Back to results

Safety Study of TissueGene-C in Degenerative Joint Disease of the Knee (TGC-03-01)

Primary Purpose

Osteoarthritis, Knee

Status

Completed

Phase

Phase 1

Locations

United States

Study Type

Interventional

Intervention

TissueGene-C

Placebo

About this trial

This is an interventional treatment trial for Osteoarthritis, Knee focused on measuring osteoarthritis, knee

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

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.
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.

Exclusion Criteria:

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.
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

Sites / Locations

Sinai Hospital of Baltimore
Commonwealth Orthopedics

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm Type

Experimental

Placebo Comparator

Arm Label

Arm Description

TissueGene-C single intraarticular injection of 3x10e6 cells/joint

TissueGene-C single intraarticular injection of 1x10e7 cells/joint

TissueGene-C single intraarticular injection of 3x10e7 cells/joint

Placebo control single intraarticular injection

Outcomes

Primary Outcome Measures

Summary of Adverse Events

The incidence of observations at the site of administration and the incidence adverse events assessed through 28 days after treatment.

Secondary Outcome Measures

Number of Patients Showing Engraftment at the Defect

Dose Response of the TG-C in Engrafting at the Defect as Compared to Placebo Control

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.

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.

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

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

Full Information

NCT ID

NCT00599248

First Posted

January 11, 2008

Last Updated

June 7, 2017

Sponsor

Kolon TissueGene, Inc.

1. Study Identification

Unique Protocol Identification Number

NCT00599248

Brief Title

Safety Study of TissueGene-C in Degenerative Joint Disease of the Knee

Acronym

TGC-03-01

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

Study Type

Interventional

2. Study Status

Record Verification Date

June 2017

Overall Recruitment Status

Completed

Study Start Date

January 2007 (undefined)

Primary Completion Date

May 2009 (Actual)

Study Completion Date

May 2010 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Kolon TissueGene, Inc.

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

Brief 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.

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Osteoarthritis, Knee

Keywords

osteoarthritis, knee

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1

Interventional Study Model

Parallel Assignment

Masking

Participant

Allocation

Randomized

Enrollment

12 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Arm Type

Experimental

Arm Description

TissueGene-C single intraarticular injection of 3x10e6 cells/joint

Arm Title

Arm Type

Experimental

Arm Description

TissueGene-C single intraarticular injection of 1x10e7 cells/joint

Arm Title

Arm Type

Experimental

Arm Description

TissueGene-C single intraarticular injection of 3x10e7 cells/joint

Arm Title

Arm Type

Placebo Comparator

Arm Description

Placebo control single intraarticular injection

Intervention Type

Biological

Intervention Name(s)

TissueGene-C

Intervention Description

TissueGene-C at 3x10e6, 1x10e7 or 3x10e7 cells/joint to be administered by intra-articular injection

Intervention Type

Biological

Intervention Name(s)

Placebo

Intervention Description

Placebo control (DMEM)

Primary Outcome Measure Information:

Title

Summary of Adverse Events

Description

The incidence of observations at the site of administration and the incidence adverse events assessed through 28 days after treatment.

Time Frame

Through 28 days post-dosing

Secondary Outcome Measure Information:

Title

Number of Patients Showing Engraftment at the Defect

Description

Dose Response of the TG-C in Engrafting at the Defect as Compared to Placebo Control

Time Frame

28 Days

Title

Number of Patients With Distribution of hChonJb#7 Cells Detected Outside of the Injection Site

Description

Number of Patients with Distribution of hChonJb#7 Cells Detected Outside of the Injection Site as determined by PCR analysis for vector DNA.

Time Frame

12 Months

Title

Number of Participants With Observable Evidence of Cartilage Regeneration

Description

The evaluation of regeneration of hyaline cartilage as determined by histological analysis of resected knee tissue and observation for engraftment and cartilage production.

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

Title

Number of Patients With Tissue Overgrowth or Transformation in the Knee Joint

Description

Visual and histological analysis of knee joint tissues to determine the number of patients with tissue overgrowth or transformation

Time Frame

Title

Number of Patients With Improvements in Pain and Function of the Knee Joint

Description

Assessment of the number of patients with improvement in pain and function of the knee joint

Time Frame

28 Days

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: 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. 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. Exclusion Criteria: 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. 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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Michael Mont, MD

Organizational Affiliation

Sinai Hospital of Baltimore

Official's Role

Principal Investigator

First Name & Middle Initial & Last Name & Degree

David Romness, MD

Organizational Affiliation

Commonwealth Orthopedics, Virginia Hospital Center

Official's Role

Principal Investigator

Facility Information:

Facility Name

Sinai Hospital of Baltimore

City

Baltimore

State/Province

Maryland

ZIP/Postal Code

21215

Country

United States

Facility Name

Commonwealth Orthopedics

City

Arlington

State/Province

Virginia

ZIP/Postal Code

22205

Country

United States

12. IPD Sharing Statement

Plan to Share IPD

Learn more about this trial

Safety Study of TissueGene-C in Degenerative Joint Disease of the Knee

We'll reach out to this number within 24 hrs