Ignite Creation Date:
2024-05-06 @ 12:30 AM
Last Modification Date:
2024-10-26 @ 10:50 AM
Study NCT ID:
NCT01590628
Status:
COMPLETED
Last Update Posted:
2022-05-20
First Post:
2012-04-15
Brief Title:
Allogeneic SCT of NiCord UCB-Derived Ex Vivo Expanded Stem and Progenitor Cells in Patients With Hemoglobinopathies
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 Patients With Hemoglobinopathies
Status:
COMPLETED
Status Verified Date:
2022-04
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:
Allogeneic Stem Cell Transplantation of NiCord Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells in Patients with Hemoglobinopathies
Detailed Description:
Umbilical cord blood UCB is an alternative stem cell source for hematopoietic stem cell transplantations HSCT and can be used for the treatment of various life-threatening diseases such as hematological malignancies or genetic blood disorders in such cases where a matched related stem cell donor is not available However the major drawback of using this valuable stem cells source is the limited cell dose in a single cord blood unit CBU which was shown to be associated with inadequate hematopoietic reconstitution and high risk of transplant-related mortality To improve outcomes and extend applicability of UCB transplantation one potential solution is ex vivo expansion of UCB-derived stem and progenitor cells NiCord is a stemprogenitor cell based product composed of ex vivo expanded allogeneic UCB cells NiCord is based on a novel technology for the 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 the broader application of UCB transplantation and improve the clinical outcomes of UCB transplantation
In Part 1 of this study NiCord will be administered to the patient in conjunction with a second unmanipulated CBU In Part 2 of this study NiCord will be administered to the patient without a second unmanipulated CBU The study duration per patient is approximately 270 days from signing of informed consent to last visit on day 180 post-transplant
The overall study objectives of part 1 of this study are to evaluate the safety and efficacy of co-transplantation of NiCord and an unmanipulated CBU in patients with Hemoglobinopathies Sickle Cell Disease SCD or thalassemia major following myeloablative therapy The overall study objectives of part 2 of this study are to evaluate the safety and efficacy of transplantation of NiCord in patients with Hemoglobinopathies Sickle Cell Disease SCD or thalassemia major following myeloablative therapy
The study hypothesis for part 1 of this study is that the co-transplantation of NiCord and an unmanipulated unrelated cord blood graft in patients with hemoglobinopathies SCD or thalassemia major following myeloablative preparative therapy will be safe and will enable cord blood engraftment The study hypothesis for part 2 of this study is that transplantation of NiCord in patients with hemoglobinopathies SCD or thalassemia major following myeloablative preparative therapy will be safe and will enable cord blood engraftment
Up to fifteen 15 evaluable patients recruited for part 1 of the study and up to five 5 patients for part 2 of the study should be 2-45 years of age at least 10 kg in weight have symptomatic SCD or thalassemia major and should be considered as candidates for allogeneic myeloablative HSCT for the treatment of SCD
In Part 1 of this study NiCord will be administered to the patient in conjunction with a second unmanipulated CBU In Part 2 of this study NiCord will be administered to the patient without a second unmanipulated CBU The study duration per patient is approximately 270 days from signing of informed consent to last visit on day 180 post-transplant
The overall study objectives of part 1 of this study are to evaluate the safety and efficacy of co-transplantation of NiCord and an unmanipulated CBU in patients with Hemoglobinopathies Sickle Cell Disease SCD or thalassemia major following myeloablative therapy The overall study objectives of part 2 of this study are to evaluate the safety and efficacy of transplantation of NiCord in patients with Hemoglobinopathies Sickle Cell Disease SCD or thalassemia major following myeloablative therapy
The study hypothesis for part 1 of this study is that the co-transplantation of NiCord and an unmanipulated unrelated cord blood graft in patients with hemoglobinopathies SCD or thalassemia major following myeloablative preparative therapy will be safe and will enable cord blood engraftment The study hypothesis for part 2 of this study is that transplantation of NiCord in patients with hemoglobinopathies SCD or thalassemia major following myeloablative preparative therapy will be safe and will enable cord blood engraftment
Up to fifteen 15 evaluable patients recruited for part 1 of the study and up to five 5 patients for part 2 of the study should be 2-45 years of age at least 10 kg in weight have symptomatic SCD or thalassemia major and should be considered as candidates for allogeneic myeloablative HSCT for the treatment of SCD
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