4-Phenylbutyrate Clinical Trial Starting Recruitment

The clinical trial pilot for 4-phenylbutyrate (4PB) is starting recruitment of the first patients. This is the first clinical trial with a therapy targeting the STXBP1 protein.

We are thankful to Jacqueline Burre, PhD and Zachary Grinspan, MD, MS and their teams at Weill Cornell, for their work that started in the lab to identify 4-phenylbutyrate through to this clinical trial.

For information on 4-phenylbutyrate and the trial, including inclusion criteria and how to contact the study team, check out our blog post.

This clinical trial has been funded by grants from the ODC's Million Dollar Bike Ride - Lulu's Crew Team STXBP1, and Clara Inspired. In addition, the STXBP1 Foundation and Clara Inspired are excited to announce that we will be offering a Patient Assistance Program for all enrolled patients in the study to cover unreimbursed medical and travel expenses up to a set amount.