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Phase 3 Completed N=1,519 Randomized Quadruple-blind Treatment

Efficacy and Safety of Tolvaptan in Subjects With Chronic Kidney Disease Between Late Stage 2 to Early Stage 4 Due to Autosomal Dominant Polycystic Kidney Disease

Source: ClinicalTrials.gov NCT02160145 ↗
Enrolled (actual)
1,519
Serious AEs
6.6%
Results posted
Aug 2018
Primary outcomePrimary: The Mean Annualized Change in eGFR From Pretreatment Baseline to Post-treatment Follow-up. — -2.339; -3.610 mL/min/1.73 m^2/year — p=<0.0001
◆ Published Evidence
Highly cited
619citations · ~69 / year
Tolvaptan in Later-Stage Autosomal Dominant Polycystic Kidney Disease.
The New England journal of medicine · 2017 · Open access · Likely link

Summary

The purpose of the study is to determine whether tolvaptan is effective and safe for the treatment of late-stage chronic kidney disease due to autosomal dominant polycystic kidney disease (ADPKD)

Linked Publications (5)

  • Tolvaptan in Later-Stage Autosomal Dominant Polycystic Kidney Disease.
    The New England journal of medicine · 2017 · 619 citations · Open access · Likely link
  • Clinical Pattern of Tolvaptan-Associated Liver Injury in Trial Participants With Autosomal Dominant Polycystic Kidney Disease (ADPKD): An Analysis of Pivotal Clinical Trials.
    American journal of kidney diseases : the official journal of the National Kidney Foundation · 2023 · 39 citations · Open access · Likely link
  • Modelling the long-term benefits of tolvaptan therapy on renal function decline in autosomal dominant polycystic kidney disease: an exploratory analysis using the ADPKD outcomes model.
    BMC nephrology · 2019 · 20 citations · Open access · Likely link
  • Tolvaptan and Kidney Function Decline in Older Individuals With Autosomal Dominant Polycystic Kidney Disease: A Pooled Analysis of Randomized Clinical Trials and Observational Studies.
    Kidney medicine · 2023 · 13 citations · Open access · Likely link
  • Interventions for preventing the progression of autosomal dominant polycystic kidney disease.
    The Cochrane database of systematic reviews · 2024 · 8 citations · Open access · Likely link

Outcome Measures

OutcomeResultp-value
PRIMARY
The Mean Annualized Change in eGFR From Pretreatment Baseline to Post-treatment Follow-up.
-2.339; -3.610 <0.0001 sig
SECONDARY
Mean Annualized Slope of eGFR Change
-3.160; -4.170 <0.0001 sig

Eligibility Criteria

Inclusion Criteria

  • Male and female subjects with eGFR between 25-65 mL/min/1.73m2 (if aged 18 to55) or eGFR between 25-44 mL/min/1.73m2 (if aged 56 to <66)
  • Tolvaptan naïve
  • Diagnosis of ADPKD by modified pei-Ravine criteria 1) 3 cysts per kidney by sonography or 5 cysts by CT or MRI with family history of ADPKD or 2) 10 cysts per kidney by any radiologic method and exclusion of other cystic kidney diseases if without family history

Exclusion Criteria

  • Women of childbearing potential who do not agree to practice 2 different methods of birth control or remain abstinent during the trial and for 30 days after the last dose of Investigational medicinal product (IMP)
  • Women who are breast-feeding and/or who have a positive pregnancy test prior to receiving IMP
  • Need for chronic diuretic use
  • Hepatic impairment or liver function abnormalities other than that expected for ADPKD with typical cystic liver disease
  • Advanced diabetes, evidence of additional significant renal disease, renal cancer, single kidney, recent renal surgery or acute kidney injury
  • Contraindications to required trial assessments
  • Medical history or medical findings inconsistent with safety or compliance with trial assessments
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT02160145) and the linked publication. 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. Informational only — not medical advice.

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