Phase 3 Completed N=90 Randomized Double-blind Treatment

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

Phenylketonurias

Source: ClinicalTrials.gov NCT00272792 ↗

Enrolled (actual)

Serious AEs

0.0%

Results posted

Jul 2009

Primary outcomePrimary: Amount of Dietary Supplemented Phenylalanine (Phe)Tolerated in Children With Phenylketonuria — 20.9; 2.9 mg/kg/day — p=<0.001

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.

Outcome Measures

Outcome	Result	p-value
PRIMARY Amount of Dietary Supplemented Phenylalanine (Phe)Tolerated in Children With Phenylketonuria	20.9; 2.9	<0.001 sig
SECONDARY Change in Phenylalanine Levels From Baseline to Week 3	-147.4; -90.3	0.009 sig

Eligibility Criteria

Inclusion Criteria

Clinical diagnosis of PKU with hyperphenylalaninemia (HPA) documented by at least one blood Phe measurement >/=360 umol/L (6 mg/dL)
Under dietary control with a Phe-restricted diet as evidenced by:· Estimated daily Phe tolerance 2 times the upper limit of normal (i.e., Grade 1 or higher based on World Health Organization Toxicity Criteria) at screening
Concurrent disease or condition that would interfere with study participation or safety (e.g., seizure disorder, oral steroid-dependent asthma or other condition requiring oral or parenteral corticosteroid administration, or insulin-dependent diabetes)

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Data sourced from ClinicalTrials.gov (NCT00272792). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.