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Phase 3 N=1,083 Treatment

Tolvaptan Extension Study in Participants With ADPKD

Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Enrolled (actual)
1,083
Serious AEs
24.1%
Results posted
Oct 2021
Primary outcome: Primary: Percent Change From the Baseline in Total Kidney Volume (TKV) for Study 156-04-251 Participants Enrolled in This Study (156-08-271) — 28.66; 30.58 percent change — p=0.358

Study Design & Population

Study type
Interventional
Phase
Phase 3
Interventions
Tolvaptan (Drug)
Age
Adult, Older Adult · 18+ yrs
Sex
All
Sponsor
Otsuka Pharmaceutical Development & Commercialization, Inc.
Primary completion
Feb 2016

Outcome Measures

OutcomeResultp-value
PRIMARY
Percent Change From the Baseline in Total Kidney Volume (TKV) for Study 156-04-251 Participants Enrolled in This Study (156-08-271)
28.66; 30.58 0.358
SECONDARY
Change From the Baseline in Estimated Glomerular Filtration Rate (eGFR) as Assessed by Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) for Study 156-04-251 Participants Enrolled in This Study (156-08-271)
-16.77; -19.92 0.0003 sig
SECONDARY
Annualized Slope of Total Kidney Volume (TKV) for Study 156-04-251 Participants Enrolled in Study 156-08-271
6.164; 4.960 0.0462 sig
SECONDARY
Annualized Slope of eGFR (CKD-EPI) for Study 156-04-251 Participants Enrolled in Study 156-08-271
-3.255; -3.142 0.7259
SECONDARY
Annualized TKV Slope for Study 156-04-251 Placebo Participants Enrolled in Study 156-08-271
4.779; 5.627 0.1080
SECONDARY
Annualized Slope of Renal Function (eGFRCKD-EPI) for Study 156-04-251 Placebo Participants Enrolled in Study 156-08-271
-3.211; -3.572 0.2265

Summary

To demonstrate whether tolvaptan modifies ADPKD progression as measured by changes from Baseline (from Study 156-04-251) in total kidney volume (TKV) and renal function.

Eligibility Criteria

Inclusion Criteria

  • Participants who had successfully completed a Phase 1, 2, or 3 tolvaptan ADPKD or renal impairment study, with a confirmed diagnosis of ADPKD from prior studies [either 156-04-251 (NCT00428948) or 156-04-250 (NCT00413777), 156-06-260, 156-09-284 (NCT01336972), 156-09-285 (NCT01210560), and 156-09-290 (NCT01451827)].

Exclusion Criteria

  • Participants unable to provide written informed consent.
  • Participants (men or women) would not adhere to the reproductive precautions as outlined in the Informed Consent Form.
  • Participants (women only) with a positive urine pregnancy test.
  • Participants who were pregnant or breast-feeding.
  • Participants unable to take oral medications.
  • Participants who had allergic reactions to tolvaptan or chemically related structures such as benzazepines (benzazepril, conivaptan, fenoldopam mesylate, or mirtazapine).
  • Participants with disorders in thirst recognition or an inability to access fluids.
  • Participants with critical electrolyte imbalances, as determined by the investigator
  • Participants with or at risk of significant hypovolemia, as determined by investigator.
  • Participants with significant anemia, as determined by investigator.
  • Participants with a history of substance abuse (within the last 3 years).
  • Participants who were taking other experimental (that is, non-marketed) therapies or were participating in another clinical drug or device study; participating in the off-drug follow-up period of another ADPKD trial with tolvaptan was permitted.
  • Participants unable to complete magnetic resonance imaging (MRI) assessments (for example, participants with ferro-magnetic prostheses, aneurysm clips, severe claustrophobia).
  • Participants who had taken a vasopressin antagonist (outside of previous participation in a tolvaptan study).
  • Participants unable to comply with anti-hypertensive or other important medical therapy.
  • Participants with advanced diabetes.
  • Participants who were taking medications or had an illness that could confound endpoint assessments (including taking approved therapies for the purpose of affecting polycystic kidney disease [PKD] cysts).
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT01214421). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.

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