Ignite Creation Date:
2024-05-05 @ 10:59 PM
Last Modification Date:
2024-10-26 @ 10:26 AM
Study NCT ID:
NCT01221857
Status:
COMPLETED
Last Update Posted:
2021-08-03
First Post:
2010-10-14
Brief Title:
Pilot Study Evaluating Safety Efficacy of DCBT NiCord UNM CBU to Patients With Hematological Malignancies
Sponsor:
Gamida Cell ltd
Organization:
Gamida Cell ltd
Study Overview
Official Title:
Allogeneic Stem Cell Transplantation of NiCord Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells in Combination With a Second Unmanipulated Cord Blood Unit in Patients With Hematological Malignancies
Status:
COMPLETED
Status Verified Date:
2021-08
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:
Pilot Study Evaluating the Safety and Efficacy of a Co-Transplantation of NiCord a UCB-derived ex Vivo Expanded Population of Stem and Progenitor Cells with a Second Unmanipulated CBU in Patients with Hematological Malignancies
Detailed Description:
Allogeneic hematopoietic stem cell transplantation HSCT is a potentially curative procedure for various hematological malignancies bone marrow failure syndromes and inherited metabolic disorders The application of allogeneic HSCT is limited by donor availability such that only approximately one-third of the otherwise appropriate candidates have suitably matched family donors Alternative donors include mismatched family members or matched unrelated donors but these approaches are often complicated by an increased risk of graft-versus-host disease GvHD and a prolonged and cumbersome search and procurement process In addition far fewer subjects of racial minorities find suitable human leukocyte antigen HLA-matched donors
Umbilical cord blood has been increasingly used as an alternative source of stem cells and has extended the availability of allogeneic HSCT to patients who would otherwise not be eligible for this curative approach In the last decade the number of cord blood transplantations from related and unrelated donors has increased dramatically It is estimated that more than 20000 patients have undergone cord blood transplantation from unrelated donors to date for a variety of genetic hematological immunological metabolic and oncologic disorders The major advantages of cord blood transplantation include easy procurement no risk to donors reduced incidence of transmitting infections immediate availability and reduced risk of acute GvHD in the setting of donor-recipient HLA mismatch Nevertheless the low cell dose remains a main limitation of this cell source leading to delayed hematopoietic reconstitution higher risk of graft failure and relatively high treatment related mortality rates as compared to other hematopoeitic cell sources To improve outcomes and extend applicability of cord blood transplantation one potential solution is ex vivo expansion of cord blood-derived stem and progenitor cells
The Sponsor has undertaken to develop NiCord which is based on a novel technology for ex vivo cell expansion of cord blood derived hematopoietic progenitor cells By increasing the number of the short and long-term reconstitution progenitor cells transplanted NiCord has the potential to enable broader application of umbilical cord blood transplantation and improve clinical outcomes in subjects with high-risk hematological malignancies
The main objective of the current study is to evaluate the safety of co-transplantation of NiCord and an unmanipulated cord blood unit in patients with hematological malignancies following myeloablative therapy
Umbilical cord blood has been increasingly used as an alternative source of stem cells and has extended the availability of allogeneic HSCT to patients who would otherwise not be eligible for this curative approach In the last decade the number of cord blood transplantations from related and unrelated donors has increased dramatically It is estimated that more than 20000 patients have undergone cord blood transplantation from unrelated donors to date for a variety of genetic hematological immunological metabolic and oncologic disorders The major advantages of cord blood transplantation include easy procurement no risk to donors reduced incidence of transmitting infections immediate availability and reduced risk of acute GvHD in the setting of donor-recipient HLA mismatch Nevertheless the low cell dose remains a main limitation of this cell source leading to delayed hematopoietic reconstitution higher risk of graft failure and relatively high treatment related mortality rates as compared to other hematopoeitic cell sources To improve outcomes and extend applicability of cord blood transplantation one potential solution is ex vivo expansion of cord blood-derived stem and progenitor cells
The Sponsor has undertaken to develop NiCord which is based on a novel technology for ex vivo cell expansion of cord blood derived hematopoietic progenitor cells By increasing the number of the short and long-term reconstitution progenitor cells transplanted NiCord has the potential to enable broader application of umbilical cord blood transplantation and improve clinical outcomes in subjects with high-risk hematological malignancies
The main objective of the current study is to evaluate the safety of co-transplantation of NiCord and an unmanipulated cord blood unit in patients with hematological malignancies following myeloablative therapy
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