BRTX-100 for Chronic Lumbar Disc Disease

Recruiting at 22 trial locations

Overseen By Zongdong Li

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: BioRestorative Therapies

Must not be taking: Anticoagulants, NSAIDs, Opioids, others

Disqualifiers: Spinal deformity, Obesity, Autoimmune, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 7

Duration: 1 day

99 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

The trial aims to test a new treatment called BRTX-100 for individuals with chronic lumbar disc disease, a condition causing long-term lower back pain often triggered by sitting, bending, or lifting. BRTX-100 is derived from special cells taken from a person's own bone marrow, which might help reduce pain and improve function. Participants will receive either BRTX-100 or a placebo (a harmless saline solution) to compare results. The trial seeks individuals who have experienced lower back pain for at least six months and have not found relief from other treatments like physical therapy or medication. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group of people.

Will I have to stop taking my current medications?

The trial requires that you do not use systemic non-steroidal anti-inflammatory drugs (NSAIDs) within 72 hours before the study treatment. If you are on anticoagulation therapy, it must be interrupted for the procedures. Other medications are not specifically mentioned, so it's best to discuss with the trial team.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that BRTX-100 appears safe for humans. In earlier studies, no serious side effects were linked to its use. Among 10 patients in a safety trial, only 3 reported a total of 9 minor side effects, which are typical in early testing. BRTX-100 is a therapy made from the patient's own cells, potentially lowering the risk of rejection. While the treatment is still under investigation, current evidence suggests it is well-tolerated. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for chronic lumbar disc disease, which often include pain medications, physical therapy, or surgery, BRTX-100 is unique because it uses hypoxic-cultured bone marrow mononuclear cells enriched with mesenchymal stem cells from the patient's own bone marrow. This innovative approach aims to leverage the body's natural healing processes to regenerate damaged disc tissue. Researchers are excited about BRTX-100 because it could offer a less invasive and potentially more effective option for patients by directly targeting the underlying cause of disc degeneration rather than just managing symptoms.

What evidence suggests that BRTX-100 might be an effective treatment for chronic lumbar disc disease?

Research has shown that BRTX-100, which participants in this trial may receive, may help treat chronic lower back disc disease. In earlier studies, patients reported over a 30% reduction in their pain levels using a simple pain rating scale. BRTX-100 uses a person's own stem cells, which may help repair damaged discs. The FDA has fast-tracked this treatment due to its potential benefits. While more research is needed, these early results are promising for people with this condition. ^{1 2 3 4 5}

Who Is on the Research Team?

Jason Lipitz, MD

Principal Investigator

BioRestorative Therapies

Are You a Good Fit for This Trial?

This trial is for adults aged 18 to 60 with chronic lower back pain due to lumbar disc disease, who have not responded to at least six months of conservative treatments. Participants should have specific MRI findings and a history of back pain provoked by activities like sitting or bending. Pregnant women, those with severe spinal conditions, recent steroid injections, drug abuse history, or certain medical conditions are excluded.

Inclusion Criteria

My condition did not improve despite following medical advice and attending educational sessions.

I have been diagnosed with degenerative disc disease.

I suspect my back pain is due to a degenerating disc.

Exclusion Criteria

Women of childbearing potential not protected by a highly-effective method of birth control

Any other condition which in the judgment of the Investigator would preclude adequate evaluation of the safety and efficacy of the study drug

My discography test showed a Grade V annular fissure.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Safety Run-In

Initial subjects dosed with BRTX-100 at 40 × 10^6 cells with a 14-day safety follow-up period between dosing of each subject

14 days per subject

1 visit per subject (in-person)

Treatment

Subjects receive a single dose intradiscal injection of BRTX-100 or saline

1 day

1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment with visits at Week 2, Week 12, Week 26, Week 52, and Week 104/Early Termination

104 weeks

5 visits (in-person)

What Are the Treatments Tested in This Trial?

Interventions

• BRTX-100
• Saline

Trial Overview The study tests BRTX-100 against saline in a double-blind setup where neither the participants nor the researchers know who receives which treatment. It's randomized and involves one dose to see if BRTX-100 can help relieve chronic lumbar disc disease symptoms better than a saline placebo.

How Is the Trial Designed?

Group I: Active Treatment- BRTX-100 Experimental Treatment 1 Intervention

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.

Group II: Sham Placebo Group 1 Intervention

Isotonic saline will be used as a control (sham) in this study. Drug: saline (0.9% sodium chloride). Please note this will be a sham procedure. This isotonic saline will not be injected intradiscally.

Who Is Running the Clinical Trial?

BioRestorative Therapies

Lead Sponsor

Trials

1

Recruited

100+

Published Research Related to This Trial

A systematic review of randomized controlled trials found that lumbar total disk replacement (TDR) has a twofold lower risk of adverse events (AEs) compared to lumbar fusion, with this trend persisting over a 5-year follow-up period.

Despite these findings, the quality of AE reporting in the studies was generally poor, making it difficult to definitively recommend one treatment over the other; standardization in AE reporting is needed for better reliability in future research.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Adverse Event Recording and Reporting in Clinical Trials Comparing Lumbar Disk Replacement with Lumbar Fusion: A Systematic Review. Hiratzka, J., Rastegar, F., Contag, AG., et al.[2022]

In a study of 11 patients with contemporary metal-on-polyethylene lumbar total disc replacements (L-TDRs), wear debris was found in a significant number of fixed-bearing devices, indicating a biological response, but overall wear particle characteristics showed improvement compared to historical designs.

The analysis revealed that contemporary L-TDRs produced 99% fewer wear particles and had 50% rounder particles than older models, suggesting that the newer materials and designs have enhanced wear resistance and potentially better long-term outcomes.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

UHMWPE wear debris and tissue reactions are reduced for contemporary designs of lumbar total disc replacements. Veruva, SY., Lanman, TH., Isaza, JE., et al.[2022]

Biologic injections, such as mesenchymal signaling cells (MSCs) and platelet-rich plasma (PRP), show potential for treating intervertebral disc disease, but they carry risks, including serious adverse events like infections and disc herniation, as highlighted by a review of 330 patients across various studies.

To reduce the risk of complications from these injections, the authors recommend using leukocyte-rich PRP and a two-needle delivery technique along with intradiscal gentamicin, emphasizing the need for further research to monitor both clinical outcomes and complication rates.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Risks of Intradiscal Orthobiologic Injections: A Review of the Literature and Case Series Presentation. Jerome, MA., Lutz, C., Lutz, GE.[2021]

In a study of 62 patients who underwent total lumbar disc replacement (TDR) with ProDisc II, significant improvements in pain and disability scores were observed post-surgery, indicating TDR is an effective treatment for degenerative disc disease.

While TDR restored disc space height (DSH), it resulted in a significant decrease in range of motion (ROM), especially at the lumbosacral junction, suggesting that while pain relief is achieved, some mobility may be sacrificed.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Interdependence between disc space height, range of motion and clinical outcome in total lumbar disc replacement. Siepe, CJ., Hitzl, W., Meschede, P., et al.[2022]

Botulinum toxin (BTX) type A has shown effectiveness in treating various chronic pain conditions, including chronic low back pain, pelvic pain, and temporomandibular joint dysfunction, based on a review of existing evidence.

While BTX is beneficial for certain conditions, such as piriformis syndrome, there is conflicting evidence regarding its efficacy for whiplash and myofascial pain, indicating that further research is needed to fully understand its potential in chronic pain management.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Evidence for the use of botulinum toxin in the chronic pain setting--a review of the literature. Jeynes, LC., Gauci, CA.[2010]

Total lumbar disc replacement (TDR) is a motion-preserving alternative to traditional spinal fusion for treating lumbar degenerative conditions, aiming to reduce complications like adjacent level disc degeneration and the need for reoperations.

Despite advancements in TDR technology and its potential benefits, reimbursement issues in the U.S. due to concerns over complications and costs have led to a decline in TDR procedures, highlighting the need for clearer guidelines on its indications and outcomes.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Lumbar disc replacement: update. Heider, FC., Mayer, HM., Siepe, CJ.[2022]

The Physio-L Artificial Lumbar Disc demonstrated safety and efficacy over a 12-month follow-up in a study of 12 patients, with no implant failures or significant complications reported.

Patients experienced significant improvements in low-back pain and functional recovery, with average satisfaction scores of 83.5 mm, outperforming other artificial discs like Charité and ProDisc-L in clinical outcomes.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Clinical performance of an elastomeric lumbar disc replacement: Minimum 12 months follow-up. Pimenta, L., Springmuller, R., Lee, CK., et al.[2022]

The study evaluates the clinical outcomes of three cervical total disc replacement prostheses: the Bryan Cervical Disc, the Bristol Disc, and the ProDisc-C, providing insights into their design and biomechanical properties.

Clinical results indicate that these prostheses are effective for treating cervical disc issues, although specific efficacy and safety data for each device were not detailed in the abstract.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Cervical total disc replacement, part two: clinical results. Bertagnoli, R., Duggal, N., Pickett, GE., et al.[2016]

In a long-term study of 51 patients who underwent total lumbar disc replacement (TDR) with the ProDisc II implant, there was a significant improvement in clinical outcomes, such as reduced pain and disability, despite a gradual decline in range of motion (ROM) over time.

The study found that while the overall mobility of the implant decreased, this reduction did not negatively affect the patients' clinical symptoms, indicating that TDR can be effective in managing low back pain without compromising patient satisfaction.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Motion preservation following total lumbar disc replacement at the lumbosacral junction: a prospective long-term clinical and radiographic investigation. Wuertinger, C., Annes, RDÀ., Hitzl, W., et al.[2022]

Lumbar total disc replacement is a safe and effective treatment for degenerative lumbar disc disease, with a high patient satisfaction rate of 90.73% based on an analysis of 38 clinical trials involving 3,180 patients over follow-up periods ranging from 5.9 to 204 months.

The success of the procedure is significantly influenced by proper preoperative indications and the selection of appropriately sized implants, as incorrect indications are associated with higher rates of revision and explantation surgeries.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Current short- and long-term results of lumbar disc replacement : update 2008]. Wiedenhöfer, B., Ewerbeck, V., Suda, AJ., et al.[2022]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT04042844

A Single Dose of BRTX 100 for Patients With Chronic ... This is a double-blind, controlled, randomized study with blinded assessments using a single dose. Subjects that have a current diagnosis of chronic lumbar disc ...

2. ir.biorestorative.com ir.biorestorative.com/news-events/press-releases/detail/218/biorestorative-reports-compelling-preliminary-data-for-fda-fast-tracked-brtx-100-an-autologous-stem-cell-therapy-to-treat-chronic-lumbar-disc-disease

an Autologous Stem Cell Therapy to Treat Chronic Lumbar ... BRTX-100 is a novel, autologous cell-based therapy designed to treat patients suffering from painful lumbosacral disc degeneration.

3. pharmacytimes.com pharmacytimes.com/view/fda-grants-fast-track-designation-to-brtx-100-to-treat-chronic-lumbar-disc-disease

FDA Grants Fast Track Designation to BRTX-100 to Treat ... BRTX-100 is a novel cell-based therapy targeting chronic lumbar disc disease, currently in phase 2 trials for safety and efficacy evaluation.

4. cgtlive.com cgtlive.com/view/biorestorative-therapies-msc-therapy-brtx-100-fast-tracked-chronic-lumbar-disc-disease

BioRestorative Therapies' MSC Therapy BRTX-100 Fast ... With regard to efficacy, it was reported that at 26 weeks posttreatment, a greater than 30% improvement in visual analog scales (VAS) score was ...

5. mountsinai.org mountsinai.org/clinical-trials/single-dose-of-brtx-100-for-patients-with-chronic-lumbar-disc-disease-cldd

A Single Dose of BRTX 100 for Patients With Chronic ... This is a double-blind, saline-controlled, and randomized study with blinded assessments using a single dose.