CDE Detailed Report
Disease: Sickle Cell Disease
Sub-Domain: Safety Data
CRF: Genotoxicity
Sub-Domain: Safety Data
CRF: Genotoxicity
| 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 |
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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 |
Free-Form Entry |
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 |
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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 |
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| 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 |
Free-Form Entry |
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| 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 |
Free-Form Entry |
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 |
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| 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 |
Free-Form Entry |
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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 |
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| 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 |
Free-Form Entry |
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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 |
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| 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 |
Free-Form Entry |
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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 |
Free-Form Entry |
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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 |
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| 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 |
Free-Form Entry |
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 |
Free-Form Entry |
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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 |
Free-Form Entry |
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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 |
Free-Form Entry |
0 | 100 | percent | ||||||||||||||||||||||||||||||
| 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 |
Free-Form Entry |
0 | 100 | percent | ||||||||||||||||||||||||||||||
| 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 |
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| 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 |
Free-Form Entry |
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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 |
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| 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 |
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| 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 |
Free-Form Entry |
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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 |
Free-Form Entry |
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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 |
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| C60521 | Gene editing conversion blood CD15 plus lineage percent value | GnEdConvBldCD15PlsLinePctVal | Value of the percentage of gene-editing conversions in the blood CD15+ lineage. | Value of the percentage of gene-editing conversions in the blood CD15+ lineage. | Percent conversion in Specific Blood Lineages: CD15+ | Numeric Values | Adult;Pediatric | Supplemental | 1.00 | 2021-04-23 11:20:12.0 | Genotoxicity | Safety Data | Adverse Events/Toxicities |
Free-Form Entry |
0 | 100 | percent | ||||||||||||||||||||||||||||||
| C60457 | Marrow hypoplasia megakaryotic text | MrwHypoplsiaMegakaryoticTxt | Text describing the megakaryotic component of bone marrow hypoplasia. | Text describing the megakaryotic component of bone marrow hypoplasia. | Megakaryotic | Alphanumeric | Adult;Pediatric | Supplemental | 1.00 | 2021-04-22 09:39:41.0 | Genotoxicity | Safety Data | Adverse Events/Toxicities | 100 |
Free-Form Entry |
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| C60553 | Gene editing any associated cell clone minimum 20 percent clone population indicator | GnEdAnAsCeClMin20PctClPopInd | Indicator of whether any cell clone(s) associated with gene editing comprise >=20 percent of the population of clones. | Indicator of whether any cell clone(s) associated with gene editing comprise >=20 percent of the population of clones. | Do any cell clone/s associated with an editing event account for >=20% of all clones? | No;Yes | No;Yes | Alphanumeric | Adult;Pediatric | Supplemental | 1.00 | 2021-04-23 13:38:09.0 | Genotoxicity | Safety Data | Adverse Events/Toxicities |
Single Pre-Defined Value Selected |
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| C60489 | Burst forming unit erythroid cell positive for integrating viral vector percent value | BFUEPostvFrIntgrtVrlVctrPctVal | Value of the percentage of burst-forming unit - erythroid (BFU-E) cells positive for the integrating viral vector. | Value of the percentage of burst-forming unit - erythroid (BFU-E) cells positive for the integrating viral vector. | Percentage of Blood Cells Positive for Integrating Vector: BFU-E | Numeric Values | Adult;Pediatric | Supplemental | 1.00 | 2021-04-22 13:33:03.0 | Genotoxicity | Safety Data | Adverse Events/Toxicities |
Free-Form Entry |
0 | 100 | percent | ||||||||||||||||||||||||||||||
| C60532 | Gene editing on target indel bone marrow mononuclear cell count | GnEdOnTgtIndelBMMCCt | Count of on-target indels in gene editing of bone marrow mononuclear cells (BMMC). | Count of on-target indels in gene editing of bone marrow mononuclear cells (BMMC). | Bone Marrow: BMMC On-target Indels | Numeric Values | Adult;Pediatric | Supplemental | 1.00 | 2021-04-23 11:40:19.0 | Genotoxicity | Safety Data | Adverse Events/Toxicities |
Free-Form Entry |
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| C60468 | Transfusion blood product type | TransfusionBloodProductTyp | Type(s) of blood product(s) introduced as part of transfusion. | Type(s) of blood product(s) introduced as part of transfusion. | Blood Product | Alphanumeric | Adult;Pediatric | Supplemental | 1.00 | 2021-04-22 10:29:39.0 | Genotoxicity | Safety Data | Adverse Events/Toxicities | 100 |
Free-Form Entry |
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| C60564 | Malignancy new follow drug product infusion indicator | MlgnNwFlwDrgPrdInfsInd | Indicator of whether new malignancies were detected following infusion of drug product. | Indicator of whether new malignancies were detected following infusion of drug product. | Since drug product infusion has there been. New malignancy(ies)? | No;Yes | No;Yes | Alphanumeric | Adult;Pediatric | Supplemental | 1.00 | 2021-04-23 14:56:07.0 | Genotoxicity | Safety Data | Adverse Events/Toxicities |
Single Pre-Defined Value Selected |
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| C60500 | Evidence polyclonal gene-modified hematopoiesis specimen type | EvidPlyclnlGnModHmtpsSpecTyp | Type of specimen in which evidence was found of polyclonal gene-modified hematopoiesis. | Type of specimen in which evidence was found of polyclonal gene-modified hematopoiesis. | Specimen Type | Alphanumeric | Adult;Pediatric | Supplemental | 1.00 | 2021-04-22 14:23:03.0 | Genotoxicity | Safety Data | Adverse Events/Toxicities | 25 |
Free-Form Entry |
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| C60447 | Hematology since previous report date status | HematolSncPrevRptDtStatus | Status of subject/participant's hematology since the date of the previous genotoxicity report. | Status of subject/participant's hematology since the date of the previous genotoxicity report. | Hematological Status since the date of last report: | Concerning;Non-concerning | Concerning;Non-concerning | Alphanumeric | Adult;Pediatric | Supplemental | 1.00 | 2021-04-21 16:43:01.0 | Genotoxicity | Safety Data | Adverse Events/Toxicities |
Single Pre-Defined Value Selected |
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| C60543 | Gene editing off target site acceptable rate confirm indicator | GnEdOfTgtSitAcceptRtCnfrmInd | Indicator of whether an acceptable editing rate was confirmed for each off-target gene editing site. | Indicator of whether an acceptable editing rate was confirmed for each off-target gene editing site. | Acceptable editing rate confirmed for each off-target editing site: | No;Yes | No;Yes | Alphanumeric | Adult;Pediatric | Supplemental | 1.00 | 2021-04-23 12:08:45.0 | Genotoxicity | Safety Data | Adverse Events/Toxicities |
Single Pre-Defined Value Selected |
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| C60479 | Integrating viral vector for hematopoietic stem progenitor cell therapy promoter enhancer component text | IntViVeFHSPCTherProEnhCompTxt | Text field for describing the promoter and/or enhancer component(s) of the integrating viral vector(s) used for genetic manipulation of hematopoietic stem and progenitor cells. | Text field for describing the promoter and/or enhancer component(s) of the integrating viral vector(s) used for genetic manipulation of hematopoietic stem and progenitor cells. | Promoter/Enhancers in vector | Alphanumeric | Adult;Pediatric | Supplemental | 1.00 | 2021-04-22 13:06:17.0 | Genotoxicity | Safety Data | Adverse Events/Toxicities | 50 |
Free-Form Entry |
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| C60511 | Cell clone proportional increase last two analysis identifier text | CelClnPrprtnIncrLst2AnlysIdTxt | Text field for identifying cell clone(s) that have increased as a proportion of the population of clones over the last two analyses. | Text field for identifying cell clone(s) that have increased as a proportion of the population of clones over the last two analyses. | Clone | Alphanumeric | Adult;Pediatric | Supplemental | 1.00 | 2021-04-22 16:51:48.0 | Genotoxicity | Safety Data | Adverse Events/Toxicities | 25 |
Free-Form Entry |
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| C60522 | Gene editing conversion blood GPA plus lineage percent value | GnEdConvBldGPAPlsLinePctVal | Value of the percentage of gene-editing conversions in the blood GPA+ lineage. | Value of the percentage of gene-editing conversions in the blood GPA+ lineage. | Percent conversion 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 |
Free-Form Entry |
0 | 100 | percent | ||||||||||||||||||||||||||||||
| C60458 | Bone marrow myeloid erythroid cell ratio text | BonMrwMyldErythrdCelRatioTxt | Text field for capturing the ratio of maturing myeloid cells to erythroid cells in the bone marrow. | Text field for capturing the ratio of maturing myeloid cells to erythroid cells in the bone marrow. | What was M:E ratio: | Alphanumeric | Adult;Pediatric | Supplemental | 1.00 | 2021-04-22 09:44:38.0 | Genotoxicity | Safety Data | Adverse Events/Toxicities | 10 |
Free-Form Entry |
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| C60554 | Gene editing off target cell clone minimum 20 percent clone population gene location | GnEdOfTgCeClMn20PcClPopGnLoc | Location of the gene containing, or nearest flanking gene to, the site of the off-target gene-editing clone that comprises >=20 percent of the population of all clones. | Location of the gene containing, or nearest flanking gene to, the site of the off-target gene-editing clone that comprises >=20 percent of the population of all clones. | List the site of off-target gene editing of dominant (=20% representation) clone(s) [e.g. gene location or flanking gene location], if known | Alphanumeric | Adult;Pediatric | Supplemental | 1.00 | 2021-04-23 13:41:43.0 | Genotoxicity | Safety Data | Adverse Events/Toxicities | 25 |
Free-Form Entry |
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| C60490 | Integrating viral vector copy number bone marrow mononuclear cell value | IntgrtVrlVCNBMMCVal | Value of the integrating viral vector copy number (VCN) in bone marrow for bone marrow mononuclear cells (BMMC). | Value of the integrating viral vector copy number (VCN) in bone marrow for bone marrow mononuclear cells (BMMC). | Bone Marrow VCN: BMMC | Numeric Values | Adult;Pediatric | Supplemental | 1.00 | 2021-04-22 13:25:18.0 | Genotoxicity | Safety Data | Adverse Events/Toxicities |
Free-Form Entry |
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| C04807 | Vital status | VitStatus | Status of participant/subject as alive or dead | Status of participant/subject as alive or dead | Survival Status | Alive;Dead | Alive;Dead | Alphanumeric | Adult;Pediatric | Supplemental | 3.00 | 2013-07-20 10:21:25.65 | Genotoxicity | Safety Data | Adverse Events/Toxicities |
Single Pre-Defined Value Selected |
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| C60533 | Gene editing on target indel bone marrow CD34 plus cell count | GnEdOnTgtIndelBMCD34PlsCellCt | Count of on-target indels in gene editing of bone marrow CD34+ cells. | Count of on-target indels in gene editing of bone marrow CD34+ cells. | Bone Marrow: CD34+ On-target Indels | Numeric Values | Adult;Pediatric | Supplemental | 1.00 | 2021-04-23 11:40:19.0 | Genotoxicity | Safety Data | Adverse Events/Toxicities |
Free-Form Entry |
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| C60469 | Transfusion frequency text | TransfusionFrequencyTxt | Text field for capturing frequency of blood transfusions | Text field for capturing frequency of blood transfusions | Transfusion frequency | Alphanumeric | Adult;Pediatric | Supplemental | 1.00 | 2021-04-22 10:31:04.0 | Genotoxicity | Safety Data | Adverse Events/Toxicities | 50 |
Free-Form Entry |
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| C60565 | Malignancy new follow drug product infusion describe text | MlgnNwFlwDrgPrdInfsDscTxt | Text describing new malignancies detected following infusion of drug product. | Text describing new malignancies detected following infusion of drug product. | Since drug product infusion has there been. New malignancy(ies)? If yes, describe: | Alphanumeric | Adult;Pediatric | Supplemental | 1.00 | 2021-04-23 14:56:07.0 | Genotoxicity | Safety Data | Adverse Events/Toxicities | 100 |
Free-Form Entry |
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| C60501 | Evidence polyclonal gene-modified hematopoiesis unique integrand count | EvPlyclnlGnMdHmtpsUniqIngrndCt | Count of unique integrands of the selected specimen type displaying evidence of gene-modified hematopoiesis. | Count of unique integrands of the selected specimen type displaying evidence of gene-modified hematopoiesis. | Number of unique integrands | Numeric Values | Adult;Pediatric | Supplemental | 1.00 | 2021-04-22 14:24:48.0 | Genotoxicity | Safety Data | Adverse Events/Toxicities |
Free-Form Entry |
0 | ||||||||||||||||||||||||||||||||
| C60448 | Bone marrow aspirate biopsy prior genetically modified hematopoietic stem cell infusion indicator | BoMwAspBpsPrGMHSCInfsInd | Indicator of whether bone marrow aspirate or biopsy was performed prior to infusion of genetically modified (GM) hematopoietic stem cells (HSC). | Indicator of whether bone marrow aspirate or biopsy was performed prior to infusion of genetically modified (GM) hematopoietic stem cells (HSC). | Was bone marrow aspirate/biopsy done prior to infusion? | No;Yes | No;Yes | Alphanumeric | Adult;Pediatric | Supplemental | 1.00 | 2021-04-21 16:53:07.0 | Genotoxicity | Safety Data | Adverse Events/Toxicities |
Single Pre-Defined Value Selected |
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| C60544 | Gene editing off target site dominant location | GnEdOffTgtSiteDominantLoc | Location(s) of the dominant off-target gene editing site(s). | Location(s) of the dominant off-target gene editing site(s). | What is the location of the dominant off-target site(s): | Alphanumeric | Adult;Pediatric | Supplemental | 1.00 | 2021-04-23 13:04:33.0 | Genotoxicity | Safety Data | Adverse Events/Toxicities | 100 |
Free-Form Entry |
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| C60480 | Integrating viral vector copy number assessment type | IntgrtVrlVctrCpyNumAssessTyp | Type of assessment(s) of vector copy number (VCN) performed for the integrating viral vector products. | Type of assessment(s) of vector copy number (VCN) performed for the integrating viral vector products. | Vector Copy Number (VCN) performed in | Blood VCN;Bone marrow VCN;VCN in specific lineages;Percentage of Blood Cells Positive for Integrating Vector;Percentage of Bone Marrow Cells Positive for Integrating Vector | Blood vector copy number (VCN);Bone marrow vector copy number (VCN);Vector copy number (VCN) in specific lineages;Percentage of Blood Cells Positive for Integrating Vector;Percentage of Bone Marrow Cells Positive for Integrating Vector | Alphanumeric | Adult;Pediatric | Supplemental | 1.00 | 2021-04-22 13:12:48.0 | Genotoxicity | Safety Data | Adverse Events/Toxicities |
Multiple Pre-Defined Values Selected |
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| C60512 | Cell clone proportional increase last two analysis previous proportion percent value | ClClnPrpIncLst2AnlPrvPrpPctVal | Value as a percent of the proportion of all cell clones, that the specified cell clone showing proportional increase over the last two analyses presented during the previous analysis. | Value as a percent of the proportion of all cell clones, that the specified cell clone showing proportional increase over the last two analyses presented during the previous analysis. | Last % representation/abundance | Numeric Values | Adult;Pediatric | Supplemental | 1.00 | 2021-04-22 16:53:58.0 | Genotoxicity | Safety Data | Adverse Events/Toxicities |
Free-Form Entry |
0 | 100 | percent |