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NCT06192576 · ClinicalTrials.gov registry record
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 clinical trial of Niemann-Pick Diseases and Acid Sphingomyelinase Deficiency, sponsored by Sanofi.
- Recruiting
- Registry status
- 10
- Enrollment target
- 5
- Study locations
NCT06192576: Recruiting study of Niemann-Pick Diseases and Acid Sphingomyelinase Deficiency, sponsored by Sanofi.
NCT06192576 is a study of Niemann-Pick Diseases and Acid Sphingomyelinase Deficiency that is actively recruiting participants, run by Sanofi. The registered enrollment target is 10 participants. The trial reports 5 study locations across 4 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT06192576, a study of Niemann-Pick Diseases and Acid Sphingomyelinase Deficiency, is actively recruiting participants, sponsored by Sanofi.
- RECRUITING
- Registry status
- 10 participants
- Enrollment target
- 5
- Study locations
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 |
What NCT06192576 shows while recruiting
NCT06192576 is an observational study that tracks outcomes without assigning an intervention. The registry caps enrollment at 10 participants, a relatively small participant target.
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.
NCT06192576 lists 5 locations in 4 states (Ohio, Illinois, Pennsylvania).
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.
What interventions are being tested in trial NCT06192576?
The interventions under investigation include: Olipudase alfa (DRUG).
Who is sponsoring clinical trial NCT06192576?
This trial is sponsored by Sanofi, which has 713 total clinical trials registered on ClinicalTrials.gov.
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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