Long-Term Follow-Up Gene Therapy Study for Leber Congenital Amaurosis OPTIRPE65 (Retinal Dystrophy Associated With Defects in RPE65)
NCT ID: NCT02946879
Last Updated: 2024-04-11
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
COMPLETED
15 participants
OBSERVATIONAL
2016-11-30
2023-07-31
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
Related Clinical Trials
Explore similar clinical trials based on study characteristics and research focus.
An Expanded Clinical Study Evaluating the AAV2-RPE65 Gene Therapy(LX101) in Patients With LCA
NCT06024057
Natural History Study in Inherited Retinal Disease Subjects Caused by Mutations in RPE65 or LRAT
NCT02575430
Safety/Proof of Concept Study of Oral QLT091001 in Subjects With Leber Congenital Amaurosis (LCA) or Retinitis Pigmentosa (RP) Due to Retinal Pigment Epithelial 65 Protein (RPE65) or Lecithin:Retinol Acyltransferase (LRAT) Mutations
NCT01014052
Single Ascending Dose Study in Participants With LCA10
NCT03872479
Repeated Treatments of QLT091001 in Subjects With Leber Congenital Amaurosis or Retinitis Pigmentosa (Extension of Study RET IRD 01)
NCT01521793
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
COHORT
PROSPECTIVE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
Low dose AAV OPTIRPE65
subretinal administration of a single low dose of AAV RPE65
AAV OPTIRPE65
comparison of different doses of AAV RPE65
Intermediate dose AAV OPTIRPE65
subretinal administration of a single intermediate dose of AAV RPE65
AAV OPTIRPE65
comparison of different doses of AAV RPE65
High dose AAV OPTIRPE65
subretinal administration of a single highdose of AAV RPE65
AAV OPTIRPE65
comparison of different doses of AAV RPE65
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
AAV OPTIRPE65
comparison of different doses of AAV RPE65
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
Exclusion Criteria
3 Years
100 Years
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
MeiraGTx UK II Ltd
INDUSTRY
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
Kellogg Eye Center, University of Michigan Health
Ann Arbor, Michigan, United States
Moorfields Eye Hospital NHS Foundation Trust
London, , United Kingdom
Countries
Review the countries where the study has at least one active or historical site.
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
2016-000898-20
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
MGT004
Identifier Type: -
Identifier Source: org_study_id
More Related Trials
Additional clinical trials that may be relevant based on similarity analysis.