Phase II Study of CUV1647 in Erythropoietic Protoporphyria (EPP)
- Conditions
- Erythropoietic Protoporphyria (EPP)Human Genetics and Inherited Disorders - Other metabolic and endocrine disorders
- Registration Number
- ACTRN12606000535572
- Lead Sponsor
- Clinuvel Pharmaceuticals
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Stopped early
- Sex
- All
- Target Recruitment
- 10
Patients with EPP (confirmed by elevated free protoporphyrin in peripheral erythrocytes and/or ferrochelatase mutation)- Fitzpatrick skin types I to IV.
Any other photodermatosis such as polymorphic light eruption (PLE), discoid lupus erythematosus (DLE) or solar urticaria- females who are pregnant, lactating or of child bearing age not using adequate methods of contraception- 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).- History of disorders of the gastrointestinal, hepatic, renal, cardiovascular, respiratory, endocrine, neurological, haematological or systemic disease judged to be clinically significant by the Investigator.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Time to appearance of artificially provoked symptoms when hands are exposed to light from a solar simulator[At baseline and then every two months.];Amount of rescue medication used to treat phototoxic reactions.[]
- Secondary Outcome Measures
Name Time Method Changes is skin melanin density[From baseline to 2, 4, 6, 8, 10 and 12 months.];Quality of life as measured by SF36v2 [At baseline and again at 12 months.];Number and severity of phototoxic reactions[Recorded over the 12 month study period.]