A Study to Learn More About Friedrich's Ataxia in People Living in the Gulf Cooperation Council (GCC) Region of the World

NCT07887828 | Not Yet Recruiting

• 1 other identifier: SA-FRA-12346
• Study Type: observational
• Target: 40
• Locations: 0 countries
• Sites: N/A

Brief 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. 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:

• 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.

Conditions

Friedreich's Ataxia

Outcome Measures

Primary Outcomes (8)

• Change From Baseline in Modified Friedreich's Ataxia Rating Scale (mFARS) Score

  Baseline up to approximately 24 months

• Change From Baseline in Activities of Daily Living (ADL) Score

  Baseline up to approximately 24 months

• Clinical History: Age of Onset

  At Baseline

• Clinical History: Family History

  At Baseline

• Clinical History: Medication

  At Baseline

• Clinical History: Treatment

  At Baseline

• Diagnostic Information: Age at Diagnosis

  At Baseline

• Diagnostic Information: Proportion of Participants With Frataxin (FXN) Mutations

  At Baseline

Secondary Outcomes (9)

• Estimation of Frequency of Friedreich's Ataxia (FRDA)

  Up to approximately 24 months

• Baseline Demographics: Age

  At Baseline

• Baseline Demographics: Sex

  At Baseline

• Baseline Demographics: Geography

  At Baseline

• Baseline Demographics: Race/Ethnicity

  At Baseline

Study Arms (1)

Genetically Confirmed FRDA Participants

Participants with genetically confirmed FRDA at baseline visit will be enrolled and followed for approximately 24 months.

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

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.

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Biogen: lead

MeSH Terms

Conditions

Friedreich Ataxia

Condition Hierarchy (Ancestors)

Spinocerebellar Degenerations; Cerebellar Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Spinal Cord Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Mitochondrial Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Medical Director

  Biogen

  STUDY DIRECTOR

Central Study Contacts

US Biogen Clinical Trial Center

CONTACT

866-633-4636; clinicaltrials@biogen.com

Global Biogen Clinical Trial Center

CONTACT

clinicaltrials@biogen.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: OTHER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 14, 2026
• First Posted: October 21, 2026
• Study Start: October 15, 2026
• Primary Completion (Estimated): January 1, 2029
• Study Completion (Estimated): January 1, 2029
• Last Updated: October 21, 2026

Record last verified: 2026-10

Data Sharing

• IPD Sharing: Will share