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Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency

NCT02004691 · Status: COMPLETED · Phase: PHASE2/PHASE3 · Type: INTERVENTIONAL · Enrollment: 36

Last updated 2024-11-07

Study results available
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Summary

Primary Objective:

The primary objective of this phase 2/3 study was to evaluate the efficacy of olipudase alfa (recombinant human acid sphingomyelinase) administered intravenously once every 2 weeks for 52 weeks in adult participants with acid sphingomyelinase deficiency (ASMD) by assessing changes in: 1) spleen volume as measured by abdominal magnetic resonance imaging (MRI) (and, for the United States \[US\] only, in association with participant perception related to spleen volume as measured by splenomegaly-related score \[SRS\]); and 2) infiltrative lung disease as measured by the pulmonary function test, diffusing capacity of the lung for carbon monoxide (DLCO).

Secondary Objectives:

* To confirm the safety of olipudase alfa administered intravenously once every 2 weeks for 52 weeks.
* To characterize the effect of olipudase alfa on the participant perception related to spleen volume as measured by the SRS after 52 weeks of study drug administration. (For the US, the effect of olipudase alfa on the SRS is part of the primary objective).
* To characterize the effect of olipudase alfa after 52 weeks of study drug administration on the following outcome measures assessed sequentially:

* The effect of olipudase alfa on liver volume;
* The effect of olipudase alfa on platelet count;
* The effect of olipudase alfa on fatigue;
* The effect of olipudase alfa on pain;
* The effect of olipudase alfa on dyspnea.

Conditions

  • Sphingomyelin Lipidosis

Interventions

DRUG

placebo (saline)

Pharmaceutical form: solution administered once every two weeks during the 52 weeks of the primary analysis period for participants randomized to placebo. Route of administration: intravenous infusion

DRUG

Olipudase alfa

Pharmaceutical form: Powder for concentrate for solution for infusion administered once every two weeks during the 52 weeks of the primary analysis period for participants randomized to olipudase alfa, and during the extension treatment period for all participants. Route of administration: intravenous infusion

Sponsors & Collaborators

  • Genzyme, a Sanofi Company

    lead INDUSTRY

Principal Investigators

  • Clinical Sciences & Operations · Sanofi

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
Yes

Timeline & Regulatory

Start
2015-12-18
Primary Completion
2021-03-15
Completion
2023-10-19
FDA Drug
Yes

Countries

  • United States
  • Argentina
  • Australia
  • Belgium
  • Brazil
  • Bulgaria
  • Chile
  • France
  • Germany
  • Italy
  • Japan
  • Netherlands
  • Portugal
  • Spain
  • Tunisia
  • Turkey (Türkiye)
  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02004691 on ClinicalTrials.gov