Phenylketonuria clinical trials at UCSF
1 research study open to eligible people
Phenylketonuria is a genetic disorder where the body can't break down an amino acid called phenylalanine. UCSF is conducting trials to test the safety of a new drug given through an IV to adults with this condition. These trials focus on understanding how the drug affects phenylketonuria symptoms.
Phase 1/Phase 2 Open-label Study to Evaluate the Safety, Tolerability, and Efficacy of a Single Intravenous Administration of SAR444836 in Adult Participants With Phenylketonuria
open to eligible people ages 18-65
This is a single group Phase 1/Phase 2, 1-arm, open-label study with SAR444836, an adeno-associated virus (AAV) vector-mediated gene transfer of human phenylalanine hydroxylase (PAH), for the treatment of adult participants with phenylketonuria…
San Francisco, California and other locations
Our lead scientists for Phenylketonuria research studies include Irene Chang.
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