Gene Therapy for Knee Osteoarthritis
(DONATELLO Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a new gene therapy treatment, GNSC-001, to determine its safety for individuals with painful knee osteoarthritis (OA). Participants will receive either a high or low dose of the treatment, sometimes combined with immune-modulating medicines, or a placebo. The trial aims to discover if a single injection can manage knee pain more effectively than standard care. It seeks participants who have experienced painful knee OA for a while, despite trying other treatments like physical therapy or medications. As a Phase 1 trial, participants will be among the first to receive this new treatment, aiding researchers in understanding its effects in people.
Will I have to stop taking my current medications?
The trial information does not specify if you need to stop taking your current medications. However, it mentions that participants should have persistent symptoms despite trying conservative therapies, which might include medications like opioids and anti-inflammatory drugs.
Is there any evidence suggesting that this trial's treatments are likely to be safe?
Research has shown that GNSC-001, a gene therapy, has been tested for knee osteoarthritis with promising safety results. A previous study found that injecting GNSC-001 into knee joints was safe. Patients tolerated it well, with no major issues reported. The study tested different doses, and participants tolerated both well.
The trial also uses a method called transient immune-modulation, which temporarily adjusts the immune system. Although specific safety data on this method is limited, the overall goal is to reduce potential side effects and maintain safety.
As this is an early-phase study, the main focus is on ensuring safety. Early trials often mean researchers are still learning about the treatment's safety, but reaching this stage suggests it has demonstrated a good safety profile so far.12345Why are researchers excited about this trial's treatments?
Researchers are excited about GNSC-001 for knee osteoarthritis because it uses gene therapy, a novel approach for this condition. Unlike standard treatments like pain relievers and corticosteroid injections that primarily address symptoms, GNSC-001 aims to modify the disease process itself by targeting the genes involved in cartilage degradation and inflammation. Additionally, the use of transient immune-modulation could potentially enhance the therapy's effectiveness and safety by managing the body's immune response to the treatment. This targeted approach may offer longer-lasting relief and possibly even slow down or reverse the progression of osteoarthritis.
What evidence suggests that this trial's treatments could be effective for knee osteoarthritis?
Research shows that the gene therapy GNSC-001 could be promising for treating knee osteoarthritis. In earlier studies, GNSC-001 was generally safe, with no serious side effects. This treatment uses a harmless virus to deliver genes directly to the knee, potentially reducing pain and improving joint function. Early results suggest it could manage osteoarthritis symptoms by altering the knee's cellular environment. In this trial, some participants will receive GNSC-001 combined with transient immune-modulation to target inflammation, a major cause of osteoarthritis pain. Although researchers are still studying its effectiveness, the initial findings are encouraging.16789
Who Is on the Research Team?
Jeymi Tambiah, MBChB
Principal Investigator
Genascence Corporation
Are You a Good Fit for This Trial?
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.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive a single intra-articular injection of GNSC-001 gene therapy or placebo
Follow-up
Participants are monitored for safety and tolerability after the injection
What Are the Treatments Tested in This Trial?
Interventions
- GNSC-001
- Placebo
- Transient Immune-modulation
Trial Overview
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.
How Is the Trial Designed?
6
Treatment groups
Experimental Treatment
Placebo Group
Find a Clinic Near You
Who Is Running the Clinical Trial?
Genascence Corporation
Lead Sponsor
California Institute for Regenerative Medicine (CIRM)
Collaborator
Citations
Safety and Pharmacodynamics of GNSC-001 Intra-articular ...
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 ...
Genascence Phase 1b DONATELLO Trial Evaluating ...
Through 12 months of follow-up, data show that GNSC-001 was well tolerated, with no treatment-emergent or treatment-related deaths, serious ...
Genascence Announces Positive 12-Month Safety and ...
Genascence Announces Positive 12-Month Safety and Biomarker Data for GNSC-001 Gene Therapy in Knee Osteoarthritis Trial | PackGene Biotech.
4.
pharmacytimes.com
pharmacytimes.com/view/fda-grants-fast-track-designation-to-potential-gene-therapy-for-osteoarthritis-of-the-kneeFDA Grants Fast Track Designation to Potential Gene ...
Phase 1 clinical trial data of GNSC-001 for the treatment of OA indicated that it is well-tolerated in patients, according to Genescence. The ...
Genascence to Present Full Data from Phase 1 Clinical ...
Data from the Phase 1 clinical trial of GNSC-001 for the treatment of osteoarthritis (OA) demonstrated that it was safe and well tolerated.
Phase 1 clinical trial testing gene therapy treatment ...
"This phase 1 clinical trial confirmed that local gene therapy in knee joints with osteoarthritis is safe and able to achieve a sustained, intra ...
POS0451 A PHASE 1B, RANDOMIZED, DOUBLE- ...
The primary objective was to evaluate the safety and tolerability of two doses GNSC-001 (1x1012 vector genomes (vg) or 1x1013 vg), administered as a single IA ...
8.
investor.pacira.com
investor.pacira.com/news-releases/news-release-details/pacira-biosciences-presents-updated-three-year-dataRelease details
Pacira BioSciences Presents Updated Three-Year Data Demonstrating Sustained Efficacy and Safety of PCRX-201 Gene Therapy for Patients with ...
Gene Therapy Trial Report
Gene Therapy Trial Report Summary Safety and Pharmacodynamics of GNSC-001 Intra-articular Injection for Knee Osteoarthritis Therapy Information
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