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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/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
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)
Secondary ID [1] 0 0
2020-004400-34
Secondary ID [2] 0 0
LTS15823
Universal Trial Number (UTN)
Trial acronym
STAGED-PKD-EXT
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Congenital Cystic Kidney Disease 0 0
Condition category
Condition code

Intervention/exposure
Study type
Interventional
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
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Percent Change in Total Kidney Volume (TKV)
Timepoint [1] 0 0
From the EFC15392 study Baseline to 24 months of open-label extension study LTS15823
Secondary outcome [1] 0 0
Change From the Baseline in Estimated Glomerular Filtration Rate (eGFR) as Assessed by Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI)
Timepoint [1] 0 0
From the EFC15392 study Baseline to 24 months of open-label extension study LTS15823
Secondary outcome [2] 0 0
Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Timepoint [2] 0 0
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)
Secondary outcome [3] 0 0
Change From Baseline in Lens Clarity During the Open-label Extension Treatment-emergent Period of LTS15823 Study
Timepoint [3] 0 0
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

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.
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
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
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
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
9/02/2021
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
13/07/2021
Sample size
Target
Accrual to date
Final
24
Recruitment in Australia
Recruitment state(s)

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Sanofi
Address
Country

Ethics approval
Ethics application status

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).
Trial website
https://clinicaltrials.gov/study/NCT04705051
Trial related presentations / publications
Public notes
This record is viewable in the ANZCTR as it had previously listed Australia and/or New Zealand as a recruitment site, however these sites have since been removed

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/NCT04705051