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Peritransplant Deoxyspergualin in Islet Transplantation in Type 1 Diabetes

Peritransplant Deoxyspergualin in Islet Transplantation in Type 1 Diabetes (CIT-03)

Type 1 diabetes is an autoimmune disease in which the insulin-producing pancreatic beta cells are destroyed, resulting in poor blood sugar control. The purpose of this study is to assess the safety and efficacy of deoxyspergualin (DSG), an immunosuppressant drug, on post-transplant islet function in people with type 1 diabetes who have not responded to intensive insulin therapy.

調査の概要

詳細な説明

Type 1 diabetes, also known as insulin-dependent diabetes, is a chronic disease in which the pancreas produces insufficient insulin to properly regulate blood sugar levels. Hypoglycemia, low blood sugar, and hyperglycemia, high blood sugar, can lead to significant complications in people with type 1 diabetes. Intensive insulin therapy has been shown to reduce the risk of chronic complications in people who achieve near normalization of glycemia. However, this therapy is labor intensive, difficult to implement, and associated with an increased frequency of severe hypoglycemia. Transplantation of islets from a healthy pancreas has been successful in restoring normal blood sugar levels and has led to initial insulin independence in people with type 1 diabetes. Rejection of these islets by the recipient's immune system, however, makes the treatment ineffective within a couple of years. Immunosuppressant drugs may be an effective way to maintain islet function post-transplant. The purpose of this study is to assess the safety and efficacy of an immunosuppressive regimen that includes DSG on post-transplant islet function in people with type 1 diabetes who have not responded to intensive insulin therapy. The study will also seek to improve the understanding of determinants of success and failure of islet transplants for type 1 diabetes.

Following screening procedures and 2 days prior to islet transplant, participants will be randomly assigned to either this Phase 2 trial or a multicenter Phase 3 trial. Participants in this study will receive up to three separate islet transplants. They will begin receiving antithymocyte globulin (ATG) and sirolimus 2 days prior to the first islet transplant. ATG will continue to be given until Day 2 post-transplant. Participants will continue taking sirolimus for the duration of the study. On the day of transplant, participants will receive DSG and etanercept, in addition to ATG and sirolimus. The DSG infusion will be administered over 3 hours and will immediately precede the islet transplant. Participants will continue receiving daily 3-hour infusions of DSG through Day 6 post-transplant. Etanercept will also be administered on Days 3, 7, and 10 post-transplant. Tacrolimus will be administered on Day 1 post-transplant and continued throughout the study.

Transplantations will involve an inpatient hospital stay and infusion of islets into a branch of the portal vein. Participants who do not achieve or maintain insulin independence by Day 75 post-transplant will be considered for a second islet transplant. Participants who remain dependent on insulin for longer than 1 month after the second transplant and who show partial graft function will be considered for a third transplant. Daclizumab or basiliximab will be used in place of ATG for the second and third transplants, if they are necessary. Participants who do not meet the criteria for a subsequent transplant and do not have a functioning graft will enter a reduced follow-up period.

There will be up to 21 study visits following each transplant. A physical exam, review of adverse events, blood collection, urine tests, and measures of immunosuppression levels will occur at most visits. An abdominal ultrasound and glomerular filtration rate testing will occur at some study visits. Participants will also self-test their glucose levels at least five times per day throughout the study. A 12-month follow-up period will take place after the participant's last transplant.

研究の種類

介入

入学 (実際)

14

段階

  • フェーズ2

連絡先と場所

このセクションには、調査を実施する担当者の連絡先の詳細と、この調査が実施されている場所に関する情報が記載されています。

研究場所

    • California
      • San Francisco、California、アメリカ、94143
        • University of Californinia, San Francisco
    • Illinois
      • Chicago、Illinois、アメリカ、60611
        • Northwestern University
    • Minnesota
      • Minneapolis、Minnesota、アメリカ、55455
        • University of Minnesota

参加基準

研究者は、適格基準と呼ばれる特定の説明に適合する人を探します。これらの基準のいくつかの例は、人の一般的な健康状態または以前の治療です。

適格基準

就学可能な年齢

18年~65年 (大人、高齢者)

健康ボランティアの受け入れ

いいえ

受講資格のある性別

全て

説明

Inclusion Criteria:

  • Mentally stable and able to comply with study procedures
  • Clinical history compatible with type 1 diabetes, with onset of disease at less than 40 years of age; insulin dependence for at least 5 years at study entry; AND sum of age and insulin-dependent diabetes duration of at least 28
  • Absent stimulated C-peptide (less than 0.3 ng/ml) 60 and 90 minutes post mixed-meal tolerance test
  • Involvement of intensive diabetes management, defined as:

    1. Self monitoring of glucose values no less than a mean of three times each day, averaged over each week
    2. Administration of three or more insulin injections each day or insulin pump therapy
    3. Under the direction of an endocrinologist, diabetologist, or diabetes specialist, with at least three clinical evaluations during the past 12 months prior to study enrollment
  • At least one episode of severe hypoglycemia, defined as an event with one of the following symptoms: memory loss; confusion; uncontrollable behavior; irrational behavior; unusual difficulty in awakening; suspected seizure; seizure; loss of consciousness; or visual symptoms, in which the participant was unable to treat him/herself and which was associated with either a blood glucose level less than 54 mg/dl or prompt recovery after an oral carbohydrate, intravenous glucose, or glucagon administration in the 12 months prior to study enrollment.
  • Reduced awareness of hypoglycemia. More information about this criterion, including the specific definition of hypoglycemia unawareness, is in the protocol.

Exclusion Criteria:

  • Body mass index (BMI) greater than 30 kg/m2 or weight less than or equal to 50 kg
  • Insulin requirement of more than 1.0 IU/kg/day or less than 15 U/day
  • HbA1c greater than 10%
  • Untreated proliferative diabetic retinopathy
  • Systolic blood pressure higher than 160 mmHg or diastolic blood pressure higher than 100 mmHg
  • Measured glomerular filtration rate using iohexol of less than 80 ml/min/1.73m2. More information about this criterion is in the protocol.
  • Presence or history of macroalbuminuria (greater than 300 mg/g creatinine)
  • Presence or history of panel-reactive anti-HLA antibody levels greater than background by flow cytometry. More information about this criterion is in the protocol.
  • Pregnant, breastfeeding, or unwilling to use effective contraception throughout the study and for 4 months after study completion
  • Active infection, including hepatitis B virus, hepatitis C virus, HIV, or tuberculosis. More information about this criterion is in the protocol.
  • Negative for Epstein-Barr virus by IgG determination
  • Invasive aspergillus, histoplasmosis, or coccidioidomycosis infection in the past year
  • History of malignancy except for completely resected squamous or basal cell carcinoma of the skin
  • Known active alcohol or substance abuse
  • Baseline Hgb below the lower limits of normal, lymphopenia, neutropenia, or thrombocytopenia
  • History of Factor V deficiency
  • Any coagulopathy or medical condition requiring long-term anticoagulant therapy after transplantation or individuals with an INR greater than 1.5
  • Severe coexisting cardiac disease, characterized by any one of the following conditions:

    1. Heart attack within the last 6 months
    2. Evidence of ischemia on functional heart exam within the year prior to study entry
    3. Left ventricular ejection fraction less than 30%
  • Persistent elevation of liver function tests at the time of study entry
  • Symptomatic cholecystolithiasis
  • Acute or chronic pancreatitis
  • Symptomatic peptic ulcer disease
  • Severe unremitting diarrhea, vomiting, or other gastrointestinal disorders that could interfere with the ability to absorb oral medications
  • Hyperlipidemia despite medical therapy, defined as fasting LDL cholesterol greater than 130 mg/dl (treated or untreated) and/or fasting triglycerides greater than 200 mg/dl
  • Currently receiving treatment for a medical condition that requires chronic use of systemic steroids except for the use of less than or equal to 5 mg prednisone daily, or an equivalent dose of hydrocortisone, for physiological replacement only
  • Treatment with any anti-diabetic medication other than insulin within 4 weeks prior to study entry
  • Use of any study medications within the past 4 weeks
  • Received a live attenuated vaccine within the past 2 months
  • Any medical condition that, in the opinion of the investigator, might interfere with safe participation in the trial
  • Treatment with any immunosuppressive regimen at the time of enrollment.
  • A previous islet transplant.
  • A previous pancreas transplant, unless the graft failed within the first week due to thrombosis, followed by pancreatectomy and the transplant occurred more than 6 months prior to enrollment

研究計画

このセクションでは、研究がどのように設計され、研究が何を測定しているかなど、研究計画の詳細を提供します。

研究はどのように設計されていますか?

デザインの詳細

  • 主な目的:処理
  • 割り当て:なし
  • 介入モデル:単一グループの割り当て
  • マスキング:なし(オープンラベル)

武器と介入

参加者グループ / アーム
介入・治療
実験的:Allogeneic Pancreatic Islet Cells
Participants in this study can receive up to three separate islet transplants. They will begin receiving antithymocyte globulin (ATG) and sirolimus 2 days prior to the first islet transplant. ATG will continue to be given until Day 2 post-transplant. Participants will continue taking sirolimus for the duration of the study. On the day of transplant, participants will receive DSG and etanercept, in addition to ATG and sirolimus. The DSG infusion will be administered over 3 hours and will immediately precede the islet transplant. Participants will continue receiving daily 3-hour infusions of DSG through Day 6 post-transplant. Etanercept will also be administered on Days 3, 7, and 10 post-transplant. Tacrolimus will be administered on Day 1 post-transplant and continued throughout the study.
維持免疫抑制療法
維持免疫抑制療法
Preparation of allogeneic pancreatic islet cells injected into the portal vein of the liver
An anti-inflammatory agent that blocks proinflammatory cytokine production and inhibits T-cells and B-cells and affects antigen presenting cells.
Immunosuppressive that selectively depletes activated T-cells and depletes resting T-cells in a dose-dependent manner.
Will replace antithymocyte globulin in all islet transplantations after the first one
Blocks TNF-alpha which is toxic to islet cells

この研究は何を測定していますか?

主要な結果の測定

結果測定
時間枠
Proportion of Insulin-independent Subjects
時間枠:75 days following the first islet transplant
75 days following the first islet transplant

二次結果の測定

結果測定
メジャーの説明
時間枠
Percent Reduction in Insulin Requirements
時間枠:75 days following the first and subsequent islet transplant
75 days following the first and subsequent islet transplant
Hemoglobin A1c (HbA1c)
時間枠:75 days following the first and subsequent islet transplant
75 days following the first and subsequent islet transplant
Mean Amplitude of Glycemic Excursions (MAGE)
時間枠:75 days following the first and subsequent islet transplant
75 days following the first and subsequent islet transplant
Glycemic Lability Index (LI)
時間枠:75 days following the first and subsequent islet transplant
75 days following the first and subsequent islet transplant
Ryan Hypoglycemia Severity Score (HYPO)
時間枠:75 days following the first and subsequent islet transplant
75 days following the first and subsequent islet transplant
Basal (fasting) and 90-minute Glucose and C-peptide Results
時間枠:75 days following the first and subsequent islet transplant
Derived from Mixed Meal Tolerance Test (MMTT)
75 days following the first and subsequent islet transplant
Beta-score
時間枠:75 days following the first and subsequent islet transplant
Assesses beta-cell function after islet transplantation
75 days following the first and subsequent islet transplant
C-peptide: Glucose Creatinine Ratio
時間枠:75 days following the first and subsequent islet transplant
75 days following the first and subsequent islet transplant
Acute Insulin Response to Glucose, Insulin Sensitivity, and Disposition Index
時間枠:75 days following the first and subsequent islet transplant
Derived from the insulin-modified frequently sampled intravenous glucose tolerance (FSIGT) test
75 days following the first and subsequent islet transplant
Glucose Variability and Hypoglycemia Duration
時間枠:75 days following the first and subsequent islet transplant
Derived from the continuous glucose monitoring system (CGMS)
75 days following the first and subsequent islet transplant
Quality of Life (QOL) Measure
時間枠:75 days following the first and subsequent islet transplant
75 days following the first and subsequent islet transplant
Incidence of Worsening Retinopathy
時間枠:365 days following the first islet transplant
365 days following the first islet transplant
Proportion of Insulin-independent Subjects
時間枠:365 days following the first and final islet transplant
365 days following the first and final islet transplant
Percent Reduction in Insulin Requirements
時間枠:365 days following the first and final islet transplant
365 days following the first and final islet transplant
Hemoglobin A1c (HbA1c)
時間枠:365 days following the first and final islet transplant
365 days following the first and final islet transplant
Mean Amplitude of Glycemic Excursions (MAGE)
時間枠:365 days following the first and final islet transplant
365 days following the first and final islet transplant
Glycemic Lability Index (LI)
時間枠:365 days following the first and final islet transplant
365 days following the first and final islet transplant
Clarke Score
時間枠:365 days following the first and final islet transplant
A hypoglycemia score
365 days following the first and final islet transplant
HYPO Score
時間枠:365 days following the first and final islet transplant
A hypoglycemia score
365 days following the first and final islet transplant
Basal (fasting) and 90-minute Glucose and C-peptide
時間枠:365 days following the first and final islet transplant
Derived from Mixed Meal Tolerance Test (MMTT)
365 days following the first and final islet transplant
Beta-score
時間枠:365 days following the first and final islet transplant
Assesses beta-cell function after islet transplantation
365 days following the first and final islet transplant
C-peptide: Glucose Creatinine Ratio
時間枠:365 days following the first and final islet transplant
365 days following the first and final islet transplant
Quality of life (QOL) Measure
時間枠:365 days following the first and final islet transplant
365 days following the first and final islet transplant
Proportion of Subjects Receiving a Second Islet Cell Transplant
時間枠:365 days following the first islet transplant
365 days following the first islet transplant
Proportion of Subjects Receiving a Third Islet Cell Transplant
時間枠:365 days following the first and final islet transplant
365 days following the first and final islet transplant
Incidence and Severity of Adverse Events Related to the Islet Cell Transplant Procedure
時間枠:75 days and 365 days following the first and final islet cell infusion
75 days and 365 days following the first and final islet cell infusion
Incidence and Severity of Adverse Events Related to the Immunosuppression Therapy
時間枠:75 days and 365 days following the first and final islet transplant
75 days and 365 days following the first and final islet transplant
Incidence of a Change in the Immunosuppression Drug Regimen
時間枠:75 days and 365 days following the first and final islet cell transplant
75 days and 365 days following the first and final islet cell transplant
Incidence of Immune Sensitization
時間枠:75 days and 365 days following the first and final islet transplant
Defined by detecting anti-HLA antibodies not present prior to transplantation
75 days and 365 days following the first and final islet transplant
Acute Insulin Response to Glucose, Insulin Sensitivity, and Disposition Index (DI)
時間枠:365 day following the first and final islet transplant
Derived from the insulin-modified frequently sampled intravenous glucose tolerance (FSIGT) test
365 day following the first and final islet transplant

協力者と研究者

ここでは、この調査に関係する人々や組織を見つけることができます。

捜査官

  • 主任研究者:Bernhard Hering, MD、University of Minnesota
  • 主任研究者:Andrew Posselt, MD, PhD、University of California, San Francisco
  • 主任研究者:Xunrong Luo, MD, PhD、Northwestern University

出版物と役立つリンク

研究に関する情報を入力する責任者は、自発的にこれらの出版物を提供します。これらは、研究に関連するあらゆるものに関するものである可能性があります。

研究記録日

これらの日付は、ClinicalTrials.gov への研究記録と要約結果の提出の進捗状況を追跡します。研究記録と報告された結果は、国立医学図書館 (NLM) によって審査され、公開 Web サイトに掲載される前に、特定の品質管理基準を満たしていることが確認されます。

主要日程の研究

研究開始

2006年10月1日

一次修了 (実際)

2011年9月1日

研究の完了 (実際)

2013年11月1日

試験登録日

最初に提出

2007年2月9日

QC基準を満たした最初の提出物

2007年2月9日

最初の投稿 (見積もり)

2007年2月13日

学習記録の更新

投稿された最後の更新 (見積もり)

2016年3月16日

QC基準を満たした最後の更新が送信されました

2016年2月17日

最終確認日

2016年2月1日

詳しくは

この情報は、Web サイト clinicaltrials.gov から変更なしで直接取得したものです。研究の詳細を変更、削除、または更新するリクエストがある場合は、register@clinicaltrials.gov。 までご連絡ください。 clinicaltrials.gov に変更が加えられるとすぐに、ウェブサイトでも自動的に更新されます。

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