Contact this trialFirst, we need to learn more about you.
Olipudase alfa arm for Niemann-Pick Disease
Recruiting1 award5 criteria
London, Ontario
US, multicenter, cohort, open label observational study with primary data collection. Ancillary protocol-specified procedures to address the study objectives (eg, assessment of ADA) may be considered outside the standard of care for acid sphingomyelinase deficiency (ASMD), but the study methodology remains non-interventional, as the additional collection of data from participants will not dictate treatment. The total overall study duration will be 5 years. The follow-up period will be a minimum of 1 year to a maximum of 3 years. The enrollment period will be up to 4 years, to allow a minimum of 1 year of follow-up for the last participant enrolled.
Fill-in a few details and create your profileHaving a profile will help us send you recommended trials and speed up your application in the future.
We will keep your details privateWe won’t share unless you give us permission. By completing this form you agree to our privacy policy
Get in touch with the study teamWe’ll send your responses to the study team and help you to setup your first screening. This is not the start of informed consent. Screening happens between you and the trial.