Autosomal Dominant Polycystic Kidney DiseasePhase 2ADPKD-SATNCT04284657
Pravastatin and Alkali Therapy in Patients With Autosomal Dominant Polycystic Kidney Disease
A Phase 2 study testing Pravastatin and sodium citrate in people with Autosomal Dominant Polycystic Kidney Disease, run by University of Southern California at 1 site in United States.
Trial summary
This is an one-year open-label study to determine treatment efficacy and feasibility of a trial that uses open-label interventions in ADPKD patients. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Autosomal Dominant Polycystic Kidney Disease
- What is being tested?
- Pravastatin and sodium citrate
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 1 participants
- Where
- 1 siteUnited States
- Study dates
- 30 Jan 2019to 4 Aug 2025
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patient voluntarily gives informed consent to participate in the study and signed study's IC and HIPAA.
- Patient is age 18 or older at the time of consent.
- If applicable, female of reproductive potential (Females who are successfully sterilized (surgical sterilization methods include hysterectomy, bilateral tubal ligation, or bilateral oophorectomy) or are postmenopausal (defined as amenorrhea for at least 12 consecutive months) are not considered to be of reproductive potential) must be non-pregnant (as confirmed by a urine pregnancy test at screening) and non-lactating, and agree:
- Either abstain from intercourse (when it is in line with their preferred and usual lifestyle), or
- Use 2 medically acceptable, highly-effective forms of contraception for the duration of study, and at least 30 days after discontinuing study drug (highly-effective forms of contraception can include approved hormonal contraceptives (oral, injectable, and implantable), and barrier methods (such as a condom or diaphragm) when used with a spermicide.))
- Patients has ADPKD diagnosed by unified criteria using a combination of ultrasound results, genotyping and MRI as needed (1, 2). Kidney ultrasound is usually used for screening because it is safe, effective, and inexpensive. Diagnostic criteria are based upon whether the genotype is known. Disease severity varies between the different genotypes. The great majority of patients at risk for ADPKD are from families with an unknown genotype. This diagnosis will take place prior to recruitment / inclusion into the study.
Show all 14Show fewer
- The following ultrasonographic criteria for the diagnosis of ADPKD are for at-risk patients from families of where the genotype is not known:
- If the patient is between 18 and 39 years of age, at least three unilateral or bilateral kidney cysts. The specificity and positive predictive value at this age-range is 100 percent. (sensitivity of 82 and 96 percent for individuals between 15 and 29 years and between 30 to 39 years of age, respectively).
- If the patient is 40 to 59 years of age, at least two cysts in each kidney (sensitivity, specificity, and positive predictive value of 90, 100, and 100 percent, respectively).
- Among individuals 60 years or older, at least four cysts in each kidney. (100 percent sensitivity and specificity).
- The above patients with estimated GFR ≥30 ml/min i.e. with stage 1-3b CKD
- Plasma bicarbonate ≤ 25 mMol/L
- Metabolic acidosis
- The patient agrees to immediately inform Investigator and research coordinator of any changes or planned changes in concomitant medication
Who cannot join
- Patients with known allergy or sensitive to Pravastatin or NaCitrate
- Acute coronary disease, liver disease, muscle disease, or a history of pulmonary edema
- Creatine Phospho Kinase (CPK) > 2ULN (2.5 ULN in African Americans). Elevated creatine phosphokinase could be a marker of rhabdomyolysis, which is a potential side effect of pravastatin. In general, patients with African American ancestry can have higher normal level of CPK
- Patients with systemic disease that impacting kidney per Investigator's decision
- Patients with known unstable cerebral aneurysm per Investigator's decision
- Pregnancy or lactation, or patients who refuse to use recommended contraception methods
Show all 24Show fewer
- Proteinuria > 500 mg/day
- History of non-compliance of medication per Investigator's decision
- Patients with uncontrolled hypertension, edema, or development of severe MA as per Investigator's decision
- History of cancer
- History of liver disease: hepatic failure/shock, cirrhosis
- Current or planned use of any of prohibited concomitant medication
- Patients with history of nephrolithiasis
- Following medications prohibited at the time of enrollment and during the study and if the patient is started on these medications then the patient will be excluded from the study:
- rapamycin or its analogues
- tolvaptan
- spironolactone
- cimetidine and ketoconazole
- erythromycin
- cyclosporine
- gemfibrozil
- colchicine
- niacin (>1 g/day)
- other lipid lowering medications in the class of statins
Trial locations and contacts
1 site in 1 country.
United States1 site
Keck School of Medicine of University of Southern California
Los Angeles, California, 90033
About this study
This is an one-year open-label study to determine treatment efficacy and feasibility of a trial that uses open-label interventions in ADPKD patients.
Read the detailed description
Polycystic Kidney Disease (PKD) is the most common genetic disease leading to End Stage Kidney Disease (ESKD), affecting between 1 in 500-1000 individuals from every ethnic group. The autosomal dominant (ADPKD) form arises from a two-hit downregulation of proteins encoded by either PKD1 or PKD2. Although many potential therapies have been studied to slow progression of ADPKD, none to date have been proven to be both safe and effective in slowing disease progression. Cholesterol-lowering agents called statins have shown promise in the treatment of younger ADPKD patients, reducing inflammation and progression as assessed by kidney growth, but their utility appears to be limited in older populations and those with more advanced chronic kidney disease (CKD). Recent evidence suggests that acidosis, as often seen in patients with worsening CKD and which may enhance CKD progression, limits the effectiveness of statins and enhances their potential toxicity. The investigators thus hypothesize that correction of acidosis along with statin treatment will be a safe and effective therapeutic regimen to slow CKD progression in the adult ADPKD population and improve overall quality of life in these patients. To test this hypothesis, the investigators will conduct a pilot open-label randomized clinical trial in ADPKD patients with estimated GFR >45 min (Stage 1-3a CKD) comparing three treatment groups: control, pravastatin (40 mg po qd), and pravastatin plus sodium citrate solution (30 mL po total daily dose) over one year. During the study period, through study visits along with serial blood draws and urinary measurements, the investigators will evaluate safety and tolerability of these treatment regimens, follow renal function and investigate the role of these treatments on acidosis, inflammatory and metabolic biomarkers in patients enrolled at an outpatient facility. This study will establish the framework for larger clinical trials in ADPKD. Moreover, if the results of this study suggest safety/tolerability or potential benefits of statins and alkali therapy in this ADPKD population, the investigators will seek extramural funding for a larger clinical trial to test this therapeutic strategy in ADPKD.
- Being tested
- Pravastatin, sodium citrate
- Keywords
- Polycystic Kidney Disease, ADPKD, PKD
- Medical subject headings
- Polycystic Kidney, Autosomal Dominant, Polycystic Kidney Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PravastatinDrug
Pravastatin 40 mg QD
sodium citrateDrug
sodium citrate up to 30 ml TID
| Study group | Role | What they receive |
|---|---|---|
| ARM I: Control group | No intervention | Standard therapy alone |
| ARM II: PRAVASTATIN | Active comparator | Standard therapy and PRAVASTATIN 40 mg QD Receives: Drug: Pravastatin |
| ARM III: PRAV + Sodium Citrate | Active comparator | Standard therapy and PRAVASTATIN 40 mg QD and Sodium Citrate (up to 30 mL TID) Receives: Drug: Pravastatin; Drug: sodium citrate |
What is being measured
The primary outcome is the main question the study is designed to answer.
Changes in kidney function in patients enrolled in different arms of the study
The investigators will estimate the effect of Pravastatin and NaCitrate on kidney function in patients with ADPKD compared to Pravastatin alone or Standard therapy: serum creatinine and serum blood urea nitrogen, serum electrolytes contribute to evaluate kidney function.
Measured: 12 months
Changes in liver function in patients enrolled in different arms of the study
The investigators will test liver function test panels in patients with ADPKD compared to Pravastatin alone or Standard therapy. The liver function panel should be within normal limits for enrollment and continuation in the study. Liver function tests include AST > 3ULN, ALT > 3ULN, Total Bilirubin > 2 ULN, or increase in prothrombin time to abnormal level INR >1.5 repeated two weeks apart
Measured: 12 months
Changes in blood pressure in patients enrolled in different arms of the study
The investigators will estimate the effect of Pravastatin and NaCitrate on blood pressure (systolic and diastolic) in patients with ADPKD compared to Pravastatin alone or Standard therapy
Measured: 12 months
Changes in muscle injury marker function in patients enrolled in different arms of the study
The investigators will estimate the effect of Pravastatin and NaCitrate on creatine phospho kinase (CPK) in patients with ADPKD compared to Pravastatin alone or Standard therapy.
Measured: 12 months
Changes in muscle tenderness in patients enrolled in the study
The investigators will estimate the effect of Pravastatin and NaCitrate on muscle tenderness in patients with ADPKD compared to Pravastatin alone or Standard therapy. The physical exam will evaluate tenderness to palpation in major muscle groups such as leg, arm and back muscles. It will be graded as presence or absence. The patients will only be enrolled if there is absence of tenderness in muscles upon palpation on physical exam. If there is tenderness on exam or the patient reports tenderness that is then confirmed by exam the patient will be removed from the study.
Measured: 12 months
Secondary outcomes (3)
Urinary alkalinization changes
These parameters will be ascertained by measurements of urinary pH and serum electrolytes.
Measured: 12 months
Inflammatory markers in blood and urine
The investigators will assess inflammatory markers (interleukins, prostaglandins and other cytokines) in leukocytes, plasma, and urine in the different study groups.
The biomarkers that will be measured are
HETE / HODE species phospho-AMPK Inflammatory and metabolic biomarkers: NGAL, KIM1 in blood and urine
Measured: 12 months
AMPK pathway activation
The investigators will determine whether and to what extent the AMPK pathway is activated in leukocytes and urine derived from patients in the study groups at the different study visits, and correlate AMPK activation with biological effects.
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Feb 2020
- Study start30 Jan 2019
- Primary completion4 Aug 2025
- Study completion4 Aug 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Autosomal Dominant Polycystic Kidney DiseaseStudies of the same condition, open and under way.
- More trials run by University of Southern CaliforniaThe 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 1 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Southern California
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Nuria M. Pastor-Soler, University of Southern California
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04284657
- Sponsor's protocol ID
- HS-18-00170
- Acronym
- ADPKD-SAT
- Status last verified
- Aug 2025
- First submitted
- 11 Apr 2019
- Last updated
- 6 Aug 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Aug 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pravastatin and Alkali Therapy in Patients With Autosomal Dominant Polycystic Kidney Disease
- ClinicalTrials.gov ID
- NCT04284657
- Acronym / sponsor ID
- ADPKD-SAT / HS-18-00170
- Keywords
- Polycystic Kidney Disease, ADPKD, PKD
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 1 participants (actual)
- Lead sponsor
- University of Southern CaliforniaUniversity, hospital or other
- Study start
- 30 Jan 2019 (actual)
- Primary completion
- 4 Aug 2025 (actual)
- Study completion
- 4 Aug 2025 (actual)
- First posted
- 26 Feb 2020 (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 is an one-year open-label study to determine treatment efficacy and feasibility of a trial that uses open-label interventions in ADPKD patients.
Detailed description
Polycystic Kidney Disease (PKD) is the most common genetic disease leading to End Stage Kidney Disease (ESKD), affecting between 1 in 500-1000 individuals from every ethnic group. The autosomal dominant (ADPKD) form arises from a two-hit downregulation of proteins encoded by either PKD1 or PKD2. Although many potential therapies have been studied to slow progression of ADPKD, none to date have been proven to be both safe and effective in slowing disease progression. Cholesterol-lowering agents called statins have shown promise in the treatment of younger ADPKD patients, reducing inflammation and progression as assessed by kidney growth, but their utility appears to be limited in older populations and those with more advanced chronic kidney disease (CKD). Recent evidence suggests that acidosis, as often seen in patients with worsening CKD and which may enhance CKD progression, limits the effectiveness of statins and enhances their potential toxicity. The investigators thus hypothesize that correction of acidosis along with statin treatment will be a safe and effective therapeutic regimen to slow CKD progression in the adult ADPKD population and improve overall quality of life in these patients. To test this hypothesis, the investigators will conduct a pilot open-label randomized clinical trial in ADPKD patients with estimated GFR >45 min (Stage 1-3a CKD) comparing three treatment groups: control, pravastatin (40 mg po qd), and pravastatin plus sodium citrate solution (30 mL po total daily dose) over one year. During the study period, through study visits along with serial blood draws and urinary measurements, the investigators will evaluate safety and tolerability of these treatment regimens, follow renal function and investigate the role of these treatments on acidosis, inflammatory and metabolic biomarkers in patients enrolled at an outpatient facility. This study will establish the framework for larger clinical trials in ADPKD. Moreover, if the results of this study suggest safety/tolerability or potential benefits of statins and alkali therapy in this ADPKD population, the investigators will seek extramural funding for a larger clinical trial to test this therapeutic strategy in ADPKD.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| ARM I: Control group | No intervention | –Standard therapy alone |
| ARM II: PRAVASTATIN | Active comparator | Drug: PravastatinStandard therapy and PRAVASTATIN 40 mg QD |
| ARM III: PRAV + Sodium Citrate | Active comparator | Drug: Pravastatin, Drug: sodium citrateStandard therapy and PRAVASTATIN 40 mg QD and Sodium Citrate (up to 30 mL TID) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (14 listed)
- Patient voluntarily gives informed consent to participate in the study and signed study's IC and HIPAA.
- Patient is age 18 or older at the time of consent.
- If applicable, female of reproductive potential (Females who are successfully sterilized (surgical sterilization methods include hysterectomy, bilateral tubal ligation, or bilateral oophorectomy) or are postmenopausal (defined as amenorrhea for at least 12 consecutive months) are not considered to be of reproductive potential) must be non-pregnant (as confirmed by a urine pregnancy test at screening) and non-lactating, and agree:
- Patients has ADPKD diagnosed by unified criteria using a combination of ultrasound results, genotyping and MRI as needed (1, 2). Kidney ultrasound is usually used for screening because it is safe, effective, and inexpensive. Diagnostic criteria are based upon whether the genotype is known. Disease severity varies between the different genotypes. The great majority of patients at risk for ADPKD are from families with an unknown genotype. This diagnosis will take place prior to recruitment / inclusion into the study.
- The following ultrasonographic criteria for the diagnosis of ADPKD are for at-risk patients from families of where the genotype is not known:
Key exclusion criteria (24 listed)
- Patients with known allergy or sensitive to Pravastatin or NaCitrate
- Acute coronary disease, liver disease, muscle disease, or a history of pulmonary edema
- Creatine Phospho Kinase (CPK) > 2ULN (2.5 ULN in African Americans). Elevated creatine phosphokinase could be a marker of rhabdomyolysis, which is a potential side effect of pravastatin. In general, patients with African American ancestry can have higher normal level of CPK
- Patients with systemic disease that impacting kidney per Investigator's decision
- Patients with known unstable cerebral aneurysm per Investigator's decision
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 |
|---|---|---|
| PravastatinPravastatin 40 mg QD | Drug | ARM II: PRAVASTATIN, ARM III: PRAV + Sodium Citrate |
| sodium citratesodium citrate up to 30 ml TID | Drug | ARM III: PRAV + Sodium Citrate |
Primary outcome
Changes in kidney function in patients enrolled in different arms of the study
Time frame: 12 months
The investigators will estimate the effect of Pravastatin and NaCitrate on kidney function in patients with ADPKD compared to Pravastatin alone or Standard therapy: serum creatinine and serum blood urea nitrogen, serum electrolytes contribute to evaluate kidney function.
Changes in liver function in patients enrolled in different arms of the study
Time frame: 12 months
The investigators will test liver function test panels in patients with ADPKD compared to Pravastatin alone or Standard therapy. The liver function panel should be within normal limits for enrollment and continuation in the study. Liver function tests include AST > 3ULN, ALT > 3ULN, Total Bilirubin > 2 ULN, or increase in prothrombin time to abnormal level INR >1.5 repeated two weeks apart
Changes in blood pressure in patients enrolled in different arms of the study
Time frame: 12 months
The investigators will estimate the effect of Pravastatin and NaCitrate on blood pressure (systolic and diastolic) in patients with ADPKD compared to Pravastatin alone or Standard therapy
Changes in muscle injury marker function in patients enrolled in different arms of the study
Time frame: 12 months
The investigators will estimate the effect of Pravastatin and NaCitrate on creatine phospho kinase (CPK) in patients with ADPKD compared to Pravastatin alone or Standard therapy.
Changes in muscle tenderness in patients enrolled in the study
Time frame: 12 months
The investigators will estimate the effect of Pravastatin and NaCitrate on muscle tenderness in patients with ADPKD compared to Pravastatin alone or Standard therapy. The physical exam will evaluate tenderness to palpation in major muscle groups such as leg, arm and back muscles. It will be graded as presence or absence. The patients will only be enrolled if there is absence of tenderness in muscles upon palpation on physical exam. If there is tenderness on exam or the patient reports tenderness that is then confirmed by exam the patient will be removed from the study.
3 secondary outcomes
Urinary alkalinization changes
Time frame: 12 months
Inflammatory markers in blood and urine
Time frame: 12 months
AMPK pathway activation
Time frame: 12 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Aug 2025.
- Individual participant dataSharing planned: No
Related trials for Autosomal Dominant Polycystic Kidney Disease
RecruitingObservational study
Hypertension in Children and Young People at Risk of Autosomal Dominant Polycystic Kidney Disease
Autosomal Dominant Polycystic Kidney Disease (ADPKD
Active, not recruitingObservational study
Role of ROS and cAMP-PKA Biomarkers in ADPKD
Autosomal Dominant Polycystic Kidney Disease
Not yet recruitingPhase 1
A Study to Investigate the Pharmacokinetics of JMKX003142 in Healthy Participants and Participants With Hepatic Impairment
ADPKD (Autosomal Dominant Polycystic Kidney Disease), Renal Edema
RecruitingPhase 1
Absorption, Distribution, Metabolism, and Excretion of 14C-VX-407 After a Single Oral Dose
Autosomal Dominant Polycystic Kidney Disease (ADPKD)
RecruitingObservational study
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Rare Disorders, Undiagnosed Disorders
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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Pravastatin and sodium citrate in people with Autosomal Dominant Polycystic Kidney Disease, run by University of Southern California 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 14 inclusion and 24 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: finished, as of the sponsor's last update on 6 Aug 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. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Changes in kidney function in patients enrolled in different arms of the study (time frame: 12 months). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 6 Aug 2025.