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)

Description

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)

Conditions

Autosomal Recessive Polycystic Kidney Disease (ARPKD)

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Study Overview

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Study Details

Study overview

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)

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)

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)

Condition
Autosomal Recessive Polycystic Kidney Disease (ARPKD)
Intervention / Treatment

-

Contacts and Locations

Washington

Children's National Medical Center, Washington, District of Columbia, United States, 20010

Atlanta

Emory University, Atlanta, Georgia, United States, 30322

Chicago

Northwestern University Feinberg School of Medicine - Ann & Robert H. Lurie Children's Hospital of Chicago - Neonatology, Chicago, Illinois, United States, 60611

Baltimore

Johns Hopkins Pediatric Specialty Clinic, Baltimore, Maryland, United States, 21287-0001

Ann Arbor

C.S. Mott Children's Hospital, Ann Arbor, Michigan, United States, 48109-5283

Rochester

Mayo Clinic, Rochester, Minnesota, United States, 55905

Cincinnati

Cincinnati Children's Hospital, Cincinnati, Ohio, United States, 45229

Cleveland

Cleveland Clinic, Cleveland, Ohio, United States, 44195

Salt Lake City

Primary Children's Hospital, Salt Lake City, Utah, United States, 84113-1103

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.

For general information about clinical research, read Learn About Studies.

Eligibility 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.
  • 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

Ages Eligible for Study

28 Days to 12 Weeks

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Otsuka Pharmaceutical Development & Commercialization, Inc.,

Olga Sergeyeva, MD, STUDY_DIRECTOR, Otsuka Pharmaceutical Development & Commercialization, Inc.

Study Record Dates

2027-10-11