CI

At a glance

ClinicalIndex Comparison Record
Phase 2Completed· 12 enrolled
Drug / intervention
Phenoptindrug
Likely dose
Phenoptin 5mg/kgfrom record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.

Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.

Search/NCT00355264
NCT00355264Phase 2Completed
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Phase 2, Multicenter, Open Label Study of Phenoptin in Subjects With Hyperphenylalaninemia Due to Primary BH4 Deficiency

BioMarin Pharmaceutical·interventional·Posted Jul 21, 2006·Updated Sep 25, 2020

In Brief

A Phase 2 clinical trial evaluating Phenoptin for Tetrahydrobiopterin Deficiencies and Hyperphenylalaninemia, Non-Phenylketonuric. Completed, enrolled 12 participants across 10 sites in 2 countries.

Detailed Summary

The purpose of this study is to evaluate the ability of Phenoptin to control blood phenylalanine levels in subjects who have hyperphenylalaninemia due to a primary BH4 deficiency and to evaluate the safety of Phenoptin in this population. Some subjects were receiving non-registered formulations of BH4 at enrollment and this treatment was suspended after Part 1 and within one day the subjects started Phenoptin at approximately the same dose.

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsTetrahydrobiopterin Deficiencies, Hyperphenylalaninemia, Non-Phenylketonuric
CountriesGermany, United States
Collaborators--

Timeline

Phase 2CompletedFinished
2006200720082009201020112012201320142015201620172018201920202021202220232024202520262027
First PostedJul 21, 2006
Enrollment StartAug 1, 2006
Primary CompletionJun 1, 2009
TodayJul 2, 2026
Enrollment to primary: 2.8 yearsPosted 19.9 years ago

Interventions

Phenoptindrug

5mg/kg/day orally, dose may be adjusted to between 5-20 mg/kg/day by investigator at week 6 to control blood Phe levels