Clin2
NCT06337864Possibly a fitRecruiting

Large neutral amino acids for adults with classical PKU

Brain DiseasesBrain Diseases, MetabolicBrain Diseases, Metabolic, InbornGenetic Diseases, InbornMetabolism, Inborn ErrorsAmino Acid Metabolism, Inborn ErrorsMetabolic DiseasePhenylketonurias

Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.

This study tests whether a specific mix of large neutral amino acids (LNAAs) can help adults with classical PKU by lowering phenylalanine levels in the brain. It includes an MRI and PET scan to see how the treatment affects brain activity.

Summary written for real people, not researchers, by Clin2.

Phase
N/A
Enrollment
30 people
Ages
18 years to 50 years
Study type
Interventional

Who can take part

  • You are 18 or older and have been diagnosed with classical PKU (a severe type of phenylketonuria).
  • You started PKU treatment within the first month of life and followed the special diet until at least age 15.
  • You have an IQ above 84 based on a recent test.
  • You are not taking sapropterin (BH4) or Pegvaliase (PALYNZIQ) and have no metal implants or other MRI restrictions.
  • You are not pregnant, breastfeeding, or planning pregnancy, and you weigh under 110 kg (about 242 pounds).

View the official record on ClinicalTrials.gov

Quick eligibility check

Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.

Similar studies

Other trials that look related to this one.

NCT06332807Recruiting· Phase 1/Phase 2
Gene therapy trial for classic PKU

This trial tests a one-time gene therapy called NGGT002 for adults with classic PKU who cannot take or do not respond well to current treatments. The goal is to see if it can safely lower blood phenylalanine levels.

Orange, California
NCT04969809Not yet recruiting
Diet program study for adults with PKU

This study looks at how nutrition affects risk factors in adults with PKU (a condition that requires a special diet to control certain blood chemicals). If you can manage your diet independently and agree to follow the study diet plan, this may help researchers find better nutritional approaches.

NCT06687733Recruiting· Phase 1/Phase 2
Gene therapy for phenylketonuria

This trial tests a new gene therapy, NGGT002, which delivers a healthy gene to help people with phenylketonuria (PKU) manage their phenylalanine levels. It may reduce the need for strict diet control.

Bengbu, Anhui
NCT05128149Recruiting
PKU care guidelines and how they affect daily well-being

This study looks at whether following specific care guidelines for phenylketonuria (PKU) leads to better metabolic control (blood phenylalanine levels) and better patient well-being. You may be asked to complete questionnaires and follow the study plan while receiving the usual PKU treatment your clinic recommends.

Zurich
NCT07241234Recruiting· Phase 1
Study of an experimental drug for phenylketonuria (PKU)

This trial tests an investigational drug (AG-181) for people with PKU who have a specific genetic change (R408W mutation). It aims to check if the drug is safe and how it works in your body.

Indianapolis, Indiana
NCT07318909Not yet recruiting· Early Phase 1
Testing GS1168 injection for adults with PKU

This study tests a new injection called GS1168 for adults with phenylketonuria (PKU) whose current treatments are not working well. The goal is to see if it can help control their PKU better.

Hefei, Anhui

Hear when a new Brain Diseases trial opens

We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.