12 Clinical Trials for Various Conditions
Background: HTLV-1 associated myelopathy/tropical spastic paraparesis (HAM/TSP) is a rare, progressive disease. It occurs in some people infected with the HTLV-1 virus. It leads to weakness in the lower limbs and other serious problems. It has no treatment. Teriflunomide is a drug used to treat multiple sclerosis. It reduces immune cells that make the disease worse. Researchers want to learn if this drug can help people with HAM/TSP. Objective: To learn the effects, immune response, safety, and tolerability of teriflunomide in people with HAM/TSP. Eligibility: Adults ages 18 and older with HAM/TSP. Design: Participants will be screened under protocol 98-N-0047. Participants will have a medical history. They will have physical and neurological exams. They will have blood and urine tests. Participants will take 1 tablet of the study drug once a day for 9 months. They will keep a drug diary. Participants will have lymphapheresis. For this, blood is drawn from a needle in one arm. A machine divides the blood into red cells, plasma, and white cells. The white cells are removed. The plasma and red cells are returned to the participant through a needle in the other arm. Participants will have lumbar punctures ( spinal taps ). For this, a thin needle is inserted into the spinal canal in the lower back. Spinal fluid is removed. Participants will have magnetic resonance imaging (MRI) of the brain and spine. The MRI scanner is a metal cylinder surrounded by a strong magnetic field. During the MRI, participants will lie on a table that can slide in and out of the scanner. Participation will last for 15 months.
This study will examine the use of the humanized Mik-Beta-1 (Hu Mik-(SqrRoot) 1) monoclonal antibody in patients with HTLV-1-associated myelopathy/tropical spastic paraparesis (HAM/TSP). Some patients infected with the human T-lymphotropic virus type 1 (HTLV-1) virus develop HAM/TSP, a disease in which the immune response to HTLV-1 becomes directed against the person's own body in what is called an autoimmune response. Hu-Mik-Beta-1 is a genetically engineered antibody that blocks the action of a chemical produced by the body during infection or inflammation called interleukin 15 (IL-15). Blocking IL-15 may prevent the autoimmune response that results in HAM/TSP. Patients 18 years of age and older with HAM/TSP may be eligible for this study. Candidates are screened with a medical history and physical examination, blood and urine tests, and an electrocardiogram. Participants undergo the following procedures: 1. Baseline visit(s): Repeat physical examination and blood and urine tests, as well as the following: * Lumbar puncture: A local anesthetic is injected to numb the skin of the lower back. A needle is inserted in the space between the bones where the cerebrospinal fluid that bathes the brain and spinal cord circulates below the spinal cord. About 4 tablespoons of fluid is collected through the needle. * Magnetic resonance imaging (MRI): This test uses radio waves and magnets to produce images of body tissues and organs. The patient lies on a table that slides into a metal cylinder surrounded by a strong magnetic field. During part of the scan, a contrast agent is injected to brighten the images. * Apheresis: This procedure is used to collect large quantities of white blood cells. Whole blood is collected through a needle in an arm vein and directed into a machine that separates it into its components by spinning. The white cells and plasma are removed and the rest of the blood (red cells and platelets) is returned to the body through the same needle. 2. Hu Mik-Beta-1 treatment: Infusions of Hu Mik-Beta-1 are given through a vein every 3 weeks for nine doses. The first treatment requires at least an overnight hospital stay; subsequent infusions are given in the outpatient clinic. 3. Blood and urine tests and a physical examination at every treatment visit and a skin test at one treatment visit. 4. Research tests at the end of the 24-week treatment period, including lumbar puncture (spinal tap), MRI scan, and apheresis. 5. After completing treatment, patients have three follow-up clinic visits for blood and urine tests, and a skin test at one follow-up visit.
This study will use three different magnetic resonance imaging (MRI) techniques to study HTLV-1 associated myelopathy/tropical spastic paraparesis (HAM/STP)-a disease of slowly progressive weakness in the legs. It is not known how the HTLV-1 virus causes this disease, but it is thought that as the body's immune system tries to destroy the virus, parts of the nervous system-primarily the spinal cord-are damaged. Patients 18 years of age and older with HAM/TSP and healthy normal volunteers may be eligible for this study. Participants will undergo diffusion tensor MRI, MR-spectroscopy, and magnetization transfer imaging to look at different compositional, architectural, and microscopic properties of the brain. All of these techniques are similar to conventional MRI, and like the conventional method they use a strong magnetic field and radio waves to measure structural and chemical changes in brain tissue. Each of the three scans will be done on separate days, each lasting about 1 hour. For the procedures, the patient or volunteer lies on a stretcher in a narrow metal cylinder (the scanner) and is asked to remain still for 15 to 30 minutes at a time. A special lightweight coil may be placed on the head to enhance the brain images. The subject can communicate with the person doing the scan at all times.
The SciExVR study will evaluate the potential benefit of autologous bone marrow derived stem cells (BMSC) in the treatment of spinal cord injury with evidence of impaired motor or sensory function. The treatment consists of bilateral paraspinal injections of the BMSC at the level of the injury as well as superior and inferior to that spinal segment followed by an intravenous injection and intranasal placement. Patients undergoing BMSC treatment may also be assigned to use of exoskeletal movement (or equivalent) or virtual reality visualization (or equivalent) to augment upper motor neuron firing and/or receptivity of the sensory neurons. http://mdstemcells.com/sciexvr/
Many people with spinal cord injury (SCI) retain at least some movement below their injury, but their muscles often have a 'mind of their own'. Typical exercise programs focus on keeping muscles strong and flexible, but don't usually focus on helping patients control their muscles. The investigators' exercise research study will compare two different programs with the specific goal of improving conscious control of patients' muscles below the injury. This study focuses on those with 'chronic' SCI - the injury occurred at least one year prior to enrolling. This is a single center study taking place in the Bronx, NY. The first phase of the study will be observational - the investigators will analyze which nerve connections might remain partially intact through the injury (even if the nerves aren't consciously controlled). Participants with all severity of SCI may participate in this first phase. The second phase of the study will involve people who retain at least slight ability to move their legs and the ability to move the arms against gravity. Each person will undergo two different exercise rehabilitation strategies: weight-supported treadmill training; and balance training combined with skilled arm or hand exercises. The investigators will compare the effects of these exercise programs on a variety of outcomes, including gait speed, balance, strength, and muscle activation in response to brain stimulation. The investigators hypothesize that participants with chronic SCI undergoing combined balance/arm/hand training will show improved outcomes when compared to traditional gait or balance training.
Safety and Efficacy of AAV9/AP4B1 For Patients with AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47): A Phase 1/2 Single-Center, Open-Label Study of Stereotactic Intra-cisterna Magna Administration. The goal of this clinical trial is to evaluate whether a gene therapy can safely treat children with SPG47, a rare genetic condition that causes progressive spasticity and developmental delays. The main questions it aims to answer are: * Is the gene therapy safe and well tolerated? * Does the gene therapy improve motor function and developmental outcomes? Participants will: * Undergo screening assessments to confirm eligibility * Receive a single dose of the gene therapy vector * Attend follow-up visits for safety monitoring and developmental assessments over the course of five years
The purpose of this study is to collect 650 blood and 300 cerebrospinal fluid (CSF) samples from people with amyotrophic lateral sclerosis (ALS), pure lower or upper motor neuron diseases, as well as other neurodegenerative diseases and from people with no neurological disorder. Through comparison of these samples, the researchers hope to learn more about the underlying cause of ALS, as well as find unique biological markers, which could be used to diagnose ALS and monitor disease progression. Additionally, up to 600 blood samples will be collected for a sub-study for DNA analysis. Studying components of the blood, such as DNA, may help us understand what happens when genes function abnormally and how it might be related to disease.
HTLV stands for human T cell leukemia virus. HTLV-1 is a virus that attacks specific kinds of white blood cells called T cells. T cells are part of the natural defense system of the body. HTLV-1 has been associated with leukemia and lymphoma. In addition, approximately 1% of all patients infected with HTLV-1 develops a condition known as HTLV-1 associated myelopathy (HAM) / tropical spastic paraparesis (TSP). Currently there is no clearly defined, effective treatment for patients with HAM/TSP. Steroids have been used as therapy but have only been able to provide temporary relief of symptoms. Human interferon is a small protein released from different kinds of cells in the body. Interferon has been known to have antiviral and immunological effects and has been used to treat hepatitis and multiple sclerosis. Interferon Beta is released from cells called fibroblasts. These cells play a role in the production of connective tissue. The purpose of this study is to evaluate the possible role of recombinant interferon beta (Avonex) in treatment of HAM/TSP. The study is broken into three phases, a pre-treatment phase, a treatment phase, and a post-treatment phase. The total duration of the study will be 44 weeks. Patients participating in this study will receive injections of Avonex 1 to 2 times a week. Throughout the study patients will regularly submit blood samples and undergo diagnostic tests such as MRI and measures of somatosensory evoked potentials.
Objective: Human T-lymphotropic virus type-I-associated myelopathy / tropical spastic paraparesis (HAM/TSP) is a rare neurologic disorder that affects less than 5% of patients infected with the HTLV-I virus. The purpose of this protocol is to study the natural history of HAM/TSP by monitoring clinical progression of patients longitudinally. Additionally, we will attempt to define the virological and immunological changes of HAM/TSP. Study Population: Patients with HAM/TSP who fulfill World Health Organization diagnostic criteria are eligible to participate in this protocol. Asymptomatic seropositive individuals and individuals with indeterminate HTLV-1 serology are also eligible to participate. Design and Outcome Measures: A longitudinal assessment of clinical, virological and immunological progression in HAM/TSP will be accomplished through periodic testing and evaluation. Asymptomatic seropositive individuals, those with seroindeterminate HTLV-I serology and normal volunteers may serve as controls. Longitudinal standardized neurological examinations will be performed. Longitudinal samples of serum, plasma, and lymphocytes may be obtained from participants. Lumbar punctures may be performed on all participants. These samples will be used virological and immunological assays. A focus is on the relationships between the characteristics of viral infection, the immune response, and the genetic makeup.
Multiple sclerosis (MS) is a disease of the nervous system. The exact cause of MS is unknown, but it is believed to be an autoimmune condition. Autoimmune conditions are diseases that cause the body's immune system and natural defenses to attack healthy cells. In the case of MS, the immune system begins attacking myelin, the cells that make up the sheath covering nerves. Without myelin, nerves are unable to transmit signals effectively and symptoms occur. This study is directed toward a better understanding of the cause of Multiple Sclerosis (MS). Researchers will evaluate patients with a tentative diagnosis of MS or other neurological diseases possibly caused by a immunological reaction. Patients will undergo a series of three MRIs, taken once a month for three months and submit blood samples for immunological studies.
This study will use a magnetic resonance imaging technique called nuclear magnetic spectroscopy (H-MRS) to define the pathology and progression of primary lateral sclerosis, hereditary spastic paraplegia and amyotrophic lateral sclerosis and assess the usefulness of this technique in evaluating patients' response to therapy. H-MRS will be used to examine metabolic changes in the parts of the brain and spinal cord (motor cortex and corticospinal tract) involved in movement. Normal volunteers and patients with primary lateral sclerosis, hereditary spastic paraplegia or amyotrophic lateral sclerosis between 21 and 65 years of age may be eligible for this study. Participants will have up to five H-MRS studies, including baseline and follow-up tests. For this procedure, the subject lies on a stretcher that is moved into a strong magnetic field. Earplugs are worn to muffle the loud knocking noise that occurs during switching of radio frequencies. The subject will be asked to lie still during each scan, for 1 to 8 minutes at a time. Total scanning time varies from 20 minutes to 2 hours, with most examinations lasting between 45 and 90 minutes. Communication with the medical staff is possible at all times during the scan.
This study will provide information about changes that occur in the motor neurons of the spinal cord (the nerve cells that control the muscles) when the motor cortex (the region of the brain that controls movement) is unable to send messages to the spinal cord and muscles in the normal way. This information will help elucidate how the nervous system adapts after injury or disease of the motor cortex. Healthy adult volunteers and adults with a spasticity disorder and moderate weakness may be eligible for this study. Patients will be screened with a medical history, physical examination and diagnostic studies as needed. Healthy volunteers will have a neurological examination. Muscle weakness and spasticity will be evaluated in both groups of subjects. All participants will have electromyography (measurement of electrical activity in muscles) during nerve stimulation and transcranial magnetic stimulation, described below. (Some patients, such as those with a pacemaker or implanted medication pumps, metal objects in the eye, history of epilepsy and others, will not have magnetic stimulation.) Electromyography - The electrical activity of muscles will be measured either by 1) using metal electrodes taped to the skin overlying a muscle, or 2) using thin wires inserted into the muscle through a needle. Nerve stimulation - The nerves will be stimulated by applying a small electrical pulse through metal disks on the skin of the arm or leg. Transcranial magnetic stimulation - A brief electrical current is passed through a wire coil placed on the scalp. This creates a magnetic pulse, which stimulates the brain. During the test, the participant may be asked to tense certain muscles slightly or perform other simple actions. Nerve block - Some patients will have a nerve block of one of the nerves in the arm. For this procedure, a local anesthetic is injected under the skin to produce numbness and weakness in some arm muscles.