A Study to See if Tolvaptan Can Delay Dialysis in Infants and Children Who at Enrollment Are 28 Days to Less Than 12 Weeks Old With Autosomal Recessive Polycystic Kidney Disease (ARPKD)

September 14, 2023 updated by: Otsuka Pharmaceutical Development & Commercialization, Inc.

A Phase 3b Multicenter Open-label Trial of the Safety, Tolerability, and Efficacy of Tolvaptan in Infants and Children 28 Days to Less Than 12 Weeks of Age With Autosomal Recessive Polycystic Kidney Disease (ARPKD)

The primary objective of this study is to evaluate the effect of tolvaptan on the need for renal replacement therapy in pediatric subjects with autosomal recessive polycystic kidney disease (ARPKD)

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

Tolvaptan has been demonstrated to delay the decline of kidney function in adults with rapidly progressing ADPKD (CKD stages 1 to 3), a closely related indication to ARPKD, as measured by estimated glomerular filtration rate (eGFR) and Total Kidney Volume (TKV).

The trial will be the first trial of tolvaptan in a pediatric ARPKD population.

Participants in this study will be assigned to tolvaptan for 24 months and closely monitored over the course of the study.

Study Type

Interventional

Enrollment (Estimated)

20

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • Belgium
      • Leuven, Belgium, 3000
        • Recruiting
        • UZ Leuven
    • Oost-Vlaanderen
      • Gent, Oost-Vlaanderen, Belgium, B-9000
        • Recruiting
        • Universitair Ziekenhuis Gent
  • Germany
    • Nordrhein-Westfalen
      • Cologne, Nordrhein-Westfalen, Germany, 50937
        • Not yet recruiting
        • University Hospital of Cologne
  • Poland
      • Białystok, Poland, 15-274
        • Not yet recruiting
        • Uniwersytecki Dzieciecy Szpital Kliniczny im. L. Zamenhofa
      • Warszawa, Poland, 04-730
        • Not yet recruiting
        • Instytut "Pomnik - Centrum Zdrowia Dziecka"
  • Spain
      • Barcelona, Spain, 08035
        • Recruiting
        • Hospital Universitari Vall d Hebron
      • Barcelona, Spain, 08950
        • Recruiting
        • Universitat de Barcelona - Hospital Sant Joan de Deu Barcelona (HSJDB)
      • Sevilla, Spain, 41013
        • Recruiting
        • Hospital Universitario Virgen del Rocio
  • United Kingdom
      • London, United Kingdom, WC1N 3JH
        • Not yet recruiting
        • Great Ormond Street
  • United States
    • District of Columbia
      • Washington, District of Columbia, United States, 20010
        • Recruiting
        • Children's National Medical Center
    • Georgia
      • Atlanta, Georgia, United States, 30322
        • Not yet recruiting
        • Emory University
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Not yet recruiting
        • Northwestern University Feinberg School of Medicine - Ann & Robert H. Lurie Children's Hospital of Chicago - Neonatology
    • Maryland
      • Baltimore, Maryland, United States, 21287-0001
        • Not yet recruiting
        • Johns Hopkins Pediatric Specialty Clinic
    • Michigan
      • Ann Arbor, Michigan, United States, 48109-5283
        • Recruiting
        • C.S. Mott Children's Hospital
    • Minnesota
      • Rochester, Minnesota, United States, 55905
        • Not yet recruiting
        • Mayo Clinic
    • Ohio
      • Cincinnati, Ohio, United States, 45229
        • Recruiting
        • Cincinnati Children's Hospital
      • Cleveland, Ohio, United States, 44195
        • Not yet recruiting
        • Cleveland Clinic
    • Utah
      • Salt Lake City, Utah, United States, 84113-1103
        • Not yet recruiting
        • Primary Children's Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 year to 1 year (Child)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. Male or female subjects between 28 days and < 12 weeks of age, inclusive at the time of enrollment.

  2. Must have clinical and imaging features that are consistent with a diagnosis of ARPKD with all the following characteristics:

    • Nephromegaly (> 2 standard deviations from age appropriate standard via ultrasound)
    • Multiple renal cysts
    • History of oligohydramnios or anhydramnios
  3. Ability for parent or 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.

Exclusion Criteria:

  1. Premature birth (≤ 32 weeks gestational age)
  2. Anuria or RRT, defined as intermittent or continuous hemodialysis, peritoneal dialysis, hemofiltration, hemodiafiltration or history of kidney transplantation
  3. Evidence of syndromic conditions associated with renal cysts (other than ARPKD)
  4. Abnormal liver function tests including ALT and AST, > 1.2 × ULN
  5. Parents with renal cystic disease
  6. Need for chronic diuretic use
  7. Cannot be monitored for fluid balance
  8. Has or at risk of having sodium and potassium electrolyte imbalances
  9. Has or at risk of having significant hypovolemia as determined by investigator
  10. Clinically significant anemia, as determined by investigator
  11. Severe systolic dysfunction defined as ejection fraction < 14%
  12. Serum sodium levels < 130 mmol/L or >145 mmol/L
  13. Taking any other experimental medications
  14. Require ventilator support
  15. Taking medications known to induce CYP3A4
  16. Having an infection including viral that would require therapy disruptive to IMP dosing
  17. Platelet count <50,000 µL
  18. Significant Portal Hypertension
  19. Bladder dysfunction or difficulty voiding
  20. Taking vasopressin agonist
  21. Having concomitant illness or taking medications that are likely to confound endpoint assessments.
  22. History of cholangitis
  23. Received or scheduled to receive a liver transplant

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Tolvaptan (OPC-41061)
Drug: Tolvaptan (OPC-41061)
Tolvaptan suspension will be administered orally or via 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 2 years.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
The percentage of subjects that will have Renal Replacement Therapy (RRT) by 1 year of age.
Time Frame: From Enrollment to 1 year of age
From Enrollment to 1 year of age

Secondary Outcome Measures

Outcome Measure
Time Frame
Rate of change of eGFR by Schwartz formula from pre-treatment to after 2 years of treatment
Time Frame: From Enrollment to 2 years of age
From Enrollment to 2 years of age
Palatability of the suspension formulation as assessed by a parent questionnaire immediately after and within 15-20 minutes after the first oral dose
Time Frame: From Enrollment to 2 years of age
From Enrollment to 2 years of age
Acceptance of the suspension formulation as assessed by a parent questionnaire immediately after and within 15-20 minutes after the first oral dose
Time Frame: From Enrollment to 2 years of age
From Enrollment to 2 years of age

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Otsuka Pharmaceutical Development & Commercialization, Inc.

Investigators

  • Study Director: Olga Sergeyeva, MD, Otsuka Pharmaceutical Development & Commercialization, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

July 1, 2022

Primary Completion (Estimated)

October 11, 2027

Study Completion (Estimated)

October 11, 2027

Study Registration Dates

First Submitted

February 19, 2021

First Submitted That Met QC Criteria

March 3, 2021

First Posted (Actual)

March 8, 2021

Study Record Updates

Last Update Posted (Actual)

September 15, 2023

Last Update Submitted That Met QC Criteria

September 14, 2023

Last Verified

August 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 156-12-204

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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