Autosomal Recessive Polycystic Kidney Disease (ARPKD)Phase 3NCT04786574
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)
A Phase 3 study testing Tolvaptan (OPC-41061) in people with Autosomal Recessive Polycystic Kidney Disease (ARPKD), run by Otsuka Pharmaceutical Development & Commercialization, Inc..
Trial 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) Read more about this study
- Who is it for?
- Ages 4 to 12 weeks
- Conditions the study is for
- Autosomal Recessive Polycystic Kidney Disease (ARPKD)
- What is being tested?
- Tolvaptan (OPC-41061)
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- 1 Jul 2022to 10 Dec 2025
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 4 to 12 weeks
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.
Who cannot join
- 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
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- 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
About this study
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)
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)
Read the 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.
- Being tested
- Tolvaptan (OPC-41061)
- Keywords
- ARPKD, Tolvaptan, Polycystic Kidney Disease, Autosomal Recessive Polycystic Kidney Disease, Renal Cysts, Nephromegaly, Oligohydramnios, Anhydramnios
- Medical subject headings
- Polycystic Kidney, Autosomal Recessive, Polycystic Kidney Diseases, Oligohydramnios
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Tolvaptan (OPC-41061)Drug
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.
| Study group | Role | What they receive |
|---|---|---|
| Tolvaptan (OPC-41061) | Experimental | Not described Receives: Drug: Tolvaptan (OPC-41061) |
What is being measured
The primary outcome is the main question the study is designed to answer.
The percentage of subjects that will have Renal Replacement Therapy (RRT) by 1 year of age.
Measured: From Enrollment to 1 year of age
Secondary outcomes (3)
Rate of change of eGFR by Schwartz formula from pre-treatment to after 2 years of treatment
Measured: 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
Measured: 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
Measured: From Enrollment to 2 years of age
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Mar 2021
- Study start1 Jul 2022
- Primary completion10 Dec 2025
- Study completion10 Dec 2025
Why it stopped: Study stopped before enrolling any participants.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Autosomal Recessive Polycystic Kidney Disease (ARPKD)Studies of the same condition, open and under way.
- More trials run by Otsuka Pharmaceutical Development & Commercialization, Inc.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Olga Sergeyeva, MD, Study director, Otsuka Pharmaceutical Development & Commercialization, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04786574
- Sponsor's protocol ID
- 156-12-204
- Status last verified
- Dec 2025
- First submitted
- 19 Feb 2021
- Last updated
- 24 Dec 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT04786574
- Acronym / sponsor ID
- 156-12-204
- Keywords
- ARPKD, Tolvaptan, Polycystic Kidney Disease, Autosomal Recessive Polycystic Kidney Disease, Renal Cysts, Nephromegaly, Oligohydramnios, Anhydramnios
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Study stopped before enrolling any participants.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.Industry (pharma, biotech, devices)
- Study start
- 1 Jul 2022 (actual)
- Primary completion
- 10 Dec 2025 (actual)
- Study completion
- 10 Dec 2025 (actual)
- First posted
- 8 Mar 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Tolvaptan (OPC-41061) | Experimental | Drug: Tolvaptan (OPC-41061) |
Who may qualify
- Ages
- 4 to 12 weeks
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- 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:
- 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.
Key exclusion criteria (23 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 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. | Drug | Tolvaptan (OPC-41061) |
Primary outcome
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
3 secondary outcomes
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
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Dec 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Olga Sergeyeva, MD study director
Otsuka Pharmaceutical Development & Commercialization, Inc.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Tolvaptan (OPC-41061) in people with Autosomal Recessive Polycystic Kidney Disease (ARPKD), run by Otsuka Pharmaceutical Development & Commercialization, Inc..
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 23 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 24 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Dec 2025.