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

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT01319331
First Posted: March 21, 2011
Last Update Posted: March 24, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Collaborator:
Omni Bio Pharmaceutical, Inc.
Information provided by (Responsible Party):
University of Colorado, Denver
  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: Intervention Model: Single Group Assignment
Masking: None (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 ]

Secondary Outcome Measures:
  • 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. ]

Enrollment: 12
Study Start Date: October 2010
Study Completion Date: May 2016
Primary Completion Date: October 2015 (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

Information from the National Library of Medicine

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Ages Eligible for Study:   6 Years to 45 Years   (Child, Adult)
Sexes Eligible for Study:   All
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
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01319331


Locations
United States, Colorado
Barbara Davis Center for Childhood Diabetes
Aurora, Colorado, United States, 80045
Sponsors and Collaborators
University of Colorado, Denver
Omni Bio Pharmaceutical, Inc.
Investigators
Principal Investigator: Peter A Gottlieb, MD University of Colorado, Denver
  More Information

Additional Information:
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
First Submitted: March 17, 2011
First Posted: March 21, 2011
Last Update Posted: March 24, 2017
Last Verified: March 2017

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