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Looking for participantsPhase3

A Study to See if Tolvaptan is Safe in Infants and Children Who at Enrollment Are 28 Days to Less Than 18 Years Old With Autosomal Recessive Polycystic Kidney Disease (ARPKD)

Sponsor: Otsuka Pharmaceutical Development & Commercialization, Inc.

NCT ID: NCT04782258

View on ClinicalTrials.gov ↗

At a glance

What the study gets you
Access to the study treatment being tested
Type of study
Interventional (receives a drug or procedure)
Time in hospital
In-person visits at study sites — visit count not specified by the sponsor
Drug or intervention
Tolvaptan Suspension (drug), Tolvaptan Tablets (drug)
How long the study runs
Study runs about 67 months (dates as stated)
About the drug or intervention
Tolvaptan Suspension — drug: Syrup · Tolvaptan Tablets — drug: Tolvaptan (OPC-41061) Tolvaptan tablets will be administered orally as split-dose regimens (15/7.5 mg, 30/15 mg, and 45/15 mg) upon awakening and 8 hours later (twice daily) based on weight if able to swallow tablets.
Patient visit burden
Not specified by the sponsor

In plain English

This study looks at whether tolvaptan is safe for babies, children and young people aged 28 days to under 18 years who have autosomal recessive polycystic kidney disease (ARPKD) — an inherited condition where cysts form in the kidneys. It is funded by Otsaka Pharmaceutical Development & Commercialization, Inc.

Who can take part

  • Aged 28 days to under 18 years, with signs that fit a diagnosis of autosomal recessive polycystic kidney disease (ARPKD)
  • A parent or legal guardian can give written permission (consent) for the child to take part, and children old enough can give their own written agreement (assent)
  • The family can follow all the trial requirements, in the opinion of the lead researcher

Who may not be able to

  • Babies born early (at or before 32 weeks of pregnancy) who are aged 28 days to under 12 weeks
  • Not producing urine, or needing dialysis or a kidney transplant (or having had one)
  • Having another condition with cysts in the kidneys that is not ARPKD
  • Abnormal liver blood test results (ALT and AST higher than 1.2 times the normal limit)
  • An enlarged spleen or high blood pressure in the liver's blood vessels (portal hypertension)
  • Parents with cystic kidney disease
  • Taking water tablets (diuretics) long-term that cannot be adjusted after starting tolvaptan
  • Cannot have fluid levels monitored
  • Has or is at risk of salt (sodium) or potassium imbalances in the blood
  • Has or is at risk of serious dehydration or low blood volume
  • Serious anaemia (low red blood cells)
  • Low platelet count (below 50,000 per microlitre)
  • Severe heart pumping problems (ejection fraction below 14%)
  • Blood sodium levels below 130 or above 145 mmol/L
  • Taking other experimental medicines
  • Needing help from a ventilator to breathe
  • Taking medicines that speed up a liver enzyme called CYP3A4
  • An infection needing treatment that would disrupt trial medicine dosing
  • Girls who are breastfeeding or have a positive pregnancy test before receiving the trial medicine
  • History of substance abuse in the last 6 months
  • Bladder problems or difficulty passing urine
  • Taking a vasopressin-type medicine (for example, desmopressin)
  • A history of not taking important medicines as prescribed
  • Taking medicines (including approved ones for kidney cysts, such as tolvaptan, vasopressin antagonists, anti-sense RNA therapies, rapamycin, sirolimus, everolimus, or somatostatin analogues like octreotide) or having other illnesses that could affect the trial results
  • Has had or is scheduled to have a liver transplant
  • A liver bile duct infection (cholangitis) in the last 6 months
  • Signs of serious portal hypertension, such as swollen veins (varices), bleeding from varices, or an overactive spleen causing low platelets
  • Not agreeing to avoid sex or use two highly effective forms of contraception for at least 28 days before the first dose, during the trial, and for at least 30 days after the last dose

What taking part involves

  • • The trial involves taking a medicine called tolvaptan (the investigational medicinal product)
  • • Not stated — ask the trial team about how the medicine is given and how often

Time commitment: Not stated — ask the trial team about how long the trial lasts, how many visits are needed, and what taking part involves.

Plain-English summary (AI-generated) from registry data. Not eligibility advice — only the trial team can confirm whether you can take part. Use the eligibility checker to see how your health profile matches this trial.

Type of study
Testing a treatment
Ages
28 Days to 18 Years
Who
All
Number of participants
20
Started
2023-01-23
Last checked
2026-08

Plain English Summary

What is this study?

  • • Testing a new treatment for autosomal recessive polycystic kidney (arpkd)
  • • Phase3 - 20 participants
  • • To evaluate the pharmacodynamics and safety of tolvaptan in pediatric subjects with ARPKD

Who can take part?

  • • Ages 28 Days to 18 Years
  • • Diagnosed with autosomal recessive polycystic kidney (arpkd)

Where?

  • • London - Great Ormond Street Hospital for Children NHS Trust

This is a simplified summary. Always discuss with your doctor before making any decisions.

About This Trial

To evaluate the pharmacodynamics and safety of tolvaptan in pediatric subjects with ARPKD

More detail

This study is a multinational, multicenter, open-label, non-randomized trial. The study consist of three periods: Screening Period, Treatment period and Follow-up period. Tolvaptan has been demonstrated to delay the decline of kidney function in adults with rapidly progressing ADPKD (CKD stages 1 to 4), a closely related indication to ARPKD, as measured by estimated glomerular filtration rate (eGFR) and Total Kidney Volume (TKV). Participants in this study will be assigned to tolvaptan and followed for 24 months over the course of the study. The overall trial duration is expected to be approximately 5 years.

Autosomal Recessive Polycystic Kidney (ARPKD)

How this trial compares with your answers

Answer 2 more questions to improve match

What we know so far

Condition· Matched your search
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Still need:

  • • Tell us your age for better matching
  • • Tell us your sex for better matching

Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.

Eligibility at a Glance

Key info

  • Age: 28 Days - 18 Years
  • Who can join: All genders

Biomarkers mentioned

have a positive

What the study is looking for

  • ✓Male or female subjects between 28 days and less than 18 years of age, with clinical features that are consistent...

Who cannot take part

  • ✗Premature birth (≤ 32 weeks gestational age) for infants 28 days to \< 12 weeks of age.
  • ✗Anuria or RRT defined as intermittent or continuous hemodialysis, peritoneal dialysis, hemofiltration,...
  • ✗Evidence of syndromic conditions associated with kidney cysts (other than ARPKD).
  • ✗Abnormal liver function tests including liver enzymes and liver enzymes, \> 1.2 × ULN (upper limit of normal).
  • ✗Has splenomegaly or portal hypertension (HTN).
See the full criteria
Inclusion Criteria: 1. Male or female subjects between 28 days and less than 18 years of age, with clinical features that are consistent with a diagnosis of ARPKD. 2. Ability for parent/legal 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. Ability to provide written informed assent from all subjects old enough per local laws to provide assent. Exclusion Criteria: 1. Premature birth (≤ 32 weeks gestational age) for infants 28 days to \< 12 weeks of 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 (upper limit of normal). 5. Has splenomegaly or portal hypertension (HTN). 6. Parents with renal cystic disease. 7. Receiving chronic diuretic that could not be adjusted after tolvaptan initiation. 8. Cannot be monitored for fluid balance. 9. Has or at risk of having sodium and potassium electrolyte imbalances, as determined by the investigator. 10. Has or at risk of having significant hypovolemia as determined by investigator. 11. Clinically significant anemia, as determined by investigator. 12. Platelets \< 50000 µL. 13. Severe systolic dysfunction defined as ejection fraction \< 14%. 14. Serum sodium levels \< 130 mmol/L or \>145 mmol/L. 15. Taking any other experimental medications. 16. Require ventilator support. 17. Taking medications known to induce CYP3A4 (CYP = Cytochrome P). 18. Having an infection including viral that would require therapy disruptive to IMP (Investigational Medicinal Product) dosing. 19. Females who are breast-feeding or who have a positive pregnancy test result prior to receiving IMP. 20. Subjects with a history of substance abuse (within the last 6 months). 21. Subjects who have bladder dysfunction and/or difficulty voiding. 22. Subjects taking a vasopressin agonist (eg, desmopressin). 23. Subjects with a history of persistent noncompliance with antihypertensive or other important medical therapy. 24. Subjects taking medications or having concomitant illnesses likely to confound endpoint assessments, including taking approved (ie, marketed) therapies for the purpose of affecting PKD cysts such as tolvaptan, vasopressin antagonists, anti-sense ribonucleic acid (RNA) therapies, rapamycin, sirolimus, everolimus, or somatostatin analogs (ie, octreotide, sandostatin). 25. Received or are scheduled to receive a liver transplant. 26. History of cholangitis within the last 6 months. 27. Has findings consistent with clinically significant portal hypertension (eg, varices, variceal bleeding, hypersplenism indicated by thrombocytopenia). 28. Subjects who do not agree to remain abstinent or assent to use a combination of 2 of the following highly effective birth control methods for at least 28 days before the first dose of IMP, during the trial (including during IMP dose interruptions), and for at least 30 days after the last dose of IMP: * Barrier method of contraception: condoms (male or female) with or without a spermicidal agent, diaphragm or cervical cap with spermicide * Intrauterine device * Hormone-based contraceptives which are associated with inhibition of ovulation.

Where Is This Study? (1 UK site)

Great Ormond Street Hospital for Children NHS Trust

London WC1N 3JH, United Kingdom

Recruiting
Hospital R&D contact (matched)

Main Email: Research.Governance@gosh.nhs.uk

Research.Governance@gosh.nhs.uk0207 905 2700

How to Get in Touch

Otsuka Call Center

Sponsor contact

CONTACT

844-687-8522 Otsuka-ProfessionalServices@otsuka-us.com
Data sourced from ClinicalTrials.gov · Last verified: 2026-08