Phase II Confirmatory Study in Erythropoietic Protoporphyria (EPP)

What Was Tested

The trial (phase 2) is built around Melanotan I.

The number and severity of phototoxic reactions will be determined Days 60, 120, and 180.

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

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 not yet available on the market.

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.

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

Who Took Part

Enrollment: 77 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 photosensitivity of EPP symptoms and positive diagnosis of EPP confirmed by laboratory result of elevated total protoporphyrin IX. 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 ple, dle or solar urticaria.

How the Study Was Run

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

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

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 2010-04 with completion listed for 2011-04.

It ran at 6 sites in United States (6).

Its stated primary purpose is treatment.

What Was Measured

Researchers chose this as the main measure: Time in Direct Sunlight Between 10:00-15:00 on Pain-free Days (time frame: Daily for 6 months).

It also tracked 6 secondary outcomes, for example Maximum Severity of Phototoxic Reaction Experienced by Participants, Quality of Life Measured by Participant Completed Questionnaire, Change of Total Protoporphyrin IX Level in Participants, Number of Participants With Phototoxic Reactions With Likert Severity Scores ≥ 4 and ≥ 7, Number of Phototoxic Reactions Experienced by Participants and Total Severity of Phototoxic Reactions Experienced by Participants Over the Entire Study.

What This Means in Plain Language

Put simply, this is a phase 2, randomized trial of Melanotan I in 77 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.