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Clinical Trial Details — Status: Approved for marketing

Administrative data

NCT number NCT04877132
Other study ID # RHASHC09706
Secondary ID
Status Approved for marketing
Phase
First received
Last updated

Study information

Verified date September 2022
Source Sanofi
Contact n/a
Is FDA regulated No
Health authority
Study type Expanded Access

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.


Recruitment information / eligibility

Status Approved for marketing
Enrollment 0
Est. completion date
Est. primary completion date
Accepts healthy volunteers
Gender All
Age group 3 Years and older
Eligibility Inclusion Criteria: All patients (adult and pediatric) - Unsolicited request for the patient from a site with at least 3 years of experience in administration and safety management of ERT. - Written informed consent signed by the patient or the patient's parent(s)/guardian(s), where applicable. - Documented deficiency of acid sphingomyelinase in peripheral leukocytes, lymphocytes, or cultured fibroblasts. Adult patients - Age = 18 years. - Clinically documented advanced disease evidenced by defined thresholds for lung, spleen, liver, and hematologic parameters. Pediatric patients - Age >3 years and <18 years or clinical diagnosis consistent with ASMD Type A/B or Type B. Exclusion Criteria: All patients (adult and pediatric) - Active serious intercurrent illness which will preclude enrollment, significant liver disease with etiology other than ASMD, Malignancy with poor prognosis, serious medical or psychiatric condition that may preclude participation, or circumstances that may interfere with compliance in this compassionate use program, requirement for recurrent dose adjustment of anticoagulation treatment over the last 6 months. - Pregnancy or breastfeeding. - For female patients of childbearing potential, a positive serum pregnancy (ß human chorionic gonadotropin [HCG]) test result. - For female patients of childbearing potential and sexually active male patients, unwillingness to abstain from heterosexual intercourse in accordance with their preferred and usual lifestyle, or to use 2 acceptable, effective contraceptive methods, while participating in this program and for 15 days after the last infusion of olipudase alfa. - For pediatric patients, clinical diagnosis, or suspicion of infantile onset ASMD. Genotype compatible with ASMD type A.

Study Design


Intervention

Drug:
olipudase alfa (GZ402665)
Patients will receive intravenous (IV) infusion of olipudase alfa

Locations

Country Name City State
n/a

Sponsors (1)

Lead Sponsor Collaborator
Sanofi
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)