Clinical Trials

Date: 2016-08-17

Type of information: Recruitment of the first patient

phase: 1

Announcement: recruitment of the first patient

Company: Audentes Therapeutics (USA - CA)

Product: AT132

Action mechanism:

gene therapy. X-Linked Myotubular Myopathy (XLMTM) is caused by mutations in the MTM1 gene, which encodes a protein called myotubularin. Myotubularin plays an important role in the development, maintenance and function of muscle cells. AT132 is a recombinant serotype 8 adeno-associated virus (AAV8) vector expressing murine myotubularin. Audentes is developing AT132 for the treatment of XLMTM in collaboration with Genethon.

Disease: X-linked myotubular myopathy (XLMTM)

Therapeutic area: Rare diseases - Genetic diseases - Neuromuscular diseases

Country: Canada, France, Germany, UK, USA

Trial details:

INCEPTUS is an international study of boys with XLMTM, ages three years or younger, designed to characterize their individual disease presentation, with a specific focus on respiratory measurements and assessment of muscle strength and function. Patients enrolled in INCEPTUS will be evaluated over a three to twelve-month period prior to their potential participation in ASPIRO. INCEPTUS is designed to serve as a longitudinal baseline and within-patient control for subjects who enroll in the ASPIRO study, while also facilitating the operational aspects of ASPIRO once initiated. (NCT02704273)

Latest news:

• On August 17, 2016, Audentes Therapeutics reported that the first patient has been enrolled in INCEPTUS, a prospective study designed to characterize the disease presentation in children living with X-Linked Myotubular Myopathy (XLMTM). The study is evaluating subjects prior to potential enrollment in ASPIRO, the planned Phase 1 / 2 clinical study intended to evaluate the safety and preliminary efficacy of AT132, the Audentes product candidate for treatment of XLMTM. Audentes plans to file investigational new drug applications for AT132 with North American and European regulatory authorities in the first quarter of 2017. The company expects preliminary data from the ASPIRO study in the fourth quarter of 2017.