An observational study in Friedreich's Ataxia, sponsored by Biogen. Not yet recruiting. Per ClinicalTrials.gov, last updated 2026-10-21.
Sponsored by Biogen · Observational
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).
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:
Researchers will also learn more about:
This study will be done as follows:
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.
111 studies on the registry are indexed under Friedreich Ataxia; 28 are open to participants now.
This study's planned enrollment of 40 is below the median of 78 across 32 observational studies indexed under Friedreich Ataxia.
Browse Friedreich Ataxia studies →Biogen is the lead sponsor of 511 studies on the registry; 24 are open to participants now.
Of its 100 completed or terminated interventional studies of FDA-regulated products, 49 (49%) have results posted.
Counted across the registry records on this site, refreshed daily.
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.
Key Inclusion Criteria:
Key Exclusion Criteria:
NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
Participants with genetically confirmed FRDA at baseline visit will be enrolled and followed for approximately 24 months.
Change From Baseline in Modified Friedreich's Ataxia Rating Scale (mFARS) Score
Time frame: Baseline up to approximately 24 months
Change From Baseline in Activities of Daily Living (ADL) Score
Time frame: Baseline up to approximately 24 months
Clinical History: Age of Onset
Time frame: At Baseline
Clinical History: Family History
Time frame: At Baseline
Clinical History: Medication
Time frame: At Baseline
Clinical History: Treatment
Time frame: At Baseline
Diagnostic Information: Age at Diagnosis
Time frame: At Baseline
Diagnostic Information: Proportion of Participants With Frataxin (FXN) Mutations
Time frame: At Baseline
Estimation of Frequency of Friedreich's Ataxia (FRDA)
Time frame: Up to approximately 24 months
Baseline Demographics: Age
Time frame: At Baseline
Baseline Demographics: Sex
Time frame: At Baseline
Baseline Demographics: Geography
Time frame: At Baseline
Baseline Demographics: Race/Ethnicity
Time frame: At Baseline
Baseline Demographics: Height
Time frame: At Baseline
Baseline Demographics: Weight
Time frame: At Baseline
Number of Participants With Associated Comorbidities
Time frame: Up to approximately 24 months
Number of Participants With Clinical History of FRDA
Time frame: Up to approximately 24 months
No study locations are listed for this record.
Plan to share: Yes — In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on htps://www.biogentrialtransparency.com/
Data sharing details ↗ · vivli.org
No publications or documents are linked to this record.
Start is estimated for Oct 2026; primary completion for Jan 2029, 26 months later.
How these figures are worked out, and which studies are left out of them.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
This study is not yet recruiting, as verified in Oct 2026. You cannot join it, but the record below documents what was studied.
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