Trial · NCT02819557
Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy
NCT02819557, Phase 2, Completed, PTC Therapeutics; results first posted 2020-08-28
3 sourcestrials-2026-11-12-0.4.0
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ConductScience Library. Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy. Trials, record NCT02819557. Release trials-2026-11-12-0.4.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02819557
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy
- NCT number
- NCT02819557
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 14 participants, actual
- Lead sponsor
- PTC Therapeutics
- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- Ataluren
- First posted
- 2016-06-30
- Last updated
- 2020-08-28
- Results first posted
- 2020-08-28
Source ClinicalTrials.gov
Registered outcomes14
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), TEAEs Leading to Discontinuation, and Serious Adverse Events (SAEs) | Baseline up to Week 56 | A TEAE was any untoward medical occurrence or undesirable event that begins or worsens following administration of study drug, whether or not considered related to study drug by Investigator. An 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 dystrophinopathy. An event was not reported as an SAE, if event was exclusively a relapse or expected change or progression of baseline dystrophinopathy. AEs included both SAEs and nonserious AEs. AEs classified according to National Cancer Institute Common Terminology Criteria for Adverse Events version 4.0 and coded using Medical Dictionary for Regulatory Activities. A summary of SAEs and all non-serious AEs, regardless of causality, is located in the Reported Adverse Events section. |
| Primary | Number of Participants With a Clinically Meaningful Abnormal Clinical Laboratory (Biochemistry, Hematology, and Urinalysis) Parameter | Baseline up to Week 56 | Clinical laboratory results that were considered clinically meaningful were to be determined by the Investigator and Sponsor. Biochemistry parameters included sodium, potassium, chloride, bicarbonate, blood urea nitrogen, creatinine, magnesium, calcium, phosphorus, uric acid, glucose, total protein, albumin, bilirubin (total, direct, and indirect), aspartate aminotransferase, alanine aminotransferase, gamma-glutamyl transferase, creatine kinase, lactate dehydrogenase, alkaline phosphatase, total cholesterol, high-density lipoprotein, low-density lipoprotein, triglycerides, and cystatin C. Hematology parameters included white blood cell count with differential, hemoglobin, hematocrit, other red cell parameters, and platelet count. Urinalysis parameters included pH, specific gravity, glucose, ketones, blood, protein, urobilinogen, bilirubin, nitrite, and leukocyte esterase. A summary of other non-serious AEs and all serious AEs, regardless of causality is located in Reported AE section. |
| Primary | Number of Participants With a Clinically Meaningful Abnormal Electrocardiogram (ECG) Test Results | Baseline up to Week 56 | ECG results that were considered clinically meaningful were to be determined by the Investigator. A summary of other non-serious AEs and all serious AEs, regardless of causality is located in Reported AE section. |
| Primary | Number of Participants With a Dose-Limiting Toxicity as Measured by Hepatic and Renal Toxicity | Baseline up to Week 56 | Dose-limiting toxicity was measured through clinical evaluations for potential hepatic and renal toxicities. The clinical evaluations included the following: * Hepatic: The participant's medical history, hepatitis screening results, all clinical blood values (particularly serum bilirubin, gamma-glutamyl transferase \[GGT\], aspartate aminotransferase \[AST\], and alanine aminotransferase \[ALT\] values), and all concomitant medications were reviewed. * Renal: The participant's medical history, all clinical blood and urine renal values, serum electrolytes, medications, and potential pre- or post-renal conditions were reviewed. |
| Secondary | Pharmacokinetics: Maximum Observed Plasma Concentration From Time Zero up to 6 Hours After the Morning Dose (Cmax0-6hr) | 0 (predose), 1, 2, 4, and 6 (postdose) hours on Days 1 and 28 | Ataluren concentrations in plasma were analyzed using a validated high performance liquid chromatography-tandem mass spectrometry (HPLC-MS/MS) method. |
| Secondary | Pharmacokinetics: Time to Reach Maximum Observed Plasma Concentration From Time Zero up to 6 Hours After the Morning Dose (Tmax0-6hr) | 0 (predose), 1, 2, 4, and 6 (postdose) hours on Days 1 and 28 | Ataluren concentrations in plasma were analyzed using a validated HPLC-MS/MS method. |
| Secondary | Area Under the Plasma Concentration Time Curve From Time Zero up to 10 Hours After the Morning Dose (AUC0-10hr) | 0 (predose), 1, 2, 4, 6, 8, and 10 (postdose) hours on Days 1 and 28 | Ataluren concentrations in plasma were analyzed using a validated HPLC-MS/MS method. AUC0-10hr was measured using the linear trapezoidal rule during the ascending portion of the curve and the log-trapezoidal rule during the descending portion of the curve. |
| Secondary | Pharmacokinetics: Concentration at the End of the First (Morning) Dose Interval (Ctrough6hr) | 0 (predose), 1, 2, 4, and 6 (postdose) hours on Days 1 and 28 | Ataluren concentrations in plasma were analyzed using a validated HPLC-MS/MS method. |
| Secondary | Change From Baseline in Proximal Muscle Function as Assessed by Speed During TFTs | Baseline, Week 28 and Week 52 | TFTs included time to stand from supine position (rise to standing), time to run/walk 10 meters (m), and time to ascend/descend 4 stairs. A decrease from baseline reflects faster completion of the functional task and, thus, better muscle function. If the time taken to perform a test exceeded 30 seconds or if a participant could not perform the test due to disease progression (PD), a value of 30 seconds was used. |
| Secondary | Change From Baseline in Physical Function as Measured by the NSAA | Baseline, Week 28 and Week 52 | NSAA consists of 17 activities, including items assessing abilities necessary to remain functionally ambulant (that is, ability to rise from floor, to get from lying to sitting/sitting to standing, and that are known to progressively deteriorate); items that can be partly present in DMD early stages (that is, assessing head raise and standing on heels); and a number of activities such as hopping, jumping, and running. Since the boys were \<5 years old, revised 16 point, 8-point, and 3-point scales were used over the 17 point scale. Scores for evaluations=0 (Unable to achieve independently), 1 (Modified method but achieved goal independent of physical assistance), or 2 (Normal, no obvious modification of activity). Maximum total score for the 16-point scale=32, 8-point scale=16, and 3-point scale=6. If an activity couldn't be performed due to PD/loss of ambulation, a score of 0 was assigned. Change from Baseline calculated by subtracting Baseline value from value at Week 28 and Week 52. |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 registered outcomesOutcomes14
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), TEAEs Leading to Discontinuation, and Serious Adverse Events (SAEs) | Baseline up to Week 56 | All participants who received at least 1 dose of ataluren (Safety Population). | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With a Clinically Meaningful Abnormal Clinical Laboratory (Biochemistry, Hematology, and Urinalysis) Parameter | Baseline up to Week 56 | All participants who received at least 1 dose of ataluren (Safety Population). | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With a Clinically Meaningful Abnormal Electrocardiogram (ECG) Test Results | Baseline up to Week 56 | All participants who received at least 1 dose of ataluren (Safety Population). | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With a Dose-Limiting Toxicity as Measured by Hepatic and Renal Toxicity | Baseline up to Week 56 | All participants who received at least 1 dose of ataluren (Safety Population). | participants | NUMBER |
| SECONDARY | Pharmacokinetics: Maximum Observed Plasma Concentration From Time Zero up to 6 Hours After the Morning Dose (Cmax0-6hr) | 0 (predose), 1, 2, 4, and 6 (postdose) hours on Days 1 and 28 | All participants who received at least 1 dose of ataluren and had at least 1 PK concentration datum (PK Population). | microgram/milliliter (µg/mL) | MEAN |
| SECONDARY | Pharmacokinetics: Time to Reach Maximum Observed Plasma Concentration From Time Zero up to 6 Hours After the Morning Dose (Tmax0-6hr) | 0 (predose), 1, 2, 4, and 6 (postdose) hours on Days 1 and 28 | All participants who received at least 1 dose of ataluren and had at least 1 PK concentration datum (PK Population). | hours | MEAN |
| SECONDARY | Area Under the Plasma Concentration Time Curve From Time Zero up to 10 Hours After the Morning Dose (AUC0-10hr) | 0 (predose), 1, 2, 4, 6, 8, and 10 (postdose) hours on Days 1 and 28 | All participants who received at least 1 dose of ataluren and had at least 1 PK concentration datum (PK Population). | hour*μg/mL | MEAN |
| SECONDARY | Pharmacokinetics: Concentration at the End of the First (Morning) Dose Interval (Ctrough6hr) | 0 (predose), 1, 2, 4, and 6 (postdose) hours on Days 1 and 28 | All participants who received at least 1 dose of ataluren and had at least 1 PK concentration datum (PK Population). | microgram per milliliter (µg/ml) | MEAN |
| SECONDARY | Change From Baseline in Proximal Muscle Function as Assessed by Speed During TFTs | Baseline, Week 28 and Week 52 | All participants who received at least 1 dose of ataluren and had a baseline and at least 1 postbaseline measurement for TFT, NSAA, or response to the palatability of ataluren questions (Evaluable Population) and had evaluable data for the applicable timed function test. | seconds | MEAN |
| SECONDARY | Change From Baseline in Physical Function as Measured by the NSAA | Baseline, Week 28 and Week 52 | All participants who received at least 1 dose of ataluren and had a baseline and at least 1 postbaseline measurement for TFT, NSAA, or response to the palatability of ataluren questions (Evaluable Population) and had evaluable NSAA data. | units on a scale | MEAN |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 outcomesResults by group78
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), TEAEs Leading to Discontinuation, and Serious Adverse Events (SAEs) | Ataluren | At least 1 TEAE | 14 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs), TEAEs Leading to Discontinuation, and Serious Adverse Events (SAEs) | Ataluren | Mild TEAE | 5 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs), TEAEs Leading to Discontinuation, and Serious Adverse Events (SAEs) | Ataluren | Moderate TEAE | 8 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs), TEAEs Leading to Discontinuation, and Serious Adverse Events (SAEs) | Ataluren | Severe TEAE | 1 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs), TEAEs Leading to Discontinuation, and Serious Adverse Events (SAEs) | Ataluren | TEAE Related to Study Drug | 5 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs), TEAEs Leading to Discontinuation, and Serious Adverse Events (SAEs) | Ataluren | TEAE Leading to Participant Study Discontinuation | 0 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs), TEAEs Leading to Discontinuation, and Serious Adverse Events (SAEs) | Ataluren | Serious TEAE | 0 | Participants | |
| Number of Participants With a Clinically Meaningful Abnormal Clinical Laboratory (Biochemistry, Hematology, and Urinalysis) Parameter | Ataluren | 0 | Participants | ||
| Number of Participants With a Clinically Meaningful Abnormal Electrocardiogram (ECG) Test Results | Ataluren | 0 | Participants | ||
| Number of Participants With a Dose-Limiting Toxicity as Measured by Hepatic and Renal Toxicity | Ataluren | 0 | participants |
1 to 10 of 78Source ClinicalTrials.gov
See all 78 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Ataluren | 14 | |
| Overall Study | Safety Population | Ataluren | 14 | |
| Overall Study | Pharmacokinetics (PK) Population | Ataluren | 14 | |
| Overall Study | Evaluable Population | Ataluren | 14 | |
| Overall Study | COMPLETED | Ataluren | 14 | |
| Overall Study | NOT COMPLETED | Ataluren | 0 |
Source ClinicalTrials.gov
Adverse events47
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 Upper | Gastrointestinal disorders | Ataluren | 2 | 14 | |
| other | Constipation | Gastrointestinal disorders | Ataluren | 2 | 14 | |
| other | Flatulence | Gastrointestinal disorders | Ataluren | 2 | 14 | |
| other | Nausea | Gastrointestinal disorders | Ataluren | 1 | 14 | |
| other | Stomatitis | Gastrointestinal disorders | Ataluren | 1 | 14 | |
| other | Vomiting | Gastrointestinal disorders | Ataluren | 3 | 14 | |
| other | Fatigue | General disorders | Ataluren | 1 | 14 | |
| other | Gait Disturbance | General disorders | Ataluren | 1 | 14 | |
| other | Pyrexia | General disorders | Ataluren | 6 | 14 | |
| other | Bronchitis Viral | Infections and infestations | Ataluren | 1 | 14 |
1 to 10 of 47Source ClinicalTrials.gov
See all 47 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Ataluren | 0 | 14 |
| Total, other adverse events | Ataluren | 14 | 14 |
| Total, serious adverse events | Ataluren | 0 | 14 |
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.
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.
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GET https://conductscience.org/api/trials/v1/NCT02819557/results?page=2