Uncontrolled, Open Label, Pilot and Feasibility Study of Niacinamide in Polycystic Kidney Disease (NIAC-PKD1)

November 30, 2016 updated by: Alan Yu, MB, BChir
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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Actual)

10

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Kansas
      • Kansas City, Kansas, United States, 66160
        • University of Kansas Medical Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

14 years to 46 years (Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Niacinamide
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.
Dietary supplement: Niacinamide
Other Names:
  • Vitamin B3

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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))
Change from Baseline to 12 Months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Sirtuin deacetylase activity
Time Frame: Change from Baseline to 6 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))
Change from Baseline to 6 Months
Estimated Glomerular Filtration Rate (eGFR)
Time Frame: 12 Months
Measurements at each visit using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation
12 Months
Height-adjusted total kidney volumes (htTKV)
Time Frame: Change from Baseline to 12 Months
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.
Change from Baseline to 12 Months
Biomarker levels
Time Frame: 12 Months
Aliquots of the urine samples from visits to be tested for a panel of biomarkers related to cyst growth compared to baseline levels.
12 Months
Subject pain
Time Frame: Change from Baseline to 12 Months
Subject feelings collected via abbreviated pain questionnaire. Questionnaire will be used to calculate pain score.
Change from Baseline to 12 Months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Alan Yu, MB, BChir

Investigators

  • Principal Investigator: Alan S Yu, MB, BChir, University of Kansas Medical Center

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

May 1, 2014

Primary Completion (Actual)

February 1, 2016

Study Registration Dates

First Submitted

May 14, 2014

First Submitted That Met QC Criteria

May 14, 2014

First Posted (Estimate)

May 16, 2014

Study Record Updates

Last Update Posted (Estimate)

December 1, 2016

Last Update Submitted That Met QC Criteria

November 30, 2016

Last Verified

November 1, 2016

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • STUDY00000874

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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