Long Term Treatment Effect of the Safety, Tolerability and Efficacy of AAT in Type 1 Diabetes (AAT Extension)
At a previous study the investigators have assessed the safety and efficacy of treatment with AAT(Alpha 1 Antitrypsin)in newly diagnosed type 1 diabetes subjects aiming at beta cells preservation .
Since treatment with AAT is expected to be a chronic treatment; stopping treatment will probably result in eventual loss of the preserved beta-cell function. Indeed, other investigational drugs aiming at beta cells preservation have shown that patients who were initially treated and maintained their initial beta-cell function, required continuation of treatment or they lost the beta-cell function.
Therefore, in this extension study, patients who were previously treated with AAT and maintained clinically significant beta-cell function are offered a continuation of treatment, since they are likely to benefit from use of the medication.
The proposed study is aimed to assess the long term effect of AAT in subjects with type 1 diabetes mellitus: safety and tolerability of treatment, and effect on beta-cell function.
Subjects who have completed all visits of the 008 study will be offered to participate in the extension study.
The study will be consist off two main arms as following:
Arm 1: Subjects who maintained peak stimulated C-peptide secretion ≥ 0.2 nmol/L will continue treatment with AAT for up to 18 treatments according to the dosage group they were allocated to in the 008 study.
Subjects who have not maintained peak stimulated C-peptide secretion ≥ 0.2nmol/L and subjects with peak stimulated C -peptide secretion ≥ 0.2 nmol/L who are reluctant to receive additional study drug.
Clinical follow up for all subjects in both arms will be for 3 years
|Type 1 Diabetes Beta Cell Preservation||Drug: AAT( Alpha 1 Antitrypsin)||Phase 2|
|Study Design:||Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Prevention
|Official Title:||Open Label Study (Extension 001)to Evaluate Long Term Treatment Effect of the Safety, Tolerability and Efficacy of Intervenous ALPHA-1 ANTITRYSIN (AAT)Glasia™ in Type 1 Diabetes Mellitus (Extension to KAMADA AAt 008, PHASE I/II Study)|
- Safety and tolerability of AAT in terms of adverse events and serious adverse events [ Time Frame: At month 36 ]We will assess at each visit until final visit (month 36)the safety and tolerability of study drug in terms of adverse events and serious adverse events
- Safety and tolerability of the AAT in terms of laboratory values [ Time Frame: At month 36 ]We will assess at each visit until final visit (month 36)the safety and tolerability of study drug in terms of laboratory values
- Beta cell function-AUC (Area Under the Curve) of stimulated C-Peptide from stimulated MMTT (mixed meal tolerance test) [ Time Frame: at month 36 ]
- Percentage of patients that maintain stimulated peak C-peptide >=0.2 nmol/L [ Time Frame: at month 36 ]
- Percentage of patients that achieve glycemic target of HbA1c <=7.5% [ Time Frame: At month 36 ]
- Daily insulin dose adjusted to body weight [ Time Frame: At month 36 ]
|Study Start Date:||January 2013|
|Study Completion Date:||January 2017|
|Primary Completion Date:||January 2017 (Final data collection date for primary outcome measure)|
Experimental: AAT( Alpha 1 Antitrypsin)
Subjects who maintained peak stimulated C-peptide secretion ≥ 0.2nmol/L will continue treatment with AAT according to the dosage group they were allocated to at the previous study(40mg/kg or 60mg/kg or 80mg/kg), intravenously, once a week for 6 consecutive weeks, at 24-week intervals for a duration of ~54 weeks.
|Drug: AAT( Alpha 1 Antitrypsin)|
No Intervention: Follow up group
Subjects who have not maintained peak stimulated C-peptide secretion ≥ 0.2nmol/L or subjects with peak stimulated C -peptide secretion ≥ 0.2nmol/L who are reluctant to receive additional study drug, will be followed up only with no administration of investigational product
Please refer to this study by its ClinicalTrials.gov identifier: NCT01661192
|Schneider Children's Medical Center|
|Petah-Tikva, Israel, 49202|
|Assaf Haroffeh Medical Center|
|Principal Investigator:||Yael Lebenthal, MD||Rabin Medical Center|
|Principal Investigator:||Mariana Rachmiel, MD||Assaf Haroffe Medical Center|