Facioscapulohumeral Muscular Dystrophy (FSHD)Phase 1/2NCT02239224
Safety, Tolerability, Pharmacokinetics, and Biological Activity of ATYR1940 in Adult Participants With Muscular Dystrophy
A Phase 1/2 study testing ATYR1940 in people with Facioscapulohumeral Muscular Dystrophy (FSHD), run by aTyr Pharma, Inc. at 5 sites in 4 countries.
Trial summary
The purpose of this study is to assess the safety and tolerability profile of ATYR1940 in the treatment of adult participants with molecularly defined genetic muscular dystrophies Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Facioscapulohumeral Muscular Dystrophy (FSHD)
- What is being tested?
- ATYR1940
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 20 participants
- Where
- 5 sitesin 4 countries
- Study dates
- 4 Sep 2014to 14 Dec 2015
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Participant is a male or female aged 18 to 65 years, inclusive.
- Participant has an established, genetically-confirmed, diagnosis of FSHD with clinical findings meeting existing criteria.
- Participant has provided written informed consent after the nature of the study has been explained and prior to the performance of any research-related procedures.
- Participant is, in the Investigator's opinion, willing and able to comply with all study procedures.
- Cohorts ≥2 only: Participant has imaging findings meeting defined criteria for muscle inflammation in at least 1 skeletal muscle.
Who cannot join
- Participant is currently receiving treatment with an immunomodulatory agent or has a history of such treatment, including targeted biological therapies (for example, etanercept, omalizumab) within the 3 months before Baseline; corticosteroids within 4 weeks before Baseline; or high-dose non-steroidal anti-inflammatory agents (NSAIDs) within 2 weeks before Baseline.
- Participant is currently receiving curcumin or albuterol or requires such treatment during study participation.
- Participant has evidence of an alternative diagnosis other than FSHD, based on prior muscle biopsy or genetic test findings.
- Participant has a presumptive diagnosis of FSHD, based on clinical assessment, but does not yet have genetic confirmation of the diagnosis.
- Participant has a severe retinopathy.
- Participant has a history of obstructive or restrictive lung disease (including interstitial lung disease, pulmonary fibrosis, or asthma), or evidence for interstitial lung disease on Screening chest radiograph.
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- Participant has a history of anti-synthetase syndrome, prior Jo-1 antibody (Ab)-positivity, or has a positive or equivocally positive Jo-1 Ab test result during Screening.
- Participant has acute or clinically relevant Epstein-Barr virus or cytomegalovirus infection or re-activation.
- Participant has a chronic infection such as hepatitis B virus, hepatitis C virus, or human immunodeficiency virus or a history of tuberculosis.
- Participant has received a vaccination within 8 weeks before Baseline or vaccination is planned during study participation.
- Participant has symptomatic cardiomyopathy or severe cardiac arrhythmia that may, in the Investigator's opinion, limit the participant's ability to complete the study protocol.
- Participant has anemia (as defined for participant's age and gender by local laboratory range).
- Participant has gamma-glutamyl transferase (GGT) or serum creatinine levels >2 × the upper limit of normal (ULN).
- Participant has abnormal baseline findings, medical condition(s), or laboratory findings that, in the Investigator's opinion, might jeopardize participant's safety or decrease the chance of obtaining satisfactory data needed to achieve the objectives of the study.
- Participant has evidence of clinically significant cardiovascular, pulmonary, hepatic, renal, hematological, metabolic, dermatological, or gastrointestinal disease, or has a condition that requires immediate surgical intervention or other treatment or may not allow safe participation.
- Participant has used any investigational product or device (other than a mobility assistance device) within 30 days before Baseline.
- Participant has received a product intended to enhance muscle growth within 30 days before Baseline.
- Participant underwent muscle biopsy within 30 days before Baseline.
- Participant initiated treatment with a statin or had a significant adjustment to their statin regimen within 3 months before Baseline. (Stable, chronic statin use is permissible.)
- Participant has received a product that putatively enhances muscle growth (for example, insulin-like growth factor, growth hormone) or activity (for example, Coenzyme A) on a chronic basis within 4 weeks before Baseline.
- Participant is unwilling to abstain from strenuous physical activity for 24 hours prior to each study center visit.
- Participant previously received ATYR1940.
- If female and of childbearing potential (premenopausal and not surgically sterile), participant has a positive pregnancy test at Screening or is unwilling to use contraception from the time of Screening through 1 month after the last Study Drug dose. Acceptable methods of birth control include abstinence, barrier methods, hormones, or intra-uterine device.
- If male, participant is unwilling to use a condom plus spermicide during sexual intercourse from the time of Screening through 1 month after the last Study Drug dose.
- Cohorts ≥2 only: Participant has a known contraindication for magnetic resonance imaging (MRI) assessments (for example metal prosthesis or pacemaker) as per local site MRI protocol
Trial locations and contacts
5 sites in 4 countries.
5 sites in 4 countries
United States2 sites
aTyr Pharma Investigative Site
Columbus, Ohio, 43210
aTyr Pharma Investigative Site
Rochester, New York, 14642
About this study
Official title: A Placebo-Controlled, Randomized, Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics (PK), and Biological Activity of ATYR1940 in Adult Patients With Molecularly Defined Genetic Muscular Dystrophies
The purpose of this study is to assess the safety and tolerability profile of ATYR1940 in the treatment of adult participants with molecularly defined genetic muscular dystrophies
Read the detailed description
Study ATYR1940-C-002 is a multi-national, multi-center, double-blind, randomized, placebo-controlled, ascending dose study designed to evaluate the safety, tolerability, PK, immunogenicity, and pharmacodynamic effects of ATYR1940 in participants with FSHD. Up to 44 participants are planned to be enrolled at multiple study centers in the United States and Europe; the actual number of participants enrolled will depend on the number of cohorts initiated.
Participants will be screened for study eligibility during the Screening period within 3 weeks before Baseline (that is, Day 1, the first day of Study Drug administration). Eligible participants, based on Screening assessments, will be randomly assigned to treatment with ATYR1940 or placebo. Participants who are randomized will be considered enrolled in the study.
- Being tested
- Placebo, ATYR1940
- Keywords
- FSHD
- Medical subject headings
- Muscular Dystrophy, Facioscapulohumeral
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PlaceboBiological
Concentrate for solution for infusion
ATYR1940Biological
Concentrate for solution for infusion
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1: ATYR1940 0.3 mg/kg | Experimental | Participants will receive ATYR1940 0.3 milligrams/kilograms (mg/kg) intravenous (IV) infusion once weekly for 4 weeks. Receives: Biological: ATYR1940 |
| Cohort 2: ATYR1940 1.0 mg/kg | Experimental | Participants will receive ATYR1940 1.0 mg/kg IV infusion once weekly for 4 weeks. Receives: Biological: ATYR1940 |
| Cohort 3: ATYR1940 3.0 mg/kg | Experimental | Participants will receive ATYR1940 3.0 mg/kg IV infusion once weekly for 12 weeks. Receives: Biological: ATYR1940 |
| Placebo | Placebo comparator | Participants will receive placebo matched to ATYR1940 IV infusion once weekly for 4 weeks in Cohorts 1 and 2 and for 12 weeks in Cohort 3. Receives: Biological: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Treatment Emergent Adverse Events (TEAEs)
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.
Measured: Up to End of Study (up to Week 25)
Number of Participants With Positive Anti-Drug Antibodies (ADA)
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.
Measured: Baseline up to Week 14
Number of Participants With a Positive or Equivocal Jo-1 Antibody (Ab) Test Result
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.
Measured: Baseline up to Week 14
Number of Participants With a Clinically Significant Laboratory Abnormality
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.
Measured: Baseline up to Week 14
Number of Participants With a Physical Examination Abnormality
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.
Measured: Up to End of Study (up to Week 25)
Number of Participants With a Vital Sign-Related Event Resulting in a TEAE
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.
Measured: Up to End of Study (Up to Week 25)
Number of Participants With a Pulmonary Function Event Resulting in a TEAE
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.
Measured: Up to End of Study (Up to Week 25)
Secondary outcomes (6)
Percent Change From Baseline in Manual Muscle Testing (MMT) Score at 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.
Measured: Baseline, Week 6 and Week 14
Change From Baseline in Individualized Neuromuscular Quality of Life (INQoL) - Overall QoL at 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.
Measured: Baseline, Week 6 and Week 14
Maximum Observed Plasma Concentration (Cmax) of ATYR1940
Measured: 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)
Time to Reach Maximum Observed Plasma Concentration (Tmax) of ATYR1940
Measured: 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)
Area Under the Plasma Concentration Time Curve From Time 0 to Time t (AUC 0-t) of ATYR1940
Measured: 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)
Average of Half-life (T1/2) of ATYR1940
Measured: 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)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Sep 2014 (estimated)
- Study start4 Sep 2014
- Primary completion14 Dec 2015
- Study completion14 Dec 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Facioscapulohumeral Muscular Dystrophy (FSHD)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- aTyr Pharma, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Sanjay Shukla, MD, Study director, aTyr Pharma
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02239224
- Sponsor's protocol ID
- ATYR1940-C-002
- Other IDs
- 2014-001753-17, 2014
- Status last verified
- Jul 2021
- First submitted
- 7 Sep 2014
- Last updated
- 11 Aug 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Aug 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Placebo-Controlled, Randomized, Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics (PK), and Biological Activity of ATYR1940 in Adult Patients With Molecularly Defined Genetic Muscular Dystrophies
- ClinicalTrials.gov ID
- NCT02239224
- Acronym / sponsor ID
- ATYR1940-C-002
- Keywords
- FSHD
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- aTyr Pharma, Inc.Industry (pharma, biotech, devices)
- Study start
- 4 Sep 2014 (actual)
- Primary completion
- 14 Dec 2015 (actual)
- Study completion
- 14 Dec 2015 (actual)
- First posted
- 12 Sep 2014 (estimated)
- Results
- Posted 11 Aug 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to assess the safety and tolerability profile of ATYR1940 in the treatment of adult participants with molecularly defined genetic muscular dystrophies
Detailed description
Study ATYR1940-C-002 is a multi-national, multi-center, double-blind, randomized, placebo-controlled, ascending dose study designed to evaluate the safety, tolerability, PK, immunogenicity, and pharmacodynamic effects of ATYR1940 in participants with FSHD. Up to 44 participants are planned to be enrolled at multiple study centers in the United States and Europe; the actual number of participants enrolled will depend on the number of cohorts initiated.
Participants will be screened for study eligibility during the Screening period within 3 weeks before Baseline (that is, Day 1, the first day of Study Drug administration). Eligible participants, based on Screening assessments, will be randomly assigned to treatment with ATYR1940 or placebo. Participants who are randomized will be considered enrolled in the study.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Cohort 1: ATYR1940 0.3 mg/kg | Experimental | Biological: ATYR1940Participants will receive ATYR1940 0.3 milligrams/kilograms (mg/kg) intravenous (IV) infusion once weekly for 4 weeks. |
| Cohort 2: ATYR1940 1.0 mg/kg | Experimental | Biological: ATYR1940Participants will receive ATYR1940 1.0 mg/kg IV infusion once weekly for 4 weeks. |
| Cohort 3: ATYR1940 3.0 mg/kg | Experimental | Biological: ATYR1940Participants will receive ATYR1940 3.0 mg/kg IV infusion once weekly for 12 weeks. |
| Placebo | Placebo comparator | Biological: PlaceboParticipants will receive placebo matched to ATYR1940 IV infusion once weekly for 4 weeks in Cohorts 1 and 2 and for 12 weeks in Cohort 3. |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Participant is a male or female aged 18 to 65 years, inclusive.
- Participant has an established, genetically-confirmed, diagnosis of FSHD with clinical findings meeting existing criteria.
- Participant has provided written informed consent after the nature of the study has been explained and prior to the performance of any research-related procedures.
- Participant is, in the Investigator's opinion, willing and able to comply with all study procedures.
- Cohorts ≥2 only: Participant has imaging findings meeting defined criteria for muscle inflammation in at least 1 skeletal muscle.
Key exclusion criteria (25 listed)
- Participant is currently receiving treatment with an immunomodulatory agent or has a history of such treatment, including targeted biological therapies (for example, etanercept, omalizumab) within the 3 months before Baseline; corticosteroids within 4 weeks before Baseline; or high-dose non-steroidal anti-inflammatory agents (NSAIDs) within 2 weeks before Baseline.
- Participant is currently receiving curcumin or albuterol or requires such treatment during study participation.
- Participant has evidence of an alternative diagnosis other than FSHD, based on prior muscle biopsy or genetic test findings.
- Participant has a presumptive diagnosis of FSHD, based on clinical assessment, but does not yet have genetic confirmation of the diagnosis.
- Participant has a severe retinopathy.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PlaceboConcentrate for solution for infusion | Biological | Placebo |
| ATYR1940Concentrate for solution for infusion | Biological | Cohort 1: ATYR1940 0.3 mg/kg, Cohort 2: ATYR1940 1.0 mg/kg, Cohort 3: ATYR1940 3.0 mg/kg |
Primary outcome
Number of Participants With Treatment Emergent Adverse Events (TEAEs)
Time frame: 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.
Number of Participants With Positive Anti-Drug Antibodies (ADA)
Time frame: 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.
Number of Participants With a Positive or Equivocal Jo-1 Antibody (Ab) Test Result
Time frame: 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.
Number of Participants With a Clinically Significant Laboratory Abnormality
Time frame: 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.
Number of Participants With a Physical Examination Abnormality
Time frame: 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.
Number of Participants With a Vital Sign-Related Event Resulting in a TEAE
Time frame: 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.
Number of Participants With a Pulmonary Function Event Resulting in a TEAE
Time frame: 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.
6 secondary outcomes
Percent Change From Baseline in Manual Muscle Testing (MMT) Score at Week 6 and Week 14
Time frame: Baseline, Week 6 and Week 14
Change From Baseline in Individualized Neuromuscular Quality of Life (INQoL) - Overall QoL at Week 6 and Week 14
Time frame: Baseline, Week 6 and Week 14
Maximum Observed Plasma Concentration (Cmax) of ATYR1940
Time frame: 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)
Time to Reach Maximum Observed Plasma Concentration (Tmax) of ATYR1940
Time frame: 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)
Area Under the Plasma Concentration Time Curve From Time 0 to Time t (AUC 0-t) of ATYR1940
Time frame: 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)
Average of Half-life (T1/2) of ATYR1940
Time frame: 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)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Aug 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Sanjay Shukla, MD study director
aTyr Pharma
5 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing ATYR1940 in people with Facioscapulohumeral Muscular Dystrophy (FSHD), run by aTyr Pharma, Inc. at 5 sites in 4 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 25 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 11 Aug 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in United States, France, Italy, Netherlands. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 11 Aug 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With Treatment Emergent Adverse Events (TEAEs) (time frame: Up to End of Study (up to Week 25)). The study lists 7 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Aug 2021.