Steroid-Free and Long-Term Calcineurin-Free Trial in Islet Cell Transplantation
| Status: | Archived |
|---|---|
| Conditions: | Diabetes |
| Therapuetic Areas: | Endocrinology |
| Healthy: | No |
| Age Range: | Any |
| Updated: | 7/1/2011 |
The purposes of this study are:
1. To reverse hyperglycemia and insulin dependency in patients with type 1 diabetes
mellitus through islet transplantation utilizing steroid free, calcineurin-inhibitor
free immunosuppression.
2. To assess the long-term function of successful islet transplants in patients with type
1 diabetes mellitus utilizing islets that have undergone a period of culture.
3. To determine whether the natural history of the microvascular, macrovascular, and
neuropathic complications are altered following the successful transplantation of
islets.
STUDY DESIGN:
The initial proposal submitted to the JDRFI was to compare 3 different groups of patients
receiving islet cell transplants utilizing steroid-free, calcineurin-free protocols. The 3
groups were as follows:
1. Zenapax, Rapamycin & MMF
2. Campath, Rapamycin & MMF, and
3. Thymoglobulin, Rapamycin & MMF.
The grant was awarded in December 2003, however the recommendations were to focus on a
single group (group 3 or 4) in order to determine the relative efficacy and toxicity of a
new immunosuppressive drug combination. We elected to perform the group utilizing Campath,
since we have a similar protocol utilizing the same immunosuppressive regimen with the
addition of CD34+ enriched donor bone marrow cells (2000/0024). The results of this trial
utilizing a steroid-free/calcineurin-free protocol will be compared with the standard
"Edmonton Protocol" (2000/0196), which we are currently conducting (14 patients have been
transplanted). In addition, the results will be compared with those in 2000/0024.
Protocol 2000/0024 (utilizing the same immunosuppressive regimen; Campath, Rapamycin,
Tacrolimus-switched to MMF at 3 months) is being followed by a DSMB established at the NIH.
We propose to evaluate 12 patients with steroid free, long term calcineurin inhibitor free
immunosuppression regimens which can be directly compared to our historical group of
patients who underwent the Miami version of the Edmonton protocol (Islet Cell
Transplantation Alone in Patients with Type 1 Diabetes Mellitus: Steroid-Free
Immunosuppression - Protocol # 2000/196) and with the concurrent tolerogenic protocol (Islet
Cell Transplantation Alone and CD34+ Enriched Donor Bone Marrow Cell Infusion in Patients
with Type 1 Diabetes Mellitus; Steroid Free Regimen - Protocol # 2000/0024) which uses the
same immunosuppressive regimen combined with CD34+ stem cell enriched donor bone marrow
infusions.
The regimen will consist of Campath 1-H induction, maintenance immunosuppression with
sirolimus and tacrolimus for 3 months with subsequent introduction of mycophenolate mofetil
(MMF) and removal of tacrolimus completely and TNF-alpha inhibition (etanercept) in the
peri-transplant period.
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