Effect of Large Neutral Amino Acids in Adults With Classical Phenylketonuria
Recruiting
N/A
Interventional Study
Brain Diseases
Brain Diseases, Metabolic
Brain Diseases, Metabolic, Inborn
Genetic Diseases, Inborn
Metabolism, Inborn Errors
Amino Acid Metabolism, Inborn Errors
Metabolic Disease
Phenylketonurias
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Healthy Volunteers Welcome
You do not need to have the condition being studied to take part.
At a Glance
- Age
- 18 – 50
- Sex
- Any
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 30 (estimated)
- Sponsor
- Rigshospitalet, Denmark · Other
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About This Trial
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, …
Trial Locations
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Eligibility Criteria
Patients ≥ 18 years of age with Classical PKU molecularly confirmed via the finding of two pathogenic variants in the phenylalanine hydroxylase (PAH) gene and/or historical evidence of Phe concentrations ≥1200 μmol/L in the medical history
Inclusion Criteria:
* Treatment initiation within the firs…
Contacts