PER-059-22招募中2 期
A Phase 2/3, Randomized, Double-Blinded, Placebo-Controlled, Parallel-Group, 2-Arm, Multicenter, Operationally Seamless Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacodynamics, Pharmacokinetics, and Immunogenicity of Efgartigimod PH20 SC in Participants Aged 18 Years and Older With Active Idiopathic Inflammatory Myopathy.
适应症
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- Argenx BV
- 入组人数
- 1
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 性别
- All
入选标准
- •1.Aged at least 18 years (or the legal age of consent in the jurisdiction in which the study is taking place) at the time of signing the informed consent
- •2.A definite or probable clinical diagnosis of IIM under the 2017 EULAR/ACR classification criteria
- •3.One of the following medical histories:
- •a.Diagnosis of DM or juvenile DM (JDM), fulfilling the 2017 EULAR/ACR criteria for DM or JDM (age of disease onset <18 years of age). The diagnosis date for JDM should not be >5 years from the screening date.
- •b.Diagnosis of PM (including ASyS), having either of the following:
- •i.A prior muscle biopsy diagnostic for IIM
- •ii.A positive test at the central laboratory for at least 1 anti-aminoacyl-tRNA synthetase MSA (-Jo-1, -PL-7, -PL-12, -EJ, -OJ)
- •c.Diagnosis of IMNM, meeting the 2017 European Neuromuscular Center classification criteria,11 and having any of the following:
- •i.Anti–signal recognition particle (SRP) positive (at the central laboratory) myopathy
- •ii.Anti–3-hydroxy-3-methylglutaryl coenzyme A reductase (HMGCR) positive (at the central laboratory) myopathy
- •iii.A prior muscle biopsy with pathological features of IMNM
- •4.Active disease as defined by the presence of at least 1 of the following criteria:
- •a.Abnormal levels of at least 1 of the following enzymes: CK (=4×upper limit of normal [ULN]), aldolase (=4×ULN), LDH (=4×ULN), AST (=4×ULN), or ALT (=4×ULN), based on central laboratory results
- •b.Electromyography demonstrating active disease within the past 3 months
- •c.Active DM skin rash (Gottron’s papules, Gottron’s signs, or heliotrope rash) or CDASI =7 (phase 2 stage) or =14 (phase 3 stage) at screening and baseline
- •d.Muscle biopsy indicative of active IIM in the past 3 months
- •e.Magnetic resonance imaging within the past 3 months indicative of active inflammation
- •5.Muscle weakness as assessed by an MMT8 score of <142/150 and abnormalities in any 2 of the other 5 CSMs:
- •c.Extramuscular global =2
- •d.HAQ-DI =0.25
- •e.Muscle enzyme =1.5×ULN
- •6.Receiving a permitted background treatment for IIM. Permitted background treatment includes: OCS; 1 antimalarial (hydroxychloroquine, quinacrine, or chloroquine); or 1 of the following immunosuppressants: methotrexate, azathioprine, mycophenolate mofetil, mycophenolic acid, tacrolimus, cyclosporine, leflunomide, or mizoribine. Participants may receive a combination of either OCS and up to 1 antimalarial or OCS and up to 1 immunosuppressant.
- •7.Contraceptive use by nonsterilized male participants and WOCBP will be consistent with local regulations, where available, for individuals participating in clinical studies. WOCBP must have a negative serum pregnancy test during screening and a negative urine pregnancy test at baseline to establish the nonpregnant state before receiving IMP.
- •The contraceptive requirements for nonsterilized males and WOCBP are described in Section 10.4.2. of the protocol.
- •8.Capable of giving signed informed consent, as described in Appendix 1, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and this protocol.
排除标准
- •1.A clinically significant uncontrolled active or chronic bacterial, viral, or fungal infection at screening
- •2.A COVID-19 polymerase chain reaction (PCR)-positive test <72 hours before enrollment
- •3.Any other known autoimmune disease that, in the investigator’s opinion, would interfere with an accurate assessment of clinical symptoms of IIM or put the patient at undue risk
- •4.A history of malignancy unless considered cured by adequate treatment, with no evidence of recurrence for =3 years before the first administration of IMP. Adequately treated participants with the following cancers can be included at any time:
- •a.Basal cell or squamous cell skin cancer
- •b.Carcinoma in situ of the cervix
- •c.Carcinoma in situ of the breast
- •d.Incidental histological finding of prostate cancer (TNM stage T1a or T1b)
- •5.Severe muscle damage defined as a global muscle damage score of >5 on a 10 cm visual analog scale (VAS) scale on the Myositis Damage Index (MDI)
- •6.Glucocorticoid-induced myopathy that the investigator considers the primary cause of muscle weakness or permanent weakness linked to a non-IIM cause
- •7.JDM diagnosed >5 years from screening or JDM with extensive calcinosis (defined as calcinosis involving the torso or 2 extremities) or severe calcinosis (indicated by a calcinosis associated with severe loss of function)
- •8.Uncontrolled interstitial lung disease or any other uncontrolled IIM manifestation that, in the opinion of the investigator, would be likely to require treatment with prohibited medication (eg, cyclophosphamide) during the study
- •9.Other inflammatory and noninflammatory myopathies: inclusion body myositis (based on the biopsy or when the weakness affects the finger and/or the wrist flexors out of proportion to shoulder abductors), overlap myositis (connective tissue disease–associated myositis, except an overlap with Sjögren’s syndrome, which is allowed provided that the biopsy does not demonstrate any of the features listed in inclusion criterion 3b), metabolic myopathies, muscle dystrophies or a family history of muscle dystrophy, drug-induced or endocrine-induced myositis (except statin-induced IMNM), and juvenile myositis (other than JDM)
- •10.Clinically significant disease, recent major surgery (within 3 months of screening) or intends to have surgery during the study, or has any other condition in the opinion of the investigator that could confound the results of the study or put the patient at undue risk
- •11.Known hypersensitivity reaction to IMP or 1 of its excipients
- •12.Received a live or live-attenuated vaccine less than 4 weeks before screening. The receipt of other types of vaccines at any time before screening is not considered exclusionary. It is recommended that participants are up to date with vaccinations before the first dose of IMP.
- •13.Lack of clinical response to plasmapheresis/plasma exchange (PLEX)
- •14.Positive serum test at screening for active viral infection with any of the following conditions (refer to Section 10.2.1):
- •a.Hepatitis B virus (HBV) indicative of an acute or chronic infection, unless associated with a negative HBV DNA test (https://www.cdc.gov/hepatitis/HBV/PDFs/SerologicChartv8.pdf)
- •b.Hepatitis C virus (HCV) based on HCV antibody assay, unless associated with a negative HCV RNA test
- •c.HIV based on test results associated with either:
- •i.An AIDS-defining condition or a CD4 count of =200 cells/mm3
- •ii.No adequate
研究者
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