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)
- Conditions
- ADPKDkidney disease10038430
- Registration Number
- NL-OMON51229
- Lead Sponsor
- Genzyme Europe BV
- Brief Summary
Trial ended prematurely
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 32
- Male or female adult with ADPKD who has completed the treatment period in
Stage 1 or Stage 2 of Study EFC15392.
- The patient has an eGFR >30 mL/min/1.73 m2:
a) 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.
b) 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 use by men and women should be consistent with local
regulations regarding the methods of contraception for those participating in
clinical studies.
a) Male participants must agree 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.
b) Female participants must have a negative urine pregnancy test at the
Baseline visit and agree 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.
For participants who have lag phase between the end of the EFC15392 study and
Screening visit (Visit 0) in the LTS15823 study:
-The patient has a new clinically significant, uncontrolled medical condition
that, in the opinion of the Investigator, would put the safety of the patient
at risk through participation, or which would affect the efficacy or safety
analysis if the condition exacerbated during the study, or that may
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 patient is 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 patient has received strong or moderate inducers or inhibitors of CYP3A4
within 14 days or 5 half lives, whichever is 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 is 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 patient has the
diagnosis of Gilbert syndrome. Patients with the Gilbert syndrome should have
no additional symptoms or signs which suggest hepatobiliary disease and serum
total bilirubin level no more than 3 mg/dL (51 *mol/L) with conjugated
bilirubin less than 20% of the total bilirubin fraction.
For participants with or without lag phase between the end of EFC15392 study
and entry into LTS15823 study:
-The patient is pregnant or lactating.
-Presence of severe depression as measured by Beck Depression Inventory 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.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>Percent change in TKV based on magnetic resonance imaging (MRI) from the<br /><br>EFC15392 study baseline to 24 months of open-label extension study, in<br /><br>early-treated and delayed-treated participants.</p><br>
- Secondary Outcome Measures
Name Time Method <p>- Change in eGFR (Chronic Kidney Disease Epidemiology Collaboration [CKD-EPI]<br /><br>equation) from the EFC15392 study baseline to 24 months of open-label extension<br /><br>study, in early-treated and delayed-treated participants.<br /><br>- Safety in terms of treatment-emergent adverse events (TEAEs), adverse events<br /><br>(AEs), serious adverse events (SAEs), laboratory parameters, vital signs,<br /><br>electrocardiogram and findings from physical examination will be assessed<br /><br>through the study and will be reported in the electronic case report form<br /><br>(eCRF).<br /><br>- Change from EFC15392 study baseline in the lens clarity by ophthalmological<br /><br>examination during the open-label extension treatment-emergent period.<br /><br>- Change from EFC15392 study baseline in BDI-II score during the open-label<br /><br>extension treatment-emergent period.</p><br>