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

Phase 1

Recruiting

• Conditions: Amyotrophic Lateral Sclerosis Als
• Interventions: Drug: RJK002 Intrathecal injection

• Registration Number: NCT06493279
• Lead Sponsor: RJK Biopharma Ltd

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).

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2024-07-02
• Study First Submitted QC: 2024-07-02
• Study First Posted: 2024-07-09
• Study Start: 2024-09-24
• Status Verified: 2024-11
• Last Update Submitted: 2024-11-17
• Last Update Posted: 2024-11-19
• Primary Completion: 2025-11-30
• Study Completion: 2031-05-02

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 9

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
RJK002 Intrathecal injection	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.

Primary Outcome Measures

Name	Time	Method
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 Outcome Measures

Name	Time	Method
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.

Trial Locations

Locations (1)

Peking University Third Hospital; 🇨🇳 Beijing, China