NCT01319331已完成1 期
The Effects of Open Label Alpha-1 Antitrypsin on the Progression of Type 1 Diabetes in Subjects With Detectable C-peptide
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 12
- 试验地点
- 1
- 主要终点
- To assess participant safety & feasibility of study drug administration
研究概览
简要总结
The purpose of this study is to determine if the drug Alpha-1 Antitrypsin (AAT, Aralast NP) will preserve beta-cell function and help slow the progression of type 1 diabetes.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 6 Years 至 45 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of Type 1 Diabetes Mellitus based on ADA Criteria for fewer than 5 years but more than 100 days
- •6-45 years of age, inclusive. To assess safety, we will initially enroll 8 patients over the age of
- •Following the last infusion of the 8th patient, we will assess adverse events. As long as there are no stopping criteria met for these 8 patients we will decrease the age criteria down to 6 years old.
- •C-peptide increase during screening mixed meal tolerance test with a minimal stimulated value of ≥ 0.2 pmol/mL.
- •Positive for antibodies to insulin (if insulin autoantibody positive only, determination must be within two weeks of insulin initiation), GAD-65, IA-2 or ZnT8
- •Agree to intensive management of diabetes with an HgbA1c goal of < 7.0%
- •If female, (a) surgically sterile or (b) postmenopausal or (c) if of reproductive potential, willing to use medically acceptable birth control (e.g. female hormonal contraception, barrier methods or sterilization. ) until 3 months after completion of any treatment period
- •If male and of reproductive potential, willing to use medically acceptable birth control until 3 months after completion of any treatment period, unless the female partner is postmenopausal or surgically sterile
- •Serum creatinine ≤ 1.5 x upper limit of normal
- •AST < 2 times the upper limit of normal
- •Hematology:WBC > 3000 x 109/L; platelets > 100 x 109/L; hemoglobin > 10.0 g/dL.
排除标准
- •Unable or unwilling to comply with the requirements of the study protocol
- •Body Mass Index (BMI) > 30 kg/m2
- •Unstable blood sugar control defined as one or more episodes of severe hypoglycemia (defined as hypoglycemia that required the assistance of another person) within the last 30 days
- •Previous immunotherapy for T1D
- •Administration of an experimental agent for T1D at any time or use of an experimental device for T1D within 30 days of screening, unless approved by the study PI
- •History of any organ transplant, including islet cell transplant
- •Active autoimmune or immune deficiency disorder (e.g. sarcoidosis, rheumatoid arthritis)
- •Serum bilirubin > ULN, except those subjects whose abnormal values were attributed to any stable, benign condition (such as Gilbert's Syndrome) may be included
- •TSH outside the normal range at screening, except those subjects on stable doses of thyroid hormone replacement therapy may be included
- •Known HIV positivity, active hepatitis B or active hepatitis C infection
- •Anticipated pregnancy during active dosing or within 3 months after completion of active dosing phase
- •History of a malignant neoplasm within the previous 5 years (except in situ cervical cancer and curable non-melanoma skin malignancy)
- •Any social condition or medical condition that would, in the opinion of the investigator, prevent complete participation in the study or that would pose a significant hazard to the subjects' participation
- •History of active substance abuse within 12 months of screening
- •A psychiatric or medical disorder that would prevent giving informed consent
- •Individuals with a history of IgA deficiency
- •Individuals with a history of hypersensitivity to AAT
研究组 & 干预措施
Alpha-1 Antitrypsin (AAT, Aralast NP)
Experimental
Alpha-1 Antitrypsin (AAT, Aralast NP) as prescribed for study duration
干预措施: Alpha 1-Antitrypsin (AAT, Aralast NP) (Drug)
结局指标
主要结局
To assess participant safety & feasibility of study drug administration
时间窗: Study duration is 2 years
次要结局
- To assess AAT treatment on the maintenance of c-peptide production(Stimulated c-peptide at year one and two.)
- Assess the effects of AAT on glycemic variability and A1c.(Continuous Glucose Monitoring at one and two years.)
研究者
研究点 (1)
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