Clinical trial NCT02140814: Uncontrolled, Open Label, Pilot and Feasibility Study of Niacinamide in Polycystic Kidney Disease
Official title: Uncontrolled, Open Label, Pilot and Feasibility Study of Niacinamide in Polycystic Kidney Disease
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Polycystic Kidney Disease
Keywords: PKD
niacinamide
Vitamin B3
Vitamin B
Lead sponsor: University of Kansas Medical Center
Sponsor class: OTHER
First posted: 2014-05-16
Enrollment: 10
Sex: ALL
Minimum age (years): 18
Maximum age (years): 50
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

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.

Eligibility criteria: 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

Primary outcomes:
measure: Sirtuin deacetylase activity
timeFrame: Change from Baseline to 12 Months
Secondary outcomes:
measure: Sirtuin deacetylase activity
timeFrame: Change from Baseline to 6 Months
measure: Estimated Glomerular Filtration Rate (eGFR)
timeFrame: 12 Months
measure: Height-adjusted total kidney volumes (htTKV)
timeFrame: Change from Baseline to 12 Months
measure: Biomarker levels
timeFrame: 12 Months
measure: Subject pain
timeFrame: Change from Baseline to 12 Months
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 S Yu, MB, BChir
role: PRINCIPAL_INVESTIGATOR
References: