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)

Conditions: Niemann-Pick Diseases, Acid Sphingomyelinase Deficiency

Sex: All

Ages: N/A – 2 Years

Healthy volunteers: No

Enrollment: 10

Sponsor: Sanofi

Location: Ann and Robert H Lurie Children's Hospital of Chicago- Site Number: 001002 Chicago Illinois

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.

Eligibility Criteria

Inclusion Criteria: * The participant must have ASMD type A/B or B and must be \

Interested in this study? View the official listing for contact and enrollment details.

View on ClinicalTrials.gov

Source: ClinicalTrials.gov (NCT06192576). StuddyBuddy aggregates publicly available trial information.