- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT01319331
The Effects of Alpha-1 Antitrypsin (AAT) on the Progression of Type 1 Diabetes
March 23, 2017 updated by: University of Colorado, Denver
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.
Study Overview
Status
Completed
Conditions
Intervention / Treatment
Study Type
Interventional
Enrollment (Actual)
12
Phase
- Phase 1
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
-
-
Colorado
-
Aurora, Colorado, United States, 80045
- Barbara Davis Center for Childhood Diabetes
-
-
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
6 years to 45 years (Child, Adult)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
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
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Alpha-1 Antitrypsin (AAT, Aralast NP)
Alpha-1 Antitrypsin (AAT, Aralast NP) as prescribed for study duration
|
Eligible subjects will be treated once a week for 8 weeks (8 total treatments).
Other Names:
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
To assess participant safety & feasibility of study drug administration
Time Frame: Study duration is 2 years
|
Study duration is 2 years
|
Secondary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
To assess AAT treatment on the maintenance of c-peptide production
Time Frame: Stimulated c-peptide at year one and two.
|
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.
|
Continuous Glucose Monitoring at one and two years.
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Collaborators
Investigators
- Principal Investigator: Peter A Gottlieb, MD, University of Colorado, Denver
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
Helpful Links
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start
October 1, 2010
Primary Completion (Actual)
October 1, 2015
Study Completion (Actual)
May 1, 2016
Study Registration Dates
First Submitted
March 17, 2011
First Submitted That Met QC Criteria
March 18, 2011
First Posted (Estimate)
March 21, 2011
Study Record Updates
Last Update Posted (Actual)
March 24, 2017
Last Update Submitted That Met QC Criteria
March 23, 2017
Last Verified
March 1, 2017
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
- Digestive System Diseases
- Pathologic Processes
- Glucose Metabolism Disorders
- Metabolic Diseases
- Respiratory Tract Diseases
- Immune System Diseases
- Autoimmune Diseases
- Lung Diseases
- Endocrine System Diseases
- Liver Diseases
- Genetic Diseases, Inborn
- Subcutaneous Emphysema
- Emphysema
- Diabetes Mellitus
- Diabetes Mellitus, Type 1
- Alpha 1-Antitrypsin Deficiency
- Molecular Mechanisms of Pharmacological Action
- Enzyme Inhibitors
- Protease Inhibitors
- Serine Proteinase Inhibitors
- Trypsin Inhibitors
- Alpha 1-Antitrypsin
- Protein C Inhibitor
Other Study ID Numbers
- 09-0667
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Diabetes
-
University of Colorado, DenverMassachusetts General Hospital; Beta Bionics, Inc.CompletedDiabetes Mellitus, Type 1 | Type 1 Diabetes | Diabetes type1 | Type 1 Diabetes Mellitus | Autoimmune Diabetes | Diabetes Mellitus, Insulin-Dependent | Juvenile-Onset Diabetes | Diabetes, Autoimmune | Insulin-Dependent Diabetes Mellitus 1 | Diabetes Mellitus, Insulin-Dependent, 1 | Diabetes Mellitus, Brittle | Diabetes Mellitus, Juvenile-Onset and other conditionsUnited States
-
Guang NingRecruitingType 2 Diabetes Mellitus | Type1 Diabetes Mellitus | Monogenetic Diabetes | Pancreatogenic Diabetes | Drug-Induced Diabetes Mellitus | Other Forms of Diabetes MellitusChina
-
University of Trás-os-Montes and Alto DouroCompletedType 2 Diabetes Mellitus | Diabetes-Related ComplicationsPortugal
-
Northern Care Alliance NHS Foundation TrustBrighter ABCompletedDiabetes type1 | Diabetes type2United Kingdom
-
VeraLight, Inc.InLight SolutionsUnknownGestational Diabetes | Insulin Dependent Diabetes | Non Insulin Dependent DiabetesUnited States
-
Garvan Institute of Medical ResearchWeizmann Institute of ScienceActive, not recruitingType 2 Diabetes Mellitus | Pre DiabetesAustralia
-
Oregon State UniversitySanofiCompletedType I or Type II Diabetes (Excludes Gestational Diabetes)
-
Taichung Veterans General HospitalNational Health Research Institutes, TaiwanRecruitingDiabetes Complications | Type 2 Diabetes | Maturity-Onset Diabetes of the Young (MODY)Taiwan
-
University of RoehamptonRecruitingType2 Diabetes Mellitus | Pre DiabetesUnited Kingdom
-
Peking Union Medical College HospitalUnknownType 2 Diabetes Mellitus | Type 1 Diabetes Mellitus | Gestational Diabetes Mellitus | Pancreatogenic Diabetes Mellitus | Pregestational Diabetes Mellitus | Diabetes Patients in Perioperative PeriodChina
Clinical Trials on Alpha 1-Antitrypsin (AAT, Aralast NP)
-
National Institute of Allergy and Infectious Diseases...Juvenile Diabetes Research Foundation; Immune Tolerance Network (ITN)WithdrawnDiabetes Mellitus, Type 1United States
-
National Institute of Allergy and Infectious Diseases...Juvenile Diabetes Research Foundation; Immune Tolerance Network (ITN)TerminatedDiabetes Mellitus, Type 1United States
-
Baxalta now part of ShireCompletedAlpha 1-Antitrypsin DeficiencyNew Zealand, Australia
-
Kamada, Ltd.CompletedType 1 Diabetes MellitusIsrael
-
Rabin Medical CenterUnknownLung Transplantation | Bronchiolitis Obliterable Syndrome
-
Kamada, Ltd.CompletedNew Onset Type-1 DiabetesIsrael
-
Universität des SaarlandesCompleted
-
Baxalta now part of ShireBaxalta Innovations GmbH, now part of ShireTerminatedChronic Obstructive Pulmonary Disease | Alpha1-antitrypsin DeficiencyUnited States, Canada, Australia
-
Virginia Commonwealth UniversityCompletedAcute Myocardial InfarctionUnited States
-
Blessing Corporate Services, IncTakeda Pharmaceuticals North America, Inc.Withdrawn