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).

Conditions

Autosomal Recessive Polycystic Kidney Disease

Brief summary

Primary Efficacy: Percentage of subjects having RRT by 1 year of age

Detailed description

Main secondary efficacy endpoint: Rate of change of eGFR (eGFR Schwartz formula = 0.413 × height [or length, cm] /serum creatinine mg/dL) from pretreatment to post‑treatment after 2 years of treatment., Age-appropriate assessment of palatability and acceptability of suspension formulation., Change in sNa+ from baseline at each visit., Percentage of change in kidney size of height adjusted TKV at every time point from baseline to 24 months of treatment via ultrasound using ellipsoid methodology., Change from baseline (last assessment prior to dosing) in growth percentile trajectories for height (stature), weight, and head circumference at 3 months, 6 months, 12 months, 18 months, and 24 months., Summary of vital signs data., Adverse events (AEs), including rate of aquaretic AEs., Changes from baseline in serum creatinine and laboratory values including liver function tests (total bilirubin, ALT, AST, ALP, GGT).

Related papers

No related papers found yet.

Interventions

DRUGTolvaptan

Eligibility

Sex/Gender

All

Age

0 Years to 17 Years

Design outcomes

Primary

Measure	Time frame
Primary Efficacy: Percentage of subjects having RRT by 1 year of age	—

Secondary

Measure	Time frame
Main secondary efficacy endpoint: Rate of change of eGFR (eGFR Schwartz formula = 0.413 × height [or length, cm] /serum creatinine mg/dL) from pretreatment to post‑treatment after 2 years of treatment., Age-appropriate assessment of palatability and acceptability of suspension formulation., Change in sNa+ from baseline at each visit., Percentage of change in kidney size of height adjusted TKV at every time point from baseline to 24 months of treatment via ultrasound using ellipsoid methodology., Change from baseline (last assessment prior to dosing) in growth percentile trajectories for height (stature), weight, and head circumference at 3 months, 6 months, 12 months, 18 months, and 24 months., Summary of vital signs data., Adverse events (AEs), including rate of aquaretic AEs., Changes from baseline in serum creatinine and laboratory values including liver function tests (total bilirubin, ALT, AST, ALP, GGT).	—

Measure

Time frame

Main secondary efficacy endpoint: Rate of change of eGFR (eGFR Schwartz formula = 0.413 × height [or length, cm] /serum creatinine mg/dL) from pretreatment to post‑treatment after 2 years of treatment., Age-appropriate assessment of palatability and acceptability of suspension formulation., Change in sNa+ from baseline at each visit., Percentage of change in kidney size of height adjusted TKV at every time point from baseline to 24 months of treatment via ultrasound using ellipsoid methodology., Change from baseline (last assessment prior to dosing) in growth percentile trajectories for height (stature), weight, and head circumference at 3 months, 6 months, 12 months, 18 months, and 24 months., Summary of vital signs data., Adverse events (AEs), including rate of aquaretic AEs., Changes from baseline in serum creatinine and laboratory values including liver function tests (total bilirubin, ALT, AST, ALP, GGT).

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Countries

Belgium, Germany, Poland, Spain

Outcome results

None listed