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Gene therapy RJK002 injected into spine for ALS – first human test begins

NCT ID NCT06493279

Disease control Sponsor: RJK Biopharma Ltd Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This early-stage trial tests a single injection of RJK002, a gene therapy, into the spinal fluid of 9 people with ALS. The main goal is to see if it is safe and to find the best dose for future studies. Researchers will also track changes in daily function, but the trial is too small to prove whether it works.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
RJK002 (gene therapy given as a single injection into the spinal fluid)
What this could lead to
If safe, this could point toward a treatment that slows or stabilizes ALS progression.
What could go wrong
This is a very early, small trial (9 people) focused only on safety, not effectiveness. Gene therapies can have serious side effects, and the dose may not be strong enough to help.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1

The first testing in people. Mainly checks safety and dose, usually in a small group.

Participants

About 9 people

The number the study aims to enrol. It can still change while the study runs.

Started

Sep 2024

Expected to finish

Nov 2030

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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;

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Conditions

The condition(s) this trial relates to.

amyotrophic lateral sclerosis

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Amyotrophic lateral sclerosis (ALS)

Contacts and locations

Locations

  • Peking University Third Hospital

    Beijing, China

More trials for these conditions

Other studies related to the condition(s) this trial covers.