Uncontrolled, Open Label, Pilot and Feasibility Study of Niacinamide in Polycystic Kidney Disease

Polycystic Kidney Disease Clinical Trial

— NIAC-PKD1  

Official title:

Uncontrolled, Open Label, Pilot and Feasibility Study of Niacinamide in Polycystic Kidney Disease

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02140814
• Other study ID #: STUDY00000874
• Status: Completed
• Phase: Phase 2
• First received: May 14, 2014
• Last updated: November 30, 2016
• Start date: May 2014

Study information

• Verified date: November 2016
• Source: University of Kansas Medical Center
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Institutional Review Board
• Study type: Interventional

Clinical 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 function.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 10
• Est. primary completion date: February 2016
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years to 50 Years

Eligibility

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 Design

Endpoint Classification: Pharmacodynamics Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Kidney Diseases
• Polycystic Kidney Disease
• Polycystic Kidney Diseases

Intervention

Dietary Supplement:
• Niacinamide

Locations

Country	Name	City	State
United States	University of Kansas Medical Center	Kansas City	Kansas

Sponsors (1)

Lead Sponsor	Collaborator
Alan Yu, MB, BChir

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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))	Change from Baseline to 12 Months	No
Secondary	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))	Change from Baseline to 6 Months	No
Secondary	Estimated Glomerular Filtration Rate (eGFR)	Measurements at each visit using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation	12 Months	No
Secondary	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.	Change from Baseline to 12 Months	No
Secondary	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.	12 Months	No
Secondary	Subject pain	Subject feelings collected via abbreviated pain questionnaire. Questionnaire will be used to calculate pain score.	Change from Baseline to 12 Months	No