Trial · Interventional

CompletedPhase 3Results postedRegistry data

Phase III Confirmatory Study in Erythropoietic Protoporphyria (EPP)

NCTNCT00979745Other idCUV029

Open at ClinicalTrials.govAsk about this trial

Overview

Status: Completed
Phase: Phase 3
Enrollment: 74 participants (actual)
Sponsor: Clinuvel Pharmaceuticals Limited · Industry
Design: Randomized · Quadruple masking
Countries: 6 countries
Finland, France, Germany, Ireland, Netherlands, United Kingdom
Start: Sept 2009
Primary completion: May 2011
Completion: May 2011
First posted: 18 Sept 2009 (estimated)
Last update posted: 12 Oct 2021
Conditions: Erythropoietic Protoporphyria

Timeline

1 trial · 1 completed · 1 with posted results

Completed
Ongoing
Not yet recruiting
Stopped
Estimated dates (est.)
Results posted

2009201020112012

Ph 3
Phase III Confirmatory Study in Erythropoietic Protoporphyria (EPP)
NCT00979745
Sept 2009 to May 2011

Registry summary

Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.

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.

Interventions

AfamelanotideDrug
PlaceboDrug

Arms2

Experimental
Afamelanotide
Placebo comparator
Placebo

Primary outcomes1

Outcomes the sponsor listed as primary.

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

Secondary outcomes(4)

Number of Phototoxic Reactions
Time frame · 9 months
Quality of Life Measured by Patient Completed Questionnaire
Time frame · 9 months
Free Protoporphyrin IX Level
Time frame · 9 months
Treatment Emergent Adverse Events
Time frame · 9 months

Posted results

Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.

Primary outcome results

Primary outcomeMedian· From baseline to Day 270

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)

Group	Value (Hours)
Experimental: Afamelanotide	6.0
Placebo	0.75

Secondary and other outcome results(4)

Secondary outcome· 9 months

Number of Phototoxic Reactions

Group	Value

Secondary outcome· 9 months

Quality of Life Measured by Patient Completed Questionnaire

Group	Value

Secondary outcome· 9 months

Free Protoporphyrin IX Level

Group	Value

Secondary outcome· 9 months

Treatment Emergent Adverse Events

Group	Value

Adverse events, as posted

Adverse events as posted: participants affected out of participants at risk, by group
Group	Deaths	Serious events	Other events
Experimental: AfamelanotideAfamelanotide implants administered on Days 0, 60, 120, 180 and 240.	–	0 / 38	16 / 38
PlaceboPlacebo implants administered on Days 0, 60, 120, 180 and 240.	–	0 / 36	12 / 36

Each cell reads participants affected / participants at risk, as the registry posted them.

Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works

Studies1

Studies that cite this trial.

Studies with results

1 study on this page

Indexing hint: Randomized controlled trial · 2015

6 results

Afamelanotide for Erythropoietic Protoporphyria.

Langendonk et al. 2015The New England journal of medicinecited by 203

PMID 26132941PMC4780255DOI 10.1056/nejmoa1411481PUB-26132941

Rights unknown6 results

Source link See results (6)

Eligibility

Who could join, as posted.

Sex: All
Age: from 18 Years up to 70 Years
Healthy volunteers: Not accepted

Inclusion and exclusion criteria, as posted

Inclusion Criteria: * 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. Exclusion Criteria: * Any allergy to afamelanotide or the polymer contained in the implant or to lignocaine or other local anaesthetic to be used during the administration of study medication. * EPP patients with significant hepatic involvement. * 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. * Any evidence of clinically significant organ dysfunction or any clinically significant deviation from normal in the clinical or laboratory determinations. * Acute history of drug or alcohol abuse (in the last 12 months). * Patient assessed as not suitable for the study in the opinion of the Investigator (e.g. noncompliance history, allergic to local anaesthetics, faints when given injections or giving blood). * Female who is pregnant (confirmed by positive serum β-HCG pregnancy test prior to baseline) or lactating. * Females of child-bearing potential (pre-menopausal, not surgically sterile) not using adequate contraceptive measures (i.e. oral contraceptives, diaphragm plus spermicide, intrauterine device). * Sexually active men with partners of child bearing potential not using barrier contraception during the trial and for a period of three months thereafter. * Participation in a clinical trial of an investigational agent within 30 days prior to the screening visit. * Prior and concomitant therapy with medications which may interfere with the objectives of the study, including drugs that cause photosensitivity or skin pigmentation.

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