Pitt-Hopkins Syndrome

Arizona State University
Trials underway

Dr. Adams and Dr. Krajmalnik-Brown led a Phase 2, double-blind, placebo-controlled trial assessing the impact of Microbiota Transfer Therapy in patients with Pitt-Hopkins Disease, a genetic disease associated with a severe form of Autism. More details on the study are available here. The study demonstrated microbiota transfer therapy is safe in Pitt-Hopkins patients with improvements in gastrointestinal and Pitt-Hopkins symptoms. The FDA is currently reviewing the treatment protocol for approval. Another trial is in development with a next-generation microbiota therapeutic to allow more patients access who are unable to swallow the standard microbiota therapeutic capsules.