An Open-Label, Multicenter, Ascending Dose Study Of The Tolerability And Safety Of Recombinant Human Acid Sphingomyelinase (rhASM) In Patients With Acid Sphingomyelinase Deficiency (ASMD)

ID Number 12-1897

Principal Investigator(s)
Melissa P. Wasserstein

Department(s) or Division(s)
Genetics and Genomic Sciences


This is a study designed to find out if a drug called recombinant human acid sphingomyelinase (rhASM) is safe to give to humans.  This drug has been developed to treat a disease called Niemann Pick Disease type B or Acid Sphingomyelinase Deficiency (ASMD).  If the safety of the drug is shown additional studies will be carried out to see of it reverses and prevents the symptoms of ASMD.

Contact Information
Natalie Lippa, MS, CGC
(212) 659-1456

Recruiting Patients: No