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

Displaying 1 - 50 of 137
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
C60559 Whole genome sequencing follow genotoxic event detection indicator WhlGnmSqncFlwGntxcEvntDtctnInd Indicator of whether whole genome sequencing was performed following detection of a genotoxic event. Indicator of whether whole genome sequencing was performed following detection of a genotoxic event. If genotoxic event detected was whole genome sequencing used? No;Yes No;Yes Alphanumeric Adult;Pediatric Supplemental 1.00 2021-04-23 14:37:50.0 Genotoxicity Safety Data Adverse Events/Toxicities

Single Pre-Defined Value Selected

C60495 Bone marrow colony forming cell positive for integrating viral vector percent value BMCFCPstvFrIntVrlVctPctVal Value of the percentage of bone marrow colony forming cells (CFC) positive for the integrating viral vector. Value of the percentage of bone marrow colony forming cells (CFC) positive for the integrating viral vector. Percentage of Bone Marrow Cells Positive for Integrating Vector: CFC 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
C60527 Gene editing blood GPA plus lineage on target indel count GnEdBldGPAPlsLinOnTgtIndelCt Count of gene-editing on-target indels in the blood GPA+ lineage. Count of gene-editing on-target indels in the blood GPA+ lineage. On-target Indels in Specific Blood Lineages: GPA+ Numeric Values Adult;Pediatric Supplemental 1.00 2021-04-23 11:20:12.0 Genotoxicity Safety Data Adverse Events/Toxicities

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C60463 Genetic mutational panel indicator GeneticMutationalPanelInd Indicator of whether a genetic mutational panel was performed. Indicator of whether a genetic mutational panel was performed. Was a genetic mutational panel performed? No;Yes No;Yes Alphanumeric Adult;Pediatric Supplemental 1.00 2021-04-22 10:16:11.0 Genotoxicity Safety Data Adverse Events/Toxicities

Single Pre-Defined Value Selected

C60474 Bone marrow aspirate result type BonMarowAspiratResultTyp Type(s) of results obtained from bone marrow aspiration. Type(s) of results obtained from bone marrow aspiration. Bone Marrow Aspirate Performed? Abnormal cytogenetics;Normal cytogenetics;Dysplastic morphology;Malignant morphology;Normal morphology Abnormal cytogenetics;Normal cytogenetics;Dysplastic morphology;Malignant morphology;Normal morphology Alphanumeric Adult;Pediatric Supplemental 1.00 2021-04-22 12:02:30.0 Genotoxicity Safety Data Adverse Events/Toxicities

Multiple Pre-Defined Values Selected

C60570 Hematologic disorder new incidence follow drug product infusion indicator HmDsNwInFlwDrPrInfInd Indicator of whether there was a new incidence of hematologic disorder following drug product infusion. Indicator of whether there was a new incidence of hematologic disorder following drug product infusion. Since drug product infusion has there been new incidence of a hematologic disorder. 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

C60506 Clone integrant minimum 20 percent representation integration site gene location ClnIntgrMn20PctRpIntgrtStGnLoc Location of the integration site (gene or flanking gene) of clone(s) or integrant(s) having >=20 percent representation in the clone population. Location of the integration site (gene or flanking gene) of clone(s) or integrant(s) having >=20 percent representation in the clone population. List the site of integration of dominant (>=20% representation) clone(s) [e.g. gene location or flanking gene location] Alphanumeric Adult;Pediatric Supplemental 1.00 2021-04-22 14:42:22.0 Genotoxicity Safety Data Adverse Events/Toxicities 25

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C59958 White blood cell count WhiteBloodCellCt Count of white blood cells (WBC) in the subject/participant's complete blood cell (CBC) sample. Count of white blood cells (WBC) in the subject/participant's complete blood cell (CBC) sample. The latest Complete Blood Count WBC Numeric Values Adult;Pediatric Supplemental 1.00 2020-09-01 13:36:15.0 Genotoxicity Safety Data Adverse Events/Toxicities

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CIBMTR n/a Form 2031 revision 2 10. WBC:
C60538 Gene editing off target site confirm count GnEdOffTgtSiteConfirmCt Count of off-target gene editing sites confirmed. Count of off-target gene editing sites confirmed. Number of off-target editing sites confirmed: Numeric Values Adult;Pediatric Supplemental 1.00 2021-04-23 11:57:22.0 Genotoxicity Safety Data Adverse Events/Toxicities

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C60485 Integrating viral vector copy number blood CD15 plus lineage value IntgrtVrlVCNBldCD15PlsLneagVal Value of the integrating viral vector copy number (VCN) in blood for the CD15+ lineage Value of the integrating viral vector copy number (VCN) in blood for the CD15+ lineage VCN in Specific Lineages: CD15+ Numeric Values Adult;Pediatric Supplemental 1.00 2021-04-22 13:25:18.0 Genotoxicity Safety Data Adverse Events/Toxicities

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C60517 Gene edited hematopoietic stem progenitor cells blood CD3 depleted conversion percent value GenEdHSPCBldCD3DepltCnvPctVal Value of the percentage of conversions related to CD3 depletion in gene-edited hematopoietic stem and progenitor cells (HSPC) in blood. Value of the percentage of conversions related to CD3 depletion in gene-edited hematopoietic stem and progenitor cells (HSPC) in blood. Percent Conversion Numeric Values Adult;Pediatric Supplemental 1.00 2021-04-23 11:09:53.0 Genotoxicity Safety Data Adverse Events/Toxicities

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0 100 percent
C60453 Blast bone marrow type BlastBoneMarrowTyp Type of blast(s) present in bone marrow aspirate/biopsy. Type of blast(s) present in bone marrow aspirate/biopsy. Blasts type Alphanumeric Adult;Pediatric Supplemental 1.00 2021-04-22 09:28:44.0 Genotoxicity Safety Data Adverse Events/Toxicities 255

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C60549 Gene editing site translocation on off target droplet digital polymerase chain reaction detection limit text GnEdStTrOnOfTgDrDgPCRDtLmTxt Text field for capturing the droplet digital polymerase chain reaction (PCR) detection limit for translocations of on target and off target gene editing sites. Text field for capturing the droplet digital polymerase chain reaction (PCR) detection limit for translocations of on target and off target gene editing sites. Translocations between on-target and off-target editing sites detected by: Droplet Digital PCR: If no, what is the limit of detection: Alphanumeric Adult;Pediatric Supplemental 1.00 2021-04-23 13:24:46.0 Genotoxicity Safety Data Adverse Events/Toxicities 50

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C60560 Post genotoxic event oncogene panel whole exome genome sequencing result text PstGntxcEvOncPnlWhlExGnSqRsTxt Text describing the results of oncogene panel, whole exome sequencing, or whole genome sequencing performed following a genotoxic event. Text describing the results of oncogene panel, whole exome sequencing, or whole genome sequencing performed following a genotoxic event. State results Alphanumeric Adult;Pediatric Supplemental 1.00 2021-04-23 14:43:36.0 Genotoxicity Safety Data Adverse Events/Toxicities 255

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C60496 Vector integration site analysis substrate type VISASubstrateTyp Type of substrate(s) on which Vector Integration Site Analysis (VISA) was performed. Type of substrate(s) on which Vector Integration Site Analysis (VISA) was performed. Vector Integration Site Analysis (VISA) performed in Blood;Bone marrow;Sorted/fractionated cell populations Blood;Bone marrow;Sorted/fractionated cell populations Alphanumeric Adult;Pediatric Supplemental 1.00 2021-04-22 14:01:20.0 Genotoxicity Safety Data Adverse Events/Toxicities

Multiple Pre-Defined Values Selected

C60528 Gene editing blood CD14 plus lineage on target indel count GnEdBldCD14PlsLinOnTgtIndelCt Count of gene-editing on-target indels in the blood CD14+ lineage. Count of gene-editing on-target indels in the blood CD14+ lineage. On-target Indels in Specific Blood Lineages: CD14+ Numeric Values Adult;Pediatric Supplemental 1.00 2021-04-23 11:20:12.0 Genotoxicity Safety Data Adverse Events/Toxicities

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C60464 Abnormal hematopoiesis best descriptor type AbnormHematopoieBstDscrpTyp Type of descriptor that best characterizes the abnormal hematopoiesis identified. Type of descriptor that best characterizes the abnormal hematopoiesis identified. What best describes the abnormal hematopoiesis? MDS;Leukemia/lymphoma Myelodysplastic syndrome;Leukemia/lymphoma Alphanumeric Adult;Pediatric Supplemental 1.00 2021-04-22 10:17:46.0 Genotoxicity Safety Data Adverse Events/Toxicities

Single Pre-Defined Value Selected

C60475 Bone marrow aspirate cytogenetic abnormality describe text BnMrwAsprtCytgntcAbnormDscrTxt Text field for describing cytogenetic abnormalities identified in bone marrow aspirate. Text field for describing cytogenetic abnormalities identified in bone marrow aspirate. Cytogenetic Abnormalities (describe) Alphanumeric Adult;Pediatric Supplemental 1.00 2021-04-22 12:06:45.0 Genotoxicity Safety Data Adverse Events/Toxicities 255

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C60571 Hematologic disorder new incidence follow drug product infusion describe text HmDsNwInFlwDrPrInfDscTxt Text describing new incidence of hematologic disorder following drug product infusion. Text describing new incidence of hematologic disorder following drug product infusion. Since drug product infusion has there been new incidence of a hematologic disorder. 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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C60507 Clone integrant minimum 20 percent representation value ClnIntgrntMin20PctRepVal Value, as a percentage, of the representation among all clones/integrants of the clones/integrants at the specified gene or flanking gene location, for clones/integrants comprising >=20 percent of all clones/integrants. Value, as a percentage, of the representation among all clones/integrants of the clones/integrants at the specified gene or flanking gene location, for clones/integrants comprising >=20 percent of all clones/integrants. Percent Representation/Relative Abundance Numeric Values Adult;Pediatric Supplemental 1.00 2021-04-22 14:46:13.0 Genotoxicity Safety Data Adverse Events/Toxicities

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20 100 percent
C59968 Platelet count value PlateletCountVal Value of the count of platelets in the subject/participant's complete blood cell (CBC) sample. Value of the count of platelets in the subject/participant's complete blood cell (CBC) sample. The latest Complete Blood Count Plt Numeric Values Adult;Pediatric Supplemental 1.00 2020-09-01 13:36:15.0 Genotoxicity Safety Data Adverse Events/Toxicities

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CIBMTR n/a Form 2031 revision 2 15: Platelets
C60539 Gene editing off target site confirm GUIDE seq check indicator GnEdOfTgtSitCnfrmGUIDESeqChInd Indicator of whether confirmation of off-target gene editing sites involved checking by GUIDE-seq. Indicator of whether confirmation of off-target gene editing sites involved checking by GUIDE-seq. Checked by GUIDE-seq? 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

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
C60550 Gene editing site translocation on off target droplet digital polymerase chain reaction detection frequency text GnEdStTrOnOfTgDrDgPCRDtFrqTxt Text field for reporting the frequency of detection of translocation of on target and off target gene editing sites using droplet digital polymerase chain reaction (PCR). Text field for reporting the frequency of detection of translocation of on target and off target gene editing sites using droplet digital polymerase chain reaction (PCR). Translocations between on-target and off-target editing sites detected by: Droplet Digital PCR: If yes, what is the frequency of detection: Alphanumeric Adult;Pediatric Supplemental 1.00 2021-04-23 13:29:02.0 Genotoxicity Safety Data Adverse Events/Toxicities 50

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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
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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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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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

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

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

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
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

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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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

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
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