Study to Determine the Safety and Tolerability of TG-C in Subjects With Back Pain Due to Degenerative Disc Disease

A Phase I, Double-blind, Randomized, Sham-controlled, Dose-response, Study Evaluating the Safety and Tolerability of TG-C in Subjects With Chronic Discogenic Lumbar Back Pain Due to Degenerative Disc Disease at 6 and 12 Months

The goal of this study is to evaluate the safety and tolerability of TG-C in subjects with chronic discogenic lumbar back pain due to degenerative disc disease. Participants will be administered a single intradiscal injection or subcutaneous injection for sham and followed up with in-clinic visits and telephone calls for 24 months.

Study Overview

• Status: Not yet recruiting
• Conditions: Degenerative Disc Disease
• Intervention / Treatment: Biological: TG-C Low Dose; Biological: TG-C Mid Dose; Biological: TG-C High Dose; Biological: Sham Control

Detailed Description

A Phase I, Double-blind, Randomized, Sham-controlled, Dose-response, Study Evaluating the Safety and Tolerability of TG-C in Subjects with Chronic Discogenic Lumbar Back Pain due to Degenerative Disc Disease at 6 and 12 Months, male or female subjects with chronic discogenic lumbar back pain due to degenerative disc disease at one level. TG-C is to be administered by a single intradiscal injection to the damaged disc via fluoroscopic guidance. Sham will be a normal saline subcutaneous injection. Subjects will be followed for 24 months with in-clinic visits and telephone calls after study drug administration.

• Study Type: Interventional
• Enrollment (Estimated): 24
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: Diana M Halim, MS; Phone Number: 187 (301) 921-6000; Email: dhalim@tissuegene.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Between the ages of >= 22 and <= 70
• Provides written informed consent before undergoing any study specific procedures
• Chronic lower back pain for at least 6 months. Back pain twice as great as leg pain measured using NRS
• VAS between >= 40 and <= 90
• ODI Index >30 and <= 80
• Inadequate response to at least one medication (e.g., NSAID, acetaminophen, muscle relaxant) and some form of formal physical therapy over a period of at least 6 months, within 6-9 months of screening.
• Diagnosis of DDD from L1 to S1, confirmed by patient history and radiographic studies
• Modified Pfirrmann score of 3-7 on MRI
• With or without contained disc herniations of <3 mm protrusion
• If more than 1 degenerative disc is identified. PD must be performed 30 days prior and ensure only one level is the pain generator
• BMI >15 and < 30 kg/m2
• Use birth control

Exclusion Criteria:

• Co-morbid medical condition of the spine or upper extremities
• Grade 2 or higher spondylolisthesis and type III Modic changes around target disc
• Suspicion of full thickness annular tear at disc
• History of endocrine or metabolic disorder
• Rheumatoid or psoriatic arthritis
• Compressive pathology due to stenosis or herniated or sequestered discs
• Symptomatic involvement of more than one lumber disc
• Intact disc bulge/protrusion at >3 mm
• Lumbar intervertebral foraminal stenosis
• Previous surgery at the target disc level
• Epidural or facet joint steroid, platelet rich plasma (PRP) or bone marrow concentrate (BMC) injections, or radio frequency ablation (RFA), within 6 months prior to baseline.
• Pregnant
• Presence of ferromagnetic implants
• Involved in current or pending spinal litigations
• Care is provided under a Worker's Compensation claim
• Physical or mental conditions
• 3 or more of the 5 Waddell signs
• Positive screen for HIV
• Immediate family member of other participating patients
• Participated within 3 months or is concurrently enrolled in non-interventional research
• Transient or has a history of any substance use disorder
• Currently incarcerated
• Investigator site personnel or immediate family or sponsor employee
• On chronic anti-coagulation therapy or have confirmed coagulopathy
• Tested positive on RCR testing at screening

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

4

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Active Comparator: Active Treatment 1; Low dose TG-C 1.5 x 10e6 cells	Biological: TG-C Low Dose; Low Dose - A volume of 9 mL CS10 and 1 mL combined TG-C cells. 1.0 mL volume of the mixed TG-C cells is injected
Active Comparator: Active Treatment 2; Middle dose TG-C 5.0 x 10e6 cells	Biological: TG-C Mid Dose; Middle Dose - A volume of 2 mL CS10 and 1 mL combined TG-C cells. 1.0 mL volume of the mixed TG-C cells is injected
Active Comparator: Active Treatment 3; High dose TG-C 1.5 x 10e7 cells	Biological: TG-C High Dose; High Dose - 1 mL of the combined TG-C cells
Sham Comparator: Sham Control; single subcutaneous injection of normal saline	Biological: Sham Control; Single subcutaneous injection of normal saline

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Treatment-emergent adverse events	The safety and tolerability of the TG-C injection for the treatment of degenerative disc disease resulting in chronic discogenic lumbar back pain will be evaluated by summarizing adverse events by treatment	6 and 12 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Compare pain severity using a Visual Analogue Scale	Assessment of change from baseline in target back 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.	6 and 12 months
Oswestry Disability Index survey	Survey results compared to patient baseline	6 and 12 months

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Treatment-emergent adverse events	The safety and tolerability of the TG-C injection for the treatment of degenerative disc disease resulting in chronic discogenic lumbar back pain will be evaluated by summarizing adverse events by treatment	18 and 24 months
Compare pain severity using a Visual Analogue Scale	Assessment of change from baseline in target back 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.	18 and 24 months
Oswestry Disability Index survey	Survey results compared to patient baseline	18 and 24 months
Numerical Rating Scale	Compare severity of lower back pain to patient baseline using a Numerical Rating Scale (NRS). Patients rate their pain at baseline and 6 and 12 months after treatment. Patients will use an eleven point numerical scale to record their pain severity with 0 being no pain and 10 being worst possible pain.	6, 12, 18, and 24 months
Patient Global Impression of Changes	Patient Global Impression of Changes (PGI-C) is a 7 point scale depicting a patient's rating of overall improvement and their belief about the efficacy of treatment. The score ranges from 1 (very much worse) to 7 (very much improved)	6, 12, 18, and 24 months
Patient-Reported Outcomes Measurement Information System	Patient-Reported Outcomes Measurement Information System (PROMIS) is a way to measure patient-reported outcomes (PROs), such as pain, fatigue, physical functioning, emotional distress, and social role participation that have a major impact on quality-of-life. Here patients will answer a survey that consists of 16 questions that measure their physical functions after treatment. Patients will use a 5 point scale to answer the survey questions, with 1 being unable to do and 5 being without any difficulty.	6, 12, 18, and 24 months
MRI Assessment of Structural Changes of the Spine	Magnetic Resonance Imaging (MRI) of the lumbar spine will be performed using a 1.5 or 3T MRI machine. The modified Pfirrmann grading system will be used to evaluate MRI signals from the nucleus and inner annular fibers. The modified Pfirrmann grading scale will be used for MRI interpretation. The grading scale uses 8 grades with 1 being uniformly hyperintense and 8 being hyperintense. MRI results will be assessed at 6 and 12 months to observe structural changes to the spine.	1, 6, 12, and 24 months

Collaborators and Investigators

• Sponsor: Kolon TissueGene, Inc.
• Investigators: Study Chair: Moon Jong Noh, PhD, Kolon TissueGene

Study record dates

Study Major Dates

• Study Start (Estimated): November 1, 2025
• Primary Completion (Estimated): November 1, 2027
• Study Completion (Estimated): November 1, 2027

Study Registration Dates

• First Submitted: October 31, 2023
• First Submitted That Met QC Criteria: November 17, 2023
• First Posted (Actual): November 22, 2023

Study Record Updates

• Last Update Posted (Actual): January 29, 2024
• Last Update Submitted That Met QC Criteria: January 26, 2024
• Last Verified: January 1, 2024

More Information

Terms related to this study

• Keywords: Degenerative Disc Disease
• Additional Relevant MeSH Terms: Pain; Neurologic Manifestations; Musculoskeletal Diseases; Bone Diseases; Back Pain; Intervertebral Disc Degeneration; Spinal Diseases
• Other Study ID Numbers: TGC-DDD-101

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No