Erythropoietic Protoporphyria (EPP) MedDRA version: 9.1 Level: LLT Classification code 10015289 Term: Erythropoietic protoporphyria
Conditions
Interventions
Product Name: CUV1647
Product Code: CUV1647
Pharmaceutical Form: Implant
Pharmaceutical form of the placebo: Implant
Route of administration of the placebo: Subcutaneous use
Sponsors
Clinuvel Pharmaceuticals Limited
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: -Male or female subjects with a positive diagnosis of EPP (confirmed by elevated free protoporphyrin in peripheral erythrocytes) 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
Exclusion criteria: -Allergy to CUV1647 or the polymer contained in the implant or to lignocaine or other local anaesthetic to be used during the administration of the study medication -Any other photodermatosis such as PLE, DLE or solar urticaria. -Female who is pregnant, lactating or of childbearing potential and not using adequate form(s) of contraception. -Any evidence of clinically significant organ dysfunction or any clinically significant deviation from normal in the clinical or laboratory determinations. -Current Bowen’s disease, basal cell carcinoma, squamous cell carcinoma, or other malignant or premalignant skin lesions. -Personal history of melanoma or dysplastic nevus syndrome.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: -determine whether CUV1647 can reduce the number of phototoxic reactions in patients with EPP -determine whether CUV1647 can reduce the severity of phototoxic reactions in patients with EPP ;Secondary Objective: -determine whether CUV1647 can increase the duration of sunlight tolerated by EPP patients -determine whether CUV1647 increases melanin density in the skin at several specified body sites -evaluate the safety and tolerability of CUV1647 by measuring treatment-emergent adverse events (AEs) -determine whether CUV1647 can improve the quality of life of EPP patients -in a subset of patients, determine whether CUV1647 implants can reduce the susceptibility to provocation with a standardized light source (time to appearance of provoked symptoms) ;Primary end point(s): -The mean number of phototoxic reactions that occur whilst patients are on active compared with placebo implants. That is, the mean number of phototoxic reaction in (active) Group A between Days 0-60, 120-180, 240-300 plus Group B between Days 60-120, 180-240, 300-360 compared with (placebo) Group A between Days 60-120, 180-240, 300-360 plus Group B between Days 0-60, 120-180, 240-300 -The mean severity score for phototoxic reactions that occur whilst patients are on active compared with placebo implants. That is, the mean severity score in (active) Group A between Days 0-60, 120-180, 240-300 plus Group B between Days 60-120, 180-240, 300-360 compared with (placebo) Group A between Days 60-120, 180-240, 300-360 plus Group B between Days 0-60, 120-180, 240-300 Null Hypothesis: there is no difference between the mean number and severity of phototoxic reactions that occurred in patients treated with active and placebo. | — |
Countries
France, Germany, Italy, Netherlands, Sweden, United Kingdom
Outcome results
None listed