Back to resultsUncontrolled, Open Label, Pilot and Feasibility Study of Niacinamide in Polycystic Kidney Disease (NIAC-PKD1)
Primary Purpose
Polycystic Kidney Disease
Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Niacinamide
Sponsored by
About this trial
This is an interventional treatment trial for Polycystic Kidney Disease focused on measuring PKD, niacinamide, Vitamin B3, Vitamin B
Eligibility Criteria
Inclusion Criteria:
- 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
Exclusion Criteria:
- 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
- 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
Sites / Locations
- University of Kansas Medical Center
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Niacinamide
Arm Description
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.
Outcomes
Primary Outcome Measures
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))
Secondary Outcome Measures
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))
Estimated Glomerular Filtration Rate (eGFR)
Measurements at each visit using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation
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.
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.
Subject pain
Subject feelings collected via abbreviated pain questionnaire. Questionnaire will be used to calculate pain score.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT02140814
Brief Title
Uncontrolled, Open Label, Pilot and Feasibility Study of Niacinamide in Polycystic Kidney Disease
Acronym
NIAC-PKD1
Official Title
Uncontrolled, Open Label, Pilot and Feasibility Study of Niacinamide in Polycystic Kidney Disease
Study Type
Interventional
2. Study Status
Record Verification Date
November 2016
Overall Recruitment Status
Completed
Study Start Date
May 2014 (undefined)
Primary Completion Date
February 2016 (Actual)
Study Completion Date
undefined (undefined)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor-Investigator
Name of the Sponsor
Alan Yu, MB, BChir
4. Oversight
Data Monitoring Committee
No
5. Study Description
Brief 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 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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Polycystic Kidney Disease
Keywords
PKD, niacinamide, Vitamin B3, Vitamin B
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
10 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Niacinamide
Arm Type
Experimental
Arm Description
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.
Intervention Type
Dietary Supplement
Intervention Name(s)
Niacinamide
Other Intervention Name(s)
Vitamin B3
Primary Outcome Measure Information:
Title
Sirtuin deacetylase activity
Description
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))
Time Frame
Change from Baseline to 12 Months
Secondary Outcome Measure Information:
Title
Sirtuin deacetylase activity
Description
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))
Time Frame
Change from Baseline to 6 Months
Title
Estimated Glomerular Filtration Rate (eGFR)
Description
Measurements at each visit using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation
Time Frame
12 Months
Title
Height-adjusted total kidney volumes (htTKV)
Description
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.
Time Frame
Change from Baseline to 12 Months
Title
Biomarker levels
Description
Aliquots of the urine samples from visits to be tested for a panel of biomarkers related to cyst growth compared to baseline levels.
Time Frame
12 Months
Title
Subject pain
Description
Subject feelings collected via abbreviated pain questionnaire. Questionnaire will be used to calculate pain score.
Time Frame
Change from Baseline to 12 Months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
50 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
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
Exclusion Criteria:
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
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Alan S Yu, MB, BChir
Organizational Affiliation
University of Kansas Medical Center
Official's Role
Principal Investigator
Facility Information:
Facility Name
University of Kansas Medical Center
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
66160
Country
United States
12. IPD Sharing Statement
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Uncontrolled, Open Label, Pilot and Feasibility Study of Niacinamide in Polycystic Kidney Disease
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