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 (PERIDOT)

March 27, 2024 updated by: Sanofi

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 Participants ≥16 Years of Age With Fabry Disease Who Are Treatment-naïve or Untreated for at Least 6 Months

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

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)

Study Type

Interventional

Enrollment (Estimated)

114

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

  • Name: Trial Transparency email recommended (Toll free for US & Canada)
  • Phone Number: option 6 800-633-1610
  • Email: Contact-US@sanofi.com

Study Locations

  • Argentina
      • La Rioja, Argentina, F5300
        • Recruiting
        • Fundacion Cori para la Investigación y Prevención del Cancer_Investigational Site Number: 0320002
        • Contact:
        • Principal Investigator:
          • Juan Marcelo Muraro, MD
      • Pergamino, Argentina, B2700CPM
        • Recruiting
        • Instituto de Nefrologia Pergamino_Investigational Site Number: 0320001
        • Contact:
        • Principal Investigator:
          • Norberto Antongiovanni
      • Quilmes, Argentina, B1878GEG
        • Recruiting
        • Instituto de Investigaciones Clínicas Quilmes (IICQ) SRL_Investigational Site Number: 0320003
        • Contact:
        • Contact:
          • Phone Number: +549113580-4733
        • Principal Investigator:
          • Alberto Fernandez, Dr
  • Australia
    • Victoria
      • Parkville, Victoria, Australia, 3050
        • Recruiting
        • Investigational Site Number : 0360001
  • Austria
      • Wien, Austria, 1090
        • Recruiting
        • Investigational Site Number: 0400001
  • Brazil
    • Rio Grande Do Sul
      • Porto Alegre, Rio Grande Do Sul, Brazil, 90035-903
        • Recruiting
        • Hospital de Clínicas de Porto Alegre_Investigational Site Number: 0760001
        • Contact:
        • Contact:
          • Backup Phone
          • Phone Number: +55 5133596343 /5133596340
        • Principal Investigator:
          • Roberto Giugliani, Dr.
  • Canada
    • Alberta
      • Calgary, Alberta, Canada, T2E7Z4
        • Recruiting
        • M.A.G.I.C Calgary LTD_Investigational Site Number: 1240003
        • Principal Investigator:
          • Aneal Khan, MD
        • Contact:
    • Nova Scotia
      • Halifax, Nova Scotia, Canada, B3H 2Y9
        • Recruiting
        • Medicine Dalhousie University_Investigational Site Number : 1240001
        • Contact:
          • Michael L. West, Dr.
          • Phone Number: 902 473-4023
          • Email: mlwest@dal.ca
        • Principal Investigator:
          • Michael L. West, Dr.
    • Ontario
      • Toronto, Ontario, Canada, M5T 3L9
        • Recruiting
        • University Health Network_Investigational Site Number : 1240005
        • Contact:
        • Principal Investigator:
          • Chantal Morel, Dr.
  • China
      • Beijing, China, 100034
        • Recruiting
        • No.8, Xishiku Street, Xicheng District_Site Number: 1560001
        • Contact:
      • Shanghai, China, 200010
        • Recruiting
        • No.197,2nd Ruijin road, Huangpu district_Site Number: 1560003
        • Contact:
      • Taiyuan, China, 030001
        • Recruiting
        • No.85 South Jiefang road, Yingze District_Site Number: 1560004
        • Contact:
      • Zhengzhou, China, 450008
        • Recruiting
        • Investigational Site Number : 1560006
  • Denmark
      • Copenhagen, Denmark, 2100
        • Recruiting
        • Investigational Site Number: 2080001
  • Finland
      • Turku, Finland, FI-20520
        • Recruiting
        • Investigational Site Number: 2460001
  • France
      • Garches, France, 92380
        • Recruiting
        • Investigational Site Number : 2500001
  • Germany
      • Hochheim Am Main, Germany, 65239
        • Recruiting
        • ISphinCS GmbH_Investigational Site Number: 2760004
        • Contact:
      • München, Germany, 80336
        • Recruiting
        • Investigational Site Number: 2760003
      • Wurzburg, Germany, 97080
        • Recruiting
        • Investigational Site Number: 2760001
  • Greece
      • Heraklion, Greece, 711 10
        • Recruiting
        • University Hospital of Heraklion_Investigational Site Number: 3000001
        • Contact:
        • Principal Investigator:
          • Konstantinos Stylianou, Dr.
      • Ioannina, Greece, 455 00
        • Recruiting
        • University Hospital of Ioannina_Investigational Site Number: 3000002
        • Contact:
        • Principal Investigator:
          • Evangelia Ntounousi, Dr.
  • Italy
      • Bologna, Italy, 40138
        • Recruiting
        • Investigational Site Number : 3800005
      • Monza, Italy, 20052
        • Recruiting
        • Fondazione IRCCS San Gerardo dei Tintori, S.C. Nefrologia - Clinica Nefrologica_Investigational Site Number: 3800002
        • Contact:
        • Principal Investigator:
          • Federico Pieruzzi
      • Napoli, Italy, 80138
        • Recruiting
        • Azienda Ospedaliera Universitaria "Federico II", U.O. di Nefrologia- Diparimento di Sanità Pubblica_Investigational Site Number: 3800001
        • Contact:
        • Contact:
        • Principal Investigator:
          • Antonio Pisani
      • Palermo, Italy, 90127
        • Recruiting
        • Azienda Ospedaliera Universitaria_Investigational Site Number: 3800003
        • Contact:
        • Principal Investigator:
          • Antonino Tuttolomondo
      • Roma, Italy, 00168
        • Recruiting
        • Fondazione Policlinico Universitario_Investigational Site Number: 3800004
        • Contact:
        • Principal Investigator:
          • Elena Verrecchia, Dr
  • Japan
      • Fukuoka-shi, Fukuoka, Japan, 814-0180
        • Recruiting
        • Fukuoka University Hospital_Investigational Site Number: 3920004
      • Minato-ku, Tokyo, Japan, 105-8471
        • Recruiting
        • The Jikei University Hospital_Investigational Site Number: 3920003
      • Sendai-shi, Miyagi, Japan, 980-8574
        • Recruiting
        • Tohoku University Hospital_Investigational Site Number: 3920001
    • Kanagawa
      • Kawasaki-shi, Kanagawa, Japan, 215-0026
        • Recruiting
        • Investigational Site Number : 3920005
  • Mexico
      • Ciudad de Mexico, Mexico, 06700
        • Recruiting
        • Odette del Carmen DIAZ-AVENDAÑO Clinstile, S.A. de C.V. Durango_Investigational Site Number: 4840002
        • Contact:
          • Odette del Carmen DIAZ-AVENDAÑO
          • Phone Number: 52-1-55-3200-2515
          • Email: oddimx@hotmail.com
        • Principal Investigator:
          • Odette del Carmen DIAZ-AVENDAÑO
    • Nuevo León
      • Monterrey, Nuevo León, Mexico, 64460
        • Recruiting
        • Hospital Universitario "Dr. José Eleuterio González" Departamento de Genética Centro Universitario contra el cáncer_Investigational Site Number: 4840001
        • Contact:
        • Principal Investigator:
          • Marisol IBARRA-RAMIREZ
  • Norway
      • Bergen, Norway, 5021
        • Recruiting
        • Investigational Site Number: 5780001
  • Poland
      • Lodz, Poland, 92-213
        • Recruiting
        • Investigational Site Number: 6160001
      • Wroclaw, Poland, 50-556
        • Recruiting
        • Investigational Site Number: 6160003
    • Wielkopolskie
      • Poznan, Wielkopolskie, Poland, 60-355
        • Recruiting
        • Investigational Site Number : 6160002
  • Romania
      • Bucuresti, Romania, 22328
        • Recruiting
        • Institutul Clinic Fundeni_Investigational Site Number: 6420001
        • Contact:
  • Switzerland
      • Zürich, Switzerland, 8091
        • Recruiting
        • Investigational Site Number : 7560001
  • Turkey
      • Ankara, Turkey, 06500
        • Recruiting
        • Investigational Site Number : 7920001
      • Kocaeli, Turkey, 41380
        • Recruiting
        • Investigational Site Number : 7920002
      • Malatya, Turkey, 44280
        • Recruiting
        • Investigational Site Number : 7920004
  • United Kingdom
      • Cambridge, United Kingdom, CB2 2QQ
        • Recruiting
        • Investigational Site Number: 8260001
      • London, United Kingdom, NW3 2QG
        • Recruiting
        • Investigational Site Number: 8260002
  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35233
        • Recruiting
        • Nephrology Clinic at Kirklin Clinic of UAB Hospital_Investigational Site Number: 8400011
        • Contact:
        • Principal Investigator:
          • Eric Wallace
    • California
      • Los Angeles, California, United States, 90095
        • Recruiting
        • UCLA Medical Center_Investigational Site Number: 8400006
        • Principal Investigator:
          • Anjay Rastogi
        • Contact:
      • Orange, California, United States, 92868
        • Recruiting
        • University of California Irvine Medical Center Site Number : 8400019
    • Florida
      • Orlando, Florida, United States, 32804
        • Recruiting
        • Advent Health Orlando_Investigational Site Number: 8400008
        • Contact:
        • Principal Investigator:
          • Majed Dasouki, Dr.
    • Georgia
      • Atlanta, Georgia, United States, 30322
        • Recruiting
        • Emory Genetics Site Number : 8400010
    • New York
      • Hawthorne, New York, United States, 10532
        • Recruiting
        • Westchester Medical Center Healthcare Corporation Site Number : 8400001
    • Ohio
      • Cincinnati, Ohio, United States, 45229-3039
        • Recruiting
        • Cincinnati Children's Hospital Medical Center - PIN Site Number : 8400013
    • Pennsylvania
      • Pittsburgh, Pennsylvania, United States, 15213
        • Recruiting
        • Children's Hospital Of Pittsburgh Site Number : 8400009
    • Texas
      • Dallas, Texas, United States, 75235
        • Recruiting
        • Renal Disease Research Institute, An affiliate of: Dallas Nephrology Associates_Investigational Site Number: 8400012
        • Contact:
        • Principal Investigator:
          • Ankit Mehta, Dr.
    • Virginia
      • Fairfax, Virginia, United States, 22030
        • Recruiting
        • Lysosomal and Rare Disorders Research and Treatment Center_Investigational Site Number: 8400004
        • Principal Investigator:
          • Ozlam Goker-Alpan, MD
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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.

The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial.

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?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Double

Arms and Interventions

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 Measures

Outcome Measure
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)
Time Frame: From baseline to 6 months
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)
Time Frame: From baseline to 12 months
From baseline to 12 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Percent change in plasma globotriaosylsphingosine (lyso-GL-3)
Time Frame: From baseline to 6 month and 12 months
From baseline to 6 month and 12 months
Frequency of rescue pain medication use
Time Frame: From baseline to 6 months and 12 months
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)
Time Frame: From baseline to 6 month and 12 months
From baseline to 6 month and 12 months
Percent change in tiredness component of FD-PRO
Time Frame: From baseline to 6 month and 12 months
From baseline to 6 month and 12 months
Proportion of responders in neuropathic or abdominal pain, as assessed by FD-PRO
Time Frame: At 6 months and 12 months
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)
Time Frame: From baseline to 6 month and 12 months
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)
Time Frame: From baseline to 12 months
From baseline to 12 months
Change in Beck Depression Inventory-II (BDI-II) score
Time Frame: From baseline to 6 month and 12 months
From baseline to 6 month and 12 months
Plasma venglustat concentrations at prespecified visits over the study duration
Time Frame: From baseline to 6 month and 12 months
From baseline to 6 month and 12 months
Maximum venglustat plasma concentration (Cmax)
Time Frame: From baseline to 6 month and 12 months
From baseline to 6 month and 12 months
Time to maximum venglustat plasma concentration (tmax)
Time Frame: From baseline to 6 month and 12 months
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)
Time Frame: From baseline to 6 month and 12 months
From baseline to 6 month and 12 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sanofi

Investigators

  • Study Director: Clinical Sciences & Operations, Sanofi

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

March 11, 2022

Primary Completion (Estimated)

November 30, 2024

Study Completion (Estimated)

July 8, 2026

Study Registration Dates

First Submitted

October 20, 2021

First Submitted That Met QC Criteria

January 24, 2022

First Posted (Actual)

January 25, 2022

Study Record Updates

Last Update Posted (Actual)

March 28, 2024

Last Update Submitted That Met QC Criteria

March 27, 2024

Last Verified

March 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • EFC17045
  • 2021-002350-90 (EudraCT Number)
  • U1111-1256-9310 (Registry Identifier: ICTRP)
  • 2024-511990-31 (Registry Identifier: CTIS)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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