PERIDOT

• Research type

  Research Study

• Full title

  A randomized, double-blind, placebo-controlled, 12 month Phase 3 study to evaluate the effect of venglustat on neuropathic and abdominal pain in male and female adults with Fabry disease who are treatment-naïve or untreated for at least 6 months

• IRAS ID

  1004207

• Contact name

  Marine Baixauli

• Contact email

  Marine.Baixauli@sanofi.com

• Sponsor organisation

  Sanofi-Aventis Recherche & Développement

• Eudract number

  2021-002350-90

• Clinicaltrials.gov Identifier

  NCT05206773

• Research summary

  Fabry disease is a rare X-linked inborn error of glycosphingolipid catabolism caused by deficient activity of lysosomal α-galactosidase A (α-GAL or α-Gal A). The deficiency of α-Gal A leads to accumulation of GL-3 in the lysosomes of various cell types, leading over decades to cellular pathology and various clinical manifestations, including progression to renal failure and potentially fatal cerebrovascular and cardiovascular events. Fabry disease has a multisystemic nature, with heterogeneous clinical manifestations and a highly variable rate of disease progression. Although participants with Fabry disease may experience a heterogenous mix of symptoms, neuropathic pain and abdominal pain are among the most commonly experienced and severe symptoms in the target population

  Current treatment for Fabry disease includes enzyme replacement therapy (ERT), which requires intravenous (IV) infusions every 2 weeks that are often accompanied by infusion reactions, and the oral agent migalastat, for a subset of participants with amenable mutations. Given the lifelong nature of Fabry disease, challenges to compliance with an IV treatment, and the restriction of the only available oral therapy to a subset of participants, there is an unmet need for additional oral agents. Venglustat is an oral glucosylceramide synthase (GCS) inhibitor in development for treatment of Fabry disease.
  This double-blind, placebo-controlled study is designed to assess the effect of up to 12 months of treatment with venglustat on symptom burden, focusing on neuropathic and abdominal pain, using the Fabry Disease Patient-Reported Outcome (FD-PRO) instrument. Approximately 114 participants will be randomly assigned to venglustat or placebo in a 1:1 ratio.

• REC name

  East of England - Cambridge East Research Ethics Committee

• REC reference

  22/EE/0028

• Date of REC Opinion

  24 Mar 2022

• REC opinion

  Further Information Favourable Opinion