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).
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
9
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.
Peking University Third Hospital
Beijing, China
incidence of adverse events (AEs)/serious adverse events (SAEs)
Time frame: Each visit within 5 years after administration
Dose limiting toxicities (DLT)
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.
Time frame: 28 days after administration
Change in the ALS Functional Rating Scale-revised (ALSFRSr) Score
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.
Time frame: 28 days, 56 days, 84 days, 6 months, 12months, 18 months after administration
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.