AchromatopsiaPhase 1/2CNGB3NCT03001310
Gene Therapy for Achromatopsia (CNGB3)
A Phase 1/2 study testing AAV - CNGB3 in people with Achromatopsia, run by MeiraGTx UK II Ltd at 2 sites in 2 countries.
Trial summary
A clinical trial of AAV - CNGB3 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?
- AAV - CNGB3
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 23 participants
- Where
- 2 sitesin 2 countries
- Study dates
- 16 Jan 2017to 25 Oct 2019
- 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 3 years or older
- Have achromatopsia confirmed by a retinal specialist (CI or PI)
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 CI/PI considers makes them inappropriate for entry into the trial
Trial locations and contacts
2 sites in 2 countries.
2 sites in 2 countries
United States1 site
University of Michigan Kellog Eye Centre
Ann Arbor, Michigan, MI 48105
United Kingdom1 site
Moorfields Eye Hospital NHS Foundation Trust
London
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-hCARp.hCNGB3) for Gene Therapy of Adults and Children With Achromatopsia Owing to Defects in CNGB3
A clinical trial of AAV - CNGB3 retinal gene therapy for patients with achromatopsia
Read the detailed description
CNGB3 retinal gene therapy for patients with achromatopsia
- Conditions
- Achromatopsia
- Being tested
- AAV - CNGB3
- 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.
AAV - CNGB3Biological
Comparison of different dosages of AAV-CNGB3
| Study group | Role | What they receive |
|---|---|---|
| Biological-Low dose AAV - CNGB3 | Experimental | Subretinal administration of a single low dose of AAV - CNGB3 Receives: Biological: AAV - CNGB3 |
| Biological-Medium dose AAV - CNGB3 | Experimental | Subretinal administration of a single intermediate dose of AAV - CNGB3 Receives: Biological: AAV - CNGB3 |
| Biological-High dose AAV - CNGB3 | Experimental | Subretinal administration of a single high dose of AAV - CNGB3 Receives: Biological: AAV - CNGB3 |
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 in the treated eye. 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-5D-Y 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 change from baseline reflects worsening.
Measured: 6 months
Quality of Life Measured by QoL Questionnaires in Adults
Change from baseline to Week 24 in EuroQol-5D-5L 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 change from baseline 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 posted23 Dec 2016 (estimated)
- Study start16 Jan 2017
- Primary completion25 Oct 2019
- Study completion25 Oct 2019
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.
- Publications (1)Papers the sponsor has linked to this study.
- 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
- Sequential
- Masking
- None (open label)
- Enrolment
- 23 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, Prof, Principal investigator, Chief Investigator
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03001310
- Sponsor's protocol ID
- MGT006
- Other IDs
- 2016-002290-35, 2016
- Acronym
- CNGB3
- Status last verified
- Feb 2023
- First submitted
- 22 Nov 2016
- Last updated
- 8 Mar 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
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.
PubMed 37172884
Study at a glance
From the registry record, last updated by the sponsor on 8 Mar 2023. 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-hCARp.hCNGB3) for Gene Therapy of Adults and Children With Achromatopsia Owing to Defects in CNGB3
- ClinicalTrials.gov ID
- NCT03001310
- Acronym / sponsor ID
- CNGB3 / MGT006
- Conditions
- Achromatopsia
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 23 participants (actual)
- Lead sponsor
- MeiraGTx UK II LtdIndustry (pharma, biotech, devices)
- Collaborators
- Janssen Research & Development, LLC
- Study start
- 16 Jan 2017 (actual)
- Primary completion
- 25 Oct 2019 (actual)
- Study completion
- 25 Oct 2019 (actual)
- First posted
- 23 Dec 2016 (estimated)
- Results
- Posted 8 Mar 2023
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 AAV - CNGB3 retinal gene therapy for patients with achromatopsia
Detailed description
CNGB3 retinal gene therapy for patients with achromatopsia
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Biological-Low dose AAV - CNGB3 | Experimental | Biological: AAV - CNGB3Subretinal administration of a single low dose of AAV - CNGB3 |
| Biological-Medium dose AAV - CNGB3 | Experimental | Biological: AAV - CNGB3Subretinal administration of a single intermediate dose of AAV - CNGB3 |
| Biological-High dose AAV - CNGB3 | Experimental | Biological: AAV - CNGB3Subretinal administration of a single high dose of AAV - CNGB3 |
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 3 years or older
- Have achromatopsia confirmed by a retinal specialist (CI or PI)
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 CI/PI 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 |
|---|---|---|
| AAV - CNGB3Comparison of different dosages of AAV-CNGB3 | Biological | Biological-High dose AAV - CNGB3, Biological-Low dose AAV - CNGB3, Biological-Medium dose AAV - CNGB3 |
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 8 Mar 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 37172884Michaelides 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.
- Individual participant dataSharing planned: No
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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, Prof 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 AAV - CNGB3 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 8 Mar 2023. 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 8 Mar 2023). 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?
On the ClinicalTrials.gov record for NCT03001310, which also lists 1 linked publication.
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 8 Mar 2023.