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Gene Therapy Trial Report

Summary

Efficacy and Safety of GNSC-001 Intra-articular Injection for Knee Osteoarthritis


NCTID NCT07831577 (View at clinicaltrials.gov)
Description
Development Status 🔄 Active
Indication 🔄 Osteoarthritis, Knee
Disease Ontology Term 🔄 DOID:8398
Drug Product Name 🔄 GNSC-001
Drug Product Description 🔄 AAV-CMV-IL1RN
Sponsor Genascence Corporation
Funder Type Industry
Recruitment Status
Not yet recruiting
Enrollment Count 240 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant 🔄 IL1RN
Regulatory Element CMV (promoter)
Therapeutic Modality 🔄 Gene transfer
Therapy Route 🔄 in vivo
Mechanism of Action 🔄 Overexpression of protective allele/gene
Route of Administration 🔄 Intra-articular
Drug Product Type 🔄 Viral vector
Gene Delivery System Type 🔄 Viral transduction
Vector Type 🔄 Adeno-associated virus
Viral Vector Subtype AAV2.5
Dose 1 🔄 Phase 1 study: 1E11 vg/knee, 1E12 vg/knee, 1E13 vg/knee
Dose 2 🔄 Phase 1b study (DONATELLO): 1E12 vg/knee (n=23), 1E13 vg/knee (n=24)

Study Record Dates


Current Phase Phase2, Phase3
Submit Date 2026-09-15
Completion Date 2030-10
Last Update 2026-09-21

Participation Criteria


Eligible Age 40 Years - 80 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
Key Inclusion Criteria: * OA in the target knee confirmed by radiograph and with a Kellgren-Lawrence (KL) score of 2 or 3 * OA symptoms affecting the target knee present for at least 6 months * NRS pain score 5-9 (0-10 scale) in the target knee within the 24 hours prior to the time of consent * Synovitis and/or effusion detectable on imaging * Willing to stop use of any medication taken for knee OA pain for the duration of the study, including but not limited to nonsteroidal anti-inflammatory drugs (NSAIDs) * Able to provide written informed consent and comply with the requirements of the study Key Exclusion Criteria: * Body mass index (BMI) \> 38 kg/m2 * NRS pain score in other (non-target) knee ≥ 4 * Large effusion, substantial malalignment, structural compromise, prior partial or total joint replacement of target knee * Surgery on the target knee within the 6 months prior to screening or the anticipated need for treatment of either knee prior to the Month 12 Visit. * Current or history of inflammatory conditions (e.g., gout or rheumatoid arthritis), other joint disorders, septic arthritis, nerve damage or injury, chronic pain not associated with knee OA (e.g., fibromyalgia, hip OA), or otherwise immunocompromised * Active infection * Uncontrolled diabetes mellitus, glycosylated hemoglobin (HbA1c) \>7.0% at screening * Initiation of GLP-1RA, or similar medication, within the last 12 months, or discontinued within last 6 months prior to Screening * Use of opioid medications (including tramadol or tapentadol) or medical/recreational cannabinoids (oral/inhaled) for \> 5 days within the last 30 days * Use of medications for the treatment of chronic pain, immunosuppressant medications, or intra-articular injections into the target knee within 90 days of Day 1 * Current use of anticoagulant therapy * Received live vaccine within 6 weeks of Day 1 * Participation in another interventional clinical study with experimental therapy within 90 days prior to screening * Relies on a walking aids (e.g., cane, crutch, walker, wheelchair) or a personal assistant * Pregnant, breastfeeding, or intending to become pregnant during the study
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites
Locations

Regulatory Information


Has US IND True
FDA Designations 🔄 Fast Track, Regenerative Medicine Advanced Therapy (RMAT)
Recent Updates The last patient exited the study in March 2025, and data are being analyzed

Resources/Links