measured (primary outcome): The Duration of Direct Sunlight Exposure Between 10:00 and 15:00 Hours on Days When Patients Did Not Report Phototoxicity-related Pain (Likert Pain Scale Score of 0) (time frame: From baseline to Day 270).

It also tracked 4 secondary outcomes, for example Number of Phototoxic Reactions, Quality of Life Measured by Patient Completed Questionnaire, Free Protoporphyrin IX Level and Treatment Emergent Adverse Events.

What This Means in Plain Language

Put simply, this is a phase 3, randomized trial of Melanotan I in 74 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.