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 Approved for marketing

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, that could not participate in the olipudase clinical trials. The program will provide access to olipudase alfa prior to registration and the availability of commercial product (including reimbursement where applicable) in the country of the patient.

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT04877132
Study type	Expanded Access
Source	Sanofi
Contact
Status	Approved for marketing
Phase

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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
Available	`NCT04799522` - Compassionate Use Program for Olipudase Alfa Enzyme Replacement Therapy for Patients With Chronic Acid Sphingomyelinase Deficiency (ASMD)