A Phase 2 interventional study of M5049 dose and Placebo in Dermatomyositis and Polymyositis, sponsored by EMD Serono Research & Development Institute, Inc.. Terminated at 31 sites in 7 countries. Open to participants aged 18 Years to 75 Years. Per ClinicalTrials.gov, last updated 2026-09-09.
Sponsored by EMD Serono Research & Development Institute, Inc. (part of Merck KGaA, Darmstadt, Germany) · Phase 2, Interventional, and Treatment
The purpose of this study is to evaluate the efficacy and safety of orally administered M5049 in idiopathic inflammatory myopathies, specifically dermatomyositis (DM) and polymyositis (PM) participants for 24 weeks.
184 studies on the registry are indexed under Dermatomyositis; 63 are open to participants now.
This study's enrollment of 20 is below the median of 35 across 130 interventional studies indexed under Dermatomyositis.
Browse Dermatomyositis studies →EMD Serono Research & Development Institute, Inc. is the lead sponsor of 87 studies on the registry; 8 are open to participants now.
Of its 47 completed or terminated interventional studies of FDA-regulated products, 38 (81%) have results posted.
First submitted after EMD Serono Research & Development Institute, Inc. became part of Merck KGaA, Darmstadt, Germany.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Weight in kilograms divided by height in metres squared. Example: 70 kg and 1.75 m gives 70 ÷ (1.75 × 1.75) ≈ 22.9.
What counts as overweight or obesity differs between countries (some use lower cut-offs), so go by the range the study states.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Biopsy and archival tissue
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Immunosuppression
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
BMI (body mass index)
Weight in kilograms divided by height in metres squared. Example: 70 kg and 1.75 m gives 70 ÷ (1.75 × 1.75) ≈ 22.9.
What counts as overweight or obesity differs between countries (some use lower cut-offs), so go by the range the study states.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
GFR and eGFR (kidney filtering)
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Drug: M5049 dose
Drug: Placebo
Drug: M5049 dose
Participants will receive film-coated tablets of M5049 dose orally, twice daily up to 24 weeks.
Also known as: Enpatoran
Participants will receive placebo matched to M5049 orally, twice daily up to 24 weeks.
Double Blind Placebo Control Period (DBPC): American College of Rheumatology/European League Against Rheumatism (ACR/EULAR) Total Improvement Score (TIS) at Week 24
The ACR/EULAR TIS was derived from evaluation of results from 6 Core Set Measures(CSMs) of myositis disease activity in participants with Dermatomyositis and Polymyositis.TIS was determined by summing scores for each CSM,based on improvement in absolute percent change which is calculated by subtracting the baseline value from the final value,dividing difference by the range, and then multiplying the result by100 and relative weight of each core set measure. The TIS scale ranges from 0 to 100 with thresholds for minimal \[more than or equal to(\>= 20)\],moderate(\>= 40) and major(\>= 60)improvement in combined 6 CSM.The CSMs include,physician global activity visual analogue scale(VAS) score(0-10), patient global activity VAS score(0-10), manual muscle testing-8 score(0-150), health assessment questionnaire-disability index(0-3) along with checks for most abnormal muscle-associated enzymes and extra muscular activity assessment derived using myositis disease activity assessment tool(0-10).
Time frame: At Week 24 (end of DBPC period)
DBPC Period: Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs and Adverse Events of Special Interest (AESIs)
An AE was defined as any untoward medical occurrence in a participant administered a pharmaceutical product and which did not necessarily have a causal relationship with this treatment. An AE could therefore be any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a medicinal product or protocol-specified procedure, whether considered related to the medicinal product or protocol-specified procedure. Serious AE was defined AE that resulted in any of the following outcomes: death; life threatening; persistent/significant disability/incapacity; initial/prolonged inpatient hospitalization; congenital anomaly/birth defect. TEAE was defined as events with onset date or worsening during the on-treatment period. TEAEs included serious TEAEs and non-serious TEAEs. Adverse events of special interest (AESI) are serious or non-serious AEs that are of clinical interest and were closely followed.
Time frame: Up to Week 26 (Safety follow up)
DBPC Period: Number of Participants With Clinically Meaningful Changes From Baseline in Laboratory Parameters
Laboratory parameters included hematology, biochemistry, urinalysis and reflex testing for HBV DNA. Number of participants with clinically meaningful changes from baseline in laboratory parameters were reported. Clinical meaningfulness was assessed by the investigator.
Time frame: Up to Week 26 (Safety follow up)
DBPC Period: Number of Participants With Clinically Meaningful Changes From Baseline in Vital Signs
Vital signs included body temperature, systolic and diastolic blood pressure, respiration rate, pulse rate, weight, height and body mass index. Number of participants with clinically meaningful changes from baseline in vital signs were reported. Clinical meaningfulness was assessed by the investigator.
Time frame: Up to Week 26 (Safety follow up)
DBPC Period: Number of Participants With Clinically Meaningful Changes From Baseline in Electrocardiograms (ECG) Parameters
12-lead ECG recordings included rhythm, heart rate (as measured by RR interval), PR interval, QRS duration, and QT interval. Number of participants with clinically meaningful changes from baseline in ECG parameters were reported. Clinical meaningfulness was assessed by the investigator.
Time frame: Up to Week 26 (Safety follow up)
DBPC Period: Number of Participants With American College of Rheumatology/European League Against Rheumatism (ACR/EULAR) Total Improvement Score (TIS) Greater Than or Equal to (>=) 20, >= 40 and >= 60
The ACR/EULAR TIS was derived from evaluation of results from 6 Core Set Measures(CSMs) of myositis disease activity in participants with Dermatomyositis and Polymyositis.TIS was determined by summing scores for each CSM,based on improvement in absolute percent change which is calculated by subtracting the baseline value from the final value,dividing difference by the range, and then multiplying the result by100 and relative weight of each core set measure. The TIS scale ranges from 0 to 100 with thresholds for minimal \[more than or equal to(\>= 20)\],moderate(\>=40) and major(\>=60)improvement in combined 6 CSM.The CSMs include,physician global activity visual analogue scale(VAS) score(0-10), patient global activity VAS score(0-10), manual muscle testing-8 score(0-150), health assessment questionnaire-disability index(0-3) along with checks for most abnormal muscle-associated enzymes and extra muscular activity assessment derived using myositis disease activity assessment tool(0-10).
Time frame: At Week 16 and Week 24
DBPC Period: Participant's Total Improvement Score (TIS)
The ACR/EULAR TIS was derived from evaluation of results from 6 Core Set Measures(CSMs) of myositis disease activity in participants with Dermatomyositis and Polymyositis.TIS was determined by summing scores for each CSM,based on improvement in absolute percent change which is calculated by subtracting the baseline value from the final value,dividing difference by the range, and then multiplying the result by100 and relative weight of each core set measure. The TIS scale ranges from 0 to 100 with thresholds for minimal \[more than or equal to(\>= 20)\],moderate(\>=40) and major(\>=60)improvement in combined 6 CSM.The CSMs include,physician global activity visual analogue scale(VAS) score(0-10), patient global activity VAS score(0-10), manual muscle testing-8 score(0-150), health assessment questionnaire-disability index(0-3) along with checks for most abnormal muscle-associated enzymes and extra muscular activity assessment derived using myositis disease activity assessment tool(0-10).
Time frame: Week 4, 8, 12, 16, 20 and 24
DBPC Period: Change From Baseline in Physician Global Activity (PGA) Scores From Myositis Disease Activity Assessment Tool (MDAAT)
The Physician's Global Assessment of Disease Activity was an assessment of overall global disease activity by the physician that was taken from Myositis Disease Activity Assessment Tool (MDAAT). Physician rated participant's disease activity on a 0-10 centimeter (cm) visual analog scale (VAS). Extra-muscular activity ranged between 0 and 10, where, 0 cm = absent and 10 cm = maximum disease activity.
Time frame: Baseline, Week 4, 8, 12, 16, 20 and 24
DBPC Period: Change From Baseline in Patient Global Activity (PtGA) Visual Analog Scale (VAS) Score
PtGA was the assessment of the severity of disease by the participant/participant's guardian, using a VAS from 0 mm (no evidence of disease activity) to 100 mm (extremely active or severe disease activity). Higher score indicated worse status.
Time frame: Baseline, Week 4, 8, 12, 16, 20 and 24
DBPC Period: Change From Baseline in Extra Muscular Global Assessment From Myositis Disease Activity Assessment Tool (MDAAT) Score
MDAAT was a combined tool capturing physician's assessment of disease activity of various organ systems- constitutional, cutaneous, pulmonary, gastrointestinal, joints and cardiovascular, muscular using: Myositis Intention to Treat Activity Index (MITAX), a scale from 0("Not present in the last 4 weeks") to 4("New - in the last 4 weeks \[compared to the previous 4 weeks\]") and Myositis Disease Activity Assessment Visual Analogue Scales (MYOACT), a 10 cm VAS assessing disease activity in the last 4 weeks of various organ systems from 0 cm("No evidence of disease activity") to 10 cm("Extremely active or severe disease activity"), disease activity being defined as potentially reversible pathology or physiology resulting from the myositis. Left end of line = no evidence of disease activity, midpoint of line = moderate disease activity, and right end of line = extreme or maximum disease activity. An assessment of extramuscular activity assessment was taken from MDAAT using a 10cm VAS scale.
Time frame: Baseline, Week 4, 8, 12, 16, 20 and 24
DBPC Period: Change From Baseline in Manual Muscle Testing-8 (MMT-8) Score
MMT-8 was an International Myositis Assessment and Clinical Studies (IMACS) Core Set Measure used in the calculation of the IMACS Definition of Improvement (DOI) and ACR/EULAR TIS. It is a set of 8 designated muscles tested unilaterally (generally on right side) -which include 1 axial, 5 proximal (2 upper extremity, 3 lower extremity), and 2 distal muscles (1 upper, 1 lower extremity). Total score for unilateral testing ranges from 0 to 80, and bilateral testing ranges from 0 to 150 with normal strength represented by a higher score at or near the top of the scale. The total score for bilateral testing (item "MMT8 score (0 - 150)" from the MANUAL MUSCLE TESTING-8 (mmt8) form) is used in the derivation of the Total Improvement Score.
Time frame: Baseline, Week 4, 8, 12, 16, 20 and 24
DBPC Period: Change From Baseline in Health Assessment Questionnaire -Disability Index (HAQ-DI) Score
HAQ-DI was an IMACS Core Set Measure used in the calculation of the IMACS DOI and ACR/EULAR TIS. It is a generic rather than a disease-specific instrument comprised of 8 sections (dressing, arising, eating, walking, hygiene, reach, grip, and activities), with 2 to 3 questions per section. Scoring within each section is from 0 (without any difficulty) to 3 (unable to do). For each section the score given to that section is the worst score within the section. The 8 scores of the 8 sections are summed and divided by 8. Overall score ranges from 0-3.
Time frame: Baseline, Week 4, 8, 12, 16, 20 and 24
DBPC Period: Relative Change From Baseline in Most Abnormal Muscle-associated Enzyme
For each participant, the most abnormal muscle-associated enzyme was defined as the baseline muscle-associated enzyme with the highest ratio of the observed value to the upper limit of the normal range (ULN) among creatinine kinase, lactate dehydrogenase, aspartate aminotransferase, alanine aminotransferase, and aldolase. The relative abnormality could be expressed as a percentage of ULN, calculated as (enzyme value divided by ULN) multiplied by 100.
Time frame: Baseline, Week 4, 8, 12, 16, 20 and 24
DBPC Period: Number of Participants Who Achieved International Myositis Assessment and Clinical Studies (IMACS) Response
A participant was defined as an International Myositis Assessment and Clinical Studies (IMACS) Responder if 3 of any 6 IMACS Core Set Measures (CSMs) have been improved by more than equal to (\>= 20) percent (%), with no more than 2 IMACS CSMs worse by \>= 25%. The CSMs include, physician global activity visual analogue scale(VAS) score(0-10), patient global activity VAS score(0-10), manual muscle testing-8 score(0-150), health assessment questionnaire-disability index (0-3) along with checks for muscle-associated enzymes and extra muscular activity assessment derived using myositis disease activity assessment tool(0-10).
Time frame: At Week 16 and 24
DBPC Period: Change From Baseline in Cutaneous Dermatomyositis Area and Severity Index-A (CDASI-A) and Cutaneous Dermatomyositis Area and Severity Index-D (CDASI-D) Subscale Score
CDASI is a clinician-scored single page instrument that separately measures activity and damage in the skin of DM participants. CDASI has 3 activity measures (CDASI-A: erythema, scale, and erosion/ulceration) and 2 damage measures (CDASI-D: poikiloderma and calcinosis) which are assessed over 15 body areas. In addition, Gottron's papules on the hands, periungual changes and alopecia are evaluated separately both for activity and damage. Activity and Damage Subscale scores range from 0 to 100 and 0 to 32, respectively, where higher scores indicate greater disease severity.
Time frame: Baseline, Week 4, 8, 12, 16, 20 and 24
DBPC Period: Change From Baseline in Investigator's Global Assessment (IGA) Skin Activity Score
The IGA skin activity is a five-point score that defines the level of disease severity based on overall lesion characteristics where 0 is "clear" and "4" is severe.
Time frame: Baseline, Week 4, 8, 12, 16, 20 and 24
Open-label Extension (OLE) Period: Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs and Adverse Events of Special Interest (AESIs)
An AE was defined as any untoward medical occurrence in a participant administered a pharmaceutical product and which did not necessarily have a causal relationship with this treatment. An AE could therefore be any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a medicinal product or protocol-specified procedure, whether considered related to the medicinal product or protocol-specified procedure. Serious AE was defined AE that resulted in any of the following outcomes: death; life threatening; persistent/significant disability/incapacity; initial/prolonged inpatient hospitalization; congenital anomaly/birth defect. TEAE was defined as events with onset date or worsening during the on-treatment period. TEAEs included serious TEAEs and non-serious TEAEs. Adverse events of special interest (AESI) are serious or non-serious AEs that are of clinical interest and were closely followed.
Time frame: From start of OLE period (Week 1) up to safety follow up (Week 26)
OLE Period: Number of Participants With Clinically Meaningful Changes From Baseline in Laboratory Parameters
Laboratory parameters included hematology, biochemistry, urinalysis and reflex testing for HBV DNA. Number of participants with clinically meaningful changes from baseline in laboratory parameters were reported. Clinical meaningfulness was assessed by the investigator.
Time frame: From start of OLE period (Week 1) up to safety follow up (Week 26)
OLE Period: Number of Participants With Clinically Meaningful Changes From Baseline in Vital Signs
Vital signs included body temperature, systolic and diastolic blood pressure, respiration rate, pulse rate, weight, height and body mass index. Number of participants with clinically meaningful changes from baseline in vital signs were reported. Clinical meaningfulness was assessed by the investigator.
Time frame: From start of OLE period (Week 1) up to safety follow up (Week 26)
OLE Period: Number of Participants With Clinically Meaningful Changes From Baseline in Electrocardiograms (ECG) Parameters
12-lead ECG recordings included rhythm, heart rate (as measured by RR interval), PR interval, QRS duration, and QT interval. Number of participants with clinically meaningful changes from baseline in ECG parameters were reported. Clinical meaningfulness was assessed by the investigator.
Time frame: From start of OLE period (Week 1) up to safety follow up (Week 26)
| Milestone | Placebo | Enpatoran 100 mg BID |
|---|---|---|
| Started | 9 | 11 |
| Completed | 6 | 6 |
| Not completed | 3 | 5 |
| Withdrew: Adverse event | 2 | 2 |
| Withdrew: Withdrawal by subject | 0 | 1 |
| Withdrew: Other | 1 | 2 |
| Milestone | Placebo | Enpatoran 100 mg BID |
|---|---|---|
| Started | 5 | 6 |
| Completed | 4 | 4 |
| Not completed | 1 | 2 |
| Withdrew: Other | 1 | 2 |
The ACR/EULAR TIS was derived from evaluation of results from 6 Core Set Measures(CSMs) of myositis disease activity in participants with Dermatomyositis and Polymyositis.TIS was determined by summing scores for each CSM,based on improvement in absolute percent change which is calculated by subtracting the baseline value from the final value,dividing difference by the range, and then multiplying the result by100 and relative weight of each core set measure. The TIS scale ranges from 0 to 100 with thresholds for minimal \[more than or equal to(\>= 20)\],moderate(\>= 40) and major(\>= 60)improvement in combined 6 CSM.The CSMs include,physician global activity visual analogue scale(VAS) score(0-10), patient global activity VAS score(0-10), manual muscle testing-8 score(0-150), health assessment questionnaire-disability index(0-3) along with checks for most abnormal muscle-associated enzymes and extra muscular activity assessment derived using myositis disease activity assessment tool(0-10).
| scores on a scale | Placebo | Enpatoran 100 mg BID |
|---|---|---|
| Double Blind Placebo Control Period (DBPC): American College of Rheumatology/European League Against Rheumatism (ACR/EULAR) Total Improvement Score (TIS) at Week 24 | 51.3 ± 26.96 | 30.4 ± 20.76 |
An AE was defined as any untoward medical occurrence in a participant administered a pharmaceutical product and which did not necessarily have a causal relationship with this treatment. An AE could therefore be any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a medicinal product or protocol-specified procedure, whether considered related to the medicinal product or protocol-specified procedure. Serious AE was defined AE that resulted in any of the following outcomes: death; life threatening; persistent/significant disability/incapacity; initial/prolonged inpatient hospitalization; congenital anomaly/birth defect. TEAE was defined as events with onset date or worsening during the on-treatment period. TEAEs included serious TEAEs and non-serious TEAEs. Adverse events of special interest (AESI) are serious or non-serious AEs that are of clinical interest and were closely followed.
| Participants | Placebo | Enpatoran 100 mg BID |
|---|---|---|
| TEAEs | 7 | 7 |
| Serious TEAEs | 0 | 2 |
| AESI | 0 | 1 |
Laboratory parameters included hematology, biochemistry, urinalysis and reflex testing for HBV DNA. Number of participants with clinically meaningful changes from baseline in laboratory parameters were reported. Clinical meaningfulness was assessed by the investigator.
| Participants | Placebo | Enpatoran 100 mg BID |
|---|---|---|
| DBPC Period: Number of Participants With Clinically Meaningful Changes From Baseline in Laboratory Parameters | 0 | 0 |
Vital signs included body temperature, systolic and diastolic blood pressure, respiration rate, pulse rate, weight, height and body mass index. Number of participants with clinically meaningful changes from baseline in vital signs were reported. Clinical meaningfulness was assessed by the investigator.
| Participants | Placebo | Enpatoran 100 mg BID |
|---|---|---|
| DBPC Period: Number of Participants With Clinically Meaningful Changes From Baseline in Vital Signs | 0 | 0 |
12-lead ECG recordings included rhythm, heart rate (as measured by RR interval), PR interval, QRS duration, and QT interval. Number of participants with clinically meaningful changes from baseline in ECG parameters were reported. Clinical meaningfulness was assessed by the investigator.
| Participants | Placebo | Enpatoran 100 mg BID |
|---|---|---|
| DBPC Period: Number of Participants With Clinically Meaningful Changes From Baseline in Electrocardiograms (ECG) Parameters | 0 | 0 |
The ACR/EULAR TIS was derived from evaluation of results from 6 Core Set Measures(CSMs) of myositis disease activity in participants with Dermatomyositis and Polymyositis.TIS was determined by summing scores for each CSM,based on improvement in absolute percent change which is calculated by subtracting the baseline value from the final value,dividing difference by the range, and then multiplying the result by100 and relative weight of each core set measure. The TIS scale ranges from 0 to 100 with thresholds for minimal \[more than or equal to(\>= 20)\],moderate(\>=40) and major(\>=60)improvement in combined 6 CSM.The CSMs include,physician global activity visual analogue scale(VAS) score(0-10), patient global activity VAS score(0-10), manual muscle testing-8 score(0-150), health assessment questionnaire-disability index(0-3) along with checks for most abnormal muscle-associated enzymes and extra muscular activity assessment derived using myositis disease activity assessment tool(0-10).
| Participants | Placebo | Enpatoran 100 mg BID |
|---|---|---|
| TIS >= 20: Week 16 | 5 | 4 |
| TIS >= 40: Week 16 | 4 | 3 |
| TIS >= 60: Week 16 | 2 | 1 |
| TIS >= 20: Week 24 | 4 | 1 |
| TIS >= 40: Week 24 | 2 | 1 |
| TIS >= 60: Week 24 | 1 | 1 |
The ACR/EULAR TIS was derived from evaluation of results from 6 Core Set Measures(CSMs) of myositis disease activity in participants with Dermatomyositis and Polymyositis.TIS was determined by summing scores for each CSM,based on improvement in absolute percent change which is calculated by subtracting the baseline value from the final value,dividing difference by the range, and then multiplying the result by100 and relative weight of each core set measure. The TIS scale ranges from 0 to 100 with thresholds for minimal \[more than or equal to(\>= 20)\],moderate(\>=40) and major(\>=60)improvement in combined 6 CSM.The CSMs include,physician global activity visual analogue scale(VAS) score(0-10), patient global activity VAS score(0-10), manual muscle testing-8 score(0-150), health assessment questionnaire-disability index(0-3) along with checks for most abnormal muscle-associated enzymes and extra muscular activity assessment derived using myositis disease activity assessment tool(0-10).
| scores on a scale | Placebo | Enpatoran 100 mg BID |
|---|---|---|
| Week 4 | 27.5 ± 23.78 | 17.3 ± 19.60 |
| Week 8 | 36.1 ± 28.46 | 25.4 ± 23.43 |
| Week 12 | 48.2 ± 27.87 | 31.9 ± 18.06 |
| Week 16 | 46.4 ± 29.33 | 38.0 ± 23.61 |
| Week 20 | 51.4 ± 25.61 | 36.5 ± 16.45 |
| Week 24 | 51.3 ± 26.96 | 30.4 ± 20.76 |
The Physician's Global Assessment of Disease Activity was an assessment of overall global disease activity by the physician that was taken from Myositis Disease Activity Assessment Tool (MDAAT). Physician rated participant's disease activity on a 0-10 centimeter (cm) visual analog scale (VAS). Extra-muscular activity ranged between 0 and 10, where, 0 cm = absent and 10 cm = maximum disease activity.
| centimeter (cm) | Placebo | Enpatoran 100 mg BID |
|---|---|---|
| Change at Week 4 | -1.2 ± 1.65 | -0.1 ± 1.74 |
| Change at Week 8 | -1.3 ± 1.50 | -1.0 ± 1.74 |
| Change at Week 12 | -2.0 ± 1.60 | -1.0 ± 1.52 |
| Change at Week 16 | -2.1 ± 1.20 | -1.2 ± 1.73 |
| Change at Week 20 | -2.5 ± 1.58 | -0.7 ± 1.36 |
| Change at Week 24 | -1.3 ± 2.39 | -1.0 ± 1.52 |
PtGA was the assessment of the severity of disease by the participant/participant's guardian, using a VAS from 0 mm (no evidence of disease activity) to 100 mm (extremely active or severe disease activity). Higher score indicated worse status.
| scores on a scale | Placebo | Enpatoran 100 mg BID |
|---|---|---|
| Change at Week 4 | -1.1 ± 1.54 | -0.5 ± 2.31 |
| Change at Week 8 | -0.2 ± 2.31 | -0.9 ± 1.82 |
| Change at Week 12 | -2.5 ± 3.78 | -1.3 ± 1.52 |
| Change at Week 16 | -2.1 ± 3.19 | -2.4 ± 1.62 |
| Change at Week 20 | -2.9 ± 2.99 | -1.9 ± 1.96 |
| Change at Week 24 | -2.0 ± 3.17 | -1.5 ± 2.64 |
MDAAT was a combined tool capturing physician's assessment of disease activity of various organ systems- constitutional, cutaneous, pulmonary, gastrointestinal, joints and cardiovascular, muscular using: Myositis Intention to Treat Activity Index (MITAX), a scale from 0("Not present in the last 4 weeks") to 4("New - in the last 4 weeks \[compared to the previous 4 weeks\]") and Myositis Disease Activity Assessment Visual Analogue Scales (MYOACT), a 10 cm VAS assessing disease activity in the last 4 weeks of various organ systems from 0 cm("No evidence of disease activity") to 10 cm("Extremely active or severe disease activity"), disease activity being defined as potentially reversible pathology or physiology resulting from the myositis. Left end of line = no evidence of disease activity, midpoint of line = moderate disease activity, and right end of line = extreme or maximum disease activity. An assessment of extramuscular activity assessment was taken from MDAAT using a 10cm VAS scale.
| scores on a scale | Placebo | Enpatoran 100 mg BID |
|---|---|---|
| Change at Week 4 | -0.4 ± 2.20 | 0.0 ± 2.02 |
| Change at Week 8 | -1.3 ± 3.18 | -0.3 ± 1.44 |
| Change at Week 12 | -2.2 ± 2.07 | -0.3 ± 1.42 |
| Change at Week 16 | -2.4 ± 2.09 | -0.5 ± 1.68 |
| Change at Week 20 | -2.6 ± 1.68 | -0.3 ± 1.60 |
| Change at Week 24 | -1.9 ± 2.24 | -0.4 ± 1.72 |
MMT-8 was an International Myositis Assessment and Clinical Studies (IMACS) Core Set Measure used in the calculation of the IMACS Definition of Improvement (DOI) and ACR/EULAR TIS. It is a set of 8 designated muscles tested unilaterally (generally on right side) -which include 1 axial, 5 proximal (2 upper extremity, 3 lower extremity), and 2 distal muscles (1 upper, 1 lower extremity). Total score for unilateral testing ranges from 0 to 80, and bilateral testing ranges from 0 to 150 with normal strength represented by a higher score at or near the top of the scale. The total score for bilateral testing (item "MMT8 score (0 - 150)" from the MANUAL MUSCLE TESTING-8 (mmt8) form) is used in the derivation of the Total Improvement Score.
| scores on a scale | Placebo | Enpatoran 100 mg BID |
|---|---|---|
| Change at Week 4 | 6.6 ± 7.72 | 4.1 ± 9.91 |
| Change at Week 8 | 9.3 ± 8.40 | 9.1 ± 9.70 |
| Change at Week 12 | 15.0 ± 8.94 | 10.6 ± 10.03 |
| Change at Week 16 | 14.4 ± 10.49 | 9.9 ± 9.81 |
| Change at Week 20 | 15.7 ± 9.16 | 8.0 ± 5.39 |
| Change at Week 24 | 16.0 ± 10.03 | 10.9 ± 9.48 |
HAQ-DI was an IMACS Core Set Measure used in the calculation of the IMACS DOI and ACR/EULAR TIS. It is a generic rather than a disease-specific instrument comprised of 8 sections (dressing, arising, eating, walking, hygiene, reach, grip, and activities), with 2 to 3 questions per section. Scoring within each section is from 0 (without any difficulty) to 3 (unable to do). For each section the score given to that section is the worst score within the section. The 8 scores of the 8 sections are summed and divided by 8. Overall score ranges from 0-3.
| scores on a scale | Placebo | Enpatoran 100 mg BID |
|---|---|---|
| Change at Week 4 | -0.2 ± 0.39 | 0.2 ± 0.48 |
| Change at Week 8 | -0.2 ± 0.40 | -0.1 ± 0.28 |
| Change at Week 12 | -0.4 ± 0.59 | -0.2 ± 0.33 |
| Change at Week 16 | -0.4 ± 0.60 | -0.2 ± 0.42 |
| Change at Week 20 | -0.5 ± 0.56 | -0.3 ± 0.34 |
| Change at Week 24 | -0.4 ± 0.54 | -0.1 ± 0.35 |
For each participant, the most abnormal muscle-associated enzyme was defined as the baseline muscle-associated enzyme with the highest ratio of the observed value to the upper limit of the normal range (ULN) among creatinine kinase, lactate dehydrogenase, aspartate aminotransferase, alanine aminotransferase, and aldolase. The relative abnormality could be expressed as a percentage of ULN, calculated as (enzyme value divided by ULN) multiplied by 100.
| percent change | Placebo | Enpatoran 100 mg BID |
|---|---|---|
| Change at Week 4 | -5.8 ± 14.82 | -3.4 ± 17.04 |
| Change at Week 8 | -2.6 ± 26.83 | -11.4 ± 32.32 |
| Change at Week 12 | -13.9 ± 29.30 | -4.7 ± 21.11 |
| Change at Week 16 | 2.6 ± 40.80 | -24.6 ± 39.36 |
| Change at Week 20 | -18.0 ± 32.39 | -11.5 ± 39.60 |
| Change at Week 24 | -4.0 ± 40.31 | -16.8 ± 37.57 |
A participant was defined as an International Myositis Assessment and Clinical Studies (IMACS) Responder if 3 of any 6 IMACS Core Set Measures (CSMs) have been improved by more than equal to (\>= 20) percent (%), with no more than 2 IMACS CSMs worse by \>= 25%. The CSMs include, physician global activity visual analogue scale(VAS) score(0-10), patient global activity VAS score(0-10), manual muscle testing-8 score(0-150), health assessment questionnaire-disability index (0-3) along with checks for muscle-associated enzymes and extra muscular activity assessment derived using myositis disease activity assessment tool(0-10).
| Participants | Placebo | Enpatoran 100 mg BID |
|---|---|---|
| Week 16 | 2 | 2 |
| Week 24 | 1 | 1 |
CDASI is a clinician-scored single page instrument that separately measures activity and damage in the skin of DM participants. CDASI has 3 activity measures (CDASI-A: erythema, scale, and erosion/ulceration) and 2 damage measures (CDASI-D: poikiloderma and calcinosis) which are assessed over 15 body areas. In addition, Gottron's papules on the hands, periungual changes and alopecia are evaluated separately both for activity and damage. Activity and Damage Subscale scores range from 0 to 100 and 0 to 32, respectively, where higher scores indicate greater disease severity.
| scores on a scale | Placebo | Enpatoran 100 mg BID |
|---|---|---|
| CDASI-A Week 4 | -3.8 ± 7.59 | -3.7 ± 9.53 |
| CDASI-A Week 8 | -6.8 ± 6.24 | -8.1 ± 10.68 |
| CDASI-A Week 12 | -11.5 ± 9.69 | -9.4 ± 9.07 |
| CDASI-A Week 16 | -12.3 ± 10.15 | -5.3 ± 8.36 |
| CLASI-A Week 20 | -12.3 ± 9.56 | -3.5 ± 6.38 |
| CDASI-A Week 24 | -11.2 ± 9.06 | -4.7 ± 8.98 |
| CDASI-D Week 4 | -0.8 ± 1.39 | 0.6 ± 1.71 |
| CDASI-D Week 8 | -0.3 ± 0.52 | 0.3 ± 1.41 |
| CDASI-D Week 12 | -1.5 ± 2.07 | 0.1 ± 0.90 |
| CDASI-D Week 16 | -1.5 ± 2.07 | 1.0 ± 1.26 |
| CDASI-D Week 20 | -1.5 ± 2.07 | 1.3 ± 2.07 |
| CDASI-D Week 24 | -0.7 ± 1.21 | 1.2 ± 2.04 |
The IGA skin activity is a five-point score that defines the level of disease severity based on overall lesion characteristics where 0 is "clear" and "4" is severe.
| scores on a scale | Placebo | Enpatoran 100 mg BID |
|---|---|---|
| Week 4 | -0.3 ± 0.46 | -0.2 ± 0.79 |
| Week 8 | -0.3 ± 0.52 | -0.4 ± 0.73 |
| Week 12 | -0.8 ± 0.98 | -0.9 ± 0.90 |
| Week 16 | -0.8 ± 0.98 | -0.3 ± 1.03 |
| Week 20 | -1.0 ± 0.89 | -0.3 ± 1.03 |
| Week 24 | -0.7 ± 1.03 | -0.3 ± 0.82 |
An AE was defined as any untoward medical occurrence in a participant administered a pharmaceutical product and which did not necessarily have a causal relationship with this treatment. An AE could therefore be any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a medicinal product or protocol-specified procedure, whether considered related to the medicinal product or protocol-specified procedure. Serious AE was defined AE that resulted in any of the following outcomes: death; life threatening; persistent/significant disability/incapacity; initial/prolonged inpatient hospitalization; congenital anomaly/birth defect. TEAE was defined as events with onset date or worsening during the on-treatment period. TEAEs included serious TEAEs and non-serious TEAEs. Adverse events of special interest (AESI) are serious or non-serious AEs that are of clinical interest and were closely followed.
| Participants | Placebo to Enpatoran 100 mg | Enpatoran 100 mg BID |
|---|---|---|
| TEAEs | 4 | 4 |
| Serious TEAEs | 0 | 0 |
| AESI | 1 | 0 |
Laboratory parameters included hematology, biochemistry, urinalysis and reflex testing for HBV DNA. Number of participants with clinically meaningful changes from baseline in laboratory parameters were reported. Clinical meaningfulness was assessed by the investigator.
| Participants | Placebo to Enpatoran 100 mg | Enpatoran 100 mg |
|---|---|---|
| OLE Period: Number of Participants With Clinically Meaningful Changes From Baseline in Laboratory Parameters | 0 | 0 |
Vital signs included body temperature, systolic and diastolic blood pressure, respiration rate, pulse rate, weight, height and body mass index. Number of participants with clinically meaningful changes from baseline in vital signs were reported. Clinical meaningfulness was assessed by the investigator.
| Participants | Placebo to Enpatoran 100 mg | Enpatoran 100 mg |
|---|---|---|
| OLE Period: Number of Participants With Clinically Meaningful Changes From Baseline in Vital Signs | 0 | 0 |
12-lead ECG recordings included rhythm, heart rate (as measured by RR interval), PR interval, QRS duration, and QT interval. Number of participants with clinically meaningful changes from baseline in ECG parameters were reported. Clinical meaningfulness was assessed by the investigator.
| Participants | Placebo to Enpatoran 100 mg | Enpatoran 100 mg |
|---|---|---|
| OLE Period: Number of Participants With Clinically Meaningful Changes From Baseline in Electrocardiograms (ECG) Parameters | 0 | 0 |
Collected over From 24-week DBPC Treatment Period followed by a 2-week Safety Follow-up period. OLE period: 26 weeks (24-week treatment period + 2 weeks Safety Follow-up), approximately 57 weeks.. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Placebo: DBPC Period | 0/9 (0%) | 0/9 (0%) | 7/9 (77.8%) |
| Enpatoran 100mg: Double Blind Placebo Controlled Period | 0/11 (0%) | 2/11 (18.2%) | 7/11 (63.6%) |
| Enpatoran 100mg: Open-label Expension Period | 0/11 (0%) | 0/11 (0%) | 8/11 (72.7%) |
| Event | Placebo: DBPC Period | Enpatoran 100mg: Double Blind Placebo Controlled Period | Enpatoran 100mg: Open-label Expension Period |
|---|---|---|---|
| CellulitisInfections and infestations | 0/9 | 1/11 | 0/11 |
| SepsisInfections and infestations | 0/9 | 1/11 | 0/11 |
| Glomerulonephritis membranousRenal and urinary disorders | 0/9 | 1/11 | 0/11 |
| Event | Placebo: DBPC Period | Enpatoran 100mg: Double Blind Placebo Controlled Period | Enpatoran 100mg: Open-label Expension Period |
|---|---|---|---|
| HeadacheNervous system disorders | 4/9 | 0/11 | 1/11 |
| Upper respiratory tract infectionInfections and infestations | 0/9 | 3/11 | 0/11 |
| NauseaGastrointestinal disorders | 2/9 | 0/11 | 1/11 |
| SinusitisInfections and infestations | 2/9 | 1/11 | 0/11 |
| ArthralgiaMusculoskeletal and connective tissue disorders | 2/9 | 0/11 | 0/11 |
| Oedema peripheralGeneral disorders | 0/9 | 2/11 | 0/11 |
| PruritusSkin and subcutaneous tissue disorders | 0/9 | 0/11 | 2/11 |
| LymphadenopathyBlood and lymphatic system disorders | 1/9 | 0/11 | 0/11 |
| Abdominal painGastrointestinal disorders | 1/9 | 0/11 | 0/11 |
| DiarrhoeaGastrointestinal disorders | 1/9 | 0/11 | 0/11 |
| Age, Continuous(Years) | Placebo | Enpatoran 100 mg BID | Total |
|---|---|---|---|
| Mean | 53 ± 11.5 | 55 ± 11.3 | 54 ± 11.2 |
| Sex: Female, Male(Participants) | Placebo | Enpatoran 100 mg BID | Total |
|---|---|---|---|
| Female | 7 | 8 | 15 |
| Male | 2 | 3 | 5 |
| Ethnicity (NIH/OMB)(Participants) | Placebo | Enpatoran 100 mg BID | Total |
|---|---|---|---|
| Hispanic or Latino | 2 | 1 | 3 |
| Not Hispanic or Latino | 7 | 10 | 17 |
| Unknown or Not Reported | 0 | 0 | 0 |
| Race (NIH/OMB)(Participants) | Placebo | Enpatoran 100 mg BID | Total |
|---|---|---|---|
| American Indian or Alaska Native | 0 | 1 | 1 |
| Asian | 0 | 0 | 0 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 |
| Black or African American | 0 | 1 | 1 |
| White | 9 | 9 | 18 |
| More than one race | 0 | 0 | 0 |
| Unknown or Not Reported | 0 | 0 | 0 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: Yes — IPD supporting publicly available results will be evaluated for sharing.
Supporting information: Study protocol, Statistical analysis plan, Clinical study report, Analytic code
Time frame: IPD from completed trials will be publicly available within 6 months after all of the following events: Approval of a product/new indication by major authorities (FDA, EMA, PMDA if requested) with no pending submissions Public results via primary manuscript or trial registry disclosure Legal authority to share data Privacy protections are in place If approval is not sought or development is globally discontinued, data will be publicly available within 18 months after global trial completion. Further information on how to request data can be found on our website bit.ly/IPD21
Access criteria: Qualified researchers may propose access to IPD from sponsored trials via https://vivli.org/members/ourmembers/.
Data sharing details ↗ · vivli.org
Ran from Jan 2023 to primary completion in Jun 2025, 29 months.
Results were first posted in Sep 2026, 1 year 2 months after primary completion.
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Studies of the drug tested here: Enpatoran
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