RecruitingPhase 3

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)

United States20 participantsStarted 2023-01-23

Plain-language summary

To evaluate the safety of tolvaptan in pediatric subjects with ARPKD

Age range28 Days – 18 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. 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.

✕. 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).
✕. Has splenomegaly or portal hypertension (HTN).
✕. Parents with renal cystic disease.
✕. Receiving chronic diuretic that could not be adjusted after tolvaptan initiation.
✕. Cannot be monitored for fluid balance.

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

Timeframe: Enrollment up to 7 days post last dose

NCT IDNCT04782258

SponsorOtsuka Pharmaceutical Development & Commercialization, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2028-07-31

Otsuka Call Center

Who can participate

Age range28 Days – 18 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. 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.

✕. 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).
✕. Has splenomegaly or portal hypertension (HTN).
✕. Parents with renal cystic disease.
✕. Receiving chronic diuretic that could not be adjusted after tolvaptan initiation.
✕. Cannot be monitored for fluid balance.