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| ID | Type | Description | Link |
|---|---|---|---|
| 0911M74035 | Other Identifier | IRB, University of Minnesota |
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This is an open-label, single institution, phase II study in patients with epidermolysis bullosa (EB). The underlying hypothesis is that the infusion of bone marrow or umbilical cord blood from a healthy unaffected donor will correct the collagen, laminin, integrin, or plakin deficiency and reduce the skin fragility characteristic of severe forms of EB. A secondary hypothesis is that mesenchymal stem cells from a healthy donor will enhance the safety and efficacy of the allogeneic hematopoietic stem cell transplant as well as serve as a source of renewable cells for the treatment of focal areas of residual blistering.
The primary objective of this study is to estimate the event-free survival rate by 1 year post-transplant with an event defined as a death or failure to have a demonstrable increase in collagen, laminin, integrin, keratin or plakin deposition by 1 year post-transplant or other biochemical, structural or physical measure of improvement.
The secondary objectives of this study are to i) determine the incidence of transplant-related mortality (TRM) at 180 days; ii) describe the pattern of biochemical improvement as measured by an increase in protein expression (collagen, laminin, integrin, keratin or plakin) and related structural and physical changes; iii) describe health quality of life at day 365 and 730 as compared to pretreatment results; iv) describe the pattern and durability of HSC and third party MSC engraftment in the skin; v) determine the probability of survival at 1 year.
Patients with severe epidermolysis bullosa will be screened to meet the eligibility requirements, related or unrelated donor marrow or UCB will be infused, and subjects will be followed for a minimum of 5 years after stem cell transplant. A target accrual of 75 subjects over 5 years will be recruited to the study.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| RDEB Mac | Experimental | Recessive Dystrophic EB (RDEB) - Myeloablative conditioning (MAC) Participants receive Myeloablative Busulfan (targeting AUC 1000 umol/min), Fludarabine 75 mg/m2, and Cyclophosphamide 200 mg/kg |
|
| RDEB RIC | Experimental | Recessive Dystrophic EB (RDEB) - Reduced Intensity Condition Participants received Fludarabine 500 mg/m2, Cyclosphosphamide 50 mg/kg, equine Anti-thymocyte globulin 90 mg/kg, and low dose total body irradiation (either 200 or 300 cGy) |
|
| JEB MAC | Experimental | Junctional EB (JEB) - Myeloablative conditioning (MAC) Participants receive Myeloablative Busulfan (targeting AUC 1000 umol/min), Fludarabine 75 mg/m2, and Cyclophosphamide 200 mg/kg |
|
| JEB RIC | Experimental |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Cyclophosphamide | Drug | Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants With Event-free Survival | Event-free survival rate, with an event defined as death or failure to have a demonstrable increase in collagen, laminin, intergrin, keratin or plakin deposition. Assessed at follow up appointments through questionnaire and patient samples. | 1 year and 2 Years Post-transplant |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants Transplant-related Mortality (TRM) | Incidence of transplant-related mortality (TRM) | 180 Days Post Transplant |
| Average Biochemical Improvement | Pattern of biochemical improvement measured by cumulative increase in protein expression and related structural and physical changes |
Not provided
Inclusion Criteria:
Diagnosis of severe form of epidermolysis bullosa (EB) characterized by collagen, laminin, integrin, keratin or plakin deficiency. Assessment criteria for severe EB:
Adequate Organ Function Criteria
Available Healthy HSC Donor (order of preference)
Related Donor (marrow or UCB)
Unrelated Donor
Marrow
UCB
Voluntary written consent
Absence of Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Jakub Tolar, MD, PhD | Masonic Cancer Center, University of Minnesota | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Minnesota Masonic Cancer Center and Medical Center | Minneapolis | Minnesota | 55455 | United States |
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| ID | Title | Description |
|---|---|---|
| FG000 | RDEB Mac | Recessive Dystrophic EB (RDEB) - Myeloablative conditioning (MAC) Participants receive Myeloablative Busulfan (targeting AUC 1000 umol/min), Fludarabine 75 mg/m2, and Cyclophosphamide 200 mg/kg Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6. Fludarabine: 40 mg/m^2/day intravenously on Days -6, -5, -4, -3 and -2. Myeloablative Busulfan: Targeting AUC 1000 umol/min Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0 Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip. |
| 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 | Nov 21, 2016 |
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Junctional EB (JEB) - Reduced Intensity Condition Participants received Fludarabine 500 mg/m2, Cyclosphosphamide 50 mg/kg, equine Anti-thymocyte globulin 90 mg/kg, and low dose total body irradiation (either 200 or 300 cGy)
|
|
| Fludarabine | Drug | 40 mg/m^2/day intravenously on Days -6, -5, -4, -3 and -2. |
|
|
| Anti-thymocyte globulin | Drug | 30 mg/kg on Days -4, -3 and -2. |
|
|
| Myeloablative Busulfan | Drug | Targeting AUC 1000 umol/min |
|
| Mesenchymal stem cell transplantation | Procedure | infused via intravenous drip on Day 0 |
|
|
| Total body irradiation | Radiation | 300 cGY on Day -1 administered in a single fraction at a dose rate of 10-19 cGy/minute prescribed to the midplane of the patient at the level of the umbilicus. |
|
| Bone marrow or umbilical cord blood (UCG) stem cell transplantation | Procedure | Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip. |
|
|
| 1 Year Post-Transplant |
| Measure Patients Quality of Life Using a Questionnaire | Health quality of life questionnaire as compared to pretreatment results. Scores can range from 0 to 100. The QOLS scores are summed so that a higher score indicates higher quality of life. | Pretreatment and 1 year |
| Durability of HSC Donor Engraftment in the Skin | Incidence of HSC donor engraftment in the skin | 100 Days |
| Probability of Survival | Surviving patients one year after engraftment | 1 Year |
| Percentage of Participants Who Experienced Acute GVHD | Incidence of acute GCHD | 100 Days |
| FG001 | RDEB RIC | Recessive Dystrophic EB (RDEB) - Reduced Intensity Condition Participants received Fludarabine 500 mg/m2, Cyclosphosphamide 50 mg/kg, equine Anti-thymocyte globulin 90 mg/kg, and low dose total body irradiation (either 200 or 300 cGy) Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6. Fludarabine: 40 mg/m^2/day intravenously on Days -6, -5, -4, -3 and -2. Anti-thymocyte globulin: 30 mg/kg on Days -4, -3 and -2. Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0 Total body irradiation: 300 cGY on Day -1 administered in a single fraction at a dose rate of 10-19 cGy/minute prescribed to the midplane of the patient at the level of the umbilicus. Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip. |
| FG002 | JEB MAC | Junctional EB (JEB) - Myeloablative conditioning (MAC) Participants receive Myeloablative Busulfan (targeting AUC 1000 umol/min), Fludarabine 75 mg/m2, and Cyclophosphamide 200 mg/kg Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6. Fludarabine: 40 mg/m^2/day intravenously on Days -6, -5, -4, -3 and -2. Myeloablative Busulfan: Targeting AUC 1000 umol/min Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0 Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip. |
| FG003 | JEB RIC | Junctional EB (JEB) - Reduced Intensity Condition Participants received Fludarabine 500 mg/m2, Cyclosphosphamide 50 mg/kg, equine Anti-thymocyte globulin 90 mg/kg, and low dose total body irradiation (either 200 or 300 cGy) Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6. Fludarabine: 40 mg/m^2/day intravenously on Days -6, -5, -4, -3 and -2. Anti-thymocyte globulin: 30 mg/kg on Days -4, -3 and -2. Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0 Total body irradiation: 300 cGY on Day -1 administered in a single fraction at a dose rate of 10-19 cGy/minute prescribed to the midplane of the patient at the level of the umbilicus. Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip. |
| COMPLETED |
|
| NOT COMPLETED |
|
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | RDEB Mac | Recessive Dystrophic EB (RDEB) - Myeloablative conditioning (MAC) Participants receive Myeloablative Busulfan (targeting AUC 1000 umol/min), Fludarabine 75 mg/m2, and Cyclophosphamide 200 mg/kg Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6. Fludarabine: 40 mg/m^2/day intravenously on Days -6, -5, -4, -3 and -2. Myeloablative Busulfan: Targeting AUC 1000 umol/min Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0 Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip. |
| BG001 | RDEB RIC | Recessive Dystrophic EB (RDEB) - Reduced Intensity Condition Participants received Fludarabine 500 mg/m2, Cyclosphosphamide 50 mg/kg, equine Anti-thymocyte globulin 90 mg/kg, and low dose total body irradiation (either 200 or 300 cGy) Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6. Fludarabine: 40 mg/m^2/day intravenously on Days -6, -5, -4, -3 and -2. Anti-thymocyte globulin: 30 mg/kg on Days -4, -3 and -2. Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0 Total body irradiation: 300 cGY on Day -1 administered in a single fraction at a dose rate of 10-19 cGy/minute prescribed to the midplane of the patient at the level of the umbilicus. Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip. |
| BG002 | JEB MAC | Junctional EB (JEB) - Myeloablative conditioning (MAC) Participants receive Myeloablative Busulfan (targeting AUC 1000 umol/min), Fludarabine 75 mg/m2, and Cyclophosphamide 200 mg/kg Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6. Fludarabine: 40 mg/m^2/day intravenously on Days -6, -5, -4, -3 and -2. Myeloablative Busulfan: Targeting AUC 1000 umol/min Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0 Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip. |
| BG003 | JEB RIC | Junctional EB (JEB) - Reduced Intensity Condition Participants received Fludarabine 500 mg/m2, Cyclosphosphamide 50 mg/kg, equine Anti-thymocyte globulin 90 mg/kg, and low dose total body irradiation (either 200 or 300 cGy) Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6. Fludarabine: 40 mg/m^2/day intravenously on Days -6, -5, -4, -3 and -2. Anti-thymocyte globulin: 30 mg/kg on Days -4, -3 and -2. Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0 Total body irradiation: 300 cGY on Day -1 administered in a single fraction at a dose rate of 10-19 cGy/minute prescribed to the midplane of the patient at the level of the umbilicus. Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip. |
| BG004 | Total | Total of all reporting groups |
| 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 |
| ||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Race (NIH/OMB) | 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 | Percentage of Participants With Event-free Survival | Event-free survival rate, with an event defined as death or failure to have a demonstrable increase in collagen, laminin, intergrin, keratin or plakin deposition. Assessed at follow up appointments through questionnaire and patient samples. | Posted | Number | 95% Confidence Interval | Percentage of participants | 1 year and 2 Years Post-transplant |
|
|
| |||||||||||||||||||||||||||||||||||
| Secondary | Percentage of Participants Transplant-related Mortality (TRM) | Incidence of transplant-related mortality (TRM) | Posted | Number | 95% Confidence Interval | Percentage of participants | 180 Days Post Transplant |
| |||||||||||||||||||||||||||||||||||||
| Secondary | Average Biochemical Improvement | Pattern of biochemical improvement measured by cumulative increase in protein expression and related structural and physical changes | Posted | Number | 95% Confidence Interval | Percentage of participants | 1 Year Post-Transplant |
| |||||||||||||||||||||||||||||||||||||
| Secondary | Measure Patients Quality of Life Using a Questionnaire | Health quality of life questionnaire as compared to pretreatment results. Scores can range from 0 to 100. The QOLS scores are summed so that a higher score indicates higher quality of life. | Participants unnable to complete quality of life questionnaires at various time points. | Posted | Median | Full Range | Scores on a scale | Pretreatment and 1 year |
| ||||||||||||||||||||||||||||||||||||
| Secondary | Durability of HSC Donor Engraftment in the Skin | Incidence of HSC donor engraftment in the skin | Posted | Number | 95% Confidence Interval | Percentage of Incidence | 100 Days |
| |||||||||||||||||||||||||||||||||||||
| Secondary | Probability of Survival | Surviving patients one year after engraftment | Posted | Number | 95% Confidence Interval | Percentage of participants | 1 Year |
| |||||||||||||||||||||||||||||||||||||
| Secondary | Percentage of Participants Who Experienced Acute GVHD | Incidence of acute GCHD | Posted | Number | 95% Confidence Interval | Percentage of participants | 100 Days |
|
1 year through treatment
Not provided
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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 | RDEB Mac | Recessive Dystrophic EB (RDEB) - Myeloablative conditioning (MAC) Participants receive Myeloablative Busulfan (targeting AUC 1000 umol/min), Fludarabine 75 mg/m2, and Cyclophosphamide 200 mg/kg Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6. Fludarabine: 40 mg/m^2/day intravenously on Days -6, -5, -4, -3 and -2. Myeloablative Busulfan: Targeting AUC 1000 umol/min Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0 Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip. | 4 | 7 | 7 | 7 | 7 | 7 |
| EG001 | RDEB RIC | Recessive Dystrophic EB (RDEB) - Reduced Intensity Condition Participants received Fludarabine 500 mg/m2, Cyclosphosphamide 50 mg/kg, equine Anti-thymocyte globulin 90 mg/kg, and low dose total body irradiation (either 200 or 300 cGy) Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6. Fludarabine: 40 mg/m^2/day intravenously on Days -6, -5, -4, -3 and -2. Anti-thymocyte globulin: 30 mg/kg on Days -4, -3 and -2. Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0 Total body irradiation: 300 cGY on Day -1 administered in a single fraction at a dose rate of 10-19 cGy/minute prescribed to the midplane of the patient at the level of the umbilicus. Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip. | 3 | 16 | 16 | 16 | 16 | 16 |
| EG002 | JEB MAC | Junctional EB (JEB) - Myeloablative conditioning (MAC) Participants receive Myeloablative Busulfan (targeting AUC 1000 umol/min), Fludarabine 75 mg/m2, and Cyclophosphamide 200 mg/kg Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6. Fludarabine: 40 mg/m^2/day intravenously on Days -6, -5, -4, -3 and -2. Myeloablative Busulfan: Targeting AUC 1000 umol/min Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0 Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip. | 1 | 2 | 2 | 2 | 2 | 2 |
| EG003 | JEB RIC | Junctional EB (JEB) - Reduced Intensity Condition Participants received Fludarabine 500 mg/m2, Cyclosphosphamide 50 mg/kg, equine Anti-thymocyte globulin 90 mg/kg, and low dose total body irradiation (either 200 or 300 cGy) Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6. Fludarabine: 40 mg/m^2/day intravenously on Days -6, -5, -4, -3 and -2. Anti-thymocyte globulin: 30 mg/kg on Days -4, -3 and -2. Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0 Total body irradiation: 300 cGY on Day -1 administered in a single fraction at a dose rate of 10-19 cGy/minute prescribed to the midplane of the patient at the level of the umbilicus. Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip. | 6 | 7 | 7 | 7 | 7 | 7 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Bone marrow hypocellular | Blood and lymphatic system disorders | Systematic Assessment |
| ||
| Blood and lymphatic system disorders - Other, specify | Blood and lymphatic system disorders | Systematic Assessment |
| ||
| Febrile neutropenia | Blood and lymphatic system disorders | Systematic Assessment |
| ||
| Cardiac disorders - Other, specify | Cardiac disorders | Systematic Assessment |
| ||
| Heart failure | Cardiac disorders | Systematic Assessment |
| ||
| Diarrhea | Gastrointestinal disorders | Systematic Assessment |
| ||
| Gastrointestinal disorders - Other, specify | Gastrointestinal disorders | Systematic Assessment |
| ||
| Mucositis oral | Gastrointestinal disorders | Systematic Assessment |
| ||
| Vomiting | Gastrointestinal disorders | Systematic Assessment |
| ||
| Esophageal stenosis | Gastrointestinal disorders | Systematic Assessment |
| ||
| Fever | General disorders | Systematic Assessment |
| ||
| General disorders and administration site conditions - Other, specify | General disorders | Systematic Assessment |
| ||
| Multi-organ failure | General disorders | Systematic Assessment |
| ||
| Portal hypertension | Hepatobiliary disorders | Systematic Assessment |
| ||
| Hepatobiliary disorders - Other, specify | Hepatobiliary disorders | Systematic Assessment |
| ||
| Catheter related infection | Infections and infestations | Systematic Assessment |
| ||
| Sepsis | Infections and infestations | Systematic Assessment |
| ||
| Skin infection | Infections and infestations | Systematic Assessment |
| ||
| Upper respiratory infection | Infections and infestations | Systematic Assessment |
| ||
| Wound infection | Infections and infestations | Systematic Assessment |
| ||
| Infections and infestations - Other, specify | Infections and infestations | Systematic Assessment |
| ||
| Metabolism and nutrition disorders - Other, specify | Metabolism and nutrition disorders | Systematic Assessment |
| ||
| Intracranial hemorrhage | Nervous system disorders | Systematic Assessment |
| ||
| Seizure | Nervous system disorders | Systematic Assessment |
| ||
| Acute kidney injury | Renal and urinary disorders | Systematic Assessment |
| ||
| Hypoxia | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Laryngeal edema | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Stridor | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Bronchopulmonary hemorrhage | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Pulmonary edema | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Rash maculo-papular | Skin and subcutaneous tissue disorders | Systematic Assessment |
| ||
| Toxic epidermal necrolysis | Skin and subcutaneous tissue disorders | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Dyspnea | Respiratory, thoracic and mediastinal disorders | Dyspnea | Systematic Assessment |
| |
| Hypoxia | General disorders | Hypoxia | Systematic Assessment |
| |
| Febrile neutropenia | Blood and lymphatic system disorders | Febrile neutropenia | Systematic Assessment |
| |
| Chills | General disorders | Chills | Systematic Assessment |
| |
| Hypertension | Vascular disorders | Hypertension | Systematic Assessment |
| |
| Hypotension | Vascular disorders | Hypotension | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | Rash/Desquamation | Systematic Assessment |
| |
| Infections and infestations - Other | Infections and infestations | Other | Systematic Assessment |
| |
| General disorders and administration site conditions - Other, specify | Gastrointestinal disorders | Systematic Assessment |
| ||
| Sinus Tachycardia | Cardiac disorders | Sinus Tachycardia | Systematic Assessment |
| |
| Pain | General disorders | Pain | Systematic Assessment |
| |
| General disorders - Other | General disorders | Fluid overload | Systematic Assessment |
| |
| Stridor | Respiratory, thoracic and mediastinal disorders | Stridor | Systematic Assessment |
| |
| Tachypnea | Respiratory, thoracic and mediastinal disorders | Tachypnea | Systematic Assessment |
| |
| Wheezing | Respiratory, thoracic and mediastinal disorders | Wheezing | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | Vomiting | Systematic Assessment |
| |
| Agitation | Psychiatric disorders | Agitation | Systematic Assessment |
|
Not provided
Not provided
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Dr. Jakub Tolar | University of Minnesota, Masonic Cancer Center | (612) 626-5654 | tolar003@umn.edu |
| Dec 4, 2023 |
| Prot_SAP_000.pdf |
| ICF | No | No | Yes | Informed Consent Form | May 6, 2018 | Dec 4, 2023 | ICF_001.pdf |
| ID | Term |
|---|---|
| D004820 | Epidermolysis Bullosa |
| ID | Term |
|---|---|
| D012868 | Skin Abnormalities |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D012873 | Skin Diseases, Genetic |
| D030342 | Genetic Diseases, Inborn |
| D012871 | Skin Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D012872 | Skin Diseases, Vesiculobullous |
Not provided
Not provided
| ID | Term |
|---|---|
| D003520 | Cyclophosphamide |
| C024352 | fludarabine |
| C042382 | fludarabine phosphate |
| D000961 | Antilymphocyte Serum |
| D045164 | Mesenchymal Stem Cell Transplantation |
| D014916 | Whole-Body Irradiation |
| D033581 | Stem Cell Transplantation |
| 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 |
| D007106 | Immune Sera |
| D000906 | Antibodies |
| D007136 | Immunoglobulins |
| D007162 | Immunoproteins |
| D001798 | Blood Proteins |
| D011506 | Proteins |
| D000602 | Amino Acids, Peptides, and Proteins |
| D012712 | Serum Globulins |
| D005916 | Globulins |
| D001688 | Biological Products |
| D045424 | Complex Mixtures |
| D017690 | Cell Transplantation |
| D064987 | Cell- and Tissue-Based Therapy |
| D001691 | Biological Therapy |
| D013812 | Therapeutics |
| D014180 | Transplantation |
| D013514 | Surgical Procedures, Operative |
| D011878 | Radiotherapy |
| D008919 | Investigative Techniques |
Not provided
Not provided
| Between 18 and 65 years |
|
| >=65 years |
|
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Asian |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
| 2 year |
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| OG002 | JEB MAC | Junctional EB (JEB) - Myeloablative conditioning (MAC) Participants receive Myeloablative Busulfan (targeting AUC 1000 umol/min), Fludarabine 75 mg/m2, and Cyclophosphamide 200 mg/kg Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6. Fludarabine: 40 mg/m^2/day intravenously on Days -6, -5, -4, -3 and -2. Myeloablative Busulfan: Targeting AUC 1000 umol/min Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0 Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip. |
| OG003 | JEB RIC | Junctional EB (JEB) - Reduced Intensity Condition Participants received Fludarabine 500 mg/m2, Cyclosphosphamide 50 mg/kg, equine Anti-thymocyte globulin 90 mg/kg, and low dose total body irradiation (either 200 or 300 cGy) Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6. Fludarabine: 40 mg/m^2/day intravenously on Days -6, -5, -4, -3 and -2. Anti-thymocyte globulin: 30 mg/kg on Days -4, -3 and -2. Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0 Total body irradiation: 300 cGY on Day -1 administered in a single fraction at a dose rate of 10-19 cGy/minute prescribed to the midplane of the patient at the level of the umbilicus. Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip. |
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| OG002 | JEB MAC | Junctional EB (JEB) - Myeloablative conditioning (MAC) Participants receive Myeloablative Busulfan (targeting AUC 1000 umol/min), Fludarabine 75 mg/m2, and Cyclophosphamide 200 mg/kg Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6. Fludarabine: 40 mg/m^2/day intravenously on Days -6, -5, -4, -3 and -2. Myeloablative Busulfan: Targeting AUC 1000 umol/min Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0 Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip. |
| OG003 | JEB RIC | Junctional EB (JEB) - Reduced Intensity Condition Participants received Fludarabine 500 mg/m2, Cyclosphosphamide 50 mg/kg, equine Anti-thymocyte globulin 90 mg/kg, and low dose total body irradiation (either 200 or 300 cGy) Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6. Fludarabine: 40 mg/m^2/day intravenously on Days -6, -5, -4, -3 and -2. Anti-thymocyte globulin: 30 mg/kg on Days -4, -3 and -2. Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0 Total body irradiation: 300 cGY on Day -1 administered in a single fraction at a dose rate of 10-19 cGy/minute prescribed to the midplane of the patient at the level of the umbilicus. Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip. |
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| OG002 | JEB MAC | Junctional EB (JEB) - Myeloablative conditioning (MAC) Participants receive Myeloablative Busulfan (targeting AUC 1000 umol/min), Fludarabine 75 mg/m2, and Cyclophosphamide 200 mg/kg Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6. Fludarabine: 40 mg/m^2/day intravenously on Days -6, -5, -4, -3 and -2. Myeloablative Busulfan: Targeting AUC 1000 umol/min Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0 Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip. |
| OG003 | JEB RIC | Junctional EB (JEB) - Reduced Intensity Condition Participants received Fludarabine 500 mg/m2, Cyclosphosphamide 50 mg/kg, equine Anti-thymocyte globulin 90 mg/kg, and low dose total body irradiation (either 200 or 300 cGy) Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6. Fludarabine: 40 mg/m^2/day intravenously on Days -6, -5, -4, -3 and -2. Anti-thymocyte globulin: 30 mg/kg on Days -4, -3 and -2. Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0 Total body irradiation: 300 cGY on Day -1 administered in a single fraction at a dose rate of 10-19 cGy/minute prescribed to the midplane of the patient at the level of the umbilicus. Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip. |
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| OG002 | JEB MAC | Junctional EB (JEB) - Myeloablative conditioning (MAC) Participants receive Myeloablative Busulfan (targeting AUC 1000 umol/min), Fludarabine 75 mg/m2, and Cyclophosphamide 200 mg/kg Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6. Fludarabine: 40 mg/m^2/day intravenously on Days -6, -5, -4, -3 and -2. Myeloablative Busulfan: Targeting AUC 1000 umol/min Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0 Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip. |
| OG003 | JEB RIC | Junctional EB (JEB) - Reduced Intensity Condition Participants received Fludarabine 500 mg/m2, Cyclosphosphamide 50 mg/kg, equine Anti-thymocyte globulin 90 mg/kg, and low dose total body irradiation (either 200 or 300 cGy) Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6. Fludarabine: 40 mg/m^2/day intravenously on Days -6, -5, -4, -3 and -2. Anti-thymocyte globulin: 30 mg/kg on Days -4, -3 and -2. Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0 Total body irradiation: 300 cGY on Day -1 administered in a single fraction at a dose rate of 10-19 cGy/minute prescribed to the midplane of the patient at the level of the umbilicus. Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip. |
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| OG002 | JEB MAC | Junctional EB (JEB) - Myeloablative conditioning (MAC) Participants receive Myeloablative Busulfan (targeting AUC 1000 umol/min), Fludarabine 75 mg/m2, and Cyclophosphamide 200 mg/kg Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6. Fludarabine: 40 mg/m^2/day intravenously on Days -6, -5, -4, -3 and -2. Myeloablative Busulfan: Targeting AUC 1000 umol/min Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0 Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip. |
| OG003 | JEB RIC | Junctional EB (JEB) - Reduced Intensity Condition Participants received Fludarabine 500 mg/m2, Cyclosphosphamide 50 mg/kg, equine Anti-thymocyte globulin 90 mg/kg, and low dose total body irradiation (either 200 or 300 cGy) Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6. Fludarabine: 40 mg/m^2/day intravenously on Days -6, -5, -4, -3 and -2. Anti-thymocyte globulin: 30 mg/kg on Days -4, -3 and -2. Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0 Total body irradiation: 300 cGY on Day -1 administered in a single fraction at a dose rate of 10-19 cGy/minute prescribed to the midplane of the patient at the level of the umbilicus. Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip. |
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| OG002 | JEB MAC | Junctional EB (JEB) - Myeloablative conditioning (MAC) Participants receive Myeloablative Busulfan (targeting AUC 1000 umol/min), Fludarabine 75 mg/m2, and Cyclophosphamide 200 mg/kg Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6. Fludarabine: 40 mg/m^2/day intravenously on Days -6, -5, -4, -3 and -2. Myeloablative Busulfan: Targeting AUC 1000 umol/min Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0 Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip. |
| OG003 | JEB RIC | Junctional EB (JEB) - Reduced Intensity Condition Participants received Fludarabine 500 mg/m2, Cyclosphosphamide 50 mg/kg, equine Anti-thymocyte globulin 90 mg/kg, and low dose total body irradiation (either 200 or 300 cGy) Cyclophosphamide: Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6. Fludarabine: 40 mg/m^2/day intravenously on Days -6, -5, -4, -3 and -2. Anti-thymocyte globulin: 30 mg/kg on Days -4, -3 and -2. Mesenchymal stem cell transplantation: infused via intravenous drip on Day 0 Total body irradiation: 300 cGY on Day -1 administered in a single fraction at a dose rate of 10-19 cGy/minute prescribed to the midplane of the patient at the level of the umbilicus. Bone marrow or umbilical cord blood (UCG) stem cell transplantation: Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip. |
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