Clinical Application of Stem Cell Educator Therapy in Type 1 Diabetes

Throne Biotechnologies

Status and phase

Enrolling

Phase 3

Conditions

Type 1 Diabetes

Treatments

Combination Product: Stem Cell Educator therapy

Study type

Interventional

Funder types

Other

Industry

Identifiers

NCT04011020

2019-TH-002

Details and patient eligibility

About

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 300 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-SC) in vitro, and returns the "educated" autologous immune cells to the patient's circulation.

Full description

The SCE device is made of a hydrophobic material from FDA-approved (USP Class VI) dishes that tightly binds stem cells CB-SC without interfering with their immune modulating capability. During SCE therapy, a patient's peripheral blood mononuclear cells (PBMC) are collected by leukapheresis and co-cultured with adherent cord-blood-derived multipotent stem cells (CB-SC) in vitro, after which the "educated" PBMC are returned to the patient's circulation via infusion. Over the last 15 years, this technology has been evaluated through international multicenter clinical studies conducted in the United States, China, and Spain. These studies have demonstrated the clinical safety and efficacy of SCE therapy in treating type 1 diabetes (T1D), alopecia areata (AA), and other autoimmune conditions, including reversal of T1D-associated autoimmunity and treatment of other immune dysfunction-associated diseases, such as alopecia areata, type 2 diabetes (T2D), Crohn's disease and long-COVID.

Enrollment

80 estimated patients

Sex

All

Ages

10+ years old

Volunteers

No Healthy Volunteers

Inclusion criteria

Pediatric and adult patients (>10 years).
Must have a diagnosis of type 1 diabetes mellitus based on the American Diabetes Association (ADA) Guidelines.
Must have a blood test confirming the presence of at least one autoantibody to pancreatic islet beta Cells (IAA, IA2, GAD 65, ZnT8).
Fasting C-peptide level > 0.3 ng/ml.
Adequate venous access for apheresis.
Ability to provide informed consent or assent.
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, Human Immunodeficiency Virus (HIV), HTLV (human T-cell leukemia-lymphoma virus cAB), or active tuberculosis.
Pregnant or breastfeeding women.
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 (Hashimoto's thyroiditis, celiac disease, lupus, rheumatoid arthritis, scleroderma, etc.).
Anticoagulation other than ASA.
Hemoglobin < 10 g/dl or platelets < 100 k/ml.
Subjects with leukopenia, total white blood cells (WBC) count < 4000/uL.
Patient or patient's surrogate is unable or unwilling to voluntarily sign an informed consent form.
Presence of any other physical or psychological medical condition that, in the opinion of the investigator, would preclude participation.