Ignite Creation Date:
2024-05-06 @ 3:34 PM
Last Modification Date:
2024-10-26 @ 1:51 PM
Study NCT ID:
NCT04671212
Status:
RECRUITING
Last Update Posted:
2024-04-08
First Post:
2020-12-02
Brief Title:
Discarded Bone Marrow for Hematology Research
Sponsor:
St Jude Childrens Research Hospital
Organization:
St Jude Childrens Research Hospital
Study Overview
Official Title:
Discarded Bone Marrow for Hematology Research
Status:
RECRUITING
Status Verified Date:
2024-04
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:
The primary objective of this study is to establish a mechanism to obtain discarded bone marrow-containing bone samples from hemoglobinopathy as well as non-hemoglobinopathy individuals The processing of samples will help to understand how best to manipulate HSPCs from hemoglobinopathy patients with gene therapy and gene technologies in the laboratory environment It will also allow us to establish a reservoir of samples that can be studied in the future to assess cellular function and fitness for transplant
Secondary objectives
To develop gene transfer and gene editing strategies as potentially curative therapy for hemoglobinopathies eg sickle cell disease SCD and β-thalassemia
To develop a drug treatment strategy which elevates the expression of fetal hemoglobin to a potentially curative level for hemoglobinopathies
To examine the biology of bone marrow cells isolated from patients with hemoglobinopathies
Secondary objectives
To develop gene transfer and gene editing strategies as potentially curative therapy for hemoglobinopathies eg sickle cell disease SCD and β-thalassemia
To develop a drug treatment strategy which elevates the expression of fetal hemoglobin to a potentially curative level for hemoglobinopathies
To examine the biology of bone marrow cells isolated from patients with hemoglobinopathies
Detailed Description:
The hemoglobinopathies eg sickle cell disease SCD and thalassemia are devastating inherited anemias that shorten and reduce quality of life The only current curative therapy for SCD is bone marrow transplantation However many patients lack access to suitable donors for transplant Alternative treatments based on gene therapy gene editing and novel drugs are currently being developed and show great promise for hemoglobinopathies Gene therapy and gene editing are especially appealing because they eliminate both the need for donors and the potentially devastating side effects of Graft-versus-Host Disease because they take advantage of the patients own cells In gene therapy approaches hematopoietic stem and progenitor cells HSPCs are collected from a patient and then treated to correct or replace the disease-causing mutation However much work remains to develop optimal gene therapy and gene editing protocols as well as better understand the inherent biology of HSPCs in patients with hemoglobinopathies
Researchers at St Jude want to learn how to best manipulate HSPCs from hemoglobinopathy patients with gene therapy and gene editing technologies to achieve optimal gene correction andor replacement as well as optimal engraftment of corrected HSPCs after transplantation St Jude researchers also seek to test candidate drugs on SCD HSPCs that might ameliorate the symptoms of SCD Finally St Jude researchers seek to thoroughly characterize the basic biology and function of HSPCs isolated from hemoglobinopathy patients
Bone marrow-containing bone samples that are typically discarded during orthopedic surgery will be saved from hemoglobinopathy patients as well as non- hemoglobinopathy patients undergoing these surgeries These samples will be shipped to the St Jude Hematology Department for experimental research aimed at addressing the primary and secondary objectives of this protocol
Researchers at St Jude want to learn how to best manipulate HSPCs from hemoglobinopathy patients with gene therapy and gene editing technologies to achieve optimal gene correction andor replacement as well as optimal engraftment of corrected HSPCs after transplantation St Jude researchers also seek to test candidate drugs on SCD HSPCs that might ameliorate the symptoms of SCD Finally St Jude researchers seek to thoroughly characterize the basic biology and function of HSPCs isolated from hemoglobinopathy patients
Bone marrow-containing bone samples that are typically discarded during orthopedic surgery will be saved from hemoglobinopathy patients as well as non- hemoglobinopathy patients undergoing these surgeries These samples will be shipped to the St Jude Hematology Department for experimental research aimed at addressing the primary and secondary objectives of this protocol
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None