- Conditions
- Batten Disease, Late-Infantile Neuronal Ceroid Lipofuscinosis
- Interventions
- AAVrh.10CUhCLN2 vector 9.0x10^11 genome copies, AAVrh.10CUhCLN2 vector 2.85x10^11 genome copies
- Biological
- Lead sponsor
- Weill Medical College of Cornell University
- Other
- Eligibility
- 2 Years to 18 Years
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2020
- U.S. locations
- 1
- States / cities
- New York, New York
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Showing 1–8
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- Conditions
- Late Infantile Neuronal Ceroid Lipofuscinosis, Batten Disease
- Interventions
- AAVrh.10CUCLN2
- Biological
- Lead sponsor
- Weill Medical College of Cornell University
- Other
- Eligibility
- 3 Years to 18 Years
- Enrollment
- 8 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2017
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2
- Interventions
- Cerliponase Alfa, Administration Kit
- Drug · Device
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- Not listed
- Enrollment
- 35 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2030
- U.S. locations
- 16
- States / cities
- Little Rock, Arkansas • Orange, California • Aurora, Colorado + 12 more
- Conditions
- Jansky-Bielschowsky Disease, Batten Disease, Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2, CLN2 Disease, CLN2 Disorder
- Interventions
- BMN 190, Intracerebroventricular (ICV) access device
- Biological · Device
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 3 Years to 16 Years
- Enrollment
- 23 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2020
- U.S. locations
- 1
- States / cities
- Columbus, Ohio
- Conditions
- Batten Disease, CLN2, Neuronal Ceroid-Lipofuscinoses
- Interventions
- CLN2 Treatment
- Other
- Lead sponsor
- Jessica Scherr
- Other
- Eligibility
- Not listed
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2025
- U.S. locations
- 1
- States / cities
- Columbus, Ohio
- Conditions
- Neuronal Ceroid Lipofuscinosis Type 2
- Interventions
- Cerliponase Alfa
- Drug
- Lead sponsor
- David L Rogers, MD
- Other
- Eligibility
- 24 Months to 72 Months
- Enrollment
- 5 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2027
- U.S. locations
- 1
- States / cities
- Columbus, Ohio
- Conditions
- Jansky-Bielschowsky Disease, Batten Disease, Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2, CLN2 Disease, CLN2 Disorder
- Interventions
- BMN 190 recombinant human tripeptidyl peptidase-1 (rhTPP1), Intracerebroventricular access device
- Biological · Device
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- Up to 17 Years
- Enrollment
- 14 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2022
- U.S. locations
- 1
- States / cities
- Columbus, Ohio
- Conditions
- Batten Disease, Late Infantile Neuronal Ceroid Lipofuscinosis
- Interventions
- AAV2CUhCLN2 (3x10^12 particle units)
- Biological
- Lead sponsor
- Weill Medical College of Cornell University
- Other
- Eligibility
- 3 Years to 18 Years
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2004 – 2019
- U.S. locations
- 1
- States / cities
- New York, New York