Gene Therapy for Knee Osteoarthritis (DONATELLO Trial)

Recruiting in Palo Alto (17 mi)

+9 other locations

Age: 18+

Sex: Any

Travel: May be covered

Time Reimbursement: Varies

Trial Phase: Phase 1

Waitlist Available

Sponsor: Genascence Corporation

Trial Summary

What is the purpose of this trial?This trial is testing a new treatment for people with painful knee osteoarthritis. The treatment uses a special virus to deliver a helpful gene directly into the knee. Researchers want to see if this single injection is safe and well-tolerated.

Eligibility Criteria

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.

Participant Groups

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.

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