NCT00789568

A Phase 1 Study to Evaluate Effects of Sapropterin Dihydrochloride on QTc Intervals in Healthy Adult Subjects

A Phase 1, Randomized, Placebo- and Active-controlled Crossover Study to Evaluate the Effects of Sapropterin Dihydrochloride Oral Administration on QTc Intervals in Healthy Adult Subjects

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 56 (actual)
• Sponsor: BioMarin Pharmaceutical · Industry
• Sex: All
• Age: 18 Years – 50 Years
• Healthy volunteers: Accepted

Summary

Sapropterin dihydrochloride (subsequently referred to as sapropterin) (Kuvan®) was approved by the FDA for the treatment of hyperphenylalaninemia in 2007. Preclinical and clinical studies and post-marketing surveillance have not demonstrated any specific cardiovascular concerns with sapropterin (Kuvan®). Nonetheless, nonantiarrhythmic drugs may have the potential to prolong QT interval, leading to potentially fatal ventricular tachycardias, including torsades de pointes. As part of the post-marketing commitment, a thorough QT/QTc study will be conducted according to ICH guidelines.

Conditions

• Phenylketonuria

Interventions

Type	Name	Description
DRUG	sapropterin dihydrochloride	20 mg/kg and 100 mg/kg
DRUG	Moxifloxacin	Moxifloxacin is included as a positive control to demonstrate the assay sensitivity based on the expected increased QTc response.
DRUG	Moxifloxacin placebo	Moxifloxacin placebo tablet

Timeline

• Start date: 2008-10-01
• Primary completion: 2009-01-01
• Completion: 2009-10-01
• First posted: 2008-11-13
• Last updated: 2021-07-23

Locations

1 site across 1 country: United States