Date: 2015-02-13
Type of information: Submission of a clinical trial application
phase: 3
Announcement: Submission of a clinical trial application
Company: Horizon Pharma (Ireland)
Product: Actimmune® (interferon gamma-1b)
Action
mechanism: protein. Actimmune® is a synthetic version of interferon gamma-1b, a naturally occurring biologic response modifier. The drug is already approved in the US to decrease the number and severity of infections in patients with chronic granulomatous disease and to delay the progression of severe, malignant osteopetrosis, a genetic disorder that affects normal bone formation.
Disease: Friedreich\'s ataxia
Therapeutic area: Rare diseases - Genetic diseases - Neurodegenerative diseases
Country: USA
Trial details:
Latest
news: * On February 13, 2015, Horizon Pharma announced it has submitted an Investigational New Drug (IND) application to the FDA for Actimmune® (interferon gamma-1b) in the treatment of Friedreich\'s Ataxia (FA). The Company has simultaneously requested Fast Track Designation. The Company plans to begin a Phase 3 study in the second quarter in collaboration with the Friedreich\'s Ataxia Research Alliance (FARA) and the investigators and clinics of FARA\'s Collaborative Clinical Research Network (CCRN) in FA. The Phase 3 study is expected to be a randomized, double-blind, multicenter, placebo-controlled, 26-week study evaluating Actimmune® in approximately 90 male and non-pregnant female subjects between the ages of 10 and 25 years, inclusive, with an FA functional stage of greater than 1 to less than 5 and the ability to walk 25 feet with or without an assistive device. Subjects will be randomized 1:1 to receive either Actimmune® or matching placebo. The primary endpoint will be the change in the Friedreich\'s Ataxia Rating Scale-modified neurological exam score (FARS-mNeuro), which is a measure of disease activity and correlates significantly with functional disability, from baseline to 26 weeks for patients treated with Actimmune® compared to placebo. It is anticipated that it will take 18 months to completely enroll the study. A six-month open-label extension study will be offered to those FA patients completing the Phase 3 study.
