NCT04569084终止3 期
A Phase 3b, Multicenter, Randomized, Double-Blind Study to Evaluate Efficacy and Safety of Oral Edaravone Administered for a Period of 48 Weeks in Subjects With Amyotrophic Lateral Sclerosis (ALS)
Shionogi189 个研究点 分布在 3 个国家目标入组 384 人开始时间: 2020年11月13日最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 终止
- 发起方
- 入组人数
- 384
- 试验地点
- 189
- 主要终点
- CAFS Score at Week 48
研究概览
简要总结
To evaluate and compare the efficacy of two dosing regimens of oral edaravone in subjects with amyotrophic lateral sclerosis (ALS) based on the change in ALS Functional Rating Scale- Revised (ALSFRS-R) score from baseline up to Week 48:
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subjects must provide a signed and dated informed consent form (ICF) to participate in the study. Subjects must be able (in the judgment of the Investigator) to understand the nature of the study and all risks involved with participation in the study. Subjects must be willing to cooperate and comply with all protocol restrictions and requirements.
- •Subjects will be male or female, ≥ 18 to 75 years of age at the time the ICF is signed.
- •Subjects will be diagnosed with Definite ALS or Probable ALS according to the El Escorial revised criteria for the diagnosis of ALS.
- •Subjects with a baseline score ≥ 2 points on each individual item of the ALSFRS- R at screening and baseline visits.
- •Subjects have a screening and baseline %forced vital capacity (FVC) ≥ 70%.
- •Subjects with 1- to 4-point decline for 8 weeks (±7 days) in ALSFRS-R total score between screening and baseline visits.
- •Subjects whose first symptom of ALS has occurred within 2 years of providing written informed consent.
排除标准
- •Exclusions Related to Primary Diagnosis
- •Subjects with a history of spinal surgery after the onset of ALS, such as surgery for cervical spondylosis or a herniated disc, or plans for such surgery during the study period.
- •Exclusions Related to Other Neurological Disorders (including, but not limited to the following)
- •Subjects with the possibility that the current symptoms may be symptoms of a disease requiring differential diagnosis, such as cervical spondylosis and multifocal motor neuropathy, cannot be ruled out.
- •Exclusions Related to General Health or Concomitant Conditions
- •Subjects undergoing treatment for a malignancy.
- •Subjects with a complication that could have a significant effect on efficacy evaluations, such as Parkinson's disease or syndrome, schizophrenia, bipolar disorder, and dementia.
- •Subjects who have the presence or history of any clinically significant (CS) disease (except ALS) that could interfere with the objectives of the study (the assessment of safety and efficacy) or the safety of the subject, as judged by the Investigator.
- •Subjects who are female, of childbearing potential, and pregnant (a positive pregnancy test) or lactating at the screening visit (Visit 1).
- •Subjects of childbearing potential unwilling to use acceptable method of contraception from the screening visit until 3 months after the last dose of study medication. Subjects who are sexually active who do not agree to use contraception during the study period.
- •Subjects who have a significant risk of suicidality. Subjects with any suicidal behavior or suicidal ideation of type 4 (active suicidal ideation with some intent to act, without a specific plan) or type 5 (active suicidal ideation with specific plan and intent) based on the Columbia-Suicide Severity Rating Scale (C-SSRS) within the 3 months before the screening visit.
- •Subjects who have alanine aminotransferase (ALT) or aspartate aminotransferase (AST) elevations greater than 2 times the upper limit of normal (ULN) at screening.
- •Subjects with a Glomerular Filtration Rate (GFR) < 30 mL/Min Per 1.73 m2 at screening, using the Larsson Equation.
- •Exclusions Related to Medications
- •Subjects with history of hypersensitivity to edaravone, any of the additives or inactive ingredients of edaravone, or sulfites.
- •Subjects with hereditary problems of fructose intolerance (eg, fructose, sucrose, invert sugar, and sorbitol).
- •Subjects who participated in another study and were administered an investigational product within 1 month or 5 half-lives of the investigational agent, whichever is longer, before providing informed consent for the present study.
- •Subjects who have received any previous treatment with edaravone.
- •Subjects who have received stem cell therapy.
- •Subjects who are unable to take their medications orally at baseline (Visit 2).
研究组 & 干预措施
MT-1186 and Placebo
Experimental
干预措施: MT-1186 (Drug)
MT-1186 and Placebo
Experimental
干预措施: Placebo (Drug)
MT-1186
Experimental
干预措施: MT-1186 (Drug)
结局指标
主要结局
CAFS Score at Week 48
时间窗: up to 48 Weeks
CAFS ranks patients' clinical outcomes based on survival time and change in the ALSFRS-R score. To calculate a patient's CAFS, each patient is compared individually to all other patients in the study. The summary score for each patient is the sum of the comparisons ( 1, 0, 1) against all other patients. After that, patients' summary scores are ranked. The CAFS rank is 1-383 and a higher CAFS rank indicates a better outcome than does a lower CAFS. Since CAFS were calculated using the imputed ALSFRS-R scores with multiple imputation method, and the maximum varies for each simulation, 383 is the maximum possible value of full range for measure of dispersion.
次要结局
- Change From Baseline in % Slow Vital Capacity (SVC) at Week 48(up to 48 Weeks)
- Change From Baseline in Amyotrophic Lateral Sclerosis Assessment Questionnaire (ALSAQ)40 at Week 48(up to 48 Weeks)
- Time to Death, Tracheostomy or Permanent Assisted Mechanical Ventilation (≥ 23 Hours/Day)(up to 48 Weeks)
- Time to Death or PAMV (≥ 23 Hours/Day)(up to 48 Weeks)
- Time to Death(up to 48 Weeks)
研究者
研究点 (189)
Loading locations...
相似试验
终止
3 期
Efficacy and Safety Extension Study of Oral Edaravone Administered in Subjects With ALSALSNCT05151471Shionogi202
已完成
1 期
Clinical Pharmacology Study of Oral Edaravone in Patients With Amyotrophic Lateral SclerosisJapanese Patients With ALSNCT04176224Shionogi9
终止
3 期
Phase III Efficacy and Tolerability Trial of the Fixed Dose Combination of Etodolac 400 mg and Cyclobenzaprine 10 mg Versus Isolated Active Substances in Pain Control After Impacted Third Molar ExtractionPain, AcuteNCT03127592Apsen Farmaceutica S.A.140
已完成
3 期
A Study to Determine the Efficacy, Safety and Tolerability of Aztreonam-Avibactam (ATM-AVI) ± Metronidazole (MTZ) Versus Meropenem (MER) ± Colistin (COL) for the Treatment of Serious Infections Due to Gram Negative Bacteria.Hosptial Acquired PneumoniaVentilator Associated PneumoniaComplicated Intra-abdominal InfectionNCT03329092Pfizer422
尚未招募
不适用
Efficacy and Safety of Intravenous + Oral Vs. Oral Amiodarone in Preventing Recurrence After Catheter Ablation in Patients with Persistent Atrial FibrillationAtrial Fibrillation (AF)NCT06742866Shanghai Chest Hospital418