E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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E.1.1.1 | Medical condition in easily understood language |
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E.1.1.2 | Therapeutic area | Diseases [C] - Eye Diseases [C11] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10008791 |
E.1.2 | Term | Choroideremia |
E.1.2 | System Organ Class | 100000004853 |
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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 |
To evaluate the safety of bilateral, sequential sub-retinal administration
of a single dose of AAV2-REP1 in subjects with choroideremia (CHM).
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E.2.2 | Secondary objectives of the trial |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
During the Screening Period, subjects will be found eligible for study participation if they meet all of the following inclusion criteria.
1. Are willing and able to give informed consent for participation in the study to have both eyes treated
2. Are male and ≥18 years of age
3. Have documentation of a genetically-confirmed diagnosis of CHM
4. Have active disease clinically visible within the macular region of both eyes
5. Have a BCVA of ≥34 ETDRS letters (20/200 or better Snellen acuity) in both eyes, or in the untreated eye, if the other eye was previously treated with AAV2-REP1*
*If previously treated with AAV2-REP1 in an antecedent study, subjects may be eligible for participation following Sponsor approval.
6. For subjects who received treatment with AAV2-REP1 in an antecedent study, have biological samples available to complete an adequate immunology profile
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E.4 | Principal exclusion criteria |
Exclusion Criteria: At the Screening Visit, subjects will be found not eligible for study participation if they meet any of the following exclusion criteria.
1. Have a history of amblyopia or inflammatory disorder in either eye
2. Are unwilling to use barrier contraception methods, or abstain from sexual intercourse for a period of 3 months following treatment with AAV2-REP1 in either eye
3. Have had previous intraocular surgery performed within 3 months of the Screening Visit in either eye
4. Have any other significant ocular or non-ocular disease/disorder which, in the opinion of the Investigator, may either put the subjects at risk because of participation in the study, or may influence the results of the study, or the subject’s ability to participate in the study. This includes but is not limited to a potential subject:
• with a contraindication to oral corticosteroid (e.g. prednisolone/prednisone)
• with clinically significant cataract in either eye
• who, in the clinical opinion of the Patient Eligibility Review Committee or Investigator, is not an appropriate candidate for sub-retinal surgery
5. Have participated in another research study involving an investigational product in the past 12 weeks or received a gene/cell-based therapy at any time previously, except if treated within in an antecedent study with AAV2-REP1.
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E.5 End points |
E.5.1 | Primary end point(s) |
1. The evaluation of safety following bilateral administration of AAV2-
REP1.
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Assessments will be performed at timepoints described in the protocol.
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E.5.2 | Secondary end point(s) |
1. Change from baseline in visual function.
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Assessments will be performed at timepoints described in the protocol.
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
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 | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
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 | No |
E.8.2.3 | Other | No |
E.8.3 |
The trial involves single site in the Member State concerned
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 2 |
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 | No |
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 |
United States |
France |
Germany |
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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
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 2 |
E.8.9.1 | In the Member State concerned months | 7 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 2 |
E.8.9.2 | In all countries concerned by the trial months | 7 |