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

March 29, 2024 updated by: Genascence Corporation

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

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

65

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Alabama
      • Mobile, Alabama, United States, 36608
        • Recruiting
        • Alliance for Medical Research Mobile
        • Contact:
        • Principal Investigator:
          • Russel Hudgens, MD
    • California
      • La Mesa, California, United States, 91942
        • Recruiting
        • Biosolutions Clinical Research Center
        • Contact:
        • Principal Investigator:
          • Pete Hanson, MD
      • San Diego, California, United States, 92037
        • Recruiting
        • UC San Diego
        • Contact:
        • Principal Investigator:
          • Susan Bukata, MD
    • Florida
      • Miami, Florida, United States, 33173
        • Recruiting
        • Well Pharma Medical Research
        • Principal Investigator:
          • Eddie Armas, MD
        • Contact:
      • Miami Springs, Florida, United States, 33166
        • Recruiting
        • South Florida Research
        • Contact:
        • Principal Investigator:
          • Belkis Delgado, MD
      • The Villages, Florida, United States, 32159
        • Recruiting
        • Premier Medical Associates
        • Principal Investigator:
          • Sanjay Shah, MD
        • Contact:
    • Illinois
      • Oak Brook, Illinois, United States, 60523
        • Recruiting
        • Affinity Health
        • Contact:
        • Principal Investigator:
          • Sana Meah, DO
    • Missouri
      • Kansas City, Missouri, United States, 64114
        • Recruiting
        • Alliance for Multispecialty Research Kansas City
        • Contact:
        • Principal Investigator:
          • Sue Fanning, MD
    • North Carolina
      • Morehead City, North Carolina, United States, 28557
        • Recruiting
        • West Clinical Research
        • Principal Investigator:
          • Jeffrey Moore, MD
        • Contact:
    • Pennsylvania
      • Duncansville, Pennsylvania, United States, 16635
        • Recruiting
        • Altoona Center For Clinical Research
        • Contact:
        • Principal Investigator:
          • Alan Kivitz, MD, CPI

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Placebo Comparator: Placebo
Drug: Placebo
intra-articular injection
Experimental: GNSC-001 (low dose)
Genetic: GNSC-001
intra-articular injection
Other Names:
  • sc-rAAV2.5IL-1Ra
Experimental: GNSC-001 (high dose)
Genetic: GNSC-001
intra-articular injection
Other Names:
  • sc-rAAV2.5IL-1Ra
Experimental: GNSC-001 (low dose) + transient immune-modulation (oral)
Genetic: GNSC-001
intra-articular injection
Other Names:
  • sc-rAAV2.5IL-1Ra
Drug: transient immune-modulation
oral
Drug: transient immune-modulation
intra-articular injection
Experimental: GNSC-001 (high dose) + transient immune-modulation (oral)
Genetic: GNSC-001
intra-articular injection
Other Names:
  • sc-rAAV2.5IL-1Ra
Drug: transient immune-modulation
oral
Drug: transient immune-modulation
intra-articular injection
Experimental: GNSC-001 (high dose) + transient immune-modulation (oral + injectable)
Genetic: GNSC-001
intra-articular injection
Other Names:
  • sc-rAAV2.5IL-1Ra
Drug: transient immune-modulation
oral
Drug: transient immune-modulation
intra-articular injection

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Incidence of treatment emergent adverse events (TEAEs) and serious adverse events (SAEs)
Time Frame: Month 12
Month 12
Severity of TEAEs and SAEs
Time Frame: Month 12
Month 12

Secondary Outcome Measures

Outcome Measure
Time Frame
Change in synovial Interleukin-1 Receptor Antagonist (IL-1Ra) levels over time, from Baseline
Time Frame: Baseline to Month 1, 3, 6, 12, and 24
Baseline to Month 1, 3, 6, 12, and 24

Other Outcome Measures

Outcome Measure
Time Frame
Immunogenicity of a single dose of GNSC-001
Time Frame: Month 6 and 12
Month 6 and 12

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Genascence Corporation

Collaborators

California Institute for Regenerative Medicine (CIRM)

Investigators

  • Study Director: Lachy McLean, MD, Genascence Corporation

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 12, 2023

Primary Completion (Estimated)

May 1, 2025

Study Completion (Estimated)

May 1, 2029

Study Registration Dates

First Submitted

April 19, 2023

First Submitted That Met QC Criteria

April 19, 2023

First Posted (Actual)

April 28, 2023

Study Record Updates

Last Update Posted (Actual)

April 2, 2024

Last Update Submitted That Met QC Criteria

March 29, 2024

Last Verified

March 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CPRO-22-001
  • CLIN2-14265 (Other Grant/Funding Number: California Institute for Regenerative Medicine)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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