Safety and Pharmacodynamics of GNSC-001 Intra-articular Injection for Knee Osteoarthritis

Phase 1

Active, not recruiting

• Conditions: Osteoarthritis, Knee; Osteo Arthritis Knee; Knee Osteoarthritis
• Interventions: Genetic: GNSC-001; Drug: Placebo; Drug: transient immune-modulation

• Registration Number: NCT05835895
• Lead Sponsor: Genascence Corporation

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2023-04-19
• Study First Submitted QC: 2023-04-19
• Study First Posted: 2023-04-28
• Study Start: 2023-06-12
• Status Verified: 2024-12
• Last Update Submitted: 2024-12-20
• Last Update Posted: 2024-12-27
• Primary Completion: 2025-05
• Study Completion: 2029-05

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 67

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
GNSC-001 (low dose) + transient immune-modulation (oral)	GNSC-001	-
GNSC-001 (low dose) + transient immune-modulation (oral)	transient immune-modulation	-
GNSC-001 (high dose) + transient immune-modulation (oral)	transient immune-modulation	-
GNSC-001 (high dose) + transient immune-modulation (oral + injectable)	GNSC-001	-
Placebo	Placebo	-
GNSC-001 (high dose) + transient immune-modulation (oral)	GNSC-001	-
GNSC-001 (low dose)	GNSC-001	-
GNSC-001 (high dose) + transient immune-modulation (oral + injectable)	transient immune-modulation	-
GNSC-001 (high dose)	GNSC-001	-

Primary Outcome Measures

Name	Time	Method
Incidence of treatment emergent adverse events (TEAEs) and serious adverse events (SAEs)	Month 12
Severity of TEAEs and SAEs	Month 12

Secondary Outcome Measures

Name	Time	Method
Synovial fluid Interleukin-1 Receptor Antagonist (IL-1Ra) levels	Months 1, 3, 6, and 24 months
Change in synovial Interleukin-1 Receptor Antagonist (IL-1Ra) levels over time	Baseline to Months 1, 3, 6, 12 and 24

Trial Locations

Locations (9)

Well Pharma Medical Research; 🇺🇸 Miami, Florida, United States

South Florida Research; 🇺🇸 Miami Springs, Florida, United States

Premier Medical Associates; 🇺🇸 The Villages, Florida, United States

Biosolutions Clinical Research Center; 🇺🇸 La Mesa, California, United States

UC San Diego; 🇺🇸 San Diego, California, United States

Alliance for Multispecialty Research Kansas City; 🇺🇸 Kansas City, Missouri, United States

West Clinical Research; 🇺🇸 Morehead City, North Carolina, United States

Altoona Center for Clinical Research; 🇺🇸 Duncansville, Pennsylvania, United States

Alliance for Medical Research Mobile; 🇺🇸 Mobile, Alabama, United States