NCT00532844

A Phase 2, Pharmacokinetic (PK) Study of 6R-BH4 Alone or 6R-BH4 With Vitamin C in Subjects With Endothelial Dysfunction

A Phase 2 Randomized Open-label 2-Treatment 2-Sequence 2-Period Crossover PK Study to Compare Plasma Concentrations of BH4 in Subjects With Endothelial Dysfunction Following 14 Days of Treatment by 2 Regimens: 6R-BH4 With Vitamin C and 6R-BH4 Alone

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 52 (actual)
• Sponsor: BioMarin Pharmaceutical · Industry
• Sex: All
• Age: 18 Years – 75 Years
• Healthy volunteers: Not accepted

Summary

This Phase 2, randomized, open-label, 2-treatment, 2-sequence, 2-period crossover, pharmacokinetic (PK) study will compare plasma concentrations of BH4 in subjects with endothelial dysfunction following 14 days of treatment by each of 2 regimens: sapropterin dihydrochloride with vitamin C and sapropterin dihydrochloride alone.

Detailed description

This was a Phase 2, randomized, open-label, 2-treatment, 2-sequence, 2-period crossover, pharmacokinetic (PK) study designed to compare the plasma concentrations of BH4 in subjects with endothelial dysfunction following 14 days of treatment by each of 2 regimens: sapropterin dihydrochloride with vitamin C and sapropterin dihydrochloride alone. Each subject received each regimen; the 2 treatment groups varied only in the sequence of the 2 regimens. The washout period between treatment regimens comprised the 1 day period between the last dose of study drug under the first regimen and the first dose of study drug under the second regimen (Day 14 morning to Day 15 morning).

Conditions

• Endothelial Dysfunction

Interventions

Type	Name	Description
DRUG	Sapropterin Dihydrochloride	Sapropterin Dihydrochloride 5 mg/kg twice a day administered as whole tablets orally within 1 hour after morning and evening meals for 13 days and the last dose within 1 hour after a morning meal on Day 14 and Day 28.
DRUG	Sapropterin Dihydrochloride and Vitamin C	Sapropterin Dihydrochloride 5 mg/kg and 500 mg Vitamin C twice a day administered as whole tablets orally within 1 hour after morning and evening meals for 13 days and the last dose within 1 hour after a morning meal on Day 14 and Day 28.

Timeline

• Start date: 2007-09-01
• Primary completion: 2008-11-01
• Completion: 2009-03-01
• First posted: 2007-09-20
• Last updated: 2021-05-17
• Results posted: 2021-05-17

Locations

1 site across 1 country: United States