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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


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

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)