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Clinical Application of Stem Cell Educator Therapy in Type 1 Diabetes

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ClinicalTrials.gov Identifier: NCT04011020
Recruitment Status : Not yet recruiting
First Posted : July 8, 2019
Last Update Posted : July 9, 2019
Sponsor:
Collaborator:
Hackensack Meridian Health
Information provided by (Responsible Party):
Tianhe Stem Cell Biotechnologies Inc.

Brief Summary:
Type 1 diabetes (T1D) is a T cell-mediated autoimmune disease that causes a deficit of pancreatic islet beta cells. Millions of individuals worldwide have T1D, and incidence increases annually. Several recent clinical trials point to the need for an approach that produces comprehensive immune modulation at both the local pancreatic and systemic levels. Stem Cell Educator (SCE) therapy offers comprehensive immune modulation at both the local and systemic levels in T1D by using a patient's own immune cells (including platelets) that are "educated" by cord blood stem cells. Tested clinically in more than 200 patients, SCE therapy has shown lasting reversal in autoimmunity in T1D patients, including improved C-peptide levels, reduced median glycated hemoglobin A1C (HbA1C) values, and decreased median daily usage of insulin. SCE therapy circulates a patient's blood through a blood cell separator, briefly cocultures the patient's immune cells with adherent Cord Blood Stem Cells (CB-SCs) in vitro, and returns the "educated" autologous immune cells to the patient's circulation.

Condition or disease Intervention/treatment Phase
Type 1 Diabetes Combination Product: Stem Cell Educator therapy Phase 2 Phase 3

Detailed Description:

The SCE device is made of a hydrophobic material from FDA-approved (USP Class VI) dishes that tightly binds stem cells CB-SCs without interfering with their immune modulating capability. We originally designed a chamber for co-culture of lymphocytes and CB-SCs that included nine discs of the material with a flow pathway and adherent CB-SCs sandwiched between a top cover plate and a bottom collecting plate. In this trial, we are going to use the 12-layer SCE device.

The SCE therapy carried a lower risk of infection than a typical blood transfusion, and did not introduce stem cells or reagents into the patients. In addition, CB-SCs have very low immunogenicity, and the CB-SCs cultured in the device are a highly restricted population and contain no CD3+ T cells or other lymphocyte subsets, eliminating the need for human leukocyte antigen (HLA) matching prior to treatment. This innovative approach has the potential to provide CB-SC-mediated immune modulation therapy for multiple autoimmune diseases while mitigating the safety and ethical concerns associated with other approaches such as T1D, type 2 diabetes (T2D), and alopecia areata (AA) in clinics. The relative simplicity of the approach may also provide cost and time savings relative to other approaches.


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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 50 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Masking Description: All recruited T1D subjects will receive the treatment with Stem Cell Educator therapy.
Primary Purpose: Treatment
Official Title: Clinical Application of Stem Cell Educator Therapy in Type 1 Diabetes
Estimated Study Start Date : November 1, 2019
Estimated Primary Completion Date : October 31, 2021
Estimated Study Completion Date : December 20, 2021

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Diabetes Type 1

Arm Intervention/treatment
Experimental: Treatment of T1D with Stem Cell Educator therapy
Recruited T1D subjects will receive one treatment with SCE therapy.
Combination Product: Stem Cell Educator therapy
Patients with T1D will be evaluated by the study principal investigator or co-investigators. Informed consent will be obtained at the initial screening visit. The initial screening visit will occur within 30 days of initiation of SCE therapy. The second screening visit will occur within 7 days of therapy. Subjects who meet all criteria will be scheduled for treatment. All enrolled subjects will receive treatment with the SCE system consisting of a single session of mononuclear cells (MNC) collection by apheresis where 10 L of blood will be processed on day -1. The MNC product will then be exposed over to the SCE and on day 0 the product will be infused intravenously back to the patient.




Primary Outcome Measures :
  1. Incidence of Treatment Adverse Events in T1D Subjects [ Time Frame: 6 month ]
    The occurrence of treatment-related adverse events will be evaluated post the treatment with SCE therapy.


Secondary Outcome Measures :
  1. Preliminary efficacy of SCE therapy to improve beta cell function [ Time Frame: 12 months ]
    Preliminary efficacy as measured by Area under the C-peptide curve (AUC) over the first 2 hours of a 3-hour mixed meal tolerance test (MMTT)

  2. Preliminary efficacy of SCE therapy to improve glucose control [ Time Frame: 12 months ]
    Change in HbA1C levels over time

  3. Preliminary efficacy of SCE therapy to reduce insulin dose [ Time Frame: 12 months ]
    Change in daily insulin requirements

  4. Efficacy of SCE therapy in immune modulation [ Time Frame: 12 month ]
    Measurements of immune markers at baseline, 1, 3, 6, 9, and 12 months. Peripheral blood mononuclear cells (PBMC) will be collected and tested by flow cytometry.



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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Adult patients ( 12 years)
  2. Must have a diagnosis of type 1 diabetes mellitus based on the 2015 American Diabetes Association criteria for the Clarification and Diagnosis of diabetes.
  3. Must have a blood test confirming the presence of at least one autoantibody to pancreatic islet Cells (IAA, IA2, GAD 65, ZnT8).
  4. Fasting C-peptide level > 0.3 ng/ml
  5. HbA1C < 10% at enrollment
  6. Recent diagnosis (within two years of enrollment)
  7. Adequate venous access for apheresis
  8. Must be equipped with a continuous glucose monitoring system (CGMS)
  9. Ability to provide informed consent
  10. For female patients only, willingness to use FDA-recommended birth control (http://www.fda.gov/downloads/ForConsumers/ByAudience/ForWomen/FreePublications/UCM356451.pdf) until 6 months post treatment.
  11. Must agree to comply with all study requirements and be willing to complete all study visits

Exclusion Criteria:

  1. AST or ALT 2 > x upper limit of normal.
  2. Abnormal bilirubin (total bilirubin > 1.2 mg/dL, direct bilirubin > 0.4 mg/dL)
  3. Creatinine > 2.0 mg/dl.
  4. Known coronary artery disease or EKG suggestive of coronary artery disease unless cardiac clearance for apheresis is obtained from a cardiologist.
  5. Known active infection such as Hepatitis B, Hepatitis C, or Human Immunodeficiency Virus (HIV)
  6. Pregnancy assessed by a positive serum pregnancy test or breastfeeding mothers
  7. Use of immunosuppressive medication within one month of enrollment including but not limited to prednisone, cyclosporine, tacrolimus, sirolimus, and chemotherapy.
  8. Presence of any other autoimmune diseases (lupus, rheumatoid arthritis, scleroderma, etc.)
  9. Anticoagulation other than ASA.
  10. Hemoglobin < 10 g/dl or platelets < 100 k/ml
  11. Is unable or unwilling to provide informed consent
  12. Presence of any other physical or psychological medical condition that, in the opinion of the investigator, would preclude participation

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): NCT04011020


Contacts
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Contact: YONG I ZHAO, MD,PhD 201 880 0290 Zhao@tianhecell.com

Locations
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United States, New Jersey
Hackensack Meridian Health
Hackensack, New Jersey, United States, 07601
Sponsors and Collaborators
Tianhe Stem Cell Biotechnologies Inc.
Hackensack Meridian Health
Investigators
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Study Chair: YONG ZHAO, MD,PhD Tianhe Stem Cell Biotechnologies Inc.

Additional Information:
Publications of Results:
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Responsible Party: Tianhe Stem Cell Biotechnologies Inc.
ClinicalTrials.gov Identifier: NCT04011020     History of Changes
Other Study ID Numbers: 2019-TH-002
First Posted: July 8, 2019    Key Record Dates
Last Update Posted: July 9, 2019
Last Verified: July 2019
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: Yes
Device Product Not Approved or Cleared by U.S. FDA: No
Pediatric Postmarket Surveillance of a Device Product: No

Additional relevant MeSH terms:
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Diabetes Mellitus
Diabetes Mellitus, Type 1
Glucose Metabolism Disorders
Metabolic Diseases
Endocrine System Diseases
Autoimmune Diseases
Immune System Diseases