Tracking a lifesaving Treatment's safety in the tiniest patients
NCT ID NCT06192576
Summary
This study aims to monitor the long-term safety and how the body's immune system responds to olipudase alfa, an enzyme replacement therapy, in children under 2 years old with acid sphingomyelinase deficiency (ASMD), a rare and serious genetic disorder. It will follow up to 10 children for up to 5 years during their routine clinical care to watch for side effects and immune reactions. The goal is to gather more information to help manage this lifelong condition safely in very young patients.
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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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