Trial · NCT00592553

Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)

NCT00592553, Phase 2, Completed, PTC Therapeutics; results first posted 2020-04-07

3 sourcestrials-2026-11-12-0.4.0

Cite this record

ConductScience Library. Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD). Trials, record NCT00592553. Release trials-2026-11-12-0.4.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00592553

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Trial

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

Title: Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)
NCT number: NCT00592553
Status: Completed
Phases: Phase 2
Enrollment: 174 participants, actual
Lead sponsor: PTC Therapeutics
Conditions: Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
Interventions: Ataluren, Placebo
First posted: 2008-01-14
Last updated: 2020-04-07
Other registries' IDs: eudract:2007-005478-29
Results first posted: 2020-04-07

Source ClinicalTrials.gov

Registered outcomes23

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

List	Outcome	Time frame	Description
Primary	Change From Baseline in 6MWD at Week 48	Baseline, Week 48	The 6MWD test was performed in a 30 meters long flat corridor, where the participant was 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 by measuring the 6MWD in meters. Participants were not permitted to use assistive devices (walker, long leg braces, or short leg braces) during the 6MWD test.
Secondary	Change From Baseline in Time to Stand From Supine Position at Week 48	Baseline, Week 48	If the time taken to perform this test exceeded 30 seconds or if a participant could not perform this test due to disease progression, a value of 30 seconds was used. Change from baseline data has been reported.
Secondary	Change From Baseline in Time to Walk/Run 10 Meters at Week 48	Baseline, Week 48	If the time taken to perform this test exceeded 30 seconds or if a participant could not perform this test due to disease progression, a value of 30 seconds was used. Change from baseline data has been reported.
Secondary	Change From Baseline in Time to Climb 4 Stairs at Week 48	Baseline, Week 48	If the time taken to perform this test exceeded 30 seconds or if a participant could not perform this test due to disease progression, a value of 30 seconds was used. Change from baseline data has been reported.
Secondary	Change From Baseline in Time to Descend 4 Stairs at Week 48	Baseline, Week 48	If the time taken to perform this test exceeded 30 seconds or if a participant could not perform this test due to disease progression, a value of 30 seconds was used. Change from baseline data has been reported.
Secondary	Change From Baseline in Force Exerted During Knee Flexion and Extension, Elbow Flexion and Extension, and Shoulder Abduction at Week 48, as Assessed by Myometry	Baseline, Week 48	Upper and lower extremity myometry was performed using a myometer following standardized procedures. Muscle groups evaluated included knee flexors, knee extensors, elbow flexors, elbow extensors, and shoulder abductors. Bilateral assessments were done and 3 measurements were recorded from each muscle group on each side if possible. Mean values for the left and right sides were calculated.
Secondary	Change From Baseline in Mean Activity Period/Day/Visit at Week 48, as Assessed by Step Activity Monitoring (SAM)	Baseline, Week 48	The SAM is a pedometer (worn on the ankle) that continuously records the number of steps per time interval. Participants were instructed to continue to wear the SAM for at least 9 consecutive days. SAM was used to record the number of strides/minute following each visit. A stride is the leg motion that begins when the foot with SAM leaves the floor and ends when the same foot touches the floor again (that is, a stride generally equals 2 steps). Mean activity period/day/visit was computed for each participant. Mean obtained during Screening (Week -6 to -1) and following Week 1 visit were used as baseline data for analysis. For each day, an active period was defined as the first time after 3:00 AM that greater than (\>) 2 strides/minute were recorded to the last time prior to midnight that \>2 strides/minute were recorded. Days were deleted on which such an active period was less than (\<) 50 percent (%) of the mean active period across all days for that participant's visit.
Secondary	Change From Baseline in Mean Total Step Count/Day/Visit During the Active Periods at Week 48, as Assessed by SAM	Baseline, Week 48	The SAM is a pedometer (worn on the ankle) that continuously records the number of steps per time interval. Participants were instructed to continue to wear the SAM for at least 9 consecutive days. SAM was used to record the number of strides/minute following each visit. A stride is the leg motion that begins when the foot with SAM leaves the floor and ends when the same foot touches the floor again (that is, a stride generally equals 2 steps). Mean total step count/day/visit during the active periods was computed for each participant. Mean obtained during Screening (Week -6 to -1) and following Week 1 visit were used as baseline data for analysis. For each day, an active period was defined as the first time after 3:00 AM that \>2 strides/minute were recorded to the last time prior to midnight that \>2 strides/minute were recorded. Days were deleted on which such an active period was \<50% of the mean active period across all days for that participant's visit.
Secondary	Change From Baseline in Mean Total Step Count/Hour During the Active Period at Week 48, as Assessed by SAM	Baseline, Week 48	The SAM is a pedometer (worn on the ankle) that continuously records the number of steps per time interval. Participants were instructed to continue to wear the SAM for at least 9 consecutive days. SAM was used to record the number of strides/minute following each visit. A stride is the leg motion that begins when the foot with SAM leaves the floor and ends when the same foot touches the floor again (that is, a stride generally equals 2 steps). Mean total step count/hour during the active periods for the days in a visit was computed for each participant. Mean obtained during Screening (Week -6 to -1) and following Week 1 visit were used as baseline data for analysis. For each day, an active period was defined as the first time after 3:00 AM that \>2 strides/minute were recorded to the last time prior to midnight that \>2 strides/minute were recorded. Days were deleted on which such an active period was \<50% of the mean active period across all days for that participant's visit.
Secondary	Change From Baseline in Maximum Continuous 10-minute, 20-minute, 30-minute, and 60-minute Total Step Count at Week 48, as Assessed by SAM	Baseline, Week 48	SAM is a pedometer (worn on the ankle) that continuously records the number of steps per time interval. Participants were instructed to continue to wear the SAM for at least 9 consecutive days. SAM was used to record the number of strides/minute following each visit. A stride is the leg motion that begins when the foot with SAM leaves the floor and ends when the same foot touches the floor again (that is, a stride generally equals 2 steps). The maximum continuous 10-minute, 20-minute, 30-minute, and 60-minute total step counts were computed for each participant. Mean obtained during Screening (Week -6 to -1) and following Week 1 visit were used as baseline data for analysis. For each day, an active period was defined as the first time after 3:00 AM that \>2 strides/minute were recorded to the last time prior to midnight that \>2 strides/minute were recorded. Days were deleted on which such an active period was \<50% of the mean active period across all days for that participant's visit.

1 to 10 of 23Source ClinicalTrials.gov

See all 23 registered outcomes

Outcomes23

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

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Change From Baseline in 6MWD at Week 48	Baseline, Week 48	Intent-to-treat (ITT) population included all participants who were randomized and received any study treatment; and had a valid baseline, and at least one valid post-baseline 6MWD value. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure.	meters	MEAN
SECONDARY	Change From Baseline in Time to Stand From Supine Position at Week 48	Baseline, Week 48	ITT population included all participants who were randomized and received any study treatment; and had a valid baseline, and at least one valid post-baseline 6MWD value. Here, 'Number analyzed' signifies participants evaluable at specified timepoint.	seconds	MEAN
SECONDARY	Change From Baseline in Time to Walk/Run 10 Meters at Week 48	Baseline, Week 48	ITT population included all participants who were randomized and received any study treatment; and had a valid baseline, and at least one valid post-baseline 6MWD value. Here, 'Number analyzed' signifies participants evaluable at specified timepoint.	seconds	MEAN
SECONDARY	Change From Baseline in Time to Climb 4 Stairs at Week 48	Baseline, Week 48	ITT population included all participants who were randomized and received any study treatment; and had a valid baseline, and at least one valid post-baseline 6MWD value. Here, 'Number analyzed' signifies participants evaluable at specified timepoint.	seconds	MEAN
SECONDARY	Change From Baseline in Time to Descend 4 Stairs at Week 48	Baseline, Week 48	ITT population included all participants who were randomized and received any study treatment; and had a valid baseline, and at least one valid post-baseline 6MWD value. Here, 'Number analyzed' signifies participants evaluable at specified timepoint.	seconds	MEAN
SECONDARY	Change From Baseline in Force Exerted During Knee Flexion and Extension, Elbow Flexion and Extension, and Shoulder Abduction at Week 48, as Assessed by Myometry	Baseline, Week 48	ITT population included all participants who were randomized and received any study treatment; and had a valid baseline, and at least one valid post-baseline 6MWD value. Here, 'Number analyzed' signifies participants evaluable for specified categories.	pounds	MEAN
SECONDARY	Change From Baseline in Mean Activity Period/Day/Visit at Week 48, as Assessed by Step Activity Monitoring (SAM)	Baseline, Week 48	ITT population included all participants who were randomized and received any study treatment; and had a valid baseline, and at least one valid post-baseline 6MWD value. Here, 'Number analyzed' signifies participants evaluable for this outcome measure at specified timepoint.	minutes	MEAN
SECONDARY	Change From Baseline in Mean Total Step Count/Day/Visit During the Active Periods at Week 48, as Assessed by SAM	Baseline, Week 48	ITT population included all participants who were randomized and received any study treatment; and had a valid baseline, and at least one valid post-baseline 6MWD value. Here, 'Number analyzed' signifies participants evaluable for this outcome measure at specified timepoint.	steps/day	MEAN
SECONDARY	Change From Baseline in Mean Total Step Count/Hour During the Active Period at Week 48, as Assessed by SAM	Baseline, Week 48	ITT population included all participants who were randomized and received any study treatment; and had a valid baseline, and at least one valid post-baseline 6MWD value. Here, 'Number analyzed' signifies participants evaluable for this outcome measure at specified timepoint.	steps/hour	MEAN
SECONDARY	Change From Baseline in Maximum Continuous 10-minute, 20-minute, 30-minute, and 60-minute Total Step Count at Week 48, as Assessed by SAM	Baseline, Week 48	ITT population included all participants who were randomized and received any study treatment; and had a valid baseline, and at least one valid post-baseline 6MWD value. Here, 'Number analyzed' signifies participants evaluable for this outcome measure for specified categories.	steps	MEAN

1 to 10 of 23Source ClinicalTrials.gov

See all 23 outcomes

Results by group237

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

Outcome	Group	Category	Value	Spread	Units
Change From Baseline in 6MWD at Week 48	High-Dose Ataluren		-41.81	Standard Deviation 89.234	meters
Change From Baseline in 6MWD at Week 48	Low-Dose Ataluren		-12.86	Standard Deviation 72.007	meters
Change From Baseline in 6MWD at Week 48	Placebo		-42.56	Standard Deviation 90.046	meters
Change From Baseline in Time to Stand From Supine Position at Week 48	High-Dose Ataluren	Baseline	12.25	Standard Deviation 11.191	seconds
Change From Baseline in Time to Stand From Supine Position at Week 48	Low-Dose Ataluren	Baseline	10.80	Standard Deviation 9.924	seconds
Change From Baseline in Time to Stand From Supine Position at Week 48	Placebo	Baseline	11.50	Standard Deviation 11.440	seconds
Change From Baseline in Time to Stand From Supine Position at Week 48	High-Dose Ataluren	Change at Week 48	3.00	Standard Deviation 5.686	seconds
Change From Baseline in Time to Stand From Supine Position at Week 48	Low-Dose Ataluren	Change at Week 48	3.23	Standard Deviation 5.761	seconds
Change From Baseline in Time to Stand From Supine Position at Week 48	Placebo	Change at Week 48	3.24	Standard Deviation 7.253	seconds
Change From Baseline in Time to Walk/Run 10 Meters at Week 48	High-Dose Ataluren	Baseline	7.80	Standard Deviation 5.243	seconds

1 to 10 of 237Source ClinicalTrials.gov

See all 237 results by group

Participant flow15

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

Period	Milestone	Group	Participants
Overall Study	STARTED	High-Dose Ataluren	60
Overall Study	STARTED	Low-Dose Ataluren	57
Overall Study	STARTED	Placebo	57
Overall Study	As-treated Population	High-Dose Ataluren	60
Overall Study	As-treated Population	Low-Dose Ataluren	57
Overall Study	As-treated Population	Placebo	57
Overall Study	ITT Population	High-Dose Ataluren	60
Overall Study	ITT Population	Low-Dose Ataluren	57
Overall Study	ITT Population	Placebo	57
Overall Study	COMPLETED	High-Dose Ataluren	59

1 to 10 of 15Source ClinicalTrials.gov

See all 15 participant flow

Adverse events957

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	Lymphadenopathy	Blood and lymphatic system disorders	High-Dose Ataluren	0	60	0
other	Lymphadenopathy	Blood and lymphatic system disorders	Low-Dose Ataluren	2	57	2
other	Lymphadenopathy	Blood and lymphatic system disorders	Placebo	2	57	2
other	Microcytic anaemia	Blood and lymphatic system disorders	High-Dose Ataluren	1	60	1
other	Microcytic anaemia	Blood and lymphatic system disorders	Low-Dose Ataluren	0	57	0
other	Microcytic anaemia	Blood and lymphatic system disorders	Placebo	0	57	0
other	Arrhythmia	Cardiac disorders	High-Dose Ataluren	1	60	1
other	Arrhythmia	Cardiac disorders	Low-Dose Ataluren	0	57	0
other	Arrhythmia	Cardiac disorders	Placebo	0	57	0
other	Dilatation ventricular	Cardiac disorders	High-Dose Ataluren	0	60	0

1 to 10 of 957Source ClinicalTrials.gov

See all 957 adverse events

Adverse event totals9

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	High-Dose Ataluren
Total, all-cause mortality	Low-Dose Ataluren
Total, all-cause mortality	Placebo
Total, other adverse events	High-Dose Ataluren	58	60
Total, other adverse events	Low-Dose Ataluren	56	57
Total, other adverse events	Placebo	57	57
Total, serious adverse events	High-Dose Ataluren	2	60
Total, serious adverse events	Low-Dose Ataluren	2	57
Total, serious adverse events	Placebo	3	57

Source ClinicalTrials.gov

Papers3

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as	Source
32851872	DERIVED	PubMed
34693725	DERIVED	PubMed
37236246	DERIVED	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 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.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

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/NCT00592553

GET https://conductscience.org/api/trials/v1/NCT00592553/registered?page=2

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