# Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency

> **NCT02292654** · PHASE1,PHASE2 · COMPLETED · sponsor: **Genzyme, a Sanofi Company** · enrollment: 20 (actual)

## Conditions studied

- Sphingomyelin Lipidosis

## Interventions

- **DRUG:** Olipudase alfa

## Key facts

- **NCT ID:** NCT02292654
- **Lead sponsor:** Genzyme, a Sanofi Company
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE1,PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** COMPLETED
- **Start date:** 2015-05-01
- **Primary completion:** 2019-12-09
- **Final completion:** 2019-12-09
- **Target enrollment:** 20 (ACTUAL)
- **Last updated:** 2022-03-23


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT02292654

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT02292654, "Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency". Retrieved via AI Analytics 2026-07-26 from https://api.ai-analytics.org/clinical/NCT02292654. Licensed CC0.

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