FDA Approved Clinical Trials
FDA Approved Clinical Trials are underway to study treatments for Acid Sphingomyelinase Deficiency (ASMD) and Niemann-Pick Disease type C (NPC). Follow the links below for more information.
Acid Sphingomyelinase Deficiency (ASMD)
RECRUITING:
- Sanofi (Age 0-24 months with ASMD)
Niemann-Pick Disease type C (NPC)
ACTIVE - NOT RECRUITING:
- Cyclo Therapeutics, Inc.
- Azafaros (Currently, no active U.S. sites)
- National Institutes of Health (Infant NPC Trial and Study to treat liver disease)