NCT02472535

Study to Evaluate the Effects of MBX-8025 in Patients With HoFH

A 12-week, Open-label, Dose-escalating, Phase 2 Study to Evaluate the Effects of MBX-8025 in Patients With Homozygous Familial Hypercholesterolemia (HoFH)

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 13 (actual)
• Sponsor: Gilead Sciences · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

A 12-week, open-label, dose-escalating, phase 2 study to evaluate the effects of MBX-8025 in patients with Homozygous Familial Hypercholesterolemia (HoFH).

Detailed description

Open-label, single arm, non-controlled, dose ascending (50 mg/day, 100 mg/day and 200 mg/day) with three consecutive dose escalation periods. After signing an informed consent subject will enter a screening period and a run-in stabilization period. At the end of run-in period patients will enter treatment phase. MBX-8025 in ascending doses (50 mg, 100 mg, and 200 mg) will be given within three consecutive 4 weeks periods, for a total of 12 weeks. At the end of treatment, subjects will enter a follow-up period.

Conditions

• Homozygous Familial Hypercholesterolemia

Interventions

Type	Name	Description
OTHER	Run-In Period: Placebo	2 capsules, once a day for two weeks
DRUG	MBX-8025 50 mg (Dose Escalation Period 1)	1 capsule once a day for 4 weeks (MBX-8025 50 mg capsule)
DRUG	MBX-8025 50 mg or 100 mg (Dose Escalation Period 2)	1 capsule once a day for 4 weeks (MBX-8025 50 mg or 100 mg capsule)
DRUG	MBX-8025 50 mg, 100 mg or 200 mg (Dose Escalation Period 3)	1 or 2 capsules once a day for 4 weeks (MBX-8025 50 mg, 100 mg or two (2) 100 mg capsules)

Timeline

• Start date: 2015-04-01
• Primary completion: 2016-02-01
• Completion: 2016-02-01
• First posted: 2015-06-16
• Last updated: 2016-03-09

Locations

5 sites across 4 countries: Canada, France, Netherlands, Norway