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A Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, and Safety of Venglustat in Late-onset GM2 (AMETHIST)

Primary Purpose

Tay-Sachs Disease, Sandhoff Disease

Status
Active
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
venglustat GZ402671
placebo
Sponsored by
Genzyme, a Sanofi Company
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Tay-Sachs Disease

Eligibility Criteria

2 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion criteria :

  • Primary population and adult secondary population: age ≥ 18 years
  • Juvenile/adolescent secondary population: 2 ≥ age < 18 years with weight ≥ 10 kg
  • Participants with a diagnosis of late onset GM2 gangliosidosis (Tay-Sachs disease and Sandhoff disease) caused by genetic β-hexosaminidase deficiency resulting from mutations in the HEXA or HEXB genes (primary population only); a secondary population will enroll patients with diagnosis of juvenile/adolescent GM2 gangliosidosis, GM1 gangliosidosis, saposin C deficiency, sialidosis type 1 or juvenile adult galactosialidosis
  • For primary population, the participant has the ability to perform the 9-HPT at the screening visit in < = 240 seconds for the 2 consecutive trials of the dominant hand and the 2 consecutive trials of the nondominant hand.
  • Participants with a history of seizures well controlled by medication other than strong or moderate inducer or inhibitor of CYP3A4
  • Participant is cooperative, able to ingest oral medication, willing to travel to a study site (if applicable), and able to comply with all aspects of the study, including all assessments, according to the Investigator's judgement
  • Signed written informed assent/consent
  • Contraception for sexually active male participants or female patient; not pregnant or breastfeeding; no sperm donating for male participant

Exclusion criteria:

  • Participant has clinical features of Tay-Sachs or Sandhoff disease, not caused by β-hexosaminidase deficiency resulting from mutations in the HEXA or HEXB genes and/or is without clinical features
  • For primary population and participants with juvenile/adolescent late onset GM2 gangliosidosis and GM1 gangliosidosis, the participant cannot understand and perform all age-appropriate study assessments with the exception of 25FWT and PROs.
  • Relevant medical disorders that would compromise his/her safety
  • Documented diagnosis of hepatitis B, C, human immunodeficiency virus 1 or 2
  • World Health Organization (WHO) grade >= 2 cortical cataract or a grade >= 2 posterior subcapsular cataract; patients with nuclear cataracts will be accepted
  • Participant who requires invasive ventilatory support
  • Current treatment by anticoagulants, cataractogenic medications or any medications that may worsen the vision of patient with cataract
  • Previous treatment with substrate reduction therapy (SRT) within 3 months prior to study enrollment, strong or moderate inducers or inhibitors of CYP3A4 within 14 days or 5 half-lives prior to enrollment. This also includes the consumption of grapefruit, grapefruit juice or grapefruit products within 72hrs prior to starting investigational medicinal product (IMP) administration.
  • Current participation in another study
  • Use of investigational medicinal product (IMP) within 3 months or 5 half-lives, whichever is longer, before study enrollment (for N-acetyl-leucine, within 5 half-lives before study enrollment).
  • Liver enzymes (alanine aminotransferase [ALT]/aspartate aminotransferase [AST]) or total bilirubin > 2 x the upper limite of normal (ULN) at the time of screening unless the participant has the diagnosis of Gilbert syndrome and maintains a level of bilirubin < 5 mg/dl and direct bilirubin < 20% (1 mg/dl) of total bilirubin level
  • Renal insufficiency is defined by estimated glomerular filtration rate (eGFR) < 30 mL/min/1.73m2 at the screening visit

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

Sites / Locations

  • Ataxia Center and HD Center of Excellence, 300 UCLA Med Plaza, Suite B200 - Investigational site number 8400004
  • Emory Genetics - Investigational site number 8400006
  • NIH National Human Genome Research Institute - 10 Center Dr - Bldg. 10 CRC3-2551 MSC 1205 - Investigational site number 8400005
  • Massachusetts General Hospital - Charles River Plaza 175 Cambridge St. Ste 340 - Investigational site number 8400002
  • NYU Langone - 550 First Avenue-Investigational site number 8400001
  • Hospital Universitario Austral Pilar, Buenos Aires_Unidad de Investigación Clínica_investigational site number 0320002
  • Clínica Universitaria Reina Fabiola Córdoba_investigational site number 0320001
  • AKH Wien_Universitätsklinik für Innere Medizin III Klinische Abteilung für Endokrinologie & Stoffwechsel Währinger Gürtel 18-20_investigational site number 0400001
  • Hospital de Clinicas de Porto Alegre _investigational site number 0760001
  • Vseobecna Fakultni Nemocnice V Praze Metabolicke centrum U Nemocnice 2_investigational site number 2030001
  • APHP - Centre Hospitalier la Pitié Salpetrière, Service de Neurologie, 47-83 Boulevard De l'Hôpital_Investigational site number 2500001
  • Universitätsklinikum der Justus-Liebig-Universität Gießen Zentrum für Kinderheilkunde und Jugendmedizin Feulgenstraße 10-12_investigational site number 2760001
  • Investigational Site Number 3800001
  • Japanese Red Cross Akita Hospital 222-1 Nawashirosawa, Kamikitatesaruta_investigational site number 3920001
  • Tohoku Medical and Pharmaceutical University Hospital_investigation site number 3920002
  • Centro Hospitalar Universitário Lisboa Norte- Hospital Santa Maria Avenida Professor Egas Moniz_investigational site number 6200002
  • Research Center Of Neurology 80, Volokolamskoye shosse_investigational site number 6430001
  • Hospital Maternoinfantil Sant Joan de Déu Paseo de Sant Joan de Déu, 2_investigational site number 7240001
  • Hospital Universitari de Bellvitge. Feixa Llarga, S / N_investigational site number 7240004
  • Hospital Clínico Universitario de Santiago-CHUS. Travesia de Chopuana, s/n_investigational site number 7240002
  • Gazi Universitesi Tip Fakultesi Emniyet Street Mevlana Blv Besevler Yenimahalle_investigational site number 7920001
  • Cambridge University Hospitals NHS Foundation Trust Addenbrookes Hospital Hills Road_investigational site number 8260001
  • Investigational Site Number 8260003
  • Salford Royal NHS Foundation Trust Salford Royal Hospital Stott Lane_investigational site number 8260002

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

GZ402671

Placebo

Arm Description

Primary population: participant will receive venglustat dose once daily during the primary analysis period (104 weeks) and the open-label extension period (104 weeks). Secondary population: participant will receive venglustat at various doses once daily during the primary analysis period open-label (104 weeks) and the open-label extension period (104 weeks).

Primary population: participants will receive placebo once daily during the primary analysis period (104 weeks) and will receive venglustat dose once daily during the open-label extension period (104 weeks).

Outcomes

Primary Outcome Measures

Change in cerebrospinal fluid (CSF) GM2 biomarker
Percent change in CSF GM2 biomarker from baseline to Week 104 in primary population
Change in the 9-hole pegboard test (9-HPT)
Annualized rate of change in the 9-HPT from baseline to Week 104 in primary population
Assessment of pharmacodynamic (PD) response in plasma: GL-1, GM1 biomarkers
Concentration of GL-1 biomarker and pathway specific biomarker GM1 for GM1 gangliosidosis in secondary population
Assessment of PD response in plasma: GL-1, GM2 biomarkers
Concentration of GL-1 and GM2 for GM2 gangliosidosis in secondary population
Assessment of PD response in plasma: GL-1, GM2, GM3 biomarkers
Concentration of GL-1 and GM2, GM3 for sialidosis in secondary population
Assessment of PD response in plasma: GL-1, GM1, GM3 biomarkers
Concentration of GL-1 and GM1, GM3 for galactosialidosis in secondary population
Assessment of PD response in plasma: GL-1 biomarker
Concentration of GL-1 for saposin C deficiency in secondary population
Assessment of PD response in CSF: GL-1, GM1 biomarkers
Concentration of GL-1 biomarker and pathway specific biomarker GM1 for GM1 gangliosidosis in secondary population
Assessment of PD response in CSF: GL-1, GM2 biomarkers
Concentration of GL-1 and GM2 for GM2 gangliosidosis in secondary population
Assessment of PD response in CSF: GL-1, GM2, GM3 biomarkers
Concentration of GL-1 and GM2, GM3 for sialidosis in secondary population
Assessment of PD response in CSF: GL-1, GM1, GM3 biomarkers
Concentration of GL-1 and GM1, GM3 for galactosialidosis in secondary population
Assessment of PD response in CSF: GL-1 biomarker
Concentration of GL-1 for saposin C deficiency in secondary population

Secondary Outcome Measures

Safety/tolerability: Adverse events
Number of patients with adverse events
Assessment of pharmacokinetic (PK) parameters in plasma: Cmax
Maximum venglustat concentration (Cmax)
Assessment of PK parameters in plasma: tmax
Time to maximum venglustat concentration (tmax)
Assessment of PK parameters in plasma: AUC0-24h
Concentration versus time curve calculated using the trapezoidal method from time 0 to 24 hours (AUC0-24h)
Assessment of PK parameters in plasma: plasma concentrations
Plasma venglustat concentration
Assessment of PK parameters: CSF venglustat concentration
CSF venglustat concentration
Change in 25-foot walk test (FWT)
Change in timed 25-FWT from baseline to Week 104 (in patients able to walk at baseline)
Change in the neurological examination of the Friedreich's Ataxia Rating Scale (FARS)
Change in the neurological examination of the FARS score from baseline to Week 104
Change in 9-hole peg test (9-HPT)
Annualized rate of change in the 9-HPT from baseline to Week 104 in secondary population

Full Information

First Posted
January 6, 2020
Last Updated
September 22, 2023
Sponsor
Genzyme, a Sanofi Company
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1. Study Identification

Unique Protocol Identification Number
NCT04221451
Brief Title
A Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, and Safety of Venglustat in Late-onset GM2
Acronym
AMETHIST
Official Title
A Multicenter, Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, Safety, and Tolerability of Venglustat in Late-onset GM2 Gangliosidosis (Tay-Sachs Disease and Sandhoff Disease) Together With a Separate Basket for Juvenile/Adolescent Late-onset GM2 Gangliosidosis and Ultra-rare Diseases Within the Same and Similar Glucosylceramide-based Sphingolipid Pathway
Study Type
Interventional

2. Study Status

Record Verification Date
September 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
June 29, 2020 (Actual)
Primary Completion Date
February 25, 2024 (Anticipated)
Study Completion Date
February 25, 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
Primary Objectives: Primary population (adult participants with late-onset GM2 gangliosidosis): To assess the efficacy and pharmacodynamics (PD) of daily oral dosing of venglustat when administered over a 104-week period Secondary population (participants with juvenile/adolescent late-onset GM2 gangliosidosis, GM1 gangliosidosis, saposin C deficiency, sialidosis type 1 or juvenile/adult galactosialidosis): To assess PD response (plasma and CSF GL-1 biomarker and disease specific biomarkers) of venglustat when administered once daily over a 104-week period Secondary Objectives: Primary population: To assess the effect of venglustat on selected performance test and scale over a 104-week period To determine the safety and tolerability of venglustat when administered orally once daily over a 104-week period To assess the pharmacokinetics (PK) of venglustat in plasma and cerebrospinal fluid (CSF) Secondary population: To assess the effect of venglustat on selected performance tests and scale over a 104-week period To determine the safety and tolerability of venglustat when administered once daily over a 104-week period To assess the PK of venglustat in plasma and CSF
Detailed Description
The total duration is up to approximately 223 weeks, including a 60-day screening period, a 104-week primary analysis treatment period, a 104-week open-label extension treatment period and a 6-week post-treatment safety observation period.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Tay-Sachs Disease, Sandhoff Disease

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
74 (Actual)

8. Arms, Groups, and Interventions

Arm Title
GZ402671
Arm Type
Experimental
Arm Description
Primary population: participant will receive venglustat dose once daily during the primary analysis period (104 weeks) and the open-label extension period (104 weeks). Secondary population: participant will receive venglustat at various doses once daily during the primary analysis period open-label (104 weeks) and the open-label extension period (104 weeks).
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Primary population: participants will receive placebo once daily during the primary analysis period (104 weeks) and will receive venglustat dose once daily during the open-label extension period (104 weeks).
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
Change in cerebrospinal fluid (CSF) GM2 biomarker
Description
Percent change in CSF GM2 biomarker from baseline to Week 104 in primary population
Time Frame
From baseline to Week 104
Title
Change in the 9-hole pegboard test (9-HPT)
Description
Annualized rate of change in the 9-HPT from baseline to Week 104 in primary population
Time Frame
From baseline to Week 104
Title
Assessment of pharmacodynamic (PD) response in plasma: GL-1, GM1 biomarkers
Description
Concentration of GL-1 biomarker and pathway specific biomarker GM1 for GM1 gangliosidosis in secondary population
Time Frame
From baseline to Week 104
Title
Assessment of PD response in plasma: GL-1, GM2 biomarkers
Description
Concentration of GL-1 and GM2 for GM2 gangliosidosis in secondary population
Time Frame
From baseline to Week 104
Title
Assessment of PD response in plasma: GL-1, GM2, GM3 biomarkers
Description
Concentration of GL-1 and GM2, GM3 for sialidosis in secondary population
Time Frame
From baseline to Week 104
Title
Assessment of PD response in plasma: GL-1, GM1, GM3 biomarkers
Description
Concentration of GL-1 and GM1, GM3 for galactosialidosis in secondary population
Time Frame
From baseline to Week 104
Title
Assessment of PD response in plasma: GL-1 biomarker
Description
Concentration of GL-1 for saposin C deficiency in secondary population
Time Frame
From baseline to Week 104
Title
Assessment of PD response in CSF: GL-1, GM1 biomarkers
Description
Concentration of GL-1 biomarker and pathway specific biomarker GM1 for GM1 gangliosidosis in secondary population
Time Frame
From baseline to Week 104
Title
Assessment of PD response in CSF: GL-1, GM2 biomarkers
Description
Concentration of GL-1 and GM2 for GM2 gangliosidosis in secondary population
Time Frame
From baseline to Week 104
Title
Assessment of PD response in CSF: GL-1, GM2, GM3 biomarkers
Description
Concentration of GL-1 and GM2, GM3 for sialidosis in secondary population
Time Frame
From baseline to Week 104
Title
Assessment of PD response in CSF: GL-1, GM1, GM3 biomarkers
Description
Concentration of GL-1 and GM1, GM3 for galactosialidosis in secondary population
Time Frame
From baseline to Week 104
Title
Assessment of PD response in CSF: GL-1 biomarker
Description
Concentration of GL-1 for saposin C deficiency in secondary population
Time Frame
From baseline to Week 104
Secondary Outcome Measure Information:
Title
Safety/tolerability: Adverse events
Description
Number of patients with adverse events
Time Frame
From baseline to Week 104
Title
Assessment of pharmacokinetic (PK) parameters in plasma: Cmax
Description
Maximum venglustat concentration (Cmax)
Time Frame
From baseline up to Week 12
Title
Assessment of PK parameters in plasma: tmax
Description
Time to maximum venglustat concentration (tmax)
Time Frame
From baseline up to Week 12
Title
Assessment of PK parameters in plasma: AUC0-24h
Description
Concentration versus time curve calculated using the trapezoidal method from time 0 to 24 hours (AUC0-24h)
Time Frame
From baseline up to Week 12
Title
Assessment of PK parameters in plasma: plasma concentrations
Description
Plasma venglustat concentration
Time Frame
From baseline up to Week 104
Title
Assessment of PK parameters: CSF venglustat concentration
Description
CSF venglustat concentration
Time Frame
Week 104
Title
Change in 25-foot walk test (FWT)
Description
Change in timed 25-FWT from baseline to Week 104 (in patients able to walk at baseline)
Time Frame
From baseline to Week 104
Title
Change in the neurological examination of the Friedreich's Ataxia Rating Scale (FARS)
Description
Change in the neurological examination of the FARS score from baseline to Week 104
Time Frame
From baseline to Week 104
Title
Change in 9-hole peg test (9-HPT)
Description
Annualized rate of change in the 9-HPT from baseline to Week 104 in secondary population
Time Frame
From baseline to Week 104

10. Eligibility

Sex
All
Minimum Age & Unit of Time
2 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion criteria : Primary population and adult secondary population: age ≥ 18 years Juvenile/adolescent secondary population: 2 ≥ age < 18 years with weight ≥ 10 kg Participants with a diagnosis of late onset GM2 gangliosidosis (Tay-Sachs disease and Sandhoff disease) caused by genetic β-hexosaminidase deficiency resulting from mutations in the HEXA or HEXB genes (primary population only); a secondary population will enroll patients with diagnosis of juvenile/adolescent GM2 gangliosidosis, GM1 gangliosidosis, saposin C deficiency, sialidosis type 1 or juvenile adult galactosialidosis For primary population, the participant has the ability to perform the 9-HPT at the screening visit in < = 240 seconds for the 2 consecutive trials of the dominant hand and the 2 consecutive trials of the nondominant hand. Participants with a history of seizures well controlled by medication other than strong or moderate inducer or inhibitor of CYP3A4 Participant is cooperative, able to ingest oral medication, willing to travel to a study site (if applicable), and able to comply with all aspects of the study, including all assessments, according to the Investigator's judgement Signed written informed assent/consent Contraception for sexually active male participants or female patient; not pregnant or breastfeeding; no sperm donating for male participant Exclusion criteria: Participant has clinical features of Tay-Sachs or Sandhoff disease, not caused by β-hexosaminidase deficiency resulting from mutations in the HEXA or HEXB genes and/or is without clinical features For primary population and participants with juvenile/adolescent late onset GM2 gangliosidosis and GM1 gangliosidosis, the participant cannot understand and perform all age-appropriate study assessments with the exception of 25FWT and PROs. Relevant medical disorders that would compromise his/her safety Documented diagnosis of hepatitis B, C, human immunodeficiency virus 1 or 2 World Health Organization (WHO) grade >= 2 cortical cataract or a grade >= 2 posterior subcapsular cataract; patients with nuclear cataracts will be accepted Participant who requires invasive ventilatory support Current treatment by anticoagulants, cataractogenic medications or any medications that may worsen the vision of patient with cataract Previous treatment with substrate reduction therapy (SRT) within 3 months prior to study enrollment, strong or moderate inducers or inhibitors of CYP3A4 within 14 days or 5 half-lives prior to enrollment. This also includes the consumption of grapefruit, grapefruit juice or grapefruit products within 72hrs prior to starting investigational medicinal product (IMP) administration. Current participation in another study Use of investigational medicinal product (IMP) within 3 months or 5 half-lives, whichever is longer, before study enrollment (for N-acetyl-leucine, within 5 half-lives before study enrollment). Liver enzymes (alanine aminotransferase [ALT]/aspartate aminotransferase [AST]) or total bilirubin > 2 x the upper limite of normal (ULN) at the time of screening unless the participant has the diagnosis of Gilbert syndrome and maintains a level of bilirubin < 5 mg/dl and direct bilirubin < 20% (1 mg/dl) of total bilirubin level Renal insufficiency is defined by estimated glomerular filtration rate (eGFR) < 30 mL/min/1.73m2 at the screening visit The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
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
Ataxia Center and HD Center of Excellence, 300 UCLA Med Plaza, Suite B200 - Investigational site number 8400004
City
Los Angeles
State/Province
California
ZIP/Postal Code
90095
Country
United States
Facility Name
Emory Genetics - Investigational site number 8400006
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30322
Country
United States
Facility Name
NIH National Human Genome Research Institute - 10 Center Dr - Bldg. 10 CRC3-2551 MSC 1205 - Investigational site number 8400005
City
Bethesda
State/Province
Maryland
ZIP/Postal Code
20892-1205
Country
United States
Facility Name
Massachusetts General Hospital - Charles River Plaza 175 Cambridge St. Ste 340 - Investigational site number 8400002
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02114
Country
United States
Facility Name
NYU Langone - 550 First Avenue-Investigational site number 8400001
City
New York
State/Province
New York
ZIP/Postal Code
10016
Country
United States
Facility Name
Hospital Universitario Austral Pilar, Buenos Aires_Unidad de Investigación Clínica_investigational site number 0320002
City
Buenos Aires
ZIP/Postal Code
B1629AHJ
Country
Argentina
Facility Name
Clínica Universitaria Reina Fabiola Córdoba_investigational site number 0320001
City
Córdoba
ZIP/Postal Code
X5004FHP
Country
Argentina
Facility Name
AKH Wien_Universitätsklinik für Innere Medizin III Klinische Abteilung für Endokrinologie & Stoffwechsel Währinger Gürtel 18-20_investigational site number 0400001
City
Wien
ZIP/Postal Code
1090
Country
Austria
Facility Name
Hospital de Clinicas de Porto Alegre _investigational site number 0760001
City
Porto Alegre
ZIP/Postal Code
90035-903
Country
Brazil
Facility Name
Vseobecna Fakultni Nemocnice V Praze Metabolicke centrum U Nemocnice 2_investigational site number 2030001
City
Praha 2
ZIP/Postal Code
12808
Country
Czechia
Facility Name
APHP - Centre Hospitalier la Pitié Salpetrière, Service de Neurologie, 47-83 Boulevard De l'Hôpital_Investigational site number 2500001
City
Paris
ZIP/Postal Code
Cedex 13-75651
Country
France
Facility Name
Universitätsklinikum der Justus-Liebig-Universität Gießen Zentrum für Kinderheilkunde und Jugendmedizin Feulgenstraße 10-12_investigational site number 2760001
City
Gießen
ZIP/Postal Code
35389
Country
Germany
Facility Name
Investigational Site Number 3800001
City
Milano
ZIP/Postal Code
20133
Country
Italy
Facility Name
Japanese Red Cross Akita Hospital 222-1 Nawashirosawa, Kamikitatesaruta_investigational site number 3920001
City
Akita-Shi
ZIP/Postal Code
010-1495
Country
Japan
Facility Name
Tohoku Medical and Pharmaceutical University Hospital_investigation site number 3920002
City
Sendai
ZIP/Postal Code
983-8512
Country
Japan
Facility Name
Centro Hospitalar Universitário Lisboa Norte- Hospital Santa Maria Avenida Professor Egas Moniz_investigational site number 6200002
City
Lisboa
ZIP/Postal Code
1649-035
Country
Portugal
Facility Name
Research Center Of Neurology 80, Volokolamskoye shosse_investigational site number 6430001
City
Moscow
ZIP/Postal Code
125367
Country
Russian Federation
Facility Name
Hospital Maternoinfantil Sant Joan de Déu Paseo de Sant Joan de Déu, 2_investigational site number 7240001
City
Esplugues De Llobregat
ZIP/Postal Code
08950
Country
Spain
Facility Name
Hospital Universitari de Bellvitge. Feixa Llarga, S / N_investigational site number 7240004
City
Hospitalet De Llobregat
ZIP/Postal Code
08907
Country
Spain
Facility Name
Hospital Clínico Universitario de Santiago-CHUS. Travesia de Chopuana, s/n_investigational site number 7240002
City
Santiago De Compostela
ZIP/Postal Code
15706
Country
Spain
Facility Name
Gazi Universitesi Tip Fakultesi Emniyet Street Mevlana Blv Besevler Yenimahalle_investigational site number 7920001
City
Ankara
ZIP/Postal Code
06500
Country
Turkey
Facility Name
Cambridge University Hospitals NHS Foundation Trust Addenbrookes Hospital Hills Road_investigational site number 8260001
City
Cambridge
ZIP/Postal Code
CB2 0QQ
Country
United Kingdom
Facility Name
Investigational Site Number 8260003
City
Manchester
ZIP/Postal Code
M13 9WL
Country
United Kingdom
Facility Name
Salford Royal NHS Foundation Trust Salford Royal Hospital Stott Lane_investigational site number 8260002
City
Salford
ZIP/Postal Code
M6 8HD
Country
United Kingdom

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
Citations:
PubMed Identifier
34539759
Citation
Nicoli ER, Annunziata I, d'Azzo A, Platt FM, Tifft CJ, Stepien KM. GM1 Gangliosidosis-A Mini-Review. Front Genet. 2021 Sep 3;12:734878. doi: 10.3389/fgene.2021.734878. eCollection 2021.
Results Reference
derived

Learn more about this trial

A Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, and Safety of Venglustat in Late-onset GM2

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