Trial · NCT02836418
Study to Evaluate the Long-Term Safety, Tolerability, and Biological Activity of ATYR1940 in Participants With Limb Girdle and Facioscapulohumeral Muscular Dystrophy (FSHD)
NCT02836418, Phase 1, Phase 2, Completed, aTyr Pharma, Inc.; results first posted 2023-12-22
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study to Evaluate the Long-Term Safety, Tolerability, and Biological Activity of ATYR1940 in Participants With Limb Girdle and Facioscapulohumeral Muscular Dystrophy (FSHD). Trials, record NCT02836418. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02836418
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study to Evaluate the Long-Term Safety, Tolerability, and Biological Activity of ATYR1940 in Participants With Limb Girdle and Facioscapulohumeral Muscular Dystrophy (FSHD)
- NCT number
- NCT02836418
- Status
- Completed
- Phases
- Phase 1, Phase 2
- Enrollment
- 8 participants, actual
- Lead sponsor
- aTyr Pharma, Inc.
- Conditions
- Facioscapulohumeral Muscular Dystrophy, Limb Girdle Muscular Dystrophy
- Interventions
- ATYR1940
- First posted
- 2016-07-19
- Last updated
- 2023-12-22
- Results first posted
- 2023-12-22
Source ClinicalTrials.gov
Registered outcomes9
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) and Serious Adverse Events (SAEs) | Up to End of Study (up to approximately Week 39) | TEAEs were defined as adverse events (AEs) with an onset following administration of the first dose of study drug. AEs were defined as any untoward medical occurrence in a participant administered study drug and that does not necessarily have a causal relationship with the study drug. Worsening of a pre-existing medical condition 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. SAEs were defined as any AE that, in the view of either the Investigator or Sponsor, resulted in any of the following outcomes as fatal, life-threatening, required in-participant hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, a congenital anomaly/birth defect, an important medical event. A summary of all SAEs and Other AEs (nonserious) regardless of causality is located in 'Reported Adverse Events' Section. |
| Primary | Number of Participants With Positive Anti-Drug Antibodies (ADA) | Up to End of Study (up to approximately Week 39) | Summarized titers are reported below. |
| Primary | Number of Participants With a Jo-1 Antibody (Ab) Test Result ≥1.5 Units/Milliliter (U/mL) | Up to End of Study (up to approximately Week 39) | Participants with Jo-1 Ab levels ≥1.5 U/mL were to be discontinued from dosing of the study drug. |
| Primary | Number of Participants With a Clinical Laboratory Abnormality Leading to an AE | Up to End of Study (up to approximately Week 39) | Laboratory parameters included hematology (hematocrit, hemoglobin, red blood cell count, white blood cell count neutrophils, lymphocytes, monocytes, eosinophils, basophils, and platelet count); serum chemistries (blood urea nitrogen, creatinine, total bilirubin, aspartate aminotransferase, alanine aminotransferase, gamma-glutamyl transferase, alkaline phosphatase, total protein, sodium, potassium, bicarbonate, calcium, chloride, magnesium, inorganic phosphate, creatine kinase, lactate dehydrogenase, C-reactive protein, troponin, myoglobin, insulin-like growth factor 1, and cholesterol \[nonfasting\]); and urinalysis (color, pH, specific gravity, protein, glucose, ketones, and blood). Clinically significant laboratory abnormalities were based upon Investigator's discretion. 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 Clinically Significant Pulmonary Function Event Resulting in a TEAE | Up to End of Study (up to approximately Week 39) | Pulmonary evaluations included pulmonary function tests and pulse oximetry. Clinically significant changes were to be reported as adverse events. 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 Clinically Significant Electrocardiogram (ECG) Abnormality Leading to a TEAE | Up to End of Study (up to approximately Week 39) | ECG parameters that were evaluated included heart rate and PR, QR, and QT intervals. A clinically significant ECG abnormality was based upon the Investigator's discretion. 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 Vital Sign Abnormality Resulting in a TEAE | Up to End of Study (up to approximately Week 39) | 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 | Change From Baseline in Manual Muscle Testing (MMT) Score at Week 12 | Baseline, Week 12 | MMT (muscle strength) will be graded using a modified Medical Research Council scale. Scores were converted to 13-point scale (range from 0 \[on contraction palpable\] to 12 \[normal strength\]). An overall total score was calculated by summing all scores for a total possible score of 336. Decreased muscle strength was indicated by a decreased score. An overall total score was calculated as long as 24 of the 28 individual scores were non-missing. |
| Secondary | Change From Baseline in Creatinine Kinase at Week 12 | Baseline, Week 12 |
Source ClinicalTrials.gov
Outcomes9
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) and Serious Adverse Events (SAEs) | Up to End of Study (up to approximately Week 39) | Safety population was defined as all participants who received at least 1 full or partial dose of ATYR1940 in the current study (Study ATYR1940-C-006) and had a post-infusion safety observation since enrollment in parent studies. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Positive Anti-Drug Antibodies (ADA) | Up to End of Study (up to approximately Week 39) | Cumulative Safety population included all participants who received at least 1 full or partial dose of ATYR1940 in the current study (Study ATYR1940-C-006) and had a post-infusion safety observation since enrollment in parent studies, but with cumulative data from both the parent studies and the current study. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With a Jo-1 Antibody (Ab) Test Result ≥1.5 Units/Milliliter (U/mL) | Up to End of Study (up to approximately Week 39) | Cumulative Safety population included all participants who received at least 1 full or partial dose of ATYR1940 in the current study (Study ATYR1940-C-006) and had a post-infusion safety observation since enrollment in parent studies, but with cumulative data from both the parent studies and the current study. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With a Clinical Laboratory Abnormality Leading to an AE | Up to End of Study (up to approximately Week 39) | Safety population was defined as all participants who received at least 1 full or partial dose of ATYR1940 in the current study (Study ATYR1940-C-006) and had a post-infusion safety observation since enrollment in parent studies. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With a Clinically Significant Pulmonary Function Event Resulting in a TEAE | Up to End of Study (up to approximately Week 39) | Safety population was defined as all participants who received at least 1 full or partial dose of ATYR1940 in the current study (Study ATYR1940-C-006) and had a post-infusion safety observation since enrollment in parent studies. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With a Clinically Significant Electrocardiogram (ECG) Abnormality Leading to a TEAE | Up to End of Study (up to approximately Week 39) | Safety population was defined as all participants who received at least 1 full or partial dose of ATYR1940 in the current study (Study ATYR1940-C-006) and had a post-infusion safety observation since enrollment in parent studies. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Vital Sign Abnormality Resulting in a TEAE | Up to End of Study (up to approximately Week 39) | Safety population was defined as all participants who received at least 1 full or partial dose of ATYR1940 in the current study (Study ATYR1940-C-006) and had a post-infusion safety observation since enrollment in parent studies. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Change From Baseline in Manual Muscle Testing (MMT) Score at Week 12 | Baseline, Week 12 | Cumulative Safety population included all participants who received at least 1 full or partial dose of ATYR1940 in the current study (Study ATYR1940-C-006) and had a post-infusion safety observation since enrollment in parent studies, but with cumulative data from both the parent studies and the current study. Here, 'Number of Participants analyzed' signifies those who were evaluable for this outcome measure and number analyzed signifies those who were evaluable at specified time points only. | score on a scale | MEAN |
| SECONDARY | Change From Baseline in Creatinine Kinase at Week 12 | Baseline, Week 12 | Cumulative Safety population included all participants who received at least 1 full or partial dose of ATYR1940 in the current study (Study ATYR1940-C-006) and had a post-infusion safety observation since enrollment in parent studies, but with cumulative data from both the parent studies and the current study. Here, 'Number of Participants analyzed' signifies those who were evaluable for this outcome measure. | Units/liter | MEAN |
Source ClinicalTrials.gov
Results by group11
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) and Serious Adverse Events (SAEs) | ATYR1940 | TEAEs | 7 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | ATYR1940 | SAEs | 0 | Participants | |
| Number of Participants With Positive Anti-Drug Antibodies (ADA) | ATYR1940 | 3 | Participants | ||
| Number of Participants With a Jo-1 Antibody (Ab) Test Result ≥1.5 Units/Milliliter (U/mL) | ATYR1940 | 2 | Participants | ||
| Number of Participants With a Clinical Laboratory Abnormality Leading to an AE | ATYR1940 | 1 | Participants | ||
| Number of Participants With a Clinically Significant Pulmonary Function Event Resulting in a TEAE | ATYR1940 | 0 | Participants | ||
| Number of Participants With a Clinically Significant Electrocardiogram (ECG) Abnormality Leading to a TEAE | ATYR1940 | 2 | Participants | ||
| Number of Participants With Vital Sign Abnormality Resulting in a TEAE | ATYR1940 | 0 | Participants | ||
| Change From Baseline in Manual Muscle Testing (MMT) Score at Week 12 | ATYR1940 | Baseline | 229.1 | Standard Deviation 82.56 | score on a scale |
| Change From Baseline in Manual Muscle Testing (MMT) Score at Week 12 | ATYR1940 | Change at Week 12 | 4.3 | Standard Deviation 13.50 | score on a scale |
1 to 10 of 11Source ClinicalTrials.gov
See all 11 results by groupParticipant flow4
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | ATYR1940 | 8 | |
| Overall Study | Received at Least One Dose of Study Drug | ATYR1940 | 8 | |
| Overall Study | COMPLETED | ATYR1940 | 8 | |
| Overall Study | NOT COMPLETED | ATYR1940 | 0 |
Source ClinicalTrials.gov
Adverse events35
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 | Back pain | Musculoskeletal and connective tissue disorders | ATYR1940 | 3 | 8 | |
| other | Myalgia | Musculoskeletal and connective tissue disorders | ATYR1940 | 3 | 8 | |
| other | Pain in extremity | Musculoskeletal and connective tissue disorders | ATYR1940 | 3 | 8 | |
| other | Arthralgia | Musculoskeletal and connective tissue disorders | ATYR1940 | 1 | 8 | |
| other | Arthropathy | Musculoskeletal and connective tissue disorders | ATYR1940 | 1 | 8 | |
| other | Muscular weakness | Musculoskeletal and connective tissue disorders | ATYR1940 | 1 | 8 | |
| other | Musculoskeletal chest pain | Musculoskeletal and connective tissue disorders | ATYR1940 | 1 | 8 | |
| other | Musculoskeletal pain | Musculoskeletal and connective tissue disorders | ATYR1940 | 1 | 8 | |
| other | Musculoskeletal stiffness | Musculoskeletal and connective tissue disorders | ATYR1940 | 1 | 8 | |
| other | Nausea | Gastrointestinal disorders | ATYR1940 | 4 | 8 |
1 to 10 of 35Source ClinicalTrials.gov
See all 35 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | ATYR1940 | 0 | 8 |
| Total, other adverse events | ATYR1940 | 7 | 8 |
| Total, serious adverse events | ATYR1940 | 0 | 8 |
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.
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/NCT02836418
GET https://conductscience.org/api/trials/v1/NCT02836418/events?page=2