Intermediate-Size Expanded Access Trial of Autologous Hybrid TREG/Th2 Cell Therapy (RAPA-501) of Amyotrophic Lateral Sclerosis

肌萎缩侧索硬化症自体杂交 TREG/Th2 细胞疗法 (RAPA-501) 的中型扩大试验

基本信息

  • 批准号:
    10834469
  • 负责人:
  • 金额:
    $ 1120.3万
  • 依托单位:
  • 依托单位国家:
    美国
  • 项目类别:
  • 财政年份:
    2023
  • 资助国家:
    美国
  • 起止时间:
    2023-09-25 至 2026-08-31
  • 项目状态:
    未结题

项目摘要

ALS is a lethal neurodegenerative disease accelerated by neuroinflammation. Current FDA-approved therapies have modest benefits and do not address inflammation. To address this, RAPA Therapeutics, LLC (RAPA) has developed an autologous T cell therapy (RAPA-501) that reduces inflammation, with the goal of reducing ALS morbidity and mortality. RAPA-501 are manufactured ex vivo to attain dual TREG/Th2 anti- inflammatory activity and a T-stem phenotype that permits T cell therapy without conditioning chemotherapy. In an ongoing clinical trial of RAPA-501 in people with ALS (pwALS) (NCT04220190), RAPA-501 cells were found to be safe (no product-related adverse events), biologically active (diverse anti-inflammatory effects in pwALS), and showed early trends toward stabilizing pulmonary function decline. A phase 2/3 expansion cohort was added to the trial to assess whether RAPA-501 is efficacious in standard-risk pwALS. We will extend RAPA-501 therapy to pwALS not eligible for this ongoing phase 2/3 trial or other ALS trials, which nearly universally require that participants have a slow vital capacity (SVC) value of ≥50% of predicted normal. The proposed EAP will enroll pwALS who have SVC values <50%. This population of pwALS is considered “high risk” (~50% chance of respiratory failure or death within 180 days) and thus particularly suitable for experimental immune therapies such as RAPA-501. In addition, the RAPA-501-EAP will not exclude pwALS who have a prolonged time from ALS-related symptoms or low ALSFRS-R scores. Participants will receive four RAPA-501 IV infusions (every 42-days at established safe dose, 80 x 106 cells/infusion). This RAPA-501-EAP will further evaluate the safety of this therapy, expand an understanding of the RAPA-501 therapeutic mechanism of action, and evaluate signals of efficacy in this real-world population of pwALS using standard methods and Origent Data Sciences machine learning ALS prediction algorithms. The RAPA-501 EAP will be led by investigators at Mass General Hospital (MGH; Drs. Berry, Babu, and Paganoni) and sponsored by RAPA, which is responsible for RAPA-501 manufacturing and FDA regulatory filings under existing IND 019480 (Dr. Fowler, Sponsor). Clinical trial site investigators have experience with RAPA-501 therapy (MGH; Hackensack University Medical Center; and Mayo Clinic Arizona) or other cells therapies. Sites are geographically diverse and likely to accrue a significant number of underserved pwALS (U of Iowa; U of Idaho; Providence Hospital, Portland, Oregon; UC-Irvine; Columbia, NYC). In addition, several research collaborations will emanate from the intensive study of the clinically-annotated, valuable research samples obtained from the RAPA-501 EAP.
ALS是一种致命的神经退行性疾病,由神经炎症加速。目前fda批准 治疗具有适度的益处并且不能解决炎症。为了解决这个问题,RAPA Therapeutics,LLC (RAPA)开发了一种自体T细胞疗法(RAPA-501),可减少炎症, 降低ALS发病率和死亡率。RAPA-501是离体制造的,以获得双重TREG/Th 2抗- 炎性活性和T干细胞表型,其允许T细胞疗法而无需调节化疗。在 一项正在进行的RAPA-501在ALS患者中的临床试验(NCT 04220190),发现RAPA-501细胞 安全(无产品相关不良事件),生物活性(在药物中具有多种抗炎作用), 并显示出稳定肺功能下降的早期趋势。增加了2/3期扩展队列 该试验旨在评估RAPA-501是否对标准风险患者有效。 我们将把RAPA-501疗法扩展到不符合这项正在进行的2/3期试验或其他ALS试验条件的ALS患者, 这几乎普遍要求参与者的慢肺活量(SVC)值≥预测值的50% 正常拟定的EAP将招募SVC值<50%的患者。这一群体是 被认为是“高风险”(180天内呼吸衰竭或死亡的可能性约为50%),因此特别适合 用于实验性免疫疗法如RAPA-501。此外,RAPA-501-EAP将不排除 患者出现ALS相关症状的时间较长或ALSFRS-R评分较低。参与者将获得四个 RAPA-501 IV输注(每42天一次,以确定的安全剂量,80 x 106个细胞/输注)。此RAPA-501-EAP 将进一步评估这种疗法的安全性,扩大对RAPA-501治疗机制的理解 并使用标准方法评估该真实世界的MATEALS人群中的疗效信号, Origent Data Sciences机器学习ALS预测算法。 RAPA-501 EAP将由马萨诸塞州总医院(MGH; Berry,Babu和 Paganoni),并由负责RAPA-501生产和FDA监管的RAPA赞助。 在现有IND 019480下提交(Dr. Fowler,申办者)。临床试验中心研究者具有以下经验: RAPA-501疗法(MGH; Hackensack University Medical Center;和马约诊所亚利桑那州)或其它细胞 治疗研究中心的地理位置各不相同,可能会产生大量服务不足的医疗机构(美国 爱荷华州;爱达荷州;普罗维登斯医院,波特兰,俄勒冈州;加州大学欧文分校;纽约市哥伦比亚)。此外,几个 研究合作将产生于对临床注释的、有价值的研究的深入研究 从RAPA-501 EAP获得的样本。

项目成果

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Suma Babu其他文献

Suma Babu的其他文献

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{{ truncateString('Suma Babu', 18)}}的其他基金

An Intermediate-Size Expanded Access Protocol for Amyotrophic Lateral Sclerosis with Pridopidine
使用普利多匹定治疗肌萎缩侧索硬化症的中型扩展治疗方案
  • 批准号:
    10835282
  • 财政年份:
    2023
  • 资助金额:
    $ 1120.3万
  • 项目类别:
An Expanded Access Protocol of Intravenous Trehalose Injection 90 mg/mL Treatment of Patients with Amyotrophic Lateral Sclerosis
静脉注射海藻糖注射液 90 mg/mL 治疗肌萎缩侧索硬化症的扩展方案
  • 批准号:
    10649756
  • 财政年份:
    2022
  • 资助金额:
    $ 1120.3万
  • 项目类别:

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