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This study primarily evaluates the safety, tolerability, and efficacy of RAG - 17 in adult ALS patients with SOD1 - mutated genes in the real - world setting.
Based on the results of the previous investigator - initiated trial (IIT) of RAG - 17 in the treatment of SOD1 - ALS, this study further planned to conduct another small - sample real - world research. By administering regular and quantitative intrathecal injections of RAG - 17 to subjects who meet the inclusion criteria of the study, the safety, tolerability, and efficacy of RAG - 17 in adult ALS patients with SOD1 - mutated genes in the real - world setting will be evaluated.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Treatment Group | Experimental | Patients who meet the inclusion and exclusion criteria are required to come to the hospital at the following time points to complete the intrathecal bolus injection of quantitative RAG - 17, relevant examinations, and evaluations by the research physicians: baseline, 60 ± 3 days, 120 ± 3 days, 180 ± 3 days, 240 ± 3 days, 300 ± 3 days, 360 ± 3 days, 450 ± 3 days, 540 ± 3 days. An online follow - up will be conducted at 570 ± 3 days. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| A sterile aqueous solution of RAG - 17 preparation (sodium salt) | Drug | Patients who meet the inclusion and exclusion criteria are required to come to the hospital at the following time points to complete the intrathecal bolus injection of RAG - 17 for 150 mg: baseline, 60 ± 3 days, 120 ± 3 days, 180 ± 3 days, 240 ± 3 days, 300 ± 3 days, 360 ± 3 days, 450 ± 3 days, 540 ± 3 days. |
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of adverse events and serious adverse events | Incidence of adverse events and serious adverse events within 570 days after treating SOD1 - ALS patients with RAG - 17 in the real world, and number of participants with treatment-related adverse events as assessed by Common Terminology Criteria for Adverse Events (CTCAE) V5.0. | Baseline to 570 days |
| ALSFRS-R score | Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) score (ALSFRS-R scores from 0 to 48, score decline indicates worse outcome of ALS patients, or disease progression and disability) | baseline, 60±3 days, 120±3 days, 180±3 days, 240±3 days, 300±3 days, 360±3 days, 450±3 days, 540±3 days, and 570±3 days |
| Measure | Description | Time Frame |
|---|---|---|
| The level of SOD1 protein in cerebrospinal fluid | The level of SOD1 protein in the cerebrospinal fluid of ALS patients is positively correlated with the disease severity. Changes in the level of SOD1 protein in cerebrospinal fluid relative to the baseline at 60±3 days, 120±3 days, 180±3 days, 240±3 days, 300±3 days, 360±3 days, 450±3 days, and 540±3 days after treating SOD1 - ALS patients with RAG - 17 in the real - world setting |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Wang YiLong | Contact | 13911666571 | yilong528@gmail.com | |
| Chen WeiQI | Contact | 15652813380 | weiqichen@aliyun.com |
| Name | Affiliation | Role |
|---|---|---|
| Wang YiLong | Beijing Tiantan Hospital | Principal Investigator |
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| ID | Term |
|---|---|
| D000690 | Amyotrophic Lateral Sclerosis |
| ID | Term |
|---|---|
| D013118 | Spinal Cord Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D016472 | Motor Neuron Disease |
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| baseline, 60±3 days, 120±3 days, 180±3 days, 240±3 days, 300±3 days, 360±3 days, 450±3 days, and 540±3 days |
| Plasma neurofilament light chain protein (NFL) level | The level of neurofilament light chain (NFL) in blood of ALS patients. Value increase indicates neuroinflammation or nerve damage. Changes in plasma neurofilament light chain protein (NFL) levels relative to the baseline at 60±3 days, 120±3 days, 180±3 days, 240±3 days, 300±3 days, 360±3 days, 450±3 days, and 540±3 days after treating SOD1 - ALS patients with RAG - 17 in the real world | baseline, 60±3 days, 120±3 days, 180±3 days, 240±3 days, 300±3 days, 360±3 days, 450±3 days, and 540±3 days |
| Invasive mechanical ventilation | The time of occurrence of invasive mechanical ventilation after treating SOD1-ALS patients with RAG-17 in the real world | From date of randomization until the date of first documented event about invasive mechanical ventilation. The assessment period is up to 30 months. |
| Gastrostomy | The time of occurrence of gastrostomy after treating SOD1-ALS patients with RAG-17 in the real world | From date of randomization until the date of first documented event about gastrostomy. The assessment period is up to 30 months. |
| Death | The time of occurrence of death after treating SOD1-ALS patients with RAG-17 in the real world | From randomization date to date of death from any cause. The assessment period is up to 60 months. |
| Muscle strength (MRC Scale) | Medical Research Council Scale (MRC Scale) is an assessment that is used to measure the change in muscle strength. MRC Scales ranges from grade 0 to grade 5, where grade 0 means no muscle contraction, grade 5 means muscle has full strength. Changes in muscle strength relative to the baseline at 60±3 days, 120±3 days, 180±3 days, 240±3 days, 300±3 days, 360±3 days, 450±3 days and 540±3 days after treating SOD1-ALS patients with RAG-17 in the real world | baseline, 60±3 days, 120±3 days, 180±3 days, 240±3 days, 300±3 days, 360±3 days, 450±3 days and 540±3 |
| ALSAQ-40 | Change in Amyotrophic Lateral Sclerosis Assessment Questionnaire-40 (ALSAQ-40) score (from 40 to 200, score decline indicates worse outcome of ALS patients, or disease progression and disability). Changes in ALSAQ-40 relative to the baseline at 60±3 days, 120±3 days, 180±3 days, 240±3 days, 300±3 days, 360±3 days, 450±3 days, 540±3 days and 570±3 days after treating SOD1 - ALS patients with RAG - 17 in the real world | baseline，60±3 days, 120±3 days, 180±3 days, 240±3 days, 300±3 days, 360±3 days, 450±3 days, 540±3 days and 570±3 days |
| EQ-5D-5L | The EuroQol 5 Dimension 5 Level (EQ-5D-5L) is a self-report survey that is used to measure the changes in the quality of life. EQ-5D-5L contains 5 domains including mobility, self-care, usual activities, pain/discomfort, and anxiety/depression, and each domain is scored on a 5-level severity from Level1(no difficulty) to Level5 (extreme difficulty). Changes in EQ-5D-5L relative to the baseline at 60±3 days, 120±3 days, 180±3 days, 240±3 days, 300±3 days, 360±3 days, 450±3 days, 540±3 days and 570±3 days after treating SOD1 - ALS patients with RAG - 17 in the real world. | baseline，60±3 days, 120±3 days, 180±3 days, 240±3 days, 300±3 days, 360±3 days, 450±3 days, 540±3 days and 570±3 days |
| D019636 | Neurodegenerative Diseases |
| D057177 | TDP-43 Proteinopathies |
| D009468 | Neuromuscular Diseases |
| D057165 | Proteostasis Deficiencies |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |