Clinical trial NCT01825811: Efficacy and Safety Study of TissueGene-C Mixed With Fibrin-glue for the Patients With Degenerative Arthritis
Official title: A Single-blind, Randomized, Parallel-group, Multi-center Phase 2 Study to Determine the Efficacy and Safety of TissueGene-C Mixed With Fibrin-glue in Patients With Degenerative Arthritis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Degenerative Arthritis
Keywords: Cell mediated gene therapy
Osteoarthritis
Fibrin-glue
Degenerative Arthritis
Lead sponsor: Kolon Life Science
Sponsor class: INDUSTRY
First posted: 2013-04-08
Enrollment: 18
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to determine whether TissueGene-C, allogeneic human chondrocytes expressing Transforming Growth Factor(TGF)-b1, mixed with fibrin-glue is effective and safe in patients with degenerative arthritis.

Detailed description: TissueGene-C is a biological new drug which consists of non-transduced chondrocytes and transduced chondrocytes that express TGF-b1 to regenerate the damaged cartilage.

During the clinical trial Phase 2, the investigators compare low dose or high dose TissueGene-C in 12 - months trial with 18 outpatients who have degenerative arthritis. The patients are randomized to two dose levels of TissueGene-C by 1:1 ratio, and they are monitored and recorded for alleviating symptoms, sports activities, function of the knee, and the presence of adverse events.

Eligibility criteria: Inclusion Criteria:

1. Female or male aged 18 years or more
2. Diagnosed with degenerative arthritis of the knee
3. With an IKDC score of 60 or lower at the screening visit
4. With a BMI of higher than18.5 and lower than 30
5. With an International Cartilage Repair Society(ICRS) Grade IV cartilage damage in the major lesions, as confirmed through an MRI scan
6. With major lesions concentrated in one section of the knee, and with the major lesions considered the main cause of the clinical symptoms
7. With major lesions defect size 2 cm2 \~ 10cm2
8. With no alleviation of the symptoms even after at least three months of non-surgical treatment
9. Healthy, with no major findings from the physical examination, hematology, serum chemistry, and urine tests, and no significant medical history
10. Agreed to use an effective contraceptive method during the study period
11. Voluntarily agreed to participate in this study, and signed the informed consent form

Exclusion Criteria:

1. Showed clinically significant hematology, serum chemistry, and urine test results at the screening visit
2. Mechanical axis (HKA) is greater than 5°
3. Patients receiving injections to the treated knee within 2 months prior to study entry
4. Patients who are pregnant or currently breast-feeding children
5. With another joint disease (e.g., inflammatory arthritis, infectious arthritis)
6. With an infectious disease, including HIV or hepatitis
7. With any of the following clinically significant diseases:

   * heart disease \[e.g., myocardial infarction, arrhythmia, other serious heart diseases,
   * 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 past 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)
8. Participated in another clinical trial (using the investigational drug or a medical device) within 30 days before enrollment in this study
9. Considered inappropriate by the investigator for participation in this study

Primary outcomes:
measure: Changes in IKDC Subjective Knee Evaluation
timeFrame: Week 0 and 48
Secondary outcomes:
measure: Changes in WOMAC scores
timeFrame: Week 0, 24 and 48
measure: Changes in KOOS scores
timeFrame: Week 0, 24 and 48
measure: Changes in 100 mm-VAS
timeFrame: Week 0, 24 and 48
measure: Comparative Evaluation of Knee Magnetic Resonance Images (MRIs)
timeFrame: Week 0, 24 and 48
measure: Changes in ICRS Cartilage Repair Assessment
timeFrame: week 0 and 48
measure: Changes in IKDC Subjective Knee Evaluation
timeFrame: Week 0 and 24
measure: Changes in KOOS QOL Sub-scale scores
timeFrame: Week 0, 24 and 48
measure: Proportion of Patients Use of Rescue Medication
timeFrame: Week 12, 24 and 48
Other outcomes:
Locations:
city: Daegu
country: Korea, Republic of
facility: Kyungpook National University Hospital
city: Incheon
country: Korea, Republic of
facility: Inha University Hospital
city: Seoul
country: Korea, Republic of
facility: Seoul National University Hospital
city: Seoul
country: Korea, Republic of
facility: Samsung Medical Center
city: Seoul
country: Korea, Republic of
facility: Asan Medical Center
city: Seoul
country: Korea, Republic of
facility: Ewha Womans University Mokdong Hospital
Study officials:
affiliation: Seoul National University Hospital
name: Myung Chul Lee, MD, PhD
role: PRINCIPAL_INVESTIGATOR
affiliation: Samsung Medical Center
name: Chul Won Ha, MD, PhD
role: PRINCIPAL_INVESTIGATOR
affiliation: Asan Medical Center
name: Seong Il Bin, MD, PhD
role: PRINCIPAL_INVESTIGATOR
affiliation: Inha University Hospital
name: Myung Gu Kim, MD, PhD
role: PRINCIPAL_INVESTIGATOR
affiliation: Ewha Womans University Mokdong Hospital
name: Jae Doo Yoo, MD, PhD
role: PRINCIPAL_INVESTIGATOR
affiliation: Kyungpook National University Hospital
name: Hee Su Kyung, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: