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

A Single-arm, Open-label, Phase I Clinical Study to Evaluate the Safety and Efficacy of a Single Intrathecal Injection of RJK002 in Patients With Amyotrophic Lateral Sclerosis (ALS)

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 Overview

• Status: Active, not recruiting
• Conditions: Amyotrophic Lateral Sclerosis Als
• Intervention / Treatment: Drug: RJK002 Intrathecal injection

• Study Type: Interventional
• Enrollment (Estimated): 9
• Phase: Phase 1

Contacts and Locations

Study Locations

• China
  • Beijing, China
    • Peking University Third Hospital

Participation Criteria

Eligibility Criteria

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

Description

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;

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: RJK002 Intrathecal injection; 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; 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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	28 days, 56 days, 84 days, 6 months, 12months, 18 months after administration

Other Outcome Measures

Outcome Measure	Time Frame
change of concentration of neurofilament light chain (NFL) in blood and CSF	84 days, 6 months, 12months, 18 months after administration

Collaborators and Investigators

• Sponsor: RJK Biopharma Ltd
• Collaborators: Peking University Third Hospital
• Investigators: Principal Investigator: Dongsheng Fan, PhD, Peking University Third Hospital

Study record dates

Study Major Dates

• Study Start (Actual): September 24, 2024
• Primary Completion (Estimated): November 30, 2026
• Study Completion (Estimated): November 30, 2030

Study Registration Dates

• First Submitted: July 2, 2024
• First Submitted That Met QC Criteria: July 2, 2024
• First Posted (Actual): July 9, 2024

Study Record Updates

• Last Update Posted (Actual): November 20, 2025
• Last Update Submitted That Met QC Criteria: November 19, 2025
• Last Verified: November 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Central Nervous System Diseases; Nervous System Diseases; Neuromuscular Diseases; Metabolic Diseases; Neurodegenerative Diseases; Spinal Cord Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Motor Neuron Disease; Nutritional and Metabolic Diseases; Amyotrophic Lateral Sclerosis
• Other Study ID Numbers: RJK002-0001-R

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Part of the data are planed to share on Oct 2025, Oct 2026, and Sep 2032.
• IPD Sharing Supporting Information Type: CSR

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No