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Completed Phase 1Phase 2 Interventional Results available

Gene Therapy for Achromatopsia (CNGB3)

ClinicalTrials.gov ID: NCT03001310

Public ClinicalTrials.gov record NCT03001310. 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.

ClinicalTrials.gov public records Last synced Jul 21, 2026, 2:22 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Official title

An Open Label, Multi-centre, Phase I/II Dose Escalation Trial of a Recombinant Adeno-associated Virus Vector (AAV2/8-hCARp.hCNGB3) for Gene Therapy of Adults and Children With Achromatopsia Owing to Defects in CNGB3

Study identification

NCT ID
NCT03001310
Recruitment status
Completed
Study type
Interventional
Phase
Phase 1, Phase 2
Lead sponsor
MeiraGTx UK II Ltd
Industry
Enrollment
23 participants

Conditions and interventions

Conditions

Interventions

  • AAV - CNGB3 Biological

Biological

Eligibility (public fields only)

Age range
3 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
Jan 15, 2017
Primary completion
Oct 24, 2019
Completion
Oct 24, 2019
Last update posted
Mar 7, 2023

2017 – 2019

United States locations

U.S. sites
1
U.S. states
1
U.S. cities
1
Facility City State ZIP Site status
University of Michigan Kellog Eye Centre Ann Arbor Michigan MI 48105

Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.

Non-U.S. locations

This page focuses on the U.S. directory. The official record also lists 1 non-U.S. site.

About this trial record page

What this page shows
Public field values for ClinicalTrials.gov record NCT03001310, 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 Mar 7, 2023 · Synced Jul 21, 2026

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Open the official record

The complete protocol, eligibility criteria, and contact information for NCT03001310 live on ClinicalTrials.gov.

View official ClinicalTrials.gov record →