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

The recruitment status of this study is unknown because the information has not been verified recently.
Verified March 2011 by University of Colorado, Denver.
Recruitment status was  Recruiting
Sponsor:
Collaborator:
Omni Bio Pharmaceutical, Inc.
Information provided by (Responsible Party):
University of Colorado, Denver
ClinicalTrials.gov Identifier:
NCT01319331
First received: March 17, 2011
Last updated: January 7, 2013
Last verified: March 2011
  Purpose

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.


Condition Intervention Phase
Diabetes
Type 1 Diabetes
Drug: Alpha 1-Antitrypsin (AAT, Aralast NP)
Phase 1

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: The Effects of Open Label Alpha-1 Antitrypsin on the Progression of Type 1 Diabetes in Subjects With Detectable C-peptide

Resource links provided by NLM:


Further study details as provided by University of Colorado, Denver:

Primary Outcome Measures:
  • To assess participant safety & feasibility of study drug administration [ Time Frame: Study duration is 2 years ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • To assess AAT treatment on the maintenance of c-peptide production [ Time Frame: Stimulated c-peptide at year one and two. ] [ Designated as safety issue: No ]
  • Assess the effects of AAT on glycemic variability and A1c. [ Time Frame: Continuous Glucose Monitoring at one and two years. ] [ Designated as safety issue: No ]

Estimated Enrollment: 15
Study Start Date: October 2010
Estimated Study Completion Date: September 2013
Estimated Primary Completion Date: September 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Alpha-1 Antitrypsin (AAT, Aralast NP)
Alpha-1 Antitrypsin (AAT, Aralast NP) as prescribed for study duration
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

  Eligibility

Ages Eligible for Study:   6 Years to 45 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01319331

Contacts
Contact: Lisa Meyers 303-724-6893 lisa.meyers@ucdenver.edu

Locations
United States, Colorado
Barbara Davis Center for Childhood Diabetes Recruiting
Aurora, Colorado, United States, 80045
Contact: Dominic DiDomenico    303-724-5687    dominic.didomenico@ucdenver.edu   
Principal Investigator: Peter A Gottlieb, MD         
Sub-Investigator: Aaron Michels, MD         
Sponsors and Collaborators
University of Colorado, Denver
Omni Bio Pharmaceutical, Inc.
  More Information

Additional Information:
No publications provided by University of Colorado, Denver

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: University of Colorado, Denver
ClinicalTrials.gov Identifier: NCT01319331     History of Changes
Other Study ID Numbers: 09-0667
Study First Received: March 17, 2011
Last Updated: January 7, 2013
Health Authority: United States: Food and Drug Administration

Keywords provided by University of Colorado, Denver:
diabetes
type 1 diabetes
AAT
Alpha-1 Antitrypsin

Additional relevant MeSH terms:
Diabetes Mellitus
Diabetes Mellitus, Type 1
Glucose Metabolism Disorders
Metabolic Diseases
Endocrine System Diseases
Autoimmune Diseases
Immune System Diseases
Alpha 1-Antitrypsin
Protein C Inhibitor
Trypsin Inhibitors
Serine Proteinase Inhibitors
Protease Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions

ClinicalTrials.gov processed this record on April 22, 2014