for people ages 40-75 (full criteria)
at San Diego, California and other locations
study started
completion around



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.

Official Title

A Phase 1b, Randomized, Double-Blinded, Placebo-Controlled Dose Ranging Study to Evaluate Safety, Tolerability and Pharmacodynamics of a Single Intra-articular Injection of GNSC-001 Gene Therapy in Subjects With Osteoarthritis of the Knee


Osteoarthritis, Knee, Osteo Arthritis Knee, Knee Osteoarthritis, Gene therapy, Adeno-associated virus, Intra-articular, GNSC-001, Genascence, Knee pain, Knee arthritis, Osteoarthritis, Arthritis, transient immune-modulation


You can join if…

Open to people ages 40-75

  • 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])

You CAN'T join if...

  • 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


  • UC San Diego
    San Diego California 92037 United States
  • Biosolutions Clinical Research Center
    La Mesa California 91942 United States


in progress, not accepting new patients
Start Date
Completion Date
Genascence Corporation
Phase 1 research study
Study Type
About 67 people participating
Last Updated