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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 · View on ClinicalTrials.gov ↗
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 Studied
Interventions
- DRUG Olipudase alfa
Study Locations (5)
Ohio
- Children's Hospital Medical Center- Site Number: 001003 — Cincinnati
- Nationwide Children's Hospital - PIN- Site Number : 1001-1 — Columbus
Illinois
- Ann and Robert H Lurie Children's Hospital of Chicago- Site Number: 001002 — Chicago
Pennsylvania
- Pulse InfoFrame US Inc.- Site Number: 001001 — Philadelphia
Texas
- Dell Children's Medical Center- Site Number : 1001-2 — Austin
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 10 participants |
| Start Date | 2024-04-16 |
| Est. Completion | 2029-01-15 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT06192576
The ClinicalTrials.gov registry entry for NCT06192576 describes a study currently listed as recruiting. It is categorized as an unspecified phase, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 10 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Sanofi, which has 275 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 2 conditions, with Niemann-Pick Diseases appearing as the primary indexed condition, and to 1 intervention — of which Olipudase alfa is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT06192576 reports 5 study locations spanning 4 distinct geographic areas — top geographies include Ohio, Illinois, Pennsylvania. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT06192576 about?
NCT06192576 is a clinical study titled "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)". 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 ...
What is the current status of trial NCT06192576?
This trial is currently recruiting. The enrollment target is 10 participants. The study started on 2024-04-16. Estimated completion is 2029-01-15.
What conditions does trial NCT06192576 study?
This clinical trial studies the following conditions: Niemann-Pick Diseases, Acid Sphingomyelinase Deficiency. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT06192576?
The interventions under investigation include: Olipudase alfa (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT06192576?
This trial is sponsored by Sanofi, which has 275 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT06192576 being conducted?
This trial has 5 study locations across Illinois, Ohio, Pennsylvania, Texas. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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