Amyotrophic Lateral Sclerosis Als
Conditions
Brief summary
The goal of the study aims to evaluate the safety and tolerability of a single intrathecal injection of RJK002 in subjects with amyotrophic lateral sclerosis (ALS), and to determine the recommended Phase II dose (RP2D).
Interventions
Eligible subjects will receive a single intrathecal administration of investigational product with dose 3E13vg (3ml), 6E13vg(6ml), and 1.2E14 vg(12ml) per person sequentially.
Sponsors
Peking University Third Hospital
RJK Biopharma Ltd
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
Main Inclusion Criteria: 1. Female or male subjects who are ≥ 18 years of age at screening; 2. Patients with a diagnosis consistent with clinically or laboratory-supported possible, probable, or definite sporadic or familial ALSALS in accordance with Revised EI Escorial diagnostic criteria published by the World Federation of Neurology (WFN); 3. The duration of the disease from the first symptom (any ALS symptom) prior to the screening visit must be less than 2 years (inclusive); 4. The Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) score ≥30 during the screening period, and the three respiratory scores (dyspnea, upright respiration, and respiratory insufficiency) must be ≥3; 5. The forced vital capacity (FVC) of predicted during the screening period is ≥70% at screening; 6. Body mass index (BMI) greater than 18 kg/m2 at screening; Main
Exclusion criteria
1. Subjects with other neurological diseases similar to ALS that affect the evaluation of drug efficacy, such as cervical spondylotic myelopathy, syringomyelia, spinal cord and brain stem tumors, hirayama disease, multifocal motor neuropathy, multiple sclerosis, Guillain-Barre syndrome, Parkinson's disease and dementia; 2. Patients with a diagnosis consistent with clinically or laboratory-supported possible, probable, or definite sporadic or familial ALSALS in accordance with Revised EI Escorial diagnostic criteria published by the World Federation of Neurology (WFN); 3. Subjects who refuse to take food and medication by nasal feeding tube during the study period due to swallowing dysfunction;
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| incidence of adverse events (AEs)/serious adverse events (SAEs) | Each visit within 5 years after administration | — |
| Dose limiting toxicities (DLT) | 28 days after administration | Measured as any drug-related serious adverse event that meet DLT standard. If a dose has less than 33% DLTs it will be considered tolerable. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in the ALS Functional Rating Scale-revised (ALSFRSr) Score | 28 days, 56 days, 84 days, 6 months, 12months, 18 months after administration | The ALSFRSr, a questionnaire-based scale assessing daily living function ranging from 48 (best score) to 0 (worst), was administered to the patient, or to a proxy if the patient could not communicate effectively. Decline was defined as ALSFRSr at baseline minus ALSFRSr at month 9. A positive value indicates worsening. |
Other
| Measure | Time frame |
|---|---|
| change of concentration of neurofilament light chain (NFL) in blood and CSF | 84 days, 6 months, 12months, 18 months after administration |
Countries
China
Outcome results
None listed