Polycystic Kidney DiseasePhase 2PIOPKDNCT02697617
Use of Low Dose Pioglitazone to Treat Autosomal Dominant Polycystic Kidney Disease
A Phase 2 study testing Pioglitazone in people with Polycystic Kidney Disease, run by Indiana University at 1 site in United States.
Trial summary
Funding Source - FDA OOPD Pioglitazone is currently used in clinical practice to treat diabetes and this study will examine the potential use of a low dose of the same drug for the treatment of polycystic kidney disease. Read more about this study
- Who is it for?
- Ages 18 to 55 years
- Conditions the study is for
- Polycystic Kidney Disease
- What is being tested?
- Pioglitazone
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 18 participants
- Where
- 1 siteUnited States
- Study dates
- 26 Jan 2016to Jan 2020
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 to 55 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Male or female patients with autosomal dominant polycystic kidney disease (ADPKD) aged 18-55
- estimate glomerular filtration rate (GFR) at or above ≥ 50 ml/min/1.73 m2 by any GFR formula
- Normal liver enzymes (ALT/AST)
- fasting blood glucose between 70 and120
- for female patients, a willingness to use double contraception to avoid pregnancy while in study
- able to give informed consent
- In the opinion of the investigator, high likelihood of progressive kidney disease
Who cannot join
- diabetes, defined as any of the following: fasting blood sugar > 130 times two, HgbA1C > 7, on any blood sugar lowering medication, or past diagnosis of diabetes not occurring during pregnancy
- uncontrolled hypertension as determined by the examining physician
- history of impaired systolic function (ejection fraction < 50%) by previous echocardiogram or known ischemic cardiovascular disease
- findings suggestive of a kidney disease other than ADPKD
- systemic illness requiring immunosuppressive or anti-inflammatory agents
- congenital absence of a kidney or history of a total nephrectomy
Show all 14Show fewer
- history of cyst reduction or partial nephrectomy
- history of renal cyst aspiration within the previous year
- History of bladder cancer, or gross hematuria
- inability to undergo MRI due to implantable devices or foreign objects that preclude MRI
- active renal transplant
- allergy or sensitivity to any of the components of the test materials
- institutionalized
- currently pregnant or plans to become pregnant during the study
Trial locations and contacts
1 site in 1 country.
United States1 site
Indiana University Health
Indianapolis, Indiana, 46202
About this study
Official title: Use of Low Dose Pioglitazone to Treat Autosomal Dominant Polycystic Kidney
Funding Source - FDA OOPD
Pioglitazone is currently used in clinical practice to treat diabetes and this study will examine the potential use of a low dose of the same drug for the treatment of polycystic kidney disease. The purpose of this study is to determine whether the diabetes drug pioglitazone (Actos) is a safe and effective treatment of autosomal dominant polycystic kidney disease when treated in its early stages. Pioglitazone is approved by the FDA for the treatment of diabetes. Pre-clinical models of polycystic kidney disease have shown that low dose treatment with pioglitazone decreases the growth of the cysts. The studies also suggest that effective pioglitazone dosing for polycystic kidney disease may be lower than that used to treat diabetes. The purpose of this study is to see if pioglitazone might slow cyst disease in humans.
Read the detailed description
Patients will be randomize to placebo or 15 mg pioglitazone for 12 months, and then be crossed over to the other arm. Patients will undergo MRI of the liver and kidney and MRspectroscopy of the lumbar spine (if they choose as this is ancillary study) three times during the study. Assessments will be every 3 months and include blood work, blood pressure, and body water assessments.
- Conditions
- Polycystic Kidney Disease
- Being tested
- Pioglitazone, Placebo
- Medical subject headings
- Polycystic Kidney Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PioglitazoneDrug
Also called Actos
Pioglitazone
PlaceboDrug
Placebo
| Study group | Role | What they receive |
|---|---|---|
| Placebo Arm | Placebo comparator | Subject will be on placebo Receives: Drug: Placebo |
| Pioglitazone Arm | Active comparator | Subject will be on pioglitazone Receives: Drug: Pioglitazone |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety: Total Body Water
Bioimpedance analysis (BIA)(Ohms); Increase in BIA in Ohms indicates a decrease in total body water
Measured: average of 4 measures in each 12 month arm
Efficacy: Percent Change in Total Kidney Volume
Change in total kidney volume by Magnetic Resonance Imaging (MRI) from beginning to end of the 12 months
Measured: Baseline, end of year 1, and end of year 2
Secondary outcomes (5)
Safety: Hypoglycemia
number of patients with blood sugar < 70 mg/dl
Measured: measured quarterly for 12 months in pioglitazone and same in placebo
Safety: Elevated Liver Function Tests
Number of patients with elevated liver test (ALT or AST) > 2 times upper limit of normal
Measured: measured quarterly over 12 months for each arm
Efficacy: Glomerular Filtration Rate
average estimated glomerular filtration rate by chronic kidney disease (CKD) epidemiologic (epi) formula measured quarterly
Measured: average of 4 values over 12 months
Efficacy Blood Pressure
mean systolic and diastolic blood pressure
Measured: average of 4 measures over 12 months
Bone Marrow Fat
We will assess change in bone marrow fat by MR spectroscopy as an ancillary study to be done at the same time as MRI; will not be done due to person leaving institution.
Measured: Baseline, end of year 1, and end of year 2
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Mar 2016 (estimated)
- Study start26 Jan 2016
- Primary completionOct 2019
- Study completionJan 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Polycystic Kidney DiseaseStudies of the same condition, open and under way.
- More trials run by Indiana UniversityThe 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
- Crossover
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 18 participants (actual)
Sponsor and investigators
- Lead sponsor
- Indiana University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Sharon Moe, Indiana University
- Investigators
- Sharon Moe, 317-944-7580, Principal investigator, Indiana University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02697617
- Sponsor's protocol ID
- IndianaU 1308084213
- Other IDs
- FD-R-004826-01-A2
- Acronym
- PIOPKD
- Status last verified
- Dec 2020
- First submitted
- 30 Oct 2015
- Last updated
- 15 Jan 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Individual data sharing
- No
Publications
St 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 39356039
Study at a glance
From the registry record, last updated by the sponsor on 15 Jan 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Use of Low Dose Pioglitazone to Treat Autosomal Dominant Polycystic Kidney
- ClinicalTrials.gov ID
- NCT02697617
- Acronym / sponsor ID
- PIOPKD / IndianaU 1308084213
- Conditions
- Polycystic Kidney Disease
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 18 participants (actual)
- Lead sponsor
- Indiana UniversityUniversity, hospital or other
- Study start
- 26 Jan 2016 (actual)
- Primary completion
- Oct 2019 (actual)
- Study completion
- Jan 2020 (actual)
- First posted
- 3 Mar 2016 (estimated)
- Results
- Posted 15 Jan 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Funding Source - FDA OOPD
Pioglitazone is currently used in clinical practice to treat diabetes and this study will examine the potential use of a low dose of the same drug for the treatment of polycystic kidney disease. The purpose of this study is to determine whether the diabetes drug pioglitazone (Actos) is a safe and effective treatment of autosomal dominant polycystic kidney disease when treated in its early stages. Pioglitazone is approved by the FDA for the treatment of diabetes. Pre-clinical models of polycystic kidney disease have shown that low dose treatment with pioglitazone decreases the growth of the cysts. The studies also suggest that effective pioglitazone dosing for polycystic kidney disease may be lower than that used to treat diabetes. The purpose of this study is to see if pioglitazone might slow cyst disease in humans.
Detailed description
Patients will be randomize to placebo or 15 mg pioglitazone for 12 months, and then be crossed over to the other arm. Patients will undergo MRI of the liver and kidney and MRspectroscopy of the lumbar spine (if they choose as this is ancillary study) three times during the study. Assessments will be every 3 months and include blood work, blood pressure, and body water assessments.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Placebo Arm | Placebo comparator | Drug: PlaceboSubject will be on placebo |
| Pioglitazone Arm | Active comparator | Drug: PioglitazoneSubject will be on pioglitazone |
Who may qualify
- Ages
- 18 to 55 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (7 listed)
- Male or female patients with autosomal dominant polycystic kidney disease (ADPKD) aged 18-55
- estimate glomerular filtration rate (GFR) at or above ≥ 50 ml/min/1.73 m2 by any GFR formula
- Normal liver enzymes (ALT/AST)
- fasting blood glucose between 70 and120
- for female patients, a willingness to use double contraception to avoid pregnancy while in study
Key exclusion criteria (14 listed)
- diabetes, defined as any of the following: fasting blood sugar > 130 times two, HgbA1C > 7, on any blood sugar lowering medication, or past diagnosis of diabetes not occurring during pregnancy
- uncontrolled hypertension as determined by the examining physician
- history of impaired systolic function (ejection fraction < 50%) by previous echocardiogram or known ischemic cardiovascular disease
- findings suggestive of a kidney disease other than ADPKD
- systemic illness requiring immunosuppressive or anti-inflammatory agents
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 |
|---|---|---|
| PioglitazoneAlso: ActosPioglitazone | Drug | Pioglitazone Arm |
| PlaceboPlacebo | Drug | Placebo Arm |
Primary outcome
Safety: Total Body Water
Time frame: average of 4 measures in each 12 month arm
Bioimpedance analysis (BIA)(Ohms); Increase in BIA in Ohms indicates a decrease in total body water
Efficacy: Percent Change in Total Kidney Volume
Time frame: Baseline, end of year 1, and end of year 2
Change in total kidney volume by Magnetic Resonance Imaging (MRI) from beginning to end of the 12 months
5 secondary outcomes
Safety: Hypoglycemia
Time frame: measured quarterly for 12 months in pioglitazone and same in placebo
Safety: Elevated Liver Function Tests
Time frame: measured quarterly over 12 months for each arm
Efficacy: Glomerular Filtration Rate
Time frame: average of 4 values over 12 months
Efficacy Blood Pressure
Time frame: average of 4 measures over 12 months
Bone Marrow Fat
Time frame: Baseline, end of year 1, and end of year 2
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Jan 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- 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.
- Individual participant dataSharing planned: No
Related trials for 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
Recruiting
Well-Formulated Ketogenic Diet Polycystic Kidney Disease
Polycystic Kidney Disease
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)
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.
Sharon Moe, 317-944-7580 principal investigator
Indiana University
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 Pioglitazone in people with Polycystic Kidney Disease, run by Indiana University 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 7 inclusion and 14 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 15 Jan 2021. 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 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?
Yes. Results are posted in the registry (first posted 15 Jan 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Safety: Total Body Water (time frame: average of 4 measures in each 12 month arm). The study lists 2 primary outcomes in all. 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 NCT02697617, which also lists 1 linked publication.
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 15 Jan 2021.