Adrabetadex to Treat Niemann-Pick Type C1 (NPC1) Disease

Stopped early · Phase 2/Phase 3

Conditions studied: Niemann-Pick Disease, Type C

In brief

Due to different study designs, the sponsor separated Part C into this separate registration (NCT04958642), leaving Parts A/B in NCT02534844. The trial's final results for the primary outcome measure of Adverse Events (AE) will be reported here. This study is to evaluate how safe and effective adrabetadex is for participants with Niemann-Pick Type C1 (NPC1) disease who experience neurologic symptoms (listed under Keywords). In Parts A/B (NCT02534844), two out of every 3 participants will receive the study drug. The third participant will receive 1 to 2 small needle pricks at the location where the IT injection is normally made (sham control). In Part C, all participants will receive study drug.

Key facts

Study ID

NCT04958642

Run by

Mandos LLC

People needed

66

Starts

2015-12-23

Expected to finish

2022-04-11

Last updated by the study team

2023-08-29

Who can join

Age: 4 and older, up to 21. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• One of the following is required for inclusion into VTS301 Part C:
• Has agreed to convert from the monthly dosing regimen used in the NIH phase 1/2a protocol to an every 2 weeks dosing regimen
• The investigator has received prior written authorization from the sponsor for the participant to enter VTS301 Part C on an amended dose and/or regimen
• Has received prior written authorization from Vtesse to enroll directly into Part C

You may not qualify if…

• None of the inclusion criteria are applicable

Where it is running

• University of Alabama at Birmingham — Birmingham, Alabama, United States
• Children's Hospital of Orange County: CHOC Children's — Orange, California, United States
• University of California San Francisco — San Francisco, California, United States
• University of Colorado Denver — Aurora, Colorado, United States
• Children's National Medical Center — Washington D.C., District of Columbia, 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 — Randwick, New South Wales, Australia
• Monash Medical Centre — Clayton, Victoria, Australia
• Royal Melbourne Hospital — Melbourne, Victria, Australia
• Royal Perth Hospital — Perth, Western Australia, Australia
• CHU Paris Est - Hopital 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, New Zealand
• National University Hospital (Singapore) Pte, Ltd — Singapore, Singapore

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.