Clinical Trials

RTX-134 for the Treatment of Phenylketonuria (PKU)

Study Title
A Phase 1b Open-Label Single Dose Safety, Tolerability, and Pharmacokinetics Study of RTX-134 in Adults with Phenylketonuria


Brief Summary

This study will evaluate the safety and tolerability of RTX-134 in adult patients with PKU.

Primary Objectives
  1. To evaluate the safety and tolerability of RTX-134
  2. To correlate dose with percent reduction in serum phenylalanine levels relative to baseline
  3. Determine a preliminary dose to achieve serum Phe levels <360 µmol/L and <600 µmol/L
Secondary Objectives
  1. To evaluate pharmacodynamics of RTX-134, including measurement of trans-cinnamic acid.

If you are a patient or loved one interested in learning more about RTX-134 for the treatment of PKU or participating in a clinical trial, please visit our clinical trial website for more information at