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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)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT04786574
Recruitment Status : Not yet recruiting
First Posted : March 8, 2021
Last Update Posted : March 8, 2021
Information provided by (Responsible Party):
Otsuka Pharmaceutical Development & Commercialization, Inc.

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)

Condition or disease Intervention/treatment Phase
Autosomal Recessive Polycystic Kidney Disease (ARPKD) Drug: Tolvaptan (OPC-41061) Phase 3

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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)
Estimated Study Start Date : April 1, 2021
Estimated Primary Completion Date : March 31, 2025
Estimated Study Completion Date : July 1, 2025

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.

Primary Outcome Measures :
  1. The percentage of subjects that will have RRT by 1 year of age. [ Time Frame: From Enrollment to 1 year of age ]

Secondary Outcome Measures :
  1. 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 ]
  2. Acceptance and palatability of the suspension formulation will be 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 ]

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   up to 12 Weeks   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. Male or female subjects between 28 days and < 12 weeks of age, inclusive.
  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
  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. Critical electrolyte imbalances, as determined by the investigator
  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
  13. Cannot be 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT04786574

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Contact: Lindsay Getz (919) 797-9591

Sponsors and Collaborators
Otsuka Pharmaceutical Development & Commercialization, Inc.
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Study Director: Rosa Real, MD Otsuka Pharmaceutical Development & Commercialization, Inc.
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Responsible Party: Otsuka Pharmaceutical Development & Commercialization, Inc. Identifier: NCT04786574    
Other Study ID Numbers: 156-12-204
First Posted: March 8, 2021    Key Record Dates
Last Update Posted: March 8, 2021
Last Verified: January 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Otsuka Pharmaceutical Development & Commercialization, Inc.:
Polycystic Kidney Disease
Autosomal Recessive Polycystic Kidney Disease
Renal Cysts
Additional relevant MeSH terms:
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Kidney Diseases
Polycystic Kidney Diseases
Polycystic Kidney, Autosomal Recessive
Urologic Diseases
Kidney Diseases, Cystic
Abnormalities, Multiple
Congenital Abnormalities
Genetic Diseases, Inborn
Antidiuretic Hormone Receptor Antagonists
Molecular Mechanisms of Pharmacological Action
Natriuretic Agents
Physiological Effects of Drugs