This trial is active, not recruiting.

Condition polycystic kidney disease
Treatment niacinamide
Phase phase 2
Sponsor Alan Yu, MB, BChir
Start date May 2014
End date June 2016
Trial size 15 participants
Trial identifier NCT02140814, STUDY00000874


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.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Endpoint classification pharmacodynamics study
Intervention model single group assignment
Masking open label
Primary purpose treatment
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.
niacinamide Vitamin B3

Primary Outcomes

Sirtuin deacetylase activity
time frame: Change from Baseline to 12 Months

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

Eligibility Criteria

Male or female participants from 18 years up to 50 years old.

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

Additional Information

Official title Uncontrolled, Open Label, Pilot and Feasibility Study of Niacinamide in Polycystic Kidney Disease
Principal investigator Alan S Yu, MB, BChir
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.
Trial information was received from ClinicalTrials.gov and was last updated in December 2015.
Information provided to ClinicalTrials.gov by University of Kansas Medical Center.