Not recruitingRare disease

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

Verified siteRegistered drugNo placebo

Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

What is this trial about?

A plain-language summary of the goals, design and what participants do

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.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 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
Step 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
Step 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
Step 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
Step 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 trial?

12 criteria

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 trial?

15 criteria

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

Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

Tell us about your condition – we search every trial in Europe and connect you with the right site.

We usually reply within a few days

Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Fondazione I.R.C.C.S. Istituto Neurologico Besta
Verified
Milan, Italy

Trial locations

Where you can join this trial

Countries are shaded by recruitment status. Click a recruiting country to ask about joining there.

Not recruiting

Not finding your country?

Not sure what to do next?

Joining a clinical trial can seem overwhelming. We guide you step by step, so you know exactly what to expect and how we support you through the process.

See the full process and FAQ

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.

Omaveloxolone

What is already known about the treatment

RTA 408 - An investigational drug being studied for the treatment of Friedreich's ataxia, a rare genetic neurodegenerative disorder. The medication is undergoing Phase 2 clinical trials to evaluate its safety, efficacy, and pharmacodynamic properties in patients with this condition. The drug's effectiveness is being measured through changes in peak work during maximal exercise testing and modifications in the Friedreich's ataxia rating scale (mFARS) over a 48-week period. While specific administration details are not provided in the source data, this experimental treatment is being evaluated for its potential to address the underlying mechanisms of Friedreich's ataxia.

Investigated diseases

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.