- ICH GCP
- Registro degli studi clinici negli Stati Uniti
- Sperimentazione clinica NCT00446264
Islet Allotransplantation With Steroid Free Immunosuppression
Sequential Islet Transplantation With Steroid Free Immunosuppression for Type 1 Diabetes
The restoration of endogenous insulin secretion carries significant hopes for shifting the paradigm of life long exogenous insulin therapy in selected groups of patients with type 1 diabetes(T1D). After decades of frustrating clinical attempts, the Edmonton group set up in 2000 new standards for islet transplantation in patients with brittle T1D by achieving insulin independence in 80 percent of patients. These seminal results have however proved much more difficult to duplicate than initially expected.
This single center phase 2 clinical trial, duplicating the Edmonton protocol, is designed for confirming the consistent short term efficacy and safety of sequential islet allotransplantation with steroid free immunosuppression in patients with severe T1D.
Panoramica dello studio
Stato
Condizioni
Intervento / Trattamento
Descrizione dettagliata
The short term effectiveness of islet transplantation for alleviating hypoglycemia and controlling glucose homeostasis while limiting or even avoiding the nedd for exogenous insulin has been established despite protocol modifications in donor selection, islet preparation or recipient treatment, insulin independence with adequate metabolic control was however rarely prolonged beyond two years. The most frequently proposed explanations include chronic allogenic rejection, recurrence of autoimmunity and beta cell toxicity from administered immunosuppressive drugs.
Fourteen patients were enrolled in this single center phase 2 trial initiated in 2003. Eligible patients were males or females between 18 and 65 years of age, with type 1 diabeted documented for more than 5 years, arginine stimulated C-peptide lower than 0.2ng/ml, and hypoglycemia awareness or documented metabolic lability. Exclusion criteria included body mass index greater than 28Kg/m2, unstable arteriopathy or heart disease, active infection, previous transplantation, insulin daily requirements above 1.2 UI/kg, creatinin clearance below 60 ml/mn/m2 or urinary albumin excretion above 300 mg/day, malignancy, smoking, desire for pregnancy, psychiatric disorders and lack of compliance. The study primary efficacy endpoint was graft survival defined as insulin independence and HbA1c<6.5%. Secondary outcomes were graft function and metabolic control.
Tipo di studio
Iscrizione (Effettivo)
Fase
- Fase 2
Contatti e Sedi
Luoghi di studio
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-
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Lille, Francia, 59037
- University Hospital of Lille
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Criteri di partecipazione
Criteri di ammissibilità
Età idonea allo studio
Accetta volontari sani
Sessi ammissibili allo studio
Descrizione
Inclusion Criteria:
- type 1 diabetes documented for more than 5 years
- arginine stimulated C-peptide lower than 0.2 ng/mL
- one of the following:hypoglycemia unawareness OR metabolic lability documented by one or more severe hypoglycemias or two or more hospital admissions for ketoacidosis within the previous year.
Exclusion Criteria:
- body mass index greater than 28 kg/m2
- non stable arteriopathy or heart disease
- active infection
- previous transplantation
- hyperimmunization
- insulin daily needs above 1.2 U/Kg
- creatinine clearance below 60 ml/mn or urinary albumin excretion above 300 mg/d
- malignancy
- smoking
- desire for pregnancy
- psychiatric disorders
- lack of compliance
Piano di studio
Come è strutturato lo studio?
Dettagli di progettazione
- Scopo principale: Trattamento
- Assegnazione: N / A
- Modello interventistico: Assegnazione di gruppo singolo
- Mascheramento: Nessuno (etichetta aperta)
Armi e interventi
Gruppo di partecipanti / Arm |
Intervento / Trattamento |
|---|---|
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Sperimentale: islet transplantation
Each participant received up to three sequential fresh islet infusions within three months.
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Il trapianto di isole consisteva in un massimo di tre infusioni sequenziali di isole fresche entro tre mesi.
L'accesso alla vena porta è stato ottenuto in anestesia generale mediante cateterizzazione percutanea di un ramo portale periferico sotto guida ecografica o mediante cateterizzazione chirurgica di una piccola vena mesenterica.
Altri nomi:
Immunosuppressive consisted of Tacrolimus, target through level at 3-6 ng/ml, Sirolimus, target through level at 12-15 ng/ml for three months and at 7-10 ng/ml thereafter.
A five-dose induction course of Daclizumab 1mg/Kg was administered biweekly beginning one hour prior to the first infusion
Altri nomi:
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Cosa sta misurando lo studio?
Misure di risultato primarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
|---|---|---|
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Composite Criteria: Insulin Independence and Glycosylated Hemoglobin (HbA1c) Under 6.5% at One Year
Lasso di tempo: 1 year
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The percentage of insulin independents subjects with an HbA1c less than 6.5% at one year after last transplant
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1 year
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Misure di risultato secondarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
|---|---|---|
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Hypoglycemic Events
Lasso di tempo: day 0 to day 365
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Percentage of subjects free of severe hypoglycemic events from day 0 to day 365 with the day of transplant designated day 0
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day 0 to day 365
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Plasma C-peptide
Lasso di tempo: 1 year
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Level of plasma C-peptide at 1 year after the first transplant
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1 year
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HbA1c < 6.5%
Lasso di tempo: 1 year
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The percentage of subjects with HbA1c < 6.5% at 1 year after the first transplant
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1 year
|
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Percentage of Time Spent in Hypoglycemia (<0.70 mg/L)
Lasso di tempo: 1 year
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percentage of time spent in hypoglycemia derived from CGMS (Continuous Glucose Monitoring System)
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1 year
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Number of Adverse Events
Lasso di tempo: 1 year
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The number of adverse events related to the procedure and to the immunosuppression
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1 year
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Collaboratori e investigatori
Sponsor
Investigatori
- Investigatore principale: Francois Pattou, MD, University Hospital, Lille
- Investigatore principale: Marie-Christine Vantyghem, MD PhD, University Hospital, Lille
- Investigatore principale: Julie Kerr-Conte, PhD, Université de Lille 2
Pubblicazioni e link utili
Pubblicazioni generali
- Vantyghem MC, Chetboun M, Gmyr V, Jannin A, Espiard S, Le Mapihan K, Raverdy V, Delalleau N, Machuron F, Hubert T, Frimat M, Van Belle E, Hazzan M, Pigny P, Noel C, Caiazzo R, Kerr-Conte J, Pattou F; Members of the Spanish Back Pain Research Network Task Force for the Improvement of Inter-Disciplinary Management of Spinal Metastasis. Ten-Year Outcome of Islet Alone or Islet After Kidney Transplantation in Type 1 Diabetes: A Prospective Parallel-Arm Cohort Study. Diabetes Care. 2019 Nov;42(11):2042-2049. doi: 10.2337/dc19-0401. Erratum In: Diabetes Care. 2020 May;43(5):1164.
- Benomar K, Chetboun M, Espiard S, Jannin A, Le Mapihan K, Gmyr V, Caiazzo R, Torres F, Raverdy V, Bonner C, D'Herbomez M, Pigny P, Noel C, Kerr-Conte J, Pattou F, Vantyghem MC. Purity of islet preparations and 5-year metabolic outcome of allogenic islet transplantation. Am J Transplant. 2018 Apr;18(4):945-951. doi: 10.1111/ajt.14514. Epub 2017 Nov 11.
- Caiazzo R, Vantyghem MC, Raverdi V, Bonner C, Gmyr V, Defrance F, Leroy C, Sergent G, Hubert T, Ernst O, Noel C, Kerr-Conte J, Pattou F. Impact of Procedure-Related Complications on Long-term Islet Transplantation Outcome. Transplantation. 2015 May;99(5):979-84. doi: 10.1097/TP.0000000000000458.
- Vantyghem MC, Raverdy V, Balavoine AS, Defrance F, Caiazzo R, Arnalsteen L, Gmyr V, Hazzan M, Noel C, Kerr-Conte J, Pattou F. Continuous glucose monitoring after islet transplantation in type 1 diabetes: an excellent graft function (beta-score greater than 7) Is required to abrogate hyperglycemia, whereas a minimal function is necessary to suppress severe hypoglycemia (beta-score greater than 3). J Clin Endocrinol Metab. 2012 Nov;97(11):E2078-83. doi: 10.1210/jc.2012-2115. Epub 2012 Sep 20.
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Studia le date principali
Inizio studio
Completamento primario (Effettivo)
Completamento dello studio (Effettivo)
Date di iscrizione allo studio
Primo inviato
Primo inviato che soddisfa i criteri di controllo qualità
Primo Inserito (Stima)
Aggiornamenti dei record di studio
Ultimo aggiornamento pubblicato (Stima)
Ultimo aggiornamento inviato che soddisfa i criteri QC
Ultimo verificato
Maggiori informazioni
Termini relativi a questo studio
Parole chiave
Termini MeSH pertinenti aggiuntivi
- Disturbi del metabolismo del glucosio
- Malattie del sistema immunitario
- Malattie autoimmuni
- Malattie del sistema endocrino
- Diabete mellito
- Diabete mellito, tipo 1
- Ipoglicemia
- Malattie metaboliche
- Effetti fisiologici delle droghe
- Meccanismi molecolari dell'azione farmacologica
- Agenti antinfettivi
- Inibitori enzimatici
- Agenti antineoplastici
- Agenti immunosoppressivi
- Fattori immunologici
- Agenti antibatterici
- Antibiotici, Antineoplastici
- Agenti antimicotici
- Inibitori della calcineurina
- Tacrolimo
- Sirolimo
- Daclizumab
Altri numeri di identificazione dello studio
- CP 01/48
- PHRC (French Ministry of Health)
- AFSSAPS 030209 (Altro identificatore: AFSSAPS)
Queste informazioni sono state recuperate direttamente dal sito web clinicaltrials.gov senza alcuna modifica. In caso di richieste di modifica, rimozione o aggiornamento dei dettagli dello studio, contattare register@clinicaltrials.gov. Non appena verrà implementata una modifica su clinicaltrials.gov, questa verrà aggiornata automaticamente anche sul nostro sito web .