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Your journey
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More info
You can access this
clinical trial
if you have
Human Acid Sphingomyelinase Deficiency
and you are
between 18 and 65
years old
This is an early phase trial to determine
the dosage and safety of the new treatment.
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The purpose

To evaluate the safety, tolerability, pharmacokinetic, and pharmacodynamic profile of rhASM in adult patients with Acid Sphingomyelinase Deficiency (ASMD) following repeated-dose administration.

Provided treatments

  • Drug: Recombinant human acid sphingomyelinase

Locations near you

Unfortunately, there are no recruiting locations near you. Please check the list with all locations below.
Tris trial is registered with FDA with number: NCT01722526. The sponsor of the trial is Genzyme, a Sanofi Company and it is looking for 5 volunteers for the current phase.
Official trial title:
An Open-label, Multicenter, Ascending Dose Study of the Tolerability and Safety of Recombinant Human Acid Sphingomyelinase (rhASM) in Patients With Acid Sphingomyelinase Deficiency (ASMD)