Clinical trial NCT05923905: Clinical Study to Evaluate the Efficacy and Safety of FB1006 in the Treatment of ALS Patients
Official title: Randomized Double-blind Controlled Exploratory Clinical Study to Evaluate the Efficacy and Safety of FB1006 in the Treatment of ALS Patients
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE4
Conditions: Sporadic and Familial Amyotrophic Lateral Sclerosis
Lead sponsor: Peking University Third Hospital
Sponsor class: OTHER
First posted: 2023-06-28
Enrollment: 64
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Eligibility criteria: 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

Primary outcomes:
measure: ROADS (Rasch-Built Overall Amyotrophic Lateral Sclerosis Disability Scale) Score
timeFrame: 24 weeks
Secondary outcomes:
measure: ALSFRS-R (Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised) Score
timeFrame: 24 weeks
measure: ALSFRS-R Score
timeFrame: 12 weeks, 36 weeks, and 48 weeks
measure: ROADS (Rasch-Built Overall Amyotrophic Lateral Sclerosis Disability Scale) Score
timeFrame: 4 weeks, 8 weeks, 12 weeks, 16 weeks, 20 weeks, 28 weeks, 32 weeks, 36 weeks, 40 weeks, 44 weeks, and 48 weeks
measure: ALSAQ-40 (Amyotrophic Lateral Sclerosis Assessment Questionnaire-40) Score
timeFrame: 12 weeks, 24 weeks, 36 weeks, and 48 weeks
measure: Zung 's Self-Rating Depression Scale
timeFrame: 4 weeks, 8 weeks, 12 weeks, 16 weeks, 20 weeks, 28 weeks, 32 weeks, 36 weeks, 40 weeks, 44 weeks, and 48 weeks.
measure: Metabolic Level
timeFrame: 12 weeks, 24 weeks, 36 weeks, and 48 weeks.
measure: FVC% (Forced Vital Capacity)
timeFrame: 12 weeks, 24 weeks, 36 weeks, and 48 weeks.
measure: Body Weight
timeFrame: 12 weeks, 24 weeks, 36 weeks, and 48 weeks.
measure: MRC (Medical Research Council) Scale
timeFrame: 12 weeks, 24 weeks, 36 weeks, and 48 weeks.
measure: MUNIX (Motor Unit Number Index)
timeFrame: 12 weeks, 24 weeks, 36 weeks, and 48 weeks.
measure: Language Disorders
timeFrame: 12 weeks, 24 weeks, 36 weeks, and 48 weeks.
measure: Hand Delicate Function
timeFrame: 12 weeks, 24 weeks, 36 weeks, and 48 weeks.
Other outcomes:
measure: Important Events in ALS Progression
timeFrame: 12 weeks, 24 weeks, 36 weeks, and 48 weeks.
measure: ESS (Epworth Sleepiness Scale) Score
timeFrame: 12 weeks, 24 weeks, 36 weeks, and 48 weeks.
measure: PSQI (Pittsburgh Sleep Quality Index) Score
timeFrame: 12 weeks, 24 weeks, 36 weeks, and 48 weeks.
measure: Biomarkers
timeFrame: 12 weeks, 24 weeks, 36 weeks, and 48 weeks.
measure: EEG (Electroencephalogram)
timeFrame: 12 weeks, 24 weeks, 36 weeks, and 48 weeks.
measure: CMAP (Compound Muscle Action Potential)
timeFrame: 12 weeks, 24 weeks, 36 weeks, and 48 weeks.
measure: Adverse Events
timeFrame: 48 weeks
Locations:
city: Beijing
country: China
facility: Peking University Third Hospital
state: Beijing
Study officials:
affiliation: Peking University Third Hospital
name: Fan DongSheng
role: PRINCIPAL_INVESTIGATOR
References: