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A Phase 2 Study to Evaluate Safety and Effectiveness of Omaveloxolone (RTA 408) in Patients with Friedreich’s Ataxia

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What is this study about?

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.

1 Initial evaluation

Your eligibility for the study will be confirmed through genetic testing for Friedreich’s ataxia

A medical assessment will be performed, including kidney function tests and heart evaluation (echocardiogram)

Your initial mFARS score (a measure of ataxia severity) will be evaluated twice to ensure it falls between 20 and 80 points

2 Study medication – Part 1

You will receive RTA 408 capsules for oral use

During this period, you will need to maintain your current exercise routine

Your ability to perform exercise testing will be evaluated

The study will measure changes in your maximum exercise capacity

3 Study medication – Part 2

Treatment continues with Skyclarys 50 mg hard capsules taken by mouth

This phase lasts 48 weeks

Your mFARS score will be regularly evaluated to track changes in your condition

Exercise testing will continue to measure your physical capacity

4 Monitoring and assessments

Regular evaluations of your kidney function will be performed

Your heart function will be monitored through the study

The medical team will track any changes in your condition

Your impression of any changes in your condition will be recorded

5 Extension phase eligibility

After completing either 12 weeks of Part 1 or 24 weeks of Part 2

Your kidney function must remain at appropriate levels

Your heart function must maintain an ejection fraction of 40% or higher

You must have followed all study requirements without major deviations

Who Can Join the Study?

  • Must have confirmed Friedreich’s ataxia through genetic testing
  • Must be between 16 and 40 years old
  • Must have a mFARS score (a scale that measures ataxia severity) between 20 and 80 points, with two measurements being within 4.5 points of each other
  • Must have good kidney function with an estimated glomerular filtration rate (eGFR) of 60 mL/min/1.73 m² or higher
  • Must have a left ventricular ejection fraction (a measure of heart pumping ability) of 40% or higher
  • Must be able to perform maximal exercise testing
  • Must maintain the same exercise routine during the study and for 30 days before starting
  • Must be able to swallow capsules
  • Must use medically acceptable birth control methods
  • Must be willing to cooperate with all study requirements
  • Must provide written informed consent to participate in the study
  • For extension study participation: Must complete either 12 weeks of Part 1 or 24 weeks of Part 2 treatment without major protocol violations

Who Cannot Join the Study?

  • Prior participation in any clinical trial involving RTA 408 or similar investigational drugs
  • History of severe allergic reactions to medications
  • Significant heart problems or abnormal heart rhythm
  • Severe kidney or liver disease (organs not functioning properly)
  • Active cancer or history of cancer in the past 5 years
  • Pregnancy or breastfeeding
  • Use of prohibited medications that might interfere with the study drug
  • Unable to perform required exercise tests due to physical limitations
  • Mental conditions that could interfere with following study procedures
  • Drug or alcohol abuse within the past year
  • Any medical condition that, in the opinion of the study doctor, would make participation unsafe
  • Unable or unwilling to follow study procedures and visits
  • Blood test results showing significant abnormalities
  • Recent surgery (within 3 months before starting the study)
  • Current participation in another clinical trial

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

No sites found in this category

Other Sites

Site Name City Country Status
Fondazione I.R.C.C.S. Istituto Neurologico Besta Milan Italy
Mjycuhz Ubypyfufmu op Icqsixqoo Duyvhfet ou Nibvzfklwhar Dxfngqyyug os Nbijrumml Innsbruck Austria

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Austria Austria
Not recruiting
21.07.2016
Italy Italy
Not recruiting
21.07.2016

Trial locations

Investigated drugs:

RTA 408 is an investigational medication being studied for the treatment of Friedreich’s ataxia, a rare genetic disease that affects the nervous system. This medication is designed to improve muscle function and neurological symptoms in patients with this condition. It works by activating certain cellular pathways that may help protect nerve cells and improve energy production in cells. The medication aims to help patients maintain better physical function and potentially slow down the progression of the disease.

Friedreich’s ataxia – A genetic neurological disorder that affects the nervous system and causes movement problems. The condition typically begins in childhood or early teens, gradually affecting coordination, balance, and muscle strength. People with this condition experience progressive difficulty with walking, speaking clearly, and performing fine motor tasks with their hands. The disorder also commonly affects the heart, spine, and sometimes hearing and vision. This inherited condition occurs when both parents carry a mutation in the FXN gene, which leads to reduced production of an important protein called frataxin in the body’s cells.

Trial ID:
2024-517436-22-00
Protocol code:
408-C-1402
NCT ID:
NCT02255435
Trial Phase:
Therapeutic exploratory (Phase II)

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