Evaluate the Safety and Efficacy of Intrathecal Injection of RJK002 in Patients With Amyotrophic Lateral Sclerosis

NCT06493279 | Active Not Recruiting Phase 1

• 1 other identifier: RJK002-0001-R
• Study Type: interventional
• Target: 9
• Locations: 1 country
• Sites: 1

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).

Conditions

Amyotrophic Lateral Sclerosis Als

Outcome Measures

Primary Outcomes (2)

• incidence of adverse events (AEs)/serious adverse events (SAEs)

  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.

  28 days after administration

Secondary Outcomes (1)

• Change in the ALS Functional Rating Scale-revised (ALSFRSr) Score

  28 days, 56 days, 84 days, 6 months, 12months, 18 months after administration

Other Outcomes (1)

• change of concentration of neurofilament light chain (NFL) in blood and CSF

  84 days, 6 months, 12months, 18 months after administration

Study Arms (1)

RJK002 Intrathecal injection

EXPERIMENTAL

Eligible subjects will receive a single intrathecal administration of investigational product. 9 subjects will be involved in 3 doses cohort from low to high: 6E13 vg/person, 1.2E14 vg/person, and 2.4E14 vg/person, 3 subjects for each cohort.

Drug: RJK002 Intrathecal injection

Interventions

RJK002 Intrathecal injection DRUG

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.

RJK002 Intrathecal injection

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Female or male subjects who are ≥ 18 years of age at screening;
• 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);
• The duration of the disease from the first symptom (any ALS symptom) prior to the screening visit must be less than 2 years (inclusive);
• 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;
• The forced vital capacity (FVC) of predicted during the screening period is ≥70% at screening;
• Body mass index (BMI) greater than 18 kg/m2 at screening;

You may not qualify if:

• 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;
• 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);
• Subjects who refuse to take food and medication by nasal feeding tube during the study period due to swallowing dysfunction;

Sponsors & Collaborators

• RJK Biopharma Ltd: lead
• Peking University Third Hospital: collaborator

Study Sites (1)

Peking University Third Hospital

Beijing, China

Location

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Dongsheng Fan, PhD

  Peking University Third Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 2, 2024
• First Posted: July 9, 2024
• Study Start: September 24, 2024
• Primary Completion (Estimated): November 30, 2026
• Study Completion (Estimated): November 30, 2030
• Last Updated: November 20, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will share
• Shared Documents: CSR

Locations

CN (1)