ADPKDPhase 4NCT03273413
Statin Therapy in Patients With Early Stage ADPKD
A Phase 4 study testing Pravastatin in people with ADPKD and Autosomal Dominant Polycystic Kidney, run by University of Colorado, Denver at 1 site in United States.
Trial summary
This study plans to learn if pravastatin is helpful in slowing down the progression of kidney disease in adults with autosomal dominant polycystic kidney disease (ADPKD). Pravastatin has been approved by the Food and Drug Administration (FDA) for adults for treatment of hyperlipidemia (high cholesterol levels). Read more about this study
- Who is it for?
- Ages 25 to 60 years
- Conditions the study is for
- ADPKD, Autosomal Dominant Polycystic Kidney
- What is being tested?
- Pravastatin
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 150 participants
- Where
- 1 siteUnited States
- Study dates
- 31 Aug 2017to 30 Sep 2025
- Phase
- Phase 4
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 25 to 60 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Diagnosis of ADPKD
- Total kidney volume >500 mL
- Estimated glomerular filtration rate (GFR) ≥60 mL/min/1.73m^2
- Controlled blood pressure <140/80 mmHg
Who cannot join
- Uncontrolled hypertension
- Diabetes mellitus
- Renal disease, renal cancer, single kidney, recent renal surgery, or acute kidney injury
- Unstable angina
- Coronary artery disease
- Prior ischemic stroke
Show all 18Show fewer
- Other clinical indication for a statin
- History of hospitalizations within the last 3 months
- Hepatic impairment or liver function abnormalities
- Secondary hypercholesterolemia or hypocholesterolemia
- Use of tolvaptan, gemfibrozil, other fibrates, niacin, clarithromycin, or cyclosporine
- Hypersensitivity to statins
- Immunosuppressive therapy within the last year
- Clinical contraindication for an MRI (i.e. implants, pacemaker, claustrophobia)
- Hypersensitivity to iodine
- Pregnant or breast feeding
- Current tobacco use
- Alcohol abuse or dependence
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Colorado Denver Anschutz Medical Campus
Denver, Colorado, 80045
About this study
This study plans to learn if pravastatin is helpful in slowing down the progression of kidney disease in adults with autosomal dominant polycystic kidney disease (ADPKD). Pravastatin has been approved by the Food and Drug Administration (FDA) for adults for treatment of hyperlipidemia (high cholesterol levels). The investigators are using pravastatin in this study as an investigational drug for treatment of ADPKD.
Read the detailed description
This is a randomized, double-blind, placebo-controlled trial designed to assess the efficacy and benefits of pravastatin therapy in adults with ADPKD by evaluating 1) kidney volume as measured by kidney magnetic resonance imaging (MRI); 2) renal blood flow as measured by kidney magnetic resonance angiography (MRA); 3) kidney function as assessed by Glofil-125; 4) plasma and urine protein markers that will allow a better understanding of how pravastatin works in ADPKD; and 5) blood vessel stiffness as measured by carotid-femoral pulse wave velocity. These parameters will be measured at baseline and after 2 years of pravastatin or placebo treatment in 150 patients with ADPKD.
- Conditions
- ADPKD, Autosomal Dominant Polycystic Kidney
- Being tested
- Pravastatin, Placebo
- Keywords
- Pravastatin, Statin
- Medical subject headings
- Polycystic Kidney, Autosomal Dominant
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PravastatinDrug
Anti-inflammatory, anti-oxidative stress, and anti-proliferative therapy
PlaceboDrug
Inactive tablet
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | Participants will receive inactive 40 mg tablets of placebo everyday for 6 weeks. If well tolerated, participants will continue taking inactive 40 mg dose of placebo everyday for 2 years. Receives: Drug: Placebo |
| Pravastatin | Active comparator | Participants will receive 40 mg tablets of pravastatin everyday for 6 weeks. If well tolerated, participants will continue taking 40 mg dose of pravastatin everyday for 2 years. Receives: Drug: Pravastatin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Total Kidney Volume
Total kidney volume as assessed by renal MRI, at baseline and after 2 years of treatment
Measured: Baseline, 2 years
Secondary outcomes (5)
Change in Renal Blood Flow
Renal blood flow, as assessed by renal MRA, at baseline and after 2 years of treatment
Measured: Baseline, 2 years
Change in Kidney Function
Glomerular filtration rate (GFR), as assessed by Glofil-125, at baseline and after 2 years of treatment
Measured: Baseline, 2 years
Change in Circulating Inflammatory Markers
Plasma levels of inflammatory cytokines and growth factors at baseline and after 2 years of treatment
Measured: Baseline, 2 years
Change in Circulating Markers of Oxidative Stress
Plasma levels of oxidative stress markers at baseline and after 2 years of treatment
Measured: Baseline, 2 years
Change in Urinary Epithelial Cells
Urinary epithelial cell protein expression, as assessed by immunofluorescence, at baseline and after 2 years of treatment
Measured: Baseline, 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Sep 2017
- Study start31 Aug 2017
- Primary completion25 Mar 2024
- Study completion30 Sep 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for ADPKDStudies of the same condition, open and under way.
- More trials run by University of Colorado, DenverThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Randomized, double-blind, placebo-controlled, parallel study design.
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 150 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Colorado, Denver
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Michel Chonchol, MD, Principal investigator, University of Colorado, Denver
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03273413
- Sponsor's protocol ID
- 17-0678
- Status last verified
- May 2025
- First submitted
- 1 Sep 2017
- Last updated
- 8 May 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Cadnapaphornchai MA, George DM, McFann K, Wang W, Gitomer B, Strain JD, Schrier RW. Effect of pravastatin on total kidney volume, left ventricular mass index, and microalbuminuria in pediatric autosomal dominant polycystic kidney disease. Clin J Am Soc Nephrol. 2014 May;9(5):889-96. doi: 10.2215/CJN.08350813. Epub 2014 Apr 10.
PubMed 24721893Gitomer BY, Ostrow A, Wang W, George D, Coleman E, Nowak KL, Cadnapaphornchai MA, Patel NU, Steele C, Jovanovich A, Klawitter J, Farmer B, You Z, Chonchol M. A randomized controlled trial evaluated the effect of pravastatin on kidney disease outcomes in adult patients with early-stage autosomal dominant polycystic kidney disease. Kidney Int. 2026 Feb;109(2):390-397. doi: 10.1016/j.kint.2025.08.037. Epub 2025 Oct 10.
PubMed 41077128St Pierre K, Cashmore BA, Bolignano D, Zoccali C, Ruospo M, Craig JC, Strippoli GF, Mallett AJ, Green SC, Tunnicliffe DJ. Interventions for preventing the progression of autosomal dominant polycystic kidney disease. Cochrane Database Syst Rev. 2024 Oct 2;10(10):CD010294. doi: 10.1002/14651858.CD010294.pub3.
PubMed 39356039Gitomer BY, Wang W, George D, Coleman E, Nowak KL, Struemph T, Cadnapaphornchai MA, Patel NU, Jovanovich A, Klawitter J, Farmer B, Ostrow A, You Z, Chonchol M. Statin therapy in patients with early-stage autosomal dominant polycystic kidney disease: Design and baseline characteristics. Contemp Clin Trials. 2024 Feb;137:107423. doi: 10.1016/j.cct.2023.107423. Epub 2023 Dec 25.
PubMed 38151173
Study at a glance
From the registry record, last updated by the sponsor on 8 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Statin Therapy in Patients With Early Stage ADPKD
- ClinicalTrials.gov ID
- NCT03273413
- Acronym / sponsor ID
- 17-0678
- Conditions
- ADPKD, Autosomal Dominant Polycystic Kidney
- Keywords
- Pravastatin, Statin
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 150 participants (actual)
- Lead sponsor
- University of Colorado, DenverUniversity, hospital or other
- Study start
- 31 Aug 2017 (actual)
- Primary completion
- 25 Mar 2024 (actual)
- Study completion
- 30 Sep 2025 (estimated)
- First posted
- 6 Sep 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study plans to learn if pravastatin is helpful in slowing down the progression of kidney disease in adults with autosomal dominant polycystic kidney disease (ADPKD). Pravastatin has been approved by the Food and Drug Administration (FDA) for adults for treatment of hyperlipidemia (high cholesterol levels). The investigators are using pravastatin in this study as an investigational drug for treatment of ADPKD.
Detailed description
This is a randomized, double-blind, placebo-controlled trial designed to assess the efficacy and benefits of pravastatin therapy in adults with ADPKD by evaluating 1) kidney volume as measured by kidney magnetic resonance imaging (MRI); 2) renal blood flow as measured by kidney magnetic resonance angiography (MRA); 3) kidney function as assessed by Glofil-125; 4) plasma and urine protein markers that will allow a better understanding of how pravastatin works in ADPKD; and 5) blood vessel stiffness as measured by carotid-femoral pulse wave velocity. These parameters will be measured at baseline and after 2 years of pravastatin or placebo treatment in 150 patients with ADPKD.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- Randomized, double-blind, placebo-controlled, parallel study design.
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Drug: PlaceboParticipants will receive inactive 40 mg tablets of placebo everyday for 6 weeks. If well tolerated, participants will continue taking inactive 40 mg dose of placebo everyday for 2 years. |
| Pravastatin | Active comparator | Drug: PravastatinParticipants will receive 40 mg tablets of pravastatin everyday for 6 weeks. If well tolerated, participants will continue taking 40 mg dose of pravastatin everyday for 2 years. |
Who may qualify
- Ages
- 25 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (4 listed)
- Diagnosis of ADPKD
- Total kidney volume >500 mL
- Estimated glomerular filtration rate (GFR) ≥60 mL/min/1.73m^2
- Controlled blood pressure <140/80 mmHg
Key exclusion criteria (18 listed)
- Uncontrolled hypertension
- Diabetes mellitus
- Renal disease, renal cancer, single kidney, recent renal surgery, or acute kidney injury
- Unstable angina
- Coronary artery disease
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PravastatinAnti-inflammatory, anti-oxidative stress, and anti-proliferative therapy | Drug | Pravastatin |
| PlaceboInactive tablet | Drug | Placebo |
Primary outcome
Change in Total Kidney Volume
Time frame: Baseline, 2 years
Total kidney volume as assessed by renal MRI, at baseline and after 2 years of treatment
5 secondary outcomes
Change in Renal Blood Flow
Time frame: Baseline, 2 years
Change in Kidney Function
Time frame: Baseline, 2 years
Change in Circulating Inflammatory Markers
Time frame: Baseline, 2 years
Change in Circulating Markers of Oxidative Stress
Time frame: Baseline, 2 years
Change in Urinary Epithelial Cells
Time frame: Baseline, 2 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 May 2025.
- Background: PubMed 24721893Cadnapaphornchai MA, George DM, McFann K, Wang W, Gitomer B, Strain JD, Schrier RW. Effect of pravastatin on total kidney volume, left ventricular mass index, and microalbuminuria in pediatric autosomal dominant polycystic kidney disease. Clin J Am Soc Nephrol. 2014 May;9(5):889-96. doi: 10.2215/CJN.08350813. Epub 2014 Apr 10.
- Derived from this study: PubMed 41077128Gitomer BY, Ostrow A, Wang W, George D, Coleman E, Nowak KL, Cadnapaphornchai MA, Patel NU, Steele C, Jovanovich A, Klawitter J, Farmer B, You Z, Chonchol M. A randomized controlled trial evaluated the effect of pravastatin on kidney disease outcomes in adult patients with early-stage autosomal dominant polycystic kidney disease. Kidney Int. 2026 Feb;109(2):390-397. doi: 10.1016/j.kint.2025.08.037. Epub 2025 Oct 10.
- Derived from this study: PubMed 39356039St Pierre K, Cashmore BA, Bolignano D, Zoccali C, Ruospo M, Craig JC, Strippoli GF, Mallett AJ, Green SC, Tunnicliffe DJ. Interventions for preventing the progression of autosomal dominant polycystic kidney disease. Cochrane Database Syst Rev. 2024 Oct 2;10(10):CD010294. doi: 10.1002/14651858.CD010294.pub3.
- Derived from this study: PubMed 38151173Gitomer BY, Wang W, George D, Coleman E, Nowak KL, Struemph T, Cadnapaphornchai MA, Patel NU, Jovanovich A, Klawitter J, Farmer B, Ostrow A, You Z, Chonchol M. Statin therapy in patients with early-stage autosomal dominant polycystic kidney disease: Design and baseline characteristics. Contemp Clin Trials. 2024 Feb;137:107423. doi: 10.1016/j.cct.2023.107423. Epub 2023 Dec 25.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Michel Chonchol, MD principal investigator
University of Colorado, Denver
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Pravastatin in people with ADPKD and Autosomal Dominant Polycystic Kidney, run by University of Colorado, Denver at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 8 May 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Total Kidney Volume (time frame: Baseline, 2 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03273413, which also lists 4 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 May 2025.