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NCT02004691 · ClinicalTrials.gov registry record · Phase 2
Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency
A Phase 2 study, sponsored by Genzyme, a Sanofi Company.
- Completed
- Registry status
- Phase 2
- Development phase
- 36
- Enrollment target
NCT02004691 is a Phase 2 study that has completed, run by Genzyme, a Sanofi Company. The registered enrollment target is 36 participants.
The verdict
NCT02004691, a Phase 2 study, has completed, sponsored by Genzyme, a Sanofi Company.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 36 participants
- Enrollment target
Study 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.
Interventions
- DRUG Olipudase alfa
- DRUG placebo (saline)
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 36 participants |
| Start Date | 2015-12-18 |
| Est. Completion | 2023-10-19 |
| Phase | Phase 2 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT02004691
The ClinicalTrials.gov registry entry for NCT02004691 describes a study currently listed as completed, categorized as Phase 2. The registered enrollment target is 36 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Genzyme, a Sanofi Company, which has 196 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 2 interventions - of which Olipudase alfa is the first listed.
NCT02004691 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT02004691 about?
NCT02004691 is a clinical study titled "Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency". 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 change...
What is the current status of trial NCT02004691?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 36 participants. The study started on 2015-12-18. Estimated completion is 2023-10-19.
What interventions are being tested in trial NCT02004691?
The interventions under investigation include: Olipudase alfa (DRUG), placebo (saline) (DRUG).
Who is sponsoring clinical trial NCT02004691?
This trial is sponsored by Genzyme, a Sanofi Company, which has 196 total clinical trials registered on ClinicalTrials.gov.
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