122 Participants Needed

Venglustat for Fabry Disease

(PERIDOT Trial)

Recruiting at 105 trial locations
TT
Overseen ByTrial Transparency email recommended (Toll free for US & Canada)
Pivotal Trial (Near Approval)This treatment is in the last trial phase before FDA approval
Prior Safety DataThis treatment has passed at least one previous human trial

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial is testing a medication called venglustat to see if it can help reduce pain in people with Fabry disease. The medication works by targeting the root causes of nerve and abdominal pain.

Do I have to stop taking my current medications for the trial?

The trial does not specify if you must stop all current medications, but you cannot have started or changed chronic pain treatment within 3 months before joining. Also, you can't use strong or moderate inducers or inhibitors of cytochrome P450 3A within 14 days before joining.

Will I have to stop taking my current medications?

The trial does not specify if you must stop taking your current medications, but you should not have started or changed any chronic pain treatment within 3 months before joining. Also, you cannot use certain medications that affect liver enzymes within 14 days before starting the trial.

What safety data is available for Venglustat in treating Fabry Disease?

The provided research does not contain any safety data for Venglustat or its other names (GZ402671, Ibiglustat, SAR402671) in the treatment of Fabry Disease. The articles focus on the safety of lumacaftor and ivacaftor for cystic fibrosis, which is unrelated to Venglustat or Fabry Disease.12345

Is the drug Venglustat a promising treatment for Fabry Disease?

Venglustat shows promise as a treatment for Fabry Disease because it can reduce harmful substances in the body that cause organ problems. It works by blocking the production of these substances, potentially offering a new way to help people with this disease.678910

How does the drug Venglustat differ from other treatments for Fabry disease?

Venglustat is unique because it is an oral medication that works by inhibiting glucosylceramide synthase, which reduces the production of glycosphingolipids that accumulate in Fabry disease. This approach is different from enzyme replacement therapies that are typically administered intravenously and focus on replacing the deficient enzyme in patients.678910

What data supports the idea that Venglustat for Fabry Disease is an effective drug?

The available research does not provide any data supporting the effectiveness of Venglustat for Fabry Disease. The articles focus on other treatments and conditions, such as myasthenia gravis and multiple sclerosis, without mentioning Venglustat or its impact on Fabry Disease.1112131415

Who Is on the Research Team?

CS

Clinical Sciences & Operations

Principal Investigator

Sanofi

Are You a Good Fit for This Trial?

Adults with Fabry disease experiencing neuropathic or abdominal pain can join this trial if they haven't had treatments for Fabry in the last 6 months. They must have a confirmed diagnosis, be at least 18 years old, and agree to use double contraception methods. Exclusions include recent changes in pain meds, certain cardiovascular issues, uncontrolled hypertension, severe liver problems, active infections like COVID-19 within specific timeframes.

Inclusion Criteria

A signed informed consent must be provided prior to any study-related procedures
I am 16 or older with a confirmed diagnosis and symptoms of Fabry disease.
My worst symptom from Fabry disease scores 3 or more on the FD-PRO.
See 3 more

Exclusion Criteria

My blood pressure has been stable and below 150/100 for the past year.
My liver does not work well.
I have a history of liver or bile duct disease.
See 14 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

4 weeks

Treatment

Participants receive either venglustat or placebo for 12 months to evaluate its effect on neuropathic and abdominal pain

12 months
Study visits approximately every 3 months

Follow-up

Participants are monitored for safety and effectiveness after the double-blind treatment period

4 weeks

Open-label extension

Participants who completed the double-blind period may receive venglustat for up to an additional 12 months, with further treatment until a common study end date

Up to 46 months

What Are the Treatments Tested in This Trial?

Interventions

  • Placebo
  • Venglustat (GZ402671)
Trial Overview The study is testing Venglustat tablets against placebo over a year to see if they reduce neuropathic and abdominal pain in adults with Fabry disease. Participants will either get the real drug or a dummy pill without knowing which one. After this 'blind' phase, there's an extra year where everyone gets Venglustat.
How Is the Trial Designed?
2Treatment groups
Experimental Treatment
Placebo Group
Group I: VenglustatExperimental Treatment1 Intervention
Participant will receive venglustat dose once daily up to 12 months
Group II: PlaceboPlacebo Group1 Intervention
Participants will receive placebo once daily up to 12 months

Find a Clinic Near You

Who Is Running the Clinical Trial?

Sanofi

Lead Sponsor

Trials
2,246
Recruited
4,085,000+
Paul Hudson profile image

Paul Hudson

Sanofi

Chief Executive Officer since 2019

Degree in Economics from Manchester Metropolitan University

Christopher Corsico profile image

Christopher Corsico

Sanofi

Chief Medical Officer

MD from Cornell University, MPH in Chronic Disease Epidemiology from Yale University

Genzyme, a Sanofi Company

Lead Sponsor

Trials
528
Recruited
186,000+
David Meeker profile image

David Meeker

Genzyme, a Sanofi Company

Chief Executive Officer since 2011

MD from the University of Vermont Medical School, Advanced Management Program at Harvard Business School

Jean-Paul Kress profile image

Jean-Paul Kress

Genzyme, a Sanofi Company

Chief Medical Officer since 2015

MD from Faculte Necker-Enfants Malades, Paris

Published Research Related to This Trial

In a subgroup of patients with treatment-refractory generalized myasthenia gravis who had previously received chronic intravenous immunoglobulin (IVIg), eculizumab demonstrated sustained clinical improvements over 18 months compared to placebo.
Eculizumab was associated with a lower rate of disease exacerbations and was well tolerated, indicating its efficacy and safety for patients transitioning from IVIg treatment.
Response to eculizumab in patients with myasthenia gravis recently treated with chronic IVIg: a subgroup analysis of REGAIN and its open-label extension study.Jacob, S., Murai, H., Utsugisawa, K., et al.[2022]
New treatments for multiple sclerosis (MS), including teriflunomide and BG-12, are expected to be approved by the FDA soon, expanding options for patients with relapsing forms of MS.
Fingolimod, the first oral MS treatment, is now commonly used as a first-line therapy, especially for patients who cannot tolerate injections, while natalizumab is being used more widely due to the availability of JC virus antibody testing, allowing safer use in patients without the virus.
Recent advances in treating multiple sclerosis: efficacy, risks and place in therapy.Jeffery, DR.[2022]
Efgartigimod, a first-in-class neonatal Fc receptor antagonist, received its first approval in the USA in December 2021 for treating generalized myasthenia gravis in adults who are positive for anti-acetylcholine receptor antibodies.
The drug is also being evaluated for other autoimmune diseases and has been approved in Japan for generalized myasthenia gravis patients regardless of antibody status, indicating its potential broad application in autoimmune conditions.
Efgartigimod: First Approval.Heo, YA.[2022]

Citations

Response to eculizumab in patients with myasthenia gravis recently treated with chronic IVIg: a subgroup analysis of REGAIN and its open-label extension study. [2022]
Recent advances in treating multiple sclerosis: efficacy, risks and place in therapy. [2022]
Efgartigimod: First Approval. [2022]
Randomized phase 2 study of FcRn antagonist efgartigimod in generalized myasthenia gravis. [2020]
Current perspectives on FTY720. [2019]
The safety of lumacaftor and ivacaftor for the treatment of cystic fibrosis. [2018]
Clinical mechanism of the cystic fibrosis transmembrane conductance regulator potentiator ivacaftor in G551D-mediated cystic fibrosis. [2022]
Ivacaftor for patients with cystic fibrosis. [2016]
Real-Life Safety and Effectiveness of Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis. [2020]
Longitudinal effects of ivacaftor and medicine possession ratio in people with the Gly551Asp mutation: a 5-year study. [2021]
Glucosylceramide synthase inhibition with lucerastat lowers globotriaosylceramide and lysosome staining in cultured fibroblasts from Fabry patients with different mutation types. [2022]
12.United Statespubmed.ncbi.nlm.nih.gov
Venglustat, an orally administered glucosylceramide synthase inhibitor: Assessment over 3 years in adult males with classic Fabry disease in an open-label phase 2 study and its extension study. [2023]
Venglustat combined with imiglucerase for neurological disease in adults with Gaucher disease type 3: the LEAP trial. [2023]
14.United Statespubmed.ncbi.nlm.nih.gov
Pharmacokinetics, Pharmacodynamics, Safety, and Tolerability of Oral Venglustat in Healthy Volunteers. [2022]
Miglustat. Oxford GlycoSciences/Actelion. [2016]
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