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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)

NCT06192576 · Status: RECRUITING · Type: OBSERVATIONAL · Enrollment: 10

Last updated 2025-10-31

No results posted yet for this study

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.

Conditions

  • Niemann-Pick Diseases
  • Acid Sphingomyelinase Deficiency

Interventions

DRUG

Olipudase alfa

This study will not administer any treatment, only observe the treatment as prescribed in real-world clinical practice.

Sponsors & Collaborators

  • Pulse Infoframe Ltd.

    collaborator INDUSTRY

  • Sanofi

    lead INDUSTRY

Eligibility

Max Age
2 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-04-16
Primary Completion
2029-01-02
Completion
2029-01-15
FDA Drug
Yes

Countries

  • United States

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06192576 on ClinicalTrials.gov