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One shot to the knee: gene therapy tested against arthritis pain

NCT ID NCT07831577

What the study statuses mean

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Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting This study
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
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Sep 21, 2026 · Last updated Sep 21, 2026

Summary

Researchers are testing whether a single injection of an experimental gene therapy called GNSC-001 can reduce knee osteoarthritis pain and improve joint function in adults. The trial enrolls about 240 people with moderate knee arthritis confirmed by X-ray. Participants receive one injection of GNSC-001 or a placebo into the affected knee, plus a daily immune-modulating pill for the first 22 days. They track symptoms and pain medication use for two years while researchers compare pain relief and function between the two groups.

What this could mean

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

Active substance
GNSC-001, an experimental gene therapy injected into the knee joint
What this could lead to
If it works, a single knee injection could ease arthritis pain and improve movement for months or years, possibly delaying or avoiding knee replacement surgery.
What could go wrong
Gene therapy is experimental and may not reduce pain better than placebo. Injections into the knee can cause swelling, stiffness, or immune reactions, and the trial is still testing safety.

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 2/3

Runs two stages together: whether the treatment works, then large-scale confirmation.

Participants

About 240 people

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

Expected to start

Jul 2027

An estimate. Start dates often move.

Expected to finish

Oct 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

40 to 80 years

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.

Key Inclusion Criteria: * OA in the target knee confirmed by radiograph and with a Kellgren-Lawrence (KL) score of 2 or 3 * OA symptoms affecting the target knee present for at least 6 months * NRS pain score 5-9 (0-10 scale) in the target knee within the 24 hours prior to the time of consent * Synovitis and/or effusion detectable on imaging * Willing to stop use of any medication taken for knee OA pain for the duration of the study, including but not limited to nonsteroidal anti-inflammatory drugs (NSAIDs) * Able to provide written informed consent and comply with the requirements of the study Key Exclusion Criteria: * Body mass index (BMI) \> 38 kg/m2 * NRS pain score in other (non-target) knee ≥ 4 * Large effusion, substantial malalignment, structural compromise, prior partial or total joint replacement of target knee * Surgery on the target knee within the 6 months prior to screening or the anticipated need for treatment of either knee prior to the Month 12 Visit. * Current or history of inflammatory conditions (e.g., gout or rheumatoid arthritis), other joint disorders, septic arthritis, nerve damage or injury, chronic pain not associated with knee OA (e.g., fibromyalgia, hip OA), or otherwise immunocompromised * Active infection * Uncontrolled diabetes mellitus, glycosylated hemoglobin (HbA1c) \>7.0% at screening * Initiation of GLP-1RA, or similar medication, within the last 12 months, or discontinued within last 6 months prior to Screening * Use of opioid medications (including tramadol or tapentadol) or medical/recreational cannabinoids (oral/inhaled) for \> 5 days within the last 30 days * Use of medications for the treatment of chronic pain, immunosuppressant medications, or intra-articular injections into the target knee within 90 days of Day 1 * Current use of anticoagulant therapy * Received live vaccine within 6 weeks of Day 1 * Participation in another interventional clinical study with experimental therapy within 90 days prior to screening * Relies on a walking aids (e.g., cane, crutch, walker, wheelchair) or a personal assistant * Pregnant, breastfeeding, or intending to become pregnant during the study

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

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How to take part

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  1. The study's own enquiry address

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  2. The official record

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Contacts and locations

Study contacts

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