Trial Outcomes & Findings for Safety Study of TissueGene-C in Degenerative Joint Disease of the Knee (NCT NCT00599248)
NCT ID: NCT00599248
Last Updated: 2017-07-07
Results Overview
The incidence of observations at the site of administration and the incidence adverse events assessed through 28 days after treatment.
Recruitment status
COMPLETED
Study phase
PHASE1
Target enrollment
12 participants
Primary outcome timeframe
Through 28 days post-dosing
Results posted on
2017-07-07
Participant Flow
Enrollment and assignment of patients in Arms 2 and 3 (dose escalation to sequentially higher doses) was only performed after completion of review of safety data in the previous Arm (lower dose) by the Independent Data Monitoring Committee
Participant milestones
| Measure |
Active Treatment (TG-C) 1
TissueGene-C at 3x10e6 cells/joint
TissueGene-C: TissueGene-C at 3x10e6 cells/joint administered by single intra-articular injection
|
Active Treatment (TG-C) 2
TissueGene-C at 1 x 10e7 cells/joint
TissueGene-C: TissueGene-C at 1x10e7 cells/joint administered by single intra-articular injection
|
Active Treatment (TG-C) 3
TissueGene-C at 3x10e7 cells/joint
TissueGene-C: TissueGene-C at 3x10e7 cells/joint administered by single intra-articular injection
|
Placebo Control (DMEM)
Placebo Control (DMEM)
Placebo: Placebo control (DMEM) administered by a single intraarticular injection
|
|---|---|---|---|---|
|
Overall Study
STARTED
|
3
|
3
|
3
|
3
|
|
Overall Study
COMPLETED
|
3
|
3
|
3
|
3
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Safety Study of TissueGene-C in Degenerative Joint Disease of the Knee
Baseline characteristics by cohort
| Measure |
Active Treatment (TG-C)
n=9 Participants
TissueGene-C at 3x10e6, 1x10e7 or, 3x10e7 cells/joint
TissueGene-C: TissueGene-C at 3x10e6, 1x10e7 or 3x10e7 cells/joint to be administered by intra-articular injection
|
Placebo Control (DMEM)
n=3 Participants
Placebo control
Placebo: Placebo control (DMEM)
|
Total
n=12 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
|
7 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
10 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
2 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Age, Continuous
|
61.0 Years
n=5 Participants
|
60.0 Years
n=7 Participants
|
60.5 Years
n=5 Participants
|
|
Sex: Female, Male
Female
|
8 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
10 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
9 participants
n=5 Participants
|
3 participants
n=7 Participants
|
12 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Through 28 days post-dosingPopulation: Intention-to-treat
The incidence of observations at the site of administration and the incidence adverse events assessed through 28 days after treatment.
Outcome measures
| Measure |
Active Treatment (TG-C) 1
n=3 Participants
TissueGene-C at 3x10e6 cells/joint
TissueGene-C: TissueGene-C at 3x10e6 cells/joint to be administered by a single intra-articular injection
|
Active Treatment (TG-C) 2
n=3 Participants
TissueGene-C at 1x10e7 cells/joint
TissueGene-C: TissueGene-C at 1x107 cells/joint to be administered by a single intra-articular injection
|
Active Treatment (TG-C) 3
n=3 Participants
TissueGene-C at 3x10e7 cells/joint
TissueGene-C: TissueGene-C at 3x10e7 cells/joint to be administered by a single intra-articular injection
|
Placebo Control (DMEM)
n=3 Participants
Placebo control
Placebo: Placebo control (DMEM)
|
|---|---|---|---|---|
|
Summary of Adverse Events
|
2 Adverse events
|
2 Adverse events
|
3 Adverse events
|
2 Adverse events
|
SECONDARY outcome
Timeframe: 28 DaysDose Response of the TG-C in Engrafting at the Defect as Compared to Placebo Control
Outcome measures
| Measure |
Active Treatment (TG-C) 1
n=3 Participants
TissueGene-C at 3x10e6 cells/joint
TissueGene-C: TissueGene-C at 3x10e6 cells/joint to be administered by a single intra-articular injection
|
Active Treatment (TG-C) 2
n=3 Participants
TissueGene-C at 1x10e7 cells/joint
TissueGene-C: TissueGene-C at 1x107 cells/joint to be administered by a single intra-articular injection
|
Active Treatment (TG-C) 3
n=3 Participants
TissueGene-C at 3x10e7 cells/joint
TissueGene-C: TissueGene-C at 3x10e7 cells/joint to be administered by a single intra-articular injection
|
Placebo Control (DMEM)
n=3 Participants
Placebo control
Placebo: Placebo control (DMEM)
|
|---|---|---|---|---|
|
Number of Patients Showing Engraftment at the Defect
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
SECONDARY outcome
Timeframe: 12 MonthsNumber of Patients with Distribution of hChonJb#7 Cells Detected Outside of the Injection Site as determined by PCR analysis for vector DNA.
Outcome measures
| Measure |
Active Treatment (TG-C) 1
n=3 Participants
TissueGene-C at 3x10e6 cells/joint
TissueGene-C: TissueGene-C at 3x10e6 cells/joint to be administered by a single intra-articular injection
|
Active Treatment (TG-C) 2
n=3 Participants
TissueGene-C at 1x10e7 cells/joint
TissueGene-C: TissueGene-C at 1x107 cells/joint to be administered by a single intra-articular injection
|
Active Treatment (TG-C) 3
n=3 Participants
TissueGene-C at 3x10e7 cells/joint
TissueGene-C: TissueGene-C at 3x10e7 cells/joint to be administered by a single intra-articular injection
|
Placebo Control (DMEM)
n=3 Participants
Placebo control
Placebo: Placebo control (DMEM)
|
|---|---|---|---|---|
|
Number of Patients With Distribution of hChonJb#7 Cells Detected Outside of the Injection Site
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
SECONDARY outcome
Timeframe: 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 dosingPopulation: Intention-to-treat
The evaluation of regeneration of hyaline cartilage as determined by histological analysis of resected knee tissue and observation for engraftment and cartilage production.
Outcome measures
| Measure |
Active Treatment (TG-C) 1
n=3 Participants
TissueGene-C at 3x10e6 cells/joint
TissueGene-C: TissueGene-C at 3x10e6 cells/joint to be administered by a single intra-articular injection
|
Active Treatment (TG-C) 2
n=3 Participants
TissueGene-C at 1x10e7 cells/joint
TissueGene-C: TissueGene-C at 1x107 cells/joint to be administered by a single intra-articular injection
|
Active Treatment (TG-C) 3
n=3 Participants
TissueGene-C at 3x10e7 cells/joint
TissueGene-C: TissueGene-C at 3x10e7 cells/joint to be administered by a single intra-articular injection
|
Placebo Control (DMEM)
n=3 Participants
Placebo control
Placebo: Placebo control (DMEM)
|
|---|---|---|---|---|
|
Number of Participants With Observable Evidence of Cartilage Regeneration
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
SECONDARY outcome
Timeframe: 29Visual and histological analysis of knee joint tissues to determine the number of patients with tissue overgrowth or transformation
Outcome measures
| Measure |
Active Treatment (TG-C) 1
n=3 Participants
TissueGene-C at 3x10e6 cells/joint
TissueGene-C: TissueGene-C at 3x10e6 cells/joint to be administered by a single intra-articular injection
|
Active Treatment (TG-C) 2
n=3 Participants
TissueGene-C at 1x10e7 cells/joint
TissueGene-C: TissueGene-C at 1x107 cells/joint to be administered by a single intra-articular injection
|
Active Treatment (TG-C) 3
n=3 Participants
TissueGene-C at 3x10e7 cells/joint
TissueGene-C: TissueGene-C at 3x10e7 cells/joint to be administered by a single intra-articular injection
|
Placebo Control (DMEM)
n=3 Participants
Placebo control
Placebo: Placebo control (DMEM)
|
|---|---|---|---|---|
|
Number of Patients With Tissue Overgrowth or Transformation in the Knee Joint
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
SECONDARY outcome
Timeframe: 28 DaysAssessment of the number of patients with improvement in pain and function of the knee joint
Outcome measures
| Measure |
Active Treatment (TG-C) 1
n=3 Participants
TissueGene-C at 3x10e6 cells/joint
TissueGene-C: TissueGene-C at 3x10e6 cells/joint to be administered by a single intra-articular injection
|
Active Treatment (TG-C) 2
n=3 Participants
TissueGene-C at 1x10e7 cells/joint
TissueGene-C: TissueGene-C at 1x107 cells/joint to be administered by a single intra-articular injection
|
Active Treatment (TG-C) 3
n=3 Participants
TissueGene-C at 3x10e7 cells/joint
TissueGene-C: TissueGene-C at 3x10e7 cells/joint to be administered by a single intra-articular injection
|
Placebo Control (DMEM)
n=3 Participants
Placebo control
Placebo: Placebo control (DMEM)
|
|---|---|---|---|---|
|
Number of Patients With Improvements in Pain and Function of the Knee Joint
|
3 participants
|
3 participants
|
3 participants
|
3 participants
|
Adverse Events
Active Treatment (TG-C)
Serious events: 0 serious events
Other events: 7 other events
Deaths: 0 deaths
Placebo Control (DMEM)
Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Active Treatment (TG-C)
n=9 participants at risk
TissueGene-C at 3x10e6, 1x10e7 or, 3x10e7 cells/joint
TissueGene-C: TissueGene-C at 3x10e6, 1x10e7 or 3x10e7 cells/joint to be administered by intra-articular injection
|
Placebo Control (DMEM)
n=3 participants at risk
Placebo control
Placebo: Placebo control (DMEM)
|
|---|---|---|
|
Gastrointestinal disorders
Diarrhea
|
22.2%
2/9 • Number of events 2 • 1 Year
Treatment emergent adverse events
|
33.3%
1/3 • Number of events 1 • 1 Year
Treatment emergent adverse events
|
|
Gastrointestinal disorders
Gingival bleeding
|
11.1%
1/9 • Number of events 1 • 1 Year
Treatment emergent adverse events
|
0.00%
0/3 • 1 Year
Treatment emergent adverse events
|
|
Gastrointestinal disorders
Abdominal pain
|
11.1%
1/9 • Number of events 1 • 1 Year
Treatment emergent adverse events
|
0.00%
0/3 • 1 Year
Treatment emergent adverse events
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
22.2%
2/9 • Number of events 2 • 1 Year
Treatment emergent adverse events
|
0.00%
0/3 • 1 Year
Treatment emergent adverse events
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
0.00%
0/9 • 1 Year
Treatment emergent adverse events
|
33.3%
1/3 • Number of events 1 • 1 Year
Treatment emergent adverse events
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
11.1%
1/9 • Number of events 1 • 1 Year
Treatment emergent adverse events
|
0.00%
0/3 • 1 Year
Treatment emergent adverse events
|
|
Musculoskeletal and connective tissue disorders
Joint range of motion decreased
|
11.1%
1/9 • Number of events 1 • 1 Year
Treatment emergent adverse events
|
0.00%
0/3 • 1 Year
Treatment emergent adverse events
|
|
Musculoskeletal and connective tissue disorders
Joint effusion
|
11.1%
1/9 • Number of events 1 • 1 Year
Treatment emergent adverse events
|
0.00%
0/3 • 1 Year
Treatment emergent adverse events
|
|
Musculoskeletal and connective tissue disorders
Lower extremity mass
|
11.1%
1/9 • Number of events 1 • 1 Year
Treatment emergent adverse events
|
0.00%
0/3 • 1 Year
Treatment emergent adverse events
|
|
Investigations
Activated PTT prolonged
|
22.2%
2/9 • Number of events 2 • 1 Year
Treatment emergent adverse events
|
33.3%
1/3 • Number of events 1 • 1 Year
Treatment emergent adverse events
|
|
Investigations
PT Prolonged
|
11.1%
1/9 • Number of events 1 • 1 Year
Treatment emergent adverse events
|
33.3%
1/3 • Number of events 1 • 1 Year
Treatment emergent adverse events
|
|
Investigations
Blood test abnormal
|
0.00%
0/9 • 1 Year
Treatment emergent adverse events
|
33.3%
1/3 • Number of events 1 • 1 Year
Treatment emergent adverse events
|
|
General disorders
Pyrexia
|
11.1%
1/9 • Number of events 1 • 1 Year
Treatment emergent adverse events
|
0.00%
0/3 • 1 Year
Treatment emergent adverse events
|
|
General disorders
Fatigue
|
11.1%
1/9 • Number of events 1 • 1 Year
Treatment emergent adverse events
|
0.00%
0/3 • 1 Year
Treatment emergent adverse events
|
|
Nervous system disorders
Headache
|
33.3%
3/9 • Number of events 3 • 1 Year
Treatment emergent adverse events
|
0.00%
0/3 • 1 Year
Treatment emergent adverse events
|
|
Nervous system disorders
Paresthesia
|
0.00%
0/9 • 1 Year
Treatment emergent adverse events
|
33.3%
1/3 • Number of events 1 • 1 Year
Treatment emergent adverse events
|
|
Nervous system disorders
Sinus headache
|
0.00%
0/9 • 1 Year
Treatment emergent adverse events
|
33.3%
1/3 • Number of events 1 • 1 Year
Treatment emergent adverse events
|
|
Skin and subcutaneous tissue disorders
Pruritis generalized
|
11.1%
1/9 • Number of events 1 • 1 Year
Treatment emergent adverse events
|
0.00%
0/3 • 1 Year
Treatment emergent adverse events
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
11.1%
1/9 • Number of events 1 • 1 Year
Treatment emergent adverse events
|
0.00%
0/3 • 1 Year
Treatment emergent adverse events
|
|
Infections and infestations
Gastroenteritis
|
22.2%
2/9 • Number of events 2 • 1 Year
Treatment emergent adverse events
|
0.00%
0/3 • 1 Year
Treatment emergent adverse events
|
|
Respiratory, thoracic and mediastinal disorders
Rhinitis allgergic
|
11.1%
1/9 • Number of events 1 • 1 Year
Treatment emergent adverse events
|
0.00%
0/3 • 1 Year
Treatment emergent adverse events
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
11.1%
1/9 • Number of events 1 • 1 Year
Treatment emergent adverse events
|
0.00%
0/3 • 1 Year
Treatment emergent adverse events
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
11.1%
1/9 • Number of events 1 • 1 Year
Treatment emergent adverse events
|
0.00%
0/3 • 1 Year
Treatment emergent adverse events
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: GT60