Ignite Creation Date:
2024-05-05 @ 11:48 AM
Last Modification Date:
2024-10-26 @ 9:14 AM
Study NCT ID:
NCT00145587
Status:
TERMINATED
Last Update Posted:
2017-05-30
First Post:
2005-09-01
Brief Title:
Stem Cell Transplantation for Children Affected With Osteopetrosis
Sponsor:
St Jude Childrens Research Hospital
Organization:
St Jude Childrens Research Hospital
Study Overview
Official Title:
Allogeneic Hematopoietic Stem Cell Transplantation for Children Affected With Malignant Osteopetrosis A Pilot Study
Status:
TERMINATED
Status Verified Date:
2011-01
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Due to the principal investigator having left the institution
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
Malignant infantile osteopetrosis MIOP is a rare fatal genetic disorder that is characterized by the bones inability to regulate remodeling The only curative therapy is hematopoietic stem cell transplantation Stem cells provided from an HLA identical matched sibling donor is the standard of care but not feasible for the majority of patients In addition due to the potentially rapid progression of this disease the time to identify a suitable HLA matched unrelated donor is not optimal Therefore this study is designed to test the hypothesis that children with osteopetrosis can properly engraft hematopoietic stem cells that are donated from a partially matched parental donor or haploidentical stem cell donor that are processed on the investigational device CliniMACS selection system
Detailed Description:
The primary objective of this trial will be answered strictly by those patients enrolled who receive a haploidentical stem cell donor graft
Patients with a matched sibling donor will be offered participation in this clinical trial and will receive a standard myeloablative conditioning regimen followed by the infusion of an unmanipulated bone marrow graft However data from these transplant recipients will be reported in a descriptive manner only
Secondary Objectives in this trial include the following
To describe the outcome of children with MIOP who receive hematopoietic stem cells from a matched sibling donor or a haploidentical donor utilizing a uniform approach one year from transplant
To estimate the fraction of children with MIOP who have a genetic defect correlating to the osteopetrosis phenotype
To assess carrier-state of the genetic mutation in parents with an affected child
To assess carrier-state of the genetic mutation in siblings of affected children
To estimate the effect of age at the time of hematopoietic stem cell transplantation on the overall outcome of children with MIOP
To describe the kinetics of select cytokine expression before and after transplantation
Patients with a matched sibling donor will be offered participation in this clinical trial and will receive a standard myeloablative conditioning regimen followed by the infusion of an unmanipulated bone marrow graft However data from these transplant recipients will be reported in a descriptive manner only
Secondary Objectives in this trial include the following
To describe the outcome of children with MIOP who receive hematopoietic stem cells from a matched sibling donor or a haploidentical donor utilizing a uniform approach one year from transplant
To estimate the fraction of children with MIOP who have a genetic defect correlating to the osteopetrosis phenotype
To assess carrier-state of the genetic mutation in parents with an affected child
To assess carrier-state of the genetic mutation in siblings of affected children
To estimate the effect of age at the time of hematopoietic stem cell transplantation on the overall outcome of children with MIOP
To describe the kinetics of select cytokine expression before and after transplantation
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