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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT04705051
Registration number
NCT04705051
Ethics application status
Date submitted
8/01/2021
Date registered
12/01/2021
Date last updated
6/10/2022
Titles & IDs
Public title
Long-term Treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD) With Venglustat
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Scientific title
Multicenter, Open-label, Extension Study to Characterize the Long-term Efficacy and Safety of Early Versus Delayed Treatment With Venglustat (GZ/SAR402671) in Patients at Risk of Rapidly Progressive Autosomal Dominant Polycystic Kidney Disease (ADPKD)
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Secondary ID [1]
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2020-004400-34
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Secondary ID [2]
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LTS15823
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Universal Trial Number (UTN)
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Trial acronym
STAGED-PKD-EXT
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Congenital Cystic Kidney Disease
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Condition category
Condition code
Renal and Urogenital
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Kidney disease
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Renal and Urogenital
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Other renal and urogenital disorders
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Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - Venglustat GZ402671
Experimental: Venglustat - Participants were to be treated with venglustat 15 milligrams once daily orally for 24 months or until venglustat was commercially available, whichever came first.
Treatment: Drugs: Venglustat GZ402671
Pharmaceutical form: capsule Route of administration: oral
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Percent Change in Total Kidney Volume (TKV)
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Assessment method [1]
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Total kidney volume is a measure of disease progression in the ADPKD participants. Study LTS15823 was terminated prematurely by the Sponsor following a decision to terminate the parent study EFC15392 (NCT03523728). Study EFC15392 was terminated based on the results of the planned prespecified futility analysis performed for this study. Termination decision was due to a lack of efficacy in the ADPKD population and not linked to safety findings with venglustat. Due to the early termination of study LTS15823 and low enrollment numbers, efficacy analysis was not performed.
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Timepoint [1]
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From the EFC15392 study Baseline to 24 months of open-label extension study LTS15823
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Secondary outcome [1]
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Change From the Baseline in Estimated Glomerular Filtration Rate (eGFR) as Assessed by Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI)
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Assessment method [1]
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eGFR is a measure of kidney function assessed through blood/serum. Study LTS15823 was terminated prematurely by the Sponsor following a decision to terminate the parent study EFC15392 (NCT03523728). Study EFC15392 was terminated based on the results of the planned prespecified futility analysis performed for this study. Termination decision was due to a lack of efficacy in the ADPKD population and not linked to safety findings with venglustat. Due to the early termination of study LTS15823 and low enrollment numbers, efficacy analysis was not performed.
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Timepoint [1]
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From the EFC15392 study Baseline to 24 months of open-label extension study LTS15823
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Secondary outcome [2]
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Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
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Assessment method [2]
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Adverse event (AE) was defined as any untoward medical occurrence in participant who received investigational medicinal product (IMP) and did not necessarily have a causal relationship with treatment. All reported AEs were TEAEs that developed/worsened during 'TEAE period' (defined as the time from the first administration of the IMP in LTS15823 study up to the last IMP administration + 30 days). A SAEs was defined as any AE that at any dose results in: death; is life threatening; requires initial or prolonged inpatient hospitalization; results in persistent or significant disability/incapacity; is a congenital anomaly/birth defect; is a suspected transmission of any infectious agent via an authorized medicinal product; is a medically important event.
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Timepoint [2]
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From the first administration of the IMP in LTS15823 study up to the last IMP administration in LTS15823 study + 30 days (i.e., up to approximately 20 weeks)
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Secondary outcome [3]
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Change From Baseline in Lens Clarity During the Open-label Extension Treatment-emergent Period of LTS15823 Study
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Assessment method [3]
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Change from EFC15392 study Baseline in the lens clarity was assessed by ophthalmological examination with World Health Organization (WHO) simplified cataract grading system during the open label extension treatment-emergent period of LTS15823 study. Study LTS15823 was terminated prematurely by the Sponsor following a decision to terminate the parent study EFC15392 (NCT03523728). Study EFC15392 was terminated based on the results of the planned prespecified futility analysis performed for this study. Termination decision was due to a lack of efficacy in the ADPKD population and not linked to safety findings with venglustat. Baseline was defined as the values from EFC15392 study Baseline.
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Timepoint [3]
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Baseline (EFC15392 study Baseline); from first administration of IMP up to last administration of IMP of open-label extension study LTS15823 + 30 days (i.e., up to approximately 20 weeks), in comparison to the EFC15392 study Baseline
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Secondary outcome [4]
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Effect on Mood With Change From Baseline in Beck Depression Inventory (BDI-II) Score During the Open-label Extension Treatment-emergent Period of LTS15823 Study
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Assessment method [4]
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BDI-II was a validated self-reported instrument of 21 questions which were each scored on a Likert scale of 0 to 3. Total score was calculated as sum of all scores of 21 questions. Total scores ranged from 0 (minimal depression) to 63 (severe depression), with higher score totals indicating more severe depression symptoms (0 to 13: indicates minimal depression;14 to 19: indicates mild depression; 20 to 28: indicates moderate depression; and 29 to 63: indicates severe depression). A negative change from baseline indicated an improvement. Baseline was defined as the values from EFC15392 study Baseline.
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Timepoint [4]
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Baseline (EFC15392 study Baseline); from first IMP administration up to 3 months in study LTS15823, in comparison to the EFC15392 study Baseline
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Eligibility
Key inclusion criteria
Inclusion criteria :
- Male or female adult with ADPKD who had completed the treatment period in Stage 1 or
Stage 2 of Study EFC15392 (NCT03523728).
- The participants who had an eGFR >30 mL/min/1.73 m^2:
1. Measured at Visit 11 of the EFC15392 study for participant enrolled in the
LTS15823 study at the time of Visit 12 (Month 24; end-of treatment visit) of the
EFC15392 study.
2. Measured at Screening visit for participant enrolled in the LTS15823 study not
concomitantly to the Visit 12 (Month 24; end-of treatment visit) of the EFC15392
study.
- Contraceptive used by men and women should be consistent with local regulations
regarding the methods of contraception for those participating in clinical studies.
1. Male participants who agreed to practice true abstinence in line with their
preferred and usual lifestyle or to use double-contraceptive methods for the
entire duration of the study and for at least 90 days following their last dose
of IMP.
2. Female participants who had a negative urine pregnancy test at the Baseline visit
and 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 the entire duration of the study and for at least 6
weeks following their last dose of IMP.
- Capable of giving signed informed consent before performance of any study related
procedures not part of standard medical care.
- Able to read, comprehend, and respond to the study questionnaires.
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Minimum age
18
Years
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
Exclusion criteria:
- For participants who had lag phase between the end of the EFC15392 study and Screening
visit (Visit 0) in the LTS15823 study.
- The participant had a new 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.
- A history of drug abuse and/or alcohol abuse or alcohol dependence during the lag
phase between the end of the EFC15392 study and Screening visit (Visit 0) in the
LTS15823 study when applicable.
- Administration of tolvaptan or other polycystic kidney disease-modifying agents
(somatostatin analogues) within 3 months prior to the Screening visit (Visit 0) in the
LTS15823 study when applicable.
- The participant was currently 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 may 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 the Baseline visit
(including consumption of grapefruit-containing products within 72 hours of starting
venglustat administration).
- Participation in another investigational interventional study or use of IMP, within 3
months or 5 half-lives, whichever was longer, before the Baseline visit (Visit 1)
except participation in the EFC15392 study when applicable.
- 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 had no additional
symptoms or signs which suggest hepatobiliary disease and serum total bilirubin level
no more than 3 milligrams per decilitre (mg/dL) (51 micromoles per litre [µmol/L])
with conjugated bilirubin less than 20 percent (%) of the total bilirubin fraction.
For participants with or without lag phase between the end of EFC15392 study and entry into
LTS15823 study:
- The participant was pregnant or lactating.
- Presence of severe depression as measured by BDI-II >28 at Visit 1 (for participants
enrolled in the LTS15823 study at the time of the end of treatment visit of the
EFC15392 study) or at Visit 0 (for participants enrolled in the LTS15823 study after
the end-of-treatment visit of the EFC15392 study).
- Sensitivity to any of the study interventions, or components thereof, or drug or other
allergy that, in the opinion of the Investigator, contraindicates participation in the
study.
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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Study design
Purpose of the study
Treatment
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Allocation to intervention
N/A
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Terminated
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
9/02/2021
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
13/07/2021
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Sample size
Target
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Accrual to date
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Final
24
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Recruitment in Australia
Recruitment state(s)
NSW
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Recruitment hospital [1]
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Investigational Site Number :0360001 - Westmead
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Recruitment postcode(s) [1]
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2145 - Westmead
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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West Virginia
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Country [2]
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Belgium
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State/province [2]
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Leuven
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Country [3]
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Germany
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State/province [3]
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Berlin
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Country [4]
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Japan
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State/province [4]
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Hokkaido
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Country [5]
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Japan
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State/province [5]
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Osaka
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Country [6]
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Japan
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State/province [6]
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Tokyo
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Country [7]
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Korea, Republic of
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State/province [7]
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Seoul-teukbyeolsi
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Country [8]
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Netherlands
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State/province [8]
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Nijmegen
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Country [9]
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Spain
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State/province [9]
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Barcelona [Barcelona]
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Sanofi
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Address
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Country
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Ethics approval
Ethics application status
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Summary
Brief summary
Primary Objective:
-To determine the effect of early versus delayed treatment with venglustat on the total
kidney volume (TKV) in participants at risk of rapidly progressive autosomal dominant
polycystic kidney disease (ADPKD).
Secondary Objective:
- To determine the effect of early versus delayed treatment with venglustat on the renal
function (estimated glomerular filtration rate [eGFR] [Chronic Kidney Disease
Epidemiology Collaboration {CKD-EPI} equation]).
- To characterize the safety profile of venglustat.
- To evaluate the effect of venglustat on the lens by ophthalmological examination.
- To evaluate the effect of venglustat on mood using Beck Depression Inventory-II
(BDI-II).
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Trial website
https://clinicaltrials.gov/ct2/show/NCT04705051
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Clinical Sciences & Operations
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Address
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Sanofi
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Address
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Country
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Phone
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Fax
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Email
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT04705051
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