Trial · NCT03179631

Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy

NCT03179631, Phase 3, Completed, PTC Therapeutics; results first posted 2026-03-10

3 sourcestrials-2026-11-12-0.4.0

Cite this record

ConductScience Library. Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy. Trials, record NCT03179631. Release trials-2026-11-12-0.4.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03179631

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy
NCT number: NCT03179631
Status: Completed
Phases: Phase 3
Enrollment: 360 participants, actual
Lead sponsor: PTC Therapeutics
Conditions: Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Disease, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, X-Linked, Genetic Diseases, Inborn
Interventions: Ataluren, PLACEBO
First posted: 2017-06-07
Last updated: 2026-03-10
Results first posted: 2026-03-10

Source ClinicalTrials.gov

Registered outcomes39

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	DB Period: Change From Baseline in 6-Minute Walk Distance (6MWD) at Week 72 - Modified Intention-to-treat (mITT) Population	Baseline, Week 72	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).
Primary	DB Period: Change From Baseline in 6MWD at Week 72 - Intent-to-Treat (ITT) Population	Baseline, Week 72	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.
Primary	DB Period: Average Rate of Change From Baseline in 6MWD at Week 72 - mITT Population	Baseline, Week 72	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.
Primary	DB Period: Average Rate of Change From Baseline in 6MWD at Week 72 - ITT Population	Baseline, Week 72	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	DB Period: Change From Baseline in Time to Run/Walk 10 Meters at Week 72 - mITT Population	Baseline, Week 72	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.
Secondary	DB Period: Change From Baseline in Time to Run/Walk 10 Meters at Week 72 - ITT Population	Baseline, Week 72	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	DB Period: Change From Baseline in Time to Climb 4 Stairs at Week 72 - mITT Population	Baseline, Week 72	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	DB Period: Change From Baseline in Time to Climb 4 Stairs at Week 72 - ITT Population	Baseline, Week 72	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	DB Period: Change From Baseline in Time to Descend 4 Stairs at Week 72 - mITT Population	Baseline, Week 72	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	DB Period: Change From Baseline in Time to Descend 4 Stairs at Week 72 - ITT Population	Baseline, Week 72	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.

1 to 10 of 39Source ClinicalTrials.gov

See all 39 registered outcomes

Outcomes39

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	DB Period: Change From Baseline in 6-Minute Walk Distance (6MWD) at Week 72 - Modified Intention-to-treat (mITT) Population	Baseline, Week 72	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.	meters	LEAST_SQUARES_MEAN
PRIMARY	DB Period: Change From Baseline in 6MWD at Week 72 - Intent-to-Treat (ITT) Population	Baseline, Week 72	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.	meters	LEAST_SQUARES_MEAN
PRIMARY	DB Period: Average Rate of Change From Baseline in 6MWD at Week 72 - mITT Population	Baseline, Week 72	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.	meters/week	LEAST_SQUARES_MEAN
PRIMARY	DB Period: Average Rate of Change From Baseline in 6MWD at Week 72 - ITT Population	Baseline, Week 72	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.	meters/week	LEAST_SQUARES_MEAN
SECONDARY	DB Period: Change From Baseline in Time to Run/Walk 10 Meters at Week 72 - mITT Population	Baseline, Week 72	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.	seconds	LEAST_SQUARES_MEAN
SECONDARY	DB Period: Change From Baseline in Time to Run/Walk 10 Meters at Week 72 - ITT Population	Baseline, Week 72	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.	seconds	LEAST_SQUARES_MEAN
SECONDARY	DB Period: Change From Baseline in Time to Climb 4 Stairs at Week 72 - mITT Population	Baseline, Week 72	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.	seconds	LEAST_SQUARES_MEAN
SECONDARY	DB Period: Change From Baseline in Time to Climb 4 Stairs at Week 72 - ITT Population	Baseline, Week 72	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.	seconds	LEAST_SQUARES_MEAN
SECONDARY	DB Period: Change From Baseline in Time to Descend 4 Stairs at Week 72 - mITT Population	Baseline, Week 72	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.	seconds	LEAST_SQUARES_MEAN
SECONDARY	DB Period: Change From Baseline in Time to Descend 4 Stairs at Week 72 - ITT Population	Baseline, Week 72	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.	seconds	LEAST_SQUARES_MEAN

1 to 10 of 39Source ClinicalTrials.gov

See all 39 outcomes

Results by group176

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Value	Spread	Units
DB Period: Change From Baseline in 6-Minute Walk Distance (6MWD) at Week 72 - Modified Intention-to-treat (mITT) Population	Ataluren	-81.83	Standard Error 6.461	meters
DB Period: Change From Baseline in 6-Minute Walk Distance (6MWD) at Week 72 - Modified Intention-to-treat (mITT) Population	Placebo	-90.09	Standard Error 6.377	meters
DB Period: Change From Baseline in 6MWD at Week 72 - Intent-to-Treat (ITT) Population	Ataluren	-53.01	Standard Error 5.169	meters
DB Period: Change From Baseline in 6MWD at Week 72 - Intent-to-Treat (ITT) Population	Placebo	-67.43	Standard Error 5.403	meters
DB Period: Average Rate of Change From Baseline in 6MWD at Week 72 - mITT Population	Ataluren	-1.14	Standard Error 0.090	meters/week
DB Period: Average Rate of Change From Baseline in 6MWD at Week 72 - mITT Population	Placebo	-1.25	Standard Error 0.089	meters/week
DB Period: Average Rate of Change From Baseline in 6MWD at Week 72 - ITT Population	Ataluren	-0.74	Standard Error 0.072	meters/week
DB Period: Average Rate of Change From Baseline in 6MWD at Week 72 - ITT Population	Placebo	-0.94	Standard Error 0.075	meters/week
DB Period: Change From Baseline in Time to Run/Walk 10 Meters at Week 72 - mITT Population	Ataluren	3.06	Standard Error 0.393	seconds
DB Period: Change From Baseline in Time to Run/Walk 10 Meters at Week 72 - mITT Population	Placebo	3.79	Standard Error 0.389	seconds

1 to 10 of 176Source ClinicalTrials.gov

See all 176 results by group

Participant flow16

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
DB Treatment Period (72 Weeks)	STARTED	Ataluren	184
DB Treatment Period (72 Weeks)	STARTED	Placebo	176
DB Treatment Period (72 Weeks)	Intent-to-treat (ITT) Population	Ataluren	183
DB Treatment Period (72 Weeks)	Intent-to-treat (ITT) Population	Placebo	176
DB Treatment Period (72 Weeks)	Received at Least 1 Dose of Study Drug	Ataluren	184
DB Treatment Period (72 Weeks)	Received at Least 1 Dose of Study Drug	Placebo	176
DB Treatment Period (72 Weeks)	COMPLETED	Ataluren	179
DB Treatment Period (72 Weeks)	COMPLETED	Placebo	172
DB Treatment Period (72 Weeks)	NOT COMPLETED	Ataluren	5
DB Treatment Period (72 Weeks)	NOT COMPLETED	Placebo	4

1 to 10 of 16Source ClinicalTrials.gov

See all 16 participant flow

Adverse events224

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	Abdominal pain	Gastrointestinal disorders	DB Period: Ataluren	13	184	22
other	Abdominal pain	Gastrointestinal disorders	DB Period: Placebo	14	176	15
other	Abdominal pain	Gastrointestinal disorders	Ataluren /Ataluren	16	184	27
other	Abdominal pain	Gastrointestinal disorders	Ataluren/Placebo	4	172	4
other	Diarrhoea	Gastrointestinal disorders	DB Period: Ataluren	15	184	17
other	Diarrhoea	Gastrointestinal disorders	DB Period: Placebo	12	176	15
other	Diarrhoea	Gastrointestinal disorders	Ataluren /Ataluren	18	184	20
other	Diarrhoea	Gastrointestinal disorders	Ataluren/Placebo	2	172	2
other	Vomiting	Gastrointestinal disorders	DB Period: Ataluren	30	184	46
other	Vomiting	Gastrointestinal disorders	DB Period: Placebo	9	176	10

1 to 10 of 224Source ClinicalTrials.gov

See all 224 adverse events

Adverse event totals12

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	DB Period: Ataluren	0	184
Total, all-cause mortality	DB Period: Placebo	0	176
Total, all-cause mortality	Ataluren /Ataluren	0	184
Total, all-cause mortality	Ataluren/Placebo	0	172
Total, other adverse events	DB Period: Ataluren	157	184
Total, other adverse events	DB Period: Placebo	148	176
Total, other adverse events	Ataluren /Ataluren	170	184
Total, other adverse events	Ataluren/Placebo	107	172
Total, serious adverse events	DB Period: Ataluren	13	184
Total, serious adverse events	DB Period: Placebo	12	176

1 to 10 of 12Source ClinicalTrials.gov

See all 12 adverse event totals

Papers1

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as
41879641	DERIVED

Source PubMed

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 comparisons2

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

Comparison	Source
Ataluren/Ataluren vs Placebo/Ataluren: Overall Treatment Period: Number of Participants With TEAEs	Trial comparisons
Ataluren vs Placebo: DB Period: Number of Participants With Treatment-emergent Adverse Events (TEAEs)	Trial comparisons

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Source

Release: trials-2026-11-12-0.4.0
Release manifest SHA-256: 0f4a8d8c48aee2a7573ae0fa94ecbeab70c1adbe449bbc95c424bc7d3e97b9d4
Registry capture: 2026-11-12
Results copy, AACT: 2026-03-23
NIH clinical studies file: 2026-11-13

Source ClinicalTrials.gov

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNone 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/NCT03179631

GET https://conductscience.org/api/trials/v1/NCT03179631/registered?page=2

API reference For AI assistants