NCT03001310

Brief Summary

A clinical trial of AAV - CNGB3 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
23

participants targeted

Target at P25-P50 for phase_1

Timeline
Completed

Started Jan 2017

Typical duration for phase_1

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 22, 2016

Completed
1 month until next milestone

First Posted

Study publicly available on registry

December 23, 2016

Completed
24 days until next milestone

Study Start

First participant enrolled

January 16, 2017

Completed
2.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 25, 2019

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

October 25, 2019

Completed
3.4 years until next milestone

Results Posted

Study results publicly available

March 8, 2023

Completed
Last Updated

March 8, 2023

Status Verified

February 1, 2023

Enrollment Period

2.8 years

First QC Date

November 22, 2016

Results QC Date

January 3, 2023

Last Update Submit

February 9, 2023

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)

Biological-Low dose AAV - CNGB3

EXPERIMENTAL

Subretinal administration of a single low dose of AAV - CNGB3

Biological: AAV - CNGB3

Biological-Medium dose AAV - CNGB3

EXPERIMENTAL

Subretinal administration of a single intermediate dose of AAV - CNGB3

Biological: AAV - CNGB3

Biological-High dose AAV - CNGB3

EXPERIMENTAL

Subretinal administration of a single high dose of AAV - CNGB3

Biological: AAV - CNGB3

Interventions

AAV - CNGB3BIOLOGICAL

Comparison of different dosages of AAV-CNGB3

Biological-High dose AAV - CNGB3Biological-Low dose AAV - CNGB3Biological-Medium dose AAV - CNGB3

Eligibility Criteria

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

You may qualify if:

  • Are aged 3 years or older
  • Have achromatopsia confirmed by a retinal specialist (CI or PI)

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 CI/PI considers makes them inappropriate for entry into the trial

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

University of Michigan Kellog Eye Centre

Ann Arbor, Michigan, MI 48105, United States

Location

Moorfields Eye Hospital NHS Foundation Trust

London, United Kingdom

Location

Related Publications (1)

  • Michaelides M, Hirji N, Wong SC, Besirli CG, Zaman S, Kumaran N, Georgiadis A, Smith AJ, Ripamonti C, Gottlob I, Robson AG, Thiadens A, Henderson RH, Fleck P, Anglade E, Dong X, Capuano G, Lu W, Berry P, Kane T, Naylor S, Georgiou M, Kalitzeos A, Ali RR, Forbes A, Bainbridge J. First-in-Human Gene Therapy Trial of AAV8-hCARp.hCNGB3 in Adults and Children With CNGB3-associated Achromatopsia. Am J Ophthalmol. 2023 Sep;253:243-251. doi: 10.1016/j.ajo.2023.05.009. Epub 2023 May 11.

    PMID: 37172884DERIVED

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
CNGB3@meiragtx.com
0044 (0)20 3866 4320

Study Officials

  • James Bainbridge, Prof

    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
SEQUENTIAL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 22, 2016

First Posted

December 23, 2016

Study Start

January 16, 2017

Primary Completion

October 25, 2019

Study Completion

October 25, 2019

Last Updated

March 8, 2023

Results First Posted

March 8, 2023

Record last verified: 2023-02

Data Sharing

IPD Sharing
Will not share

Locations

US(1)GB(1)