Phase III Confirmatory Study in Erythropoietic Protoporphyria

What Was Tested

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

Between 75 and 100 eligible patients will be enrolled.

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

One placebo subcutaneous implant every 2 months for 6 months.

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) and is available in Europe.

The purpose of this study is to look at the type and duration of sun exposure when patients are exposed to light.

This study will also look at how the drug is tolerated when taken by people with EPP.

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

Who Took Part

93 actual participants took part.

It was open to people of any sex aged from 18 years with no upper age limit.

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

Examples of who could join: Male or female subjects with characteristic symptoms of EPP phototoxicity and a biochemically-confirmed diagnosis of EPP. Aged 18 years old and above (inclusive). Able to understand and sign the written Informed Consent Form.

The full record lists 4 inclusion and 12 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 polymorphic light eruption, actinic prurigo, discoid lupus erythematosus, chronic actinic dermatitis 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.

Groups ran in parallel: each of the participants stayed in one arm throughout.

Blinding on both sides kept the participants and researchers unaware of who received Melanotan I.

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

It started in 2012-05 with completion listed for 2013-07.

It ran at 7 sites in United States (7).

Its stated primary purpose is treatment.

What Was Measured

Researchers chose this as the main measure: Duration of Direct Sunlight Exposure Between 10:00 and 18:00 Hours on Days When no Pain Was Experienced (Pain Score of 0) (time frame: Daily for 6 months).

It also tracked 6 secondary outcomes, for example Combined Sun Exposure and Phototoxic Pain, Sun Exposure, Quality of Life Score, Photoprovocation, Maximum Severity of Phototoxic Reaction Experienced by Participants and Total Number Phototoxic Reactions Experienced by Participants.

What This Means in Plain Language

Put simply, this is a phase 3, randomized trial of Melanotan I in 93 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.