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Gene Therapy

Gene Therapy for Color Blindness

Phase 1 & 2

Waitlist Available

Research Sponsored by MeiraGTx UK II Ltd

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Received AAV2/8-hCARp.hCNGB3 or AAV2/8-hG1.7p.coCNGA3 by intraocular administration in the prior open-label, Phase I/II, dose escalation study (EudraCT 2016-002290-35 or EudraCT 2018-003431-29)

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 5 years

Awards & highlights

Study Summary

This trial is for people who have achromatopsia and have had gene therapy in the past.

Who is the study for?

This trial is for individuals who have a type of color blindness called achromatopsia and were previously treated with gene therapies AAV - CNGB3 or AAV - CNGA3. Participants must be able to consent, follow the study's long-term procedures, and have received one of these treatments in earlier Phase I/II trials.Check my eligibility

What is being tested?

The study is observing the long-term effects of two gene therapy interventions: AAV - CNGB3 and AAV - CNGA3. These therapies were administered in previous clinical trials to treat achromatopsia, a form of color blindness.See study design

What are the potential side effects?

Since this is a follow-up study, it focuses on monitoring ongoing side effects from the initial treatment which may include eye irritation or inflammation, changes in vision, and potential immune responses.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I received a specific gene therapy in my eye as part of a previous study.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and 5 years for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Incidence of Adverse Events related to the treatment

Secondary outcome measures

Improvement in quality of life

Improvement in retinal function

Improvement in the visual function

Trial Design

3Treatment groups

Experimental Treatment

Group I: Biological-medium dose of either AAV - CNGB3 or AAV - CNGA3Experimental Treatment1 Intervention

Subretinal administration of a single medium dose of either AAV - CNGB3 or AAV - CNGA3

Group II: Biological-high dose of either AAV - CNGB3 or AAV - CNGA3Experimental Treatment1 Intervention

Subretinal administration of a single high dose of either AAV - CNGB3 or AAV - CNGA3

Group III: Biological-Low dose of either AAV - CNGB3 or AAV - CNGA3Experimental Treatment1 Intervention

Subretinal administration of a single low dose of either AAV - CNGB3 or AAV - CNGA3

0 / 1Eligibility criteria met

Find a Location

Who is running the clinical trial?

Syne Qua Non LimitedIndustry Sponsor

6 Previous Clinical Trials

442 Total Patients Enrolled

EMAS PharmaIndustry Sponsor

4 Previous Clinical Trials

156 Total Patients Enrolled

MeiraGTx UK II LtdLead Sponsor

14 Previous Clinical Trials

687 Total Patients Enrolled

Media Library

AAV - CNGA3 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT03278873 — Phase 1 & 2

Color Blindness Research Study Groups: Biological-medium dose of either AAV - CNGB3 or AAV - CNGA3, Biological-high dose of either AAV - CNGB3 or AAV - CNGA3, Biological-Low dose of either AAV - CNGB3 or AAV - CNGA3

Color Blindness Clinical Trial 2023: AAV - CNGA3 Highlights & Side Effects. Trial Name: NCT03278873 — Phase 1 & 2

AAV - CNGA3 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03278873 — Phase 1 & 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is there still availability for individuals to join this research project?

"Per the information found on clinicaltrials.gov, this specific trial is not currently seeking participants. Initially published in June 2017 and amended for the last time in January 2022, it does not appear to be actively recruiting at present. Nonetheless, there are 7 other trials that require patient enrolment immediately."

Answered by AI

Might I be qualified to partake in this investigation?

"This medical trial is recruiting 34 patients between the ages of 3 and 100, who possess achromatopsia. Additionally, these persons must meet additional criteria such as being able to provide informed consent or assent (with parental/guardian guidance where applicable), receiving AAV2/8-hCARp.hCNGB3 or AAV2/8-hG1.7p.coCNGA3 through intraocular administration in a prior open-label study, demonstrating commitment to protocol adherence and longterm followup, and being unable to fulfill other requirements of the study."

Answered by AI

Does the research allow individuals under 45 years of age to participate?

"This clinical trial mandates that participants be between the ages of 3 and 100. There are also 4 trials for those under 18, as well as 7 specially tailored studies for seniors over 65 years old."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Long-Term Follow-Up Gene Therapy Study for Achromatopsia CNGB3 and CNGA3

2017. "Long-Term Follow-Up Gene Therapy Study for Achromatopsia CNGB3 and CNGA3". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03278873.

All > Achromatopsia