100 Clinical Trials for Various Conditions
The purpose of this study is to measure the safety, tolerability, PK, and PD of AZD5492 administered subcutaneously in adult participants with SLE or IIM. Study details include: • The study duration will be a minimum of 180 days in addition to the screening period. Additional follow-up visits may be required up to 12 months from study start. * Depending on the study part they are assigned to, participants will be administered AZD5492 once (Part 1) or twice (Part 2). * Study visits will occur at: Screening, Days 1-4, 8, 15, 22, 30, 60, 90, 120, 150, and 180 in Part 1, Screening, Days 1-4, 8-11, 15, 22, 29, 43, 60, 90, 120, 150, and 180 in Part 2.
A Phase 2, randomized, open-label, controlled study to evaluate the efficacy and safety of rapcabtagene autoleucel versus comparator in participants with severe refractory idiopathic inflammatory myopathies (IIM)
The purpose of this study is to determine the safety and tolerability of imvotamab in patients with Moderate-Severe Idiopathic Inflammatory Myopathies who have failed prior therapies. Participants will be given imvotamab through a vein (i.e., intravenously).
To evaluate the efficacy of Pozelimab/Cemdisiran combination therapy in patients with sIBM
RESET-Myositis: Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Subjects with Active Idiopathic Inflammatory Myopathy or Juvenile Idiopathic Inflammatory Myopathy
The purpose of this study is to measure the long-term safety and tolerability of efgartigimod PH20 SC in adult participants with IIM who previously participated in ARGX-113-2007. Secondary objectives include efficacy measures of efgartigimod PH20 SC in participants with IIM.
The purpose of the study is to understand how the study medicine PF-06823859 works in people with idiopathic inflammatory myopathies (DM and PM). These disorders cause inflammation that weakens the muscles that are important for movement and may also cause skin rash in people with DM. This study is seeking participants who: * Are 18 years of age or older or minimum legal adult age as defined per local regulation, whichever is greater * Have active DM or active PM. * Are receiving a stable dose of 1 corticosteroid taken by mouth and/or 1 traditional immunosuppressant. * Note: Corticosteroids and immunosuppressants are medicines that help reduce inflammation and may signal to the immune system not to attack the body. Dermatomyositis (DM) is a rare disease that causes muscle inflammation that results in muscle weakness and low muscle stamina. Patients with DM have a characteristic skin rash. Polymyositis (PM) is a rare disease that involves mainly muscle inflammation resulting in muscle weakness, that can sometimes be painful. Patients with DM and PM may have trouble going up the steps, walking or getting to a standing position. Some of the participants will receive the study medicine (PF-06823859) and some will receive placebo (which is similar to study medicine but contains no medicine in it). The study medicine or placebo will be given as an intravenous (IV) infusion (directly into the veins), which takes about1 hour; every 4 weeks from Day 1 to Week 48 of the study. Both PF-06823859 and placebo and will be given at the study site. The study will compare the experiences of people receiving study medication to those of the people who do not. This will help to see if PF-06823859 is safe and effective. Participants will take part in this study for about 13 months. During this time, participants will have 15 study visits. These visits will be performed at the study site.
The primary efficacy objective: To evaluate the effect of daxdilimab compared with placebo in reducing disease activity at Week 24. The secondary efficacy objectives include: 1. To evaluate the effect of daxdilimab compared with placebo in reducing disease activity at Week 24. 2. To evaluate the effect of daxdilimab compared with placebo on skin symptoms at Week 24. 3. To evaluate the effect of daxdilimab on decreasing the use of corticosteroid at Week 24. Other secondary objectives include: 1. To characterize the pharmacokinetics (PK) and immunogenicity of daxdilimab in participants. 2. To evaluate the safety and tolerability of daxdilimab in participants.
This study's purpose is to measure the treatment response from efgartigimod PH20 SC compared with placebo in participants with Idiopathic Inflammatory Myopathy (IIM). Participants with the IIM subtypes of dermatomyositis (DM), immune-mediated necrotizing myopathy (IMNM), or certain other subtypes of polymyositis (PM; including antisynthetase syndrome \[ASyS\]) will be included in the study. Treatment response will be measured by Total improvement score (TIS). Additional information can be found on https://myositis-study.com/.
The purpose of this study is to evaluate the efficacy and safety of Nipocalimab versus placebo in participants with active idiopathic inflammatory myopathies (IIM).
Trial to Evaluate the Efficacy and Safety of Abatacept subcutaneous (SC) in Combination With Standard Therapy Compared to Standard Therapy Alone in Improving Disease Activity in Adults With Active Idiopathic Inflammatory Myopathy
The goal of the trial is to evaluate the efficacy and safety of belimumab as a maintenance therapy in adults with refractory Idiopathic inflammatory myositis (IIM) as compared with standard of care. This is a multicentre double-blind, placebo-controlled trial.
This study of inflammatory muscle diseases-polymyositis and dermatomyositis and related disorders-will examine what causes these diseases and describe the clinical features (signs and symptoms) associated with them. Inflammation and degeneration of skeletal muscles in these disorders leads to weakness and muscle wasting. The skin, lungs and other organs may also be involved. Patients 16 years of age and older with polymyositis, dermatomyositis, or a related disorder may be eligible for this study. Participants will undergo a complete history and physical examination, including routine blood and urine tests. Additional procedures for diagnosis, treatment or research may include: 1. Blood sample for genetic studies. 2. Muscle biopsy-removal of a tissue sample for microscopic examination. Under local anesthetic, a 1/2- to 1-inch long incision is made in the thigh or upper arm, and a small piece of muscle is removed. 3. Electromyography-measurement of the electrical activity of a muscle. A needle is inserted through the skin into a muscle to record its electrical activity. 4. Magnetic resonance imaging-visualization of organs or tissues, using a magnetic field and radio waves. The patient lies on a table inside a narrow cylinder (the MRI scanner) with a strong magnetic field for the scanning. 5. Manual muscle strength testing by a physiotherapist. 6. Swallowing studies using ultrasound (imaging using sound waves) and X-rays (barium swallow) to evaluate swallowing and speaking abilities. 7. Questionnaires on swallowing ability and ability to perform daily living activities 8. Pulmonary function tests-measurement of movement of air in and out of the lungs. The patient breathes into a machine to evaluate lung function. 9. Chest X-rays to evaluate lung function. 10. Electrocardiogram and, if necessary, Holter monitoring (measurement of the electrical activity of the heart) and echocardiogram (ultrasound imaging of the heart) to evaluate heart function. 11. Apheresis-collection of white blood cells for research. Whole blood is collected through a needle placed in an arm vein. The blood circulates through a machine that separates it into its components. The white cells are removed and the rest of the blood is returned to the body through the same needle or through a second one placed in the other arm. 12. MR guided muscle biopsy-measurement of glycogen in muscle tissue using magnetic resonance imaging. Certain patients may undergo this experimental procedure to compare MRI findings with those of muscle biopsy. The affected muscles are identified using MRI and the biopsy incision is made. MRI is then used to guide the biopsy needle to the muscle and a small piece is removed. Patients who are eligible for experimental treatment studies will be offered the opportunity to join them. Others will be advised of treatment recommendations.
The purpose of this study is to understand how the study medicine, dazukibart, works in people with active idiopathic inflammatory myopathies (dermatomyositis \[DM\] or polymyositis \[PM\]). Idiopathic inflammatory myopathies are a group of disorders that show inflammation of the muscles used for movement. There are several types of idiopathic inflammatory myopathies, including DM and PM. DM and PM involve weakness of the muscles closest to the center of the body, such as the muscles of the hips, thighs, upper arms, and neck. People with these forms of idiopathic inflammatory myopathies may find it difficult to climb stairs, get up from a seated position, or lift items above their head. People with DM can also have a skin rash. These disorders negatively impact the quality of life and functioning of patients. In addition to the above, these disorders can affect how the lungs and heart work. This study is seeking participants who took part in a DM and PM study with dazukibart before. Some participants will receive study medicine, and some participants will not receive study medicine and only complete safety follow-up. The study medicine will be given as an intravenous (IV) infusion (directly into the veins). This takes about 1 hour, every 4 weeks, from Day 1 to Week 48 (about 12 months) of the study. This will be followed by a safety follow-up period that lasts about 4 months after the last infusion. Participants who receive study medicine will have about 18 study visits at the site over about 16 months. There will also be participants enrolled in this study who will not receive study medicine. Such participants will only take part in safety follow-up visits as they do not want to or are not eligible to receive dazukibart. These participants will not receive study medicine and will have up to 4 study visits at the site every 4 weeks to complete safety follow-up.
The purpose of this multicenter, randomized, placebo-controlled and double-blind study is to evaluate the efficacy and safety of subcutaneous anifrolumab compared with placebo on the overall disease activity in participants with moderate to severe Idiopathic Inflammatory Myopathies (IIM) \[polymyositis (PM) or dermatomyositis (DM)\] while receiving standard of care (SoC) treatment.
A Phase II/III Randomized, Double-blind, Placebo-controlled, Multicenter Study to Determine the Efficacy and Safety of ABC008 in the Treatment of Subjects with Inclusion Body Myositis
1. To assess changes in impedance parameters in Idiopathic Inflammatory Myopathies (IIMs). 2. To assess whether EIM parameters are reflective of disease severity, based on clinical outcome measures of IIMs.
Prospective, Double-blind, Randomized, Placebo-Controlled Phase III Study Evaluating Efficacy and Safety of Octagam 10% in Patients With Dermatomyositis ("ProDERM study")
Myositis is a disease, believed to be due to immune cells, cells which normally protect the body, but are now attacking the muscles and other organ systems within body. As a result, the affected muscles and organs fail to work properly causing weakness, difficulty swallowing, skin rash, respiratory problems, heart problems, joint stiffness, soft tissue calcification and vasculitis (blood circulation problems). The likelihood of progression of this disease is high. This study is designed to examine whether treating patients with high dose cyclophosphamide (a drug which reduces the function of the immune system) and ATG (a protein that kills the immune cells that are thought to be causing this disease), followed by return of previously collected blood stem cells will stop the progression of myositis.
This study will examine the abnormal immune response in patients with sporadic inclusion body myositis (s-IBM)-the most common inflammatory muscle disease in people over the age of 50. s-IBM progresses steadily and may lead to severe weakness and wasting of arm and leg muscles. Patients may become unable to perform daily living activities and be confined to wheelchairs. s-IBM is thought to be an autoimmune disease, in which the body's own immune system attacks healthy muscles. This study will explore the causes of the muscle tissue inflammation that is responsible for destruction of muscle fibers and weakness in this disease. Information from the study may help in the development of an effective treatment for this disease. Patients with s-IBM may be eligible for this study. Those who are unable to travel or who have severe cardiovascular, renal or other end-stage organ disease will be excluded. Candidates will be screened for eligibility with a medical history and physical and neurological examinations. Participants will be seen at the NIH Clinical Center every six months over a 12-month period (visits at enrollment, 6 months and 12 months) either on an inpatient or outpatient basis, depending on their disease severity. Each 2- to 3-day visit will involve the following tests and evaluations: * Blood samples for routine laboratory tests are collected at every visit. Additional blood for research studies is collected at 12 months. * Quantitative muscle strength testing is done at every visit. The patient pulls against straps connected to dynamometers (devices that measure muscle power) to evaluate strength of the main muscle groups in the arms and legs. * Lymphapheresis is done at enrollment and at 12 months. This is a procedure for collecting quantities of lymphocytes (white blood cells that are an important part of the immune system). Blood is collected through a needle placed in an arm vein and circulated through a machine that spins it, separating it into its components. The lymphocytes are removed and the rest of the blood (red cells, platelets and plasma) is returned to the body through the same needle or another needle placed in the other arm. * Electrophysiologic studies (electromyography and nerve conduction testing) are done at enrollment and 12 months. Electromyography evaluates the electrical activity of muscles. A small needle is inserted into the muscle and the patient is asked to relax or to contract the muscle. For nerve conduction testing, nerves are stimulated by electrodes (small wires taped to the skin over the muscle). * Muscle biopsy is done at enrollment and 12 months. A sample of muscle tissue (about the size of a lima bean) from an arm or leg is surgically removed to confirm the diagnosis of s-IBM and for analysis of proteins involved in the muscle inflammation process. A local anesthetic is used to numb the area before the surgery and the wound is closed with stitches.
Inflammatory myopathies are a group of muscle diseases characterized by muscle weakness, high levels of muscle enzymes in the blood, and inflammation of the tissue surrounding muscle fibers (endomysium). The diseases making up the inflammatory myopathies are grouped into three subsets: I) Polymyositis (PM) II) Dermatomyositis (DM) III) Inclusion Body Myositis (IBM) Inflammatory myopathies are thought to be autoimmune processes and are treated with steroids and immunosuppressive drugs. However, many patients who initially respond to these treatments develop resistance to the therapy or experience side effects causing the treatments to be stopped. Researchers believe that intravenous immunoglobulin (IVIg) may provide patients with PM, DM, and IBM a safer and more effective alternative to standard therapies for the diseases. IVIg is a drug that has been used successfully to treat other immune-related diseases of the nervous system. The study will take 60 patients and divide them into two groups. Group one will receive 2 injections of IVIg once a month for three months. Group two will receive 2 injections of placebo "inactive injection of sterile water" once a month for three months. Following the three months of treatment, group one will begin taking the placebo and group two will begin taking IVIg for an additional 3 months. The drug will be considered effective if patients receiving it experience a significant improvement (\>15%) in muscle strength.
ABC008-IBM-202 is an open-label, multicenter study to evaluate the safety and efficacy of long-term administration of ulviprubart (ABC008) in subjects with IBM who have completed either Study ABC008-IBM-101 or Study ABC008-IBM-201. Subjects may be enrolled in this study if they meet study eligibility criteria and: * Have completed the Part 2 (Multiple Ascending Dose \[MAD\]) End of-Treatment (EOT) Visit in Study ABC008-IBM-101; subjects who continued further on into Part 3 of the study (MAD Extension) prior to enrolling in this study are also eligible; OR * Have completed the Week 80 Follow-up Visit in Study ABC008-IBM-201.
Inclusion-Body Myositis (IBM) results in weakness and the deterioration of distal arm muscles, the symptoms of which are currently assessed through expert examination at clinical visits. Such in-clinic assessments are time-consuming, subjective, of limited sensitivity, and only provide a snapshot of a patient's disease. In this project, the investigators will conduct clinical validation of monitoring digital biomarkers of upper limb function during activities of daily living using a wearable sensor platform that enables frequent, at-home monitoring of upper limb function health in IBM and could be incorporated into IBM trials.
This research study will evaluate safety and how well the study drug, nintedanib improve symptoms in participants with myositis associated interstitial lung disease (MA-ILD). Interstitial lung disease is a disorder caused by the abnormal accumulation of cells structures between air sacs of the lungs resulting in thickening, stiffness and scarring of the tissues of the lung. This study will enroll a total of 134 participants across 15 clinical sites located in the United States. A subset of participants will be enrolled remotely via telemedicine utilizing certified mobile home research nurses and various remote monitoring devices. The research visits may include a physical exam, vital signs (such as blood pressure, heart rate, etc.), pulmonary function tests (PFT and/or home spirometry), Computerized Tomography (or CT) scans of the chest, blood draws, wearing a physical activity monitor and completing questionnaires. Some of these events may be done at home, at a local facility or remotely (via telemedicine).
Objective: To collect information and biospecimens (such as blood, muscle, and skin samples) that will be used to research causes and treatments of inflammatory muscle diseases. Eligibility: People aged 12 years and older with suspected or confirmed inflammatory muscle disease. Healthy volunteers aged 18 years and older are also needed. Design: Participants will have at least 1 clinic visit. Each visit will last 4 to 8 hours. Some may return for additional visits. All participants will undergo these procedures (unless they are unable to): * Physical exam, including blood and urine tests. * Magnetic resonance imaging (MRI) scan of the thigh. Participants will lie still on a table with padding around 1 thigh. The table will slide into a tube. The scan will last for approximately 40 minutes. Some procedures are optional: * Muscle biopsy. An area of skin will be numbed. A quarter-inch cut will be made. Several pieces of muscle tissue, about the size of grains of rice, will be removed. * Skin biopsy. An area of skin will be numbed. A piece of skin about a quarter inch in diameter will be removed. * Genetic testing. Some of the samples collected may be used for genetic testing.
This is a prospective natural history study on patients with clinically defined sIBM. Participants will be assessed every 6 months over two years (five visits total). We will include 150 participants, enrolled across 13 sites, with sporadic IBM, diagnosed according to established criteria.
This is an open-label, single arm study evaluating the safety for patients with Inclusion Body Myositis. A total of 9 subjects will be enrolled in the study. Subjects will be randomized to Part 1 or Part 2 of the study in blocks of 3 every 3 months. Stem cell injections will be given in the forearm and thigh on either the left or right side of the body, depending on which side meets criteria. The overall goal of this pilot study is to test the safety of adipose derived regenerative cells in patients with Inclusion Body Myositis. If determined safe, this trial could lead to larger Phase II trials. While this specific trial's primary endpoint is safety, it our ultimate hope that ADRC injections into the forearm and thigh of IBM patients will slow, stabilize, or even reverse the progression of muscle weakness in patients with IBM.
This is a pilot study (phase 1 clinical trial) to evaluate the safety and tolerability of phenylbutyrate in IBM. In this open label study, 10 patients with sporadic inclusion body myositis will be treated with phenylbutyrate (3 gm twice daily) for 3 months. There will be a run-in period, during which certain biomarkers will be measured at baseline and at the end of the run-in period in addition to final measurement at the end of the treatment period.
A multicenter, nonrandomized, open-label, uncontrolled clinical extension trial designed to compare the efficacy and safety of early versus delayed start of arimoclomol in the treatment of Inclusion Body Myositis (IBM)
A study looking at the effect of pioglitazone in skeletal muscle of patients with sporadic inclusion body myositis (sIBM).