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RECRUITING
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)

Sponsor: Sanofi

View on ClinicalTrials.gov

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

DRUG

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