- 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
Search Results
Search by objective public record fields.
Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results come from ClinicalTrials.gov. When the live registry is unavailable, a clearly labeled stored copy may be shown.
These results were retrieved from the live ClinicalTrials.gov registry. Filters and sort order apply to this result list.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Showing 1–8
of 8
matching trials from the live ClinicalTrials.gov search.
- 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
- 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
- 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
- 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
- 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
- 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