Trials Sponsors Conditions Drugs Pricing

Conditions

Niemann-Pick disease Acid Sphingomyelinase Deficiency

Outcome Measures

Primary Outcomes (1)

Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
An AE was any untoward medical occurrence in participant or clinical study participant temporally associated with the use of study drug, whether or not considered related to the study drug. An SAE was any AE, that at any dose: resulted in death, was life-threatening, required inpatient hospitalization/prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect or was a medically important event.
From the signature of informed consent (Day 0) up to end of safety follow-up per participant, up to approximately 40 months

Study Arms (1)

GZ402665

EXPERIMENTAL

Olipudase alfa administered intravenously every 2 weeks

Drug: Olipudase alfa

Interventions

Olipudase alfaDrug

Pharmaceutical form:Powder for concentrate for solution for infusion-Route of administration:intravenous infusion

Also known as: GZ402665

GZ402665

Xenpozyme (olipudase alfa,GZ402665)Type not reported

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+)

You may qualify if:

The participant has completed Study DFI12712 (ASCEND) or LTS13632 in France
The participant must provide signed, informed consent prior to performing any study-related procedures.

Sponsors & Collaborators

Sanofi Winthrop Industrie
Industrylead
Sanofi
Industrylead

Study Sites (2)

Investigational Site Number : 2500002

Bron, 69500, France

Investigational Site Number : 2500001

Paris, 75020, 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

Results Point of Contact

Title: Trial Transparency Team
Organization: Sanofi aventis recherche & développement

Publication Agreements

PI is Sponsor Employee: No
Restriction Type: OTHER
Restrictive Agreement: Yes

Enrollment

3 participants (actual enrollment reported by the registry).

Study Design

Study Type: interventional
Phase: phase 2
Allocation: NA
Masking: NONE
Purpose: TREATMENT
Intervention Model: SINGLE GROUP
Sponsor Type: INDUSTRY
Responsible Party: SPONSOR

Study Record Dates

First Submitted

April 22, 2025

First Posted

April 29, 2025

Study Start

November 18, 2021

Actual (reported)

Primary Completion

A Study to Evaluate Safety and Tolerability of Olipudase Alfa in Pediatric and Adult Participants With Acid Sphingomyelinase Deficiency (ASMD) Who Completed the DFI12712 or the LTS13632 Study in France

Brief Summary

Registry Overview

Trial Relationships

Study Timeline

Study Start

Primary Completion

Study Completion

First Submitted

First Posted

Results Posted

Conditions

Outcome Measures

Primary Outcomes (1)

Study Arms (1)

GZ402665

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (2)

Related Links

MeSH Terms

Conditions

Interventions

Condition Hierarchy (Ancestors)

Results Point of Contact

Publication Agreements

Enrollment

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

Source records

Documents

Locations

Brief Summary

Registry Overview

Trial Relationships

Related Scientific Literature

Study Timeline

Study Start

Primary Completion

Study Completion

First Submitted

First Posted

Results Posted

Conditions

Outcome Measures

Primary Outcomes (1)

Study Arms (1)

GZ402665

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (2)

Related Links

MeSH Terms

Conditions

Interventions

Condition Hierarchy (Ancestors)

Results Point of Contact

Publication Agreements

Enrollment

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

Source records

Documents

Locations