Clinical Study to Evaluate the Efficacy and Safety of FB1006 in the Treatment of ALS Patients
Study Details
Study Description
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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 4 |
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.
Study Design
Arms and Interventions
| 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
|
Outcome Measures
Primary Outcome Measures
- 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
- 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.
- 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.
- 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.
- 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).
- FVC% (Forced Vital Capacity) [12 weeks, 24 weeks, 36 weeks, and 48 weeks.]
Changes in FVC% during the observation period.
- Body Weight [12 weeks, 24 weeks, 36 weeks, and 48 weeks.]
Changes in body weight during the observation period.
- 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.
- 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.
- 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.
Other Outcome Measures
- Important Events in ALS Progression [12 weeks, 24 weeks, 36 weeks, and 48 weeks.]
Occurrence of important events in ALS Progression, include indwelling gastric tube/gastrostomy, respiratory support, death, etc.
- ESS (Epworth Sleepiness Scale) Score [12 weeks, 24 weeks, 36 weeks, and 48 weeks.]
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.
- PSQI (Pittsburgh Sleep Quality Index) Score [12 weeks, 24 weeks, 36 weeks, and 48 weeks.]
Changes in PSQI during the observation period. PSQI score ranges from 0 to 21 points, and higher scores indicate poorer sleep quality.
- Biomarkers [12 weeks, 24 weeks, 36 weeks, and 48 weeks.]
Changes in serum NFL, BDNF, GDNF, inflammatory factors (TNF, IL-1, IL-6, IL-18, CRP) during the observation period.
- EEG (Electroencephalogram) [12 weeks, 24 weeks, 36 weeks, and 48 weeks.]
Occurrence of clinically significant abnormalities during the observation period.
- CMAP (Compound Muscle Action Potential) [12 weeks, 24 weeks, 36 weeks, and 48 weeks.]
Changes in CMAP during the observation period. CMAP is obtained from electromyography (EMG) in terms of medianus, peroneus, radialis, tibialis, and ulnaris muscles.
- Adverse Events [48 weeks]
Occurrence of any adverse events and clinically significant laboratory abnormalities during the study period.
Eligibility Criteria
Criteria
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
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Peking University Third Hospital | Beijing | Beijing | China | 100191 |
Sponsors and Collaborators
- Peking University Third Hospital
Investigators
- Principal Investigator: Fan DongSheng, Peking University Third Hospital
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- M2022300