Trial · NCT02239224
Safety, Tolerability, Pharmacokinetics, and Biological Activity of ATYR1940 in Adult Participants With Muscular Dystrophy
NCT02239224, Phase 1, Phase 2, Completed, aTyr Pharma, Inc.; results first posted 2021-08-11
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Safety, Tolerability, Pharmacokinetics, and Biological Activity of ATYR1940 in Adult Participants With Muscular Dystrophy. Trials, record NCT02239224. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02239224
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Safety, Tolerability, Pharmacokinetics, and Biological Activity of ATYR1940 in Adult Participants With Muscular Dystrophy
- NCT number
- NCT02239224
- Status
- Completed
- Phases
- Phase 1, Phase 2
- Enrollment
- 20 participants, actual
- Lead sponsor
- aTyr Pharma, Inc.
- Conditions
- Facioscapulohumeral Muscular Dystrophy (FSHD)
- Interventions
- Placebo, ATYR1940
- First posted
- 2014-09-12
- Last updated
- 2021-08-11
- Other registries' IDs
- eudract:2014-001753-17
- Results first posted
- 2021-08-11
Source ClinicalTrials.gov
Registered outcomes13
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) | Up to End of Study (up to Week 25) | TEAEs were defined as adverse events (AEs) with an onset following administration of the first dose of study drug. An AE was defined as any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with study drug. Worsening of a pre-existing medical condition, (that is, diabetes, migraine headaches, gout) should have been considered an AE if there was either an increase in severity, frequency, or duration of the condition or an association with significantly worse outcomes. Classification of AEs was to be done by the Investigator according to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE), version 4.03. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section. |
| Primary | Number of Participants With Positive Anti-Drug Antibodies (ADA) | Baseline up to Week 14 | Titers through Week 14 are summarized. For Cohorts 1 and 2, samples that screened positive but did not confirm on confirmatory assay were not titered. |
| Primary | Number of Participants With a Positive or Equivocal Jo-1 Antibody (Ab) Test Result | Baseline up to Week 14 | Criterion for a positive Jo-1 Ab test result: \>10.0 units/milliliter (U/mL), a cut-point associated with anti-synthetase syndrome. Criterion for an equivocal Jo-1 Ab test result: 7.0 to 10.0 U/mL. Participants were required to have a negative Jo-1 Ab test result (defined as \<7 U/mL) for inclusion in the study as well as to continue dosing with study drug during the study. |
| Primary | Number of Participants With a Clinically Significant Laboratory Abnormality | Baseline up to Week 14 | Laboratory parameters included hematology (hematocrit, hemoglobin, platelet count, red blood cell count, white blood cell count, neutrophils, lymphocytes, monocytes, eosinophils, basophils); serum chemistries (blood urea nitrogen, creatinine, total bilirubin, aspartate aminotransferase, alanine aminotransferase, gamma-glutamyl transferase (GGT), alkaline phosphatase, sodium, total protein, bicarbonate, potassium, calcium, chloride, magnesium, inorganic phosphate, creatine phosphokinase \[will be fractionated if elevated\], lactic dehydrogenase, erythrocyte sedimentation rate, C-reactive protein, troponin, myoglobin, insulin-like growth factor 1, cholesterol \[non-fasting\]); Urinalysis (Color, pH, specific gravity, protein, glucose, ketones, blood). Clinically significant laboratory abnormalities were based upon Investigator's discretion. A summary of all Serious AEs and Other AEs (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section. |
| Primary | Number of Participants With a Physical Examination Abnormality | Up to End of Study (up to Week 25) | Body systems that were evaluated during the physical examination included general appearance, head, eyes, ears, nose, throat (HEENT), cardiovascular system, respiratory system, chest (breasts), gastrointestinal system (abdomen), lymphatic system, musculoskeletal system, skin, psychiatric, and neurologic. Neurologic examination included assessment of mental status, memory, cranial nerves, motor function, and reflexes, and sensory testing. The body systems with at least 1 physical abnormality is presented. One participant could be represented in more than 1 body system category. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section. |
| Primary | Number of Participants With a Vital Sign-Related Event Resulting in a TEAE | Up to End of Study (Up to Week 25) | The vital sign parameters that were evaluated included heart rate, systolic and diastolic blood pressure, and respiration rate as well as temperature. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section. |
| Primary | Number of Participants With a Pulmonary Function Event Resulting in a TEAE | Up to End of Study (Up to Week 25) | Pulmonary function testing included measurements of forced vital capacity (FVC) and forced expiratory volume in 1 second (FEV1). A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section. |
| Secondary | Percent Change From Baseline in Manual Muscle Testing (MMT) Score at Week 6 and Week 14 | Baseline, Week 6 and Week 14 | MMT was graded on a scale from 0 (no movement) to 10 (normal movement). Each side of the body and the position in which each muscle was tested were recorded for each participant. The total MMT score were calculated by averaging a converted MMT scores across all tested muscle groups. Decreased motor function was indicated by decreased individual muscle or composite MMT score. |
| Secondary | Change From Baseline in Individualized Neuromuscular Quality of Life (INQoL) - Overall QoL at Week 6 and Week 14 | Baseline, Week 6 and Week 14 | The INQoL is a validated muscle disease-specific measure of quality of life. The self-administered questionnaire consisted of 45 questions with 4 domains measuring the impact of common muscle disease symptoms (weakness, locking \[or myotonia\], pain, and fatigue); 5 domains measuring the influence of the muscle disease on particular areas of life (activities, independence, relationships, emotions, and body image); and the last domain focused on the positive and negative effects of treatment and was divided into 2 scores. All responses were given in a 7-point Likert scale, with improved QoL indicated by decreased scores. Overall QoL score was calculated from preselected 5 domains (activities, independence, relationships, emotions, and body image) by adding the scores from each domain. In summary, INQoL yielded 11 scores and 1 QoL score. The Overall scoring used a scale of 0-100, with improved QoL indicated by decreased scores. |
| Secondary | Maximum Observed Plasma Concentration (Cmax) of ATYR1940 | Cohort 1: Predose, 0.25, 0.5, 0.75, 1.0, 1.5, 2.0, 3.0, 4.0, 6.0, and 8.0 hours after the start of infusion at Weeks 2 and 5; Cohorts 2 and 3: Predose, 0.5, 1.0, 2.0, 4.0, and 6.0 hours after the start of infusion at Weeks 2, 5, and 13 (Cohort 3 only) |
1 to 10 of 13Source ClinicalTrials.gov
See all 13 registered outcomesOutcomes13
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) | Up to End of Study (up to Week 25) | ITT analysis set included all participants who were randomized and received at least 1 dose (full or partial) of study drug. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Positive Anti-Drug Antibodies (ADA) | Baseline up to Week 14 | ITT analysis set included all participants who were randomized and received at least 1 dose (full or partial) of study drug. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With a Positive or Equivocal Jo-1 Antibody (Ab) Test Result | Baseline up to Week 14 | ITT analysis set included all participants who were randomized and received at least 1 dose (full or partial) of study drug. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With a Clinically Significant Laboratory Abnormality | Baseline up to Week 14 | ITT analysis set included all participants who were randomized and received at least 1 dose (full or partial) of study drug. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With a Physical Examination Abnormality | Up to End of Study (up to Week 25) | ITT analysis set included all participants who were randomized and received at least 1 dose (full or partial) of study drug. Here, 'Overall Number of Participants Analyzed' (N) signifies number of participants evaluable for this outcome measure. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With a Vital Sign-Related Event Resulting in a TEAE | Up to End of Study (Up to Week 25) | ITT analysis set included all participants who were randomized and received at least 1 dose (full or partial) of study drug. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With a Pulmonary Function Event Resulting in a TEAE | Up to End of Study (Up to Week 25) | ITT analysis set included all participants who were randomized and received at least 1 dose (full or partial) of study drug. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Percent Change From Baseline in Manual Muscle Testing (MMT) Score at Week 6 and Week 14 | Baseline, Week 6 and Week 14 | ITT analysis set included all participants who were randomized and received at least 1 dose (full or partial) of study drug. Here, 'Number Analyzed' signifies participants evaluable at specified timepoints. | percent change | MEAN |
| SECONDARY | Change From Baseline in Individualized Neuromuscular Quality of Life (INQoL) - Overall QoL at Week 6 and Week 14 | Baseline, Week 6 and Week 14 | Per-Protocol (PP) analysis set included all participants in the ITT analysis set with no major protocol deviations and ≥1 post-baseline pharmacodynamic (PD) or clinical parameter assessed. Change from baseline included only those participants with both a baseline value and a value for the summarized time period. Here, 'Number Analyzed' signifies participants evaluable at specified timepoints. | score on a scale | MEAN |
| SECONDARY | Maximum Observed Plasma Concentration (Cmax) of ATYR1940 | Cohort 1: Predose, 0.25, 0.5, 0.75, 1.0, 1.5, 2.0, 3.0, 4.0, 6.0, and 8.0 hours after the start of infusion at Weeks 2 and 5; Cohorts 2 and 3: Predose, 0.5, 1.0, 2.0, 4.0, and 6.0 hours after the start of infusion at Weeks 2, 5, and 13 (Cohort 3 only) | Pharmacokinetic (PK) analysis set included all participants who had baseline samples and sufficient samples collected post-baseline to permit analysis of PK parameters. Here, 'Number Analyzed' signifies participants evaluable at specified timepoints. | nanogram/milliliter | MEAN |
1 to 10 of 13Source ClinicalTrials.gov
See all 13 outcomesResults by group92
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) | Cohort 1: ATYR1940 0.3 mg/kg | 3 | Participants | ||
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) | Cohort 2: ATYR1940 1.0 mg/kg | 6 | Participants | ||
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) | Cohort 3: ATYR1940 3.0 mg/kg | 6 | Participants | ||
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) | Placebo | 5 | Participants | ||
| Number of Participants With Positive Anti-Drug Antibodies (ADA) | Cohort 1: ATYR1940 0.3 mg/kg | 2 | Participants | ||
| Number of Participants With Positive Anti-Drug Antibodies (ADA) | Cohort 2: ATYR1940 1.0 mg/kg | 1 | Participants | ||
| Number of Participants With Positive Anti-Drug Antibodies (ADA) | Cohort 3: ATYR1940 3.0 mg/kg | 3 | Participants | ||
| Number of Participants With Positive Anti-Drug Antibodies (ADA) | Placebo | 0 | Participants | ||
| Number of Participants With a Positive or Equivocal Jo-1 Antibody (Ab) Test Result | Cohort 1: ATYR1940 0.3 mg/kg | 0 | Participants | ||
| Number of Participants With a Positive or Equivocal Jo-1 Antibody (Ab) Test Result | Cohort 2: ATYR1940 1.0 mg/kg | 0 | Participants |
1 to 10 of 92Source ClinicalTrials.gov
See all 92 results by groupParticipant flow16
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Cohort 1: ATYR1940 0.3 mg/kg | 3 | |
| Overall Study | STARTED | Cohort 2: ATYR1940 1.0 mg/kg | 6 | |
| Overall Study | STARTED | Cohort 3: ATYR1940 3.0 mg/kg | 6 | |
| Overall Study | STARTED | Placebo | 5 | |
| Overall Study | Received at Least One Dose of Study Drug | Cohort 1: ATYR1940 0.3 mg/kg | 3 | |
| Overall Study | Received at Least One Dose of Study Drug | Cohort 2: ATYR1940 1.0 mg/kg | 6 | |
| Overall Study | Received at Least One Dose of Study Drug | Cohort 3: ATYR1940 3.0 mg/kg | 6 | |
| Overall Study | Received at Least One Dose of Study Drug | Placebo | 5 | |
| Overall Study | COMPLETED | Cohort 1: ATYR1940 0.3 mg/kg | 3 | |
| Overall Study | COMPLETED | Cohort 2: ATYR1940 1.0 mg/kg | 6 |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 participant flowAdverse events220
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 | Eyelid Irritation | Eye disorders | Cohort 1: ATYR1940 0.3 mg/kg | 0 | 3 | |
| other | Eyelid Irritation | Eye disorders | Cohort 2: ATYR1940 1.0 mg/kg | 1 | 6 | |
| other | Eyelid Irritation | Eye disorders | Cohort 3: ATYR1940 3.0 mg/kg | 0 | 6 | |
| other | Eyelid Irritation | Eye disorders | Placebo | 0 | 5 | |
| other | Scintillating Scotoma | Eye disorders | Cohort 1: ATYR1940 0.3 mg/kg | 0 | 3 | |
| other | Scintillating Scotoma | Eye disorders | Cohort 2: ATYR1940 1.0 mg/kg | 0 | 6 | |
| other | Scintillating Scotoma | Eye disorders | Cohort 3: ATYR1940 3.0 mg/kg | 1 | 6 | |
| other | Scintillating Scotoma | Eye disorders | Placebo | 0 | 5 | |
| other | Abdominal Pain Upper | Gastrointestinal disorders | Cohort 1: ATYR1940 0.3 mg/kg | 0 | 3 | |
| other | Abdominal Pain Upper | Gastrointestinal disorders | Cohort 2: ATYR1940 1.0 mg/kg | 1 | 6 |
1 to 10 of 220Source ClinicalTrials.gov
See all 220 adverse eventsAdverse event totals12
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Cohort 1: ATYR1940 0.3 mg/kg | ||
| Total, all-cause mortality | Cohort 2: ATYR1940 1.0 mg/kg | ||
| Total, all-cause mortality | Cohort 3: ATYR1940 3.0 mg/kg | ||
| Total, all-cause mortality | Placebo | ||
| Total, other adverse events | Cohort 1: ATYR1940 0.3 mg/kg | 3 | 3 |
| Total, other adverse events | Cohort 2: ATYR1940 1.0 mg/kg | 6 | 6 |
| Total, other adverse events | Cohort 3: ATYR1940 3.0 mg/kg | 6 | 6 |
| Total, other adverse events | Placebo | 5 | 5 |
| Total, serious adverse events | Cohort 1: ATYR1940 0.3 mg/kg | 0 | 3 |
| Total, serious adverse events | Cohort 2: ATYR1940 1.0 mg/kg | 0 | 6 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 adverse event totalsPapers0
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.
Data dates
The day each source was copied for this release.
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/NCT02239224
GET https://conductscience.org/api/trials/v1/NCT02239224/results?page=2