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| Name | Class |
|---|---|
| California Institute for Regenerative Medicine (CIRM) | OTHER |
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A phase 1b, open label, multi-center trial of AB-110 in adults with hematologic malignancies, including acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), and myelodysplasia (MDS) undergoing cord blood transplantation. Subjects will receive unmanipulated cord blood (UCB) and AB-110 expanded CD34 enriched hematopoietic progenitor cells (HSPC).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Experimental | Experimental | Unmanipulated umbilical cord blood plus AB-110 |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Unmanipulated Umbilical Cord Blood (UCB) | Biological | Human leukocyte antigen (HLA) matched umbilical cord blood |
|
| Measure | Description | Time Frame |
|---|---|---|
| Occurrence of adverse events grade 4 or grade 5 as assessed by CTCAEv4 | 24 hours | |
| Graft failure defined as survival to day 42 without absolute neutrophil count greater than or equal to 500/mm3 | 42 days |
| Measure | Description | Time Frame |
|---|---|---|
| Time to neutrophil engraftment | 42 days | |
| Cumulative incidence of sustained donor-derived neutrophil engraftment | 42 days | |
| Cumulative incidence of sustained donor-derived neutrophil engraftment |
| Measure | Description | Time Frame |
|---|---|---|
| Late onset acute GVHD | 720 days | |
| Chronic GVHD | 720 days | |
| Quantitative recovery of T-cells and subsets |
Inclusion Criteria:
Patients must have received some immunosuppressive chemotherapy in the preceding 3 months.
Acute myelogenous leukemia (AML):
Acute lymphoblastic leukemia (ALL):
Other acute leukemias that are of ambiguous lineage or of other types
Any acute leukemia with marrow aplasia or without adequate count recovery.
Myelodysplastic Syndrome (MDS)
Karnofsky score > 70 %.
Calculated creatinine clearance > 60 ml/min.
Bilirubin < 1.5 mg/dL, ALT < 3 x upper limit of normal
Pulmonary function (FVC, FEV1 and corrected DLCO) > 50% predicted.
Left ventricular ejection fraction > 50%.
Albumin > 3.0 g/dL.
Negative antiviral serology:
Negative human immunodeficiency virus (HIV) antibody.
Negative human T-lymphotropic virus (HTLV)-1 and 2 antibodies.
Negative hepatitis B surface antigen (HBsAg) and undetectable hepatitis B virus (HBV) DNA
Negative hepatitis C virus (HCV) antibody or negative HCV ribonucleic acid (RNA)
For female subjects of childbearing potential:
Male subjects must be willing to use a recommended method of contraception throughout the study period, and to refrain from sperm donation throughout the study period.
Two appropriate CB units identified for the subject.
In the judgment of the investigator, participation in the protocol offers an acceptable benefit-to-risk ratio when considering current disease status, medical condition, and the potential benefits and risks of alternative treatments for the subject's cancer.
Willingness and ability to comply with scheduled visits, drug administration plan, protocol-specified laboratory tests, other study procedures, and study restrictions.
Evidence of a signed informed written consent
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Paul Finnegan, MD | Angiocrine Bioscience | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| City of Hope Comprehensive Cancer Center | Duarte | California | 91010 | United States | ||
| University of Colorado Cancer Center University of Colorado Anschutz Medical Campus |
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| AB-110 | Biological | Expanded cord blood stem cells and engineered human endothelial cells |
|
| 100 days |
| Cumulative incidence of sustained donor-derived neutrophil engraftment | 180 days |
| Incidence of engraftment syndrome | 28 days |
| Cumulative incidence of graft failure | 43 days |
| Cumulative incidence of graft failure | 180 days |
| Presence of post-transplant phenotype in hematopoietic cells of donor origin | 180 days |
| Cumulative incidence of grade II - IV acute graft versus host disease (aGVHD) | 100 days |
| Cumulative incidence of grade II - IV acute graft versus host disease (GVHD) | 180 days |
| Cumulative incidence of chronic GVHD | 100 days |
| Cumulative incidence of chronic GVHD | 180 days |
| Time to lymphoid recovery | 180 days |
| Cumulative incidence of transplant related mortality (TRM) | 100 days |
| Cumulative incidence of transplant related mortality (TRM) | 180 days |
| Overall survival (OS) | 100 days |
| Overall survival (OS) | 180 days |
| Disease Free Survival (DFS) | 100 days |
| Disease Free Survival (DFS) | 180 days |
| 1 year |
| Quantitative recovery of T-cells and subsets | 2 years |
| Transplant Related Mortality | 1 year |
| Transplant Related Mortality | 2 years |
| Recurrence of malignancy | 1 year |
| Recurrence of malignancy | 2 years |
| Overall survival | 1 year |
| Overall survival | 2 years |
| Disease free survival | 1 year |
| Disease free survival | 2 years |
| Aurora |
| Colorado |
| 80045 |
| United States |
| Memorial Sloan Kettering Cancer Center | New York | New York | 10065 | United States |
| ID | Term |
|---|---|
| D015470 | Leukemia, Myeloid, Acute |
| D009190 | Myelodysplastic Syndromes |
| ID | Term |
|---|---|
| D007951 | Leukemia, Myeloid |
| D007938 | Leukemia |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D001855 | Bone Marrow Diseases |
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| ID | Term |
|---|---|
| C056345 | epithienamycin E |
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