Clinical trials: Ataxia
Ataxia Telangiectasia
Whole Body MRI for Cancer Surveillance in A-T
Ataxia Telangiectasia (A-T) is an inherited disorder characterised by cerebellar neurodegeneration, immunodeficiency and respiratory disease.
- Trial name
- NCT05252819
- Testing
- Magnetic Resonance Imaging and an optional blood test
- Where
- United Kingdom
- Volunteers
- 37 people
- Sponsor
- University of Nottingham
Friedreich Ataxia
A Study to Learn About the Effects and Safety of RTA 408 (Omaveloxolone) in People Aged 16 to 40 With Friedreich's Ataxia
In this study, researchers are learning more about RTA 408, also known as omaveloxolone, BIIB141, or SKYCLARYS®. The main goal of this study is to learn more about the safety of RTA 408 and how it affects physical effort…
- Trial name
- NCT02255435
- Testing
- Omaveloxolone Capsules, 2.5 mg, Omaveloxolone Capsules, 5 mg, Omaveloxolone Capsules, 10 mg
- Where
- 11 sites in United States, Australia and 3 more
- Volunteers
- 172 people
- Sponsor
- Biogen
Ataxia Telangiectasia
Open-Label Extension of EryDex Study IEDAT-04-2022
This is an international, multi-center, prospective, open-label, non-comparative study aiming to provide access to treatment with EryDex to ataxia telangiectasia (A-T) patients who completed the IEDAT-04-2022 trial which studied…
- Trial name
- OLE_NEAT (NCT06664853)
- Testing
- Dexamethasone sodium phosphate
- Where
- 21 sites in United States, Denmark and 7 more
- Volunteers
- 101 people
- Sponsor
- Quince Therapeutics S.p.A.
Ataxia Telangiectasia
Evaluate the Neurological Effects of EryDex on Subjects With A-T
This is an international, multi-center, randomized, prospective, double-blind, placebo-controlled, Phase 3 study, designed to assess the effect of EryDex (dexamethasone sodium phosphate [DSP] in autologous erythrocytes)…
- Trial name
- NEAT (NCT06193200)
- Testing
- Dexamethasone sodium phosphate
- Where
- 19 sites in United States, Denmark and 7 more
- Volunteers
- 105 people
- Sponsor
- Quince Therapeutics S.p.A.
Friedreich Ataxia
A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
The primary objective of the study is to evaluate the efficacy (using the modified Friedreich Ataxia Rating Scale [mFARS]) and safety of vatiquinone in participants with Friedreich ataxia (FA).
- Trial name
- MOVE-FA (NCT04577352)
- Testing
- Vatiquinone
- Where
- 14 sites in United States, Australia and 7 more
- Volunteers
- 146 people
- Sponsor
- PTC Therapeutics
Ataxia Telangiectasia
N-Acetyl-L-Leucine for Ataxia-Telangiectasia (A-T)
This is a multinational, multicenter, open-label, rater-blinded prospective Phase II study which will assess the safety and efficacy of N-Acetyl-L-Leucine (IB1001) for the treatment of Ataxia-Telangiectasia (A-T).
- Trial name
- NCT03759678
- Testing
- IB1001
- Where
- 4 sites in United States, Germany and 2 more
- Volunteers
- 17 people
- Sponsor
- IntraBio Inc
Friedreich Ataxia 1
NAD+ and Exercise in FA
Randomized, placebo-controlled trial with a 2x2 factorial design testing the effects of an NAD+ precursor (NR) and exercise on Peak VO2 and Si in Friedreich's Ataxia (FA).
- Trial name
- ExRx in FA (NCT04192136)
- Testing
- Nicotinamide Riboside, Exercise Intervention
- Where
- United States
- Volunteers
- 75 people
- Sponsor
- Children's Hospital of Philadelphia
Friedreich Ataxia
FRDA Investigator Initiated Study (IIS) With Elamipretide
To evaluate the safety, tolerability, and activity of Elamipretide in treating vision loss in Friedreich Ataxia (FRDA).
- Trial name
- ELViS-FA (NCT05168774)
- Testing
- Elamipretide
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Children's Hospital of Philadelphia
Friedreich Ataxia
Neurology Measures in FA Children
The purpose of this study is to identify ways to follow progression of Friedreich's Ataxia (FA) and be able to measure changes over time in children with FA.
- Trial name
- NCT03418740
- Where
- 3 sites in United States
- Volunteers
- 108 people
- Sponsor
- Children's Hospital of Philadelphia
Friedreich's Ataxia
Safety, Tolerability and Efficacy of ACTIMMUNE® Dose Escalation in Friedreich's Ataxia
The purpose of this phase 3 randomized, multi-center, double-blind, placebo-controlled study is to evaluate the efficacy and safety of ACTIMMUNE® (interferon-γ 1b) in the treatment of Friedreich's Ataxia (FA) and to evaluate the…
- Trial name
- STEADFAST (NCT02415127)
- Testing
- Interferon γ-1b
- Where
- 4 sites in United States
- Volunteers
- 92 people
- Sponsor
- Amgen
Friedreich's Ataxia
Safety, Tolerability and Efficacy of ACTIMMUNE® Dose Escalation in Friedreich's Ataxia Study
The purpose of this phase 3 multi-center, open-label extension study is to evaluate the long-term safety of ACTIMMUNE® (interferon-γ 1b) in participants with Friedreich's Ataxia.
- Trial name
- STEADFAST (NCT02593773)
- Testing
- Interferon γ-1b
- Where
- 4 sites in United States
- Volunteers
- 86 people
- Sponsor
- Amgen
Friedreich's Ataxia
Long-Term Safety Extension Study of ACTIMMUNE® (Interferon γ-1b) in Children and Young Adults With Friedreich's Ataxia
The purpose of this long term extension study is to evaluate the long-term safety of ACTIMMUNE® (interferon-γ 1b) in participants with Friedreich's Ataxia (FA).
- Trial name
- STEADFAST (NCT02797080)
- Testing
- interferon γ-1b
- Where
- 4 sites in United States
- Volunteers
- 38 people
- Sponsor
- Amgen
Ataxia Telangiectasia, Genetic Syndrome
Extension Treatment Using EryDex System in Patients With AT Who Participated in the ATTeST-IEDAT-02-2015 Study
Primary Objective To monitor and evaluate the long-term safety and tolerability of EDS-EP in AT patients. Secondary Objective To evaluate the long-term effect of EDS-EP on health-related Quality of Life (QoL; EQ-5D-5L scale).
- Trial name
- OLE-IEDAT (NCT03563053)
- Testing
- EryDex System
- Where
- 17 sites in United States, Australia and 9 more
- Volunteers
- 104 people
- Sponsor
- Quince Therapeutics S.p.A.
Wolfram Syndrome, Diabetes Mellitus
A Clinical Trial of Dantrolene Sodium in Pediatric and Adult Patients With Wolfram Syndrome
Wolfram syndrome is a rare genetic disorder characterized by juvenile-onset diabetes mellitus, diabetes insipidus, optic nerve atrophy, hearing loss, and neurodegeneration.
- Conditions
- Wolfram Syndrome, Diabetes Mellitus, Optic Nerve Atrophy, Ataxia
- Trial name
- NCT02829268
- Testing
- dantrolene sodium
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- Washington University School of Medicine
Friedreich's Ataxia
Study to Assess the Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia
This study is meant to assess the effectiveness of idebenone on neurological outcome measures in patients with Friedreich's Ataxia over a 6 months period.
- Trial name
- IONIA (NCT00537680)
- Testing
- Idebenone
- Where
- 2 sites in United States
- Volunteers
- 70 people
- Sponsor
- Santhera Pharmaceuticals
Friedreich's Ataxia
Study to Assess the Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia Patients
This study is meant to assess the safety and tolerability of idebenone in patients with Friedreich's Ataxia over a 12 months period.
- Trial name
- IONIA-E (NCT00697073)
- Testing
- Idebenone
- Where
- 2 sites in United States
- Volunteers
- 68 people
- Sponsor
- Santhera Pharmaceuticals
Friedreich's Ataxia
A Study to Assess Efficacy, Long Term Safety and Tolerability of RT001 in Subjects With Friedreich's Ataxia
The purpose of this study is to assess the Efficacy, Long Term Safety and Tolerability of RT001 in subjects with Friedreich's Ataxia
- Trial name
- NCT04102501
- Testing
- RT001
- Where
- 6 sites in United States
- Volunteers
- 65 people
- Sponsor
- Biojiva LLC
Friedreich Ataxia
Interferon Gamma-1b in Friedreich Ataxia (FRDA)
Friedreich ataxia (FRDA) is a progressive neurodegenerative disease of children and adults for which there is presently no therapy.
- Trial name
- NCT01965327
- Testing
- Interferon Gamma-1b
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Children's Hospital of Philadelphia
Friedreich Ataxia
Efficacy of EGb761 in Patients Suffering From Friedreich Ataxia
The purpose of this protocol is to determine the efficacy of EGb 761 120 mg bid versus placebo in patients suffering from Friedreich Ataxia
- Trial name
- NCT00824512
- Testing
- EGb 761 120 mg
- Where
- France
- Volunteers
- 22 people
- Sponsor
- Ipsen
Friedreich's Ataxia
(+) Epicatechin to Treat Friedreich's Ataxia
This 24-week study will test the safety and effectiveness of synthetically produced (+) Epicatechin in treating patients who have Friedreich's Ataxia, a neurological disorder.
- Trial name
- NCT02660112
- Testing
- (+)-Epicatechin
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Ralitza Gavrilova