Cord Blood Product for Sacroiliac Pain

(SIJ Trial)

Overseen By Rebecca Wakeman

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: University of Florida

Must not be taking: Steroids, Regenerative medicines

Disqualifiers: Fibromyalgia, Inflammatory arthritis, Diabetes, others

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

Approved in 1 Jurisdiction This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 7

Duration: Approximately 4 weeks

9 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment made from umbilical cord blood, called PremierMaxCB®-Platinum (also known as CFL001), to determine its safety and potential effectiveness for sacroiliac joint pain, a condition causing discomfort in the lower back and pelvis. Participants will receive varying doses to identify the optimal one. Individuals who have not found relief from other treatments, such as medication and physical therapy, may be suitable candidates. As a Phase 1 trial, this research aims to understand how the treatment works in people, offering participants the chance to be among the first to receive this innovative therapy.

Will I have to stop taking my current medications?

The trial requires that any pain medication or therapy you are using must have a stable dosage for at least 3 months before starting the trial. You also need to avoid using pain medication for at least 24 hours before each follow-up evaluation.

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

Research has shown that cord blood products like PremierMaxCB®-Platinum (CFL001) have been tested in past studies for safety. This trial is in the early stages, focusing mainly on safety. Although specific data from earlier studies is not available, Phase 1 trials typically assess how well participants tolerate a new treatment and identify any side effects.

In real-world use, similar cord blood products are generally well-tolerated, with most people not experiencing serious side effects. This study tests different doses to determine the safest and most effective amount. While specific safety data for this exact product is not yet available, early trials include careful monitoring to ensure participant safety. Prospective participants should discuss potential risks and benefits with their doctor. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for sacroiliac pain?

Most treatments for sacroiliac pain, like physical therapy and anti-inflammatory medications, focus on managing symptoms rather than addressing the underlying issue. However, PremierMaxCB®-Platinum, a cord blood product, offers a unique approach by potentially regenerating damaged tissues and reducing inflammation at the source. This treatment uses stem cells from cord blood, which have the ability to transform into various cell types, potentially healing the joint itself. Researchers are excited about this approach because it targets the root cause of pain, promising longer-lasting relief compared to traditional methods.

What evidence suggests that PremierMaxCB®-Platinum (CFL001) might be an effective treatment for sacroiliac pain?

Research suggests that cord blood products like PremierMaxCB®-Platinum (CFL001), administered in varying doses during this trial, might alleviate sacroiliac pain. Cord blood contains special cells that can reduce swelling and aid in tissue repair. Similar products have shown promise in other contexts by reducing pain and improving movement. Although data specifically for sacroiliac pain is limited, early results are encouraging. The special cells from cord blood may target pain and support healing. ^{1 2 4 5 6}

Who Is on the Research Team?

Rene Przkora, MD, PHD

Principal Investigator

University of Florida

Are You a Good Fit for This Trial?

This trial is for individuals experiencing back pain, specifically sacroiliac joint syndrome. Details on who can join or reasons for exclusion are not provided.

Inclusion Criteria

I am using effective birth control during the study.

I've tried medications, daily exercises, and physical therapy for 3 months without relief from pain.

Body mass index < 40 kg/m2

Exclusion Criteria

Participated in another clinical trial within the last 6 months

An absolute value vital sign outside the following ranges: Systolic blood pressure >170 or <100, pulse rate of >100 or <50 bpm, and respiratory rate >22. Reasonable delay (i.e., one hour) may be provided at investigator's discretion to evaluate for return to acceptable parameters in the event that the subject had been subjected to a stressful circumstance prior to arrival in clinic

Active alcohol or substance abuse or any other reason that makes it unlikely that the subject will comply with study procedures

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive varying doses of CFL001 cord blood product to assess safety and tolerability

Approximately 4 weeks

3 visits (in-person) for dose administration

Follow-up

Participants are monitored for safety and effectiveness after treatment

26 weeks

4 visits (in-person) at 7, 30, 90, and 180 days post dose

What Are the Treatments Tested in This Trial?

Interventions

• PremierMaxCB®-Platinum (CFL001)

Trial Overview The study is testing the safety and potential effectiveness of a cord blood product called PremierMaxCB®-Platinum (CFL001) in treating sacroiliac joint syndrome. It's an open-label, early-phase trial without specific details on study design.

How Is the Trial Designed?

Group I: Arm 3: High Dose Experimental Treatment 1 Intervention

The next three subjects will receive the 90 x 106 TNC dose.

Group II: Arm 2: Medium Dose Experimental Treatment 1 Intervention

The next three subjects will receive the 60 x 106 TNC dose.

Group III: Arm 1: Low dose Experimental Treatment 1 Intervention

The first three subjects will receive the 30 x 106 TNC dose.

PremierMaxCB®-Platinum (CFL001) is already approved in United States for the following indications:

Approved in United States as HPC Cord Blood for:

• Blood cancers
• Genetic disorders
• Immunological disorders
• Anemia
• Leukemia

Who Is Running the Clinical Trial?

University of Florida

Lead Sponsor

Trials

1,428

Recruited

987,000+

Cord for Life, Inc.

Collaborator

Trials

1

Recruited

9+

Published Research Related to This Trial

The Milan Cord Blood Bank successfully banked 763 units of cord blood by 1995, with 8 units used for transplants in both children and adults, demonstrating the potential of cord blood in hematopoietic stem cell transplantation.

Early cord clamping after delivery was found to significantly increase the volume of cord blood collected without harming the newborn, and using 3% gelatin as a sedimenting agent yielded the best concentration of progenitor cells.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The Milan Cord Blood Bank and the Italian Cord Blood Network. Lazzari, L., Corsini, C., Curioni, C., et al.[2006]

Ex vivo expansion of cord blood hematopoietic stem cells (HSCs) can be significantly enhanced using a specific cytokine cocktail (FL + TPO + IL-6 + IL-11) in a serum-free medium, achieving a median expansion of 5205.6 fold after 10 weeks.

This study demonstrates that it is possible to maintain long-term expansion of CD34+ cells from cord blood in a simple, serum-free system, which is crucial for clinical applications and good medical practice.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Long-term expansion and maintenance of cord blood haematopoietic stem cells using thrombopoietin, Flt3-ligand, interleukin (IL)-6 and IL-11 in a serum-free and stroma-free culture system. Lazzari, L., Lucchi, S., Rebulla, P., et al.[2019]

A patient with severe mucositis after high-dose chemotherapy for non-Hodgkin lymphoma was successfully treated with daily applications of platelet gel from cord blood (CBPG), leading to significant improvement in oral mucosa after 8 days.

The treatment with CBPG showed no side effects, suggesting it is a safe option for managing severe oral mucositis in patients undergoing intensive chemotherapy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Impressive tissue regeneration of severe oral mucositis post stem cell transplantation using cord blood platelet gel. Piccin, A., Rebulla, P., Pupella, S., et al.[2018]

The study demonstrated that microencapsulated osteoblasts in a hypoxic environment significantly enhanced the expansion of hematopoietic stem/progenitor cells (HSPCs) from umbilical cord blood, achieving up to 23.4-fold increases in CD34(+) cells after 7 days of culture.

This method resulted in higher total yields of UCB mononuclear cells and improved repopulating potential, indicating a promising approach for increasing HSPC availability for therapeutic applications.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Effective expansion of umbilical cord blood hematopoietic stem/progenitor cells by regulation of microencapsulated osteoblasts under hypoxic condition. Song, K., Zhao, G., Liu, T., et al.[2009]

Cord blood (CB) plasma significantly enhances the expansion of hematopoietic progenitor cells, increasing colony-forming unit (CFU) yields by 2.23-fold, especially when the ABO blood types are unmatched.

In contrast, adult peripheral blood plasma does not promote the expansion of these progenitor cells, suggesting that specific factors in CB plasma could be beneficial for stem cell transplantation.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Cord blood plasma selectively stimulates the expansion of hematopoietic pregenitor cells in vitro]. Wu, G., Ying, D., Lin, Z., et al.[2004]

Cord blood (CB) red blood cells (RBCs) showed significant hemolysis over time, particularly in those stored with the SAG-M solution compared to the PAGGS-M solution, suggesting that PAGGS-M is a better preservation method.

The study found that leukoreduction of CB RBC units prevented significant changes in the production of TNF-alpha and TGF-beta1, indicating that white blood cells may influence cytokine release during storage.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Evaluation of nonleukoreduced red blood cell transfusion units collected at delivery from the placenta. Widing, L., Bechensteen, AG., Mirlashari, MR., et al.[2007]

Ex vivo co-culture of cord blood with bone-marrow-derived mesenchymal stem cells (MSCs) significantly enhances the output of hematopoietic progenitor cells (HPCs) compared to traditional liquid culture methods, leading to better potential for transplantation.

This co-culture technique requires less manipulation of the cells, resulting in lower initial loss of hematopoietic progenitor cells, which may improve the success rates of cord blood transplantation and reduce the risk of delayed or failed engraftment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Superior ex vivo cord blood expansion following co-culture with bone marrow-derived mesenchymal stem cells. Robinson, SN., Ng, J., Niu, T., et al.[2022]

Human umbilical cord blood (hUCB)-derived stromal cells can effectively enhance the expansion of CD34+ hematopoietic progenitor cells in vitro, significantly increasing colony-forming units compared to unexpanded CD34+ cells.

The study found that when CD34+ cells were cocultured with hUCB-derived stromal cells and cytokines, their growth was boosted by over 8-fold, indicating that these stromal cells could serve as a beneficial feeder layer for expanding hematopoietic cells for transplantation.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Human umbilical cord blood-derived stromal cell, a new resource of feeder layer to expand human umbilical cord blood CD34+ cells in vitro. Gao, L., Chen, X., Zhang, X., et al.[2006]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06415461

A Study Assessing the Safety of Cord Blood Product in ... This is a Phase 1 trial. The overall objective is to evaluate the safety and potential efficacy effect of specific type of umbilical cord blood product ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT06415461?cond=back%20pain&viewType=Table&rank=10

A Study Assessing the Safety of Cord Blood Product in ... This is a Phase 1 trial. The overall objective is to evaluate the safety and potential efficacy effect of specific type of umbilical cord blood product ...

3. withpower.com withpower.com/trial/phase-1-back-pain-4-2024-4246c

Cord Blood Product for Sacroiliac Pain (SIJ Trial) Trial Overview The study is testing the safety and potential effectiveness of a cord blood product called PremierMaxCB®-Platinum (CFL001) in treating sacroiliac ...

4. ctv.veeva.com ctv.veeva.com/study/open-label-dose-ranging-study-assessing-the-safety-of-cord-blood-product-in-sacroiliac-joint-syndrom

A Study Assessing the Safety of Cord Blood Product in ... This is a Phase 1 trial. The overall objective is to evaluate the safety and potential efficacy effect of specific type of umbilical cord blood product.

5. synapse.patsnap.com synapse.patsnap.com/drug/a869123ca7554d5a85a9441024470ea1

CFL-001 - Drug Targets, Indications, Patents ... CFL001 Cord Blood Product in Patients With Symptomatic Sacroiliac Joint Syndrome. 100 Clinical Results associated with CFL-001. Login to view more data. 100 ...

6. centerwatch.com centerwatch.com/clinical-trials/listings/NCT06415461/a-study-assessing-the-safety-of-cord-blood-product-in-sacroiliac-joint-syndrome-sij

A Study Assessing the Safety of Cord Blood Product in ... Severity of Sacroiliac Joint (SIJ) syndrome with a baseline Oswestry DisabilityIndex (ODI) score ≥ 30% and an SI joint pain score of ≥ 50 and ≤ ...