Study of Phenoptin to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet

Summary

The primary objective of this trial is to evaluate the ability of Phenoptin to increase phenylalanine (phe) tolerance in children with phenylketonuria who are following a phe-restricted diet.

Timeline

Start

2006-02

Primary completion

—

Completion

2006-11

Outcome

Met primary endpoint

paper mean (+/-SD) Phe supplement tolerated by the sapropterin group had increased significantly from the pretreatment amount (0 mg/kg/d) to 20.9 (+/-15.4) mg/kg/d (P < .001) PMID 19261295 ↗

paper the mean difference from the sapropterin group (+/-SE) was 17.7 +/- 4.5 mg/kg/d (P < .001). PMID 19261295 ↗

release “In the primary endpoint, Phenoptin enabled a mean increase of 20.9 mg/kg/day in Phe supplementation for those patients on Phenoptin (p<0.001).” biomarin.com ↗

release “Sapropterin dihydrochloride (sapropterin) increases Phenylalanine (Phe) tolerance in children with phenylketonuria (PKU) maintained on a Phe-restricted diet” biomarin.com ↗

release “Mean daily Phe intake (dietary + supplement) increased from 16.8 to 43.8mg/kg/day with sapropterin (p<0.001)” biomarin.com ↗

Publications

• Trefz FK, Burton BK, Longo N, Casanova MM, Gruskin DJ, Dorenbaum A, Kakkis ED, Crombez EA, Grange DK, Harmatz P, Lipson MH, Milanowski A, Randolph LM, Vockley J, Whitley CB, Wolff JA, Bebchuk J, Christ-Schmidt H, Hennermann JB; Sapropterin Study Group. Efficacy of sapropterin dihydrochloride in increasing phenylalanine tolerance in children with phenylketonuria: a phase III, randomized, double-blind, placebo-controlled study. J Pediatr. 2009 May;154(5):700-7. doi: 10.1016/j.jpeds.2008.11.040. Epub 2009 Mar 4.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	sapropterin	Unknown	20 mg/kg	Oral

Indications