Polycystic Kidney DiseasePhase 2NCT02558595
Pilot Study of Niacinamide in Polycystic Kidney Disease (NIAC-PKD2)
A Phase 2 study testing Niacinamide in people with Polycystic Kidney Disease, run by University of Kansas Medical Center at 1 site in United States.
Trial summary
The purpose of this study is to observe the effects of niacinamide on markers of kidney injury, inflammation, kidney cyst growth and kidney function. Read more about this study
- Who is it for?
- Ages 18 to 60 years
- Conditions the study is for
- Polycystic Kidney Disease
- What is being tested?
- Niacinamide
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 36 participants
- Where
- 1 siteUnited States
- Study dates
- 22 Sep 2015to 18 Dec 2017
- 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 to 60 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Confirmed diagnosis of autosomal dominant polycystic kidney disease (ADPKD).
- Estimated glomerular filtration rate (eGFR) > 50 ml/min/1.73m2 as determined from the serum creatinine by the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation
- Provide Informed consent
Who cannot join
- History of liver disease or abnormal liver function test
- Heavy alcohol intake
- Chronic diarrhea or malabsorption syndrome
- Thrombocytopenia
- Hypophosphatemia
- Pregnancy or lactation or plan to become pregnant during the study
Show all 14Show fewer
- Treatment with anti-epileptic drugs
- Treatment with tolvaptan, current or within 2 months prior to screening
- Participation in another interventional trial currently or within 2 months prior to screening.
- Exclusions specific to magnetic resonance (MR) imaging acquisition and measurement:
- Partial or total nephrectomy or renal cyst reduction (including aspiration) done <1 year ago
- Cardiac pacemaker.
- Presence of MR incompatible metallic clips (e.g. clipped cerebral aneurysm)
- Body weight >159 kg (350 lbs) or untreatable claustrophobia.
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Kansas Medical Center
Kansas City, Kansas, 66160
About this study
Official title: Randomized, Controlled Pilot Study of Niacinamide in Polycystic Kidney Disease
The purpose of this study is to observe the effects of niacinamide on markers of kidney injury, inflammation, kidney cyst growth and kidney function.
Read the detailed description
Polycystic Kidney Disease (PKD) is a genetic disease that affects about 1 in 500 people worldwide. It can lead to kidney failure which can lead to death. PKD causes the development of kidney cysts (fluid-filled balloons), which cause worsening kidney function. It is common for people with PKD to also have blood in the urine, kidney pain, high blood pressure, kidney stones, kidney infections, and cysts in the brain or other parts of the body.
There is currently no treatment known to stop cyst growth or a cure for the disease.
Participants in this study will be randomly assigned to one of two groups: niacinamide or placebo. Participants have an equal chance of being assigned to either of the groups. For people that qualify and decide to participate in this study, they will be asked to make five visits to the study clinic and complete two study related phone calls over the course of 12 months.
- Conditions
- Polycystic Kidney Disease
- Being tested
- Niacinamide, Placebo
- Keywords
- PKD, Kidney disease, Vitamin B3
- Medical subject headings
- Polycystic Kidney Diseases, Kidney Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NiacinamideDietary supplement
Also called Nicotinamide, Vitamin B3
PlaceboOther
Placebo pill that matches niacinamide pill is size, shape and color
| Study group | Role | What they receive |
|---|---|---|
| Niacinamide | Experimental | Participants will be asked to take niacinamide at a dose of 30 mg/kg/d orally. Receives: Dietary Supplement: Niacinamide |
| Placebo | Placebo comparator | Participants will be asked to take a placebo pill at a dose of 30 mg/kg/d orally. Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in acetylated/total p53 ratio measured by ELISA in peripheral blood mononuclear cells (PBMC)
Sirtuin 1 deacetylates p53 protein, and niacinamide inhibits sirtuin 1. So an increase in acetylated/total p53 ratio in PBMC will be used as a marker of biological efficacy.
Measured: Change from Baseline to Month 12
Secondary outcomes (4)
Change in height-adjusted total kidney volume
Measured by MRI
Measured: Change from Baseline to Month 12
Change in score on pain questionnaire
PKD-9 Pain Questionnaire: Range of scores from 13 (no pain or symptoms) to 70 (very severe symptoms almost every day)
Measured: Change from Baseline to Month 12
Change in urinary concentration of MCP-1
Measured: Change from Baseline to Month 12
Change in estimated GFR
Determined from serum creatinine concentrations using CKD-Epi equation
Measured: Change from Baseline to Month 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Sep 2015 (estimated)
- Study start22 Sep 2015
- Primary completion18 Dec 2017
- Study completion18 Dec 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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 University of Kansas Medical CenterThe 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
- Basic science
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 36 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Kansas Medical Center
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Alan Yu, MB, BChir, University of Kansas Medical Center
- Investigators
- Alan Yu, M.B., B.Chir, Principal investigator, University of Kansas Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02558595
- Sponsor's protocol ID
- STUDY00002827
- Other IDs
- R21DK104086
- Status last verified
- Feb 2019
- First submitted
- 21 Sep 2015
- Last updated
- 11 Feb 2019
- Results posted
- No
- Data monitoring committee
- Yes
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 11 Feb 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Randomized, Controlled Pilot Study of Niacinamide in Polycystic Kidney Disease
- ClinicalTrials.gov ID
- NCT02558595
- Acronym / sponsor ID
- STUDY00002827
- Conditions
- Polycystic Kidney Disease
- Keywords
- PKD, Kidney disease, Vitamin B3
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 36 participants (actual)
- Lead sponsor
- University of Kansas Medical CenterUniversity, hospital or other
- Collaborators
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
- Study start
- 22 Sep 2015 (actual)
- Primary completion
- 18 Dec 2017 (actual)
- Study completion
- 18 Dec 2017 (actual)
- First posted
- 24 Sep 2015 (estimated)
- 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.
The purpose of this study is to observe the effects of niacinamide on markers of kidney injury, inflammation, kidney cyst growth and kidney function.
Detailed description
Polycystic Kidney Disease (PKD) is a genetic disease that affects about 1 in 500 people worldwide. It can lead to kidney failure which can lead to death. PKD causes the development of kidney cysts (fluid-filled balloons), which cause worsening kidney function. It is common for people with PKD to also have blood in the urine, kidney pain, high blood pressure, kidney stones, kidney infections, and cysts in the brain or other parts of the body.
There is currently no treatment known to stop cyst growth or a cure for the disease.
Participants in this study will be randomly assigned to one of two groups: niacinamide or placebo. Participants have an equal chance of being assigned to either of the groups. For people that qualify and decide to participate in this study, they will be asked to make five visits to the study clinic and complete two study related phone calls over the course of 12 months.
Study design
- Primary purpose
- Basic science
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Niacinamide | Experimental | Dietary Supplement: NiacinamideParticipants will be asked to take niacinamide at a dose of 30 mg/kg/d orally. |
| Placebo | Placebo comparator | Other: PlaceboParticipants will be asked to take a placebo pill at a dose of 30 mg/kg/d orally. |
Who may qualify
- Ages
- 18 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (3 listed)
- Confirmed diagnosis of autosomal dominant polycystic kidney disease (ADPKD).
- Estimated glomerular filtration rate (eGFR) > 50 ml/min/1.73m2 as determined from the serum creatinine by the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation
- Provide Informed consent
Key exclusion criteria (14 listed)
- History of liver disease or abnormal liver function test
- Heavy alcohol intake
- Chronic diarrhea or malabsorption syndrome
- Thrombocytopenia
- Hypophosphatemia
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 |
|---|---|---|
| NiacinamideAlso: Nicotinamide, Vitamin B3 | Dietary supplement | Niacinamide |
| PlaceboPlacebo pill that matches niacinamide pill is size, shape and color | Other | Placebo |
Primary outcome
Change in acetylated/total p53 ratio measured by ELISA in peripheral blood mononuclear cells (PBMC)
Time frame: Change from Baseline to Month 12
Sirtuin 1 deacetylates p53 protein, and niacinamide inhibits sirtuin 1. So an increase in acetylated/total p53 ratio in PBMC will be used as a marker of biological efficacy.
4 secondary outcomes
Change in height-adjusted total kidney volume
Time frame: Change from Baseline to Month 12
Change in score on pain questionnaire
Time frame: Change from Baseline to Month 12
Change in urinary concentration of MCP-1
Time frame: Change from Baseline to Month 12
Change in estimated GFR
Time frame: Change from Baseline to Month 12
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 11 Feb 2019.
- 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.
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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.
Alan Yu, M.B., B.Chir principal investigator
University of Kansas Medical Center
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 Niacinamide in people with Polycystic Kidney Disease, run by University of Kansas Medical Center 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 3 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 11 Feb 2019. 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?
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?
Change in acetylated/total p53 ratio measured by ELISA in peripheral blood mononuclear cells (PBMC) (time frame: Change from Baseline to Month 12). 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 NCT02558595, 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 11 Feb 2019; AzTrials last refreshed its copy on 8 Nov 2026.