CI

At a glance

ClinicalIndex Comparison Record
Phase 3Completed· 89 enrolled
Drug / intervention
sapropterin dihydrochloride, 6R-BH4, tetrahydrobiopterindrug
Likely dose
Not stated in record
Key inclusion· 5
  • ✓Age 8 years or older
  • ✓Completed at least 7 of 8 doses in prior Study PKU-001
  • ✓Demonstrated responsiveness to Phenoptin™ (≥30% reduction in blood phenylalanine from baseline in prior study)
  • ✓Current blood phenylalanine level ≥450 μmol/L at screening
Key exclusion· 6
  • ✕ALT >5× upper limit of normal (WHO Grade 3 or higher) at screening
  • ✕Concurrent disease interfering with study participation or safety (seizure disorder, oral steroid-dependent asthma or other corticosteroid-dependent condition, insulin-dependent diabetes, or organ transplant recipient)
  • ✕Serious neuropsychiatric illness not currently under medical management (e.g., major depression)
  • ✕Requirement for concomitant treatment with folate synthesis inhibitors (e.g., methotrexate)

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

Search/NCT00104247
NCT00104247Phase 3Completed
CompareCT.gov

A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Phenoptin™ in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels

BioMarin Pharmaceutical·interventional·Posted Feb 25, 2005·Updated Jul 23, 2014

In Brief

A Phase 3 clinical trial evaluating sapropterin dihydrochloride, 6R-BH4, tetrahydrobiopterin for Phenylketonurias. Completed, enrolled 89 participants across 13 sites.

Detailed Summary

The primary objective of this study is to evaluate the efficacy of Phenoptin™ (sapropterin dihydrochloride) in reducing blood phenylalanine (Phe) levels in subjects with phenylketonuria.

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsPhenylketonurias
CountriesUnited States
Collaborators--

Timeline

Phase 3CompletedFinished
20052006200720082009201020112012201320142015201620172018201920202021202220232024202520262027
First PostedFeb 25, 2005
Enrollment StartMar 1, 2005
Study CompletionFeb 1, 2006
TodayJul 2, 2026
Posted 21.4 years ago

Interventions

sapropterin dihydrochloride, 6R-BH4, tetrahydrobiopterindrug