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

ConditionAutosomal Recessive Polycystic Kidney (ARPKD)
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age28-18
SponsorOtsuka Pharmaceutical Development & Commercialization, Inc.

About this trial

To evaluate the safety of tolvaptan in pediatric subjects with ARPKD

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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.

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.

Disqualifiers

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, 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 (upper limit of normal).

Trial design

Design model

Parallel

Treatments tested in this trial

  • 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.

Treatment groups

20 Participants
are divided into 2 treatment groups
Group A: Tolvaptan SuspensionExperimental treatment 1 intervention
Group B: Tolvaptan TabletsExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Number of Participants Reporting Treatment-Emergent Adverse Events (TEAEs)

Time frame
Enrollment up to 7 days post last dose

Secondary outcomes

1

Annual rate of change of eGFR (by Schwartz formula) from baseline to post-treatment after 18 months of treatment

Time frame
From Enrollment to 18 months
2

Change from baseline of eGFR (by Schwartz formula) while on treatment at Months 1, 6, 12, and 18

Time frame
1 month, 6 months, 12 months, and 18 months
3

The percentage of subjects that will receive renal replacement therapy (RRT) by 18 months.

Time frame
From Enrollment to 18 months
4

The amount of time between enrollment and 18 months that a subject requires renal replacement therapy (RRT).

Time frame
From enrollment to 18 months

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Otsuka Pharmaceutical Development & Commercialization, Inc.

Lead sponsor

Source ClinicalTrials.gov
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