Clinical trial NCT02251275: Long Term Safety of Immediate-release Tolvaptan in Subjects With Autosomal Dominant Polycystic Kidney Disease
Official title: A Phase 3b, Multi-center, Open-label Trial to Evaluate the Long Term Safety of Immediate-release Tolvaptan (OPC-41061, 30 mg to 120 mg/Day, Split Dose) in Subjects With Autosomal Dominant Polycystic Kidney Disease
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Polycystic Kidney, Autosomal Dominant
Keywords: ADPKD
Lead sponsor: Otsuka Pharmaceutical Development & Commercialization, Inc.
Sponsor class: INDUSTRY
First posted: 2014-09-29
Enrollment: 1803
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The purpose of the trial was to evaluate and describe the long term safety of tolvaptan in participants with autosomal dominant polycystic kidney disease (ADPKD).

Detailed description: This was a Phase 3b trial to evaluate and describe the long term safety of tolvaptan treatment in ADPKD participants with chronic kidney disease (CKD). Eligible participants could enroll into Trial 156-13-211 after completing the follow-up visit(s) of their previous trial (156-13-210, 156-08-271, 156-04-251, or 156-09-290). Renal function was assessed during screening by using historical laboratory values for serum creatinine levels to calculate the estimated glomerular filtration rate (eGFR).

Eligibility criteria: Inclusion Criteria:

* Male and female participants ≥ 18 years with confirmed diagnosis of ADPKD (during participation in prior tolvaptan trials) who have completed and transferred from the double-blind Trial 156-13-210 (12-month period including post treatment follow-up, regardless of whether this was on-treatment or off-treatment), or completed Trial 156-08-271 or a prior tolvaptan trial, or interrupted or discontinued treatment in a prior tolvaptan ADPKD trial other than Trial 156-13-210. Participants may be enrolled with the medical monitor approval, and additional close monitoring may be required at the beginning of the trial.
* eGFR ≥ 20 milliliter (mL)/minute (min)/1.73 meter squared (m\^2) within 3 months prior to the baseline visit. Participants who have an eGFR ≤ 20 mL/min/1.73 m\^2 may be enrolled with medical monitor approval.

Exclusion Criteria:

* Need for chronic diuretic use
* Hepatic impairment based on liver function abnormalities other than that expected for ADPKD with cystic liver disease
* 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 result prior to receiving IMP.
* Participants with contraindications to required trial assessments (contraindications to optional assessments, for example, magnetic resonance imaging \[MRI\] are not a limitation).
* Participants who in the opinion of the investigator or the medical monitor, have a medical history or medical finding inconsistent with safety or trial compliance

Primary outcomes:
measure: Number Of Participants Reporting Treatment-Emergent Adverse Events (TEAEs)
timeFrame: Baseline through end of treatment (up to 42 months) and follow-up 7 days posttreatment(+ 7 days)
Secondary outcomes:
Other outcomes:
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state: Greater Manchester
city: Salford
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city: Stevenage
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city: Inverness
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city: Leicester
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state: Leicestershire
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city: Liverpool
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city: Stoke on Trent
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state: Staffordshire
city: Newcastle upon Tyne
country: United Kingdom
state: Tyne & Wear
city: Coventry
country: United Kingdom
state: Warwickshire
city: Birmingham
country: United Kingdom
state: West Midlands
city: Middlesbrough
country: United Kingdom
city: Swansea
country: United Kingdom
Study officials:
References:
citation: Torres VE, Chapman AB, Devuyst O, Gansevoort RT, Perrone RD, Lee J, Hoke ME, Estilo A, Sergeyeva O. Multicenter Study of Long-Term Safety of Tolvaptan in Later-Stage Autosomal Dominant Polycystic Kidney Disease. Clin J Am Soc Nephrol. 2020 Dec 31;16(1):48-58. doi: 10.2215/CJN.10250620. Epub 2020 Dec 29.