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| ID | Type | Description | Link |
|---|---|---|---|
| MT2018-06 | Other Identifier | University of Minnesota Masonic Cancer Center |
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This is an single arm, open label, interventional phase II trial evaluating the efficacy of umbilical cord blood (UCB) hematopoietic stem and progenitor cells (HSPC) expanded in culture with stimulatory cytokines (SCF, Flt-3L, IL-6 and thromopoietin) on lympho-hematopoietic recovery. Patients will receive a uniform myeloablative conditioning and post-transplant immunoprophylaxis.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| FLU, CY, TBI + MGTA-456 infusion | Experimental | All patients will receive MGTA-456 on the day of transplantation after myeloablative conditioning. All patients aged 3-55 years will be conditioned with cyclophosphamide (CY) 120 mg/kg total dose, fludarabine (FLU) 75 m/m2 total dose and total body irradiation (TBI) 1320 cGy total dose as well as tacrolimus (Tac) and mycophenolate mofetil (MMF) immunoprophylaxis and granulocyte-colony stimulating factor (G-CSF) |
|
| BU,FLU, MEL + MGTA-456 infusion | Experimental | All young children <3 years of age at the time of diagnosis will receive MGTA-456 on the day of transplantation after a non-TBI containing myeloablative conditioning as TBI may have a damaging effect on brain development in the very young child. All patients aged 0-3 years will be conditioned with busulfan (BU), FLU and melphalan (MEL) as well as Tac/MMF immunoprophylaxis and G-CSF |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Fludarabine (FLU) | Drug | 25 mg/m2 IV over 1 hour (<10 kg: 0.83 mg/kg IV over 1 hour) |
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| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants With Neutrophil Recover | Percentage of participants with neutrophil recovery by day 14 after transplantation in recipients of MGTA-456. | Day 14 |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Days Alive Without Hospitalization | Number of days alive without hospitalization between days 0 and 100 after transplantation | Day 0 and Day 100 |
| Secondary Graft Failure | Incidence of secondary graft failure |
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Age, Unit Cell Dose and HLA Match Criteria
Eligible Diseases:
Acute myelogenous leukemia (AML) in morphological complete remission with:
Minimal residual disease (MRD) by flow cytometry, or
Intermediate to high risk leukemia in first (CR1) based on institutional criteria, eg. not favorable risk AML which is defined as having one of the following:
Any second or subsequent CR, or
Secondary AML with prior malignancy that has been in remission for at least 12 months.
Acute lymphocytic leukemia (ALL) at the following stages:
High risk first morphological, cytogenetic and molecular CR with:
High risk second CR based on institutional criteria (eg, for children, bone marrow relapse <36 months from induction or T-lineage bone marrow relapse or very early isolated central nervous system (CNS) relapse <6 months from diagnosis, or slow re-induction (stage M2-3 at day 28 after induction) regardless of length remission. All patients with MRD by flow cytometry.
Any third or subsequent CR.
Secondary ALL
Biphenotypic/undifferentiated leukemia in morphological, cytogenetic and molecular CR .
Chronic Myelogenous Leukemia (CML) in high risk first chronic phase (failure of two tyrosine kinase inhibitors (TKI) or TKI intolerance), accelerated phase or second chronic phase.
Myelodysplasia (MDS) IPSS Int-2 or High risk (i.e. RAEB, RAEBt <5% blasts) or other high risk features, including multiple cytopenias, high risk cytogenetics or lack of response to standard therapy..
Relapsed large-cell lymphoma, mantle-cell lymphoma and Hodgkin lymphoma that is chemotherapy sensitive and ineligible for an autologous transplant.
Burkitt's lymphoma in CR2 or subsequent CR.
Relapsed T-cell lymphoma that is chemotherapy sensitive in CR/PR that is ineligible for an autologous transplant.
Organ Specific Inclusion Criteria
Karnofsky score ≥70 (16 years and older), Lansky play score >50 (children 2-16 years, or 'adequate' score for children <2 years, as detailed in Appendix II.
Adequate organ function defined as:
Cardiac: No uncontrolled arrhythmia and left ventricular ejection fraction at rest must be >3545%.
Available 'back-up' HSPC graft (e.g, second UCB unit, haploidentical related donor).
Females of child bearing potential and sexually active males must agree to use adequate birth control during study treatment.
Voluntary written consent signed (adult or parental) before performance of any study-related procedure not part of normal medical care.
Exclusion Criteria
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Masonic Cancer Center at University of Minnesota | Minneapolis | Minnesota | 55455 | United States |
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| ID | Title | Description |
|---|---|---|
| FG000 | FLU, CY, TBI + MGTA-456 Infusion | All patients aged 3-55 years will be conditioned with cyclophosphamide (CY) 120 mg/kg total dose, fludarabine (FLU) 75 m/m2 total dose and total body irradiation (TBI) 1320 cGy total dose as well as tacrolimus (Tac) and mycophenolate mofetil (MMF) immunoprophylaxis and granulocyte-colony stimulating factor (G-CSF) |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Apr 23, 2021 |
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| Cyclophosphamide (CY) | Drug | 60 mg/kg IV over 2 hours |
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| Total Body Irradiation (TBI) | Drug | 165 cGy twice daily |
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| Tacrolimus (Tac) | Drug | Tacrolimus will start day -3 and will be administered as a continuous IV infusion at a starting dose of 0.03 mg/kg/day. Goal trough levels will be 10-15 ng/mL for the first 14 days post-transplant and then decreased to a goal of 5-10 ng/ml thereafter. |
|
| Mycophenolate Mofetil (MMF) | Drug | MMF 3 gram/day IV/PO for adult patients divided in 2 or 3 doses. Pediatric patients will receive MMF at the dose of 15 mg/kg/dose (max 1 gram per dose) every 8 hours beginning day -3. |
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| Granulocyte Colony-Stimulating Factor (G-CSF) | Drug | 5 ug/kg/d until the absolute neutrophil count (ANC) is >2500/uL for 2 consecutive days |
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| Busulfan (BU) | Drug | BU IV once daily with dose based on Pharmacokinetics (PK) calculator over 3 hours |
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| Melphalan | Drug | 50 mg/m2/day (1.7 mg/kg/day if < 10 kg) IV over 30 min |
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| MGTA 456 Infusion | Drug | The target cell dose is >10 x 106 CD34/kg with a maximum TNC 2.7 x 108/kg for children (<18 years) and 8.1 × 108 cells/kg [expanded product only] for adults based on the highest cell dose windows evaluated in prior studies. |
|
| 2 Years |
| Platelet Recovery | Incidence of platelet recovery at day 42 | Day 42 |
| Treatment Related Mortality (TRM) | Incidence of TRM at 6 months | 6 Months |
| Grades II-IV Acute GVHD | Incidence of grades II-IV acute GVHD at day 100 | Day 100 |
| Grades III-IV Acute GVHD | Incidence of grades III-IV acute GVHD at day 100 | Day 100 |
| Chronic GVHD | Incidence of chronic GVHD at 1 year | 1 Year |
| Relapse | Incidence of relapse at 2 years | 2 Years |
| Non-catheter Associated Bacterial Infections | Incidence of non-catheter associated bacterial infections by day 100 | Day 100 |
| Overall Survival (OS) | Incidence of overall survival (OS) at 2 years | 2 Years |
| Event-Free Survival (EFS) | Incidence of event-free survival (EFS) at 2 years | 2 Years |
| FG001 |
| BU,FLU, MEL + MGTA-456 Infusion |
All young children <3 years of age at the time of diagnosis will receive MGTA-456 on the day of transplantation after a non-TBI containing myeloablative conditioning as TBI may have a damaging effect on brain development in the very young child. All patients aged 0-3 years will be conditioned with busulfan (BU), FLU and melphalan (MEL) as well as Tac/MMF immunoprophylaxis and G-CSF |
| COMPLETED |
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| NOT COMPLETED |
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| ID | Title | Description |
|---|---|---|
| BG000 | FLU, CY, TBI + MGTA-456 Infusion | All patients aged 3-55 years will be conditioned with cyclophosphamide (CY) 120 mg/kg total dose, fludarabine (FLU) 75 m/m2 total dose and total body irradiation (TBI) 1320 cGy total dose as well as tacrolimus (Tac) and mycophenolate mofetil (MMF) immunoprophylaxis and granulocyte-colony stimulating factor (G-CSF) |
| BG001 | BU,FLU, MEL + MGTA-456 Infusion | All young children <3 years of age at the time of diagnosis will receive MGTA-456 on the day of transplantation after a non-TBI containing myeloablative conditioning as TBI may have a damaging effect on brain development in the very young child. All patients aged 0-3 years will be conditioned with busulfan (BU), FLU and melphalan (MEL) as well as Tac/MMF immunoprophylaxis and G-CSF |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants |
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| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| Region of Enrollment | Number | participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Percentage of Participants With Neutrophil Recover | Percentage of participants with neutrophil recovery by day 14 after transplantation in recipients of MGTA-456. | Posted | Number | 95% Confidence Interval | Percentage of participants | Day 14 |
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| Secondary | Number of Days Alive Without Hospitalization | Number of days alive without hospitalization between days 0 and 100 after transplantation | Posted | Median | Full Range | Days | Day 0 and Day 100 |
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| Secondary | Secondary Graft Failure | Incidence of secondary graft failure | Posted | Number | Percentage of participants | 2 Years |
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| Secondary | Platelet Recovery | Incidence of platelet recovery at day 42 | Posted | Number | 95% Confidence Interval | Percentage of participants | Day 42 |
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| Secondary | Treatment Related Mortality (TRM) | Incidence of TRM at 6 months | Posted | Number | 95% Confidence Interval | Percentage of participants | 6 Months |
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| Secondary | Grades II-IV Acute GVHD | Incidence of grades II-IV acute GVHD at day 100 | Posted | Number | 95% Confidence Interval | Percentage of participants | Day 100 |
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| Secondary | Grades III-IV Acute GVHD | Incidence of grades III-IV acute GVHD at day 100 | Posted | Number | 95% Confidence Interval | Percentage of participants | Day 100 |
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| Secondary | Chronic GVHD | Incidence of chronic GVHD at 1 year | Posted | Number | 95% Confidence Interval | Percentage of participants | 1 Year |
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| Secondary | Relapse | Incidence of relapse at 2 years | Posted | Number | 95% Confidence Interval | Percentage of participants | 2 Years |
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| Secondary | Non-catheter Associated Bacterial Infections | Incidence of non-catheter associated bacterial infections by day 100 | Posted | Number | Percentage of participants | Day 100 |
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| Secondary | Overall Survival (OS) | Incidence of overall survival (OS) at 2 years | Posted | Number | Percentage of participants | 2 Years |
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| Secondary | Event-Free Survival (EFS) | Incidence of event-free survival (EFS) at 2 years | Posted | Number | Percentage of participants | 2 Years |
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2 years
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | FLU, CY, TBI + MGTA-456 Infusion | All patients aged 3-55 years will be conditioned with cyclophosphamide (CY) 120 mg/kg total dose, fludarabine (FLU) 75 m/m2 total dose and total body irradiation (TBI) 1320 cGy total dose as well as tacrolimus (Tac) and mycophenolate mofetil (MMF) immunoprophylaxis and granulocyte-colony stimulating factor (G-CSF) | 5 | 16 | 0 | 16 | 0 | 16 |
| EG001 | BU,FLU, MEL + MGTA-456 Infusion | All young children <3 years of age at the time of diagnosis will receive MGTA-456 on the day of transplantation after a non-TBI containing myeloablative conditioning as TBI may have a damaging effect on brain development in the very young child. All patients aged 0-3 years will be conditioned with busulfan (BU), FLU and melphalan (MEL) as well as Tac/MMF immunoprophylaxis and G-CSF | 2 | 2 | 0 | 2 | 0 | 2 |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Dr. Margaret MacMillan | University of Minnesota, Masonic Cancer Center | (612) 626-5654 | macmi002@umn.edu |
| Dec 20, 2023 |
| Prot_SAP_000.pdf |
| ICF | No | No | Yes | Informed Consent Form | May 10, 2021 | Dec 20, 2023 | ICF_001.pdf |
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| ID | Term |
|---|---|
| D015470 | Leukemia, Myeloid, Acute |
| D054198 | Precursor Cell Lymphoblastic Leukemia-Lymphoma |
| D015464 | Leukemia, Myelogenous, Chronic, BCR-ABL Positive |
| D000754 | Anemia, Refractory, with Excess of Blasts |
| D054739 | Dendritic Cell Sarcoma, Interdigitating |
| D020522 | Lymphoma, Mantle-Cell |
| D006689 | Hodgkin Disease |
| D002051 | Burkitt Lymphoma |
| D016399 | Lymphoma, T-Cell |
| D018442 | Lymphoma, B-Cell, Marginal Zone |
| ID | Term |
|---|---|
| D007951 | Leukemia, Myeloid |
| D007938 | Leukemia |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D007945 | Leukemia, Lymphoid |
| D008232 | Lymphoproliferative Disorders |
| D008206 | Lymphatic Diseases |
| D007160 | Immunoproliferative Disorders |
| D007154 | Immune System Diseases |
| D009196 | Myeloproliferative Disorders |
| D001855 | Bone Marrow Diseases |
| D002908 | Chronic Disease |
| D020969 | Disease Attributes |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D000753 | Anemia, Refractory |
| D000740 | Anemia |
| D009190 | Myelodysplastic Syndromes |
| D015620 | Histiocytic Disorders, Malignant |
| D015614 | Histiocytosis |
| D008228 | Lymphoma, Non-Hodgkin |
| D008223 | Lymphoma |
| D020031 | Epstein-Barr Virus Infections |
| D006566 | Herpesviridae Infections |
| D004266 | DNA Virus Infections |
| D014777 | Virus Diseases |
| D007239 | Infections |
| D014412 | Tumor Virus Infections |
| D016393 | Lymphoma, B-Cell |
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| ID | Term |
|---|---|
| C024352 | fludarabine |
| D003520 | Cyclophosphamide |
| D014916 | Whole-Body Irradiation |
| D016559 | Tacrolimus |
| D014221 | Triamcinolone |
| D009173 | Mycophenolic Acid |
| D016179 | Granulocyte Colony-Stimulating Factor |
| D002066 | Busulfan |
| D008558 | Melphalan |
| ID | Term |
|---|---|
| D010752 | Phosphoramide Mustards |
| D009588 | Nitrogen Mustard Compounds |
| D009150 | Mustard Compounds |
| D006846 | Hydrocarbons, Halogenated |
| D006838 | Hydrocarbons |
| D009930 | Organic Chemicals |
| D063088 | Phosphoramides |
| D009943 | Organophosphorus Compounds |
| D011878 | Radiotherapy |
| D013812 | Therapeutics |
| D008919 | Investigative Techniques |
| D018942 | Macrolides |
| D007783 | Lactones |
| D011245 | Pregnadienes |
| D011278 | Pregnanes |
| D013256 | Steroids |
| D000072473 | Fused-Ring Compounds |
| D011083 | Polycyclic Compounds |
| D013259 | Steroids, Fluorinated |
| D002208 | Caproates |
| D000144 | Acids, Acyclic |
| D002264 | Carboxylic Acids |
| D005227 | Fatty Acids |
| D008055 | Lipids |
| D003115 | Colony-Stimulating Factors |
| D006023 | Glycoproteins |
| D006001 | Glycoconjugates |
| D002241 | Carbohydrates |
| D016298 | Hematopoietic Cell Growth Factors |
| D016207 | Cytokines |
| D036341 | Intercellular Signaling Peptides and Proteins |
| D010455 | Peptides |
| D000602 | Amino Acids, Peptides, and Proteins |
| D011506 | Proteins |
| D001685 | Biological Factors |
| D002072 | Butylene Glycols |
| D006018 | Glycols |
| D000438 | Alcohols |
| D008698 | Mesylates |
| D000476 | Alkanesulfonates |
| D017738 | Alkanesulfonic Acids |
| D000473 | Alkanes |
| D006839 | Hydrocarbons, Acyclic |
| D013451 | Sulfonic Acids |
| D013456 | Sulfur Acids |
| D013457 | Sulfur Compounds |
| D010649 | Phenylalanine |
| D024322 | Amino Acids, Aromatic |
| D000598 | Amino Acids, Cyclic |
| D000596 | Amino Acids |
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| >=65 years |
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| Male |
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| Not Hispanic or Latino |
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| Unknown or Not Reported |
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| Asian |
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| Native Hawaiian or Other Pacific Islander |
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| Black or African American |
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| White |
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| More than one race |
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| Unknown or Not Reported |
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