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Calcineurin Inhibitor (CNI)-Free Immunosuppressive Regimen in T1D Patients Receiving Islet Transplantation (ECIT-1)

9 april 2014 uppdaterad av: Piemonti Lorenzo, Ospedale San Raffaele

A Multi-step Trial Towards Single Donor Islet Transplantation in Type 1 Diabetic Patients, Using Calcineurin Inhibitor-free Immunosuppression

Our final objective is to develop an adoptive therapy with tolerogenic donor-specific Tr1 cells in T1D patients undergoing pancreatic islet transplantation (Tx). The achievement of this objective depends by the availability of an immunosuppressive treatment (IS) compatible with the survival, function, and expansion of the transferred Tr1 cells. For this purpose the investigators design a CNI-free single-group, phase 1-2 trial excluding the ATG or anti-CD25 induction therapy after the 1st islet infusion

Studieöversikt

Status

Avslutad

Betingelser

Detaljerad beskrivning

We designed the clinical trial as a single-arm, phase 1-2 trial conducted in two transplant centers (San Raffaele Scientific Institute, Milan, Italy; Cell Isolation and Transplantation Center, University of Geneva, Geneva, Switzerland) which used a common protocol for islet preparation, post-transplantation patient management and data collection. The trial is exploratory in nature and the target enrollment is 10 patients. The recruitment is competitive between the two centers and each patient is to receive at least 10,000 IE/kg. Up to three islet infusions are allowed per patients until insulin independence is reached, provided that partial islet function (i.e., fasting C-peptide ≥0.3 ng/mL) is maintained between infusions. We planned an individual follow-up of 3 years after the last islet infusion.

Patients with type 1 diabetes are eligible for this study. Major criteria for inclusion are: age 18-65 years; type 1 diabetes with onset <40 years of age; insulin treatment of at least 5 years at the time of enrollment; stimulated C-peptide in response to arginine <0.5 ng/ml; multiple (three or more) daily insulin injections or Continuous Subcutaneous Insulin Infusion; self-blood glucose monitoring ≥3 times/day; high glycemic instability and/or hypoglycemia unawareness; inability to consistently attain a glycated hemoglobin target of <7.5 % without severe hypoglycemia (defined as an hypoglycemic episode requiring the assistance by another person for its resolution) in the past 36 months despite medical management by a diabetes specialist. Major criteria for exclusion are: HbA1c >12%; BMI >30 kg/m2, or insulin requirement > 0.8 IU/kg/day; poorly controlled hypertension; untreated proliferative diabetic retinopathy; presence or history of macroalbuminuria (>300mg/g day) or estimated glomerular filtration rate <60 ml/min/1.73 m2 for females or <70 ml/min/1.73 m2 for males.

Studietyp

Interventionell

Inskrivning (Faktisk)

10

Fas

  • Fas 2
  • Fas 1

Kontakter och platser

Det här avsnittet innehåller kontaktuppgifter för dem som genomför studien och information om var denna studie genomförs.

Studieorter

      • Milan, Italien, 20132
        • IRCCS San Raffaele Scientific Institute
      • Geneve, Schweiz, 1211
        • Universite de Geneve

Deltagandekriterier

Forskare letar efter personer som passar en viss beskrivning, så kallade behörighetskriterier. Några exempel på dessa kriterier är en persons allmänna hälsotillstånd eller tidigare behandlingar.

Urvalskriterier

Åldrar som är berättigade till studier

18 år till 65 år (Vuxen, Äldre vuxen)

Tar emot friska volontärer

Nej

Kön som är behöriga för studier

Allt

Beskrivning

Inclusion Criteria:

  • Male and female patients aged 18-65yr
  • ability to provide written informed consent and comply with the study protocol procedures
  • clinical history of type 1 diabetes with onset <40yr of age, on insulin for at least 5yr at the time of enrollment
  • absent stimulated C-peptide (<0.5ng/ml) in response to arginine
  • multiple (three or more) daily insulin injections or insulin pump therapy
  • self blood glucose monitoring ≥3 times/day, supervised by a specialist physician
  • high glycemic instability and hypoglycemia unawareness
  • inability to consistently attain a HbA1c < 7.5 % target without experiencing severe hypoglycemia (assistance by another person) in the past 36 months despite appropriate medical management.

Exclusion Criteria:

  • HbA1c >12%
  • BMI >30 kg/m2, or insulin requirement of > 0.8 IU/kg/day;
  • poorly controlled hypertension;
  • untreated proliferative diabetic retinopathy;
  • presence or history of macroalbuminuria (>300mg/g day) or measured glomerular filtration rate <60 ml/min/1.73 m2 for females and <70 ml/min/1.73 m2 for males
  • for female participants: positive pregnancy test, presently breast-feeding, or unwilling to use effective contraceptive measures for the duration of the study and 3 months after discontinuation
  • for male participants: intent to procreate during the duration of the study or within 3 months after discontinuation or unwillingness to use effective measures of contraception;
  • any history of malignancy within the previous 5 years, except for completely resected squamous or basal cell carcinoma of the skin;

Studieplan

Det här avsnittet ger detaljer om studieplanen, inklusive hur studien är utformad och vad studien mäter.

Hur är studien utformad?

Designdetaljer

  • Primärt syfte: Behandling
  • Tilldelning: N/A
  • Interventionsmodell: Enskild gruppuppgift
  • Maskning: Ingen (Open Label)

Vapen och interventioner

Deltagargrupp / Arm
Intervention / Behandling
Experimentell: CNI-free single-group
Immunosuppression consisted of: (i) pre-Tx rapamycin treatment (0.1 mg/kg/day) for at least 30 days; (ii) induction therapy with ATG (1.5 mg/kg/day for 4 days starting at day -1) and a steroid bolus (methyl-prednisolone 500 mg, day -1) plus low dose steroids (prednisone, 10 mg/day) and interleukin-1 (IL-1) receptor antagonist (100 mg/day) for 2 weeks (with ATG and steroid bolus administered only prior to the 1st islet infusion; (iii) maintenance with rapamycin (0.1 mg/kg/day) plus mycophenolate mofetil (2 g/day).
Andra namn:
  • Kineret, Rapamune, Thymoglobulin, Myfortic

Vad mäter studien?

Primära resultatmått

Resultatmått
Åtgärdsbeskrivning
Tidsram
The Proportion of Insulin Free Patients 3 Years After the Last Islet Infusion
Tidsram: 3 year
Insulin independence is defined as no need for exogenous insulin, with adequate glycemic control [i.e., glycated hemoglobin <7% (normal range 3.5 - 6.0%), fasting glucose levels not exceeding 140 mg/dL (7.8 mmol/L) more than three times per week and 2-hour postprandial levels not exceeding 180 mg/dL (10 mmol/L) more than four times per week].
3 year

Sekundära resultatmått

Resultatmått
Åtgärdsbeskrivning
Tidsram
Insulin Independence With Adequate Glycemic Control Throughout Follow-up
Tidsram: up to 3 years
up to 3 years
Glycated Hemoglobin Levels Throughout Follow-up
Tidsram: up to 3 years
up to 3 years
Basal and Stimulated Blood C-peptide Levels in Response to Arginine Challenge Throughout Follow-up
Tidsram: up to 3 year
up to 3 year
the Reduction in Insulin Requirement Compared to Baseline
Tidsram: up to 3 years
up to 3 years
Severe Hypoglycemic Events Since Completion of Transplant
Tidsram: up to 3 years
up to 3 years
Any Adverse Event Throughout Follow-up
Tidsram: up to 3 years
Among study participants there were no reports of death, post-transplantation lymphoproliferative disease, cancer, or opportunistic infections. There was no evidence of cytomegalovirus disease, infection or serological activation (CMV early antigens negative during the whole follow-up), nor of Epstein-Barr clinical and serological reactivation (all patients were antibodies anti EBV positive before transplant, as per the inclusion criteria).
up to 3 years

Samarbetspartners och utredare

Det är här du hittar personer och organisationer som är involverade i denna studie.

Utredare

  • Huvudutredare: Lorenzo Piemonti, MD, Fondazione Centro San Raffaele del Monte Tabor
  • Huvudutredare: Thierry Berney, MD, Universite de Geneve
  • Studiestol: Antonio Secchi, MD, Fondazione Centro San Raffaele del Monte Tabor
  • Studierektor: Paola Maffi, MD, Cantro San Raffaele del Monte Tabor

Publikationer och användbara länkar

Den som ansvarar för att lägga in information om studien tillhandahåller frivilligt dessa publikationer. Dessa kan handla om allt som har med studien att göra.

Studieavstämningsdatum

Dessa datum spårar framstegen för inlämningar av studieposter och sammanfattande resultat till ClinicalTrials.gov. Studieposter och rapporterade resultat granskas av National Library of Medicine (NLM) för att säkerställa att de uppfyller specifika kvalitetskontrollstandarder innan de publiceras på den offentliga webbplatsen.

Studera stora datum

Studiestart

1 oktober 2006

Primärt slutförande (Faktisk)

1 juni 2009

Avslutad studie (Faktisk)

1 juni 2012

Studieregistreringsdatum

Först inskickad

29 april 2011

Först inskickad som uppfyllde QC-kriterierna

29 april 2011

Första postat (Uppskatta)

2 maj 2011

Uppdateringar av studier

Senaste uppdatering publicerad (Uppskatta)

9 maj 2014

Senaste inskickade uppdateringen som uppfyllde QC-kriterierna

9 april 2014

Senast verifierad

1 april 2014

Mer information

Denna information hämtades direkt från webbplatsen clinicaltrials.gov utan några ändringar. Om du har några önskemål om att ändra, ta bort eller uppdatera dina studieuppgifter, vänligen kontakta register@clinicaltrials.gov. Så snart en ändring har implementerats på clinicaltrials.gov, kommer denna att uppdateras automatiskt även på vår webbplats .

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