Dear Patients,
The Department of Neurology, West China Hospital, Sichuan University is conducting a clinical study – an exploratory trial of SO‑002 for the safety and efficacy in amyotrophic lateral sclerosis (ALS). This study has been approved by the Biomedical Ethics Review Committee of West China Hospital.
ALS is a disease that usually begins in middle age or later, characterized by selective progressive degeneration and loss of upper and lower motor neurons responsible for voluntary movement, leading to muscle atrophy and weakness. The estimated disease duration from onset to death is about 40 months. Currently, two drugs are approved in China to slow ALS progression: riluzole and edaravone. Riluzole is an oral drug that inhibits glutamate release, non‑competitively inhibits excitatory amino acid receptors, and blocks voltage‑dependent Na+ channels. Edaravone is a free‑radical scavenger administered by injection, but its effect is modest.
SO‑002 is a low‑molecular‑weight compound developed by SOCIUM Inc. It inhibits neuronal cell death by suppressing the aggregation of TAR DNA‑binding protein 43 (TDP‑43). SO‑002 was identified through AI analysis of gene expression data from ALS patients among a set of approved drugs. It inhibits TDP‑43 aggregation and suppresses neuronal cell death. TDP‑43 is observed in motor neurons of approximately 97% of ALS patients and is considered to play an important role in ALS pathophysiology. These effects have also been observed when SO‑002 is administered after TDP‑43 accumulation in neurons. In addition, SO‑002 inhibits TDP‑43 aggregation in motor neurons derived from induced pluripotent stem cells (iPSCs) of sporadic ALS patients. Based on these findings, SOCIUM Inc. has initiated development of SO‑002 as a therapeutic agent for ALS.
Main Inclusion Criteria:
(A) Inclusion Criteria
Patients with ALS who meet all of the following selection criteria and have the ability to provide consent.
At pre‑registration:
(1) Patients who meet the El Escorial revised Airlie House diagnostic criteria for "definite" or "probable" ALS;
(2) ALS severity stage 1 or 2 according to the King’s staging system;
(3) ALSFRS‑R score of ≥2 for each item (items (4) Writing and (5) Eating must be ≥2 on both sides);
(4) Normal respiratory function (%FVC ≥80% as measured);
(5) Patients who commit to taking riluzole at a stable dose during the observation period and double‑blind treatment period;
(6) Diagnosis of ALS for less than 2 years at the time of signing informed consent;
(7) Age between 18 and 75 years at the time of obtaining written consent;
(8) Patients who have provided written informed consent to participate in the study.
At enrollment (in addition to pre‑registration criteria):
(9) Patients with a change in ALSFRS‑R score between –1 and –4 during the 12‑week observation period.
(B) Exclusion Criteria
Patients with ALS who meet any of the following exclusion criteria will be excluded from the study.
At pre‑registration:
(1) Patients with impaired respiratory function or complaints of dyspnea (score ≤3 on any of the following three ALSFRS‑R respiratory items: 1) dyspnea, 2) orthopnea, 3) respiratory insufficiency);
(2) Patients with Parkinson’s disease or parkinsonian syndromes, schizophrenia, dementia, or other comorbidities that may significantly affect evaluation of drug efficacy;
(3) Severe renal impairment: creatinine clearance <30 mL/min (Cockcroft‑Gault formula), or known other severe renal disease;
(4) Severe hepatic impairment: ALT or AST >3 times the upper limit of normal, or other known liver diseases such as acute or chronic active hepatitis, cirrhosis, etc.;
(5) Acute myocardial infarction or interventional therapy within the last 6 months, or heart failure (NYHA class III‑IV);
(6) Patients with a history of cervical spondylosis, disc herniation, or other spinal surgery who plan to undergo surgery after ALS onset or during the study period;
(7) Patients whose current symptoms cannot rule out cervical spondylosis, multifocal motor neuropathy, or other diseases requiring differential diagnosis;
(8) Allergy to cycloserine;
(9) Patients with active malignant tumors receiving treatment;
(10) Patients receiving treatment for tuberculosis;
(11) Patients with a history of epilepsy;
(12) Patients with alcohol use or alcohol abuse;
(13) Severe complications (Grade 3 according to severity grading standards for adverse drug reactions, etc.);
(14) Patients who cannot agree to use contraception during the clinical trial;
(15) Pregnant, lactating, or potentially pregnant female patients;
(16) Patients participating in other clinical trials and having received investigational drug within 12 weeks prior to obtaining informed consent;
(17) Patients currently taking edaravone;
(18) Patients deemed unsuitable for the trial by the investigator or sub‑investigator.
At enrollment (additional exclusion):
(19) Patients who did not take riluzole regularly during the month before the end of the observation period.
You may withdraw from the study at any time without any impact on your medical care or rights.
If you are interested in this study, please contact us via the following:
Department: Neurology
Contact Physician: Dr. Liu
Phone: 86 13408518782
