Friedreich's AtaxiaNCT07887828
A Study to Learn More About Friedrich's Ataxia in People Living in the Gulf Cooperation Council (GCC) Region of the World
An observational study of Friedreich's Ataxia, run by Biogen.
Trial summary
In this study, researchers will learn more about a disease called Friedrich'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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Friedreich's Ataxia
- How many people
- 40 participants
- Where
- No sites listed yet
- Study dates
- 15 Oct 2026to 1 Jan 2029
- Phase
- Observational
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Participants who consent to participate in the study and meet all the inclusion criteria none of the exclusion criteria at screening or enrolment visit will be considered enrolled in this study.
Who may be able to join
- Informed consent is obtained from the participants and/or parents/legal representative as applicable.
- Individuals clinically and genetically diagnosed with FRDA through Frataxin (FXN) Guanine-adenine-adenine triplet-repeat expansions (GAA) repeat mutation or point mutation genetic testing.
- For participants already on treatment for FRDA, baseline data (including mFARS score) prior to treatment should be available retrospectively.
Who cannot join
- Presence of acute or ongoing medical or other conditions that would interfere with the conduct and assessments of the study.
- On FRDA treatment and has no baseline assessment.
- NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
Global Biogen Clinical Trial Center contact
About this study
Official title: Friedreich's Ataxia Observational Study
In this study, researchers will learn more about a disease called Friedrich'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.
In certain parts of the world, people with FA may not know about the disease or have access to genetic testing to get a diagnosis. To help more people get medical care for FA across the world, researchers need to learn more about any cases that do happen. Participants for this study will come from West Asia, in countries covered by the "Gulf Cooperation Council", also known as the GCC. This will include study sites in Saudi Arabia, Kuwait, Qatar, Oman, Bahrain, and the United Arab Emirates (UAE).
Read more
The main goal of this study is to learn more about the natural history and participant experience of FA in people living in the GCC region of the world. This will include learning about their experiences, symptoms, and how the disease affects their daily lives over time. To do this, researchers will collect information from participants' medical records and regular healthcare visits. Researchers hope that what they learn from this study will help them understand more about FA in this part of the world so that they can better understand people need for their FA.
The main question researchers want to answer in this study is:
- What are the experiences of people with FA, and how does the disease affect people over time in the GCC region of the world?
Researchers will also learn more about:
- About how many cases of FA are happening in this part of the world?
- How different do FA cases look like in this part of the world?
- What information in the participants' medical records helps show the difference between FA and other kinds of ataxia?
This study will be done as follows:
- Participants will join the study after signing an informed consent form, also known as an ICF.
- During the study, health information from the participants' regular visits to their doctor will be collected about every 6 months.
- Information about participants' FA symptoms, medical history, genetic test results, and daily functioning will be collected.
- For participants already receiving treatment for FA, information from before they started treatment may also be collected from medical records.
- During regular visits, researchers will also collect information about participants' FA symptoms and their ability to do their daily living activities.
- Each participant will be in the study for up to about 2 years.
Read the detailed description
The primary objective of this study is to describe the natural history and participant experience of disease for participants with Friedreich's Ataxia (FRDA) in a real-world setting. The secondary objectives of this study are to estimate FRDA frequency in the GCC countries, and to further characterize FRDA participant population profile in the GCC countries by evaluating clinical features, heterogeneity of disease presentation and genetic profile and to collect data available to differentiate FRDA from other ataxia.
- Conditions
- Friedreich's Ataxia
- Medical subject headings
- Friedreich Ataxia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Genetically Confirmed FRDA Participants | Participants with genetically confirmed FRDA at baseline visit will be enrolled and followed for approximately 24 months. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline in Modified Friedreich's Ataxia Rating Scale (mFARS) Score
Measured: Baseline up to approximately 24 months
Change From Baseline in Activities of Daily Living (ADL) Score
Measured: Baseline up to approximately 24 months
Clinical History: Age of Onset
Measured: At Baseline
Clinical History: Family History
Measured: At Baseline
Clinical History: Medication
Measured: At Baseline
Clinical History: Treatment
Measured: At Baseline
Diagnostic Information: Age at Diagnosis
Measured: At Baseline
Diagnostic Information: Proportion of Participants With Frataxin (FXN) Mutations
Measured: At Baseline
Secondary outcomes (9)
Estimation of Frequency of Friedreich's Ataxia (FRDA)
Measured: Up to approximately 24 months
Baseline Demographics: Age
Measured: At Baseline
Baseline Demographics: Sex
Measured: At Baseline
Baseline Demographics: Geography
Measured: At Baseline
Baseline Demographics: Race/Ethnicity
Measured: At Baseline
Baseline Demographics: Height
Measured: At Baseline
Baseline Demographics: Weight
Measured: At Baseline
Number of Participants With Associated Comorbidities
Measured: Up to approximately 24 months
Number of Participants With Clinical History of FRDA
Measured: Up to approximately 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Oct 2026
- Study start15 Oct 2026 (estimated)
- Primary completion1 Jan 2029 (estimated)
- Study completion1 Jan 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Friedreich's AtaxiaStudies of the same condition, open and under way.
- More trials run by BiogenThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Other
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Biogen
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Biogen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07887828
- Sponsor's protocol ID
- SA-FRA-12346
- Status last verified
- Oct 2026
- First submitted
- 14 Oct 2026
- Last updated
- 21 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on htps://www.biogentrialtransparency.com/