Clinical trials: Achromatopsia
Achromatopsia
Safety and Efficacy of rAAV.hCNGA3 Gene Therapy in Patients With CNGA3-linked Achromatopsia
The purpose of this study is to proof the safety and efficacy of a single bilateral subretinal injection of rAAV.hCNGA3 in adult and minor patients with CNGA3-linked achromatopsia.
- Trial name
- Colourbridge (NCT02610582)
- Testing
- rAAV.hCNGA3
- Where
- Germany
- Volunteers
- 13 people
- Sponsor
- STZ eyetrial
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
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
Eye Diseases Hereditary, Retinal Disease
Inherited Retinal Degenerative Disease Registry
The My Retina Tracker® Registry is sponsored by the Foundation Fighting Blindness and is for people affected by one of the rare inherited retinal degenerative diseases studied by the Foundation.
- Conditions
- Eye Diseases Hereditary, Retinal Disease, Achromatopsia, Bardet-Biedl Syndrome
- Trial name
- MRTR (NCT02435940)
- Where
- United States
- Volunteers
- 20,000 people, healthy volunteers welcome
- Sponsor
- Foundation Fighting Blindness
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
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
- Augelux Therapeutics
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
Inherited Ophthalmic Diseases, Hypopigmentation Disorder
National Ophthalmic Genotyping and Phenotyping Network (eyeGENE (Registered Trademark)), Stage 3 - Expansion of DNA and Data Repositories for Rare Inherited Ophthalmic Diseases
Background: The eyeGENE (Registered Trademark) program is a research resource for inherited eye conditions which includes genotypic and phenotypic data, imaging, and a corresponding biobank of DNA samples from people with a…
- Conditions
- Inherited Ophthalmic Diseases, Hypopigmentation Disorder, Corneal Dystrophy, Blue-cone Monochromacy
- Trial name
- NCT06491615
- Where
- 2 sites in United States
- Volunteers
- 1,000 people
- Sponsor
- National Eye Institute (NEI)
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
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