One shot to the knee: gene therapy tested against arthritis pain
NCT ID NCT07831577
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 Hide 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
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for Knee arthritis are added.
By submitting, you agree to our Terms of use
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
-
The study's own enquiry address
This study publishes an address for enquiries. See it below .
-
The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
-
A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
By submitting, you agree to our Terms of use
Study contacts
-
Contact
Email: •••••@•••••
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Platelet injections face off against placebo for knee arthritis
- Can teaching pain science rewire how older adults think about knee arthritis?
- Wrist trackers vs. feelings: the Sleep-Pain puzzle in arthritis
- One shot in the knee: can hyaluronic acid quiet osteoarthritis pain?
- Light weights, tight cuff: a new way to prep knees for surgery?
- Inside or outside the knee: which steroid injection spot eases arthritis pain better?