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A Single Dose of BRTX 100 for Patients With Chronic Lumbar Disc Disease (cLDD)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04042844
Recruitment Status : Recruiting
First Posted : August 2, 2019
Last Update Posted : December 2, 2022
Sponsor:
Information provided by (Responsible Party):
BioRestorative Therapies

Study Description
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Brief Summary:
This is a double-blind, saline-controlled, and randomized study with blinded assessments using a single dose. Subjects that have a current diagnosis of chronic lumbar disc disease and meet eligibility criteria will be enrolled. Chronic lumbar disc disease is defined as back and/or radicular pain with degeneration of the disc confirmed by patient history, physical examination, and radiographic measures such as computed tomography (CT), magnetic resonance imaging (MRI), plain film, myelography, discography, or other acceptable means.

Condition or disease Intervention/treatment Phase
Lumbar Disc Disease Biological: BRTX-100 Drug: Saline Phase 2

Detailed Description:

This is a double-blind, saline-controlled, and randomized study with blinded assessments using a single dose. Subjects that have a current diagnosis of chronic lumbar disc disease and meet eligibility criteria will be enrolled. Chronic lumbar disc disease is defined as back and/or radicular pain with degeneration of the disc confirmed by patient history, physical examination, and radiographic measures such as computed tomography (CT), magnetic resonance imaging (MRI), plain film, myelography, discography, or other acceptable means.

Subjects randomized to active treatment will undergo bone marrow harvest for processing into BRTX-100 for intradiscal injection. Subjects randomized to control will also undergo a bone marrow and blood harvest but only receive saline intradiscal injection procedures. Subjects will return to the study site for a visit at Week 2, Week 12, Week 26, Week 52 and Week 104/Early Termination.

The trial will have a Safety Run-In component that will insert a 3+3 design for the initial subjects dosed with BRTX-100 at 40 × 106 cells. Specifically, the randomization scheme will be briefly shifted from the overall trial 2:1 randomization to an initial 3:1 allotment of intradiscal BRTX-100 versus saline control. As such, four subjects will initially be randomized and administered their agents. There will be a 14 day safety follow-up period that must elapse between dosing of each of the first four (4) subjects. Dosing of each subsequent subject in the Safety Run-In component cannot occur until the independent Medical Monitor (MM) reviews the previously-dosed subject's blinded data, including but not limited to physical examination findings, laboratory values and reported adverse events (AEs) and serious adverse events (SAEs), at the completion of the 14-day visit and documents the findings. If no potential dose- limiting toxicity (DLT) is noted by the MM, the MM will approve the dosing of the next subject. If a potential DLT is noted by the MM, the MM will request that an ad hoc Data Safety Monitoring Board (DSMB) review of unblinded data occur per DSMB Charter before the next subject is dosed.

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 99 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: All subjects will be randomized (2:1) to receive either intradiscal BRTX-100 or saline.
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official Title: A Phase 2, Double-Blind, Saline-Controlled, Randomized Study to Evaluate the Safety and Preliminary Efficacy of a Single Dose Intradiscal Injection of BRTX 100 for Patients With Chronic Lumbar Disc Disease (cLDD)
Actual Study Start Date : June 30, 2022
Estimated Primary Completion Date : August 31, 2024
Estimated Study Completion Date : August 31, 2025

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Active Treatment- BRTX-100
BRTX-100 consists of a population of hypoxic-cultured bone marrow mononuclear cells highly enriched in mesenchymal stem cells from autologous bone marrow with autologous platelet lysate.
 Biological: BRTX-100
Hypoxic cultured mesenchymal stem cells (MSCs) from autologous bone marrow with autologous platelet lysate.
Placebo Comparator: Saline
Isotonic saline will be used as a control in this study. Drug: saline (0.9% sodium chloride).
 Drug: Saline
Sodium Chloride (0.9%) intravenous infusion preparation is a sterile and non-pyrogenic solution


Outcome Measures
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Primary Outcome Measures :
  1. Primary Safety Measures [ Time Frame: Baseline through Week 104 / Early Termination ]

    Report of adverse events (AEs), clinical review and questionnaires for pain, disability and quality of life at Baseline through Week 104/ Early Termination

    • Vital Signs
    • Physical Examination
    • Laboratory Evaluation (hematology and chemistry)
    • Clinical review of MRI changes from Baseline to Week 104 (MRI density measurements in T2 weighted images performed at Baseline, Week 52 and Week 104 with predetermined MRI rating scores)

  2. Primary Efficacy Measures [ Time Frame: Baseline through Week 52 ]
    • VAS for Pain Assessment
    • ODI for Functional Assessment

    Primary Efficacy Endpoint:

    Clinical response, defined as at least a 30% decrease in pain as measured on the VAS scale and at least a 30% increase in function based on the ODI at Week 52 as compared to baseline.



Eligibility Criteria
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Ages Eligible for Study:   18 Years to 60 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Subject Inclusion Criteria:

A subject is eligible for inclusion if all the following criteria are met:

  1. A high index of suspicion for discogenic pain, (i.e., painful degenerative disc(s) with or without protrusions < 5 mm)

    1. Chronic Lower Back Pain for at least 6 months
    2. Pain commonly provoked by prolonged sitting, forward bending, lifting, twisting, coughing, sneezing, or Valsalva maneuvers
    3. Failure of at least 6 months of conservative back pain care (can include any or all of the following: rest, anti-inflammatory medication, analgesics, narcotics, epidural injections or selective nerve root injections at the target level, facet joint injections, muscle relaxers, massage, acupuncture, chiropractic care)
    4. Failure of supervised therapy and education
    5. Screening of ≥ 40 mm and ≤ 80 mm on low back pain visual analog scales (VAS) (average pain in the last week)
    6. Screening Oswestry Disability Index (ODI) score ≥ 30 and < 90 on a 100-point scale
    7. No localized and significant pain below beltline (i.e., potential sacroiliac joint pain) without lumbar pain component
    8. Thigh or Leg pain, if present, is non prevailing and of nonradicular origin, i.e., not due to stimulation of nerve roots or dorsal root ganglion of a spinal nerve by compressive forces
    9. Diagnostic medial branch block or facet joint injection (bilateral unless the symptoms are purely unilateral in nature) in the last 12 months prior to the informed consent date indicates no prevailing facet joint involvement

  2. Has degenerative disc disease (DDD) as defined by the following:

    1. Changes from normal disc morphology of the affected disc as defined by radiographic evaluation
    2. Modified Pfirrmann score of 2 to 7 on MRI, may contain a contained protrusion and/or annular tear on MRI
    3. Modified Pfirrmann score of 1 must contain a contained protrusion and/or annular tear on MRI
    4. Modic Grade I or II changes or no change on MRI
    5. Maintained intervertebral disc heights of at least 50% on MRI.
    6. Discography, if not performed within the last 6 months prior to informed consent date, has to be performed if more than one degenerative disc is identified by MRI, and the symptomatic disc cannot be otherwise reasonably determined
    7. If more than one degenerative disc is identified by MRI, no disc shall demonstrate greater degenerative change than the symptomatic disc or contain a protrusion greater than 5mm
  3. Aged 18 to 60 years
  4. Willing and able to provide written informed consent
  5. No evidence of contraindications to the procedure such as pregnancy, active infection, bleeding disorder, or metastatic cancer.

Subject Exclusion Criteria

A subject is not eligible to participate if any of the following criteria are met:

  1. Spinal Deformity (Scoliosis >20 degrees, spondylolysis, clinically or radiographically significant retrolisthesis or spondylolisthesis) detected on MRI or plain film radiographic assessment
  2. Disc extrusions, sequestered fragments, facet cysts, or greater than moderate spinal stenosis on MRI.
  3. Presence of a Grade V annular fissure on discography in a subject for whom provocation discography has been performed
  4. Intervertebral disc with radiographic evidence of Modified Pfirrmann Grade 8 or greater
  5. Any bleeding disorder, intrinsic or extrinsic
  6. Required anticoagulation (with either antiplatelet agents or antithrombotics) that cannot be interrupted for harvest and injection procedures
  7. Platelet count < 100,000
  8. International Normalized Ratio (INR) > 1.5
  9. Extreme obesity, as defined by National Institute of Health (NIH) Clinical Guidelines Body Mass Index (BMI >40
  10. Clinically relevant instability on flexion-extension as determined by the investigator by overlaying films (flexion & extension films)
  11. Has undergone any previous lumbosacral spine surgery (e.g. discectomy, laminectomy, foraminotomy, fusion, intradiscal electrothermal therapy, intradiscal radiofrequency thermocoagulation.) or therapeutic percutaneous disc intervention
  12. Have any acute or chronic lumbosacral spine fracture
  13. Have a history of lumbosacral epidural steroid injections within 1 month prior to informed consent date.
  14. Planned/expected use of systemic non-steroidal anti-inflammatory drugs (NSAIDs) within 72 hours prior to study treatment.
  15. Have a known history of hypersensitivity or anaphylactic reaction to dimethyl sulfoxide (DMSO)
  16. Active significant non lumbosacral spinal orthopedic pain generators including, not limited to arthritic hip and/or knee, cervical disc disease
  17. More widespread and ill-defined myofascial pain
  18. Have had treatment with any cellular or biological investigational therapy or device within 6 months of informed consent date and/or plans to participate in any other autologous or allogeneic stem cell/progenitor cell therapy trial during the 2-year follow-up period.
  19. Have been a recipient of prior stem cell/progenitor cell therapy or other biological intervention to repair a lumbosacral intervertebral disc
  20. Are transient or has been treated in the last 6 months before enrollment for alcohol and/or drug abuse in an inpatient substance abuse program
  21. Apparent ongoing and poorly controlled psychological or somatic disease that may impact treatment outcomes
  22. Social, familial, or geographical hindrances to compliance with the study protocol or the informed consent process.
  23. Known autoimmune disease (e.g., systemic lupus erythematosus)
  24. Required chronic immunosuppression
  25. Positive hepatitis C virus (HCV) antibody test
  26. Positive human immunodeficiency virus (HIV) Ag/Ab Combo test
  27. Pregnant or lactating women
  28. Women of childbearing potential not protected by a highly-effective method of birth control
  29. Clinically significant hematology and chemistry including, but not limited to: a. Total bilirubin level ≥ 1.5 times institutional upper limit of normal (ULN) b. Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) ≥ 2 x ULN c. Absolute neutrophil count (ANC) < 1000/mm3 d. Hemoglobin ≤ 10 g/dL e. Creatinine clearance use calculated clearance (Cockcroft-Gault equation) of ≤ 50 mL/min
  30. Any other condition which in the judgment of the Investigator would preclude adequate evaluation of the safety and efficacy of the study drug
  31. Inability to comply with the requirements of the study protocol
  32. History of using nicotine delivery products (active within 3 months of study treatment prior to informed consent date)
  33. Actively on workers compensation or no-fault case for this complaint or any other active case or litigation pertaining to their lumbosacral pain
  34. History of drug abuse or documented history of noncompliance with controlled substances
  35. History of regular, long term, daily opioid drug use (>30 MME)
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04042844


Contacts
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Contact: Francisco Silva 1(949)394-0132 FSilva@biorestorative.com

Locations
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United States, Alabama
Alabama Clinical Therapeutics, LLC Not yet recruiting
Birmingham, Alabama, United States, 35235
Contact: Brad Goodman    205-833-2228    brad.goodman@actstudy.net   
United States, Colorado
Denver Back Pain Specialists, LLC Recruiting
Greenwood Village, Colorado, United States, 80111
Contact: Scott Bainbridge    303-327-5511    jscottbainbridge@gmail.com   
United States, District of Columbia
Minimally Invasive and Endovascular Neurosurgery, George Washington University Not yet recruiting
Washington, District of Columbia, United States, 20037
Contact: Shawn Sarin         
United States, Florida
Cantor Spine Institute Recruiting
Fort Lauderdale, Florida, United States, 33306
Contact: Anthony Giuffrida       Anthony.giuffrida16@gmail.com   
Mayo Clinic Not yet recruiting
Jacksonville, Florida, United States, 32224
Contact: Wenchun Qu       Qu.Wenchun@mayo.edu   
Pain Relief Centers Recruiting
Saint Petersburg, Florida, United States, 33709
Contact: Robert Guirguis    727-510-9773    drguirguis@vantagetrials.com   
Tampa Pain Relief Center Recruiting
Tampa, Florida, United States, 33603
Contact: Jose Rivera    727-510-9773    drrivera@vantagetrials.com   
Florida Pain Relief Center Recruiting
Tampa, Florida, United States, 33614
Contact: Jorge Leal    727-510-9773    drleal@vantagetrials.com   
United States, New York
Long Island Spine Rehabilitation Medicine Not yet recruiting
East Meadow, New York, United States, 11554
Contact: Jeffry Beer    516-595-0096    jbeer@lispinemed.com   
Mount Sinai Not yet recruiting
New York, New York, United States, 10029
Contact: Alexander Lee    212-241-1076    alexander.lee@mountsinai.org   
United States, North Carolina
The Center of Clinical Research, LLC Recruiting
Winston-Salem, North Carolina, United States, 27103
Contact: Richard Rauck    336-765-6181 ext 152    rrauck@ccrpain.com   
United States, Ohio
Cleveland Clinic Not yet recruiting
Cleveland, Ohio, United States, 44195
Contact: Shrif Costandi    440-695-4000    Costans2@ccf.org   
United States, Oklahoma
Clinical Investigations LLC Not yet recruiting
Edmond, Oklahoma, United States, 73013
Contact: Douglas Beall    405-601-2325    db@clinrad.org   
United States, South Carolina
Coastal Carolina Research Center Not yet recruiting
North Charleston, South Carolina, United States, 29406,
Contact: Shailesh Patel, MD    843-856-3784    spatel@coastalcarolinaresearch.com   
United States, Texas
Precision Spine Care Recruiting
Tyler, Texas, United States, 75701
Contact: Aaron Calodney    903-952-8286    aaroncalodney@me.com   
United States, Virginia
Virginia iSpine Physicians Recruiting
Richmond, Virginia, United States, 23235
Contact: Michael DePalma    840-330-3030 ext 4    michaeldepalma8@gmail.com   
Sponsors and Collaborators
BioRestorative Therapies
Investigators
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Study Chair: Jason Lipitz, MD BioRestorative Therapies
More Information
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Responsible Party: BioRestorative Therapies
ClinicalTrials.gov Identifier: NCT04042844    
Other Study ID Numbers: CLDD-001
First Posted: August 2, 2019    Key Record Dates
Last Update Posted: December 2, 2022
Last Verified: November 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Spinal Diseases
Intervertebral Disc Displacement
Intervertebral Disc Degeneration
Bone Diseases
 Musculoskeletal Diseases
Hernia
Pathological Conditions, Anatomical