Clinical Ex Vivo Expansion of Human Umbilical Cord Blood Stem and Progenitor Cells
|ClinicalTrials.gov Identifier: NCT01624701|
Recruitment Status : Terminated (Funding expired)
First Posted : June 21, 2012
Last Update Posted : November 30, 2015
|Condition or disease||Intervention/treatment||Phase|
|Acute Leukemia Chronic Leukemia Myelodysplastic Syndrome Lymphoma Myeloma||Other: Ex vivo expanded cord blood cells||Phase 1 Phase 2|
- Clinical transplantation of two cord blood units: one ex vivo expanded while another unmanipulated unit to function as a back-up.
Ten patients will be selected from those for whom:
- Allogeneic haematopoietic stem cell transplant is indicated (see details)
- No matched sibling or matched unrelated donor is available quickly enough for the transplant (no fully matched donor found within 1 month of initiation of donor search or donor found but not available for donation within 3 months of donor search).
- At least three unrelated donor cord blood unit can be identified with less than 2 antigens mismatches with the patient but with insufficient cell dose to meet the patient's requirements. If clinical efficacy of this protocol is demonstrated, we will proceed to a multicentre clinical trial with more patients.
- The investigators will obtain haplo-identical MSC from the bone marrow of sibling/parent/offspring of the patient. Although there will be some MSC co-infused with the cord blood cells, this has been shown to be safe in trials of MSC given for patients with graft versus host disease (GVHD) and human leukocyte antigen (HLA) matching of MSC and recipient has been shown to be not important. bone marrow mesenchymal stroma cells (BM-MSCs) derived from related donor bone marrow with a minimum of 2/6 HLA match have been safe for use in patients.1 If the haplo-identical MSC donor is not available, matched unrelated donor MSC would also be used.
Efficacy will be assessed by the following and compared to published literature as well as historical controls:
- Neutrophil and platelet engraftment
- Post transplant 100-day mortality
- Overall and progression-free survival If clinical efficacy of this protocol is demonstrated, the investigators will proceed to a multicentre clinical trial with more patients.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||3 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Clinical Ex Vivo Expansion of Human Umbilical Cord Blood Stem and Progenitor Cells: A Pilot Trial in Collaboration With the Massachusetts Institute of Technology|
|Study Start Date :||March 2012|
|Primary Completion Date :||July 2015|
|Estimated Study Completion Date :||July 2016|
'Ex vivo expanded cord blood cells
Other: Ex vivo expanded cord blood cells
Cord blood cells will be expanded ex vivo using a combination of stem cell factor (SCF), thrombopoietin (TPO), Flt3 ligand and IGFBP2 with mesenchymal stromal cell (MSC) co-culture.
- Safety [ Time Frame: 1 month ]Number of subjects with infusional toxicities (including fever, renal dysfunction within 72 hours of infusion) and potential immunologic competition (absent chimerism of donor cells by 21 days post transplantation).
- Engraftment [ Time Frame: 2 months ]Neutrophil engraftment (ANC>500/ul) in subjects as demonstrated by number of subjects with engraftment <=21 days.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01624701
|Singapore General Hospital|
|Singapore, Singapore, 169608|
|Principal Investigator:||William YK Hwang, MBBS, FRCP||Singapore General Hospital|