Clinical Trials in the Spotlight

Actively Recruiting Studies

This page features extramurally funded clinical trials supported by the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS). Click on a disease/condition below to find studies that are actively recruiting. Updates are made to this page as new studies get funded and begin recruitment. Please check back often to find new trials supported by NIAMS.

Please check other clinical trials supported by the NIAMS that have completed recruitment or completed with published results following the links below.
View Active Studies with Completed Recruitment
View Completed Studies with Published Results

For questions, please contact the NIAMS Clinical Trials team at: [email protected]

Disease / Condition

Anterior Cruciate Ligament (ACL) Injury

A Two Arm Non-Inferiority Randomized Clinical Trial Comparing ACL Repair with BEAR Device vs. Autograft Patellar Tendon ACL Reconstruction (BEAR – MOON Trial)

Tissues which live within joints, including the anterior cruciate ligament, rotator cuff tendon, meniscus and labrum fail to heal spontaneously after injury and have high failure rates of surgical repair. The ACL represents a good model to study the problem of intra-articular healing as there are validated preclinical models and clinical outcome measures that make it possible to critically evaluate the success or failure of strategies to enhance tissue healing. The current standard of care for ACL injuries is ACL reconstruction, which is good at stabilizing the knee but requires compromising other uninjured structures around the knee to obtain a graft that is subsequently used to replace the ACL. Further the early posttraumatic OA is not mitigated despite ACL reconstruction. The preclinical studies on ACL repair called BEAR (Bridge-Enhanced ACL Repair) demonstrated a prevention of posttraumatic OA and achieved knee stability. The positive preclinical findings of BEAR compared to ACL reconstruction provided the foundation for two FDA approved preliminary clinical trials: 1) the first-in-human cohort study (“BEAR I”), and 2) a small, single-center randomized control trial (“BEAR II”). The statistical analysis of our pilot data shows a 200 patient trial will be required to demonstrate non-inferiority of BEAR (a novel paradigm changing technology) when compared to ACL reconstruction (current gold standard) for the key outcomes of anterior-posterior (AP) knee laxity and a validated patient reported outcome for knee surgery. Therefore, the study team proposes the BEAR-MOON (Bridge-Enhanced ACL Repair) multi-center randomized non-inferiority clinical trial for co-primary outcomes AP (anterior-posterior) knee laxity and International Knee Documentation Committee (IKDC) validated patient reported outcome measure.

View more about this trial in ClinicalTrials.gov

STABILITY 2: ACL Reconstruction +/- Lateral Tenodesis with Patellar vs. Quad Tendon

Anterior cruciate ligament reconstruction (ACLR) is complicated by high failure rates in young active individuals. The surgical methods to reconstruct the ACL-deficient knee, including graft selection (autograft bone-patellar tendon-bone or quadriceps tendon) and performance of a lateral extra-articular tenodesis may reduce the risk of graft failure in young active individuals. Improved surgical methods to reduce the risk of graft failure will minimize the need for revision ACLR and the associated decreased quality of life and socioeconomic burden that occurs because of ongoing knee instability and the increased risk for post-traumatic osteoarthritis.

View more about this trial in ClinicalTrials.gov

Anti-Phospholipid Syndrome

Certolizumab to Prevent Pregnancy Complications in High-Risk Patients With APS or SLE - (IMPACT Study: IMProve Pregnancy in APS With Certolizumab Therapy)

Antiphospholipid syndrome (APS) is an autoimmune disorder that occurs most commonly in women of reproductive age and is associated with thrombosis and adverse pregnancy outcomes (APOs), such as fetal loss and preterm birth due to severe preeclampsia (PE) or placental insufficiency (PI). Traditional therapy for APS during pregnancy has been a heparin agent and low dose aspirin. However, in PROMISSE, a prospective observational study of 724 patients, 44% of pregnancies in women with APS and LAC resulted in APOs despite treatment with heparin and low dose aspirin.

Based on the observations in PROMISSE, this study hypothesizes that tumor necrosis factor-alpha blockade will significantly decrease the rate of fetal death and preterm delivery due to PE and PI in women with APS and LAC.

View more about this trial in ClinicalTrials.gov

Fibromyalgia

Mechanisms and Modification of Pain Modulatory Capacity

Chronic pain affects over 100 million Americans, with an annual cost estimated at over $500 billion. Reliance on opioid medications has led to an “opioid crisis” and the need to identify alternative treatment for chronic pain. This study hypothesizes that chronic pain represents failure or suboptimal function of pain modulatory capacity (PMC) and aims to test hypotheses about resilience to clinically relevant pain challenges, and test multisystem (psychological, behavioral, and neural) mechanisms underlying chronic pain. Furthermore, the study aims to demonstrate the trainability of PMC in asymptomatic controls, which has relevance for pain prevention, and in patients with fibromyalgia (FM) who are hypothesized to have suboptimal PMC (resilience) to pain challenges.

View more about this trial in ClinicalTrials.gov

Gout

INvestigations In Gout, Hyperuricemia, and comorbidiTies (INSIGHT) Center of Research Translation (CORT) - Improving Minority Gout Care and Clinical Research Participation in the Southeast

Gout impacts around 4% of the U.S. adult population and is the most common form of inflammatory arthritis in men. The incidence of gout is increasing worldwide and may be as high as 9% in the elderly. Gout is a significant burden on the healthcare system and is associated with both decreased work productivity and quality of life. With the ever-increasing impact of gout and its associated comorbidities on the population, investigation of novel translational mechanisms mediating gout flares as well as approaches to improving gout and hyperuricemia outcomes comprise an unmet and urgent medical need. This multi-disciplinary INvestigationS In Gout, Hyperuricemia, and comorbidiTies (INSIGHT) Center of Research Translation (CORT) includes 4 research projects and an Administrative Core focused on the theme, “Gout, Hyperuricemia, and Associated Comorbidities”.

View more about this trial in ClinicalTrials.gov

Isolated Skin Vasculitis

A Randomized Multicenter Study for Isolated Skin Vasculitis

Vasculitides are uncommon diseases which can affect almost any organ. Vasculitis frequently involves the skin, as an isolated process, or as part of systemic vasculitis. Skin vasculitis represents an important source of morbidity and symptomatology as well as an opportunity for early diagnosis and treatment. Despite the important place of isolated skin vasculitis in this group of diseases, there has historically been limited emphasis placed on understanding its pathogenic mechanisms and determining its best treatment. Whereas episodes of isolated small vessel vasculitis in the skin are often self-limited, resolve over 3-4 weeks with residual hyperpigmentation, and do not recur, up to one-third of patients have persistent or recurrent disease for up to several years. The cutaneous vasculitic lesions can be itchy, painful, and cosmetically disturbing. It can ulcerate, and then be complicated by infection and scarring. In around 8-10% of patients, chronic or recurrent small vessel vasculitis can develop. In general, systemic involvement (if found) is minimal and limited to mild arthralgias, myalgias, fatigue, low-grade fever or peripheral edema. No therapy has been proven to shorten the duration of disease or decrease its severity and the frequency of the flares.  Initial long-term treatment options mainly include colchicine, dapsone, or azathioprine.

This study proposes to compare the efficacy of three of the drugs among the most commonly used ones — colchicine, dapsone, and azathioprine — for the treatment of isolated skin vasculitis, in a multi-center sequential multiple assignment randomized trial.

View more about this trial in ClinicalTrials.gov

Knee Osteoarthritis

The Relation of Altered Pain Processing to Impact Loading and Response to a Gait Retraining Treatment in Knee Osteoarthritis

Knee Osteoarthritis (OA) is a major public health problem, causing substantial pain and functional limitations and effects ~12% of older adults. There are a few effective non-invasive treatments to prevent disease progression or decrease pain. The current proposal will provide evidence-based information in order to design novel and effective rehabilitation treatments for knee osteoarthritis.

View more about this trial at ClinicalTrials.gov

A Sequenced-Strategy for Improving Outcomes in Patients with Knee Osteoarthritis Pain

Knee osteoarthritis (KOA) is one of the leading causes of chronic pain and disability worldwide, affecting over 30% of older adults and represents a major global health and economic burden to individuals and society. The rates of KOA have more than doubled in the past 70 years and continue to grow sharply, given increases in life expectancy and population BMI. Surgery is often employed to treat KOA, but it is associated with a high rate of persistent pain and is not a permanent solution. Numerous nonsurgical therapies have been advocated to treat pain in patients with KOA. However, stand-alone conservative treatments including non-opioid medications and joint injections provide only limited pain relief and functional improvement in a subset of knee OA sufferers. This has led to a high rate of opioid use in this population. The overarching goal of this proposal is to conduct a sequential parallel group randomized controlled trial (RCT) to rigorously evaluate the comparative-effectiveness of conservative behavioral and non-opioid pharmacological treatments (Phase I) and, among non-responders, the benefits of nonsurgical procedural interventions (Phase II) in three interrelated Aims.

View more about this trial in ClinicalTrials.gov

Osteogenesis Imperfecta

Rare Diseases Clinical Research Network Brittle Bone Disease Consortium Longitudinal Study of Osteogenesis Imperfecta

Osteogenesis Imperfecta (OI) is a rare disorder that causes bones to break easily. People with OI may have broken bones with little or no trauma, dentinogenesis imperfecta (DI), and, in adult years, hearing loss. The purpose of this natural history study is to perform a long-term follow-up of a large group of people with osteogenesis imperfecta (OI) to improve the health and quality of life of people with OI.  The study team will collect information on medical history, number of broken bones, surgeries done, medications taken, ability to walk, pain, lung function and breathing, hearing, and bone mineral density. This study aims to perform DNA testing and collect natural history data on all individuals enrolled in this longitudinal study, see how often people with type I OI have vertebral compression fractures of the spine, follow people with all forms of OI to see how often they develop scoliosis (curvature of the spine), and look at dental health in people with OI.

View more about this trial in ClinicalTrials.gov

Recessive dystrophic epidermolysis bullosa (RDEB)

Intravenous Gentamicin Therapy for Recessive Dystrophic Epidermolysis Bullosa (RDEB)

Recessive dystrophic epidermolysis bullosa (RDEB) is an incurable and inherited mechano-bullous disease of the skin characterized by skin fragility, blister formation, and chronic wounds. RDEB is caused by mutations in the COL7A1 gene encoding type VII collagen (C7), the major component of anchoring fibrils (AFs) that anchor the epidermis to the dermis. AFs are attenuated, diminutive, or absent in RDEB. There is no effective treatment for RDEB except palliative measures and supportive wound care.  Based on encouraging in vitro and in vivo RDEB data, the proposed study will assess the safety and efficacy of IV gentamicin in RDEB patients with nonsense mutations.

View more about this trial in ClinicalTrials.gov

Sedentariness

Molecular Transducers of Physical Activity Consortium (MoTrPAC)

Physical inactivity is a major public health challenge underlying a broad range of health problems at all ages. While physical activity (PA) has shown to produce relevant health benefits, the underlying molecular mechanisms are poorly known. The coordinated effort of clinical and animal studies supported by bioinformatics and chemical analyses will achieve the Molecular Transducers of Physical Activity Consortium (MoTrPAC) goals of assessing the molecular changes that occur in response to PA. The Consortium Coordinating Center (CCC) for the MoTrPAC will provide support for the organization, administration, planning, standardization, documentation, monitoring and reporting activities relating to the MoTrPAC. The CCC will play a pivotal role in ensuring the cohesion of the MoTrPAC by enhancing communication and integration across all study components, including the Clinical Sites, the Preclinical Animal Study Sites, the Bioinformatics Center, the Chemical Analysis Sites, and the various study committees. The CCC will develop strategies and strategic planning processes by integrating activities of the MoTrPAC investigators and facilitate interactions and communications with junior and senior investigators outside the consortium to maximize the use of the MoTrPAC resources toward achieving the overall research goals. To accomplish these goals and maximize the progress and productivity of the MoTrPAC, the CCC will promote team science, team leadership, and innovative leadership approaches across all study components. Strategic planning that follows the principles of the dynamic theory of strategy will be fostered to evaluate alternative approaches, maintain the cutting-edge scientific focus, leverage state-of-the-art coordination technologies, anticipate challenges, and maximize future opportunities to ensure the success of the consortium.

View more about this trial in ClinicalTrials.gov

Systemic Lupus Erythematosus (SLE)

Lupus Intervention for Fatigue Trial

Systemic lupus erythematosus (SLE, lupus) is a systemic autoimmune disease characterized by pronounced inflammation that affects up to 1.5 million people in the US. While excess mortality has decreased in SLE patients since the 1970s, a major ongoing cause of morbidity in this population is chronic, debilitating fatigue that significantly decreases quality of life, and increases risk of work disability and associated health care costs. An urgent, unmet need in the management of patients with SLE is identification of effective strategies to reduce fatigue.

Preliminary data from lupus patients showed that regular aerobic exercise can improve quality of life and reduce fatigue. In considering other lifestyle behaviors, there is limited literature on effects of diet, energy balance, and/or nutrient density in SLE patients, but diet intervention has been favorably associated with managing fatigue in other disorders (e.g., cardiovascular disease and age-related functional decline). To study the effects of intervening on these two potential modifiable lifestyle behaviors (PA and diet) this study designed the Lupus Intervention Fatigue Trial (LIFT) to compare the effectiveness of a motivational interviewing program intervention versus a patient educational program control to reduce fatigue in persons with lupus.

View more about this trial in ClinicalTrials.gov

Ulnar Neuropathy

Clinical Trial for Surgery of the Ulnar Nerve (SUN) at the Elbow

Ulnar neuropathy at the elbow (UNE) is a common compressive neuropathy condition that can cause pain, tingling , and muscle loss in hand. If left untreated, it can lead to disability and job loss. Conservative treatment fails in 60% of cases, and most patients require surgery, which is often recommended to eliminate symptoms, but controversy xists over the best procedure. The two most common surgical procedures are in-situ (simple) decompression and subcutaneous anterior transposition. This study plans to conduct a multicenter randomized clinical trial to compare outcomes for two commonly used surgical procedures and develop an optimal surgical plan for individual patients.

View more about this trial in ClinicalTrials.gov

Vasculitis

Low Dose Naltrexone to Improve Physical Health in Patients With Vasculitis

Symptoms clearly attributable to inflammatory disease are controllable in the great majority of patients with vasculitis. However, most patients do not feel healthy despite effective immune-suppressive treatment. Among the residual symptoms that disrupt the lives of patients with vasculitis, fatigue, poor physical function, sleep disturbance, and satisfaction with social roles are the most common and have the greatest negative impact on quality of life. Although there are multiple potential sources of these symptoms – residual autoimmune/inflammatory disease, medication side effects, co-morbidities such as obstructive sleep apnea or depression – the cause(s) often defy identification and successful management in the individual patient. Chronic pain is also high on the list of important negative influences on quality of life of patients with vasculitis.

Naltrexone is an opioid antagonist that is used in high doses for emergency treatment of opioid overdose and is FDA approved in an oral daily dose of 50 mg to prevent recidivism in alcoholics. At much lower doses of 4 – 4.5 mg daily, however, it has been shown in small, blinded, randomized trials to improve pain in fibromyalgia, gastrointestinal symptoms in Crohn’s disease, and quality of life in patients with multiple sclerosis.  The mechanism is unclear, with proposals including not only modulation of central pain-processing pathways but also mitigation of inflammatory roles of microglia. In addition to blocking opioid receptors, naltrexone blocks TLR-4, and some studies suggest that the anti-inflammatory effects of low dose naltrexone are independent of opioid receptors.

This multi-center, randomized, double-blinded, cross-over, placebo-controlled trial aims to evaluate the efficacy of low-dose naltrexone (LDN) at 4.5 mg nightly in improving self-reported physical health in patients with vasculitis.

View more about this trial in ClinicalTrials.gov

Venous Leg Ulcers (VLU)

Developing Strategies for Effective Debridement in Patients for Venous Leg Ulcers

Chronic non-healing venous leg ulcers (VLUs) have an intrinsic healing impairment that is associated with dysfunctional gene expression patterns. The previous study has shown that tissue from the non-healing edge of chronic VLUs exhibits characteristic histopathology including epidermal hyperproliferation, dermal fibrosis, accumulation of intracellular pro-collagen and dysregulation of genes involved in epidermal differentiation, migration and proliferation. It also has demonstrated that biopsies taken from the non-healing edges of VLUs before and after debridement have distinct morphologies and distinguishable gene expression patterns, providing the biological basis and justification of debridement. These observations are supported by the findings that primary cells grown from tissue biopsies before and after debridement also have distinct and typical genomic patterns, with cells from pre-debridement edge biopsies exhibiting a non-healing phenotype as evidenced by loss of migration and loss of ability to respond to growth factor stimuli. Moreover, using a genomic approach in a clinical trial to determine mechanism of action of cell-based therapy in patients with VLU, the study team has identified a specific set of genes responsible for therapeutic reprogramming that shifts non-healing ulcer into acute wound-like healing VLU.

The goal of this project is to use genomic profiling, candidate genes and proteins to develop guided surgical debridement to improve healing in chronic non-healing VLUs and to test the efficacy of this approach.

View more about this trial in ClinicalTrials.gov

X-Linked Hypophosphatemia

Calcitriol Monotherapy for X-Linked Hypophosphatemia

X-linked hypophosphatemia (XLH) is characterized by increased FGF23, which impairs activation of vitamin D and promotes renal phosphate wasting leading to osteomalacia and rickets. Current treatment using 1,25- dihydroxyvitamin D(calcitriol) and phosphate is often complicated by hypercalcemia and nephrocalcinosis and does not always prevent hyperparathyroidism. Furthermore, it does not normalize growth. Thus, the study team undertook a pre-clinical study in the Hyp mouse model of XLH, to compare the effects of calcitriol alone vs treatment with FGF23 blocking antibodies on growth, serum and urine mineral ions as well as histological, histomorphometric, microarchitectural and biomechanical properties of bones. These studies revealed that calcitriol monotherapy improves growth, prevents rickets and improves the microarchitectural and biomechanical properties of bone without phosphate supplementation. The beneficial effects of calcitriol were superior to those of the FGF23 blocking antibody employed, perhaps because, as in humans, FGF23 blocking antibodies were not able to sustain increased levels of 1,25-dihydroxyvitamin D. It is notable that the beneficial effects of calcitriol occur in spite of a significant increase in circulating FGF23 and bone FGF23 mRNA expression.

View more about this trial in ClinicalTrials.gov

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