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

PHASE2/PHASE3CompletedINTERVENTIONAL
Enrollment

36

Participants

Timeline

Start Date

December 18, 2015

Primary Completion Date

March 15, 2021

Study Completion Date

October 19, 2023

Conditions
Sphingomyelin Lipidosis
Interventions
DRUG

placebo (saline)

"Pharmaceutical form: solution administered once every two weeks during the 52 weeks of the primary analysis period for participants randomized to placebo.~Route of administration: intravenous infusion"

DRUG

Olipudase alfa

"Pharmaceutical form: Powder for concentrate for solution for infusion administered once every two weeks during the 52 weeks of the primary analysis period for participants randomized to olipudase alfa, and during the extension treatment period for all participants.~Route of administration: intravenous infusion"

Trial Locations (24)

1007

Investigational Site Number : 788001, Tunis

1431

Investigational Site Number : 100001, Sofia

2145

Investigational Site Number : 036001, Westmead

3000

Investigational Site Number : 056001, Leuven

10467

Montefiore Medical Center Site Number : 840006, The Bronx

28034

Investigational Site Number : 724001, Madrid

30033

Emory University Site Number : 840003, Decatur

33100

Investigational Site Number : 380001, Udine

34520

Investigational Site Number : 792004, Istanbul

34722

Investigational Site Number : 792003, Istanbul

35040

Investigational Site Number : 792001, Izmir

55131

Investigational Site Number : 276001, Mainz

75020

Investigational Site Number : 250001, Paris

80131

Investigational Site Number : 380002, Napoli

94143

UCSF Medical Center Site Number : 840005, San Francisco

X5004FHP

Investigational Site Number : 032001, Córdoba

90035 003

Hospital De Clinicas De Porto Alegre Site Number : 076001, Porto Alegre

753-0204

Investigational Site Number : 152001, Santiago

960-1295

Investigational Site Number : 392001, Fukushima

1105 AZ

Investigational Site Number : 528001, Amsterdam

4099-001

Investigational Site Number : 620002, Porto

06500

Investigational Site Number : 792002, Ankara

WC1N 3JZ

Investigational Site Number : 826001, London

B15 2GW

Investigational Site Number : 826002, Birmingham

All Listed Sponsors
lead

Genzyme, a Sanofi Company

INDUSTRY

NCT02004691 - Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency | Biotech Hunter | Biotech Hunter