Open Label Dose Ranging Study Assessing the Safety of Cord Blood Product in Sacroiliac Joint Syndrome (SIJ) (SIJ)

May 13, 2024 updated by: University of Florida

A Phase 1, Open Label Dose-Ranging Study to Assess the Safety, Tolerability, Preliminary Efficacy, and Dose Effect of CFL001 Cord Blood Product in Patients With Symptomatic Sacroiliac Joint Syndrome

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 cGMP, is essentially similar to that reported in real-world experience.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

The Phase 1 trial will enroll three subjects into an initial group receiving a low dose of CFL001. Provided that these subjects tolerate this dose well, will proceed to enroll three subjects into a group receiving a middle dose of CFL001. Provided that these subjects tolerate this dose well, will proceed to enroll three subjects into a group receiving the highest dose of CFL001.

All subjects will have Symptomatic Sacroiliac Joint (SIJ) syndrome, with clinical average pain score in the month prior to enrollment ≥50 and ≤90 on a 100-point scale.

Study Type

Interventional

Enrollment (Estimated)

9

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

  • Name: Eileen M Handberg, PhD
  • Phone Number: 352-273-8944
  • Email: handbem@ufl.edu

Study Contact Backup

  • Name: Dana D Leach, DNP
  • Phone Number: 352-273-8933
  • Email: leach@ufl.edu

Study Locations

  • United States
    • Florida
      • Gainesville, Florida, United States, 32610
        • Recruiting
        • University of Florida Pain Clinic
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. Age ≥ 18 years and ≤ 90 years.
  2. Diagnosis of SIJ syndrome based on clinical findings, including the Fortin, FABER and compression sign.
  3. Severity of SIJ syndrome with a baseline ODI score ≥ 30% and an SI joint pain score of ≥ 50 and ≤ 90 on the VAS 100 mm scale.
  4. Individuals with either unilateral or bilateral SIJ arthritis can be candidates for enrollment; if both joints are deemed appropriate for administration of the test agent by all other inclusion criteria, then the SIJ which the participants reports as more painful will be treated, or if both are equally painful then we will use a random generator approach ("flip a coin") to determine which joint will be treated.

  5. ≥75% decrease in pain within 2 days after image-guided injection of only local anesthetic (with no steroid) into the SIJ within 3 months prior to screening.

    OR Established SIJ condition based on decrease in pain after image-guided injection of local anesthetic and steroid into the SIJ 3 months prior to screening.

  6. Body mass index < 40 kg/m2.
  7. Ability to comply with the requirements of the study.
  8. Ability to understand and provide written informed consent.
  9. All participants of reproductive age/capacity to confirm use of adequate contraception during the study period.
  10. All participants should have tried and failed conservative therapies such as medications (acetaminophen and/or NSAIDs or Tramadol); daily home exercise or home stretching, including hip-girdle and core exercise, with the target of 20 minutes; and guided physical therapy at a facility once weekly for six weeks, if logistically practical. Failure of the above conservative therapeutic approaches is defined as persistent pain after three months despite attempting the above.

Exclusion Criteria:

  1. Prior radiation to the SIJ.
  2. Use of any pain medication or therapy less than 15 days prior to test product administration that has not or will not have had a stable dosage, frequency, or intensity for at least 3 months prior to test agent administration. Use of scheduled pain medication other than acetaminophen for conditions unrelated to SIJ syndrome that has not had a stable dosage for at least 3 months prior to test agent administration. Unwillingness to consider avoiding the use of pain medication for at least 24 hours prior to each follow up evaluation.
  3. Intra-articular treatment with corticosteroids or systemic steroid use within 3 months prior to screening.
  4. Intra-articular treatment with regenerative medicines (e.g., plasma, stem cell, placental products) at any point prior to screening.
  5. Participated in another clinical trial within the last 6 months.
  6. 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.
  7. Intra-articular treatment with hyaluronic acid within 6 months prior to screening.
  8. Surgical intervention on the index SIJ < 12 months, or arthroscopy < 3 months prior to screening.
  9. Non-ambulatory status.
  10. Past or current diagnosis of fibromyalgia or inflammatory arthritis, gout, rheumatoid arthritis, lupus arthropathy, psoriatic arthritis, avascular necrosis, severe bone deformity, active infection of the SIJ or at the site of injection, pes anserine bursitis, neurogenic or vascular claudication, or uncontrolled diabetes mellitus (HbA1C >8%).
  11. Past or current diagnosis of concurrent diseases, including uncontrolled arrhythmias, Class 3 or 4 congestive heart failure, active hepatitis B or C, liver enzymes ≥ 2 times ULN if there is also elevation of bilirubin, hypercoagulable state, eGFR <45 mL/min by CKD-EPI, and untreated malignancy or malignancy diagnosed within 6 months.
  12. Poorly controlled condition anticipated to have a likelihood of steroid requirement during the course of the trial that could potentially confound the outcomes.
  13. Past or current diagnosis of cancer, or at a high risk of recurrence.
  14. Diagnosis of secondary arthritis due to traumatic injury in the index SIJ within 2 years of screening.
  15. SIJ effusion in the index SIJ at screening that requires drainage for diagnostic purposes or symptomatic relief.
  16. Clinically significant, ongoing illness or medical condition, that in the opinion of the investigator constitutes a safety risk for participation in the study or that could interfere with achieving the study objectives, conduct or evaluation.
  17. Females who are pregnant or lactating.
  18. Regular use of anticoagulants (daily use of aspirin < 325 mg is acceptable).
  19. Active alcohol or substance abuse or any other reason that makes it unlikely that the subject will comply with study procedures.
  20. Positive results on the urine drug screen for a banned substance or substance for which a subject does not have a valid prescription, using standard screen at clinical site.
  21. Subjects with a psychiatric illness or condition, which, in the opinion of the investigation, would interfere with the conduct of the study or the interpretation of study results. Subjects with stable anxiety and depression defined as being on stable doses of antidepressant and anxiety drugs for the last 6 months and for which no dose changes are expected during the study can be included.
  22. Clinically significant medical, surgical, psychiatric, or laboratory abnormality that, in the judgment of the investigator, is likely to adversely affect the subject's risk-benefit or interfere with study compliance or assessment of safety or efficacy.
  23. Known allergy to local anesthetics or components of the study drug, including DMSO.
  24. Known allergy to radiographic contrast.
  25. Subjects with autoimmune disease or a known history of having Acquired Immunodeficiency Syndrome (AIDS) or Human Immunodeficiency Virus (HIV).
  26. Severe back pain due to other causes (e.g., lumbar disk degeneration, spinal stenosis), that in the opinion of the clinical investigator will render assessment of the SIJ pain difficult or ambiguous.
  27. History of recent (<1 year) major trauma to the pelvis.
  28. Metabolic bone disease (either induced or idiopathic).
  29. Involvement in litigation.
  30. Receiving disability payments or worker's compensation for back or SI joint pain.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Arm 1: Low dose
The first three subjects will receive the 30 x 106 TNC dose.
Biological: PremierMaxCB®-Platinum (CFL001);
Stem Cell: Human cell, tissue, and cellular or tissue-based product (HCT/P) manufactured from umbilical cord blood
Experimental: Arm 2: Medium Dose
The next three subjects will receive the 60 x 106 TNC dose.
Biological: PremierMaxCB®-Platinum (CFL001);
Stem Cell: Human cell, tissue, and cellular or tissue-based product (HCT/P) manufactured from umbilical cord blood
Experimental: Arm 3: High Dose
The next three subjects will receive the 90 x 106 TNC dose.
Biological: PremierMaxCB®-Platinum (CFL001);
Stem Cell: Human cell, tissue, and cellular or tissue-based product (HCT/P) manufactured from umbilical cord blood

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of participants with treatment-related adverse events as assessed by CTCAE v.5 and assessment of protocol defined study endpoints
Time Frame: 7, 30, 90, and 180 days post dose

All Adverse events (types and frequencies) will be collected for all patients. Protocol defined study endpoints will also be collected on all patients. Study endpoints include:

• Stopping criteria as defined in protocol section 6.11.2.8
7, 30, 90, and 180 days post dose
Number of participants with treatment-related adverse events as assessed by CTCAE v.5 and assessment of protocol defined study endpoints
Time Frame: 7, 30, 90, and 180 days post dose
Vital Sign Measurements (changes from baseline) Blood Pressure -systolic and diastolic (mm Hg)
7, 30, 90, and 180 days post dose

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
CFL001 Efficacy
Time Frame: 7, 30, 90, and 180 days post dose
Changes from baseline in overall activity obtained by actigraphy
7, 30, 90, and 180 days post dose
CFL001 Efficacy
Time Frame: 7, 30, 90, and 180 days post dose
Changes from baseline in patient reported outcomes with respect to SIJ and lower back pain measured the using the Visual Analog Scale
7, 30, 90, and 180 days post dose
CFL001 Efficacy
Time Frame: 7, 30, 90, 180 days post dose
Changes from baseline in Quality of Life Measured by PROMISE 29
7, 30, 90, 180 days post dose

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

University of Florida

Collaborators

Cord for Life, Inc.

Investigators

  • Principal Investigator: Rene Przkora, MD, University of Florida

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

May 10, 2024

Primary Completion (Estimated)

June 30, 2025

Study Completion (Estimated)

December 30, 2025

Study Registration Dates

First Submitted

May 26, 2023

First Submitted That Met QC Criteria

May 13, 2024

First Posted (Actual)

May 16, 2024

Study Record Updates

Last Update Posted (Actual)

May 16, 2024

Last Update Submitted That Met QC Criteria

May 13, 2024

Last Verified

May 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • IRB202300181

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

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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