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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

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
Trial website
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
[email protected]
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
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
When will data be available (start and end dates)?
Available to whom?
Available for what types of analyses?
How or where can data be obtained?


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT05206773