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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT02090959
Registration number
NCT02090959
Ethics application status
Date submitted
17/03/2014
Date registered
19/03/2014
Date last updated
11/08/2020
Titles & IDs
Public title
An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy
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Scientific title
A Phase 3 Extension Study of Ataluren (PTC124) in Patients With Nonsense Mutation Dystrophinopathy
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Secondary ID [1]
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PTC124-GD-020e-DMD
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Muscular Dystrophy, Duchenne
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Muscular Dystrophies
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Muscular Disorders, Atrophic
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Muscular Diseases
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Musculoskeletal Diseases
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Neuromuscular Diseases
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Nervous System Diseases
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Genetic Diseases, X-Linked
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Genetic Diseases, Inborn
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Condition category
Condition code
Musculoskeletal
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Other muscular and skeletal disorders
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Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Neurological
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Other neurological disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - Ataluren
Experimental: Ataluren - Participants will receive ataluren suspension orally 3 times a day (TID), 10 milligrams/kilogram (mg/kg) at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for up to 144 weeks.
Treatment: Drugs: Ataluren
Ataluren will be administered per the dose and schedule specified in the arm.
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
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Assessment method [1]
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An adverse event (AE): any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Severity of AEs: graded per Common Terminology Criteria for AEs (CTCAE), Version 3.0 as Grade 1 (mild), 2 (moderate), 3 (severe), 4 (life-threatening), 5 (death). Drug-related AEs: AEs with possible, probable, unlikely relationship, or unrelated to study drug. Serious AEs (SAEs): death, a life-threatening AE, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, a congenital anomaly or birth defect, or an important medical event that jeopardized participant and required medical intervention. TEAE: an AE that occurred or worsened in the period extending from first dose of study drug in this study to 6 weeks after last dose of study drug in this study. A summary of other non-serious AEs and all SAEs, regardless of causality is located in Reported AE section.
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Timepoint [1]
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Baseline (Day 1) up to 6 weeks post-treatment (Week 150)
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Primary outcome [2]
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Number of Participants With Abnormalities in Clinical Laboratory Parameters
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Assessment method [2]
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Abnormalities in laboratory variables as pre-defined in protocol for safety-monitoring were: Hepatic (Serum alanine aminotransferase [ALT]: increase of greater than [>] 150 units/liter [U/L] with stable or decrease of creatinine kinese [CK]; Serum glutamyl amino transferase [GGT] [U/L]: Grade 2 [>2.5 - 5.0 * upper limit of normal {ULN}]), renal (Serum cystatin C miiligrams/liter [mg/L] >1.33 - 2.00 mg/L; Serum blood urea nitrogen [UREAN] [millimoles/liter {mmol/L}] greater than or equal to [=]1.5 - 3.0 * ULN; Urine occult blood: 2+ [Small], 3+ [Moderate], 4+ [Large]), and electrolytes (Serum sodium: low [mmol/L], Grade 3-4 [less than {<}130 mmol/L]; serum potassium: high [mmol/L], Grade 3-4 [>6.0 mmol/L]; and Serum bicarbonate [mmol/L]: Grade 2 [<16 - 11 mmol/L]).
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Timepoint [2]
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Baseline (Day 1) up to 6 weeks post-treatment (Week 150)
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Secondary outcome [1]
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Change From Baseline in 6MWD at Week 144
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Assessment method [1]
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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.
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Timepoint [1]
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Baseline, Week 144
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Secondary outcome [2]
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Change From Baseline in Time to Stand From Supine Position at Week 144
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Assessment method [2]
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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.
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Timepoint [2]
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Baseline, Week 144
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Secondary outcome [3]
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Change From Baseline in Time to Walk/Run 10 Meters at Week 144
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Assessment method [3]
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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.
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Timepoint [3]
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Baseline, Week 144
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Secondary outcome [4]
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Change From Baseline in Time to Climb 4 Stairs at Week 144
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Assessment method [4]
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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.
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Timepoint [4]
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Baseline, Week 144
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Secondary outcome [5]
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Change From Baseline in Time to Descend 4 Stairs at Week 144
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Assessment method [5]
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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.
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Timepoint [5]
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Baseline, Week 144
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Secondary outcome [6]
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Change From Baseline in Physical Function Total Score as Measured by NSAA at Week 144
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Assessment method [6]
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Physical function was assessed via the NSAA, a functional scale specifically designed for ambulant Duchenne muscular dystrophy (DMD) participants. The assessment comprised tests for 17 abilities of a participant, such as ability to stand, rise from the floor, get from lying to sitting, get from sitting to standing, raise one's head, stand on one's heels, hop, jump, and run. For each activity, a score of 0, 1, or 2 was recorded, with 0 = "unable to achieve independently," 1 = "modified method but achieves goal independent of physical assistance from another," or 2 = "normal- achieves goal without any assistance." The sum of these scores (except for 'raise one's head' activity score) was reported as the ordinal total score, which was transformed to a linear total score ranging from 0 (worst) to 100 (best). Participants with confirmed loss of ambulation at a particular visit were assigned a score of 0.
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Timepoint [6]
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Baseline, Week 144
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Secondary outcome [7]
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Change From Baseline in PUL Total Score at Week 144
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Assessment method [7]
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The PUL was used to assess motor performance of the upper limb. The PUL scale includes 22 items; an entry item defining the starting functional level, and 21 items subdivided into shoulder level (4 items), elbow level (9 items), and distal level (8 items) dimensions. Scoring options per item may not be uniform and may vary from 0-1 and 0-6, according to the performance, with higher values corresponding to better performance. Each dimension was scored separately with a maximum score of 16 for shoulder level, 34 for elbow level, and 24 for distal level. Total score was calculated by adding the 3 level scores, with a maximum global score of 74 (total score range = 0-74). Higher score = better outcome.
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Timepoint [7]
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Baseline, Week 144
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Secondary outcome [8]
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Change From Baseline in Percent Predicted FVC as Measured by Spirometry at Week 144
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Assessment method [8]
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FVC is a standard pulmonary function test. FVC was defined as the volume of air that can forcibly be blown out after full inspiration in the upright position, measured in liters. Percent predicted FVC (in %) = [(observed FVC)/(predicted FVC)]*100.
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Timepoint [8]
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Baseline, Week 144
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Secondary outcome [9]
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Change From Baseline in Percent Predicted FEV1 as Measured by Spirometry at Week 144
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Assessment method [9]
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FEV1 is a standard pulmonary function test. FEV1 was defined as the volume of air that can forcibly be blown out in 1 second, after full inspiration in the upright position, measured in liters. Percent predicted FEV1 (in %) = [(observed FEV1)/(predicted FEV1)]*100.
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Timepoint [9]
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Baseline, Week 144
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Secondary outcome [10]
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Change From Baseline in PEF as Measured by Spirometry at Week 144
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Assessment method [10]
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PEF was defined as the maximum airflow during a forced expiration beginning with the lungs fully inflated.
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Timepoint [10]
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Baseline, Week 144
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Secondary outcome [11]
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Change From Baseline in PCF as Measured by Spirometry at Week 144
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Assessment method [11]
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PCF measures an individual's maximum speed of expiration during cough.
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Timepoint [11]
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Baseline, Week 144
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Secondary outcome [12]
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Change From Baseline in PODCI Transfers/Basic Mobility Score at Week 144
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Assessment method [12]
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Changes in health-related quality of life (HRQL) were measured via the PODCI questionnaire that has been shown to correlate with disease progression and clinical outcome measures in DMD. PODCI includes a Global Functioning Scale and 5 core scales: Upper Extremity and Physical Function,Transfer/Basic Mobility, Sports/Physical Functioning, Pain/Comfort,and Happiness. The following PODCI domain was prespecified in the protocol for analysis:Transfers/Basic Mobility domain assesses difficulty experienced in performing routine motor activities in daily life. Each domain was scored from 0 to 100, with 0 representing a poor outcome/worse health, while 100 representing the highest level of functioning and least pain.
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Timepoint [12]
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Baseline, Week 144
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Secondary outcome [13]
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Number of Participants With Change From Baseline in Activities of Daily Living and Disease Status at Week 144, as Assessed by a Standardized Survey Administered by Site Personnel
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Assessment method [13]
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Changes in activities of daily living and disease symptoms were captured via a DMD-specific survey administered by Site personnel. At screening or baseline, the participant and/or parent/caregiver were asked to identify any activities of daily living (for example, ambulation, balance, personal hygiene/grooming, dressing and undressing, self-feeding, using the bathroom, handwriting, school performance, behavior or energy level) or symptoms that were affected by the participant's DMD. At post-baseline visit (Week 144), the same participant and/or parent/caregiver was asked to describe any changes from baseline in those activities of daily living/symptoms, within the following categories: physical functioning; general energy level; cognition/school function; emotional/social functioning; and sleep. Changes from baseline were reported on a 5-point Likert scale: 1 (much better), 2 (slightly better), 3 (unchanged), 4 (slightly worse), or 5 (much worse).
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Timepoint [13]
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Baseline, Week 144
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Secondary outcome [14]
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Ataluren Plasma Concentration
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Assessment method [14]
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Pre-dose ataluren plasma concentrations prior to morning ataluren administration at each clinic visit was assessed using a validated high performance liquid chromatography with tandem mass spectrometry (HPLC/MS-MS) method.
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Timepoint [14]
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Pre-dose at Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, and 144
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Secondary outcome [15]
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Change From Baseline in Systolic and Diastolic Blood Pressure at Week 144
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Assessment method [15]
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Blood pressure determination was performed with the participant in a sitting position after a 5-minute rest.
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Timepoint [15]
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Baseline, Week 144
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Secondary outcome [16]
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Change From Baseline in Pulse Rate at Week 144
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Assessment method [16]
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Pulse rate determination was performed with the participant in a sitting position after a 5-minute rest.
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Timepoint [16]
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Baseline, Week 144
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Secondary outcome [17]
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Change From Baseline in Body Temperature at Week 144
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Assessment method [17]
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Timepoint [17]
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Baseline, Week 144
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Eligibility
Key inclusion criteria
- Completion of study treatment in the previous Phase 3, double-blind study
(PTC124-GD-020-DMD).
- Evidence of signed and dated informed consent/assent document(s) indicating that the
participant (and/or his parent/legal guardian) has been informed of all pertinent
aspects of the trial. Note: If the study candidate is considered a child under local
regulation, a parent or legal guardian must provide written consent prior to
initiation of study screening procedures and the study candidate may be required to
provide written assent. The rules of the responsible Institutional Review
Board/Independent Ethic Committee (IRB/IEC) regarding whether 1 or both parents must
provide consent and the appropriate ages for obtaining consent and assent from the
participant should be followed.
- In participants who are sexually active, willingness to abstain from sexual
intercourse or employ a barrier or medical method of contraception during the period
of study drug administration and 6-week follow-up period.
- Willingness and ability to comply with scheduled visits, ataluren administration plan,
study procedures, laboratory tests, and study restrictions.
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Minimum age
7
Years
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Maximum age
15
Years
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Sex
Males
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Can healthy volunteers participate?
No
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Key exclusion criteria
- Known hypersensitivity to any of the ingredients or excipients of the study drug
(Litesse® UltraTM [refined polydextrose], polyethylene glycol 3350, Lutrol® micro F127
[poloxamer 407], mannitol 25C, crospovidone XL10, hydroxyethyl cellulose, vanilla,
Cab-O-Sil® M5P [colloidal silica], and magnesium stearate).
- Ongoing participation in any other therapeutic clinical trial.
- Prior or ongoing medical condition (for example, concomitant illness, psychiatric
condition, behavioral disorder, alcoholism, drug abuse), medical history, physical
findings, electrocardiogram (ECG) findings, or laboratory abnormality that, in the
investigator's opinion, could adversely affect the safety of the participant, makes it
unlikely that the course of treatment or follow-up would be completed, or could impair
the assessment of study results.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
N/A
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Terminated
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
20/03/2014
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
12/06/2018
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Sample size
Target
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Accrual to date
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Final
219
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Recruitment in Australia
Recruitment state(s)
NSW,VIC
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Recruitment hospital [1]
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The Children's Hospital at Westmead - Westmead
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Recruitment hospital [2]
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The Royal Children's Hospital - Parkville
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Recruitment postcode(s) [1]
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2145 - Westmead
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Recruitment postcode(s) [2]
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3052 - Parkville
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Recruitment outside Australia
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United States of America
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California
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United States of America
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Colorado
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United States of America
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Florida
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United States of America
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Illinois
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United States of America
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Iowa
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United States of America
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Kansas
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United States of America
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Massachusetts
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United States of America
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Minnesota
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United States of America
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Missouri
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United States of America
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New York
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United States of America
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Ohio
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United States of America
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Oregon
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United States of America
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Pennsylvania
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0
United States of America
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Texas
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United States of America
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Utah
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United States of America
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Washington
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Belgium
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Leuven
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0
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Brazil
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Rio de Janeiro
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Brazil
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São Paulo
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Bulgaria
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Sofia
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Canada
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Alberta
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Canada
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British Columbia
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Canada
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Ontario
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Chile
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State/province [24]
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Región Metropolitana
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Chile
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Santiago
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Czechia
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Brno
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Czechia
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Praha
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France
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Marseille
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France
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Nantes Cedex 1
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France
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Paris Cedex 13
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France
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Paris
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Germany
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Essen
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Germany
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Freiburg
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Germany
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Munchen
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Israel
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Jerusalem
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Italy
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Milano
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Italy
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Rome
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Italy
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Sicily
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Korea, Republic of
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Seoul
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Poland
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Warsaw
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Spain
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Barcelona
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Spain
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Valencia
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Sweden
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Goteburg
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Sweden
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Stockholm
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Switzerland
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Lausanne
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Turkey
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Ankara
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Turkey
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Talas/Kayseri
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United Kingdom
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London
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United Kingdom
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Manchester
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United Kingdom
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Newcastle upon Tyne
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
PTC Therapeutics
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Address
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Country
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Ethics approval
Ethics application status
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Summary
Brief summary
The primary objective of this study is to obtain long term safety data of ataluren in male
participants with nonsense mutation dystrophinopathy (who participated and completed a
previous Phase 3 study of ataluren [PTC124-GD-020-DMD {NCT01826487}]) to augment the overall
safety database. Screening and baseline procedures are structured to avoid a gap in treatment
between the double-blind study (PTC124-GD-020-DMD) and this extension study.
This study may be further extended by amendment until either ataluren becomes commercially
available or the clinical development of ataluren in duchenne muscular dystrophy (DMD) is
discontinued.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT02090959
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Francesco Bibbiani, M.D.
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Address
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PTC Therapeutics
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Address
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Country
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Phone
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Fax
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Email
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT02090959
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