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

Study Overview
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Brief Summary
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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.

Official Title
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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

Conditions
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New Onset Type-1 Diabetes

Intervention / Treatment
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* Biological: Alpha-1 Antitrypsin
* Other: Placebo


Participation Criteria
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Eligibility Criteria
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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.

Ages Eligible for Study
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Minimum Age: 8 Years
Maximum Age: 25 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Alpha1 Antitrypsin (Glassia)<br>60 mg/kg body weight | Biological: Alpha-1 Antitrypsin<br> <br> * Other names: AAT;|
| Experimental: Alpha-1 Antitrypsin (Glassia)<br>120 mg/kg body weight | Biological: Alpha-1 Antitrypsin<br> <br> * Other names: AAT;|
| Placebo Comparator: Placebo<br>Placebo | Other: Placebo<br>* Placebo<br>|

What is the study measuring?
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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 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Diabetes Mellitus, Type-1 Diabetes, T1D