Clinical trial NCT04518540: Explore Neuroprotective Effect of Lipoic Acid in Amyotrophic Lateral Sclerosis
Official title: Randomized, Parallel Safety and Efficacy Study of Lipoic Acid in Patients With Amyotrophic Lateral Sclerosis
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: NA
Conditions: Amyotrophic Lateral Sclerosis
Lead sponsor: Second Affiliated Hospital, School of Medicine, Zhejiang University
Sponsor class: OTHER
First posted: 2020-08-19
Enrollment: 150
Sex: ALL
Minimum age (years): 20
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: In this proposed study, the investigators will evaluate the safety and efficacy of lipoic acid in treatment of Amyotrophic lateral sclerosis (ALS). The study will recruit 150 AD patients, and then these patients will be randomized to lipoic acid group or control group (75 patients per arm) for 6 courses for about 5 months. Clinical assessment will be done at screen/baseline, 3th course and 6th course. The specific aims are to compare lipoic acid versus control on: motor function and disease progression. During the study period, clinical effect index will be recorded, including bulbar function, motor function, respiratory function, and safety index including blood and urine routine, liver and kidney function, coagulation function.

Detailed description: In this proposed study, the investigators will evaluate the safety and efficacy of Lipoic acid in treatment of ALS. The study will recruit 150 ALS patients, then these patients will be randomized to lipoic acid group or control group (75 patients per arm) for 6 courses for about 5 months. Clinical efficacy and safety assessment will be done at screen/baseline, 3th course and 6th course. The specific aims are to compare lipoic acid versus placebo on: (1) Lipoic acid could improve the motor function, delay the disease progression and extend survival time in patients with ALS, measured by the ALSFRS-R Scale, ROADS Scale, upper motor neuron Scale, Muscle strength Scale and Electromyography; (2) Lung function will be collected to prove the hypothesis lipoic acid may help respiratory function. (3) Safety index including blood and urine routine, liver and kidney function, coagulation index will be recorded.

Eligibility criteria: Inclusion Criteria:

1. Age range from 20 to 75 (including 20 and 75 years old), regardless of ethnic group or gender;
2. The subjects should meet the diagnostic criteria for ALS by El Escorial revised criteria: "Definite ALS", "Probable ALS" and "Probable, laboratory-supported ALS".
3. ALS Functional Rating Scale-Revised (ALSFRS-R 12 items) each item score ≥2 points;
4. The onset (the symptoms of limbs weakness, muscle atrophy or bulbar involvement ) of the disease is less than 2 years
5. Baseline breath function: Forced Vital Capacity≥70% .
6. Disease Progression Rate FS=(48- ALSFRS-R at "time of diagnosis")/duration from onset to diagnosis (month), progression rate FS≤1;

Exclusion Criteria:

1. Combined with one of cerebrovascular disease, spinal cord disease, spinal muscular atrophy, juvenile myoatrophy of distal upper extremity, multifocal motor neuropathy, Kennedy disease, epilepsy, etc;
2. Severe renal insufficiency: creatinine clearance rate \<30 mL/min (Cockcroft-Gault formula, urea nitrogen and (or) blood creatinine\> 1.5 times the upper limit of normal, or other known severe renal insufficiency diseases;
3. Severe liver damage: ALT, AST\> 3 times the upper limit of normal, or other known liver diseases such as acute and chronic hepatitis, cirrhosis, etc.;
4. Obvious tachycardia or bradycardia; patients with acute myocardial infarction or interventional therapy in the past 6 months (patients with grade III-IV according to NYHA classification);
5. Combined with malignant tumor, blood, digestion or other serious diseases;
6. Female patients during pregnancy and lactation;
7. Participated in other clinical trials within 30 days before randomization, or are participating in other clinical trials;

Primary outcomes:
measure: change of Amyotrophic lateral sclerosis Function Rate Scale-Revised (ALSFRS-R)
timeFrame: change from baseline to 11th week& 21th week
Secondary outcomes:
measure: change of Rasch Overall ALS Disability Scale (ROADS)
timeFrame: change from baseline to 11th week& 21th week
measure: change of Upper motor neuron scale (UMNS)
timeFrame: change from baseline to 11th week& 21th week
measure: change of Muscle strength scale
timeFrame: change from baseline to 11th week& 21th week
measure: change of Pulmonary Forced Vital Capacity (PFVC)
timeFrame: change from baseline to 11th week& 21th week
measure: change of Motor Neuron Disease Electromyography
timeFrame: change from baseline EMG to 21th week
Other outcomes:
measure: Endpoint events occur rate
timeFrame: at 21th week
measure: percentage of adverse drug reaction
timeFrame: At the baseline, 11th week and 21th week
Locations:
city: Hangzhou
country: China
facility: Second Affiliated Hospital,Zhejiang University School of Medicine
state: Zhejiang
Study officials:
References: