Knee Arthritis OsteoarthritisPhase 2/3RAPHAELNCT07831577
Efficacy and Safety of GNSC-001 Intra-articular Injection for Knee Osteoarthritis
A Phase 2/3 study testing Gene Therapy GNSC-001 in people with Knee Arthritis Osteoarthritis and Knee Arthritis, Osteoarthritis, run by Genascence Corporation.
Trial summary
The goal of this clinical trial is to learn if GNSC-001 works to treat knee osteoarthritis in adults. It will also learn about the safety of GNSC-001. The main questions it aims to answer are: 1. Does GNSC-001 lower daily knee joint pain and improve function in participants? 2. Read more about this study
- Who is it for?
- Ages 40 to 80 years
- Conditions the study is for
- Knee Arthritis Osteoarthritis, Knee Arthritis, Osteoarthritis, Knee Arthritis
- What is being tested?
- Gene Therapy GNSC-001
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 240 participants
- Where
- No sites listed yet
- Study dates
- Jul 2027to Oct 2030
- Phase
- Phase 2/3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 40 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 15Show fewer
- 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
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: A Randomized, Double-blind, Placebo-controlled, Phase 2/3, Study to Evaluate the Efficacy and Safety of an Intraarticular Injection of GNSC-001 in Participants With Osteoarthritis of the Knee
The goal of this clinical trial is to learn if GNSC-001 works to treat knee osteoarthritis in adults. It will also learn about the safety of GNSC-001. The main questions it aims to answer are:
- Does GNSC-001 lower daily knee joint pain and improve function in participants?
- What medical problems do participants have when taking GNSC-001?
Read more
Researchers will compare GNSC-001 to a placebo (a look-alike substance that contains no drug) to see if GNSC-001 works to treat knee osteoarthritis.
Participants will:
- Receive a single injection of immune-modulation, and GNSC-001 or a placebo, in their knee
- Take an oral immune-modulating pill every day for the first 22 days
- Visit the clinic at least 9 times over 2 years for checkups and tests
- Keep a diary of their symptoms and the number of times they use a rescue medication
- Conditions
- Knee Arthritis Osteoarthritis, Knee Arthritis, Osteoarthritis, Knee Arthritis, Knee Arthritis Disease, Osteo Arthritis of the Knee, Osteoarthritic Knee Pain
- Being tested
- Gene Therapy GNSC-001, Placebo
- Keywords
- Adeno-associated virus, Gene therapy, Intra-articular, GNSC-001, Genascence, Knee pain, Knee arthritis, Osteoarthritis
- Medical subject headings
- Osteoarthritis, Pain
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Gene Therapy GNSC-001Drug
Single intra-articular injection into target knee
PlaceboDrug
Single intra-articular injection into target knee
| Study group | Role | What they receive |
|---|---|---|
| GNSC-001 | Experimental | Not described Receives: Drug: Gene Therapy GNSC-001 |
| Placebo | Placebo comparator | Not described Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from baseline in OA pain as assessed by the Weekly Average of Daily Pain (WADP) Numeric Rating Scale (NRS) at Week 52
Measured: Baseline and Week 52
Change from baseline in the function subscale of the Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC) at Week 52
Measured: Baseline and Week 52
Incidence and severity of treatment emergent adverse events (TEAEs), events of clinical interest (ECIs), and serious adverse events (SAEs)
Measured: From enrollment to the end of study at 104 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Sep 2026
- Study startJul 2027 (estimated)
- Primary completionOct 2029 (estimated)
- Study completionOct 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Knee Arthritis OsteoarthritisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 240 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Genascence Corporation
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07831577
- Sponsor's protocol ID
- CPRO-25-001
- Acronym
- RAPHAEL
- Status last verified
- Sep 2026
- First submitted
- 15 Sep 2026
- Last updated
- 21 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-blind, Placebo-controlled, Phase 2/3, Study to Evaluate the Efficacy and Safety of an Intraarticular Injection of GNSC-001 in Participants With Osteoarthritis of the Knee
- ClinicalTrials.gov ID
- NCT07831577
- Acronym / sponsor ID
- RAPHAEL / CPRO-25-001
- Conditions
- Knee Arthritis Osteoarthritis, Knee Arthritis, Osteoarthritis, Knee Arthritis, Knee Arthritis Disease, Osteo Arthritis of the Knee, Osteoarthritic Knee Pain
- Keywords
- Adeno-associated virus, Gene therapy, Intra-articular, GNSC-001, Genascence, Knee pain, Knee arthritis, Osteoarthritis
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 240 participants (estimated)
- Lead sponsor
- Genascence CorporationIndustry (pharma, biotech, devices)
- Study start
- Jul 2027 (estimated)
- Primary completion
- Oct 2029 (estimated)
- Study completion
- Oct 2030 (estimated)
- First posted
- 21 Sep 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The goal of this clinical trial is to learn if GNSC-001 works to treat knee osteoarthritis in adults. It will also learn about the safety of GNSC-001. The main questions it aims to answer are:
- Does GNSC-001 lower daily knee joint pain and improve function in participants?
- What medical problems do participants have when taking GNSC-001?
Researchers will compare GNSC-001 to a placebo (a look-alike substance that contains no drug) to see if GNSC-001 works to treat knee osteoarthritis.
Participants will:
- Receive a single injection of immune-modulation, and GNSC-001 or a placebo, in their knee
- Take an oral immune-modulating pill every day for the first 22 days
- Visit the clinic at least 9 times over 2 years for checkups and tests
- Keep a diary of their symptoms and the number of times they use a rescue medication
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| GNSC-001 | Experimental | Drug: Gene Therapy GNSC-001 |
| Placebo | Placebo comparator | Drug: Placebo |
Who may qualify
- Ages
- 40 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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)
Key exclusion criteria (15 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Gene Therapy GNSC-001Single intra-articular injection into target knee | Drug | GNSC-001 |
| PlaceboSingle intra-articular injection into target knee | Drug | Placebo |
Primary outcome
Change from baseline in OA pain as assessed by the Weekly Average of Daily Pain (WADP) Numeric Rating Scale (NRS) at Week 52
Time frame: Baseline and Week 52
Change from baseline in the function subscale of the Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC) at Week 52
Time frame: Baseline and Week 52
Incidence and severity of treatment emergent adverse events (TEAEs), events of clinical interest (ECIs), and serious adverse events (SAEs)
Time frame: From enrollment to the end of study at 104 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Sep 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Gene Therapy GNSC-001 in people with Knee Arthritis Osteoarthritis and Knee Arthritis, Osteoarthritis, run by Genascence Corporation.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 21 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Head of Clinical Development (408-475-8627, inquiries@genascence.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change from baseline in OA pain as assessed by the Weekly Average of Daily Pain (WADP) Numeric Rating Scale (NRS) at Week 52 (time frame: Baseline and Week 52). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Sep 2026.