Pravastatin and Alkali Therapy in Patients With Autosomal Dominant Polycystic Kidney Disease (ADPKD-SAT)
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ClinicalTrials.gov Identifier: NCT04284657 |
Recruitment Status :
Enrolling by invitation
First Posted : February 26, 2020
Last Update Posted : January 25, 2022
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Condition or disease | Intervention/treatment | Phase |
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Autosomal Dominant Polycystic Kidney Disease | Drug: Pravastatin Drug: sodium citrate | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 30 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | Pravastatin and Alkali Therapy in Patients With Autosomal Dominant Polycystic Kidney Disease |
Actual Study Start Date : | January 30, 2019 |
Estimated Primary Completion Date : | December 31, 2022 |
Estimated Study Completion Date : | December 31, 2022 |

Arm | Intervention/treatment |
---|---|
No Intervention: ARM I: Control group
Standard therapy alone
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Active Comparator: ARM II: PRAVASTATIN
Standard therapy and PRAVASTATIN 40 mg QD
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Drug: Pravastatin
Pravastatin 40 mg QD |
Active Comparator: ARM III: PRAV + Sodium Citrate
Standard therapy and PRAVASTATIN 40 mg QD and Sodium Citrate (up to 30 mL TID)
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Drug: Pravastatin
Pravastatin 40 mg QD Drug: sodium citrate sodium citrate up to 30 ml TID |
- 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.
- Urinary alkalinization changes [ Time Frame: 12 months ]These parameters will be ascertained by measurements of urinary pH and serum electrolytes.
- Inflammatory markers in blood and urine [ Time Frame: 12 months ]
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
- AMPK pathway activation [ Time Frame: 12 months ]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.

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- 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.
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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.))
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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:
- 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
Exclusion Criteria
- 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
- 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

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04284657
United States, California | |
Keck School of Medicine of University of Southern California | |
Los Angeles, California, United States, 90033 |
Responsible Party: | Nuria M. Pastor-Soler, Associate Professor, University of Southern California |
ClinicalTrials.gov Identifier: | NCT04284657 |
Other Study ID Numbers: |
HS-18-00170 |
First Posted: | February 26, 2020 Key Record Dates |
Last Update Posted: | January 25, 2022 |
Last Verified: | January 2022 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Product Manufactured in and Exported from the U.S.: | No |
Polycystic Kidney Disease ADPKD PKD |
Arthrogryposis Kidney Diseases Polycystic Kidney Diseases Polycystic Kidney, Autosomal Dominant Urologic Diseases Joint Diseases Musculoskeletal Diseases Muscular Diseases Musculoskeletal Abnormalities Congenital Abnormalities Kidney Diseases, Cystic Abnormalities, Multiple Ciliopathies Genetic Diseases, Inborn |
Citric Acid Sodium Citrate Pravastatin Anticoagulants Calcium Chelating Agents Chelating Agents Sequestering Agents Molecular Mechanisms of Pharmacological Action Anticholesteremic Agents Hypolipidemic Agents Antimetabolites Lipid Regulating Agents Hydroxymethylglutaryl-CoA Reductase Inhibitors Enzyme Inhibitors |