| 1 |
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
|
| 2 |
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
|
| 3 |
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
|
| 4 |
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
|
| 5 |
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
|
| 6 |
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
|
| 7 |
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, 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...)
|
| 8 |
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
|
| 9 |
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
|
| 10 |
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...)
|
| 11 |
NCT00811681 |
Completed |
Effect of Pioglitazone Administered to Patients With Friedreich's Ataxia: Proof of Concept |
|
- Drug: pioglitazone
- Drug: Placebo
|
Interventional |
Phase 3 |
- Assistance Publique - Hôpitaux de Paris
|
Other |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Double (Participant, Investigator)
- Primary Purpose: Treatment
|
- evaluate the efficacy of Pioglitazone on the neurological function of FA patients. Success will be defined as a stabilisation or improvement on ICARS designed as no more than 2 points maximum increment on this scale in two year.
- tolerance of Pioglitazone
- efficacy of Pioglitazone on neurological function
- (and 2 more...)
|
40 |
All |
7 Years to 24 Years (Child, Adult) |
NCT00811681 |
P070123 |
ACTFRIE |
December 2008 |
March 2013 |
March 2013 |
December 19, 2008 |
September 4, 2013 |
|
- Hôpital Robert Debré
Paris, 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 |
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
|
| 14 |
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
|
| 15 |
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
|
| 16 |
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
|
| 17 |
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...)
|
| 18 |
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
|
| 19 |
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
|
| 20 |
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
|
| 21 |
NCT00537680 |
Completed
Has Results |
Study to Assess the Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia |
|
- 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
|
|
70 |
All |
8 Years to 17 Years (Child) |
NCT00537680 |
SNT-III-002 |
IONIA |
December 2007 |
April 2009 |
April 2009 |
October 1, 2007 |
February 3, 2012 |
December 29, 2011 |
- David Geffen School of Medicine, UCLA
Los Angeles, California, United States - The Children's Hopsital of Philadelphia
Philadelphia, Pennsylvania, United States
|
| 22 |
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
|
| 23 |
NCT01728064 |
Completed |
Safety and Efficacy of EPI-743 in Patients With Friedreich's Ataxia |
|
- Drug: Placebo
- Drug: EPI-743 400 mg
- Drug: EPI-743 200 mg
|
Interventional |
Phase 2 |
- Edison Pharmaceuticals Inc
|
Industry |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- Visual Function
- Color vision
- Neurologic function
- (and 6 more...)
|
60 |
All |
18 Years to 45 Years (Adult) |
NCT01728064 |
EPI2010-006 |
|
December 2012 |
October 2015 |
February 2016 |
November 16, 2012 |
March 21, 2016 |
|
- University of California Los Angeles
Los Angeles, California, United States - University of South Florida
Tampa, Florida, United States - Childrens Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
|
| 24 |
NCT01303406 |
Completed
Has Results |
Patient Reported Outcomes in Friedreich's Ataxia Patients After Withdrawal From Treatment With Idebenone (PROTI) |
|
- 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
|
- Patient Assessment of Treatment Assignment: Comparison of the Proportions of Patients Randomised to Idebenone and Placebo Who Assessed That They Received Idebenone
- Comparison of the Percentage of Participants Randomised to Idebenone and Placebo Who Withdrew Early Due to Recurrence or Worsening of FRDA Symptoms
|
29 |
All |
10 Years and older (Child, Adult, Senior) |
NCT01303406 |
SNT-III-004 |
PROTI |
April 2011 |
July 2012 |
July 2012 |
February 24, 2011 |
March 9, 2016 |
March 9, 2016 |
- Innsbruck, Austria
- Bonn, Germany
- München, Germany
- (and 3 more...)
|
| 25 |
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 |
|
| 26 |
NCT01898884 |
Completed
Has Results |
Safety and Pharmacology Study of VP 20629 in Adults With Friedreich's Ataxia |
|
- Drug: VP 20629
- Drug: Placebo
|
Interventional |
Phase 1 |
|
Industry |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Double (Participant, Investigator)
- Primary Purpose: Treatment
|
- Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs)
- Number of Participants With Clinically Significant Laboratory Abnormalities Recorded as Treatment-Emergent Adverse Events (TEAEs)
- Number of Participants With Vital Signs Abnormalities Recorded as Treatment-Emergent Adverse Events (TEAEs)
- (and 28 more...)
|
46 |
All |
18 Years to 45 Years (Adult) |
NCT01898884 |
20629-100 |
|
July 2013 |
July 2015 |
July 2015 |
July 15, 2013 |
October 6, 2016 |
October 6, 2016 |
- UCLA Medical Center
Los Angeles, California, United States - University of South Florida
Tampa, Florida, United States - Emory University
Atlanta, Georgia, United States - (and 2 more...)
|
| 27 |
NCT01493973 |
Completed |
Efficacy Study of Epoetin Alfa in Friedreich Ataxia |
|
- Drug: Epoetin alfa
- Drug: Placebo
|
Interventional |
Phase 2 |
- Federico II University
- Friedreich's Ataxia Research Alliance
- Associazione Italiana per la lotta alle Sindromi Atassiche (AISA)
|
Other |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- Peak oxygen uptake (VO2 max) at the cardiopulmonary exercise test (CPET)
- Secondary outcome variables at the CPET (anaerobic threshold, ventilatory efficiency, exercise duration, and power output).
- Frataxin levels in peripheral blood mononuclear cells (PBMCs).
- (and 5 more...)
|
56 |
All |
12 Years and older (Child, Adult, Senior) |
NCT01493973 |
FA_BBK_8 |
FRIEMAX |
January 2013 |
October 2014 |
June 2015 |
December 16, 2011 |
August 11, 2015 |
|
- Università di Bari
Bari, BA, Italy - Università la Sapienza, Neurologia C
Rome, RM, Italy - Dipartimento di Scienze Neurologiche
Napoli, Italy
|
| 28 |
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...)
|
| 29 |
NCT00229632 |
Completed |
Idebenone to Treat Friedreich's Ataxia |
|
|
Interventional |
Phase 2 |
- National Institute of Neurological Disorders and Stroke (NINDS)
- National Institutes of Health Clinical Center (CC)
|
NIH |
- Allocation: Randomized
- Intervention Model: Single Group Assignment
- Masking: Triple (Participant, Care Provider, Investigator)
- Primary Purpose: Treatment
|
- To examine the change in the level of oxidative stress by measuring the oxidative marker 8-hydroxy-2-deoxyguanosine from baseline and after 6 months of treatment with placebo or varying doses of idebenone
- To evaluate the safety and tolerability of idebenone
- To explore the effects of idebenone on cardiac parameters
- (and 4 more...)
|
51 |
All |
9 Years to 17 Years (Child) |
NCT00229632 |
050245
05-N-0245 |
|
September 27, 2005 |
December 17, 2007 |
December 17, 2007 |
September 29, 2005 |
March 29, 2018 |
|
- National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States
|
| 30 |
NCT01035671 |
Completed |
Safety and Efficacy Study of A0001 in Subjects With Friedreich's Ataxia |
|
- Drug: alpha-tocopherolquinone (A0001)
- Drug: placebo
|
Interventional |
Phase 2 |
- Penwest Pharmaceuticals Co.
|
Industry |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Treatment
|
- Change in Disposition Index of Glucose Regulation, determined by Frequent Sampling IV Glucose Tolerance Test
- Sensitivity index (SI) calculated from IVGTT
- Glucose effectiveness (SG) calculated from IVGTT
- (and 13 more...)
|
42 |
All |
18 Years to 60 Years (Adult) |
NCT01035671 |
FRD02 |
|
December 2009 |
February 2011 |
March 2011 |
December 21, 2009 |
April 22, 2011 |
|
- The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
|
| 31 |
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
|
| 32 |
NCT00824512 |
Completed
Has Results |
Efficacy of EGb761 in Patients Suffering From Friedreich Ataxia |
|
- Drug: EGb 761 120 mg
- Drug: Placebo
|
Interventional |
Phase 2 |
|
Industry |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Primary Purpose: Supportive Care
|
- Creatine Rephosphorylation Rate Post Exercise
- Peak Post Exercise Perfusion
- Time to Peak Perfusion
- (and 19 more...)
|
22 |
All |
12 Years to 22 Years (Child, Adult) |
NCT00824512 |
2-39-00240-133
2007-005371-34 |
|
June 2008 |
October 2011 |
October 2011 |
January 16, 2009 |
May 21, 2013 |
May 21, 2013 |
- Hospital Necker Enfants Malades
Paris, France
|
| 33 |
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
|
| 34 |
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...)
|
| 35 |
NCT00631202 |
Completed |
Efficacy of Epoetin Alfa in Patients With Friedreich's Ataxia |
|
|
Interventional |
Phase 2 |
|
Other |
- Allocation: Non-Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Primary endpoint will be the frataxin level in PBMCs from patients at different timing from a single Epoetin alfa administration.
- Echocardiography: Strain and strain rate after EPO administration at the highest study dose
- Safety laboratory parameters, adverse events and tolerability
- International cooperative ataxia rating scale (ICARS).
|
10 |
All |
18 Years to 50 Years (Adult) |
NCT00631202 |
FA_EPO_3 |
|
February 2008 |
December 2008 |
June 2009 |
March 7, 2008 |
May 27, 2010 |
|
- Dipartimento di Scienze Neurologiche
Naples, Italy
|
| 36 |
NCT00224640 |
Completed |
Iron-Chelating Therapy and Friedreich Ataxia |
|
- Drug: Iron chelating intervention
|
Interventional |
Phase 1
Phase 2 |
- Assistance Publique - Hôpitaux de Paris
|
Other |
- Allocation: Non-Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- assessment of iron overload at TO and month2 by imagery
- Clinical (monthly) and biological parameter follow- up ( blood count,
- plasma iron, ferritin, transferrin and liver enzymes)
|
15 |
All |
13 Years and older (Child, Adult, Senior) |
NCT00224640 |
P041201
PCR 05001 |
|
March 2005 |
March 2008 |
March 2008 |
September 23, 2005 |
March 4, 2009 |
|
- Necker Hospital
Paris, France
|
| 37 |
NCT00078481 |
Completed |
Phase 1 Trial of Idebenone to Treat Patients With Friedreich's Ataxia |
|
|
Interventional |
Phase 1 |
- National Institute of Neurological Disorders and Stroke (NINDS)
- National Institutes of Health Clinical Center (CC)
|
NIH |
- Primary Purpose: Treatment
|
|
16 |
All |
Child, Adult, Senior |
NCT00078481 |
040129
04-N-0129 |
|
February 2004 |
|
April 2006 |
March 1, 2004 |
March 4, 2008 |
|
- National Institute of Neurological Disorders and Stroke (NINDS)
Bethesda, Maryland, United States
|
| 38 |
NCT00056186 |
Completed |
Transitional Life Events in Patients With Friedreich's Ataxia: Implications for Genetic Counseling |
|
|
Observational |
|
- National Human Genome Research Institute (NHGRI)
- National Institutes of Health Clinical Center (CC)
|
NIH |
|
|
40 |
All |
Child, Adult, Senior |
NCT00056186 |
030118
03-HG-0118 |
|
March 2003 |
|
January 2004 |
March 7, 2003 |
March 4, 2008 |
|
- National Human Genome Research Institute (NHGRI)
Bethesda, Maryland, United States
|
| 39 |
NCT00015808 |
Completed |
Safety Study of Idebenone to Treat Friedreich's Ataxia |
|
|
Interventional |
Phase 1 |
- National Institute of Neurological Disorders and Stroke (NINDS)
- National Institutes of Health Clinical Center (CC)
|
NIH |
- Primary Purpose: Treatment
|
|
100 |
All |
Child, Adult, Senior |
NCT00015808 |
010167
01-N-0167 |
|
May 2001 |
|
April 2006 |
May 7, 2001 |
July 12, 2006 |
|
- National Institute of Neurological Disorders and Stroke (NINDS)
Bethesda, Maryland, United States
|
| 40 |
NCT02035020 |
Completed |
A Phase IIa Trial to Test Safety and Efficacy Interferon Gamma Treatment in Elevating Frataxin Levels in FRDA Patients |
|
|
Interventional |
Phase 2 |
- Azienda Policlinico Umberto I
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Change in cellular frataxin
- Safety Blood sample
|
10 |
All |
18 Years to 45 Years (Adult) |
NCT02035020 |
GIFT/1 |
|
May 2013 |
July 30, 2014 |
July 30, 2014 |
January 14, 2014 |
April 21, 2017 |
|
- Policlinico Umberto I°
Rome, Italy/Rome, Italy
|
| 41 |
NCT02797080 |
Completed |
STEADFAST Long-Term Safety Extension |
|
|
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
|
- Number of Participants with Adverse Events
|
38 |
All |
11 Years to 27 Years (Child, Adult) |
NCT02797080 |
HZNP-ACT-303 |
|
June 2016 |
March 31, 2017 |
March 31, 2017 |
June 13, 2016 |
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
|
| 42 |
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...)
|
| 43 |
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 |
February 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
|
| 44 |
NCT01589809 |
Active, not recruiting |
Effect of Nicotinamide in Friedreich's Ataxia |
- Neurodegenerative Disorders
|
|
Interventional |
Phase 2 |
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Significant upregulation of Frataxin (FXN) in patients with Friedrich ataxia using an antibody dipstick assay (interventional part)
- Assessment of impact on clinical phenotype using the SARA scale to measure degree of ataxia (interventional part of the study)
- Use of novel highly-sensitive technology to capture clinical deficit (non-interventional part)
- (and 4 more...)
|
40 |
All |
18 Years and older (Adult, Senior) |
NCT01589809 |
CR01849 |
|
June 2012 |
June 2015 |
December 2017 |
May 2, 2012 |
July 2, 2017 |
|
- NIHR/Wellcome Trust Imperial CRF
London, Hammersmith, United Kingdom - National Hospital for Neurology and Neurosurgery
London, United Kingdom
|
| 45 |
NCT00993967 |
Completed
Has Results |
Long-Term Safety and Tolerability of Idebenone in Friedreich's Ataxia Patients (MICONOS Extension) |
|
|
Interventional |
Phase 3 |
|
Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Measures of Safety and Tolerability: Nature and Frequency of Adverse Events (AEs)
- Absolute Change in The International Cooperative Ataxia Rating Scale (ICARS)
|
200 |
All |
9 Years and older (Child, Adult, Senior) |
NCT00993967 |
SNT-III-001-E |
|
June 2007 |
June 2012 |
June 2012 |
October 14, 2009 |
March 5, 2018 |
March 5, 2018 |
- Universitätsklinik Innsbruck
Innsbruck, Austria - Hôpital Erasme - Univeristé Libre de Bruxelles
Bruxelles, Belgium - Hôpital de la Salpétrière - INSERM U679, Neurologie et Thérapeutique expérimentale
Paris, France - (and 8 more...)
|
| 46 |
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, Senior) |
NCT03285204 |
ELA-AF-OCT-2014-01 |
|
July 2015 |
June 2018 |
December 2019 |
September 15, 2017 |
April 10, 2018 |
|
- Pilar Rojas Lozano
Madrid, Spain
|
| 47 |
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
|
| 48 |
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
|
| 49 |
NCT03120013 |
Active, not recruiting |
Rehabilitative Trial With Cerebello-Spinal tDCS in Neurodegenerative Ataxia |
- Ataxia, Cerebellar
- Cerebellar Ataxia
- Spinocerebellar Ataxias
- (and 8 more...)
|
- Device: Anodal cerebellar and cathodal spinal tDCS
- Device: Sham cerebellar and sham spinal tDCS
|
Interventional |
Not Applicable |
- Azienda Ospedaliera Spedali Civili di Brescia
|
Other |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: Triple (Participant, Care Provider, Investigator)
- Primary Purpose: Treatment
|
- Change in the ICARS score from baseline
- Change in the SARA score from baseline
- Change in the 9HPT score from baseline
- (and 2 more...)
|
21 |
All |
18 Years and older (Adult, Senior) |
NCT03120013 |
NP1576 |
CStDCSAtaxia |
February 6, 2017 |
December 1, 2018 |
December 23, 2018 |
April 19, 2017 |
September 6, 2017 |
|
- AO Spedali Civili
Brescia, BS, Italy
|
| 50 |
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, Senior |
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
|
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