Choroideremia MedDRA version: 20.1 Level: LLT Classification code 10008791 Term: Choroideremia System Organ Class: 100000004853
Conditions
Interventions
Product Code: AAV2-REP1
Pharmaceutical Form: Suspension for injection
INN or Proposed INN: timrepigene emparvovec
CAS Number: 1905415-02-2
Current Sponsor code: AAV2-REP1
Other descriptive name: Adeno
Sponsors
NightstaRx Ltd
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: Subjects are eligible for study participation if they meet all of the following inclusion criteria. 1. Are willing and able to provide informed consent for participation in the study. 2. Are male and =18 years of age. 3. Have a genetically-confirmed diagnosis of CHM. 4. Have active disease clinically visible within the macular region in the study eye. 5. Have reduced visual acuity in the study eye 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 140 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range 20
Exclusion criteria
Exclusion criteria: Subjects are not eligible for study participation if they meet any of the following exclusion criteria. - Previous intraocular surgery performed in the study eye within 3 months of Visit 1. - Any significant 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 subjects ability to participate in the study. - 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. - Previous intraocular surgery performed in the study eye within 6 months of Visit 1
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Timepoint(s) of evaluation of this end point: BCVA assessments will be performed as per protocol.;Main Objective: The objective of the study is to evaluate the efficacy and safety of a single sub-retinal injection of AAV2-REP1 in subjects with Choroideremia.;Secondary Objective: Not applicable;Primary end point(s): Change from baseline in best corrected visual acuity (BCVA). | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Visual assessments will be performed as per protocol.;Timepoint(s) of evaluation of this end point: Safety assessments will be performed at all timepoints. | — |
Countries
Brazil, Canada, Denmark, Finland, France, Germany, Netherlands, United Kingdom, United States
Contacts
Public ContactClinical Operations Department
NightstaRx Ltd
Outcome results
None listed