AchromatopsiaPhase 1/2CNGA3NCT03758404
Gene Therapy for Achromatopsia (CNGA3)
A Phase 1/2 study testing adeno-associated virus vector AAV- CNGA3 in people with Achromatopsia, run by MeiraGTx UK II Ltd at 2 sites in 2 countries.
Trial summary
A clinical trial of adeno-associated virus vector (AAV) CNGA3 retinal gene therapy for patients with achromatopsia Read more about this study
- Who is it for?
- Ages 3 years and older
- Conditions the study is for
- Achromatopsia
- What is being tested?
- adeno-associated virus vector AAV- CNGA3
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 11 participants
- Where
- 2 sitesin 2 countries
- Study dates
- 12 Aug 2019to 10 Jun 2021
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Are aged years or over
- Have achromatopsia confirmed by a retinal specialist investigator
Who cannot join
- 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
Trial locations and contacts
2 sites in 2 countries.
2 sites in 2 countries
United Kingdom1 site
Moorfields Eye Hospital NHS Foundation Trust
London, EC1V 2PD
About this study
Official title: An Open Label, Multi-centre, Phase I/II Dose Escalation Trial of a Recombinant Adeno-associated Virus Vector (AAV2/8-hG1.7p.coCNGA3) for Gene Therapy of Children and Adults With Achromatopsia Owing to Defects in CNGA3
A clinical trial of adeno-associated virus vector (AAV) CNGA3 retinal gene therapy for patients with achromatopsia
Read the detailed description
CNGA3 retinal gene therapy for patients with achromatopsia
- Conditions
- Achromatopsia
- Being tested
- adeno-associated virus vector AAV- CNGA3
- Medical subject headings
- Color Vision Defects
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
adeno-associated virus vector AAV- CNGA3Biological
Adeno-associated virus (AAV) gene therapy for defects in CNGA3 gene
| Study group | Role | What they receive |
|---|---|---|
| Low dose adeno-associated virus (AAV) CNGA3 | Experimental | Subretinal administration of a single low dose AAV CNGA3 Receives: Biological: adeno-associated virus vector AAV- CNGA3 |
| Intermediate dose adeno-associated virus (AAV) CNGA3 | Experimental | Subretinal administration of a single intermediate dose AAV CNGA3 Receives: Biological: adeno-associated virus vector AAV- CNGA3 |
| High dose adeno-associated virus (AAV) CNGA3 | Experimental | Subretinal administration of a single high dose AAV CNGA3 Receives: Biological: adeno-associated virus vector AAV- CNGA3 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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)
Measured: 6 Weeks
Secondary outcomes (4)
Improvements in Visual Function as Assessed by Visual Acuity
Change from baseline to Week 24 in best corrected visual acuity (BCVA) using Early Treatment Diabetic Retinopathy Study (ETDRS) chart letter score. The direction of improvement from baseline is an increase in the number of ETDRS letters read over time.
Measured: 6 Months
Improvements in Retinal Function as Assessed by Static Perimetry
Change from baseline to Week 24 in mean retinal sensitivity in the treated eye. The direction of improvement is an increase in sensitivity.
Measured: 6 Months
Quality of Life Measured by QoL Questionnaires in Children and Adolescents
Change from baseline to Week 24 in EuroQol Visual Analogue Scale (EQ-VAS) in children and adolescents. EQ-VAS uses a scale from 0 to 100, where 0 represents the worst imaginable health state and 100 represents the best imaginable health state. A positive change from baseline reflects improvement and a negative reflects worsening.
Measured: 6 Months
Quality of Life Measured by QoL Questionnaires in Adults
Change from baseline to Week 24 in EuroQol Visual Analogue Scale (EQ-VAS) in adults. EQ-VAS uses a scale from 0 to 100, where 0 represents the worst imaginable health state and 100 represents the best imaginable health state. A positive change from baseline reflects improvement and a negative reflects worsening.
Measured: 6 Months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Nov 2018
- Study start12 Aug 2019
- Primary completion10 Jun 2021
- Study completion10 Jun 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for AchromatopsiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 11 participants (actual)
Sponsor and investigators
- Lead sponsor
- MeiraGTx UK II Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Janssen Research & Development, LLC
- Responsible party
- Sponsor
- Investigators
- James Bainbridge, Principal investigator, Chief Investigator
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03758404
- Sponsor's protocol ID
- MGT012
- Acronym
- CNGA3
- Status last verified
- Nov 2022
- First submitted
- 27 Nov 2018
- Last updated
- 1 Dec 2022
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Dec 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open Label, Multi-centre, Phase I/II Dose Escalation Trial of a Recombinant Adeno-associated Virus Vector (AAV2/8-hG1.7p.coCNGA3) for Gene Therapy of Children and Adults With Achromatopsia Owing to Defects in CNGA3
- ClinicalTrials.gov ID
- NCT03758404
- Acronym / sponsor ID
- CNGA3 / MGT012
- Conditions
- Achromatopsia
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 11 participants (actual)
- Lead sponsor
- MeiraGTx UK II LtdIndustry (pharma, biotech, devices)
- Collaborators
- Janssen Research & Development, LLC
- Study start
- 12 Aug 2019 (actual)
- Primary completion
- 10 Jun 2021 (actual)
- Study completion
- 10 Jun 2021 (actual)
- First posted
- 29 Nov 2018 (actual)
- Results
- Posted 11 Feb 2022
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A clinical trial of adeno-associated virus vector (AAV) CNGA3 retinal gene therapy for patients with achromatopsia
Detailed description
CNGA3 retinal gene therapy for patients with achromatopsia
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Low dose adeno-associated virus (AAV) CNGA3 | Experimental | Biological: adeno-associated virus vector AAV- CNGA3Subretinal administration of a single low dose AAV CNGA3 |
| Intermediate dose adeno-associated virus (AAV) CNGA3 | Experimental | Biological: adeno-associated virus vector AAV- CNGA3Subretinal administration of a single intermediate dose AAV CNGA3 |
| High dose adeno-associated virus (AAV) CNGA3 | Experimental | Biological: adeno-associated virus vector AAV- CNGA3Subretinal administration of a single high dose AAV CNGA3 |
Who may qualify
- Ages
- 3 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (2 listed)
- Are aged years or over
- Have achromatopsia confirmed by a retinal specialist investigator
Key exclusion criteria (3 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| adeno-associated virus vector AAV- CNGA3Adeno-associated virus (AAV) gene therapy for defects in CNGA3 gene | Biological | High dose adeno-associated virus (AAV) CNGA3, Intermediate dose adeno-associated virus (AAV) CNGA3, Low dose adeno-associated virus (AAV) CNGA3 |
Primary outcome
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.
Time frame: 6 Weeks
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)
4 secondary outcomes
Improvements in Visual Function as Assessed by Visual Acuity
Time frame: 6 Months
Improvements in Retinal Function as Assessed by Static Perimetry
Time frame: 6 Months
Quality of Life Measured by QoL Questionnaires in Children and Adolescents
Time frame: 6 Months
Quality of Life Measured by QoL Questionnaires in Adults
Time frame: 6 Months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Dec 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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Achromatopsia
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
James Bainbridge principal investigator
Chief Investigator
2 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing adeno-associated virus vector AAV- CNGA3 in people with Achromatopsia, run by MeiraGTx UK II Ltd at 2 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 1 Dec 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 11 Feb 2022). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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. (time frame: 6 Weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Dec 2022.