Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Primary Objective:
To describe the lung, spleen and liver outcomes of olipudase alfa
Secondary Objectives:
Approximate duration of enrollment: 30 months
Total study duration: approximately 30 months
This is a national, multicenter observational retrospective and prospective cohort data collection study. Retrospective is defined as collection of data from all patients, including deceased patients, who were already on early access olipudase alfa in France before the start of this study.
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Cohort 1 | Patients with a confirmed diagnosis of ASMD under early access to olipudase alfa in France |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Olipudase alfa | Drug | GZ402665 |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change in pulmonary function diffusion capacity of lung for carbon monoxide (DLco) | From baseline to 24 months | |
| Change in spleen size | From baseline to 24 months | |
| Change in liver size | From baseline to 24 months |
| Measure | Description | Time Frame |
|---|---|---|
| Baseline patient characteristics | Demographic and baseline data [age, gender, weight, phenotype and genotype of ASMD, acid sphingomyelinase activity in peripheral leukocytes, lymphocytes, or cultured fibroblasts, age at diagnosis, age at first symptom onset, history of splenectomy (month/year), habits (i.e., smoking, alcoholism), known metabolic conditions or diseases (obesity, diabetes, familial dyslipidemias), known respiratory diseases; known hepatic diseases; others)] |
Not provided
Inclusion Criteria:
Exclusion Criteria:
The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial.
Not provided
Not provided
Not provided
Not provided
All patients with a confirmed diagnosis of ASMD under early access to olipudase alfa in France (with a written informed consent)
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Clinical Sciences & Operations | Sanofi | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Investigational site in France | France | France |
Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
Not provided
Not provided
Not provided
Not provided
Not provided
| ID | Term |
|---|---|
| D009542 | Niemann-Pick Diseases |
| ID | Term |
|---|---|
| D013106 | Sphingolipidoses |
| D020140 | Lysosomal Storage Diseases, Nervous System |
| D020739 | Brain Diseases, Metabolic, Inborn |
| D001928 | Brain Diseases, Metabolic |
Not provided
Not provided
| ID | Term |
|---|---|
| C000608782 | olipudase alfa |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| At baseline |
| Condition of olipudase alfa use | Conditions of olipudase alfa use (time to reach maximum dose [3 mg/kg] or the maximum tolerated dose for the patient, center profile, treater specialty, need of a premedication before the infusion [if yes, precise], treatment duration [start and end dates], treatment discontinuation [Yes/No] and reason of treatment discontinuation if any) | From baseline up to 3 years |
| Safety: AE | Number of Participants with Adverse events (AE) including infusion-associated reactions | From baseline up to 3 years |
| Safety: immunogenicity | Immune response assessments (antibodies anti-olipudase alfa IgG) | From baseline up to 3 years |
| Complementary effectiveness: change in pulmonary function DLco | From baseline to 12 months and 36 months |
| Complementary effectiveness: change in spleen size | From baseline to 12 months and 36 months |
| Complementary effectiveness: change in liver size | FFrom baseline to 12 months and 36 months |
| Change in interstitial pulmonary infiltration based on lung imaging (thoracic CT-scan) | From baseline to 12 months and 24 months |
| Change in platelet count | From baseline at 3, 6, 9, 12, 24 months and every year up to 3 years |
| Change in biomarkers (chitotriosidase and lysosphingomyelin) plasma levels | From baseline at 3, 6, 9, 12 months and every year up to 3 years |
| Change in liver function | Alanine transaminase (ALT), aspartate aminotransferase (AST), alkaline phosphatase (ALP), total and direct bilirubin | From baseline at 3, 6, 9, 12, 24 months and every year up to 3 years |
| Change in lipid profile | total cholesterol, high density lipoprotein (HDL) and low density lipoprotein (LDL) cholesterol | From baseline at 3, 6, 9, 12, 24 months and every year up to 3 years |
| Change in growth curve for pediatric patient | From baseline at 6, 12, 24 months and every year up to 3 years |
| Change in weight | From baseline to 12 months and 36 months |
| Number of Participants with Evolution of Comorbidities | Number of participants with evolution of comorbidities will be assessed by grade, attenuation or disappearance/absence | From baseline to 12 months, 24 months and 36 months |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D015616 | Histiocytosis, Non-Langerhans-Cell |
| D015614 | Histiocytosis |
| D008206 | Lymphatic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D008064 | Lipidoses |
| D008052 | Lipid Metabolism, Inborn Errors |
| D016464 | Lysosomal Storage Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D052439 | Lipid Metabolism Disorders |