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Venglustat (GZ402671) for Fabry Disease

Phase-Based Progress Estimates
2
Effectiveness
3
Safety
Hospital de Clínicas de Porto Alegre_ Rua Ramiro Barcelos, 2350, Santa Cecília, Porto Alegre - RS_ Site: 0760001, Porto Alegre, BrazilFabry DiseaseVenglustat (GZ402671) - Drug
Eligibility
18+
All Sexes
What conditions do you have?
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Study Summary

This trial will study the effect of venglustat on neuropathic and abdominal pain symptoms of Fabry disease in adults who have not been treated or have been treated for less than 6 months.

Eligible Conditions
  • Fabry Disease

Treatment Effectiveness

Effectiveness Progress

2 of 3
This is further along than 85% of similar trials

Similar Trials

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epetraborole + OBR
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Study Objectives

2 Primary · 12 Secondary · Reporting Duration: At 6 months and 12 months

Month 12
Proportion of responders in neuropathic or abdominal pain, as assessed by FD-PRO
Month 12
Change in the lens clarity (new or worsening lens opacities) by ophthalmological examination (by slit lamp exam at Visit 2 and Visit 6)
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)
Month 12
Area under the venglustat plasma concentration versus time curve from time 0 to 24 hours (AUC0-24)
Change in Beck Depression Inventory-II (BDI-II) score
Change in the percentage of days with at least 1 stool reflecting diarrhea (Bristol Stool Form Scale [BSFS] Type 6 or 7)
Maximum venglustat plasma concentration (Cmax)
Number of participants with adverse event (AE) and serious adverse event (SAE)
Percent change in plasma globotriaosylsphingosine (lyso-GL-3)
Percent change in tiredness component of FD-PRO
Plasma venglustat concentrations at prespecified visits over the study duration
Time to maximum venglustat plasma concentration (tmax)
Month 6
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)
Month 12
Frequency of rescue pain medication use

Trial Safety

Safety Progress

3 of 3
This is further along than 85% of similar trials

Similar Trials

1
Participants with mild hepatic impairment
2
epetraborole + OBR
1
ST-920

Trial Design

2 Treatment Groups

Venglustat
1 of 2
Placebo
1 of 2

Experimental Treatment

Non-Treatment Group

114 Total Participants · 2 Treatment Groups

Primary Treatment: Venglustat (GZ402671) · Has Placebo Group · Phase 3

Venglustat
Drug
Experimental Group · 1 Intervention: Venglustat (GZ402671) · Intervention Types: Drug
Placebo
Drug
PlaceboComparator Group · 1 Intervention: Placebo · Intervention Types: Drug

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: at 6 months and 12 months

Who is running the clinical trial?

Genzyme, a Sanofi CompanyLead Sponsor
521 Previous Clinical Trials
85,334 Total Patients Enrolled
25 Trials studying Fabry Disease
14,674 Patients Enrolled for Fabry Disease
Clinical Sciences & OperationsStudy DirectorSanofi
808 Previous Clinical Trials
1,627,342 Total Patients Enrolled
5 Trials studying Fabry Disease
271 Patients Enrolled for Fabry Disease

Eligibility Criteria

Age 18+ · All Participants · 6 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:
You have not received any approved or experimental treatment for Fabry disease in the last 6 months.
If you are a woman, you are not pregnant or breastfeeding. If you are a man or woman, you must use two forms of birth control and cannot donate sperm.
You must agree to the study and sign a form before any study-related tests are done.
You are an adult over 18 years old who has been diagnosed with Fabry disease before and have experienced symptoms of the disease.
You have high levels of lysoGL3 in your blood or previous biopsy results that show GL3 buildup in any organ.
You have pain in your arms, legs or stomach that you rate as a 3 or higher on a 0-10 scale.
References

Frequently Asked Questions

What is Venglustat's (GZ402671) official regulatory status?

"Venglustat's (GZ402671) safety is estimated to be a 3. This Phase 3 rating means that, while there isn't extensive evidence of its efficacy, multiple rounds of testing have proven it to be safe." - Anonymous Online Contributor

Unverified Answer

Are there many hospitals conducting this trial in Canada?

"Currently, there are 7 clinical trial sites operational. These locations include Advent Health Orlando 301 East Princeton St_Site Number: 8400008 in Orlando, University of Manitoba, Children's Hospital Research Institute of Manitoba, Room 511C John Buhler Research Center - site number 1240002 in Winnipeg, and Oregon Health and Sciences University - site number 8400007 in Portland." - Anonymous Online Contributor

Unverified Answer

What is the projected number of people who will be taking part in this research project?

"In order to move this study forward, 114 individuals who match the pre-determined criteria must participate. Those willing and eligible can enroll at either Advent Health Orlando or University of Manitoba." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

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