Clinical Application of Stem Cell Educator Therapy in Type 1 Diabetes
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ClinicalTrials.gov Identifier: NCT04011020 |
Recruitment Status :
Recruiting
First Posted : July 8, 2019
Last Update Posted : April 29, 2024
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Condition or disease | Intervention/treatment | Phase |
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Type 1 Diabetes | Combination Product: Stem Cell Educator therapy | Phase 2 Phase 3 |
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.
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 50 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Intervention Model Description: | Treatment group receive Stem Cell Educator therapy, control group receive conventional insulin therapy. |
Masking: | Double (Care Provider, Investigator) |
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 |
Actual Study Start Date : | September 20, 2022 |
Estimated Primary Completion Date : | October 20, 2024 |
Estimated Study Completion Date : | June 20, 2025 |
Arm | Intervention/treatment |
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Experimental: Treatment of T1D with Stem Cell Educator therapy
Recruited T1D subjects will receive one treatment with SCE therapy.
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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. |
Experimental: Conventional insulin therapy
Control group will receive conventional insulin therapy.
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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. |
- 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.
- 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)
- Preliminary efficacy of SCE therapy to improve glucose control [ Time Frame: 12 months ]Change in HbA1C levels over time
- Preliminary efficacy of SCE therapy to reduce insulin dose [ Time Frame: 12 months ]Change in daily insulin requirements
- 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: | 14 Years and older (Child, Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Adult patients ( 14 years)
- Must have a diagnosis of type 1 diabetes mellitus based on the 2015 American Diabetes Association criteria for the Clarification and Diagnosis of diabetes.
- Must have a blood test confirming the presence of at least one autoantibody to pancreatic islet Cells (IAA, IA2, GAD 65, ZnT8).
- Fasting C-peptide level > 0.3 ng/ml
- HbA1C < 10% at enrollment
- Recent diagnosis (within two years of enrollment)
- Adequate venous access for apheresis
- Must be equipped with a continuous glucose monitoring system (CGMS)
- Ability to provide informed consent
- 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.
- Must agree to comply with all study requirements and be willing to complete all study visits
Exclusion Criteria:
- AST or ALT 2 > x upper limit of normal.
- Abnormal bilirubin (total bilirubin > 1.2 mg/dL, direct bilirubin > 0.4 mg/dL)
- Creatinine > 2.0 mg/dl.
- Known coronary artery disease or EKG suggestive of coronary artery disease unless cardiac clearance for apheresis is obtained from a cardiologist.
- Known active infection such as Hepatitis B, Hepatitis C, or Human Immunodeficiency Virus (HIV)
- Pregnancy assessed by a positive serum pregnancy test or breastfeeding mothers
- Use of immunosuppressive medication within one month of enrollment including but not limited to prednisone, cyclosporine, tacrolimus, sirolimus, and chemotherapy.
- Presence of any other autoimmune diseases (lupus, rheumatoid arthritis, scleroderma, etc.)
- Anticoagulation other than ASA.
- Hemoglobin < 10 g/dl or platelets < 100 k/ml
- Is unable or unwilling to provide informed consent
- Presence of any other physical or psychological medical condition that, in the opinion of the investigator, would preclude participation
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
Contact: YONG ZHAO, MD,PhD | 201 988 0290 | Yong.Zhao@ThroneBio.com |
United States, New Jersey | |
Hackensack Meridian Health | Active, not recruiting |
Hackensack, New Jersey, United States, 07601 | |
Throne Biotechnologies | Recruiting |
Paramus, New Jersey, United States, 07652 | |
Contact: Yong Zhao, MD,PhD 201-988-0290 yong.zhao@thronebio.com | |
Principal Investigator: Boris Veysman, MD |
Study Chair: | YONG ZHAO, MD,PhD | Throne Biotechnologies Inc. |
Publications of Results:
Other Publications:
Responsible Party: | Throne Biotechnologies Inc. |
ClinicalTrials.gov Identifier: | NCT04011020 |
Other Study ID Numbers: |
2019-TH-002 |
First Posted: | July 8, 2019 Key Record Dates |
Last Update Posted: | April 29, 2024 |
Last Verified: | April 2024 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | Yes |
Diabetes Mellitus Diabetes Mellitus, Type 1 Glucose Metabolism Disorders Metabolic Diseases |
Endocrine System Diseases Autoimmune Diseases Immune System Diseases |