Back to resultsA Study to Evaluate the Effect of Venglustat Tablets on Neuropathic and Abdominal Pain in Male and Female Adult Participants With Fabry Disease (PERIDOT)
Primary Purpose
Fabry Disease
Status
Recruiting
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Venglustat (GZ402671)
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Fabry Disease
Eligibility Criteria
Inclusion Criteria:
- Male and female adult patients 18 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.
- Elevated plasma globotriaosylsphingosine (lysoGL3) at screening, or a previous biopsy of any organ that shows deposition of GL3.
- 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.
- 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.
Sites / Locations
- Nephrology Clinic at Kirklin Clinic of UAB Hospital_Investigational Site Number: 8400011Recruiting
- UCLA Medical Center_Investigational Site Number: 8400006Recruiting
- Advent Health Orlando_Investigational Site Number: 8400008Recruiting
- Oregon Health and Sciences University_Investigational Site Number: 8400007Recruiting
- Renal Disease Research Institute, An affiliate of: Dallas Nephrology Associates_Investigational Site Number: 8400012Recruiting
- Lysosomal and Rare Disorders Research and Treatment Center_Investigational Site Number: 8400004Recruiting
- Fundacion Cori para la Investigación y Prevención del Cancer_Investigational Site Number: 0320002Recruiting
- Instituto de Nefrologia Pergamino_Investigational Site Number: 0320001Recruiting
- Instituto de Investigaciones Clínicas Quilmes (IICQ) SRL_Investigational Site Number: 0320003Recruiting
- Investigational Site Number: 0400001Recruiting
- Hospital de Clínicas de Porto Alegre_Investigational Site Number: 0760001Recruiting
- M.A.G.I.C Calgary LTD_Investigational Site Number: 1240003Recruiting
- No.8, Xishiku Street, Xicheng District_Site Number: 1560001Recruiting
- No.197,2nd Ruijin road, Huangpu district_Site Number: 1560003Recruiting
- No.85 South Jiefang road, Yingze District_Site Number: 1560004Recruiting
- Investigational Site Number: 2080001Recruiting
- Investigational Site Number: 2460001Recruiting
- ISphinCS GmbH_Investigational Site Number: 2760004Recruiting
- Investigational Site Number: 2760003Recruiting
- Investigational Site Number: 2760001Recruiting
- University Hospital of Heraklion_Investigational Site Number: 3000001Recruiting
- University Hospital of Ioannina_Investigational Site Number: 3000002Recruiting
- Fondazione IRCCS San Gerardo dei Tintori, S.C. Nefrologia - Clinica Nefrologica_Investigational Site Number: 3800002Recruiting
- Azienda Ospedaliera Universitaria "Federico II", U.O. di Nefrologia- Diparimento di Sanità Pubblica_Investigational Site Number: 3800001Recruiting
- Azienda Ospedaliera Universitaria_Investigational Site Number: 3800003Recruiting
- Fondazione Policlinico Universitario_Investigational Site Number: 3800004Recruiting
- Fukuoka University Hospital_Investigational Site Number: 3920004Recruiting
- Kagoshima University Hospital_Investigational Site Number: 3920002Recruiting
- The Jikei University Hospital_Investigational Site Number: 3920003Recruiting
- Tohoku University Hospital_Investigational Site Number: 3920001Recruiting
- Hospital Universitario "Dr. José Eleuterio González" Departamento de Genética Centro Universitario contra el cáncer_Investigational Site Number: 4840001Recruiting
- Odette del Carmen DIAZ-AVENDAÑO Clinstile, S.A. de C.V. Durango_Investigational Site Number: 4840002Recruiting
- Investigational Site Number: 5780001Recruiting
- Investigational Site Number: 6160001Recruiting
- Investigational Site Number: 6160003Recruiting
- Institutul Clinic Fundeni_Investigational Site Number: 6420001Recruiting
- Investigational Site Number: 8260001Recruiting
- Investigational Site Number: 8260002Recruiting
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Placebo Comparator
Arm Label
Venglustat
Placebo
Arm Description
Participant will receive venglustat dose once daily up to 12 months
Participants will receive placebo once daily up to 12 months
Outcomes
Primary Outcome Measures
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)
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)
Secondary Outcome Measures
Percent change in plasma globotriaosylsphingosine (lyso-GL-3)
Frequency of rescue pain medication use
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.
Change in the percentage of days with at least 1 stool reflecting diarrhea (Bristol Stool Form Scale [BSFS] Type 6 or 7)
Percent change in tiredness component of FD-PRO
Proportion of responders in neuropathic or abdominal pain, as assessed by FD-PRO
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
Number of participants with adverse event (AE) and serious adverse event (SAE)
Change in the lens clarity (new or worsening lens opacities) by ophthalmological examination (by slit lamp exam at Visit 2 and Visit 6)
Change in Beck Depression Inventory-II (BDI-II) score
Plasma venglustat concentrations at prespecified visits over the study duration
Maximum venglustat plasma concentration (Cmax)
Time to maximum venglustat plasma concentration (tmax)
Area under the venglustat plasma concentration versus time curve from time 0 to 24 hours (AUC0-24)
Full Information
NCT ID
NCT05206773
First Posted
October 20, 2021
Last Updated
September 26, 2023
Sponsor
Genzyme, a Sanofi Company
1. Study Identification
Unique Protocol Identification Number
NCT05206773
Brief Title
A Study to Evaluate the Effect of Venglustat Tablets on Neuropathic and Abdominal Pain in Male and Female Adult Participants With Fabry Disease
Acronym
PERIDOT
Official 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
Study Type
Interventional
2. Study Status
Record Verification Date
September 2023
Overall Recruitment Status
Recruiting
Study Start Date
March 11, 2022 (Actual)
Primary Completion Date
November 30, 2024 (Anticipated)
Study Completion Date
July 8, 2026 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Genzyme, a Sanofi Company
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
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 adult participants 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.
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 13 months (12 month of treatment and 1 month of follow-up period)
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Fabry Disease
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantInvestigator
Masking Description
Participants enrolled in the open-label extension will be treated with venglustat only.
Allocation
Randomized
Enrollment
114 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Venglustat
Arm Type
Experimental
Arm Description
Participant will receive venglustat dose once daily up to 12 months
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Participants will receive placebo once daily up to 12 months
Intervention Type
Drug
Intervention Name(s)
Venglustat (GZ402671)
Intervention Description
Pharmaceutical form: Tablet Route of administration: Oral
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Pharmaceutical form: Tablet Route of administration: Oral
Primary Outcome Measure Information:
Title
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
Title
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
Secondary Outcome Measure Information:
Title
Percent change in plasma globotriaosylsphingosine (lyso-GL-3)
Time Frame
From baseline to 6 month and 12 months
Title
Frequency of rescue pain medication use
Description
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.
Time Frame
From baseline to 6 months and 12 months
Title
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
Title
Percent change in tiredness component of FD-PRO
Time Frame
From baseline to 6 month and 12 months
Title
Proportion of responders in neuropathic or abdominal pain, as assessed by FD-PRO
Description
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
Time Frame
At 6 months and 12 months
Title
Number of participants with adverse event (AE) and serious adverse event (SAE)
Time Frame
From baseline to 6 month and 12 months
Title
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
Title
Change in Beck Depression Inventory-II (BDI-II) score
Time Frame
From baseline to 6 month and 12 months
Title
Plasma venglustat concentrations at prespecified visits over the study duration
Time Frame
From baseline to 6 month and 12 months
Title
Maximum venglustat plasma concentration (Cmax)
Time Frame
From baseline to 6 month and 12 months
Title
Time to maximum venglustat plasma concentration (tmax)
Time Frame
From baseline to 6 month and 12 months
Title
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
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Male and female adult patients 18 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.
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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Trial Transparency email recommended (Toll free for US & Canada)
Phone
800-633-1610
Ext
option 6
Email
Contact-US@sanofi.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Clinical Sciences & Operations
Organizational Affiliation
Sanofi
Official's Role
Study Director
Facility Information:
Facility Name
Nephrology Clinic at Kirklin Clinic of UAB Hospital_Investigational Site Number: 8400011
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35233
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Maria El-Hachem
Phone
205-975-0549
Email
mehachem@uabmc.edu
First Name & Middle Initial & Last Name & Degree
Eric Wallace
Facility Name
UCLA Medical Center_Investigational Site Number: 8400006
City
Los Angeles
State/Province
California
ZIP/Postal Code
90095
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Anita Mkrttchyan
Phone
310-954-2692
Email
RastogiResearch@mednet.ucla.edu
First Name & Middle Initial & Last Name & Degree
Anjay Rastogi
Facility Name
Advent Health Orlando_Investigational Site Number: 8400008
City
Orlando
State/Province
Florida
ZIP/Postal Code
32804
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Estephanie Charria
Phone
407-609-1409
Email
Estephanie.Charria@AdventHealth.com
First Name & Middle Initial & Last Name & Degree
Majed Dasouki, Dr.
Facility Name
Oregon Health and Sciences University_Investigational Site Number: 8400007
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Amy Yang, MD
Facility Name
Renal Disease Research Institute, An affiliate of: Dallas Nephrology Associates_Investigational Site Number: 8400012
City
Dallas
State/Province
Texas
ZIP/Postal Code
75235
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Caren Swift
Phone
214-366-6094
Email
swiftc@dneph.com
First Name & Middle Initial & Last Name & Degree
Ankit Mehta, Dr.
Facility Name
Lysosomal and Rare Disorders Research and Treatment Center_Investigational Site Number: 8400004
City
Fairfax
State/Province
Virginia
ZIP/Postal Code
22030
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Omar Abu Slayeh, MD
Phone
571-407-7393
Email
oabuslayeh@ldrtc.org
First Name & Middle Initial & Last Name & Degree
Ozlam Goker-Alpan, MD
Facility Name
Fundacion Cori para la Investigación y Prevención del Cancer_Investigational Site Number: 0320002
City
La Rioja
ZIP/Postal Code
F5300
Country
Argentina
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Juan Marcelo Muraro
Phone
+54 9 380 468 7593
Email
Juanmuraro7@gmail.com
First Name & Middle Initial & Last Name & Degree
Juan Marcelo Muraro, MD
Facility Name
Instituto de Nefrologia Pergamino_Investigational Site Number: 0320001
City
Pergamino
ZIP/Postal Code
B2700CPM
Country
Argentina
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Norberto Antongiovanni
Phone
+54 9 2477 363658
Email
nantongiovanni1@yahoo.com.ar
First Name & Middle Initial & Last Name & Degree
Norberto Antongiovanni
Facility Name
Instituto de Investigaciones Clínicas Quilmes (IICQ) SRL_Investigational Site Number: 0320003
City
Quilmes
ZIP/Postal Code
B1878GEG
Country
Argentina
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Alberto Fernandez, Dr
Phone
+54116063-4883
Email
alberto.afernan@gmail.com
Phone
+549113580-4733
First Name & Middle Initial & Last Name & Degree
Alberto Fernandez, Dr
Facility Name
Investigational Site Number: 0400001
City
Wien
ZIP/Postal Code
1090
Country
Austria
Individual Site Status
Recruiting
Facility Name
Hospital de Clínicas de Porto Alegre_Investigational Site Number: 0760001
City
Porto Alegre
State/Province
Rio Grande Do Sul
ZIP/Postal Code
90035-903
Country
Brazil
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Roberto Giugliani, Dr.
Phone
+55 51 3359 6350
Email
rgigugliani@hcpa.edu.br
First Name & Middle Initial & Last Name & Degree
Backup Phone
Phone
+55 5133596343 /5133596340
First Name & Middle Initial & Last Name & Degree
Roberto Giugliani, Dr.
Facility Name
M.A.G.I.C Calgary LTD_Investigational Site Number: 1240003
City
Calgary
State/Province
Alberta
ZIP/Postal Code
T2E7Z4
Country
Canada
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Olugbenga Olunaike
Phone
587-885-1632
Email
clinic@magiccalgary.ca
First Name & Middle Initial & Last Name & Degree
Aneal Khan, MD
Facility Name
No.8, Xishiku Street, Xicheng District_Site Number: 1560001
City
Beijing
ZIP/Postal Code
100034
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Jinyue LUAN
Phone
18840728905
Email
jinyue.luan@globalaes.com
Facility Name
No.197,2nd Ruijin road, Huangpu district_Site Number: 1560003
City
Shanghai
ZIP/Postal Code
200010
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Xiangyu MENG
Phone
19892639307
Email
xiangyu.meng@globalaes.com
Facility Name
No.85 South Jiefang road, Yingze District_Site Number: 1560004
City
Taiyuan
ZIP/Postal Code
030001
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Shanting ZHANG
Phone
17703400422
Email
shaoting.zhang@globalaes.com
Facility Name
Investigational Site Number: 2080001
City
Copenhagen
ZIP/Postal Code
2100
Country
Denmark
Individual Site Status
Recruiting
Facility Name
Investigational Site Number: 2460001
City
Turku
ZIP/Postal Code
FI-20520
Country
Finland
Individual Site Status
Recruiting
Facility Name
ISphinCS GmbH_Investigational Site Number: 2760004
City
Hochheim Am Main
ZIP/Postal Code
65239
Country
Germany
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Eugen Mengel
Phone
+49 06146 904820
Email
eugen.mengel@sphincs.de
Facility Name
Investigational Site Number: 2760003
City
München
ZIP/Postal Code
80336
Country
Germany
Individual Site Status
Recruiting
Facility Name
Investigational Site Number: 2760001
City
Wurzburg
ZIP/Postal Code
97080
Country
Germany
Individual Site Status
Recruiting
Facility Name
University Hospital of Heraklion_Investigational Site Number: 3000001
City
Heraklion
ZIP/Postal Code
711 10
Country
Greece
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Konstantina Kiriakidi
Phone
+30 6944025099
Email
kiriakidi.konstantina@hotmail.com
First Name & Middle Initial & Last Name & Degree
Konstantinos Stylianou, Dr.
Facility Name
University Hospital of Ioannina_Investigational Site Number: 3000002
City
Ioannina
ZIP/Postal Code
455 00
Country
Greece
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Maria Spilia
Phone
+30 6986265705
Email
mariaspilia2@gmail.com
First Name & Middle Initial & Last Name & Degree
Evangelia Ntounousi, Dr.
Facility Name
Fondazione IRCCS San Gerardo dei Tintori, S.C. Nefrologia - Clinica Nefrologica_Investigational Site Number: 3800002
City
Monza
ZIP/Postal Code
20052
Country
Italy
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Barbara Trezzi, Dr
Email
barbara.trezzi@unimib.it
First Name & Middle Initial & Last Name & Degree
Federico Pieruzzi
Facility Name
Azienda Ospedaliera Universitaria "Federico II", U.O. di Nefrologia- Diparimento di Sanità Pubblica_Investigational Site Number: 3800001
City
Napoli
ZIP/Postal Code
80138
Country
Italy
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Antonio Pisani
Phone
+39 081 7464521
Email
antonio.pisai13@gmail.com
First Name & Middle Initial & Last Name & Degree
Eleonora Riccio
Phone
+39 3396770124
Email
eleonora.riccio83@gmail.com
First Name & Middle Initial & Last Name & Degree
Antonio Pisani
Facility Name
Azienda Ospedaliera Universitaria_Investigational Site Number: 3800003
City
Palermo
ZIP/Postal Code
90127
Country
Italy
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Irene Simonetta
Phone
+39 3476888073
Email
irene.simonetta@live.it
First Name & Middle Initial & Last Name & Degree
Antonino Tuttolomondo
Facility Name
Fondazione Policlinico Universitario_Investigational Site Number: 3800004
City
Roma
ZIP/Postal Code
00168
Country
Italy
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Elena Verrecchia, Dr
Phone
+39 0630159061
Email
elena.verrecchia@policlinicogemelli.it
First Name & Middle Initial & Last Name & Degree
Elena Verrecchia, Dr
Facility Name
Fukuoka University Hospital_Investigational Site Number: 3920004
City
Fukuoka-shi, Fukuoka
ZIP/Postal Code
814-0180
Country
Japan
Individual Site Status
Recruiting
Facility Name
Kagoshima University Hospital_Investigational Site Number: 3920002
City
Kagoshima
ZIP/Postal Code
890-8520
Country
Japan
Individual Site Status
Recruiting
Facility Name
The Jikei University Hospital_Investigational Site Number: 3920003
City
Minato-ku, Tokyo
ZIP/Postal Code
105-8471
Country
Japan
Individual Site Status
Recruiting
Facility Name
Tohoku University Hospital_Investigational Site Number: 3920001
City
Sendai-shi, Miyagi
ZIP/Postal Code
980-8574
Country
Japan
Individual Site Status
Recruiting
Facility Name
Hospital Universitario "Dr. José Eleuterio González" Departamento de Genética Centro Universitario contra el cáncer_Investigational Site Number: 4840001
City
Monterrey
State/Province
Nuevo León
ZIP/Postal Code
64460
Country
Mexico
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Marisol IBARRA-RAMIREZ
Phone
52-1-811-069-7182
Email
mibarrar@uanl.edu.mx
First Name & Middle Initial & Last Name & Degree
Marisol IBARRA-RAMIREZ
Facility Name
Odette del Carmen DIAZ-AVENDAÑO Clinstile, S.A. de C.V. Durango_Investigational Site Number: 4840002
City
Ciudad de Mexico
ZIP/Postal Code
06700
Country
Mexico
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Odette del Carmen DIAZ-AVENDAÑO
Phone
52-1-55-3200-2515
Email
oddimx@hotmail.com
First Name & Middle Initial & Last Name & Degree
Odette del Carmen DIAZ-AVENDAÑO
Facility Name
Investigational Site Number: 5780001
City
Bergen
ZIP/Postal Code
5021
Country
Norway
Individual Site Status
Recruiting
Facility Name
Investigational Site Number: 6160001
City
Lodz
ZIP/Postal Code
92-213
Country
Poland
Individual Site Status
Recruiting
Facility Name
Investigational Site Number: 6160003
City
Wroclaw
ZIP/Postal Code
50-556
Country
Poland
Individual Site Status
Recruiting
Facility Name
Institutul Clinic Fundeni_Investigational Site Number: 6420001
City
Bucuresti
ZIP/Postal Code
22328
Country
Romania
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Elena Emanuela, Dr.
Phone
+40 0723301828
Email
ela.rusu@gmail.com
Facility Name
Investigational Site Number: 8260001
City
Cambridge
ZIP/Postal Code
CB2 2QQ
Country
United Kingdom
Individual Site Status
Recruiting
Facility Name
Investigational Site Number: 8260002
City
London
ZIP/Postal Code
NW3 2QG
Country
United Kingdom
Individual Site Status
Recruiting
12. IPD Sharing Statement
Plan to Share IPD
Yes
IPD Sharing 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
Learn more about this trial
A Study to Evaluate the Effect of Venglustat Tablets on Neuropathic and Abdominal Pain in Male and Female Adult Participants With Fabry Disease
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