NCT06192576

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)

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

• Status: Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 10 (estimated)
• Sponsor: Sanofi · Industry
• Sex: All
• Age: 2 Years
• Healthy volunteers: Not accepted

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.

Detailed description

This is a hybrid study design. Patients need not travel to existing study sites in order to enroll.

Conditions

• Niemann-Pick Diseases
• Acid Sphingomyelinase Deficiency

Interventions

Type	Name	Description
DRUG	Olipudase alfa	This study will not administer any treatment, only observe the treatment as prescribed in real-world clinical practice.

Timeline

• Start date: 2024-04-16
• Primary completion: 2029-01-02
• Completion: 2029-01-15
• First posted: 2024-01-05
• Last updated: 2025-10-31

Locations

5 sites across 1 country: United States

Regulatory

• FDA-regulated drug study