Phase II Study to Evaluate the Efficacy and Safety of Glassia® in Type-1 Diabetes

Phase II Study to Evaluate the Efficacy and Safety of Human, Alpha-1 Antitrypsin (AAT) [Glassia®] in the Treatment of New Onset Type-1 Diabetes

A Phase II, Double-Blind, Randomized, Placebo-Controlled, Multicenter, Study Evaluating the Efficacy and Safety of Human, Alpha-1 Antitrypsin (AAT) [Glassia®] in the Treatment of New Onset Type-1 Diabetes.

The study objectives are:

• To assess the efficacy of intravenous AAT in treatment of new onset Type 1 Diabetes
• To assess the safety and tolerability of intravenous AAT in new onset Type 1 Diabetes pediatric and young adult population.

Study Overview

• Status: Completed
• Conditions: New Onset Type-1 Diabetes
• Intervention / Treatment: Other: Placebo; Biological: Alpha-1 Antitrypsin

• Study Type: Interventional
• Enrollment (Actual): 70
• Phase: Phase 2

Contacts and Locations

Study Locations

• Israel
  • Beer Sheva, Israel
    • Soroka Medical Center
  • Haifa, Israel
    • Rambam Medical Center
  • Pethach Tikva, Israel
    • Schneider Children's Medical Center
  • Zerifin, Israel
    • Assaf Harofe Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 8 years to 25 years (Child, Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Main Inclusion Criteria:

• Subject (or parent/guardian) willing and able to sign an informed consent
• Age 8-25 (inclusive) years
• Recently diagnosed with T1DM
• Basal C-peptide ≥ 0.2 pmol/mL
• Positive for at least one diabetes-related autoantibody
• Ability and consent to comply with completion of patient diary
• No significant abnormalities in serum hematology, serum chemistry
• No significant abnormalities in urinalysis
• No significant abnormalities in ECG
• For women of child bearing potential, non-pregnant, non-lactating female patients

Main Exclusion Criteria:

• IgA deficient subjects
• Subjects who have received an active/ live virus vaccine within 4 weeks of the screening date
• Subjects who have received treatment with corticosteroid medication within 2 months prior to screening or any immunosuppressant or cytostatic agent within 6 months prior to screening
• Individuals with a history of severe immediate hypersensitivity reactions, including anaphylaxis, to plasma products
• Clinically significant intercurrent illnesses
• Pregnant or lactating women
• Current use of any medication known to influence glucose tolerance
• Current or prior (within the last 60 days prior to screening visit) use of metformin, sulfonylureas, glinides, thiazolidinediones, exenatide, liraglutide, DPP-IV inhibitors or amylin.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Double

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Placebo	Other: Placebo; Placebo
Experimental: Alpha1 Antitrypsin (Glassia); 60 mg/kg body weight	Biological: Alpha-1 Antitrypsin; Other Names: AAT; API; Humman Alpha-1 Antitrypsin; Alpha-1 Proteinase Inhibitor
Experimental: Alpha-1 Antitrypsin (Glassia); 120 mg/kg body weight	Biological: Alpha-1 Antitrypsin; Other Names: AAT; API; Humman Alpha-1 Antitrypsin; Alpha-1 Proteinase Inhibitor

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Beta cell function	Beta cell function (measured by C peptide)	12 months from baseline

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Glycemic control	Glycemic control expressed in HbA1c level	12 months from baseline
Beta cell function		12 months from baseline
Insulin dose		12 months from baseline
Hypoglycemic episodes		12 months from baseline
Safety parameters	Adverse events, vital signs, physical examination	12 months from baseline

Collaborators and Investigators

• Sponsor: Kamada, Ltd.

Study record dates

Study Major Dates

• Study Start: April 1, 2014
• Primary Completion (Actual): February 1, 2017
• Study Completion (Actual): February 1, 2017

Study Registration Dates

• First Submitted: November 27, 2013
• First Submitted That Met QC Criteria: December 4, 2013
• First Posted (Estimate): December 9, 2013

Study Record Updates

• Last Update Posted (Actual): October 11, 2018
• Last Update Submitted That Met QC Criteria: October 10, 2018
• Last Verified: October 1, 2018

More Information

Terms related to this study

• Keywords: Diabetes Mellitus; T1D; Type-1 Diabetes
• Additional Relevant MeSH Terms: Glucose Metabolism Disorders; Metabolic Diseases; Immune System Diseases; Autoimmune Diseases; Endocrine System Diseases; Diabetes Mellitus; Diabetes Mellitus, Type 1; Molecular Mechanisms of Pharmacological Action; Enzyme Inhibitors; Serine Proteinase Inhibitors; Trypsin Inhibitors; Protease Inhibitors; Alpha 1-Antitrypsin; Protein C Inhibitor
• Other Study ID Numbers: Kamada-AAT(IV)-011