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Study of Human Plasma-Derived Alpha1-Proteinase Inhibitor in Subjects With New-Onset Type 1 Diabetes Mellitus

2018年8月6日 更新者：Grifols Therapeutics LLC

A Multicenter, Randomized, Partial-Blinded, Placebo-Controlled Study to Evaluate the Safety and Efficacy of a Human Plasma-Derived Alpha1-Proteinase Inhibitor in Subjects With New-Onset Type 1 Diabetes Mellitus

This is a multicenter, randomized, partial-blinded, five-arm, placebo-controlled study of human plasma-derived alpha1-proteinase inhibitor (alpha1-PI) in children (ages 6-11 years old) and teens/adults (ages 12-35 years old) with new onset Type 1 Diabetes Mellitus (T1DM). Currently enrolling ages 12-35 only. Once 25 patients are randomized and data is reviewed enrollment will be opened to the child cohort. The purpose of this study is to evaluate the safety and efficacy of four dosing regimens of human plasma-derived alpha1-PI in T1DM.

研究概览

地位

终止

条件

干预/治疗

研究类型

介入性

注册 (实际的)

76

阶段

  • 阶段2

联系人和位置

本节提供了进行研究的人员的详细联系信息，以及有关进行该研究的地点的信息。

学习地点

  • 美国
    • Arizona
      • Tucson、Arizona、美国、85724
        • University of Arizona
    • California
      • Los Angeles、California、美国、90015
        • Clinica Medica San Miguel
      • Orange、California、美国、92868
        • Diabetes Associates Medical Group
      • San Diego、California、美国、92123
        • Rady Children's Hospital San Diego
      • Tarzana、California、美国、91356
        • Metabolic Institute of America
      • Ventura、California、美国、93003
        • Ronald H Chochinov MD
    • Connecticut
      • New Haven、Connecticut、美国、06511
        • Yale New Haven Hospital
    • Delaware
      • Newark、Delaware、美国、19713
        • Christiana Care Health Services
    • Florida
      • Jacksonville、Florida、美国、32258
        • Solutions Through Advanced Research Inc.
      • Miami、Florida、美国、33133
        • CCM Clinical Research
      • Miami、Florida、美国、33136
        • Advanced Pharma CR LLC
    • Georgia
      • Atlanta、Georgia、美国、30309
        • Atlanta Diabetes Associates
    • Idaho
      • Idaho Falls、Idaho、美国、83404
        • Rocky Mountain Diabetes and Osteoporosis Center
    • Illinois
      • Chicago、Illinois、美国、60612
        • Cook County Hospital
    • Indiana
      • Indianapolis、Indiana、美国、46202
        • Methodist Research Institute
    • Iowa
      • Iowa City、Iowa、美国、52242
        • University of Iowa Hospitals and Clinics
    • Kentucky
      • Louisville、Kentucky、美国、40202
        • University of Louisville
    • Maryland
      • Baltimore、Maryland、美国、21229
        • Barry J. Reiner MD, LLC.
    • Massachusetts
      • Worcester、Massachusetts、美国、01655
        • UMass Memorial Medical Center
    • Michigan
      • Detroit、Michigan、美国、48201
        • Wayne State University
    • Minnesota
      • Saint Paul、Minnesota、美国、55102
        • Children's Hospitals and Clinics of Minnesota
    • New Jersey
      • Morristown、New Jersey、美国、07962
        • Morristown Medical Center
    • New Mexico
      • Albuquerque、New Mexico、美国、87131-0001
        • University of New Mexico, Health Sciences Center
    • New York
      • Buffalo、New York、美国、14222
        • Women and Children's Hospital
    • North Carolina
      • Raleigh、North Carolina、美国、27610
        • WakeMed Children's Hospital
    • Ohio
      • Columbus、Ohio、美国、43210
        • Ohio State University
      • Columbus、Ohio、美国、43201
        • Endocrinology Associates Inc
    • Oklahoma
      • Oklahoma City、Oklahoma、美国、73112
        • Pediatric Endocrinology, Genetics & Metabolism
    • Pennsylvania
      • Philadelphia、Pennsylvania、美国、19106
        • Children's Hospital of Philadelphia
      • Pittsburgh、Pennsylvania、美国、15224
        • Children's Hospital of Pittsburgh
    • South Dakota
      • Rapid City、South Dakota、美国、57701
        • Rapid City Regional Hospital/Health Clinical Research
    • Texas
      • Dallas、Texas、美国、75390
        • University of Texas Southwestern Medical Center
      • Dallas、Texas、美国、75231
        • Research Institute of Dallas
      • San Antonio、Texas、美国、78229
        • University of Texas Health Science Center
      • San Antonio、Texas、美国、78154
        • Northeast Clinical Research of San Antonio LLC
      • San Antonio、Texas、美国、78232
        • Consano Clinical Research

参与标准

研究人员寻找符合特定描述的人，称为资格标准。这些标准的一些例子是一个人的一般健康状况或先前的治疗。

资格标准

适合学习的年龄

6年 至 35年 (孩子、成人)

接受健康志愿者

不

有资格学习的性别

全部

描述

Inclusion Criteria:

  • Diagnosis of T1DM according to the ADA criteria.

  • Current use of injected insulin therapy and one positive result on testing for any of the following antibodies (If not currently on insulin therapy, must have positive result for at least two of the below antibodies):

    • Anti-islet-cell antibodies (islet cell antigen 512, insulinoma associated protein 2),
    • Anti-glutamic acid decarboxylase antibodies, or
    • Anti-insulin antibodies (unless received insulin therapy for > 7 days).
  • Body Mass Index (BMI) ≤ 28 kg/m2 for adults (≥ 20 years of age) OR ≤ 90th percentile in accordance with the Centers for Disease Control BMI assessment for children and teens (2 through 19 years old).

Exclusion Criteria:

  • History of or current diabetic retinopathy, neuropathy, or nephropathy.
  • Known thrombophilia or history of thrombosis.
  • Malignant disease (including malignant melanoma; however, other forms of skin cancer are allowed) within five years of randomization.
  • Active Hepatitis A virus, Hepatitis B virus, Hepatitis C virus, or Human Immunodeficiency Virus infection.
  • History of anaphylaxis or severe systemic response to any plasma-derived alpha1-PI preparation or other blood product(s).
  • Known selective or severe Immunoglobulin A deficiency.
  • Elevated liver enzymes (aspartate transaminase, alanine aminotransferase, and alkaline phosphatase) equal to or greater than 2.5 times the upper limit of normal.
  • Therapy with exenatide or any other agents that stimulate pancreatic β cell regeneration or insulin secretion, or any antidiabetic agents (oral or parenteral) other than insulin within one month prior to screening.
  • Use of omega-3 fatty acid supplements, including fish oil, within seven days prior to screening.
  • Current or planned therapy with inhaled insulin, if it becomes available.
  • Chronic use of systemic steroids, with the exception of inhaled steroids, above a stable dose equivalent to 5 mg/day prednisone (e.g., 10 mg every 2 days) within 4 weeks prior to randomization. It is recommended to maintain the same dose throughout the study. (Note: Subjects with autoimmune conditions (i.e., asthma) necessitating treatment with systemic short-term corticosteroids and administered a rapid taper are eligible per protocol with the caveat that the tapering is complete or decreased to the minimum requirement (i.e., 5 mg/day) at least 1 week prior to the Baseline visit (when randomization occurs) to ensure the subject is stable. For longer term steroid usage, please consult the Grifols Medical Monitor before considering the subject for study participation.)
  • Treatment with immunosuppressants or cytostatic agents within 6 months of randomization.

学习计划

本节提供研究计划的详细信息，包括研究的设计方式和研究的衡量标准。

研究是如何设计的？

设计细节

  • 主要用途：治疗
  • 分配：随机化
  • 介入模型：并行分配
  • 屏蔽：四人间

武器和干预

参与者组/臂
干预/治疗
实验性的：Alpha1-PI 180 mg/kg/wk, 26 weeks
180 mg/kg weekly infusions of Alpha1-PI for 26 weeks.
生物：180 mg/kg Alpha1-PI
其他名称：
  • 前列腺素C
  • Alpha-1 MP
  • Alpha1-antitrypsin
  • Alpha1-Proteinase Inhibitor (human), Modified Process
实验性的：90 mg/kg/wk Alpha1-PI, 26 weeks
90 mg/kg weekly infusions of Alpha1-PI for 26 weeks.
生物：90 mg/kg Alpha1-PI
其他名称：
  • 前列腺素C
  • Alpha-1 MP
  • Alpha1-antitrypsin
  • Alpha1-Proteinase Inhibitor (human), Modified Process
安慰剂比较：Placebo, 26 weeks
Weekly infusions of placebo for 26 weeks.
生物：安慰剂
实验性的：180 mg/kg/wk Alpha1-PI, 13 weeks
180 mg/kg weekly infusions of Alpha1-PI for 13 weeks.
生物：180 mg/kg Alpha1-PI
其他名称：
  • 前列腺素C
  • Alpha-1 MP
  • Alpha1-antitrypsin
  • Alpha1-Proteinase Inhibitor (human), Modified Process
实验性的：90 mg/kg/wk Alpha1-PI, 13 weeks
90 mg/kg weekly infusions of Alpha1-PI for 13 weeks
生物：90 mg/kg Alpha1-PI
其他名称：
  • 前列腺素C
  • Alpha-1 MP
  • Alpha1-antitrypsin
  • Alpha1-Proteinase Inhibitor (human), Modified Process
安慰剂比较：Placebo, 13 weeks
Weekly infusions of placebo for 13 weeks.
生物：安慰剂

研究衡量的是什么？

主要结果指标

结果测量
措施说明
大体时间
Change From Baseline in Mixed Meal Tolerance Test (MMTMT) Stimulated C-peptide 2 Hour Area Under the Concentration-time Curve (AUC)
大体时间：Baseline, Week 52 (pre-high protein drink and 15, 30, 60, 90, 120 minutes post-drink)
C-peptide concentration during MMTT with high protein energy drink. "Dose" for time frame refers to intake of high protein energy drink.
Baseline, Week 52 (pre-high protein drink and 15, 30, 60, 90, 120 minutes post-drink)

次要结果测量

结果测量
措施说明
大体时间
Change From Baseline for MMTT Stimulated C-peptide 2h AUC
大体时间：Baseline, Weeks 14, 27, 39, 69, 87, and 104 (pre-high protein drink and 15, 30, 60, 90, 120 minutes post-drink)
Baseline, Weeks 14, 27, 39, 69, 87, and 104 (pre-high protein drink and 15, 30, 60, 90, 120 minutes post-drink)
Change From Baseline for HbA1c Levels
大体时间：Baseline, Weeks 14, 27, 39, 52, 69, 87, and 104
Baseline, Weeks 14, 27, 39, 52, 69, 87, and 104
Number of Subjects With Overall Severe Hypoglycemic Episodes
大体时间：104 weeks
Severe hypoglycemia defined according the ADA Workgroup on Hypoglycemia definition, as follows: An event requiring assistance of another person to actively administer carbohydrate, glucagons, or other resuscitative actions.
104 weeks
Change From Baseline for Mean Daily Insulin Dose Requirements
大体时间：Baseline, Weeks 2, 4, 14, 27, 39, 52, 69, 87, and 104
Baseline, Weeks 2, 4, 14, 27, 39, 52, 69, 87, and 104
Change From Baseline for Mean Daily Glucose Levels Prior to Meals and Bedtime
大体时间：Baseline, Weeks 2, 4, 14, 27, 39, 52, 69, 87, and 104
For each visit, the mean daily glucose levels were calculated over the previous 3-7 days prior to the study visit from blood glucose levels recorded daily prior to meals and bedtime.
Baseline, Weeks 2, 4, 14, 27, 39, 52, 69, 87, and 104

合作者和调查者

在这里您可以找到参与这项研究的人员和组织。

赞助

Grifols Therapeutics LLC

出版物和有用的链接

负责输入研究信息的人员自愿提供这些出版物。这些可能与研究有关。

一般刊物

研究记录日期

这些日期跟踪向 ClinicalTrials.gov 提交研究记录和摘要结果的进度。研究记录和报告的结果由国家医学图书馆 (NLM) 审查，以确保它们在发布到公共网站之前符合特定的质量控制标准。

研究主要日期

学习开始

2014年3月1日

初级完成 (实际的)

2017年1月1日

研究完成 (实际的)

2017年6月1日

研究注册日期

首次提交

2014年3月11日

首先提交符合 QC 标准的

2014年3月18日

首次发布 (估计)

2014年3月20日

研究记录更新

最后更新发布 (实际的)

2018年9月5日

上次提交的符合 QC 标准的更新

2018年8月6日

最后验证

2018年8月1日

更多信息

与本研究相关的术语

关键字

其他相关的 MeSH 术语

其他研究编号

  • GTI1302

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