Ignite Creation Date:
2024-05-05 @ 10:51 PM
Last Modification Date:
2024-10-26 @ 10:25 AM
Study NCT ID:
NCT01200017
Status:
NO_LONGER_AVAILABLE
Last Update Posted:
2021-05-27
First Post:
2010-09-09
Brief Title:
Expanded Access Protocol EAP Using the CliniMACS Device for Pediatric Haplocompatible Donor Stem Cell Transplant
Sponsor:
Christopher Dvorak
Organization:
University of California San Francisco
Study Overview
Official Title:
An Expanded Access Study of the Feasibility of Using the CliniMACS Device for CD34 Cell Selection and T Cell Depletion for Graft-versus-Host Disease Prophylaxis in Alternative Donor Stem Cell Transplant Recipients
Status:
NO_LONGER_AVAILABLE
Status Verified Date:
2021-05
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
This protocol provides expanded access to bone marrow transplants for children who lack a histocompatible tissue matched stem cell or bone marrow donor when an alternative donor unrelated donor or half-matched related donor is available to donate In this procedure some of the blood forming cells the stem cells are collected from the blood of a partially human leukocyte antigen HLA matched haploidentical donor and are transplanted into the patient the recipient after administration of a conditioning regimen A conditioning regimen consists of chemotherapy and sometimes radiation to the entire body total body irradiation or TBI which is meant to destroy the cancer cells and suppress the recipients immune system to allow the transplanted cells to take grow A major problem after a transplant from an alternative donor is increased risk of Graft-versus-Host Disease GVHD which occurs when donor T cells white blood cells that are involved with the bodys immune response attack other tissues or organs like the skin liver and intestines of the transplant recipient In this study stem cells that are obtained from a partially-matched donor will be highly purified using the investigational CliniMACS stem cell selection device in an effort to achieve specific T cell target values The primary aim of the study is to help improve overall survival with haploidentical stem cell transplant in a high risk patient population by limiting the complication of GVHD
Detailed Description:
Patients will be enrolled with alternative mismatchedhaplocompatible related donors or unrelated donors either for an initial transplant or as a rescue following rejection of a previous graft or relapse following a previous transplant For patients with mismatched related donors the majority of clinical experience has been with a T cell-depleted PBSC product Currently no FDA-approved method for T cell depletion exists Recent experience with the CliniMACS device has produced excellent results with a 70-75 survival in children many of whom were high risk patients
Patients that receive transplants from unrelated donors usually receive stem cells that are not T cell-depleted However this is associated with a high risk of GVHD The excellent results with mismatched related donor transplants justify expanding this approach to unrelated donor transplant recipients if the HLA mismatch is sufficiently great It is anticipated that the use of the CliniMACS device will result in a very low risk of GVHD without the need for post-transplant immunosuppression The outcomes in relatively small studies for children receiving unrelated donor transplants using the CliniMACS have been comparable to or better than those receiving T replete transplants with post-transplant immunosuppression
This protocol will allow the use of patient-specific conditioning regimens Some patients have contraindications to certain components of the conditioning regimen used for our ongoing study under BB-IND 8817 CC 01151 An example is a patient with pre-existing organ dysfunction that would be better served by the use of a reduced intensity conditioning regimen Another example is a patient for whom total body irradiation is contraindicated due to very young age or prior radiation therapy Finally patients who would be otherwise eligible for the predecessor study but who do not have an eligible related donor or a closely matched unrelated donor would be eligible for this study The target CD3 T cell dose that will be given will be 3 x 104kg The University of California San Francisco Protocol CC01151 uses a dose of 3 x 104kg The T cell dose in the graft is usually 1 x 104kg after processing and T cells are added to the product
Patients that receive transplants from unrelated donors usually receive stem cells that are not T cell-depleted However this is associated with a high risk of GVHD The excellent results with mismatched related donor transplants justify expanding this approach to unrelated donor transplant recipients if the HLA mismatch is sufficiently great It is anticipated that the use of the CliniMACS device will result in a very low risk of GVHD without the need for post-transplant immunosuppression The outcomes in relatively small studies for children receiving unrelated donor transplants using the CliniMACS have been comparable to or better than those receiving T replete transplants with post-transplant immunosuppression
This protocol will allow the use of patient-specific conditioning regimens Some patients have contraindications to certain components of the conditioning regimen used for our ongoing study under BB-IND 8817 CC 01151 An example is a patient with pre-existing organ dysfunction that would be better served by the use of a reduced intensity conditioning regimen Another example is a patient for whom total body irradiation is contraindicated due to very young age or prior radiation therapy Finally patients who would be otherwise eligible for the predecessor study but who do not have an eligible related donor or a closely matched unrelated donor would be eligible for this study The target CD3 T cell dose that will be given will be 3 x 104kg The University of California San Francisco Protocol CC01151 uses a dose of 3 x 104kg The T cell dose in the graft is usually 1 x 104kg after processing and T cells are added to the product
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| NCI-2020-02478 | REGISTRY | NCI Clinical Trials Reporting Program CTRP | None |