A Multicenter, Randomized, Double-Blind, Placebo-Controlled Phase II Study to Evaluate the Efficacy and Safety of FHND1002 Granules in Patients With Amyotrophic Lateral Sclerosis (ALS)
试验速览
- 阶段
- 2 期
- 状态
- 尚未招募
- 发起方
- 入组人数
- 180
- 主要终点
- Change from Baseline in Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) Total Score at Week 48
研究概览
简要总结
This is a Phase II clinical trial evaluating the effectiveness and safety of an investigational drug, FHND1002 granules, in adults with Amyotrophic Lateral Sclerosis (ALS).
The main goals are:
To determine if FHND1002 can slow the progression of ALS compared to a placebo.
To assess the safety and tolerability of two different doses of FHND1002 (100mg and 200mg) in ALS patients.
Approximately 180 participants will be randomly assigned (like flipping a coin) to one of three groups:
FHND1002 100mg once daily
FHND1002 200mg once daily
Placebo (an inactive substance) once daily Assignment will consider disease severity (ALSFRS-R score) and where symptoms started (Limb vs. Bulbar). Participants can continue taking stable doses of approved ALS medications (like riluzole or edaravone) or be on no medication.
The study consists of:
A Screening Period (up to 4 weeks).
A Double-Blind Treatment Period (48 weeks).
During the 48-week treatment period:
Participants will take their assigned granules orally once daily (with or without food).
They will attend clinic visits at Weeks 2, 4, 12, 24, 36, and 48 for safety checks.
Effectiveness will be measured at Weeks 12, 24, 36, and 48 using standard ALS assessments, including the ALS Functional Rating Scale-Revised (ALSFRS-R), breathing tests (FVC%), and quality of life/questionnaires (ROADS, ALSAQ-5).
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 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
- •Subjects must be ≥18 years of age at the time of signing the informed consent form.
- •Subjects must have a diagnosis of Amyotrophic Lateral Sclerosis (ALS) meeting the revised El Escorial World Federation of Neurology criteria categories including Clinically Definite ALS, Clinically Probable ALS, Laboratory-supported Probable ALS, or Clinically Possible ALS.
- •Subjects must demonstrate a documented mean monthly decline of ≥0.5 points in the ALS Functional Rating Scale-Revised (ALSFRS-R) score since initial diagnosis.
- •During screening, subjects must exhibit a percent predicted forced vital capacity (FVC%) ≥70%; note that use of non-invasive ventilation (NIV) is exclusionary.
- •Disease duration must be ≤2 years calculated from the onset of the first ALS-related symptom.
- •At screening, subjects must have an ALSFRS-R total score ≥30 with a swallowing function subscore ≥2 and all respiratory-related subscores at 4 points.
- •Subjects must have a Body Mass Index (BMI) ≥18.5 kg/m².
- •Subjects may or may not be receiving stable therapeutic doses of approved ALS medications (e.g., edaravone, riluzole) prior to enrollment, provided any existing regimen remains unchanged throughout the study.
- •Subjects must demonstrate ability to understand study procedures, willingness to comply, voluntary participation, and provide signed informed consent.
排除标准
- •Subjects with coexisting neurological disorders that may mimic ALS symptoms or interfere with efficacy assessment (e.g., cervical/lumbar spondylosis, dementia, history of seizures except childhood febrile seizures) will be excluded.
- •Subjects exhibiting motor conduction block or sensory nerve conduction abnormalities on electromyography (EMG) testing will be excluded.
- •Subjects with any history of spinal surgery within 3 months prior to screening will be excluded.
- •Subjects showing clinically significant laboratory abnormalities at screening including aspartate aminotransferase (AST) or alanine aminotransferase (ALT) >1.5 × upper limit of normal (ULN), or serum creatinine (Scr) > ULN will be excluded.
- •Subjects with severe/uncontrolled cardiac, hepatic, renal, hematologic, or neoplastic diseases, or active severe psychiatric disorders will be excluded.
- •Exclusion applies to pregnant or lactating women, and subjects planning pregnancy during the study or within 3 months post-treatment; participants of childbearing potential unwilling to use highly effective contraception throughout the study and for 3 months after last dose are excluded.
- •Subjects with known or suspected hypersensitivity to FHND1002 or its excipients will be excluded.
- •Subjects who have participated in another investigational drug/device trial within 1 month prior to screening will be excluded.
- •Subjects deemed by the Investigator to have any condition compromising safety, data integrity, or study compliance will be excluded.
研究组 & 干预措施
FHND1002 100mg
Participants receive FHND1002 granules 100mg orally once daily for 48 weeks. Administration may occur fasting or with food. Concurrent stable ALS medications (e.g., edaravone/riluzole) are permitted if maintained ≥4 weeks prior to enrollment and unchanged during the study. Standard ALS care continues throughout.
干预措施: FHND1002 100mg (Drug)
FHND1002 200mg
Participants receive FHND1002 granules 200mg orally once daily for 48 weeks. Administration may occur fasting or with food. Pre-existing stable ALS therapies (edaravone/riluzole) are allowed if dose-stable for ≥4 weeks pre-randomization and maintained during the trial. Standard supportive care is provided.
干预措施: FHND1002 200mg (Drug)
Placebo
Participants receive matching placebo granules orally once daily for 48 weeks. Administration conditions (fasting/with food) mirror active arms. Background ALS medications (edaravone/riluzole) are permitted if stabilized ≥4 weeks before enrollment and unchanged throughout the study. Standard ALS management continues.
干预措施: Placebo (Drug)
结局指标
主要结局
Change from Baseline in Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) Total Score at Week 48
时间窗: Baseline to Week 48
The difference in least-squares mean (LSMean) change from baseline to Week 48 in ALSFRS-R total score between FHND1002 treatment groups (combined or individual doses) and placebo. Missing data due to early discontinuation will be handled using multiple imputation based on observed data from the same treatment group. Analysis will use a repeated measures mixed-effects model (MMRM) adjusted for baseline ALSFRS-R score, site of onset (limb vs. bulbar), treatment group, visit, and treatment-by-visit interaction, with subject as a random effect.
次要结局
未报告次要终点