Sporadic and Familial Amyotrophic Lateral Sclerosis
Conditions
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.
Interventions
DRUGFB1006
30mg/day
DRUGPlacebo
30mg/day
Sponsors
Peking University Third Hospital
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to 80 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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. |
| 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. |
| 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. |
| 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. |
Other
| Measure | Time frame | Description |
|---|---|---|
| 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. |
| 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. |
| 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. |
Countries
China
Contacts
Primary ContactSun Can
Outcome results
None listed