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

> **NCT02004691** · PHASE2,PHASE3 · COMPLETED · sponsor: **Genzyme, a Sanofi Company** · enrollment: 36 (actual)

## Conditions studied

- Sphingomyelin Lipidosis

## Interventions

- **DRUG:** placebo (saline)
- **DRUG:** Olipudase alfa

## Key facts

- **NCT ID:** NCT02004691
- **Lead sponsor:** Genzyme, a Sanofi Company
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE2,PHASE3
- **Study type:** INTERVENTIONAL
- **Status:** COMPLETED
- **Start date:** 2015-12-18
- **Primary completion:** 2021-03-15
- **Final completion:** 2023-10-19
- **Target enrollment:** 36 (ACTUAL)
- **Last updated:** 2024-11-07


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT02004691

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT02004691, "Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency". Retrieved via AI Analytics 2026-05-26 from https://api.ai-analytics.org/clinical/NCT02004691. Licensed CC0.

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*[Clinical trials dataset](/datasets/clinical-trials) · CC0 1.0*