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NCT Number: NCT05923905

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.

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Key information

Conditions

Sporadic and Familial Amyotrophic Lateral Sclerosis Amyotrophic Lateral Sclerosis Central Nervous System Diseases Metabolic Diseases Motor Neuron Disease Nervous System Diseases Neurodegenerative Diseases Neuromuscular Diseases Nutritional and Metabolic Diseases Proteostasis Deficiencies Spinal Cord Diseases TDP-43 Proteinopathies

Age range

18 year–80 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 4

Primary location

Peking University Third Hospital

Beijing, Beijing Municipality, 100191, China

Location status: Recruiting

Location contact

Sun Can

CONTACT

[email protected]

+86-10-82265791

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

FB1006

Drug

30mg/day

Placebo

Drug

30mg/day

Primary outcomes

  1. ROADS (Rasch-Built Overall Amyotrophic Lateral Sclerosis Disability Scale) Score

    Time frame: 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 outcomes

  1. ALSFRS-R (Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised) Score

    Time frame: 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.

  2. ALSFRS-R Score

    Time frame: 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.

  3. ROADS (Rasch-Built Overall Amyotrophic Lateral Sclerosis Disability Scale) Score

    Time frame: 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.

  4. ALSAQ-40 (Amyotrophic Lateral Sclerosis Assessment Questionnaire-40) Score

    Time frame: 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.

  5. Zung 's Self-Rating Depression Scale

    Time frame: 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.

  6. Metabolic Level

    Time frame: 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).

  7. FVC% (Forced Vital Capacity)

    Time frame: 12 weeks, 24 weeks, 36 weeks, and 48 weeks.

    Changes in FVC% during the observation period.

  8. Body Weight

    Time frame: 12 weeks, 24 weeks, 36 weeks, and 48 weeks.

    Changes in body weight during the observation period.

  9. MRC (Medical Research Council) Scale

    Time frame: 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.

  10. MUNIX (Motor Unit Number Index)

    Time frame: 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.

  11. Language Disorders

    Time frame: 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.

  12. Hand Delicate Function

    Time frame: 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 outcomes

  1. Important Events in ALS Progression

    Time frame: 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.

  2. ESS (Epworth Sleepiness Scale) Score

    Time frame: 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.

  3. PSQI (Pittsburgh Sleep Quality Index) Score

    Time frame: 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.

  4. Biomarkers

    Time frame: 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.

  5. EEG (Electroencephalogram)

    Time frame: 12 weeks, 24 weeks, 36 weeks, and 48 weeks.

    Occurrence of clinically significant abnormalities during the observation period.

  6. CMAP (Compound Muscle Action Potential)

    Time frame: 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.

  7. Adverse Events

    Time frame: 48 weeks

    Occurrence of any adverse events and clinically significant laboratory abnormalities during the study period.

Study contacts

Contact information is provided by the study sponsor or research team.

Sun Can

CONTACT

[email protected]

+86-10-82265791

Sponsors and collaborators

Lead sponsor

Peking University Third Hospital

Other

Registry information

Official study title

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

Acronym: FB1006

Important dates

Study start
2023
Primary completion
2027
Study completion
2027
First posted
Jun 28, 2023
Registry last updated
Nov 22, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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