Ongoing Clinical Trials for Friedreich’s Ataxia
Currently, there are 3 ongoing clinical trials exploring treatments for Friedreich’s ataxia, a rare genetic disorder affecting the nervous system and causing progressive problems with movement and coordination. These trials are testing three different investigational medications: vatiquinone, omaveloxolone, and dimethyl fumarate, with studies taking place across several European countries including Italy, Germany, France, Spain, and Austria.
Clinical trial locations
- Austria
- France
- Germany
- Italy
- Long-term safety and effectiveness study of vatiquinone (oral capsules) in patients with Friedreich ataxia who have previously received this treatment
- A Phase 2 Study to Evaluate Safety and Effectiveness of Omaveloxolone (RTA 408) in Patients with Friedreich’s Ataxia
- Study on the Effects of Dimethyl Fumarate for Patients with Friedreich’s Ataxia
- Spain
Long-term safety and effectiveness study of vatiquinone (oral capsules) in patients with Friedreich ataxia who have previously received this treatment
This study is designed for people who have already taken vatiquinone in a previous clinical trial. The main purpose is to monitor the long-term safety of this medication, which is being developed to help protect nerve cells and support cellular energy production.
Who can participate: This trial is open to adults and adolescents who previously completed a clinical study with vatiquinone. Participants must be willing to use effective birth control methods throughout the study and for 30 days after the last dose. Both men and women can join, and all participants must provide informed consent.
Who cannot participate: You cannot join if you have any medical condition that could make participation unsafe, have a history of significant allergies to vatiquinone or similar medications, are pregnant or breastfeeding, have participated in another clinical trial within the past 30 days, or have unstable medical conditions requiring immediate treatment. People with substance abuse history within the past year or mental conditions affecting their ability to provide consent are also excluded.
Study focus and goals: Researchers will evaluate the long-term safety of vatiquinone by monitoring participants for any health changes over up to 36 months. The study will assess various aspects of physical function, including balance while standing, movement of arms and legs, and speech function. These assessments will help determine how abilities may change over time while taking the medication.
Investigational drug: Vatiquinone is an oral medication that supports mitochondrial function and may help reduce oxidative stress, which plays a role in disease progression. Participants will receive up to 200 mg daily in capsule form.
A Phase 2 Study to Evaluate Safety and Effectiveness of Omaveloxolone (RTA 408) in Patients with Friedreich’s Ataxia
This study examines whether omaveloxolone can improve physical function and movement abilities in people with Friedreich’s ataxia. The trial is divided into two parts, each with different evaluation goals.
Who can participate: Eligible participants must be between 16 and 40 years old with genetically confirmed Friedreich’s ataxia. You must have a specific severity score on a movement assessment scale (mFARS) between 20 and 80 points. Good kidney function and heart pumping ability are required, and you must be able to perform exercise testing and swallow capsules. Participants must maintain their current exercise routine and use medically acceptable birth control methods.
Who cannot participate: You cannot join if you have previously participated in trials involving omaveloxolone, have severe heart problems or abnormal heart rhythms, significant kidney or liver disease, active cancer or cancer history in the past 5 years, are pregnant or breastfeeding, or have drug or alcohol abuse within the past year. Recent surgery within 3 months or inability to perform required exercise tests also excludes participation.
Study focus and goals: The first part of the study evaluates how well patients perform during exercise testing, measuring maximum exercise capacity. The second part, lasting 48 weeks, examines changes in movement and coordination using the modified Friedreich’s ataxia rating scale. Throughout both phases, doctors will monitor kidney and heart function, track physical capacity, and assess any changes in the patient’s condition.
Investigational drug: Omaveloxolone (RTA 408) is an oral medication available in different strengths (2.5 mg, 10 mg, and 50 mg). It works by activating cellular pathways that may help protect nerve cells and improve energy production in cells, potentially helping patients maintain better physical function.
Study on the Effects of Dimethyl Fumarate for Patients with Friedreich’s Ataxia
This clinical trial tests whether dimethyl fumarate can increase the production of frataxin, a protein that is typically low in people with Friedreich’s ataxia. The study is a double-blind trial, meaning neither participants nor researchers know who receives the actual medication versus a placebo.
Who can participate: You must be 12 years or older, weigh at least 30 kg (about 66 pounds), and have a molecular diagnosis of Friedreich’s ataxia with a specific genetic change (homozygous GAA expansion). You must be able to read and sign the informed consent form.
Who cannot participate: People without Friedreich’s ataxia, those outside the specified age range, or those unable to follow study procedures cannot participate. Patients with other medical conditions that might interfere with the study, those taking medications that could affect results, pregnant or breastfeeding women, and those who recently participated in another clinical trial are excluded.
Study focus and goals: The core phase lasts 12 weeks, during which researchers monitor how the medication affects frataxin protein levels and gene expression. Regular assessments include physical exams, blood tests, and evaluations to measure the medication’s impact on the condition. The study aims to understand the effectiveness, safety, and tolerability of dimethyl fumarate in treating Friedreich’s ataxia.
Investigational drug: Dimethyl fumarate is administered orally as gastro-resistant tablets designed to pass through the stomach and dissolve in the intestines. It works as an immunomodulatory and neuroprotective agent by activating a pathway that helps protect cells from stress and damage.
Summary
The three ongoing clinical trials for Friedreich’s ataxia represent different approaches to treating this rare genetic disorder. Italy stands out as the location where all three trials are being conducted, making it a significant center for this research. Two trials focus on medications that work at the cellular level to protect nerve cells and support energy production (vatiquinone and omaveloxolone), while one trial examines whether increasing frataxin protein levels can help manage the condition (dimethyl fumarate).
The trials vary in their design and requirements. One is specifically for patients who have already received treatment with vatiquinone, while the others are open to newly enrolled participants. The age ranges also differ, with one trial accepting participants as young as 12 years old, while another restricts enrollment to those aged 16 to 40. All three studies emphasize careful monitoring of participants’ safety and physical function throughout the treatment period.
These trials offer hope for developing better treatments for Friedreich’s ataxia, a condition that currently has limited therapeutic options. Each study contributes valuable information about different potential treatment approaches, from long-term safety monitoring to evaluating new therapeutic mechanisms.
