Evaluating a new injection for ALS treatment

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)

This study tests a new injection for people with early-stage ALS to see if it’s safe and how well it works.

Quick facts

• Phase: PHASE1
• Study type: Interventional
• Enrollment: 9 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: RJK Biopharma Ltd (industry)
• Locations: 1 site (Beijing)
• Trial ID: NCT06493279 on ClinicalTrials.gov

What this trial studies

This study aims to assess the safety and tolerability of a single intrathecal injection of RJK002 in patients diagnosed with amyotrophic lateral sclerosis (ALS). It will also determine the recommended Phase II dose for future studies. Participants will be monitored for any adverse effects and overall response to the treatment. The study focuses on individuals with early-stage ALS, ensuring they meet specific health criteria.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18 and older with a diagnosis of sporadic or familial ALS and specific functional and respiratory criteria.

Not a fit: Patients with other neurological diseases that may interfere with the evaluation of RJK002's efficacy will not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for patients with ALS, potentially slowing disease progression.

How similar studies have performed: While this approach is novel, similar studies targeting ALS treatments have shown promise in the past.

Eligibility 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;

Where this trial is running

Beijing

• Peking University Third Hospital — Beijing, China (RECRUITING)

Study contacts

• Principal investigator: Dongsheng Fan, PhD — Peking University Third Hospital
• Study coordinator: Chanel CI

Conditions: Amyotrophic Lateral Sclerosis Als