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

Phase 4

Recruiting

• Conditions: Sporadic and Familial Amyotrophic Lateral Sclerosis
• Interventions: Drug: Placebo; Drug: FB1006

• Registration Number: NCT05923905
• Lead Sponsor: Peking University Third Hospital

Brief Summary

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

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.

Study Timeline

• Status Verified: 2023-01
• Study Start: 2023-01-18
• Study First Submitted: 2023-02-06
• Study First Submitted QC: 2023-06-20
• Study First Posted: 2023-06-28
• Last Update Submitted: 2023-11-20
• Last Update Posted: 2023-11-22
• Primary Completion: 2027-09-30
• Study Completion: 2027-09-30

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 64

Inclusion Criteria

1. 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)
2. Age 18 to 80 years old
3. ALS duration no longer than 18 months(from day of onset)
4. Patient 's ALSFRS-R total scored ≥27，Each single item is scored at least 2（dyspnoea, orthopnea and respiratory insufficiency ≥3）
5. Forced vital capacity (FVC%) no less than 70% of predicted normal for gender, height and age
6. According to brain function AI analysis in accordance with depressive EEG characteristics
7. Women and men of childbearing potential should use medically acceptable contraception
8. Voluntarily participate, and sign an informed consent form

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Exclusion Criteria

1. 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
2. Pregnant women and lactating women
3. Suicide attempt or attempted suicide
4. 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.
5. Patients with history of spinal surgery after ALS onset
6. ALT or AST > 2 times ULN，creatinine clearance < 60 mL/min/1.73m2 (MDRD)
7. Patients who are allergic to the investigational product
8. Having participated in other clinical studies within 3 months before randomization
9. Patients that the investigator considers unsuitable for participation in the study

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
placebo group	Placebo	Take placebo at night,30mg/day
FB1006 test group	FB1006	Take FB1006 at night,30mg/day

Primary Outcome Measures

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

Secondary Outcome Measures

Name	Time	Method
ALSFRS-R (Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised) Score	24 weeks	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.
FVC% (Forced Vital Capacity)	12 weeks, 24 weeks, 36 weeks, and 48 weeks.	Changes in FVC% during the observation period.
Metabolic Level	12 weeks, 24 weeks, 36 weeks, and 48 weeks.	Changes in metabolic equivalent (MET) value during the observation period. MET is measured by cardiopulmonary exercise test (CPET).
MRC (Medical Research Council) Scale	12 weeks, 24 weeks, 36 weeks, and 48 weeks.	Changes in MRC classification during the observation period. MRC ranges from 0 to V grades, and higher grades indicate higher muscle strength.
Hand Delicate Function	12 weeks, 24 weeks, 36 weeks, and 48 weeks.	Changes in hand delicate function during the observation period. Hand delicate function is detected by AI (Artificial Intelligence) analysis from wearable electromyographic device.
ALSFRS-R Score	12 weeks, 36 weeks, and 48 weeks	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.
MUNIX (Motor Unit Number Index)	12 weeks, 24 weeks, 36 weeks, and 48 weeks.	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.
Language Disorders	12 weeks, 24 weeks, 36 weeks, and 48 weeks.	Occurrence of language disorders during the observation period. Language disorders is detected by AI (Artificial Intelligence) analysis from wearable electroencephalographic device.
ROADS (Rasch-Built Overall Amyotrophic Lateral Sclerosis Disability Scale) Score	4 weeks, 8 weeks, 12 weeks, 16 weeks, 20 weeks, 28 weeks, 32 weeks, 36 weeks, 40 weeks, 44 weeks, and 48 weeks	Changes in ROADS during the observation period. ROADS score ranges from 0 to 56 points, and higher scores indicate better basic daily function.
ALSAQ-40 (Amyotrophic Lateral Sclerosis Assessment Questionnaire-40) Score	12 weeks, 24 weeks, 36 weeks, and 48 weeks	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.
Zung 's Self-Rating Depression Scale	4 weeks, 8 weeks, 12 weeks, 16 weeks, 20 weeks, 28 weeks, 32 weeks, 36 weeks, 40 weeks, 44 weeks, and 48 weeks.	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.
Body Weight	12 weeks, 24 weeks, 36 weeks, and 48 weeks.	Changes in body weight during the observation period.

Trial Locations

Locations (1)

Peking University Third Hospital; 🇨🇳 Beijing, Beijing, China