Study of the Safety and Efficacy of Intravenous Alpha-1 Antitrypsin in Type 1 Diabetes Mellitus
Open Label, Proof of Concept, Phase I/II Study of the Safety, Tolerability and Efficacy of Intravenous Alpha-1 Antitrypsin (AAT) [Trade Name Glassia™] in Type 1 Diabetes Mellitus
1 other identifier
interventional
24
1 country
2
Brief Summary
Alpha-1 Antitrypsin (AAT), trade name (Glassia ®), is being explored in this phase I/II trial as a potential disease modifying agent in Type 1 Diabetes Mellitus (T1DM) based on its anti-inflammatory properties. AAT is an acute stress reactant protein that increases during inflammation. In T1DM inflammation serves a major role in disease progression.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_1
Started Jun 2011
2 active sites
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
First Submitted
Initial submission to the registry
February 24, 2011
CompletedFirst Posted
Study publicly available on registry
February 25, 2011
CompletedStudy Start
First participant enrolled
June 1, 2011
CompletedPrimary Completion
Last participant's last visit for primary outcome
November 1, 2012
CompletedStudy Completion
Last participant's last visit for all outcomes
November 1, 2012
CompletedJune 9, 2016
February 1, 2013
1.4 years
February 24, 2011
June 8, 2016
Conditions
Outcome Measures
Primary Outcomes (1)
Safety and Tolerability
Safety and Tolerability: assessed by vital signs(systolic/diastolic blood pressure and heart rate), physical examination, routine safety lab tests, AEs and SAEs.
Approximately 1 year
Secondary Outcomes (1)
Efficacy
Approximately 1 year
Study Arms (3)
Alpha-1 Antitrypsin 40mg (AAT, Glassia®)
EXPERIMENTALSubjects in this arm will receive a dose of 40 mg/kg throughout the study.
Alpha-1 Antitrypsin 60mg (AAT, Glassia®)
EXPERIMENTALSubjects in this arm will receive a dose of 60 mg/kg throughout the study.
Alpha-1 Antitrypsin 80mg (AAT, Glassia®)
EXPERIMENTALSubjects in this arm will receive a dose of 80 mg/kg throughout the study.
Interventions
Each study group will undergo 3 treatment periods:12 weeks, 8 weeks and 4 weeks.
Each study group will undergo 3 treatment periods:12 weeks, 8 weeks and 4 weeks.
Each study group will undergo 3 treatment periods:12 weeks, 8 weeks and 4 weeks.
Eligibility Criteria
You may qualify if:
- Subject (or parent/guardian) willing and able to sign an informed consent
- Age 10-25 (inclusive) years
- Diagnosed with T1DM within the previous 6 months
- Level of C-peptide ≥ 0.2 pmol/mL during MMTT(maximal level)
- Positive for at least one diabetes-related autoantibody(except for insulin autoantibody)
- No significant abnormalities in serum hematology,serum chemistry according to the Investigator's judgment, taking into considerations the potential effects of the diabetic illness.
- No significant abnormalities in urinalysis according to the Principal Investigator's judgment, taking into considerations the potential effects of the diabetic illness.
- No significant abnormalities in ECG per investigator judgment
- Negative for HBsAg and antibodies to HCV, HIV-1
- Non-pregnant, non-lactating female patients, whose screening pregnancy test is negative and who are using contraceptive methods deemed reliable by the investigator.
You may not qualify if:
- 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
- IgA deficient subjects
- Individuals with a history of severe immediate hypersensitivity reactions, including anaphylaxis, to plasma products
- Planned major surgery within the study period
- Clinically significant intercurrent illnesses, including(but not limited to): cardiac, hepatic, renal,neurological, hematological, neoplastic, immunological, skeletal or other) that in the opinion of the investigator, could interfere with the safety, compliance or other aspects of this study. Patients with well-controlled, chronic diseases could be possibly included after consultation with the treating physician and the sponsor.
- Pregnant or lactating women at entry to study and those who are unwilling to agree to continue to use acceptable methods of contraception throughout the study.
- Presence of psychiatric/ mental disorder or any other medical disorder which might impair the patient's ability to give informed consent or to comply with the requirements of the study protocol.
- Evidence of ongoing viral infection with HCV, HBV and/or HIV-1.
- Evidence of alcohol abuse or history of alcohol abuse or illegal and/or legally prescribed drugs.
- Participation in another interventional clinical trial within 30 days prior to baseline visit.
- Inability to attend scheduled clinic visits and/or comply with the study protocol.
- Any other factor that, in the opinion of the investigator, would prevent the patient form complying with the requirements of the protocol.
- 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).
- Current or prior (within the last 30 days prior to screening visit) use of metformin, sulfonylureas, glinides, thiazolidinediones, exenatide, liraglutide, DPP-IV inhibitors or amylin.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Kamada, Ltd.lead
Study Sites (2)
Institute for Endocrinology and Diabetes, National Center for Childhood Diabetes at Schneider Children's Medical Center of Israel
Petah Tikva, Israel, 49202, Israel
Assaf Haroffeh Medical Center
Ẕerifin, Israel, 70300, Israel
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
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Mariana Rachmiel, B.Med.Sc
Assaf Haroffeh Medical Center, Zerifin, Israel
- PRINCIPAL INVESTIGATOR
Yael Lebenthal, MD
Institute for Endocrinology & Diabetes, Schneider Children's Medical Center, Israel
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
February 24, 2011
First Posted
February 25, 2011
Study Start
June 1, 2011
Primary Completion
November 1, 2012
Study Completion
November 1, 2012
Last Updated
June 9, 2016
Record last verified: 2013-02
Data Sharing
- IPD Sharing
- Will not share