VTS-270 to Treat Niemann-Pick Type C1 (NPC1) Disease
Completed · Phase 2/Phase 3 · Has a placebo group
Conditions studied: Niemann-Pick Disease, Type C
In brief
Due to different study designs, the sponsor separated Part C into a separate registration (NCT04958642), leaving Parts A/B here in NCT02534844. This study is to find out how safe and effective VTS-270 is for patients with Niemann-Pick Type C1 (NPC1) disease who have neurologic symptoms (listed under Keywords). In Parts A/B, two out of every three patients will receive the study drug. The third patient will receive 1 to 2 small needle pricks at the location where the LP and IT injection is normally made (sham control). In Part C, all participants will receive study drug, as described in the Part C registration record. Start date for this record is the first day a participant was enrolled in Parts A/B. The trial is actually continuing until the last primary outcome measure of safety data are collected from Part C participants. The last primary outcome measure of safety, along with final adverse events results will be posted in the separate Part C registration record.
Key facts
- Study ID
- NCT02534844
- Run by
- Mandos LLC
- People needed
- 56
- Starts
- 2015-10-01
- Expected to finish
- 2018-03-28
- Last updated by the study team
- 2023-02-22
Who can join
Age: 4 and older, up to 21. Sex: any. Healthy volunteers: not accepted.
Where it is running
- University of Alabama at Birmingham — Birmingham, Alabama, United States
- Children's Hospital of Orange County — Orange, California, United States
- University of California San Francisco — San Francisco, California, United States
- University of Colorado Denver — Aurora, Colorado, United States
- Shands Children's Hospital — Gainesville, Florida, United States
- Rush University Medical Center — Chicago, Illinois, United States
- Eunice Kennedy Shriver National Institute of Child Health and Human Development — Bethesda, Maryland, United States
- Boston Children's Hospital — Boston, Massachusetts, United States
- Montefiore Medical Center — The Bronx, New York, United States
- University of North Carolina — Chapel Hill, North Carolina, United States
- Lehigh Valley Health Network — Allentown, Pennsylvania, United States
- The Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
- Dell Children's Medical Center of Central Texas — Austin, Texas, United States
- Multicare Institute for Research and Innovation — Tacoma, Washington, United States
- The Prince of Wales Hospital — Sydney, New South Wales, Australia
- Monash Medical Centre — Clayton, Victoria, Australia
- Royal Melbourne Hospital — Parkville, Victoria, Australia
- Royal Perth Hospital — Perth, Western Australia, Australia
- CHU Paris Est - Hospital d'Enfants Armand-Trousseau — Paris, Cedex 12, France
- Katholisches Klinikim Bochum gGmbH — Bochum, Germany
- Universitaetsklinikum Mainz — Mainz, Germany
- Universitaetsklinikum Muenster — Münster, Germany
- Waikato Hospital — Hamilton West, New Zealand
- National University Hospital (Singapore) Pte, Ltd — Singapore, Singapore
- Hospital Universitario del Valle Hebron — Barcelona, Spain
Full record on ClinicalTrials.gov
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