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Phase III Confirmatory Study in Erythropoietic Protoporphyria (EPP)

NCT00979745 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 74

Last updated 2021-10-12

Study results available
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Summary

Afamelanotide is a man-made drug being studied for use as a preventative medication for EPP sufferers. It is a synthetically produced analogue of human alpha melanocyte stimulating hormone (alpha-MSH) and is not yet available on the market.

The purpose of this study is to look at whether afamelanotide can reduce the number and severity of EPP symptoms 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 (approximately 2 cm x 0.15 cm) to be administered under the skin. The implant may contain the study drug afamelanotide or a placebo (inactive medication).

Over 450 subjects have been treated with afamelanotide to date with no serious safety concerns identified. For this study, afamelanotide has been formulated as a controlled release depot injection (implant). This means that the afamelanotide will be released slowly into the body over a few days. Once inserted, the implant will remain in the body after afamelanotide has been released and will slowly dissolve.

This study will help to provide more information about afamelanotide. This information will be used to determine the safety and efficacy (the ability of the drug to produce an effect) of this drug in EPP sufferers.

Up to 70 people will participate in this study from study sites across Europe.

Conditions

Interventions

DRUG

Afamelanotide

One 16mg subcutaneous implant every 2 months for 9 months.

DRUG

Placebo

One 16mg subcutaneous implant every 2 months for 9 months.

Sponsors & Collaborators

  • Clinuvel Pharmaceuticals Limited

    lead INDUSTRY

Principal Investigators

  • Alex Anstey, MBBS, FRCP · St Woolos Hospital, Newport

  • Jorge Frank, MD, PhD · Academisch Ziekenhuis Maastricht

  • Raili Kauppinen, MD, PhD · University Central Hospital of Helsinki

  • Eric JG Sijbrands, MD, PhD · Erasmus Medical Center

  • Jean-Charles Deybach, MD. PhD · Centre Francais des Porphyries, Hopital Louis Mourier, Colombes, France

  • Sandra Hanneken, MD · Heinrich-Heine Universität, Düsseldorf, Germany

  • Gillian M Murphy, MD PhD · Beaumont Hospital, Dublin, Ireland

  • Lesley E Rhodes, MD PhD · Hope Hospital, University of Manchester, UK

Study Design

Allocation
RANDOMIZED
Purpose
PREVENTION
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Max Age
70 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2009-09-30
Primary Completion
2011-05-31
Completion
2011-05-31

Countries

  • Finland
  • France
  • Germany
  • Ireland
  • Netherlands
  • United Kingdom

Study Locations

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Entities

Drugs
Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00979745 on ClinicalTrials.gov