Back to resultsCompassionate Use Program for Olipudase Alfa Enzyme Replacement Therapy for Patients With Chronic Acid Sphingomyelinase Deficiency (ASMD)
Primary Purpose
Sphingomyelin Lipidosis
Status
Approved for marketing
Phase
Locations
Study Type
Expanded Access
Intervention
olipudase alfa (GZ402665)
Sponsored by
About this trial
This is an expanded access trial for Sphingomyelin Lipidosis
Eligibility Criteria
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 (β 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.
Sites / Locations
Outcomes
Primary Outcome Measures
Secondary Outcome Measures
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT04877132
Brief Title
Compassionate Use Program for Olipudase Alfa Enzyme Replacement Therapy for Patients With Chronic Acid Sphingomyelinase Deficiency (ASMD)
Official Title
Compassionate Use Program for Olipudase Alfa Enzyme Replacement Therapy for Patients With Chronic Acid Sphingomyelinase Deficiency (ASMD)
Study Type
Expanded Access
2. Study Status
Record Verification Date
September 2022
Overall Recruitment Status
Approved for marketing
Study Start Date
undefined (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
undefined (undefined)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Sanofi
4. Oversight
5. Study Description
Brief 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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Sphingomyelin Lipidosis
7. Study Design
8. Arms, Groups, and Interventions
Intervention Type
Drug
Intervention Name(s)
olipudase alfa (GZ402665)
Intervention Description
Patients will receive intravenous (IV) infusion of olipudase alfa
10. Eligibility
Sex
All
Minimum Age & Unit of Time
3 Years
Eligibility Criteria
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 (β 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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Clinical Sciences & Operations
Organizational Affiliation
Sanofi
Official's Role
Study Director
12. IPD Sharing Statement
Learn more about this trial
Compassionate Use Program for Olipudase Alfa Enzyme Replacement Therapy for Patients With Chronic Acid Sphingomyelinase Deficiency (ASMD)
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