- To evaluate the effect of venglustat on mood using Beck Depression Inventory II (BDI-II) (Stages 1 and 2).

Phase 3 trials include large numbers of people to make sure that the result is valid. All rights reserved. - To evaluate the pharmacokinetics (PK) of venglustat in Autosomal Dominant Polycystic Kidney Disease patients (Stages 1 and 2).

A phase 2 study assessed the safety, pharmacokinetics (PK), pharmacodynamics (PD), and exploratory efficacy of Ibiglustat (GZ/SAR402671) in enzyme replacement therapy treatment-naïve adult male participants diagnosed with Fabry disease: GZ/SAR402671 15 mg once daily orally for 26 weeks. AbstractBackground and Aims. The Sanofi Genzyme proof of concept study, [NCT02906020][1], will assess safety, tolerability, and efficacy of oral venglustat in more than 200 patients worldwide.

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A phase 2/3 study is being initiated on venglustat, an oral glucosylceramide synthase (GCS) inhibitor, in ADPKD (9). Mayo Clinic does not endorse any of the third party products and services advertised.A single copy of these materials may be reprinted for noncommercial personal use only. Ab etwa Januar 2019 beginnt in Deutschland eine Phase-II / 3-Studie zur Evaluierung von Venglustat, einem oralen Glucosylceramid-Synthase (GCS) -Inhibitor, der bei Patienten mit dem Risiko einer schnell fortschreitenden autosomal dominanten polyzystischen Nierenerkrankung (ADPKD) starten soll.

In the later phase (phase 3), researchers study whether the treatment works better than the current standard therapy. Items shown with a ✜ denote a Phase 1/2 trial. Sanofi expects full Phase 1 results and the recommended Phase 2 dose by 2021. Types include:During the early phases (phases 1 and 2), researchers assess safety, side effects, optimal dosages and risks/benefits. Mayo Clinic is a nonprofit organization and proceeds from Web advertising help support our mission. For details on venglustat trials, see There are also less common very early (phase 0) and later (phase 4) phases. PHASE III: Olipudase Alfa† Acid Sphingomyelinase Deficiency (ASMD) - …

Ibiglustat (GZ/SAR402671 or Genz-682452) is a small molecule inhibitor of glucosylceramide synthase. InChIKey=YFHRCLAKZBDRHN-MRXNPFEDSA-N Phase 2 Phase 3 Detailed Description: Study duration per participant is 26 months (maximal) per stage, including a screening period of 15 days, run-in period of 2 weeks, a 24-month treatment period, and a follow-up 30 days after final dose of investigational medicinal product (IMP). Venglustat is a small‐molecule glucosylceramide synthase (GCS) inhibitor designed to reduce the production of glucosylceramide (GL‐1) and thus is expected to substantially reduce formation of …

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Evaluate short-term (Part 2) and long-term (Part 3) safety and tolerability of GZ/SAR402671 in combination with Cerezyme in adult GD3 patients. are being developed in collaboration with Regeneron, are being developed in collaboration with Sangamo Therapeutics,  are being developed in collaboration with Roche, are being developed in collaboration with BioNtech, are being developed in collaboration with Immunext, are being developed in collaboration with Denali, ➤ are being developed in collaboration with Lead Pharma and ♦ are being developed in collaboration with Principia Biopharma Inc.

Venglustat is being developed for lysosomal storage diseases, including Gaucher’s, Fabry, and for Parkinson’s caused by mutations in the GBA1 gene. Sanofi Genzyme is currently recruiting for a phase 2/3 study of venglustat in patients with ADPKD between the ages of 18-50.

There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. evaluation phase in adult GD3 and adult GD1 patients, as well as a screening phase for adult GD3 patients prior to entering Part 2. A Phase 2 trial has been approved by the Food and Drug Administration and will begin enrolling this month, with completion scheduled for 2022, Sardi said. Genzyme, a Sanofi Company is developing Ibiglustat for the treatment of Parkinson's Disease, Gaucher Disease, and Fabry Disease. A phase 2 study assessed the safety, pharmacokinetics (PK), pharmacodynamics (PD), and exploratory efficacy of Ibiglustat (GZ/SAR402671) in enzyme replacement therapy treatment-naïve adult male participants diagnosed with Fabry disease: GZ/SAR402671 15 mg once daily orally for 26 weeks.



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