Double blind placebo control trial of large neutral amino acids in treatment of PKU: Effect on blood phenylalanine

Reuben Matalon, K. Michals-Matalon, G. Bhatia, A. B. Burlina, A. P. Burlina, C. Braga, L. Fiori, M. Giovannini, E. Grechanina, P. Novikov, J. Grady, S. K. Tyring, F. Guttler

Research output: Contribution to journalArticle

59 Citations (Scopus)

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.

Original languageEnglish (US)
Pages (from-to)153-158
Number of pages6
JournalJournal of Inherited Metabolic Disease
Volume30
Issue number2
DOIs
StatePublished - Apr 2007

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Neutral Amino Acids
Phenylketonurias
Phenylalanine
Placebos
Ukraine
Russia
Therapeutics
Safety
Brain

ASJC Scopus subject areas

  • Genetics(clinical)
  • Genetics
  • Endocrinology

Cite this

Double blind placebo control trial of large neutral amino acids in treatment of PKU : Effect on blood phenylalanine. / Matalon, Reuben; Michals-Matalon, K.; Bhatia, G.; Burlina, A. B.; Burlina, A. P.; Braga, C.; Fiori, L.; Giovannini, M.; Grechanina, E.; Novikov, P.; Grady, J.; Tyring, S. K.; Guttler, F.

In: Journal of Inherited Metabolic Disease, Vol. 30, No. 2, 04.2007, p. 153-158.

Research output: Contribution to journalArticle

Matalon, R, Michals-Matalon, K, Bhatia, G, Burlina, AB, Burlina, AP, Braga, C, Fiori, L, Giovannini, M, Grechanina, E, Novikov, P, Grady, J, Tyring, SK & Guttler, F 2007, 'Double blind placebo control trial of large neutral amino acids in treatment of PKU: Effect on blood phenylalanine', Journal of Inherited Metabolic Disease, vol. 30, no. 2, pp. 153-158. https://doi.org/10.1007/s10545-007-0556-4
Matalon, Reuben ; Michals-Matalon, K. ; Bhatia, G. ; Burlina, A. B. ; Burlina, A. P. ; Braga, C. ; Fiori, L. ; Giovannini, M. ; Grechanina, E. ; Novikov, P. ; Grady, J. ; Tyring, S. K. ; Guttler, F. / Double blind placebo control trial of large neutral amino acids in treatment of PKU : Effect on blood phenylalanine. In: Journal of Inherited Metabolic Disease. 2007 ; Vol. 30, No. 2. pp. 153-158.
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