RECRUITING

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)

Conditions

Autosomal Recessive Polycystic Kidney Disease (ARPKD)ARPKD Tolvaptan Polycystic Kidney Disease Autosomal Recessive Polycystic Kidney Disease Renal Cysts Nephromegaly Oligohydramnios Anhydramnios

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

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)

Official Title

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)

Quick Facts

Study Start:2022-07-01

Study Completion:2027-10-11

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT04786574

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.

Ages Eligible for Study:28 Days to 12 Weeks

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD

Inclusion Criteria	Exclusion 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

Inclusion Criteria

Exclusion 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

Contacts and Locations

Study Contact

Leslyn Hermonstine

CONTACT

240.683.3157

Leslyn.Hermonstine@otsuka-us.com

Linda Cappiello

CONTACT

+1 (609) 6084545

linda.cappiello-cw@otsuka-us.com

Principal Investigator

Olga Sergeyeva, MD

STUDY_DIRECTOR

Otsuka Pharmaceutical Development & Commercialization, Inc.

Study Locations (Sites)

Children's National Medical Center

Washington, District of Columbia, 20010

United States

Emory University

Atlanta, Georgia, 30322

United States

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

Chicago, Illinois, 60611

United States

Johns Hopkins Pediatric Specialty Clinic

Baltimore, Maryland, 21287-0001

United States

C.S. Mott Children's Hospital

Ann Arbor, Michigan, 48109-5283

United States

Mayo Clinic

Rochester, Minnesota, 55905

United States

Cincinnati Children's Hospital

Cincinnati, Ohio, 45229

United States

Cleveland Clinic

Cleveland, Ohio, 44195

United States

Primary Children's Hospital

Salt Lake City, Utah, 84113-1103

United States

Collaborators and Investigators

Sponsor: Otsuka Pharmaceutical Development & Commercialization, Inc.

Olga Sergeyeva, MD, STUDY_DIRECTOR, 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 Registration Dates

Study Start Date2022-07-01

Study Completion Date2027-10-11

Study Record Updates

Study Start Date2022-07-01

Study Completion Date2027-10-11

Terms related to this study

Keywords Provided by Researchers

ARPKD
Tolvaptan
Polycystic Kidney Disease
Autosomal Recessive Polycystic Kidney Disease
Renal Cysts
Nephromegaly
Oligohydramnios
Anhydramnios

Additional Relevant MeSH Terms

Autosomal Recessive Polycystic Kidney Disease (ARPKD)