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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03523728




Registration number
NCT03523728
Ethics application status
Date submitted
1/05/2018
Date registered
14/05/2018
Date last updated
3/02/2023

Titles & IDs
Public title
A Medical Research Study Designed to Determine if Venglustat Can be a Future Treatment for ADPKD Patients
Scientific title
Multicenter, Randomized, Double-blind, Placebo-controlled Two Stage Study to Characterize the Efficacy, Safety, Tolerability and Pharmacokinetics of GZ/SAR402671 in Patients at Risk of Rapidly Progressive Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Secondary ID [1] 0 0
2017-004084-12
Secondary ID [2] 0 0
EFC15392
Universal Trial Number (UTN)
Trial acronym
STAGED-PKD
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Polycystic Kidney, Autosomal Dominant 0 0
Condition category
Condition code
Renal and Urogenital 0 0 0 0
Other renal and urogenital disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Venglustat
Treatment: Drugs - Placebo

Placebo comparator: 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.

Experimental: Stage 1- Venglustat 8 mg - 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.

Experimental: 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.

Placebo comparator: 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.

Experimental: 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.


Treatment: Drugs: Venglustat
Pharmaceutical form: capsule; Route of administration: oral

Treatment: Drugs: Placebo
Pharmaceutical form: capsule; Route of administration: oral
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Annualized Slope of Change in Total Kidney Volume (TKV) From Baseline to Month 18: Stage 1
Timepoint [1] 0 0
From Baseline to Month 18
Primary outcome [2] 0 0
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
Timepoint [2] 0 0
From Baseline to Month 24
Secondary outcome [1] 0 0
Annualized Rate of Change in Estimated Glomerular Filtration Rate (eGFR) From Baseline to Month 24: Stage 1
Timepoint [1] 0 0
From Baseline to Month 24
Secondary outcome [2] 0 0
Annualized Slope of Change in Total Kidney Volume (TKV) From Baseline to Month 18: Combined Stage 1 and Stage 2
Timepoint [2] 0 0
From Baseline to Month 18
Secondary outcome [3] 0 0
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
Timepoint [3] 0 0
From Baseline to Month 18
Secondary outcome [4] 0 0
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
Timepoint [4] 0 0
From Baseline to Month 24
Secondary outcome [5] 0 0
Change in Fatigue Severity as Measured by Brief Fatigue Inventory (BFI-SF)-Item 3 Scale Score From Baseline to Month 18: Stage 1
Timepoint [5] 0 0
From Baseline to Month 18
Secondary outcome [6] 0 0
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
Timepoint [6] 0 0
From Baseline to Month 24
Secondary outcome [7] 0 0
Pharmacokinetics: Plasma Concentration of Venglustat: Stage 1
Timepoint [7] 0 0
Day 1: 3 hours Post-Dose, Month 1: Pre-Dose and 3 hours Post-Dose, Month 6: Pre-Dose, Month 18: Pre-Dose
Secondary outcome [8] 0 0
Pharmacokinetics: Plasma Concentration of Venglustat: Stage 2
Timepoint [8] 0 0
Month 1: Pre-dose and 3 hours Post-dose
Secondary outcome [9] 0 0
Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs): Stage 1
Timepoint [9] 0 0
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
Secondary outcome [10] 0 0
Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs): Combined Stage 1 and Stage 2
Timepoint [10] 0 0
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
Secondary outcome [11] 0 0
Number of Participants With Potentially Clinically Significant Abnormalities: Hematology: Combined Stage 1 and Stage 2
Timepoint [11] 0 0
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
Secondary outcome [12] 0 0
Number of Participants With Potentially Clinically Significant Abnormalities: Clinical Chemistry: Combined Stage 1 and Stage 2
Timepoint [12] 0 0
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
Secondary outcome [13] 0 0
Number of Participants With Potentially and Clinically Significant Abnormalities: Urinalysis: Combined Stage 1 and Stage 2
Timepoint [13] 0 0
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
Secondary outcome [14] 0 0
Number of Participants With Potentially and Clinically Significant Abnormalities: Vital Signs: Combined Stage 1 and Stage 2
Timepoint [14] 0 0
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
Secondary outcome [15] 0 0
Number of Participants With Potentially Clinically Significant Abnormalities: Electrocardiogram: Combined Stage 1 and Stage 2
Timepoint [15] 0 0
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
Secondary outcome [16] 0 0
Number of Participants With Physical Examination Abnormalities: Combined Stage 1 and Stage 2
Timepoint [16] 0 0
Baseline, Month 18, Month 24
Secondary outcome [17] 0 0
Change From Baseline in Beck Depression Inventory-II (BDI-II) Score: Combined Stage 1 and Stage 2
Timepoint [17] 0 0
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)
Secondary outcome [18] 0 0
Number of Participants With Worsening Lens Opacity From Baseline During the Treatment-emergent Period: Combined Stage 1 and Stage 2
Timepoint [18] 0 0
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

Eligibility
Key inclusion criteria
Inclusion criteria:

* Male or female adult with ADPKD with age at the time the consent was signed:

1. between 18 to 50 years (both inclusive) for participants from Stage 1.
2. between 18 to 50 years (both inclusive) for participants from Stage 2 with eGFR between 45 and 89.9 milliliters per minute per 1.73 meter square (mL/min/1.73 m^2) during screening period.*
3. between 18 to 55 years (both inclusive) for participants from Stage 2 with eGFR between 30 and 44.9 mL/min/1.73 m^2 during screening period.*
* 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 (ß-human chorionic gonadotropin [ß-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.
Minimum age
18 Years
Maximum age
55 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Exclusion criteria:

* Systolic blood pressure greater than (>) 160 millimeters of Mercury (mmHg) at Run-in and Baseline visits.
* Administration within 3 months prior to the screening visit of tolvaptan or other Polycystic Kidney Disease-modifying agents (somatostatin analogues).
* Participation in another investigational interventional study or use of IMP, within 3 months or 5 half lives, whichever was longer, before randomization.
* The participant had a positive result of any of the following tests: hepatitis B surface antigen (HBsAg), anti-hepatitis C virus (anti HCV) antibodies, anti-human immunodeficiency virus 1 and 2 antibodies (anti HIV1 and anti HIV2 Ab). Participants with a positive hepatitis B surface antibody (HBsAb) test were eligible if other criteria were met (i.e., negative tests for: HBsAg, hepatitis B core antibody [HBcAb]). Participants immune due to natural infection (positive hepatitis B surface antibody (HBsAb), negative hepatitis B surface antigen (HBsAg) and positive hepatitis B core antibody [HBcAb]) were eligible if they had negative hepatitis B vaccine (HBV) deoxyribonucleic acid (DNA) test.
* A history of drug and/or alcohol abuse within the past year prior to the screening visit. A history of alcohol dependence within the 5 years prior to the screening visit.
* The participant was scheduled for in-patient hospitalization including elective surgery, during the study.
* The participant had a clinically significant, uncontrolled medical condition that, in the opinion of the investigator, would put the safety of the participant at risk through participation, or which would affect the efficacy or safety analysis if the condition exacerbated during the study, or that might significantly interfere with study compliance, including all prescribed evaluations and follow-up activities.
* The participants, in the opinion of the investigator, was unable to adhere to the requirements of the study or unable to undergo study assessments (e.g., had contraindications to pupillary dilation or unable to undergo magnetic resonance imaging (MRI) [For example: participant's weight exceeds weight capacity of the MRI, ferromagnetic metal prostheses, aneurysm clips, severe claustrophobia, large abdominal/back tattoos, etc.]).
* Any country-related specific regulation that would prevent the participant from entering the study.
* The participants did not adhere to treatment (less than [<] 70 percent [%] compliance rate) in the run-in.
* The participant had, according to World Health Organization (WHO) Grading, a cortical cataract greater than or equal to (>=)one-quarter of the lens circumference (Grade cortical cataract-2 [COR-2]) or a posterior subcapsular cataract >=2 millimeter (Grade posterior subcapsular cataract-2 [PSC-2]). Participant with nuclear cataracts would not be excluded.
* The participant was then receiving potentially cataractogenic medications, including a chronic regimen (more frequently than every 2 weeks) of any route of corticosteroids (including medium and high potency topical steroids) or any medication that might cause cataract, according to the Prescribing Information.
* The participant had received strong or moderate inducers or inhibitors of CYP3A4 within 14 days or 5 half-lives, whichever was longer, prior to randomization. This also included the consumption of grapefruit, grapefruit juice, or grapefruit containing products within 72 hours of starting venglustat administration.
* The participant was pregnant, or lactating.
* Liver enzymes (alanine aminotransferase /aspartate aminotransferase ) or total bilirubin >2 times the upper limit of normal unless the participant had the diagnosis of Gilbert syndrome. Participants with the Gilbert syndrome should have had no additional symptoms or signs which suggested hepatobiliary disease and serum total bilirubin level no more than 3 milligrams per deciliter (mg/dL) (51 [micromoles per Liter] mcmol/L) with conjugated bilirubin less than 20% of the total bilirubin fraction.
* Presence of severe depression as measured by Beck Depression Inventory-II (BDI-II) >28 and/or a history of a major affective disorder within 1 year of the screening visit.
* Known hypersensitivity to venglustat or any component of the excipients.

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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s

The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Stopped early
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
4/10/2018
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
3/08/2021
Sample size
Target
Accrual to date
Final
478
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Investigational Site Number 0360002 - Herston
Recruitment hospital [2] 0 0
Investigational Site Number 0360003 - Nedlands
Recruitment hospital [3] 0 0
Investigational Site Number 0360001 - Westmead
Recruitment postcode(s) [1] 0 0
4029 - Herston
Recruitment postcode(s) [2] 0 0
6009 - Nedlands
Recruitment postcode(s) [3] 0 0
2145 - Westmead
Recruitment outside Australia
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United States of America
State/province [1] 0 0
Alabama
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State/province [2] 0 0
California
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Colorado
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Connecticut
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Graz
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Bruxelles
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Leuven
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Edmonton
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Osaka-Shi
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Shinjuku-Ku
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Japan
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Toyoake-Shi
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Korea, Republic of
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Seoul
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Netherlands
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Amsterdam
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Groningen
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Netherlands
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Nijmegen
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Poland
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Warszawa
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Poland
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Wroclaw
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Lódz
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Almada
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Carnaxide
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Loures
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Romania
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Bucuresti
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Oradea
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Timisoara
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Barcelona
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Madrid
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Taichung
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Taiwan
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Taipei
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Turkey
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Istanbul
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Turkey
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Kayseri
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Turkey
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Kocaeli
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United Kingdom
State/province [86] 0 0
Sheffield

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Genzyme, a Sanofi Company
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
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:

* To determine the effect of venglustat on the rate of renal function decline (Stage 1) and on the rate of TKV growth (Stage 2).
* To evaluate the pharmacokinetics (PK) of venglustat in ADPKD participants (Stages 1 and 2).
* To determine the effect of venglustat on pain and fatigue, based on participant reported diary (Stages 1 and 2).
* Safety/tolerability objectives:
* To characterize the safety profile of venglustat (Stages 1 and 2).
* To evaluate the effect of venglustat on mood using Beck Depression Inventory II (BDI-II) (Stages 1 and 2).
* To evaluate the effect of venglustat on the lens by ophthalmological examination (Stages 1 and 2).
Trial website
https://clinicaltrials.gov/study/NCT03523728
Trial related presentations / publications
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.
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.
Public notes

Contacts
Principal investigator
Name 0 0
Clinical Sciences & Operations
Address 0 0
Sanofi
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT03523728