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)
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ClinicalTrials.gov Identifier: NCT04786574 |
Recruitment Status :
Recruiting
First Posted : March 8, 2021
Last Update Posted : April 20, 2023
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Autosomal Recessive Polycystic Kidney Disease (ARPKD) | Drug: Tolvaptan (OPC-41061) | Phase 3 |
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 (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) |
Actual Study Start Date : | July 1, 2022 |
Estimated Primary Completion Date : | October 11, 2027 |
Estimated Study Completion Date : | October 11, 2027 |

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. |
- 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 ]
- 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 ]
- 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 ]
- 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 ]

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Ages Eligible for Study: | 28 Days to 12 Weeks (Child) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Male or female subjects between 28 days and < 12 weeks of age, inclusive at the time of enrollment.
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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 Criteria:
- 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

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 ClinicalTrials.gov identifier (NCT number): NCT04786574
Contact: Leslyn Hermonstine | 240.683.3157 | Leslyn.Hermonstine@otsuka-us.com | |
Contact: Linda Cappiello | +1 (609) 6084545 | linda.cappiello-cw@otsuka-us.com |

Study Director: | Olga Sergeyeva, MD | Otsuka Pharmaceutical Development & Commercialization, Inc. |
Responsible Party: | Otsuka Pharmaceutical Development & Commercialization, Inc. |
ClinicalTrials.gov Identifier: | NCT04786574 |
Other Study ID Numbers: |
156-12-204 |
First Posted: | March 8, 2021 Key Record Dates |
Last Update Posted: | April 20, 2023 |
Last Verified: | June 2022 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
ARPKD Tolvaptan Polycystic Kidney Disease Autosomal Recessive Polycystic Kidney Disease |
Renal Cysts Nephromegaly Oligohydramnios Anhydramnios |
Kidney Diseases Polycystic Kidney Diseases Polycystic Kidney, Autosomal Recessive Urologic Diseases Female Urogenital Diseases Female Urogenital Diseases and Pregnancy Complications Urogenital Diseases Male Urogenital Diseases Kidney Diseases, Cystic |
Abnormalities, Multiple Congenital Abnormalities Ciliopathies Genetic Diseases, Inborn Tolvaptan Antidiuretic Hormone Receptor Antagonists Molecular Mechanisms of Pharmacological Action Natriuretic Agents Physiological Effects of Drugs |