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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

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
Autoimmune Diseases
Endocrine System Diseases
Glucose Metabolism Disorders
Immune System Diseases
Metabolic Diseases
Alpha 1-Antitrypsin
Protein C Inhibitor
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Protease Inhibitors
Serine Proteinase Inhibitors
Trypsin Inhibitors

ClinicalTrials.gov processed this record on October 21, 2014