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IV adrabetadex (VTS-270) for NPC

Official Title

Phase 1/2a study of 2-Hydroxypropyl-Beta-Cyclodextrin Therapy for Infantile Liver Disease Associated with Niemann-Pick Disease, type C

Study Details

The purpose of the study is to learn how well adrabetadex can reduce liver damage in infants diagnosed with Niemann-Pick (NPC) disease, type C.

Principal Investigator

Dr. Patricia DIckson

IRB Number

201708114

Eligibility

1. Participants must be 0 to 6 months of age
2. Participants must have diagnosis of NPC
3. Participants and their guardians must be able to travel to Washington University in St. Louis

Volunteer Information

For more information about this study, contact Volunteer for Health at:

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