NCT05835895

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.

Trial Health

75
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
67

participants targeted

Target at P75+ for phase_1

Timeline
37mo left

Started Jun 2023

Longer than P75 for phase_1

Geographic Reach
1 country

9 active sites

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress49%
Jun 2023May 2029

First Submitted

Initial submission to the registry

April 19, 2023

Completed
9 days until next milestone

First Posted

Study publicly available on registry

April 28, 2023

Completed
2 months until next milestone

Study Start

First participant enrolled

June 12, 2023

Completed
1.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 8, 2025

Completed
4.1 years until next milestone

Study Completion

Last participant's last visit for all outcomes

May 1, 2029

Expected
Last Updated

August 1, 2025

Status Verified

December 1, 2024

Enrollment Period

1.8 years

First QC Date

April 19, 2023

Last Update Submit

July 30, 2025

Conditions

Osteoarthritis, KneeOsteo Arthritis KneeKnee Osteoarthritis

Keywords

Gene therapyAdeno-associated virusIntra-articularGNSC-001GenascenceKnee painKnee arthritisOsteoarthritis

Outcome Measures

Primary Outcomes (2)

  • Incidence of treatment emergent adverse events (TEAEs) and serious adverse events (SAEs)

    Month 12

  • Severity of TEAEs and SAEs

    Month 12

Secondary Outcomes (3)

  • Synovial fluid Interleukin-1 Receptor Antagonist (IL-1Ra) levels

    Month 12

  • 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

Other Outcomes (1)

  • Immunogenicity of a single dose of GNSC-001

    Month 6 and 12

Study Arms (6)

GNSC-001 (low dose)

EXPERIMENTAL
Genetic: GNSC-001

GNSC-001 (low dose) + transient immune-modulation (oral)

EXPERIMENTAL
Genetic: GNSC-001Drug: transient immune-modulation

GNSC-001 (high dose)

EXPERIMENTAL
Genetic: GNSC-001

GNSC-001 (high dose) + transient immune-modulation (oral)

EXPERIMENTAL
Genetic: GNSC-001Drug: transient immune-modulation

GNSC-001 (high dose) + transient immune-modulation (oral + injectable)

EXPERIMENTAL
Genetic: GNSC-001Drug: transient immune-modulation

Placebo

PLACEBO COMPARATOR
Drug: Placebo

Interventions

GNSC-001GENETIC

intra-articular injection

Also known as: sc-rAAV2.5IL-1Ra
GNSC-001 (high dose)GNSC-001 (high dose) + transient immune-modulation (oral + injectable)GNSC-001 (high dose) + transient immune-modulation (oral)GNSC-001 (low dose)GNSC-001 (low dose) + transient immune-modulation (oral)
transient immune-modulationDRUG

oral

GNSC-001 (high dose) + transient immune-modulation (oral + injectable)GNSC-001 (high dose) + transient immune-modulation (oral)GNSC-001 (low dose) + transient immune-modulation (oral)
PlaceboDRUG

intra-articular injection

Placebo

Eligibility Criteria

Age40 Years - 75 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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 may not qualify 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (9)

Alliance for Medical Research Mobile

Mobile, Alabama, 36608, United States

Location

Biosolutions Clinical Research Center

La Mesa, California, 91942, United States

Location

UC San Diego

San Diego, California, 92037, United States

Location

Well Pharma Medical Research

Miami, Florida, 33173, United States

Location

South Florida Research

Miami Springs, Florida, 33166, United States

Location

Premier Medical Associates

The Villages, Florida, 32159, United States

Location

Alliance for Multispecialty Research Kansas City

Kansas City, Missouri, 64114, United States

Location

West Clinical Research

Morehead City, North Carolina, 28557, United States

Location

Altoona Center for Clinical Research

Duncansville, Pennsylvania, 16635, United States

Location

Related Publications (3)

  • Evans CH, Ghivizzani SC, Robbins PD. Osteoarthritis Gene Therapy: A 30-Year Journey From Concept to Clinical Trials. J Orthop Res. 2026 Feb;44(2). doi: 10.1002/jor.70141.

    PMID: 41607103DERIVED

  • Chen L, Huang FL, Tang Q, Zhao ZK, Ye ZY, Liang JH. Targeted therapy for knee osteoarthritis: From basic to clinics. Medicine (Baltimore). 2025 Aug 15;104(33):e43686. doi: 10.1097/MD.0000000000043686.

    PMID: 40826764DERIVED

  • Evans CH, Ghivizzani SC, Robbins PD. The 2024 OREF Clinical Research Award: Progress Toward a Gene Therapy for Arthritis. J Am Acad Orthop Surg. 2024 Dec 1;32(23):1052-1060. doi: 10.5435/JAAOS-D-24-00831. Epub 2024 Oct 17.

    PMID: 39284030DERIVED

MeSH Terms

Conditions

Osteoarthritis, KneeOsteoarthritis

Condition Hierarchy (Ancestors)

ArthritisJoint DiseasesMusculoskeletal DiseasesRheumatic Diseases

Study Officials

  • Jeymi Tambiah, MBChB

    Genascence Corporation

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

April 19, 2023

First Posted

April 28, 2023

Study Start

June 12, 2023

Primary Completion

April 8, 2025

Study Completion (Estimated)

May 1, 2029

Last Updated

August 1, 2025

Record last verified: 2024-12

Data Sharing

IPD Sharing
Will not share

Locations

US(9)