Trials Sponsors Conditions Drugs Pricing

Conditions

Acid Sphingomyelinase Deficiency (ASMD)

Outcome Measures

Primary Outcomes (3)

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

Secondary Outcomes (15)

Baseline patient characteristics
At baseline
Condition of olipudase alfa use
From baseline up to 3 years
Safety: AE
From baseline up to 3 years
Safety: immunogenicity
From baseline up to 3 years
Complementary effectiveness: change in pulmonary function DLco
From baseline to 12 months and 36 months
+10 more secondary outcomes

Study Arms (1)

Cohort 1

Patients with a confirmed diagnosis of ASMD under early access to olipudase alfa in France

Drug: Olipudase alfa

Interventions

Olipudase alfaDrug

GZ402665

Cohort 1

Registry-reported names are shown in full. Names without a link have not yet been matched to a Syfrah profile.

Eligibility Criteria

Sexall

Healthy VolunteersNo

Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

Sampling MethodNon-Probability Sample

Study Population

All patients with a confirmed diagnosis of ASMD under early access to olipudase alfa in France (with a written informed consent)

You may qualify if:

The patient, or the patient's parent(s)/guardian(s), has signed written informed consent.

Sponsors & Collaborators

Sanofi
Industrylead

Study Sites (1)

Investigational site in France

France, France

MeSH Terms

Conditions

Niemann-Pick Diseases

Interventions

olipudase alfa

Condition Hierarchy (Ancestors)

SphingolipidosesLysosomal Storage Diseases, Nervous SystemBrain Diseases, Metabolic, InbornBrain Diseases, MetabolicBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesHistiocytosis, Non-Langerhans-CellHistiocytosisLymphatic DiseasesHemic and Lymphatic DiseasesMetabolism, Inborn Errors

Study Officials

Clinical Sciences & Operations
Sanofi
STUDY DIRECTOR

Enrollment

40 participants (actual enrollment reported by the registry).

Study Design

Study Type: observational
Observational Model: COHORT
Time Perspective: PROSPECTIVE
Sponsor Type: INDUSTRY
Responsible Party: SPONSOR

Study Record Dates

First Submitted

April 28, 2022

First Posted

May 3, 2022

Study Start

June 10, 2022

Actual (reported)

Data Analysis of Adult and Pediatric Participants With Acid Sphingomyelinase Deficiency (ASMD) on Early Access to Olipudase Alfa in France

Brief Summary

Registry Overview

Trial Relationships

Study Timeline

First Submitted

First Posted

Study Start

Primary Completion

Study Completion

Conditions

Outcome Measures

Primary Outcomes (3)

Secondary Outcomes (15)

Study Arms (1)

Cohort 1

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (1)

MeSH Terms

Conditions

Interventions

Condition Hierarchy (Ancestors)

Study Officials

Enrollment

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

Source record

Locations

Brief Summary

Registry Overview

Trial Relationships

Related Scientific Literature

Study Timeline

First Submitted

First Posted

Study Start

Primary Completion

Study Completion

Conditions

Outcome Measures

Primary Outcomes (3)

Secondary Outcomes (15)

Study Arms (1)

Cohort 1

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (1)

MeSH Terms

Conditions

Interventions

Condition Hierarchy (Ancestors)

Study Officials

Enrollment

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

Source record

Locations