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The Effects of Alpha-1 Antitrypsin (AAT) on the Progression of Type 1 Diabetes

This study has been completed.
Omni Bio Pharmaceutical, Inc.
Information provided by (Responsible Party):
University of Colorado, Denver Identifier:
First received: March 17, 2011
Last updated: March 23, 2017
Last verified: March 2017

March 17, 2011
March 23, 2017
October 2010
October 2015   (Final data collection date for primary outcome measure)
To assess participant safety & feasibility of study drug administration [ Time Frame: Study duration is 2 years ]
Same as current
Complete list of historical versions of study NCT01319331 on Archive Site
  • To assess AAT treatment on the maintenance of c-peptide production [ Time Frame: Stimulated c-peptide at year one and two. ]
  • Assess the effects of AAT on glycemic variability and A1c. [ Time Frame: Continuous Glucose Monitoring at one and two years. ]
Same as current
Not Provided
Not Provided
The Effects of Alpha-1 Antitrypsin (AAT) on the Progression of Type 1 Diabetes
The Effects of Open Label Alpha-1 Antitrypsin on the Progression of Type 1 Diabetes in Subjects With Detectable C-peptide
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.
Not Provided
Phase 1
Intervention Model: Single Group Assignment
Masking: No masking
Primary Purpose: Treatment
  • Diabetes
  • Type 1 Diabetes
Drug: Alpha 1-Antitrypsin (AAT, Aralast NP)
Eligible subjects will be treated once a week for 8 weeks (8 total treatments).
Other Names:
  • Alpha-1 Antitrypsin
  • AAT
  • Aralast NP
Experimental: Alpha-1 Antitrypsin (AAT, Aralast NP)
Alpha-1 Antitrypsin (AAT, Aralast NP) as prescribed for study duration
Intervention: Drug: Alpha 1-Antitrypsin (AAT, Aralast NP)
Ozeri E, Mizrahi M, Shahaf G, Lewis EC. α-1 antitrypsin promotes semimature, IL-10-producing and readily migrating tolerogenic dendritic cells. J Immunol. 2012 Jul 1;189(1):146-53. doi: 10.4049/jimmunol.1101340. Epub 2012 May 25.

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
May 2016
October 2015   (Final data collection date for primary outcome measure)

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
Sexes Eligible for Study: All
6 Years to 45 Years   (Child, Adult)
Contact information is only displayed when the study is recruiting subjects
United States
Not Provided
Not Provided
Not Provided
University of Colorado, Denver
University of Colorado, Denver
Omni Bio Pharmaceutical, Inc.
Principal Investigator: Peter A Gottlieb, MD University of Colorado, Denver
University of Colorado, Denver
March 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP