CDE Detailed Report
Disease: Sickle Cell Disease
Sub-Domain: Safety Data
CRF: Genotoxicity

137 results.
CDE ID CDE Name Variable Name Definition Short Description Additional Notes (Question Text) Permissible Values Description Data Type Disease Specific Instructions Disease Specific Reference Population Classification (e.g., Core) Version Number Version Date CRF Name (CRF Module / Guideline) Sub Domain Name Domain Name Size Input Restrictions Min Value Max Value Measurement Type Source Form Set Form Field Domain CDASH Variable CDASH Definition CDASH Label Controlled Terminology Prompt Essentiality Question Text CDASH imp guidance SDTM IG target csDSR PhenX Data Type CRF Completion Inst SDTMIG Target Var SDTMIG Target Map Codelist Name PVs Pre Pop Value Query Display List Style
C60486 Integrating viral vector copy number blood GPA plus lineage value IntgrtVrlVCNBldGPAPlsLneagVal Value of the integrating viral vector copy number (VCN) in blood for the GPA+ lineage Value of the integrating viral vector copy number (VCN) in blood for the GPA+ lineage VCN in Specific Lineages: GPA+ Numeric Values Adult;Pediatric Supplemental 1.00 2021-04-22 13:25:18.0 Genotoxicity Safety Data Adverse Events/Toxicities

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C60518 Gene edited hematopoietic stem progenitor cells blood CD3 depleted on target indel count GnEdHSPCBldCD3DpltOnTgtIndCt Count of on-target indels related to CD3 depletion in gene-edited hematopoietic stem and progenitor cells (HSPC) in blood. Count of on-target indels related to CD3 depletion in gene-edited hematopoietic stem and progenitor cells (HSPC) in blood. On-target Indels Numeric Values Adult;Pediatric Supplemental 1.00 2021-04-23 11:12:55.0 Genotoxicity Safety Data Adverse Events/Toxicities

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C60454 Blast bone marrow percent value BlastBoneMarrowPctVal Value of percentage of blast(s) present in bone marrow aspirate/biopsy. Value of percentage of blast(s) present in bone marrow aspirate/biopsy. Blasts Percentage Numeric Values Adult;Pediatric Supplemental 1.00 2021-04-22 09:28:44.0 Genotoxicity Safety Data Adverse Events/Toxicities

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0 100 percent
C60465 Abnormal hematopoiesis onset date AbnormalHematopoiesisOnsetDate Date of onset of the abnormal hematopoiesis Date of onset of the abnormal hematopoiesis Date of onset: Date or Date & Time Adult;Pediatric Supplemental 1.00 2021-04-22 10:22:12.0 Genotoxicity Safety Data Adverse Events/Toxicities

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C60561 Genotoxicity pre manipulated cell genetic analysis chromatin immunoprecipitation indicator GntxcPrMnpCeGenAnlChIPInd Indicator of whether genetic analysis for chromatin immunoprecipitation (ChIP) was performed on pre-manipulated cells following detection of genotoxicity. Indicator of whether genetic analysis for chromatin immunoprecipitation (ChIP) was performed on pre-manipulated cells following detection of genotoxicity. If genotoxicity developed, were pre-manipulated cells genetically analyzed for ChIP? No;Yes No;Yes Alphanumeric Adult;Pediatric Supplemental 1.00 2021-04-23 14:49:40.0 Genotoxicity Safety Data Adverse Events/Toxicities

Single Pre-Defined Value Selected

C60497 Vector integration site analysis sorted fractionated cell population lineage text VISASortFracCellPopLinTxt Text field for identifying the sorted/fractionated cell population lineages on which Vector Integration Site Analysis (VISA) was performed. Text field for identifying the sorted/fractionated cell population lineages on which Vector Integration Site Analysis (VISA) was performed. Sorted/fractionated Cell Populations (specify lineages) Alphanumeric Adult;Pediatric Supplemental 1.00 2021-04-22 14:04:17.0 Genotoxicity Safety Data Adverse Events/Toxicities 100

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C60529 Gene editing conversion bone marrow mononuclear cell percent value GnEdConvBMMCPctVal Value of the percentage of gene editing conversions in bone marrow mononuclear cells (BMMC). Value of the percentage of gene editing conversions in bone marrow mononuclear cells (BMMC). Bone Marrow: BMMC Percent Conversion Numeric Values Adult;Pediatric Supplemental 1.00 2021-04-23 11:35:20.0 Genotoxicity Safety Data Adverse Events/Toxicities

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0 100 percent
C60540 Gene editing off target site confirm next generation sequencing indicator GnEdOfTgtSitCnfrmNGSInd Indicator of whether confirmation of off-target gene editing sites was obtained by next-generation sequencing (NGS). Indicator of whether confirmation of off-target gene editing sites was obtained by next-generation sequencing (NGS). Confirmed by NGS? No;Yes No;Yes Alphanumeric Adult;Pediatric Supplemental 1.00 2021-04-23 11:59:58.0 Genotoxicity Safety Data Adverse Events/Toxicities

Single Pre-Defined Value Selected

C60476 Abnormal hematopoiesis resolve indicator AbnrmlHmtopoiesisReslvInd Indicator of whether abnormal hematopoiesis has been resolved. Indicator of whether abnormal hematopoiesis has been resolved. Has the abnormal hematopoiesis resolved? No;Yes No;Yes Alphanumeric Adult;Pediatric Supplemental 1.00 2021-04-22 12:44:34.0 Genotoxicity Safety Data Adverse Events/Toxicities

Single Pre-Defined Value Selected

C60572 Infection new incidence potential product relate follow drug product infusion indicator InfNwInPtPrRlFlwDrPrInfInd Indicator of whether there was a new incidence of infection that is potentially product-related following drug product infusion. Indicator of whether there was a new incidence of infection that is potentially product-related following drug product infusion. Since drug product infusion has there been new incidence of infection (potentially product-related) No;Yes No;Yes Alphanumeric Adult;Pediatric Supplemental 1.00 2021-04-23 15:02:28.0 Genotoxicity Safety Data Adverse Events/Toxicities

Single Pre-Defined Value Selected

C60508 Clone integrant minimum 20 percent representation genetic location ClnIngrntMin20PctRepGeneLoc Location, relative to other genes on a DNA strand, of any clone(s) or integrant(s) having >=20 percent representation within the population of clones and integrants. Location, relative to other genes on a DNA strand, of any clone(s) or integrant(s) having >=20 percent representation within the population of clones and integrants. Is the location of the dominant integrant(s) Within or <50Kb of a known oncogene;Within or <50Kb of a Transcriptional Unit of any gene;Within or <50Kb of Cell signaling/proliferation Gene;Within or near the following genes known to be associated with insertional oncogenesis: LMO2, IKZF1, CCND2, HMGA2, MECOM Within, or fewer than 50 kilobases away from, of a known oncogene;Within, or fewer than 50 kilobases away from, a Transcriptional Unit of any gene;Within, or fewer than 50 kilobases away from, a Cell signaling/proliferation Gene;Within or near the following genes known to be associated with insertional oncogenesis: LMO2, IKZF1, CCND2, HMGA2, MECOM Alphanumeric Adult;Pediatric Supplemental 1.00 2021-04-22 16:30:50.0 Genotoxicity Safety Data Adverse Events/Toxicities

Multiple Pre-Defined Values Selected

C60093 Reticulocyte count ReticulocyteCt Count of reticulocytes Count of reticulocytes The latest Complete Blood Count Retic Count Numeric Values Adult;Pediatric Supplemental 1.00 2021-01-14 13:06:05.0 Genotoxicity Safety Data Adverse Events/Toxicities

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C60551 Gene editing site translocation clonality monitor method type GnEdTrClonMonMethTyp Type of method used to monitor clonality in translocation of gene editing sites. Type of method used to monitor clonality in translocation of gene editing sites. What method was used to monitor clonality: On-target indels;Whole exome sequencing;Other On-target indels;Whole exome sequencing;Other Alphanumeric Adult;Pediatric Supplemental 1.00 2021-04-23 13:32:03.0 Genotoxicity Safety Data Adverse Events/Toxicities

Multiple Pre-Defined Values Selected

C60487 Integrating viral vector copy number blood CD14 plus lineage value IntgrtVrlVCNBldCD14PlsLneagVal Value of the integrating viral vector copy number (VCN) in blood for the CD14+ lineage Value of the integrating viral vector copy number (VCN) in blood for the CD14+ lineage VCN in Specific Lineages: CD14+ Numeric Values Adult;Pediatric Supplemental 1.00 2021-04-22 13:25:18.0 Genotoxicity Safety Data Adverse Events/Toxicities

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C60519 Gene editing conversion blood CD3 plus lineage percent value GnEdConvBldCD3PlsLinePctVal Value of the percentage of gene-editing conversions in the blood CD3+ lineage. Value of the percentage of gene-editing conversions in the blood CD3+ lineage. Percent conversion in Specific Blood Lineages: CD3+ Numeric Values Adult;Pediatric Supplemental 1.00 2021-04-23 11:20:12.0 Genotoxicity Safety Data Adverse Events/Toxicities

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0 100 percent
C60455 Marrow hypoplasia indicator MarrowHypoplasiaInd Indicator of the presence of bone marrow hypoplasia Indicator of the presence of bone marrow hypoplasia Is there marrow hypoplasia? No;Yes No;Yes Alphanumeric Adult;Pediatric Supplemental 1.00 2021-04-22 09:35:25.0 Genotoxicity Safety Data Adverse Events/Toxicities

Single Pre-Defined Value Selected

C60466 Abnormal hematopoiesis treatment indicator AbnormalHematopoiesisTxInd Indicator of whether the abnormal hematopoiesis is being treated. Indicator of whether the abnormal hematopoiesis is being treated. Is the event being treated? No;Yes No;Yes Alphanumeric Adult;Pediatric Supplemental 1.00 2021-04-22 10:23:25.0 Genotoxicity Safety Data Adverse Events/Toxicities

Single Pre-Defined Value Selected

C60562 Genotoxicity pre manipulated cell genetic analysis chromatin immunoprecipitation method text GntxcPrMnpCeGenAnlChIPMthTxt Text describing the method used in genetic analysis for chromatin immunoprecipitation (ChIP) performed on pre-manipulated cells following detection of genotoxicity. Text describing the method used in genetic analysis for chromatin immunoprecipitation (ChIP) performed on pre-manipulated cells following detection of genotoxicity. If genotoxicity developed, were pre-manipulated cells genetically analyzed for ChIP? How: Alphanumeric Adult;Pediatric Supplemental 1.00 2021-04-23 14:49:40.0 Genotoxicity Safety Data Adverse Events/Toxicities 255

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C60498 Polyclonal population over 999 unique integration minimally fractionated cell specimen demonstrate gene modified hematopoiesis indicator PPO999UIMFCSDGMHInd Indicator of whether there is a polyclonal population of >= 1000 unique integrations of minimally fractionated cell specimens demonstrating gene-modified hematopoiesis. Indicator of whether there is a polyclonal population of >= 1000 unique integrations of minimally fractionated cell specimens demonstrating gene-modified hematopoiesis. Is there a "Rich" (polyclonal population with =1000 unique integrations) of minimally fractionated cell specimens (whole Bone Marrow, CD34+ cell product, whole blood) No;Yes No;Yes Alphanumeric Adult;Pediatric Supplemental 1.00 2021-04-22 14:11:42.0 Genotoxicity Safety Data Adverse Events/Toxicities

Single Pre-Defined Value Selected

C60530 Gene editing conversion bone marrow CD34 plus percent value GnEdConvBMCD34PlsPctVal Value of the percentage of gene editing conversions in bone marrow CD34+ cells. Value of the percentage of gene editing conversions in bone marrow CD34+ cells. Bone Marrow: CD34+ Percent Conversion Numeric Values Adult;Pediatric Supplemental 1.00 2021-04-23 11:35:20.0 Genotoxicity Safety Data Adverse Events/Toxicities

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0 100 percent
C60541 Gene editing off target site confirm other method indicator GnEdOfTgtSitCnfrmOthMethInd Indicator of whether confirmation of off-target gene editing sites was obtained by another method. Indicator of whether confirmation of off-target gene editing sites was obtained by another method. Confirmed by other method: No;Yes No;Yes Alphanumeric Adult;Pediatric Supplemental 1.00 2021-04-23 11:59:58.0 Genotoxicity Safety Data Adverse Events/Toxicities

Single Pre-Defined Value Selected

C60477 Abnormal hematopoiesis resolve date AbnrmlHmtopoiesResolvDate Date on which abnormal hematopoiesis was determined to have been resolved. Date on which abnormal hematopoiesis was determined to have been resolved. Date of resolution Date or Date & Time Adult;Pediatric Supplemental 1.00 2021-04-22 12:46:18.0 Genotoxicity Safety Data Adverse Events/Toxicities

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C60573 Infection new incidence potential product relate follow drug product infusion describe text InfNwInPtPrRlFlwDrPrInfDscTxt Text describing new incidence of infection that is potentially product-related following drug product infusion. Text describing new incidence of infection that is potentially product-related following drug product infusion. Since drug product infusion has there been new incidence of infection (potentially product-related)? If yes, describe: Alphanumeric Adult;Pediatric Supplemental 1.00 2021-04-23 15:02:28.0 Genotoxicity Safety Data Adverse Events/Toxicities 255

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C60509 Insertional oncogene near clone integrant minimum 20 percent representation name InsOncgnNrClnIntMin20PctRpName Name(s) of the gene(s) associated with insertional oncogenesis within or near which are located clone(s) or integrant(s) that have >=20 percent representation among the population of all clones and integrants. Name(s) of the gene(s) associated with insertional oncogenesis within or near which are located clone(s) or integrant(s) that have >=20 percent representation among the population of all clones and integrants. Is the location of the dominant integrant(s) within or near the following genes known to be associated with insertional oncogenesis LMO2;MECOM;HMGA2;CCND2;IKZF1 LMO2;MECOM;HMGA2;CCND2;IKZF1 Alphanumeric Adult;Pediatric Supplemental 1.00 2021-04-22 16:41:29.0 Genotoxicity Safety Data Adverse Events/Toxicities

Multiple Pre-Defined Values Selected

C60445 Genetically modified hematopoietic stem cell infusion date GMHemtpcStemCellInfsnDate Date of infusion of genetically modified (GM) hematopoietic stem cells (HSC). Date of infusion of genetically modified (GM) hematopoietic stem cells (HSC). Date of Genetically Modified HSC Infusion Date or Date & Time Adult;Pediatric Supplemental 1.00 2019-01-14 10:27:55.0 Genotoxicity Safety Data Adverse Events/Toxicities

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C60552 Gene editing site translocation clonality monitor method type other text GnEdTrClonMonMethTypOTH The free-text field related to "Gene editing site translocation clonality monitor method type", specifying other text. The free-text field related to "Gene editing site translocation clonality monitor method type", specifying other text. What method was used to monitor clonality: Other: Alphanumeric Adult;Pediatric Supplemental 1.00 2021-04-23 13:35:25.0 Genotoxicity Safety Data Adverse Events/Toxicities 4000

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C60488 Peripheral blood mononuclear cell positive for integrating viral vector percent value PBMCPostvFrIntgrtVrlVctrPctVal Value of the percentage of peripheral blood mononuclear cells (PBMC) positive for the integrating viral vector. Value of the percentage of peripheral blood mononuclear cells (PBMC) positive for the integrating viral vector. Percentage of Blood Cells Positive for Integrating Vector: PBMC Numeric Values Adult;Pediatric Supplemental 1.00 2021-04-22 13:33:03.0 Genotoxicity Safety Data Adverse Events/Toxicities

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0 100 percent
C60520 Gene editing conversion blood CD19 plus lineage percent value GnEdConvBldCD19PlsLinePctVal Value of the percentage of gene-editing conversions in the blood CD19+ lineage. Value of the percentage of gene-editing conversions in the blood CD19+ lineage. Percent conversion in Specific Blood Lineages: CD19+ Numeric Values Adult;Pediatric Supplemental 1.00 2021-04-23 11:20:12.0 Genotoxicity Safety Data Adverse Events/Toxicities

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0 100 percent
C60456 Marrow hypoplasia myeloid lineage name MrwHypoplsiaMyeldLineName Name of the myeloid lineage present in bone marrow hypoplasia. Name of the myeloid lineage present in bone marrow hypoplasia. Myeloid lineage Alphanumeric Adult;Pediatric Supplemental 1.00 2021-04-22 09:39:41.0 Genotoxicity Safety Data Adverse Events/Toxicities 100

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C60467 Abnormal hematopoiesis treatment type AbnormalHematopoiesisTxTyp Type of treatment undertaken for abnormal hematopoiesis. Type of treatment undertaken for abnormal hematopoiesis. Treatment HSC transplant;Chemotherapy;Transfusions Hematopoietic stem cell (HSC) transplant;Chemotherapy;Transfusions Alphanumeric Adult;Pediatric Supplemental 1.00 2021-04-22 10:25:09.0 Genotoxicity Safety Data Adverse Events/Toxicities

Multiple Pre-Defined Values Selected

C60563 Genotoxicity pre manipulated cell genetic analysis chromatin immunoprecipitation result text GntxcPrMnpCeGenAnlChIPRstTxt Text describing the result of genetic analysis for chromatin immunoprecipitation (ChIP) performed on pre-manipulated cells following detection of genotoxicity. Text describing the result of genetic analysis for chromatin immunoprecipitation (ChIP) performed on pre-manipulated cells following detection of genotoxicity. If genotoxicity developed, were pre-manipulated cells genetically analyzed for ChIP? State results: Alphanumeric Adult;Pediatric Supplemental 1.00 2021-04-23 14:49:40.0 Genotoxicity Safety Data Adverse Events/Toxicities 255

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C60499 Evidence polyclonal gene-modified hematopoiesis elapsed time since infusion category EvPoGeModHmtpsElTmSnInfCat Category of elapsed time since infusion when evidence was found of polyclonal gene-modified hematopoiesis. Category of elapsed time since infusion when evidence was found of polyclonal gene-modified hematopoiesis. Time Point 0/0.5 yr;1/1.5 yr;2/2.5 yr;3/3.5 yr;4/4.5 yr;5 yr;6 yr;7 yr;8 yr;9 yr;10 yr;11 yr;12 yr;13 yr;14 yr;15 yr 0/0.5 yr;1/1.5 yr;2/2.5 yr;3/3.5 yr;4/4.5 yr;5 yr;6 yr;7 yr;8 yr;9 yr;10 yr;11 yr;12 yr;13 yr;14 yr;15 yr Alphanumeric Adult;Pediatric Supplemental 1.00 2021-04-22 14:16:52.0 Genotoxicity Safety Data Adverse Events/Toxicities

Single Pre-Defined Value Selected

C60531 Gene editing conversion bone marrow colony forming cell percent value GnEdConvBMCFCPctVal Value of the percentage of gene editing conversions in bone marrow colony forming cells (CFC). Value of the percentage of gene editing conversions in bone marrow colony forming cells (CFC). Bone Marrow: CFC Percent Conversion Numeric Values Adult;Pediatric Supplemental 1.00 2021-04-23 11:35:20.0 Genotoxicity Safety Data Adverse Events/Toxicities

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0 100 percent
C60542 Gene editing off target site confirm other method indicator other text GnEdOfTgtSitCnfrmOthMethIndOTH The free-text field related to "Gene editing off target site confirm other method indicator" specifying other text. The free-text field related to "Gene editing off target site confirm other method indicator" specifying other text. Confirmed by other method: If yes, list: Alphanumeric Adult;Pediatric Supplemental 1.00 2021-04-23 11:59:58.0 Genotoxicity Safety Data Adverse Events/Toxicities 4000

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C60478 Integrating viral vector for hematopoietic stem progenitor cell therapy transgene component text IntViVeFHSPCTherTrnsgnCompTxt Text field for describing the transgene component(s) of the integrating viral vector(s) used for genetic manipulation of hematopoietic stem and progenitor cells. Text field for describing the transgene component(s) of the integrating viral vector(s) used for genetic manipulation of hematopoietic stem and progenitor cells. Transgene in the vector Alphanumeric Adult;Pediatric Supplemental 1.00 2021-04-22 13:06:17.0 Genotoxicity Safety Data Adverse Events/Toxicities 50

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C60510 Cell clone proportional increase last two analysis indicator CelClnProportnIncLst2AnlysInd Indicator of whether any cell clones have increased in proportion over the last two analyses. Indicator of whether any cell clones have increased in proportion over the last two analyses. Is there evidence of clonal expansion/Are any cell clones increasing in proportion over the last two analyses? No;Yes No;Yes Alphanumeric Adult;Pediatric Supplemental 1.00 2021-04-22 16:49:24.0 Genotoxicity Safety Data Adverse Events/Toxicities

Single Pre-Defined Value Selected

C60446 Autologous transplant genetically modified hematopoietic stem cell infusion elapsed time category AutlgTPGMHSCInfsElpsTimCat Category of time elapsed since autologous transplant (TP) and genetically modified (GM) hematopoietic stem cell (HSC) infusion. Category of time elapsed since autologous transplant (TP) and genetically modified (GM) hematopoietic stem cell (HSC) infusion. Time since autologous transplant and Genetically Modified HSC Infusion): Day 0;3 months;6 months;1/1.5 yr;2/2.5 yr;3/3.5 yr;4/4.5 yr;5 yr;6 yr;7 yr;8 yr;9 yr;10 yr;11 yr;12 yr;13 yr;14 yr;15 yr Day 0;3 months;6 months;1/1.5 yr;2/2.5 yr;3/3.5 yr;4/4.5 yr;5 yr;6 yr;7 yr;8 yr;9 yr;10 yr;11 yr;12 yr;13 yr;14 yr;15 yr Alphanumeric Adult;Pediatric Supplemental 1.00 2021-04-21 16:32:23.0 Genotoxicity Safety Data Adverse Events/Toxicities

Single Pre-Defined Value Selected

137 results.
CSV