Osteoarthritis, KneePhase 1TGC-03-01NCT00599248
Safety Study of TissueGene-C in Degenerative Joint Disease of the Knee
A Phase 1 study testing TissueGene-C in people with Osteoarthritis, Knee, run by Kolon TissueGene, Inc. at 2 sites in United States.
Trial summary
The study is being conducted to investigate the safety and activity of TissueGene-C injected into the knee joint of patients with chronic degenerative joint disease (DJD) who will be undergoing total knee replacement. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Osteoarthritis, Knee
- What is being tested?
- TissueGene-C
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 12 participants
- Where
- 2 sitesUnited States
- Study dates
- Jan 2007to May 2010
- Phase
- Phase 1
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male or Female subjects
- Age 18 years and older
- In general good health as evidenced by physical examination, normal hematology, serum chemistry, and urinalysis screening laboratory results, and a negative history of significant organ system disorders. All laboratory values must be within 20% of normal ranges.
- Patients with Degenerative Joint Disease (DJD) of the knee that is refractory to existing drug therapies and who are scheduled for Total Knee Arthroplasty.
- Based on Radiographic findings, defect should be more than 2 cm.
- Patients providing written informed consent, after the nature of the study, are fully explained.
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- Body Mass Index (BMI) should be between '18.5 - 45.5'. The applied scale is the same for both men and women.
- Blood Pressure measurements - Systolic Blood Pressure (SBP) should be between 90-160mm. Hg, and Diastolic Blood Pressure (DBP) between 50-90mm.Hg,
- Osteoarthritis confirmed by the Radiographic Criteria of Kellgren and Lawrence
- Symptom of pain for more than four (4) consecutive months and intensity greater than Grade 4 on the 11-point numeric scale.
Who cannot join
- Patients with abnormal hematology, serum chemistry, or urinalysis screening laboratory results
- Patients taking anti-inflammatory medications (prescription or over-the-counter), including herbal therapies, within 14 days of baseline visit
- Patients with a recent (within 1 year) history of drug abuse and/or a positive urine drug/alcohol test at the time of screening
- Patients receiving injections to the treated knee within 2 months prior to study entry
- Patients who are pregnant or currently breast-feeding children
- Patients with systemic, rheumatic, or inflammatory disease of the knee or chondrocalcinosis, hemochromatosis, inflammatory arthritis, necrosis of the femoral condyle, arthropathy of the knee associated with juxta-articular Paget's disease of the femur or tibia, ochronosis, hemophilic arthropathy, infectious arthritis, Charcot's knee joint, villonodular synovitis, and synovial chondromatosis.
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- Patients with ongoing infectious disease, including HIV and hepatitis
- Patients with clinically significant cardiovascular, renal, hepatic, endocrine disease, cancer, or Type I diabetes
- Patients participating in a study of an experimental drug or medical device within 30 days of study entry.
- Positive drug screen at screening visit
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Commonwealth Orthopedics
Arlington, Virginia, 22205
Sinai Hospital of Baltimore
Baltimore, Maryland, 21215
About this study
Official title: A Phase 1 Study to Determine the Safety and Biological Activity of Cell-Mediated Gene Therapy Using TissueGene-C in Patients With Degenerative Joint Disease Prior to Total Knee Arthroplasty
The study is being conducted to investigate the safety and activity of TissueGene-C injected into the knee joint of patients with chronic degenerative joint disease (DJD) who will be undergoing total knee replacement.
Read the detailed description
The purpose of this first human study is to investigate the safety and biological activity of intra-articularly applied TissueGene-C in patients with chronic degenerative joint disease (DJD) who will be undergoing total knee replacement. In addition, data on joint pain, range of motion, and functionality will be obtained in this patient population prior to total knee replacement.
The primary objective of this study is to evaluate the safety and biological activity of intra-articularly administered TissueGene-C as evidenced by observation of the injection site for irritation or other abnormalities affecting the incidence and severity of adverse events, and the changes in physical examination findings and laboratory tests as compared to the placebo control.
The secondary objectives of this study are to:
- Evaluate the dose response of the hChonJb#7 cells in grafting at the defect as compared to the placebo control.
- Evaluate distribution of hChonJb#7 cells out of the injection site.
- Evaluate the regeneration of hyaline cartilage as determined by the histological analysis of the resected knee tissue.
- Evaluate the joint for evidence of tissue overgrowth or transformation.
- Evaluate the biological activity of TissueGene-C on joint pain, range of motion and functionality as compared to the placebo control
- Conditions
- Osteoarthritis, Knee
- Being tested
- TissueGene-C, Placebo
- Keywords
- osteoarthritis, knee
- Medical subject headings
- Osteoarthritis, Knee
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TissueGene-CBiological
TissueGene-C at 3x10e6, 1x10e7 or 3x10e7 cells/joint to be administered by intra-articular injection
PlaceboBiological
Placebo control (DMEM)
| Study group | Role | What they receive |
|---|---|---|
| 1 | Experimental | TissueGene-C single intraarticular injection of 3x10e6 cells/joint Receives: Biological: TissueGene-C |
| 2 | Experimental | TissueGene-C single intraarticular injection of 1x10e7 cells/joint Receives: Biological: TissueGene-C |
| 3 | Experimental | TissueGene-C single intraarticular injection of 3x10e7 cells/joint Receives: Biological: TissueGene-C |
| 4 | Placebo comparator | Placebo control single intraarticular injection Receives: Biological: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Summary of Adverse Events
The incidence of observations at the site of administration and the incidence adverse events assessed through 28 days after treatment.
Measured: Through 28 days post-dosing
Secondary outcomes (5)
Number of Patients Showing Engraftment at the Defect
Dose Response of the TG-C in Engrafting at the Defect as Compared to Placebo Control
Measured: 28 Days
Number of Patients With Distribution of hChonJb#7 Cells Detected Outside of the Injection Site
Number of Patients with Distribution of hChonJb#7 Cells Detected Outside of the Injection Site as determined by PCR analysis for vector DNA.
Measured: 12 Months
Number of Participants With Observable Evidence of Cartilage Regeneration
The evaluation of regeneration of hyaline cartilage as determined by histological analysis of resected knee tissue and observation for engraftment and cartilage production.
Measured: Days 0, 3, 7, 11, 28 (prior to surgery), and day 29 (one day post-surgery) following dosing. Follow-up patient monitoring will be performed at 3, 6, 9, and 12 months following dosing
Number of Patients With Tissue Overgrowth or Transformation in the Knee Joint
Visual and histological analysis of knee joint tissues to determine the number of patients with tissue overgrowth or transformation
Measured: 29
Number of Patients With Improvements in Pain and Function of the Knee Joint
Assessment of the number of patients with improvement in pain and function of the knee joint
Measured: 28 Days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Jan 2008 (estimated)
- Study startJan 2007
- Primary completionMay 2009
- Study completionMay 2010
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Osteoarthritis, KneeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Participant
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Kolon TissueGene, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Michael Mont, MD, Principal investigator, Sinai Hospital of BaltimoreDavid Romness, MD, Principal investigator, Commonwealth Orthopedics, Virginia Hospital Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00599248
- Sponsor's protocol ID
- TGC-03-01
- Acronym
- TGC-03-01
- Status last verified
- Jun 2017
- First submitted
- 11 Jan 2008
- Last updated
- 7 Jul 2017
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 Jul 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1 Study to Determine the Safety and Biological Activity of Cell-Mediated Gene Therapy Using TissueGene-C in Patients With Degenerative Joint Disease Prior to Total Knee Arthroplasty
- ClinicalTrials.gov ID
- NCT00599248
- Acronym / sponsor ID
- TGC-03-01 / TGC-03-01
- Conditions
- Osteoarthritis, Knee
- Keywords
- osteoarthritis, knee
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Kolon TissueGene, Inc.Industry (pharma, biotech, devices)
- Study start
- Jan 2007
- Primary completion
- May 2009 (actual)
- Study completion
- May 2010 (actual)
- First posted
- 23 Jan 2008 (estimated)
- Results
- Posted 30 Jun 2016
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The study is being conducted to investigate the safety and activity of TissueGene-C injected into the knee joint of patients with chronic degenerative joint disease (DJD) who will be undergoing total knee replacement.
Detailed description
The purpose of this first human study is to investigate the safety and biological activity of intra-articularly applied TissueGene-C in patients with chronic degenerative joint disease (DJD) who will be undergoing total knee replacement. In addition, data on joint pain, range of motion, and functionality will be obtained in this patient population prior to total knee replacement.
The primary objective of this study is to evaluate the safety and biological activity of intra-articularly administered TissueGene-C as evidenced by observation of the injection site for irritation or other abnormalities affecting the incidence and severity of adverse events, and the changes in physical examination findings and laboratory tests as compared to the placebo control.
The secondary objectives of this study are to:
- Evaluate the dose response of the hChonJb#7 cells in grafting at the defect as compared to the placebo control.
- Evaluate distribution of hChonJb#7 cells out of the injection site.
- Evaluate the regeneration of hyaline cartilage as determined by the histological analysis of the resected knee tissue.
- Evaluate the joint for evidence of tissue overgrowth or transformation.
- Evaluate the biological activity of TissueGene-C on joint pain, range of motion and functionality as compared to the placebo control
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (participant)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| 1 | Experimental | Biological: TissueGene-CTissueGene-C single intraarticular injection of 3x10e6 cells/joint |
| 2 | Experimental | Biological: TissueGene-CTissueGene-C single intraarticular injection of 1x10e7 cells/joint |
| 3 | Experimental | Biological: TissueGene-CTissueGene-C single intraarticular injection of 3x10e7 cells/joint |
| 4 | Placebo comparator | Biological: PlaceboPlacebo control single intraarticular injection |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Male or Female subjects
- Age 18 years and older
- In general good health as evidenced by physical examination, normal hematology, serum chemistry, and urinalysis screening laboratory results, and a negative history of significant organ system disorders. All laboratory values must be within 20% of normal ranges.
- Patients with Degenerative Joint Disease (DJD) of the knee that is refractory to existing drug therapies and who are scheduled for Total Knee Arthroplasty.
- Based on Radiographic findings, defect should be more than 2 cm.
Key exclusion criteria (10 listed)
- Patients with abnormal hematology, serum chemistry, or urinalysis screening laboratory results
- Patients taking anti-inflammatory medications (prescription or over-the-counter), including herbal therapies, within 14 days of baseline visit
- Patients with a recent (within 1 year) history of drug abuse and/or a positive urine drug/alcohol test at the time of screening
- Patients receiving injections to the treated knee within 2 months prior to study entry
- Patients who are pregnant or currently breast-feeding children
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| TissueGene-CTissueGene-C at 3x10e6, 1x10e7 or 3x10e7 cells/joint to be administered by intra-articular injection | Biological | 1, 2, 3 |
| PlaceboPlacebo control (DMEM) | Biological | 4 |
Primary outcome
Summary of Adverse Events
Time frame: Through 28 days post-dosing
The incidence of observations at the site of administration and the incidence adverse events assessed through 28 days after treatment.
5 secondary outcomes
Number of Patients Showing Engraftment at the Defect
Time frame: 28 Days
Number of Patients With Distribution of hChonJb#7 Cells Detected Outside of the Injection Site
Time frame: 12 Months
Number of Participants With Observable Evidence of Cartilage Regeneration
Time frame: Days 0, 3, 7, 11, 28 (prior to surgery), and day 29 (one day post-surgery) following dosing. Follow-up patient monitoring will be performed at 3, 6, 9, and 12 months following dosing
Number of Patients With Tissue Overgrowth or Transformation in the Knee Joint
Time frame: 29
Number of Patients With Improvements in Pain and Function of the Knee Joint
Time frame: 28 Days
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Jul 2017.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Michael Mont, MD principal investigator
Sinai Hospital of Baltimore
David Romness, MD principal investigator
Commonwealth Orthopedics, Virginia Hospital Center
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing TissueGene-C in people with Osteoarthritis, Knee, run by Kolon TissueGene, Inc. at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 7 Jul 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 30 Jun 2016). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Summary of Adverse Events (time frame: Through 28 days post-dosing). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Jul 2017.