Polycystic Kidney DiseasePhase 2NIAC-PKD1NCT02140814
Uncontrolled, Open Label, Pilot and Feasibility Study of Niacinamide in Polycystic Kidney Disease
A Phase 2 study testing Niacinamide in people with Polycystic Kidney Disease, run by Alan Yu, MB, BChir at 1 site in United States.
Trial summary
The goal of this pilot study is to evaluate the feasibility of administering niacinamide to patients with autosomal dominant polycystic kidney disease, to develop methods to assess the biological efficacy of niacinamide, and to perform a preliminary exploration of its clinical effect on kidney cyst growth and kidney… Read more about this study
- Who is it for?
- Ages 18 to 50 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?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteUnited States
- Study dates
- May 2014to Feb 2016
- 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 50 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Confirmed diagnosis of autosomal dominant polycystic kidney disease
- eGFR > 90ml/min/1.73m2 as determined from the serum creatinine by the CKD-EPI equation
- Ability to give informed consent in English
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 13Show 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 30 days prior to screening
- Partial or total nephrectomy or renal cyst reduction (including aspiration) done <1 year ago
- Cardiac pacemaker
- Presence of magnetic resonance-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
The goal of this pilot study is to evaluate the feasibility of administering niacinamide to patients with autosomal dominant polycystic kidney disease, to develop methods to assess the biological efficacy of niacinamide, and to perform a preliminary exploration of its clinical effect on kidney cyst growth and kidney function.
Read the detailed description
Niacinamide is a form of vitamin B3. Vitamin B3 is found in many foods including yeast, meat, fish, milk, eggs, green vegetables, beans, and cereal grains. Recent studies in mice have shown that niacinamide, at high doses, may slow kidney cyst growth from polycystic kidney disease (PKD).
By doing this study, the researchers will determine if a larger, long-term study to test whether niacinamide slows progression of PKD is justified.
- Conditions
- Polycystic Kidney Disease
- Being tested
- Niacinamide
- Keywords
- PKD, niacinamide, Vitamin B3, Vitamin B
- 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.
NiacinamideDietary supplement
Also called Vitamin B3
| Study group | Role | What they receive |
|---|---|---|
| Niacinamide | Experimental | All subjects in this study will take niacinamide at a dose of 30 mg per kilogram of body weight by mouth daily, in two divided daily doses, for 12 months. Receives: Dietary Supplement: Niacinamide |
What is being measured
The primary outcome is the main question the study is designed to answer.
Sirtuin deacetylase activity
Assessed by the level of post-translational modifications of two intracellular markers (1. Acetylated and total p53 protein concentrations, 2. Phosphorylated and total retinoblastoma protein (Rb))
Measured: Change from Baseline to 12 Months
Secondary outcomes (5)
Sirtuin deacetylase activity
Assessed by the level of post-translational modifications of two intracellular markers (1. Acetylated and total p53 protein concentrations, 2. Phosphorylated and total retinoblastoma protein (Rb))
Measured: Change from Baseline to 6 Months
Estimated Glomerular Filtration Rate (eGFR)
Measurements at each visit using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation
Measured: 12 Months
Height-adjusted total kidney volumes (htTKV)
Measurements will be taken from MRI images, and the annual percent change in htTKV will be compared to historical values reported in a separate study of a similar study population.
Measured: Change from Baseline to 12 Months
Biomarker levels
Aliquots of the urine samples from visits to be tested for a panel of biomarkers related to cyst growth compared to baseline levels.
Measured: 12 Months
Subject pain
Subject feelings collected via abbreviated pain questionnaire. Questionnaire will be used to calculate pain score.
Measured: Change from Baseline to 12 Months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 May 2014 (estimated)
- Study startMay 2014
- Primary completionFeb 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Polycystic Kidney DiseaseStudies of the same condition, open and under way.
- 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- Alan Yu, MB, BChir
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Alan Yu, MB, BChir, University of Kansas Medical Center
- Investigators
- Alan S Yu, MB, BChir, Principal investigator, University of Kansas Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02140814
- Sponsor's protocol ID
- STUDY00000874
- Acronym
- NIAC-PKD1
- Status last verified
- Nov 2016
- First submitted
- 14 May 2014
- Last updated
- 1 Dec 2016
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Dec 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Uncontrolled, Open Label, Pilot and Feasibility Study of Niacinamide in Polycystic Kidney Disease
- ClinicalTrials.gov ID
- NCT02140814
- Acronym / sponsor ID
- NIAC-PKD1 / STUDY00000874
- Conditions
- Polycystic Kidney Disease
- Keywords
- PKD, niacinamide, Vitamin B3, Vitamin B
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- Alan Yu, MB, BChirUniversity, hospital or other
- Study start
- May 2014
- Primary completion
- Feb 2016 (actual)
- First posted
- 16 May 2014 (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 goal of this pilot study is to evaluate the feasibility of administering niacinamide to patients with autosomal dominant polycystic kidney disease, to develop methods to assess the biological efficacy of niacinamide, and to perform a preliminary exploration of its clinical effect on kidney cyst growth and kidney function.
Detailed description
Niacinamide is a form of vitamin B3. Vitamin B3 is found in many foods including yeast, meat, fish, milk, eggs, green vegetables, beans, and cereal grains. Recent studies in mice have shown that niacinamide, at high doses, may slow kidney cyst growth from polycystic kidney disease (PKD).
By doing this study, the researchers will determine if a larger, long-term study to test whether niacinamide slows progression of PKD is justified.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Niacinamide | Experimental | Dietary Supplement: NiacinamideAll subjects in this study will take niacinamide at a dose of 30 mg per kilogram of body weight by mouth daily, in two divided daily doses, for 12 months. |
Who may qualify
- Ages
- 18 to 50 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (3 listed)
- Confirmed diagnosis of autosomal dominant polycystic kidney disease
- eGFR > 90ml/min/1.73m2 as determined from the serum creatinine by the CKD-EPI equation
- Ability to give informed consent in English
Key exclusion criteria (13 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: Vitamin B3 | Dietary supplement | Niacinamide |
Primary outcome
Sirtuin deacetylase activity
Time frame: Change from Baseline to 12 Months
Assessed by the level of post-translational modifications of two intracellular markers (1. Acetylated and total p53 protein concentrations, 2. Phosphorylated and total retinoblastoma protein (Rb))
5 secondary outcomes
Sirtuin deacetylase activity
Time frame: Change from Baseline to 6 Months
Estimated Glomerular Filtration Rate (eGFR)
Time frame: 12 Months
Height-adjusted total kidney volumes (htTKV)
Time frame: Change from Baseline to 12 Months
Biomarker levels
Time frame: 12 Months
Subject pain
Time frame: Change from Baseline to 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 1 Dec 2016.
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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 S Yu, MB, BChir 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 Alan Yu, MB, BChir 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 13 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 1 Dec 2016. 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?
Sirtuin deacetylase activity (time frame: Change from Baseline to 12 Months). 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 1 Dec 2016.