NCT06337864 Effect of Large Neutral Amino Acids in Adults With Classical Phenylketonuria
| NCT ID | NCT06337864 |
| Status | Recruiting |
| Phase | — |
| Sponsor | Rigshospitalet, Denmark |
| Condition | Brain Diseases |
| Study Type | INTERVENTIONAL |
| Enrollment | 30 participants |
| Start Date | 2024-11-01 |
| Primary Completion | 2026-09 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
This trial targets 30 participants in total. It began in 2024-11-01 with a primary completion date of 2026-09.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief 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 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 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
Contact & Investigator
Allan Lund, Professor, MD, DMSc
PRINCIPAL INVESTIGATOR
Rigshospitalet, Denmark
Frequently Asked Questions
Who can join the NCT06337864 clinical trial?
This trial is open to participants of all sexes, aged 18 Years or older, up to 50 Years, studying Brain Diseases. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT06337864 currently recruiting?
Yes, NCT06337864 is actively recruiting participants. Contact the research team at allan.lund@regionh.dk for enrollment information.
Where is the NCT06337864 trial being conducted?
This trial is being conducted at Copenhagen, Denmark, Copenhagen, Denmark.
Who is sponsoring the NCT06337864 clinical trial?
NCT06337864 is sponsored by Rigshospitalet, Denmark. The principal investigator is Allan Lund, Professor, MD, DMSc at Rigshospitalet, Denmark. The trial plans to enroll 30 participants.