What side effects are associated with this type of intervention?

Common treatments for osteoarthritis that aim to modify the disease process or alleviate symptoms, such as tissue gene therapy, can have various side effects. For example, tissue gene therapy involving transforming growth factor-beta (TGF-beta) has shown statistically significant improvements in pain and function, but trends of structural improvement were not statistically significant. General side effects of gene therapy can include immune responses to the vector, local inflammation, and potential off-target effects. Platelet-rich plasma (PRP) injections, another investigational approach, may cause minor and transient risks such as pain, bleeding, tenderness, swelling, and bruising at the injection site. However, there is also a risk of local infection associated with intraarticular injections.

What prior FDA approvals exist?

Currently, there are no FDA-approved gene therapies specifically for the treatment of osteoarthritis. Traditional treatments for osteoarthritis include NSAIDs, corticosteroid injections, and physical therapy. Investigational approaches, such as the AAV gene therapy (GNSC-001), aim to modify the disease process or alleviate symptoms by delivering therapeutic genes directly to the affected tissues. These advanced therapies are still in clinical trials and have shown promise in preclinical studies, but they have not yet received FDA approval.

What other types of research exist?

Promising novel alternatives to AAV Gene Therapy (GNSC-001) for osteoarthritis patients in 2024 include: 1) Biologic treatments such as monoclonal antibodies targeting inflammatory cytokines (e.g., IL-1, TNF-alpha inhibitors). 2) Mesenchymal stem cell (MSC) therapy, which aims to repair and regenerate damaged cartilage. 3) Small molecule inhibitors targeting specific pathways involved in cartilage degradation and inflammation (e.g., MMP inhibitors, JAK inhibitors). 4) RNA-based therapies, including siRNA and antisense oligonucleotides, to modulate gene expression related to osteoarthritis pathology.

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Gene Therapy

Gene Therapy for Knee Osteoarthritis (DONATELLO Trial)

Phase 1

Waitlist Available

Research Sponsored by Genascence Corporation

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be older than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline to month 1, 3, 6, 12, and 24

Awards & highlights

DONATELLO Trial Summary

This trial will test a gene therapy injection to see if it helps reduce knee pain from osteoarthritis.

Who is the study for?

This trial is for adults with moderate to severe knee osteoarthritis, confirmed by X-ray and pain scores. Participants must have tried at least two conservative treatments without success and be able to provide a synovial fluid sample. Exclusions include recent knee surgery, BMI over 38, significant lab abnormalities, planned surgeries or injections in the next year, inflammatory arthritis history, joint replacement in the target knee, or degeneration from other syndromes.Check my eligibility

What is being tested?

The study tests if a single injection of GNSC-001 gene therapy is safe compared to placebo for those with painful knee osteoarthritis. It explores different dose levels of this AAV gene therapy which aims for transient immune-modulation to alleviate symptoms.See study design

What are the potential side effects?

While specific side effects are not listed here, typical concerns may include local reactions at the injection site such as pain or swelling, potential allergic responses to components of the therapy and systemic effects related to immune modulation.

DONATELLO Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline to month 1, 3, 6, 12, and 24

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline to month 1, 3, 6, 12, and 24

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline to month 1, 3, 6, 12, and 24 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

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

Severity of TEAEs and SAEs

Secondary outcome measures

Change in synovial Interleukin-1 Receptor Antagonist (IL-1Ra) levels over time, from Baseline

Other outcome measures

Immunogenicity of a single dose of GNSC-001

DONATELLO Trial Design

6Treatment groups

Experimental Treatment

Placebo Group

Group I: GNSC-001 (low dose) + transient immune-modulation (oral)Experimental Treatment2 Interventions

Group II: GNSC-001 (low dose)Experimental Treatment1 Intervention

Group III: GNSC-001 (high dose) + transient immune-modulation (oral)Experimental Treatment2 Interventions

Group IV: GNSC-001 (high dose) + transient immune-modulation (oral + injectable)Experimental Treatment2 Interventions

Group V: GNSC-001 (high dose)Experimental Treatment1 Intervention

Group VI: PlaceboPlacebo Group1 Intervention

Do I qualify?Apply below to find out

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Common treatments for osteoarthritis (OA) primarily aim to alleviate symptoms and manage pain. Analgesics like acetaminophen and NSAIDs such as ibuprofen and naproxen reduce pain and inflammation by inhibiting enzymes involved in the inflammatory process. Glucocorticoids, administered orally or via intraarticular injections, provide short-term relief by suppressing inflammation. However, these treatments do not alter the disease's structural progression. Investigational approaches, such as gene therapy (e.g., AAV Gene Therapy GNSC-001), aim to modify the disease process by delivering genes that can produce therapeutic proteins directly in the joint, potentially addressing the underlying causes of OA and offering longer-term benefits. This is significant for OA patients as it represents a shift from merely managing symptoms to potentially altering the disease course.

Knee osteoarthritis: pathophysiology and current treatment modalities.

Find a Location

Who is running the clinical trial?

Genascence CorporationLead Sponsor

California Institute for Regenerative Medicine (CIRM)OTHER

65 Previous Clinical Trials

3,108 Total Patients Enrolled

1 Trials studying Osteoarthritis

60 Patients Enrolled for Osteoarthritis

Lachy McLean, MDStudy DirectorGenascence Corporation

Media Library

GNSC-001 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT05835895 — Phase 1

Osteoarthritis Research Study Groups: GNSC-001 (high dose) + transient immune-modulation (oral), GNSC-001 (high dose) + transient immune-modulation (oral + injectable), GNSC-001 (low dose) + transient immune-modulation (oral), GNSC-001 (low dose), GNSC-001 (high dose), Placebo

Osteoarthritis Clinical Trial 2023: GNSC-001 Highlights & Side Effects. Trial Name: NCT05835895 — Phase 1

GNSC-001 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05835895 — Phase 1

~30 spots leftby May 2025

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