Safety and Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatric Symptoms in Phenylketonuria (PKU) Patients

Phase 3 Completed 206 enrolled BioMarin Pharmaceutical

RandomizedParallel-groupQuadruple-blindTreatment

Summary

This double-blind, placebo-controlled, randomized study is designed to evaluate the safety and therapeutic effects of sapropterin dihydrochloride on neuropsychiatric symptoms in subjects with PKU.

Timeline

Start: 2010-08
Primary completion: 2013-03
Completion: 2013-03

Outcome

Missed primary endpoint

registry p = 0.085; Mean Difference (Final Values) -4.2 (95% CI -8.9 to 0.6); ANCOVA NCT01114737 ↗

registry p = 0.67; Relative Risk 0.87 (95% CI 0.46 to 1.64); Cochran-Mantel-Haenszel NCT01114737 ↗

Publications

Grant ML, Jurecki ER, McCandless SE, Stahl SM, Bilder DA, Sanchez-Valle A, Dimmock D. Neuropsychiatric Function Improvement in Pediatric Patients with Phenylketonuria. J Pediatr. 2023 Sep;260:113526. doi: 10.1016/j.jpeds.2023.113526. Epub 2023 May 30.

Drugs

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