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

Knee Osteoarthritis Clinical Trial

— DONATELLO  

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT05835895
• Other study ID #: CPRO-22-001
• Secondary ID: CLIN2-14265
• Status: Active, not recruiting
• Phase: Phase 1
• Start date: June 12, 2023
• Est. completion date: May 2029

Study information

• Verified date: May 2024
• Source: Genascence Corporation
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 67
• Est. completion date: May 2029
• Est. primary completion date: May 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 40 Years to 75 Years

Eligibility

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

Related Conditions & MeSH terms

• Arthritis
• Knee Osteoarthritis
• Osteo Arthritis Knee
• Osteoarthritis
• Osteoarthritis, Knee

Intervention

Genetic:
• GNSC-001
intra-articular injection

Drug:
• transient immune-modulation
oral
• transient immune-modulation
intra-articular injection
• Placebo
intra-articular injection

Locations

Country	Name	City	State
United States	Altoona Center for Clinical Research	Duncansville	Pennsylvania
United States	Alliance for Multispecialty Research Kansas City	Kansas City	Missouri
United States	Biosolutions Clinical Research Center	La Mesa	California
United States	Well Pharma Medical Research	Miami	Florida
United States	South Florida Research	Miami Springs	Florida
United States	Alliance for Medical Research Mobile	Mobile	Alabama
United States	West Clinical Research	Morehead City	North Carolina
United States	UC San Diego	San Diego	California
United States	Premier Medical Associates	The Villages	Florida

Sponsors (2)

Lead Sponsor	Collaborator
Genascence Corporation	California Institute for Regenerative Medicine (CIRM)

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Immunogenicity of a single dose of GNSC-001		Month 6 and 12
Primary	Incidence of treatment emergent adverse events (TEAEs) and serious adverse events (SAEs)		Month 12
Primary	Severity of TEAEs and SAEs		Month 12
Secondary	Change in synovial Interleukin-1 Receptor Antagonist (IL-1Ra) levels over time, from Baseline		Baseline to Month 1, 3, 6, 12, and 24