PHENYLKETONURIAS
Clinical trials for PHENYLKETONURIAS explained in plain language.
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New gene therapy aims to tame PKU in first human test
Disease control Recruiting nowThis early-phase study tests a single IV dose of NGGT002 gene therapy in 15 adults with phenylketonuria (PKU), a genetic disorder that causes harmful buildup of phenylalanine. The main goal is to check safety over one year, with long-term follow-up for five years. Researchers wil…
Matched conditions: PHENYLKETONURIAS
Phase: EARLY_PHASE1 • Sponsor: The First Affiliated Hospital of Bengbu Medical University • Aim: Disease control
Last updated Jun 27, 2026 09:10 UTC
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One-Time gene fix could ease PKU's strict diet burden
Disease control Recruiting nowThis early-stage trial tests a gene therapy called NGGT002 for adults with classic PKU, a genetic disorder that makes it hard to break down an amino acid called phenylalanine. The treatment delivers a working copy of the missing gene via a single IV infusion. Researchers will mon…
Matched conditions: PHENYLKETONURIAS
Phase: PHASE1, PHASE2 • Sponsor: NGGT INC. • Aim: Disease control
Last updated Jun 27, 2026 08:01 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-term behavioral program called Show Me FIRST to help teens with PKU manage anxiety and depression. Thirty adolescents will be randomly assigned to receive the program right away or after a wait period. The researchers will also check if new phone-based te…
Matched conditions: PHENYLKETONURIAS
Phase: NA • Sponsor: University of Missouri-Columbia • Aim: Symptom relief
Last updated Jun 27, 2026 08:06 UTC
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PKU drug palynziq under the microscope: what it does to diet and brain
Knowledge-focused Recruiting nowThis study follows 45 people with PKU who are taking Palynziq to see how the drug changes their diet, brain health, and nutrition. Researchers will track protein intake and blood levels over time. The goal is to understand the full impact of Palynziq beyond just lowering phenylal…
Matched conditions: PHENYLKETONURIAS
Sponsor: Emory University • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:11 UTC