Clinical Study to Evaluate the Efficacy and Safety of FB1006 in the Treatment of ALS Patients

Study Overview
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Brief Summary
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This is a randomized double-blind controlled exploratory clinical study to evaluate the efficacy and safety of FB1006 in the treatment of amyotrophic lateral sclerosis (ALS) patients.

Detailed Description
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This is a randomized double-blind controlled exploratory clinical study to evaluate the efficacy and safety of FB1006 in the treatment of amyotrophic lateral sclerosis (ALS) patients. The study has two phases: the first phase is 24 weeks, using a randomized double-blind placebo-controlled design; the second phase is 24 weeks, using an open-label study design. FB1006 was approved by the National Medical Products Administration（NMPA）on June 21, 2021.

Official Title
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Randomized Double-blind Controlled Exploratory Clinical Study to Evaluate the Efficacy and Safety of FB1006 in the Treatment of ALS Patients

Conditions
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Sporadic and Familial Amyotrophic Lateral Sclerosis

Intervention / Treatment
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* Drug: FB1006
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: World Federation of Neurology modified El Ecorial criteria for diagnosis of patients with laboratory support probable, clinically probable, or definite sporadic and familial amyotrophic lateral sclerosis (ALS) Age 18 to 80 years old ALS duration no longer than 18 months(from day of onset) Patient 's ALSFRS-R total scored ≥27，Each single item is scored at least 2（dyspnoea, orthopnea and respiratory insufficiency ≥3） Forced vital capacity (FVC%) no less than 70% of predicted normal for gender, height and age According to brain function AI analysis in accordance with depressive EEG characteristics Women and men of childbearing potential should use medically acceptable contraception Voluntarily participate, and sign an informed consent form Exclusion Criteria: Patients with dementia or severe neurological, psychiatric or systemic disease that is poorly controlled or may interfere with the conduct of the trial or the results of the trial Pregnant women and lactating women Suicide attempt or attempted suicide Combined with other neurological diseases similar to ALS symptoms, or affecting the evaluation of drug efficacy, such as cervical spondylotic myelopathy, lumbar spondylosis, dementia, etc. Patients with history of spinal surgery after ALS onset ALT or AST > 2 times ULN，creatinine clearance < 60 mL/min/1.73m2 (MDRD) Patients who are allergic to the investigational product Having participated in other clinical studies within 3 months before randomization Patients that the investigator considers unsuitable for participation in the study

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 80 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: FB1006 test group<br>Take FB1006 at night,30mg/day | Drug: FB1006<br>* 30mg/day<br>|
| Placebo Comparator: placebo group<br>Take placebo at night,30mg/day | Drug: Placebo<br>* 30mg/day<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| ROADS (Rasch-Built Overall Amyotrophic Lateral Sclerosis Disability Scale) Score | Changes in ROADS from baseline to 24 weeks. ROADS score ranges from 0 to 56 points, and higher scores indicate better basic daily function. | 24 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| ALSFRS-R (Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised) Score | Changes in ALSFRS-R from baseline to 24 weeks. ALSFRS-R score ranges from 0 to 48 points, and higher scores indicate milder functional impairment. | 24 weeks | 
| ALSFRS-R Score | Changes in ALSFRS-R and slope of score decrease during the observation period. ALSFRS-R score ranges from 0 to 48 points, and higher scores indicate better function. | 12 weeks, 36 weeks, and 48 weeks | 
| ROADS (Rasch-Built Overall Amyotrophic Lateral Sclerosis Disability Scale) Score | Changes in ROADS during the observation period. ROADS score ranges from 0 to 56 points, and higher scores indicate better basic daily function. | 4 weeks, 8 weeks, 12 weeks, 16 weeks, 20 weeks, 28 weeks, 32 weeks, 36 weeks, 40 weeks, 44 weeks, and 48 weeks | 
| ALSAQ-40 (Amyotrophic Lateral Sclerosis Assessment Questionnaire-40) Score | Changes in ALSAQ-40 during the observation period. ALSAQ-40 score ranges from 0 to 160 points, and higher scores indicate better quality of life. | 12 weeks, 24 weeks, 36 weeks, and 48 weeks | 
| Zung 's Self-Rating Depression Scale | Changes in Zung 's score during the observation period. Zung 's score ranges from 0 to 100 points, and higher scores indicate more severity of depression. | 4 weeks, 8 weeks, 12 weeks, 16 weeks, 20 weeks, 28 weeks, 32 weeks, 36 weeks, 40 weeks, 44 weeks, and 48 weeks. | 
| Metabolic Level | Changes in metabolic equivalent (MET) value during the observation period. MET is measured by cardiopulmonary exercise test (CPET). | 12 weeks, 24 weeks, 36 weeks, and 48 weeks. | 
| FVC% (Forced Vital Capacity) | Changes in FVC% during the observation period. | 12 weeks, 24 weeks, 36 weeks, and 48 weeks. | 
| Body Weight | Changes in body weight during the observation period. | 12 weeks, 24 weeks, 36 weeks, and 48 weeks. | 
| MRC (Medical Research Council) Scale | Changes in MRC classification during the observation period. MRC ranges from 0 to V grades, and higher grades indicate higher muscle strength. | 12 weeks, 24 weeks, 36 weeks, and 48 weeks. | 
| MUNIX (Motor Unit Number Index) | Changes in MUNIX during the observation period. MUNIX is obtained from electromyography (EMG) in terms of quadriceps, deltoid, abductor digiti minimi (ADM), and tibialis anterior (TA) muscles. | 12 weeks, 24 weeks, 36 weeks, and 48 weeks. | 
| Language Disorders | Occurrence of language disorders during the observation period. Language disorders is detected by AI (Artificial Intelligence) analysis from wearable electroencephalographic device. | 12 weeks, 24 weeks, 36 weeks, and 48 weeks. | 
| Hand Delicate Function | Changes in hand delicate function during the observation period. Hand delicate function is detected by AI (Artificial Intelligence) analysis from wearable electromyographic device. | 12 weeks, 24 weeks, 36 weeks, and 48 weeks. |