Clinical trials: Degenerative Brain Disorder
Friedreich Ataxia
A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
The primary objective of the study is to evaluate the efficacy (using the modified Friedreich Ataxia Rating Scale [mFARS]) and safety of vatiquinone in participants with Friedreich ataxia (FA).
- Trial name
- MOVE-FA (NCT04577352)
- Testing
- Vatiquinone
- Where
- 14 sites in United States, Australia and 7 more
- Volunteers
- 146 people
- Sponsor
- PTC Therapeutics
Rare Disorders, Undiagnosed Disorders
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
The Coordination of Rare Diseases at Sanford (CoRDS) is an international patient registry that serves as a centralized resource for rare disease research.
- Conditions
- Rare Disorders, Undiagnosed Disorders, Disorders of Unknown Prevalence, Cornelia De Lange Syndrome
- Trial name
- CoRDS (NCT01793168)
- Where
- United States
- Volunteers
- 20,000 people
- Sponsor
- Sanford Health
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
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 Type 3, Machado Joseph Disease
Safety and Efficacy of Lithium Carbonate in Patients With Spinocerebellar Ataxia Type 3
Design: Phase II-III, double-blind, parallel, placebo controlled randomized Clinical trial Background: Spinocerebellar ataxia type 3 (SCA-3) is an autosomal dominant adult-onset neurodegenerative disorder for which there is no…
- Trial name
- NCT01096082
- Testing
- Lithium Carbonate
- Where
- Brazil
- Volunteers
- 62 people
- Sponsor
- Hospital de Clinicas de Porto Alegre
Primary Progressive Aphasia, Neurodegenerative Diseases
Home-Based Exercise in Primary Progressive Aphasia (HEPPA)
This pilot study aims to evaluate the feasibility of a novel home-based multicomponent exercise program in adults clinically diagnosed with Primary Progressive Aphasia
- Conditions
- Primary Progressive Aphasia, Neurodegenerative Diseases, Logopenic Progressive Aphasia, Semantic Dementia
- Trial name
- HEPPA (NCT06181500)
- Testing
- Home-based multicomponent exercise intervention program
- Where
- 2 sites in Canada
- Volunteers
- 13 people
- Sponsor
- Montreal Heart Institute
Wilson Disease
Natural History of Wilson Disease
The purpose of the registry/repository is to provide a mechanism to store data and specimens to support the conduct of future research about Wilson disease (WD).
- Trial name
- NCT03334292
- Where
- 6 sites in United States, Germany and 1 more
- Volunteers
- 300 people
- Sponsor
- Yale University
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
Hereditary Ataxia
Safety and Efficacy of Umbilical Cord Mesenchymal Stem Cell Therapy for Patients With Hereditary Ataxia
The Hereditary Ataxias are a group of genetic disorders characterized by slowly progressive incoordination of gait and often associated with poor coordination of hands, speech, and eye movements.
- Trial name
- NCT01360164
- Testing
- human umbilical cord mesenchymal stem cells
- Where
- China
- Volunteers
- 20 people
- Sponsor
- Shenzhen Beike Bio-Technology Co., Ltd.
Friedreich Ataxia, Cardiomyopathies
Phase IA and IB Study of AAVrh.10hFXN Gene Therapy for the Cardiomyopathy of Friedreich's Ataxia
The 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).
- Conditions
- Friedreich Ataxia, Cardiomyopathies, Cardiac Hypertrophy, Myocardial Fibrosis
- Trial name
- NCT05302271
- Testing
- AAVrh.10hFXN, serotype rh.10 adeno-associated virus (AAV) gene transfer vector expressing the cDNA coding for human FXN, Prednisone
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Weill Medical College of Cornell University
Friedreich Ataxia
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…
- Trial name
- ARTEMIS (NCT04921930)
- Testing
- Artesunate Oral Product
- Where
- France
- Volunteers
- 20 people
- Sponsor
- Institut National de la Santé Et de la Recherche Médicale, France
Friedreich Ataxia, Cardiomyopathy
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)
- Trial name
- NCT06865482
- Where
- 19 sites in United States, Brazil and 6 more
- Volunteers
- 65 people
- Sponsor
- Lexeo Therapeutics
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
Wilson Disease
A Clinical Study to Evaluate the Safety and Efficacy of LY-M003 Injection in Patients With Wilson Disease
Wilson's disease (WD), also known as Wilson's disease, is a rare autosomal recessive metabolic disorder caused by a mutation of the copper transport ATPase β (ATP7B) gene located on the long arm of chromosome 13 (13q14.3).
- Trial name
- WD (NCT06650319)
- Testing
- LY-M003
- Where
- China
- Volunteers
- 18 people
- Sponsor
- Chaohui Yu
Friedreich Ataxia
Neurology Measures in FA Children
The purpose of this study is to identify ways to follow progression of Friedreich's Ataxia (FA) and be able to measure changes over time in children with FA.
- Trial name
- NCT03418740
- Where
- 3 sites in United States
- Volunteers
- 108 people
- Sponsor
- Children's Hospital of Philadelphia
Friedreich Ataxia
Methylprednisolone Treatment of Friedreich Ataxia
This study will explore whether methylprednisolone treatment is safe, well-tolerated, and beneficial in patients that are diagnosed with Friedreich Ataxia (FRDA).
- Trial name
- NCT02424435
- Testing
- Methylprednisolone
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- Children's Hospital of Philadelphia
Friedreich Ataxia
A Clinical Study to Evaluate the Effect of MIN-102 on the Progression of Friedreich's Ataxia in Male and Female Patients
Randomized, double-blind, placebo-controlled study on the effects of MIN-102 on Biochemical, Imaging, neurophysiological, and clinical markers in patients with Friedreich's Ataxia
- Trial name
- FRAMES (NCT03917225)
- Testing
- MIN-102
- Where
- 5 sites in Belgium, France and 2 more
- Volunteers
- 39 people
- Sponsor
- Minoryx Therapeutics, S.L.
Wilson Disease
Role for Biochemical Assays and Kayser-Fleischer Rings in Diagnosis of Wilson Disease
The investigators aimed to identify factors associated with symptoms and features of Wilson disease from a large cohort during long-term follow-up
- Trial name
- NCT04965545
- Testing
- low copper diet
- Where
- China
- Volunteers
- 1,000 people
- Sponsor
- Second Affiliated Hospital, Zhejiang University, School of Medicine
Friedreich Ataxia
Safety and Efficacy of Etravirine in Friedreich Ataxia Patients
A drug repositioning effort provided evidence supporting the possible use of Etravirine, a drug approved for the treatment of HIV infections in patients starting from 2 years of age, as a treatment for FA.
- Trial name
- FAEST1 (NCT04273165)
- Testing
- Etravirine Tablets
- Where
- Italy
- Volunteers
- 30 people
- Sponsor
- IRCCS Eugenio Medea
Neurodegeneration, G-CSF
Neuroregenerative Potential of Intravenous G-CSF and Autologous Peripheral Blood Stem Cells
The current study describes a randomized, double-blind, cross-over study of intravenous G-CSF followed by infusion with autologous mobilized peripheral blood mononuclear cells (mPBMCs) in children with cerebral palsy (CP) to…
- Conditions
- Neurodegeneration, G-CSF, Peripheral Blood Mononuclear Cells, Cerebral Palsy
- Trial name
- NCT02983708
- Testing
- Peripheral blood mononuclear cells (mPBMC), G-CSF
- Volunteers
- 57 people
- Sponsor
- Hanyang University Seoul Hospital