Safety and Efficacy of Olesoxime (TRO19622) in 3-25 Years SMA Patients.
Spinal Muscular Atrophy Type IISpinal Muscular Atrophy Type III Non AmbulantAssess the efficacy and the safety of olesoxime in SMA type 2 or type 3 non ambulant patients aged 3-25 years
An Open-label Safety, Tolerability, and Dose-range Finding Study of Nusinersen (ISIS 396443) in...
Spinal Muscular AtrophyThis objectives of this study are to evaluate the safety, tolerability, and pharmacokinetics of a single dose of nusinersen (ISIS 396443) administered intrathecally to participants with Spinal Muscular Atrophy (SMA).
A Pilot Therapeutic Trial Using Hydroxyurea in Type II and Type III Spinal Muscular Atrophy Patients...
Muscular AtrophySpinalThe objectives of this trial are: to establish a safety profile for use of Hydroxyurea in children with Types II and III Spinal Muscular Atrophy; to identify reliable outcome measures for HU treatment in Types II and III SMA; and to detect the clinical efficacy of HU treatment in children with Types II and III SMA.
CARNIVAL Type I: Valproic Acid and Carnitine in Infants With Spinal Muscular Atrophy (SMA) Type...
Spinal Muscular Atrophy Type IThis is a multi-center trial to test safety and evaluate early treatment intervention with valproic acid and carnitine in moderating SMA symptoms of Type I infants.
Valproic Acid in Ambulant Adults With Spinal Muscular Atrophy
Spinal Muscular AtrophyThe primary objective of this proposal is to determine whether oral VPA is effective in treating SMA in adult patients.
Phase II Study of Leuprolide and Testosterone for Men With Kennedy's Disease or Other Motor Neuron...
Spinal Muscular AtrophyAmyotrophic Lateral Sclerosis1 moreOBJECTIVES: I. Evaluate the effects of androgen suppression with leuprolide and androgen replacement with testosterone enanthate on muscle strength in men with Kennedy's disease or other motor neuron disease.
Effects of Standing on Non-Ambulatory Children With Spinal Muscular Atrophy
Spinal Muscular DystrophyNeuromuscular DisabilityNon-ambulatory children with a neuromuscular disability such as spinal muscular atrophy (SMA) are at significant risk for poor bone health as defined by low bone mineral density (BMD) and increased propensity to fracture. Poor bone health is thought to be related, at least in part, to abnormally low levels of load experienced by the skeleton. A common physical approach for increasing bone density is to stimulate the musculoskeletal system by increasing the amount and duration of weight-bearing in the lower extremities. For non-ambulatory individuals, this takes the form of using an assisted standing device to enable the child to spend time in a standing position with some degree of weight placed on the lower limbs. Some of these physical interventions demonstrate variable improvement in BMD in children with neuromuscular conditions, and some do not. A serious limitation in the previous work in this area is a failure to objectively measure the magnitude and duration of the loading experienced by the lower extremities. Thus, a lack of change in BMD may be due to the extremities experiencing only a fraction of the body weight (due to load-sharing with the assistive device) for an inadequate duration of standing time. In order to investigate the efficacy of standing treatment for increasing BMD, the investigators will develop a simple, portable and inexpensive transducer that will measure the magnitude and time course of the load experienced by the lower extremities of individuals with SMA who use a stationary assisted standing device. The specific goal of this proposed project will be to develop, validate and establish the initial feasibility of such a measurement device.
An Open-label Safety, Tolerability and Dose-Range Finding Study of Multiple Doses of Nusinersen...
Spinal Muscular AtrophyThis study will test the safety, tolerability, and pharmacokinetics of escalating doses of nusinersen (ISIS 396443) administered into the spinal fluid either two or three times over the duration of the trial, in participants with spinal muscular atrophy (SMA). Four dose levels will be evaluated sequentially. Each dose level will be studied in a cohort of approximately 8 participants, where all participants will receive active drug.
Safety and Efficacy Study of Pyridostigmine on Patients With Spinal Muscular Atrophy Type 3
Spinal Muscular Atrophy Type 3The purpose of this study is to evaluate safety and efficacy of anti-cholinesterase therapy on the motor function in SMA type 3 patients with impaired neuromuscular junction (NMJ).
A Study for Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in Nusinersen...
Spinal Muscular AtrophyThe primary objective is to evaluate the long-term safety and tolerability of nusinersen (ISIS 396443) administered by intrathecal (IT) injection to participants with Spinal Muscular Atrophy (SMA) who previously participated in investigational studies of nusinersen. The secondary objective is to examine the long-term efficacy of nusinersen administered by IT injection to participants with SMA who previously participated in investigational studies of nusinersen.