TY - JOUR
T1 - Double blind placebo control trial of large neutral amino acids in treatment of PKU
T2 - Effect on blood phenylalanine
AU - Matalon, R.
AU - Michals-Matalon, K.
AU - Bhatia, G.
AU - Burlina, A. B.
AU - Burlina, A. P.
AU - Braga, C.
AU - Fiori, L.
AU - Giovannini, M.
AU - Grechanina, E.
AU - Novikov, P.
AU - Grady, J.
AU - Tyring, S. K.
AU - Guttler, F.
N1 - Funding Information:
Acknowledgement This work was supported in part by the Genetics Research Trust, the Mid-Atlantic Connection for PKU and Allied Disorders (MACPAD), the South Texas Association for PKU and Allied Disorders (STAPAD), and PKU and Allied Disorders of Wisconsin (PADOW). The authors would like to acknowledge the generous donation of PreKUNil and NeoPhe by PreKU lab, Denmark.
PY - 2007/4
Y1 - 2007/4
N2 - 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.
AB - 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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U2 - 10.1007/s10545-007-0556-4
DO - 10.1007/s10545-007-0556-4
M3 - Article
C2 - 17334706
AN - SCOPUS:33947613027
SN - 0141-8955
VL - 30
SP - 153
EP - 158
JO - Journal of Inherited Metabolic Disease
JF - Journal of Inherited Metabolic Disease
IS - 2
ER -