Phase 3
N=74
Phase III Confirmatory Study in Erythropoietic Protoporphyria (EPP)
Erythropoietic Protoporphyria
Bottom Line
View on ClinicalTrials.gov: NCT00979745 ↗Enrolled (actual)
74
Serious AEs
0.0%
Results posted
Oct 2021
Primary outcome: 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) — 6.0; 0.75 Hours
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Afamelanotide (Drug); Placebo (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Clinuvel Pharmaceuticals Limited
- Primary completion
- May 2011
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| 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) |
6.0; 0.75 | — |
| SECONDARY Number of Phototoxic Reactions |
— | — |
| SECONDARY Quality of Life Measured by Patient Completed Questionnaire |
— | — |
| SECONDARY Free Protoporphyrin IX Level |
— | — |
| SECONDARY Treatment Emergent Adverse Events |
— | — |
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.
Eligibility Criteria
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.
Data sourced from ClinicalTrials.gov (NCT00979745). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.