NCT03758404

Brief Summary

A clinical trial of adeno-associated virus vector (AAV) CNGA3 retinal gene therapy for patients with achromatopsia

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
11

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Aug 2019

Geographic Reach
2 countries

2 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

November 27, 2018

Completed
2 days until next milestone

First Posted

Study publicly available on registry

November 29, 2018

Completed
9 months until next milestone

Study Start

First participant enrolled

August 12, 2019

Completed
1.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 10, 2021

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

June 10, 2021

Completed
8 months until next milestone

Results Posted

Study results publicly available

February 11, 2022

Completed
Last Updated

December 1, 2022

Status Verified

November 1, 2022

Enrollment Period

1.8 years

First QC Date

November 27, 2018

Results QC Date

January 6, 2022

Last Update Submit

November 3, 2022

Conditions

Achromatopsia

Outcome Measures

Primary Outcomes (1)

  • 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.

    The primary outcome is defined as any of the below occurring during the 6 weeks following administration, at least possibly related to the Advanced Therapy Investigational Medicinal Products (ATIMP), not surgery alone: * Reduction in visual acuity by 15 Early Treatment Diabetic Retinopathy Study (ETDRS) letters or more that fails to resolve to within 15 letters of baseline in a 4-week period once prophylactic treatment commences * Severe unresponsive inflammation * Infective endophthalmitis * Ocular malignancy * Grade III or above non-ocular Suspected Unexpected Serious Adverse Reaction (SUSAR)

    6 Weeks

Secondary Outcomes (4)

  • Improvements in Visual Function as Assessed by Visual Acuity

    6 Months

  • Improvements in Retinal Function as Assessed by Static Perimetry

    6 Months

  • Quality of Life Measured by QoL Questionnaires in Children and Adolescents

    6 Months

  • Quality of Life Measured by QoL Questionnaires in Adults

    6 Months

Study Arms (3)

Low dose adeno-associated virus (AAV) CNGA3

EXPERIMENTAL

Subretinal administration of a single low dose AAV CNGA3

Biological: adeno-associated virus vector AAV- CNGA3

Intermediate dose adeno-associated virus (AAV) CNGA3

EXPERIMENTAL

Subretinal administration of a single intermediate dose AAV CNGA3

Biological: adeno-associated virus vector AAV- CNGA3

High dose adeno-associated virus (AAV) CNGA3

EXPERIMENTAL

Subretinal administration of a single high dose AAV CNGA3

Biological: adeno-associated virus vector AAV- CNGA3

Interventions

adeno-associated virus vector AAV- CNGA3BIOLOGICAL

Adeno-associated virus (AAV) gene therapy for defects in CNGA3 gene

High dose adeno-associated virus (AAV) CNGA3Intermediate dose adeno-associated virus (AAV) CNGA3Low dose adeno-associated virus (AAV) CNGA3

Eligibility Criteria

Age3 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Are aged years or over
  • Have achromatopsia confirmed by a retinal specialist investigator

You may not qualify if:

  • 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 investigator considers makes them inappropriate for entry into the trial

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

Kellogg Eye Center

Ann Arbor, Michigan, 48105, United States

Location

Moorfields Eye Hospital NHS Foundation Trust

London, EC1V 2PD, United Kingdom

Location

MeSH Terms

Conditions

Color Vision Defects

Condition Hierarchy (Ancestors)

Vision DisordersSensation DisordersNeurologic ManifestationsNervous System DiseasesCone DystrophyEye Diseases, HereditaryEye DiseasesSigns and SymptomsPathological Conditions, Signs and Symptoms

Results Point of Contact

Title
Program Manager
Organization
MeiraGTx
CNGA3@meiragtx.com
020 3866 4320

Study Officials

  • James Bainbridge

    Chief Investigator

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
No
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 27, 2018

First Posted

November 29, 2018

Study Start

August 12, 2019

Primary Completion

June 10, 2021

Study Completion

June 10, 2021

Last Updated

December 1, 2022

Results First Posted

February 11, 2022

Record last verified: 2022-11

Data Sharing

IPD Sharing
Will not share

Locations

US(1)GB(1)