A Safety Extension Study in Patients With Erythropoietic Protoporphyria (EPP)

November 2, 2020 updated by: Clinuvel Pharmaceuticals Limited

A Phase III, Safety Extension Study in Patients With Erythropoietic Protoporphyria (EPP)

The main objectives of the study were to evaluate the safety and tolerability of afamelanotide by measuring treatment emergent adverse events (AEs) and to determine whether afamelanotide can improve the quality of life of EPP patients.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

16

Phase

  • Phase 3

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 75 years (ADULT, OLDER_ADULT)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • male or female subjects with a positive diagnosis of EPP who successfully completed the CUV017 or CUV029 studies;
  • aged 18-75 years (inclusive);
  • provide written informed patient consent prior to the performance of any study-specific procedure.

Exclusion Criteria:

  • any serious adverse event considered to be related to afamelanotide or the polymer contained in the implant;
  • any allergy to lignocaine or other local anaesthetic to be used during the administration of the 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 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);
  • female who is pregnant (confirmed by positive serum β-Human Chorionic Gonadotropin (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 for 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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: NA
  • Interventional Model: SINGLE_GROUP
  • Masking: NONE

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
EXPERIMENTAL: Afamelanotide
Drug: Afamelanotide

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change in Quality of Life Measured Using the EPP-quality of Life Questionnaire (EPP-QoL).
Time Frame: Baseline, Day 60, Day 120, Day 180 or early termination (ET) (if applicable)
The EPP-QoL is presented as 15 questions aiming at measuring how the condition has affected the patient over the last 2 months. Each question is answered on a categorical scale. The maximum score is calculated by summing the score of each question resulting in a maximum of 100 and a minimum of 0. The higher the score, the better the quality of life.
Baseline, Day 60, Day 120, Day 180 or early termination (ET) (if applicable)
Change in Quality of Life Measured Using the Dermatology Life Quality Index Questionnaire (DLQI)
Time Frame: Baseline, Day 60, Day 120, Day 180 or early termination (ET) (if applicable)
The DLQI questionnaire consists of 10 questions, each answered on a categorical scale. The DLQI is calculated by summing the score of each question resulting in a maximum of 30 and a minimum of 0. The higher the score, the more quality of life is impaired.
Baseline, Day 60, Day 120, Day 180 or early termination (ET) (if applicable)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Clinuvel Pharmaceuticals Limited

Investigators

  • Study Director: Pilar Bilbao, Clinuvel Pharmaceuticals Limited

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (ACTUAL)

July 11, 2011

Primary Completion (ACTUAL)

February 27, 2014

Study Completion (ACTUAL)

February 27, 2014

Study Registration Dates

First Submitted

October 5, 2020

First Submitted That Met QC Criteria

October 5, 2020

First Posted (ACTUAL)

October 8, 2020

Study Record Updates

Last Update Posted (ACTUAL)

November 19, 2020

Last Update Submitted That Met QC Criteria

November 2, 2020

Last Verified

November 1, 2020

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CUV037

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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