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

NCT04578496 | Completed Phase 3 Results

• 1 other identifier: CUV037
• Study Type: interventional
• Target: 16
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Erythropoietic Protoporphyria

Outcome Measures

Primary Outcomes (2)

• 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.

  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)

  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)

Study Arms (1)

Afamelanotide

EXPERIMENTAL

Drug: Afamelanotide

Interventions

Afamelanotide DRUG

Afamelanotide

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Clinuvel Pharmaceuticals Limited: lead

MeSH Terms

Conditions

Protoporphyria, Erythropoietic

Interventions

afamelanotide

Condition Hierarchy (Ancestors)

Porphyrias, Hepatic; Liver Diseases; Digestive System Diseases; Skin Diseases, Genetic; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases; Skin and Connective Tissue Diseases; Porphyrias; Metabolic Diseases; Nutritional and Metabolic Diseases

Results Point of Contact

• Title: Clinical Operations Manager
• Organization: CLINUVEL PHARMACEUTICALS LIMITED

mail@clinuvel.com

Study Officials

• Pilar Bilbao

  Clinuvel Pharmaceuticals Limited

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 5, 2020
• First Posted: October 8, 2020
• Study Start: July 11, 2011
• Primary Completion: February 27, 2014
• Study Completion: February 27, 2014
• Last Updated: November 19, 2020
• Results First Posted: November 2, 2020

Record last verified: 2020-11