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

NCT02140814 | Completed Phase 2

• 1 other identifier: STUDY00000874
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

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.

Conditions

Polycystic Kidney Disease

Keywords

PKD; niacinamide; Vitamin B3; Vitamin B

Outcome Measures

Primary Outcomes (1)

• 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

Secondary Outcomes (5)

• Sirtuin deacetylase activity

  Change from Baseline to 6 Months

• Estimated Glomerular Filtration Rate (eGFR)

  12 Months

• Height-adjusted total kidney volumes (htTKV)

  Change from Baseline to 12 Months

• Biomarker levels

  12 Months

• Subject pain

  Change from Baseline to 12 Months

Study Arms (1)

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.

Dietary Supplement: Niacinamide

Interventions

Niacinamide DIETARY_SUPPLEMENT

Also known as: Vitamin B3

Niacinamide

Eligibility Criteria

• Age: 18 Years - 50 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Alan Yu, MB, BChir: lead

Study Sites (1)

University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

Location

MeSH Terms

Conditions

Polycystic Kidney Diseases

Interventions

Niacinamide

Condition Hierarchy (Ancestors)

Kidney Diseases, Cystic; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Ciliopathies; Genetic Diseases, Inborn

Intervention Hierarchy (Ancestors)

Nicotinic Acids; Acids, Heterocyclic; Heterocyclic Compounds; Pyridines; Heterocyclic Compounds, 1-Ring

Study Officials

• Alan S Yu, MB, BChir

  University of Kansas Medical Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Professor and Director, Kidney Institute

Study Record Dates

• First Submitted: May 14, 2014
• First Posted: May 16, 2014
• Study Start: May 1, 2014
• Primary Completion: February 1, 2016
• Last Updated: December 1, 2016

Record last verified: 2016-11

Locations

US (1)