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NCT Number: NCT01661192

Long Term Treatment Effect of the Safety, Tolerability and Efficacy of AAT in Type 1 Diabetes

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.

Arm 2:

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

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Key information

Conditions

Type 1 Diabetes Autoimmune Diseases Beta Cell Preservation Diabetes Mellitus Diabetes Mellitus, Type 1 Endocrine System Diseases Glucose Metabolism Disorders Immune System Diseases Metabolic Diseases Nutritional and Metabolic Diseases

Age range

10 year–25 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Schneider Children's Medical Center, Petah Tikva, Israel

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Subject (or parent/guardian) willing and able to sign an informed consent
  • Ability to comply with all study requirements.
  • A patient that participated in Study 008 and received all doses of study medication, per protocol.
  • Evidence of clinically significant residual beta-cell function demonstrated by MMTT peak stimulated C-peptide concentrations ≥ 0.20 nmol/L (Arm 1 only).
  • Age 10-25 (inclusive) years
  • If a female is of childbearing potential, the subject is not pregnant or lactating, and will use oral hormonal contraception or other equally effective contraceptive methods throughout the study.

Exclusion criteria

  • IgA (immunoglobulin A ) deficient subjects.
  • Individuals with a history of severe immediate hypersensitivity reactions, including anaphylaxis, to plasma products.
  • History of life threatening allergy, anaphylactic reaction, or systemic response to human plasma derived products.
  • The subject is receiving immunosuppressive or immunomodulating agents or cytotoxic therapy or any medication that in the opinion of the Investigator might interfere with the study.
  • 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.

Treatment and study plan

AAT( Alpha 1 Antitrypsin)

Drug

Primary outcomes

  1. 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

  2. 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

Secondary outcomes

  1. Beta cell function-AUC (Area Under the Curve) of stimulated C-Peptide from stimulated MMTT (mixed meal tolerance test)

    Time frame: at month 36

  2. Percentage of patients that maintain stimulated peak C-peptide >=0.2 nmol/L

    Time frame: at month 36

  3. Percentage of patients that achieve glycemic target of HbA1c <=7.5%

    Time frame: At month 36

  4. Daily insulin dose adjusted to body weight

    Time frame: At month 36

Sponsors and collaborators

Lead sponsor

Rabin Medical Center

Other

Collaborators

  • Kamada, Ltd.

Registry information

Official study 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)

Acronym: AAT Extension

Important dates

Study start
2013
Primary completion
2017
Study completion
2017
First posted
Aug 9, 2012
Registry last updated
Jan 11, 2017

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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