Trials Sponsors Conditions Drugs Pricing

Conditions

Gaucher's Disease Type III

Outcome Measures

Primary Outcomes (2)

Change in Scale for Assessment and Rating of Ataxia (SARA) modified total score
From baseline to Week 52
Change in Repeatable Battery for the Assessment of Neuropsychological Status (RBANS) total scale index score
From baseline to Week 52

Secondary Outcomes (9)

Percent change in spleen volume
From baseline to Week 52
Percent change in liver volume
From baseline to Week 52
Change in hemoglobin level
From baseline to Week 52
Percent change in platelet count
From baseline to Week 52
Percent change in CSF GL-1 and lyso-GL-1 levels
From baseline to Week 52
+4 more secondary outcomes

Study Arms (2)

Venglustat

EXPERIMENTAL

Venglustat

Drug: Venglustat

Cerezyme

ACTIVE COMPARATOR

Cerezyme

Drug: imiglucerase

Interventions

VenglustatDrug

tablet; oral

Venglustat

imigluceraseDrug

sterile lyophilized product; intravenous

Also known as: Cerezyme®

Cerezyme

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

Eligibility Criteria

Age12 Years+

Sexall

Healthy VolunteersNo

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

You may qualify if:

The participant has received ERT (Cerezyme or other ERT; as deemed appropriate by local regulations) for at least 3 years prior to enrollment, on a stable dose for at least 6 months, is deemed clinically stable for at least 1 year by the Investigator and is within the therapeutic goals as all of the following:

Sponsors & Collaborators

Sanofi
Industrylead

Study Sites (22)

Yale University School of Medicine - Investigational Site Number: 8400003

New Haven, Connecticut, 06511, United States

University of Iowa - Investigational Site Number: 8400002

Iowa City, Iowa, 52242, United States

Texas Oncology - Medical City Dallas Site Number : 8400008

Dallas, Texas, 75230, United States

Lysosomal & Rare Disorders Research & Treatment Center, Inc - Investigational Site Number: 8400001

Fairfax, Virginia, 22030, United States

Hospital de Ninos - Investigational Site Number: 320001

Buenos Aires, 1426, Argentina

Children's Hospital Research Institute of Manitoba - Investigational Site Number: 1240001

Winnipeg, Manitoba, R3E 3P4, Canada

MeSH Terms

Conditions

Gaucher Disease

Interventions

venglustatimiglucerase

Condition Hierarchy (Ancestors)

SphingolipidosesLysosomal Storage Diseases, Nervous SystemBrain Diseases, Metabolic, InbornBrain Diseases, MetabolicBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

Clinical Sciences & Operations
Sanofi
STUDY DIRECTOR

Enrollment

43 participants (actual enrollment reported by the registry).

Study Design

Study Type: interventional
Phase: phase 3
Allocation: RANDOMIZED
Masking: QUADRUPLE
Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Masking Details: During the open-label extended treatment period all participants will receive open label venglustat.
Purpose: TREATMENT
Intervention Model: PARALLEL
Sponsor Type: INDUSTRY
Responsible Party: SPONSOR
Expanded Access: Yes

Study Record Dates

First Submitted

January 20, 2022

First Posted

February 3, 2022

Study Start

You may not qualify if:

The participant is blood transfusion-dependent.
Prior esophageal varices or liver infarction or current liver enzymes (alanine aminotransferase \[ALT\]/ aspartate aminotransferase \[AST\]) or total bilirubin \>2 times the upper limit of normal, unless the participant has a diagnosis of Gilbert Syndrome.
The participant has any clinically significant disease, other than GD, including cardiovascular (congenital cardiac defect, coronary artery disease, valve disease or left sided heart failure; clinically significant arrhythmias or conduction defect), hepatic, gastrointestinal, pulmonary, neurologic, endocrine, metabolic (eg, hypokalemia, hypomagnesemia) or psychiatric disease, other medical conditions, or serious intercurrent illnesses that may preclude participation in the opinion of the Investigator.
The participant has renal insufficiency, as defined by an estimated glomerular filtration rate \<30 mL/min/1.73m2 at the screening visit.
The participant has a history of cancer, except for basal cell carcinoma.
The participant has progressive myoclonic epilepsy.
The participant is pregnant (has a positive serum beta-human chronic gonadotropin \[β-hCG\]) or lactating.
The participant requires use of invasive ventilatory support.
The participant requires use of noninvasive ventilator support while awake for longer than 12 hours daily.
The participant is scheduled for in-patient hospitalization including elective surgery, during the study.
The participant has had a major organ transplant (eg, bone marrow or liver).
A history of drug and/or alcohol abuse within the past year prior to the screening visit.
Chaperone therapy within 6 months, substrate reduction therapy other than venglustat within 6 months or venglustat substrate reduction therapy prior to enrollment.
Exposure to any investigational drug within the last 30 days or 5 half-lives from screening, whichever is longer.
The participant has received strong or moderate inducers or inhibitors of CYP3A within 14 days or 5 half-lives from screening, whichever is longer, prior to screening. This also includes the consumption of grapefruit, grapefruit juice, or grapefruit containing products within 72 hours of starting venglustat. The participant is unwilling to abstain from consumption of grapefruit, grapefruit juice, or grapefruit containing products for the duration of the treatment period.
+5 more criteria

Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3

Brief Summary

Registry Overview

Trial Relationships

Study Timeline

First Submitted

First Posted

Study Start

Primary Completion

Study Completion

Conditions

Outcome Measures

Primary Outcomes (2)

Secondary Outcomes (9)

Study Arms (2)

Venglustat

Cerezyme

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (22)

Related Links

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 (2)

Secondary Outcomes (9)

Study Arms (2)

Venglustat

Cerezyme

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (22)

Related Links

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