Trial Outcomes & Findings for Effect of Statin Therapy on Disease Progression in Autosomal Dominant Polycystic Kidney Disease (ADPKD) (NCT NCT00456365)

NCT ID: NCT00456365

Last Updated: 2018-03-09

Results Overview

Percent of participants demonstrating 20% or more increase in total kidney volume corrected for height, left ventricular mass index, or urinary albumin excretion over the three year study period

• Recruitment status: COMPLETED
• Study phase: PHASE3
• Target enrollment: 110 participants
• Primary outcome timeframe: 3 years
• Results posted on: 2018-03-09

Participant Flow

Participant milestones

Measure	Pravastatin Pravastatin pravastatin: Pravastatin 20 mg daily (subject age 8-12 years) or 40 mg daily (subject age 13-21 years)	Placebo Placebo Placebo: Placebo daily
Overall Study STARTED	56	54
Overall Study COMPLETED	49	42
Overall Study NOT COMPLETED	7	12

Reasons for withdrawal

Measure	Pravastatin Pravastatin pravastatin: Pravastatin 20 mg daily (subject age 8-12 years) or 40 mg daily (subject age 13-21 years)	Placebo Placebo Placebo: Placebo daily
Overall Study Lost to Follow-up	1	3
Overall Study Withdrawal by Subject	4	7
Overall Study Pregnancy	1	1
Overall Study oncologic diagnosis	1	1

Baseline Characteristics

Effect of Statin Therapy on Disease Progression in Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Baseline characteristics by cohort

Measure	Pravastatin n=56 Participants Pravastatin pravastatin: Pravastatin 20 mg daily (subject age 8-12 years) or 40 mg daily (subject age 13-21 years)	Placebo n=54 Participants Placebo Placebo: Placebo daily	Total n=110 Participants Total of all reporting groups
Age, Continuous	16 years STANDARD_DEVIATION 4 • n=5 Participants	16 years STANDARD_DEVIATION 4 • n=7 Participants	16 years STANDARD_DEVIATION 4 • n=5 Participants
Sex: Female, Male Female	34 Participants n=5 Participants	34 Participants n=7 Participants	68 Participants n=5 Participants
Sex: Female, Male Male	22 Participants n=5 Participants	20 Participants n=7 Participants	42 Participants n=5 Participants
Region of Enrollment United States	56 participants n=5 Participants	54 participants n=7 Participants	110 participants n=5 Participants
Total kidney volume corrected for height	336 ml/m STANDARD_DEVIATION 187 • n=5 Participants	315 ml/m STANDARD_DEVIATION 175 • n=7 Participants	326 ml/m STANDARD_DEVIATION 181 • n=5 Participants
Urinary albumin excretion	27 mcg/min STANDARD_DEVIATION 37 • n=5 Participants	51 mcg/min STANDARD_DEVIATION 123 • n=7 Participants	39 mcg/min STANDARD_DEVIATION 79 • n=5 Participants
Left ventricular mass index	55 g/m^2 STANDARD_DEVIATION 12 • n=5 Participants	53 g/m^2 STANDARD_DEVIATION 10 • n=7 Participants	54 g/m^2 STANDARD_DEVIATION 11 • n=5 Participants
Total cholesterol	145 mg/dL STANDARD_DEVIATION 30 • n=5 Participants	151 mg/dL STANDARD_DEVIATION 27 • n=7 Participants	148 mg/dL STANDARD_DEVIATION 29 • n=5 Participants
LDL cholesterol	84 mg/dL STANDARD_DEVIATION 23 • n=5 Participants	90 mg/dL STANDARD_DEVIATION 21 • n=7 Participants	86 mg/dL STANDARD_DEVIATION 22 • n=5 Participants

PRIMARY outcome

Timeframe: 3 years

Population: Intention to treat

Percent of participants demonstrating 20% or more increase in total kidney volume corrected for height, left ventricular mass index, or urinary albumin excretion over the three year study period

Outcome measures

Measure	Pravastatin n=49 Participants Pravastatin pravastatin: Pravastatin 20 mg daily (subject age 8-12 years) or 40 mg daily (subject age 13-21 years)	Placebo n=42 Participants Placebo Placebo: Placebo daily
Percent of Participants Demonstrating 20% or More Increase in Total Kidney Volume	69 percentage of participants	88 percentage of participants

SECONDARY outcome

Timeframe: 3 years

Population: ITT

Outcome measures

Measure	Pravastatin n=49 Participants Pravastatin pravastatin: Pravastatin 20 mg daily (subject age 8-12 years) or 40 mg daily (subject age 13-21 years)	Placebo n=42 Participants Placebo Placebo: Placebo daily
Percentage Change in Total Kidney Volume Corrected for Height	23 Percent change Standard Deviation 3	31 Percent change Standard Deviation 3

SECONDARY outcome

Timeframe: 3 years

left ventricular mass index in g/m^2 by MRI

Outcome measures

Measure	Pravastatin n=49 Participants Pravastatin pravastatin: Pravastatin 20 mg daily (subject age 8-12 years) or 40 mg daily (subject age 13-21 years)	Placebo n=42 Participants Placebo Placebo: Placebo daily
Left Ventricular Mass Index	60 g/m^2 Standard Deviation 15	58 g/m^2 Standard Deviation 13

SECONDARY outcome

Timeframe: 3 years

Outcome measures

Measure	Pravastatin n=49 Participants Pravastatin pravastatin: Pravastatin 20 mg daily (subject age 8-12 years) or 40 mg daily (subject age 13-21 years)	Placebo n=42 Participants Placebo Placebo: Placebo daily
Urinary Albumin Excretion	29 mcg/min Standard Deviation 39	49 mcg/min Standard Deviation 99

Adverse Events

Pravastatin

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Placebo

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Melissa Cadnapaphornchai, MD

PKD Research Group, University of Colorado Anschutz Medical Campus

Phone: 3037241690

Email: pkd@ucdenver.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place