Randomized Trial of ENT-01 for Constipation in Parkinson Disease

A benefit was seen, but adverse effects may limit treatment and the mechanism remains in question.

The disease process in Parkinson disease (PD) has been associated with deposition of α-synuclein (αS) in enteric neurons. ENT-01 (squalamine phosphate) is an investigational (not FDA-approved) oral drug that inhibits the formation of αS aggregates and may improve constipation. In this phase 2b, randomized, placebo-controlled trial of safety and efficacy of ENT-01 for constipation and neurologic symptoms, 150 patients with PD received ENT-01 or placebo once daily for up to 25 days. Dosing was escalated to 250 mg or to tolerability. The primary outcome was the number of complete spontaneous bowel movements (CSBMs) per week during the fixed treatment-dose period (9.1 days).

From a baseline rate of 0.7 per week, CSBM rate improved significantly …

Reviewing Author

Michael S. Okun, MD

Disclosures

Consultant/Advisory BoardParkinson’s Foundation; Tourette Association of America

Speaker’s BureauMedscape/WebMD CME, Movement Disorders Society CME, American Academy of Neurology CME

RoyaltiesBooks4Patients; Demos; Cambridge; Taylor and Francis; Robert Rose; Public Affairs

Grant/Research SupportNational Institutes of Health; Parkinson’s Foundation; Tourette Association of America; University of Florida Foundation

Editorial BoardsTremor and Other Hyperkinetic Movements; JAMA Neurology

Leadership Positions in Professional SocietiesParkinson’s Foundation (Medical Advisor)