A Study to See Iftolvaptan is Safe in Infants and Children Who at Enrollment Are 28 Days to Less Than 18 Years Old withAutosomal Recessive Polycystic Kidney Disease (ARPKD)

NCT04782258 | Recruiting Phase 3 FDA Drug

• 2 other identifiers: 156-201-003072020-005992-10
• Study Type: interventional
• Target: 20
• Locations: 6 countries
• Sites: 23

Brief Summary

To evaluate the safety of tolvaptan in pediatric subjects with ARPKD

Conditions

Autosomal Recessive Polycystic Kidney (ARPKD)

Keywords

ARPKD; TOLVAPTAN; Polycystic Kidney Disease; Autosomal Recessive Polycystic Kidney Disease; Adolescent; Renal Cysts; Oligohydramnios; Anhydramnios

Outcome Measures

Primary Outcomes (1)

• Number of Participants Reporting Treatment-Emergent Adverse Events (TEAEs)

  Enrollment up to 7 days post last dose

Secondary Outcomes (4)

• Annual rate of change of eGFR (by Schwartz formula) from baseline to post-treatment after 18 months of treatment

  From Enrollment to 18 months

• Change from baseline of eGFR (by Schwartz formula) while on treatment at Months 1, 6, 12, and 18

  1 month, 6 months, 12 months, and 18 months

• The percentage of subjects that will receive renal replacement therapy (RRT) by 18 months.

  From Enrollment to 18 months

• The amount of time between enrollment and 18 months that a subject requires renal replacement therapy (RRT).

  From enrollment to 18 months

Study Arms (2)

Tolvaptan Suspension

EXPERIMENTAL

Tolvaptan suspension will be administered orally or via feeding/nasogastric tube at doses of 0.15 mg/kg once daily in the AM, 0.30 mg/kg once daily in the AM, 0.5 mg/kg once daily in the AM, 0.75 mg/kg split dose (0.5 mg/kg AM and 0.25 mg/kg 8 hours later), and 1 mg/kg split dose (0.67 mg/kg AM and 0.33 mg/kg 8 hours later) based on age. Treatment duration is 18 months.

Drug: Tolvaptan Suspension

Tolvaptan Tablets

EXPERIMENTAL

Tolvaptan tablets will be administered orally as split-dose regimens (15/7.5 mg, 30/15 mg, and 45/15 mg) upon awakening and 8 hours later (twice daily) based on weight if able to swallow tablets. Treatment duration is 18 months.

Drug: Tolvaptan Tablets

Interventions

Tolvaptan Suspension DRUG

Syrup

Tolvaptan Suspension

Tolvaptan Tablets DRUG

Tolvaptan (OPC-41061) Tolvaptan tablets will be administered orally as split-dose regimens (15/7.5 mg, 30/15 mg, and 45/15 mg) upon awakening and 8 hours later (twice daily) based on weight if able to swallow tablets.

Tolvaptan Tablets

Eligibility Criteria

• Age: 28 Days - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Male or female subjects between 28 days and less than 18 years of age, with clinical features that are consistent with a diagnosis of ARPKD.
• Ability for parent/legal guardian to provide written, informed consent prior to initiation of any trial-related procedures, and ability, in the opinion of the principal investigator, to comply with all the requirements of the trial. Ability to provide written informed assent from all subjects old enough per local laws to provide assent.

You may not qualify if:

• Premature birth (≤ 32 weeks gestational age) for infants 28 days to \< 12 weeks of age.
• Anuria or RRT defined as intermittent or continuous hemodialysis, peritoneal dialysis, hemofiltration, hemodiafiltration or history of kidney transplantation.
• Evidence of syndromic conditions associated with renal cysts (other than ARPKD).
• Abnormal liver function tests including ALT and AST, \> 1.2 × ULN (upper limit of normal).
• Has splenomegaly or portal hypertension (HTN).
• Parents with renal cystic disease.
• Receiving chronic diuretic that could not be adjusted after tolvaptan initiation.
• Cannot be monitored for fluid balance.
• Has or at risk of having sodium and potassium electrolyte imbalances, as determined by the investigator.
• Has or at risk of having significant hypovolemia as determined by investigator.
• Clinically significant anemia, as determined by investigator.
• Platelets \< 50000 µL.
• Severe systolic dysfunction defined as ejection fraction \< 14%.
• Serum sodium levels \< 130 mmol/L or \>145 mmol/L.
• Taking any other experimental medications.

Sponsors & Collaborators

• Otsuka Pharmaceutical Development & Commercialization, Inc.: lead

Study Sites (23)

Children's National Medical Center

Washington D.C., District of Columbia, 20010, United States

RECRUITING

Emory University Hospital

Atlanta, Georgia, 30322, United States

WITHDRAWN

Northwestern University Feinberg School of Medicine - Ann & Robert H. Lurie Children's Hospital of Chicago - Neonatology

Chicago, Illinois, 60611, United States

WITHDRAWN

Riley Hospital for Children

Indianapolis, Indiana, 46202-5119, United States

WITHDRAWN

Children's Hospital - New Orleans

New Orleans, Louisiana, 70118, United States

WITHDRAWN

Johns Hopkins Pediatric Specialty Clinic

Baltimore, Maryland, 21287, United States

RECRUITING

C.S. Mott Children's Hospital

Ann Arbor, Michigan, 48109-5000, United States

RECRUITING

Mayo Clinic - Rochester

Rochester, Minnesota, 55905, United States

RECRUITING

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229-3039, United States

RECRUITING

Cleveland Clinic

Cleveland, Ohio, 44195, United States

RECRUITING

Children's Hospital of Pittsburgh of UPMC

Pittsburgh, Pennsylvania, 15213, United States

WITHDRAWN

Primary Children's Hospital

Salt Lake City, Utah, 84113, United States

WITHDRAWN

Université Catholique De Louvain And Cliniques St Luc

Brussels, Brussels Capital, 1200, Belgium

RECRUITING

Universitair Ziekenhuis Gent

Ghent, Oost-Vlaanderen, 9000, Belgium

RECRUITING

UZ Leuven

Leuven, Vlaams Brabant, 3000, Belgium

RECRUITING

Universitätsklinikum Köln

Cologne, North Rhine-Westphalia, 50937, Germany

RECRUITING

Instytut "Pomnik - Centrum Zdrowia Dziecka"

Warsaw, Masovian Voivodeship, 04-730, Poland

WITHDRAWN

Uniwersytecki Dzieciecy Szpital Kliniczny im. L. Zamenhofa

Bialystok, 15-274, Poland

WITHDRAWN

Universitat de Barcelona - Hospital Sant Joan de Deu Barcelona (HSJDB)

Esplugues de Llobregat, Barcelona, 8950, Spain

RECRUITING

Hospital Universitari Parc Tauli

Sabadell, Barcelona, 08208, Spain

WITHDRAWN

Hospital Universitari Vall D Hebron

Barcelona, 8035, Spain

RECRUITING

Hospital Universitario Virgen del Rocío Avenida Manuel Siurot

Seville, 41013, Spain

WITHDRAWN

Great Ormond Street Hospital for Children NHS Trust

London, WC1N 3JH, United Kingdom

RECRUITING

Related Links

• Otsuka Clinical Trials Website

MeSH Terms

Conditions

Polycystic Kidney, Autosomal Recessive; Polycystic Kidney Diseases; Oligohydramnios

Interventions

Tolvaptan

Condition Hierarchy (Ancestors)

Kidney Diseases, Cystic; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Ciliopathies; Genetic Diseases, Inborn; Pregnancy Complications

Intervention Hierarchy (Ancestors)

Benzazepines; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Heterocyclic Compounds

Central Study Contacts

Otsuka Call Center

CONTACT

844-687-8522; Otsuka-ProfessionalServices@otsuka-us.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 19, 2021
• First Posted: March 4, 2021
• Study Start: January 23, 2023
• Primary Completion (Estimated): July 31, 2028
• Study Completion (Estimated): August 14, 2028
• Last Updated: February 17, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

PL (2); GB (1); US (12); BE (3); ES (4); DE (1)