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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 up to 5 years
Summary
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.
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.
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.
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.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ up to 5 years
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 5 years
Treatment Details
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
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.
Knee osteoarthritis: pathophysiology and current treatment modalities.
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Who is running the clinical trial?
Genascence CorporationLead Sponsor
California Institute for Regenerative Medicine (CIRM)OTHER
69 Previous Clinical Trials
3,291 Total Patients Enrolled
1 Trials studying Osteoarthritis
60 Patients Enrolled for Osteoarthritis
Jeymi Tambiah, MBChBStudy DirectorGenascence Corporation
Lachy McLean, MDStudy DirectorGenascence Corporation
Anke Fierlinger, MDStudy DirectorGenascence Corporation
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I have or had inflammatory arthritis, connective tissue inflammation, or an immune disease.I plan to have surgery or injections in my knee or other joints within a year.I have not had knee surgery in the last 6 months.I have joint issues due to a condition like Ehler's Danlos.My knee arthritis is moderate to severe, confirmed by an X-ray.My knee pain score is 20 or more out of 50.My doctor can likely extract fluid from my joints.My knee pain remains severe despite trying multiple treatments for over 3 months.I have had a partial or total knee replacement.
Research Study Groups:
This trial has the following groups:- Group 1: GNSC-001 (high dose) + transient immune-modulation (oral + injectable)
- Group 2: GNSC-001 (low dose) + transient immune-modulation (oral)
- Group 3: GNSC-001 (high dose) + transient immune-modulation (oral)
- Group 4: GNSC-001 (low dose)
- Group 5: GNSC-001 (high dose)
- Group 6: Placebo
Awards:
This trial has 0 awards, including:Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.