Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
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: Completed Phase 2 study, sponsored by Genzyme, a Sanofi Company.
NCT02004691 is a Phase 2 study that has completed, run by Genzyme, a Sanofi Company. The registered enrollment target is 36 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (73% lower). According to ClinicalTrials.gov, the official US trial registry.
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.
Primary Outcome
Percent predicted Hb and Altitude-adjusted DLco was calculated as: 100\*Adjusted DLco/Predicted DLco in unit of mL CO/min/mmHg where, adjusted DLco = Observed DLco (in mL CO/min/mmHg) times Hemoglobin-adjusted factor times Altitude-adjustment factor.
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 |
What the finished NCT02004691 record still lists
NCT02004691 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 36 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (73% lower).
The record links to 0 conditions, and to 2 interventions - of which Olipudase alfa is the first listed.
NCT02004691 does not publish any study locations in the registry export this page uses.
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.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT02004691's enrollment target sits among peer trials
36 1496th of 2000 higher than 477 of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 2 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
Nationwide trials with similar profiles
Cross-condition peers matched on enrollment target and registry start date, not the same-condition list above.
Similar enrollment target
- NCT00929006 · 36 participants · Early Phase 1
Acute Progesterone Suppression of Wake vs. Sleep Luteinizing Hormone Pulse Frequency in Pubertal Girls With and Without Hyperandrogenism
- NCT01891318 · 36 participants · Phase 1
Neoadjuvant Radiosurgery for Resectable Brain Metastases: Phase I/II Study
- NCT02291848 · 36 participants · Phase 1
Tumor-Associated Antigen-Specific Cytotoxic T-Lymphocytes for Multiple Myeloma
- NCT02366819 · 36 participants · Phase 4
Genetic Analysis-Guided Irinotecan Hydrochloride Dosing of mFOLFIRINOX in Treating Patients With Locally Advanced Gastroesophageal or Stomach Cancer
Similar registry start date
- NCT05962346 · started 2026-12 · NA
Fetal Endoscopic Tracheal Occlusion for Congenital Diaphragmatic Hernia
- NCT07125183 · started 2026-12 · Phase 2
Study on Efficacy and Tolerability of Weekly Doxorubicin in Elderly Patients With Advanced or Metastatic Leiomyosarcoma
- NCT07292298 · started 2026-11 · Phase 2
Phase 2 Single-Arm Rectal Cancer Brachytherapy for Patients With Low-Lying Residual Adenocarcinoma After Total Neoadjuvant Therapy to Improve Organ Preservation Rates
- NCT04263285 · started 2026-10 · NA
Treatment of Depression Post-SCI