| 1 |
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)
- Ataxia Study Group
|
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, Senior) |
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...)
|
| 2 |
NCT02179333 |
Completed |
Preliminary Study of the Scale To Assess Ataxia and Neurologic Dysfunction (STAND) |
- Spinocerebellar Ataxia - All Sub-types
- Friedreich's Ataxia
|
- Other: Ataxia rating scale
|
Observational |
|
- University of South Florida
|
Other |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- Inter- and intra-rater agreement
|
30 |
All |
18 Years to 80 Years (Adult, Senior) |
NCT02179333 |
STAND2012 |
STAND |
October 2012 |
June 2016 |
|
July 1, 2014 |
June 29, 2016 |
|
- University of South Florida
Tampa, Florida, United States
|
| 3 |
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, Senior) |
NCT03347344 |
P160927J
2017-001481-23 |
ATRIL |
November 2017 |
November 2019 |
November 2019 |
November 20, 2017 |
November 20, 2017 |
|
|
| 4 |
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, Senior) |
NCT02497534 |
IRB201500369-N
UL1TR000064 |
|
September 2015 |
August 2018 |
August 2018 |
July 14, 2015 |
April 5, 2018 |
|
- University of Florida
Gainesville, Florida, United States
|
| 5 |
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, Senior) |
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
|
| 6 |
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, Senior) |
NCT03341416 |
102178/2015 |
|
September 1, 2017 |
July 1, 2018 |
January 1, 2019 |
November 14, 2017 |
November 17, 2017 |
|
- Rubens Cury
São Paulo, SP, Brazil
|
| 7 |
NCT01965327 |
Completed
Has Results |
Interferon Gamma-1b in Friedreich Ataxia (FRDA) |
|
- Drug: Interferon Gamma-1b
|
Interventional |
Phase 2 |
- Children's Hospital of Philadelphia
- Friedreich's Ataxia Research Alliance
- Vidara Therapeutics Research Ltd
|
Other / Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Change in Whole Blood Frataxin Levels
- Change in Total Friedreich Ataxia Rating Scale (FARS) Score
|
12 |
All |
5 Years to 17 Years (Child) |
NCT01965327 |
13-010121 |
|
August 2013 |
March 2014 |
October 2014 |
October 18, 2013 |
May 15, 2015 |
April 30, 2015 |
- Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
|
| 8 |
NCT02424435 |
Active, not recruiting |
Methylprednisolone Treatment of Friedreich Ataxia |
|
|
Interventional |
Early Phase 1 |
- Children's Hospital of Philadelphia
- Friedreich's Ataxia Research Alliance
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Change in the Timed 25 Foot Walk (T25FW) Score
- Change in the Friedreich Ataxia Rating Scale (FARS) Score
- Change in The 1-Minute Walk Distance
- Change in the Change in the 9-Hole Peg Test (9HPT) Time
|
11 |
All |
5 Years and older (Child, Adult, Senior) |
NCT02424435 |
15-011801 |
|
June 2015 |
December 2017 |
April 2018 |
April 23, 2015 |
February 14, 2018 |
|
- Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
|
| 9 |
NCT01543750 |
Unknown † |
4-Aminopyridine in Episodic Ataxia Type 2 |
|
- Drug: 4-Aminopyridine
- Drug: Placebo
|
Interventional |
Phase 2 |
- University of California, Los Angeles
- University of Rochester
- University of South Florida
|
Other |
- Allocation: Randomized
- Intervention Model: Crossover Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- the frequency of ataxia episodes
- impact on daily activities
- duration of ataxia episodes
- (and 4 more...)
|
20 |
All |
18 Years and older (Adult, Senior) |
NCT01543750 |
CINCH-EA2
R01FD003923 |
4AP in EA2 |
April 2013 |
April 2016 |
April 2016 |
March 5, 2012 |
February 10, 2014 |
|
- University of California, Los Angeles (UCLA)
Los Angeles, California, United States - University of South Florida
Tampa, Florida, United States - University of Rochester School of Medicine
Rochester, New York, United States
|
| 10 |
NCT02887703 |
Completed |
Augmenting Balance in Individuals With Cerebellar Ataxias |
|
- Behavioral: Sensory Augmentation
|
Interventional |
Not Applicable |
- Kathleen Sienko
- University of Michigan
|
Other |
- Allocation: Randomized
- Intervention Model: Crossover Assignment
- Masking: Single (Outcomes Assessor)
- Primary Purpose: Treatment
|
- Change in Scale for the assessment and rating of ataxia (SARA)
- Change in 10-meter Preferred Gait Speed
- Change in 10-meter Fast Gait Speed
|
10 |
All |
18 Years and older (Adult, Senior) |
NCT02887703 |
HUM00116756 |
|
September 2016 |
January 31, 2018 |
January 31, 2018 |
September 2, 2016 |
March 7, 2018 |
|
- Department of Mechanical Engineering, University of Michigan
Ann Arbor, Michigan, United States
|
| 11 |
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 |
|
|
| 12 |
NCT02705547 |
Completed |
Rosuvastatin (Crestor) in Friedreich Ataxia |
|
|
Interventional |
Early Phase 1 |
- Children's Hospital of Philadelphia
- Friedreich's Ataxia Research Alliance
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Change in ApoA-1 serum protein levels from baseline to Week 12 visit
- Change in frataxin levels from baseline to Week 12 visit
- Change in platelet metabolism from baseline to Week 12 visit
|
12 |
All |
18 Years to 65 Years (Adult) |
NCT02705547 |
16-012659 |
|
May 2016 |
August 4, 2017 |
August 4, 2017 |
March 10, 2016 |
January 10, 2018 |
|
- Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
|
| 13 |
NCT02415127 |
Completed
Has Results |
Safety, Tolerability and Efficacy of ACTIMMUNE® Dose Escalation in Friedreich's Ataxia |
|
- Drug: Interferon γ-1b
- Drug: Placebo
|
Interventional |
Phase 3 |
- Horizon Pharma Ireland, Ltd., Dublin Ireland
- Friedreich's Ataxia Research Alliance
|
Industry / Other |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Double (Participant, Investigator)
- Primary Purpose: Treatment
|
- Change From Baseline to Week 26 in the Friedreich's Ataxia Rating Scale (FARS)-mNeuro Score
- Change From Baseline to Week 26 in Activities of Daily Living (ADL) Score
- Change From Baseline at Week 26 in Timed 25-Foot Walk (T25FW)
- (and 2 more...)
|
92 |
All |
10 Years to 25 Years (Child, Adult) |
NCT02415127 |
HZNP-ACT-301 |
STEADFAST |
June 2015 |
November 2016 |
November 2016 |
April 14, 2015 |
December 8, 2017 |
December 8, 2017 |
- University of California, Los Angeles Neurology Clinic
Los Angeles, California, United States - University of Florida - Clinical Research Center
Gainesville, Florida, United States - University of Iowa Children's Hospital
Iowa City, Iowa, United States - Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
|
| 14 |
NCT01958177 |
Unknown † |
Clinical Study to Evaluate the Safety and Efficacy BMMNC in Cerebellar Ataxia |
|
- Biological: Intravenous transfer of of Bone Marrow derived Mono Nuclear Stem Cell (BMMNCs)
|
Interventional |
Phase 1
Phase 2 |
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- improve in INTERNATIONAL CO-OPERATIVE ATAXIA RATING SCALE
- Balance test
- Tremor Rating Scale
- quality of life
|
20 |
All |
18 Years to 65 Years (Adult) |
NCT01958177 |
CSCC/BMCA/2013//01 |
|
September 2014 |
December 2016 |
December 2016 |
October 9, 2013 |
September 17, 2014 |
|
- Chaitanya Hospital
Pune,, Maharashtra, India
|
| 15 |
NCT03122925 |
Enrolling by invitation |
TCA Cycle in the Dentate in Friedreich's Ataxia |
|
|
Observational |
|
- University of Minnesota - Clinical and Translational Science Institute
|
Other |
- Observational Model: Case-Control
- Time Perspective: Prospective
|
- TCA cycle rate in the dentate nucleus
|
20 |
All |
18 Years and older (Adult, Senior) |
NCT03122925 |
STUDY00000143 |
|
August 15, 2017 |
July 1, 2018 |
July 1, 2019 |
April 21, 2017 |
August 25, 2017 |
|
- CMRR
Minneapolis, Minnesota, United States
|
| 16 |
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...)
|
| 17 |
NCT01104649 |
Completed |
Efficacy of Riluzole in Hereditary Cerebellar Ataxia |
|
- Drug: riluzole
- Drug: Placebo comparator
|
Interventional |
Phase 2
Phase 3 |
- S. Andrea Hospital
- Agenzia Italiana del Farmaco
|
Other |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Double (Participant, Investigator)
- Primary Purpose: Treatment
|
- Scale for the assessment and rating of ataxia (SARA)
- Baropodometric parameters
- Quality of life
- Depression
|
60 |
All |
14 Years to 70 Years (Child, Adult, Senior) |
NCT01104649 |
FARM7KAJM7 |
|
April 2010 |
February 2014 |
March 2014 |
April 15, 2010 |
April 21, 2015 |
|
- Center for Experimental Neurological Therapies (CENTERS), S. Andrea Hospital, II Faculty of Medicine, "Sapienza" University of Rome
Rome, Italy
|
| 18 |
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 |
|
|
| 19 |
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
|
| 20 |
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, Senior) |
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
|
| 21 |
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, Senior) |
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...)
|
| 22 |
NCT01962363 |
Completed |
EPI-743 in Friedreich's Ataxia Point Mutations |
|
|
Interventional |
Phase 2 |
- University of South Florida
- Edison Pharmaceuticals Inc
- Friedreich's Ataxia Research Alliance
|
Other / Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Visual function
- Safety parameters
- Neurologic function
- (and 6 more...)
|
4 |
All |
18 Years to 65 Years (Adult) |
NCT01962363 |
EPI-743 PM |
|
October 2013 |
June 2016 |
June 2016 |
October 14, 2013 |
June 29, 2016 |
|
- University of South Florida
Tampa, Florida, United States
|
| 23 |
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, Senior) |
NCT02540655 |
IB02 |
|
September 2015 |
November 2017 |
February 2018 |
September 4, 2015 |
November 1, 2016 |
|
- Taipei Veterans General Hospital
Taipei, Taiwan
|
| 24 |
NCT02593773 |
Completed |
Safety, Tolerability and Efficacy of ACTIMMUNE Dose Escalation in Friedreich's Ataxia Study |
|
|
Interventional |
Phase 3 |
- Horizon Pharma Ireland, Ltd., Dublin Ireland
- Friedreich's Ataxia Research Alliance
|
Industry / Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- To evaluate the long-term safety effect of ACTIMMUNE® (interferon [IFN]-γ 1b) in subjects with FA
- To evaluate the effect of ACTIMMUNE® (interferon [IFN]-γ 1b) on the Friedreich's Ataxia Rating Scale (FARS-mNeuro score)
- The effect of ACTIMMUNE® on Activities of Daily Living (ADL)
- (and 3 more...)
|
86 |
All |
10 Years to 26 Years (Child, Adult) |
NCT02593773 |
HZNP-ACT-302 |
STEADFAST |
December 2015 |
March 21, 2017 |
March 21, 2017 |
November 2, 2015 |
April 21, 2017 |
|
- University of California, Los Angeles Neurology Clinic
Los Angeles, California, United States - University of Florida - Clinical Research Center
Gainesville, Florida, United States - University of Iowa Children's Hospital
Iowa City, Iowa, United States - Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
|
| 25 |
NCT00957216 |
Completed |
Coenzyme Q10 in Adult-Onset Ataxia |
|
- Drug: Placebo (sugar pill)
- Drug: Coenzyme Q10
|
Interventional |
Phase 1 |
- The University of Texas Medical Branch, Galveston
- University of Florida
|
Other |
- Allocation: Randomized
- Intervention Model: Single Group Assignment
- Masking: Triple (Participant, Care Provider, Investigator)
- Primary Purpose: Supportive Care
|
- Measure cerebellar functions by a validated scale (SARA)
|
1 |
All |
18 Years to 80 Years (Adult, Senior) |
NCT00957216 |
08-129
08-SAOA-001 |
CoQATAX |
April 2008 |
August 2009 |
August 2009 |
August 12, 2009 |
April 16, 2010 |
|
- The Universtity of Texas, Galveston
Galveston, Texas, United States
|
| 26 |
NCT01921868 |
Unknown † |
An Open-label Study of the Effects of Acetyl-L-Carnitine on Cardiovascular Outcomes in Friedreich's Ataxia |
|
|
Interventional |
Not Applicable |
- University of South Florida
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- To observe the changes in cardiac functioning in patients with Friedreich's Ataxia between study endpoint and baseline.
- To assess the changes in Friedreich's Ataxia symptoms and severity (as measured by clinical rating scales) compared to baseline.
- Changes in patient global impression of improvement
- (and 4 more...)
|
20 |
All |
18 Years to 80 Years (Adult, Senior) |
NCT01921868 |
ALCAR-8499 |
|
August 2013 |
June 2017 |
June 2017 |
August 13, 2013 |
April 12, 2016 |
|
- University of South Florida
Tampa, Florida, United States
|
| 27 |
NCT02345200 |
Completed |
Body Composition and Hormonal Status in Ataxia Telangiectasia |
|
- Procedure: bioelectrical impedance analysis
- Procedure: blood draw
|
Interventional |
Not Applicable |
- Johann Wolfgang Goethe University Hospital
|
Other |
- Allocation: Non-Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Supportive Care
|
|
52 |
All |
2 Years to 45 Years (Child, Adult) |
NCT02345200 |
BIA_AT2014 |
|
April 2013 |
April 2014 |
April 2014 |
January 26, 2015 |
January 26, 2015 |
|
|
| 28 |
NCT01975909 |
Completed
Has Results |
Transcranial Magnetic Stimulation in Spino-Cerebellar Ataxia |
|
- Device: Transcranial Magnetic Stimulation
|
Interventional |
Not Applicable |
- Beth Israel Deaconess Medical Center
- National Institute of Neurological Disorders and Stroke (NINDS)
|
Other / NIH |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Triple (Participant, Care Provider, Outcomes Assessor)
- Primary Purpose: Treatment
|
- Percent Change From Baseline to Post Treatment on the Scale for the Assessment and Rating of Ataxia (SARA)
- Percent Change From Baseline to Post Treatment on the Timed 25-Foot Walk
- Percent Change From Baseline to Post Treatment on the 9-hole Peg Test
|
20 |
All |
18 Years to 70 Years (Adult, Senior) |
NCT01975909 |
2013P000233
1R21NS085491-01 |
TMS |
September 2013 |
October 2016 |
November 2016 |
November 5, 2013 |
May 30, 2017 |
May 30, 2017 |
- Beth Israel Deaconess Medical Center
Boston, Massachusetts, United States
|
| 29 |
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, Senior) |
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
|
| 30 |
NCT02069509 |
Recruiting |
Patient Registry of the European Friedreich's Ataxia Consortium for Translational Studies (EFACTS) |
|
|
Observational |
|
- European Friedreich's Ataxia Consortium for Translational Studies
- 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, Senior |
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...)
|
| 31 |
NCT02840669 |
Active, not recruiting |
A Study to Characterize the Cardiac Phenotype of Individuals With Friedreich's Ataxia (CARFA Study) |
|
- Procedure: Cardiac magnetic resonance imaging (CMR)
- Procedure: Exercise-stress test
- Procedure: Echocardiography (ECHO)
- Procedure: Cardiac-related blood studies
|
Interventional |
Not Applicable |
- Adverum Biotechnologies, Inc.
- Adverum Biotechnologies SAS, a wholly owned subsidiary of Adverum Biotechnologies, Inc.
- Weill Medical College of Cornell University
|
Industry / Other |
- Intervention Model: Factorial Assignment
- Masking: None (Open Label)
- Primary Purpose: Diagnostic
|
- Exercise-stress test
- Cardiac magnetic resonance imaging (CMR)
- Echocardiogram
- (and 2 more...)
|
40 |
All |
18 Years and older (Adult, Senior) |
NCT02840669 |
CM-FA-101 |
|
July 2016 |
June 2018 |
September 2018 |
July 21, 2016 |
July 11, 2017 |
|
- Hôpital Pitié-Salpêtrière, AP-HP
Paris, France
|
| 32 |
NCT02316314 |
Recruiting |
Characterization of the Cardiac Phenotype of Friedreich's Ataxia (FRDA) |
|
- Procedure: Cardiac magnetic resonance imaging (CMR)
- Procedure: Exercise-stress test
- Procedure: Echocardiogram (ECHO)
- Procedure: Cardiac-related blood studies
|
Observational |
|
- Weill Medical College of Cornell University
|
Other |
- Observational Model: Case-Control
- Time Perspective: Cross-Sectional
|
- Level of troponin, BNP, and CPK in blood
- echocardiogram
- exercise-stress test
|
45 |
All |
18 Years to 30 Years (Adult) |
NCT02316314 |
1408015429 |
|
December 2014 |
December 2017 |
December 2017 |
December 12, 2014 |
May 25, 2017 |
|
- Weill Cornell Medical College
New York, New York, United States
|
| 33 |
NCT00530127 |
Completed |
A Study Investigating the Safety and Tolerability of Deferiprone in Patients With Friedreich's Ataxia |
|
- Drug: placebo
- Drug: deferiprone
|
Interventional |
Phase 1
Phase 2 |
|
Industry |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Double (Participant, Investigator)
- Primary Purpose: Treatment
|
- The patient's tolerance of treatment will be determined, as assessed by the occurrence of adverse events
- The efficacy endpoints will be change in the score for 9-Hole Peg Test (9HPT), Timed 25-Foot Walk (T25FW), Low-Contrast Letter Acuity test (LCLA), International Cooperative Ataxia Rating Scale (ICARS), and Friedreich's Ataxia Rating Scale (FARS).
|
80 |
All |
7 Years to 35 Years (Child, Adult) |
NCT00530127 |
LA29-0207 |
|
April 2008 |
July 2009 |
July 2009 |
September 17, 2007 |
June 2, 2010 |
|
- Murdoch Children's Research Institute
Victoria, Australia - Hospital Erasme
Brussels, Belgium - McMaster University
Hamilton, Ontario, Canada - (and 3 more...)
|
| 34 |
NCT02660112 |
Active, not recruiting |
(+) Epicatechin to Treat Friedreich's Ataxia |
|
|
Interventional |
Phase 2 |
- Ralitza Gavrilova
- Cardero Therapeutics, Inc.
- Mayo Clinic
|
Other / Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Change from baseline in Friedreich Ataxia Rating Scale (FARS) composite score
- Change from baseline in ventricular hypertrophy as shown on cardiac MRI
|
10 |
All |
10 Years to 50 Years (Child, Adult) |
NCT02660112 |
15-006845 |
|
September 2016 |
December 2018 |
December 2018 |
January 21, 2016 |
November 1, 2017 |
|
- Mayo Clinic
Rochester, Minnesota, United States
|
| 35 |
NCT02733679 |
Completed |
Response of Individuals With Ataxia-Telangiectasia to Metformin and Pioglitazone |
|
- Drug: Metformin
- Drug: Pioglitazone
|
Interventional |
Phase 4 |
- NHS Tayside
- University of Dundee
|
Other |
- Allocation: Non-Randomized
- Intervention Model: Crossover Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Change in insulin sensitivity after taking metformin in A-T compared to controls
- Difference in insulin sensitivity at baseline between groups.
- Change in insulin sensitivity after taking pioglitazone in A-T compared to controls
- Difference in fat distribution between groups
|
27 |
All |
18 Years to 30 Years (Adult) |
NCT02733679 |
2016GE03 |
RAMP |
September 29, 2016 |
August 30, 2017 |
August 30, 2017 |
April 11, 2016 |
December 4, 2017 |
|
- Ninewells Hospital
Dundee, Angus, United Kingdom
|
| 36 |
NCT01716221 |
Completed
Has Results |
An Objective Double-blind Evaluation of Bupropion and Citalopram in an Individual With Friedreich Ataxia |
|
- Drug: bupropion & Citalopram
- Drug: Bupropion & Placebo
- Drug: Placebo & Citalopram
- Drug: Placebo & Placebo
|
Interventional |
Phase 4 |
- University of Colorado, Denver
|
Other |
- Allocation: Randomized
- Intervention Model: Crossover Assignment
- Masking: Triple (Participant, Investigator, Outcomes Assessor)
- Primary Purpose: Supportive Care
|
- International Cooperative Ataxia Rating Scale (ICARS)
- Friedreich Ataxia Rating Scale (FARS)
- Comparison of FARS and ICARS
|
1 |
Female |
Child, Adult, Senior |
NCT01716221 |
11-1141 |
|
October 2012 |
March 2013 |
March 2013 |
October 29, 2012 |
September 19, 2016 |
September 19, 2016 |
|
| 37 |
NCT02287064 |
Recruiting |
An Open-label Trial of Intravenous Immune Globulin (IVIG)in Treating Spinocerebellar Ataxias |
|
- Drug: Intravenous Immune Globulin (IVIG)
|
Interventional |
Phase 1 |
- University of South Florida
- Baxter Healthcare Corporation
|
Other / Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Changes in Scale for the Assessment and Rating Ataxia (SARA)
- clinician and patient global impression of improvement (CGI and PGI)
- Neurologic dysfunction as assessed by STAND scores
- 9-hole peg test
|
10 |
All |
18 Years to 80 Years (Adult, Senior) |
NCT02287064 |
SCA IVIG 2014 |
|
April 2015 |
December 2016 |
|
November 10, 2014 |
June 8, 2016 |
|
- University of South Florida
Tampa, Florida, United States
|
| 38 |
NCT01776164 |
Enrolling by invitation |
Early and Longitudinal Assessment of Neurodegeneration in the Brain and Spinal Cord in Friedreich's Ataxia |
|
|
Observational |
|
- University of Minnesota - Clinical and Translational Science Institute
- Bob Allison Ataxia Research Center
- Friedreich's Ataxia Research Alliance
|
Other |
- Observational Model: Cohort
- Time Perspective: Cross-Sectional
|
- Difference in connectivity (apparent coefficient of diffusion, fractional anisotropy, radial and axial diffusivity), anatomy (cortical thickness, volumetry analysis) and biochemistry (metabolite concentrations) between patients and controls
|
32 |
All |
10 Years and older (Child, Adult, Senior) |
NCT01776164 |
FRDA_MRI |
|
January 2013 |
October 2018 |
October 2018 |
January 28, 2013 |
January 17, 2018 |
|
- University of Minnesota
Minneapolis, Minnesota, United States
|
| 39 |
NCT01339884 |
Completed |
A Study of Resveratrol as Treatment for Friedreich Ataxia |
|
|
Interventional |
Phase 1
Phase 2 |
- Murdoch Childrens Research Institute
- Friedreich's Ataxia Research Alliance
|
Other |
- Allocation: Non-Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Lymphocyte frataxin level
- Oxidative stress markers
- Clinical rating scales of ataxia
- (and 2 more...)
|
27 |
All |
18 Years and older (Adult, Senior) |
NCT01339884 |
10358B |
|
April 2011 |
August 2012 |
December 2012 |
April 21, 2011 |
January 22, 2014 |
|
- Monash Medical Centre, Southern Health
Clayton, Melbourne, Victoria, Australia
|
| 40 |
NCT01470729 |
Completed |
Biomarkers in Autosomal Dominant Cerebellar Ataxia |
- Spinocerebellar Ataxia Type 1
- Spinocerebellar Ataxia Type 2
- Spinocerebellar Ataxia, Autosomal Recessive 3
- Episodic Ataxia, Type 7
|
- Other: metabolic and imaging biomarkers in SCA1,2,3 and 7 patients
|
Observational |
|
- Assistance Publique - Hôpitaux de Paris
|
Other |
- Time Perspective: Prospective
|
- metabolic biomarkers of SCA
- imaging biomarkers of SCA
|
102 |
All |
18 Years and older (Adult, Senior) |
NCT01470729 |
P100125 |
BIOSCA |
November 2011 |
December 2015 |
December 2015 |
November 11, 2011 |
December 16, 2015 |
|
- Groupe Hospitalier Pitié Salpêtrière
Paris, France
|
| 41 |
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, Senior) |
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...)
|
| 42 |
NCT01016366 |
Completed |
Safety Study of Carbamylated Erythropoietin to Treat Patients With the Neurodegenerative Disorder Friedreich's Ataxia |
|
- Drug: Lu AA24493
- Drug: Placebo
|
Interventional |
Phase 2 |
|
Industry |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Double (Participant, Investigator)
- Primary Purpose: Treatment
|
- To evaluate the safety and tolerability of 2 weeks treatment with Lu AA24493 in patients with Friedreich's Ataxia
- To explore biomarkers of efficacy, including frataxin, 8-OHdG & peroxides
- To explore efficacy by neurological assessment (Scale for the Assessment and Rating of Ataxia (SARA), Friedreich's Ataxia Rating Scale (FARS))
- (and 3 more...)
|
36 |
All |
18 Years and older (Adult, Senior) |
NCT01016366 |
12631A
2008-003662-25 |
|
October 2009 |
March 2011 |
April 2011 |
November 19, 2009 |
November 8, 2016 |
|
- AT001
Innsbruck, Austria - DE004
Bochum, Germany - DE002
Bonn, Germany - (and 4 more...)
|
| 43 |
NCT00803868 |
Terminated |
Pilot Study of Varenicline (Chantix®) in the Treatment of Friedreich's Ataxia |
|
- Drug: varenicline
- Drug: placebo
|
Interventional |
Phase 2
Phase 3 |
- University of South Florida
- Friedreich's Ataxia Research Alliance
- Pfizer
|
Other / Industry |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- Friedreich Ataxia Rating Scale (FARS)
|
28 |
All |
18 Years to 80 Years (Adult, Senior) |
NCT00803868 |
6 |
|
May 2009 |
May 2010 |
May 2010 |
December 8, 2008 |
July 24, 2012 |
|
- University of South Florida
Tampa, Florida, United States - The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
|
| 44 |
NCT02960893 |
Active, not recruiting |
Trial in Adult Subjects With Spinocerebellar Ataxia |
- Spinocerebellar Ataxias
- Spinocerebellar Ataxia Type 1
- Spinocerebellar Ataxia Type 2
- (and 5 more...)
|
- Drug: BHV-4157
- Drug: Placebo Comparator
|
Interventional |
Phase 2
Phase 3 |
- Biohaven Pharmaceutical Holding Company Ltd.
- Cognitive Research Corporation
- Cytel Inc.
|
Industry / Other |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- To measure the change in total score on the Scale for Assessment and Rating of Ataxia (SARA)
- • To assess the safety and tolerability of BHV-4157 in subjects with SCA by measuring the frequency and severity of adverse events and discontinuations of adverse events.
- To compare efficacy of BHV-4157 with placebo on patient impression of benefit via use of the PGI-C
|
120 |
All |
18 Years to 75 Years (Adult, Senior) |
NCT02960893 |
BHV4157-201 |
|
December 2016 |
August 18, 2017 |
August 2018 |
November 10, 2016 |
August 24, 2017 |
|
- St. Joseph's Hospital and Medical Center
Phoenix, Arizona, United States - CNS Trial
Long Beach, California, United States - University of California, Los Angeles
Los Angeles, California, United States - (and 15 more...)
|
| 45 |
NCT00697073 |
Completed
Has Results |
Study to Assess the Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia Patients |
|
|
Interventional |
Phase 3 |
|
Industry |
- Allocation: Non-Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Change in ICARS
- FARS (Friedreich's Ataxia Rating Scale)
- Nature and Frequency of Adverse Events
|
68 |
All |
8 Years to 18 Years (Child, Adult) |
NCT00697073 |
SNT-III-002-E |
IONIA-E |
July 2008 |
May 2010 |
May 2010 |
June 13, 2008 |
June 4, 2013 |
August 4, 2011 |
- David Geffen School of Medicine, UCLA
Los Angeles, California, United States - The Children's Hopsital of Philadelphia
Philadelphia, Pennsylvania, United States
|
| 46 |
NCT02445794 |
Completed |
A First in Human Study of RT001 in Patients With Friedreich's Ataxia |
|
- Drug: RT001
- Drug: RT001 comparator
|
Interventional |
Phase 1
Phase 2 |
|
Industry |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- Number of patients with adverse events
- Pharmacokinetics - Area under the concentration-time curve after a single dose
- Pharmacokinetics - Maximum observed plasma concentration after a single dose
- (and 14 more...)
|
19 |
All |
18 Years to 50 Years (Adult) |
NCT02445794 |
RT001-002 |
|
August 2015 |
June 2016 |
July 2016 |
May 15, 2015 |
October 4, 2016 |
|
- Collaborative Neuroscience Network, LLC
Long Beach, California, United States - University of South Florida
Tampa, Florida, United States
|
| 47 |
NCT01350440 |
Completed |
Safety and Efficacy of Intravenous Immune Globulin in Treating Spinocerebellar Ataxia |
|
|
Interventional |
Phase 2 |
- University of South Florida
- Baxter Healthcare Corporation
|
Other / Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Scale for the Assessment and Rating of Ataxia
- Timed 25 foot walk
- Clinical Global impression
- (and 5 more...)
|
5 |
All |
10 Years to 50 Years (Child, Adult) |
NCT01350440 |
10 |
|
August 2011 |
July 2013 |
July 2013 |
May 9, 2011 |
July 23, 2013 |
|
- University of South Florida
Tampa, Florida, United States
|
| 48 |
NCT00905268 |
Completed
Has Results |
A Study of Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia (FRDA) Patients |
|
- Drug: idebenone
- Drug: Placebo
|
Interventional |
Phase 3 |
|
Industry |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- Absolute Change in International Cooperative Ataxia Rating Scale (ICARS) Scores From Baseline Assessment to Week 52
- Absolute Change in Friedreich's Ataxia Rating Scale (FARS) Scores From Baseline Assessment to Week 52
- Proportion of Patients Improving (Responding) on ICARS by a Clinically Relevant Margin
- (and 3 more...)
|
232 |
All |
8 Years and older (Child, Adult, Senior) |
NCT00905268 |
SNT-III-001 |
MICONOS |
April 2006 |
January 2010 |
January 2010 |
May 20, 2009 |
June 27, 2016 |
June 27, 2016 |
- Universitätsklinik Innsbruck
Innsbruck, Austria - Hôpital Erasme - Université Libre de Bruxelles
Bruxelles, Belgium - Hôpital de la Salpêtrière - INSERM U679, Neurologie et Thérapeutique expérimentale
Paris, France - (and 10 more...)
|
| 49 |
NCT01360164 |
Unknown † |
Safety and Efficacy of Umbilical Cord Mesenchymal Stem Cell Therapy for Patients With Hereditary Ataxia |
|
- Biological: human umbilical cord mesenchymal stem cells
|
Interventional |
Phase 1
Phase 2 |
- Shenzhen Beike Bio-Technology Co., Ltd.
- The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical School
- The Affiliated Nanjing Brain Hospital of Nanjing University Medical School
- (and 3 more...)
|
Industry / Other |
- Allocation: Non-Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- scores of International Cooperative Ataxia Rating Scale （ICARS） scale and Berg Scale
- the volume of Cerebellum of Brain Magnetic Resonance Imaging （MRI） Scan
- Number of Participants with Adverse Events as a Measure of Safety and Tolerability
- Number of Participants with Serious Adverse Events as a Measure of Safety and Tolerability
|
20 |
All |
16 Years to 65 Years (Child, Adult) |
NCT01360164 |
BKCR-HA-1.0(2010) |
|
January 2010 |
December 2012 |
December 2013 |
May 25, 2011 |
November 28, 2012 |
|
- Nanjing University Medical College Affiliated Drum Tower Hospital
Nanjing, Jiangsu, China
|
| 50 |
NCT00897221 |
Completed |
A Study Investigating the Long-term Safety and Efficacy of Deferiprone in Patients With Friedreich's Ataxia |
|
- Drug: Deferiprone oral solution 100mg/mL
- Drug: Deferiprone oral solution 100 mg/mL
|
Interventional |
Phase 2 |
|
Industry |
- Allocation: Non-Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- The patient's long-term tolerance of treatment will be assessed by the occurence of adverse events.
- The long-term efficacy of deferiprone will be assessed. Efficacy measures include the 9HPT, T25FW, LCLA, ICARS and FARS.
|
36 |
All |
7 Years to 35 Years (Child, Adult) |
NCT00897221 |
LA29-EXT |
|
June 2009 |
March 2011 |
March 2011 |
May 12, 2009 |
June 27, 2011 |
|
- Hospital Erasme
Brussels, Belgium - Hospital Necker-Enfants Malades
Paris, France - Fondazione IRCCS Istituto Neurologico "C. Besta"
Milan, Italy - La Fundacion Para la Investigacion Biomedica
Madrid, Spain
|
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