Clinical trial NCT05835895: Safety and Pharmacodynamics of GNSC-001 Intra-articular Injection for Knee Osteoarthritis
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
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE1
Conditions: Osteoarthritis, Knee
Osteo Arthritis Knee
Knee Osteoarthritis
Keywords: Gene therapy
Adeno-associated virus
Intra-articular
GNSC-001
Genascence
Knee pain
Knee arthritis
Osteoarthritis
Lead sponsor: Genascence Corporation
Sponsor class: INDUSTRY
Collaborators: name: California Institute for Regenerative Medicine (CIRM)
First posted: 2023-04-28
Enrollment: 67
Sex: ALL
Minimum age (years): 40
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


Eligibility 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

Primary outcomes:
measure: Incidence of treatment emergent adverse events (TEAEs) and serious adverse events (SAEs)
timeFrame: Month 12
measure: Severity of TEAEs and SAEs
timeFrame: Month 12
Secondary outcomes:
measure: Synovial fluid Interleukin-1 Receptor Antagonist (IL-1Ra) levels
timeFrame: Month 12
measure: Synovial fluid Interleukin-1 Receptor Antagonist (IL-1Ra) levels
timeFrame: Months 1, 3, 6, and 24 months
measure: Change in synovial Interleukin-1 Receptor Antagonist (IL-1Ra) levels over time
timeFrame: Baseline to Months 1, 3, 6, 12 and 24
Other outcomes:
measure: Immunogenicity of a single dose of GNSC-001
timeFrame: Month 6 and 12
Locations:
city: Mobile
country: United States
facility: Alliance for Medical Research Mobile
state: Alabama
city: La Mesa
country: United States
facility: Biosolutions Clinical Research Center
state: California
city: San Diego
country: United States
facility: UC San Diego
state: California
city: Miami Springs
country: United States
facility: South Florida Research
state: Florida
city: Miami
country: United States
facility: Well Pharma Medical Research
state: Florida
city: The Villages
country: United States
facility: Premier Medical Associates
state: Florida
city: Kansas City
country: United States
facility: Alliance for Multispecialty Research Kansas City
state: Missouri
city: Morehead City
country: United States
facility: West Clinical Research
state: North Carolina
city: Duncansville
country: United States
facility: Altoona Center for Clinical Research
state: Pennsylvania
Study officials:
affiliation: Genascence Corporation
name: Jeymi Tambiah, MBChB
role: STUDY_DIRECTOR
References: