2022-501351-82-00已完成2 期
A Phase IIa, Randomized, Parallel, Double-Blind, Placebo Controlled Study to Evaluate the Efficacy and Safety of Enpatoran in Dermatomyositis and Polymyositis Participants receiving Standard of Care
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 15
- 试验地点
- 14
- 主要终点
- DBPC Period: American College of Rheumatology/European League Against Rheumatism (ACR/EULAR) Total Improvement Score (TIS) at Week 24
研究概览
简要总结
(1) To evaluate the effect of enpatoran compared to placebo on the American College of Rheumatology/European League Against Rheumatism (ACR/EULAR) Total Improvement Score (TIS) at Week 24 of the Double-Blind Placebo Controlled (DBPC) period in DM and PM participants (2) To evaluate the safety and tolerability of enpatoran in DM and PM participants during the DBPC period
研究设计
- 分配方式
- Not Applicable
- 主要目的
- Optional OLE Period
- 盲法
- None
入排标准
- 年龄范围
- 18 years 至 65+ years（18-64 Years, 65+ Years）
- 接受健康志愿者
- 是
入选标准
- •Diagnosis of probable or definite DM or PM as per 2017 ACR/EULAR classification criteria, with positive autoantibody status. Anti-synthetase syndrome (ASyS) participants that meet classification criteria are allowed
- •Active disease on standard of care (SoC), must meet one of the criteria within 6 months prior to Screening: Pathological evidence of active myositis in muscle biopsy; Evidence of active myositis by Electromyography (EMG); Magnetic resonance imaging (MRI) with evidence of active myositis; or any muscle enzyme greater than or equal to (>=) 4 × upper limit of normal (ULN) at time of Screening; Active PM/DM skin rash as per cutaneous dermatomyositis area and severity index-A (CDASI-A) >= 7 at time of Screening
- •Minimum disease severity defined by: moderate to severe myopathy with manual muscle testing-8 (MMT-8) >= 80 and less than or equal to (<=) 142 AND at least 2 of the following core set measures (CSM) abnormalities: Patient Global Activity (PtGA) >= 2 centimeters (cm); Physician Global Activity (PGA) derived from myositis disease activity assessment tool (MDAAT) >= 2 cm; Extramuscular Activity Assessment derived from MDAAT >= 2 cm; At least one muscle enzyme > 1.5 times ULN; health assessment questionnaire-disability index (HAQ-DI) >= 0.25
- •Stable doses of oral corticosteroids (CS) and/or maximum of 1 non-corticosteroid immunosuppressive/immunomodulatory medications (methotrexate, 6 mercaptopurine, sulfasalazine, mycophenolate mofetil or sodium, azathioprine, leflunomide, cyclosporine, oral tacrolimus) for DM or PM
- •Participants have a body mass index (BMI) lower or Equal to 40.0 kilograms per square meter (kg/m^2)
- •Other protocol defined inclusion criteria could apply
排除标准
- •Primary diagnosis of inclusion body myositis (IBM), malignancy-associated myositis (defined as diagnosis of myositis within 3 years of cancer), immune mediated necrotizing myopathy (IMNM) with a biopsy characterized as necrotizing biopsy or IMNM with positive anti-signal recognition particle antibody (SRP) or anti 3-hydroxy-3-methylglutaryl-coenzyme A reductase (HMGCR) auto antibodies. Participants with anti-transcription intermediary factor 1 (TIF1) gamma antibody or newly diagnosed (within 1 year) anti MDAT5 antibody should have had adequate screening for cancer within 12 months of Day
- •Adequate screening of cancer is defined as up-to-date age and gender appropriate screening as per national guidelines
- •Primary diagnosis of juvenile DM, or adult participants previously diagnosed with juvenile DM
- •Any other active concurrent connective tissue disease associated with inflammatory myopathy in the Investigator’s opinion. Eligibility of participants with diagnosis of concurrent connective tissue disease(s) will be reviewed and approved by an idiopathic inflammatory myopathies (IIM) expert committee
- •Severe interstitial lung disease defined as supplemental oxygen required at rest, or forced vital capacity (FVC) of <60 percent (%) predicted. Participants within 1 year of PM/DM diagnosis and anti-MDA5 antibody, should have been evaluated for interstitial lung disease (ILD) with high resolution computed tomography (HRCT) Chest.
- •Any uncontrolled disease (for example [e.g.], severe respiratory, cardiovascular, gastrointestinal, neurological, psychiatric, hematological, metabolic [including thyroiditis with increased/decreased thyroid stimulating hormone (TSH)], renal [Estimated glomerular filtration rate < 40 milliliter per minute/1.73 m^2 as calculated by the Modification of Diet in Renal Disease equation by the central laboratory], hepatic, endocrine/reproductive organ disease) other than DM/PM, that in the Investigator’s or Sponsor/designee’s opinion constitutes an inappropriate risk or contraindication for participation in the study or that could interfere with the study objectives, conduct, or evaluation
- •Other protocol defined exclusion criteria could apply
结局指标
主要结局
DBPC Period: American College of Rheumatology/European League Against Rheumatism (ACR/EULAR) Total Improvement Score (TIS) at Week 24
DBPC Period: American College of Rheumatology/European League Against Rheumatism (ACR/EULAR) Total Improvement Score (TIS) at Week 24
DBPC Period: Number of Participants with Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs and Adverse Events of Special Interest (AESIs)
DBPC Period: Number of Participants with Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs and Adverse Events of Special Interest (AESIs)
DBPC Period: Number of Participants with Clinically Significant Changes from Baseline in Laboratory Parameters, Vital Signs and 12-Lead Electrocardiogram (ECG) Measurements
DBPC Period: Number of Participants with Clinically Significant Changes from Baseline in Laboratory Parameters, Vital Signs and 12-Lead Electrocardiogram (ECG) Measurements
次要结局
- 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
- DBPC Period: Total Improvement Score (TIS)
- DBPC Period: Mean Score for Core Set Measures (CSM) from Week 4 up to Week 24
- DBPC Period: Percent Change from Baseline in Most Abnormal Muscle-associated Enzyme at Weeks 4, 8, 12, 16, 20 and 24
- DBPC Period: Absolute Change from Baseline in the Core Set Measures (CSM) at Weeks 4, 8, 12, 16, 20 and 24
- DBPC Period: Percent Change from Baseline in Core Set Measures (CSM) at Weeks 4, 8, 12, 16, 20 and 24
- DBPC Period: Number of Participants with International Myositis Assessment and Clinical Studies (IMACS) Response
- DBPC Period: Change from Baseline in Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI) A and CDASI-D at Weeks 4, 8, 12, 16, 20 and 24
- DBPC Period: Percent Change from Baseline in Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI) A and CDASI-D at Weeks 4, 8, 12, 16, 20 and 24
- DBPC Period: Change from Baseline in Investigator’s Global Assessment (IGA) Skin Activity Score at Weeks 4, 8, 12, 16, 20 and 24
- DBPC Period: Percent Change from Baseline in Investigator’s Global Assessment (IGA) Skin Activity Score at Weeks 4, 8, 12, 16, 20 and 24
- OLE Period: Number of Participants with Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs and Adverse Events of Special Interest (AESIs)
- OLE Period: Number of Participants with Clinically Significant Changes from Baseline in Laboratory Parameters, Vital Signs and 12-Lead Electrocardiogram (ECG) Measurements
研究者
Global Regulatory Affairs
Scientific
Merck Healthcare KGaA
研究点 (14)
Loading locations...
相似试验
招募中
2 期
A Study to Investigate Efficacy and Safety of AZD1163 in Participants With Rheumatoid ArthritisRheumatoid ArthritisNCT07276581AstraZeneca320
招募中
4 期
A study to investigate the course of synovial hypertrophy in patients with haemophilia A on efanesoctocog alfa prophylaxis2024-512066-33-00Swedish Orphan Biovitrum AB (publ)35
招募中
1 期
SA1211 Injection Phase 1 StudyNCT07275918Suzhou Siran Biotechnology Co.,Ltd.80
尚未招募
2 期
Losartan and Paclitaxel in Platinum Resistant Ovarian CancerNCT07265739Massachusetts General Hospital27
招募中
4 期
A Phase 3b/4 Randomized, Double-Blind, Double-Dummy, Active Comparator-Controlled Study, Comparing the Efficacy and Safety of Upadacitinib Versus Adalimumab in Subjects with Moderate to Severe Rheumatoid Arthritis on a Stable Background of MTX and who had an Inadequate Response or Intolerance to a Single TNF Inhibitor (SELECT- SWITCH)Rheumatoid Arthritis2022-502578-18-00Abbvie Deutschland GmbH & Co. KG165