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Type 1 DiabetesPhase 3STABILOTNCT02854696

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

A Phase 3 study testing Islet graft and best medical care in people with Type 1 Diabetes, run by University Hospital, Grenoble at 11 sites in 2 countries.

Unknown status

Status not confirmed. The sponsor has not verified this record for over two years, so it may be out of date.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 18 to 65 years
Conditions the study is for
Type 1 Diabetes
What is being tested?
Islet graft and best medical care
Type of treatment
Surgery or procedure
Could I receive a placebo?
No, everyone receives an active treatment
How many people
42 participants
Where
11 sitesin 2 countries
Study dates
7 Jul 2016to Dec 2023
Phase
Phase 3
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 65 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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

Who cannot join

  • 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

Trial locations and contacts

11 sites in 2 countries.

11 sites in 2 countries

France10 sites
  • University Hospital of Besançon

    Besançon

  • university hospital of Clermont Ferrand

    Clermont-Ferrand

  • Grenoble University Hospital

    Grenoble, 38000

  • University hospital of Lille

    Lille, 59000

  • University Hospital of Lyon

    Lyon, 69000

  • University Hospital of Montpellier

    Montpellier, 34000

  • University hospital of Nancy

    Nancy, 54511

  • university hospital of Nantes

    Nantes, 44093

  • APHP

    Paris, 75010

  • University hospital of Strasbourg

    Strasbourg, 67000

Other trials in France

Switzerland1 site
  • University Hospital of Geneva

    Geneva, CH-1211

Other trials in Switzerland

About this study

Official title: 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.

Read the 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.

Conditions
Type 1 Diabetes
Being tested
Islet graft, best medical care
Keywords
Islet cell Transplantation, cost-effectiveness analysis, Cost-utility analysis, Type 1 Diabetes
Medical subject headings
Diabetes Mellitus, Type 1

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Islet graftProcedure or surgery

    Also called islet cell transplantation

    Patients will be transplanted with pancreatic islet cells

  • best medical careDrug

    Also called insulin treatment

    Patients will continue their insulin treatment

Study groups in NCT02854696
Study groupRoleWhat they receive
Islet graftExperimentalPatients who receive islet graft Intervention : Procedure/Surgery
Receives: Procedure: Islet graft
Best medical careActive comparatorPatients who continue their optimal medical treatment (insulin pump therapy coupled with real time continuous glucose monitoring) Intervention : insulin treatment
Receives: Drug: best medical care

What is being measured

The primary outcome is the main question the study is designed to answer.

  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.

    Measured: 1 year

Secondary outcomes (7)
  1. Cost-effectiveness ratio at 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

    Measured: 1 year

  2. Assessment of individual medical benefit of quality of life

    Evaluate with DQOL questionnaire

    Measured: 6 months and 1year

  3. Assessment of individual medical benefit in terms of metabolic efficacy

    measured from the following criteria: severe hypoglycemia, HbA1c, stimulated C-peptide, fasting glucose, insulin dose or oral diabetes, glycemic variability

    Measured: 6 months and 1 year

  4. Assessment and comparison of individual medical benefit in terms of complications of islet cell transplantation between the two groups

    measured from in insulin independence, hospitalizations

    Measured: 6 months and 1 year

  5. Assessment and comparison of clinical benefit for patients with brittle type 1 diabetes with impairment of vital prognosis before and after islet cell transplantation

    measured from DQOL, insulin independence, complications of islet cell transplantation

    Measured: 1 year

  6. Assessment and comparison of costs for patients with brittle type 1 diabetes with impairment of vital prognosis before and after islet cell transplantation

    measured from hospitalizations

    Measured: 1 year

  7. Assessment of total cost of islet cell transplantation

    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.

    Measured: 1 year

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted3 Aug 2016 (estimated)
  2. Study start7 Jul 2016
  3. Primary completionMay 2023 (estimated)
  4. Study completionDec 2023 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
42 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02854696
Sponsor's protocol ID
38RC14.453
Acronym
STABILOT
Status last verified
Jun 2022
First submitted
4 Mar 2016
Last updated
24 Jun 2022
Results posted
No
Data monitoring committee
Yes
Individual data sharing
No

Publications

  • 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.

    PubMed 22723582
  • 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.

    PubMed 21323736
  • 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.

    PubMed 18808408
  • 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.

    PubMed 17627243
  • 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.

    PubMed 28219959

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Islet graft and best medical care in people with Type 1 Diabetes, run by University Hospital, Grenoble at 11 sites in 2 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 7 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: status not confirmed, as of the sponsor's last update on 24 Jun 2022. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 11 sites in France, Switzerland. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Incremental cost- utility ratio at 1 year (time frame: 1 year). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02854696, which also lists 5 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Jun 2022.

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