At a glance
ClinicalIndex Comparison Record- ✓Clinical diagnosis of PKU with hyperphenylalaninemia documented by blood phenylalanine ≥360 μmol/L (6 mg/dL)
- ✓Under dietary control with phenylalanine-restricted diet and estimated daily phenylalanine tolerance ≤1000 mg/day
- ✓At least 6 months of blood phenylalanine control (mean level ≤480 μmol/L) prior to enrollment
- ✓Aged 4 to 12 years inclusive at screening
- ✕Any condition that renders subject at high risk for treatment non-compliance or inability to complete the study
- ✕Prior history of organ transplantation
- ✕Perceived to be unreliable or unavailable for study participation
- ✕Use of investigational agent within 30 days prior to screening or requirement for investigational agent/vaccine before study completion
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
A Phase 3, Double-blind, Placebo-controlled Study to Evaluate the Safety and Efficacy of Phenoptin to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet
In Brief
A Phase 3 clinical trial evaluating Sapropterin Dihydrochloride and Placebo for Phenylketonurias. Completed, enrolled 45 participants across 9 sites.
Detailed 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.
Study Details
Timeline
Interventions
Phenoptin, provided in tablets containing 100 mg of sapropterin dihydrochloride each, was administered orally once daily in the morning as the number of tablets equivalent to a 20mg/kg/day dose dissolved in 4-8 oz (120-240 mL) of water or apple juice for 6 weeks. A follow-up call or visit was made 4 weeks later during this double-blind, placebo-controlled study.
Placebo, provided as tablets similar to Phenoptin tablets, was administered orally once daily in the morning as the number of tablets equivalent to a 20mg/kg/day dose dissolved in 4 8 oz (120-240 mL) of water or apple juice. for 6 weeks. A follow-up call or visit was made 4 weeks later during this double-blind, placebo-controlled study.