Clinical Trial Results:
A Phase 2a Study of Ataluren (PTC124) in Nonambulatory Patients with Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy
Summary
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EudraCT number |
2009-013169-24 |
Trial protocol |
GB |
Global end of trial date |
09 Jul 2010
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Results information
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Results version number |
v1(current) |
This version publication date |
11 Jul 2020
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First version publication date |
11 Jul 2020
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Other versions |
Trial Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
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Trial identification
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Sponsor protocol code |
PTC124-GD-008-DMD
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
NCT01009294 | ||
WHO universal trial number (UTN) |
- | ||
Sponsors
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Sponsor organisation name |
PTC Therapeutics, Inc.
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Sponsor organisation address |
100 Corporate Court, South Plainfield, United States, 07080
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Public contact |
Medical Information, PTC Therapeutics, Inc., +011 44 1-866-562-4620, medinfo@ptcbio.com
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Scientific contact |
Medical Information, PTC Therapeutics International Limited, +353 19068700, medinfo@ptcbio.com
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Paediatric regulatory details
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Is trial part of an agreed paediatric investigation plan (PIP) |
Yes
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EMA paediatric investigation plan number(s) |
EMEA-000115-PIP01-09 | ||
Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial? |
Yes
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Results analysis stage
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Analysis stage |
Final
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Date of interim/final analysis |
23 Mar 2010
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Is this the analysis of the primary completion data? |
Yes
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Primary completion date |
23 Mar 2010
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Global end of trial reached? |
Yes
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Global end of trial date |
09 Jul 2010
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Was the trial ended prematurely? |
Yes
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General information about the trial
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Main objective of the trial |
The primary objective of this study was to assess the safety and torlerability of ataluren in nonambulatory participants with Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDBMD).
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Protection of trial subjects |
The trial was conducted in accordance with Declaration of Helsinki (revised version of Edinburgh, Scotland, 2000), FDA GCP regulations, and the International Conference on Harmonisation (ICH) GCP guidance documents.
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Background therapy |
- | ||
Evidence for comparator |
- | ||
Actual start date of recruitment |
13 Jan 2010
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Long term follow-up planned |
Yes
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Long term follow-up rationale |
Safety | ||
Long term follow-up duration |
5 Years | ||
Independent data monitoring committee (IDMC) involvement? |
Yes
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Population of trial subjects
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Number of subjects enrolled per country |
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Country: Number of subjects enrolled |
United States: 6
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Worldwide total number of subjects |
6
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EEA total number of subjects |
0
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Number of subjects enrolled per age group |
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In utero |
0
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Preterm newborn - gestational age < 37 wk |
0
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Newborns (0-27 days) |
0
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Infants and toddlers (28 days-23 months) |
0
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Children (2-11 years) |
0
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Adolescents (12-17 years) |
3
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Adults (18-64 years) |
3
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From 65 to 84 years |
0
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85 years and over |
0
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Recruitment
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Recruitment details |
A total of 11 participants with nmDBMD and were nonambulatory signed the informed consent form and were screened for eligibility. Six of these participants were enrolled at 2 sites. Three of the participants were receiving chronic corticosteroid therapy and a stable corticosteroid regimen was to be maintained during the study. | ||||||||||||
Pre-assignment
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Screening details |
When the Sponsor terminated the study, the participants were told to discontinue ataluren treatment, and to return all unused ataluren to the site for return to the Sponsor. Because of difficulty of traveling to the clinic for these nonambulatory participants, the planned final visits were not performed. | ||||||||||||
Period 1
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Period 1 title |
Overall Study (overall period)
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Is this the baseline period? |
Yes | ||||||||||||
Allocation method |
Not applicable
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Blinding used |
Not blinded | ||||||||||||
Arms
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Arm title
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Ataluren | ||||||||||||
Arm description |
Ataluren was provided as a vanilla-flavored powder to be mixed with water, apple juice, or milk. Study drug dosing was based on milligrams of drug per kilogram of body weight. The dose level for ataluren was 20 milligrams/kilograms (mg/kg) in the morning, 20 mg/kg at midday, and 40 mg/kg in the evening. Administration within 30 minutes after a meal was recommended. Study drug was taken for up to 50 days. | ||||||||||||
Arm type |
Experimental | ||||||||||||
Investigational medicinal product name |
Ataluren
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Investigational medicinal product code |
PTC124
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Other name |
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Pharmaceutical forms |
Powder for oral suspension
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Routes of administration |
Oral use
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Dosage and administration details |
Ataluren was administered as per the dose and schedule specified in the respective arms.
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Baseline characteristics reporting groups
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Reporting group title |
Ataluren
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Reporting group description |
Ataluren was provided as a vanilla-flavored powder to be mixed with water, apple juice, or milk. Study drug dosing was based on milligrams of drug per kilogram of body weight. The dose level for ataluren was 20 milligrams/kilograms (mg/kg) in the morning, 20 mg/kg at midday, and 40 mg/kg in the evening. Administration within 30 minutes after a meal was recommended. Study drug was taken for up to 50 days. | |||||||||||||||||||||||||||||||||||||||
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End points reporting groups
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Reporting group title |
Ataluren
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Reporting group description |
Ataluren was provided as a vanilla-flavored powder to be mixed with water, apple juice, or milk. Study drug dosing was based on milligrams of drug per kilogram of body weight. The dose level for ataluren was 20 milligrams/kilograms (mg/kg) in the morning, 20 mg/kg at midday, and 40 mg/kg in the evening. Administration within 30 minutes after a meal was recommended. Study drug was taken for up to 50 days. |
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End point title |
Number of Participants With Treatment Emergent Adverse Events (TEAEs) [1] | ||||||||||||
End point description |
A TEAE is any untoward medical occurrence or undesirable event(s) experienced in a participant that begins or worsens following administration of study drug, whether or not considered related to study drug by Investigator. A serious adverse event (SAE) was an adverse event (AE) resulting in any of the following outcomes or deemed significant for any other reason, death, initial or prolonged inpatient hospitalization, life-threatening experience (immediate risk of dying), or persistent or significant disability/incapacity not related to nmDBMD. AEs included both SAEs and non-serious AEs. AEs classified according to National Cancer Institute Common Terminology Criteria for Adverse Events version 3.0 and coded using the Medical Dictionary for Regulatory Activities (MedDRA). A summary of serious and all other non-serious AEs, regardless of causality, is located in the Reported Adverse Events module. Population included all enrolled participants who received at least 1 dose of study drug.
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End point type |
Primary
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End point timeframe |
Baseline up to Day 50
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Notes [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Justification: Statistical analyses not applicable for this endpoint. |
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No statistical analyses for this end point |
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End point title |
Time to Complete Upper Limb Function Tasks as Measured by the Jebsen Test | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
End point description |
Arm and hand function were assessed using the Jebsen test, a standardized clinical evaluation of tasks important to daily living. The test comprises of unilateral subtests performed with each hand (the dominant [DOM] hand and the non-DOM hand): moving and stacking light (250 grams) and heavy (500 grams) objects; picking up small, commonly encountered objects; stacking checkers; simulated feeding; simulated page turning; and writing. Participant performance of each task was timed. Longer time to complete the test indicates worse hand function. Population included all enrolled participants who received at least 1 dose of study drug and with evaluable upper limb function tasks data.
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End point type |
Secondary
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End point timeframe |
Baseline and Week 6
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No statistical analyses for this end point |
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End point title |
Upper Limb Function as Measured by the Brooke Upper Extremity Functional Rating Scale | ||||||||||||
End point description |
Upper extremity function was assessed using the Brooke Upper Extremity Functional Rating Scale, following standardized procedures. The Brooke Upper Extremity Functional Rating Scale graded arm and shoulder function from 1 to 6, with higher values indicating less function. A rating of "1" was used when the participant was able to abduct his arms in a full circle until they touch above his head, whereas a rating of "6" was used when the participant was unable to raise his hands to his mouth and had no useful function of hands. Population included all enrolled participants who received at least 1 dose of study drug and with evaluable upper limb function tasks data.
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End point type |
Secondary
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End point timeframe |
Baseline and Week 6
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No statistical analyses for this end point |
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End point title |
Participant Activities of Daily Living as Assessed Using the Egen Klassifikation (EK) Scale | ||||||||||||||||||||||||||||||||||||||||||||||||
End point description |
Activities of daily living after loss of ambulation were measured using the EK scale. The EK scale is an ordinal scale ranging from 0 to 30 points where 0 represents the highest level of independent function and 30 the lowest. The scale consists of 10 categories (each scored 0 to 3), involving different functional domains including 1) ability to use wheelchair, 2) ability to transfer from wheelchair, 3) ability to stand, 4) ability to balance in the wheelchair, 5) ability to move arms, 6) ability to use hands and arms when eating, 7) ability to turn in bed, 8) ability to cough, 9) ability to speak, and 10) physical well-being. The administration of the EK scale consisted of an interview of the participant to capture how he performs the tasks of daily life (as described by Categories 1 to 9) and how he perceives his wellbeing (as described by Category 10). Population included all enrolled participants who received at least 1 dose of study drug and with evaluable EK Scale data.
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End point type |
Secondary
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End point timeframe |
Baseline and Week 6
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No statistical analyses for this end point |
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End point title |
Shoulder, Elbow, and Wrist Passive and Active Range of Motion as Measured by Goniometry | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
End point description |
Goniometry was performed to test active and passive range-of motion (RoM) of the left (L) and right (R) shoulder, elbow, and wrist following standardized procedures. The observed angle for passive and active motion for each joint was measured in degrees (0-180). Greater degree of motion indicates better response. Population included all enrolled participants who received at least 1 dose of study drug and with evaluable range of motion data.
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End point type |
Secondary
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End point timeframe |
Baseline and Week 6
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No statistical analyses for this end point |
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End point title |
Force Exerted During Elbow Flexion and Extension, Shoulder Abduction, Hand Grip, Key Grip, and Finger Pinch as Assessed by Myometry | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||
End point description |
Upper extremity myometry was performed using a hand-held dynamometer following standardized procedures. Evaluators judged the strength of each muscle using a scoring system. Bilateral assessments were done, and 3 measurements were recorded from each muscle group on each side, when possible. When the measurements were done in duplicate or triplicate, the best value was used. Greater value indicates better measurement. Population included all enrolled participants who received at least 1 dose of study drug and with evaluable myometry data.
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End point type |
Secondary
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End point timeframe |
Baseline and Week 6
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No statistical analyses for this end point |
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End point title |
Time to Complete Hand Fine Motor Coordination and Dexterity Tasks as Measured by 9-Hole Peg Test (9HPT) | ||||||||||||||||
End point description |
Hand fine motor coordination and dexterity were assessed using the 9HPT using standardized procedures. The 9HPT is a unilateral test in which 9 pegs were placed in a board and then removed with the dominate and non-dominate hand within a 5-minute time limit. The amount of time required to put the pegs in the holes and remove them again with each hand was recorded. Each test was conducted twice per hand. Longer time to complete the test indicates worse hand fine motor coordination and dexterity. Population included all enrolled participants who received at least 1 dose of study drug and with evaluable 9HPT data.
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End point type |
Secondary
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End point timeframe |
Baseline and Week 6
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No statistical analyses for this end point |
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End point title |
Forced Vital Capacity (FVC) as Measured by Spirometry | ||||||||||||
End point description |
Pulmonary function was assessed as FVC in participants by spirometry using a study-specific spirometer. Multiple tests were conducted, if needed. Population included all enrolled participants who received at least 1 dose of study drug and with evaluable spirometry data.
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End point type |
Secondary
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End point timeframe |
Baseline and Week 6
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No statistical analyses for this end point |
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End point title |
Systolic and Diastolic Function as Measured by Echocardiography with Tissue Doppler | ||||||||||||
End point description |
Cardiac function was assessed by echocardiography, which included standard parameters (for example, ejection fraction, left ventricle diastolic and systolic dimensions), as well as parameters integrating Doppler flow analysis with imaging to evaluate perturbations in wall motion. A standardized data collection process harmonized data from all participating institutions and allowed for centralized review. Since the study was terminated early, echocardiography data were not collected after the start of study drug administration. Population included all enrolled participants who received at least 1 dose of study drug and with evaluable echocardiography data.
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End point type |
Secondary
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End point timeframe |
Week 24 and Week 48
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Notes [2] - Study terminated early and echocardiography data were not collected after start of study drug. |
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No statistical analyses for this end point |
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End point title |
Heart Rate as Assessed by Radial Pulse | ||||||||||||
End point description |
Heart rate was measured with the radial pulse. Following the Jebsen test, the participant rested for 5 minutes in a sitting position, and the heart rate for the last minute of this rest period was collected as the resting heart rate. Population included all enrolled participants who received at least 1 dose of study drug and with evaluable heart rate data.
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End point type |
Secondary
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End point timeframe |
Baseline and Week 6
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No statistical analyses for this end point |
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End point title |
Verbal Memory and Attention as Assessed by the Digit Span Task | ||||||||||||||||||||||
End point description |
A series of digits (0-9) were presented to the participant in an auditory format only. The task had 2 parts: in the Forward Condition, the participant was requested to repeat back the digits in the order they were presented, and in the Backward Condition, he was requested to reverse the order of presentation. Population included all enrolled participants who received at least 1 dose of study drug and with evaluable verbal memory and attention data. The test was repeated until the participant had 0 correct responses, which was up to 7 times for the Forward Condition and up to 5 times for the Backward Condition at Baseline and at Week 6.
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End point type |
Secondary
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End point timeframe |
Baseline and Week 6
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No statistical analyses for this end point |
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End point title |
HRQL as Measured by the PedsQL Inventory Generic Core Scale | ||||||||||||||||||||||||
End point description |
Health-related quality of life (HRQL) was measured by the Pediatric Quality of Life Inventory (PedsQL) Inventory Generic Core Scale. The generic core module comprised of 23 questions evaluating physical, emotional, social, and school functioning. Examples of items in each of the generic core module scales included: “It is hard for me to run”; “I feel sad or blue”; “I cannot do things that other kids my age can do;” and “It is hard to pay attention in class.” Each of the generic core module items was scored on a 5-point response scale from 0 (never a problem) to 4 (almost always a problem). The appropriate age-specific version was completed. PedsQL Inventory Generic Core Scale data at Week 6 is presented. Population included all enrolled participants who received at least 1 dose of study drug and with evaluable PedsQL Inventory Generic Core Scale data at Week 6.
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End point type |
Secondary
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End point timeframe |
Week 6
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No statistical analyses for this end point |
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End point title |
HRQL as Measured by the PedsQL Multidimensional Fatigue Scale | ||||||||||||||||||||
End point description |
Health-related quality of life (HRQL) was measured by the Pediatric Quality of Life Inventory (PedsQL) Multidimensional Fatigue Scale. The fatigue-specific module comprised of 18 questions evaluating general fatigue, sleep/rest fatigue, and cognitive fatigue. Fatigue-specific module obtains information relating to items such as: “I feel too tired to do things that I like to do”; “I spend a lot of time in bed”; and “I have trouble remembering more than one thing at a time.” Each of the fatigue-specific module items was scored on a 5-point response scale from 0 (never a problem) to 4 (almost always a problem). The appropriate age-specific version was completed. PedsQL Multidimensional Fatigue Scale data at Week 6 is presented. Population included all enrolled participants who received at least 1 dose of study drug and with evaluable PedsQL Multidimensional Fatigue Scale data at Week 6.
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End point type |
Secondary
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End point timeframe |
Week 6
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No statistical analyses for this end point |
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End point title |
HRQL as Measured by the INQoL | ||||||||||||
End point description |
HRQL was measured by the Individualized Neuromuscular Quality of Life Questionnaire (INQoL). The INQoL consisted of 45 questions within 10 sections. Four of the sections evaluate key muscle disease symptoms (that is, weakness, locking [myotonia], pain, and fatigue), 5 sections evaluate the degree and importance of the impact of muscle disease on particular areas of life, and 1 section asks about the positive and negative effects of treatment. A higher score indicates greater symptom impact or worse HRQL, with a range of 0-7. Since the study was terminated early, INQoL data were not collected. Population included all enrolled participants who received at least 1 dose of study drug and with evaluable INQoL data.
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End point type |
Secondary
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End point timeframe |
Week 24 and Week 48
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Notes [3] - Study terminated early and INQoL data were not collected. |
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No statistical analyses for this end point |
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End point title |
Muscle Fragility as Determined by Serum Creatine Kinase (CK) Levels | ||||||||||||||||||||
End point description |
Blood samples collected for chemistry assays were used to quantify serum CK concentrations. Serum CK was assessed as a potential biomarker for muscle fragility, with a reduction in serum CK considered to be a positive outcome. The reference range was based on the age of the participant. Population included all enrolled participants who received at least 1 dose of study drug and with evaluable CK data.
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End point type |
Secondary
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End point timeframe |
Baseline and Week 6
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No statistical analyses for this end point |
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End point title |
Gastrocnemius Muscle Dystrophin Expression as Determined by Immunofluoresence or by Western Blotting Techniques | ||||||||
End point description |
The gastrocnemius muscle was to be biopsied from 1 leg to assess for the production of dystrophin at Week 36. The production of dystrophin was to be measured by immunofluorescene staining of the sarcolemmal membrane or by Western blotting techniques with an antibody to the C-terminal portion of the dystrophin protein (excluding revertant fibers). Since the study was terminated early, gastrocnemius muscle dystrophin expression data were not collected after the start of study drug administration. Population included all enrolled participants who received at least 1 dose of study drug and with evaluable dystrophin production data.
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End point type |
Secondary
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End point timeframe |
Week 36
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Notes [4] - Study terminated early. Data for this outcome measure were not collected after start of study drug. |
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No statistical analyses for this end point |
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End point title |
Study Drug Compliance | ||||||||||||||||||
End point description |
Study drug compliance was assessed by the participant daily diary and quantification of used and unused study drug. Compliance was assessed in terms of the amount of drug actually taken relative to the amount that should have been taken during the study. Population included all enrolled participants who received at least 1 dose of study drug and with evaluable study drug compliance data.
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End point type |
Secondary
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End point timeframe |
Baseline to Day 50
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No statistical analyses for this end point |
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End point title |
Pharmacokinetics: Ataluren Plasma Exposure | ||||||||
End point description |
Blood for ataluren concentrations over a 24-hour period was to be collected on Days 2 and 3 of Week 6. Analysis of the blood samples was to be conducted using a validated high performance liquid chromatography with tandem mass spectrometry (HPLC-MS/MS) method. Since the study was terminated early, steady state data were not collected at Week 6. Population included all enrolled participants who received at least 1 dose of study drug and with evaluable plasma data.
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End point type |
Secondary
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End point timeframe |
0, 2, 3, 6, 8, 9, 12, 14, 15, and 24 hours after the morning dose
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Notes [5] - Since the study was terminated early, steady state data were not collected at Week 6. |
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No statistical analyses for this end point |
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Adverse events information
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Timeframe for reporting adverse events |
Baseline up to Day 50
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Assessment type |
Systematic | ||||||||||||||||||||||||||||||
Dictionary used for adverse event reporting
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Dictionary name |
11.1 | ||||||||||||||||||||||||||||||
Dictionary version |
11.1
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Reporting groups
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Reporting group title |
Ataluren
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Reporting group description |
Ataluren was provided as a vanilla-flavored powder to be mixed with water, apple juice, or milk. Study drug dosing was based on milligrams of drug per kilogram of body weight. The dose level for ataluren was 20 mg/kg in the morning, 20 mg/kg at midday, and 40 mg/kg in the evening. Administration within 30 minutes after a meal was recommended. Study drug was taken for up to 50 days. | ||||||||||||||||||||||||||||||
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Frequency threshold for reporting non-serious adverse events: 0% | |||||||||||||||||||||||||||||||
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Substantial protocol amendments (globally) |
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Were there any global substantial amendments to the protocol? No | |||
Interruptions (globally) |
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Were there any global interruptions to the trial? No | |||
Limitations and caveats |
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Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data. | |||
None reported |