Studies
| Study First Submitted Date | 2021-05-03 |
| Study First Posted Date | 2021-05-07 |
| Last Update Posted Date | 2022-09-19 |
| Verification Month Year | September 2022 |
| Verification Date | 2022-09-30 |
| Last Update Posted Date | 2022-09-19 |
Conditions
| Sequence: | 52418873 |
| Name | Sphingomyelin Lipidosis |
| Downcase Name | sphingomyelin lipidosis |
Id Information
| Sequence: | 40333963 |
| Id Source | org_study_id |
| Id Value | RHASHC09706 |
Interventions
| Sequence: | 52727346 |
| Intervention Type | Drug |
| Name | olipudase alfa (GZ402665) |
| Description | Patients will receive intravenous (IV) infusion of olipudase alfa |
Browse Conditions
| Sequence: | 194427072 | Sequence: | 194427073 | Sequence: | 194427074 | Sequence: | 194427075 | Sequence: | 194427076 | Sequence: | 194427077 | Sequence: | 194427078 | Sequence: | 194427079 | Sequence: | 194427080 | Sequence: | 194427081 | Sequence: | 194427082 | Sequence: | 194427083 | Sequence: | 194427084 | Sequence: | 194427085 | Sequence: | 194427086 | Sequence: | 194427087 | Sequence: | 194427088 | Sequence: | 194427089 | Sequence: | 194427090 | Sequence: | 194427091 |
| Mesh Term | Niemann-Pick Disease, Type A | Mesh Term | Niemann-Pick Diseases | Mesh Term | Niemann-Pick Disease, Type C | Mesh Term | Lipidoses | Mesh Term | Sphingolipidoses | Mesh Term | Lysosomal Storage Diseases, Nervous System | Mesh Term | Brain Diseases, Metabolic, Inborn | Mesh Term | Brain Diseases, Metabolic | Mesh Term | Brain Diseases | Mesh Term | Central Nervous System Diseases | Mesh Term | Nervous System Diseases | Mesh Term | Histiocytosis, Non-Langerhans-Cell | Mesh Term | Histiocytosis | Mesh Term | Lymphatic Diseases | Mesh Term | Metabolism, Inborn Errors | Mesh Term | Genetic Diseases, Inborn | Mesh Term | Lipid Metabolism, Inborn Errors | Mesh Term | Lysosomal Storage Diseases | Mesh Term | Metabolic Diseases | Mesh Term | Lipid Metabolism Disorders |
| Downcase Mesh Term | niemann-pick disease, type a | Downcase Mesh Term | niemann-pick diseases | Downcase Mesh Term | niemann-pick disease, type c | Downcase Mesh Term | lipidoses | Downcase Mesh Term | sphingolipidoses | Downcase Mesh Term | lysosomal storage diseases, nervous system | Downcase Mesh Term | brain diseases, metabolic, inborn | Downcase Mesh Term | brain diseases, metabolic | Downcase Mesh Term | brain diseases | Downcase Mesh Term | central nervous system diseases | Downcase Mesh Term | nervous system diseases | Downcase Mesh Term | histiocytosis, non-langerhans-cell | Downcase Mesh Term | histiocytosis | Downcase Mesh Term | lymphatic diseases | Downcase Mesh Term | metabolism, inborn errors | Downcase Mesh Term | genetic diseases, inborn | Downcase Mesh Term | lipid metabolism, inborn errors | Downcase Mesh Term | lysosomal storage diseases | Downcase Mesh Term | metabolic diseases | Downcase Mesh Term | lipid metabolism disorders |
| Mesh Type | mesh-list | Mesh Type | mesh-list | Mesh Type | mesh-list | Mesh Type | mesh-list | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor |
Sponsors
| Sequence: | 48549067 |
| Agency Class | INDUSTRY |
| Lead Or Collaborator | lead |
| Name | Sanofi |
Overall Officials
| Sequence: | 29413771 |
| Role | Study Director |
| Name | Clinical Sciences & Operations |
| Affiliation | Sanofi |
Eligibilities
| Sequence: | 30907612 |
| Gender | All |
| Minimum Age | 3 Years |
| Maximum Age | N/A |
| 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. |
| Adult | True |
| Child | True |
| Older Adult | True |
Calculated Values
| Sequence: | 254166610 |
| Registered In Calendar Year | 2021 |
| Were Results Reported | False |
| Has Single Facility | False |
| Minimum Age Num | 3 |
| Minimum Age Unit | Years |
Responsible Parties
| Sequence: | 29019979 |
| Responsible Party Type | Sponsor |