6 Clinical Trials for Various Conditions
This proposal seeks to determine whether near infrared spectroscopy (NIRS) can differentiate between patients with confirmed SAMS and those with non-specific muscle complaints. NIRS is a non-invasive technique of assessing skeletal muscle tissue oxygenation and mitochondrial function. Mitochondrial dysfunction is a possible cause of SAMS, but NIRS has never been evaluated as a diagnostic tool for SAMS. Investigators will enroll 40 patients with a history of SAMS in an 8 wk randomized, double-blind crossover trial of simvastatin 20 mg/d and placebo separated by a 4 wk washout phase. Tissue oxygenation will be measured using NIRS during a short handgrip exercise protocol before and after each treatment period. Investigators will query patients about muscle complaints weekly during both phases of the study with a validated survey to assess muscle pain. Investigators will classify patients as testing positive for SAMS if they report pain on simvastatin and not placebo. Investigators hypothesize that these patients, vs. patients experiencing pain on both treatments, placebo, or neither treatment, will be distinguished by reduced tissue oxygenation during simvastatin treatment relative to placebo, demonstrating efficacy of NIRS as a clinical tool that can be eventually used for the diagnosis and ultimately treatment of SAMS.
This is a single arm Phase 2 trial to evaluate the efficacy and safety of RP-A501, a recombinant adeno-associated virus serotype 9 (AAV9) containing the human lysosome-associated membrane protein 2 isoform B (LAMP2B) transgene, in male patients with Danon Disease.
This is a non-randomized open-label Phase 1 study to evaluate the safety and toxicity of gene therapy using a recombinant adeno-associated virus serotype 9 (AAV9) containing the human lysosome-associated membrane protein 2 isoform B (LAMP2B) transgene (investigational product (IP), RP-A501) in male patients with Danon Disease (DD).
People with heart failure often have weakness in their leg muscles. This study will determine whether the leg weakness is due to very high adrenaline levels and whether the medication clonidine can improve leg weakness.
The goal of this proposal is two-fold: (1) to further develop and validate a technology, magnetic resonance elastography (MRE), for quantitatively imaging mechanical properties and tension distribution in muscle and (2) to apply the technique for in vivo evaluation of patients with four common, and clinically significant muscle disorders (spasticity, disuse atrophy, myofascial pain and a metabolic myopathy). These studies will employ a magnetic resonance imaging sequence with synchronous motion-sensitizing gradients to map propagating shear waves in the muscle. The technique will assess the mechanical properties of the muscle and its tension distribution. Specifically, the study can be divided into three specific aims. Aim 1: Optimize MRE methods of acquisition and analysis for the assessment of muscle, including electromechanical drivers, data acquisition techniques, and methods for image analysis. Advanced techniques for very rapid MRE assessment of muscle will continue to be developed. Aim 2: Validate the MRE assessment of muscle properties and tension with phantom, ex-vivo muscle, and Finite Element Modeling (FEM) techniques. Finite Element Analysis will be performed by using both phantom and bovine muscles to better correlate MRE wave-length findings as function of muscle properties, tension and fiber architecture. Aim 3: Study In Vivo Normal and Abnormal Muscle. The MRE technique will be applied in vivo to provide elastographic images of abnormal muscle with known disorders. The patient groups chosen for study are each important in their own right, and furnish unique information across the spectrum of muscular disease and dysfunction. Groups to be studied include individuals with new onset of spasticity following an ischemic, hemispheric stroke, disuse atrophy as a result of immobilization, metabolic (hyperthyroid) myopathy and myofascial pain for trigger point identification. The overall hypothesis of this work is that will bring benefits to both basic research and clinical care.
This study is researching an experimental drug called andecaliximab. The study will include pediatric and adult patients with fibrodysplasia ossificans progressiva (FOP). The study will evaluate how safe and effective andecaliximab is in patients with FOP. The study is looking at several research questions, including: * Safety of andecaliximab in participants with FOP * Whether andecaliximab reduces the number of new heterotopic bone lesions (Heterotopic Ossification; HO) * Whether andecaliximab reduces the number or severity of flare-ups * Pharmacokinetics/pharmacodynamics (PK/PD): How much study drug is in your blood at different times and its impact on blood biomarker(s) * Whether the body makes antibodies against the study drug (which could make the drug less effective or could lead to side effects)