Cord Blood Product for Sacroiliac Pain

(SIJ Trial)

Overseen By Eileen M Handberg, PhD

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

Trial Summary

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.

What data supports the effectiveness of the treatment PremierMaxCB®-Platinum (CFL001) for sacroiliac pain?

Research shows that expanding cord blood cells can improve their effectiveness in treatments, as seen in studies where cord blood cells were expanded using various techniques, leading to increased cell numbers and improved outcomes in other conditions. This suggests potential benefits for treatments like PremierMaxCB®-Platinum. ^{1 2 3 4 5}

How is the treatment PremierMaxCB®-Platinum (CFL001) unique for sacroiliac pain?

PremierMaxCB®-Platinum (CFL001) is unique because it uses cord blood, which contains hematopoietic stem cells, potentially offering regenerative properties that are not typical in standard treatments for sacroiliac pain. This approach may provide a novel mechanism of action by promoting tissue repair and regeneration. ^{2 3 6 7 8}

What is the purpose of this trial?

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 (CFL001), which, other than specific modifications in manufacturing to render it compatible with current Good Manufacuring Practice (cGMP), is essentially similar to that reported in real-world experience.

Research Team

Rene Przkora, MD

Principal Investigator

University of Florida

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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+

Findings from Research

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]

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]

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]

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]

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]

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]

References

1. England pubmed.ncbi.nlm.nih.gov

Superior ex vivo cord blood expansion following co-culture with bone marrow-derived mesenchymal stem cells. [2022]

2. United States pubmed.ncbi.nlm.nih.gov

The Milan Cord Blood Bank and the Italian Cord Blood Network. [2006]

3. England 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. [2019]

4. United States 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. [2006]

5. Netherlands pubmed.ncbi.nlm.nih.gov

Effective expansion of umbilical cord blood hematopoietic stem/progenitor cells by regulation of microencapsulated osteoblasts under hypoxic condition. [2009]

6. United States pubmed.ncbi.nlm.nih.gov

Impressive tissue regeneration of severe oral mucositis post stem cell transplantation using cord blood platelet gel. [2018]

7. China pubmed.ncbi.nlm.nih.gov

[Cord blood plasma selectively stimulates the expansion of hematopoietic pregenitor cells in vitro]. [2004]

8. United States pubmed.ncbi.nlm.nih.gov

Evaluation of nonleukoreduced red blood cell transfusion units collected at delivery from the placenta. [2007]