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Showing 1–5 of 5 matching trials from the live ClinicalTrials.gov search.
Conditions
Batten Disease, CLN6
Interventions
Natural history
Other
Lead sponsor
Emily de los Reyes
Other
Eligibility
2 Years to 25 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2026
U.S. locations
1
States / cities
Columbus, Ohio
Enrolling by invitation Phase 1Phase 2 Interventional

Gene Therapy for SLC6A1 Neurodevelopmental Disorder

NCT07173153
Conditions
SLC6A1
Interventions
AAV9.SLC6A1 Gene Therapy
Biological
Lead sponsor
Emily de los Reyes
Other
Eligibility
Not listed
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
1
States / cities
Columbus, Ohio
Conditions
Variant Late-Infantile Neuronal Ceroid Lipofuscinosis
Interventions
AT-GTX-501
Genetic
Lead sponsor
Emily de los Reyes
Other
Eligibility
1 Year and older
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2024
U.S. locations
1
States / cities
Columbus, Ohio
Conditions
CLN6, Batten Disease
Interventions
AT-GTX-501
Genetic
Lead sponsor
Emily de los Reyes
Other
Eligibility
12 Months and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2027
U.S. locations
1
States / cities
Columbus, Ohio
Terminated No phase listed Observational

Natural History Study of Batten Disease

NCT04644549
Conditions
Batten Disease, Neuronal Ceroid Lipofuscinosis CLN6, Neuronal Ceroid Lipofuscinosis CLN3
Interventions
Not listed
Lead sponsor
Emily de los Reyes
Other
Eligibility
Not listed
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
1
States / cities
Rochester, New York