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

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

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.

Study Overview

• Status: Recruiting
• Conditions: Sporadic and Familial Amyotrophic Lateral Sclerosis
• Intervention / Treatment: Drug: FB1006; Drug: Placebo

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 Type: Interventional
• Enrollment (Estimated): 64
• Phase: Phase 4

Contacts and Locations

• Study Contact: Name: Sun Can; Phone Number: +86-10-82265791; Email: scdoctor@qq.com

Study Locations

• China
  • Beijing
    • Beijing, Beijing, China, 100191
      • Recruiting
      • Peking University Third Hospital
      • Contact: Sun Can; Phone Number: +86-10-82265791; Email: scdoctor@qq.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: FB1006 test group; Take FB1006 at night,30mg/day	Drug: FB1006; 30mg/day
Placebo Comparator: placebo group; Take placebo at night,30mg/day	Drug: Placebo; 30mg/day

What is the study measuring?

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.

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Important Events in ALS Progression	Occurrence of important events in ALS Progression, include indwelling gastric tube/gastrostomy, respiratory support, death, etc.	12 weeks, 24 weeks, 36 weeks, and 48 weeks.
ESS (Epworth Sleepiness Scale) Score	Changes in ESS score during the observation period. ESS score ranges from 0 to 24 points, and higher scores indicate more severity of daytime somnolence.	12 weeks, 24 weeks, 36 weeks, and 48 weeks.
PSQI (Pittsburgh Sleep Quality Index) Score	Changes in PSQI during the observation period. PSQI score ranges from 0 to 21 points, and higher scores indicate poorer sleep quality.	12 weeks, 24 weeks, 36 weeks, and 48 weeks.
Biomarkers	Changes in serum NFL, BDNF, GDNF, inflammatory factors (TNF, IL-1, IL-6, IL-18, CRP) during the observation period.	12 weeks, 24 weeks, 36 weeks, and 48 weeks.
EEG (Electroencephalogram)	Occurrence of clinically significant abnormalities during the observation period.	12 weeks, 24 weeks, 36 weeks, and 48 weeks.
CMAP (Compound Muscle Action Potential)	Changes in CMAP during the observation period. CMAP is obtained from electromyography (EMG) in terms of medianus, peroneus, radialis, tibialis, and ulnaris muscles.	12 weeks, 24 weeks, 36 weeks, and 48 weeks.
Adverse Events	Occurrence of any adverse events and clinically significant laboratory abnormalities during the study period.	48 weeks

Collaborators and Investigators

• Sponsor: Peking University Third Hospital
• Investigators: Principal Investigator: Fan DongSheng, Peking University Third Hospital

Study record dates

Study Major Dates

• Study Start (Actual): January 18, 2023
• Primary Completion (Estimated): September 30, 2027
• Study Completion (Estimated): September 30, 2027

Study Registration Dates

• First Submitted: February 6, 2023
• First Submitted That Met QC Criteria: June 20, 2023
• First Posted (Actual): June 28, 2023

Study Record Updates

• Last Update Posted (Estimated): November 22, 2023
• Last Update Submitted That Met QC Criteria: November 20, 2023
• Last Verified: January 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Metabolic Diseases; Central Nervous System Diseases; Nervous System Diseases; Neuromuscular Diseases; Neurodegenerative Diseases; Spinal Cord Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Motor Neuron Disease; Amyotrophic Lateral Sclerosis
• Other Study ID Numbers: M2022300

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No