The Myelin Disorders Biorepository Project (MDBP) seeks to collect and analyze clinical data and biological samples from leukodystrophy patients worldwide to support ongoing and future research projects. The MDBP is one of the world's largest leukodystrophy biorepositories, having enrolled nearly 2,000 affected individuals since it was launched over a decade ago.
Researchers working in the biorepository hope to use these materials to uncover new genetic etiologies for various leukodystrophies, develop biomarkers for use in future clinical trials, and better understand the natural history of these disorders. The knowledge gained from these efforts may help improve the diagnostic tools and treatment options available to patients in the future.
Leukodystrophy· Children's Hospital of Philadelphia· Atlanta, Georgia · Chicago, Illinois +2 more
This open-label extension study will provide post-trial access to pelacarsen (TQJ230) to participants who have successfully completed the double-blind parent study (CTQJ230A12301).
The central objective of this application is to explore the neural substrate of personalized tDCS (ptDCS) and to determine whether the paradigm for each stroke patient can predict the amount of sustained clinical improvement through increased connectivity as measured by a biomarker of plasticity.
Stroke· The Methodist Hospital Research Institute· Houston, Texas· Ages 18–80
The overall goal of the DISCOVERY study is to better understand what factors contribute to changes in cognitive (i.e., thinking and memory) abilities in patients who experienced a stroke. The purpose of the study is to help doctors identify patients at risk for dementia (decline in memory, thinking and other mental abilities that significantly affects daily functioning) after their stroke so that future treatments may be developed to improve outcomes in stroke patients. For this study, a "stroke" is defined as either (1) an acute ischemic stroke (AIS, or blood clot in the brain), (2) an intracerebral hemorrhage (ICH, or bleeding in the brain), (3) or an aneurysmal subarachnoid hemorrhage (aSAH, or bleeding around the brain caused by an abnormal bulge in a blood vessel that bursts).
The investigators hypothesize that:
1. The size, type and location of the stroke play an important role in recovery of thinking and memory abilities after stroke, and pre-existing indicators of brain health further determine the extent of this recovery.
2. Specific stroke events occurring in individuals with underlying genetic or biological risk factors can cause further declines in brain heath, leading to changes in thinking and memory abilities after stroke.
3. Studying thinking and memory alongside brain imaging and blood samples in patients who have had a stroke allows for earlier identification of declining brain health and development of individualized treatment plans to improve patient outcomes in the future.
Ischemic Stroke· Massachusetts General Hospital· Phoenix, Arizona · Los Angeles, California +3 more
The KHENERFIN study aims to determine whether the study medicine, sonlicromanol, is able to reduce symptoms of fatigue and the impact of fatigue on daily life, and whether sonlicromanol is able to improve physical abilities of people like balance control and lower limb skeletal muscle strength in people with mitochondrial disease.
In this study, the effects of sonlicromanol are compared against a placebo, a tablet identical in appearance and taste but without the active drug. Participants take either sonlicromanol or placebo twice daily for a treatment duration of 52 weeks.
In addition to these primary objectives, the study evaluates the efficacy of sonlicromanol on secondary and exploratory outcomes, as well as its safety and tolerability after one year of treatment.
This study will assess the safety and effectiveness of GORE® CARDIOFORM Septal Occluder in a post approval setting and evaluate the quality of operator education and training and transferability of trial experience to a post-market setting.
Stroke· W.L.Gore & Associates· La Jolla, California · Loma Linda, California +3 more· Ages 18–70
The objective of this study is to develop Neuro-Intermuscular Coordination Enhancement (NICE) rehabilitation, a novel neuromuscular control signal-guided strategy that visually guides stroke patients to individually activate motor modules through human-machine interaction. Ultimately, the development will lead to better clinical motor recovery, better quality of life, and lowered healthcare costs associated with the impairment.
Chronic Stroke-related Upper-extremity Motor Impairment· University of Houston· Houston, Texas· Ages 21–80
This is a Phase 2/3 study evaluating the safety and efficacy of DM199 (rinvecalinase alfa) in treating participants with moderate stroke severity, who present within 24 hours of Acute Ischemic Stroke (AIS) onset due to small and medium vessel occlusions. This study focuses on participants with limited treatment options. Participants who have or will receive mechanical thrombectomy (MT) are not eligible for participation. Additionally, participants who have received fibrinolytics are excluded unless they experience a persistent neurological deficit of moderate severity six or more hours after fibrinolytic treatment. Participants considered for this trial should not be denied the use of standard of care (SoC) AIS therapies, such as fibrinolytics or MT, when appropriate. The double-blinded study will be randomized and placebo-controlled at up to approximately 100 sites.
This is an observational study to better understand the risk factors and progression of CADASIL, a leading cause of vascular cognitive impairment and dementia (VCID). 575 participants will be enrolled and can expect to be on study for up to 5 years.
CADASIL· University of Wisconsin, Madison· Chicago, Illinois
The purpose of this clinical trial is to study the two main types of approaches used in stroke thrombectomy and to investigate if one approach is more effective than the other, as this is currently not known. This study will be conducted in adults who have been diagnosed with an acute ischemic stroke and who are undergoing a thrombectomy for the treatment of their stroke. The main questions it aims to answer are:
* Does the use of a balloon guide catheter versus a conventional guide catheter lower the time needed to restore blood flow in the blocked vessel in the brain
* To help researchers better understand the technical, clinical, and procedural outcomes associated with using a balloon guide catheter versus a conventional guide catheter in stroke thrombectomy
Participants will be asked to
* Share their medical history and imaging data that is collected as part of their routine medical care
* Undergo a mechanical thrombectomy as part of their routine medical care
* Answer some questions about their neurological functioning at 3 months (90 days) post hospitalization
Acute Ischemic Stroke AIS· University of South Florida· Tampa, Florida · Houston, Texas
This single-session study aims to evaluate a novel gait training protocol that integrates mechanical constraints and sensory feedback to enhance paretic leg propulsion in individuals post-stroke. The study will include 15 individuals who have experienced a stroke and 15 healthy adults, each aged 20 years or older. Participants will walk on both tied-belt and split-belt treadmills under various training conditions, including backward-directed resistance (applied at the pelvis, ankle, or both) and real-time sensory feedback (visual, auditory, or combined). These interventions will be applied individually and in combination to identify the most effective environment for promoting symmetrical gait patterns. Each session will last approximately two hours. The equipment used is non-invasive, and the risk to participants is minimal.
Stroke· The University of Texas Medical Branch, Galveston· Galveston, Texas
The North American Mitochondrial Disease Consortium (NAMDC) maintains a patient contact registry and tissue biorepository for patients with mitochondrial disorders.
Mitochondrial Disorders· Columbia University· San Diego, California · Stanford, California +3 more
Extensive research is being conducted in search of neuroprotective agents for possible use in the acute phase of stroke and agents that can be used for neurorepair in later stages of stroke. Several trials have been conducted and are in progress using different pharmacological agents, but none of the studies involve the stimulation of ETB receptors to treat cerebral ischemic stroke. Sovateltide (IRL-1620, PMZ-1620) has been effective in animal models of cerebral ischemic stroke. Its safety and tolerability have been demonstrated in a human phase I study with 7 subjects. Clinical phase II and III results indicate that sovateltide is a novel, first-in-class, highly effective drug candidate for treating cerebral ischemic stroke. Safety and significant efficacy in improving the National Institutes of Health Stroke Scale (NIHSS), Modified Rankin scale (mRS), and Barthel index (BI) obtained in phase II and III studies in patients with cerebral ischemic stroke in India are convincing and encouraged us to investigate its safety and efficacy in cerebral ischemic stroke patients in the United States. Therefore, the plan is to conduct a phase III clinical study to evaluate the safety and efficacy of sovateltide therapy along with standard of care in patients of acute ischemic stroke.
The overall goal of this study is to attain the highest possible health-related quality of life for individuals with lower-limb impairment through a clinical trial examining three different modern carbon fiber ankle-foot orthosis (AFO) treatment options targeting the rehabilitation of individuals post-stroke. To achieve this goal, the investigators will:
1. Identify the factors that significantly contribute to an individual's highest potential quality of life when considering each of the three available AFO design options, and
2. Develop prediction models of clinical performance using biomechanical function linked to the three AFO designs.
Participants will be asked to:
* Wear each of the three modern AFO designs for one month, after receiving therapy training and
* Complete questionnaires and performance tests with each AFO.
In addition, the subset of individuals participating in goal 2) will also be asked to:
* Perform biomechanical analyses using high-speed cameras and force plates during different walking and balance tests with each AFO design.
Ankle Foot Orthosis (AFO)· University of Texas at Austin· Downey, California · Jacksonville, Florida +2 more
SISTER is a Phase-II, prospective, randomized, placebo-controlled, blinded, dose finding trial that aims to determine the safety and preliminary efficacy of TS23, a monoclonal antibody against the alpha-2 antiplasmin (a2-AP), in acute ischemic stroke.
TESTED will compare the risks and benefits of endovascular thrombectomy (EVT) to medical management (no EVT) in ischemic stroke patients who have a blockage in one of the large blood vessels in the brain and have a moderate-to-severe disability prior to their stroke.
Stroke· University of Cincinnati· Phoenix, Arizona · Atlanta, Georgia +2 more
VERIFY will validate biomarkers of upper extremity (UE) motor outcome in the acute ischemic stroke window for immediate use in clinical trials, and explore these biomarkers in acute intracerebral hemorrhage. VERIFY will create the first multicenter, large-scale, prospective dataset of clinical, transmagnetic stimulation (TMS), and MRI measures in the acute stroke time window.
Stroke· Yale University· Birmingham, Alabama · Tucson, Arizona +2 more
The purpose of this study is to evaluate the safety and tolerability of ascending doses (Part A) and selected doses (Part B) of BB-031 in acute ischemic stroke patients presenting within 24 hours of stroke onset.
Participants will be randomized to receive one dose of either the investigational drug or placebo and will be followed for 90 days. A total of 228 patients are planned in this study.
The overall objective of the project is to determine the effectiveness of tele-delivered behavioral activation (BA) by trained lay counselors (Tele-BA-S) to prevent Post-stroke depression (PSD) in low-income, older stroke survivors with subthreshold depression (SD).
Post-stroke Depression· The University of Texas Health Science Center, Houston· Houston, Texas