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Recruiting Not applicable

Effect of Large Neutral Amino Acids in Adults With Classical Phenylketonuria

NCT06337864 · tracked via the Priya Life Science Denmark tracker
Phase
Not applicable
Started
2024-11-01
Last updated
2025-04-06

Condition(s) studied

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

Investigational drug(s) / intervention(s)

PreKUnil® LNAA Medical Food for PKU

PreKUnil® LNAA Medical Food for PKU: PreKUnil® LNAA Medical Food for PKU is a commercially available active LNAA treatment product for PKU.

Study summary

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.

Eligibility

Sex
ALL
Min age
18 Years
Max age
50 Years
Healthy volunteers
Accepted
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 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 Exclusion Criteria: * 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

Primary outcome measure(s)

  • Dynamic positron emission tomography (PET) imaging with the fluorine-18-labeled tracer [18F]-(E)-N-(3-iodoprop-2-enyl)-2β-carbofluoroethoxy-3β-(4'-methyl phenyl)nortropane ([18F]FE-PE2I) — Crossover study: at 8, 10 and 18 weeks from baseline, Extension study: at 12 months from baseline
    Change in specific binding ratio of dopamine transporter (DaT) with \[18F\]FE-PE2I

Trial sites (2)

FacilityCityRegionStatus
Center for Inherited Metabolic Diseases Copenhagen Denmark Recruiting
Copenhagen University Hospital, Rigshospitalet Copenhagen Denmark Not Yet Recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06337864 on ClinicalTrials.gov ↗ ← All trials in Denmark