FA Clinical Outcome Measures

Status Recruiting

Clinical Trial Summary

This multicenter natural history study aims to expand the network of clinical research centers in FA, and to provide a framework for facilitating therapeutic interventions. In addition, this study will lead to the development of valid yet sensitive clinical measures crucial to outcome assessment of patients with Friedreich's Ataxia. This study will support genetic modifier studies, biomarker studies, and frataxin protein level assessments by building a sample repository.

Clinical Trial Description

Friedreich's ataxia (FA) is a rare autosomal recessive degenerative disorder characterized by ataxia, dysarthria, sensory loss, diabetes and cardiomyopathy. The discovery of the abnormal gene in FA and its product (frataxin) has provided insight into possible pathophysiological mechanisms and novel approaches to treatments in this disease. While such methods for assessing disease progression may be useful, evaluation in clinical trials will require specific clinical outcome measures. This is a multicenter natural history study which aims to expand the network of clinical research centers specializing in Friedreich's Ataxia and to advance clinical care, research and therapeutic approaches in FA through the development and validation of clinical outcome measures. Study sites aim to collect quantitative serial clinical data on patients with FA and expand the existing research network. In addition, the study will support various genetic modifier studies, biomarker studies, and frataxin protein level assessments in patients with FA, in carriers, and in controls. This study will recruit up to 2000 patients with Friedreich ataxia worldwide, to be assessed annually for up to 15 years. All individuals with a genetic or clinical diagnosis of FA can participate. Study participation involves yearly assessments of a core set of clinical measures and quality of life assessment measures in addition to optional collection of a cheek swab and/or blood sample. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT03090789
Study type	Observational [Patient Registry]
Source	Friedreich's Ataxia Research Alliance
Contact	Cait Monette
Phone	651-329-1892
Email	cait.monette@curefa.org
Status	Recruiting
Phase
Start date	January 1, 2001
Completion date	January 1, 2030

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See also
Status	Clinical Trial	Phase
Completed	`NCT05573698` - Study to Evaluate Multiple Ascending Dose and Multi-Dose of DT-216 in Adult Patients With Friedreich Ataxia	Phase 1
Completed	`NCT00229632` - Idebenone to Treat Friedreich's Ataxia	Phase 2
Completed	`NCT05579691` - A Double-Blind, Placebo-Controlled, Dose Exploration Study of CTI-1601 in Adult Subjects With Friedreich's Ataxia	Phase 2
Completed	`NCT04273165` - Safety and Efficacy of Etravirine in Friedreich Ataxia Patients	Phase 2
Not yet recruiting	`NCT05874388` - Characterisation of the Cognitive Profile of Patients Suffering From Friedreich's Ataxia	N/A
Completed	`NCT02594917` - Genetic and Environmental Determinants That Control Metabolism in Pulmonary Hypertension
Completed	`NCT01716221` - An Objective Double-blind Evaluation of Bupropion and Citalopram in an Individual With Friedreich Ataxia	Phase 4
Active, not recruiting	`NCT05485987` - A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia	Phase 2
Completed	`NCT03214588` - Efficacy, Tolerability, and Pharmacokinetics of Multiple Doses of Oral TAK-831 in Adults With Friedreich Ataxia	Phase 2
Completed	`NCT03418740` - Neurology Measures in FA Children
Not yet recruiting	`NCT06054893` - A Study of Omaveloxolone in Children With Friedreich's Ataxia	Phase 1
Recruiting	`NCT04921930` - Evaluation of the Effect of Artesunate in Friedreich Ataxia (FA)	Phase 1/Phase 2
Active, not recruiting	`NCT03933163` - Micronised Resveratrol as a Treatment for Friedreich Ataxia	Phase 2
Completed	`NCT02705547` - Rosuvastatin (Crestor) in Friedreich Ataxia	Early Phase 1
Completed	`NCT02035020` - A Phase IIa Trial to Test Safety and Efficacy Interferon Gamma Treatment in Elevating Frataxin Levels in FRDA Patients	Phase 2
Completed	`NCT00015808` - Safety Study of Idebenone to Treat Friedreich's Ataxia	Phase 1
Completed	`NCT04817111` - NAD+ Precursor Supplementation in Friedreich's Ataxia	Phase 2
Active, not recruiting	`NCT05168774` - FRDA Investigator Initiated Study (IIS) With Elamipretide	Phase 1/Phase 2
Completed	`NCT03917225` - A Clinical Study to Evaluate the Effect of MIN-102 on the Progression of Friedreich's Ataxia in Male and Female Patients	Phase 2
Completed	`NCT00224640` - Iron-Chelating Therapy and Friedreich Ataxia	Phase 1/Phase 2

Clinical trials are conducted in a series of steps, called phases - each phase is designed to answer a separate research question.

Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.

FA Clinical Outcome Measures — FA-COMS

Clinical Trial Summary

Clinical Trial Description

Study Design

Related Conditions & MeSH terms