Trial Outcomes & Findings for Study of TG-C in Patients With Grade 3 Degenerative Joint Disease of the Knee (NCT NCT01221441)
NCT ID: NCT01221441
Last Updated: 2016-02-15
Results Overview
Symptoms, pain and function of the knee joint determined and scored using the International Knee Documentation Committee (IKDC) Subjective Knee Evaluation (Total Score, range 0-100 with higher scores better). Linear mixed model used for analysis.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
102 participants
Primary outcome timeframe
1 Year
Results posted on
2016-02-15
Participant Flow
Participant milestones
| Measure |
TissueGene-C
TissueGene-C at 3 x 10e7 cells per injection (intraarticular)
TissueGene-C: Single intraarticular injection at 3 x 10e7 cells
|
Placebo Control
Normal Saline injection
Normal Saline: Single intraarticular injection of normal saline as a placebo control
|
|---|---|---|
|
Overall Study
STARTED
|
67
|
35
|
|
Overall Study
COMPLETED
|
45
|
23
|
|
Overall Study
NOT COMPLETED
|
22
|
12
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Study of TG-C in Patients With Grade 3 Degenerative Joint Disease of the Knee
Baseline characteristics by cohort
| Measure |
TissueGene-C
n=67 Participants
TissueGene-C at 3 x 10e7 cells per injection (intraarticular)
TissueGene-C: Single intraarticular injection at 3 x 10e7 cells
|
Placebo Control
n=35 Participants
Normal Saline injection
Normal Saline: Single intraarticular injection of normal saline as a placebo control
|
Total
n=102 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
39 Participants
n=5 Participants
|
20 Participants
n=7 Participants
|
59 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
28 Participants
n=5 Participants
|
15 Participants
n=7 Participants
|
43 Participants
n=5 Participants
|
|
Age, Continuous
|
56.7 Years
STANDARD_DEVIATION 7.89 • n=5 Participants
|
56.5 Years
STANDARD_DEVIATION 8.62 • n=7 Participants
|
56.6 Years
STANDARD_DEVIATION 8.21 • n=5 Participants
|
|
Sex: Female, Male
Female
|
43 Participants
n=5 Participants
|
21 Participants
n=7 Participants
|
64 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
24 Participants
n=5 Participants
|
14 Participants
n=7 Participants
|
38 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Caucasian/White
|
53 participants
n=5 Participants
|
29 participants
n=7 Participants
|
82 participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Black
|
12 participants
n=5 Participants
|
4 participants
n=7 Participants
|
16 participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Hispanic
|
2 participants
n=5 Participants
|
2 participants
n=7 Participants
|
4 participants
n=5 Participants
|
|
Region of Enrollment
United States
|
67 participants
n=5 Participants
|
35 participants
n=7 Participants
|
102 participants
n=5 Participants
|
|
Body Mass Index (BMI)
|
29.6 kg/m^2
STANDARD_DEVIATION 5.87 • n=5 Participants
|
29.6 kg/m^2
STANDARD_DEVIATION 5.62 • n=7 Participants
|
29.6 kg/m^2
STANDARD_DEVIATION 5.70 • n=5 Participants
|
PRIMARY outcome
Timeframe: 1 YearPopulation: Patients with available baseline IKDC scores
Symptoms, pain and function of the knee joint determined and scored using the International Knee Documentation Committee (IKDC) Subjective Knee Evaluation (Total Score, range 0-100 with higher scores better). Linear mixed model used for analysis.
Outcome measures
| Measure |
TissueGene-C
n=38 Participants
TissueGene-C at 3 x 10e7 cells per injection (intraarticular)
TissueGene-C: Single intraarticular injection at 3 x 10e7 cells
|
Placebo Control
n=20 Participants
Normal Saline injection
Normal Saline: Single intraarticular injection of normal saline as a placebo control
|
|---|---|---|
|
Change From Baseline in the International Knee Documentation Committee (IKDC) Subjective Knee Evaluation Score at 1 Year
|
23.3 scores on a scale
Interval 17.3 to 29.3
|
9.9 scores on a scale
Interval 1.7 to 18.1
|
PRIMARY outcome
Timeframe: 1 YearPopulation: Patients with available baseline and 1 year VAS score
Reduction in pain as measured by a 100 mm visual analog scale (0= no pain; 100 = extreme pain) from Baseline to 1 Year. Linear mixed model used for analysis.
Outcome measures
| Measure |
TissueGene-C
n=49 Participants
TissueGene-C at 3 x 10e7 cells per injection (intraarticular)
TissueGene-C: Single intraarticular injection at 3 x 10e7 cells
|
Placebo Control
n=24 Participants
Normal Saline injection
Normal Saline: Single intraarticular injection of normal saline as a placebo control
|
|---|---|---|
|
Change From Baseline in Visual Analog Scale (VAS) Score at 1 Year
|
-39.7 units on a scale
Interval -46.7 to -32.7
|
-24.3 units on a scale
Interval -34.1 to -14.5
|
SECONDARY outcome
Timeframe: 2 YearsSymptoms, pain and functionality of the knee joint as determined by Total Score of the Knee Injury and Osteoarthritis Outcome Score (KOOS) (Range 0-100 with higher scores indicating healthier outcomes). Linear mixed model used for analysis.
Outcome measures
| Measure |
TissueGene-C
n=45 Participants
TissueGene-C at 3 x 10e7 cells per injection (intraarticular)
TissueGene-C: Single intraarticular injection at 3 x 10e7 cells
|
Placebo Control
n=23 Participants
Normal Saline injection
Normal Saline: Single intraarticular injection of normal saline as a placebo control
|
|---|---|---|
|
Change From Baseline in Knee Injury and Osteoarthritis Outcome Score (KOOS) at 2 Years
|
10.0 scores on a scale
Interval 6.0 to 13.9
|
10.5 scores on a scale
Interval 5.0 to 16.0
|
SECONDARY outcome
Timeframe: 2 YearsMeasurement to assess outcomes of various chondral disorders of the knee determined by the Lysholm Knee Scale (Range 0-100 with higher scores better). Linear mixed model used for analysis.
Outcome measures
| Measure |
TissueGene-C
n=45 Participants
TissueGene-C at 3 x 10e7 cells per injection (intraarticular)
TissueGene-C: Single intraarticular injection at 3 x 10e7 cells
|
Placebo Control
n=23 Participants
Normal Saline injection
Normal Saline: Single intraarticular injection of normal saline as a placebo control
|
|---|---|---|
|
Change From Baseline in Articular Cartilage Damage in the Knee as Determined by the Lysholm Knee Score at 2 Years
|
20.1 scores on a scale
Interval 14.8 to 25.5
|
11.4 scores on a scale
Interval 4.1 to 18.8
|
SECONDARY outcome
Timeframe: 1 YearPopulation: Patients with available baseline and 1-year MRIs
Comparison of pre-procedure 3T MRI scans to those obtained at months 12 following dose administration by an independent radiographic reviewer. Evaluations will be scored using Whole Organ Magnetic Resonance imaging Score (WORMS) Cartilage Morphology Subscore (Range 0-6, with higher scores being worse)
Outcome measures
| Measure |
TissueGene-C
n=32 Participants
TissueGene-C at 3 x 10e7 cells per injection (intraarticular)
TissueGene-C: Single intraarticular injection at 3 x 10e7 cells
|
Placebo Control
n=22 Participants
Normal Saline injection
Normal Saline: Single intraarticular injection of normal saline as a placebo control
|
|---|---|---|
|
Comparative Evaluation of Knee Magnetic Resonance Images (MRIs) From Baseline to 1 Year
|
0.03 scores on a scale
Interval -1.22 to 1.29
|
-0.49 scores on a scale
Interval -2.07 to 1.08
|
SECONDARY outcome
Timeframe: 2 YearsPopulation: Patients that completed 2 year follow-up
Change in pain severity (on a scale from 1 to 4) from baseline to 2 years as measured by a questionnaire (lower scores better)
Outcome measures
| Measure |
TissueGene-C
n=33 Participants
TissueGene-C at 3 x 10e7 cells per injection (intraarticular)
TissueGene-C: Single intraarticular injection at 3 x 10e7 cells
|
Placebo Control
n=18 Participants
Normal Saline injection
Normal Saline: Single intraarticular injection of normal saline as a placebo control
|
|---|---|---|
|
Change in Pain Severity From Baseline to 2 Years as Assessed by Questionnaire
|
-0.87 scores on a scale
Standard Deviation 0.844
|
-0.30 scores on a scale
Standard Deviation 1.031
|
SECONDARY outcome
Timeframe: 2 YearsPopulation: Patients taking at least one analgesia medication
The number of participants that had a change in pain severity as measured by the incidence and dose of analgesic medications
Outcome measures
| Measure |
TissueGene-C
n=67 Participants
TissueGene-C at 3 x 10e7 cells per injection (intraarticular)
TissueGene-C: Single intraarticular injection at 3 x 10e7 cells
|
Placebo Control
n=35 Participants
Normal Saline injection
Normal Saline: Single intraarticular injection of normal saline as a placebo control
|
|---|---|---|
|
Number of Participants With Change in Pain Severity Measured by Incidence and Dose of Analgesia
|
37 participants
|
20 participants
|
SECONDARY outcome
Timeframe: 2 YearsPopulation: Patients that completed 2-year follow-up
Assessment of knee function as determined by the Lower Extremity Functional Scale (LEFS); change from baseline to 2 years (Range 0-80 with higher scores signifying lower difficulty in performing knee functions)
Outcome measures
| Measure |
TissueGene-C
n=45 Participants
TissueGene-C at 3 x 10e7 cells per injection (intraarticular)
TissueGene-C: Single intraarticular injection at 3 x 10e7 cells
|
Placebo Control
n=23 Participants
Normal Saline injection
Normal Saline: Single intraarticular injection of normal saline as a placebo control
|
|---|---|---|
|
Change From Baseline in Knee Function as Determined by the Lower Extremity Functional Scale at 2 Years
|
16.7 scores on a scale
Interval 11.9 to 21.5
|
13.8 scores on a scale
Interval 7.1 to 20.4
|
SECONDARY outcome
Timeframe: 2 YearsPopulation: All patients that participated in the study
Quantification of the incidence of total knee arthroplasty of the treated knee subsequent to treatment with TissueGene-C
Outcome measures
| Measure |
TissueGene-C
n=67 Participants
TissueGene-C at 3 x 10e7 cells per injection (intraarticular)
TissueGene-C: Single intraarticular injection at 3 x 10e7 cells
|
Placebo Control
n=35 Participants
Normal Saline injection
Normal Saline: Single intraarticular injection of normal saline as a placebo control
|
|---|---|---|
|
The Incidence of Total Knee Arthroplasty
|
1 participants
|
1 participants
|
SECONDARY outcome
Timeframe: 2 YearsPopulation: All participants receiving placebo or active treatment
The number of patients with observations of the administration site deemed related to treatment with either active or placebo, including arthralgia, swelling, irritation, pain, stiffness or abnormalities
Outcome measures
| Measure |
TissueGene-C
n=67 Participants
TissueGene-C at 3 x 10e7 cells per injection (intraarticular)
TissueGene-C: Single intraarticular injection at 3 x 10e7 cells
|
Placebo Control
n=35 Participants
Normal Saline injection
Normal Saline: Single intraarticular injection of normal saline as a placebo control
|
|---|---|---|
|
The Number of Patients Experiencing Injection Site Reactions Related to Treatment
|
35 participants
|
5 participants
|
SECONDARY outcome
Timeframe: 2 YearsPopulation: All patients receiving treatment with either active or placebo
The incidence and severity of adverse events assessed through 104 weeks (2 years) after dose administration
Outcome measures
| Measure |
TissueGene-C
n=67 Participants
TissueGene-C at 3 x 10e7 cells per injection (intraarticular)
TissueGene-C: Single intraarticular injection at 3 x 10e7 cells
|
Placebo Control
n=35 Participants
Normal Saline injection
Normal Saline: Single intraarticular injection of normal saline as a placebo control
|
|---|---|---|
|
The Incidence and Severity of Adverse Events in Treated Patients
Mild
|
12 participants
|
7 participants
|
|
The Incidence and Severity of Adverse Events in Treated Patients
Moderate
|
41 participants
|
18 participants
|
|
The Incidence and Severity of Adverse Events in Treated Patients
Severe
|
8 participants
|
4 participants
|
SECONDARY outcome
Timeframe: 2 YearsPopulation: All patients
The number of participants with changes in clinical hematology, chemistry, and urinalysis test results through 2 years that were considered Adverse Events
Outcome measures
| Measure |
TissueGene-C
n=67 Participants
TissueGene-C at 3 x 10e7 cells per injection (intraarticular)
TissueGene-C: Single intraarticular injection at 3 x 10e7 cells
|
Placebo Control
n=35 Participants
Normal Saline injection
Normal Saline: Single intraarticular injection of normal saline as a placebo control
|
|---|---|---|
|
Number of Participants With Adverse Events Due to Clinically Significant Changes in Hematology and Urinalysis Tests
|
17 participants
|
5 participants
|
SECONDARY outcome
Timeframe: 2 YearsPopulation: Patients with completed SF-36 questionnaire at 104 weeks
Overall assessment of general health as determined by scoring use an SF-36 Questionnaire (Range 0-100 with higher scores better - indicating less disability)
Outcome measures
| Measure |
TissueGene-C
n=45 Participants
TissueGene-C at 3 x 10e7 cells per injection (intraarticular)
TissueGene-C: Single intraarticular injection at 3 x 10e7 cells
|
Placebo Control
n=22 Participants
Normal Saline injection
Normal Saline: Single intraarticular injection of normal saline as a placebo control
|
|---|---|---|
|
Change in SF-36 General Health Assessment Questionnaire (Overall Score) From Baseline to 2 Years
|
7.6 scores on a scale
Interval 4.4 to 10.8
|
6.7 scores on a scale
Interval 2.2 to 11.2
|
Adverse Events
TissueGene-C
Serious events: 7 serious events
Other events: 45 other events
Deaths: 0 deaths
Placebo Control
Serious events: 4 serious events
Other events: 8 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
TissueGene-C
n=67 participants at risk
TissueGene-C at 3 x 10e7 cells per injection (intraarticular)
TissueGene-C: Single intraarticular injection at 3 x 10e7 cells
|
Placebo Control
n=35 participants at risk
Normal Saline injection
Normal Saline: Single intraarticular injection of normal saline as a placebo control
|
|---|---|---|
|
Nervous system disorders
Sciatica
|
3.0%
2/67 • Number of events 2
|
0.00%
0/35
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
3.0%
2/67 • Number of events 2
|
0.00%
0/35
|
|
Musculoskeletal and connective tissue disorders
Radius Fracture
|
1.5%
1/67 • Number of events 1
|
0.00%
0/35
|
|
Surgical and medical procedures
Total Knee Arthroplasty
|
3.0%
2/67 • Number of events 2
|
2.9%
1/35 • Number of events 1
|
|
Musculoskeletal and connective tissue disorders
Hiatal hernia
|
1.5%
1/67 • Number of events 1
|
0.00%
0/35
|
|
Gastrointestinal disorders
Gastritis
|
1.5%
1/67 • Number of events 1
|
0.00%
0/35
|
|
Gastrointestinal disorders
Abdominal Pain
|
1.5%
1/67 • Number of events 1
|
0.00%
0/35
|
|
Musculoskeletal and connective tissue disorders
Invertebral Disc Protrusion
|
1.5%
1/67 • Number of events 1
|
0.00%
0/35
|
|
Musculoskeletal and connective tissue disorders
Microdiscectomy
|
1.5%
1/67 • Number of events 1
|
0.00%
0/35
|
|
Musculoskeletal and connective tissue disorders
Lumbar spinal stenosis
|
1.5%
1/67 • Number of events 1
|
0.00%
0/35
|
|
Surgical and medical procedures
Prostatectomy
|
0.00%
0/67
|
2.9%
1/35 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary embolism
|
0.00%
0/67
|
2.9%
1/35 • Number of events 1
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal Chest Pain
|
1.5%
1/67 • Number of events 1
|
0.00%
0/35
|
|
Musculoskeletal and connective tissue disorders
Spinal Column Stenosis
|
1.5%
1/67 • Number of events 1
|
0.00%
0/35
|
|
Surgical and medical procedures
Spinal Fusion Surgery
|
1.5%
1/67 • Number of events 1
|
2.9%
1/35 • Number of events 1
|
|
Surgical and medical procedures
Spinal Laminectomy
|
1.5%
1/67 • Number of events 1
|
2.9%
1/35 • Number of events 1
|
|
Surgical and medical procedures
Invertebral Disc Operation
|
1.5%
1/67 • Number of events 1
|
0.00%
0/35
|
|
Musculoskeletal and connective tissue disorders
Lumbar Degenerative Disc Disease
|
0.00%
0/67
|
2.9%
1/35 • Number of events 1
|
|
Nervous system disorders
Paraesthesia
|
1.5%
1/67 • Number of events 1
|
0.00%
0/35
|
Other adverse events
| Measure |
TissueGene-C
n=67 participants at risk
TissueGene-C at 3 x 10e7 cells per injection (intraarticular)
TissueGene-C: Single intraarticular injection at 3 x 10e7 cells
|
Placebo Control
n=35 participants at risk
Normal Saline injection
Normal Saline: Single intraarticular injection of normal saline as a placebo control
|
|---|---|---|
|
Gastrointestinal disorders
Nausea
|
1.5%
1/67 • Number of events 1
|
0.00%
0/35
|
|
General disorders
Chills
|
1.5%
1/67 • Number of events 1
|
0.00%
0/35
|
|
General disorders
Fatigue
|
1.5%
1/67 • Number of events 1
|
0.00%
0/35
|
|
General disorders
Feeling Cold
|
1.5%
1/67 • Number of events 1
|
0.00%
0/35
|
|
General disorders
Pain
|
1.5%
1/67 • Number of events 1
|
0.00%
0/35
|
|
Investigations
C-reactive Protein Increased
|
1.5%
1/67 • Number of events 1
|
0.00%
0/35
|
|
Investigations
Eosinophil Count Increased
|
1.5%
1/67 • Number of events 1
|
0.00%
0/35
|
|
Investigations
Interleukin Level Increased
|
7.5%
5/67 • Number of events 5
|
2.9%
1/35 • Number of events 1
|
|
Investigations
Red Blood Cell Sedimentation Increased
|
3.0%
2/67 • Number of events 2
|
2.9%
1/35 • Number of events 1
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
22.4%
15/67 • Number of events 15
|
5.7%
2/35 • Number of events 2
|
|
Musculoskeletal and connective tissue disorders
Arthritis
|
28.4%
19/67 • Number of events 19
|
2.9%
1/35 • Number of events 1
|
|
Musculoskeletal and connective tissue disorders
Arthropathy
|
1.5%
1/67 • Number of events 1
|
0.00%
0/35
|
|
Musculoskeletal and connective tissue disorders
Joint Effusions
|
19.4%
13/67 • Number of events 13
|
5.7%
2/35 • Number of events 2
|
|
Musculoskeletal and connective tissue disorders
Muscular Weakness
|
1.5%
1/67 • Number of events 1
|
0.00%
0/35
|
|
Musculoskeletal and connective tissue disorders
Pain in Extremity
|
1.5%
1/67 • Number of events 1
|
0.00%
0/35
|
|
Musculoskeletal and connective tissue disorders
Weight Bearing Difficulty
|
7.5%
5/67 • Number of events 5
|
0.00%
0/35
|
|
Nervous system disorders
Presyncope
|
1.5%
1/67 • Number of events 1
|
0.00%
0/35
|
|
Nervous system disorders
Syncope
|
1.5%
1/67 • Number of events 1
|
0.00%
0/35
|
|
Skin and subcutaneous tissue disorders
Eczema
|
1.5%
1/67 • Number of events 1
|
0.00%
0/35
|
|
Skin and subcutaneous tissue disorders
Hair Growth Abnormal
|
1.5%
1/67 • Number of events 1
|
0.00%
0/35
|
|
Skin and subcutaneous tissue disorders
Skin Warm
|
1.5%
1/67 • Number of events 1
|
0.00%
0/35
|
|
Investigations
Increased White Blood Cells/Neutrophils
|
1.5%
1/67 • Number of events 1
|
0.00%
0/35
|
|
Musculoskeletal and connective tissue disorders
Right Knee Swelling
|
1.5%
1/67 • Number of events 1
|
0.00%
0/35
|
|
General disorders
Asthenia
|
0.00%
0/67
|
2.9%
1/35 • Number of events 1
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place