5 and older, male only, with Duchenne Muscular Dystrophy or Becker Muscular Dystrophy. 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.
Number of Participants With Treatment-Emergent Adverse Events (AEs)Primary· Baseline (Week 48 of Study 007) up to Week 102
An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Severity of an AE was classified as: mild (does not interfere with usual function), moderate (interferes to some extent with usual function) and severe (interferes significantly with usual function). Drug-related AEs: AEs with a possible or probable relationship to study drug. Serious AEs: death, a life-threatening AE, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, a congenital anomal
Any AEs
Group
Value
95% CI
High-Dose Ataluren/High-Dose Ataluren
52
Low-Dose Ataluren/High-Dose Ataluren
49
Placebo/High-Dose Ataluren
48
Overall Participants: High-Dose Ataluren
149
Drug-related AEs
Group
Value
95% CI
High-Dose Ataluren/High-Dose Ataluren
10
Low-Dose Ataluren/High-Dose Ataluren
10
Placebo/High-Dose Ataluren
22
Overall Participants: High-Dose Ataluren
42
Serious AEs
Group
Value
95% CI
High-Dose Ataluren/High-Dose Ataluren
0
Low-Dose Ataluren/High-Dose Ataluren
1
Placebo/High-Dose Ataluren
2
Overall Participants: High-Dose Ataluren
3
Mild AEs
Group
Value
95% CI
High-Dose Ataluren/High-Dose Ataluren
34
Low-Dose Ataluren/High-Dose Ataluren
23
Placebo/High-Dose Ataluren
26
Overall Participants: High-Dose Ataluren
83
Moderate AEs
Group
Value
95% CI
High-Dose Ataluren/High-Dose Ataluren
13
Low-Dose Ataluren/High-Dose Ataluren
22
Placebo/High-Dose Ataluren
19
Overall Participants: High-Dose Ataluren
54
Severe AEs
Group
Value
95% CI
High-Dose Ataluren/High-Dose Ataluren
5
Low-Dose Ataluren/High-Dose Ataluren
4
Placebo/High-Dose Ataluren
3
Overall Participants: High-Dose Ataluren
12
Number of Participants With Clinically Significant Abnormal Laboratory ParametersPrimary· Baseline (Week 48 of Study 007) up to Week 102
Laboratory parameters tests included hematology, biochemistry assay (hepatic, renal, and serum electrolyte values), adrenal assays, and urinalysis. Clinical significance was defined as per investigator's judgement.
Group
Value
95% CI
High-Dose Ataluren/High-Dose Ataluren
0
Low-Dose Ataluren/High-Dose Ataluren
0
Placebo/High-Dose Ataluren
0
Overall Participants: High-Dose Ataluren
0
Change From Baseline in 6-Minute Walk Distance (6MWD) at Week 60Secondary· Baseline (Week 48 of Study 007), Week 60
The 6MWD test was performed in a 30 meters long flat corridor, where the participant was instructed to walk as far as possible, back and forth around two cones, with the permission to slow down, rest, or stop if needed. Ambulation was assessed via the 6MWD test following standardized procedures by measuring the 6MWD in meters. Participants were not permitted to use assistive devices (walker, long leg braces, or short leg braces) during the 6MWD test.
Baseline
Group
Value
95% CI
High-Dose Ataluren/High-Dose Ataluren
351.60
± 130.312
Low-Dose Ataluren/High-Dose Ataluren
375.95
± 98.976
Placebo/High-Dose Ataluren
356.25
± 109.369
Overall Participants: High-Dose Ataluren
361.11
± 113.760
Change at Week 60
Group
Value
95% CI
High-Dose Ataluren/High-Dose Ataluren
-17.23
± 39.608
Low-Dose Ataluren/High-Dose Ataluren
-10.50
± 41.390
Placebo/High-Dose Ataluren
-7.06
± 50.121
Overall Participants: High-Dose Ataluren
-11.56
± 43.881
Change From Baseline in Mean Activity Period/Day/Visit at Week 60, as Assessed by Step Activity Monitoring (SAM)Secondary· Baseline (Week 48 of Study 007), Week 60
The SAM is a pedometer (worn on the ankle) that continuously records the number of steps per time interval. Participants were instructed to continue to wear the SAM for at least 9 consecutive days. SAM was used to record the number of strides/minute following each visit. A stride is the leg motion that begins when the foot with SAM leaves the floor and ends when the same foot touches the floor again (that is, a stride generally equals 2 steps). Mean activity period/day/visit was computed for each participant. For each day, an active period was defined as the first time after 3:00 AM that great
Baseline
Group
Value
95% CI
High-Dose Ataluren/High-Dose Ataluren
759.715
± 77.5210
Low-Dose Ataluren/High-Dose Ataluren
742.223
± 107.7264
Placebo/High-Dose Ataluren
747.464
± 102.6531
Overall Participants: High-Dose Ataluren
750.016
± 96.1057
Change at Week 60
Group
Value
95% CI
High-Dose Ataluren/High-Dose Ataluren
1.946
± 61.2247
Low-Dose Ataluren/High-Dose Ataluren
34.283
± 88.1587
Placebo/High-Dose Ataluren
-1.866
± 93.0531
Overall Participants: High-Dose Ataluren
10.991
± 82.6356
Change From Baseline in Mean Total Step Count/Day/Visit During the Active Periods at Week 60, as Assessed by SAMSecondary· Baseline (Week 48 of Study 007), Week 60
The SAM is a pedometer (worn on the ankle) that continuously records the number of steps per time interval. Participants were instructed to continue to wear the SAM for at least 9 consecutive days. SAM was used to record the number of strides/minute following each visit. A stride is the leg motion that begins when the foot with SAM leaves the floor and ends when the same foot touches the floor again (that is, a stride generally equals 2 steps). Mean total step count/day/visit during the active periods was computed for each participant. For each day, an active period was defined as the first ti
Baseline
Group
Value
95% CI
High-Dose Ataluren/High-Dose Ataluren
4934.575
± 2433.230
Low-Dose Ataluren/High-Dose Ataluren
4543.766
± 2051.300
Placebo/High-Dose Ataluren
5175.330
± 2307.436
Overall Participants: High-Dose Ataluren
4883.613
± 2272.108
Change at Week 60
Group
Value
95% CI
High-Dose Ataluren/High-Dose Ataluren
-357.679
± 1232.109
Low-Dose Ataluren/High-Dose Ataluren
-215.062
± 1761.773
Placebo/High-Dose Ataluren
-647.504
± 1680.522
Overall Participants: High-Dose Ataluren
-409.558
± 1566.386
Change From Baseline in Mean Total Step Count/Hour During the Active Period at Week 60, as Assessed by SAMSecondary· Baseline (Week 48 of Study 007), Week 60
The SAM is a pedometer (worn on the ankle) that continuously records the number of steps per time interval. Participants were instructed to continue to wear the SAM for at least 9 consecutive days. SAM was used to record the number of strides/minute following each visit. A stride is the leg motion that begins when the foot with SAM leaves the floor and ends when the same foot touches the floor again (that is, a stride generally equals 2 steps). Mean total step count/hour during the active periods for the days in a visit was computed for each participant. For each day, an active period was defi
Baseline
Group
Value
95% CI
High-Dose Ataluren/High-Dose Ataluren
395.491
± 200.7671
Low-Dose Ataluren/High-Dose Ataluren
368.672
± 155.6648
Placebo/High-Dose Ataluren
420.684
± 188.9124
Overall Participants: High-Dose Ataluren
394.787
± 183.0994
Change at Week 60
Group
Value
95% CI
High-Dose Ataluren/High-Dose Ataluren
-27.650
± 87.8797
Low-Dose Ataluren/High-Dose Ataluren
-34.693
± 128.0609
Placebo/High-Dose Ataluren
-54.755
± 122.4142
Overall Participants: High-Dose Ataluren
-39.010
± 113.3481
Change From Baseline in Maximum Continuous 10-minute, 20-minute, 30-minute, and 60-minute Total Step Count at Week 60, as Assessed by SAMSecondary· Baseline (Week 48 of Study 007), Week 60
SAM is a pedometer (worn on the ankle) that continuously records the number of steps per time interval. Participants were instructed to continue to wear the SAM for at least 9 consecutive days. SAM was used to record the number of strides/minute following each visit. A stride is the leg motion that begins when the foot with SAM leaves the floor and ends when the same foot touches the floor again (that is, a stride generally equals 2 steps). The maximum continuous 10-minute, 20-minute, 30-minute, and 60-minute total step counts were computed for each participant. For each day, an active period
Baseline: 10-minute total step count
Group
Value
95% CI
High-Dose Ataluren/High-Dose Ataluren
34.603
± 12.4886
Low-Dose Ataluren/High-Dose Ataluren
32.863
± 8.8533
Placebo/High-Dose Ataluren
35.256
± 10.0379
Overall Participants: High-Dose Ataluren
34.241
± 10.5911
Change at Week 60: 10-minute total step count
Group
Value
95% CI
High-Dose Ataluren/High-Dose Ataluren
-1.140
± 6.9584
Low-Dose Ataluren/High-Dose Ataluren
-0.607
± 7.2946
Placebo/High-Dose Ataluren
-2.372
± 7.1735
Overall Participants: High-Dose Ataluren
-1.384
± 7.1170
Baseline: 20-minute total step count
Group
Value
95% CI
High-Dose Ataluren/High-Dose Ataluren
28.138
± 11.0629
Low-Dose Ataluren/High-Dose Ataluren
26.096
± 7.9168
Placebo/High-Dose Ataluren
28.323
± 9.3133
Overall Participants: High-Dose Ataluren
27.526
± 9.5426
Change at Week 60: 20-minute total step count
Group
Value
95% CI
High-Dose Ataluren/High-Dose Ataluren
-1.459
± 6.7165
Low-Dose Ataluren/High-Dose Ataluren
-0.662
± 6.2171
Placebo/High-Dose Ataluren
-2.510
± 6.4346
Overall Participants: High-Dose Ataluren
-1.558
± 6.4549
Baseline: 30-minute total step count
Group
Value
95% CI
High-Dose Ataluren/High-Dose Ataluren
24.222
± 9.9576
Low-Dose Ataluren/High-Dose Ataluren
22.205
± 7.1045
Placebo/High-Dose Ataluren
24.498
± 8.4938
Overall Participants: High-Dose Ataluren
23.648
± 8.6306
Change at Week 60: 30-minute total step count
Group
Value
95% CI
High-Dose Ataluren/High-Dose Ataluren
-1.400
± 5.7605
Low-Dose Ataluren/High-Dose Ataluren
-0.450
± 5.6811
Placebo/High-Dose Ataluren
-2.457
± 5.9579
Overall Participants: High-Dose Ataluren
-1.452
± 5.8110
Baseline: 60-minute total step count
Group
Value
95% CI
High-Dose Ataluren/High-Dose Ataluren
18.019
± 7.8145
Low-Dose Ataluren/High-Dose Ataluren
16.453
± 5.6625
Placebo/High-Dose Ataluren
18.599
± 6.9509
Overall Participants: High-Dose Ataluren
17.691
± 6.8991
Change at Week 60: 60-minute total step count
Group
Value
95% CI
High-Dose Ataluren/High-Dose Ataluren
-0.977
± 3.7328
Low-Dose Ataluren/High-Dose Ataluren
-0.210
± 4.7035
Placebo/High-Dose Ataluren
-2.257
± 5.1038
Overall Participants: High-Dose Ataluren
-1.162
± 4.5803
Change From Baseline in Percentage of Time During Active Period Spent at No Activity (0 Steps/Minute[Min]), Low Activity (Less Than or Equal to [≤]15 Steps/Min), Medium Activity (16-30 Steps/Min), and High Activity (Greater Than[>]30 Steps/Min) at Week 60Secondary· Baseline (Week 48 of Study 007), Week 60
SAM is a pedometer (worn on the ankle) that continuously records the number of steps per time interval. Participants were instructed to continue to wear SAM for at least 9 consecutive days. SAM was used to record number of strides/minute following each visit. A stride is leg motion that begins when the foot with SAM leaves the floor and ends when the same foot touches the floor again. Percentage of time during active periods spent at no activity (0 steps/min), low activity (≤15 steps/min), medium activity (16-30 steps/min), and high activity (\>30 steps/min) were computed for each participant.
Baseline: Time spent at no activity
Group
Value
95% CI
High-Dose Ataluren/High-Dose Ataluren
52.818
± 14.4129
Low-Dose Ataluren/High-Dose Ataluren
53.372
± 12.2352
Placebo/High-Dose Ataluren
50.006
± 12.5263
Overall Participants: High-Dose Ataluren
52.094
± 13.1188
Change at Week 60: Time spent at no activity
Group
Value
95% CI
High-Dose Ataluren/High-Dose Ataluren
-0.051
± 8.1776
Low-Dose Ataluren/High-Dose Ataluren
0.830
± 11.2481
Placebo/High-Dose Ataluren
2.194
± 7.8652
Overall Participants: High-Dose Ataluren
0.985
± 9.1467
Baseline: Time spent at low activity
Group
Value
95% CI
High-Dose Ataluren/High-Dose Ataluren
30.677
± 7.8531
Low-Dose Ataluren/High-Dose Ataluren
31.170
± 7.2431
Placebo/High-Dose Ataluren
32.393
± 7.2426
Overall Participants: High-Dose Ataluren
31.392
± 7.4462
Change at Week 60: Time spent at low activity
Group
Value
95% CI
High-Dose Ataluren/High-Dose Ataluren
1.270
± 5.8178
Low-Dose Ataluren/High-Dose Ataluren
1.172
± 8.3050
Placebo/High-Dose Ataluren
0.252
± 5.5885
Overall Participants: High-Dose Ataluren
0.896
± 6.6058
Baseline: Time spent at medium activity
Group
Value
95% CI
High-Dose Ataluren/High-Dose Ataluren
10.248
± 4.7620
Low-Dose Ataluren/High-Dose Ataluren
10.119
± 4.4344
Placebo/High-Dose Ataluren
10.983
± 4.6049
Overall Participants: High-Dose Ataluren
10.442
± 4.5901
Change at Week 60: Time spent at medium activity
Group
Value
95% CI
High-Dose Ataluren/High-Dose Ataluren
-0.542
± 2.0960
Low-Dose Ataluren/High-Dose Ataluren
-1.356
± 3.7830
Placebo/High-Dose Ataluren
-0.965
± 3.3763
Overall Participants: High-Dose Ataluren
-0.944
± 3.1392
Baseline: Time spent at high activity
Group
Value
95% CI
High-Dose Ataluren/High-Dose Ataluren
6.404
± 4.5938
Low-Dose Ataluren/High-Dose Ataluren
5.370
± 3.4243
Placebo/High-Dose Ataluren
6.673
± 4.5847
Overall Participants: High-Dose Ataluren
6.149
± 4.2476
Change at Week 60: Time spent at high activity
Group
Value
95% CI
High-Dose Ataluren/High-Dose Ataluren
-0.643
± 2.2656
Low-Dose Ataluren/High-Dose Ataluren
-0.520
± 2.7196
Placebo/High-Dose Ataluren
-1.506
± 2.9904
Overall Participants: High-Dose Ataluren
-0.898
± 2.6913
Change From Baseline in Time to Stand From Supine Position at Week 60Secondary· Baseline (Week 48 of Study 007), Week 60
If the time taken to perform this test exceeded 30 seconds or if a participant could not perform this test due to disease progression, a value of 30 seconds was used. Change from baseline data has been reported.
Baseline
Group
Value
95% CI
High-Dose Ataluren/High-Dose Ataluren
13.736
± 11.3353
Low-Dose Ataluren/High-Dose Ataluren
11.796
± 10.3544
Placebo/High-Dose Ataluren
12.398
± 11.6010
Overall Participants: High-Dose Ataluren
12.666
± 11.0678
Change at Week 60
Group
Value
95% CI
High-Dose Ataluren/High-Dose Ataluren
0.665
± 3.0080
Low-Dose Ataluren/High-Dose Ataluren
-0.187
± 4.6289
Placebo/High-Dose Ataluren
0.002
± 4.6322
Overall Participants: High-Dose Ataluren
0.166
± 4.1373
Change From Baseline in Time to Walk/Run 10 Meters at Week 60Secondary· Baseline (Week 48 of Study 007), Week 60
If the time taken to perform this test exceeded 30 seconds or if a participant could not perform this test due to disease progression, a value of 30 seconds was used. Change from baseline data has been reported.
Baseline
Group
Value
95% CI
High-Dose Ataluren/High-Dose Ataluren
8.143
± 5.1475
Low-Dose Ataluren/High-Dose Ataluren
6.986
± 2.9995
Placebo/High-Dose Ataluren
6.967
± 3.5789
Overall Participants: High-Dose Ataluren
7.384
± 4.0530
Change at Week 60
Group
Value
95% CI
High-Dose Ataluren/High-Dose Ataluren
1.441
± 4.4822
Low-Dose Ataluren/High-Dose Ataluren
0.547
± 2.2058
Placebo/High-Dose Ataluren
0.758
± 2.6862
Overall Participants: High-Dose Ataluren
0.922
± 3.2905
Change From Baseline in Time to Climb 4 Stairs at Week 60Secondary· Baseline (Week 48 of Study 007), Week 60
If the time taken to perform this test exceeded 30 seconds or if a participant could not perform this test due to disease progression, a value of 30 seconds was used. Change from baseline data has been reported.
Baseline
Group
Value
95% CI
High-Dose Ataluren/High-Dose Ataluren
9.077
± 9.2543
Low-Dose Ataluren/High-Dose Ataluren
6.738
± 6.4069
Placebo/High-Dose Ataluren
8.071
± 9.2057
Overall Participants: High-Dose Ataluren
7.984
± 8.4076
Change at Week 60
Group
Value
95% CI
High-Dose Ataluren/High-Dose Ataluren
0.831
± 2.4664
Low-Dose Ataluren/High-Dose Ataluren
0.696
± 4.0707
Placebo/High-Dose Ataluren
0.194
± 2.5268
Overall Participants: High-Dose Ataluren
0.574
± 3.0898
Change From Baseline in Time to Descend 4 Stairs at Week 60Secondary· Baseline (Week 48 of Study 007), Week 60
If the time taken to perform this test exceeded 30 seconds or if a participant could not perform this test due to disease progression, a value of 30 seconds was used. Change from baseline data has been reported.
Baseline
Group
Value
95% CI
High-Dose Ataluren/High-Dose Ataluren
7.685
± 8.9934
Low-Dose Ataluren/High-Dose Ataluren
5.638
± 5.5635
Placebo/High-Dose Ataluren
6.641
± 7.8591
Overall Participants: High-Dose Ataluren
6.675
± 7.6319
Change at Week 60
Group
Value
95% CI
High-Dose Ataluren/High-Dose Ataluren
1.167
± 4.7624
Low-Dose Ataluren/High-Dose Ataluren
0.626
± 5.2567
Placebo/High-Dose Ataluren
-0.648
± 4.9202
Overall Participants: High-Dose Ataluren
0.385
± 5.0045
Adverse events — posted to ClinicalTrials.gov
Time frame: Baseline (Week 48 of Study 007) up to Week 102.
Reporting threshold: 0%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
High-Dose Ataluren/High-Dose Ataluren
Serious: 0/59 (0%)
Deaths: —
Low-Dose Ataluren/High-Dose Ataluren
Serious: 1/57 (2%)
Deaths: —
Placebo/High-Dose Ataluren
Serious: 2/57 (4%)
Deaths: —
Overall Participants: High-Dose Ataluren
Serious: 3/173 (2%)
Deaths: —
Serious adverse events (2 terms)
Reaction
System
High-Dose Ataluren/High-Do…
Low-Dose Ataluren/High-Dos…
Placebo/High-Dose Ataluren
Overall Participants: High…
Femur fracture
Injury, poisoning and procedural complications
—
—
—
—
Hypertension
Vascular disorders
—
—
—
—
Other adverse events (210 terms — click to expand)
Reaction
System
High-Dose Ataluren/High-Do…
Low-Dose Ataluren/High-Dos…
Placebo/High-Dose Ataluren
Overall Participants: High…
Vomiting
Gastrointestinal disorders
—
—
—
—
Headache
Nervous system disorders
—
—
—
—
Diarrhoea
Gastrointestinal disorders
—
—
—
—
Disease progression
General disorders
—
—
—
—
Influenza
Infections and infestations
—
—
—
—
Abdominal pain
Gastrointestinal disorders
—
—
—
—
Nasopharyngitis
Infections and infestations
—
—
—
—
Abdominal pain upper
Gastrointestinal disorders
—
—
—
—
Flatulence
Gastrointestinal disorders
—
—
—
—
Upper respiratory tract infection
Infections and infestations
—
—
—
—
Fall
Injury, poisoning and procedural complications
—
—
—
—
Cough
Respiratory, thoracic and mediastinal disorders
—
—
—
—
Nausea
Gastrointestinal disorders
—
—
—
—
Back pain
Musculoskeletal and connective tissue disorders
—
—
—
—
Muscular weakness
Musculoskeletal and connective tissue disorders
—
—
—
—
Pain in extremity
Musculoskeletal and connective tissue disorders
—
—
—
—
Asthenia
General disorders
—
—
—
—
Gait disturbance
General disorders
—
—
—
—
Pyrexia
General disorders
—
—
—
—
Joint contracture
Musculoskeletal and connective tissue disorders
—
—
—
—
Ear infection
Infections and infestations
—
—
—
—
Gastroenteritis viral
Infections and infestations
—
—
—
—
Arthralgia
Musculoskeletal and connective tissue disorders
—
—
—
—
Oropharyngeal pain
Respiratory, thoracic and mediastinal disorders
—
—
—
—
Muscle tightness
Musculoskeletal and connective tissue disorders
—
—
—
—
Skin papilloma
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Duchenne/Becker muscular dystrophy (DMD/BMD) is a genetic disorder that develops in boys. It is caused by a mutation in the gene for dystrophin, a protein that is important for maintaining normal muscle structure and function. Loss of dystrophin causes muscle fragility that leads to weakness and loss of walking ability during childhood and teenage years. A specific type of mutation, called a nonsense (premature stop codon) mutation is the cause of DMD/BMD in approximately 10-15% of boys with the disease. Ataluren (PTC124) is an orally delivered, investigational drug that has the potential to overcome the effects of the nonsense mutation. This study is a Phase 2b extension trial that will evaluate the long-term safety of ataluren (PTC124) in boys with nonsense mutation DMD/BMD, as determined by adverse events and laboratory abnormalities. The study will also assess changes in walking, muscle function, and other important clinical and laboratory measures.
Publications & conference data
6 peer-reviewed publications reference this trial (live from Europe PMC):
NCT04336826 — A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From ≥6 Months to <2 Years of Age With N
· Phase 2
· completed
NCT03796637 — A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have
· Phase 2
· completed
NCT04117880 — A Phase 2 Open Label Extension Study in Participants With Nonsense Mutation Aniridia
· Phase 2
· withdrawn
NCT03648827 — A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
· Phase 2
· completed
NCT03179631 — Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy
· Phase 3
· completed
Other recruiting trials for Duchenne Muscular Dystrophy
Currently open trials in the same condition.
NCT07287189 — Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients
· Phase 2
· recruiting
NCT06817382 — A Study to Investigate the Safety and Biodistribution of a Single Intrathecal (IT) Injection of INS1201 in Ambulatory Ma
· Phase 1
· recruiting
NCT06402942 — Gamified Occupational Therapy for Adolescents With Duchenne Muscular Dystrophy
· NA
· recruiting
NCT06450639 — A Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy (DMD)
· Phase 2
· active not recruiting
NCT06692426 — Trial of Cell Based Therapy for DMD
· 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: 15 July 2020
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/NCT00847379.