A Real-world Long-term Safety and Immunogenicity Study of Olipudase Alfa Therapy in Pediatric Patients Less Than 2 Years of Age With Acid Sphingomyelinase Deficiency (ASMD)

Recruiting Observational Study
Niemann-Pick Diseases Acid Sphingomyelinase Deficiency
No Placebo Group Every participant receives an active treatment — no one gets a placebo. No Study Drug Researchers observe your health over time — no experimental treatment is given.
Ready to participate?

Review the details below, then apply to join this clinical trial.

At a Glance
Age
Up to 2
Sex
Any
Study type
Observational
Participants needed
10 (estimated)
Sponsor
Sanofi · Industry
Think this trial could be right for you?

Answer a few quick questions to see if you may meet the eligibility requirements.

Check Your Eligibility
About This Trial
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 …
Trial Locations
Loading…

Loading trial locations…

Eligibility Criteria
Inclusion Criteria: * The participant must have ASMD type A/B or B and must be \<2 years of age at the time of treatment initiation, OR ASMD type A (without age restriction). * The participant must weigh ≥ 2 kg \[The United States Prescribing Information (USPI)\] for olipudase alfa specifies this m…
Contacts

Trial Transparency email recommended (Toll free for US & Canada)

800-633-1610

contact-us@sanofi.com

CONTACT