NCT04011020

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.

Trial Health

57
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
50

participants targeted

Target at P25-P50 for phase_2

Timeline
Completed

Started Sep 2022

Geographic Reach
1 country

2 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

July 4, 2019

Completed
4 days until next milestone

First Posted

Study publicly available on registry

July 8, 2019

Completed
3.2 years until next milestone

Study Start

First participant enrolled

September 20, 2022

Completed
2.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 20, 2024

Completed
8 months until next milestone

Study Completion

Last participant's last visit for all outcomes

June 20, 2025

Completed
Last Updated

April 29, 2024

Status Verified

April 1, 2024

Enrollment Period

2.1 years

First QC Date

July 4, 2019

Last Update Submit

April 25, 2024

Conditions

Type 1 Diabetes

Outcome Measures

Primary Outcomes (1)

  • Incidence of Treatment Adverse Events in T1D Subjects

    The occurrence of treatment-related adverse events will be evaluated post the treatment with SCE therapy.

    6 month

Secondary Outcomes (4)

  • Preliminary efficacy of SCE therapy to improve beta cell function

    12 months

  • Preliminary efficacy of SCE therapy to improve glucose control

    12 months

  • Preliminary efficacy of SCE therapy to reduce insulin dose

    12 months

  • Efficacy of SCE therapy in immune modulation

    12 month

Study Arms (2)

Treatment of T1D with Stem Cell Educator therapy

EXPERIMENTAL

Recruited T1D subjects will receive one treatment with SCE therapy.

Combination Product: Stem Cell Educator therapy

Conventional insulin therapy

EXPERIMENTAL

Control group will receive conventional insulin therapy.

Combination Product: Stem Cell Educator therapy

Interventions

Stem Cell Educator therapyCOMBINATION_PRODUCT

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.

Conventional insulin therapyTreatment of T1D with Stem Cell Educator therapy

Eligibility Criteria

Age14 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

Hackensack Meridian Health

Hackensack, New Jersey, 07601, United States

ACTIVE NOT RECRUITING

Throne Biotechnologies

Paramus, New Jersey, 07652, United States

RECRUITING

Related Publications (5)

  • Zhao Y, Knight CM, Jiang Z, Delgado E, Van Hoven AM, Ghanny S, Zhou Z, Zhou H, Yu H, Hu W, Li H, Li X, Perez-Basterrechea M, Zhao L, Zhao Y, Giangola J, Weinberg R, Mazzone T. Stem Cell Educator therapy in type 1 diabetes: From the bench to clinical trials. Autoimmun Rev. 2022 May;21(5):103058. doi: 10.1016/j.autrev.2022.103058. Epub 2022 Jan 31.

    PMID: 35108619BACKGROUND

  • Zhao Y, Jiang Z, Zhao T, Ye M, Hu C, Yin Z, Li H, Zhang Y, Diao Y, Li Y, Chen Y, Sun X, Fisk MB, Skidgel R, Holterman M, Prabhakar B, Mazzone T. Reversal of type 1 diabetes via islet beta cell regeneration following immune modulation by cord blood-derived multipotent stem cells. BMC Med. 2012 Jan 10;10:3. doi: 10.1186/1741-7015-10-3.

    PMID: 22233865RESULT

  • Zhao Y. Stem cell educator therapy and induction of immune balance. Curr Diab Rep. 2012 Oct;12(5):517-23. doi: 10.1007/s11892-012-0308-1.

    PMID: 22833322RESULT

  • Delgado E, Perez-Basterrechea M, Suarez-Alvarez B, Zhou H, Revuelta EM, Garcia-Gala JM, Perez S, Alvarez-Viejo M, Menendez E, Lopez-Larrea C, Tang R, Zhu Z, Hu W, Moss T, Guindi E, Otero J, Zhao Y. Modulation of Autoimmune T-Cell Memory by Stem Cell Educator Therapy: Phase 1/2 Clinical Trial. EBioMedicine. 2015 Nov 5;2(12):2024-36. doi: 10.1016/j.ebiom.2015.11.003. eCollection 2015 Dec.

    PMID: 26844283RESULT

  • Zhao Y, Jiang Z, Delgado E, Li H, Zhou H, Hu W, Perez-Basterrechea M, Janostakova A, Tan Q, Wang J, Mao M, Yin Z, Zhang Y, Li Y, Li Q, Zhou J, Li Y, Martinez Revuelta E, Maria Garcia-Gala J, Wang H, Perez-Lopez S, Alvarez-Viejo M, Menendez E, Moss T, Guindi E, Otero J. Platelet-Derived Mitochondria Display Embryonic Stem Cell Markers and Improve Pancreatic Islet beta-cell Function in Humans. Stem Cells Transl Med. 2017 Aug;6(8):1684-1697. doi: 10.1002/sctm.17-0078. Epub 2017 Jul 7.

    PMID: 28685960RESULT

Related Links

MeSH Terms

Conditions

Diabetes Mellitus, Type 1

Condition Hierarchy (Ancestors)

Diabetes MellitusGlucose Metabolism DisordersMetabolic DiseasesNutritional and Metabolic DiseasesEndocrine System DiseasesAutoimmune DiseasesImmune System Diseases

Study Officials

  • YONG ZHAO, MD,PhD

    Throne Biotechnologies Inc.

    STUDY CHAIR

Central Study Contacts

YONG ZHAO, MD,PhD

CONTACT

201 988 0290Yong.Zhao@ThroneBio.com

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
DOUBLE
Who Masked
CARE PROVIDER, INVESTIGATOR
Masking Details
All recruited T1D subjects will receive the treatment with Stem Cell Educator therapy.
Purpose
TREATMENT
Intervention Model
PARALLEL
Model Details: Treatment group receive Stem Cell Educator therapy, control group receive conventional insulin therapy.
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

July 4, 2019

First Posted

July 8, 2019

Study Start

September 20, 2022

Primary Completion

October 20, 2024

Study Completion

June 20, 2025

Last Updated

April 29, 2024

Record last verified: 2024-04

Data Sharing

IPD Sharing
Will not share

Locations

US(2)