A Phase 1, Open-Label Study Assessing the Safety, Pharmacokinetics, Pharmacodynamics, and Biomarkers of Single Ascending Dose Regimens of XT-150 for the Treatment of Amyotrophic Lateral Sclerosis (ALS)
试验速览
- 阶段
- 1 期
- 状态
- 尚未招募
- 入组人数
- 8
- 试验地点
- 6
- 主要终点
- Number of participants reporting adverse events (AEs), Serious Adverse Events (SAEs) and AEs of Special Interest (AESIs)
研究概览
简要总结
This is a Phase 1, open-label, multi-center safety study of XT-150 in adult participants with Amyotrophic Lateral Sclerosis (ALS).
Participants providing informed consent and meeting all study eligibility criteria will be enrolled in the study and will receive a single injection of XT-150 at the Baseline visit. Follow-up visits will occur over 180 days (6 months) after the injection.
8 participants (4 participants per dose level) will be enrolled sequentially in up to 2 ascending, single dose cohorts: Cohort 1: 1.5 mg XT-150 Cohort 2: 4.5 mg XT-150
详细描述
The main questions this study is aiming to answer are:
- What adverse events are reported during this study? An adverse event is any "side effect" that participants have during a study. Adverse events may or may not be caused by XT-150. The study doctors will check participants' ALS and general health throughout the trial.
- Does XT-150 have any effects on participants' physical examination findings, vital signs, or laboratory values?
In addition to evaluating the safety of XT-150, this study will also collect samples to provide information on the following:
- The levels of XT-150 in your body
- The effect of XT-150 on cytokines including interleukin (IL)-10
- The effect of XT-150 on relevant ALS biomarkers such as Neurofilament Light (NfL)
In addition, ALS functional score, breathing capacity and strength will be collected over the course of the study.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Adults between 18 and 80 years of age
- •Male or female, if of childbearing potential or sexually active, strict contraception required
- •Have ALS diagnosed by a doctor (specifically, sporadic or familial ALS diagnosed as clinically probable, lab-supported probable or definite ALS defined by the El Escorial criteria)
- •Have had symptoms of ALS (muscle weakness) within 36 months of starting this study
- •Have the ability to slowly exhale a volume of air at least 60% of what is expected for the participant's sex, height and age
- •Have not received treatment for ALS or are currently on a stable dose of an approved treatment for ALS. Patients currently receiving Tofersen are not eligible.
- •Able to receive the study injection intrathecally, determined by the study doctor
- •Able to undergo the study procedures and adhere to the study visit schedule at the time of study entry, with an estimated life expectancy of 6 months or greater
排除标准
- •Have an implanted shunt to drain cerebrospinal fluid (CSF) or an implanted CNS catheter
- •Have an implanted of diaphragm pacing system
- •Tracheostomy
- •History or current diagnosis of cardiac conditions or ECG abnormalities indicating significant risk of safety for participants in the study
- •History or current diagnosis of respiratory conditions such as COPD
- •History or current diagnosis of cancer, chemical meningitis, HIV, Hep B, Hep C, uncontrolled diabetes
- •Presence of an autoimmune condition (for example, rheumatoid arthritis or lupus) requiring treatment or immunodeficiency
- •Clinical or laboratory evidence of hepatic or renal disease/injury.
- •Taking any prohibited medications
- •Women who are pregnant or nursing
- •Use of any investigational drugs or devices within 30 days or 5 half-lives of the study agent (whichever is longer). Exception: Observational, non-interventional clinical studies are allowed in the opinion of the study doctor.
- •Any other condition that the study doctor feels could compromise the participant's safety, ability to communicate with the study staff, or the quality of the data
研究组 & 干预措施
Cohort 2: 4.5 mg XT-150
Participants will be administered 1.5 mg of XT-150 once by intrathecal injection on Day 1. An intrathecal injection is an injection using a thin needle inserted into the spinal canal at the base of your spinal cord performed by your study doctor.
干预措施: XT-150 (Biological)
Cohort 1: 1.5 mg XT-150
Participants will be administered 1.5 mg of XT-150 once by intrathecal injection on Day 1. An intrathecal injection is an injection using a thin needle inserted into the spinal canal at the base of your spinal cord performed by your study doctor.
干预措施: XT-150 (Biological)
结局指标
主要结局
Number of participants reporting adverse events (AEs), Serious Adverse Events (SAEs) and AEs of Special Interest (AESIs)
时间窗: Up to Day 180
Number of participants with clinically significant physical examination findings
时间窗: Up to Day 180
Number of participants with clinically significant vital sign abnormalities
时间窗: Up to Day 180
Vital signs include temperature, heart rate, respiratory rate and blood pressure
Number of participants with clinically significant laboratory abnormalities
时间窗: Up to Day 180
Clinical laboratory assessments include hematology, chemistry, coagulation, lipids, C-reactive protein, erythrocyte sedimentation rate and CSF total protein, cell count and glucose
次要结局
未报告次要终点