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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)
Sponsor: Sanofi
Summary
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.
Official title: A Prospective Observational Study to Assess the Long-term Safety and Immunogenicity of Olipudase Alfa Therapy During Routine Clinical Care in Pediatric Patients Less Than 2 Years of Age With Acid Sphingomyelinase Deficiency
Key Details
Gender
All
Age Range
Any - 2 Years
Study Type
OBSERVATIONAL
Enrollment
10
Start Date
2024-04-16
Completion Date
2029-01-15
Last Updated
2025-10-31
Healthy Volunteers
No
Interventions
Olipudase alfa
This study will not administer any treatment, only observe the treatment as prescribed in real-world clinical practice.
Locations (5)
Ann and Robert H Lurie Children's Hospital of Chicago- Site Number: 001002
Chicago, Illinois, United States
Children's Hospital Medical Center- Site Number: 001003
Cincinnati, Ohio, United States
Nationwide Children's Hospital - PIN- Site Number : 1001-1
Columbus, Ohio, United States
Pulse InfoFrame US Inc.- Site Number: 001001
Philadelphia, Pennsylvania, United States
Dell Children's Medical Center- Site Number : 1001-2
Austin, Texas, United States