New study tracks safety of enzyme therapy in babies with rare genetic disease

NCT ID NCT06192576

Recruiting now Knowledge-focused Sponsor: Sanofi Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

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.

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.

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Conditions

The condition(s) this trial relates to.

acid sphingomyelinase deficiency Niemann-Pick disease Niemann-Pick disease type A Niemann-Pick disease type B

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

ACID SPHINGOMYELINASE DEFICIENCY NIEMANN-PICK DISEASES

Contacts and locations

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Study contacts

Contact

Email: •••••@•••••

Locations

Ann and Robert H Lurie Children's Hospital of Chicago- Site Number: 001002
RECRUITING

Chicago, Illinois, 60611, United States
Children's Hospital Medical Center- Site Number: 001003
RECRUITING

Cincinnati, Ohio, 45229-3039, United States
Dell Children's Medical Center- Site Number : 1001-2
RECRUITING

Austin, Texas, 78723, United States
Nationwide Children's Hospital - PIN- Site Number : 1001-1
RECRUITING

Columbus, Ohio, 43205-2664, United States
Pulse InfoFrame US Inc.- Site Number: 001001
RECRUITING

Philadelphia, Pennsylvania, 19104, United States

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