Leber Congenital AmaurosisPhase 1/2OPTIRPE65NCT02781480
Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA)
A Phase 1/2 study testing AAV RPE65 in people with Leber Congenital Amaurosis, run by MeiraGTx UK II Ltd at 2 sites in 2 countries.
Trial summary
A clinical trial of AAV2/5 vector for patients with Defects in RPE65 Read more about this study
- Who is it for?
- Ages 3 years and older
- Conditions the study is for
- Leber Congenital Amaurosis
- What is being tested?
- AAV RPE65
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 2 sitesin 2 countries
- Study dates
- Apr 2016to Dec 2018
- 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
- Aged 3 years or older
- Early-onset severe retinal dystrophy consistent with RPE65 deficiency
Who cannot join
- Females who are pregnant or breastfeeding
- Have participated in another research study involving an investigational therapy for ocular disease within the last 6 months.
Trial locations and contacts
2 sites in 2 countries.
2 sites in 2 countries
United States1 site
Kellogg Eye Centre, University of Michigan
Ann Arbor, Michigan, 48105
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 an Adeno Associated Virus Vector for Gene Therapy of Adults And Children With Retinal Dystrophy Associated With Defects in RPE65 (LCA)
A clinical trial of AAV2/5 vector for patients with Defects in RPE65
Read the detailed description
A dose escalation and dose expansion (Phase I/II) trial of adults and children with retinal dystrophy associated with defects in RPE65. ATIMP will be administered to one eye only in a single sub-retinal procedure
- Conditions
- Leber Congenital Amaurosis
- Being tested
- AAV RPE65
- Medical subject headings
- Leber Congenital Amaurosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AAV RPE65Biological
Comparison of different dosages of AAV RPE65
| Study group | Role | What they receive |
|---|---|---|
| Low dose AAV-RPE65 | Experimental | Subretinal administration of a single low dose of range AAV-RPE65 Receives: Biological: AAV RPE65 |
| Intermediate dose AAV-RPE65 | Experimental | Subretinal administration of a single intermediate dose of range AAV-RPE65 Receives: Biological: AAV RPE65 |
| High dose AAV-RPE65 | Experimental | Subretinal administration of a single high dose of range AAV-RPE65 Receives: Biological: AAV RPE65 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall Safety of Adeno-Associated Virus Vector (AAV-OPTIRPE65) - Number of Participants With a Safety Event
Safety was defined as an advanced therapy investigational medicinal product (ATIMP) related:
- Reduction in visual acuity by 15 Early Treatment Diabetic Retinopathy Study (ETDRS) letters or more.
- Severe unresponsive inflammation.
- Infective endophthalmitis.
- Ocular malignancy.
- Grade III or above non-ocular suspected unexpected serious adverse reaction (SUSAR).
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 posted24 May 2016 (estimated)
- Study startApr 2016
- Primary completionDec 2018
- Study completionDec 2018
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 Leber Congenital AmaurosisStudies 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
- Model description
- AAV2/5-OPTIRPE65
- Masking
- None (open label)
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- MeiraGTx UK II Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02781480
- Sponsor's protocol ID
- MGT003
- Acronym
- OPTIRPE65
- Status last verified
- Jun 2021
- First submitted
- 28 Apr 2016
- Last updated
- 12 Jul 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 12 Jul 2021. 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 an Adeno Associated Virus Vector for Gene Therapy of Adults And Children With Retinal Dystrophy Associated With Defects in RPE65 (LCA)
- ClinicalTrials.gov ID
- NCT02781480
- Acronym / sponsor ID
- OPTIRPE65 / MGT003
- Conditions
- Leber Congenital Amaurosis
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- MeiraGTx UK II LtdIndustry (pharma, biotech, devices)
- Study start
- Apr 2016
- Primary completion
- Dec 2018 (actual)
- Study completion
- Dec 2018 (actual)
- First posted
- 24 May 2016 (estimated)
- Results
- Posted 8 Apr 2021
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 AAV2/5 vector for patients with Defects in RPE65
Detailed description
A dose escalation and dose expansion (Phase I/II) trial of adults and children with retinal dystrophy associated with defects in RPE65. ATIMP will be administered to one eye only in a single sub-retinal procedure
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
- Model description
- AAV2/5-OPTIRPE65
| Arm | Type | Receives |
|---|---|---|
| Low dose AAV-RPE65 | Experimental | Biological: AAV RPE65Subretinal administration of a single low dose of range AAV-RPE65 |
| Intermediate dose AAV-RPE65 | Experimental | Biological: AAV RPE65Subretinal administration of a single intermediate dose of range AAV-RPE65 |
| High dose AAV-RPE65 | Experimental | Biological: AAV RPE65Subretinal administration of a single high dose of range AAV-RPE65 |
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)
- Aged 3 years or older
- Early-onset severe retinal dystrophy consistent with RPE65 deficiency
Key exclusion criteria (2 listed)
- Females who are pregnant or breastfeeding
- Have participated in another research study involving an investigational therapy for ocular disease within the last 6 months.
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 RPE65Comparison of different dosages of AAV RPE65 | Biological | High dose AAV-RPE65, Intermediate dose AAV-RPE65, Low dose AAV-RPE65 |
Primary outcome
Overall Safety of Adeno-Associated Virus Vector (AAV-OPTIRPE65) - Number of Participants With a Safety Event
Time frame: 6 months
Safety was defined as an advanced therapy investigational medicinal product (ATIMP) related: * Reduction in visual acuity by 15 Early Treatment Diabetic Retinopathy Study (ETDRS) letters or more. * Severe unresponsive inflammation. * Infective endophthalmitis. * Ocular malignancy. * Grade III or above non-ocular suspected unexpected serious adverse reaction (SUSAR).
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Jul 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: Undecided
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Active, not recruitingPhase 1/2
Study of Subretinally Injected ATSN-101 Administered in Patients With Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2D
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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.
The record lists no contact. Use the official record to reach the sponsor.
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 RPE65 in people with Leber Congenital Amaurosis, 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 2 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 12 Jul 2021. 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 Apr 2021). 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?
Overall Safety of Adeno-Associated Virus Vector (AAV-OPTIRPE65) - Number of Participants With a Safety Event (time frame: 6 months). 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 12 Jul 2021.