| ID | Type | Description | Link |
|---|---|---|---|
| U54HL081030-02 | U.S. NIH Grant/Contract | View source |
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Study stopped because of toxicity concerns.
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| Name | Class |
|---|---|
| National Heart, Lung, and Blood Institute (NHLBI) | NIH |
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The purpose of this study is to determine whether the addition of parathyroid hormone after a sequential cord blood transplant will improve engraftment, which is the ability of the transplanted stem cells to grow and to successfully begin producing new blood cells.
In this phase II, single stage study, participants will include 40 adults who are candidates for a hematopoietic stem cell transplant. All participants will undergo a sequential cord blood transplant using a well-known myeloablative regimen of fludarabine, cyclophosphamide, and total body irradiation, which is appropriate for those individuals who are likely to benefit from an ablative regimen. Tacrolimus will be combined with mycophenolate mofetil (MMF) for the graft-versus-host disease (GVHD) prophylaxis regimen. Parathyroid hormone (PTH) will be added to this regimen in an attempt to improve engraftment. PTH is an approved drug with minimal side effects in individuals with osteoporosis; the dose of PTH has been determined from a phase I study in individuals with hematologic cancer.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Parathyroid Hormone (teriparatide) | Experimental | Parathyroid hormone after double umbilical cord blood transplant. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Parathyroid Hormone (teriparatide) | Drug | Day +1: PTH 40 mcg, Day +2: PTH 60 mcg, Day +3: PTH 80 mcg, Day +4 to Day +29 or until ANC>2000/microL: PTH 100 mcg |
|
| Measure | Description | Time Frame |
|---|---|---|
| Median Time to Neutrophil Engraftment (Defined as an Absolute Neutrophil Count [ANC] Greater Than 500) | Median time to neutrophil engraftment (defined as an absolute neutrophil count [ANC] greater than 500) | Statistic is calculated at Day 42 but ANC counts are measured daily up through discharge. |
| Measure | Description | Time Frame |
|---|---|---|
| Cumulative Incidence of Acute GVHD Grades II-IV at Day 100 | Cumulative Incidence of Acute GVHD Grades II-IV at day 100 | Measured at Day 100 |
| Cumulative Incidence of Chronic GVHD | Cumulative Incidence of Chronic GVHD |
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Inclusion Criteria:
One of the following diagnoses:
No prior autologous stem cell transplant
Eastern Cooperative Oncology Group (ECOG) performance status of less than 2
Lack of 6/6 or 5/6 matched related donor OR lack of 10/10 matched unrelated donor OR no available donor in the appropriate time frame to perform a potentially curative stem cell transplant
Diffusing capacity of the lung for carbon monoxide (DLCO) greater than 50% of predicted value
Left ventricular ejection fraction (LVEF) greater than 50% of predicted value
Calcium levels less than 10.5 mg/dl
Phosphate levels greater than 1.6 mg/dl
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Karen K. Ballen, MD | Massachusetts General Hospital | Principal Investigator |
| Joseph Antin, MD | Dana-Farber Cancer Institute | Principal Investigator |
| David Avigan, MD | Beth Israel Deaconess Medical Center | Principal Investigator |
| Elizabeth J Shpall, MD | MD Anderson Cancer Research Center | Principal Investigator |
| Colleen Delaney, MD | Fred Hutchinson Cancer Center | Principal Investigator |
| Ram Kamble, MD | Baylor College of Medicine | Principal Investigator |
| Katarzyna Jamieson, M.D. | University of Florida | Principal Investigator |
| Philip McCarthy, M.D. | Roswell Park Cancer Institute | Principal Investigator |
| Edward Ball, M.D. | University of California, San Diego | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Florida | Gainesville | Florida | 32610 | United States | ||
| Massachusetts General Hospital |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 22766223 | Derived | Ballen K, Mendizabal AM, Cutler C, Politikos I, Jamieson K, Shpall EJ, Dey BR, Attar E, McAfee S, Delaney C, McCarthy P, Ball ED, Kamble R, Avigan D, Maziarz RT, Ho VT, Koreth J, Alyea E, Soiffer R, Wingard JR, Boussiotis V, Spitzer TR, Antin JH. Phase II trial of parathyroid hormone after double umbilical cord blood transplantation. Biol Blood Marrow Transplant. 2012 Dec;18(12):1851-8. doi: 10.1016/j.bbmt.2012.06.016. Epub 2012 Jul 2. |
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Thirteen patients who were enrolled proceeded to the cord blood transplant.
Thirteen patients were enrolled on the study between October 25, 2006 and March 27, 2008 from three centers. Dana Farber Cancer Institute enrolled 10 patients, University of Florida enrolled 2 patients and Massachusetts General Hospital enrolled 1 patient.
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| ID | Title | Description |
|---|---|---|
| FG000 | Parathyroid Hormone (Teriparatide) | Parathyroid hormone after double umbilical cord blood transplant. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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|
| Measured at 2 years |
| Platelet Engraftment (Greater Than 20,000) | Platelet engraftment (greater than 20,000) | Measured at Day 180 |
| 100-day Transplant-related Mortality | 100-day transplant-related mortality | Measured at Day 100 |
| Cumulative Incidence of Relapse | Cumulative Incidence of Relapse | Measured at 2 years |
| Overall Survival | Overall Survival | Measured at 2 years |
| Disease-free Survival | Disease-free survival | Measured at 1 year |
| Richard Maziarz, M.D. |
| Oregon Health and Science University |
| Principal Investigator |
| Boston |
| Massachusetts |
| 02114 |
| United States |
| Beth Israel Deaconess Medical Center | Boston | Massachusetts | 02115 | United States |
| Dana Farber Cancer Institute | Boston | Massachusetts | 02115 | United States |
| COMPLETED |
|
| NOT COMPLETED |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Parathyroid Hormone (Teriparatide) | Parathyroid hormone after double umbilical cord blood transplant. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants |
| ||||||||||||||||||||
| Age Continuous | Median Age at Transplant | Median | Full Range | years |
| ||||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||||||
| Race (NIH/OMB) | Race among patients transplanted. | Count of Participants | Participants |
| |||||||||||||||||||
| Region of Enrollment | Number | participants |
|
| 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 | Median Time to Neutrophil Engraftment (Defined as an Absolute Neutrophil Count [ANC] Greater Than 500) | Median time to neutrophil engraftment (defined as an absolute neutrophil count [ANC] greater than 500) | 13 patients received the transplant. | Posted | Median | Full Range | days | Statistic is calculated at Day 42 but ANC counts are measured daily up through discharge. |
|
|
| |||||||||||||||||||||||||
| Secondary | Cumulative Incidence of Acute GVHD Grades II-IV at Day 100 | Cumulative Incidence of Acute GVHD Grades II-IV at day 100 | 13 patients were transplanted | Posted | Number | 95% Confidence Interval | percentage of participants | Measured at Day 100 |
|
| ||||||||||||||||||||||||||
| Secondary | Cumulative Incidence of Chronic GVHD | Cumulative Incidence of Chronic GVHD | 13 patients were transplanted. | Posted | Number | 95% Confidence Interval | percentage of participants | Measured at 2 years |
|
| ||||||||||||||||||||||||||
| Secondary | Platelet Engraftment (Greater Than 20,000) | Platelet engraftment (greater than 20,000) | 13 patients were transplanted. | Posted | Number | 95% Confidence Interval | percentage of participants | Measured at Day 180 |
|
| ||||||||||||||||||||||||||
| Secondary | 100-day Transplant-related Mortality | 100-day transplant-related mortality | 13 patients were transplanted | Posted | Number | participants | Measured at Day 100 |
|
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| ||||||||||||||||||||||||||
| Secondary | Cumulative Incidence of Relapse | Cumulative Incidence of Relapse | 13 patients were transplanted. | Posted | Number | 95% Confidence Interval | percentage of participants | Measured at 2 years |
|
| ||||||||||||||||||||||||||
| Secondary | Overall Survival | Overall Survival | 13 patients were transplanted. | Posted | Number | 95% Confidence Interval | percentage of participants | Measured at 2 years |
|
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| |||||||||||||||||||||||||
| Secondary | Disease-free Survival | Disease-free survival | 13 patients were transplanted. | Posted | Number | 95% Confidence Interval | percentage of participants | Measured at 1 year |
|
|
|
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 | Parathyroid Hormone (Teriparatide) | Parathyroid hormone after double umbilical cord blood transplant. | 8 | 13 | 8 | 13 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Death | General disorders | Systematic Assessment | Death |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Death | General disorders |
|
The study was closed prematurely due to early mortality and lack of efficacy.
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Adam Mendizabal, Statistician | The EMMES Corporation | 301-251-1161 | 221 | amendizabal@emmes.com |
| ID | Term |
|---|---|
| D015464 | Leukemia, Myelogenous, Chronic, BCR-ABL Positive |
| D000741 | Anemia, Aplastic |
| D055728 | Primary Myelofibrosis |
| D008223 | Lymphoma |
| D006689 | Hodgkin Disease |
| D015451 | Leukemia, Lymphocytic, Chronic, B-Cell |
| D015470 | Leukemia, Myeloid, Acute |
| D054198 | Precursor Cell Lymphoblastic Leukemia-Lymphoma |
| D000754 | Anemia, Refractory, with Excess of Blasts |
| ID | Term |
|---|---|
| D007951 | Leukemia, Myeloid |
| D007938 | Leukemia |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D009196 | Myeloproliferative Disorders |
| D001855 | Bone Marrow Diseases |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D002908 | Chronic Disease |
| D020969 | Disease Attributes |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D000740 | Anemia |
| D000080983 | Bone Marrow Failure Disorders |
| D008232 | Lymphoproliferative Disorders |
| D008206 | Lymphatic Diseases |
| D007160 | Immunoproliferative Disorders |
| D007154 | Immune System Diseases |
| D015448 | Leukemia, B-Cell |
| D007945 | Leukemia, Lymphoid |
| D000753 | Anemia, Refractory |
| D009190 | Myelodysplastic Syndromes |
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| ID | Term |
|---|---|
| D010281 | Parathyroid Hormone |
| D019379 | Teriparatide |
| ID | Term |
|---|---|
| D036361 | Peptide Hormones |
| D006728 | Hormones |
| D006730 | Hormones, Hormone Substitutes, and Hormone Antagonists |
| D010455 | Peptides |
| D000602 | Amino Acids, Peptides, and Proteins |
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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 |
|
| Unknown or Not Reported |
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