Safety and Efficacy Trial of AAV Gene Therapy in Patients With CNGB3 Achromatopsia (A Clarity Clinical Trial)
Public ClinicalTrials.gov record NCT02599922. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
A Multiple-Site, Phase 1/2, Safety and Efficacy Trial of a Recombinant Adeno-associated Virus Vector Expressing CNGB3 in Patients With Congenital Achromatopsia Caused by Mutations in the CNGB3 Gene
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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. The primary study endpoint will be safety and the secondary study endpoint will be efficacy.
Study identification
- NCT ID
- NCT02599922
- Recruitment status
- Not listed
- Study type
- Interventional
- Phase
- Phase 1, Phase 2
- Enrollment
- 32 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 4 Years and older
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Apr 11, 2016 (Actual)
- Primary completion
- Jul 2022 (Estimated)
- Completion
- Jul 2026 (Estimated)
- Last update posted
- Jul 22, 2022
2016 – 2026
United States locations
- U.S. sites
- 8
- U.S. states
- 6
- U.S. cities
- 7
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| VitreoRetinal Associates | Gainesville | Florida | 32607 | — |
| Bascom Palmer Eye Institute | Miami | Florida | 33136 | — |
| Pangere Center for Inherited Retinal Diseases, The Chicago Lighthouse for People Who Are Blind or Visually Imp | Chicago | Illinois | 60608 | — |
| Massachusetts Eye and Ear Infirmary | Boston | Massachusetts | 02114 | — |
| Boston Children's Hospital | Boston | Massachusetts | 02115 | — |
| Duke Eye Center, Duke University Medical Center | Durham | North Carolina | 27710 | — |
| Cincinnati Eye Institute | Cincinnati | Ohio | 45242 | — |
| Casey Eye Institute, Oregon Health and Sciences University | Portland | Oregon | 97239 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT02599922, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Jul 22, 2022 · Synced Oct 21, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT02599922 live on ClinicalTrials.gov.