Clinical trial NCT02558595: Pilot Study of Niacinamide in Polycystic Kidney Disease (NIAC-PKD2)
Official title: Randomized, Controlled Pilot Study of Niacinamide in Polycystic Kidney Disease
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Polycystic Kidney Disease
Keywords: PKD
Kidney disease
Vitamin B3
Lead sponsor: University of Kansas Medical Center
Sponsor class: OTHER
Collaborators: name: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
First posted: 2015-09-24
Enrollment: 36
Sex: ALL
Minimum age (years): 18
Maximum age (years): 60
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: BASIC_SCIENCE

Brief summary: The purpose of this study is to observe the effects of niacinamide on markers of kidney injury, inflammation, kidney cyst growth and kidney function.

Detailed description: Polycystic Kidney Disease (PKD) is a genetic disease that affects about 1 in 500 people worldwide. It can lead to kidney failure which can lead to death. PKD causes the development of kidney cysts (fluid-filled balloons), which cause worsening kidney function. It is common for people with PKD to also have blood in the urine, kidney pain, high blood pressure, kidney stones, kidney infections, and cysts in the brain or other parts of the body.

There is currently no treatment known to stop cyst growth or a cure for the disease.

Participants in this study will be randomly assigned to one of two groups: niacinamide or placebo. Participants have an equal chance of being assigned to either of the groups. For people that qualify and decide to participate in this study, they will be asked to make five visits to the study clinic and complete two study related phone calls over the course of 12 months.

Eligibility criteria: Inclusion Criteria:

* Confirmed diagnosis of autosomal dominant polycystic kidney disease (ADPKD).
* Estimated glomerular filtration rate (eGFR) \> 50 ml/min/1.73m2 as determined from the serum creatinine by the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation
* Provide Informed consent

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 2 months prior to screening.

Exclusions specific to magnetic resonance (MR) imaging acquisition and measurement:

* Partial or total nephrectomy or renal cyst reduction (including aspiration) done \<1 year ago
* Cardiac pacemaker.
* Presence of MR incompatible metallic clips (e.g. clipped cerebral aneurysm)
* Body weight \>159 kg (350 lbs) or untreatable claustrophobia.

Primary outcomes:
measure: Change in acetylated/total p53 ratio measured by ELISA in peripheral blood mononuclear cells (PBMC)
timeFrame: Change from Baseline to Month 12
Secondary outcomes:
measure: Change in height-adjusted total kidney volume
timeFrame: Change from Baseline to Month 12
measure: Change in score on pain questionnaire
timeFrame: Change from Baseline to Month 12
measure: Change in urinary concentration of MCP-1
timeFrame: Change from Baseline to Month 12
measure: Change in estimated GFR
timeFrame: Change from Baseline to Month 12
Other outcomes:
Locations:
city: Kansas City
country: United States
facility: University of Kansas Medical Center
state: Kansas
Study officials:
affiliation: University of Kansas Medical Center
name: Alan Yu, M.B., B.Chir
role: PRINCIPAL_INVESTIGATOR
References: