Diabetes Mellitus Type 1
Conditions
Keywords
Diabetes
Brief summary
This is a prospective, single arm, open-label, single-center pilot study to assess the safety, feasibility, and efficacy of Stem Cell Educator therapy for the treatment of patients with Type 1 Diabetes.
Detailed description
Our previous work demonstrated that human cord blood-derived multipotent stem cells (CB-SCs) are a unique type of stem cell identified from human cord blood, distinct from other stem cell types including hematopoietic stem cells (HSCs), and mesenchymal stem cells (MSCs). The stem cells and harnessed some of their unique properties with Stem Cell Educator therapy by using CB-SCs in a closed-loop system that circulates a patient's blood through a blood cell separator, briefly co-cultures the patient's lymphocytes with adherent CB-SCs in vitro, and returns the "educated" lymphocytes (but not the CB-SCs) to the patient's circulation . This treatment leads to global immune modulations and immune balance as demonstrated by clinical data and animal studies. The Stem Cell Educator therapy may revolutionize the clinical treatment of diabetes and other immune-related diseases through CB-SCs' immune education and induction of immune balance, without the safety and ethical concerns associated with conventional stem cell-based approaches
Interventions
BIOLOGICALStem Cell Educator Therapy
Patients will receive apheresis and then have their own blood returned to them with the "educated" lymphocytes
Sponsors
Hackensack Meridian Health
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Adult patients \>/=18 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 * Adequate venous access for apheresis * Ability to provide informed consent * 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. * 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 * Pregnancy 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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Treatment Related Adverse Events | 12 months | The primary study endpoint will be the occurrence of treatment-related adverse effects. Adverse events that occur during therapy (especially those that necessitate temporary or permanent discontinuation of therapy) and over the 12-month follow-up period will be assessed. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of Patients Unable to Complete Therapy | One week | Number of patients who were unable to complete SCE Therapy. |
Countries
United States
Contacts
PRINCIPAL_INVESTIGATORMichelle Donato, MD
Hackensack Meridian Health
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Stem Cell Educator Therapy Patients will have apheresis performed and then have their own blood returned to them with the educated lymphocytes
Stem Cell Educator Therapy: Patients will receive apheresis and then have their own blood returned to them with the educated lymphocytes | 13 |
| Total | 13 |
Baseline characteristics
| Characteristic | Stem Cell Educator Therapy |
|---|---|
| Age, Categorical <=18 years | 0 Participants |
| Age, Categorical >=65 years | 0 Participants |
| Age, Categorical Between 18 and 65 years | 13 Participants |
| Age, Continuous | 24 years |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 11 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 2 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 1 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 1 Participants |
| Race (NIH/OMB) White | 11 Participants |
| Region of Enrollment United States | 13 participants |
| Sex: Female, Male Female | 1 Participants |
| Sex: Female, Male Male | 12 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 8 |
| other Total, other adverse events | 4 / 8 |
| serious Total, serious adverse events | 1 / 8 |
Outcome results
Primary
Number of Participants With Treatment Related Adverse Events
The primary study endpoint will be the occurrence of treatment-related adverse effects. Adverse events that occur during therapy (especially those that necessitate temporary or permanent discontinuation of therapy) and over the 12-month follow-up period will be assessed.
Time frame: 12 months
Population: Patients Experiencing Treatment Related Adverse Events
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Stem Cell Educator Therapy | Number of Participants With Treatment Related Adverse Events | 1 Participants |
Secondary
Number of Patients Unable to Complete Therapy
Number of patients who were unable to complete SCE Therapy.
Time frame: One week
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Stem Cell Educator Therapy | Number of Patients Unable to Complete Therapy | 0 Participants |