New study tracks safety of enzyme therapy in babies with rare disease
NCT ID NCT06192576
First seen Nov 01, 2025 · Last updated May 01, 2026 · Updated 28 times
Summary
This study follows up to 10 children under 2 years old with acid sphingomyelinase deficiency (ASMD) who are receiving olipudase alfa as part of their routine care. Researchers will monitor side effects and the body's immune response to the drug over 1 to 3 years. The goal is to gather more safety data, not to test a new treatment.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Ann and Robert H Lurie Children's Hospital of Chicago- Site Number: 001002
RECRUITINGChicago, Illinois, 60611, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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Children's Hospital Medical Center- Site Number: 001003
RECRUITINGCincinnati, Ohio, 45229-3039, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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Dell Children's Medical Center- Site Number : 1001-2
RECRUITINGAustin, Texas, 78723, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Nationwide Children's Hospital - PIN- Site Number : 1001-1
RECRUITINGColumbus, Ohio, 43205-2664, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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Pulse InfoFrame US Inc.- Site Number: 001001
RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
Conditions
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