NCT01319331

Brief Summary

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
12

participants targeted

Target at below P25 for phase_1 diabetes

Timeline
Completed

Started Oct 2010

Longer than P75 for phase_1 diabetes

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

October 1, 2010

Completed
6 months until next milestone

First Submitted

Initial submission to the registry

March 17, 2011

Completed
4 days until next milestone

First Posted

Study publicly available on registry

March 21, 2011

Completed
4.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 1, 2015

Completed
7 months until next milestone

Study Completion

Last participant's last visit for all outcomes

May 1, 2016

Completed
Last Updated

March 24, 2017

Status Verified

March 1, 2017

Enrollment Period

5 years

First QC Date

March 17, 2011

Last Update Submit

March 23, 2017

Conditions

DiabetesType 1 Diabetes

Keywords

diabetestype 1 diabetesAATAlpha-1 Antitrypsin

Outcome Measures

Primary Outcomes (1)

  • To assess participant safety & feasibility of study drug administration

    Study duration is 2 years

Secondary Outcomes (2)

  • To assess AAT treatment on the maintenance of c-peptide production

    Stimulated c-peptide at year one and two.

  • Assess the effects of AAT on glycemic variability and A1c.

    Continuous Glucose Monitoring at one and two years.

Study Arms (1)

Alpha-1 Antitrypsin (AAT, Aralast NP)

EXPERIMENTAL

Alpha-1 Antitrypsin (AAT, Aralast NP) as prescribed for study duration

Drug: Alpha 1-Antitrypsin (AAT, Aralast NP)

Interventions

Alpha 1-Antitrypsin (AAT, Aralast NP)DRUG

Eligible subjects will be treated once a week for 8 weeks (8 total treatments).

Also known as: Alpha-1 Antitrypsin, AAT, Aralast NP
Alpha-1 Antitrypsin (AAT, Aralast NP)

Eligibility Criteria

Age6 Years - 45 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Diagnosis of Type 1 Diabetes Mellitus based on ADA Criteria for fewer than 5 years but more than 100 days
  • 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.

You may not qualify if:

  • 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
  • +2 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Barbara Davis Center for Childhood Diabetes

Aurora, Colorado, 80045, United States

Location

Related Publications (1)

  • Ozeri E, Mizrahi M, Shahaf G, Lewis EC. alpha-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.

    PMID: 22634621DERIVED

Related Links

MeSH Terms

Conditions

Diabetes MellitusDiabetes Mellitus, Type 1alpha 1-Antitrypsin Deficiency

Interventions

alpha 1-Antitrypsin

Condition Hierarchy (Ancestors)

Glucose Metabolism DisordersMetabolic DiseasesNutritional and Metabolic DiseasesEndocrine System DiseasesAutoimmune DiseasesImmune System DiseasesLiver DiseasesDigestive System DiseasesLung DiseasesRespiratory Tract DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesSubcutaneous EmphysemaEmphysemaPathologic ProcessesPathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

GlycoproteinsGlycoconjugatesCarbohydratesSerpinsPeptidesAmino Acids, Peptides, and ProteinsAcute-Phase ProteinsBlood ProteinsProteinsAlpha-GlobulinsSerum GlobulinsGlobulins

Study Officials

  • Peter A Gottlieb, MD

    University of Colorado, Denver

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

March 17, 2011

First Posted

March 21, 2011

Study Start

October 1, 2010

Primary Completion

October 1, 2015

Study Completion

May 1, 2016

Last Updated

March 24, 2017

Record last verified: 2017-03

Locations

US(1)