Clinical trials: FRDA
Friedreich Ataxia
A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
The primary objective of the study is to evaluate the efficacy (using the modified Friedreich Ataxia Rating Scale [mFARS]) and safety of vatiquinone in participants with Friedreich ataxia (FA).
- Trial name
- MOVE-FA (NCT04577352)
- Testing
- Vatiquinone
- Where
- 14 sites in United States, Australia and 7 more
- Volunteers
- 146 people
- Sponsor
- PTC Therapeutics
Rare Disorders, Undiagnosed Disorders
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
The Coordination of Rare Diseases at Sanford (CoRDS) is an international patient registry that serves as a centralized resource for rare disease research.
- Conditions
- Rare Disorders, Undiagnosed Disorders, Disorders of Unknown Prevalence, Cornelia De Lange Syndrome
- Trial name
- CoRDS (NCT01793168)
- Where
- United States
- Volunteers
- 20,000 people
- Sponsor
- Sanford Health
Friedreich's Ataxia
Long-Term Efficacy Study of Vatiquinone for the Treatment of Friedreich's Ataxia (FA)
The purpose of this study is to confirm the treatment effects of vatiquinone on the key measures of FA disease progression.
- Trial name
- PROVE-FA (NCT07681713)
- Testing
- Vatiquinone
- Where
- 9 sites in United States, Belgium and 4 more
- Volunteers
- 120 people
- Sponsor
- PTC Therapeutics
Friedreich's Ataxia
A First in Human Study of RT001 in Patients With Friedreich's Ataxia
The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics of RT001 in patients with Friedreich's ataxia.
- Trial name
- NCT02445794
- Testing
- Low dose cohort, High dose cohort
- Where
- 2 sites in United States
- Volunteers
- 19 people
- Sponsor
- Biojiva LLC
Friedreich Ataxia
Single Ascending Dose Study of CTI-1601 Versus Placebo in Subjects With Friedreich's Ataxia
To evaluate the safety and tolerability of single ascending doses of CTI-1601 in participants with Friedreich's ataxia
- Trial name
- NCT04176991
- Testing
- CTI-1601
- Where
- United States
- Volunteers
- 28 people
- Sponsor
- Larimar Therapeutics, Inc.
Friedreich Ataxia
Efficacy of Stabilometric Platform to Improve Standing Balance in Patients With Friedreich's Ataxia
The primary objective is to evaluate the potential effectiveness of an individualized intensive rehabilitation intervention using the "Prokin 252" stabilometric platform in the treatment of adolescent and adult patients with…
- Trial name
- NCT06692296
- Testing
- Balance training over stabilometric platform, Balance training by means of conventional rehabilitation
- Where
- Italy
- Volunteers
- 24 people
- Sponsor
- IRCCS Eugenio Medea
Friedreich Ataxia, Cardiomyopathies
Phase IA and IB Study of AAVrh.10hFXN Gene Therapy for the Cardiomyopathy of Friedreich's Ataxia
The purpose of this study is to test the safety and preliminary efficacy of AAVrh.10hFXN to treat the cardiomyopathy associated with Friedreich's ataxia (FA).
- Conditions
- Friedreich Ataxia, Cardiomyopathies, Cardiac Hypertrophy, Myocardial Fibrosis
- Trial name
- NCT05302271
- Testing
- AAVrh.10hFXN, serotype rh.10 adeno-associated virus (AAV) gene transfer vector expressing the cDNA coding for human FXN, Prednisone
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Weill Medical College of Cornell University
Friedreich Ataxia
Evaluation of the Effect of Artesunate in Friedreich Ataxia (FA)
This dose-escalation study is aimed at investigating a novel application for artesunate in the treatment of Friedreich ataxia. It will evaluate this novel application of oral artesunate using a surrogate biological marker as…
- Trial name
- ARTEMIS (NCT04921930)
- Testing
- Artesunate Oral Product
- Where
- France
- Volunteers
- 20 people
- Sponsor
- Institut National de la Santé Et de la Recherche Médicale, France
Friedreich Ataxia, Cardiomyopathy
Clinical Course Of Disease In Participants With FA-CM
Characteristics and clinical course of disease In participants with cardiomyopathy associated with Friedreich Ataxia (CLARITY-FA)
- Trial name
- NCT06865482
- Where
- 19 sites in United States, Brazil and 6 more
- Volunteers
- 65 people
- Sponsor
- Lexeo Therapeutics
Friedreich Ataxia
Study to Evaluate Multiple Ascending Dose and Multi-Dose of DT-216 in Adult Patients With Friedreich Ataxia
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetic, and pharmacodynamic effects of multiple doses of intravenous DT-216 in adult patients with Friedrich Ataxia.
- Trial name
- NCT05573698
- Testing
- DT-216
- Where
- United States
- Volunteers
- 32 people
- Sponsor
- Design Therapeutics, Inc.
Spinocerebellar Ataxia - All Sub-types, Friedreich's Ataxia
Preliminary Study of the Scale To Assess Ataxia and Neurologic Dysfunction (STAND)
The objectives of this study are: To validate the inter-rater and intra-rater reliability of a new scale for the assessment of ataxia and neurologic dysfunction (STAND) To assess common constructs and correlation between STAND…
- Trial name
- STAND (NCT02179333)
- Testing
- Ataxia rating scale
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- University of South Florida
Friedreich Ataxia
Neurology Measures in FA Children
The purpose of this study is to identify ways to follow progression of Friedreich's Ataxia (FA) and be able to measure changes over time in children with FA.
- Trial name
- NCT03418740
- Where
- 3 sites in United States
- Volunteers
- 108 people
- Sponsor
- Children's Hospital of Philadelphia
Friedreich Ataxia
Methylprednisolone Treatment of Friedreich Ataxia
This study will explore whether methylprednisolone treatment is safe, well-tolerated, and beneficial in patients that are diagnosed with Friedreich Ataxia (FRDA).
- Trial name
- NCT02424435
- Testing
- Methylprednisolone
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- Children's Hospital of Philadelphia
Friedreich Ataxia
A Clinical Study to Evaluate the Effect of MIN-102 on the Progression of Friedreich's Ataxia in Male and Female Patients
Randomized, double-blind, placebo-controlled study on the effects of MIN-102 on Biochemical, Imaging, neurophysiological, and clinical markers in patients with Friedreich's Ataxia
- Trial name
- FRAMES (NCT03917225)
- Testing
- MIN-102
- Where
- 5 sites in Belgium, France and 2 more
- Volunteers
- 39 people
- Sponsor
- Minoryx Therapeutics, S.L.
Friedreich Ataxia
Safety and Efficacy of Etravirine in Friedreich Ataxia Patients
A drug repositioning effort provided evidence supporting the possible use of Etravirine, a drug approved for the treatment of HIV infections in patients starting from 2 years of age, as a treatment for FA.
- Trial name
- FAEST1 (NCT04273165)
- Testing
- Etravirine Tablets
- Where
- Italy
- Volunteers
- 30 people
- Sponsor
- IRCCS Eugenio Medea
Friedreich Ataxia
Characterisation of the Cognitive Profile of Patients Suffering From Friedreich's Ataxia
Friedreich's Ataxia (FA) Friedreich's Ataxia is a neurodegenerative disease caused by a homozygous expansion of the GAA triplet repeats of the frataxin gene (FXN). FA usually begins in childhood or adolescence.
- Trial name
- CPCAF (NCT05874388)
- Where
- France
- Volunteers
- 70 people, healthy volunteers welcome
- Sponsor
- Institut National de la Santé Et de la Recherche Médicale, France
Friedreich Ataxia
Friedreich Ataxia Nerve Ultrasund
"Friedreich ataxia is the most common inherited autosomal recessive ataxia. It is caused by a GAA repeat expansion in the frataxin gene on chromosome 9q21.11.
- Trial name
- FAUST (NCT07508631)
- Testing
- Nerve ultrasound - routine exam
- Where
- France
- Volunteers
- 14 people
- Sponsor
- Centre Hospitalier Universitaire de Nice
Friedreich's Ataxia, Steroidogenesis
FRIEDREICH ATAXIA- STEROIDOGENESIS
Friedreich's ataxia (FA) is a rare autosomal recessive disorder caused by GAA repeat expansion in the FXN gene, leading to impaired iron-sulfur (Fe-S) cluster biosynthesis and mitochondrial dysfunction.
- Trial name
- FRIEDSTERO (NCT07123142)
- Where
- Turkey (Türkiye)
- Volunteers
- 25 people, healthy volunteers welcome
- Sponsor
- Istanbul University
Friedreich Ataxia
NAD+ Precursor Supplementation in Friedreich's Ataxia
The primary objective is to test the safety and tolerability of short-term therapy with a nicotinamide adenine dinucleotide (NAD+) precursor (MIB-626) in adults with Friedreich's Ataxia (FA) without overt heart failure and with a…
- Trial name
- NCT04817111
- Testing
- MIB-626
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- Metro International Biotech, LLC
Friedreich Ataxia
A Double-Blind, Placebo-Controlled, Dose Exploration Study of CTI-1601 in Adult Subjects With Friedreich's Ataxia
To evaluate the safety and tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of subcutaneous (SC) administration of CTI-1601 over 28 days in subjects with Friedreich's ataxia (FRDA).
- Trial name
- NCT05579691
- Testing
- CTI-1601
- Where
- United States
- Volunteers
- 28 people
- Sponsor
- Larimar Therapeutics, Inc.