FDA Approved Clinical Trials
FDA Approved Clinical Trials are underway to study treatments for Niemann-Pick disease. Follow the links below for information.
ASMD Clinical Trial
There are no current clinical trials for ASMD in the U.S. at this time. For information on Xenpozyme, approved for the treatment of non–central nervous system manifestations of acid sphingomyelinase deficiency (ASMD) in adult and pediatric patients, visit XENPOZYME® (olipudase alfa-rpcp)
NPC Clinical Trials
Recruiting:
- Azafaros
- Cyclo Therapeutics, Inc.
- National Institutes of Health (Infant NPC Trial and Study to treat liver disease now recruiting)
Other FDA Approved Clinical Trials
Clinical trial listings on clinicaltrials.gov
Learn how to participate in a clinical trial
Clinical Trial Update Procedure
The NNPDF posts new information regarding clinical trial updates as soon as it is received. Please review the following Clinical Trial Update procedure for alerting you to these updates as they are made:
The NNPDF office will post information received about trials and expanded access therapies from companies on the NNPDF website under the appropriate pharmaceutical company.
When information is received it is:
- Posted on the NNPDF website
- Shared via email announcement
- Summary post is made on the NNPDF Facebook Business page, NNPDF Facebook Awareness Group, NNPDF Facebook Staff page, Instagram and Twitter with a link back to our website where you can find more detailed information.
All updates from pharmaceutical companies are included in the next monthly e-newsletter with links back to the original posting on the website.