Long-term Function of Beta Cell Allografts in Non-uremic Type 1 Diabetic Patients

NCT ID: NCT00798785

Last Updated: 2013-12-30

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

UNKNOWN

Clinical Phase

PHASE1/PHASE2

Total Enrollment

50 participants

Study Classification

INTERVENTIONAL

Study Start Date

2006-10-31

Study Completion Date

2014-12-31

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

The present proof of concept study addresses the following specific aims:

The general objectives of this work are:

1. To increase and maintain the functional beta-cell mass after islet transplantation under a condition of low-dose tacrolimus
2. To co-investigate the potential of alternative sites for encapsulated beta-cells

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

1. Aim 1: To increase functional beta cell mass by adding rituximab at first implantation
2. Aim 2: To increase functional beta cell mass by adding basilixumab at second implantation
3. Aim 3: To assess the influence of down-tapering the tacrolimus dose during posttransplant years 2-5 on these data, on metabolic control, on the prevalence of hypoglycemia and on safety parameters.
4. Aim 4: To investigate the potential of the peritoneum and omentum as an alternative site for encapsulated beta-cells.
5. Aim 5: To investigate the potential of the brachioradial muscle as an alternative site for encapsulated beta-cells.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Diabetes Mellitus, Type 1

Keywords

Explore important study keywords that can help with search, categorization, and topic discovery.

diabetes mellitus type 1 pancreatic beta cell transplantation

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

group I ATG-MMF-TAC

Two clinical implants in the liver:

First implant: ATG-fresenium Maintained immunosuppression: MMF-TAC n=30

Group Type EXPERIMENTAL

ATG-MMF-TAC

Intervention Type DRUG

ATG-fresenium for max 6 consecutive days depending on CD3 count, starting the day before transplantation.

Tacrolimus levels for 2 years between 8-10 ng/ml.

At year 2: randomization:

group A: till 48 months: tacrolimus levels 8-10 ng/ml 48-60 months: tacrolimus levels 6-8 ng/ml group B: 24-36 months: 6-8 ng/ml 36-60 months: 4-6 ng/ml A subgroup of these patients will receive a sub clinical implant subcutaneous (total n=5) at the time of the first clinical implant in the liver.

group II ATG-Rituximab-MMF-TAC

Two clinical implants in the liver:

First Implant: ATG fresenium + Rituximab Maintained immunosuppression: MMF-TAC n=5

Group Type EXPERIMENTAL

ATG-Rituximab-MMF-TAC

Intervention Type DRUG

ATG-fresenium for max 6 consecutive days depending on CD3 count, starting the day before transplantation.

Rituximab: the day before transplantation, day 5; 12 and 19 after implantation. Tacrolimus levels for 2 years between 8-10 ng/ml.

At year 2: randomization:

group A: till 48 months: tacrolimus levels 8-10 ng/ml 48-60 months: tacrolimus levels 6-8 ng/ml group B: 24-36 months: 6-8 ng/ml 36-60 months: 4-6 ng/ml A subgroup of these patients will receive a sub clinical implant, subcutaneous at the time of the first clinical implant in the liver.

group III ATG-Basilixumab-MMF-TAC

Two clinical implants in the liver:

First implant: ATG-fresenium Second implant: basilixumab Maintained immunosuppression: MMF-TAC n=5

Group Type EXPERIMENTAL

ATG-basilixumab-MMF-TAC

Intervention Type DRUG

First transplantation:

ATG-fresenium for max 6 consecutive days depending on CD3 count, starting the day before transplantation.

Second transplantation:

Basilixumab:

the day before the second transplantation followed by 4days after transplantation.

Tacrolimus levels for 2 years between 8-10 ng/ml.

At year 2: randomization:

group A: till 48 months: tacrolimus levels 8-10 ng/ml 48-60 months: tacrolimus levels 6-8 ng/ml group B: 24-36 months: 6-8 ng/ml 36-60 months: 4-6 ng/ml A subgroup of these patients will receive a sub clinical implant, subcutaneous at the time of the first clinical implant in the liver.

group IV omentum

Two clinical implants: first in the omentum followed by a clinical implant in the liver:

First implant: ATG-fresenium Maintained immunosuppression: MMF-TAC n=10

Group Type EXPERIMENTAL

omentum

Intervention Type PROCEDURE

Two clinical implants: first in omentum followed by a clinical implant in the liver:

In a group of 10 patients, a clinical implant in the omentum will be implanted. If random C-peptide levels \>= 0.5 ng/ml are measured at 2 months post-transplantation, a second omental implant will be done. If no clinical relevant beta cell graft function is measured, two intraportal implants will be given as a compassionate use procedure. An interim analysis after 5 patients has to shown clinical relevant function at month 2 in 3 out of 5 patients before the subsequent 5 patients can be transplanted in the omentum.

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

ATG-MMF-TAC

ATG-fresenium for max 6 consecutive days depending on CD3 count, starting the day before transplantation.

Tacrolimus levels for 2 years between 8-10 ng/ml.

At year 2: randomization:

group A: till 48 months: tacrolimus levels 8-10 ng/ml 48-60 months: tacrolimus levels 6-8 ng/ml group B: 24-36 months: 6-8 ng/ml 36-60 months: 4-6 ng/ml A subgroup of these patients will receive a sub clinical implant subcutaneous (total n=5) at the time of the first clinical implant in the liver.

Intervention Type DRUG

ATG-Rituximab-MMF-TAC

ATG-fresenium for max 6 consecutive days depending on CD3 count, starting the day before transplantation.

Rituximab: the day before transplantation, day 5; 12 and 19 after implantation. Tacrolimus levels for 2 years between 8-10 ng/ml.

At year 2: randomization:

group A: till 48 months: tacrolimus levels 8-10 ng/ml 48-60 months: tacrolimus levels 6-8 ng/ml group B: 24-36 months: 6-8 ng/ml 36-60 months: 4-6 ng/ml A subgroup of these patients will receive a sub clinical implant, subcutaneous at the time of the first clinical implant in the liver.

Intervention Type DRUG

ATG-basilixumab-MMF-TAC

First transplantation:

ATG-fresenium for max 6 consecutive days depending on CD3 count, starting the day before transplantation.

Second transplantation:

Basilixumab:

the day before the second transplantation followed by 4days after transplantation.

Tacrolimus levels for 2 years between 8-10 ng/ml.

At year 2: randomization:

group A: till 48 months: tacrolimus levels 8-10 ng/ml 48-60 months: tacrolimus levels 6-8 ng/ml group B: 24-36 months: 6-8 ng/ml 36-60 months: 4-6 ng/ml A subgroup of these patients will receive a sub clinical implant, subcutaneous at the time of the first clinical implant in the liver.

Intervention Type DRUG

omentum

Two clinical implants: first in omentum followed by a clinical implant in the liver:

In a group of 10 patients, a clinical implant in the omentum will be implanted. If random C-peptide levels \>= 0.5 ng/ml are measured at 2 months post-transplantation, a second omental implant will be done. If no clinical relevant beta cell graft function is measured, two intraportal implants will be given as a compassionate use procedure. An interim analysis after 5 patients has to shown clinical relevant function at month 2 in 3 out of 5 patients before the subsequent 5 patients can be transplanted in the omentum.

Intervention Type PROCEDURE

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

* Age 18-65 years, male or female, Caucasian or not; only subjects \< 50 yrs will be allocated to the rituximab treatment arm
* Body weight \< 100 kg; patients with a bodyweight of \< 80kg, will receive priority
* Patients with a BMI ≤ 27 kg/m2 will receive priority
* Type 1 insulin-dependent diabetes
* C-peptide \< 0.07 nmol/l (\<0.2 µg/l) 6 min. after glucagon IV (1mg) (glycemia \> 180 mg/dl)
* Intensive insulin therapy for more than two years, patients with insulin pump during at least 2 months before inclusion will receive priority
* Patients should have at least one of the following chronic complications of diabetes:

* Plasma creatinine \<2 mg/dl and albuminuria 30-1000 mg/ 24hrs on 3 separate determinations (\>1 month) outside an episode of illness, despite intake of ACE inhibitors; mean systolic blood pressure should be under 130 mmHg and mean diastolic blood pressure under 85 mmHg, when measured at home with ambulatory BP monitoring
* Moderate or severe non-proliferative or proliferative retinopathy
* Hypoglycemic unawareness
* Cooperative and reliable patient giving informed consent by signature

Exclusion Criteria

* Smoker
* EBV antibody negativity
* HIV 1 \& 2 antibody positivity
* CMV IgM positivity
* Plasma creatinine ≥ 2 mg/dl and/or albuminuria ≥1000 mg/24 hrs
* History of thrombosis or pulmonary embolism
* History of malignancy, tuberculosis or chronic viral hepatitis
* History of any other serious illness which could be relevant for the protocol
* Presence of HLA antibodies
* Blood donation within one month prior to screening or during the study
* Symptoms and/or signs of infection, particularly (present or past) endocarditis, osteomyelitis, past tuberculosis with requirement for therapy
* Any history of hepatic or neoplastic disease
* Any history of renal disease (except diabetes)
* Abnormal liver function tests and /or NMR of liver
* Hemoglobinopathy
* History of any illness that, in the opinion of the investigator, might confound the results of the study or pose additional risks to the patient
* Pregnancy or use of inadequate contraception by female patients of childbearing potential
* Use of illicit drugs or overconsumption of alcohol (\> 3 beers/day) or history of drug or alcohol abuse
* Being legally incapacitated, having significant emotional problems at the time of the study, or having a history of psychiatric disorders
* Having received antidepressant medications during the last 6 months
* Having participated the last 12 months or participating in another clinical study
Minimum Eligible Age

18 Years

Maximum Eligible Age

65 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

AZ-VUB

OTHER

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Bart Keymeulen

UZ Brussels

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Bart Keymeulen, MD PhD

Role: PRINCIPAL_INVESTIGATOR

University Hospital Brussel

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Universitair Ziekenhuis Antwerpen

Antwerp, , Belgium

Site Status RECRUITING

University Hospital Brussels

Brussels, , Belgium

Site Status RECRUITING

Hopital Erasme

Brussels, , Belgium

Site Status RECRUITING

University Hospital Leuven

Leuven, , Belgium

Site Status RECRUITING

Countries

Review the countries where the study has at least one active or historical site.

Belgium

Central Contacts

Reach out to these primary contacts for questions about participation or study logistics.

Bart Keymeulen, MD PhD

Role: CONTACT

Phone: +32 2 477 61 11

Email: [email protected]

Bart Keymeulen, MD Phd

Role: CONTACT

Email: [email protected]

Facility Contacts

Find local site contact details for specific facilities participating in the trial.

Christophe De Block, MD,PhD

Role: primary

Bart Keymeulen, MD PhD

Role: primary

Laurent Crenier, MD

Role: primary

Pieter Gillard, MD PhD

Role: primary

Da Hae Lee, MD

Role: backup

References

Explore related publications, articles, or registry entries linked to this study.

Lee D, Keymeulen B, Hilbrands R, Ling Z, Van de Velde U, Jacobs-Tulleneers-Thevissen D, Maleux G, Lapauw B, Crenier L, De Block C, Mathieu C, Pipeleers D, Gillard P. Age and Early Graft Function Relate With Risk-Benefit Ratio of Allogenic Islet Transplantation Under Antithymocyte Globulin-Mycophenolate Mofetil-Tacrolimus Immune Suppression. Transplantation. 2017 Sep;101(9):2218-2227. doi: 10.1097/TP.0000000000001543.

Reference Type DERIVED
PMID: 27779572 (View on PubMed)

Keymeulen B, Gillard P, Mathieu C, Movahedi B, Maleux G, Delvaux G, Ysebaert D, Roep B, Vandemeulebroucke E, Marichal M, In 't Veld P, Bogdani M, Hendrieckx C, Gorus F, Ling Z, van Rood J, Pipeleers D. Correlation between beta cell mass and glycemic control in type 1 diabetic recipients of islet cell graft. Proc Natl Acad Sci U S A. 2006 Nov 14;103(46):17444-9. doi: 10.1073/pnas.0608141103. Epub 2006 Nov 7.

Reference Type BACKGROUND
PMID: 17090674 (View on PubMed)

Movahedi B, Keymeulen B, Lauwers MH, Goes E, Cools N, Delvaux G. Laparoscopic approach for human islet transplantation into a defined liver segment in type-1 diabetic patients. Transpl Int. 2003 Mar;16(3):186-90. doi: 10.1007/s00147-002-0517-7. Epub 2003 Feb 15.

Reference Type BACKGROUND
PMID: 12664214 (View on PubMed)

Maleux G, Gillard P, Keymeulen B, Pipeleers D, Ling Z, Heye S, Thijs M, Mathieu C, Marchal G. Feasibility, safety, and efficacy of percutaneous transhepatic injection of beta-cell grafts. J Vasc Interv Radiol. 2005 Dec;16(12):1693-7. doi: 10.1097/01.RVI.0000182506.88739.39.

Reference Type BACKGROUND
PMID: 16371537 (View on PubMed)

Lee D, Gillard P, Hilbrands R, Ling Z, Van de Velde U, Jacobs-Tulleneers-Thevissen D, Maleux G, Lapauw B, Crenier L, De Block C, Mathieu C, Pipeleers D, Keymeulen B. Use of Culture to Reach Metabolically Adequate Beta-cell Dose by Combining Donor Islet Cell Isolates for Transplantation in Type 1 Diabetes Patients. Transplantation. 2020 Oct;104(10):e295-e302. doi: 10.1097/TP.0000000000003321.

Reference Type DERIVED
PMID: 32433237 (View on PubMed)

Balke EM, Demeester S, Lee D, Gillard P, Hilbrands R, Van de Velde U, Van der Auwera BJ, Ling Z, Roep BO, Pipeleers DG, Keymeulen B, Gorus FK. SLC30A8 polymorphism and BMI complement HLA-A*24 as risk factors for poor graft function in islet allograft recipients. Diabetologia. 2018 Jul;61(7):1623-1632. doi: 10.1007/s00125-018-4609-z. Epub 2018 Apr 20.

Reference Type DERIVED
PMID: 29679103 (View on PubMed)

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

BK_TX_06

Identifier Type: -

Identifier Source: org_study_id