Phase 3
N=90
Study of Phenoptin to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet
Phenylketonurias
Bottom Line
View on ClinicalTrials.gov: NCT00272792 ↗Enrolled (actual)
90
Serious AEs
0.0%
Results posted
Jul 2009
Primary outcome: Primary: Amount of Dietary Supplemented Phenylalanine (Phe)Tolerated in Children With Phenylketonuria — 20.9; 2.9 mg/kg/day — p=<0.001
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Sapropterin Dihydrochloride (Drug); Placebo (Drug)
- Age
- Pediatric · 4+ yrs
- Sex
- All
- Sponsor
- BioMarin Pharmaceutical
- Primary completion
- —
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 |
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.
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)
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.