- Conditions
- Juvenile Neuronal Ceroid Lipofuscinosis, Smith-Lemli-Opitz Syndrome, Creatine Transporter Deficiency, Niemann-Pick Disease Type C1
- Interventions
- Not listed
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- NIH
- Eligibility
- 6 Months to 110 Years
- Enrollment
- 73 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2025
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
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 25–29
of 29
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Niemann-Pick Disease, Type C1
- Interventions
- VTS-270
- Drug
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- NIH
- Eligibility
- 3 Years to 60 Years
- Enrollment
- 2 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2021
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Niemann-Pick Disease, Type C
- Interventions
- IB1001
- Drug
- Lead sponsor
- IntraBio Inc
- Industry
- Eligibility
- 6 Years and older
- Enrollment
- 33 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2022
- U.S. locations
- 1
- States / cities
- Rochester, Minnesota
- Conditions
- Niemann-Pick Disease, Type C
- Interventions
- Adrabetadex
- Drug
- Lead sponsor
- Mandos LLC
- Industry
- Eligibility
- 4 Years to 21 Years
- Enrollment
- 66 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2022
- U.S. locations
- 15
- States / cities
- Birmingham, Alabama • Orange, California • San Francisco, California + 12 more
- Conditions
- Niemann-Pick Disease, Type C
- Interventions
- Arimoclomol
- Drug
- Lead sponsor
- ZevraDenmark
- Industry
- Eligibility
- 2 Years and older
- U.S. locations
- 13
- States / cities
- Birmingham, Alabama • Oakland, California • Orange, California + 10 more