Anti-Thymocyte Globulin, Cyclosporine, and RAD in Islet Transplantation

NCT ID: NCT00286624

Last Updated: 2008-05-08

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

6 participants

Study Classification

INTERVENTIONAL

Study Start Date

2003-03-31

Study Completion Date

2006-08-31

Brief Summary

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This study was designed to test the safety and efficacy of up to 3 pancreatic alloislet transplants in type 1 diabetic patients with hypoglycemia unawareness. 6 subjects were transplanted under this protocol using anti-thymocyte globulin induction immunosuppression and everolimus with cyclosporine maintenance immunosuppression.

Detailed Description

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This is a Phase I/II study designed to assess the safety and efficacy of sequential islet allotransplantation for the reestablishment of stable glycemic control in type 1 diabetic recipients. A total of 6 patients with type 1 diabetes have received up to three transplants of islets from different donor pancreases.

Potential candidates for islet allotransplantation included patients age 18 and older with type 1 diabetes. Induction immunotherapy for the first transplant consisted of anti-thymocyte globulin; basiliximab was used for any subsequent transplants. Peritransplant anti-inflammatory treatment with etanercept was given for each islet transplant. Maintenance immunosuppression is with cyclosporine and RAD. It is felt that those patients in whom metabolic lability/instability, reduced awareness of hypoglycemia, poor glycemic control, and progressive secondary complications persist despite continued and intensive efforts made in close cooperation with their diabetes care team are particularly likely to have a favorable benefit/risk ratio.

Adverse events, irrespective of their presumed relationship to the transplantation of allogeneic islets and/or protocol-regulated treatment products (concomitant therapy), are being monitored and recorded throughout the first year after the final islet transplant.

The proportion of single and sequential donor islet allograft recipients with full (insulin independence and HbA1c \<7%) and partial (insulin dependence, basal or arginine-stimulated C-peptide levels of greater or equal to 0.5 ng/mL and HbA1c \<7%) islet graft function at one year after the final islet transplant will be assessed. The impact of islet transplantation on quality of life will also be assessed.

The predictive value for posttransplant insulin independence of factors such as insulin resistance before and at intervals after pancreatectomy, cellular composition of the transplant, number of beta cells transplanted; and viability and insulin secretory response of isolated islets are being assessed.

Conditions

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Type 1 Diabetes Mellitus Hypoglycemia

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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1

Allogeneic islet transplantation with anti-thymocyte globulin induction and cyclosporine and RAD maintenance immunosuppression

Group Type EXPERIMENTAL

Allogeneic Islets of Langerhans

Intervention Type BIOLOGICAL

Up to 3 intraportal infusions of cadaveric pancreatic islets of Langerhans. First infusion to contain at least 5,000 islet equivalents/kg body weight. Subsequent infusions to contain at least 3,000 islet equivalents/kg body weight.

Everolimus

Intervention Type DRUG

Loading dose of 3 mg PO on day -2 relative to transplant, followed at least 12 hours later by dose of 1.5 mg PO BID. The daily dose will be adjusted according to the whole blood 12-hr trough to target 3-15 ng/ml for the first 3 months and 3-12 ng/ml thereafter.

anti-thymocyte globulin

Intervention Type DRUG

A total of 6 mg/kg IV over 12 hours on days -2, -1, 0, +1, and +2. The dose will be 0.5 mg/kg on day -2, 1.0 mg/kg on day -1, and 1.5 mg/kg on days 0, +1, and +2.

Cyclosporine

Intervention Type DRUG

Cyclosporine started on day +1 relative to the first islet transplant. Initial dose of 3 mg/kg/day administered in 2 divided doses; then adjusted to maintain target levels of 400 (350-500) ng/mL for the first three months following islet transplant and 300 (200-350) ng/mL thereafter.

Interventions

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Allogeneic Islets of Langerhans

Up to 3 intraportal infusions of cadaveric pancreatic islets of Langerhans. First infusion to contain at least 5,000 islet equivalents/kg body weight. Subsequent infusions to contain at least 3,000 islet equivalents/kg body weight.

Intervention Type BIOLOGICAL

Everolimus

Loading dose of 3 mg PO on day -2 relative to transplant, followed at least 12 hours later by dose of 1.5 mg PO BID. The daily dose will be adjusted according to the whole blood 12-hr trough to target 3-15 ng/ml for the first 3 months and 3-12 ng/ml thereafter.

Intervention Type DRUG

anti-thymocyte globulin

A total of 6 mg/kg IV over 12 hours on days -2, -1, 0, +1, and +2. The dose will be 0.5 mg/kg on day -2, 1.0 mg/kg on day -1, and 1.5 mg/kg on days 0, +1, and +2.

Intervention Type DRUG

Cyclosporine

Cyclosporine started on day +1 relative to the first islet transplant. Initial dose of 3 mg/kg/day administered in 2 divided doses; then adjusted to maintain target levels of 400 (350-500) ng/mL for the first three months following islet transplant and 300 (200-350) ng/mL thereafter.

Intervention Type DRUG

Other Intervention Names

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Islets RAD ATG Thymogloblin Neoral

Eligibility Criteria

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Inclusion Criteria

* Primary islet allotransplant
* Patients with type 1 diabetes mellitus under intensive insulin management
* Age 18 or older
* Ability to give written informed consent

Exclusion Criteria

* Age less than 18 years.
* BMI \>26 kg/m2.
* Insulin requirement of \> 50 IU per day.
* Positive C-peptide response to intravenous arginine stimulation.
* Untreated proliferative retinopathy.
* Creatinine clearance \< 60 ml/min/1.73 m2 for females and 70 ml/min/1.73 m2 for males.
* Serum creatinine \>1.3 mg/dl for females, \>1.5 mg/dl for males.
* Previous pancreas or islet transplant.
* Presence of history of panel-reactive anti-HLA antibodies \>10%.
* Positive pregnancy test, or presently breast-feeding, or failure to follow effective contraceptive measures.
* Active infection including hepatitis C, hepatitis B, HIV, or TB (or under treatment for suspected TB).
* Negative screen for Epstein-Barr Virus (EBV).
* Invasive aspergillus infection within year prior to study entry.
* History of malignancy.
* Active alcohol or substance abuse
* History of non-adherence to prescribed regimens.
* Psychiatric disorder making the subject not a suitable candidate for transplantation.
* Inability to provide informed consent.
* Baseline Hgb \< 11.7 g/dl in females, or \< 13 g/dl in males; lymphopenia (\<1,000/microL), or leukopenia (\<3,000 total leukocytes/microL), or an absolute CD4+ count \<500/microL., or platelets \<150,000/microL
* History of coagulopathy or medical condition requiring long-term anticoagulant therapy after transplantation or patient with INR \>1.5.
* Severe co-existing cardiac disease.
* Baseline liver function tests outside of normal range or history of significant liver disease.
* Active peptic ulcer disease.
* Severe unremitting diarrhea or other gastrointestinal disorders potentially interfering with the ability to absorb oral medications.
* Presence of severe allergy requiring acute or chronic treatment, or hypersensitivity to drugs similar to RAD (e.g., macrolides).
* Known hypersensitivity to rabbit proteins.
* Hyperlipidemia (fasting LDL cholesterol \> 130 mg/dl, treated or untreated; and/or fasting triglycerides \> 200 mg/dl).
* Addison's disease.
* Under treatment requiring chronic use of systemic steroids.
* Any medical condition that, in the opinion of the investigator, will interfere with the safe completion of the trial.
Minimum Eligible Age

18 Years

Maximum Eligible Age

65 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Juvenile Diabetes Research Foundation

OTHER

Sponsor Role collaborator

National Institutes of Health (NIH)

NIH

Sponsor Role collaborator

Novartis

INDUSTRY

Sponsor Role collaborator

University of Minnesota

OTHER

Sponsor Role lead

Responsible Party

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University of Minnesota

Principal Investigators

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Bernhard J. Hering, M.D.

Role: PRINCIPAL_INVESTIGATOR

University of Minnesota

Locations

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University of Minnesota

Minneapolis, Minnesota, United States

Site Status

Countries

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United States

Related Links

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http://www.diabetesinstitute.org

Diabetes Institute for Immunology and Transplantation - U of M

Other Identifiers

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0207M29841

Identifier Type: -

Identifier Source: org_study_id