Study of the Safety and Efficacy of Intravenous Alpha-1 Antitrypsin in Type 1 Diabetes Mellitus

• Status: Completed
• Trial end date: 2012-11-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Kamada, Ltd.
• Treatments: Alpha 1-Antitrypsin; Protein C Inhibitor

Criteria

Inclusion Criteria:

• 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.

Exclusion Criteria:

• 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.