- Conditions
- Neuronal Ceroid Lipofuscinosis, Neuronal Ceroid Lipofuscinosis CLN1, Neuronal Ceroid Lipofuscinosis CLN2, Neuronal Ceroid Lipofuscinosis CLN3, Neuronal Ceroid Lipofuscinosis CLN5, Neuronal Ceroid Lipofuscinosis CLN6, Neuronal Ceroid Lipofuscinosis CLN7, Neuronal Ceroid Lipofuscinosis CLN8, Neuronal Ceroid Lipofuscinosis CLN10, Batten Disease
- Interventions
- Not listed
- Lead sponsor
- University of Rochester
- Other
- Eligibility
- Not listed
- Enrollment
- 500 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2004 – 2035
- U.S. locations
- 1
- States / cities
- Rochester, New York
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Showing 1–6
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- 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, Batten's Disease, Neuronal Ceroid Lipofuscinosis CLN6, Neuronal Ceroid Lipofuscinosis
- Interventions
- scAAV9.CB.CLN6 (dose: 1.5E14 vector genomes)
- Drug
- Lead sponsor
- The Charlotte and Gwenyth Gray Foundation
- Other
- Eligibility
- 4 Months and older
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2028
- U.S. locations
- 1
- States / cities
- La Jolla, California
- 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
- 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
- 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