Leber Congenital Amaurosis (LCA)NCT02946879
Long-Term Follow-Up Gene Therapy Study for Leber Congenital Amaurosis OPTIRPE65 (Retinal Dystrophy Associated With Defects in RPE65)
An observational study of Leber Congenital Amaurosis (LCA) and Eye Diseases, run by MeiraGTx UK II Ltd at 2 sites in 2 countries.
Trial summary
This study is a longer-term follow-up study for patients who have been administered AAV2/5-OPTIRPE65 in the Phase I/II, open label, non-randomised, two-centre, dose escalation trial in adults and children with retinal dystrophy associated with defects in RPE65. Read more about this study
- Who is it for?
- Ages 3 to 100 years
- Conditions the study is for
- Leber Congenital Amaurosis (LCA), Eye Diseases, Eye Diseases, Hereditary
- What is being tested?
- AAV OPTIRPE65
- How many people
- 15 participants
- Where
- 2 sitesin 2 countries
- Study dates
- Nov 2016to Jul 2023
- Phase
- Observational
- Are there results?
- None posted in the registry
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 to 100 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: The patient population are those with Leber Congenital Amaurosis (LCA) with gene mutation RPE65 who have participated in the OPTIRPE65 trial.
Who may be able to join
- Were enrolled and treated in the prior open-label, Phase I/II, dose escalation study involving intraocular administration of AAV2/5-OPTIRPE65
Who cannot join
- Individuals will be excluded if they are unwilling or unable to meet with the requirements of the study.
Trial locations and contacts
2 sites in 2 countries.
2 sites in 2 countries
United States1 site
Kellogg Eye Center, University of Michigan Health
Ann Arbor, Michigan, 48105
United Kingdom1 site
Moorfields Eye Hospital NHS Foundation Trust
London
About this study
Official title: Long-term Follow-up Study of Participants Following an Open Label, Multi-centre, Phase I/II Dose Escalation Trial of an Adeno-associated Virus Vector (AAV2/5-OPTIRPE65) for Gene Therapy of Adults and Children With Retinal Dystrophy Owing to Defects in RPE65 (LCA2)
This study is a longer-term follow-up study for patients who have been administered AAV2/5-OPTIRPE65 in the Phase I/II, open label, non-randomised, two-centre, dose escalation trial in adults and children with retinal dystrophy associated with defects in RPE65.
Read the detailed description
The follow up study is designed to collect data on longer-term safety and efficacy of AAV2/5-OPTIRPE65 administration in the OPTIRPE65 trial.
- Conditions
- Leber Congenital Amaurosis (LCA), Eye Diseases, Eye Diseases, Hereditary, Retinal Diseases
- Being tested
- AAV OPTIRPE65
- Medical subject headings
- Leber Congenital Amaurosis, Eye Diseases, Eye Diseases, Hereditary, Retinal Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AAV OPTIRPE65Biological
comparison of different doses of AAV RPE65
| Study group | Role | What they receive |
|---|---|---|
| Low dose AAV OPTIRPE65 | subretinal administration of a single low dose of AAV RPE65 Receives: Biological: AAV OPTIRPE65 | |
| Intermediate dose AAV OPTIRPE65 | subretinal administration of a single intermediate dose of AAV RPE65 Receives: Biological: AAV OPTIRPE65 | |
| High dose AAV OPTIRPE65 | subretinal administration of a single highdose of AAV RPE65 Receives: Biological: AAV OPTIRPE65 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Adverse Events related to the treatment
Safety is defined as the absence of ATIMP-related safety events
Measured: 5 years
Secondary outcomes (3)
Improvement in the retinal function
Improvements in visual function as assessed by visual assessment
Measured: 5 years
Improvement in the visual function
Improvements in retinal function as assessed by visual assessment
Measured: 5 years
Improvement in quality of life
Improvement in the participant's quality of life which is measurable by QoL questionnaire
Measured: 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Oct 2016 (estimated)
- Study startNov 2016
- Primary completionJul 2023
- Study completionJul 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Leber Congenital Amaurosis (LCA)Studies 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- 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
- NCT02946879
- Sponsor's protocol ID
- MGT004
- Other IDs
- 2016-000898-20, 2016
- Status last verified
- Apr 2024
- First submitted
- 22 Jul 2016
- Last updated
- 11 Apr 2024
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 11 Apr 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Long-term Follow-up Study of Participants Following an Open Label, Multi-centre, Phase I/II Dose Escalation Trial of an Adeno-associated Virus Vector (AAV2/5-OPTIRPE65) for Gene Therapy of Adults and Children With Retinal Dystrophy Owing to Defects in RPE65 (LCA2)
- ClinicalTrials.gov ID
- NCT02946879
- Acronym / sponsor ID
- MGT004
- Conditions
- Leber Congenital Amaurosis (LCA), Eye Diseases, Eye Diseases, Hereditary, Retinal Diseases
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- MeiraGTx UK II LtdIndustry (pharma, biotech, devices)
- Study start
- Nov 2016
- Primary completion
- Jul 2023 (actual)
- Study completion
- Jul 2023 (actual)
- First posted
- 27 Oct 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study is a longer-term follow-up study for patients who have been administered AAV2/5-OPTIRPE65 in the Phase I/II, open label, non-randomised, two-centre, dose escalation trial in adults and children with retinal dystrophy associated with defects in RPE65.
Detailed description
The follow up study is designed to collect data on longer-term safety and efficacy of AAV2/5-OPTIRPE65 administration in the OPTIRPE65 trial.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Low dose AAV OPTIRPE65 | – | Biological: AAV OPTIRPE65subretinal administration of a single low dose of AAV RPE65 |
| Intermediate dose AAV OPTIRPE65 | – | Biological: AAV OPTIRPE65subretinal administration of a single intermediate dose of AAV RPE65 |
| High dose AAV OPTIRPE65 | – | Biological: AAV OPTIRPE65subretinal administration of a single highdose of AAV RPE65 |
Who may qualify
- Ages
- 3 to 100 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- The patient population are those with Leber Congenital Amaurosis (LCA) with gene mutation RPE65 who have participated in the OPTIRPE65 trial.
Key inclusion criteria (1 listed)
- Were enrolled and treated in the prior open-label, Phase I/II, dose escalation study involving intraocular administration of AAV2/5-OPTIRPE65
Key exclusion criteria (1 listed)
- Individuals will be excluded if they are unwilling or unable to meet with the requirements of the study.
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 OPTIRPE65comparison of different doses of AAV RPE65 | Biological | High dose AAV OPTIRPE65, Intermediate dose AAV OPTIRPE65, Low dose AAV OPTIRPE65 |
Primary outcome
Incidence of Adverse Events related to the treatment
Time frame: 5 years
Safety is defined as the absence of ATIMP-related safety events
3 secondary outcomes
Improvement in the retinal function
Time frame: 5 years
Improvement in the visual function
Time frame: 5 years
Improvement in quality of life
Time frame: 5 years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Apr 2024.
- Individual participant dataSharing planned: Undecided
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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?
An observational study of Leber Congenital Amaurosis (LCA) and Eye Diseases, 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 1 inclusion and 1 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 11 Apr 2024. 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.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Incidence of Adverse Events related to the treatment (time frame: 5 years). 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 11 Apr 2024; AzTrials last refreshed its copy on 6 Nov 2026.