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.
At a Glance
- Age
- Up to 2
- Sex
- Any
- Study type
- Observational
- Participants needed
- 10 (estimated)
- Sponsor
- Sanofi · Industry
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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
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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
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