Tiny study tracks safety of enzyme drug for rare disease

NCT ID NCT06949358

Completed Disease control Sponsor: Sanofi Source: ClinicalTrials.gov ↗

First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study followed 3 people with acid sphingomyelinase deficiency (ASMD) who had already completed earlier trials of olipudase alfa. The goal was to monitor safety and side effects while they continued receiving the enzyme replacement therapy every two weeks until the drug was officially available in France. The study focused on adverse events and serious adverse events.

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 (enzyme replacement therapy)
What this could lead to: If successful, this could confirm that long-term use of olipudase alfa is safe and tolerable for people with acid sphingomyelinase deficiency.
What could go wrong: This is a very small study (3 people) and only looked at safety, not how well the drug works. It may not apply to all patients.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for ACID SPHINGOMYELINASE DEFICIENCY are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

Conditions

The condition(s) this trial relates to.

acid sphingomyelinase deficiency Niemann-Pick disease

As listed by the trial registrant

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

ACID SPHINGOMYELINASE DEFICIENCY

Contacts and locations

Show contact details

Locations

Investigational Site Number : 2500001

Paris, 75020, France
Investigational Site Number : 2500002

Bron, 69500, France

More trials for these conditions

Other studies related to the condition(s) this trial covers.