PHENYLKETONURIAS
Clinical trials for PHENYLKETONURIAS explained in plain language.
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Gene therapy hope for PKU: first human safety trial launches
Disease control Recruiting nowThis early-stage study tests a single intravenous dose of NGGT002, a gene therapy for adults with phenylketonuria (PKU), a genetic disorder that prevents the breakdown of the amino acid phenylalanine. Fifteen participants will be monitored for safety and changes in blood phenylal…
Matched conditions: PHENYLKETONURIAS
Phase: EARLY_PHASE1 • Sponsor: The First Affiliated Hospital of Bengbu Medical University • Aim: Disease control
Last updated May 26, 2026 08:14 UTC
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One-Time gene shot aims to tame rare metabolic disorder
Disease control Recruiting nowThis early-stage trial tests a single dose of NGGT002, a gene therapy that delivers a working copy of the PAH gene, in 12 adults with classic PKU. The goal is to see if it safely lowers harmful phenylalanine levels in the blood. Participants will be monitored for 5 years to check…
Matched conditions: PHENYLKETONURIAS
Phase: PHASE1, PHASE2 • Sponsor: NGGT INC. • Aim: Disease control
Last updated May 26, 2026 08:07 UTC
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New drug lets PKU patients eat more protein – but lifelong treatment still needed
Disease control Recruiting nowThis study looks at how Palynziq, a medication for phenylketonuria (PKU), improves diet, brain function, and nutrition. PKU is a genetic disorder that makes it hard to break down an amino acid called phenylalanine, which can build up and cause brain damage. The study will follow …
Matched conditions: PHENYLKETONURIAS
Sponsor: Emory University • Aim: Disease control
Last updated May 15, 2026 11:57 UTC
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New therapy aims to ease anxiety in teens with rare metabolic disorder
Symptom relief Recruiting nowThis study tests a short, skills-based therapy program (Show Me FIRST) to help reduce anxiety and depression in 30 adolescents with PKU, a rare metabolic disorder. Participants will be randomly assigned to receive the therapy right away or after a waiting period. The goal is to s…
Matched conditions: PHENYLKETONURIAS
Phase: NA • Sponsor: University of Missouri-Columbia • Aim: Symptom relief
Last updated May 26, 2026 08:13 UTC