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

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ClinicalTrials.gov Identifier: NCT04877132
Expanded Access Status : Available
First Posted : May 7, 2021
Last Update Posted : May 7, 2021
Sponsor:
Information provided by (Responsible Party):
Sanofi

Study Description
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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.

Condition or disease Intervention/treatment
Sphingomyelin Lipidosis Drug: olipudase alfa (GZ402665)

Study Design
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Study Type : Expanded Access
Expanded Access Type : Intermediate-size Population
  See clinical trials of the intervention/treatment in this expanded access record.
Official Title: Compassionate Use Program for Olipudase Alfa Enzyme Replacement Therapy for Patients With Chronic Acid Sphingomyelinase Deficiency (ASMD)

Resource links provided by the National Library of Medicine


Interventions
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Intervention Details:
  • Drug: olipudase alfa (GZ402665)
    Patients will receive intravenous (IV) infusion of olipudase alfa
Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   3 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
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.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04877132


Contacts
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Contact: Trial Transparency email recommended: Contact-US@sanofi.com OR Toll free number for US & Canada 800-633-1610 ext option 6 Contact-Us@sanofi.com

Sponsors and Collaborators
Sanofi
Investigators
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Study Director: Clinical Sciences & Operations Sanofi
More Information
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Responsible Party: Sanofi
ClinicalTrials.gov Identifier: NCT04877132    
Other Study ID Numbers: RHASHC09706
First Posted: May 7, 2021    Key Record Dates
Last Update Posted: May 7, 2021
Last Verified: May 2021
Additional relevant MeSH terms:
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Niemann-Pick Disease, Type A
Niemann-Pick Diseases
Niemann-Pick Disease, Type C
Lipidoses
Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lipid Metabolism Disorders
Metabolic Diseases
Sphingolipidoses
 Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Histiocytosis, Non-Langerhans-Cell
Histiocytosis
Lymphatic Diseases
Lysosomal Storage Diseases