ClinicalTrials.gov record

Completed Phase 2Phase 3 Interventional Accepts healthy volunteers Results available

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

ClinicalTrials.gov ID: NCT02004691

Public ClinicalTrials.gov record NCT02004691. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.

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ClinicalTrials.gov public records Last synced Jul 21, 2026, 4:43 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Official title

A Phase 2/3, Multicenter, Randomized, Double-blinded, Placebo-controlled, Repeat-dose Study to Evaluate the Efficacy, Safety, Pharmacodynamics, and Pharmacokinetics of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency

Study identification

NCT ID: NCT02004691
Recruitment status: Completed
Study type: Interventional
Phase: Phase 2, Phase 3
Lead sponsor: Genzyme, a Sanofi Company; Industry
Enrollment: 36 participants

Conditions and interventions

Conditions

Sphingomyelin Lipidosis

Interventions

placebo (saline) Drug
Olipudase alfa Drug

Drug

Eligibility (public fields only)

Age range: 18 Years and older
Sex: All
Healthy volunteers: Accepts healthy volunteers

This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.

Study timeline

Start date: Dec 17, 2015
Primary completion: Mar 14, 2021
Completion: Oct 18, 2023
Last update posted: Nov 6, 2024

2015 – 2023

United States locations

U.S. sites: 3
U.S. states: 3
U.S. cities: 3

Facility	City	State	ZIP	Site status
UCSF Medical Center Site Number : 840005	San Francisco	California	94143	—
Emory University Site Number : 840003	Decatur	Georgia	30033	—
Montefiore Medical Center Site Number : 840006	The Bronx	New York	10467	—

Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.

Non-U.S. locations

This page focuses on the U.S. directory. The official record also lists 21 non-U.S. sites.

About this trial record page

What this page shows: Public field values for ClinicalTrials.gov record NCT02004691, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
What this page does not do: No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
Where the data comes from: Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
Last refresh: Last update posted Nov 6, 2024 · Synced Jul 21, 2026

Open the official record

The complete protocol, eligibility criteria, and contact information for NCT02004691 live on ClinicalTrials.gov.

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