Health Economic Analysis of Islet Cell Transplantation for the Stabilization of the Severe Forms of Type 1 Diabetes
STABILOT
1 other identifier
interventional
42
2 countries
11
Brief Summary
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_3
Started Jul 2016
Longer than P75 for phase_3
11 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
March 4, 2016
CompletedStudy Start
First participant enrolled
July 7, 2016
CompletedFirst Posted
Study publicly available on registry
August 3, 2016
CompletedPrimary Completion
Last participant's last visit for primary outcome
May 1, 2023
CompletedStudy Completion
Last participant's last visit for all outcomes
December 1, 2023
CompletedJune 24, 2022
June 1, 2022
6.8 years
March 4, 2016
June 17, 2022
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Incremental cost- utility ratio at 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 Outcomes (7)
Cost-effectiveness ratio at 1 year
1 year
Assessment of individual medical benefit of quality of life
6 months and 1year
Assessment of individual medical benefit in terms of metabolic efficacy
6 months and 1 year
Assessment and comparison of individual medical benefit in terms of complications of islet cell transplantation between the two groups
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
1 year
- +2 more secondary outcomes
Study Arms (2)
Islet graft
EXPERIMENTALPatients who receive islet graft Intervention : Procedure/Surgery
Best medical care
ACTIVE COMPARATORPatients who continue their optimal medical treatment (insulin pump therapy coupled with real time continuous glucose monitoring) Intervention : insulin treatment
Interventions
Patients will be transplanted with pancreatic islet cells
Patients will continue their insulin treatment
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- Hemostatic disorders, pre-existing liver disease (PAL, Gamma-GT, ASAT-ALAT \>2N) or vesicular lithiasis.
- 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.
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (11)
University Hospital of Besançon
Besançon, France
university hospital of Clermont Ferrand
Clermont-Ferrand, France
Grenoble University Hospital
Grenoble, 38000, France
University hospital of Lille
Lille, 59000, France
University Hospital of Lyon
Lyon, 69000, France
University Hospital of Montpellier
Montpellier, 34000, France
University hospital of Nancy
Nancy, 54511, France
university hospital of Nantes
Nantes, 44093, France
APHP
Paris, 75010, France
University hospital of Strasbourg
Strasbourg, 67000, France
University Hospital of Geneva
Geneva, CH-1211, Switzerland
Related Publications (5)
Barton FB, Rickels MR, Alejandro R, Hering BJ, Wease S, Naziruddin B, Oberholzer J, Odorico JS, Garfinkel MR, Levy M, Pattou F, Berney T, Secchi A, Messinger S, Senior PA, Maffi P, Posselt A, Stock PG, Kaufman DB, Luo X, Kandeel F, Cagliero E, Turgeon NA, Witkowski P, Naji A, O'Connell PJ, Greenbaum C, Kudva YC, Brayman KL, Aull MJ, Larsen C, Kay TW, Fernandez LA, Vantyghem MC, Bellin M, Shapiro AM. Improvement in outcomes of clinical islet transplantation: 1999-2010. Diabetes Care. 2012 Jul;35(7):1436-45. doi: 10.2337/dc12-0063.
PMID: 22723582BACKGROUNDBeckwith J, Nyman JA, Flanagan B, Schrover R, Schuurman HJ. A health economic analysis of clinical islet transplantation. Clin Transplant. 2012 Jan-Feb;26(1):23-33. doi: 10.1111/j.1399-0012.2011.01411.x. Epub 2011 Feb 16.
PMID: 21323736BACKGROUNDBellin MD, Kandaswamy R, Parkey J, Zhang HJ, Liu B, Ihm SH, Ansite JD, Witson J, Bansal-Pakala P, Balamurugan AN, Papas KK, Sutherland DE, Moran A, Hering BJ. Prolonged insulin independence after islet allotransplants in recipients with type 1 diabetes. Am J Transplant. 2008 Nov;8(11):2463-70. doi: 10.1111/j.1600-6143.2008.02404.x. Epub 2008 Sep 19.
PMID: 18808408BACKGROUNDBadet L, Benhamou PY, Wojtusciszyn A, Baertschiger R, Milliat-Guittard L, Kessler L, Penfornis A, Thivolet C, Renard E, Bosco D, Morel P, Morelon E, Bayle F, Colin C, Berney T; GRAGIL Group. Expectations and strategies regarding islet transplantation: metabolic data from the GRAGIL 2 trial. Transplantation. 2007 Jul 15;84(1):89-96. doi: 10.1097/01.tp.0000268511.64428.d8.
PMID: 17627243BACKGROUNDLablanche S, David-Tchouda S, Margier J, Schir E, Wojtusciszyn A, Borot S, Kessler L, Morelon E, Thivolet C, Pattou F, Vantyghem MC, Berney T, Benhamou PY. Randomised, prospective, medico-economic nationwide French study of islet transplantation in patients with severely unstable type 1 diabetes: the STABILOT study protocol. BMJ Open. 2017 Feb 20;7(2):e013434. doi: 10.1136/bmjopen-2016-013434.
PMID: 28219959DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Pierre-Yves BENHAMOU, MD, PhD
University Hospital, Grenoble
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
March 4, 2016
First Posted
August 3, 2016
Study Start
July 7, 2016
Primary Completion
May 1, 2023
Study Completion
December 1, 2023
Last Updated
June 24, 2022
Record last verified: 2022-06
Data Sharing
- IPD Sharing
- Will not share