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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05206773




Registration number
NCT05206773
Ethics application status
Date submitted
20/10/2021
Date registered
25/01/2022
Date last updated
21/06/2024

Titles & IDs
Public title
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
Scientific 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 Participants =16 Years of Age With Fabry Disease Who Are Treatment-naïve or Untreated for at Least 6 Months
Secondary ID [1] 0 0
U1111-1256-9310
Secondary ID [2] 0 0
EFC17045
Universal Trial Number (UTN)
Trial acronym
PERIDOT
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Fabry Disease 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Metabolic and Endocrine 0 0 0 0
Metabolic disorders
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders
Oral and Gastrointestinal 0 0 0 0
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Venglustat (GZ402671)
Treatment: Drugs - Placebo

Experimental: Venglustat - Participant will receive venglustat dose once daily up to 12 months

Placebo comparator: Placebo - Participants will receive placebo once daily up to 12 months


Treatment: Drugs: Venglustat (GZ402671)
Pharmaceutical form: Tablet Route of administration: Oral

Treatment: Drugs: Placebo
Pharmaceutical form: Tablet Route of administration: Oral
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
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)
Timepoint [1] 0 0
From baseline to 6 months
Primary outcome [2] 0 0
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)
Timepoint [2] 0 0
From baseline to 12 months
Secondary outcome [1] 0 0
Percent change in plasma globotriaosylsphingosine (lyso-GL-3)
Timepoint [1] 0 0
From baseline to 6 month and 12 months
Secondary outcome [2] 0 0
Frequency of rescue pain medication use
Timepoint [2] 0 0
From baseline to 6 months and 12 months
Secondary outcome [3] 0 0
Change in the percentage of days with at least 1 stool reflecting diarrhea (Bristol Stool Form Scale [BSFS] Type 6 or 7)
Timepoint [3] 0 0
From baseline to 6 month and 12 months
Secondary outcome [4] 0 0
Percent change in tiredness component of FD-PRO
Timepoint [4] 0 0
From baseline to 6 month and 12 months
Secondary outcome [5] 0 0
Proportion of responders in neuropathic or abdominal pain, as assessed by FD-PRO
Timepoint [5] 0 0
At 6 months and 12 months
Secondary outcome [6] 0 0
Number of participants with adverse event (AE) and serious adverse event (SAE)
Timepoint [6] 0 0
From baseline to 6 month and 12 months
Secondary outcome [7] 0 0
Change in the lens clarity (new or worsening lens opacities) by ophthalmological examination (by slit lamp exam at Visit 2 and Visit 6)
Timepoint [7] 0 0
From baseline to 12 months
Secondary outcome [8] 0 0
Change in Beck Depression Inventory-II (BDI-II) score
Timepoint [8] 0 0
From baseline to 6 month and 12 months
Secondary outcome [9] 0 0
Plasma venglustat concentrations at prespecified visits over the study duration
Timepoint [9] 0 0
From baseline to 6 month and 12 months
Secondary outcome [10] 0 0
Maximum venglustat plasma concentration (Cmax)
Timepoint [10] 0 0
From baseline to 6 month and 12 months
Secondary outcome [11] 0 0
Time to maximum venglustat plasma concentration (tmax)
Timepoint [11] 0 0
From baseline to 6 month and 12 months
Secondary outcome [12] 0 0
Area under the venglustat plasma concentration versus time curve from time 0 to 24 hours (AUC0-24)
Timepoint [12] 0 0
From baseline to 6 month and 12 months

Eligibility
Key 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.
Minimum age
16 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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 design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s


The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
11/03/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
6/10/2025
Actual
Sample size
Target
114
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Investigational Site Number : 0360001 - Parkville
Recruitment postcode(s) [1] 0 0
3050 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Alabama
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Florida
Country [4] 0 0
United States of America
State/province [4] 0 0
Georgia
Country [5] 0 0
United States of America
State/province [5] 0 0
New York
Country [6] 0 0
United States of America
State/province [6] 0 0
Ohio
Country [7] 0 0
United States of America
State/province [7] 0 0
Pennsylvania
Country [8] 0 0
United States of America
State/province [8] 0 0
Texas
Country [9] 0 0
United States of America
State/province [9] 0 0
Utah
Country [10] 0 0
United States of America
State/province [10] 0 0
Virginia
Country [11] 0 0
Argentina
State/province [11] 0 0
La Rioja
Country [12] 0 0
Argentina
State/province [12] 0 0
Pergamino
Country [13] 0 0
Argentina
State/province [13] 0 0
Quilmes
Country [14] 0 0
Austria
State/province [14] 0 0
Wien
Country [15] 0 0
Brazil
State/province [15] 0 0
Rio Grande Do Sul
Country [16] 0 0
Canada
State/province [16] 0 0
Alberta
Country [17] 0 0
Canada
State/province [17] 0 0
Nova Scotia
Country [18] 0 0
Canada
State/province [18] 0 0
Ontario
Country [19] 0 0
China
State/province [19] 0 0
Beijing
Country [20] 0 0
China
State/province [20] 0 0
Shanghai
Country [21] 0 0
China
State/province [21] 0 0
Taiyuan
Country [22] 0 0
China
State/province [22] 0 0
Zhengzhou
Country [23] 0 0
Denmark
State/province [23] 0 0
Copenhagen
Country [24] 0 0
Finland
State/province [24] 0 0
Turku
Country [25] 0 0
France
State/province [25] 0 0
Garches
Country [26] 0 0
Germany
State/province [26] 0 0
Hochheim Am Main
Country [27] 0 0
Germany
State/province [27] 0 0
Mainz
Country [28] 0 0
Germany
State/province [28] 0 0
München
Country [29] 0 0
Germany
State/province [29] 0 0
Wurzburg
Country [30] 0 0
Greece
State/province [30] 0 0
Heraklion
Country [31] 0 0
Greece
State/province [31] 0 0
Ioannina
Country [32] 0 0
Italy
State/province [32] 0 0
Bologna
Country [33] 0 0
Italy
State/province [33] 0 0
Monza
Country [34] 0 0
Italy
State/province [34] 0 0
Napoli
Country [35] 0 0
Italy
State/province [35] 0 0
Palermo
Country [36] 0 0
Italy
State/province [36] 0 0
Roma
Country [37] 0 0
Japan
State/province [37] 0 0
Kanagawa
Country [38] 0 0
Japan
State/province [38] 0 0
Fukuoka-shi, Fukuoka
Country [39] 0 0
Japan
State/province [39] 0 0
Minato-ku, Tokyo
Country [40] 0 0
Japan
State/province [40] 0 0
Sendai-shi, Miyagi
Country [41] 0 0
Mexico
State/province [41] 0 0
Nuevo León
Country [42] 0 0
Mexico
State/province [42] 0 0
Ciudad de Mexico
Country [43] 0 0
Norway
State/province [43] 0 0
Bergen
Country [44] 0 0
Poland
State/province [44] 0 0
Wielkopolskie
Country [45] 0 0
Poland
State/province [45] 0 0
Lodz
Country [46] 0 0
Poland
State/province [46] 0 0
Wroclaw
Country [47] 0 0
Romania
State/province [47] 0 0
Bucuresti
Country [48] 0 0
Switzerland
State/province [48] 0 0
Zürich
Country [49] 0 0
Turkey
State/province [49] 0 0
Ankara
Country [50] 0 0
Turkey
State/province [50] 0 0
Kocaeli
Country [51] 0 0
Turkey
State/province [51] 0 0
Malatya
Country [52] 0 0
United Kingdom
State/province [52] 0 0
Cambridge
Country [53] 0 0
United Kingdom
State/province [53] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Sanofi
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
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.
Trial website
https://clinicaltrials.gov/study/NCT05206773
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Clinical Sciences & Operations
Address 0 0
Sanofi
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Trial Transparency email recommended (Toll free for US & Canada)
Address 0 0
Country 0 0
Phone 0 0
800-633-1610
Fax 0 0
Email 0 0
Contact-US@sanofi.com
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT05206773