Monosialoganglioside in Acute Ischemic Stroke: a Randomized, Blinded and Multicenter Confirmatory Study

A Randomized, Double-blind, Placebo-controlled, Multicenter Phase III Clinical Trial to Evaluate the Efficacy and Safety of Monosialoganglioside in the Treatment of Acute Ischemic Stroke

A multicenter, randomized, double-blind, parallel, placebo-controlled trial design is used to evaluate the efficacy and safety of GM1 injection in the treatment of patients with acute ischemic stroke.

It is estimated that 1706 patients will be enrolled and randomly assigned to the treatment group and the placebo control group in a 1:1 ratio. Normally, the duration of this study is approximately 90 days.

Treatment was continued for 12~14 days, and follow-up was conducted until the 90th day from the first dose.

The trial is divided into three phases: screening/baseline phase, treatment phase, and follow-up phase.

Screening/baseline period: After the subjects sign the informed consent form, they enter the screening/baseline period for screening examination.

Treatment period: Qualified subjects are randomly divided into groups in a 1:1 ratio and receive continuous treatment for 12~14 days (12~14 times) with GM1 injection and placebo respectively. During the treatment period, relevant examinations and evaluations required by the protocol will be carried out.

Follow-up period: Subjects who have completed treatment will enter the follow-up period until the 90th day of treatment.

Study Overview

• Status: Completed
• Conditions: Acute Ischemic Stroke
• Intervention / Treatment: Drug: Monosialoganglioside; Drug: Placebo

• Study Type: Interventional
• Enrollment (Actual): 1232
• Phase: Phase 3

Contacts and Locations

Study Locations

• China
  • Nanjing, China
    • Nanjing Gulou Medical College Affiliated Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Age: 18-80 years old；
• Patients with anterior circulation cerebral infarction; first stroke onset or past stroke without obvious neurological deficit (mRS score≤1)；
• Within 24 hours of onset；
• 7 ≤NIHSS score ≤ 20；
• Signed informed consent.

Exclusion Criteria:

• Patients with hereditary abnormal glucose and lipid metabolism (gangliosidosis, such as Tay-Sachs disease and retinal degeneration);
• Hemorrhagic stroke;
• Disturbance of consciousness (NIHSS1a≥1)，or cerebral arteritis, brain tumor, brain trauma, intracranial infectious diseases;
• Planed endovascular treatment;
• Uncontrolled hypertension: systolic pressure ≥200 mmHg or diastolic pressure ≥110 mmHg;
• Bleeding tendency (except for thrombolysis) or severe bleeding within 3 months;
• Patients with malignant tumor or serious diseases;
• Along with epilepsy, arthritis and other disease, which have effect on neurological assessment;
• History of autoimmune diseases, spinal trauma, various demyelinating diseases, including acute inflammatory demyelinating polyneuropathy (Guillain Barre syndrome);
• Unable or unwilling to cooperate due to mental diseases;
• Abnormal liver and renal function: ALT, AST > 2 times of the upper limit of normal value, or Cr > 1.5 times of the upper limit of normal value；
• Hypersensitivity to monosialoganglioside and excipients of test drug;
• History of drug abuse；
• Pregnant or lactating women, pregnant plan or unwilling to use effective contraception during the trial period;
• Participating in other clinical trials within 3 months;
• Other conditions which are unsuitable for this trial assessed by researcher.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Monosialoganglioside; Monosialoganglioside GM1, 200 mg/day, for 12-14 days	Drug: Monosialoganglioside; Monosialoganglioside diluted with 0.9% normal saline 100ml, iv, daily for 12-14 days
Placebo Comparator: Placebo; Placebo, for 12-14 days	Drug: Placebo; Placebo diluted with 0.9% normal saline 100ml, iv, daily for 12-14 days

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Proportion of modified Rankin Scale (mRS) 0-2	the minimum and maximum values of mRS are 0 and 6, respectively; higher mRS mean a worse outcome	90 days

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Proportion of modified Rankin Scale (mRS) 0-1	the minimum and maximum values of mRS are 0 and 6, respectively; higher mRS mean a worse outcome	90 days

Collaborators and Investigators

• Sponsor: Qilu Pharmaceutical Co., Ltd.

Study record dates

Study Major Dates

• Study Start (Actual): November 3, 2020
• Primary Completion (Actual): August 30, 2023
• Study Completion (Actual): August 30, 2023

Study Registration Dates

• First Submitted: December 16, 2024
• First Submitted That Met QC Criteria: December 16, 2024
• First Posted (Actual): March 25, 2025

Study Record Updates

• Last Update Posted (Actual): March 25, 2025
• Last Update Submitted That Met QC Criteria: December 16, 2024
• Last Verified: December 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Cerebrovascular Disorders; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Vascular Diseases; Cardiovascular Diseases; Pathologic Processes; Brain Infarction; Brain Ischemia; Infarction; Necrosis; Ischemic Stroke; Stroke; Cerebral Infarction; Ischemia
• Other Study ID Numbers: QLGM1-AIS-301

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No