An observational study in Friedreich Ataxia, sponsored by German Center for Neurodegenerative Diseases (DZNE). Recruiting at 6 sites in 3 countries. Open to participants aged 12 Years and older. Per ClinicalTrials.gov, last updated 2026-04-01.
Sponsored by German Center for Neurodegenerative Diseases (DZNE) · Observational
Help build ClinicalTrials.gg — short surveys, remote interviews or testing, in your language. Take part →
Among 17 studies in Friedreich Ataxia with a site in Germany — 6 open to participants now, 9 under way, 2 of them phase 3. Friedreich Ataxia · Germany.
Among the 17 studies in Friedreich Ataxia with a site in Germany, this one is one of 3 that started in 2023, is one of 8 observational ones, and was the 10th registered.
111 studies on the registry are indexed under Friedreich Ataxia; 28 are open to participants now.
Browse Friedreich Ataxia studies →6 of the 17 studies in Friedreich Ataxia with a site in Germany are open now. Recruiting first, the newest first:
Lexeo Therapeutics · Sites in Germany, United States, Brazil and 5 more countries
Ages 6+
Biogen · Sites in Germany, United States, Austria and 4 more countries
Ages 16+
Friedreich's Ataxia Research Alliance · Sites in Germany, United States, Australia and 14 more countries
Healthy volunteers
LMU Klinikum · Sites in Germany
Ages 65–65
LMU Klinikum · Sites in Germany
Ages 18–65
A study can be open while its sites in Germany have not opened yet or have stopped. Only those recruiting at a site in Germany →
Of the 18 Friedreich Ataxia studies recruiting now, 9 (50%) set no upper age limit and 9 (50%) stop at 75 or younger; 15 take children under 18; 3 take healthy volunteers.
This study: 12 Years and older · all sexes · no healthy volunteers.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
17 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 2 4 · Phase 3 5
First in 2006; this study’s year is marked. 3 started in 2023.
By drug class, each counting the studies that test any of its medicines:
This study's planned enrollment of 200 is above the median of 70 across 31 observational studies indexed under Friedreich Ataxia.
Median enrollment 39 across the 9 interventional studies, 250 across the 8 observational. Of the 6 that completed or were terminated, 3 (50%) have results posted.
This tab as a report to print or save as PDF → With an account.
9 of 17 have an industry lead sponsor. The 10 most frequent:
German Center for Neurodegenerative Diseases (DZNE) is the lead sponsor of 15 studies on the registry; 2 are open to participants now.
German Center for Neurodegenerative Diseases (DZNE) has 1 study filed under Friedreich Ataxia — fewer than ten, so there is no comparison to draw.
4 of the studies in Friedreich Ataxia with a site in Germany that are under way reach primary completion within a year, 1 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT06953583Phase 3BiogenA Study to Learn More About the Effects and Long-Term Safety of Omaveloxolone (BIIB141) in Children and Teens With Friedreich's Ataxia | 2027-11-16 |
| NCT07072676LMU KlinikumThe Use of Assistive Gait Devices Can Reduce the Risk of Falls in Patients With Neuromuscular Diseases Following a Training Period. | 2027-05-01 |
| NCT06605612LMU KlinikumDevelopment and Validation of the FBIndex to Determine the Risk of Falls for Patients With Neuromuscular Disorders | 2027-08-01 |
| NCT06865482Lexeo TherapeuticsClinical Course Of Disease In Participants With FA-CM | 2027-09 |
Add all 4 to your calendar (.ics) → With an account.
Among the 17 studies in Friedreich Ataxia with a site in Germany, from their registry updates:
Nothing recorded among them since 2026-09-25.
This tab as a report to print or save as PDF → With an account.
The PROFA study is an international, multi-centric observational and validation study to assess the patient-reported, psychosocial and economic outcomes of patients with Friedreich Ataxia (FA). Eligible patients will be recruited from six study centers in Germany, Austria and France. Patients will complete a baseline assessment via face-to-face interviews at the study centers and multiple momentary follow-up assessments via a mobile-health app at home daily to monthly for six months. Study results will gain essential and in-depth insights into the daily life of patients with FA.
There is a lack of knowledge about the patient-reported, psychosocial, and economic impact of Friedreich's Ataxia (FA). The few previous studies were based on small sample sizes and annual cross-sectional data, limiting the generalizability of the results.
Therefore, the PROFA study aims
Thus, the PROFA study will gain a comprehensive understanding of the individual, societal, and economic burden of FA, identifying determinants of health and social life and efficient use of healthcare resources. This is crucial to improve the treatment, care, and everyday life of FA patients and their families.
This validation and observational study will recruit patients from six study centers (Germany, France, and Austria). The data assessment will be based on (i) a baseline assessment via interviews at the study centers and (ii) a subsequent remote momentary data assessment via a mobile-health app on a daily to monthly basis for six months, covering assessments of ataxia severity, HRQoL, psychosocial health, speech and hearing disabilities, health services utilization, and specific health events. Descriptive and multivariate statistics will be used to describe the impact of FA on the patient-reported HRQoL, psychosocial health and economic outcomes.
Patients in this study have a Friedreich Ataxia (FA) disease confirmed by molecular genetic testing with disease severity of ≤30 points according to the Scale of the Assessment and Rating of Ataxia. FA is the most common hereditary ataxia in Europe. The genetic mutation that underlies almost all FA cases is a homozygous guanine-adenine-adenine triplet repeat expansion in the first intron of the FXN gene, which encodes the mitochondrial protein frataxin. Clinical onset of FA occurs most often around puberty, but in a few cases, symptoms develop in adulthood. FA is characterized by muscle weakness, imbalance, poor coordination, sensory loss, and speech problems. FA could cause wheelchair dependency and reduced life expectancy.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Usability of the mobile-health app as a remote monitoring momentary data assessment tool
Completeness of data
Time frame: Six months
Acceptability of the mobile-health app as a remote monitoring momentary data assessment tool
Acceptability is assessed by a self-developed questionnaire (asking patients to rate the app based on user experience)
Time frame: Month 6
Total societal costs
Aggregated healthcare costs of utilized healthcare services, informal care and productivity losses
Time frame: Six months
Health-related quality of life
Health-related quality of life assessed by the "Patient Reported Outcome Measures for Ataxia" Short-Form (PROM-ATAX), with a score range between 0 to 40 with higher scores indicating lower health-related quality of life
Time frame: Month 1
Health-related quality of life
Health-related quality of life assessed by the "Patient Reported Outcome Measures for Ataxia" Short-Form (PROM-ATAX), with a score range between 0 to 40 with higher scores indicating lower health-related quality of life
Time frame: Month 3
Health-related quality of life
Health-related quality of life assessed by the "Patient Reported Outcome Measures for Ataxia" Short-Form (PROM-ATAX), with a score range between 0 to 40 with higher scores indicating lower health-related quality of life
Time frame: Month 5
Health-related quality of life
Health-related quality of life assessed by the "Patient Reported Outcome Measures for Ataxia" Short-Form (PROM-ATAX), with a score range between 0 to 40 with higher scores indicating lower health-related quality of life
Time frame: Month 6
Psychosocial health due to communication handicaps caused by speech and hearing disabilities
Psychosocial impact of hearing and speech disabilities assessed by the "Scale for the psychosocial impact of hearing and speech disabilities in Friedreich Ataxia" (COM-ATAX) with a score range between 0 to 68 (higher scores indicate more difficulties)
Time frame: Month 1
Psychosocial health due to communication handicaps caused by speech and hearing disabilities
Psychosocial impact of hearing and speech disabilities assessed by the "Scale for the psychosocial impact of hearing and speech disabilities in Friedreich Ataxia" (COM-ATAX) with a score range between 0 to 68 (higher scores indicate more difficulties)
Time frame: Month 2
Psychosocial health due to communication handicaps caused by speech and hearing disabilities
Psychosocial impact of hearing and speech disabilities assessed by the "Scale for the psychosocial impact of hearing and speech disabilities in Friedreich Ataxia" (COM-ATAX) with a score range between 0 to 68 (higher scores indicate more difficulties)
Time frame: Month 3
Psychosocial health due to communication handicaps caused by speech and hearing disabilities
Psychosocial impact of hearing and speech disabilities assessed by the "Scale for the psychosocial impact of hearing and speech disabilities in Friedreich Ataxia" (COM-ATAX) with a score range between 0 to 68 (higher scores indicate more difficulties)
Time frame: Month 4
Psychosocial health due to communication handicaps caused by speech and hearing disabilities
Psychosocial impact of hearing and speech disabilities assessed by the "Scale for the psychosocial impact of hearing and speech disabilities in Friedreich Ataxia" (COM-ATAX) with a score range between 0 to 68 (higher scores indicate more difficulties)
Time frame: Month 5
Psychosocial health due to communication handicaps caused by speech and hearing disabilities
Psychosocial impact of hearing and speech disabilities assessed by the "Scale for the psychosocial impact of hearing and speech disabilities in Friedreich Ataxia" (COM-ATAX) with a score range between 0 to 68 (higher scores indicate more difficulties)
Time frame: Month 6
Fluctuation of health-related quality of life
Change in health-related quality of life measured by the 5-level EQ-5D version (EQ-5D-5L) with a score range between 0 and 1 (higher values indicate higher health-related quality of life)
Time frame: Day one, two and three in months 1
Fluctuation of health-related quality of life
Change in health-related quality of life measured by the 5-level EQ-5D version (EQ-5D-5L) with a score range between 0 and 1 (higher values indicate higher health-related quality of life)
Time frame: Day one, two and three in months 3
Fluctuation of health-related quality of life
Change in health-related quality of life measured by the 5-level EQ-5D version (EQ-5D-5L) with a score range between 0 and 1 (higher values indicate higher health-related quality of life)
Time frame: Day one, two and three in months 5
Fluctuation of health-related quality of life
Change in health-related quality of life measured by the 5-level EQ-5D version (EQ-5D-5L) with a score range between 0 and 1 (higher values indicate higher health-related quality of life)
Time frame: Day one, two and three in months 6
Started Jun 2023 (actual). Primary completion is estimated for Jun 2028, 60 months after the start.
How these figures are worked out, and which studies are left out of them.
Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.
Contact study teamGet an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
German Center for Neurodegenerative Diseases (DZNE)