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.
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
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
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
Where Is This Study? (1 UK site)
Great Ormond Street Hospital for Children NHS Trust
London WC1N 3JH, United Kingdom
Main Email: Research.Governance@gosh.nhs.uk
Research.Governance@gosh.nhs.uk0207 905 2700How to Get in Touch
Otsuka Call Center
Sponsor contactCONTACT
