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Study to Determine the Efficacy and Safety of TG-C in Subjects with Kellgren/Lawrence Grade 2 or 3 OA of the Knee

Phase 3
Active, not recruiting
Conditions
Degenerative Osteoarthritis
Interventions
Biological: Placebo Control
Registration Number
NCT03291470
Lead Sponsor
Kolon TissueGene, Inc.
Brief Summary

A randomized, double-blind, placebo-controlled, multi-center, phase 3 study to determine the efficacy and safety of TG-C in subjects with Kellgren and Lawrence Grade 2 or 3 Osteoarthritis of the Knee, Osteoarthritis Research Society International (OARSI) medial joint space narrowing (JSN) Grade 1 or 2 of the knee joint. TG-C is to be administered by a single intra-articular injection to the damaged joint area via ultrasound guidance. Patients will be followed for 24 months for safety and efficacy.

Detailed Description

This protocol is designed to assess the safety and efficacy of TG-C in treating symptoms of osteoarthritis of the knee and determining disease modifying effects of TG-C. This study is intended for the treatment of patients with K\&L Grade 2 and 3 osteoarthritis and OARSI JSN Grade 1 and 2. Assessments will be made on both the symptomatic effects of pain (VAS), function (WOMAC), and Quality of Life (SF-12) as well as the physical effects (JSW, MRI evaluations of whole knee joint organ tissues and structure) and biochemical biomarkers. This is a placebo-controlled study, TG-C will be compared to normal saline as a control. Additionally, this protocol is designed to evaluate the safety of TG-C. Safety will be evaluated by observation of the injection site for irritation or other effects, the incidence and severity of adverse events, and changes in physical examination findings, radiographic criteria, and laboratory tests. Patients will be followed for 24 months for both safety and efficacy with annual cancer surveillance questionnaires through 15 years post dose for subjects who do not enroll in the Long Term Safety study.

Recruitment & Eligibility

Status
ACTIVE_NOT_RECRUITING
Sex
All
Target Recruitment
531
Inclusion Criteria
  • Aged 40 or older
  • BMI between 18.5 and 40
  • KL Grade 2 or 3 knee OA
  • OARSI Grade 1 or 2 medial JSN
  • Pain >= 40 on VAS scale
  • Written informed consent
  • Using birth control
Exclusion Criteria
  • Knee symptoms that result in difficulty or inability to walk
  • Knee effusion >2+
  • Has Grade 3 OARSI JSN
  • MRI exam indicates fracture or tumor
  • Has a positive result on RCR testing at screening
  • Has taken NSAIDS with 14 days of baseline
  • Has taken steroidal anti-inflammatory or biologic therapy medication within 2 months of baseline
  • Chronic (>21 days) narcotic use
  • Recent history (within 1 year) of drug or alcohol abuse
  • Pregnant or lactating
  • Has received injection to target knee within 2 months prior to study entry
  • History of various disorders of the knee including but not limited to rheumatoid arthritis, psoriatic arthritis, autoimmune disorder, chondrocalcinosis, gout, hemochromatosis, villonodular synovitis, or synovial chondromatosis
  • Severe hip osteoarthritis ipsilateral to the target knee
  • Ongoing infectious disease including but not limited to HIV, HTLV-1, VSV-G, HBV, or HCV.
  • Clinically significant congestive heart failure, hepatic disease, kidney disease, adrenal insufficiency, acromegaly, uncontrolled hyper- or hypothyroidism, or non-specified uncontrolled endocrine disorder.
  • Uncontrolled diabetes based on a HbA1c > 8% at screening.
  • Documented active malignancy within the last 5 years except for basal cell carcinoma, squamous cell carcinoma, and benign pigmented nevi.

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Active Treatment (TG-C)TG-CTG-C at 3 x 10e7 cells per single 2 mL intraarticular injection
Placebo Control (Normal Saline)Placebo ControlNormal saline, single 2 mL intraarticular injection
Primary Outcome Measures
NameTimeMethod
Change in Knee Pain as Assessed by VAS12 months

Assessment of change from baseline in target knee pain using the Visual Analog Scale (VAS). The VAS is a method for the assessment of the intensity of pain. The VAS is a horizontal line, 100 mm in length and anchored by word descriptors at each end (no pain, very severe pain). The patient marks on the line at the point that represents their current state. The VAS score is determined by measuring in millimeters from the left hand end to the point of the mark. The score ranges from "0" or no pain to "100" very severe pain.

Change in Knee Function as Assessed by the Western Ontario and McMaster Universities Index (WOMAC)12 months

Assessment of change from baseline in target knee function using the WOMAC Osteoarthritis Index. The WOMAC is a proprietary set of standardized questionnaires used by health professionals to evaluate the condition of patients with osteoarthritis of the knee including pain, stiffness, and physical functioning of the joints. The response to the questions is in the form of a checked box associated with the response; None, Mild, Moderate, Severe and Extreme.

Secondary Outcome Measures
NameTimeMethod
MRI Assessment of Target Knee12 months

Assessment of knee organ tissues and structure by comparing change in MRI scoring using WORMS and partial MRI Osteoarthritis Knee Score (MOAKS)

PCS of the SF-12 Questionnaire12 months

Evaluation of the change from baseline in physical component score (PCS) of the SF-12v2 questionnaire.

WOMAC Total Score24 Months

Evaluation of the efficacy of TG-C with regard to knee function via WOMAC total score

Health Assessment Questionnaire Disability Index12 months

Evaluation of the change from baseline in the disability index of the Health Assessment Questionnaire (HAQ-DI)

Trial Locations

Locations (27)

Haresh Boghara

🇺🇸

Red Oak, Texas, United States

Igor Grosman

🇺🇸

Brooklyn, New York, United States

David Scott

🇺🇸

Spokane, Washington, United States

Priyesh Mehta

🇺🇸

Dayton, Ohio, United States

Samy Metyas

🇺🇸

Covina, California, United States

Jeremy Hoff

🇺🇸

Wilmington, North Carolina, United States

Jonathan Samuels

🇺🇸

New York, New York, United States

Bassil Aish

🇺🇸

Huntington Beach, California, United States

Peter Hanson

🇺🇸

La Mesa, California, United States

Larkin Wadsworth

🇺🇸

Saint Louis, Missouri, United States

Steve Sitar

🇺🇸

Anaheim, California, United States

John Beckes

🇺🇸

San Diego, California, United States

Mira Baron

🇺🇸

West Palm Beach, Florida, United States

Thomas Schnitzer

🇺🇸

Chicago, Illinois, United States

Duane C Anderson

🇺🇸

Las Vegas, Nevada, United States

James Lilly

🇺🇸

Dallas, Texas, United States

Nebojsa Skrepnik

🇺🇸

Tucson, Arizona, United States

Edward Tavel

🇺🇸

Charleston, South Carolina, United States

Richard Radnovich

🇺🇸

Boise, Idaho, United States

Nathan Rimmke

🇺🇸

Rochester Hills, Michigan, United States

John Solic

🇺🇸

Durham, North Carolina, United States

Charles Andrews

🇺🇸

San Antonio, Texas, United States

Leonel Reyes

🇺🇸

San Antonio, Texas, United States

Miguel Trevino

🇺🇸

Clearwater, Florida, United States

Jan Hommen

🇺🇸

Doral, Florida, United States

Stuart Silverman

🇺🇸

Beverly Hills, California, United States

Manjoo Sharma

🇺🇸

Lewisville, Texas, United States

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