175 Clinical Trials for Various Conditions
The purpose of this study is to examine the effects of high intensity stepping training on gait recovery, including walking speed and endurance, in patients with cerebellar ataxia. The hypothesis is that there will be a significant improvement in gait outcome measures (6 Minute Walk Test and 10 Meter Walk Test) in patients who receive high-intensity stepping training during physical therapy.
The aim of the research is to improve motor function in people with cerebellar ataxia by using neuroimaging methods and mental imagery to "exercise" motor networks in the brain. The relevance of this research to public health is that results have the potential to reduce motor deficits associated with cerebellar atrophy, thereby enhancing the quality of life and promoting independence.
This project will study the feasibility of motor rehabilitation in people with cerebellar ataxia using real-time functional magnetic resonance imaging neurofeedback (rt-fMRI NF) in conjunction with motor imagery. To do so, data will be collected from healthy adults in this protocol, to be compared with data from cerebellar ataxia participants.
Engage-Ataxia will implement a physical activity coaching program for people with cerebellar ataxia at Teachers College, Columbia University. This program expands upon the current Engage program for people with Parkinson's disease (Engage-PD), an exercise coaching program for people with early stage Parkinson's disease to target individuals with early stage cerebellar ataxia. Engage-Ataxia will utilize a physical or occupational therapist to provide up to five one-on-one coaching sessions for individuals newly diagnosed with cerebellar ataxia. Therapists will work with participants to provide individualized structured support to facilitate and optimize exercise uptake as one part of comprehensive disease management. Participants will undertake two assessments three months apart, and will receive coaching interventions via Zoom healthcare platform. The primary objective of this program is to increase physical activity and exercise engagement in individuals with early stage cerebellar ataxia. This feasibility study will provide preliminary data and insight into the benefits of a remote coaching intervention for people with cerebellar ataxia.
Balance and aerobic training show promise as treatments for degenerative cerebellar diseases, but the neural effects of both training methods are unknown. The goal of this project is to evaluate how each training method impacts the brain, and particularly, the degenerating cerebellum. Various neuroimaging techniques will be used to accomplish this goal and test the hypothesis that balance training impacts brain structures outside the cerebellum whereas aerobic training causes more neuroplastic changes within the cerebellum.
The primary aim is to show balance training improves DCD individual's ability to compensate for their activity limitations, but does not impact disease progression. The second aim is to demonstrate aerobic exercise improves balance and gait in DCD persons by affecting brain processes and slowing cerebellar atrophy.
The purpose of this study is to create a repository for cerebellar ataxia and nucleotide repeat diseases in order to fully investigate the genetic and phenotypic presentations of both.
This is a randomized, double-blind, placebo-controlled Phase 2 study evaluating oral administration of CAD-1883 in the treatment of adults with a genotypic diagnosis of Spinocerebellar Ataxia (SCA). This study offers the opportunity to understand the safety, tolerability, and efficacy of CAD-1883 in the SCA patient population.
The purpose of this study is to test for benefits of reinforcement based training paradigm versus standard practice over weeks for improving reaching movements in people with ataxia.
The proposed study aims to characterize ataxia occurring in essential tremor and essential tremor with DBS.
The first aim is to show aerobic training improves degenerative cerebellar patients functionally The second aim is to compare the effects of balance and aerobic training on degenerative cerebellar disease.
The first aim is to show balance training improves DCD individual's ability to compensate for their activity limitations, but does not impact disease progression. The second aim is to demonstrate aerobic exercise improves balance and gait in DCD persons by affecting brain processes and slowing cerebellar atrophy.
This study evaluates the effectiveness of a 12-week in home balance training program with and without sensory augmentation for individuals with ataxia. Subjects wear a belt while performing balance exercises three times per week for 12 weeks. The belt measures body motion and has small vibrating elements called tactors mounted inside that when turned on, feel like a cell phone set to vibrate. The tactors provide information about body motion and indicate when and how to make a postural correction. Subjects will receive six weeks of balance training with the tactors turned on and six weeks of balance training with the tactors turned off.
Spinocerebellar Ataxia (SCA) refers to a family of genetic diseases that cause progressive problems with gait and balance, as well as other debilitating symptoms. This is a randomized controlled pilot study to test a novel therapeutic intervention that uses noninvasive magnetic brain stimulation to improve functional outcomes in patients with SCA. The study will include quantitative evaluations of gait, balance, and brain physiology to examine possible objective end-points for a future, larger multi-site clinical trial. The investigators anticipate that patients receiving the real intervention will show a functional gain.
The purpose of this study is to determine whether a person's ability to adapt (i.e. short term motor learning) predicts their ability to benefit from physical therapy exercises.
Episodic ataxia (EA) is a rare genetic disease characterized by episodes of imbalance, incoordination, and slurring of speech. The underlying cause of EA is only partly understood, and currently there are no established treatments. There is also little information about the link between EA's clinical features and its genetic basis. The purpose of this study is to better characterize EA and disease progression. In turn, this may direct the development of future treatments.
This study will screen patients with cerebellar ataxia to check for antibodies that indicate allergy to gluten (wheat protein) and will study the effect of a gluten-free diet in patients with these antibodies. Patients with cerebellar ataxia have problems with coordination, resulting in "clumsiness" and unsteadiness of posture and walking. There are many known causes of cerebellar ataxia, but in many patients the cause is unknown and there are no available treatments. Cerebellar ataxia has been recognized as a complication of celiac disease, a syndrome characterized by sensitivity to gluten. Recognizing gluten sensitivity in patients with cerebellar ataxia would be important for two reasons: it would be one of the rare causes of the disease that are potentially treatable, and it would identify patients at risk for developing gastrointestinal cancers, particularly intestinal lymphoma. Patients with cerebellar ataxia of known or unknown cause and normal healthy volunteers of any age are eligible for this study. All participants will have a medical history, physical examination, blood drawn (30 milliliters, or 2 tablespoons) to check for celiac disease antibodies, and possibly other lab tests. This completes the participation of normal volunteers. All patients will have magnetic resonance imaging (MRI) of the brain. This diagnostic tool uses a strong magnetic field and radio waves instead of X-rays to show structural and chemical changes in tissues. During the scanning, the patient lies on a table in a narrow cylinder containing a magnetic field. He or she can speak with a staff member via an intercom system at all times during the procedure. Scanning times vary from 20 minutes to 2 hours. Patients who have celiac disease antibodies will have an upper gastrointestinal (GI) endoscopy intestinal biopsy. For this procedure, a flexible tube is inserted into the mouth and down the throat into the stomach and duodenum (the upper part of the small intestine), where a small tissue sample is taken for microscopic examination. Patients with these antibodies will be put on a gluten-free diet and will be followed at NIH every 3 months for 12 months. On the first visit, patients will have their ataxia evaluated using NINDS's ataxia scale and will meet with a dietitian for instructions for a gluten-free diet. On the second through fifth visits (after 3, 6, 9 and 12 months, respectively, on the gluten-free diet), patients will have their ataxia evaluated, speak with a dietitian to assess their nutritional status, weight, and compliance with the diet, and provide a blood sample for celiac disease antibody testing. At the completion of the study, patients may choose to continue or stop the gluten-free diet. If the ataxia assessments show improvement, patients will be advised to continue the gluten-free diet permanently.
In this study, researchers will learn more about the effects and safety of BIIB141, also known as omaveloxolone or SKYCLARYS®. This drug has been approved, or made available for doctors to prescribe, for people with Friedreich's Ataxia (FA) who are at least 16 years old. But, it is not yet available for children and teens with FA who are younger than 16 years old. The main objective of this study is to learn how BIIB141 works in the body and about its safety in children and teens who are 2 to 15 years old. The main questions researchers want to answer in this study are: * How does BIIB141 affect the participants' FA symptoms balance and stability? * How many participants have medical problems during the study? * Are there any changes in the participants' overall health during the study? * Are there any changes in the participants' heart health? * Are there any changes in how the participants move through puberty? Puberty is the time in someone's life when their body changes from a child to an adult. Researchers will also learn more about: - How the body processes BIIB141 in children and teens This study will be done as follows: * Participants will be screened to check if they can join the study. The screening period will be up to 28 days, after which participants will check into their study research center. * There are 2 parts in this study. During Part 1, participants will take either BIIB141 or a placebo once a day. * In Part 1, participants will take BIIB141 or the placebo in a study research center on Day 1, and then at in-person visits at Week 4, Week 12, Week 26, and Week 52. On all other days, they will take BIIB141 or the placebo at home. Part 1 lasts up to 52 weeks. * During Part 2, participants from Part 1 will either continue taking BIIB141 or start it if they were taking the placebo. Part 2 will last up to 104 weeks. * In Part 1, participants will have up to 10 visits to their study research center and a phone call at Week 2. In Part 2, participants will have visits at Weeks 4, 8,12, 26, and every 26 weeks after that until they leave the study, and a phone call at Week 2. There will be a final phone call to check on the participants' health 31 days after their last dose. * Each participant will be in the study for up to about 3 years
The goal of this clinical trial is to evaluate the safety and tolerability of nomlabofusp (CTI-1601) in adolescents and children with Friedreich's ataxia (FRDA).
A pivotal, randomized, double-blind, placebo-controlled, multi-center therapeutic study for patients age 4 and older with a confirmed diagnosis of Ataxia-Telangiectasia (A-T). The objective of this study is to evaluate the safety, tolerability and efficacy of N-acetyl-L-leucine (IB1001) compared to standard of care.
In this study, researchers will learn more about the safety of BIIB141, also known as omaveloxolone or SKYCLARYS. This is a drug available for doctors to prescribe for people with Friedrich's Ataxia, also known as FA. This is known as an "observational" study, which collects health information about study participants without changing their medical care. Participants for this study will have taken BIIB141 at any time during pregnancy and/or while breastfeeding or pumping up through the first year after delivery. Participants can join this study on their own or they may be enrolled by their regular doctors. This study is also known as the "SKYCLARYS (Omaveloxolone) Pregnancy and Lactation Surveillance Program." The main objective of this study is to learn more about how BIIB141 may affect pregnancy, as well as any effects on the health of the mother and of the baby during its first year of life. The main question researchers want to answer in this study is: · Does taking BIIB141 during pregnancy or breastfeeding lead to any major birth defects? Researchers will also learn more about: * Does taking BIIB141 during pregnancy or breastfeeding lead to any minor birth defects? * Does taking BIIB141 during pregnancy or breastfeeding affect the following: * Gestational diabetes, a disease that can happen during pregnancy that affects how your body uses sugar * Pre-eclampsia, a pregnancy-related high blood pressure disease * Unborn baby being small for its expected age (usually in weeks) * Loss of an unborn baby * Live birth * Premature birth * Loss of a newborn * Growth or developmental delays in the baby * Serious illness in the baby resulting in hospitalization * Serious infections in the baby, or ones in babies with a weakened immune system This study will be done as follows: * Participants will join the study after signing an informed consent form, also known as an ICF. * During the study, health information from the participants' regular visits to their doctor will be collected based on whether participant joined the study while pregnant or after the baby is born. * Each participant will be in the study for up to 1 year after the birth of their child, unless they decide to leave early. Overall, this study is expected to last at least 10 years.
In this study, researchers will learn more about the safety of BIIB141, also known as omaveloxolone or SKYCLARYS®. This is a drug available for doctors to prescribe for people with Friedreich's Ataxia, also known as FA. This is known as an "observational" study, which collects health information about study participants without changing their medical care. Participants for this study will be found using a group called the Friedreich's Ataxia Global Clinical Consortium (FA GCC) UNIFIED Natural History Study (UNIFAI). The FA-GCC is a group of study research centers that helps provide clinical care for FA patients and also helps researchers learn more about how FA affects patients over a long time. The main objective of this study is to collect safety information in participants with FA from UNIFAI. Some of the participants in this study will be prescribed BIIB141 for the first time by their own doctors. Some of the participants will have started taking BIIB141 after joining UNIFAI, but less than 12 months before joining this study. The main questions researchers want to answer in this study are: * How many participants had serious adverse events (SAEs)? An adverse event is considered serious when it results in death, is life-threatening, causes lasting problems, or requires hospital care. * How many participants had adverse events (AEs) related to heart failure or liver damage caused by the drug? Researchers will also learn more about : • Why and when participants stopped treatment, left the study, or took more of the drug than was prescribed This study will be done as follows: * Participants will be screened to check if they can join the study. * After joining the study, the participants who had never started BIIB141 treatment before must start it within 6 months. Otherwise, all participants will take BIIB141 throughout this study as prescribed by their own doctor. * During the study, each participant's doctor will decide how often the participant visits the study research center to check on their health. This will be based on the doctor's own clinical judgment and what is recommended by the drug's label. * Data from the participants' regular visits to their doctor will be collected at 1 month, 2 months, 3 months, 6 months, 12 months, 24 months, 36 months, 48 months, and 60 months. * Each participant will be in the study for up to 5 years.
Multicenter, prospective, observational natural history and outcome measure study of children and young adults with Friedreich ataxia.
This is a prospective interventional study of patients with Friedreich's Ataxia that receive respiratory strength training for a period of 12 weeks with two research visits at the beginning and at the end of the study period. Visits include swallowing evaluation with fiberoptic endoscopic evaluation of swallowing, pulmonary function testing, surface electromyography and patient surveys.
The purpose of this study is to leverage two sources of real-world data (RWD) to assess the effectiveness of troriluzole after three years of treatment in patients with SCA by comparison to an external control of untreated patients who were followed in a natural history cohort. Real world evidence of effectiveness will be assessed from the RWD sources to examine the treatment effects of toriluzole in SCA out to 3 years. Progression rates of SCA differ by genotype and long-term follow-up is needed to assess for potential efficacy in this rare disease.
Friedreich Ataxia is a rare condition that causes damage to the nervous system and muscles. People with Friedreich Ataxia have difficulty walking, lose sensation in their arms and legs, and have slurred speech. It can also affect the heart and many people with Friedrich Ataxia develop serious heart problems. Friedreich Ataxia is a genetic condition which means a faulty gene is passed down through families. This type of gene therapy treats a genetic condition by providing a healthy copy of the gene. At the time this study started, there was no approved treatment for heart problems in people with Friedreich Ataxia. In this study, ASP2016 is being tested in humans for the first time. The people taking part are adults with Friedreich Ataxia who have heart problems. The main aims of the study are to check the safety of ASP2016 and how people cope with (tolerate) ASP2016. ASP2016 is given as a slow injection into a vein. This is called an infusion. People will also take tablets of a medicine called prednisolone. This is taken to stop the immune system interfering with ASP2016. Each person in the study will be given 1 single infusion of ASP2016. Different small groups will receive lower or higher doses of ASP2016. Each person will stay overnight in the clinic for at least 1 night after their infusion. For the first few months, people will visit the clinic regularly. There may be the option of home visits by a study nurse at some visits. At the 6-month and 12-month visits extra tests, procedures, and scans will be done. One of these is an ECHO (echocardiogram) scan. This is like an ultrasound scan for the heart. Another is an endomyocardial biopsy. A tiny piece of their heart tissue is removed (biopsy). A flexible hollow tube (catheter) goes into the blood vessels up to the heart. Then, a small device on the end of the catheter takes a tiny piece of heart tissue (about the size of a pencil tip). Another is a cardiac MRI. This takes pictures of the inside of the heart using a powerful magnet. Another is a cardiopulmonary exercise test (CPET). This involves moving a specially designed set of bicycle pedals using hands and arms. This will check how the lungs, heart and muscles are affected during exercise. After the 12-month visit, people will visit the clinic every few months for up to a few years.
This is an open-label extension (OLE) study designed to evaluate the long-term safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and clinical effects of subcutaneous (SC) administration of CTI-1601, also known as nomlabofusp, in subjects with Friedreich's ataxia (FRDA). The objectives of this OLE study are: * To evaluate the safety of long-term subcutaneous (SC) administration of CTI-1601 in subjects with FRDA * To evaluate the PK of long-term subcutaneous (SC) administration of CTI-1601 in subjects with FRDA * To evaluate the effect of long-term subcutaneous (SC) administration of CTI-1601 in subjects with FRDA on: * Tissue FXN concentrations * Clinical evaluations of FRDA * Gene Expression and select lipids
Spinocerebellar ataxias are a group of disorders that cause severe disability and can be fatal. There are currently no known disease-modifying treatments available for use, and there is a critical need to find treatments that slow disease progression and allow affected individuals to live more functional lives. Aerobic training show promise as a treatment for these diseases, but it is unclear if training induces neuroplastic changes within the damaged cerebellum to enhance motor learning, or if improvements are primarily caused by changes in leg strength, fatigue, and endurance. It is crucial to understand how the training impacts the brain, and particularly the cerebellum, in order to determine the most effective training regimen. To examine the impact of aerobic exercise on the brain, we propose using eyeblink conditioning, a form of motor learning that is dependent on the cerebellum. We will utilize BlinkLab, a newly developed smartphone application, that overcomes the typical barriers of testing eyeblink conditioning by allowing in-home assessments without the need for expensive equipment. We hypothesize that: 1) individuals with spinocerebellar ataxia will have impaired eyeblink conditioning, and 2) aerobic exercise, but not balance training, will improve eyeblink conditioning in this population. If these hypotheses are found to be true, it would further support that aerobic exercise is able to enhance motor learning in individuals with cerebellar damage. In AIM 1, we will test eyeblink conditioning in individuals with ataxias and follow them over time to see if eyeblink conditioning might be a biomarker for cerebellar ataxia disease progression. We will then use these preliminary results to devise a larger study to further validate eyeblink conditioning as a biomarker for ataxia disease progression. In AIM 2, we will determine the impact of training on eyeblink conditioning. We expect that aerobic training, but not balance training, will enhance eyeblink conditioning in spinocerebellar ataxia. Finally, in AIM 3, we will explore the use of eyeblink conditioning as a biomarker of neuroplasticity.
In this study, researchers will learn more about BIIB141, also known as omaveloxolone or SKYCLARYS®. This drug has been approved, or made available for doctors to prescribe, for people with Friedrich's Ataxia (FA) who are at least 16 years old. But, it is not yet available for children and teens with FA who are younger than 16 years old. The main objective of this study is to learn how BIIB141 is processed in the body of children and teens who are 2 to 15 years old. The main question researchers want to answer in this study is: * How does the body process BIIB141 in children and teens? * How many participants have medical problems during the study? * Are there any changes in the participants' overall health during the study? * Are there any changes in the participants' heart health? * Are there any changes in how the participants move through puberty? Puberty is the time in someone's life when their body changes from a child to an adult. This study will be done as follows: * Participants will be screened to see if they can join the study. The screening period will be up to 14 days, after which participants will check into their study research center. * There are 2 parts to this study. During Part 1, participants will take a single dose of BIIB141. Participants will be in 1 of 7 different groups based on their age: * Group A1: 12 to 15 years old, taking 150 milligrams (mg) of BIIB141 * Group A2: 12 to 15 years old, taking a dose of BIIB141 based on the data from Group A1 * Group B1: 7 to 11 years old, taking a dose of BIIB141 based on Group A1 data * Group C1: 2 to 6 years old, taking a dose of BIIB141 based on Groups A1, A2, and B1 data * Group A3: 12 to 15 years old, taking a dose of BIIB141 based on Groups A1, A2, and B1 data * Group B2: 7 to 11 years old, taking a dose of BIIB141 based on Groups A1, A2, and B1 data * Group C2: 2 to 6 years old, taking a dose of BIIB141 based on Group A1, A2, A3, B1, B2, and C1 data. * During Part 2, participants from Part 1 will take BIIB141 once in the study research center. Cohort A1 will take 150 mg of BIIB141. Dose of Cohorts A2 and B1 will be based on data from Cohort A1, dose of Cohorts C1, A3 and B2 will be based on data from Cohorts A1, A2 and B1, while Cohort C2's dose will be based on all the other groups. Participants will then take it once a day at home. * After leaving the study research center in Part 2, participants will return for tests at Week 4, Week 12, Week 24, and then every 24 weeks. Participants will also be contacted by telephone at Week 2, Week 8, and Week 18. * Participants will be in this study for up to 240 weeks.
This project is a global, multicenter, prospective, longitudinal, observational natural history study that can be used to understand the disease progression and support the development of safe and effective drugs and biological products for Friedreich ataxia.