Tiny study tracks safety of enzyme therapy for rare disease
NCT ID NCT06949358
First seen Apr 14, 2026 · Last updated Jun 20, 2026 · Updated 14 times
Summary
This study followed 3 people with acid sphingomyelinase deficiency (ASMD) who had already completed earlier trials of olipudase alfa. They received the enzyme replacement therapy every 2 weeks to see if it remained safe and tolerable over the long term. The study ended when the drug became reimbursed in France or after 5 years.
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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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Locations
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Investigational Site Number : 2500001
Paris, 75020, France
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Investigational Site Number : 2500002
Bron, 69500, France
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 given by infusion every 2 weeks)
What this could lead to
If successful, this could confirm that long-term use of olipudase alfa is safe for managing acid sphingomyelinase deficiency.
What could go wrong
This is a very small study (only 3 patients) and was designed mainly to check safety, not to prove the drug works. It also only included patients who had already completed earlier studies, so results may not apply to everyone.
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.