7 and older, any sex, with Friedreich Ataxia. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Change From Baseline in the mFARS Score at Week 72 - Modified Intent-to-treat (mITT) Analysis SetPrimary· Baseline, Week 72
mFARS is a 93-item scale; comprised of neurologic component of FARS. For each item, responses categorize the corresponding neurological finding, with a score ranging from 0 to 3, 4, or 5 with 0 being normal and higher numbers indicative of greater impairment. Total mFARS scores for each subscale: bulbar (0 to 5), upper limb coordination (0 to 36), lower limb coordination (0 to 16), and upright stability (0 to 36). mFARS total score was a composite score of all 4 subscales, ranging from 0 (normal) to 93 (greater impairment). A lower score = better neurological function. Missing data was imputed
Group
Value
95% CI
Vatiquinone
1.218
± 0.9652
Placebo
2.828
± 0.9994
Change From Baseline in the mFARS Score at Week 72 - Intent-to-treat (ITT) Analysis SetPrimary· Baseline, Week 72
mFARS is a 93-item scale; comprised of neurologic component of FARS. For each item, responses categorize the corresponding neurological finding, with a score ranging from 0 to 3, 4, or 5 with 0 being normal and higher numbers indicative of greater impairment. Total mFARS scores for each subscale: bulbar (0 to 5), upper limb coordination (0 to 36), lower limb coordination (0 to 16), and upright stability (0 to 36). mFARS total score was a composite score of all 4 subscales, ranging from 0 (normal) to 93 (greater impairment). A lower score = better neurological function. Missing data was imputed
Group
Value
95% CI
Vatiquinone
0.898
± 0.8500
Placebo
2.555
± 0.8770
Change From Baseline in Friedreich Ataxia Rating Scale Activities of Daily Living (FARS-ADL) Score at Week 72 - mITT Analysis SetSecondary· Baseline, Week 72
The ADL component of the FARS includes 9 subscales: speech, swallowing, cutting food and handling utensils, dressing, personal hygiene, falling, walking, quality of sitting position, and bladder function. Each of these subscales is rated on a 5-point scale where 0=normal to 4=severe disability/inability to carry out activity independently for a total possible score of 0 to 36, with higher scores representing greater disability/dependency. Missing data was imputed using pattern mix model multiple imputation. LS mean and SE was calculated using MMRM.
Group
Value
95% CI
Vatiquinone
0.759
± 0.5992
Placebo
1.677
± 0.6202
Change From Baseline in FARS-ADL Score at Week 72 - ITT Analysis SetSecondary· Baseline, Week 72
The ADL component of the FARS includes 9 subscales: speech, swallowing, cutting food and handling utensils, dressing, personal hygiene, falling, walking, quality of sitting position, and bladder function. Each of these subscales is rated on a 5-point scale where 0=normal to 4=severe disability/inability to carry out activity independently for a total possible score of 0 to 36, with higher scores representing greater disability/dependency. Missing data was imputed using pattern mix model multiple imputation. LS mean and SE was calculated using MMRM.
Group
Value
95% CI
Vatiquinone
0.707
± 0.5219
Placebo
1.692
± 0.5402
Change From Baseline in 1-Minute Walk Test (1MWT) at Week 72 - mITT Analysis SetSecondary· Baseline, Week 72
The 1MWT is a timed performance test used to measure functional ability, walking endurance, balance, and muscle performance by measuring maximal walking speed in 1 minute. Participants were instructed to walk as quickly as possible for 1 minute without running. Maximal walking speed was measured upon completion of the walk and distance was recorded. The mean change from baseline in the distance the participant walked is reported. Missing data was imputed using pattern mix model multiple imputation. LS mean and SE was calculated using MMRM.
Group
Value
95% CI
Vatiquinone
-4.324
± 2.5882
Placebo
-9.290
± 2.6843
Change From Baseline in 1MWT at Week 72 - ITT Analysis SetSecondary· Baseline, Week 72
The 1MWT is a timed performance test used to measure functional ability, walking endurance, balance, and muscle performance by measuring maximal walking speed in 1 minute. Participants were instructed to walk as quickly as possible for 1 minute without running. Maximal walking speed was measured upon completion of the walk and distance was recorded. The mean change from baseline in the distance the participant walked is reported. Missing data was imputed using pattern mix model multiple imputation. LS mean and SE was calculated using MMRM.
Group
Value
95% CI
Vatiquinone
-4.623
± 2.2113
Placebo
-9.462
± 2.2687
Number of Falls Per 28 Days Over Every 24-Week Period - mITT Analysis SetSecondary· Week 1-24, Week 25-48, and Week 49-72
Each participant was required to maintain a fall log, which included the date and time of each fall. Falls as defined by World Health Organization as "inadvertently coming to rest on the ground, floor or other lower level, excluding intentional change in position to rest in furniture, wall or other objects," were reported. Number of falls per 28 days during a time interval was calculated as the number of falls during the period divided by the number of days during the interval, and multiplied by 28. The falls that occurred on or after the first Loss of Ambulation visit were excluded from the a
Week 1-24
Group
Value
95% CI
Vatiquinone
5.029
± 14.4686
Placebo
3.999
± 6.9569
Week 25-48
Group
Value
95% CI
Vatiquinone
4.098
± 11.5289
Placebo
3.900
± 9.0212
Week 49-72
Group
Value
95% CI
Vatiquinone
3.732
± 10.0790
Placebo
4.462
± 12.1785
Number of Falls Per 28 Days Over Every 24-Week Period - ITT Analysis SetSecondary· Week 1-24, Week 25-48, and Week 49-72
Each participant was required to maintain a fall log, which included the date and time of each fall. Falls as defined by World Health Organization as "inadvertently coming to rest on the ground, floor or other lower level, excluding intentional change in position to rest in furniture, wall or other objects," were reported. Number of falls per 28 days during a time interval was calculated as the number of falls during the period divided by the number of days during the interval, and multiplied by 28. The falls that occurred on or after the first Loss of Ambulation visit were excluded from the a
Week 1-24
Group
Value
95% CI
Vatiquinone
4.464
± 13.5618
Placebo
3.535
± 6.5231
Week 25-48
Group
Value
95% CI
Vatiquinone
3.920
± 10.9850
Placebo
3.518
± 8.4701
Week 49-72
Group
Value
95% CI
Vatiquinone
3.407
± 9.6043
Placebo
3.974
± 11.4690
Change From Baseline in the Upright Stability Subscale of the mFARS at Week 72 - mITT Analysis SetSecondary· Baseline, Week 72
mFARS is a 93-item scale; comprised of neurologic component of FARS. For each item, responses categorize the corresponding neurological finding, with a score ranging from 0 to 3, 4, or 5 with 0 being normal and higher numbers indicative of greater impairment. The Upright Stability subscale of the mFARS includes following items: 1. Sitting posture (score: 0 to 4); 2a. Stance with feet apart and eyes open (score: 0 to 4); 2b. Stance with feet apart and eyes closed (score: 0 to 4); 3a. Stance with feet together and eyes open (score: 0 to 4); 3b. Stance with feet together and eyes closed (score: 0
Group
Value
95% CI
Vatiquinone
1.734
± 0.4911
Placebo
2.991
± 0.5094
Change From Baseline in the Upright Stability Subscale of the mFARS at Week 72 - ITT Analysis SetSecondary· Baseline, Week 72
mFARS is a 93-item scale; comprised of neurologic component of FARS. For each item, responses categorize the corresponding neurological finding, with a score ranging from 0 to 3, 4, or 5 with 0 being normal and higher numbers indicative of greater impairment. The Upright Stability subscale of the mFARS includes following items: 1. Sitting posture (score: 0 to 4); 2a. Stance with feet apart and eyes open (score: 0 to 4); 2b. Stance with feet apart and eyes closed (score: 0 to 4); 3a. Stance with feet together and eyes open (score: 0 to 4); 3b. Stance with feet together and eyes closed (score: 0
Group
Value
95% CI
Vatiquinone
1.379
± 0.4321
Placebo
2.489
± 0.4468
Change From Baseline in the Modified Fatigue Impact Scale (MFIS) Score at Week 72 - mITT Analysis SetSecondary· Baseline, Week 72
The MFIS is a 21-item, reliable, validated instrument that has been utilized in many neurological disorders. It is a modified form of the Fatigue Impact Scale, a component of the Multiple Sclerosis Quality of Life Inventory. Each item was scored on a scale of 0 (never) to 4 (almost always). The total score was the sum of all item's score and ranged from 0 (no fatigue impact) to 84 (almost always impacted by fatigue). Higher scores indicated greater impact of fatigue on participant function.
Group
Value
95% CI
Vatiquinone
-0.756
± 2.2197
Placebo
4.291
± 2.3344
Change From Baseline in the MFIS Score at Week 72 - ITT Analysis SetSecondary· Baseline, Week 72
The MFIS is a 21-item, reliable, validated instrument that has been utilized in many neurological disorders. It is a modified form of the Fatigue Impact Scale, a component of the Multiple Sclerosis Quality of Life Inventory. Each item was scored on a scale of 0 (never) to 4 (almost always). The total score was the sum of all item's score and ranged from 0 (no fatigue impact) to 84 (almost always impacted by fatigue). Higher scores indicated greater impact of fatigue on participant function.
Group
Value
95% CI
Vatiquinone
-1.200
± 1.9897
Placebo
4.354
± 2.0758
Adverse events — posted to ClinicalTrials.gov
Time frame: Baseline up to Week 100.
Reporting threshold: 5%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
The primary objective of the study is to evaluate the efficacy (using the modified Friedreich Ataxia Rating Scale \[mFARS\]) and safety of vatiquinone in participants with Friedreich ataxia (FA).
Publications & conference data
6 peer-reviewed publications reference this trial (live from Europe PMC):
NCT05515536 — A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia
· Phase 3
· active not recruiting
NCT05485987 — A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
· Phase 2
· completed
NCT05218655 — A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease
· Phase 3
· completed
NCT04378075 — A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractor
· Phase 2, PHASE3
· terminated
NCT07159139 — Vatiquinone Expanded Access Protocol
· no longer available
Other recruiting trials for Friedreich Ataxia
Currently open trials in the same condition.
NCT06628687 — A Study to Learn How BIIB141 (Omaveloxolone) Affects the Health of Participants With Friedrich's Ataxia Who Took it Duri
· recruiting
NCT06953583 — A Study to Learn More About the Effects and Long-Term Safety of BIIB141 (Omaveloxolone) in Participants With Friedreich'
· Phase 3
· recruiting
NCT06623890 — A Study to Learn More About the Long-Term Safety of BIIB141 (Omaveloxolone) in Participants With Friedreich's Ataxia Who
· recruiting
NCT06054893 — A Study to Find Out How BIIB141 (Omaveloxolone) is Processed in the Body and to Learn More About Its Safety in Participa
· Phase 1
· recruiting
Other PTC Therapeutics trials
Trials by the same sponsor.
NCT06302348 — A Study of Sepiapterin in Participants With Phenylketonuria (PKU)
· Phase 3
· recruiting
NCT05515536 — A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia
· Phase 3
· active not recruiting
NCT05485987 — A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
· Phase 2
· completed
NCT05218655 — A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease
· Phase 3
· completed
NCT05358717 — A Study to Evaluate the Safety and Efficacy of PTC518 in Participants With Huntington's Disease (HD)
· Phase 2
· completed
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by PTC Therapeutics
Last refreshed: 13 April 2026
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT04577352.