Status:
WITHDRAWN
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)
Lead Sponsor:
Otsuka Pharmaceutical Development & Commercialization, Inc.
Conditions:
Autosomal Recessive Polycystic Kidney Disease (ARPKD)
Eligibility:
All Genders
4-12 years
Phase:
PHASE3
Brief Summary
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)
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 gl...
Eligibility Criteria
Inclusion
- Male or female subjects between 28 days and \< 12 weeks of age, inclusive at the time of enrollment.
- 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
- 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
- Premature birth (≤ 32 weeks gestational 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
- Parents with renal cystic disease
- Need for chronic diuretic use
- Cannot be monitored for fluid balance
- Has or at risk of having sodium and potassium electrolyte imbalances
- Has or at risk of having significant hypovolemia as determined by investigator
- Clinically significant anemia, as determined by investigator
- Severe systolic dysfunction defined as ejection fraction \< 14%
- Serum sodium levels \< 130 mmol/L or \>145 mmol/L
- Taking any other experimental medications
- Require ventilator support
- Taking medications known to induce CYP3A4
- Having an infection including viral that would require therapy disruptive to IMP dosing
- Platelet count \<50,000 µL
- Significant Portal Hypertension
- Bladder dysfunction or difficulty voiding
- Taking vasopressin agonist
- Having concomitant illness or taking medications that are likely to confound endpoint assessments.
- History of cholangitis
- Received or scheduled to receive a liver transplant
Key Trial Info
Start Date :
July 1 2022
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 10 2025
Estimated Enrollment :
Patients enrolled
Trial Details
Trial ID
NCT04786574
Start Date
July 1 2022
End Date
December 10 2025
Last Update
December 24 2025
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