Recruiting

Gene therapy to help adults with severe inherited PKU

Plain-English translation of NCT06332807 on ClinicalTrials.gov ↗ · Source last updated August 2026 · Translation generated August 2026 · How we translate trials

Phase 1/2 — A combined trial that checks safety and dosing while also starting to look at whether the treatment works.

What is this trial?

This trial is testing a new gene therapy called NGGT002 designed to treat adults with classic phenylketonuria (PKU), a rare inherited condition where the body cannot properly process a protein building block called phenylalanine. The medication is given as a single intravenous infusion and works by helping your body produce an enzyme it's missing. Researchers want to find the right dose and confirm the treatment is safe and effective.

Classic PKU is a severe condition with limited treatment options, and some patients don't respond well to the medications currently available. This trial exists to explore whether this new gene therapy could offer hope to adults who have struggled to control their phenylalanine levels with existing treatments.

Do you qualify?

You likely qualify if…

you have been diagnosed with classic PKU caused by specific genetic mutations with no remaining enzyme activity
you are between 18 and 55 years old
you have not benefited from or cannot tolerate current PKU medications like Kuvan or Palynziq
your phenylalanine levels have remained high (at least 600 μmol/L) in the past 6 months despite treatment
you are willing to keep diet records and work with a dietitian throughout the study
you can commit to using reliable birth control for at least one year after receiving the treatment

You likely don't qualify if…

you have PKU caused by something other than PAH gene mutations
you have antibodies that would fight off the gene therapy
your liver or blood cell counts are too abnormal before the study starts
you are currently taking PKU medications and doing well with good disease control
you are unable or unwilling to follow study procedures and safety requirements

What participation looks like

You will receive a single infusion of the medication through an IV line at a medical center. The study has two phases: first, the research team will treat a small group at a lower dose to ensure safety, then potentially move to a higher dose with another group. You'll have regular blood tests and clinic visits to monitor your phenylalanine levels and overall health, and you'll be asked to keep track of what you eat and follow guidance from a dietitian throughout the trial.

AI-generated summary from trial data · Aug 26, 2026 · Not medical advice

Trial locations(5 sites)

United States

Children's Hospital of Orange County Hospital, Orange, California

University of Minnesota, Minneapolis, Minnesota

Atlantic Health System, Morristown, New Jersey

University of Pittsburgh Medical Center, Pittsburgh, Pennsylvania

University or Texas, Southwestern medical Center, Dallas, Texas