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

Genzyme

Status and phase: Completed; Phase 2

Conditions: Sphingomyelin Lipidosis

Treatments: Drug: GZ402665

Study type: Interventional

Funder types: Industry

Identifiers

NCT02004704

LTS13632

2013-000051-40 (EudraCT Number)

Details and patient eligibility

About

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.

Full description

LTS13632 is a multicenter, nonrandomized, open-label, long-term extension study of participants with ASMD who have previously participated in a study of olipudase alfa. (DFI13803 for pediatric participants and DFI13412 for adult participants).

The maximum study duration per participant was 9 years or until olipudase alfa becomes commercially accessible. Notwithstanding the above, every pediatric participant were treated in LTS13632 study for at least 3 years to comply with the requirements agreed in the olipudase alfa Pediatric Investigational Plan.

Enrollment: 25 patients

Sex: All

Volunteers: No Healthy Volunteers

Inclusion criteria

• 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.

Exclusion criteria

• 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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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25 participants in 1 patient group

GZ402665

Experimental group

Description:

GZ402665 administered intravenously once every 2 weeks at the dose each participant was receiving at the end of their previous olipudase alfa study, for 9 years or until olipudase alfa becomes commercially accessible, whichever comes first, unless the participant decides to enter another olipudase alfa clinical trial within the 9-year period prior to when olipudase alfa is commercially accessible.

Treatment:

Drug: GZ402665