Friedreich's Ataxia clinical trials at University of California Health
9 in progress, 3 open to eligible people
CTI-1601 in Subjects With Friedreich's Ataxia
open to eligible people ages 2-60
An open label study designed to evaluate the safety, PK, PD, and clinical effects of long-term daily administration of CTI-1601 enrolling adolescent and adult patients with FRDA who have participated in a prior clinical study of CTI-1601 as well as children (age 2 years and older), adolescents and adults with FRDA who have not participated in a prior clinical study of CTI-1601.
at UCLA
Clinical Course Of Disease In Participants With FA-CM
open to eligible people ages 6 years and up
Characteristics and clinical course of disease In participants with cardiomyopathy associated with Friedreich Ataxia (CLARITY-FA)
at UCSD
Friedreich Ataxia Global Clinical Consortium UNIFIED Natural History Study
open to all eligible people
This project is a global, multicenter, prospective, longitudinal, observational natural history study that can be used to understand the disease progression and support the development of safe and effective drugs and biological products for Friedreich ataxia.
at UCLA
SGT-212 Gene Therapy in Friedreich's Ataxia
Sorry, not currently recruiting here
This is a phase 1b, first in-human, open-label, dose-finding study investigating the safety and tolerability of SGT-212 in participants with Friedreich's ataxia (FA). It will be delivered via dual intradentate nucleus (IDN) and intravenous (IV) administration to participants with FA. All participants will receive SGT-212 and will be enrolled in the study for approximately 5 years.
at UCLA
Vatiquinone in Participants With Friedreich Ataxia
Sorry, in progress, not accepting new patients
The primary objective of this study is to assess the long-term safety of vatiquinone in participants with Friedreich ataxia (FA) previously exposed to vatiquinone.
at UCLA
Effects and Long-Term Safety of Omaveloxolone (BIIB141) in Children and Teens With Friedreich's Ataxia
Sorry, not currently recruiting here
In this study, researchers will learn more about omaveloxolone, also known as BIIB141 or SKYCLARYS®. Omaveloxolone is already approved for people with Friedreich's Ataxia (FA) who are 16 years of age or older. However, it is not yet available for younger teens and children. The main goal of this study is to learn how omaveloxolone affects symptoms of FA and its safety in younger participants between the ages of 2 and 15 years old. The main questions researchers want to answer in this study are: - How does omaveloxolone affect the participants' FA symptoms? - How many participants have adverse events during the study? - Are there any changes in the participants' overall health or heart health? Adverse events are health problems that may or may not be caused by the study drug. Researchers will use the modified Friedreich's Ataxia Rating Scale (mFARS) to test nerve function. The mFARS tests movement ability, balance, coordination, speech, and arm and leg functions. They will also use a number of questionnaires to learn more about participants' quality of life, muscle strength, and ability to perform daily tasks. Researchers will also note any changes as participants go through puberty. Finally, researchers will learn more about how the body processes omaveloxolone in children and teenagers. This study will be done in 2 parts as follows: - Participants will be screened for up to 4 weeks to check if they can join the study. - In Part 1, participants will be randomly assigned to take either omaveloxolone or a placebo by mouth once a day for about 1 year. A placebo looks like the study drug but contains no real medicine. - Part 1 will be double blind. This means that the participants, study doctor, and site staff will not know if the participants are receiving omaveloxolone or a placebo. - Including screening, participants will have up to 9 clinic visits and 1 phone call during Part 1. If a participant does not join Part 2, they will have another safety follow-up phone call a month after their last dose of omaveloxolone. - Participants who complete Part 1 will move onto Part 2 where everyone will receive omaveloxolone for about 2 years. - During Part 2, participants will have up to 8 clinic visits and 1 phone call. Participants will also have a follow-up phone call about a month after they stop taking omaveloxolone. - In total, participants will have up to 17 clinic visits and 3 phone calls. Each participant will be in the study for up to 3 years.
at UCLA
Gene Therapy for Cardiomyopathy Associated With Friedreich's Ataxia
Sorry, in progress, not accepting new patients
This is a Phase 1/2, open-label, dose-ascending, multicenter study of the safety and efficacy of LX2006 for participants who have Friedreich's Ataxia with evidence of cardiomyopathy. The study will evaluate up to three doses of single administration of LX2006 (AAVrh.10hFXN), an adeno-associated virus (AAV) gene therapy designed to intravenously deliver the human frataxin (hFXN) gene to cardiac cells over a 52-week period. Long-term safety and efficacy will be evaluated for an additional 4-years for a total of 5-years post LX2006 treatment.
at UCLA
Long-Term Safety of BIIB141 (Omaveloxolone) in Participants With Friedreich's Ataxia Who Are Prescribed it by Their Own Doctors
Sorry, not currently recruiting here
In this study, researchers will learn more about the safety of BIIB141, also known as omaveloxolone or SKYCLARYS®. This is a drug available for doctors to prescribe for people with Friedreich's Ataxia, also known as FA. This is known as an "observational" study, which collects health information about study participants without changing their medical care. Participants for this study will be found using a group called the Friedreich's Ataxia Global Clinical Consortium (FA GCC) UNIFIED Natural History Study (UNIFAI). The FA-GCC is a group of study research centers that helps provide clinical care for FA patients and also helps researchers learn more about how FA affects patients over a long time. The main objective of this study is to collect safety information in participants with FA from UNIFAI. Some of the participants in this study will be prescribed BIIB141 for the first time by their own doctors. Some of the participants will have started taking BIIB141 after joining UNIFAI, but less than 12 months before joining this study. The main questions researchers want to answer in this study are: - How many participants had serious adverse events (SAEs)? An adverse event is considered serious when it results in death, is life-threatening, causes lasting problems, or requires hospital care. - How many participants had adverse events (AEs) related to heart failure or liver damage caused by the drug? Researchers will also learn more about : • Why and when participants stopped treatment, left the study, or took more of the drug than was prescribed This study will be done as follows: - Participants will be screened to check if they can join the study. - After joining the study, the participants who had never started BIIB141 treatment before must start it within 6 months. Otherwise, all participants will take BIIB141 throughout this study as prescribed by their own doctor. - During the study, each participant's doctor will decide how often the participant visits the study research center to check on their health. This will be based on the doctor's own clinical judgment and what is recommended by the drug's label. - Data from the participants' regular visits to their doctor will be collected at 1 month, 2 months, 3 months, 6 months, 12 months, 24 months, 36 months, 48 months, and 60 months. - Each participant will be in the study for up to 5 years.
at UCLA
FA Clinical Outcome Measures
Sorry, in progress, not accepting new patients
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. This natural history study is no longer recruiting under this protocol NCT03090789 but remains actively recruiting under the harmonized study (UNIFAI) NCT06016946.
at UCLA
Our lead scientists for Friedreich's Ataxia research studies include Susan Perlman, MD.
Last updated: