Phase 2
N=23
Gene Therapy for Achromatopsia (CNGB3)
Achromatopsia
Bottom Line
View on ClinicalTrials.gov: NCT03001310 ↗Enrolled (actual)
23
Serious AEs
8.7%
Results posted
Mar 2023
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; 1; 0; 2 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- AAV - CNGB3 (Biological)
- Age
- Pediatric, Adult, Older Adult · 3+ yrs
- Sex
- All
- Sponsor
- MeiraGTx UK II Ltd
- Primary completion
- Oct 2019
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; 1; 0; 2 | — |
| SECONDARY Improvements in Visual Function as Assessed by Visual Acuity |
-0.44; -0.58; 1.78; 1.45 | — |
| SECONDARY Improvements in Retinal Function as Assessed by Static Perimetry |
-1.58; -0.39; 8.13; -0.77 | — |
| SECONDARY Quality of Life Measured by QoL Questionnaires in Children and Adolescents |
6.9; 2.3; -9.0 | — |
| SECONDARY Quality of Life Measured by QoL Questionnaires in Adults |
0.0; -3.8; 4.3 | — |
Summary
A clinical trial of AAV - CNGB3 retinal gene therapy for patients with achromatopsia
Eligibility Criteria
Inclusion Criteria
- Are aged 3 years or older
- Have achromatopsia confirmed by a retinal specialist (CI or PI)
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 CI/PI considers makes them inappropriate for entry into the trial
Data sourced from ClinicalTrials.gov (NCT03001310). 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.