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Study · Randomized controlled trial · n = 168 · 2015

6 results

Afamelanotide for Erythropoietic Protoporphyria.

Langendonk JG, Balwani M, Anderson KE, Bonkovsky HL, Anstey AV, Bissell DM, Bloomer J, Edwards C, Neumann NJ, Parker C, Phillips JD, Lim HW and 14 more

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Hamzavi I, Deybach JC, Kauppinen R, Rhodes LE, Frank J, Murphy GM, Karstens FPJ, Sijbrands EJG, de Rooij FWM, Lebwohl M, Naik H, Goding CR, Wilson JHP, Desnick RJ

The New England journal of medicine1 Jul 2015Cited by 203 (Europe PMC)

PMID26132941 (opens in a new tab)PMCIDPMC4780255 (opens in a new tab)DOI10.1056/nejmoa1411481 (opens in a new tab)PepBankPUB-26132941
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Summary

The text of this summary is not shown here, because the article license does not allow reuse. Read it at the source (opens in a new tab)

Text rights: Publisher copyright. Results still quote short attributed sentences.

Results from this study6

6 results from studies that gave the peptide. 6 results from people.

  1. 01HumanRandomized controlled trialEfficacy outcome

    In US patients with erythropoietic protoporphyria, median pain-free time in direct sunlight after 6 months was longer with afamelanotide than with placebo (69.4 vs. 40.8 hours, P=0.04).

    In plain wordsIn US patients with erythropoietic protoporphyria, afamelanotide gave more pain-free time in the sun than placebo.

    Peptide
    Melanotan I (afamelanotide)
    Outcome
    IncreasePain-free sun exposure time, US study (primary endpoint)
    Study

    n = 94

    Comparator
    Placebo
    Analysis
    Primary
    Outcome group
    Photosensitivity and sun tolerance · Skin and wound
    Studied dose
    16 mg · subcutaneous implant · 6 monthsAs given in this study (human).

    Measurements

    Median pain-free time
    69.4 hours
    afamelanotide, US study, 6 months
    Median pain-free time
    40.8 hours
    placebo, US study, 6 months

    Source sentences

    Summary
    In the U.S. study, the duration of pain-free time after 6 months was longer in the afamelanotide group (median, 69.4 hours, vs. 40.8 hours in the placebo group; P=0.04).
    1 context sentence
    Summary · context
    We conducted two multicenter, randomized, double-blind, placebo-controlled trials of subcutaneous implants containing 16 mg of afamelanotide.
  2. 02HumanRandomized controlled trialEfficacy outcome

    In European Union patients with erythropoietic protoporphyria, median pain-free time in direct sunlight after 9 months was longer with afamelanotide than with placebo (6.0 vs. 0.8 hours, P=0.005).

    In plain wordsIn European patients with erythropoietic protoporphyria, afamelanotide gave more pain-free time in the sun than placebo.

    Peptide
    Melanotan I (afamelanotide)
    Outcome
    IncreasePain-free sun exposure time, EU study (primary endpoint)
    Study

    n = 74

    Comparator
    Placebo
    Analysis
    Primary
    Outcome group
    Photosensitivity and sun tolerance · Skin and wound
    Studied dose
    16 mg · subcutaneous implant · 9 monthsAs given in this study (human).

    Measurements

    Median pain-free time
    6 hours
    afamelanotide, EU study, 9 months
    Median pain-free time
    0.8 hours
    placebo, EU study, 9 months

    Source sentences

    Summary
    In the European Union study, the duration of pain-free time after 9 months was also longer in the afamelanotide group than in the placebo group (median, 6.0 hours vs. 0.8 hours; P=0.005
    1 context sentence
    Summary · context
    We conducted two multicenter, randomized, double-blind, placebo-controlled trials of subcutaneous implants containing 16 mg of afamelanotide.
  3. 03HumanRandomized controlled trialEfficacy outcome

    In patients with erythropoietic protoporphyria in both trials, quality of life improved with afamelanotide therapy.

    In plain wordsIn patients with erythropoietic protoporphyria, quality of life improved with afamelanotide in both trials.

    Peptide
    Melanotan I (afamelanotide)
    Outcome
    IncreaseQuality of life (validated questionnaires), both trials
    Study

    n = 168

    Comparator
    Placebo
    Outcome group
    Quality of life · Skin and wound
    Studied dose
    16 mg · subcutaneous implantAs given in this study (human).

    Source sentences

    Summary
    In both trials, quality of life improved with afamelanotide therapy.
    1 context sentence
    Summary · context
    We conducted two multicenter, randomized, double-blind, placebo-controlled trials of subcutaneous implants containing 16 mg of afamelanotide.
  4. 04HumanRandomized controlled trialSafety outcome

    In patients with erythropoietic protoporphyria, adverse events with afamelanotide were mostly mild.

    In plain wordsIn patients with erythropoietic protoporphyria, side effects of afamelanotide were mostly mild.

    Peptide
    Melanotan I (afamelanotide)
    Outcome
    Not applicableAdverse events severity
    Study

    n = 168

    Comparator
    Placebo
    Outcome group
    Adverse events · Skin and wound
    Studied dose
    16 mg · subcutaneous implantAs given in this study (human).

    Source sentences

    Summary
    Adverse events were mostly mild
    1 context sentence
    Summary · context
    We conducted two multicenter, randomized, double-blind, placebo-controlled trials of subcutaneous implants containing 16 mg of afamelanotide.
  5. 05HumanRandomized controlled trialSafety outcome

    In patients with erythropoietic protoporphyria, serious adverse events were not thought to be related to the study drug.

    In plain wordsIn patients with erythropoietic protoporphyria, serious side effects were not thought to be caused by afamelanotide.

    Peptide
    Melanotan I (afamelanotide)
    Outcome
    Not applicableSerious adverse events relation to study drug
    Study

    n = 168

    Comparator
    Placebo
    Outcome group
    Adverse events · Skin and wound
    Studied dose
    16 mg · subcutaneous implantAs given in this study (human).

    Source sentences

    Summary
    serious adverse events were not thought to be related to the study drug.
    1 context sentence
    Summary · context
    We conducted two multicenter, randomized, double-blind, placebo-controlled trials of subcutaneous implants containing 16 mg of afamelanotide.
  6. 06HumanRandomized controlled trialEfficacy outcome

    In European Union patients with erythropoietic protoporphyria, the number of phototoxic reactions was lower with afamelanotide than with placebo (77 vs. 146, P=0.04).

    In plain wordsIn European patients with erythropoietic protoporphyria, afamelanotide led to fewer sun-related skin reactions than placebo.

    Peptide
    Melanotan I (afamelanotide)
    Outcome
    DecreaseNumber of phototoxic reactions, EU study
    Study

    n = 74

    Comparator
    Placebo
    Outcome group
    Photosensitivity and sun tolerance · Skin and wound
    Studied dose
    16 mg · subcutaneous implant · 9 monthsAs given in this study (human).

    Measurements

    Phototoxic reactions
    77 reactions
    afamelanotide, EU study
    Phototoxic reactions
    146 reactions
    placebo, EU study

    Source sentences

    Summary
    the number of phototoxic reactions was lower in the the afamelanotide group (77 vs. 146, P=0.04).
    1 context sentence
    Summary · context
    We conducted two multicenter, randomized, double-blind, placebo-controlled trials of subcutaneous implants containing 16 mg of afamelanotide.

AI wrote and checked these results. AI can make mistakes, so check important results against the source.

Registered trials2

Trials this study reports on, or that list this study as a reference. Registry data from ClinicalTrials.gov.

2 trials · 2 completed · 2 with posted results · source ClinicalTrials.gov (opens in a new tab)

CompletedPhase 3Results postedNCT01605136

Phase III Confirmatory Study in Erythropoietic Protoporphyria

Clinuvel Pharmaceuticals Limitedn = 93May 2012 to Jul 2013

Erythropoietic Protoporphyria

Trial pageClinicalTrials.govUpdated 19 Sept 2019
CompletedPhase 3Results postedNCT00979745

Phase III Confirmatory Study in Erythropoietic Protoporphyria (EPP)

Clinuvel Pharmaceuticals Limitedn = 74Sept 2009 to May 2011

Erythropoietic Protoporphyria

Trial pageClinicalTrials.govUpdated 12 Oct 2021

Version history

PepBank keeps every version of the data on this study. Status changes from Retraction Watch, Europe PMC and Crossref are listed with their source.

  1. Version 2Current

    13 Oct 2026, 21:44 UTC

    Study results updated

  2. Version 1

    13 Oct 2026, 16:31 UTC

    First added · source update from Europe PMC

Subjects

Terms as the source lists them.

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