Date: 2015-07-20
Type of information: Treatment of the first patient
phase: 1
Announcement: treatment of the first patient
Company: Santhera Pharmaceuticals (Switzerland)
Product: omigapil
Action
mechanism: neuronal apoptosis inhibitor. Omigapil is a deprenyl analog and has anti-apoptotic properties. The compound interacts with the glycolytic enzyme glyceraldehyde 3-phosphate dehydrogenase (GAPDH) which has been implicated in programmed cell death (apoptosis). In preclinical research, Santhera demonstrated that omigapil prevents apoptosis in muscle tissue, ameliorates muscle histology, prevents loss of muscle tissue and increases body weight and survival of disease-relevant animal models for congenital muscular dystrophy. Omigapil is a drug candidate in-licensed from Novartis and repositioned by Santhera for development in congenital muscular dystrophy.
Disease: congenital muscular dystrophy
Therapeutic area: Neuromuscular diseases - Rare diseases - Genetic diseases
Country: USA
Trial
details: The Phase I study (CALLISTO) will evaluate the pharmacokinetic profile, safety and tolerability of oral omigapil in pediatric and adolescent CMD patients and establish the feasibility of conducting disease-relevant clinical assessments for the design of future efficacy trials. A new liquid formulation of omigapil has been developed by Santhera specifically for this patient population. CALLISTO is being conducted at the NIH's National Institute of Neurological Disorders and Stroke (NINDS) in Bethesda, Maryland, and will evaluate use of the compound in 20 ambulatory and non-ambulatory patients aged 5 to 16 years affected by either Ullrich or MDC1A subtypes of CMD. All patients have been selected, pre-screened and randomly assigned to one of the three study cohorts that are starting sequentially with ascending doses of omigapil. Following a 4-week vehicle run-in phase, each patient will receive one of three doses of omigapil (0.02, 0.08 or 0.2 mg/kg/day) for 12 weeks, during which period the pharmacokinetics of omigapil will be assessed as well as the patients' respiratory function, muscle strength and motor function. To ensure the collection of as much information as possible from both subtypes of CMD, Ullrich or MDC1A, all patients have been identified and randomized prior to the first dosing. (NCT01805024)
Latest
news: * On July 20, 2015, Santhera Pharmaceuticals announced that the first patient in the CALLISTO Phase I study assessing the pharmacokinetics, safety and tolerability of oral omigapil in patients with Congenital Muscular Dystrophy (CMD) has been dosed and all participating patients have been recruited. This study, which is being conducted at the US National Institutes of Health (NIH), will also evaluate the feasibility of conducting disease-relevant clinical assessments that could be used as endpoints in future efficacy trials in pediatric and adolescent CMD patients. The first patient received the starting dose of 0.02 mg/kg/day, applied in a new liquid formulation developed by Santhera for this patient population. Only after an independent data and safety monitoring board (DSMB) has assessed the pharmacokinetic and safety data in each completed cohort, dosing in subsequent cohorts will follow at ascending doses. Due to the staggered dosing of patients, the study is expected to run until the end of 2016. * On July 17, 2014, Santhera Pharmaceuticals announced the initiation of a clinical program with omigapil, a drug candidate in-licensed from Novartis and repositioned for therapeutic use in Congenital Muscular Dystrophy (CMD). The clinical development program will be initiated with a Phase I study in pediatric CMD patients, to be conducted at the National Institute of Neurological Disorders and Stroke (NINDS), a component of the US National Institutes of Health (NIH). The program is supported financially in the amount of CHF 1.3 million by EndoStem, an EU 7th Framework Programme, and two patient organizations, the US-based Cure CMD and the Swiss Foundation for Research on Muscle Diseases. Patient enrolment is expected to start in late 2014. The Phase I study (CALLISTO) will evaluate the pharmacokinetic profile, safety and tolerability of oral omigapil in pediatric and adolescent CMD patients and establish the feasibility of conducting disease-relevant clinical assessments for the design of future efficacy trials. A new liquid formulation of omigapil has been developed by Santhera specifically for this patient population. CALLISTO will be conducted at the NIH's National Institute of Neurological Disorders and Stroke (NINDS) in Bethesda, Maryland, and will include 20 ambulatory and non-ambulatory patients aged between 5 and 16 years suffering either from the Ullrich or from MDC1A subtypes of CMD who will be treated for 12 weeks. An independent drug safety monitoring board (DSMB) will monitor patient safety and study progress.
