Knee Osteoarthritis, Osteoarthritis, Knee, Osteo Arthritis Knee
Conditions
Keywords
Gene therapy, Adeno-associated virus, Intra-articular, GNSC-001, Genascence, Knee pain, Knee arthritis, Osteoarthritis
Brief summary
The goal of this clinical study is to determine if a single injection of 1 or more dose levels of adeno-associated virus (AAV) gene therapy (GNSC-001) is safe and tolerated compared to placebo in participants with painful osteoarthritis (OA) of the knee.
Interventions
GENETICGNSC-001
intra-articular injection
DRUGtransient immune-modulation
oral
DRUGPlacebo
intra-articular injection
Sponsors
California Institute for Regenerative Medicine (CIRM)
Genascence Corporation
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
40 Years to 75 Years
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: * OA in at least one knee (the 'target knee'), confirmed using centrally read X-ray with a Kellgren-Lawrence (KL) score of 2 or 3 * Western Ontario & McMaster University Osteoarthritis Index (WOMAC) pain score ≥ 20 (on a 50-point numerical rating scale) in the target knee during the seven days prior to Screening * Persistent moderate to severe symptoms in the target knee, despite failure or intolerance of a 3-month trial of at least 2 conservative therapies (e.g., activity modification, weight loss, physical therapy, opioids, and anti-inflammatory medications, and injection of hyaluronic acid or steroids) * Successful extraction of synovial fluid at Screening * Negative pregnancy test at Screening and Baseline (subjects of childbearing potential \[SOCBP\]) Key
Exclusion criteria
* Current or documented history of inflammatory arthritis (such as gout or rheumatoid arthritis), inflammation of connective tissue, or other immunological disease * Any form of joint degeneration as part of another syndrome (e.g., Ehler's Danlos, Stickler syndrome, etc.) * Surgery in the target knee within the 6 months prior to screening * Previous partial or total joint replacement in target knee * Significant and clinically evident malalignment of the target knee that would impact the subject's function, as determined by the Investigator * Body mass index (BMI) \> 38 kg/m2 * Clinically significant abnormal laboratory values at Screening in laboratory parameters that in the opinion of the Investigator, could represent a potential risk to participant safety: * Plans to have, elective surgery of other joints or spine or intra-articular injections in the target knee, within the next 12 months
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence of treatment emergent adverse events (TEAEs) and serious adverse events (SAEs) | Month 12 |
| Severity of TEAEs and SAEs | Month 12 |
Secondary
| Measure | Time frame |
|---|---|
| Synovial fluid Interleukin-1 Receptor Antagonist (IL-1Ra) levels | Month 12 |
| Change in synovial Interleukin-1 Receptor Antagonist (IL-1Ra) levels over time | Baseline to Months 1, 3, 6, 12 and 24 |
Other
| Measure | Time frame |
|---|---|
| Immunogenicity of a single dose of GNSC-001 | Month 6 and 12 |
Countries
United States
Outcome results
None listed