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)
Public ClinicalTrials.gov record NCT06192576. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
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
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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.
Study identification
- NCT ID
- NCT06192576
- Recruitment status
- Recruiting
- Study type
- Observational
- Phase
- Not listed
- Enrollment
- 10 participants
Conditions and interventions
Eligibility (public fields only)
- Age range
- Up to 2 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Apr 15, 2024
- Primary completion
- Jan 1, 2029
- Completion
- Jan 14, 2029
- Last update posted
- Oct 30, 2025
2024 – 2029
United States locations
- U.S. sites
- 5
- U.S. states
- 4
- U.S. cities
- 5
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Ann and Robert H Lurie Children's Hospital of Chicago- Site Number: 001002 | Chicago | Illinois | 60611 | Recruiting |
| Children's Hospital Medical Center- Site Number: 001003 | Cincinnati | Ohio | 45229-3039 | Recruiting |
| Nationwide Children's Hospital - PIN- Site Number : 1001-1 | Columbus | Ohio | 43205-2664 | Recruiting |
| Pulse InfoFrame US Inc.- Site Number: 001001 | Philadelphia | Pennsylvania | 19104 | Recruiting |
| Dell Children's Medical Center- Site Number : 1001-2 | Austin | Texas | 78723 | Recruiting |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT06192576, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Oct 30, 2025 · Synced Sep 8, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT06192576 live on ClinicalTrials.gov.