A Research Trial of Aralast NP in New Onset Diabetes (RETAIN) - Part II

• Status: Withdrawn
• Trial end date: 2014-08-01
• Target enrollment: 0
• Participant gender: All

Summary

Aralast NP (alpha-1 antitrypsin, AAT), an alpha-1 proteinase inhibitor (human), was the drug to be tested in this clinical trial. Aralast NP is an anti-inflammatory drug that affects the cells that are thought to be involved in the development of type 1 diabetes mellitus (T1DM, T1D). This study, known as RETAIN, was planned as a two-part trial to investigate the effect of Aralast NP on preserving beta cell function and to determine if the intervention would slow the progression of type 1 diabetes.

Part I of this trial (NCT 01183468) was an open-label, safety and dose level study consisting of two groups. After completion of Part I, including a satisfactory safety review, enrollment in Part II was to begin. Part II was designed as a two-arm, double-blind, placebo-controlled clinical trial, and participants were to be randomly assigned to either the Aralast NP treatment or placebo group.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: National Institute of Allergy and Infectious Diseases (NIAID)
• Collaborators: Immune Tolerance Network (ITN); Juvenile Diabetes Research Foundation
• Treatments: Alpha 1-Antitrypsin; Protease Inhibitors; Protein C Inhibitor

Criteria

Inclusion Criteria:

• Diagnosed with type 1 diabetes (T1D) within the past 100 days

• Positive for at least one diabetes-related autoantibody (anti-GAD; anti-insulin, if obtained within 10 days of the onset of insulin therapy; IA-2 antibody and/or ICA, or ZnT8)

• Peak stimulated C-peptide level greater than (>) 0.2 pmol/mL following a mixed meal tolerance test (MMTT)

Exclusion Criteria:

• Severe active disease (hepatitis, cardiac or pulmonary disease, hypertension, immunodeficiency)

• History of any bleeding or clotting factor deficiencies, or stroke

• History of vascular disease or significant vascular abnormalities

• Positive serology exposure to hepatitis B virus (HBV), hepatitis C virus (HCV), human immunodeficiency virus (HIV) or toxoplasmosis

• Clinically active infection with Epstein-Barr virus (EBV), cytomegalovirus (CMV), or tuberculosis (TB)

• Prior or current use of oral, inhaled or intranasal glucocorticoids, or any medication known to cause a significant, ongoing change in the course of T1D or immunologic status

• Prior treatment with alpha1-antitrypsin (AAT) or hypersensitivity to AAT or human plasma-derived products

• Current or prior (within the last 30 days) use of metformin, sulfonylureas, glinides, thiazolidinediones, exenatide, liraglutide, DPP-IV inhibitors or amylin

• Current use of any medication known to influence glucose tolerance (e.g., beta-blockers, angiotensin-converting enzyme inhibitors, interferons, quinidine anti-malarial drugs, lithium, niacin)

• Females who are pregnant or lactating, or are unwilling to defer pregnancy during study participation

• Immunoglobulin A (IgA) deficiency

• Uncontrolled hypertension

• Current life-threatening malignancy

• Any condition that in the investigator's opinion may compromise study participation or may confound the interpretation of the study results