Clinical trials: Cerebellar Ataxia, open to volunteers
Autosomal Recessive Cerebellar Ataxia
Validation of the RADIAL Algorithm for Diagnosis of Autosomal Recessive Cerebellar Ataxia
RADIAL is an algorithm which has been developed following a review of the literature on 67 autosomal recessive cerebellar ataxias (ARCA) and personal clinical experience.
- Trial name
- RADIAL-VALID (NCT04261127)
- Testing
- Genetic diagnosis (PMDA panel), Use of RADIAL algorithm
- Where
- 8 sites in France
- Volunteers
- 400 people, healthy volunteers welcome
- Sponsor
- University Hospital, Strasbourg, France
Ataxia
Impact of Foot Orthoses on Gait in Individuals With Ataxia
This study is the first to assess the impact of lower extremity orthoses on gait and balance in individuals with CA. By investigating how orthoses influence mobility and stability, this research will provide critical data to…
- Trial name
- NCT07720700
- Testing
- Home wearing schedule
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- University of Texas Southwestern Medical Center
CANVAS Syndrome
Pathogenic Insights and Search for Biomarkers in RFC1-ataxia/CANVAS
CANVAS (Cerebellar Ataxia, Neuropathy, Vestibular Areflexia Syndrome), also referred to as RFC1-ataxia, is a recently molecularly characterized neurodegenerative disorder caused by a biallelic expansion of an AAGGG…
- Trial name
- INSIDE-CANVAS (NCT07156214)
- Testing
- Assessment of markers of oxidative stress in peripheral blood samples
- Where
- 2 sites in Italy
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- Catholic University of the Sacred Heart
PolG, Primary Mitochondrial Myopathy
Prospective Natural History Study of POLG Disease
The PIONEER study is a prospective, natural history study dedicated to characterizing the clinical progression of POLG-related disorders.
- Trial name
- PIONEER (NCT07775872)
- Where
- 21 sites in United States, Australia and 7 more
- Volunteers
- 300 people
- Sponsor
- The POLG Foundation
Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia Type 2
Natural History Study of and Genetic Modifiers in Spinocerebellar Ataxias
Spinocerebellar ataxias (SCA) are genetic neurological diseases that cause imbalance, poor coordination, and speech difficulties.
- Conditions
- Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia Type 2, Spinocerebellar Ataxia Type 3, Spinocerebellar Ataxia Type 6
- Trial name
- NCT01060371
- Testing
- Genetic Testing, Blood Collection, Magnetic Resonance Imaging (MRI) Scan
- Where
- 17 sites in United States, Canada
- Volunteers
- 1,400 people, healthy volunteers welcome
- Sponsor
- Lauren Moore
Friedreich Ataxia
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…
- Trial name
- NCT06447025
- Testing
- CTI-1601
- Where
- 10 sites in United States
- Volunteers
- 85 people
- Sponsor
- Larimar Therapeutics, Inc.
Dyssynergia, Constipation
Fecobionics in Biofeedback Therapy in Dyssynergia Patients
Constipation affects 12-19% of Americans. Pelvic floor dyssynergia is considered to play an important role in constipation but the underlying mechanisms are not well understood in individual patients.
- Trial name
- NCT05807321
- Testing
- Fecobionics
- Where
- 2 sites in United States
- Volunteers
- 24 people
- Sponsor
- The California Medical Innovations Institute, Inc.
Pancreas Cancer, Peutz-Jeghers Syndrome (PJS)
The Cancer of the Pancreas Screening-5 CAPS5)Study
Johns Hopkins clinical research office quality assurance group will monitor and audit this study at Johns Hopkins. The Sub Investigator at each site will be responsible for internal monitoring at their site.
- Conditions
- Pancreas Cancer, Peutz-Jeghers Syndrome (PJS), Gene Mutation, Germline Mutation Carrier
- Trial name
- CAPS5 (NCT02000089)
- Testing
- Secretin, MRI, Tumor marker gene test with CA19-9
- Where
- 9 sites in United States
- Volunteers
- 9,000 people
- Sponsor
- Johns Hopkins University
Ataxia, Degenerative Disease
Rehabilitation Program on Genetic and Degenerative Ataxia
Cerebellar ataxia is a pathology linked to the lesion of the cerebellum or the afferent and/or efferent cerebellar pathways. The aetiology can be an acquired cerebral lesion, following a chemical poisoning or a genetic…
- Conditions
- Ataxia, Degenerative Disease, Genetic Disease
- Trial name
- RAPP (NCT06089863)
- Testing
- PAMPERO program
- Where
- 3 sites in France
- Volunteers
- 48 people
- Sponsor
- Hospices Civils de Lyon
Adenine Phosphoribosyltransferase Deficiency, AH Amyloidosis
National Registry of Rare Kidney Diseases
The goal of this National Registry is to is to collect information from patients with rare kidney diseases, so that it that can be used for research.
- Conditions
- Adenine Phosphoribosyltransferase Deficiency, AH Amyloidosis, AHL Amyloidosis, AL Amyloidosis
- Trial name
- RaDaR (NCT06065852)
- Where
- United Kingdom
- Volunteers
- 35,000 people
- Sponsor
- UK Kidney Association
Ataxia
Effects of Cerebellar tACS-iTBS in Ataxia
Ataxia refers to a group of neurological disorders characterized by impaired coordination and balance due to dysfunction in the cerebellum or its connections.
- Trial name
- EtABeta (NCT06420271)
- Testing
- Real (real iTBS/tACS + exergaming)
- Where
- Italy
- Volunteers
- 30 people
- Sponsor
- I.R.C.C.S. Fondazione Santa Lucia
Cerebellar Ataxia
A Registered Cohort Study on Cerebellar Ataxia in the Organization in South-East China for Cerebellar Ataxia Research (OSCCAR)
Cerebellar ataxia is a form of ataxia originating in the cerebellum. Cerebellar ataxia can occur as a result of many diseases and may present with symptoms of an inability to coordinate balance, gait, extremity and eye movements.
- Trial name
- NCT04010214
- Testing
- No intervention
- Where
- China
- Volunteers
- 1,500 people, healthy volunteers welcome
- Sponsor
- Ning Wang, MD., PhD.
Mitochondrial Disorders, Mitochondrial Genetic Disorders
North American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC)
The North American Mitochondrial Disease Consortium (NAMDC) maintains a patient contact registry and tissue biorepository for patients with mitochondrial disorders.
- Conditions
- Mitochondrial Disorders, Mitochondrial Genetic Disorders, Mitochondrial Diseases, Disorder of Mitochondrial Respiratory Chain Complexes
- Trial name
- NAMDC (NCT01694940)
- Where
- 17 sites in United States, Canada
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- Columbia University
Spinocerebellar Ataxia Type 27B
Spinocerebellar Ataxia Type 27B Natural History Study (SCA27B-NHS)
This international, multi-center, multi-modal, and prospective observational cohort study aims to validate trial outcomes for capturing disease progression in Spinocerebellar Ataxia Type 27B (SCA27B), with combined multi-modal…
- Trial name
- NCT06472557
- Where
- 5 sites in Czechia, Germany and 2 more
- Volunteers
- 300 people, healthy volunteers welcome
- Sponsor
- University Hospital Tuebingen
Friedreich Ataxia, Friedreich's Ataxia
A Study to Evaluate the Efficacy and Safety of Subcutaneous Nomlabofusp in Subjects With Friedreich's Ataxia
To evaluate the efficacy and safety of subcutaneous nomlabofusp in adult and pediatric subjects with Friedreich's ataxia
- Trial name
- FORWARD-FA (NCT07778836)
- Testing
- Nomlabofusp
- Where
- 4 sites in United States
- Volunteers
- 150 people
- Sponsor
- Larimar Therapeutics, Inc.
Autosomal Recessive Spastic Ataxia of Charlevoix-Saguenay, Spastic Paraplegia 7
Effects of a Supervised Rehabilitation Program on Disease Severity in Spastic Ataxias
Spastic 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.
- Trial name
- NCT06261424
- Testing
- IMPACT - rehabIlitation prograM for sPAstiC aTaxias
- Where
- 5 sites in Canada
- Volunteers
- 84 people
- Sponsor
- Laval University
CLIFAHDD, Congenital Contractures of the Limbs and Face, Hypotonia, and Developmental Delay
Aprepitant for the Treatment of CLIFAHDD
There are no drug treatments for individuals with Congenital Contractures of the Limb and Face, Hypotonia, and Developmental Delay (CLIFAHDD), an ultra-rare and severe neurodevelopmental disease.
- Conditions
- CLIFAHDD, Congenital Contractures of the Limbs and Face, Hypotonia, and Developmental Delay, NALCN Channelopathy, Ataxia - Other
- Trial name
- NCT07845825
- Testing
- Aprepitant Powder for Oral Suspension
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- The University of Texas Health Science Center at San Antonio
Ataxia, Parkinson Disease
Home-based Gait and Balance Training in Patients With Movement Disorders
The research project is an experimental study with three study visits at the study site at the University Hospital Düsseldorf (UKD) / Heinrich Heine University Düsseldorf (HHU) and a three-week training phase in a parallel…
- Trial name
- NCT06617884
- Testing
- Gait and balance training
- Where
- Germany
- Volunteers
- 80 people
- Sponsor
- Forschungszentrum Juelich
Cerebellar Ataxia, Cerebellar Diseases
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.
- Conditions
- Cerebellar Ataxia, Cerebellar Diseases, Cerebellar Stroke, Cerebellar Tumor
- Trial name
- IMPACT (NCT07642856)
- Testing
- Therapy
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Friedreich's Ataxia
A Study to Learn More About Friedrich's Ataxia in People Living in the Gulf Cooperation Council (GCC) Region of the World
In this study, researchers will learn more about a disease called Friedrich's Ataxia, also known as FA. This is known as an "observational" study, which collects health information about study participants without changing their…
- Trial name
- NCT07887828
- Volunteers
- 40 people
- Sponsor
- Biogen