Health Economic Analysis of Islet Cell Transplantation for the Stabilization of the Severe Forms of Type 1 Diabetes (STABILOT)

June 17, 2022 updated by: University Hospital, Grenoble

Health Economic Analysis of Islet Cell Transplantation for Patients With Severe Forms of Brittle Type 1 Diabetes

The main goal is to perform a cost-utility analysis to compare islet cell transplantation versus best medical treatment (defined as Sensor augmented pump therapy) for patients with brittle type1 diabetes.

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Detailed Description

The main goal is to perform a cost-utility analysis to compare islet cell transplantation versus best medical treatment (defined as Sensor augmented insulin pump therapy) for patients with brittle type1 diabetes.

Study Type

Interventional

Enrollment (Actual)

42

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • France
      • Besancon, France
        • University Hospital of Besancon
      • Clermont Ferrand, France
        • University Hospital of Clermont Ferrand
      • Grenoble, France, 38000
        • Grenoble University Hospital
      • Lille, France, 59000
        • University Hospital of Lille
      • Lyon, France, 69000
        • University Hospital of Lyon
      • Montpellier, France, 34000
        • University Hospital of Montpellier
      • Nancy, France, 54511
        • University Hospital Of Nancy
      • Nantes, France, 44093
        • University Hospital of Nantes
      • Paris, France, 75010
        • APHP
      • Strasbourg, France, 67000
        • University Hospital of Strasbourg
  • Switzerland
      • Geneva, Switzerland, CH-1211
        • University Hospital of Geneva

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 65 years (ADULT, OLDER_ADULT)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria :

  • Suffering from diabetes since more than 5 years
  • Patient with brittle type 1 diabetes despite an optimized insulin treatment and educational training will be included. A patient will be considered as experiencing a brittle type 1 diabetes if at least two criteria are present among: persistence of severe hypoglycemia, occurrence of ketoacidosis events without obvious etiology, diagnosis of unaware hypoglycemic episodes < 3 mmol/l based on CGM or self-monitoring blood glucose data, a mean blood glucose standard deviation>50%, MAGE index (Mean amplitude of glucose excursions)>60 mg/dl, LBGI index (low blood glucose index)>5, Clarke score≥4 or HYPOSCORE>800.
  • Insulin needs < 0,85 U/kg/day
  • HbA1c < 12% ;
  • No residual insulin secretion (plasmatic basal and stimulated C-peptide < 0.3 ng/ml)
  • Social Security membership or benefit from Social Security
  • Patients who signed the consent form

Exclusion Criteria :

Exclusion criteria related to islet infusion:

  • Hemostatic disorders, pre-existing liver disease (PAL, Gamma-GT, ASAT-ALAT >2N) or vesicular lithiasis.

Exclusion criteria related to diabetic complications:

  • Evolutive proliferative retinopathy, evolutive nephropathy (Glomerular filtration rate <30 ml/min/1.73m2 and/or proteinuria >0.5g/day), evolutive cardiopathy or obliterative arteriopathy with trophic cutaneous lesions.

Exclusion criteria related to immunosuppressant use:

  • Hemoglobin < 110mg/dL in women and < 120 mg/dL in men, leuconeutropenia, thrombopenia, systemic infection including chronic hepatitis B, C and VIH, neoplasia disease and hypertension>160/100 mmHg.
  • Corticoid treatment (except for patient that benefited from a kidney graft with maintenance steroid therapy)
  • Presence of anti-HLA antibody directed against the donor
  • Positive B or T cells crossmatch

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: RANDOMIZED
  • Interventional Model: PARALLEL
  • Masking: NONE

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
EXPERIMENTAL: Islet graft
Patients who receive islet graft Intervention : Procedure/Surgery
Procedure: Islet graft
Patients will be transplanted with pancreatic islet cells
Other Names:
  • islet cell transplantation
ACTIVE_COMPARATOR: Best medical care
Patients who continue their optimal medical treatment (insulin pump therapy coupled with real time continuous glucose monitoring) Intervention : insulin treatment
Drug: best medical care
Patients will continue their insulin treatment
Other Names:
  • insulin treatment

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Incremental cost- utility ratio at 1 year
Time Frame: 1 year
The primary endpoint will be the incremental cost-effectiveness ratio at one year for islet transplantation versus Best Medical Treatment of brittle type 1 diabetes.The effectiveness will be expressed as quality adjusted life years (QALYs) in a cost-utility analysis. QALYs are a composite measure of outcomes where utilities for health states (on 0-1 scale, where 0 corresponds to death and 1 to full health) act as qualitative weights to combine quantity and quality of life. The number of QALYs in each group will be assessed with the EuroQol 5 Dimensions questionnaire (EQ5D). The EQ-5D measures health status in terms of mobility, self-care, usual activities, pain/discomfort and anxiety/depression.
1 year

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Cost-effectiveness ratio at 1 year
Time Frame: 1 year
Assessment of the cost-effectiveness ratio at 1 year between islet cell transplantation versus best medical treatment (SAP therapy) for patients with brittle type 1 diabetes without impairment of vital prognosis. Two criteria of effectiveness will be used : the life years gained and the number of hypoglycemia
1 year
Assessment of individual medical benefit of quality of life
Time Frame: 6 months and 1year
Evaluate with DQOL questionnaire
6 months and 1year
Assessment of individual medical benefit in terms of metabolic efficacy
Time Frame: 6 months and 1 year
measured from the following criteria: severe hypoglycemia, HbA1c, stimulated C-peptide, fasting glucose, insulin dose or oral diabetes, glycemic variability
6 months and 1 year
Assessment and comparison of individual medical benefit in terms of complications of islet cell transplantation between the two groups
Time Frame: 6 months and 1 year
measured from in insulin independence, hospitalizations
6 months and 1 year
Assessment and comparison of clinical benefit for patients with brittle type 1 diabetes with impairment of vital prognosis before and after islet cell transplantation
Time Frame: 1 year
measured from DQOL, insulin independence, complications of islet cell transplantation
1 year
Assessment and comparison of costs for patients with brittle type 1 diabetes with impairment of vital prognosis before and after islet cell transplantation
Time Frame: 1 year
measured from hospitalizations
1 year
Assessment of total cost of islet cell transplantation
Time Frame: 1 year
Assessment of total cost of islet cell transplantation for patients with type 1 diabetes without impairment of vital prognosis, from pre-transplant period until 1 year after the last injection. Two perspectives will be used: French health care system and hospital.
1 year

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

University Hospital, Grenoble

Investigators

  • Principal Investigator: Pierre-Yves BENHAMOU, MD, PhD, University Hospital, Grenoble

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (ACTUAL)

July 7, 2016

Primary Completion (ANTICIPATED)

May 1, 2023

Study Completion (ANTICIPATED)

December 1, 2023

Study Registration Dates

First Submitted

March 4, 2016

First Submitted That Met QC Criteria

July 29, 2016

First Posted (ESTIMATE)

August 3, 2016

Study Record Updates

Last Update Posted (ACTUAL)

June 24, 2022

Last Update Submitted That Met QC Criteria

June 17, 2022

Last Verified

June 1, 2022

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 38RC14.453

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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