Efficacy and Safety Study of TissueGene-C to Degenerative Arthritis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01671072
Recruitment Status : Completed
First Posted : August 23, 2012
Last Update Posted : January 22, 2015
Information provided by (Responsible Party):
Kolon Life Science

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

Condition or disease Intervention/treatment Phase
Degenerative Arthritis Biological: TissueGene-C Drug: Normal Saline Phase 2

Detailed Description:

TissueGene-C is a biological new drug which consists of normal chondrocytes and transduced chondrocytes that express growth factors to regenerate the damaged cartilage tissues.

In the clinical trial Phase 2b, the investigators compared TissueGene-C to placebo during 6 - months trial with 54 outpatients who have had degenerative arthritis. The outpatients are assigned to TissueGene-C or placebo in 1:1 ratio, and will be monitored and recorded for improving clinical symptoms, sports activities, and function of the knee, and for the presence of adverse events.

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 54 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Single (Participant)
Primary Purpose: Treatment
Official Title: A Placebo Controlled, Single-blind, Randomized, Multi-center Phase 2B Study to Determine the Efficacy and Safety of TissueGene-C, an Allogeneic Human Chondrocytes Expressing TGF-b1, in Patients With Degenerative Arthritis
Study Start Date : July 2011
Actual Primary Completion Date : October 2012
Actual Study Completion Date : January 2013

Resource links provided by the National Library of Medicine

U.S. FDA Resources

Arm Intervention/treatment
Experimental: TissueGene-C
Single intra-articular injection to the damaged knee joint a dose of 1.8 x 10^7 cells
Biological: TissueGene-C
TissueGene-C at 1.8 x 10^7 cells
Placebo Comparator: Normal Saline
Single intra-articular injection to the damaged knee joint at the same volume
Drug: Normal Saline
Sodium chloride 0.9%, 3.5ml

Primary Outcome Measures :
  1. Changes in IKDC Subjective Score. [ Time Frame: Week 0 and 24 ]
    Symptoms, sports activities, and function of the knee will be measured by the International Knee Documentation Committee (IKDC)

Secondary Outcome Measures :
  1. Changes in WOMAC scores. [ Time Frame: Week 0 and 24 ]
    Pain, stiffness, and physical function of the knee will be measured by the Western Ontario and McMaster Universities (WOMAC)

  2. Changes in KOOS scores. [ Time Frame: Week 0 and 24 ]
    Symptoms, pain and functionality of the knee joint will be evaluated via the Knee Injury and Osteoarthritis Outcome Score (KOOS)

  3. Changes in 100 mm-VAS. [ Time Frame: Week 0 and 24 ]
    Pain of the knee will be measured by the 100mm Visual Analogue Scale (VAS)

  4. MRI scan [ Time Frame: Week 0 and 24 ]
    Comparison of pre-procedure MRI scans to those obtained at pre-dosing and at months 6 and 12 following dosing by an independent radiographic reviewer.

  5. Proportion of Patients Use of Rescue Medication [ Time Frame: Week 4, 12 and 24 ]

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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 Grade 2 or 3 osteoarthritis as determined by the radiographic criteria of Kellgren and Lawrence
  5. With a Body Mass Index(BMI) of higher than18.5 and lower than 30
  6. With an International Cartilage Repair Society(ICRS) Grade IV cartilage damage in the major lesions, as confirmed through an MRI scan
  7. With major lesions concentrated in one section of the knee, and with the major lesions considered the main cause of the clinical symptoms
  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. Falls under Inclusion Criteria 6 but with the damage in the relevant lesion considered ICRS Grade IV sized more than 6 cm2
  3. Took glucosamine, chondroitin, or any natural medicine within 14 days before the injection of the investigational product (but if the 14-day washout period had elapsed, the patient can be enrolled in this study)
  4. Has taken anti-inflammatory drugs (prescribed or over-the-counter) within 14 days before the injection of the investigational product (but if the 14-day washout period had elapsed, the patient can be enrolled in this study)
  5. Has taken immunosuppressive agents, including antirheumatic drugs (e.g., methotrexate or antimetabolites) within 3 months before enrollment in this study
  6. With a history of drug abuse within one year prior to enrollment in this study, or showed positive results in the urine drug test or the serum alcohol test at the screening visit
  7. Received an injection in the target knee within two months before enrollment in this study
  8. Pregnant or breastfeeding female
  9. 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 tibia, ochronosis, hemophilic arthropathy, infectious arthritis, Charcot's disease in the knee joint, villonodular synovitis, synovial chondromas)
  10. With an infectious disease, including HIV or hepatitis
  11. With any of the following clinically significant diseases:

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01671072

Korea, Republic of
Samsung Medical Center
Gangnam-gu, Seoul, Korea, Republic of, 135-710
Seoul National Univ. Hospital
Seoul, Korea, Republic of, 110-744
Asan Medical Center
Seoul, Korea, Republic of, 138-736
Sponsors and Collaborators
Kolon Life Science
Principal Investigator: Chul Won Ha, MD, PhD Samsung Medical Center
Principal Investigator: Seong Il Bin, MD, PhD Asan Medical Center
Principal Investigator: Myung Chul Lee, MD, PhD Seoul National Univ. Hospital

Responsible Party: Kolon Life Science Identifier: NCT01671072     History of Changes
Other Study ID Numbers: KS-TGC-01-2B
First Posted: August 23, 2012    Key Record Dates
Last Update Posted: January 22, 2015
Last Verified: January 2015

Keywords provided by Kolon Life Science:
Chondrocyte cells
Gene therapy

Additional relevant MeSH terms:
Joint Diseases
Musculoskeletal Diseases
Rheumatic Diseases