A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency

Completed · Phase 2

Conditions studied: Sphingomyelin Lipidosis

In brief

The primary objective of this study was to obtain data regarding the safety of olipudase alfa in participants with acid sphingomyelinase deficiency (ASMD) who were exposed to long term treatment with olipudase alfa. The secondary objectives of this study were to obtain data regarding the efficacy of olipudase alfa and to characterize olipudase alfa pharmacodynamics (PD) and pharmacokinetics (PK) following long-term administration.

Key facts

Study ID

NCT02004704

Run by

Genzyme, a Sanofi Company

People needed

25

Starts

2013-12-04

Expected to finish

2023-09-06

Last updated by the study team

2024-07-03

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• The participant completed the treatment period of a previous study of olipudase alfa with an acceptable safety profile in the opinion of the investigator and sponsor.
• The participant and/or the participant's parent(s)/legal guardian(s) was willing and able to provide signed written informed consent.
• The participant who is female and of childbearing potential must have had a negative urine pregnancy test for beta human chorionic gonadotropin (β HCG).
• Female participants of childbearing potential and sexually mature male participants must have been willing to practice true abstinence in line with their preferred and usual lifestyle or use 2 acceptable effective methods of contraception up to 15 days following their last dose of study drug.

You may not qualify if…

• The participant had any new condition or worsening of an existing condition which in the opinion of the investigator would make the participant unsuitable for enrollment, or could interfere with the participation or completion the study.
• The participant, in the opinion of the investigator, was unable to adhere to the requirements of the study.
• The participant was unwilling or unable to abstain from the use of alcohol for 1 day prior to and 3 days after each olipudase alfa infusion for the duration of the treatment period.
• The participant was unwilling or unable to avoid, for 10 days before and 3 days after liver biopsies, medications or herbal supplements that are potentially hepatotoxic (only participants who previously participated in the DFI13412 study).
• The participant required medication(s) that may decrease olipudase alfa activity (eg, fluoxetine, chlorpromazine; tricyclic antidepressants [eg, imipramine, desipramine]).
• The above information was not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.

Where it is running

• Investigational Site Number 840001 — New York, New York, United States
• Investigational Site Number 056001 — Leuven, Belgium
• Investigational Site Number 076001 — Porto Alegre, Brazil
• Investigational Site Number 250002 — Bron, France
• Investigational Site Number 276002 — Hochheim am Main, Germany
• Investigational Site Number 380002 — Sassari, Italy
• Investigational Site Number 380001 — Udine, Italy
• Investigational Site Number 826001 — London, United Kingdom
• Investigational Site Number 826002 — Manchester, United Kingdom

Full record on ClinicalTrials.gov

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