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| ID | Type | Description | Link |
|---|---|---|---|
| 2017-004084-12 | EudraCT Number | ||
| U1111-1202-0775 | Other Identifier | UTN |
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Interim analysis for futility of the Stage 1 of the EFC15392 study met the protocol specified stopping rule based on the primary endpoint. EFC15392 study was stopped for futility based on prespecified criteria and recommendation from DMC
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Primary Objective:
To determine the effect of venglustat on the rate of total kidney volume (TKV) growth (Stage 1) and estimated glomerular filtration rate (eGFR) decline in participants at risk of rapidly progressive Autosomal Dominant Polycystic Kidney Disease (ADPKD) (Stage 2).
Secondary Objectives:
Study duration per participant was 26 months (maximal) that included a screening period of 15 days, run-in period of 2 weeks, a 24-month treatment period, and a follow-up 30 days after final dose of investigational medicinal product (IMP).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Stage 1- Placebo | Placebo Comparator | Participants from Stage 1 were randomized to receive 2 capsules of placebo matched to venglustat once daily for treatment period of 24 months. |
|
| Stage 1- Venglustat 8 mg | Experimental | Participants from Stage 1 were randomized to receive venglustat 8 milligrams (mg) (i.e., 2 capsules of 4 mg) once daily for treatment period of 24 months. |
|
| Stage 1- Venglustat 15 mg | Experimental | Participants from Stage 1 were randomized to receive 1 capsule of venglustat 15 mg and 1 capsule of placebo matched to venglustat once daily for treatment period of 24 months. |
|
| Stage 2- Placebo | Placebo Comparator | Participants from Stage 2 were randomized to receive 1 capsule of placebo matched to venglustat once daily for treatment period of 24 months. |
|
| Stage 2- Venglustat 15 mg | Experimental | Participants from Stage 2 were randomized to receive 1 capsule of venglustat 15 mg once daily for treatment period of 24 months. |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Venglustat | Drug | Pharmaceutical form: capsule; Route of administration: oral |
|
| Measure | Description | Time Frame |
|---|---|---|
| Annualized Slope of Change in Total Kidney Volume (TKV) From Baseline to Month 18: Stage 1 | Total kidney volume is a measure for assessing disease progression in participants with ADPKD, a prognostic biomarker of renal function decline and progression to end-stage renal disease. Kidney volume was assessed using magnetic resonance imaging (MRI). The annualized slope of change in TKV (in percentage [%] per year) in each treatment group was obtained from the back-transformation of the mean slope of log10-transformed TKV obtained from the linear mixed effect model. The model included fix effects of treatment (venglustat 15 mg, venglustat 8 mg or placebo), mayo imaging classification (as per randomization stratification factor: class 1C versus 1D versus 1E), time (as continuous variable in years), treatment * time interaction and mayo imaging classification * time interaction and included random intercept and slope. | From Baseline to Month 18 |
| Annualized Rate of Change in Estimated Glomerular Filtration Rate (eGFR) (Chronic Kidney Disease Epidemiology Collaboration [CKD-EPI]) From Baseline to Month 24: Combined Stage 1 and Stage 2 | An eGFR was used to measure level of kidney function and determine the stage of kidney disease in ADPKD participants. Baseline eGFR was defined for each participant as the average of eGFR values assessed prior or equal to first dose of study drug or randomization for participants randomized and not exposed. Annualized rate of change in eGFR in each treatment group was obtained from the linear mixed effect model including the fixed categorical effects of treatment group (venglustat 15 mg, venglustat 8 mg or placebo), mayo imaging classification (as per interactive response technology [IRT]: 1C, 1D, 1E), time (as continuous variable in years), treatment-by-time, mayo imaging classification-by-time, and included random intercept and slope. | From Baseline to Month 24 |
| Measure | Description | Time Frame |
|---|---|---|
| Annualized Rate of Change in Estimated Glomerular Filtration Rate (eGFR) From Baseline to Month 24: Stage 1 | An eGFR was used to measure level of kidney function and determine the stage of kidney disease in ADPKD participants. Baseline eGFR was defined for each participant as the average of eGFR values assessed prior or equal to first dose of study drug or randomization for participants randomized and not exposed. Annualized rate of change in eGFR was obtained from the linear mixed effect model including the fixed categorical effects of treatment group, mayo imaging classification (as per IRT), time (as continuous variable in years), treatment-by-time, mayo imaging classification-by-time, and included random intercept and slope. Due to early termination of study for futility, the two-steps analysis initially planned was not applicable, then the annualized rate of change from baseline in eGFR in Stage 1 population were assessed using all data available up to database lock (i.e., including Month 24 assessment). As this is a slope, it allowed to have more data and to reduce variability. |
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Inclusion criteria:
Male or female adult with ADPKD with age at the time the consent was signed:
Diagnosis of ADPKD in participants with a family history would be based on unified Pei criteria. In the absence of a family history, the diagnosis would be based on the presence of renal cysts bilaterally, totaling at least 20, in the absence of findings suggestive of other cystic renal diseases.
Mayo Imaging Classification of ADPKD Class 1C, 1D or 1E**
**Total kidney volume (TKV) had confirmed by a central reader prior to Visit 3.
Estimated glomerular filtration rate between 45 to 89.9 mL/min/1.73 m^2 during screening period* (Chronic Kidney Disease Epidemiology Collaboration [CKD-EPI] equation) for Stage 1.
Estimated glomerular filtration rate between 30 to 89.9 mL/min/1.73 m^2 during screening period* (CKD-EPI equation) for Stage 2.
*Eligibility would be confirmed by eGFR value from one of the two first pre-randomization eGFR measurements.
Stable treatment regimen of antihypertensive therapy for at least 30 days prior to the screening visit for hypertensive participant.
Able to read, comprehend, and respond to the study questionnaires.
Participant had given voluntary written informed consent before performance of any study related procedures not part of standard medical care.
Participant had no access to tolvaptan at the time of study start or tolvaptan was not indicated for treatment of participant according to treating physician (participant does not meet recommended criteria for treatment, refuses to initiate or does not tolerate treatment with tolvaptan).
The participants, if female of childbearing potential, must have had a negative blood pregnancy test (β-human chorionic gonadotropin [β-hCG]) at the screening visit and a negative urine pregnancy test at the baseline visit.
Female participants of childbearing potential and male participants must have had agreed to practice true abstinence in line with their preferred and usual lifestyle or to use double-contraceptive methods (including a highly effective method of contraception for female participants of childbearing potential) for the entire duration of the study and for at least 6 weeks for females and 90 days for males following their last dose of study drug.
Exclusion criteria:
The above information was not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.
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| Name | Affiliation | Role |
|---|---|---|
| Clinical Sciences & Operations | Sanofi | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Investigational Site Number 8400002 | Birmingham | Alabama | 35294 | United States | ||
| Investigational Site Number 8400017 |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 36535535 | Result | Gansevoort RT, Hariri A, Minini P, Ahn C, Chapman AB, Horie S, Knebelmann B, Mrug M, Ong ACM, Pei YPC, Torres VE, Modur V, Antonshchuk I, Perrone RD. Venglustat, a Novel Glucosylceramide Synthase Inhibitor, in Patients at Risk of Rapidly Progressing ADPKD: Primary Results of a Double-Blind, Placebo-Controlled, Phase 2/3 Randomized Clinical Trial. Am J Kidney Dis. 2023 May;81(5):517-527.e1. doi: 10.1053/j.ajkd.2022.10.016. Epub 2022 Dec 17. | |
| 39356039 |
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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
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Participants who completed 24 months of treatment in either Stage 1 or Stage 2 of EFC15392 had option to enroll in an open-label long-term extension study LTS15823 (NCT ID: NCT04705051).
This study was conducted at 93 sites that enrolled participants in 23 countries. A total of 478 participants were enrolled from 04 October 2018 to 01 June 2021. Study was conducted in 2 stages: Stage 1 and Stage 2.
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| ID | Title | Description |
|---|---|---|
| FG000 | Stage 1- Placebo | Participants from Stage 1 were randomized to receive 2 capsules of placebo matched to venglustat once daily for treatment period of 24 months. |
| FG001 | Stage 1- Venglustat 8 mg |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Stage 1 (24 Months) |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Apr 19, 2021 | Jul 29, 2022 |
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| Placebo | Drug | Pharmaceutical form: capsule; Route of administration: oral |
|
| From Baseline to Month 24 |
| Annualized Slope of Change in Total Kidney Volume (TKV) From Baseline to Month 18: Combined Stage 1 and Stage 2 | Total kidney volume is a measure for assessing disease progression in participants with ADPKD, a prognostic biomarker of renal function decline and progression to end-stage renal disease. Kidney volume was assessed using MRI. The annualized slope of change in TKV (in % per year) in each treatment group was obtained from the back-transformation of the mean slope of log10-transformed TKV obtained from the linear mixed effect model. The model included fix effects of treatment (venglustat 15 mg, venglustat 8 mg or placebo), mayo imaging classification (as per randomization stratification factor: class 1C versus 1D versus 1E), time (as continuous variable in years), treatment * time interaction and mayo imaging classification * time interaction and included random intercept and slope. | From Baseline to Month 18 |
| Change in Pain Severity as Measured by Brief Pain Inventory-Short Form (BPI-SF)-Item 3 Scale Score From Baseline to Month 18: Stage 1 | The BPI-SF is a validated, self-administered questionnaire designed to measure a participant's perceived level of pain. The BPI-SF consisted of 15 items that use a numeric rating scale (NRS) to assess pain severity and pain interference in the past 24 hours and the past week. BPI-SF Item 3 asks participants to "Please rate your pain by marking the box beside the number that best describes your pain at its worst in the past 24 hours." The NRS ranged from 0 (no pain) to 10 (worst imaginable pain), where higher scores indicates greater intensity of pain. Least-squares (LS) means, and standard errors (SE) were estimated from mixed-effect model with repeated measures (MMRM) analysis. | From Baseline to Month 18 |
| Change in Pain Severity as Measured by Brief Pain Inventory-Short Form (BPI-SF)-Item 3 Scale Score From Baseline to Month 24: Combined Stage 1 and Stage 2 | The BPI-SF is a validated, self-administered questionnaire designed to measure a participant's perceived level of pain. The BPI-SF consisted of 15 items that use a NRS to assess pain severity and pain interference in the past 24 hours and the past week. BPI-SF Item 3 asks participants to "Please rate your pain by marking the box beside the number that best describes your pain at its worst in the past 24 hours." The NRS ranged from 0 (no pain) to 10 (worst imaginable pain), where higher scores indicate greater intensity of pain. LS means and SE were estimated from MMRM analysis. | From Baseline to Month 24 |
| Change in Fatigue Severity as Measured by Brief Fatigue Inventory (BFI-SF)-Item 3 Scale Score From Baseline to Month 18: Stage 1 | The BFI-SF is a 10-item, validated, self-administered questionnaire that was originally developed to assess fatigue severity. The 10-items were measured on a 0-10 scale, with 0 being 'does not interfere' and 10 being 'completely interferes.' BFI - Item 3 asks participants to 'Please rate your fatigue (weariness, tiredness) by circling the one number that best describes your worst level of fatigue during the past 24 hours. The NRS ranged from 0 (no fatigue) to 10 (worst imaginable fatigue). Higher global scores were associated with more severe fatigue. LS means and SE were estimated from MMRM analysis. | From Baseline to Month 18 |
| Change in Fatigue Severity as Measured by Brief Fatigue Inventory (BFI-SF)-Item 3 Scale Score From Baseline to Month 24: Combined Stage 1 and Stage 2 | The BFI-SF is a 10-item, validated, self-administered questionnaire that was originally developed to assess fatigue severity. The 10-items were measured on a 0-10 scale, with 0 being 'does not interfere' and 10 being 'completely interferes.' BFI - Item 3 asks participants to "Please rate your fatigue (weariness, tiredness) by circling the one number that best describes your worst level of fatigue during the past 24 hours. The NRS ranged from 0 (no fatigue) to 10 (worst imaginable fatigue). Higher global scores were associated with more severe fatigue. LS means and SE were estimated from MMRM analysis. | From Baseline to Month 24 |
| Pharmacokinetics: Plasma Concentration of Venglustat: Stage 1 | Venglustat plasma concentrations was determined using a validated liquid chromatography tandem mass spectrometry (LC-MS/MS) method. Data for this outcome measure was not planned to be collected and analyzed for placebo arm. | Day 1: 3 hours Post-Dose, Month 1: Pre-Dose and 3 hours Post-Dose, Month 6: Pre-Dose, Month 18: Pre-Dose |
| Pharmacokinetics: Plasma Concentration of Venglustat: Stage 2 | Venglustat plasma concentrations was determined using a validated LC-MS/MS method. Data for this outcome measure was not planned to be collected and analyzed for placebo arm. | Month 1: Pre-dose and 3 hours Post-dose |
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs): Stage 1 | An adverse event (AE) was any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with this treatment. A serious adverse event (SAE) was any untoward medical occurrence that at any dose: results in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect, or was a medically important event. TEAEs were AEs that developed, worsened or became serious during the treatment-emergent (TE) period (defined as the time from the first investigational medicinal product [IMP] administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). | From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier |
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs): Combined Stage 1 and Stage 2 | An AE was any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with this treatment. SAE was any untoward medical occurrence that at any dose: results in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect, or was a medically important event. TEAEs were AEs that developed, worsened or became serious during the TE period (defined as the time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). | From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier |
| Number of Participants With Potentially Clinically Significant Abnormalities: Hematology: Combined Stage 1 and Stage 2 | Criteria for potentially clinically significant abnormalities: Hemoglobin: less than or equal to (<=) 115 grams per liter (g/L) [Male]; <=95 g/L [Female]; greater than or equal to (>=) 185 g/L [Male]; >=165 g/L [Female]; Decrease from baseline >=20 g/L; Hematocrit: <=0.37 volume/volume (v/v) [Male]; <=0.32 v/v [Female]; >=0.55 v/v [Male]; >=0.5 v/v [Female]; Erythrocyte (red blood cells [RBC]): >=6*10^12 per liter (/L); Platelet: less than (<) 100*10^9/L; >=700*10^9/L; Leukocyte (white blood cells [WBC]): <3*10^9/L [Non-Black]; <2*10^9/L [Black], >=16*10^9/L; Neutrophils: <1.5*10^9/L [Non-Black]; <1*10^9/L [Black]; Lymphocytes: greater than (>) 4*10^9/L, Monocytes: >0.7*10^9/L; Basophils: >0.1*10^9/L; and Eosinophils: >0.5*10^9/L or >upper limit of normal (ULN) (if ULN >=0.5*10^9/L). | From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier |
| Number of Participants With Potentially Clinically Significant Abnormalities: Clinical Chemistry: Combined Stage 1 and Stage 2 | Criteria for potentially clinically significant abnormalities: Glucose: <=3.9 millimoles per liter (mmol/L) and <lower limit of normal (LLN): >=11.1 mmol/L (unfasted); >=7 mmol/L (fasted); Albumin:<=25 g/L; Sodium: <=129 mmol/L; >=160 mmol/L; Potassium: <3 mmol/L; >=5.5 mmol/L; Chloride: <80 mmol/L, >115 mmol/L; Creatinine: >=150 micro millimoles per liter (mcmol/L) (Adults); >=30% change from Baseline; >=100% change from Baseline, Urea Nitrogen: >=17 mmol/L; Alanine Aminotransferase (ALT): >3 ULN; Aspartate Aminotransferase (AST): >3 ULN; Alkaline Phosphatase: >1.5 ULN; Total Bilirubin: >1.5 ULN, >2 ULN; ALT >3 ULN and Bilirubin >2 ULN; and Direct Bilirubin >35% Bilirubin and Bilirubin >1.5 ULN. | From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier |
| Number of Participants With Potentially and Clinically Significant Abnormalities: Urinalysis: Combined Stage 1 and Stage 2 | Criteria for potentially clinically significant abnormalities: Urine pH: <=4.6 and >=8. | From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier |
| Number of Participants With Potentially and Clinically Significant Abnormalities: Vital Signs: Combined Stage 1 and Stage 2 | Criteria for potentially clinically significant abnormalities: Sitting Systolic Blood Pressure: <=95 millimeters of Mercury (mmHg) and decrease from Baseline >=20 mmHg; >=160 mmHg and increase from Baseline >=20 mmHg; Sitting Diastolic Blood Pressure: <=45 mmHg and decrease from Baseline >=10 mmHg, >=110 mmHg and increase from Baseline >=10 mmHg; Sitting Heart Rate: <=50 beats/minute and decrease from Baseline >=20 beats/minute; >=120 beats/minute and increase from Baseline >=20 beats/minute; and Weight: >=5% decrease from Baseline; >=5% increase from Baseline. | From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier |
| Number of Participants With Potentially Clinically Significant Abnormalities: Electrocardiogram: Combined Stage 1 and Stage 2 | Criteria for potentially clinically significant abnormalities: Heart Rate: <50 beats/minute; <50 beats/minute and decrease from Baseline >=20 beats/minute; <40 beats/minute; <40 beats/minute and decrease from Baseline >=20 beats/min; <30 beats/minute; >90 beats/minute; >90 beats/minute and increase from Baseline >=20 beats/minute; >100 beats/minute; >100 beats/minute and increase from Baseline >=20 beats/minute; >120 beats/minute; >120 beats/minute, increase from Baseline >=20 beats/minute; PR Interval: >200 milliseconds (msec); >200 msec and increase from Baseline >=25%; >220 msec, >240 msec; QRS Interval: >110 msec; >110 msec and increase from Baseline >=25%; >120 msec; >120 msec and increase from Baseline >=25%; QT Interval: >500 msec; QT corrected for heart rate (QTc) Bazett: >450 msec; >480 msec; increase from Baseline (30-60) msec; increase from Baseline >60 msec; QTc Fridericia: >450 msec; >480 msec; increase from Baseline (30-60) msec and increase from Baseline > 60 msec. | From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier |
| Number of Participants With Physical Examination Abnormalities: Combined Stage 1 and Stage 2 | Physical examination included: Head, Heart, Lung, Abdomen, Musculo/Skeletal, Skin, and Mental Status. Abnormality in physical examination was based on investigator's discretion. Results are based on the treatment emergent period which was defined as time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier. | Baseline, Month 18, Month 24 |
| Change From Baseline in Beck Depression Inventory-II (BDI-II) Score: Combined Stage 1 and Stage 2 | The Beck Depression Inventory-II (BDI-II) is a 21-item questionnaire used to assess depression. Most items are rated on a 4-point scale from 0 to 3, and a few items are rated on a 7-point scale. Individual item scores are added to get a total BDI-II score from 0 to 63. The higher the total score, the more severe the depression, and the lower the total score, the less severe the depression. | Baseline, Months 3, 6, 9, 12, 15, 18, 21, and 24, Last on-treatment value up to last IMP + 1 day (anytime during the maximum duration of 25 months) |
| Number of Participants With Worsening Lens Opacity From Baseline During the Treatment-emergent Period: Combined Stage 1 and Stage 2 | Worsening of lens opacity classification system (LOCS) III score or World Health Organization (WHO) grade in nuclear opacification, cortical opacification and posterior subcapsular opacification were assessed for 'Any eye', 'Unilateral' and 'Bilateral' separately. A participant could be counted in all the 3 categories. In each category, the worst case was taken into account. To be evaluable for 'Any', a participant had to have at least one eye evaluable, whereas, for 'Unilateral' and 'Bilateral', a participant had to have both eyes evaluable. Therefore, the sum of 'Unilateral' + 'Bilateral' is not necessarily equal to 'Any' in the below table. The difference observed in 'Nuclear Opacification' in 15 mg group, comes from that 1 participant who had Unilateral worsening at a given visit and a Bilateral worsening at another visit. Therefore, this participant was counted as 'Unilateral', 'Bilateral' and counted only once in 'Any'. Results are based on the TE period. | From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier |
| Los Angeles |
| California |
| 90024 |
| United States |
| Investigational Site Number 8400001 | San Francisco | California | 94143 | United States |
| Investigational Site Number 8400008 | Aurora | Colorado | 80045 | United States |
| Investigational Site Number 8400010 | New Haven | Connecticut | 06510 | United States |
| Investigational Site Number 8400004 | Atlanta | Georgia | 30322 | United States |
| Investigational Site Number 8400007 | Chicago | Illinois | 60637 | United States |
| Investigational Site Number 8400014 | Iowa City | Iowa | 52242 | United States |
| Investigational Site Number 8400003 | Kansas City | Kansas | 66103 | United States |
| Investigational Site Number 8400021 | Baltimore | Maryland | 21201 | United States |
| Investigational Site Number 8400016 | Boston | Massachusetts | 02111 | United States |
| Investigational Site Number 8400020 | Rochester | Minnesota | 55905 | United States |
| Investigational Site Number 8400027 | Kansas City | Missouri | 64111 | United States |
| Investigational Site Number 8400011 | Philadelphia | Pennsylvania | 19104 | United States |
| Investigational Site Number 8400015 | San Antonio | Texas | 78215 | United States |
| Investigational Site Number 8400019 | Morgantown | West Virginia | 26506 | United States |
| Investigational Site Number 8400005 | Madison | Wisconsin | 53792 | United States |
| Investigational Site Number 8400006 | Milwaukee | Wisconsin | 53226 | United States |
| Investigational Site Number 0320001 | Buenos Aires | C1429BWN | Argentina |
| Investigational Site Number 0320003 | Santa Fe | S3000EPV | Argentina |
| Investigational Site Number 0360002 | Herston | 4029 | Australia |
| Investigational Site Number 0360003 | Nedlands | 6009 | Australia |
| Investigational Site Number 0360001 | Westmead | 2145 | Australia |
| Investigational Site Number 0400001 | Graz | 8036 | Austria |
| Investigational Site Number 0400004 | Vienna | 1090 | Austria |
| Investigational Site Number 0560001 | Brussels | 1200 | Belgium |
| Investigational Site Number 0560002 | Leuven | 3000 | Belgium |
| Investigational Site Number 1240002 | Edmonton | T6G 2B7 | Canada |
| Investigational Site Number 1240003 | Montreal | H2W 1R7 | Canada |
| Investigational Site Number 1240001 | Toronto | M5G 2N2 | Canada |
| Investigational Site Number 1560005 | Beijing | 100853 | China |
| Investigational Site Number 1560004 | Chengdu | 610041 | China |
| Investigational Site Number 1560009 | Guangzhou | 510080 | China |
| Investigational Site Number 1560006 | Hangzhou | 310003 | China |
| Investigational Site Number 1560002 | Hefei | 230022 | China |
| Investigational Site Number 1560007 | Nanjing | 210009 | China |
| Investigational Site Number 1560008 | Nanjing | 210029 | China |
| Investigational Site Number 1560001 | Shanghai | 200003 | China |
| Investigational Site Number 1560003 | Shenyang | 110004 | China |
| Investigational Site Number 2030001 | Prague | 12808 | Czechia |
| Investigational Site Number 2030002 | Prague | 14021 | Czechia |
| Investigational Site Number 2080001 | Copenhagen | 2100 | Denmark |
| Investigational Site Number 2080002 | Roskilde | 4000 | Denmark |
| Investigational Site Number 2500004 | Bordeaux | 33076 | France |
| Investigational Site Number 2500003 | Brest | 29609 | France |
| Investigational Site Number 2500002 | Paris | 75015 | France |
| Investigational Site Number 2500001 | Toulouse | 31403 | France |
| Investigational Site Number 2760001 | Berlin | 10117 | Germany |
| Investigational Site Number 2760003 | Cologne | 50937 | Germany |
| Investigational Site Number 2760002 | Dresden | 01307 | Germany |
| Investigational Site Number 2760010 | Dresden | 01307 | Germany |
| Investigational Site Number 2760007 | Düsseldorf | 40210 | Germany |
| Investigational Site Number 2760009 | Essen | 45122 | Germany |
| Investigational Site Number 2760005 | Hanover | 30625 | Germany |
| Investigational Site Number 2760011 | Leipzig | 04103 | Germany |
| Investigational Site Number 2760012 | Mainz | 55131 | Germany |
| Investigational Site Number 2760004 | München | 81675 | Germany |
| Investigational Site Number 3760003 | Ashdod | 7747629 | Israel |
| Investigational Site Number 3760002 | Rehovot | 76100 | Israel |
| Investigational Site Number 3800001 | Brescia | 25123 | Italy |
| Investigational Site Number 3800002 | Milan | 20132 | Italy |
| Investigational Site Number 3800003 | Naples | 80131 | Italy |
| Investigational Site Number 3920002 | Bunkyō City | Japan |
| Investigational Site Number 3920005 | Kamakura-Shi | Japan |
| Investigational Site Number 3920006 | Kawasaki-Shi | Japan |
| Investigational Site Number 3920010 | Kyoto | Japan |
| Investigational Site Number 3920009 | Nagoya | Japan |
| Investigational Site Number 3920003 | Niigata | Japan |
| Investigational Site Number 3920007 | Osaka | Japan |
| Investigational Site Number 3920001 | Sapporo | Japan |
| Investigational Site Number 3920004 | Shinjuku-Ku | Japan |
| Investigational Site Number 3920008 | Toyoake-Shi | Japan |
| Investigational Site Number 5280003 | Amsterdam | 1105AZ | Netherlands |
| Investigational Site Number 5280001 | Groningen | 9713 GZ | Netherlands |
| Investigational Site Number 5280002 | Nijmegen | 6525 GA | Netherlands |
| Investigational Site Number 6160001 | Lodz | 92-213 | Poland |
| Investigational Site Number 6160003 | Warsaw | 04-141 | Poland |
| Investigational Site Number 6160002 | Wroclaw | 50-556 | Poland |
| Investigational Site Number 6200004 | Almada | 2801-951 | Portugal |
| Investigational Site Number 6200005 | Carnaxide | 2790-134 | Portugal |
| Investigational Site Number 6200001 | Loures | 2674-514 | Portugal |
| Investigational Site Number 6420002 | Bucharest | 022328 | Romania |
| Investigational Site Number 6420004 | Oradea | 410469 | Romania |
| Investigational Site Number 6420001 | Timișoara | 300723 | Romania |
| Investigational Site Number 4100001 | Seoul | 03080 | South Korea |
| Investigational Site Number 4100002 | Seoul | 07061 | South Korea |
| Investigational Site Number 7240003 | Barcelona | 08003 | Spain |
| Investigational Site Number 7240001 | Barcelona | 08025 | Spain |
| Investigational Site Number 7240002 | Madrid | 28040 | Spain |
| Investigational Site Number 1580001 | Taichung | 40447 | Taiwan |
| Investigational Site Number 1580002 | Taipei | 10043 | Taiwan |
| Investigational Site Number 7920001 | Istanbul | Turkey (Türkiye) |
| Investigational Site Number 7920002 | Kayseri | 38039 | Turkey (Türkiye) |
| Investigational Site Number 7920003 | Kocaeli | 41380 | Turkey (Türkiye) |
| Investigational Site Number 8260001 | Sheffield | S5 7AU | United Kingdom |
| Derived |
| St Pierre K, Cashmore BA, Bolignano D, Zoccali C, Ruospo M, Craig JC, Strippoli GF, Mallett AJ, Green SC, Tunnicliffe DJ. Interventions for preventing the progression of autosomal dominant polycystic kidney disease. Cochrane Database Syst Rev. 2024 Oct 2;10(10):CD010294. doi: 10.1002/14651858.CD010294.pub3. |
| 36204243 | Derived | Perrone RD, Hariri A, Minini P, Ahn C, Chapman AB, Horie S, Knebelmann B, Mrug M, Ong ACM, Pei YPC, Torres VE, Modur V, Gansevoort RT. The STAGED-PKD 2-Stage Adaptive Study With a Patient Enrichment Strategy and Treatment Effect Modeling for Improved Study Design Efficiency in Patients With ADPKD. Kidney Med. 2022 Aug 27;4(10):100538. doi: 10.1016/j.xkme.2022.100538. eCollection 2022 Oct. |
Participants from Stage 1 were randomized to receive venglustat 8 milligrams (mg) (i.e., 2 capsules of 4 mg) once daily for treatment period of 24 months.
| FG002 | Stage 1- Venglustat 15 mg | Participants from Stage 1 were randomized to receive 1 capsule of venglustat 15 mg and 1 capsule of placebo matched to venglustat once daily for treatment period of 24 months. |
| FG003 | Stage 2- Placebo | Participants from Stage 2 were randomized to receive 1 capsule of placebo matched to venglustat once daily for treatment period of 24 months. |
| FG004 | Stage 2- Venglustat 15 mg | Participants from Stage 2 were randomized to receive 1 capsule of venglustat 15 mg once daily for treatment period of 24 months. |
| COMPLETED |
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| NOT COMPLETED |
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| Stage 2 (24 Months) |
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Analysis was performed on all participants randomized in the study.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Stage 1- Placebo | Participants from Stage 1 were randomized to receive 2 capsules of placebo matched to venglustat once daily for treatment period of 24 months. |
| BG001 | Stage 1- Venglustat 8 mg | Participants from Stage 1 were randomized to receive venglustat 8 mg (i.e., 2 capsules of 4 mg) once daily for treatment period of 24 months. |
| BG002 | Stage 1- Venglustat 15 mg | Participants from Stage 1 were randomized to receive 1 capsule of venglustat 15 mg and 1 capsule of placebo matched to venglustat once daily for treatment period of 24 months. |
| BG003 | Stage 2- Placebo | Participants from Stage 2 were randomized to receive 1 capsule of placebo matched to venglustat once daily for treatment period of 24 months. |
| BG004 | Stage 2- Venglustat 15 mg | Participants from Stage 2 were randomized to receive 1 capsule of venglustat 15 mg once daily for treatment period of 24 months. |
| BG005 | Total Title |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
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| Age, Continuous | Mean | Standard Deviation | years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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| Primary | Annualized Slope of Change in Total Kidney Volume (TKV) From Baseline to Month 18: Stage 1 | Total kidney volume is a measure for assessing disease progression in participants with ADPKD, a prognostic biomarker of renal function decline and progression to end-stage renal disease. Kidney volume was assessed using magnetic resonance imaging (MRI). The annualized slope of change in TKV (in percentage [%] per year) in each treatment group was obtained from the back-transformation of the mean slope of log10-transformed TKV obtained from the linear mixed effect model. The model included fix effects of treatment (venglustat 15 mg, venglustat 8 mg or placebo), mayo imaging classification (as per randomization stratification factor: class 1C versus 1D versus 1E), time (as continuous variable in years), treatment * time interaction and mayo imaging classification * time interaction and included random intercept and slope. | Analysis was performed on Stage 1 intent-to-treat (ITT) population that includes all participants randomized in Stage 1 and were analyzed according to the intervention allocated by randomization (venglustat 15 mg, venglustat 8 mg or placebo). Here, 'Overall number of participants analyzed' signifies participants who were evaluable for the outcome measure. | Posted | Geometric Least Squares Mean | 95% Confidence Interval | percent change in TKV/year | From Baseline to Month 18 |
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| Primary | Annualized Rate of Change in Estimated Glomerular Filtration Rate (eGFR) (Chronic Kidney Disease Epidemiology Collaboration [CKD-EPI]) From Baseline to Month 24: Combined Stage 1 and Stage 2 | An eGFR was used to measure level of kidney function and determine the stage of kidney disease in ADPKD participants. Baseline eGFR was defined for each participant as the average of eGFR values assessed prior or equal to first dose of study drug or randomization for participants randomized and not exposed. Annualized rate of change in eGFR in each treatment group was obtained from the linear mixed effect model including the fixed categorical effects of treatment group (venglustat 15 mg, venglustat 8 mg or placebo), mayo imaging classification (as per interactive response technology [IRT]: 1C, 1D, 1E), time (as continuous variable in years), treatment-by-time, mayo imaging classification-by-time, and included random intercept and slope. | Analysis was performed on Combined Stage 1 and Stage 2 ITT population that included all participants with an eGFR between 45 and 89.9 mL/min/1.73 m^2 at screening who are randomized in Stage 1 or Stage 2 and were analyzed according to the intervention allocated by randomization (venglustat 15 mg, venglustat 8 mg or placebo). Data for this outcome measure (OM) was planned to be collected and analyzed for combined Stage 1 and Stage 2 population as pre-specified in protocol. | Posted | Least Squares Mean | 95% Confidence Interval | mL/min/1.73 m^2/year | From Baseline to Month 24 |
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| Secondary | Annualized Rate of Change in Estimated Glomerular Filtration Rate (eGFR) From Baseline to Month 24: Stage 1 | An eGFR was used to measure level of kidney function and determine the stage of kidney disease in ADPKD participants. Baseline eGFR was defined for each participant as the average of eGFR values assessed prior or equal to first dose of study drug or randomization for participants randomized and not exposed. Annualized rate of change in eGFR was obtained from the linear mixed effect model including the fixed categorical effects of treatment group, mayo imaging classification (as per IRT), time (as continuous variable in years), treatment-by-time, mayo imaging classification-by-time, and included random intercept and slope. Due to early termination of study for futility, the two-steps analysis initially planned was not applicable, then the annualized rate of change from baseline in eGFR in Stage 1 population were assessed using all data available up to database lock (i.e., including Month 24 assessment). As this is a slope, it allowed to have more data and to reduce variability. | Analysis was performed on Stage 1 ITT population. | Posted | Least Squares Mean | 95% Confidence Interval | mL/min/1.73 m^2/year | From Baseline to Month 24 |
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| Secondary | Annualized Slope of Change in Total Kidney Volume (TKV) From Baseline to Month 18: Combined Stage 1 and Stage 2 | Total kidney volume is a measure for assessing disease progression in participants with ADPKD, a prognostic biomarker of renal function decline and progression to end-stage renal disease. Kidney volume was assessed using MRI. The annualized slope of change in TKV (in % per year) in each treatment group was obtained from the back-transformation of the mean slope of log10-transformed TKV obtained from the linear mixed effect model. The model included fix effects of treatment (venglustat 15 mg, venglustat 8 mg or placebo), mayo imaging classification (as per randomization stratification factor: class 1C versus 1D versus 1E), time (as continuous variable in years), treatment * time interaction and mayo imaging classification * time interaction and included random intercept and slope. | As no post-baseline MRI data were collected in Stage 2 due to early study discontinuation, the analysis of TKV in Combined Stage 1 and Stage 2 was not done. | Posted | From Baseline to Month 18 |
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| Secondary | Change in Pain Severity as Measured by Brief Pain Inventory-Short Form (BPI-SF)-Item 3 Scale Score From Baseline to Month 18: Stage 1 | The BPI-SF is a validated, self-administered questionnaire designed to measure a participant's perceived level of pain. The BPI-SF consisted of 15 items that use a numeric rating scale (NRS) to assess pain severity and pain interference in the past 24 hours and the past week. BPI-SF Item 3 asks participants to "Please rate your pain by marking the box beside the number that best describes your pain at its worst in the past 24 hours." The NRS ranged from 0 (no pain) to 10 (worst imaginable pain), where higher scores indicates greater intensity of pain. Least-squares (LS) means, and standard errors (SE) were estimated from mixed-effect model with repeated measures (MMRM) analysis. | Analysis was performed on Stage 1 ITT population. Here, 'Overall number of participants analyzed' signifies participants who were evaluable for the outcome measure. | Posted | Least Squares Mean | Standard Error | score on a scale | From Baseline to Month 18 |
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| Secondary | Change in Pain Severity as Measured by Brief Pain Inventory-Short Form (BPI-SF)-Item 3 Scale Score From Baseline to Month 24: Combined Stage 1 and Stage 2 | The BPI-SF is a validated, self-administered questionnaire designed to measure a participant's perceived level of pain. The BPI-SF consisted of 15 items that use a NRS to assess pain severity and pain interference in the past 24 hours and the past week. BPI-SF Item 3 asks participants to "Please rate your pain by marking the box beside the number that best describes your pain at its worst in the past 24 hours." The NRS ranged from 0 (no pain) to 10 (worst imaginable pain), where higher scores indicate greater intensity of pain. LS means and SE were estimated from MMRM analysis. | Analysis was performed on Combined Stage 1 and Stage 2 ITT population. Here, 'Overall number of participants analyzed' signifies participants who were evaluable for the outcome measure. Data for this OM was planned to be collected and analyzed for combined Stage 1 and Stage 2 population as pre-specified in protocol. | Posted | Least Squares Mean | Standard Error | score on a scale | From Baseline to Month 24 |
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| Secondary | Change in Fatigue Severity as Measured by Brief Fatigue Inventory (BFI-SF)-Item 3 Scale Score From Baseline to Month 18: Stage 1 | The BFI-SF is a 10-item, validated, self-administered questionnaire that was originally developed to assess fatigue severity. The 10-items were measured on a 0-10 scale, with 0 being 'does not interfere' and 10 being 'completely interferes.' BFI - Item 3 asks participants to 'Please rate your fatigue (weariness, tiredness) by circling the one number that best describes your worst level of fatigue during the past 24 hours. The NRS ranged from 0 (no fatigue) to 10 (worst imaginable fatigue). Higher global scores were associated with more severe fatigue. LS means and SE were estimated from MMRM analysis. | Analysis was performed on Stage 1 ITT population. Here, 'Overall number of participants analyzed' signifies participants who were evaluable for the outcome measure. | Posted | Least Squares Mean | Standard Error | score on a scale | From Baseline to Month 18 |
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| Secondary | Change in Fatigue Severity as Measured by Brief Fatigue Inventory (BFI-SF)-Item 3 Scale Score From Baseline to Month 24: Combined Stage 1 and Stage 2 | The BFI-SF is a 10-item, validated, self-administered questionnaire that was originally developed to assess fatigue severity. The 10-items were measured on a 0-10 scale, with 0 being 'does not interfere' and 10 being 'completely interferes.' BFI - Item 3 asks participants to "Please rate your fatigue (weariness, tiredness) by circling the one number that best describes your worst level of fatigue during the past 24 hours. The NRS ranged from 0 (no fatigue) to 10 (worst imaginable fatigue). Higher global scores were associated with more severe fatigue. LS means and SE were estimated from MMRM analysis. | Analysis was performed on Combined Stage 1 and Stage 2 ITT population. Here, 'Overall number of participants analyzed' signifies participants who were evaluable for the outcome measure. Data for this OM was planned to be collected and analyzed for combined Stage 1 and Stage 2 population as pre-specified in protocol. | Posted | Least Squares Mean | Standard Error | score on a scale | From Baseline to Month 24 |
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| Secondary | Pharmacokinetics: Plasma Concentration of Venglustat: Stage 1 | Venglustat plasma concentrations was determined using a validated liquid chromatography tandem mass spectrometry (LC-MS/MS) method. Data for this outcome measure was not planned to be collected and analyzed for placebo arm. | Analysed on Stage 1 pharmacokinetic (PK) population that included all participants randomized in Stage 1 who took at least one dose of study drug and had at least one post-baseline PK assessment; analyzed according to the intervention they actually received. Here, "number analyzed" signifies participants with available data for each specified category. | Posted | Mean | Standard Deviation | nanograms per milliliter | Day 1: 3 hours Post-Dose, Month 1: Pre-Dose and 3 hours Post-Dose, Month 6: Pre-Dose, Month 18: Pre-Dose |
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| Secondary | Pharmacokinetics: Plasma Concentration of Venglustat: Stage 2 | Venglustat plasma concentrations was determined using a validated LC-MS/MS method. Data for this outcome measure was not planned to be collected and analyzed for placebo arm. | Analysis was performed on Stage 2 PK population that included all participants with an eGFR between 45 and 89.9 mL/min/1.73 m^2 at screening who were randomized in Stage 2, who took at least one dose of study drug and who had at least one post-baseline PK assessment. Participants were analyzed according to study drug they actually received. Here, "number analyzed" signifies participants with available data for each specified category. | Posted | Mean | Standard Deviation | nanograms per milliliter | Month 1: Pre-dose and 3 hours Post-dose |
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| Secondary | Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs): Stage 1 | An adverse event (AE) was any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with this treatment. A serious adverse event (SAE) was any untoward medical occurrence that at any dose: results in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect, or was a medically important event. TEAEs were AEs that developed, worsened or became serious during the treatment-emergent (TE) period (defined as the time from the first investigational medicinal product [IMP] administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). | Analysis was performed on Stage 1 safety population that included all participants randomized in Stage 1 who took at least one dose or part of a dose of the double-blind IMP, analyzed according to the treatment actually received (venglustat 15 mg, venglustat 8 mg, or placebo) | Posted | Count of Participants | Participants | From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier |
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| Secondary | Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs): Combined Stage 1 and Stage 2 | An AE was any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with this treatment. SAE was any untoward medical occurrence that at any dose: results in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect, or was a medically important event. TEAEs were AEs that developed, worsened or became serious during the TE period (defined as the time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). | Analysis was performed on extended Combined Stage 1 and Stage 2 safety population that included all participants with an eGFR between 45 and 89.9 mL/min/1.73 m^2 at screening who were randomized in Stage 1 or Stage 2 and who took at least one dose or part of a dose of IMP, analyzed according to treatment actually received. Data for this OM was planned to be collected and analyzed for combined Stage 1 and Stage 2 population as pre-specified in protocol. | Posted | Count of Participants | Participants | From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier |
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| Secondary | Number of Participants With Potentially Clinically Significant Abnormalities: Hematology: Combined Stage 1 and Stage 2 | Criteria for potentially clinically significant abnormalities: Hemoglobin: less than or equal to (<=) 115 grams per liter (g/L) [Male]; <=95 g/L [Female]; greater than or equal to (>=) 185 g/L [Male]; >=165 g/L [Female]; Decrease from baseline >=20 g/L; Hematocrit: <=0.37 volume/volume (v/v) [Male]; <=0.32 v/v [Female]; >=0.55 v/v [Male]; >=0.5 v/v [Female]; Erythrocyte (red blood cells [RBC]): >=6*10^12 per liter (/L); Platelet: less than (<) 100*10^9/L; >=700*10^9/L; Leukocyte (white blood cells [WBC]): <3*10^9/L [Non-Black]; <2*10^9/L [Black], >=16*10^9/L; Neutrophils: <1.5*10^9/L [Non-Black]; <1*10^9/L [Black]; Lymphocytes: greater than (>) 4*10^9/L, Monocytes: >0.7*10^9/L; Basophils: >0.1*10^9/L; and Eosinophils: >0.5*10^9/L or >upper limit of normal (ULN) (if ULN >=0.5*10^9/L). | Analysis was performed on extended Combined Stage 1 and Stage 2 safety population. Here, "number analyzed" signifies participants with available data for specified category. Data for this OM was planned to be collected and analyzed for combined Stage 1 and Stage 2 population as pre-specified in protocol. | Posted | Count of Participants | Participants | From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier |
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| Secondary | Number of Participants With Potentially Clinically Significant Abnormalities: Clinical Chemistry: Combined Stage 1 and Stage 2 | Criteria for potentially clinically significant abnormalities: Glucose: <=3.9 millimoles per liter (mmol/L) and <lower limit of normal (LLN): >=11.1 mmol/L (unfasted); >=7 mmol/L (fasted); Albumin:<=25 g/L; Sodium: <=129 mmol/L; >=160 mmol/L; Potassium: <3 mmol/L; >=5.5 mmol/L; Chloride: <80 mmol/L, >115 mmol/L; Creatinine: >=150 micro millimoles per liter (mcmol/L) (Adults); >=30% change from Baseline; >=100% change from Baseline, Urea Nitrogen: >=17 mmol/L; Alanine Aminotransferase (ALT): >3 ULN; Aspartate Aminotransferase (AST): >3 ULN; Alkaline Phosphatase: >1.5 ULN; Total Bilirubin: >1.5 ULN, >2 ULN; ALT >3 ULN and Bilirubin >2 ULN; and Direct Bilirubin >35% Bilirubin and Bilirubin >1.5 ULN. | Analysis was performed on extended Combined Stage 1 and Stage 2 safety population. Here, "number analyzed" signifies participants with available data for each specified category. Data for this OM was planned to be collected and analyzed for combined Stage 1 and Stage 2 population as pre-specified in protocol. | Posted | Count of Participants | Participants | From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier |
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| Secondary | Number of Participants With Potentially and Clinically Significant Abnormalities: Urinalysis: Combined Stage 1 and Stage 2 | Criteria for potentially clinically significant abnormalities: Urine pH: <=4.6 and >=8. | Analysis was performed on extended Combined Stage 1 and Stage 2 safety population. Here, "number analyzed" signifies participants with available data for each specified category. Data for this OM was planned to be collected and analyzed for combined Stage 1 and Stage 2 population as pre-specified in protocol. | Posted | Count of Participants | Participants | From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier |
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| Secondary | Number of Participants With Potentially and Clinically Significant Abnormalities: Vital Signs: Combined Stage 1 and Stage 2 | Criteria for potentially clinically significant abnormalities: Sitting Systolic Blood Pressure: <=95 millimeters of Mercury (mmHg) and decrease from Baseline >=20 mmHg; >=160 mmHg and increase from Baseline >=20 mmHg; Sitting Diastolic Blood Pressure: <=45 mmHg and decrease from Baseline >=10 mmHg, >=110 mmHg and increase from Baseline >=10 mmHg; Sitting Heart Rate: <=50 beats/minute and decrease from Baseline >=20 beats/minute; >=120 beats/minute and increase from Baseline >=20 beats/minute; and Weight: >=5% decrease from Baseline; >=5% increase from Baseline. | Analysis was performed on extended Combined Stage 1 and Stage 2 safety population. Here, "number analyzed" signifies participants with available data for each specified category. Data for this OM was planned to be collected and analyzed for combined Stage 1 and Stage 2 population as pre-specified in protocol. | Posted | Count of Participants | Participants | From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier |
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| Secondary | Number of Participants With Potentially Clinically Significant Abnormalities: Electrocardiogram: Combined Stage 1 and Stage 2 | Criteria for potentially clinically significant abnormalities: Heart Rate: <50 beats/minute; <50 beats/minute and decrease from Baseline >=20 beats/minute; <40 beats/minute; <40 beats/minute and decrease from Baseline >=20 beats/min; <30 beats/minute; >90 beats/minute; >90 beats/minute and increase from Baseline >=20 beats/minute; >100 beats/minute; >100 beats/minute and increase from Baseline >=20 beats/minute; >120 beats/minute; >120 beats/minute, increase from Baseline >=20 beats/minute; PR Interval: >200 milliseconds (msec); >200 msec and increase from Baseline >=25%; >220 msec, >240 msec; QRS Interval: >110 msec; >110 msec and increase from Baseline >=25%; >120 msec; >120 msec and increase from Baseline >=25%; QT Interval: >500 msec; QT corrected for heart rate (QTc) Bazett: >450 msec; >480 msec; increase from Baseline (30-60) msec; increase from Baseline >60 msec; QTc Fridericia: >450 msec; >480 msec; increase from Baseline (30-60) msec and increase from Baseline > 60 msec. | Analysis was performed on extended Combined Stage 1 and Stage 2 safety population. Here, "number analyzed" signifies participants with available data for each specified category. Data for this OM was planned to be collected and analyzed for combined Stage 1 and Stage 2 population as pre-specified in protocol. | Posted | Count of Participants | Participants | From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier |
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| Secondary | Number of Participants With Physical Examination Abnormalities: Combined Stage 1 and Stage 2 | Physical examination included: Head, Heart, Lung, Abdomen, Musculo/Skeletal, Skin, and Mental Status. Abnormality in physical examination was based on investigator's discretion. Results are based on the treatment emergent period which was defined as time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier. | Analysis was performed on extended Combined Stage 1 and Stage 2 safety population. Here, "number analyzed" signifies participants with available data for each specified category. Data for this OM was planned to be collected and analyzed for combined Stage 1 and Stage 2 population as pre-specified in protocol. | Posted | Count of Participants | Participants | Baseline, Month 18, Month 24 |
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| Secondary | Change From Baseline in Beck Depression Inventory-II (BDI-II) Score: Combined Stage 1 and Stage 2 | The Beck Depression Inventory-II (BDI-II) is a 21-item questionnaire used to assess depression. Most items are rated on a 4-point scale from 0 to 3, and a few items are rated on a 7-point scale. Individual item scores are added to get a total BDI-II score from 0 to 63. The higher the total score, the more severe the depression, and the lower the total score, the less severe the depression. | Analysis was performed on extended Combined Stage 1 and Stage 2 safety population. Here, "number analyzed" signifies participants with available data for each specified category. Data for this OM was planned to be collected and analyzed for combined Stage 1 and Stage 2 population as pre-specified in protocol. | Posted | Mean | Standard Deviation | score on a scale | Baseline, Months 3, 6, 9, 12, 15, 18, 21, and 24, Last on-treatment value up to last IMP + 1 day (anytime during the maximum duration of 25 months) |
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| Secondary | Number of Participants With Worsening Lens Opacity From Baseline During the Treatment-emergent Period: Combined Stage 1 and Stage 2 | Worsening of lens opacity classification system (LOCS) III score or World Health Organization (WHO) grade in nuclear opacification, cortical opacification and posterior subcapsular opacification were assessed for 'Any eye', 'Unilateral' and 'Bilateral' separately. A participant could be counted in all the 3 categories. In each category, the worst case was taken into account. To be evaluable for 'Any', a participant had to have at least one eye evaluable, whereas, for 'Unilateral' and 'Bilateral', a participant had to have both eyes evaluable. Therefore, the sum of 'Unilateral' + 'Bilateral' is not necessarily equal to 'Any' in the below table. The difference observed in 'Nuclear Opacification' in 15 mg group, comes from that 1 participant who had Unilateral worsening at a given visit and a Bilateral worsening at another visit. Therefore, this participant was counted as 'Unilateral', 'Bilateral' and counted only once in 'Any'. Results are based on the TE period. | Analysis was performed on extended Combined Stage 1 and Stage 2 safety population. Here, "number analyzed" signifies participants with available data for each specified category. Data for this OM was planned to be collected and analyzed for combined Stage 1 and Stage 2 population as pre-specified in protocol. TE period: time from first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier. | Posted | Count of Participants | Participants | From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier |
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From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier
Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
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| EG000 | Stage 1- Placebo | Participants from Stage 1 were randomized to receive 2 capsules of placebo matched to venglustat once daily for treatment period of 24 months. | 0 | 78 | 8 | 78 | 42 | 78 |
| EG001 | Stage 1- Venglustat 8 mg | Participants from Stage 1 were randomized to receive venglustat 8 mg (i.e., 2 capsules of 4 mg) once daily for treatment period of 24 months. | 0 | 78 | 15 | 78 | 46 | 78 |
| EG002 | Stage 1- Venglustat 15 mg | Participants from Stage 1 were randomized to receive 1 capsule of venglustat 15 mg and 1 capsule of placebo matched to venglustat once daily for treatment period of 24 months. | 0 | 80 | 19 | 80 | 48 | 80 |
| EG003 | Stage 2- Placebo | Participants from Stage 2 were randomized to receive 1 capsule of placebo matched to venglustat once daily for treatment period of 24 months. | 0 | 122 | 6 | 122 | 32 | 122 |
| EG004 | Stage 2- Venglustat 15 mg | Participants from Stage 2 were randomized to receive 1 capsule of venglustat 15 mg once daily for treatment period of 24 months. | 0 | 119 | 7 | 119 | 48 | 119 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Appendicitis | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
| |
| Covid-19 | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
| |
| Diverticulitis | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
| |
| Gastroenteritis Viral | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
| |
| Infected Cyst | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
| |
| Kidney Infection | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
| |
| Renal Cyst Infection | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
| |
| Urinary Tract Infection | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
| |
| Basal Cell Carcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 24.0 | Systematic Assessment |
| |
| Breast Cancer | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 24.0 | Systematic Assessment |
| |
| Intraductal Proliferative Breast Lesion | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 24.0 | Systematic Assessment |
| |
| Invasive Ductal Breast Carcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 24.0 | Systematic Assessment |
| |
| Mediastinum Neoplasm | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 24.0 | Systematic Assessment |
| |
| Fluid Overload | Metabolism and nutrition disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Hyperkalaemia | Metabolism and nutrition disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Depression | Psychiatric disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Suicidal Ideation | Psychiatric disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Carpal Tunnel Syndrome | Nervous system disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Intracranial Aneurysm | Nervous system disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Loss Of Consciousness | Nervous system disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Polyneuropathy | Nervous system disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Syncope | Nervous system disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Arteriosclerosis Coronary Artery | Cardiac disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Coronary Artery Stenosis | Cardiac disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Mitral Valve Incompetence | Cardiac disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Supraventricular Tachycardia | Cardiac disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Dental Cyst | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Inguinal Hernia | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Large Intestinal Stenosis | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Pancreatitis Acute | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Rectal Haemorrhage | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Hepatic Pain | Hepatobiliary disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Flank Pain | Musculoskeletal and connective tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Osteoarthritis | Musculoskeletal and connective tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Rotator Cuff Syndrome | Musculoskeletal and connective tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Acute Kidney Injury | Renal and urinary disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Calculus Urinary | Renal and urinary disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Chronic Kidney Disease | Renal and urinary disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Haematuria | Renal and urinary disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Renal Cyst Haemorrhage | Renal and urinary disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Renal Cyst Ruptured | Renal and urinary disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Renal Haemorrhage | Renal and urinary disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Ureterolithiasis | Renal and urinary disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Adenomyosis | Reproductive system and breast disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Haemorrhagic Cyst | General disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Non-Cardiac Chest Pain | General disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Back Injury | Injury, poisoning and procedural complications | MedDRA 24.0 | Systematic Assessment |
| |
| Hand Fracture | Injury, poisoning and procedural complications | MedDRA 24.0 | Systematic Assessment |
| |
| Jaw Fracture | Injury, poisoning and procedural complications | MedDRA 24.0 | Systematic Assessment |
| |
| Subdural Haematoma | Injury, poisoning and procedural complications | MedDRA 24.0 | Systematic Assessment |
| |
| Tibia Fracture | Injury, poisoning and procedural complications | MedDRA 24.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Covid-19 | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
| |
| Conjunctivitis | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
| |
| Upper Respiratory Tract Infection | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
| |
| Urinary Tract Infection | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Cataract Cortical | Eye disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Cataract Nuclear | Eye disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Dry Eye | Eye disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Dyspepsia | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Back Pain | Musculoskeletal and connective tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Flank Pain | Musculoskeletal and connective tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Pain In Extremity | Musculoskeletal and connective tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Haematuria | Renal and urinary disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Influenza Like Illness | General disorders | MedDRA 24.0 | Systematic Assessment |
|
Following decision to discontinue EFC15392 based on the futility analysis, the two-steps analysis initially planned were not applicable, therefore only the final analysis of all safety endpoints (laboratory, vital sign, ECG, physical examination, BDI-II, and lens opacity) were performed on extended combined Stage 1 and Stage 2 safety population only.
The Sponsor supports publication of clinical trial results but may request that investigators temporarily delay or alter publications in order to protect proprietary information. The Sponsor may also require that the results of multicenter studies be published only in their entirety and not as individual site data.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Trial Transparency Team | Sanofi aventis recherche & développement | 800-633-1610 | 6# | Contact-US@sanofi.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Jun 22, 2021 | Jul 29, 2022 | SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| D016891 | Polycystic Kidney, Autosomal Dominant |
| ID | Term |
|---|---|
| D007690 | Polycystic Kidney Diseases |
| D052177 | Kidney Diseases, Cystic |
| D007674 | Kidney Diseases |
| D014570 | Urologic Diseases |
| D052776 | Female Urogenital Diseases |
| D005261 | Female Urogenital Diseases and Pregnancy Complications |
| D000091642 | Urogenital Diseases |
| D052801 | Male Urogenital Diseases |
| D000015 | Abnormalities, Multiple |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D000072661 | Ciliopathies |
| D030342 | Genetic Diseases, Inborn |
Not provided
Not provided
| ID | Term |
|---|---|
| C000608118 | venglustat |
Not provided
Not provided
Not provided
| Withdrawal by Subject |
|
| Study terminated by sponsor |
|
| Other-Unspecified |
|
| Randomized and not treated |
|
| Male |
|
| Asian |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
| Relative difference |
| 0.34 |
| 2-Sided |
| 95 |
| -24.57 |
| 33.36 |
| Superiority |
| OG001 | Venglustat 8 mg | Participants from Stage 1 were randomized to receive venglustat 8 mg (i.e., 2 capsules of 4 mg) once daily for treatment period of 24 months. |
| OG002 | Venglustat 15 mg | All participants from Stage 1 or Stage 2 were randomized to receive 1 capsule of venglustat 15 mg once daily for treatment period of 24 months. |
|
|
|
Participants from Stage 1 were randomized to receive venglustat 8 mg (i.e., 2 capsules of 4 mg) once daily for treatment period of 24 months.
| OG002 | Stage 1- Venglustat 15 mg | Participants from Stage 1 were randomized to receive 1 capsule of venglustat 15 mg and 1 capsule of placebo matched to venglustat once daily for treatment period of 24 months. |
|
|
|
| OG002 | Venglustat 15 mg | All participants from Stage 1 or Stage 2 were randomized to receive 1 capsule of venglustat 15 mg once daily for treatment period of 24 months. |
|
| OG002 | Stage 1- Venglustat 15 mg | Participants from Stage 1 were randomized to receive 1 capsule of venglustat 15 mg and 1 capsule of placebo matched to venglustat once daily for treatment period of 24 months. |
|
|
|
| OG002 | Venglustat 15 mg | All participants from Stage 1 or Stage 2 were randomized to receive 1 capsule of venglustat 15 mg once daily for treatment period of 24 months. |
|
|
|
| OG002 |
| Stage I- Venglustat 15 mg |
Participants from Stage 1 were randomized to receive 1 capsule of venglustat 15 mg and 1 capsule of placebo matched to venglustat once daily for treatment period of 24 months. |
|
|
|
| OG002 | Venglustat 15 mg | All participants from Stage 1 or Stage 2 were randomized to receive 1 capsule of venglustat 15 mg once daily for treatment period of 24 months. |
|
|
|
| Units |
|---|
| Counts |
|---|
| Participants |
|
|
|
| OG001 | Stage 1- Venglustat 8 mg | Participants from Stage 1 were randomized to receive venglustat 8 mg (i.e., 2 capsules of 4 mg) once daily for treatment period of 24 months. |
| OG002 | Stage 1- Venglustat 15 mg | Participants from Stage 1 were randomized to receive 1 capsule of venglustat 15 mg and 1 capsule of placebo matched to venglustat once daily for treatment period of 24 months. |
|
|
| OG001 | Venglustat 8 mg | Participants from Stage 1 were randomized to receive venglustat 8 mg (i.e., 2 capsules of 4 mg) once daily for treatment period of 24 months. |
| OG002 | Venglustat 15 mg | All participants from Stage 1 or Stage 2 were randomized to receive 1 capsule of venglustat 15 mg once daily for treatment period of 24 months. |
|
|
| OG001 | Venglustat 8 mg | Participants from Stage 1 were randomized to receive venglustat 8 mg (i.e., 2 capsules of 4 mg) once daily for treatment period of 24 months. |
| OG002 | Venglustat 15 mg | All participants from Stage 1 or Stage 2 were randomized to receive 1 capsule of venglustat 15 mg once daily for treatment period of 24 months. |
|
|
| Venglustat 8 mg |
Participants from Stage 1 were randomized to receive venglustat 8 mg (i.e., 2 capsules of 4 mg) once daily for treatment period of 24 months. |
| OG002 | Venglustat 15 mg | All participants from Stage 1 or Stage 2 were randomized to receive 1 capsule of venglustat 15 mg once daily for treatment period of 24 months. |
|
|
|
|
Participants from Stage 1 were randomized to receive venglustat 8 mg (i.e., 2 capsules of 4 mg) once daily for treatment period of 24 months.
| OG002 | Venglustat 15 mg | All participants from Stage 1 or Stage 2 were randomized to receive 1 capsule of venglustat 15 mg once daily for treatment period of 24 months. |
|
|
All participants from Stage 1 or Stage 2 were randomized to receive placebo matched to venglustat once daily for treatment period of 24 months.
| OG001 | Venglustat 8 mg | Participants from Stage 1 were randomized to receive venglustat 8 mg (i.e., 2 capsules of 4 mg) once daily for treatment period of 24 months. |
| OG002 | Venglustat 15 mg | All participants from Stage 1 or Stage 2 were randomized to receive 1 capsule of venglustat 15 mg once daily for treatment period of 24 months. |
|
|
All participants from Stage 1 or Stage 2 were randomized to receive 1 capsule of venglustat 15 mg once daily for treatment period of 24 months. |
|
|
| Venglustat 15 mg |
All participants from Stage 1 or Stage 2 were randomized to receive 1 capsule of venglustat 15 mg once daily for treatment period of 24 months. |
|
|
| Placebo |
All participants from Stage 1 or Stage 2 were randomized to receive placebo matched to venglustat once daily for treatment period of 24 months. |
| OG001 | Venglustat 8 mg | Participants from Stage 1 were randomized to receive venglustat 8 mg (i.e., 2 capsules of 4 mg) once daily for treatment period of 24 months. |
| OG002 | Venglustat 15 mg | All participants from Stage 1 or Stage 2 were randomized to receive 1 capsule of venglustat 15 mg once daily for treatment period of 24 months. |
|
|