Studies That Have Completed Recruitment
Trials that are no longer recruiting but remain active to complete other study activities are found here. Click on a disease/condition below to learn more about a range of clinical trials studying new and existing interventions for prevention and treatment of arthritis, musculoskeletal and skin diseases.
For questions, please contact the NIAMS Clinical Trials team at: NIAMSclinicaltrials@mail.nih.gov
Disease / Condition
The study aims to develop a rAAVrh74.MCK.GALGT2 as a surrogate gene therapy that can provide significant clinical benefit to boys affected by Duchenne muscular dystrophy (DMD). The study objective is to perform first-in-human studies demonstrating the safety and expression of the vector following intramuscular injection. The central hypothesis is that following intramuscular injection into the extensor digitorum brevis (EDB) muscle, CT antigen expression will be widely identifiable at the sarcolemmal membrane, and no significant inflammation will be seen. The study's specific aims are to perform a first-in-human safety study of intramuscular gene transfer of rAAVrh74.MCK.GALGT2 and to assess the degree of and the effects of CT antigen expression in EDB muscles.
Herpes zoster (HZ), also known as "shingles", is caused by reactivation and multiplication of the ubiquitous varicella zoster virus (VZV) that remains latent in everyone's sensory neurons following varicella, or "chickenpox". Among individuals who live to age 85, the lifetime risk for HZ is 50%, and more than one in five individuals affected by zoster develop post-herpetic neuralgia, resulting in chronic pain. Other serious complications include encephalitis, permanent vision loss, or more rarely, dissemination and death. Fortunately, a live attenuated vaccine is available and can reduce HZ risk by up to 70%. For patients with rheumatoid arthritis (RA), this vaccine has great potential to provide improved quality of life by reducing the incidence and complications associated with zoster. Due to the underlying disease and/or treatments (e.g. steroids) for rheumatoid arthritis (RA), the risk of herpes zoster in RA patients is approximately double in the general population. This increased risk should make prevention of zoster and vaccination exceedingly important for RA patients.
In light of 1) a substantial elevated HZ risk among RA patients; 2) national data showing most RA patients are not vaccinated for HZ; and 3) the high effectiveness of this vaccine in the general population, the investigators propose to conduct the Varicella zostER VaccinE (VERVE) trial, a randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability, and long-term effectiveness of the live herpes zoster vaccine.
The development of gene therapy has advanced to a point where a cure for Pompe disease can be foreseen. Pompe disease (glycogen storage disease type II; acid maltase deficiency) is a devastating muscle disease resulting from acid alpha-glucosidase (GAA) deficiency in striated and smooth muscle. Despite the availability of enzyme replacement therapy (ERT) with recombinant human (rh) GAA, many patients have poor outcomes including death due to clinically significant anti-GAA antibody response. The limitations of ERT have prompted the preclinical development of gene therapy for Pompe disease. Clinical translation of efficacious gene therapy will greatly advance treatment for Pompe disease by correcting GAA deficiency and suppressing immune responses against rhGAA.
The long-term goal of the study is to develop curative therapy for Pompe disease. Toward this end we have developed gene therapy with an adeno-associated virus (AAV) vector that expresses GAA specifically in the liver accompanied by GAA secretion and receptor-mediated uptake of GAA in the heart and skeletal muscle. It is the central hypothesis that continuous GAA production from a liver depot will surpass the benefits achieved with ERT in Pompe disease.
Systemic sclerosis (SSc) is a multisystem autoimmune illness characterized by vasculopathy, immune system activation and fibrosis of the skin and internal organs. SSc affects approximately 240 people per million in the U.S. but is a disease for which there is no FDA approved medication. Current hypothesis of pathogenesis suggests that a vascular injury with endothelial dysfunction may be an inciting event contributing to immunologic activation and fibrosis in the pathogenesis of the disease. More than 90% of individuals with SSc have vascular complications including Raynaud phenomenon, digital ulcers or gangrene and pulmonary hypertension; with microvascular abnormalities felt to contribute to Raynaud and digital ulcerations.
Statin medications are well-recognized to have pleiotropic effects which may modify all three aspects of SSc pathogenesis. Early diagnosis and treatment of microvascular endothelial dysfunction and Raynaud phenomenon may have the greatest effect in early disease. Thus, this study hypothesizes that treatment with atorvastatin in a well-defined cohort of early diffuse systemic sclerosis will produce beneficial results.
A Double-blinded, Placebo-controlled Pilot Study of Dimethyl Fumarate (DMF) in Pulmonary Arterial Hypertension (PAH) Associated With Systemic Sclerosis (SSc-PAH): The Effect of DMF on Clinical Disease and Biomarkers of Oxidative Stress
Systemic sclerosis (SSc) is a complex, multifactorial autoimmune disease characterized by fibrosis and vasculopathy in skin and various internal organs such as the lungs, kidneys and heart disease with no specific treatment. Pulmonary arterial hypertension (PAH) is a common complication of SSc, and compared to other subgroups of PAH patients, responds poorly to currently approved vasodilators. Oxidative damage is a well-defined component of lung disease in SSc as well as in other organs and pathology studies have shown evidence of oxidative damage in SSc lungs. Dimethyl fumarate (DMF), recently been approved as a treatment for multiple sclerosis, triggers the general response to oxidative damage through Nrf2, resulting in the induction of multiple enzymes designed to counteract the oxidative process. Preclinical studies by the study team as well as other groups have shown that DMF inhibits PAH in murine models. In addition, multiple markers of oxidative stress are elevated in patients with SSc and SSc-PAH, suggesting that this is an important pathway mediating vascular damage in SSc-PAH. On the basis of these studies, the study team proposes a double-blinded, placebo-controlled study of DMF added to stable background, standard of care PAH therapy in SSc‐PAH patients, comparing the change in 6-minute walk distance (6MWD) at 24 weeks to baseline in DMF compared to placebo-treated patients.
Traumatic knee injury is common and highly debilitating. Surgical reconstruction/repair improves knee biomechanics and function, but neuromuscular dysfunction persists for years despite rehabilitation, hindering resumption of normal activities, increasing the risk of further injury and, in a majority of patients, hastening the development of knee osteoarthritis (OA). The period from the injury through the early, post-surgical period is critical for the development of functional deficits, as the trauma of the injury and surgery combine with muscle disuse to reduce skeletal muscle size and intrinsic function.
This study aims to evaluate the utility of neuromuscular electrical stimulation (NMES), initiated following injury and maintained through the early post-surgical period, to prevent muscle atrophy and intrinsic contractile dysfunction.