Trial · NCT01009294
Study of Ataluren (PTC124) in Nonambulatory Participants With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD)
NCT01009294, Phase 2, Terminated, PTC Therapeutics; results first posted 2020-07-29
3 sourcestrials-2026-11-12-0.4.0
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ConductScience Library. Study of Ataluren (PTC124) in Nonambulatory Participants With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD). Trials, record NCT01009294. Release trials-2026-11-12-0.4.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01009294
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study of Ataluren (PTC124) in Nonambulatory Participants With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD)
- NCT number
- NCT01009294
- Status
- Terminated
- Phases
- Phase 2
- Enrollment
- 6 participants, actual
- Lead sponsor
- PTC Therapeutics
- Conditions
- Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
- Interventions
- Ataluren, Chronic Corticosteroid Therapy
- First posted
- 2009-11-06
- Last updated
- 2020-07-29
- Other registries' IDs
- eudract:2009-013169-24
- Results first posted
- 2020-07-29
Source ClinicalTrials.gov
Registered outcomes18
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With Treatment Emergent Adverse Events (TEAEs) | Baseline up to Day 50 | A TEAE is any untoward medical occurrence or undesirable event(s) experienced in a participant that begins or worsens following administration of the study drug or study treatment, whether or not considered related to the treatment by the 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 were classified according to National Cancer Institute Common Terminology Criteria for Adverse Events version 3.0 (CTCAE) and coded using the Medical Dictionary for Regulatory Activities (MedDRA). A summary of serious and all other non-serious adverse events, regardless of causality, is located in the Reported Adverse Events module. |
| Secondary | Time to Complete Upper Limb Function Tasks as Measured by the Jebsen Test | Baseline and Week 6 | 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. |
| Secondary | Upper Limb Function as Measured by the Brooke Upper Extremity Functional Rating Scale | Baseline and Week 6 | 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. |
| Secondary | Participant Activities of Daily Living as Assessed Using the Egen Klassifikation (EK) Scale | Baseline and Week 6 | 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). The interviewer observed the participant and assigned the final score for the tasks that could be observed in the clinic. |
| Secondary | Shoulder, Elbow, and Wrist Passive and Active Range of Motion as Measured by Goniometry | Baseline and Week 6 | 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. Greater degree of motion indicates better response. |
| Secondary | Force Exerted During Elbow Flexion and Extension, Shoulder Abduction, Hand Grip, Key Grip, and Finger Pinch as Assessed by Upper Extremity Myometry | Baseline and Week 6 | Upper extremity myometry was performed using a hand-held dynamometer following standardized procedures. The measured strength (peak force) was reported in Newtons. There are 0.22 pounds (lbs) in 1 Newton and approximately 10 Newton (9.80665 Newton) in 1 kilogram (kg). The threshold/range of the hand-held dynamometer is 0 to 500 Newtons. 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. |
| Secondary | Time to Complete Hand Fine Motor Coordination and Dexterity Tasks as Measured by 9-Hole Peg Test (9HPT) | Baseline and Week 6 | 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. |
| Secondary | Forced Vital Capacity (FVC) as Measured by Spirometry | Baseline and Week 6 | Pulmonary function was assessed as FVC in participants by spirometry using a study-specific spirometer. Multiple tests were conducted, if needed. |
| Secondary | Systolic and Diastolic Function as Measured by Echocardiography With Tissue Doppler | Week 24 and Week 48 | 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. |
| Secondary | Heart Rate as Assessed by Radial Pulse | Baseline and Week 6 | 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. |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 registered outcomesOutcomes18
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants With Treatment Emergent Adverse Events (TEAEs) | Baseline up to Day 50 | All enrolled participants who received at least 1 dose of study drug. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Time to Complete Upper Limb Function Tasks as Measured by the Jebsen Test | Baseline and Week 6 | All enrolled participants who received at least 1 dose of study drug and with evaluable upper limb function tasks data. | seconds | MEDIAN |
| SECONDARY | Upper Limb Function as Measured by the Brooke Upper Extremity Functional Rating Scale | Baseline and Week 6 | All enrolled participants who received at least 1 dose of study drug and with evaluable upper limb function tasks data. | score on a scale | MEDIAN |
| SECONDARY | Participant Activities of Daily Living as Assessed Using the Egen Klassifikation (EK) Scale | Baseline and Week 6 | All enrolled participants who received at least 1 dose of study drug and with evaluable EK Scale data. | score on a scale | MEDIAN |
| SECONDARY | Shoulder, Elbow, and Wrist Passive and Active Range of Motion as Measured by Goniometry | Baseline and Week 6 | All enrolled participants who received at least 1 dose of study drug and with evaluable range of motion data. | degrees | MEDIAN |
| SECONDARY | Force Exerted During Elbow Flexion and Extension, Shoulder Abduction, Hand Grip, Key Grip, and Finger Pinch as Assessed by Upper Extremity Myometry | Baseline and Week 6 | All enrolled participants who received at least 1 dose of study drug and with evaluable upper extremity myometry data. | Newton | MEDIAN |
| SECONDARY | Time to Complete Hand Fine Motor Coordination and Dexterity Tasks as Measured by 9-Hole Peg Test (9HPT) | Baseline and Week 6 | All enrolled participants who received at least 1 dose of study drug and with evaluable 9HPT data. | seconds | MEDIAN |
| SECONDARY | Forced Vital Capacity (FVC) as Measured by Spirometry | Baseline and Week 6 | All enrolled participants who received at least 1 dose of study drug and with evaluable spirometry data. Data was not collected at Week 6 as no participants were evaluable at this timepoint. | liters | MEDIAN |
| SECONDARY | Systolic and Diastolic Function as Measured by Echocardiography With Tissue Doppler | Week 24 and Week 48 | All enrolled participants who received at least 1 dose of study drug and with evaluable echocardiography data. Since the study was terminated early, echocardiography data were not collected after the start of study drug administration. | ||
| SECONDARY | Heart Rate as Assessed by Radial Pulse | Baseline and Week 6 | All enrolled participants who received at least 1 dose of study drug and with evaluable heart rate data. | beats per minute | MEDIAN |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 outcomesResults by group167
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) | Ataluren | TEAEs | 1 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) | Ataluren | Treatment Emergent SAEs | 0 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) | Ataluren | AEs Related to Study Treatment | 0 | Participants | |
| Time to Complete Upper Limb Function Tasks as Measured by the Jebsen Test | Ataluren | Lifting Large Heavy Objects, DOM Hand, Baseline | 11 | Full Range 5 to 120 | seconds |
| Time to Complete Upper Limb Function Tasks as Measured by the Jebsen Test | Ataluren | Lifting Large Heavy Objects, DOM Hand, Week 6 | 10 | Full Range 9 to 11 | seconds |
| Time to Complete Upper Limb Function Tasks as Measured by the Jebsen Test | Ataluren | Lifting Large Heavy Objects, Non-DOM Hand Baseline | 11 | Full Range 5 to 120 | seconds |
| Time to Complete Upper Limb Function Tasks as Measured by the Jebsen Test | Ataluren | Lifting Large Heavy Objects, Non-DOM Hand, Week 6 | 11 | Full Range 7 to 14 | seconds |
| Time to Complete Upper Limb Function Tasks as Measured by the Jebsen Test | Ataluren | Lifting Large Light Objects, DOM Hand, Baseline | 9 | Full Range 4 to 120 | seconds |
| Time to Complete Upper Limb Function Tasks as Measured by the Jebsen Test | Ataluren | Lifting Large Light Objects, DOM Hand, Week 6 | 15 | Full Range 7 to 22 | seconds |
| Time to Complete Upper Limb Function Tasks as Measured by the Jebsen Test | Ataluren | Lifting Large Light Objects, Non-DOM Hand Baseline | 7 | Full Range 3 to 120 | seconds |
1 to 10 of 167Source ClinicalTrials.gov
See all 167 results by groupParticipant flow4
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Ataluren | 6 | |
| Overall Study | Received at Least 1 Dose of Study Drug | Ataluren | 6 | |
| Overall Study | COMPLETED | Ataluren | 0 | |
| Overall Study | NOT COMPLETED | Ataluren | 6 |
Source ClinicalTrials.gov
Adverse events3
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Diarrhoea | Gastrointestinal disorders | Ataluren | 1 | 6 | |
| other | Nasopharyngitis | Infections and infestations | Ataluren | 1 | 6 | |
| other | Dysuria | Renal and urinary disorders | Ataluren | 1 | 6 |
Source ClinicalTrials.gov
Adverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Ataluren | ||
| Total, other adverse events | Ataluren | 1 | 6 |
| Total, serious adverse events | Ataluren | 0 | 6 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests1
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
| Test | Named as |
|---|---|
| Digit Span Test | Secondary outcome |
Source ConductCognition
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT01009294
GET https://conductscience.org/api/trials/v1/NCT01009294/results?page=2