Clinical trials: Achromatopsia
Achromatopsia
Long-Term Follow-Up Gene Therapy Study for Achromatopsia CNGB3 and CNGA3
This is a longer-term follow-up study of patients with achromatopsia associated with defects in CNGA3 who participated in a clinical trial in which they received AAV-CNGA3 retinal gene therapy, or of patients with achromatopsia…
- Trial name
- NCT03278873
- Testing
- Prior exposure to AAV-CNGA3 or AAV-CNGB3
- Where
- 2 sites in United States, United Kingdom
- Volunteers
- 34 people
- Sponsor
- MeiraGTx UK II Ltd
Achromatopsia
Gene Therapy for Achromatopsia (CNGB3)
A clinical trial of AAV - CNGB3 retinal gene therapy for patients with achromatopsia
- Trial name
- CNGB3 (NCT03001310)
- Testing
- AAV - CNGB3
- Where
- 2 sites in United States, United Kingdom
- Volunteers
- 23 people
- Sponsor
- MeiraGTx UK II Ltd
Achromatopsia
Natural History Study for Achromatopsia
In preparation for human clinical trials we intend to undertake a detailed phenotypic study to help to identify patients who may be suitable for therapeutic intervention.
- Trial name
- NCT04124185
- Testing
- Ocular assessments
- Where
- United Kingdom
- Volunteers
- 85 people
- Sponsor
- MeiraGTx UK II Ltd
Achromatopsia
Gene Therapy for Achromatopsia (CNGA3)
A clinical trial of adeno-associated virus vector (AAV) CNGA3 retinal gene therapy for patients with achromatopsia
- Trial name
- CNGA3 (NCT03758404)
- Testing
- adeno-associated virus vector AAV- CNGA3
- Where
- 2 sites in United States, United Kingdom
- Volunteers
- 11 people
- Sponsor
- MeiraGTx UK II Ltd
Retinal Dystrophies, Color Vision Defects
Natural History Study of Inherited Retinal Diseases
This prospective, observational investigation seeks to delineate the interplay between chromatic vision deficits and both functional visual outcomes and anatomical retinal biomarkers in individuals affected by Inherited Retinal…
- Conditions
- Retinal Dystrophies, Color Vision Defects, Vision Disorders, Macular Degeneration
- Trial name
- MojiLVCDT (NCT07085533)
- Where
- China
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- Zhongmou Therapeutics
Achromatopsia
Safety and Efficacy Trial of AAV Gene Therapy in Patients With CNGB3 Achromatopsia (A Clarity Clinical Trial)
This will be a non-randomized, open-label, Phase 1/2 study of the safety and efficacy of AGTC-401 administered to one eye by subretinal injection in individuals with achromatopsia caused by mutations in the CNGB3 gene.
- Trial name
- NCT02599922
- Testing
- rAAV2tYF-PR1.7-hCNGB3
- Where
- 8 sites in United States
- Volunteers
- 32 people
- Sponsor
- Beacon Therapeutics
Achromatopsia
Safety and Efficacy Trial of AAV Gene Therapy in Patients With CNGA3 Achromatopsia (A Clarity Clinical Trial)
This will be a non-randomized, open-label, Phase 1/2 study of the safety and efficacy of AGTC-402, administered to one eye by subretinal injection in individuals with achromatopsia caused by mutations in the CNGA3 gene.
- Trial name
- NCT02935517
- Testing
- AGTC-402
- Where
- 6 sites in United States, Israel
- Volunteers
- 24 people
- Sponsor
- Beacon Therapeutics
Achromatopsia
Clinical and Genetic Characterization of Individuals With Achromatopsia
The purpose of this study is to identify individuals with achromatopsia caused by mutations in the CNGB3 gene and characterize their clinical condition using several tests of visual function every 6 months for up to 1.5 years.
- Trial name
- NCT01846052
- Where
- 5 sites in United States
- Volunteers
- 56 people
- Sponsor
- Beacon Therapeutics