| 1 |
NCT03112408 |
Not yet recruiting |
Eye Movements and Visuo-spatial Perception |
|
- Behavioral: Forward adaptation of reactive saccades
- Behavioral: Backward adaptation of reactive saccades
- Behavioral: Execution of reactive saccades
- Behavioral: Generation of reactive saccades
|
Interventional
|
Not Applicable |
|
Other |
- Allocation: Randomized
- Intervention Model: Factorial Assignment
- Masking: Single (Participant)
- Primary Purpose: Basic Science
|
- Saccadic adaptation efficiency
- Evolution of localization-task performances
- Evolution of attentional performances assessed by neuropsychological tests of neglect
|
75 |
All |
18 Years to 80 Years (Adult, Older Adult) |
NCT03112408 |
69HCL17_0109 |
|
May 1, 2018 |
August 1, 2020 |
August 1, 2020 |
April 13, 2017 |
April 5, 2018 |
|
- Hospices Civils de Lyon
Bron, France
|
|
| 2 |
NCT02185313 |
Recruiting |
Gaze Holding in Cerebellar Patients |
- Gaze Holding in Humans
- Rebound in Humans
|
- Other: visual stimulus to follow
|
Interventional
|
Not Applicable |
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Basic Science
|
- eye velocity in relation to eye position
- symmetry of eye velocity for right vs. left gaze
|
90 |
All |
18 Years to 85 Years (Adult, Older Adult) |
NCT02185313 |
gaze_holding_cerebellar_zurich |
|
August 2013 |
July 2017 |
August 2017 |
July 9, 2014 |
October 27, 2016 |
|
- University Hospital Zurich, Division of Neurology
Zurich, ZH, Switzerland
|
|
| 3 |
NCT02440763 |
Recruiting |
The EUROSCA Natural History Study |
|
|
Observational
|
|
|
Other |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- Scale for the assessment and rating of ataxia (SARA)
- Disease stages
- Inventory of non-ataxia signs (INAS)
- (and 3 more...)
|
400 |
All |
18 Years and older (Adult, Older Adult) |
NCT02440763 |
010/05 |
EUROSCA-NHS |
July 2005 |
July 2050 |
July 2050 |
May 12, 2015 |
June 29, 2017 |
|
- Department of Neurology, Medical University, Innsbruck
Innsbruck, Austria - Université Libre de Bruxelles (ULB), Neurology Service - ULB Hôpital Erasme, ULB Laboratory of Experimental Neurology
Brussels, Belgium - Hôpital de la Pitié-Salpêtrière, Département de Génétique
Paris, France - (and 13 more...)
|
|
| 4 |
NCT03563053 |
Not yet recruiting
New |
Open-label, Long-term, Extension Treatment Using Intra-Erythrocyte Dexamethasone Sodium Phosphate in Patients With Ataxia Telangiectasia Who Participated in the IEDAT-02-2015 Study |
|
- Combination Product: EryDex System
|
Interventional
|
Phase 3 |
|
Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Long term safety/tolerability of EDS-EP: # of pts with treatment related Adverse Events (CTCAE4.0)
- Long term safety/tolerability of EDS-EP: lab parameters according to clinical practice such as hematology, biochemistry, urinalysis, HbA1c (haemoglobin A1c or glycated haemoglobin test), CD4+ lymphocytes count, CRP (C reactive protein)
- Long term safety/tolerability of EDS-EP: 12-lead ECG (Electrocardiography)
- (and 6 more...)
|
50 |
All |
6 Years and older (Child, Adult, Older Adult) |
NCT03563053 |
IEDAT-03-2018 |
OLE-IEDAT |
June 2018 |
August 2019 |
August 2019 |
June 20, 2018 |
June 20, 2018 |
|
|
|
| 5 |
NCT03487367 |
Not yet recruiting |
Clinical Trial Readiness for SCA1 and SCA3 |
- Spinocerebellar Ataxia Type 1
- Spinocerebellar Ataxia 3
|
|
Observational
|
|
- Tetsuo Ashizawa, MD
- University of Michigan
- University of Minnesota - Clinical and Translational Science Institute
- (and 18 more...)
|
Other |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- Change in disease progression in SCA1 and SCA3 as determined by change in scale for the assessment and rating of ataxia (SARA) score over time.
- Change in disease progression in SCA1 and SCA3 as determined by change in Composite Cerebellar Functional Severity Score (CCFS) total score over time.
- Change in disease progression in SCA1 and SCA3 as determined by change in timed 25 foot walk test (T25FW) over time.
- (and 16 more...)
|
200 |
All |
18 Years to 65 Years (Adult, Older Adult) |
NCT03487367 |
Pro00017836
U01 - Ashizawa 2016 |
|
May 31, 2018 |
December 2022 |
December 2022 |
April 4, 2018 |
April 10, 2018 |
|
|
|
| 6 |
NCT03418740 |
Recruiting |
Neurology Measures in FA Children |
|
|
Observational
|
|
- Children's Hospital of Philadelphia
- Food and Drug Administration (FDA)
- University of Florida
- University of California, Los Angeles
|
Other / U.S. Fed |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- Clinical markers in disease progression in children with FA
- Biochemical assessment of frataxin deficiency in children with FA
|
100 |
All |
2 Years to 18 Years (Child, Adult) |
NCT03418740 |
17-014390 |
|
November 20, 2017 |
December 31, 2021 |
December 31, 2021 |
February 1, 2018 |
June 6, 2018 |
|
- University of California, Los Angeles
Los Angeles, California, United States - University of Florida
Gainesville, Florida, United States - Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
|
|
| 7 |
NCT03378414 |
Not yet recruiting |
Umbilical Cord Mesenchymal Stem Cells Therapy for Patients With Spinocerebellar Ataxia |
- Spinocerebellar Ataxia Type 1
- Spinocerebellar Ataxia Type 2
- Spinocerebellar Ataxia Type 3
- Spinocerebellar Ataxia Type 6
|
- Procedure: Intravenous infusion
- Procedure: Intrathecal injection
- Biological: umbilical cord mesenchymal stem cell
|
Interventional
|
Phase 2 |
- Sclnow Biotechnology Co., Ltd.
|
Industry |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Scale for the assessment anf rating of ataxia (SARA)
- Image examinations
- Inventory of Non-Ataxia Symptoms (INAS) score
- Cerebrospinal fluid (csf) routine
|
45 |
All |
16 Years to 60 Years (Child, Adult) |
NCT03378414 |
SCLnow-XY-03 |
|
January 2018 |
January 2019 |
December 2019 |
December 19, 2017 |
December 19, 2017 |
|
|
|
| 8 |
NCT03347344 |
Not yet recruiting |
Clinical Trial With Riluzole in Spinocerebellar Ataxia Type 2 (ATRIL) |
- Spinocerebellar Ataxia Type 2
|
- Drug: Riluzole
- Drug: Placebo
|
Interventional
|
Phase 3 |
- Assistance Publique - Hôpitaux de Paris
|
Other |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Triple (Participant, Care Provider, Investigator)
- Primary Purpose: Treatment
|
- Change in Ataxia symptoms (Scale for the Assessment and Rating of Ataxia (SARA))
- Change in Ataxia symptoms (Composite Cerebellar Functional Severity (CCFS) score)
- Change in extracerebellar symptoms (Inventory of Non-Ataxia Signs (INAS))
- 12 months survival
|
42 |
All |
18 Years and older (Adult, Older Adult) |
NCT03347344 |
P160927J
2017-001481-23 |
ATRIL |
November 2017 |
November 2019 |
November 2019 |
November 20, 2017 |
November 20, 2017 |
|
|
|
| 9 |
NCT03341416 |
Recruiting |
Effects of Deep Brain Stimulation of the Dentate Nucleus on Cerebellar Ataxia |
|
- Device: deep brain stimulation on stimulation - active
- Device: deep brain stimulation off stimulation - sham
|
Interventional
|
Not Applicable |
- University of Sao Paulo General Hospital
|
Other |
- Allocation: Randomized
- Intervention Model: Crossover Assignment
- Masking: Triple (Participant, Care Provider, Outcomes Assessor)
- Primary Purpose: Treatment
|
- Primary outcome - change in Scale for the assessment and rating of ataxia - SARA
- Secondary outcome - change in tremor score using the Fahn Tolosa Marin Scale
- Secondary outcome - quality of life
|
10 |
All |
18 Years to 80 Years (Adult, Older Adult) |
NCT03341416 |
102178/2015 |
|
September 1, 2017 |
July 1, 2018 |
January 1, 2019 |
November 14, 2017 |
November 17, 2017 |
|
- Rubens Cury
São Paulo, SP, Brazil
|
|
| 10 |
NCT03336008 |
Recruiting |
Hong Kong Spinocerebellar Ataxias Registry |
|
|
Observational
|
|
- Chinese University of Hong Kong
|
Other |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- Scale for the assessment and rating of ataxia (SARA) score
- Unified Huntington's Disease Rating Scale IV
- EQ5D Health questionnaire
- Patient Health Questionnaire-9 (PHQ-9)
|
70 |
All |
18 Years to 90 Years (Adult, Older Adult) |
NCT03336008 |
HK_SCA_Registry |
HK_SCA_Reg |
December 7, 2012 |
December 31, 2018 |
December 31, 2018 |
November 8, 2017 |
November 8, 2017 |
|
- Prince of Wales Hospital
Hong Kong, Shatin, Hong Kong
|
|
| 11 |
NCT03286686 |
Recruiting |
Sensori-motor and Perceptual Functions of the PPC. |
- Healthy Volunteers
- Optic Ataxia
|
- Behavioral: point targets
- Behavioral: tactile stimulation
- Behavioral: visual images
|
Interventional
|
Not Applicable |
|
Other |
- Allocation: Non-Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Basic Science
|
- response error - number of pointing error
- response error -measure of reaction times
- response error - percentage of right responses
|
155 |
All |
18 Years to 75 Years (Adult, Older Adult) |
NCT03286686 |
69HCL17_0341 |
PPCfonctions |
February 22, 2018 |
February 22, 2024 |
February 22, 2024 |
September 18, 2017 |
February 28, 2018 |
|
- U1028 INSERM - CNRS UMR 5292 Equipe ImpAct
Bron, France
|
|
| 12 |
NCT03214588 |
Recruiting |
Efficacy, Tolerability, and Pharmacokinetics of Multiple Doses of Oral TAK-831 in Adults With Friedreich Ataxia |
|
- Drug: TAK-831
- Drug: TAK-831 Placebo
|
Interventional
|
Phase 2 |
|
Industry |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- Change from Baseline in Inverse Time to Complete 9-Hole Peg Test (9-HPT-1) at Week 12
- Change from Baseline on the Activities of Daily Living (ADL) Component of the Friedreich Ataxia Rating Scale (FARS)
- Change from Baseline on the 9-HPT-1
- (and 16 more...)
|
65 |
All |
18 Years to 55 Years (Adult) |
NCT03214588 |
TAK-831-1501
U1111-1189-7951 |
|
November 8, 2017 |
February 27, 2019 |
February 27, 2019 |
July 11, 2017 |
November 27, 2017 |
|
- UCLA Ataxia Center
Los Angeles, California, United States - University of Florida Center for Movement Disorders
Gainesville, Florida, United States - USF College of Medicine
Tampa, Florida, United States - (and 3 more...)
|
|
| 13 |
NCT03213106 |
Recruiting |
Cerebellar Non-invasive Stimulation in Ataxias |
|
- Device: Transcranial Magnetic Stimulation (TMS)
- Device: Sham Stimulation
|
Interventional
|
Not Applicable |
- University of Sao Paulo General Hospital
|
Other |
- Allocation: Randomized
- Intervention Model: Crossover Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- Percent Change From Baseline to Post Treatment on the Scale for the Assessment and Rating of Ataxia (SARA)
|
30 |
All |
18 Years and older (Adult, Older Adult) |
NCT03213106 |
1.310.275 |
|
December 1, 2016 |
December 1, 2017 |
December 1, 2019 |
July 11, 2017 |
July 11, 2017 |
|
- Hospital das Clínicas da Faculdade de Medicina da USP
São Paulo, SP, Brazil
|
|
| 14 |
NCT03090789 |
Recruiting |
FA Clinical Outcome Measures |
- Friedreich Ataxia
- Neuro-Degenerative Disease
|
|
Observational
|
|
- Children's Hospital of Philadelphia
- Friedreich's Ataxia Research Alliance
- University of Rochester
|
Other |
- Observational Model: Case-Control
- Time Perspective: Prospective
|
- Friedreich Ataxia Rating Scale
- 9-hole peg test
- timed 25 foot walk
- (and 2 more...)
|
1500 |
All |
4 Years to 80 Years (Child, Adult, Older Adult) |
NCT03090789 |
01-002609 |
FA-COMS |
January 1, 2001 |
January 1, 2030 |
January 1, 2030 |
March 27, 2017 |
February 9, 2018 |
|
- UCLA Ataxia Center
Los Angeles, California, United States - University of Florida - Neurology
Gainesville, Florida, United States - USF Ataxia Research Center
Tampa, Florida, United States - (and 6 more...)
|
|
| 15 |
NCT02903290 |
Recruiting |
Longitudinal Monitoring of Energy Expenditure, Dynamic Stability and Fatigue During Gait in Patients With Cerebellar Ataxia Gene |
|
- Other: Longitudinal Monitoring of Energy Expenditure, Dynamic Stability and Fatigue During Gait
- Other: walking track GAITRite,manual dynamometer, and treadmill
|
Interventional
|
Not Applicable |
- Centre Hospitalier Universitaire de Nice
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Pre physical activity scores (Gilette Gait Index (GVI)) to pre exercise walking
- Oxygen consumption to pre exercise walking
- Pre physical activity scores (Functional Ambulation Performance Score (FAPS)) to pre exercise walking
- (and 2 more...)
|
15 |
All |
18 Years to 60 Years (Adult) |
NCT02903290 |
15-AOI-10 |
|
January 2017 |
January 2017 |
December 2019 |
September 16, 2016 |
December 18, 2017 |
|
|
|
| 16 |
NCT02741440 |
Recruiting |
Natural History of Spinocerebellar Ataxia Type 7 (SCA7) |
|
|
Observational
|
|
- National Eye Institute (NEI)
- National Institutes of Health Clinical Center (CC)
|
NIH |
- Observational Model: Cohort
- Time Perspective: Other
|
- The primary outcome for this study is determination of the amplitude and time of photopic and scotopic responses on electroretinogram.
- Secondary outcomes include changes in visual acuity, microperimetry, peripheral visual field, color vision, macular thickness, and neurologic outcome variables.
- Exploratory outcomes include: 1)formulation of clinical outcome measures for future studies and 2)acquisition and preliminary analysis of data that may advance our understanding of the progression of retinal and neurodegeneration associated SCA7...
|
25 |
All |
12 Years and older (Child, Adult, Older Adult) |
NCT02741440 |
160090
16-EI-0090 |
|
April 13, 2016 |
February 28, 2019 |
March 2, 2019 |
April 18, 2016 |
March 29, 2018 |
|
- National Institutes of Health Clinical Center
Bethesda, Maryland, United States
|
|
| 17 |
NCT02701036 |
Recruiting |
Sporadic Degenerative Ataxia With Adult Onset: Natural History Study |
- Late Onset Sporadic Cerebellar Ataxia
|
|
Observational
|
|
- Ataxia Study Group
- German Center for Neurodegenerative Diseases (DZNE)
|
Other |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- Scale for the assessment and rating of ataxia (SARA)
- Inventory of non-ataxia signs (INAS)
- spinocerebellar ataxia functional index (SCAFI)
- (and 6 more...)
|
300 |
All |
40 Years and older (Adult, Older Adult) |
NCT02701036 |
SPORTAX 010/05 |
SPORTAX-NHS |
April 2010 |
December 2030 |
December 2030 |
March 8, 2016 |
June 29, 2017 |
|
- Department of Neurology, Medical University, Innsbruck
Innsbruck, Austria - Universitätsmedizin Berlin Charité
Berlin, Germany - Department of Neurology, University of Bonn
Bonn, Germany - (and 11 more...)
|
|
| 18 |
NCT03357978 |
Recruiting |
Susceptibility to Infections, Tumor Risk and Liver Disease in Patients With Ataxia Telangiectasia |
|
- Diagnostic Test: bioelectrical impedance analysis
- Diagnostic Test: blood draw
- Diagnostic Test: transient elastography (FibroScan)
- (and 2 more...)
|
Interventional
|
Not Applicable |
- Johann Wolfgang Goethe University Hospital
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Diagnostic
|
- Infections in A-T
- Liver disease
- Cancer risk
|
30 |
All |
2 Years to 45 Years (Child, Adult) |
NCT03357978 |
InfectionandLiver_AT2016 |
|
October 1, 2016 |
September 30, 2018 |
September 30, 2019 |
November 30, 2017 |
November 30, 2017 |
|
- Children's Hospital, Allergology, Pneumology and Cystic Fibrosis, Goethe University Frankfurt
Frankfurt, Hessen, Germany
|
|
| 19 |
NCT02540655 |
Recruiting |
Efficacy and Safety Study of Stemchymal® in Polyglutamine Spinocerebellar Ataxia |
|
- Biological: Stemchymal®
- Procedure: Excipients
|
Interventional
|
Phase 2 |
- Steminent Biotherapeutics Inc.
|
Industry |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Triple (Participant, Care Provider, Investigator)
- Primary Purpose: Treatment
|
- To assess the changes from the baseline to all visits on Scale for the Assessment and Rating of Ataxia (SARA) score.
- To assess the safety by incidence of adverse events, changes of vital signs, blood biochemistry tests, complete blood count, immunoactivity assay, urinalysis and magnetic resonance imaging (MRI).
- To assess the changes from the baseline to all visits on SCA functional index (SCAFI)
- (and 4 more...)
|
60 |
All |
20 Years to 70 Years (Adult, Older Adult) |
NCT02540655 |
IB02 |
|
September 2015 |
November 2017 |
February 2018 |
September 4, 2015 |
November 1, 2016 |
|
- Taipei Veterans General Hospital
Taipei, Taiwan
|
|
| 20 |
NCT02497534 |
Recruiting |
Biomarkers in Friedreich's Ataxia |
|
|
Observational
|
|
- University of Florida
- Children's Miracle Network
- National Institutes of Health (NIH)
- Friedreich's Ataxia Research Alliance
|
Other / NIH |
- Observational Model: Case-Control
- Time Perspective: Cross-Sectional
|
- Cardiac MRI
- Echocardiogram
- Friedreich's Ataxia Rating Scale (FARS)
- (and 6 more...)
|
100 |
All |
8 Years to 70 Years (Child, Adult, Older Adult) |
NCT02497534 |
IRB201500369-N
UL1TR000064 |
|
September 2015 |
August 2018 |
August 2018 |
July 14, 2015 |
May 2, 2018 |
|
- University of Florida
Gainesville, Florida, United States
|
|
| 21 |
NCT03285204 |
Recruiting |
Relationship Between Neurological Disability and Visual Impairment in Patients With ALS or Friedreich's Ataxia |
- Amyotrophic Lateral Sclerosis
|
|
Observational
|
|
- Jose Luis Urcelay Segura
- Hospital General Universitario Gregorio Marañon
|
Other |
- Observational Model: Case-Control
- Time Perspective: Prospective
|
- early biomarker of amyotrophic lateral sclerosis and Friedreich's Ataxia
|
90 |
All |
18 Years and older (Adult, Older Adult) |
NCT03285204 |
ELA-AF-OCT-2014-01 |
|
July 2015 |
June 2018 |
December 2019 |
September 15, 2017 |
April 10, 2018 |
|
- Pilar Rojas Lozano
Madrid, Spain
|
|
| 22 |
NCT03269201 |
Not yet recruiting |
Brain Network Activation in Patients With Movement Disorders |
- Parkinson Disease
- Essential Tremor
- Dystonia
- (and 6 more...)
|
|
Observational
|
|
|
Other |
- Observational Model: Case-Only
- Time Perspective: Cross-Sectional
|
- MDS-UPDRS part III score
- International Cooperative Ataxia Rating Scale
- TETRAS
- (and 2 more...)
|
300 |
All |
18 Years to 85 Years (Adult, Older Adult) |
NCT03269201 |
4408-17 |
BNA-MDi |
September 15, 2017 |
September 15, 2020 |
September 15, 2022 |
August 31, 2017 |
August 31, 2017 |
|
|
|
| 23 |
NCT02255435 |
Recruiting |
RTA 408 Capsules in Patients With Friedreich's Ataxia - MOXIe |
|
- Drug: Omaveloxolone Capsules, 2.5 mg
- Drug: Omaveloxolone Capsules, 5 mg
- Drug: Omaveloxolone Capsules, 10 mg
- (and 7 more...)
|
Interventional
|
Phase 2 |
- Reata Pharmaceuticals, Inc.
- AbbVie
- Friedreich's Ataxia Research Alliance
|
Industry / Other |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Double (Participant, Investigator)
- Primary Purpose: Treatment
|
- Measure the change in the modified Friedreich's ataxia rating scale (FARS)
- Measure the change of peak workload (in watts/kg) during exercise testing
|
172 |
All |
16 Years to 40 Years (Child, Adult) |
NCT02255435 |
RTA 408-C-1402 |
|
January 2015 |
January 2020 |
December 2022 |
October 2, 2014 |
April 4, 2018 |
|
- UCLA
Los Angeles, California, United States - University of Florida - Neurology
Gainesville, Florida, United States - USF Ataxia Research Center
Tampa, Florida, United States - (and 9 more...)
|
|
| 24 |
NCT02246491 |
Recruiting |
Cell-Based Approaches For Modeling and Treating Ataxia-Telangiectasia |
- Ataxia-Telangiectasia (A-T)
|
- Other: with gene correction
- Other: without gene correction
|
Interventional
|
Not Applicable |
- Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
|
Other |
- Allocation: Non-Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Basic Science
|
- Number of samples of primary A-T fibroblast samples that can be successfully reprogrammed to iPSCs
- Number of samples of patient A-T fibroblasts that can be reprogrammed to iPSCs with and without gene correction
- Quantification of the cloning efficiency of primary cells haploinsufficient for ATM relative to healthy controls
|
20 |
All |
3 Years to 100 Years (Child, Adult, Older Adult) |
NCT02246491 |
J1491
IRB00038916 |
|
September 2014 |
September 2017 |
September 2018 |
September 22, 2014 |
March 24, 2016 |
|
- SKCCC at Johns Hopkins
Baltimore, Maryland, United States
|
|
| 25 |
NCT02069509 |
Recruiting |
Patient Registry of the European Friedreich's Ataxia Consortium for Translational Studies (EFACTS) |
|
|
Observational
|
|
- European Friedreich's Ataxia Consortium for Translational Studies
|
Other |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- Disease progression as assessed by clinical examination
|
900 |
All |
Child, Adult, Older Adult |
NCT02069509 |
HEALTH-F2-2010- 242193 |
EFACTS |
May 2010 |
December 2018 |
December 2018 |
February 24, 2014 |
May 11, 2017 |
|
- Medical University Innsbruck, Department of Neurology
Innsbruck, Austria - Université Libre de Bruxelles
Bruxelles, Belgium - Hôpital Pitié Salpêtrière
Paris, France - (and 9 more...)
|
|
| 26 |
NCT02594917 |
Recruiting |
Genetic and Environmental Determinants That Control Metabolism in Pulmonary Hypertension |
- Iron-sulfur Cluster Deficiency
- Pulmonary Hypertension
- Friedreich Ataxia
|
- Procedure: Right Heart Catheterization
|
Observational
|
|
|
Other |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- 6 minute walk distance (meters)
- Pulmonary arterial pressure (mm Hg)
|
20 |
All |
18 Years to 60 Years (Adult) |
NCT02594917 |
PRO15070005 |
|
October 2015 |
May 2019 |
May 2019 |
November 3, 2015 |
April 18, 2018 |
|
- University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, United States
|
|
| 27 |
NCT02867969 |
Recruiting |
Slowing Down Disease Progression in Premanifest SCA: a Piloting Interventional Exergame Trial |
|
|
Interventional
|
Not Applicable |
- University Hospital Tuebingen
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Supportive Care
|
- Gait score
- Brain networks captured
- Quantitative movement parameters
- (and 3 more...)
|
26 |
All |
18 Years to 80 Years (Adult, Older Adult) |
NCT02867969 |
SlowSCA |
SlowSCA |
August 2016 |
February 2017 |
May 2017 |
August 16, 2016 |
August 16, 2016 |
|
- University Hospital
Tuebingen, Baden-Württemberg, Germany
|
|
| 28 |
NCT02857894 |
Recruiting |
Genetic Factors of Idiopathic Polypoidal Vasculopathies in the ATM Gene (Ataxia Telangiectasia Mutated) |
- Choroidal Neovascularization
|
|
Observational
|
|
- Fondation Ophtalmologique Adolphe de Rothschild
|
Other |
- Observational Model: Cohort
- Time Perspective: Prospective
|
|
40 |
All |
18 Years and older (Adult, Older Adult) |
NCT02857894 |
MMT_2015_4 |
ATM |
November 5, 2015 |
May 2018 |
May 2018 |
August 5, 2016 |
March 1, 2018 |
|
- Fondation Ophtalmologique A. de Rotchschild
Paris, France
|
|
| 29 |
NCT02488031 |
Recruiting |
Functional and Structural Imaging and Motor Control in Spinocerebellar Ataxia |
|
- Behavioral: Error-reduction
- Behavioral: International Cooperative Ataxia Rating Scale
- Behavioral: Scale for the Assessment and Rating of Ataxia
- (and 11 more...)
|
Interventional
|
Not Applicable |
- University of Florida
- National Institute of Neurological Disorders and Stroke (NINDS)
- University of South Florida
|
Other / NIH |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Change in the Location of the Movement Endpoint Relative to the Target in the Motor Task
- International Cooperative Ataxia Rating Scale(ICARS) Assessment
- Change in Motor Unit Discharge Rate Variability
- Change in Blood-oxygen-level-dependent(BOLD) Activity of Motor Cortex
|
60 |
All |
21 Years to 85 Years (Adult, Older Adult) |
NCT02488031 |
IRB201500202-N
R21NS094946 |
SCA |
March 2016 |
August 2019 |
August 2019 |
July 2, 2015 |
April 5, 2018 |
|
- University of Florida
Gainesville, Florida, United States
|
|
| 30 |
NCT02770807 |
Recruiting |
EDS in Ataxia Telangiectasia Patients |
- Nervous System Disease
- Genetic Syndrome
|
- Drug: EDS-EP dose range of ~5-10 mg DSP/infusion
- Drug: EDS-EP dose range of ~14-22 mg DSP/infusion
- Drug: Placebo
|
Interventional
|
Phase 3 |
|
Industry |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Triple (Participant, Care Provider, Investigator)
- Primary Purpose: Treatment
|
- Modified International Cooperative Ataxia Rating Scale (mICARS)
- Clinical Global Impression of Change (CGI-C) key secondary outcome measure
- Clinical Global Impression of Severity (CGI-S) of neurological symptoms of AT
- Vineland Adaptive Behavior Scales (VABS)
|
180 |
All |
6 Years and older (Child, Adult, Older Adult) |
NCT02770807 |
IEDAT-02-2015 |
ATTeST |
March 2, 2017 |
August 2018 |
March 2019 |
May 12, 2016 |
October 31, 2017 |
|
- UCLA
Los Angeles, California, United States - The Ataxia-Telangiectasia Clinical Center, The Johns Hopkins Hospital
Baltimore, Maryland, United States - Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States - (and 18 more...)
|
|
| 31 |
NCT02014246 |
Recruiting |
Genetic Characterization of Movement Disorders and Dementias |
- Ataxia
- Dystonia
- Parkinson's Disease
- (and 12 more...)
|
|
Observational
|
|
- National Institute on Aging (NIA)
- National Institutes of Health Clinical Center (CC)
|
NIH |
- Observational Model: Case-Control
- Time Perspective: Other
|
- Identify and characterize genetic contributions to etiology for movement disorders, such as dystonia, Parkinson's disease, and dementias, such as Alzheimer's disease, Lewy Body Dementia, frontotemporal dementia.
|
12000 |
All |
18 Years and older (Adult, Older Adult) |
NCT02014246 |
999903329
03-AG-N329 |
|
February 12, 2003 |
December 31, 2018 |
December 31, 2018 |
December 18, 2013 |
May 11, 2018 |
|
- National Institute of Aging, Clinical Research Unit
Baltimore, Maryland, United States
|
|
| 32 |
NCT01793168 |
Recruiting |
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford |
- Rare Disorders
- Undiagnosed Disorders
- Disorders of Unknown Prevalence
- (and 260 more...)
|
|
Observational
|
|
- Sanford Health
- National Ataxia Foundation
- International WAGR Syndrome Association
- (and 23 more...)
|
Other |
- Observational Model: Case-Only
- Time Perspective: Prospective
|
- To accelerate research into rare disorders by connecting individuals who are interested in research and who have been diagnosed with a rare disorder (or a disorder of unknown prevalence, or who are undiagnosed) with researchers who study rare diseases.
|
20000 |
All |
Child, Adult, Older Adult |
NCT01793168 |
03-10-014
Hypersomnia Foundation
National Ataxia Foundation
4p- Support Group
CdLS Foundation
Hyperacusis Research Limited
Kabuki Syndrome Network
Kawasaki Disease Foundation
Klippel-Feil Syndrome Freedom
Leiomyosarcoma Direct Research
MSS Support Group
ML4 Foundation
Stickler Involved People
IWSA
Soft Bones
PWN4PWN
aHUS
Klippel-Feil Syndrome Alliance
American MEN Support
Kleine-Levin Syndrome
All Things Kabuki
WSS Foundation
BIVA
ABDA
PROS Foundation (HLH) |
CoRDS |
July 2010 |
December 2100 |
December 2100 |
February 15, 2013 |
October 2, 2017 |
|
- Sanford Health
Sioux Falls, South Dakota, United States
|
|
| 33 |
NCT01060371 |
Recruiting |
Natural History Study of and Genetic Modifiers in Spinocerebellar Ataxias |
- Spinocerebellar Ataxia Type 1
- Spinocerebellar Ataxia Type 2
- Spinocerebellar Ataxia Type 3
- Spinocerebellar Ataxia Type 6
|
- Genetic: All Participants
|
Observational
|
|
- University of Florida
- National Ataxia Foundation
- University of California, Los Angeles
|
Other |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- The disease's progression over time using clinical rating scales and timed performance measures.
- Relation between the genetic modifiers and the age at onset of disease and disease progression rates.
- The effects of the disease on the Activities of Daily Living (ADL)in patients with Spinocerebellar Ataxias
|
800 |
All |
6 Years and older (Child, Adult, Older Adult) |
NCT01060371 |
IRB201700740
505-2009 |
|
April 2010 |
June 2018 |
June 2018 |
February 2, 2010 |
August 21, 2017 |
|
- University of California Los Angeles
Los Angeles, California, United States - University of California San Francisco
San Francisco, California, United States - University of Florida
Gainesville, Florida, United States - (and 9 more...)
|
|
| 34 |
NCT01582763 |
Recruiting |
International Guillain-Barré Syndrome Outcome Study |
- Guillain-Barré Syndrome
- Miller Fisher Syndrome
|
|
Observational
|
|
|
Other |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- Guillain-Barre Syndrome(GBS) disability score and Medical Research Council(MRC) sumscore
- Overall Neuropathy Limitations Scale (ONLS)
- Fatigue Severity Scale (FSS)
- (and 2 more...)
|
4000 |
All |
Child, Adult, Older Adult |
NCT01582763 |
MEC-2011-477
3290 |
IGOS |
May 2012 |
June 2017 |
January 2019 |
April 23, 2012 |
January 5, 2017 |
|
- Barrows Neurological Institute
Phoenix, Arizona, United States - UC Irvine Health
Orange, California, United States - Yale University School of medicine
New Haven, Connecticut, United States - (and 125 more...)
|
|
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