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

NCT ID: NCT04578496

Last Updated: 2020-11-19

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 16 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2011-07-11
• Study Completion Date: 2014-02-27

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

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Afamelanotide

Group Type: EXPERIMENTAL

Afamelanotide

Intervention Type: DRUG

Interventions

Afamelanotide

Intervention Type: DRUG

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.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 75 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Clinuvel Pharmaceuticals Limited

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Pilar Bilbao

Role: STUDY_DIRECTOR

Clinuvel Pharmaceuticals Limited

Other Identifiers

CUV037

Identifier Type: -

Identifier Source: org_study_id