Clinical Trials for Cerebellar Disorders

Source: ClinicalTrials.gov — U.S. National Library of Medicine — A database of privately and publicly funded clinical studies conducted around the world (U.S. National Library of Medicine, public domain).

This page lists clinical trials related to Cerebellar Disorders from the ClinicalTrials.gov database.

Trial Summary

• Total studies: 50
• Recruiting: 22
• Active, not recruiting: 9
• Completed: 8
• Other: 11

Results are refreshed periodically and may not reflect the most current information available from ClinicalTrials.gov.

Recruiting Trials

NCT00018889 — Phenotype/Genotype Correlations in Movement Disorders

The goal of this protocol is to identify families with inherited movement disorders and evaluate disease manifestations to establish an accurate clinical diagnosis by using newest technological advances and investigate the underlying molecular mechanisms. Studies of inherited movement disorders in large families with good genealogical records are especially valuable. Patients with diseases of k…

NCT01401998 — ARPKD Database Study

Hepato-renal fibrocystic diseases (HRFD) is a term developed that encompasses rare diseases such as Autosomal Recessive Polycystic Kidney Disease (ARPKD), and other diseases with common features (Joubert syndrome, Bardet Biedl syndrome, Meckel-Gruber syndrome, congenital hepatic fibrosis (CHF), Caroli syndrome (CS), polycystic liver disease, oro-facial-digital syndrome, nephronophithisis (NPHP)…

NCT04446442 — Cerebellar tDCS in Children With Autism Spectrum Disorder

The purpose of this research study is to investigate whether tDCS to the cerebellum (specifically, the right crus I/II area of the cerebellum) of children and young adults with autism spectrum disorders (ASD) is safe and to examine its effects on some of the symptoms of ASD, such as repetitive behaviors and hyperactivity.

NCT03879018 — Retraining Reaching in Cerebellar Ataxia

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.

NCT03911388 — HSV G207 in Children With Recurrent or Refractory Cerebellar Brain Tumors

This study is a clinical trial to determine the safety of inoculating G207 (an experimental virus therapy) into a recurrent or refractory cerebellar brain tumor. The safety of combining G207 with a single low dose of radiation, designed to enhance virus replication, tumor cell killing, and an anti-tumor immune response, will also be tested.

Funding Source- FDA OOPD

NCT05732207 — Cerebellar Involvement in Alcohol Use Disorder (AUD)

The goal of this observational and interventional study is to better understand the involvement of the cerebellum in the brain reward system in persons with alcohol use disorder (AUD). The main questions it aims to answer are:

1. What is the nature of cerebellar input to the ventral tegmental area (VTA) in the brain reward system, and how is it perturbed in AUD?
2. What is the relationship bet…

NCT06447025 — An Open-Label Study of CTI-1601 in Subjects With Friedreich’s Ataxia

An open label study designed to evaluate the safety, PK, PD, and clinical effects of long-term daily administration of CTI-1601 enrolling adolescent and adult patients with FRDA who have participated in a prior clinical study of CTI-1601 as well as children (age 2 years and older), adolescents and adults with FRDA who have not participated in a prior clinical study of CTI-1601.

NCT06122675 — Cerebellar Deep Brain Stimulation for Movement Disorders in Cerebral Palsy in Children and Young Adults

The purpose of this study is to test the safety of placing Deep Brain Stimulators (DBS) in a part of the brain called the cerebellum and using electrical stimulation of that part of the brain to treat movement symptoms related to cerebral palsy. Ten children and young adults with dyskinetic cerebral palsy will be implanted with a Medtronic Percept Primary Cell Neurostimulator. We will pilot vid…

NCT06457230 — Cerebellum-Prefrontal Paired-Pulse Stimulation for Methamphetamine Use Disorder

To investigate the mechanism and efficacy of a novel repetitive transcranial magnetic stimulation (rTMS) intervention model with paired cerebellar-medial prefrontal cortex (mPFC) pulses on methamphetamine use disorder patients and to develop a novel physiotherapeutic intervention to optimise the treatment and management.

NCT06701851 — Neural Correlates of Movement Disorders Associated With PRRT2 Related Paroxysmal Kinesigenic Dyskinesia - an Ancillary Study of AMEDYST Research

The main objective of this study is to investigate in real-time the neuronal correlates of paroxysmal dyskinesia episodes related to the PRRT2 mutation within this subgroup of patients (who can control paroxysmal dyskinesia episodes), and more specifically, the pathological role of the reciprocal influence between the striatum and the cerebellum in paroxysmal dyskinesia episodes.

NCT06993571 — Cerebellar Transcranial Alternating Current Stimulation (tACS) to Modulate Parkinson’s Disease Tremor

Parkinson’s disease (PD) is a prevalent neurodegenerative disorder characterized by different motor symptoms, including tremor, which is particularly difficult to manage. Common treatments, such as dopaminergic therapy, can have limitations in efficacy. Recent advancements in non-invasive brain stimulation, specifically phase-adaptive transcranial alternating current stimulation (tACS), offer a…

NCT06458153 — Imaging Speech in Neurotypical Adults and Individuals With Cerebellar Stroke

The goal of this research study is to learn how the brain areas that plan and control movement interact with the areas responsible for hearing and perceiving speech in healthy adults and people who have had cerebellar strokes. The main questions it aims to answer are:

1. What regions of the brain’s sensory systems show changes in their activity related to speech?
2. To what extent do these reg…

NCT07013292 — Efficacy of Omaveloxolone Treatment for Dysphagia in French Patients With Friedreich’s Ataxia

Friedreich’s ataxia (FA) is a rare, inherited neurodegenerative disease that typically begins in children and young people. It primarily affects the spinal cord, peripheral nerves and cerebellum of the brain. Clinical manifestations include progressive gait and limb ataxia, auditory and optic neuropathy, cardiomyopathy, scoliosis, dysarthria, and dysphagia. In advanced stages, individuals may b…

NCT06953583 — A Study to Learn More About the Effects and Long-Term Safety of Omaveloxolone (BIIB141) in Children and Teens With Friedreich’s Ataxia

In this study, researchers will learn more about omaveloxolone, also known as BIIB141 or SKYCLARYS®. Omaveloxolone is already approved for people with Friedreich’s Ataxia (FA) who are 16 years of age or older. However, it is not yet available for younger teens and children. The main goal of this study is to learn how omaveloxolone affects symptoms of FA and its safety in younger participants be…

NCT06865482 — Clinical Course Of Disease In Participants With FA-CM

Characteristics and clinical course of disease In participants with cardiomyopathy associated with Friedreich Ataxia (CLARITY-FA)

NCT07180355 — A Study of SGT-212 Gene Therapy in Friedreich’s Ataxia

This is a phase 1b, first in-human, open-label, dose-finding study investigating the safety and tolerability of SGT-212 in participants with Friedreich’s ataxia (FA). It will be delivered via dual intradentate nucleus (IDN) and intravenous (IV) administration to participants with FA.

All participants will receive SGT-212 and will be enrolled in the study for approximately 5 years.

NCT07483294 — Cerebellum-based Imaging Neural Markers for Antipsychotic Response

This study plans to collect 120 patients with first-episode psychosis. Participants will receive multimodal MRI scans (naturalistic fMRI, structural, diffusional) and comprehensive clinical assessments at baseline, and subsequently be treated with either risperidone or aripiprazole for 12 weeks. Clinical assessments for psychotic symptoms will be followed multiple times during the treatment unt…

NCT07619703 — Effects of a Neurorehabilitation Program Based on CIMT and Non-invasive Neuromodulation Through C-tDCS on the Recovery of Paretic Upper Limb Function, Quality of Life, and Therapeutic Alliance in Women From the Araucanía Region Who Suffered a Stroke (CIMT + tDCS).

General Objective To determine the effects of a neurorehabilitation program based on Constraint-Induced Movement Therapy (CIMT) and non-invasive neuromodulation through cerebellar transcranial direct current stimulation (C-tDCS) on the recovery of paretic upper limb function, quality of life, and therapeutic alliance in women from the Araucanía Region who suffered a stroke. Methodology An exper…

NCT07540572 — A Study to Investigate the Safety, Pharmacokinetics, and Preliminary Efficacy of IDE574 Therapy in Adult Participants With Advanced Solid Tumors

IDE574 is a synthetically manufactured small molecule inhibitor that co-targets the lysine acetyltransferase enzymes KAT6 and KAT7.

The purpose of this study is to evaluate the safety, preliminary efficacy, pharmacokinetics (PK), and pharmacodynamics (PD) of IDE574 as monotherapy in participants with locally advanced or metastatic solid tumors and as combination therapy with fulvestrant in par…

NCT07325487 — Interposed Nucleus aDBS for Ataxia

This is a single-center, open-label study designed to evaluate the feasibility, safety, and preliminary efficacy of cerebellar adaptive deep brain stimulation (aDBS) in adults with spinocerebellar ataxia type 6 (SCA6). A total of 5 participants will be enrolled.

Participants will undergo surgical implantation of deep brain stimulation (DBS) leads targeting the motor interposed nucleus of the c…

NCT07642856 — Individualized Neuro-Modulation Paired With Cerebellar Therapy

The purpose of this project is to evaluate feasibility and preliminary efficacy of pairing personalized transcranial direct current stimulation (tDCS) with individualized rehabilitation therapy in people with cerebellar damage.

NCT07215416 — Safety and Efficacy of Mutation-targeted Precision Genetic Therapy for Ataxia-Telangiectasia (A-T)

This project aims to evaluate the safety and efficacy of precision genetic therapy for patients with Ataxia-telangiectasia (A-T), a rare neurodegenerative disease caused by mutations in the ATM gene. The investigators will conduct a clinical trial to study the safety and efficacy of intrathecal administration of atipeksen, a targeted genetic therapy that restores ATM gene function in A-T indivi…

Active, Not Recruiting

NCT02994719 — Gait Analysis in Neurological Disease

The purpose of this study is to investigate whether speed-dependent measures of gait (so called gait signatures) can be identified in patients with neurological conditions that affect gait, particularly in subjects with parkinsonian disorders.

NCT02741440 — Natural History of Spinocerebellar Ataxia Type 7 (SCA7)

Background:

Spinocerebellar ataxia type 7 (SCA7) is a disease in which people have problems with coordination, balance, speech and vision. It is caused by a change in the ATXN7 gene. A mutation in this ATXN7 gene causes changes in eye cells, which can lead to vision loss. There is no cure for SCA7 but researchers are looking for possible treatments. Researchers need more information about SCA7…

NCT03701399 — Troriluzole in Adult Participants With Spinocerebellar Ataxia

The purpose of this study is to compare the efficacy of Troriluzole (200 mg once daily) versus placebo after 48 weeks of treatment in subjects with spinocerebellar ataxia (SCA).

NCT06529146 — Real-World Data Study of Troriluzole-Treated Patients With Spinocerebellar Ataxia (SCA) Compared to a Matched Natural History Control

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 torilu…

NCT03975829 — Pediatric Long-Term Follow-up and Rollover Study

A roll-over study to assess long-term effect in pediatric patients treated with dabrafenib and/or trametinib.

NCT04463979 — Perioperative Evaluation of Cerebellar Tumors

This is a prospective, cohort study to evaluate the impact of cerebellar functional topography on perioperative outcomes related to cognition and motor ataxia in patients with cerebellar tumors.

NCT06054893 — A Study to Find Out How BIIB141 (Omaveloxolone) is Processed in the Body and to Learn More About Its Safety in Participants With Friedreich’s Ataxia Aged 2 to 15 Years Old

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 …

NCT06573866 — Enhancement of Quality of Work And Life

Work participation is essential for quality of life, providing purpose, social interaction, financial security, and shaping social status. Work participation is increasingly compromised in people with slowly progressive chronic disorders (hereafter referred to as progressive disorders). This negatively impacts their quality of life. Early work-related support, focused on sustainable work-retent…

NCT06672445 — Study of ARO-ATXN2 Injection in Adults With Spinocerebellar Ataxia Type 2

Adult 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.

Not Yet Recruiting

NCT07580066 — Cerebellum and Neglect

The goal of this clinical trial is to learn if a non-invasive brain stimulation method called continuous theta burst stimulation (cTBS) can improve attention in adults who have hemispatial neglect after stroke. Hemispatial neglect is a common consequence of stroke where a person fails to notice things on one side of their environment and body after brain damage despite having intact sensory org…

NCT07621978 — Variable Intense Training for Ataxia and Locomotion

The goal of this study is to evaluate the feasibility and potential preliminary efficiency of high-intensity training focused on stepping in variable contexts as compared to conventional training in individuals with cerebellar disease

NCT07670936 — Effect of Cerebellar Fastigial Nucleus Stimulation Combined With Sling Exercise on Motor Function in Hemiplegic Stroke Patients

This study aims to investigate the rehabilitative effects and synergistic potential of combining Fastigial Nucleus Stimulation (FNS) with Sling Exercise Training (SET) on motor function in patients with post-stroke hemiplegia. Hemiplegia after stroke often results in unilateral motor impairment, balance dysfunction, and decreased proprioception. Although traditional rehabilitation methods can i…

NCT07681713 — Long-Term Efficacy Study of Vatiquinone for the Treatment of Friedreich’s Ataxia (FA)

The purpose of this study is to confirm the treatment effects of vatiquinone on the key measures of FA disease progression.

NCT07384442 — Effects of Targeted Temporal Interference Stimulation of Cerebellar Nuclei on Tremor and Gait Disturbance in Parkinson’s Disease Patients

The goal of this clinical trial is to explore the effects of cerebellar nuclei TIS stimulation on improving tremor and gait disorders in PD patients. Through randomized double-blind grouping, the differences in efficacy between TIS intervention and sham stimulation intervention for tremor and gait disorders in PD patients will be compared.

NCT07221292 — Pivotal Study of N-acetyl-L-leucine for CACNA1A

A pivotal, randomized, double-blind, placebo-controlled, multi-center therapeutic study for patients age 4 and older with a confirmed diagnosis of CACNA1A. The objective of this study is to evaluate the safety, tolerability and efficacy of N-acetyl-L-leucine (IB1001) compared to standard of care.

NCT07218575 — Double-Blind Trial of Everolimus for Improving Social Abilities in PTEN Germline Mutations

The goal of this study is to examine the safety and treatment effects of everolimus in adults and children with PTEN Hamartoma Tumor Syndrome (PHTS) who experience social difficulties. The study will measure if everolimus can safely improve social abilities and functioning in this study population.

PTEN Hamartoma Tumor Syndrome (PHTS) is a genetic condition that results from alteration (germli…

NCT07555379 — Bilateral Anodal Cerebellar tDCS for Multidomain Dysfunctions in Patients With Multiple Sclerosis

The goal of this clinical trial is to learn if brain stimulation can improve movement and daily function in people with multiple sclerosis (MS). The study will also look at how this treatment affects fatigue, sleep, memory and attention, and quality of life.

The main questions this study aims to answer are the following:

Does this treatment improve coordination and balance? Does it reduce fat…

Enrolling by Invitation

NCT07635030 — GABA and GSH in FRDA

The goal of this study is to obtain gamma-aminobutyric acid (GABA) and glutathione (GSH) assessment derived from magnetic resonance spectroscopy (MRS), to be used as a potential biomarker in patients with Friedreich Ataxia (FRDA) prior to (Aim 1), and after taking Omaveloxolone (Aim 2). Analysis will consist of:

A. Comparison of values in controls with those of FRDA patients (Aim 1) B. Longitu…

Completed Trials

NCT01860339 — Child to Adult Neurodevelopment in Gene Expanded Huntington’s Disease

Huntington’s Disease (HD) is an autosomal dominant disease manifested in a triad of cognitive, psychiatric, and motor signs and symptoms. HD is caused by a triplet repeat (CAG)expansion in the gene Huntingtin (HTT). This disease has classically been conceptualized as a neurodegenerative disease. However, recent evidence suggests that abnormal brain development may play an important role in the …

NCT03515486 — Cerebellar Stroke and Mood Disorders

Post-stroke mood disorders (PSMD), including depression, anxiety and apathy, are observed in about 30 % of stroke patients at follow-up 3 or 4 months after stroke occurrence. They impair the functional outcome of the patients and their quality of life. Among the different brain structures involved in PSMD the role of the cerebellum has been under-evaluated while it is now well-known to be invol…

NCT04346238 — Characterization of the Interruptions of the GAA Expansion and Study of Their Influence on the Severity of Friedreich’s Ataxia

Friedreich’s ataxia (FA) is the most frequent recessive genetic ataxia with an estimated prevalence of 1/50 000. The first symptoms appear around the age of 10 years with a progressive course and the need for an armchair 10- 15 years after the first symptoms. More rarely the disease can present with a late onset (after the age of 25) with a picture characterized by spastic paraparesis and slowe…

NCT05013762 — Fast Arm Motor Skill Training in Chronic Stroke Survivors

Every year, almost 800,000 people experience a stroke in the United States, which lead to upper-limb impairments, making recovery of motor function a priority in stroke rehabilitation. 1) The primary objective of this study is to determine whether fast arm movement training on a tracking task (“Speed-training”), in chronic stroke survivors with mild to moderate paresis, will generalize to impro…

NCT04874909 — Classification, Functional Stratification and Biomarkers in Ciliopathy (CILLICORIRCM)

The purpose of the C’IL-LICO RICM study is to develop innovative and transformative diagnostic and prognostic for patients suffering from ciliopathies leading to renal failure.

The objectives is to decipher disease mechanisms and highlight signaling pathways altered in at-risk to develop renal failure patient groups and to produce a prognostic biomarker-based kit to predict the evolution of ci…

NCT04921930 — Evaluation of the Effect of Artesunate in Friedreich Ataxia (FA)

This dose-escalation study is aimed at investigating a novel application for artesunate in the treatment of Friedreich ataxia. It will evaluate this novel application of oral artesunate using a surrogate biological marker as primary endpoint in a phase I-II open trial

NCT07601685 — MERC Proteins in Saliva and GCF in Periodontal Disease (ELISA Study)

This observational cross-sectional study investigates the levels of mitochondria-endoplasmic reticulum contact site (MERC) proteins in saliva and gingival crevicular fluid (GCF) of individuals with different periodontal conditions. MERCs are specialized regions where mitochondria and the endoplasmic reticulum physically connect, and they play important roles in calcium signaling, oxidative stre…

NCT06193200 — Evaluate the Neurological Effects of EryDex on Subjects With A-T

This 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).

Other (Terminated)

NCT06664853 — Open-Label Extension of EryDex Study IEDAT-04-2022

This is an international, multi-center, prospective, open-label, non-comparative study aiming to provide access to treatment with EryDex to ataxia telangiectasia (A-T) patients who completed the IEDAT-04-2022 trial which studied the neurological effects of EryDex on subjects with ataxia telangiectasia (NEAT trial).

Other (Withdrawn)

NCT06177626 — Use of a New Smartphone Application to Determine Changes in Eyeblink Conditioning From Home Training in Individuals With Spinocerebellar Ataxia

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 i…