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Official title Effect of Large Neutral Amino Acids in Adults With Classical Phenylketonuria
ClinicalTrials.gov ID: NCT06337864
What this study is testing
- What it's testing
- 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.
- Which group you join is decided by chance.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 to 50. Healthy volunteers may be eligible.
You may be able to join if
- Patients ≥ 18 years of age with Classical PKU molecularly confirmed via the finding of two pathogenic variants in the phenylalanine hydroxylase (PAH)...
- 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
You likely can't join 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
See the full 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...
- 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
- 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
The study team makes the final eligibility decision.
Where it's taking place
- Copenhagen, Denmark
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years to 50 years. Healthy volunteers may be eligible. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Copenhagen, Denmark. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.