Clinical trial NCT02854696: Health Economic Analysis of Islet Cell Transplantation for the Stabilization of the Severe Forms of Type 1 Diabetes
Official title: Health Economic Analysis of Islet Cell Transplantation for Patients With Severe Forms of Brittle Type 1 Diabetes
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE3
Conditions: Type 1 Diabetes
Keywords: Islet cell Transplantation
cost-effectiveness analysis
Cost-utility analysis
Type 1 Diabetes
Lead sponsor: University Hospital, Grenoble
Sponsor class: OTHER
First posted: 2016-08-03
Enrollment: 42
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

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.

Eligibility criteria: 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

Primary outcomes:
measure: Incremental cost- utility ratio at 1 year
timeFrame: 1 year
Secondary outcomes:
measure: Cost-effectiveness ratio at 1 year
timeFrame: 1 year
measure: Assessment of individual medical benefit of quality of life
timeFrame: 6 months and 1year
measure: Assessment of individual medical benefit in terms of metabolic efficacy
timeFrame: 6 months and 1 year
measure: Assessment and comparison of individual medical benefit in terms of complications of islet cell transplantation between the two groups
timeFrame: 6 months and 1 year
measure: Assessment and comparison of clinical benefit for patients with brittle type 1 diabetes with impairment of vital prognosis before and after islet cell transplantation
timeFrame: 1 year
measure: Assessment and comparison of costs for patients with brittle type 1 diabetes with impairment of vital prognosis before and after islet cell transplantation
timeFrame: 1 year
measure: Assessment of total cost of islet cell transplantation
timeFrame: 1 year
Other outcomes:
Locations:
city: Besancon
country: France
facility: University Hospital of Besançon
city: Clermont Ferrand
country: France
facility: university hospital of Clermont Ferrand
city: Grenoble
country: France
facility: Grenoble University Hospital
city: Lille
country: France
facility: University hospital of Lille
city: Lyon
country: France
facility: University Hospital of Lyon
city: Montpellier
country: France
facility: University Hospital of Montpellier
city: Nancy
country: France
facility: University hospital of Nancy
city: Nantes
country: France
facility: university hospital of Nantes
city: Paris
country: France
facility: APHP
city: Strasbourg
country: France
facility: University hospital of Strasbourg
city: Geneva
country: Switzerland
facility: University Hospital of Geneva
Study officials:
affiliation: University Hospital, Grenoble
name: Pierre-Yves BENHAMOU, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References:
citation: 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.
citation: Beckwith 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.
citation: Bellin 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.
citation: Badet 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.
citation: Lablanche 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.