Trial reportJournal of inherited metabolic disease2007
Double blind placebo control trial of large neutral amino acids in treatment of PKU: effect on blood phenylalanine.
Trial report in Journal of inherited metabolic disease, 2007. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 44 papers, 1 of them a synthesis that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
44 citing papers in PubMed, 1 synthesis or guideline pooled it, 102 citations in OpenAlex.
- Protein substitute for children and adults with phenylketonuria.The Cochrane database of systematic reviews · 2015Pooled it
- Breakfast with glycomacropeptide compared with amino acids suppresses plasma ghrelin levels in individuals with phenylketonuria.Molecular genetics and metabolism · 2010Trial
- Improved nutritional management of phenylketonuria by using a diet containing glycomacropeptide compared with amino acids.The American journal of clinical nutrition · 2009Trial
- Current state of the treatment landscape of phenylketonuria.Orphanet journal of rare diseases · 2025Review
- Amino Acid Profile Alterations in Phenylketonuria: Implications for Clinical Practice.Metabolites · 2024Article
- Protein substitutions as new-generation pharmanutrition approach to managing phenylketonuria.Clinical and experimental pediatrics · 2023Article
- The effect of casein glycomacropeptide versus free synthetic amino acids for early treatment of phenylketonuria in a mice model.PloS one · 2022Article
- Beneficial Effects of Slow-Release Large Neutral Amino Acids after a Phenylalanine Oral Load in Patients with Phenylketonuria.Nutrients · 2021Article
- A quantitative model for metabolic intervention using gut microbes.Biotechnology progress · 2021Article
- The Impact of a Slow-Release Large Neutral Amino Acids Supplement on Treatment Adherence in Adult Patients with Phenylketonuria.Nutrients · 2020Article
- Large Neutral Amino Acids (LNAAs) Supplementation Improves Neuropsychological Performances in Adult Patients with Phenylketonuria.Nutrients · 2020Article
- Outcomes in pediatric studies of medium-chain acyl-coA dehydrogenase (MCAD) deficiency and phenylketonuria (PKU): a review.Orphanet journal of rare diseases · 2020Review
- Large Neutral Amino Acid Therapy Increases Tyrosine Levels in Adult Patients with Phenylketonuria: A Long-Term Study.Nutrients · 2019Article
- Article
- Nutritional management of phenylalanine hydroxylase (PAH) deficiency in pediatric patients in Canada: a survey of dietitians' current practices.Orphanet journal of rare diseases · 2019Article
- Effect of Blood Phenylalanine Levels on Oxidative Stress in Classical Phenylketonuric Patients.Cellular and molecular neurobiology · 2018Article
- Comparison of Glycomacropeptide with Phenylalanine Free-Synthetic Amino Acids in Test Meals to PKU Patients: No Significant Differences in Biomarkers, Including Plasma Phe Levels.Journal of nutrition and metabolism · 2018Article
- Article
- Effect of natural PAL-enzyme on the quality of egg white and mushroom flour and study its impact on the expression of PKU related genes and phenylalanine reduction in mice fed on.Journal, genetic engineering & biotechnology · 2017Article
- The complete European guidelines on phenylketonuria: diagnosis and treatment.Orphanet journal of rare diseases · 2017Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
13 authors at 7 institutions in 4 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Large neutral amino acids (LNAA) have been used on a limited number of patients with phenylketonuria (PKU) with the purpose of decreasing the influx of phenylalanine (Phe) to the brain. In an open-label study using LNAA, a surprising decline of blood Phe concentration was found in patients with PKU in metabolic treatment centres in Russia, the Ukraine, and the United States. To validate the data obtained from this trial, a short-term double-blind placebo control study was done using LNAA in patients with PKU, with the participation of three additional metabolic centres--Milan, Padua and Rio de Janeiro. The results of the short trial showed significant lowering of blood Phe concentration by an average of 39% from baseline. The data from the double-blind placebo control are encouraging, establishing proof of principle of the role of orally administered LNAA in lowering blood Phe concentrations in patients with PKU. Long-term studies will be needed to validate the acceptability, efficacy and safety of such treatment.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.