Phase II Confirmatory Study in Erythropoietic Protoporphyria (EPP)

Status Completed

Clinical Trial Summary

This is a randomized placebo-controlled study to be conducted in two parallel study arms for a six month period (three doses). Approximately 10 eligible patients per center will be enrolled and will receive afamelanotide (16 mg implants) or placebo according to the following dosing regimen:

- Group A will be administered afamelanotide implants on Days 0, 60 and 120

- Group B will be administered placebo implants on Days 0, 60 and 120

To determine eligibility for study inclusion, patients will undergo a screening evaluation 7 to 14 days prior to the administration of the first dose. The number and severity of phototoxic reactions will be determined Days 60, 120, and 180. Quality of life will be measured using the EPP specific questionnaire (EPP-QoL) every 60 days and the DLQI questionnaire every 7 days, beginning at Day 0 until Day 180. Participants will visit the clinic on Days 60, 120 and 180 for assessments of adverse events.

Clinical Trial Description

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 between 10:00 and 20:00 hours. 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).

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. ;

Study Design

Related Conditions & MeSH terms

Erythropoietic Protoporphyria

Clinical Trial Details

NCT number	NCT01097044
Study type	Interventional
Source	Clinuvel Pharmaceuticals Limited
Contact
Status	Completed
Phase	Phase 2
Start date	April 2010
Completion date	April 2011

View Details

See also
Status	Clinical Trial	Phase
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Completed	`NCT00004831` - Study of Cysteine Hydrochloride for Erythropoietic Protoporphyria	N/A
Completed	`NCT05572125` - Iron Therapy in Erythropoietic Protoporphyria
Completed	`NCT01605136` - Phase III Confirmatory Study in Erythropoietic Protoporphyria	Phase 3
Completed	`NCT01422915` - Sorbent Therapy of the Cutaneous Porphyrias	Phase 2/Phase 3
Completed	`NCT04053270` - Multicentre Phase III Erythropoietic Protoporphyria Study	Phase 3
Completed	`NCT00004940` - Phase III Study of L-Cysteine in Patients With Erythropoietic Protoporphyria	Phase 3
Recruiting	`NCT06388642` - Pharmacokinetics of Afamelanotide in EPP Patients	Phase 1/Phase 2
Recruiting	`NCT05020184` - Effect of Oral Cimetidine in the Protoporphyrias	Phase 2
Completed	`NCT01688895` - Erythropoietic Protoporphyrias: Studies of the Natural History, Genotype-Phenotype Correlations, and Psychosocial Impact
Completed	`NCT04578496` - A Safety Extension Study in Patients With Erythropoietic Protoporphyria (EPP)	Phase 3
Recruiting	`NCT00206869` - Does Exercise and Heat Increase the Lightsensibility in Patients With Erythropoietic Protoporphyria	N/A
Recruiting	`NCT05780840` - Protection Against Visible Light by Dihydroxyacetone in Erythropoietic Protoporphyria	N/A
Completed	`NCT02979249` - Oral Iron for Erythropoietic Protoporphyrias	N/A
Enrolling by invitation	`NCT05883748` - Open-Label, Long-Term Extension Study to Investigate the Safety, Tolerability, and Efficacy of DISC-1459 (Bitopertin) in Participants With EPP	Phase 2/Phase 3
Completed	`NCT03682731` - Light Exposure Patterns and Symptoms Among Patients With Erythropoietic Protoporphyria

Clinical trials are conducted in a series of steps, called phases - each phase is designed to answer a separate research question.

Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.