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

RJK Biopharma Ltd

Status and phase: Enrolling; Phase 1

Conditions: Amyotrophic Lateral Sclerosis Als

Treatments: Drug: RJK002 Intrathecal injection

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT06493279

RJK002-0001-R

Details and patient eligibility

About

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).

Enrollment: 9 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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;

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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9 participants in 1 patient group

RJK002 Intrathecal injection

Experimental group

Description:

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.

Treatment:

Drug: RJK002 Intrathecal injection