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HALT Progression of Polycystic Kidney Disease Study A

Phase 3
Completed
Conditions
Kidney, Polycystic
Interventions
Drug: Placebo
Other: Low Blood Pressure Control
Other: Standard Blood Pressure Control
Registration Number
NCT00283686
Lead Sponsor
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Brief Summary

The efficacy of interruption of the renin-angiotensin-aldosterone system (RAAS) on the progression of cystic disease and on the decline in renal function in autosomal dominant kidney disease (ADPKD) will be assessed in two multicenter randomized clinical trials targeting different levels of kidney function: 1) early disease defined by GFR \>60 mL/min/1.73 m2 (Study A); and 2) moderately advanced disease defined by GFR 25-60 mL/min/1.73 m2 (Study B; NCT01885559). Participants will be recruited and enrolled, either to Study A or B, over the first three years. Participants enrolled in Study A will be followed for at least 5 years, while those enrolled in Study B will be followed for five-to-eight years, with the average length of follow-up being six and a half years. The two concurrent randomized clinical trials differ by eligibility criteria, interventions and outcomes to be studied.

Detailed Description

\* Specific Aims of Study A

To study the efficacy of angiotensin-converting-enzyme inhibitor (ACE-I) and angiotensin-receptor blockade (ARB) combination therapy as compared to ACE-I monotherapy and usual vs. low blood pressure targets on the percent change in kidney volume in participants with preserved renal function (GFR \>60 mL/min/1.73m2)and high-normal blood pressure or hypertension (\>130/80 mm Hg).

\* Hypotheses to be tested in Study A

In ADPKD individuals with hypertension or high-normal blood pressure and relatively preserved renal function (GFR \>60 mL/min/1.73 m2), multi-level blockade of the RAAS using ACE-I/ARB combination therapy will delay progression of cystic disease as compared to ACE-I monotherapy, and a low blood pressure goal will delay progression as compared with standard control.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
558
Inclusion Criteria
  • Diagnosis of ADPKD.
  • Age 15-49 (Study A); Age 18-64 (Study B).
  • GFR >60 mL/min/1.73 m2 (Study A); GFR 25-60 mL/min/1.73 m2 (Study B).
  • BP ≥130/80 or receiving treatment for hypertension.
  • Informed Consent.
Exclusion Criteria
  • Pregnant/intention to become pregnant in 4-6 yrs.
  • Documented renal vascular disease.
  • Spot urine albumin-to-creatinine ratio of >0.5 (Study A) or ≥1.0 (Study B) and/or findings suggestive of kidney disease other than ADPKD.
  • Diabetes requiring insulin or oral hypoglycemic agents / fasting serum glucose of >126 mg/dl / random non-fasting glucose of >200 mg/dl.
  • Serum potassium >5.5 milliequivalent per liter (mEq/L) for participants currently on ACE-I or ARB; >5.0 mEq/L for participants not currently on ACE-I or ARB.
  • History of angioneurotic edema or other absolute contraindication for ACE-I or ARB. Intolerable cough associated with ACE-I is defined as a cough developing within six months of initiation of ACE-I in the absence of other causes and resolving upon discontinuation of the ACE-I.
  • Indication (other than hypertension) for β-blocker or calcium channel blocker therapy (e.g. angina, past myocardial infarction, arrhythmia), unless approved by the site principal investigator. (PI may choose to accept an individual who is on only a small dose of one of these agents and would otherwise be eligible.)
  • Systemic illness necessitating nonsteroidal antiinflammatory drugs (NSAIDs), immunosuppressant or immunomodulatory medications.
  • Systemic illness with renal involvement.
  • Hospitalized for acute illness in past 2 months.
  • Life expectancy <2 years.
  • History of non-compliance.
  • Unclipped cerebral aneurysm >7mm diameter.
  • Creatine supplements within 3 months of screening visit.
  • Congenital absence of a kidney (also total nephrectomy for Study B).
  • Known allergy to sorbitol or sodium polystyrene sulfonate.
  • Exclusions specific to magnetic resonance imaging (Study A).

Study & Design

Study Type
INTERVENTIONAL
Study Design
FACTORIAL
Arm && Interventions
GroupInterventionDescription
Study A, Arm 1LisinoprilLisinopril + Telmisartan (ACE-I + ARB) and standard blood pressure control of 120-130/70-80 mm Hg
Study A, Arm 2Low Blood Pressure ControlLisinopril + Telmisartan (ACE-I + ARB) and low blood pressure control of 95-110/60-75 mm Hg
Study A, Arm 1Standard Blood Pressure ControlLisinopril + Telmisartan (ACE-I + ARB) and standard blood pressure control of 120-130/70-80 mm Hg
Study A, Arm 3PlaceboLisinopril + Placebo (ACE-I + Placebo) and standard blood pressure control of 120-130/70-80 mm Hg
Study A, Arm 3Standard Blood Pressure ControlLisinopril + Placebo (ACE-I + Placebo) and standard blood pressure control of 120-130/70-80 mm Hg
Study A, Arm 4PlaceboLisinopril + Placebo (ACE-I + Placebo) and low blood pressure control of 95-110/60-75 mm Hg
Study A, Arm 4Low Blood Pressure ControlLisinopril + Placebo (ACE-I + Placebo) and low blood pressure control of 95-110/60-75 mm Hg
Study A, Arm 1TelmisartanLisinopril + Telmisartan (ACE-I + ARB) and standard blood pressure control of 120-130/70-80 mm Hg
Study A, Arm 2LisinoprilLisinopril + Telmisartan (ACE-I + ARB) and low blood pressure control of 95-110/60-75 mm Hg
Study A, Arm 2TelmisartanLisinopril + Telmisartan (ACE-I + ARB) and low blood pressure control of 95-110/60-75 mm Hg
Study A, Arm 3LisinoprilLisinopril + Placebo (ACE-I + Placebo) and standard blood pressure control of 120-130/70-80 mm Hg
Study A, Arm 4LisinoprilLisinopril + Placebo (ACE-I + Placebo) and low blood pressure control of 95-110/60-75 mm Hg
Primary Outcome Measures
NameTimeMethod
Study A: Percent Annual Change in Total Kidney VolumeBaseline and 2-, 4- and 5-year follow-up

Annual percentage change in total kidney volume as assessed by abdominal magnetic resonance imaging (MRI) at baseline, 2 years, 4 years, and 5 years follow-up.

Secondary Outcome Measures
NameTimeMethod
All-Cause HospitalizationsUp to 96 months
Quality of Life Mental Component Summarybaseline, 12, 24, 36, 48, 60, 72, 84, and 96 months (assessed annually)

Short Form-36 Quality of LIfe Mental Component Summary ranges from 0 (worst possible outcome) to 100 (best possible outcome)

AldosteroneUp to 96 months (assessed annually)

Urinary aldosterone excretion, centrally processed, 24 hour urine collection

Quality of Life Physical Component Summarybaseline, 12, 24, 36, 48, 60, 72, 84, and 96 months (assessed annually)

Short Form-36 Quality of Life Physical Component Summary ranges from 0 (worst possible outcome) to 100 (best possible outcome)

Kidney Function (eGFR)Up to 96 months (6 month assessments)

The estimated GFR was calculated by means of the Chronic Kidney Disease Epidemiology Collaboration equation with the use of central serum creatinine measurements.

Left Ventricular Mass Index0, 24 months, 48 months, 60 months

Left ventricular mass index (g/m\^2) measured by MRI, centrally reviewed and measured

AlbuminuriaUp to 96 months (assessed annually)

Urine albumin excretion, centrally processed from 24 hour urine collection

Renal Blood Flow0, 24 months, 48 months, 60 months

renal blood flow (mL/min/1.73 m\^2) from MRI, centrally reviewed and measured. This outcome was more difficult to measure resulting in more missing data than other MRI outcomes such as total kidney volume (TKV) and left ventricular mass index (LVMI).

Trial Locations

Locations (7)

University of Colorado Health Sciences Center

🇺🇸

Aurora, Colorado, United States

Tufts University-New England Medical Center

🇺🇸

Boston, Massachusetts, United States

Beth Israel Deaconess Medical Center

🇺🇸

Boston, Massachusetts, United States

Cleveland Clinic Foundation

🇺🇸

Cleveland, Ohio, United States

Mayo Clinic

🇺🇸

Rochester, Minnesota, United States

Emory University School of Medicine

🇺🇸

Atlanta, Georgia, United States

University of Kansas Medical Center

🇺🇸

Kansas City, Kansas, United States

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