Trial · NCT03179631
Registered outcomes39
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
21 to 39 of 39
Download this page as JSON| List | Outcome | Time frame | Description |
|---|---|---|---|
| Secondary | DB Period: Time to Loss of Stair-Climbing - mITT Population | Baseline up to approximately Week 72 (Due to COVID, many participants attended the protocol-defined Week 72 visit late) | Time to loss of stair-climbing was defined as persistent inability to perform the 4-stair climb test within 30 seconds at any post-baseline visit and for all remaining visits. Due to COVID, there were many participants who participated in the protocol-defined Week 72 visit late. Due to this reason, the calculated median and 95% CI for the time to event for the Ataluren arm in the DB period were greater than Week 72. |
| Secondary | DB Period: Time to Loss of Stair-Climbing - ITT Population | Baseline up to approximately Week 72 (Due to COVID, many participants attended the protocol-defined Week 72 visit late) | Time to loss of stair-climbing was defined as persistent inability to perform the 4-stair climb test within 30 seconds at any post-baseline visit and for all remaining visits. Due to COVID, there were many participants who participated in the protocol-defined Week 72 visit late. Due to this reason, the calculated median and 95% CI for the time to event for the Ataluren arm in the DB period were greater than Week 72. |
| Secondary | DB Period: Time to Loss of Stair-Descending - mITT Population | Baseline up to approximately Week 72 (Due to COVID, many participants attended the protocol-defined Week 72 visit late) | Time to loss of stair-descending was defined as persistent inability to perform the 4-stair descend test within 30 seconds at any post-baseline visit and for all remaining visits. Due to COVID, there were many participants who participated in the protocol-defined Week 72 visit late. Due to this reason, the calculated median and 95% CI for the time to event for the Ataluren arm in the DB period were greater than Week 72. |
| Secondary | DB Period: Time to Loss of Stair-Descending - ITT Population | Baseline up to approximately Week 72 (Due to COVID, many participants attended the protocol-defined Week 72 visit late) | Time to loss of stair-descending was defined as persistent inability to perform the 4-stair descend test within 30 seconds at any post-baseline visit and for all remaining visits. Due to COVID, there were many participants who participated in the protocol-defined Week 72 visit late. Due to this reason, the calculated median and 95% CI for the time to event for the Ataluren arm in the DB period were greater than Week 72. |
| Secondary | DB Period: Number of Participants With Function Loss of NSAA Items at Week 72 - mITT Population | Week 72 | Function loss was defined as a drop of score from 1 or 2 at baseline to a score of 0 at the specified post-baseline of NSAA items. The missing assessments at post baseline visits were imputed using last observation carried forward (LOCF). |
| Secondary | DB Period: Number of Participants With Function Loss of NSAA Items at Week 72 - ITT Population | Week 72 | Function loss was defined as a drop of score from 1 or 2 at baseline to a score of 0 at the specified post-baseline of NSAA items. The missing assessments at post baseline visits were imputed using LOCF. |
| Secondary | DB Period: Number of Participants With Treatment-emergent Adverse Events (TEAEs) | Baseline up to Week 76 | An adverse event (AE) was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse event (SAE) was an 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); persistent or significant disability/incapacity; congenital anomaly. AEs included both SAEs and non-serious AEs. A TEAE was defined as an AE that occurred or worsened on or after the first dose of study drug and up to 4 weeks after the last dose of double-blind study drug and prior to the first dose of open-label treatment. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE section'. |
| Secondary | Overall Treatment Period: Change From Baseline in 6MWD at Week 144 | Baseline, Week 144 | The 6MWD test is a non-encouraged test performed in a 30 meters long flat corridor, where the participant is 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. Participants were not permitted to use assistive devices (walker, long leg braces, or short leg braces) during the 6MWD test. Participants with confirmed loss of ambulation at a particular visit were assigned a 6MWD result of 0. Baseline was defined as the maximum measurement of valid Day 1 and Day 2 6MWD values. LS mean and SE was calculated using the MMRM. |
| Secondary | Overall Treatment Period: Composite of Average Change From Baseline in TFTs at Week 144 | Baseline, Week 144 | The composite TFT was defined as the average in times to run/walk 10 meters, climb 4 stairs, and descend 4 stairs. Participants who could not perform a TFT within 30 seconds, including those who loss of ambulation or the TFT was above 30 seconds, a value of 30 seconds was used. Baseline was defined as the last observed measurement (average in times to run/walk 10 meters, climb 4 stairs, and descend 4 stairs) on or prior to the first dose of study drug. LS mean and SE was calculated using the MMRM. |
| Secondary | Overall Treatment Period: Change From Baseline in Time to Run/Walk 10 Meters at Week 144 | Baseline, Week 144 | During the test for walking/running 10 meters, the method of walk/run used by the participant was categorized as follows: 1. Unable to walk independently; 2. Unable to walk independently but can walk with support from a person or with assistive device (full leg calipers \[knee-ankle-foot orthoses \] or walker); 3. Highly adapted gait, wide-based lordotic gait, cannot increase walking speed; 4. Moderately adapted gait, can pick up speed but cannot run; 5. Able to pick up speed but runs with a double stance phase (that is, cannot achieve both feet off the ground); 6. Runs and gets both feet off the ground (with no double stance phase). Participants who could not perform a TFT within 30 seconds, including those who loss of ambulation or the TFT was above 30 seconds, a value of 30 seconds was used. Baseline was defined as the last observed measurement on or prior to the first dose of study drug. LS mean and SE was calculated using the MMRM. |
| Secondary | Overall Treatment Period: Change From Baseline in Time to Climb 4 Stairs at Week 144 | Baseline, Week 144 | During the test for stair-climbing, the method of climbing used by the participant was categorized as follows: 1. Unable to up climb 4 standard stairs; 2. Climbs 4 standard stairs "marking time" (climbs one foot at a time, with both feet on a step before moving to next step), using both arms on one or both handrails; 3. Climbs 4 standard stairs "marking time", using one arm on one handrail; 4. Climbs 4 standard stairs "marking time", not needing handrail; 5. Climbs 4 standard stairs alternating feet, needs handrail for support; 6. Climbs 4 standard stairs alternating feet, not needing handrail support. Participants who could not perform a TFT within 30 seconds, including those who loss of ambulation or the TFT was above 30 seconds, a value of 30 seconds was used. Baseline was defined as the last observed measurement on or prior to the first dose of study drug. LS mean and SE was calculated using the MMRM. |
| Secondary | Overall Treatment Period: Change From Baseline in Time to Descend 4 Stairs at Week 144 | Baseline, Week 144 | During the test for stair-descending, the method of descending used by the participant was categorized as follows: 1. Unable to descend 4 standard stairs; 2. Descends 4 standard stairs "marking time" (climbs one foot at a time, with both feet on a step before moving to next step), using both arms on one or both handrails; 3. Descends 4 standard stairs "marking time", using one arm on one handrail; 4. Descends 4 standard stairs "marking time", not needing handrail; 5. Descends 4 standard stairs alternating feet, needs handrail for support; 6. Descends 4 standard stairs alternating feet, not needing handrail support. Participants who could not perform a TFT within 30 seconds, including those who loss of ambulation or the TFT was above 30 seconds, a value of 30 seconds was used. Baseline was defined as the last observed measurement on or prior to the first dose of study drug. LS mean and SE was calculated using the MMRM. |
| Secondary | Overall Treatment Period: Change From Baseline in NSAA Total Score at Week 144 | Baseline, Week 144 | The NSAA total score in the original scale is the sum of scores from 16 activities (excluding head lift). Each activity was scored as 0 (activity couldn't be performed), 1 (modified method, achieved goal without assistance), or 2 (normal, achieved goal without assistance). The total score ranges from 0 to 32, where higher scores indicate better functioning. If fewer than 13 of the 16 activities were performed, the total score was considered missing. If from 13 to 16 activities were performed, the total score was standardized by (observed total score / number of non-missing activities) x 16. If an activity could not be performed due to disease progression, a score of 0 was assigned. Baseline was defined as the last observed measurement on or prior to the first dose of study drug. LS mean and SE was calculated using analysis of covariation (ANCOVA) with multiple imputation. |
| Secondary | Overall Treatment Period: Time to Loss of Ambulation Over 144 Weeks | Baseline up to Week 144 | Time to loss of ambulation was defined as persistent inability to perform the 10-meter run/walk test within 30 seconds at any post-baseline visit and for all remaining visits. |
| Secondary | Overall Treatment Period: Time to Loss of Stair-Climbing Over 144 Weeks | Baseline up to Week 144 | Time to loss of stair-climbing was defined as persistent inability to perform the 4-stair climb test within 30 seconds at any post-baseline visit and for all remaining visits. |
| Secondary | Overall Treatment Period: Time to Loss of Stair- Descending Over 144 Weeks | Baseline up to Week 144 | Time to loss of stair-descending was defined as persistent inability to perform the 4-stair descend test within 30 seconds at any post-baseline visit and for all remaining visits. |
| Secondary | Overall Treatment Period: Number of Participants With Function Loss of NSAA Items at Week 144 | Week 144 | Function loss was defined as a drop of score from 1 or 2 at baseline to a score of 0 at the specified post-baseline in NSAA items. The missing assessments at post baseline visits were imputed using LOCF. |
| Secondary | Overall Treatment Period: Number of Participants With TEAEs | Baseline up to Week 148 | An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. An SAE was an 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); persistent or significant disability/incapacity; congenital anomaly. AEs included both SAEs and non-serious AEs. A TEAE was defined as an AE that occurs or worsens on or after the first dose of ataluren (regardless of double-blind or open-label) and up to 4 weeks after the last dose of ataluren. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE section'. |
| Secondary | OL Period: Plasma Pharmacokinetic (PK) Concentration of Ataluren | Predose and 2 hours postdose at Week 144 |
Source ClinicalTrials.govtrials-2026-11-12-0.4.0
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.
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