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A Study to Evaluate the Effect of Venglustat Tablets on Neuropathic and Abdominal Pain in Male and Female Participants ≥16 Years of Age With Fabry Disease

PHASE3RECRUITING

This is a 12-month, parallel treatment, Phase 3, double-blind, randomized, placebo controlled study to evaluate the effect of venglustat on neuropathic and abdominal pain symptoms of Fabry disease in participants ≥16 years of age with Fabry disease who are treatment-naïve or untreated for at least 6 months. * Study visits will take place approximately every 3 months. * The double-blind period will be followed by an open-label extension (OLE) during which participants who have completed the double-blind period will be treated with venglustat for up to an additional 12 months.

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Study details:

Double blind period: the total duration will be up to approximately of 14 months (1 month of screening 12 month of treatment period, and a possible follow-up period of 1 month if no participation in the open label extension period). Open-label extension period: the total duration will be approximately of 31 months (12 month of OLE treatment, additional OLE treatment until a common study end of treatment date (CSEOTD, approximately 18 months), and 1 month of follow-up period).

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Eligibility criteria

Researchers look for people who fit a certain description, called eligibility criteria. See if you qualify.

Inclusion criteria

Male and female adult patients 16 year of age or older, who have had a previously confirmed diagnosis of Fabry disease and a history of clinical symptoms of Fabry disease

Patients who are treatment-naïve or without prior treatment with an approved or experimental therapy for Fabry disease within at least 6 months prior to screening.

Average score of ≥3 (0=no symptom, 10=symptom as bad as you can imagine) on the participant-defined most-bothersome symptom (among neuropathic pain in upper extremities, neuropathic pain in lower extremities, or abdominal pain), as measured by the Fabry Disease Patient-Reported Outcome (FD-PRO) at screening.

Contraception (with double contraception methods) for male and female participants; not pregnant or breastfeeding for female participants; no sperm donation for male participants.

Weight ≥30 Kg

A signed informed consent must be provided prior to any study-related procedures.

Exclusion criteria

Any manifestations of Fabry disease that preclude placebo administration.

History of transient ischemic attack, stroke, myocardial infarction, heart failure, evidence of left ventricular hypertrophy and/or cardiac fibrosis, major cardiovascular surgery, or kidney transplantation.

History of clinically significant cardiac arrhythmia. Atrial fibrillation that is well controlled on a stable medical regimen for at least 12 months is not an exclusion if the CHA2DS2-VASc score is 0 for males or 1 for females.

Patients with hepatitis C, HIV, or hepatitis B infection.

Neuropathic pain in upper or lower extremities, or abdominal pain not related to Fabry disease.

History of seizures currently requiring treatment.

Uncontrolled hypertension over the past 12 months prior to screening, or systolic BP >=150 or diastolic BP >=100 at screening.

Estimated glomerular filtration rate <60 mL/min/1.73m².

Urine protein to creatinine ratio >= 1 g/g at screening.

Presence of severe depression as measured by Beck's Depression Inventory (BDI)-II >28 and/or a history of an untreated, unstable major affective disorder within 1 year of the screening visit.

Positive SARS-CoV-2 virus test within 2 weeks of enrollment, or COVID 19 requiring hospitalization within 6 months of enrollment.

Moderate to severe hepatic impairment.

History of drug and/or alcohol abuse.

History of or active hepatobiliary disease.

Liver enzymes (alanine aminotransferase (ALT)/aspartate aminotransferase (AST)) or total bilirubin >2 times the upper limit of normal (ULN).

Initiation of chronic treatment for pain, or change in pain medication regimen, within 3 months prior to randomization.

Strong or moderate inducers or inhibitors of cytochrome P450 3A within 14 days or 5 half-lives, whichever is longer, prior to randomization.

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Eligibility

Age eligible for study : 16 and older

Healthy volunteers accepted : No

Gender eligible for study: All

Things to know

Study dates

Study start: 2022-03-11

Primary completion: 2024-11-30

Study completion finish: 2025-10-06

Study type

TREATMENT

Phase

PHASE3

Trial ID

NCT05206773

Intervention or treatment

DRUG: Venglustat (GZ402671)

DRUG: Placebo

Conditions

• Fabry Disease

Condition: Fabry Disease
DRUG: Venglustat (GZ402671) and other drugs
Parkville, Victoria, Australia
Sponsor: Sanofi

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Find a site

Closest Location:

Investigational Site Number : 0360001

Research sites nearby

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Investigational Site Number : 0360001
Parkville, Victoria, Australia

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Participant Group/Arm	Intervention/Treatment
EXPERIMENTAL: Venglustat Participant will receive venglustat dose once daily up to 12 months	DRUG: Venglustat (GZ402671) Pharmaceutical form: Tablet Route of administration: Oral
PLACEBO_COMPARATOR: Placebo Participants will receive placebo once daily up to 12 months	DRUG: Placebo Pharmaceutical form: Tablet Route of administration: Oral

What is the study measuring?

Primary outcome

Primary Outcome Measure	Primary Outcome Description	Primary Outcome Time Frame
Percent change from baseline at 6 months in the most bothersome symptom of 3 Fabry Disease Patient-Reported Outcome (FD-PRO) items (neuropathic pain in upper extremities, neuropathic pain in lower extremities, and abdominal pain)	Not Specified	From baseline to 6 months
Percent change from baseline at 12 months in the most bothersome symptom of 3 Fabry Disease Patient-Reported Outcome (FD-PRO) items (neuropathic pain in upper extremities, neuropathic pain in lower extremities, and abdominal pain)	Not Specified	From baseline to 12 months

Secondary outcome

Secondary Outcome Measure	Secondary Outcome Description	Secondary Outcome Time Frame
Percent change in plasma globotriaosylsphingosine (lyso-GL-3)	Not Specified	From baseline to 6 month and 12 months
Frequency of rescue pain medication use	Number of days with use of rescue pain medications during the 6-month treatment period, divided by duration of the 6-month treatment period and multiplied by 100. The same definition will be used for the 12-month period.	From baseline to 6 months and 12 months
Change in the percentage of days with at least 1 stool reflecting diarrhea (Bristol Stool Form Scale [BSFS] Type 6 or 7)	Not Specified	From baseline to 6 month and 12 months
Percent change in tiredness component of FD-PRO	Not Specified	From baseline to 6 month and 12 months
Proportion of responders in neuropathic or abdominal pain, as assessed by FD-PRO	Response is defined as at least a 30% decrease from baseline in the most bothersome of 3 FD-PRO items between neuropathic pain in upper extremities, neuropathic pain in lower extremities, and abdominal pain	At 6 months and 12 months
Number of participants with adverse event (AE) and serious adverse event (SAE)	Not Specified	From baseline to 6 month and 12 months
Change in the lens clarity (new or worsening lens opacities) by ophthalmological examination (by slit lamp exam at Visit 2 and Visit 6)	Not Specified	From baseline to 12 months
Change in Beck Depression Inventory-II (BDI-II) score	Not Specified	From baseline to 6 month and 12 months
Plasma venglustat concentrations at prespecified visits over the study duration	Not Specified	From baseline to 6 month and 12 months
Maximum venglustat plasma concentration (Cmax)	Not Specified	From baseline to 6 month and 12 months
Time to maximum venglustat plasma concentration (tmax)	Not Specified	From baseline to 6 month and 12 months
Area under the venglustat plasma concentration versus time curve from time 0 to 24 hours (AUC0-24)	Not Specified	From baseline to 6 month and 12 months

Frequently Asked Questions

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References

Clinical Trials Gov: A Study to Evaluate the Effect of Venglustat Tablets on Neuropathic and Abdominal Pain in Male and Female Participants ≥16 Years of Age With Fabry Disease