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

• Register: EUCTR
• Last refreshed on: 3 April 2012
• Main ID: EUCTR2009-011018-51-GB
• Date of registration: 27/04/2009
• Prospective Registration: Yes
• Primary sponsor: Clinuvel Pharmaceuticals Limited
• Public title: A Phase III, Safety Extension Study in Patients with Erythropoietic Protoporphyria (EPP) - Phase III EPP Safety Extension Study
• Scientific title: A Phase III, Safety Extension Study in Patients with Erythropoietic Protoporphyria (EPP) - Phase III EPP Safety Extension Study
• Date of first enrolment: 15/06/2009
• Target sample size: 15
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2009-011018-51
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: yes Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no

Countries of recruitment

Finland, Ireland, Netherlands, United Kingdom

Contacts

• Name: Dennis J. Wright
• Affiliation: Clinuvel Pharmaceuticals Ltd

• Name: Dennis J. Wright
• Affiliation: Clinuvel Pharmaceuticals Ltd

Key inclusion & exclusion criteria

Inclusion criteria:
- Male or female subjects with a positive diagnosis of EPP who successfully completed the CUV029 study
- Aged 18-75 years (inclusive)
- Provide written Informed Patient Consent prior to the performance of any study-specific procedure.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range

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 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 another 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.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Erythropoietic Protoporphyria (EPP)
MedDRA version: 13.1 Level: LLT Classification code 10015289 Term: Erythropoietic protoporphyria System Organ Class: 10010331 - Congenital, familial and genetic disorders

Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17]

Intervention(s)

Product Name: Afamelanotide
Product Code: CUV1647
Pharmaceutical Form: Implant
INN or Proposed INN: afamelanotide
CAS Number: 75921-69-6
Current Sponsor code: CUV1647
Other descriptive name: Trade Name: SCENESSE
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 16-

Primary Outcome(s)

Primary end point(s): Quality of life measured with DLQI questionnaire and supplementary EPP specific questions

Secondary Objective: Determine whether afamelanotide can improve the quality of life of EPP patients

Main Objective: To further evaluate the safety and tolerability of afamelanotide by measuring treatmentemergent
adverse events (AEs)

Timepoint(s) of evaluation of this end point: Days 0, 60, 120 and 180

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: Days 0, 60, 120 and 180.

Secondary end point(s): Change in quality of life (measured with DLQI questionnaire and supplementary EPP specific questions at Days 0, 60, 120 and 180. Both questionnaires should also be completed at Premature Termination Visit if applicable.)

Secondary ID(s)

CUV029

2009-011018-51-NL

Source(s) of Monetary Support

Clinuvel