Trial · NCT02906020
A Global Study to Assess the Drug Dynamics, Efficacy, and Safety of Venglustat (GZ/SAR402671) in Parkinson's Disease Patients Carrying a Glucocerebrosidase (GBA) Gene Mutation
NCT02906020, Phase 2, Terminated, Genzyme, a Sanofi Company; results first posted 2022-02-28
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Global Study to Assess the Drug Dynamics, Efficacy, and Safety of Venglustat (GZ/SAR402671) in Parkinson's Disease Patients Carrying a Glucocerebrosidase (GBA) Gene Mutation. Trials, record NCT02906020. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02906020
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Global Study to Assess the Drug Dynamics, Efficacy, and Safety of Venglustat (GZ/SAR402671) in Parkinson's Disease Patients Carrying a Glucocerebrosidase (GBA) Gene Mutation
- NCT number
- NCT02906020
- Status
- Terminated
- Phases
- Phase 2
- Enrollment
- 273 participants, actual
- Lead sponsor
- Genzyme, a Sanofi Company
- Conditions
- Parkinson's Disease
- Interventions
- venglustat GZ/SAR402671, Placebo
- First posted
- 2016-09-19
- Last updated
- 2022-05-24
- Other registries' IDs
- eudract:2016-000657-12
- Results first posted
- 2022-02-28
- Limitations and caveats
- This study did not achieve its primary or secondary endpoints at the time of the analysis of the double-blind 52-week treatment period of Part 2 and was terminated early.
Source ClinicalTrials.gov
Registered outcomes15
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Part 1: Number of Participants With Treatment-Emergent Adverse Events (TEAEs), and Treatment-Emergent Serious Adverse Events (TESAEs) | From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants) | An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug and does not necessarily had to have a causal relationship with the treatment. Serious AEs (SAEs) were any untoward medical occurrence that resulted in any of the following outcomes: death, life-threatening, required initial or prolonged in-patient hospitalization, persistent or significant disability/incapacity, congenital anomaly/birth defect, or considered as medically important event. TEAEs were the AEs that developed or worsened or became serious during the TEAE period (defined as the period from the time of first investigational medicinal product \[IMP\] administration up of 6 weeks after the last administration of the IMP). |
| Primary | Part 1: Number of Participants With Abnormal Physical Examination Findings | From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants) | Physical examination included following observations/measurements: general appearance; heart, skin, respiratory auscultation; head, eyes, ears, nose, and throat, extremities/joints, and abdomen. New onset of abnormal physical examination was defined as a normal physical examination at Baseline and an abnormal physical examination during the treatment-emergent (TE) period (defined as the period from the time of first IMP administration up of 6 weeks after the last administration of the IMP). Abnormalities in physical examination were based on investigator's evaluation. |
| Primary | Part 1: Number of Participants With Abnormal Neurological Examination Findings | From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants) | Neurological examination included at least assessments of the participant's cranial nerves, motor system (including muscle atrophy, tone, and power), mental status, deep tendon reflex, sensation, and cerebellar function. Abnormalities in neurological examination were based on investigator's evaluation. |
| Primary | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants) | Criteria for potentially clinically significant abnormalities (PCSA): Hemoglobin: less than or equal to (\<=) 115 grams per liter (g/L) (Male\[M\]) or \<=95 g/L (Female\[F\]), greater than or equal to (\>=) 185 g/L (M) or \>=165 g/L (F), Decrease from baseline (DFB) \>=20 g/L; Hematocrit: \<=0.37 volume/volume (v/v) (M) or \<=0.32 v/v (F), \>=0.55 v/v (M) or \>=0.5 v/v (F); Red blood cells (RBC): \>=6 Tera/L; Platelets: less than (\<) 100 Giga/L, \>=700 Giga/L; White blood cells (WBC): \<3.0 Giga/L (Non-Black \[NB\]) or \<2.0 Giga/L (Black \[B\]), \>=16.0 Giga/L; Neutrophils: \<1.5 Giga/L (NB) or \<1.0 Giga/L (B); Lymphocytes: \<lower limit of normal (LLN), greater than (\>) 4.0 Giga/L; Monocytes: \<LLN, \>0.7 Giga/L; Basophils: \>0.1 Giga/L; Eosinophils: \>0.5 Giga/L or \>upper limit of normal (ULN) (if ULN \>=0.5 Giga/L). |
| Primary | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants) | Criteria for PCSA: Alanine Aminotransferase (ALT): \>3 ULN, \>5 ULN; Aspartate aminotransferase (AST): \>3 ULN; Alkaline phosphatase: \>1.5 ULN; Total Bilirubin: \>1.5 ULN; ALT and Bilirubin: \>3 ULN and \>2 ULN; Direct Bilirubin and Bilirubin: \>35% and \>1.5 ULN. |
| Primary | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Renal Parameters | From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants) | Criteria for PCSA: Creatinine: \>=150 micromoles per liter (mcmol/L) (adults), \>=30% change from baseline, \>=100% change from baseline; Blood urea nitrogen: \>=17 millimoles (mmol)/L. |
| Primary | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Metabolic Parameters | From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants) | Criteria for PCSA: Glucose: \<=3.9 mmol/L and \<LLN; \>=11.1 mmol/L (unfasted \[unfas\]) or \>=7 mmol/L (fasted \[fas\]); Albumin: \<=25 g/L. |
| Primary | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Electrolytes Parameters | From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants) | Criteria for PCSA: Sodium: \<=129 mmol/L, \>=160 mmol/L; Potassium: \<3 mmol/L, \>=5.5 mmol/L and Chloride: \<80 mmol/L, \>115 mmol/L. |
| Primary | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants) | Criteria for PCSA: Systolic blood pressure (SBP) supine: \<=95 millimeters of mercury (mmHg) and DFB \>=20 mmHg; \>=160 mmHg and increase from baseline (IFB) \>=20 mmHg; Diastolic blood pressure (DBP) supine: \<=45 mmHg and DFB \>=10 mmHg; \>=110 mmHg and IFB \>=10 mmHg; SBP (Orthostatic): \<=-20 mmHg; DBP (Orthostatic): \<=-10 mmHg; Heart rate (HR) supine: \<=50 beats per minute (bpm) and DFB \>=20 bpm; \>=120 bpm and IFB \>=20 bpm; Weight: \>=5% DFB; \>=5% IFB. |
| Primary | Part 1: Number of Participants With Potentially Clinically Significant Ophthalmological Abnormalities | From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants) | Clinically significant observations in left eye, right eye and any eye (any eye among left and right) were assessed by the investigator based on methods like visual acuity, slit lamp examination, examination of the cornea, lens, and retina. Any abnormal clinically significant ophthalmological examination finding (which was present at screening or not) on any eye during the treatment-emergent period corresponded to cornea verticillata, cataract and abnormal overall evaluation |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 registered outcomesOutcomes15
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Part 1: Number of Participants With Treatment-Emergent Adverse Events (TEAEs), and Treatment-Emergent Serious Adverse Events (TESAEs) | From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants) | Analysis was performed on safety population which included both non-Japanese (ROW) and Japanese participants who received at least 1 dose of study medication in Part 1 of the study. This outcome measure (OM) was planned to be analyzed and reported for Part 1 only and not for Part 2, as pre-specified in protocol. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Part 1: Number of Participants With Abnormal Physical Examination Findings | From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants) | Analysis was performed on safety population. This OM was planned to be analyzed and reported for Part 1 only and not for Part 2, as pre-specified in protocol. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Part 1: Number of Participants With Abnormal Neurological Examination Findings | From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants) | Analysis was performed on safety population. This OM was planned to be analyzed and reported for Part 1 only and not for Part 2, as pre-specified in protocol. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants) | Analysis was performed on safety population. This OM was planned to be analyzed and reported for Part 1 only and not for Part 2, as pre-specified in protocol. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants) | Analysis was performed on safety population. This OM was planned to be analyzed and reported for Part 1 only and not for Part 2, as pre-specified in protocol. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Renal Parameters | From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants) | Analysis was performed on safety population. This OM was planned to be analyzed and reported for Part 1 only and not for Part 2, as pre-specified in protocol. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Metabolic Parameters | From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants) | Analysis was performed on safety population. This OM was planned to be analyzed and reported for Part 1 only and not for Part 2, as pre-specified in protocol. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Electrolytes Parameters | From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants) | Analysis was performed on safety population. This OM was planned to be analyzed and reported for Part 1 only and not for Part 2, as pre-specified in protocol. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants) | Analysis was performed on safety population. This OM was planned to be analyzed and reported for Part 1 only and not for Part 2, as pre-specified in protocol. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Part 1: Number of Participants With Potentially Clinically Significant Ophthalmological Abnormalities | From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants) | Analysis was performed on safety population. This OM was planned to be analyzed and reported for Part 1 only and not for Part 2, as pre-specified in protocol. | Participants | COUNT_OF_PARTICIPANTS |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 outcomesResults by group664
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Part 1: Number of Participants With Treatment-Emergent Adverse Events (TEAEs), and Treatment-Emergent Serious Adverse Events (TESAEs) | Part 1: Placebo (ROW) | Any TEAE | 4 | Participants | |
| Part 1: Number of Participants With Treatment-Emergent Adverse Events (TEAEs), and Treatment-Emergent Serious Adverse Events (TESAEs) | Part 1: Venglustat 4 mg (ROW) | Any TEAE | 4 | Participants | |
| Part 1: Number of Participants With Treatment-Emergent Adverse Events (TEAEs), and Treatment-Emergent Serious Adverse Events (TESAEs) | Part 1: Venglustat 8 mg (ROW) | Any TEAE | 4 | Participants | |
| Part 1: Number of Participants With Treatment-Emergent Adverse Events (TEAEs), and Treatment-Emergent Serious Adverse Events (TESAEs) | Part 1: Venglustat 15 mg (ROW) | Any TEAE | 4 | Participants | |
| Part 1: Number of Participants With Treatment-Emergent Adverse Events (TEAEs), and Treatment-Emergent Serious Adverse Events (TESAEs) | Part 1: Placebo (Japan Only) | Any TEAE | 2 | Participants | |
| Part 1: Number of Participants With Treatment-Emergent Adverse Events (TEAEs), and Treatment-Emergent Serious Adverse Events (TESAEs) | Part 1: Venglustat 4 mg (Japan Only) | Any TEAE | 3 | Participants | |
| Part 1: Number of Participants With Treatment-Emergent Adverse Events (TEAEs), and Treatment-Emergent Serious Adverse Events (TESAEs) | Part 1: Venglustat 8 mg (Japan Only) | Any TEAE | 3 | Participants | |
| Part 1: Number of Participants With Treatment-Emergent Adverse Events (TEAEs), and Treatment-Emergent Serious Adverse Events (TESAEs) | Part 1: Venglustat 15 mg (Japan Only) | Any TEAE | 2 | Participants | |
| Part 1: Number of Participants With Treatment-Emergent Adverse Events (TEAEs), and Treatment-Emergent Serious Adverse Events (TESAEs) | Part 1: Placebo (ROW) | Any TESAE | 0 | Participants | |
| Part 1: Number of Participants With Treatment-Emergent Adverse Events (TEAEs), and Treatment-Emergent Serious Adverse Events (TESAEs) | Part 1: Venglustat 4 mg (ROW) | Any TESAE | 0 | Participants |
1 to 10 of 664Source ClinicalTrials.gov
See all 664 results by groupParticipant flow192
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Part 1: Dose Escalation Period | STARTED | Part 1: Placebo (ROW) | 4 | |
| Part 1: Dose Escalation Period | STARTED | Part 1: Venglustat 4 mg (ROW) | 4 | |
| Part 1: Dose Escalation Period | STARTED | Part 1: Venglustat 8 mg (ROW) | 5 | |
| Part 1: Dose Escalation Period | STARTED | Part 1: Venglustat 15 mg (ROW) | 4 | |
| Part 1: Dose Escalation Period | STARTED | Part 1: Placebo (Japan Only) | 3 | |
| Part 1: Dose Escalation Period | STARTED | Part 1: Venglustat 4 mg (Japan Only) | 3 | |
| Part 1: Dose Escalation Period | STARTED | Part 1: Venglustat 8 mg (Japan Only) | 3 | |
| Part 1: Dose Escalation Period | STARTED | Part 1: Venglustat 15 mg (Japan Only) | 3 | |
| Part 1: Dose Escalation Period | STARTED | Part 2, DB Period: Placebo | 0 | |
| Part 1: Dose Escalation Period | STARTED | Part 2, DB Period: Venglustat 15 mg | 0 |
1 to 10 of 192Source ClinicalTrials.gov
See all 192 participant flowAdverse events2,912
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Tinnitus | Ear and labyrinth disorders | Part 1: Placebo (ROW) | 0 | 4 | 0 |
| other | Tinnitus | Ear and labyrinth disorders | Part 1: Venglustat 4 mg (ROW) | 0 | 4 | 0 |
| other | Tinnitus | Ear and labyrinth disorders | Part 1: Venglustat 8 mg (ROW) | 0 | 5 | 0 |
| other | Tinnitus | Ear and labyrinth disorders | Part 1: Venglustat 15 mg (ROW) | 0 | 4 | 0 |
| other | Tinnitus | Ear and labyrinth disorders | Part 1: Placebo (Japan Only) | 0 | 3 | 0 |
| other | Tinnitus | Ear and labyrinth disorders | Part 1: Venglustat 4 mg (Japan Only) | 0 | 3 | 0 |
| other | Tinnitus | Ear and labyrinth disorders | Part 1: Venglustat 8 mg (Japan Only) | 0 | 3 | 0 |
| other | Tinnitus | Ear and labyrinth disorders | Part 1: Venglustat 15 mg (Japan Only) | 0 | 3 | 0 |
| other | Tinnitus | Ear and labyrinth disorders | Part 2, DB Period: Placebo | 1 | 111 | 1 |
| other | Tinnitus | Ear and labyrinth disorders | Part 2, DB Period: Venglustat 15 mg | 0 | 110 | 0 |
1 to 10 of 2,912Source ClinicalTrials.gov
See all 2,912 adverse eventsAdverse event totals48
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Part 1: Placebo (ROW) | 0 | 4 |
| Total, all-cause mortality | Part 1: Venglustat 4 mg (ROW) | 0 | 4 |
| Total, all-cause mortality | Part 1: Venglustat 8 mg (ROW) | 0 | 5 |
| Total, all-cause mortality | Part 1: Venglustat 15 mg (ROW) | 0 | 4 |
| Total, all-cause mortality | Part 1: Placebo (Japan Only) | 0 | 3 |
| Total, all-cause mortality | Part 1: Venglustat 4 mg (Japan Only) | 0 | 3 |
| Total, all-cause mortality | Part 1: Venglustat 8 mg (Japan Only) | 0 | 3 |
| Total, all-cause mortality | Part 1: Venglustat 15 mg (Japan Only) | 0 | 3 |
| Total, all-cause mortality | Part 2, DB Period: Placebo | 0 | 111 |
| Total, all-cause mortality | Part 2, DB Period: Venglustat 15 mg | 1 | 110 |
1 to 10 of 48Source ClinicalTrials.gov
See all 48 adverse event totalsPapers3
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests4
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
| Test | Named as | Source |
|---|---|---|
| Clock Drawing Task | Secondary outcome | ConductCognition |
| Delayed Recall Module | Secondary outcome | ConductCognition |
| Picture Naming Test | Secondary outcome | ConductCognition |
| Verbal Fluency | Secondary outcome | ConductCognition |
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT02906020
GET https://conductscience.org/api/trials/v1/NCT02906020/results?page=2