Diabetes, Type 1 Diabetes
Conditions
Keywords
diabetes, type 1 diabetes, AAT, Alpha-1 Antitrypsin
Brief summary
The purpose of this study is to determine if the drug Alpha-1 Antitrypsin (AAT, Aralast NP) will preserve beta-cell function and help slow the progression of type 1 diabetes.
Interventions
DRUGAlpha 1-Antitrypsin (AAT, Aralast NP)
Eligible subjects will be treated once a week for 8 weeks (8 total treatments).
Sponsors
Omni Bio Pharmaceutical, Inc.
University of Colorado, Denver
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
6 Years to 45 Years
Healthy volunteers
No
Inclusion criteria
* Diagnosis of Type 1 Diabetes Mellitus based on ADA Criteria for fewer than 5 years but more than 100 days * 6-45 years of age, inclusive. To assess safety, we will initially enroll 8 patients over the age of 16. Following the last infusion of the 8th patient, we will assess adverse events. As long as there are no stopping criteria met for these 8 patients we will decrease the age criteria down to 6 years old. * C-peptide increase during screening mixed meal tolerance test with a minimal stimulated value of ≥ 0.2 pmol/mL. * Positive for antibodies to insulin (if insulin autoantibody positive only, determination must be within two weeks of insulin initiation), GAD-65, IA-2 or ZnT8 * Agree to intensive management of diabetes with an HgbA1c goal of \< 7.0% * If female, (a) surgically sterile or (b) postmenopausal or (c) if of reproductive potential, willing to use medically acceptable birth control (e.g. female hormonal contraception, barrier methods or sterilization. ) until 3 months after completion of any treatment period * If male and of reproductive potential, willing to use medically acceptable birth control until 3 months after completion of any treatment period, unless the female partner is postmenopausal or surgically sterile * Serum creatinine ≤ 1.5 x upper limit of normal * AST \< 2 times the upper limit of normal * Hematology:WBC \> 3000 x 109/L; platelets \> 100 x 109/L; hemoglobin \> 10.0 g/dL.
Exclusion criteria
* Unable or unwilling to comply with the requirements of the study protocol * Body Mass Index (BMI) \> 30 kg/m2 * Unstable blood sugar control defined as one or more episodes of severe hypoglycemia (defined as hypoglycemia that required the assistance of another person) within the last 30 days * Previous immunotherapy for T1D * Administration of an experimental agent for T1D at any time or use of an experimental device for T1D within 30 days of screening, unless approved by the study PI * History of any organ transplant, including islet cell transplant * Active autoimmune or immune deficiency disorder (e.g. sarcoidosis, rheumatoid arthritis) * Serum bilirubin \> ULN, except those subjects whose abnormal values were attributed to any stable, benign condition (such as Gilbert's Syndrome) may be included * TSH outside the normal range at screening, except those subjects on stable doses of thyroid hormone replacement therapy may be included * Known HIV positivity, active hepatitis B or active hepatitis C infection * Anticipated pregnancy during active dosing or within 3 months after completion of active dosing phase * History of a malignant neoplasm within the previous 5 years (except in situ cervical cancer and curable non-melanoma skin malignancy) * Any social condition or medical condition that would, in the opinion of the investigator, prevent complete participation in the study or that would pose a significant hazard to the subjects' participation * History of active substance abuse within 12 months of screening * A psychiatric or medical disorder that would prevent giving informed consent * Individuals with a history of IgA deficiency * Individuals with a history of hypersensitivity to AAT
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| To assess participant safety & feasibility of study drug administration | Study duration is 2 years |
Secondary
| Measure | Time frame |
|---|---|
| To assess AAT treatment on the maintenance of c-peptide production | Stimulated c-peptide at year one and two. |
| Assess the effects of AAT on glycemic variability and A1c. | Continuous Glucose Monitoring at one and two years. |
Countries
United States
Outcome results
None listed