New study tracks safety of enzyme therapy in babies with rare genetic disease
NCT ID NCT06192576
First seen Nov 01, 2025 · Last updated Jun 22, 2026 · Updated 33 times
Summary
This study follows up to 10 children under 2 years old with acid sphingomyelinase deficiency (ASMD) who are already receiving olipudase alfa (Xenpozyme®) as part of their routine care. Researchers will monitor side effects and immune responses over 1 to 3 years. No new treatment is given; the study simply observes what happens in real-world medical practice.
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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.
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
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
olipudase alfa (Xenpozyme®)
What this could lead to
If successful, this study could confirm that olipudase alfa is safe and well-tolerated in very young children with ASMD, supporting its continued use in this age group.
What could go wrong
This is a small observational study (10 participants) with no control group, so results may not apply to all patients. It only monitors safety, not effectiveness, and side effects like allergic reactions are possible.
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.