A Phase 2a Open-Label, Multi-Center Study to Evaluate the Safety and Tolerability of Multiple Doses of AT-1501 in Adults With ALS
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 54
- 试验地点
- 13
- 主要终点
- Safety and Tolerability
研究概览
简要总结
This is a Phase 2a, multi-center, open label, multiple dose study of AT-1501, a humanized monoclonal antibody antagonist to CD40 ligand (CD40L). Approximately 54 adults with Amyotrophic Lateral Sclerosis (ALS) will be enrolled into the study in the United States and Canada at approximately 13 ALS treatment sites.
Participants will be enrolled into one of four ascending doses.
详细描述
This is a Phase 2a, multi-center, open label, multiple dose study of AT-1501, a humanized monoclonal antibody antagonist to CD40L. Approximately 54 adults with ALS will be enrolled into the study in the United States and Canada at approximately 13 ALS treatment sites.
Four ascending doses of AT-1501 will be administered as an IV infusion to sequentially enrolling cohorts. Each participant will receive 6 bi-weekly (every other week) infusions of AT-1501 over an 11-week period.
The study is estimated to take 19 weeks for participants.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •ALS diagnosed as possible, laboratory supported probable, probable, or definite as defined by revised El Escorial criteria
- •ALS Functional Rating Scale - Revised (ALSFRS-R) Aggregate score of 37 or greater
- •No more than 24 months from diagnosis
排除标准
- •Any other central or peripheral nervous system disease that may interfere with the evaluation of ALS or its progression
- •Presence of a tracheostomy, or use of permanent assistive ventilation (ventilatory support for 23 hours per day or more)
- •History of malignancy within the previous 5 years, except for localized non-melanoma skin cancers
- •Abnormal function of the immune system resulting from:
- •Clinical conditions affecting the immune system (e.g. HIV infection, agammaglobulinemia),
- •Systemic administration of corticosteroids (PO/IV/IM) at a dose equivalent to 20 mg/day of prednisone for more than 14 consecutive days within 90 days prior to screening,
- •Administration of anti-neoplastic and/or immunomodulating agents (e.g. Tumor necrosis factor alpha (TNF α) antagonists or anti-B cell antibodies) or radiotherapy within 1 year prior to screening.
- •Recipient of Stem Cell or Gene Therapy
- •Positive test for Hepatitis B surface antigen, Hepatitis C antibody, or HIV.
- •History of deep venous thrombosis or pulmonary embolism
- •History of active substance abuse within the past 2 years
- •History of stroke, poorly controlled or significant cardiovascular disease, diabetes
研究组 & 干预措施
AT-1501
4 sequential dose cohorts
干预措施: AT-1501 (Drug)
结局指标
主要结局
Safety and Tolerability
时间窗: Up to 18 Weeks
Incidence of adverse events (AEs) reported as number of TEAEs (Treatment Emergent Adverse Events) having occurred.
次要结局
未报告次要终点