This study is a long-term study of ataluren in participants with nonsense mutation Duchenne muscular dystrophy.

Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy

Muscular Dystrophy, Duchenne

Muscular Dystrophies

Muscular Disorders, Atrophic

Muscular Diseases

Musculoskeletal Disease

Neuromuscular Diseases

Nervous System Diseases

Genetic Diseases, X-Linked

Genetic Diseases, Inborn

This study is a randomized, double-blind, placebo-controlled, 72-week study, followed by a 72-week open-label period. The purpose is to characterize the long-term effects of ataluren-mediated dystrophin restoration on disease progression. Participants will be randomized in a 1:1 ratio to ataluren or placebo. Participants will receive blinded study drug three times daily (TID) at morning, midday, and evening for 72 weeks, after which all participants will receive open-label ataluren for an additional 72 weeks (144 weeks in total). Study assessments will be performed at clinic visits every 12 weeks during the double-blind period and every 24 weeks during the open-label period.

Ataluren

PLACEBO

A Phase 3, Randomized, Double-blind, Placebo-controlled Efficacy and Safety Study of Ataluren in Patients With Nonsense Mutation Duchenne Muscular Dystrophy and Open-Label Extension

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. Least square (LS) mean and standard error (SE) was calculated using the mixed-model repeated measures (MMRM).

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.

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 timed function test (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.

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.

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.

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.

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.

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. Time to 10% persistent worsening in 6MWD was defined as the last time that 6MWD was not 10% worse compared with baseline. Participants who did not have 10% 6MWD worsening were censored at the time of the last 6-minute walk test during the DB period.

6MWD test is a non-encouraged test performed in a 30 meters long flat corridor, where participant is instructed to walk as far as possible, back and forth around two cones, with permission to slow down, rest, or stop if needed. Ambulation was assessed via 6MWD test following standardized procedures. Participants were not permitted to use assistive devices (walker, long leg braces, or short leg braces) during 6MWD test. Time to 10% persistent worsening in 6MWD was defined as the last time that 6MWD was not 10% worse compared with baseline. Participants who did not have 10% 6MWD worsening were censored at the time of last 6-minute walk test during DB period. Due to COVID, there were many participants who participated in the protocol-defined Week 72 visit late. Due to this reason, the calculated median for the time to event for the Ataluren arm in the DB period were greater than Week 72.

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 the MMRM.

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. 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.

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.

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.

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.

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).

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.

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'.

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.

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.

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.

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.

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'.

Participants will receive ataluren oral suspension 10 milligrams (mg)/kilogram (kg) in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening each day for 72 weeks in DB treatment period and for an additional 72 weeks in open-label treatment period.

Participants will receive placebo matched to ataluren oral suspension for 72 weeks in DB treatment period. After completion of DB treatment period, participants will receive ataluren oral suspension 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening each day for 72 weeks in open-label treatment period.

Phoenix Childrens Hospital

Children's Hospital of Los Angeles

University of California, San Francisco (UCSF) - Benioff Children's Hospital - Oakland

Stanford University Medical Center

University of California (UC) Davis Medical Center

Loma Linda University Children's Hospital

Northwest Florida Clinical Research Group, LLC

Indiana University Health - Riley Child Neurology

University of Kansas Medical Center

University of Michigan - CS Mott Children's Hospital

Children's Hospital of Michigan

University of Minnesota

Columbia University College of Physicians & Surgeons

Cincinnati Children's Hospital Medical Center

Shriners Hospital for Children

University of Pittsburgh School of Medicine

Cook Childrens Medical Center

Texas Children's Hospital

University of Texas Health Science Center at San Antonio

University Of Utah

Children's Hospital of the King's Daughters

Seattle Children's Hospital

The Childrens Hospital at Westmead

The Royal Childrens Hospital

Perth Children's Hospital

Queensland Children's Hospital

Hospital de Clínicas da Universidade Federal de Minas Gerais

Universidade Federal do Rio de Janeiro

Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo - FMUSP

UMHAT Sofiamed

Childrens Hospital London Health Sciences Centre

Childrens Hospital of Eastern Ontario

General Hospital of Chinese Armed Police Forces

The First Affiliated Hospital of Fujian Medical University

Xiangya Hospital Central South University

Children's Hospital of Fudan University

Shenzhen Children's Hospital

Queen Mary Hospital

Panchshil Hospital

National Institute of Mental Health and Neurosciences

P.D. Hinduja Hospital

Apollo Children's Hospital Chennai

Christian Medical College Hospital Vellore

Nizam's Institute of Medical Sciences (NIMS)

Apollo Gleneagles Hospital

Postgraduate Institute of Medical Education and Research

All India Institute of Medical Sciences

PTC Clinical Site

Hospital Tunku Azizah Kuala Lumpur

University Malaya Medical Centre (UMMC)

Hospital Angeles Chihuahua

Instituto Nacional de Pediatría

Instituto Nacional de Rehabilitacion

Samodzielny Publiczny Centralny Szpital Kliniczny w Warszawie

University of Puerto Rico - School of Medicine

Russian National Research Medical University n.a. N.I.Pirogov, structural branch - Research Clinical Institute of Pediatrics n.a. Academician Yu. E. Veltishchev

"Saint Petersburg State Paediatric Medical University" based at Consultative and Diagnostic Centre

Pusan National University Yangsan Hospital

Samsung Medical Center

Seoul National University Hospital

Kaohsiung Medical University Chung-Ho Memorial Hospital

National Taiwan University Hospital

Siriraj Hospital

Istanbul University- Instanbul Medical Faculty

As-treated population included all randomized participants who received study treatment, with treatment assignments designated according to actual treatment received. As pre-specified, AEs were summarized separately for DB period and for the overall ataluren experience, which included all participants who received ataluren throughout the study (DB and OL Period).

Participants received ataluren oral suspension 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening each day for 72 weeks in DB treatment period.

DB Period: Ataluren

Participants received placebo matched to ataluren oral suspension for 72 weeks in DB treatment period.

DB Period: Placebo

Participants received ataluren oral suspension 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening each day for 72 weeks in DB treatment period and for an additional 72 weeks in OL treatment period.

Ataluren /Ataluren

Participants received placebo matched to ataluren oral suspension for 72 weeks in DB treatment period. After completion of DB treatment period, participants received ataluren oral suspension 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening each day for 72 weeks in OL treatment period.

Ataluren/Placebo

Placebo

Total

Age, Continuous

Sex: Female, Male

Ethnicity (NIH/OMB)

Race (NIH/OMB)

As-treated population included all randomized participants who received study treatment, with treatment assignments designated according to actual treatment received.

Patient Advocacy

The mITT population included all randomized participants who met the following additional criteria: 7 to 16 years old with 6MWD ≥300 meters and time to stand from supine ≥5 seconds at baseline.

DB Period: Change From Baseline in 6-Minute Walk Distance (6MWD) at Week 72 - Modified Intention-to-treat (mITT) Population

The ITT population included all participants who were randomized, with treatment assignments designated according to initial randomization, regardless of whether participants received a different study treatment from the one randomized.

DB Period: Change From Baseline in 6MWD at Week 72 - Intent-to-Treat (ITT) Population

DB Period: Average Rate of Change From Baseline in 6MWD at Week 72 - mITT Population

DB Period: Average Rate of Change From Baseline in 6MWD at Week 72 - ITT Population

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 timed function test (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.

DB Period: Change From Baseline in Time to Run/Walk 10 Meters at Week 72 - mITT Population

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.

DB Period: Change From Baseline in Time to Run/Walk 10 Meters at Week 72 - ITT Population

DB Period: Change From Baseline in Time to Climb 4 Stairs at Week 72 - mITT Population

DB Period: Change From Baseline in Time to Climb 4 Stairs at Week 72 - ITT Population

DB Period: Change From Baseline in Time to Descend 4 Stairs at Week 72 - mITT Population

DB Period: Change From Baseline in Time to Descend 4 Stairs at Week 72 - ITT Population

DB Period: Composite of Average Change From Baseline in TFTs at Week 72 - mITT Population

DB Period: Composite of Average Change From Baseline in TFTs at Week 72 - ITT Population

DB Period: Composite of Average Rate of Change From Baseline in TFTs at Week 72 - mITT Population

DB Period: Composite of Average Rate of Change From Baseline in TFTs at Week 72 - ITT Population

The mITT population included all randomized participants who met the following additional criteria: 7 to 16 years old with 6MWD ≥300 meters and time to stand from supine ≥5 seconds at baseline. Here, 'Overall number of participants analyzed' = participants with 10% persistent worsening by Week 72.

DB Period: Time to Persistent 10% Worsening in 6MWD at Week 72 - mITT Population

The ITT population included all participants who were randomized, with treatment assignments designated according to initial randomization, regardless of whether participants received a different study treatment from the one randomized. 'Overall number of participants analyzed' = participants with 10% persistent worsening by Week 72.

DB Period: Time to Persistent 10% Worsening in 6MWD - ITT Population

DB Period: Change From Baseline in North Start Ambulatory Assessment (NSAA) Total Score at Week 72 - mITT Population

DB Period: Change From Baseline in NSAA Total Score at Week 72 - ITT Population

The mITT population included all randomized participants who met the following additional criteria: 7 to 16 years old with 6MWD ≥300 meters and time to stand from supine ≥5 seconds at baseline. Here, 'Overall number of participants analyzed' = participants with loss of ambulation by Week 72.

DB Period: Time to Loss of Ambulation - mITT Population

The ITT population included all participants who were randomized, with treatment assignments designated according to initial randomization, regardless of whether participants received a different study treatment from the one randomized. Here, 'Overall number of participants analyzed' = participants with loss of ambulation by Week 72.

DB Period: Time to Loss of Ambulation Over 72 Weeks - ITT Population

The mITT population included all randomized participants who met the following additional criteria: 7 to 16 years old with 6MWD ≥300 meters and time to stand from supine ≥5 seconds at baseline. Here, 'Overall number of participants analyzed' = participants with loss of stair-climbing by Week 72.

DB Period: Time to Loss of Stair-Climbing - mITT Population

The ITT population included all participants who were randomized, with treatment assignments designated according to initial randomization, regardless of whether participants received a different study treatment from the one randomized. Here, 'Overall number of participants analyzed' = participants with loss of stair-climbing by Week 72.

DB Period: Time to Loss of Stair-Climbing - ITT Population

The mITT population included all randomized participants who met the following additional criteria: 7 to 16 years old with 6MWD ≥300 meters and time to stand from supine ≥5 seconds at baseline. Here, 'Overall number of participants analyzed' = participants with loss of stair-descending by Week 72.

DB Period: Time to Loss of Stair-Descending - mITT Population

The ITT population included all participants who were randomized, with treatment assignments designated according to initial randomization, regardless of whether participants received a different study treatment from the one randomized. Here, 'Overall number of participants analyzed' = participants with loss of stair-descending by Week 72.

DB Period: Time to Loss of Stair-Descending - ITT Population

Stand

Walk (10m)

Stand up from chair

Stand on one leg - right

Stand on one leg - left

Climb box step - right

Descend box step - right

Climb box step - left

Descend box step - left

Lifts head

Gets to sitting

Rise from floor

Stand on heels

Jump

Hop right

Hop left

Run (10m)

The mITT population included all randomized participants who met the following additional criteria: 7 to 16 years old with 6MWD ≥300 meters and time to stand from supine ≥5 seconds at baseline. Here, 'Number analyzed' = number of participants with baseline score of 2 or 1 of NSAA items and at least one post-baseline assessment for the specific activity.

DB Period: Number of Participants With Function Loss of NSAA Items at Week 72 - mITT Population

The ITT population included all participants who were randomized, with treatment assignments designated according to initial randomization, regardless of whether participants received a different study treatment from the one randomized. Here, 'Number analyzed' = number of participants with baseline score of 2 or 1 and at least one post-baseline assessment for the specific activity of NSAA items.

DB Period: Number of Participants With Function Loss of NSAA Items at Week 72 - ITT Population

The as-treated population included all randomized participants who received study treatment, with treatment assignments designated according to actual treatment received.

DB Period: Number of Participants With Treatment-emergent Adverse Events (TEAEs)

Ataluren/Ataluren

Placebo/Ataluren

Overall Treatment Period: Change From Baseline in 6MWD at Week 144

Overall Treatment Period: Composite of Average Change From Baseline in TFTs at Week 144

Overall Treatment Period: Change From Baseline in Time to Run/Walk 10 Meters at Week 144

Overall Treatment Period: Change From Baseline in Time to Climb 4 Stairs at Week 144

Overall Treatment Period: Change From Baseline in Time to Descend 4 Stairs at Week 144

Overall Treatment Period: Change From Baseline in NSAA Total Score at Week 144

The ITT population included all participants who were randomized, with treatment assignments designated according to initial randomization, regardless of whether participants received a different study treatment from the one randomized. Here, 'Overall number of participants analyzed' = participants with loss of ambulation by Week 144.

Overall Treatment Period: Time to Loss of Ambulation Over 144 Weeks

The ITT population included all participants who were randomized, with treatment assignments designated according to initial randomization, regardless of whether participants received a different study treatment from the one randomized. Here, 'Overall number of participants analyzed' = participants with loss of stair-climbing by Week 144.

Overall Treatment Period: Time to Loss of Stair-Climbing Over 144 Weeks

The ITT population included all participants who were randomized, with treatment assignments designated according to initial randomization, regardless of whether participants received a different study treatment from the one randomized. Here, 'Overall number of participants analyzed' = participants with loss of stair-descending by Week 144.

Overall Treatment Period: Time to Loss of Stair- Descending Over 144 Weeks

The ITT population included all participants who were randomized, with treatment assignments designated according to initial randomization, regardless of whether participants received a different study treatment from the one randomized. Here, 'Number analyzed' = number of participants with baseline score of 2 or 1 and at least one post-baseline assessment for the specific activity in NSAA items.

Overall Treatment Period: Number of Participants With Function Loss of NSAA Items at Week 144

The as-treated-OA population included all randomized participants who received at least 1 dose of ataluren anytime during the study.

Overall Treatment Period: Number of Participants With TEAEs

Predose

2 hours postdose

The ITT population included all participants who were randomized, with treatment assignments designated according to initial randomization, regardless of whether participants received a different study treatment from the one randomized. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure. 'Number analyzed' = participants evaluable at specified timepoint.

Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy

About this trial

Study ID

Status

Type

Phase

Placebo

Accepting

Trial Timing

What is this trial about?

What are the participation requirements?