Degenerative Arthritis
Conditions
Keywords
chondrocyte cells, Osteoarthritis, gene therapy
Brief summary
The primary purpose of the this study is to evaluate the safety of TissueGene-C, a gene therapy product that uses allogenic human chondrocytes expressing Transforming Growth Factor(TGF)-β1 by assessing the inflammation at the injection site, the incidence and severity of the adverse events, the physical examination findings, and the laboratory test results after the intra-articular injection of TissueGene-C. And Secondary purpose is to evaluate the biological efficacy (knee pain, range of motion, functional tests, and MRI) of TissueGene-C and the distribution of TissueGene-C outside the injection site.
Detailed description
TissueGene-C is a biological new drug which consists of normal chondrocyte cells and transduced chondrocyte cells that express growth factor to modify osteoarthritis symptom for long term period. During clinical trial Phase 1, we compare three different dose levels of TisssueGene-C in 6 months with 12 outpatients with degenerative arthritis. The patients are randomized by three different dose levels of TisssueGene-C in 1:1:1 ratio, and they are monitored and recorded for alleviating symptoms, sports activities, function of the knee, and the presence of adverse events.
Interventions
BIOLOGICALTissueGene-C(Low dose)
3.0 x 10\^6 cells
BIOLOGICALTissueGene-C(Medium dose)
1.0 x 10\^7 cells
BIOLOGICALTissueGene-C(High dose)
3.0 x 10\^7 cells
Sponsors
Kolon Life Science
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
SINGLE (Subject)
Eligibility
Sex/Gender
ALL
Age
45 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Male or female patients 2. Patients aged 45 years or more with Grade IV \[based on the International Cartilage Repair Society(ICRS) evaluation criteria from the MRI scan results\] degenerative arthritis of the knee, whose symptoms had not been relieved by the conventional symptomatic treatment 3. Healthy, with no major physical examination, hematology, serum chemistry, and urine test findings and no significant medical history 4. Patients with cartilage damage sized 2-6 cm2 (based on the ICRS evaluation criteria from the MRI scan results) 5. Voluntarily agreed to participate in this study and signed the informed consent form
Exclusion criteria
1. Showed clinically significant hematology, serum chemistry, or urine test results at the screening visit 2. Took an anti-inflammatory medication (prescribed or over-the-counter), including natural medicine, within 14 days from the administration of the investigational product 3. Has a history of drug abuse within one year from the enrollment, or the urine test or blood alcohol test result was positive at the screening visit 4. Received any injection in the target knee within two months before the initiation of the study 5. Pregnant or breastfeeding female 6. With another joint disease apart from degenerative arthritis (e.g., systemic rheumatic inflammatory disease associated with the knee or chondrocalcinosis, hemachromatosis, inflammatory arthritis, necrosis of the trochanter, Paget's disease adjacent to a joint in the femur or the tibia, ochronosis, hemophilic arthropathy, infectious arthritis, Charcot's disease in the knee joint, villonodular synovitis, and synovial chondromas) 7. With an infectious disease, including HIV or hepatitis (HBV/HCV) 8. With a history any of the following clinically significant diseases: * heart disease \[e.g., myocardial infarction, arrhythmia, other serious heart disease, coronary artery bypass graft (CABG)\] * kidney disease (e.g., chronic renal failure, glomerulonephritis) * liver disease (e.g., liver cirrhosis, fatty liver, acute or chronic liver disease) * endocrine disease (e.g., hyperthyroidism, hypothyroidism, thyroiditis, diabetes insipidus, Cushing's disease) * insulin-dependent diabetes mellitus * medical history of or current malignant tumor In particular, the tumors that TissueGene-C may aggravate can be screened using the following tests * Leukemia : White Blood Cell level in the hematology * Osteochondroma, Chondromas, Chondroblastoma, Chondromyxoid fibroma, Chondrosarcoma : Alkaline phosphatase level in the hematology 9. Participated in another clinical trial (using the investigational drug or a medical device) within 30 days before enrollment in this study 10. Showed positive drug test results at the screening visit 11. Did not agree to use a contraceptive method (male and female)
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants with Adverse Events after the administration of TissueGene-C | 6 months | Number of Participants with Adverse Events after the administration of TissueGene-C |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in the Knee Society Clinical Rating System(KSCRS) test results | before and 7 days, 14 days, 21 days, 28 days, 3 months, and 6 months | Comparative Evaluation of knee exam |
| Changes in WOMAC scores | before and 7 days, 14 days, 21 days, 28 days, 3 months, and 6 months | Pain, stiffness, and physical function of the knee will be measured by the Western Ontario and McMaster Universities(WOMAC) |
| Changes in MRI scan | before and 28 days, 3 months, and 6 months | Change in the MRI scan results after the administration of TissueGene-C |
| Analysis of the TGF-β1 expression using Enzyme-linked Immunosorbent Assay(ELISA) and Polymerase Chain Reaction(PCR) | before and 7 days, 14 days, 21 days, 28 days, 3 months, and 6 months | Analysis of the TGF-β1 expression using Enzyme-linked Immunosorbent Assay(ELISA) and Polymerase Chain Reaction(PCR) |
| Replication-competent retrovirus test (RCR test) | before and 7 days, 14 days, 21 days, 28 days, 3 months, and 6 months | Replication-competent retrovirus test (RCR test) |
| Changes in 100 mm-VAS | before and 7 days, 14 days, 21 days, 28 days, 3 months, and 6 months | Pain of the knee will be measured by the 100mm Visual Analogue Scale (VAS) |
Countries
South Korea
Outcome results
None listed