Summary

Eligibility
for people ages 40-75 (full criteria)
Location
at UCSD
Dates
study started
study ends around

Description

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.

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

Keywords

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

Eligibility

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

Locations

  • UC San Diego
    San Diego 5391811 California 5332921 92037 United States
  • Biosolutions Clinical Research Center
    La Mesa 5363990 California 5332921 91942 United States

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Genascence Corporation
ID
NCT05835895
Phase
Phase 1 research study
Study Type
Interventional
Participants
About 67 people participating
Last Updated