Friedreich’s ataxia – European Clinical Trials Information Network https://clinicaltrials.eu Bridging Patients with Clinical Trials Wed, 17 Jun 2026 04:02:28 +0000 en-US hourly 1 https://wordpress.org/?v=7.0 https://clinicaltrials.eu/wp-content/uploads/2024/12/cropped-EU_icon-32x32.png Friedreich’s ataxia – European Clinical Trials Information Network https://clinicaltrials.eu 32 32 A Phase 3 Study Evaluating the Efficacy and Safety of Subcutaneous Nomlabofusp in Adults and Children with Friedreich’s Ataxia https://clinicaltrials.eu/trial/a-phase-3-study-evaluating-the-efficacy-and-safety-of-subcutaneous-nomlabofusp-in-adults-and-children-with-friedreich-s-ataxia/ Wed, 27 May 2026 04:05:21 +0000 https://clinicaltrials.eu/trial/a-phase-3-study-evaluating-the-efficacy-and-safety-of-subcutaneous-nomlabofusp-in-adults-and-children-with-friedreich-s-ataxia/ Friedreich’s Ataxia is a rare inherited condition that causes progressive problems with balance, coordination, and heart function because the body lacks enough of a protein called frataxin. The study evaluates the drug Nomlabofusp, given as a subcutaneous injection (an injection just under the skin), compared with a placebo that looks identical but contains no active medicine.

The purpose of the study is to find out whether Nomlabofusp can improve movement ability in children and adults with Friedreich’s Ataxia. Participants are randomly assigned to receive either the active medication or the placebo, and neither the participants nor the investigators know which treatment is given. Over roughly a year and a half, participants attend regular clinic visits where they undergo simple tests that measure walking distance, hand coordination, and overall disease severity using a scoring system called the modified Friedreich’s Ataxia Rating Scale, which is explained to participants as a way to track changes in their condition.

Throughout the study, safety is closely watched by recording any side effects, and blood samples are taken to check for immune reactions against the drug. Additional assessments include heart imaging to observe any changes in heart size and measurements of frataxin levels from skin and cheek samples. The study concludes after the final assessment at week 72, at which point the collected data are analyzed to determine the drug’s effectiveness and safety.

]]> A study testing omaveloxolone compared to placebo for children and adolescents aged 2 to 15 years with Friedreich’s ataxia https://clinicaltrials.eu/trial/a-study-testing-omaveloxolone-compared-to-placebo-for-children-and-adolescents-aged-2-to-15-years-with-friedreichs-ataxia/ Thu, 30 Apr 2026 13:08:16 +0000 https://clinicaltrials.eu/trial/a-study-testing-omaveloxolone-compared-to-placebo-for-children-and-adolescents-aged-2-to-15-years-with-friedreichs-ataxia/ This study is looking at a condition called Friedreich’s ataxia, which is an inherited disease that affects the nervous system and causes problems with movement and coordination. The study will test a medication called omaveloxolone, which is also known by its code name BIIB141, and will be given as hard capsules that are taken by mouth. Some participants will receive the actual medication while others will receive a placebo. The purpose of the study is to see how well omaveloxolone works in children and teenagers with Friedreich’s ataxia who are between 2 and 16 years old, and to check if the medication is safe when used over a long period of time.

The study is divided into two parts. In the first part, participants will be randomly assigned to receive either omaveloxolone or placebo for 52 weeks. During this time, doctors will measure changes in movement abilities and other symptoms of the disease. In the second part, all participants will have the opportunity to receive omaveloxolone in what is called an open-label extension, which means everyone will know they are getting the actual medication. This second part will continue for a longer time to see how safe the medication is when used over many months or years.

Throughout the study, doctors will regularly check participants for any side effects and will monitor their heart function, growth measurements including height and weight, and overall well-being. The study will also measure the amount of medication in the blood at different times to understand how the body processes it. Participants and their caregivers will be asked about changes in daily activities and how they feel the disease is affecting them. The study will help doctors understand whether omaveloxolone can help slow down or improve the symptoms of Friedreich’s ataxia in younger patients.

]]> Study to evaluate the effect of omaveloxolone on mitochondrial function in patients with Friedreich’s ataxia https://clinicaltrials.eu/trial/study-to-evaluate-the-effect-of-omaveloxolone-on-mitochondrial-function-in-patients-with-friedreich-s-ataxia/ Wed, 29 Apr 2026 15:09:43 +0000 https://clinicaltrials.eu/trial/study-to-evaluate-the-effect-of-omaveloxolone-on-mitochondrial-function-in-patients-with-friedreich-s-ataxia/ This study focuses on Friedreich’s ataxia, which is a rare genetic condition that affects the nervous system and can impact physical coordination. The research involves testing a medication called omaveloxolone. The purpose of the study is to evaluate how this drug affects mitochondrial function, which refers to how the small parts of cells that produce energy work within the body.

During the study, certain biomarkers, which are measurable indicators of a biological process, will be monitored to see how they respond to the treatment. Specifically, the study looks at substances like lipofuscin-like pigments and various proteins related to how cells manage energy and waste. These observations are compared to individuals who are not receiving the medication to identify reliable ways to track the effectiveness of the treatment over a period of 12 months.

]]> A Phase 2 Study to Evaluate Safety and Effectiveness of Omaveloxolone (RTA 408) in Patients with Friedreich’s Ataxia https://clinicaltrials.eu/trial/study-on-the-effects-of-omaveloxolone-for-patients-with-friedreichs-ataxia/ Wed, 29 Apr 2026 14:57:45 +0000 https://clinicaltrials.eu/trial/study-on-the-effects-of-omaveloxolone-for-patients-with-friedreichs-ataxia/ This study focuses on evaluating a medication called omaveloxolone (also known as RTA 408) in people with Friedreich’s ataxia, which is a rare genetic disease that affects coordination, muscle weakness, and movement. The purpose of the study is to determine if the medication can help improve physical function and movement abilities in patients with this condition.

The medication comes in the form of capsules taken by mouth and is available in different strengths (2.5 mg, 10 mg, and 50 mg). The study is divided into two parts, with different goals for each part. The first part looks at how well patients can perform during exercise testing, while the second part examines changes in movement and coordination over 48 weeks using a special rating scale called modified Friedreich’s ataxia rating scale.

Throughout the study, doctors will monitor patients’ health and safety while taking the medication. The study involves regular visits to evaluate how participants are responding to the treatment. The medication’s effects will be measured through various tests that assess physical function and overall well-being. Patients will need to maintain their usual exercise routines during the study period.

]]> Study on the Effects of Dimethyl Fumarate for Patients with Friedreich’s Ataxia https://clinicaltrials.eu/trial/study-on-the-effects-of-dimethyl-fumarate-for-patients-with-friedreichs-ataxia/ Wed, 29 Apr 2026 14:24:26 +0000 https://clinicaltrials.eu/trial/study-on-the-effects-of-dimethyl-fumarate-for-patients-with-friedreichs-ataxia/ This clinical trial is focused on studying Friedreich’s Ataxia, a rare genetic disease that affects the nervous system and movement. The trial will test the effectiveness, safety, and tolerability of a medication called Dimethyl Fumarate, also known by its code names BG00012 and FP 187. Participants in the study will receive either the medication or a placebo, which looks like the medication but does not contain the active ingredient.

The purpose of the study is to understand how Dimethyl Fumarate affects the production of certain proteins in the body that are important for people with Friedreich’s Ataxia. The study will last for several months, during which participants will take the medication or placebo orally in the form of gastro-resistant tablets. These tablets are designed to pass through the stomach and dissolve in the intestines, which helps to protect the active ingredient until it reaches the right part of the body.

Throughout the study, researchers will monitor participants to see how the medication affects their health and any symptoms of Friedreich’s Ataxia. This includes looking at changes in specific proteins and genes, as well as assessing physical abilities and overall well-being. The study aims to provide valuable information that could lead to better treatments for this condition in the future.

]]> Long-term safety and effectiveness study of vatiquinone (oral capsules) in patients with Friedreich ataxia who have previously received this treatment https://clinicaltrials.eu/trial/study-on-the-long-term-safety-and-effects-of-vatiquinone-for-patients-with-friedreich-ataxia/ Wed, 29 Apr 2026 14:22:43 +0000 https://clinicaltrials.eu/trial/study-on-the-long-term-safety-and-effects-of-vatiquinone-for-patients-with-friedreich-ataxia/ This study focuses on Friedreich ataxia, a rare genetic disorder that affects the nervous system and causes progressive damage to the spinal cord and nerves. The study will evaluate a medication called vatiquinone (also known as PTC743), which is being developed to treat conditions affecting cellular energy production.

The purpose of this research is to assess the long-term safety of vatiquinone in people with Friedreich ataxia who have previously received this medication in other studies. The medication comes in the form of capsules that are taken by mouth. Participants will receive vatiquinone for up to 36 months, with a maximum daily dose of 200 mg.

During the study, participants will be monitored for any health changes and will undergo various medical assessments. These assessments will include checking their ability to maintain balance while standing, movement of arms and legs, and speech function. The study will track how these abilities may change over time while taking the medication.

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