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25 Cerebellar Ataxia Trials
Power is an online platform that helps thousands of Cerebellar Ataxia patients discover FDA-reviewed trials every day. Every trial we feature meets safety and ethical standards, giving patients an easy way to discover promising new treatments in the research stage.
CTI-1601 for Friedreich's Ataxia
Columbus, OhioThis 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
No Placebo Group
Trial Details
Trial Status:Enrolling By Invitation
Trial Phase:Phase 2
Age:18+
Sex:All
75 Participants Needed
Omaveloxolone for Friedreich's Ataxia
Columbus, OhioThis trial is testing omaveloxolone, a drug that may help people with Friedreich's ataxia by boosting their body's natural defenses against cell damage. The study focuses on patients with this genetic condition because they have weakened antioxidant systems. Omaveloxolone works by activating a protein that helps protect cells from damage. It aims to improve cell function and reduce harmful effects in people with Friedreich's ataxia.
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Age:16 - 40
Sex:All
172 Participants Needed
EryDex for Ataxia Telangiectasia
Cincinnati, OhioThis is an international, multi-center, randomized, prospective, double-blind, placebo-controlled, Phase 3 study, designed to assess the effect of EryDex (dexamethasone sodium phosphate \[DSP\] in autologous erythrocytes), administered by intravenous (IV) infusion once every 28 days, on neurological symptoms of patients with Ataxia Telangiectasia (A-T).
Pivotal Trial
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 3
Age:6+
Sex:All
Key Eligibility Criteria
Disqualifiers:Immune Impairment, Neoplastic Disease, Others
Must Not Be Taking:Steroids
106 Participants Needed
ASP2016 + Prednisolone for Friedreich Ataxia
Cincinnati, OhioFriedreich 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.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1
Age:18 - 40
Sex:All
14 Participants Needed
Troriluzole for Spinocerebellar Ataxia
Ann Arbor, MichiganThis trial is testing whether a medication called Troriluzole can help people with spinocerebellar ataxia by balancing a brain chemical to prevent damage. Troriluzole is related to riluzole, which has been shown to prolong survival and slow functional deterioration in patients with ALS.
Pivotal Trial
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:18 - 75
Sex:All
299 Participants Needed
Nomlabofusp for Friedreich's Ataxia
Chevy Chase, MarylandThe 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).
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1
Age:2 - 17
Sex:All
Key Eligibility Criteria
Disqualifiers:Compound Heterozygous FRDA, Cardiac Condition, Others
Must Not Be Taking:Omaveloxolone
30 Participants Needed
Visual Feedback Reach Training for Ataxia
Baltimore, MarylandThis trial compares two training methods to improve arm movements in people with cerebellar ataxia. It targets individuals who struggle with movement coordination due to cerebellum damage. The methods involve practicing reaching movements with feedback to enhance coordination.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Unphased
Age:22 - 80
Sex:All
Key Eligibility Criteria
Disqualifiers:Dementia, Vision Loss, Others
18 Participants Needed
Home Exercise for Neurodegenerative Disease
Baltimore, MarylandThe primary goal of this study is to address the need for targeted therapeutic interventions for impairments that impact walking in related neurodegenerative diseases.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Unphased
Age:5 - 75
Sex:All
Key Eligibility Criteria
Disqualifiers:Uncontrolled Hypertension, Diabetes, Dementia, Others
30 Participants Needed
TMS and MRI for Cerebellar Ataxia
Madison, WisconsinThis study will investigate the how the cerebellum is involved in speech motor learning over time and short-term corrections in patients with cerebellar ataxia and healthy controls. This will be accomplished through three approaches: behavioral studies, magnetic resonance imaging (MRI), and transcranial magnetic stimulation (TMS). During behavioral studies, participants will be asked to speak into a microphone while their voice is played back over earphones, and to do other speaking tasks. MRI will be acquired to perform a detailed analysis on brain function and anatomy related to speech and the cerebellum. In healthy controls, TMS will also be performed to temporarily disrupt the cerebellum before, during, or after the participant performs speaking tasks. Patients with cerebellar ataxia and healthy volunteers will be asked to complete behavioral studies and/or MRI; healthy volunteers may be asked to additionally participate in TMS.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Unphased
Age:18 - 75
Sex:All
660 Participants Needed
Vatiquinone for Friedreich Ataxia
Philadelphia, PennsylvaniaThis trial is testing the long-term safety and effectiveness of vatiquinone in people with Friedreich ataxia who have already used this medication. The goal is to ensure it remains safe and helpful over an extended period. Idebenone, a predecessor to vatiquinone, has been used in Friedreich's ataxia patients and shown positive effects on cardiac hypertrophy.
No Placebo Group
Pivotal Trial
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:All
Sex:All
130 Participants Needed
Gene Therapy for Cardiomyopathy in Friedreich's Ataxia
Philadelphia, PennsylvaniaThis is a Phase 1/2, open-label, dose-ascending, multicenter study of the safety and efficacy of LX2006 for participants who have Friedreich's Ataxia with evidence of cardiomyopathy. The study will evaluate up to three doses of single administration of LX2006 (AAVrh.10hFXN), an adeno-associated virus (AAV) gene therapy designed to intravenously deliver the human frataxin (hFXN) gene to cardiac cells over a 52-week period. Long-term safety and efficacy will be evaluated for an additional 4-years for a total of 5-years post LX2006 treatment.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 1, 2
Age:18 - 50
Sex:All
8 Participants Needed
Omaveloxolone for Friedreich's Ataxia
Philadelphia, PennsylvaniaIn 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 4 different groups based on their age:
* Group 1A: 12 to 15 years old, taking 150 milligrams (mg) of BIIB141
* Group 1B: 12 to 15 years old, taking a dose of BIIB141 based on the data from Group 1A
* Group 2: 6 to 11 years old, taking a dose of BIIB141 based on Group 1 data
* Group 3: 2 to 5 years old, taking a dose of BIIB141 based on Group 2 data
* During Part 2, participants from Part 1 will take BIIB141 once in the study research center. Their dose will be based on what they could safely take during Part 1. 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.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1
Age:2 - 15
Sex:All
Key Eligibility Criteria
Disqualifiers:Uncontrolled Diabetes, Heart Disease, Liver Disease, Others
Must Not Be Taking:CYP3A4 Inhibitors, CYP3A4 Inducers
20 Participants Needed
TMS for Cerebellar Ataxia
Elkins Park, PennsylvaniaThis trial aims to see if brain activity can predict and improve motor learning in people with cerebellar ataxia. It uses non-invasive brain stimulation to adjust brain activity and tests if this helps patients learn new movement skills better.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Unphased
Age:18 - 80
Sex:All
12 Participants Needed
Exercise for Spinocerebellar Ataxias
New York, New YorkSpinocerebellar 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.
No Placebo Group
Trial Details
Trial Status:Not Yet Recruiting
Trial Phase:Unphased
Age:18 - 65
Sex:All
30 Participants Needed
Exercise Therapy for Cerebellar Ataxia
New York, New YorkBalance 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.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Unphased
Age:18+
Sex:All
48 Participants Needed
Physical Activity Coaching for Ataxia
New York, New YorkEngage-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.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 1
Age:18 - 85
Sex:All
30 Participants Needed
Gene Therapy for Cardiomyopathy in Friedreich's Ataxia
New York, New YorkThe purpose of this study is to test the safety and preliminary efficacy of AAVrh.10hFXN to treat the cardiomyopathy associated with Friedreich's ataxia (FA). AAVrh.10hFXN is a serotype rh.10 adeno-associated virus gene transfer vector coding for Frataxin (FXN). The drug is administered intravenously. This is a phase 1, open label, dose escalation study with a total of 25 participants.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1
Age:18 - 50
Sex:All
Key Eligibility Criteria
Disqualifiers:Diabetes, Heart Failure, Hypertension, Others
Must Not Be Taking:Corticosteroids, Immunosuppressives
25 Participants Needed
Exercise Intensity and Balance Training for Spinocerebellar Ataxia
New York, New YorkThis trial is testing two different online exercise routines for people with spinocerebellar ataxias (SCAs). One routine involves intense cardio workouts before balance exercises, while the other involves light exercises like stretching before balance exercises. The goal is to see which routine better improves balance and motor skills in people with SCAs.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Unphased
Age:18 - 85
Sex:All
20 Participants Needed
Transcranial Magnetic Stimulation for Schizophrenia
New York, New YorkThis is a single-site, sham-controlled, randomized trial in a total of 60 subjects between ages 18 and 40 years with schizophrenia. This study will investigate the effects of 4-week rTMS treatment on brain and cognitive functions in patients. Subjects will be randomized to one of the following arms:
Arm 1: Standard of Care (SOC) and active rTMS Arm 2: Standard of Care (SOC) and sham rTMS
Each participant will receive rTMS five days per week, for four consecutive weeks. Functional magnetic resonance imaging (fMRI) scans, clinical assessments, and cognitive tests will be performed at baseline, end of the 2nd week, and end of the 4th week.
Trial Details
Trial Status:Recruiting
Trial Phase:Unphased
Age:18 - 40
Sex:All
Key Eligibility Criteria
Disqualifiers:Substance Abuse, Seizure History, Others
Must Be Taking:Antipsychotics
60 Participants Needed
ARO-ATXN2 for Spinocerebellar Ataxias
Montréal, QuebecAdult participants with spinocerebellar ataxia type 2 (SCA2) who carry ≥33 cytosine, adenine, guanine (CAG) repeats in the ATXN2 gene, and who have met all protocol eligibility criteria will be randomized to receive a single dose of ARO-ATXN2 or placebo and be evaluated for safety, tolerability, pharmacokinetic (PK) and pharmacodynamic (PD) parameters.
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1
Age:18 - 65
Sex:All
Key Eligibility Criteria
Disqualifiers:Uncontrolled Hypertension, Cardiac, Liver, Renal, Others
36 Participants Needed
Rehabilitation Program for Spastic Ataxia
Montréal, QuebecSpastic ataxias are a group of diseases causing symptoms such as walking difficulties and balance impairments that lead to a high risk of falls. No pharmacological treatments exist to treat these diseases. Unfortunately, little effort is made to develop non-pharmacological treatments specific to spastic ataxias despite the detrimental impact of the disease on several aspects of an individual's life and the high cost of falls for society each year. The three objectives of this project are: 1) to determine the effect of a 12-week rehabilitation program on disease severity as compared with usual care for individuals with spastic ataxias; 2) to identify which factors can help (or not) the implementation of the program in the clinical settings ("reel world"); and 3) to explore the cost-benefits of IMPACT \[rehabIlitation prograM for sPAstiC aTaxias\]. The team has developed the program to specifically target symptoms present in these patients and was previously pilot-tested. Based on the results obtained in this pilot project, positive effects are expected concerning the disease severity of participants. The investigators want, with this project, provide to health care professionals an option to offer better-suited services to people living with spastic ataxia worldwide.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Unphased
Age:16+
Sex:All
Key Eligibility Criteria
Disqualifiers:Rehab Program, Physical Limitations, Pregnancy, Others
84 Participants Needed
N-Acetyl-L-Leucine for A-T
Boston, MassachusettsA 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.
Pivotal Trial
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 3
Age:4+
Sex:All
60 Participants Needed
Respiratory Training for Friedreich's Ataxia
Gainesville, FloridaThis 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.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Unphased
Age:18 - 65
Sex:All
8 Participants Needed
Stem Cell Transplant for Autoimmune Neurological Diseases
Denver, ColoradoThis trial tests a combination of strong chemotherapy and an immune-suppressing medicine followed by a stem cell transplant. It targets patients with autoimmune neurological diseases that haven't improved with other treatments. The treatment aims to weaken the immune system to stop it from attacking the nervous system and then helps the body recover by making new blood cells. High-dose chemotherapy followed by a stem cell transplant has been shown to improve various autoimmune diseases.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Age:< 71
Sex:All
53 Participants Needed
IB1001 for Ataxia
Los Angeles, CaliforniaThis is a multinational, multicenter, open-label, rater-blinded prospective Phase II study which will assess the safety and efficacy of N-Acetyl-L-Leucine (IB1001) for the treatment of Ataxia-Telangiectasia (A-T).
There are two phases to this study: the Parent Study, and the Extension Phase.
The Parent Study evaluates the safety and efficacy of N-Acetyl-L-Leucine (IB1001) for the symptomatic treatment of A-T.
The Extension Phase evaluates the long-term safety and efficacy of IB1001 for the neuroprotective, disease-modifying treatment of A-T.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:6+
Sex:All
39 Participants Needed
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Frequently Asked Questions
How much do Cerebellar Ataxia clinical trials pay?
Each trial will compensate patients a different amount, but $50-100 for each visit is a fairly common range for Phase 2–4 trials (Phase 1 trials often pay substantially more). Further, most trials will cover the costs of a travel to-and-from the clinic.
How do Cerebellar Ataxia clinical trials work?
After a researcher reviews your profile, they may choose to invite you in to a screening appointment, where they'll determine if you meet 100% of the eligibility requirements. If you do, you'll be sorted into one of the treatment groups, and receive your study drug. For some trials, there is a chance you'll receive a placebo. Across Cerebellar Ataxia trials 30% of clinical trials have a placebo. Typically, you'll be required to check-in with the clinic every month or so. The average trial length for Cerebellar Ataxia is 12 months.
How do I participate in a study as a "healthy volunteer"?
Not all studies recruit healthy volunteers: usually, Phase 1 studies do. Participating as a healthy volunteer means you will go to a research facility several times over a few days or weeks to receive a dose of either the test treatment or a "placebo," which is a harmless substance that helps researchers compare results. You will have routine tests during these visits, and you'll be compensated for your time and travel, with the number of appointments and details varying by study.
What does the "phase" of a clinical trial mean?
The phase of a trial reveals what stage the drug is in to get approval for a specific condition. Phase 1 trials are the trials to collect safety data in humans. Phase 2 trials are those where the drug has some data showing safety in humans, but where further human data is needed on drug effectiveness. Phase 3 trials are in the final step before approval. The drug already has data showing both safety and effectiveness. As a general rule, Phase 3 trials are more promising than Phase 2, and Phase 2 trials are more promising than phase 1.
Do I need to be insured to participate in a Cerebellar Ataxia medical study ?
Clinical trials are almost always free to participants, and so do not require insurance. The only exception here are trials focused on cancer, because only a small part of the typical treatment plan is actually experimental. For these cancer trials, participants typically need insurance to cover all the non-experimental components.
What are the newest Cerebellar Ataxia clinical trials ?
Most recently, we added Exercise for Spinocerebellar Ataxias, N-Acetyl-L-Leucine for A-T and ARO-ATXN2 for Spinocerebellar Ataxias to the Power online platform.