Clinical trials: Degenerative Brain Disorder
Frontotemporal Dementia, Alzheimer Disease
Young Onset Dementia - the Difficult Diagnosis and the Stressful Life for the Whole Family
People diagnosed with young onset dementia are today mostly assigned to the same healthcare services as people developing dementia at an older age.
- Trial name
- NCT02055092
- Where
- Norway
- Volunteers
- 250 people
- Sponsor
- Norwegian Centre for Ageing and Health
Canavan Disease
A Study of AAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial)
The main objective of this trial is to evaluate the safety, tolerability, and pharmacodynamic activity of BBP-812, an investigational AAV9-based gene therapy, in pediatric participants with Canavan disease.
- Trial name
- NCT04998396
- Testing
- AAV9 BBP-812
- Where
- 4 sites in United States
- Volunteers
- 26 people
- Sponsor
- Aspa Therapeutics
Friedreich Ataxia
Digital Outcome Assessment Using AI Active Gaming and Motion Capture in Friedreich Ataxia
Friedreich ataxia (FA) is a rare, inherited condition that progressively affects balance, coordination, and walking. Clinical trials of new FA treatments rely largely on standard clinical rating scales, but these can be tiring…
- Trial name
- STEP-OUT FA (NCT07731971)
- Testing
- Laboratory validation session (VSimulator), Home-based active gaming assessment
- Where
- United Kingdom
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- University of Exeter
Friedreich Ataxia
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…
- Trial name
- BOLD (NCT06054893)
- Testing
- Omaveloxolone
- Where
- United States
- Volunteers
- 33 people
- Sponsor
- Biogen
Amyotrophic Lateral Sclerosis, Frontotemporal Dementia
Genetic Study of Familial and Sporadic ALS/Motor Neuron Disease, Miyoshi Myopathy and Other Neuromuscular Disorders
The investigators laboratory has been studying families with a history of ALS for more than 30 years and is continuing to use new ways to understand how genes may play a role in ALS, motor neuron disease and other neuromuscular…
- Conditions
- Amyotrophic Lateral Sclerosis, Frontotemporal Dementia, PLS, Motor Neuron Disease
- Trial name
- NCT01459302
- Where
- United States
- Volunteers
- healthy volunteers welcome
- Sponsor
- University of Massachusetts, Worcester
Ataxia, Gait
Ataxia GAA-FGF14 - Descriptive Genetic and Clinical Study
Cerebellar ataxias of late onset are of undetermined etiology in many cases. A new cause of late-onset cerebellar ataxia was discovered in January 2023 corresponding to an expansion of GAA triplets in intron 1 of the FGF14 gene.
- Trial name
- GAA-FGF14 (NCT05884086)
- Where
- France
- Volunteers
- 20 people
- Sponsor
- Central Hospital, Nancy, France
Motor Neuron Diseases, Spinocerebellar Ataxias
Rare Disease Registry Database for Movement Disorders in Southern Anhui Province
According to the World Health Organization (WHO) definition, a rare disease is a disease affecting fewer than 6.5 people per 10,000; in China, it generally refers to diseases with a prevalence of less than 1/10,000 (i.e., with a…
- Conditions
- Motor Neuron Diseases, Spinocerebellar Ataxias, Hereditary Muscular Dystrophy, Hereditary Spastic Paraplegia
- Trial name
- W-MoSCA (NCT07838883)
- Where
- China
- Volunteers
- 180 people
- Sponsor
- First Affiliated Hospital of Wannan Medical College
Friedreich Ataxia
Efficacy Study of Epoetin Alfa in Friedreich Ataxia
Friedreich's ataxia (FRDA) is a rare genetic disorder characterised by severe neurological disability and cardiomyopathy. Friedreich's ataxia is the consequence of frataxin deficiency.
- Trial name
- FRIEMAX (NCT01493973)
- Testing
- Epoetin alfa
- Where
- 3 sites in Italy
- Volunteers
- 56 people
- Sponsor
- Federico II University
Behavioral Variant Frontotemporal Dementia (bvFTD)
Safety and Efficacy Study Evaluating TRx0237 in Subjects With Behavioral Variant Frontotemporal Dementia (bvFTD)
The purpose of this study is to demonstrate the safety and efficacy of TRx0237 in the treatment of patients with behavioral variant frontotemporal dementia (bvFTD).
- Trial name
- NCT01626378
- Testing
- TRx0237
- Where
- 67 sites in United States, Australia and 10 more
- Volunteers
- 220 people
- Sponsor
- TauRx Therapeutics Ltd
Spinocerebellar Ataxia
Safety and Efficacy of Intravenous Immune Globulin in Treating Spinocerebellar Ataxia
This is a preliminary study to determine the safety and efficacy of intravenous immune globulin in treating Spinocerebellar Ataxia.
- Trial name
- NCT01350440
- Testing
- IVIG
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- University of South Florida
Friedreich Ataxia
A Study to Learn More About the Long-Term Safety of BIIB141 (Omaveloxolone) in Participants With Friedreich's Ataxia Who Are Prescribed it by Their Own Doctors
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.
- Trial name
- NCT06623890
- Testing
- Omaveloxolone
- Where
- 14 sites in United States, Austria and 5 more
- Volunteers
- 300 people
- Sponsor
- Biogen
Friedreich Ataxia
FRDA Investigator Initiated Study (IIS) With Elamipretide
To evaluate the safety, tolerability, and activity of Elamipretide in treating vision loss in Friedreich Ataxia (FRDA).
- Trial name
- ELViS-FA (NCT05168774)
- Testing
- Elamipretide
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Children's Hospital of Philadelphia
Friedreich Ataxia
Efficacy of Stabilometric Platform to Improve Standing Balance in Patients With Friedreich's Ataxia
The primary objective is to evaluate the potential effectiveness of an individualized intensive rehabilitation intervention using the "Prokin 252" stabilometric platform in the treatment of adolescent and adult patients with…
- Trial name
- NCT06692296
- Testing
- Balance training over stabilometric platform, Balance training by means of conventional rehabilitation
- Where
- Italy
- Volunteers
- 24 people
- Sponsor
- IRCCS Eugenio Medea
Friedreich's Ataxia
Patient Registry of the European Friedreich's Ataxia Consortium for Translational Studies (EFACTS)
This is a multi-centre, multi-national, prospective, observational study of Friedreich's Ataxia (FRDA) with a control group to: obtain natural history data on individuals affected by FRDA relate clinical assessments and results…
- Trial name
- EFACTS (NCT02069509)
- Where
- 16 sites in Austria, Belgium and 8 more
- Volunteers
- 1,200 people, healthy volunteers welcome
- Sponsor
- European Friedreich's Ataxia Consortium for Translational Studies
Wilson's Disease
A Phase 1 Study to Assess the Effects in the Body of a Single Dose of Trientine Dihydrochloride in Wilson's Disease Patients
To evaluate the effects of a single dose of trientine in children ≥6 years and adult patients with Wilson's disease.
- Trial name
- NCT01874028
- Testing
- trientine dihydrochloride
- Where
- Germany
- Volunteers
- 20 people
- Sponsor
- Univar BV
Friedreich's Ataxia
Biomarkers in Friedreich's Ataxia
The purpose of this project is to characterize measures of cardiac performance and neuromuscular physiology in FA patients using novel techniques, including echocardiography and magnetic resonance imaging (MRI), metabolic…
- Trial name
- NCT02497534
- Where
- United States
- Volunteers
- 203 people, healthy volunteers welcome
- Sponsor
- University of Florida
Wilson's Disease
A Registered Cohort Study on Wilson's Disease
The aim of this study is to determine the clinical spectrum and natural progression of Wilson's Disease in a prospective multicenter natural history study, to assess the clinical, genetic, epigenetic features and biomarkers of…
- Trial name
- NCT04012658
- Testing
- No intervention
- Where
- China
- Volunteers
- 2,000 people, healthy volunteers welcome
- Sponsor
- Wan-Jin Chen
Friedreich's Ataxia
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.
- Trial name
- PROVE-FA (NCT07681713)
- Testing
- Vatiquinone
- Where
- 9 sites in United States, Belgium and 4 more
- Volunteers
- 120 people
- Sponsor
- PTC Therapeutics
Pick Disease of the Brain
Far Infrared Irradiation for the Management, Control and Treatment of Frontotemporal Dementia
Pick's disease, also known as Pick disease,or FTD is a rare fronto-temporal neurodegenerative disease. This study will investigate the use of far infrared radiation for the control, management and treatment of Pick's disease.
- Trial name
- NCT00674960
- Testing
- Radiation: Far Infrared Radiation (5μm to 20μm wavelength)
- Where
- Canada
- Sponsor
- GAAD Medical Research Institute Inc.
Primary Progressive Aphasia, Frontotemporal Dementia
Communication Bridge Speech Therapy Research Study
This is a study on Internet-based video-practice speech and language therapy for persons with primary progressive aphasia (PPA), behavioral-variant frontotemporal dementia (bvFTD), or related conditions.
- Trial name
- CB (NCT02439853)
- Testing
- Monthly Check-in Session
- Where
- United States
- Volunteers
- 57 people
- Sponsor
- University of Chicago