Safety & Efficacy of AlloRx SC® in PTHS Patients

Summary

This study will evaluate the safety of AlloRx Stem Cells® in subjects with Pitt Hopkins syndrome ages 2-45 with a molecularly confirmed pathogenic mutation in TCF4 or 18q deletion including TCF4. Participants will receive mesenchymal stem cells infusions every 3 months for a year with completion of outcome measures to assess the efficacy of the product.

Timeline

Start

2021-12

Primary completion

2022-12

Completion

2023-12

Drugs

Indications