NCT01729598

Valproic Acid in Subjects With Intact Cognition - Proof of Concept Study

Safety And Target Engagement Of Clu1 By Valproic Acid In Subjects With Intact Cognition: Proof Of Concept For The Development Of A Prevention Trial For Alzheimer's Disease

• Status: Completed
• Phase: EARLY_Phase 1
• Study type: Interventional
• Enrollment: 14 (actual)
• Sponsor: Gregory Jicha, 323-5550 · Academic / Other
• Sex: All
• Age: 65 Years – 90 Years
• Healthy volunteers: Accepted

Summary

The purpose of this study is to evaluate the safety of administration and effects of valproic acid on clusterin expression in cognitively-intact, healthy, elderly subjects. Clusterin mutations have recently been identified as a risk factor for the development of Alzheimer's Disease and changes in clusterin expression are seen in the elderly who develop Alzheimer's disease irrespective of whether they carry these genetic mutations or not. Valproic acid may prevent or reverse these changes. Fourteen subjects with normal memory and thinking will participate in this study. Ten of these subjects will receive valproic acid and 4 will receive a "placebo" capsule with no active medicine. Participants will take study medication or placebo for 28 days and be followed for a total 35 days in this trial.

Detailed description

This is a placebo-controlled, single-center, multiple ascending dose study. Seven healthy volunteers will be enrolled into 2 sequential cohorts, for a total of 14 enrolled subjects. The study will be conducted using two doses of valproate (250 mg PO bid, followed by 500 mg PO bid). At each dose level, 7 cognitively intact normal elderly subjects will be entered into the study with two subjects randomly selected to receive placebo while the other five subjects receive the designated dose of valproate. Study procedures will include routine assessment of adverse events, safety labs, baseline MRI, physical and neurological exams, and cerebrospinal fluid collection. Other investigational medication or devices are prohibited during this study.

Conditions

• Alzheimer's Disease

Interventions

Type	Name	Description
DRUG	Valproic Acid	generic valproic acid tablets packaged in placebo-matched capsules.
DRUG	Placebo	Placebo capsule without active study medication in identical capsules as experimental medicine.

Timeline

• Start date: 2012-04-01
• Primary completion: 2014-10-01
• Completion: 2014-10-01
• First posted: 2012-11-20
• Last updated: 2019-10-09
• Results posted: 2019-10-09

Locations

1 site across 1 country: United States