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Phase II Confirmatory Study in Erythropoietic Protoporphyria (EPP)

2014-08-27 03:14:51 | BioPortfolio

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 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.

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. 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

Allocation: Randomized, Control: Placebo Control, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Conditions

Erythropoietic Protoporphyria

Intervention

Afamelanotide, Placebo

Location

University of Alabama
Birmingham
Alabama
United States
35294

Status

Recruiting

Source

Clinuvel Pharmaceuticals Limited

Results (where available)

View Results

Links

Published on BioPortfolio: 2014-08-27T03:14:51-0400

Clinical Trials [21 Associated Clinical Trials listed on BioPortfolio]

Phase III Confirmatory Study in Erythropoietic Protoporphyria (EPP)

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 hormon...

Phase III Study of L-Cysteine in Patients With Erythropoietic Protoporphyria

OBJECTIVES: I. Determine the long-term efficacy and safety of L-cysteine in the prevention photosensitivity in patients with erythropoietic protoporphyria.

Erythropoietic Protoporphyrias: Studies of the Natural History, Genotype-Phenotype Correlations, and Psychosocial Impact

The initial objective of this protocol is to assemble a well-documented group of patients with confirmed diagnoses of the erythropoietic protoporphyrias, including autosomal recessive Eryt...

Study of Cysteine Hydrochloride for Erythropoietic Protoporphyria

OBJECTIVES: I. Determine the efficacy of cysteine hydrochloride in preventing or decreasing photosensitivity in patients with erythropoietic protoporphyria.

Does Exercise and Heat Increase the Lightsensibility in Patients With Erythropoietic Protoporphyria

The purpose of the study is to evaluate the effect of exercise and heat on the light sensibility of patients with erythropoietic protoporphyria

PubMed Articles [911 Associated PubMed Articles listed on BioPortfolio]

Liver Metabolomics in a Mouse Model of Erythropoietic Protoporphyria.

Erythropoietic protoporphyria (EPP) is a genetic disease that results from the defective mutation in the gene encoding ferrochelatase (FECH), the enzyme that converts protoporphyrin IX (PPIX) to heme....

Diffuse 18F-FDG Avidity in Liver Associated With X-Linked Protoporphyria on PET/CT.

An F-FDG PET/CT was performed on a 43-year-old woman with photosensitive skin rash, abnormal liver function, and pancytopenia, which demonstrated prominent hepatomegaly, splenomegaly, and diffuse live...

Intravenous Iron Does Not Augment the Haemoglobin Mass Response to Simulated Hypoxia.

Iron is integral for erythropoietic adaptation to hypoxia, yet the importance of supplementary iron compared to existing stores is poorly understood. The aim of the present study was to compare the ma...

Placebo analgesia persists during sleep: An experimental study.

Although placebo analgesia is a well-recognized phenomenon with important clinical implications, the possibility that placebo effects occur during sleep has received little attention. This experimenta...

Prescribing Placebos: An Experimental Examination of the Role of Dose, Expectancies, and Adherence in Open-Label Placebo Effects.

Recent evidence indicates that placebo effects can occur even when patients know that they are taking a placebo, termed the open-label placebo effect.

Medical and Biotech [MESH] Definitions

A carotenoid that is a precursor of VITAMIN A. It is administered to reduce the severity of photosensitivity reactions in patients with erythropoietic protoporphyria (PORPHYRIA, ERYTHROPOIETIC). (From Reynolds JEF(Ed): Martindale: The Extra Pharmacopoeia (electronic version). Micromedex, Inc, Engewood, CO, 1995.)

An autosomal dominant porphyria that is due to a deficiency of FERROCHELATASE (heme synthetase) in both the LIVER and the BONE MARROW, the last enzyme in the 8-enzyme biosynthetic pathway of HEME. Clinical features include mainly neurological symptoms, rarely cutaneous lesions, and elevated levels of protoporphyrin and COPROPORPHYRINS in the feces.

A mitochondrial enzyme found in a wide variety of cells and tissues. It is the final enzyme in the 8-enzyme biosynthetic pathway of HEME. Ferrochelatase catalyzes ferrous insertion into protoporphyrin IX to form protoheme or heme. Deficiency in this enzyme results in ERYTHROPOIETIC PROTOPORPHYRIA.

An autosomal recessive porphyria that is due to a deficiency of UROPORPHYRINOGEN III SYNTHASE in the BONE MARROW; also known as congenital erythropoietic porphyria. This disease is characterized by SPLENOMEGALY; ANEMIA; photosensitivity; cutaneous lesions; accumulation of hydroxymethylbilane; and increased excretion of UROPORPHYRINS and COPROPORPHYRINS.

Misunderstanding among individuals, frequently research subjects, of scientific methods such as randomization and placebo controls.

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