E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Erythropoietic Protoporphyria (EPP) |
|
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10015289 |
E.1.2 | Term | Erythropoietic protoporphyria |
|
E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
-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
|
|
E.2.2 | Secondary objectives of the trial |
-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)
|
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
-Male or female subjects with a positive diagnosis of EPP (confirmed by elevated free protoporphyrin in peripheral erythrocytes) |
|
E.4 | Principal 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. |
|
E.5 End points |
E.5.1 | 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.
|
|
E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | Yes |
E.6.3 | Therapy | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | Yes |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | Yes |
E.8.2.3 | Other | No |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 1 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 9 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
|
E.8.7 | Trial has a data monitoring committee | Yes |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
|
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 1 |
E.8.9.1 | In the Member State concerned months | 6 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 1 |
E.8.9.2 | In all countries concerned by the trial months | 6 |