● Recruiting

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)

Condition: Niemann-Pick Diseases · Acid Sphingomyelinase Deficiency · Sponsor: Sanofi

PhaseN/A

Planned participants10

Who can joinAll sexes, N/A to 2 Years

Healthy volunteersNo

About this study

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.

This description comes directly from the study's public registry record.

Talk to the study team

Trial Transparency email recommended (Toll free for US & Canada) · 800-633-1610 · contact-us@sanofi.com

Always discuss trial participation with your own doctor first.

Locations (5)

Ann and Robert H Lurie Children's Hospital of Chicago- Site Number: 001002	Chicago, Illinois, United States	Recruiting
Children's Hospital Medical Center- Site Number: 001003	Cincinnati, Ohio, United States	Recruiting
Nationwide Children's Hospital - PIN- Site Number : 1001-1	Columbus, Ohio, United States	Recruiting
Pulse InfoFrame US Inc.- Site Number: 001001	Philadelphia, Pennsylvania, United States	Recruiting
Dell Children's Medical Center- Site Number : 1001-2	Austin, Texas, United States	Recruiting

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Source record: clinicaltrials.gov/study/NCT06192576