Phase 2
N=11
Gene Therapy for Achromatopsia (CNGA3)
Achromatopsia
Bottom Line
View on ClinicalTrials.gov: NCT03758404 ↗Enrolled (actual)
11
Serious AEs
0.0%
Results posted
Feb 2022
Primary outcome: Primary: Number of Participants Meeting the Primary Outcome Defined as Any of the Below Events Occurring During the 6 Weeks Following Administration, at Least Possibly Related to the Advanced Therapy Investigational Medicinal Products (ATIMP), Not Surgery Alone. — 0; 0; 0 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- adeno-associated virus vector AAV- CNGA3 (Biological)
- Age
- Pediatric, Adult, Older Adult · 3+ yrs
- Sex
- All
- Sponsor
- MeiraGTx UK II Ltd
- Primary completion
- Jun 2021
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants Meeting the Primary Outcome Defined as Any of the Below Events Occurring During the 6 Weeks Following Administration, at Least Possibly Related to the Advanced Therapy Investigational Medicinal Products (ATIMP), Not Surgery Alone. |
0; 0; 0 | — |
| SECONDARY Improvements in Visual Function as Assessed by Visual Acuity |
0.22; 2.44; 2.40; 1.82 | — |
| SECONDARY Improvements in Retinal Function as Assessed by Static Perimetry |
1.17; -1.83; 0.15; -0.01 | — |
| SECONDARY Quality of Life Measured by QoL Questionnaires in Children and Adolescents |
-15; 18.3; 8.0; 9.3 | — |
| SECONDARY Quality of Life Measured by QoL Questionnaires in Adults |
1.5; 1.5 | — |
Summary
A clinical trial of adeno-associated virus vector (AAV) CNGA3 retinal gene therapy for patients with achromatopsia
Eligibility Criteria
Inclusion Criteria
- Are aged years or over
- Have achromatopsia confirmed by a retinal specialist investigator
Exclusion Criteria
- Are females who are pregnant or breastfeeding
- Have participated in another research study involving an investigational medicinal therapy for ocular disease within the last 6 months
- Have any other condition that the investigator considers makes them inappropriate for entry into the trial
Data sourced from ClinicalTrials.gov (NCT03758404). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.