Back to resultsA Safety Extension Study in Patients With Erythropoietic Protoporphyria (EPP)
Primary Purpose
Erythropoietic Protoporphyria
Status
Completed
Phase
Phase 3
Locations
Study Type
Interventional
Intervention
Afamelanotide
Sponsored by
About this trial
This is an interventional treatment trial for Erythropoietic Protoporphyria
Eligibility Criteria
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.
Sites / Locations
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Afamelanotide
Arm Description
Outcomes
Primary Outcome Measures
Change in Quality of Life Measured Using the EPP-quality of Life Questionnaire (EPP-QoL).
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.
Change in Quality of Life Measured Using the Dermatology Life Quality Index Questionnaire (DLQI)
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.
Secondary Outcome Measures
Full Information
NCT ID
NCT04578496
First Posted
October 5, 2020
Last Updated
November 2, 2020
Sponsor
Clinuvel Pharmaceuticals Limited
1. Study Identification
Unique Protocol Identification Number
NCT04578496
Brief Title
A Safety Extension Study in Patients With Erythropoietic Protoporphyria (EPP)
Official Title
A Phase III, Safety Extension Study in Patients With Erythropoietic Protoporphyria (EPP)
Study Type
Interventional
2. Study Status
Record Verification Date
November 2020
Overall Recruitment Status
Completed
Study Start Date
July 11, 2011 (Actual)
Primary Completion Date
February 27, 2014 (Actual)
Study Completion Date
February 27, 2014 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Clinuvel Pharmaceuticals Limited
4. Oversight
5. Study Description
Brief Summary
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Erythropoietic Protoporphyria
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
16 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Afamelanotide
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
Afamelanotide
Primary Outcome Measure Information:
Title
Change in Quality of Life Measured Using the EPP-quality of Life Questionnaire (EPP-QoL).
Description
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.
Time Frame
Baseline, Day 60, Day 120, Day 180 or early termination (ET) (if applicable)
Title
Change in Quality of Life Measured Using the Dermatology Life Quality Index Questionnaire (DLQI)
Description
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.
Time Frame
Baseline, Day 60, Day 120, Day 180 or early termination (ET) (if applicable)
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
75 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Pilar Bilbao
Organizational Affiliation
Clinuvel Pharmaceuticals Limited
Official's Role
Study Director
12. IPD Sharing Statement
Learn more about this trial
A Safety Extension Study in Patients With Erythropoietic Protoporphyria (EPP)
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