2024-514137-38-00招募中2 期
A Phase 2, randomized, open-label, controlled study to evaluate the efficacy and safety of rapcabtagene autoleucelversus comparator in participants with severe refractory idiopathic inflammatory myopathies
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 43
- 试验地点
- 34
- 主要终点
- Achieving at least moderate improvement in TIS (TIS ≥ 40) at Week 52 (Yes/No)
研究概览
简要总结
To demonstrate the superiority of rapcabtagene autoleucel at a target dose of xx CAR-positive viable T cells, as a single infusion, over comparator (Investigator choice of treatment) with respect to achieving at least a moderate improvement in the Total Improvement Score (TIS) at Week 52 for MSA-positive participants
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
- •Signed informed consent must be obtained prior to participation in the study
- •Male or female participants aged >18 and ≤65 years on the day of signing informed consent.
- •A diagnosis of probable or definite myositis according to American College of Rheumatology/European League Against Rheumatism 2017 (ACR/EULAR 2017) criteria.
- •Participant must be positive for MSA based on an historical result or for any of the MSAs listed below, based on the central laboratory result during Screening. If the participant is xx then the participant must fit in one of the following two categories: a) xx OR b) xx Note: Lead-in Cohort 1 participants must be MSA-positive (by a historical laboratory result) to be enrolled. In Cohort 2, xx MSA-positive participants have been randomized, whichever comes first.
- •Participants who had inadequate response to high-dose GC xx, unless there are safety concerns regarding use of high-dose GC due to comorbidities. Additionally, participants must have had inadequate response to treatment with two therapeutic agents, with xx. Note: a. xx b. xx c. xx
- •Diagnosed with active disease such as presence of at least 1 of the following criteria and confirmed by an adjudication committee for criteria b through e prior to randomization: xx
- •At screening, severe myositis that meets criteria a, b or c: xx
排除标准
- •BMI at Screening of ≤18.5 or ≥35 kg/m2
- •Have severe muscle damage at Screening, as xx
- •Participants treated with CYC within 12 weeks or RTX within 6 months prior to Baseline.
- •Inadequate organ function (one retest during Screening of any of the below is permitted): Inadequate renal function (performed by central laboratory) defined as: • eGFR < 60 ml/min/1.73m2 using the CKD-EPI formula. Inadequate hepatic function defined as any of the following: • ALT and AST >1.5 × ULN (performed by central laboratory), except if clinically assessed as secondary to IIM • Total Bilirubin >1.5 × ULN (performed by central laboratory). Participants with Gilbert's syndrome may be included if their total bilirubin is ≤ 3.0 × ULN and direct bilirubin ≤1.5 × ULN. • International normalized ratio (INR) >1.5 (performed by central laboratory) Inadequate cardiac function defined as: • Left ventricular ejection fraction (LVEF) <50% as determined by echocardiogram (ECHO) Inadequate hematologic function (performed by central laboratory) defined as: • Absolute neutrophil count (ANC) <1500/mm3 • Platelets <100,000/μL • White blood cells count (WBC) <3,000 cells/μL • Absolute lymphocyte count <700/μL • Hemoglobin < 8.0 g/dL (< 4.9 mmol/L) Inadequate pulmonary function defined as: • Oxygen saturation measured by pulse oximetry < 92% xx • Hemoglobin corrected DLCO xx • FVC% xx of predicted.
- •Any participant who failed all available treatment options in the comparator arm or for whom these options are clinically inappropriate in the opinion of the Investigator.
- •Hypersensitivity and/or contraindications to any product (including its ingredients) to be given to the participant as per the study protocol (e.g., rapcabtagene autoleucel, comparator arm treatments, tocilizumab, lymphodepleting agents, etc.), to the excipients of rapcabtagene autoleucel (e.g., xx), to any other drug product as advised for administration in the study protocol (e.g., lymphodepleting agents, tocilizumab), or to any condition that, in the Investigator's opinion, precludes lymphodepleting chemotherapy.
结局指标
主要结局
Achieving at least moderate improvement in TIS (TIS ≥ 40) at Week 52 (Yes/No)
Achieving at least moderate improvement in TIS (TIS ≥ 40) at Week 52 (Yes/No)
次要结局
- Achieving at least moderate improvement in TIS (≥ 40) at Week 52 (Yes/No), as defined for primary endpoint
- Adjusted annual cumulative GC dose up to Week 52
- Change from baseline in percent predicted Forced Vital Capacity (FVC%) at Week 52
- Achieving major improvement in TIS (≥ 60) at Week 52 (Yes/No), as defined for primary endpoint but with greater improvement required
- Change from baseline in Patient-Reported Outcome Measurement Information System (PROMIS)-Fatigue 7a at Week 52
- Safety parameters include vital signs, adverse events, laboratory parameters and ECG evaluation
研究者
Novartis Pharma Arzneimittel GmbH
Scientific
Novartis Pharma AG
研究点 (34)
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