Effect of Large Neutral Amino Acids in Adults With Classical Phenylketonuria
Recruiting now · Not applicable
Conditions studied: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Genetic Diseases, Inborn, Metabolism, Inborn Errors, Amino Acid Metabolism, Inborn Errors, Metabolic Disease, Phenylketonurias
In brief
The overall aim of this study is to evaluate LNAA treatment as a potential alternative to conventional dietary treatment for PKU. This study investigates the effects of LNAA treatment compared to the classic dietary treatment on cerebral dopamine synthesis in patients with classic PKU. We will assess LNAAs effectiveness on neurotransmitter synthesis, cognitive function, mental health, and safety, compared to the standard diet.
Key facts
- Study ID
- NCT06337864
- Run by
- Rigshospitalet, Denmark
- People needed
- 30
- Starts
- 2024-11-01
- Expected to finish
- 2026-09-30
- Last updated by the study team
- 2025-04-06
Who can join
Age: 18 and older, up to 50. Sex: any. Healthy volunteers: accepted.
You may qualify if…
- Treatment initiation within the first month of life
- Intelligence quotient over 84, based upon the baseline neuropsychological evaluation
- Conventional dietary treatment up to minimum 15 years of age
- Signed informed consent
- Willing and able to comply with the protocol and study procedures
You may not qualify if…
- Unable or unwilling to adhere to the requirements of the study
- A female who is pregnant or breastfeeding or planning to get pregnant during the study period
- Concomitant medication that may interfere with the PET analysis, as judged by the investigator
- A serious neuropsychiatric disease that could interfere with the subject's ability to participate in the study at the discretion of the investigator
- Concomitant treatment with BH4 supplementation (sapropterin) or Pegvaliase-pqpz (PALYNZIQ)
- Failing to submit at least one blood Phe home sample during the year before study initiation
- Standard MRI contraindications
- Body weight over 110 kg
Where it is running
- Center for Inherited Metabolic Diseases — Copenhagen, Denmark (enrolling)
- Copenhagen University Hospital, Rigshospitalet — Copenhagen, Denmark
Full record on ClinicalTrials.gov
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