Trial Outcomes & Findings for HALT Progression of Polycystic Kidney Disease Study B (NCT NCT01885559)

NCT ID: NCT01885559

Last Updated: 2020-04-22

Results Overview

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

486 participants

Primary outcome timeframe

Patients followed for 5-8 years with average of 6.5 years follow up

Results posted on

2020-04-22

Participant Flow

Recruitment for HALT PKD Study B occurred at seven clinical sites between February 2006 and June 2009.

Participant milestones

Participant milestones
Measure	ACE-I + Placebo ACE-I + placebo and standard blood pressure control of 110-130/80 mm Hg Lisinopril and Placebo: Lisinopril titrated to 5mg, 10mg, 20mg, 40mg and placebo titrated to 40mg and 80mg, as tolerated by participants, to achieve standard blood pressure control of 110-130/80 mm Hg.	ACE-I + Angiotensin Receptor Blocker (ARB) ACE-I + angiotensin receptor blocker (ARB) and standard blood pressure control of 110-130/80 mm Hg Lisinopril and Telmisartan: Lisinopril titrated to 5mg, 10mg, 20mg, 40mg and telmisartan titrated to 40mg and 80mg, as tolerated by participants, to achieve standard blood pressure control of 110-130/80 mm Hg.
Overall Study STARTED	242	244
Overall Study COMPLETED	218	208
Overall Study NOT COMPLETED	24	36

Reasons for withdrawal

Reasons for withdrawal
Measure	ACE-I + Placebo ACE-I + placebo and standard blood pressure control of 110-130/80 mm Hg Lisinopril and Placebo: Lisinopril titrated to 5mg, 10mg, 20mg, 40mg and placebo titrated to 40mg and 80mg, as tolerated by participants, to achieve standard blood pressure control of 110-130/80 mm Hg.	ACE-I + Angiotensin Receptor Blocker (ARB) ACE-I + angiotensin receptor blocker (ARB) and standard blood pressure control of 110-130/80 mm Hg Lisinopril and Telmisartan: Lisinopril titrated to 5mg, 10mg, 20mg, 40mg and telmisartan titrated to 40mg and 80mg, as tolerated by participants, to achieve standard blood pressure control of 110-130/80 mm Hg.
Overall Study Lost to Follow-up	15	16
Overall Study Less than full participation	9	19
Overall Study Protocol Violation	0	1

Baseline Characteristics

HALT Progression of Polycystic Kidney Disease Study B

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	ACE-I + Placebo n=242 Participants ACE-I + placebo and standard blood pressure control of 110-130/80 mm Hg Lisinopril and Placebo: Lisinopril titrated to 5mg, 10mg, 20mg, 40mg and placebo titrated to 40mg and 80mg, as tolerated by participants, to achieve standard blood pressure control of 110-130/80 mm Hg.	ACE-I + ARB n=244 Participants ACE-I + ARB and standard blood pressure control of 110-130/80 mm Hg Lisinopril and Telmisartan: Lisinopril titrated to 5mg, 10mg, 20mg, 40mg and telmisartan titrated to 40mg and 80mg, as tolerated by participants, to achieve standard blood pressure control of 110-130/80 mm Hg.	Total n=486 Participants Total of all reporting groups
Age, Continuous	48.9 years STANDARD_DEVIATION 8.1 • n=5 Participants	48.6 years STANDARD_DEVIATION 8.5 • n=7 Participants	48.7 years STANDARD_DEVIATION 8.3 • n=5 Participants
Sex: Female, Male Female	122 Participants n=5 Participants	129 Participants n=7 Participants	251 Participants n=5 Participants
Sex: Female, Male Male	120 Participants n=5 Participants	115 Participants n=7 Participants	235 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	2 Participants n=5 Participants	2 Participants n=7 Participants	4 Participants n=5 Participants
Race (NIH/OMB) Asian	5 Participants n=5 Participants	2 Participants n=7 Participants	7 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	7 Participants n=5 Participants	5 Participants n=7 Participants	12 Participants n=5 Participants
Race (NIH/OMB) White	224 Participants n=5 Participants	230 Participants n=7 Participants	454 Participants n=5 Participants
Race (NIH/OMB) More than one race	4 Participants n=5 Participants	4 Participants n=7 Participants	8 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants
PKD Genotype PKD1	183 participants n=5 Participants	179 participants n=7 Participants	362 participants n=5 Participants
PKD Genotype PKD2	30 participants n=5 Participants	30 participants n=7 Participants	60 participants n=5 Participants
PKD Genotype No Mutation Detected	11 participants n=5 Participants	14 participants n=7 Participants	25 participants n=5 Participants
PKD Genotype No data	18 participants n=5 Participants	21 participants n=7 Participants	39 participants n=5 Participants
Age at Diagnosis of Autosomal Dominant Polycystic Kidney Disease (yrs)	33.5 years STANDARD_DEVIATION 12.4 • n=5 Participants	33.0 years STANDARD_DEVIATION 12.0 • n=7 Participants	33.2 years STANDARD_DEVIATION 12.2 • n=5 Participants
Body Mass Index (kg/m2)	28.0 kg/m2 STANDARD_DEVIATION 5.5 • n=5 Participants	28.0 kg/m2 STANDARD_DEVIATION 4.9 • n=7 Participants	28.0 kg/m2 STANDARD_DEVIATION 5.2 • n=5 Participants
Serum Creatinine (mg/dl)	1.6 mg/dl STANDARD_DEVIATION 0.4 • n=5 Participants	1.5 mg/dl STANDARD_DEVIATION 0.4 • n=7 Participants	1.6 mg/dl STANDARD_DEVIATION 0.4 • n=5 Participants
Chronic Kidney Disease Epidemiology Collaboration (CKD EPI) eGFR (ml/min/m^2)	47.9 ml/min/m^2 STANDARD_DEVIATION 12.2 • n=5 Participants	48.5 ml/min/m^2 STANDARD_DEVIATION 11.5 • n=7 Participants	48.2 ml/min/m^2 STANDARD_DEVIATION 11.8 • n=5 Participants

PRIMARY outcome

Timeframe: Patients followed for 5-8 years with average of 6.5 years follow up

Population: Intention to Treat analysis was used for the primary outcome

Outcome measures

Outcome measures
Measure	ACE-I + Placebo n=242 Participants ACE-I + placebo and standard blood pressure control of 110-130/80 mm Hg Lisinopril and Placebo: Lisinopril titrated to 5mg, 10mg, 20mg, 40mg and placebo titrated to 40mg and 80mg, as tolerated by participants, to achieve standard blood pressure control of 110-130/80 mm Hg.	ACE-I + ARB n=243 Participants ACE-I + ARB and standard blood pressure control of 110-130/80 mm Hg Lisinopril and Telmisartan: Lisinopril titrated to 5mg, 10mg, 20mg, 40mg and telmisartan titrated to 40mg and 80mg, as tolerated by participants, to achieve standard blood pressure control of 110-130/80 mm Hg.
Number of Participants With 50% Reduction of Baseline eGFR, End Stage Renal Disease (ESRD, Initiation of Dialysis or Preemptive Transplant), or Death.	116 participants	115 participants

SECONDARY outcome

Timeframe: up to 8 years (annually assessed)

Population: Intention to treat analysis

Annual percent change in 24 hour urine albumin, centrally processed. Data from multiple years were analyzed with the primary focus on the change over time for the measure (from the slope of the model). The measure presented is the average annual percent change across the 8 years.

Outcome measures

Outcome measures
Measure	ACE-I + Placebo n=242 Participants ACE-I + placebo and standard blood pressure control of 110-130/80 mm Hg Lisinopril and Placebo: Lisinopril titrated to 5mg, 10mg, 20mg, 40mg and placebo titrated to 40mg and 80mg, as tolerated by participants, to achieve standard blood pressure control of 110-130/80 mm Hg.	ACE-I + ARB n=243 Participants ACE-I + ARB and standard blood pressure control of 110-130/80 mm Hg Lisinopril and Telmisartan: Lisinopril titrated to 5mg, 10mg, 20mg, 40mg and telmisartan titrated to 40mg and 80mg, as tolerated by participants, to achieve standard blood pressure control of 110-130/80 mm Hg.
Albuminuria	7.5 annual percent change Interval 5.2 to 9.9	7.3 annual percent change Interval 4.9 to 9.7

SECONDARY outcome

Timeframe: up at 8 years (annually assessed)

Population: Intention to treat analyses

Annual percent change in urinary aldosterone, centrally processed measure. Data from multiple years were analyzed with the primary focus on the change over time for the measure (from the slope for time from the model). The measure presented is the average annual percent change across the 8 years.

Outcome measures

Outcome measures
Measure	ACE-I + Placebo n=242 Participants ACE-I + placebo and standard blood pressure control of 110-130/80 mm Hg Lisinopril and Placebo: Lisinopril titrated to 5mg, 10mg, 20mg, 40mg and placebo titrated to 40mg and 80mg, as tolerated by participants, to achieve standard blood pressure control of 110-130/80 mm Hg.	ACE-I + ARB n=243 Participants ACE-I + ARB and standard blood pressure control of 110-130/80 mm Hg Lisinopril and Telmisartan: Lisinopril titrated to 5mg, 10mg, 20mg, 40mg and telmisartan titrated to 40mg and 80mg, as tolerated by participants, to achieve standard blood pressure control of 110-130/80 mm Hg.
Aldosterone	-8.8 annual percent change Interval -10.5 to -7.2	-10.2 annual percent change Interval -11.9 to -8.6

SECONDARY outcome

Timeframe: up to 8 years

Population: Intention to treat analysis

Hospitalization for any cause

Outcome measures

Outcome measures
Measure	ACE-I + Placebo n=242 Participants ACE-I + placebo and standard blood pressure control of 110-130/80 mm Hg Lisinopril and Placebo: Lisinopril titrated to 5mg, 10mg, 20mg, 40mg and placebo titrated to 40mg and 80mg, as tolerated by participants, to achieve standard blood pressure control of 110-130/80 mm Hg.	ACE-I + ARB n=243 Participants ACE-I + ARB and standard blood pressure control of 110-130/80 mm Hg Lisinopril and Telmisartan: Lisinopril titrated to 5mg, 10mg, 20mg, 40mg and telmisartan titrated to 40mg and 80mg, as tolerated by participants, to achieve standard blood pressure control of 110-130/80 mm Hg.
Hospitalizations	173 events	136 events

SECONDARY outcome

Timeframe: up to 8 years

Population: Intention to Treat analysis

Cause-specific hospitalizations (cardiovascular)

Outcome measures

Outcome measures
Measure	ACE-I + Placebo n=242 Participants ACE-I + placebo and standard blood pressure control of 110-130/80 mm Hg Lisinopril and Placebo: Lisinopril titrated to 5mg, 10mg, 20mg, 40mg and placebo titrated to 40mg and 80mg, as tolerated by participants, to achieve standard blood pressure control of 110-130/80 mm Hg.	ACE-I + ARB n=243 Participants ACE-I + ARB and standard blood pressure control of 110-130/80 mm Hg Lisinopril and Telmisartan: Lisinopril titrated to 5mg, 10mg, 20mg, 40mg and telmisartan titrated to 40mg and 80mg, as tolerated by participants, to achieve standard blood pressure control of 110-130/80 mm Hg.
Cardiovascular Hospitalizations	29 events	16 events

SECONDARY outcome

Timeframe: up to 8 years (annually assessed)

Population: Intention to Treat analysis

Short Form-36 Quality of Life Physical Component Summary ranges from 0 (worst possible outcome) to 100 (best possible outcome). Data from multiple years were analyzed with the primary focus on the change over time for the measure (from the slope for time from the model). The measure presented is the average annual change across the 8 years.

Outcome measures

Outcome measures
Measure	ACE-I + Placebo n=242 Participants ACE-I + placebo and standard blood pressure control of 110-130/80 mm Hg Lisinopril and Placebo: Lisinopril titrated to 5mg, 10mg, 20mg, 40mg and placebo titrated to 40mg and 80mg, as tolerated by participants, to achieve standard blood pressure control of 110-130/80 mm Hg.	ACE-I + ARB n=243 Participants ACE-I + ARB and standard blood pressure control of 110-130/80 mm Hg Lisinopril and Telmisartan: Lisinopril titrated to 5mg, 10mg, 20mg, 40mg and telmisartan titrated to 40mg and 80mg, as tolerated by participants, to achieve standard blood pressure control of 110-130/80 mm Hg.
Quality of Life Physical Component Summary	-0.64 units on a scale per year Interval -0.79 to -0.5	-0.68 units on a scale per year Interval -0.84 to -0.52

SECONDARY outcome

Timeframe: up to 8 years (annually assessed)

Population: Intention to treat analysis

Short Form-36 Quality of Life Mental Component Summary ranges from 0 (worst possible outcome) to 100 (best possible outcome). Data from multiple years were analyzed with the primary focus on the change over time for the measure (from the slope for time from the model). The measure presented is the average annual change across the 8 years.

Outcome measures

Outcome measures
Measure	ACE-I + Placebo n=242 Participants ACE-I + placebo and standard blood pressure control of 110-130/80 mm Hg Lisinopril and Placebo: Lisinopril titrated to 5mg, 10mg, 20mg, 40mg and placebo titrated to 40mg and 80mg, as tolerated by participants, to achieve standard blood pressure control of 110-130/80 mm Hg.	ACE-I + ARB n=243 Participants ACE-I + ARB and standard blood pressure control of 110-130/80 mm Hg Lisinopril and Telmisartan: Lisinopril titrated to 5mg, 10mg, 20mg, 40mg and telmisartan titrated to 40mg and 80mg, as tolerated by participants, to achieve standard blood pressure control of 110-130/80 mm Hg.
Quality of Life Mental Component Summary	-0.031 units on a scale per year Interval -0.19 to 0.12	-0.079 units on a scale per year Interval -0.24 to 0.08

SECONDARY outcome

Timeframe: 48 months

Population: Cross sectional analysis at 48 months is reported only for those participants responding at that time point. Intention to treat analysis was used for in the modeling over time to incorporate all repeated measures.

Report of back or flank pain since the last visit (yes or no)

Outcome measures

Outcome measures
Measure	ACE-I + Placebo n=184 Participants ACE-I + placebo and standard blood pressure control of 110-130/80 mm Hg Lisinopril and Placebo: Lisinopril titrated to 5mg, 10mg, 20mg, 40mg and placebo titrated to 40mg and 80mg, as tolerated by participants, to achieve standard blood pressure control of 110-130/80 mm Hg.	ACE-I + ARB n=179 Participants ACE-I + ARB and standard blood pressure control of 110-130/80 mm Hg Lisinopril and Telmisartan: Lisinopril titrated to 5mg, 10mg, 20mg, 40mg and telmisartan titrated to 40mg and 80mg, as tolerated by participants, to achieve standard blood pressure control of 110-130/80 mm Hg.
Back or Flank Pain	43 percentage of participants at 48 months Interval 36.0 to 50.0	46 percentage of participants at 48 months Interval 39.0 to 53.0

Adverse Events

ACE-I + Placebo

Serious events: 88 serious events

Other events: 66 other events

Deaths: 0 deaths

ACE-I + ARB

Serious events: 88 serious events

Other events: 60 other events

Deaths: 0 deaths

Serious adverse events

Other adverse events

Other adverse events
Measure	ACE-I + Placebo n=242 participants at risk ACE-I + placebo and standard blood pressure control of 110-130/80 mm Hg Lisinopril and Placebo: Lisinopril titrated to 5mg, 10mg, 20mg, 40mg and placebo titrated to 40mg and 80mg, as tolerated by participants, to achieve standard blood pressure control of 110-130/80 mm Hg.	ACE-I + ARB n=244 participants at risk ACE-I + ARB and standard blood pressure control of 110-130/80 mm Hg Lisinopril and Telmisartan: Lisinopril titrated to 5mg, 10mg, 20mg, 40mg and telmisartan titrated to 40mg and 80mg, as tolerated by participants, to achieve standard blood pressure control of 110-130/80 mm Hg.
Renal and urinary disorders Acute Kidney Injury	13.2% 32/242 • Number of events 51 • Serious adverse event data were collected during follow up between 5-8 years with average follow up time of 5.2 years.	9.0% 22/244 • Number of events 31 • Serious adverse event data were collected during follow up between 5-8 years with average follow up time of 5.2 years.
Metabolism and nutrition disorders Hyperkalemia	16.9% 41/242 • Number of events 65 • Serious adverse event data were collected during follow up between 5-8 years with average follow up time of 5.2 years.	18.9% 46/244 • Number of events 70 • Serious adverse event data were collected during follow up between 5-8 years with average follow up time of 5.2 years.

Additional Information

Dr. Vicente Torres

Mayo Clinic

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place