Compassionate Use Program for Olipudase Alfa Enzyme Replacement Therapy for Patients With Chronic Acid Sphingomyelinase Deficiency (ASMD)

Sphingomyelin Lipidosis Clinical Trial

Official title:
Compassionate Use Program for Olipudase Alfa Enzyme Replacement Therapy for Patients With Chronic Acid Sphingomyelinase Deficiency (ASMD)

Status Available

Clinical Trial Summary

The objective of this program is to provide access to enzyme replacement therapy (ERT) with olipudase alfa for certain patients with ASMD, a severe, life threatening disease, during the period between completion of the clinical trials necessary to support submission of a request for marketing authorization and receipt of a final response from the applicable regulatory authority in the country where this program is available.

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT04799522
Study type	Expanded Access
Source	Sanofi
Contact	Trial Transparency email recommended: Contact-US@sanofi.com OR T
Phone	800-633-1610
Email	Contact-Us@sanofi.com
Status	Available
Phase

View Details

See also
Status	Clinical Trial	Phase
Completed	`NCT04106544` - A Prospective and Retrospective Cohort Study in Patients With Chronic Forms of Acid Sphingomyelinase Deficiency (ASMD)	N/A
Completed	`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	Phase 1/Phase 2
Completed	`NCT02004704` - A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency	Phase 2
Completed	`NCT02004691` - Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency	Phase 2/Phase 3
Approved for marketing	`NCT04877132` - Compassionate Use Program for Olipudase Alfa Enzyme Replacement Therapy for Patients With Chronic Acid Sphingomyelinase Deficiency (ASMD)