Ignite Creation Date:
2025-12-25 @ 4:11 AM
Ignite Modification Date:
2025-12-26 @ 3:09 AM
Study NCT ID:
NCT02162420
Status:
COMPLETED
Last Update Posted:
2025-05-08
First Post:
2014-06-10
Is NOT Gene Therapy:
False
Has Adverse Events:
True
Brief Title:
Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic Anemia
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D019871', 'term': 'Dyskeratosis Congenita'}, {'id': 'D000741', 'term': 'Anemia, Aplastic'}], 'ancestors': [{'id': 'D000080984', 'term': 'Congenital Bone Marrow Failure Syndromes'}, {'id': 'D000080983', 'term': 'Bone Marrow Failure Disorders'}, {'id': 'D001855', 'term': 'Bone Marrow Diseases'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D012868', 'term': 'Skin Abnormalities'}, {'id': 'D000013', 'term': 'Congenital Abnormalities'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D040181', 'term': 'Genetic Diseases, X-Linked'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D012873', 'term': 'Skin Diseases, Genetic'}, {'id': 'D012871', 'term': 'Skin Diseases'}, {'id': 'D017437', 'term': 'Skin and Connective Tissue Diseases'}, {'id': 'D000740', 'term': 'Anemia'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D000074323', 'term': 'Alemtuzumab'}, {'id': 'C024352', 'term': 'fludarabine'}, {'id': 'C042382', 'term': 'fludarabine phosphate'}, {'id': 'D003520', 'term': 'Cyclophosphamide'}, {'id': 'D014916', 'term': 'Whole-Body Irradiation'}, {'id': 'D033581', 'term': 'Stem Cell Transplantation'}, {'id': 'D000961', 'term': 'Antilymphocyte Serum'}, {'id': 'C512542', 'term': 'thymoglobulin'}], 'ancestors': [{'id': 'D061067', 'term': 'Antibodies, Monoclonal, Humanized'}, {'id': 'D000911', 'term': 'Antibodies, Monoclonal'}, {'id': 'D000906', 'term': 'Antibodies'}, {'id': 'D007136', 'term': 'Immunoglobulins'}, {'id': 'D007162', 'term': 'Immunoproteins'}, {'id': 'D001798', 'term': 'Blood Proteins'}, {'id': 'D011506', 'term': 'Proteins'}, {'id': 'D000602', 'term': 'Amino Acids, Peptides, and Proteins'}, {'id': 'D012712', 'term': 'Serum Globulins'}, {'id': 'D005916', 'term': 'Globulins'}, {'id': 'D010752', 'term': 'Phosphoramide Mustards'}, {'id': 'D009588', 'term': 'Nitrogen Mustard Compounds'}, {'id': 'D009150', 'term': 'Mustard Compounds'}, {'id': 'D006846', 'term': 'Hydrocarbons, Halogenated'}, {'id': 'D006838', 'term': 'Hydrocarbons'}, {'id': 'D009930', 'term': 'Organic Chemicals'}, {'id': 'D063088', 'term': 'Phosphoramides'}, {'id': 'D009943', 'term': 'Organophosphorus Compounds'}, {'id': 'D011878', 'term': 'Radiotherapy'}, {'id': 'D013812', 'term': 'Therapeutics'}, {'id': 'D008919', 'term': 'Investigative Techniques'}, {'id': 'D017690', 'term': 'Cell Transplantation'}, {'id': 'D064987', 'term': 'Cell- and Tissue-Based Therapy'}, {'id': 'D001691', 'term': 'Biological Therapy'}, {'id': 'D014180', 'term': 'Transplantation'}, {'id': 'D013514', 'term': 'Surgical Procedures, Operative'}, {'id': 'D007106', 'term': 'Immune Sera'}, {'id': 'D001688', 'term': 'Biological Products'}, {'id': 'D045424', 'term': 'Complex Mixtures'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'ebens012@umn.edu', 'phone': '612-624-4511', 'title': 'Christen Ebens, MD, MPH', 'organization': 'Masonic Cancer Center'}, 'certainAgreement': {'piSponsorEmployee': True}}, 'adverseEventsModule': {'timeFrame': '2 years', 'description': 'Adverse event and serious adverse event reporting is focused on protocol related endpoint data, as well as risks associated with research data collection.\n\nProtocol related endpoint data includes:\n\n* neutrophil engraftment and platelet engraftment\n* regimen related morality\n* acute GVHD at\n* chronic GVHD at\n* secondary malignancies\n\nThere were no reportable adverse events in regards to research data collection (e.g. breach in confidentiality, etc.).', 'eventGroups': [{'id': 'EG000', 'title': 'Arm A: Dyskeratosis Congenita, Non-Haploidentical Donor', 'description': 'Arm A Dyskeratosis Congenita (DKC) (non-haploidentical donor)', 'otherNumAtRisk': 12, 'deathsNumAtRisk': 12, 'otherNumAffected': 0, 'seriousNumAtRisk': 12, 'deathsNumAffected': 1, 'seriousNumAffected': 0}, {'id': 'EG001', 'title': 'Arm B: Severe Aplastic Anemia, Non-Matched Related, Non-Haploidentical Donor', 'description': 'Arm B: Severe Aplastic Anemia (SAA ) (non-matched related, non-haploidentical donor)', 'otherNumAtRisk': 18, 'deathsNumAtRisk': 18, 'otherNumAffected': 0, 'seriousNumAtRisk': 18, 'deathsNumAffected': 3, 'seriousNumAffected': 0}, {'id': 'EG002', 'title': 'Arm C: Severe Aplastic Anemia, Matched Related Donor', 'description': 'Arm C: Severe Aplastic Anemia (matched related donor)', 'otherNumAtRisk': 23, 'deathsNumAtRisk': 23, 'otherNumAffected': 0, 'seriousNumAtRisk': 23, 'deathsNumAffected': 1, 'seriousNumAffected': 0}, {'id': 'EG003', 'title': 'Arm D: Dyskeratosis Congenita, PTCy Platform', 'description': 'Arm D: Dyskeratosis Congenita (DKC), PTCy platform', 'otherNumAtRisk': 0, 'deathsNumAtRisk': 0, 'otherNumAffected': 0, 'seriousNumAtRisk': 0, 'deathsNumAffected': 0, 'seriousNumAffected': 0}, {'id': 'EG004', 'title': 'Arm E: Severe Aplastic Anemia, PTCy Platform', 'description': 'Arm E: Severe Aplastic Anemia (SAA),PTCy platform', 'otherNumAtRisk': 8, 'deathsNumAtRisk': 8, 'otherNumAffected': 0, 'seriousNumAtRisk': 8, 'deathsNumAffected': 0, 'seriousNumAffected': 0}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Incidence of Neutrophil Engraftment', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}, {'value': '18', 'groupId': 'OG001'}, {'value': '23', 'groupId': 'OG002'}, {'value': '8', 'groupId': 'OG003'}]}], 'groups': [{'id': 'OG000', 'title': 'Arm A: Dyskeratosis Congenita, Non-Haploidentical Donor', 'description': 'Arm A Dyskeratosis Congenita (DKC) (non-haploidentical donor)'}, {'id': 'OG001', 'title': 'Arm B: Severe Aplastic Anemia, Non-Matched Related, Non-Haploidentical Donor', 'description': 'Arm B: Severe Aplastic Anemia (SAA ) (non-matched related, non-haploidentical donor)'}, {'id': 'OG002', 'title': 'Arm C: Severe Aplastic Anemia, Matched Related Donor', 'description': 'Arm C: Severe Aplastic Anemia (matched related donor)'}, {'id': 'OG003', 'title': 'Arm E: Severe Aplastic Anemia, PTCy Platform', 'description': 'Arm E: Severe Aplastic Anemia (SAA), PTCy platform'}], 'classes': [{'categories': [{'measurements': [{'value': '100', 'groupId': 'OG000', 'lowerLimit': '0', 'upperLimit': '100'}, {'value': '100', 'groupId': 'OG001', 'lowerLimit': '0', 'upperLimit': '100'}, {'value': '96', 'groupId': 'OG002', 'lowerLimit': '82', 'upperLimit': '100'}, {'value': '100', 'groupId': 'OG003', 'lowerLimit': '0', 'upperLimit': '100'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Day 42', 'description': 'Incidence of neutrophil engraftment by day 42.', 'unitOfMeasure': 'Percentage of participants', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED'}, {'type': 'PRIMARY', 'title': 'Incidence of Platelet Engraftment', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}, {'value': '18', 'groupId': 'OG001'}, {'value': '23', 'groupId': 'OG002'}, {'value': '8', 'groupId': 'OG003'}]}], 'groups': [{'id': 'OG000', 'title': 'Arm A: Dyskeratosis Congenita, Non-Haploidentical Donor', 'description': 'Arm A Dyskeratosis Congenita (DKC) (non-haploidentical donor)'}, {'id': 'OG001', 'title': 'Arm B: Severe Aplastic Anemia, Non-Matched Related, Non-Haploidentical Donor', 'description': 'Arm B: Severe Aplastic Anemia (SAA ) (non-matched related, non-haploidentical donor)'}, {'id': 'OG002', 'title': 'Arm C: Severe Aplastic Anemia, Matched Related Donor', 'description': 'Arm C: Severe Aplastic Anemia (matched related donor)'}, {'id': 'OG003', 'title': 'Arm E: Severe Aplastic Anemia, Haploidentical Donor', 'description': 'Arm E: Severe Aplastic Anemia (SAA), Haploidentical Donor'}], 'classes': [{'categories': [{'measurements': [{'value': '100', 'groupId': 'OG000', 'lowerLimit': '0', 'upperLimit': '100'}, {'value': '100', 'groupId': 'OG001', 'lowerLimit': '0', 'upperLimit': '100'}, {'value': '96', 'groupId': 'OG002', 'lowerLimit': '82', 'upperLimit': '100'}, {'value': '100', 'groupId': 'OG003', 'lowerLimit': '0', 'upperLimit': '100'}]}]}], 'paramType': 'NUMBER', 'timeFrame': '1 year', 'description': 'Incidence of platelet engraftment at 1 year', 'unitOfMeasure': 'Percentage of participants', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Incidence of Regimen Related Mortality', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}, {'value': '18', 'groupId': 'OG001'}, {'value': '23', 'groupId': 'OG002'}, {'value': '8', 'groupId': 'OG003'}]}], 'groups': [{'id': 'OG000', 'title': 'Arm A: Dyskeratosis Congenita, Non-Haploidentical Donor', 'description': 'Arm A Dyskeratosis Congenita (DKC) (non-haploidentical donor)'}, {'id': 'OG001', 'title': 'Arm B: Severe Aplastic Anemia, Non-Matched Related, Non-Haploidentical Donor', 'description': 'Arm B: Severe Aplastic Anemia (SAA ) (non-matched related, non-haploidentical donor)'}, {'id': 'OG002', 'title': 'Arm C: Severe Aplastic Anemia, Matched Related Donor', 'description': 'Arm C: Severe Aplastic Anemia (matched related donor)'}, {'id': 'OG003', 'title': 'Arm E: Severe Aplastic Anemia, PTCy Platform', 'description': 'Arm E: Severe Aplastic Anemia (SAA), PTCy platform'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000', 'lowerLimit': '0', 'upperLimit': '0'}, {'value': '6', 'groupId': 'OG001', 'lowerLimit': '1', 'upperLimit': '33'}, {'value': '6', 'groupId': 'OG002', 'lowerLimit': '1', 'upperLimit': '33'}, {'value': '0', 'groupId': 'OG003', 'lowerLimit': '0', 'upperLimit': '0'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Day 100', 'description': 'Incidence of regimen related mortality by day 100.', 'unitOfMeasure': 'Percentage of participants', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Incidence of Acute Graft-versus-host Disease', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}, {'value': '18', 'groupId': 'OG001'}, {'value': '23', 'groupId': 'OG002'}, {'value': '8', 'groupId': 'OG003'}]}], 'groups': [{'id': 'OG000', 'title': 'Arm A: Dyskeratosis Congenita, Non-Haploidentical Donor', 'description': 'Arm A Dyskeratosis Congenita (DKC) (non-haploidentical donor)'}, {'id': 'OG001', 'title': 'Arm B: Severe Aplastic Anemia, Non-Matched Related, Non-Haploidentical Donor', 'description': 'Arm B: Severe Aplastic Anemia (SAA ) (non-matched related, non-haploidentical donor)'}, {'id': 'OG002', 'title': 'Arm C: Severe Aplastic Anemia, Matched Related Donor', 'description': 'Arm C: Severe Aplastic Anemia (matched related donor)'}, {'id': 'OG003', 'title': 'Arm E: Severe Aplastic Anemia, PTCy Platform', 'description': 'Arm E: Severe Aplastic Anemia (SAA), PTCy platform'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000', 'lowerLimit': '0', 'upperLimit': '0'}, {'value': '11', 'groupId': 'OG001', 'lowerLimit': '0', 'upperLimit': '25'}, {'value': '9', 'groupId': 'OG002', 'lowerLimit': '0', 'upperLimit': '21'}, {'value': '0', 'groupId': 'OG003', 'lowerLimit': '0', 'upperLimit': '0'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Day 100', 'description': 'Incidence of acute graft-versus-host disease by day 100.', 'unitOfMeasure': 'Percentage of participants', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Incidence of Chronic Graft-versus-host Disease', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}, {'value': '18', 'groupId': 'OG001'}, {'value': '23', 'groupId': 'OG002'}, {'value': '8', 'groupId': 'OG003'}]}], 'groups': [{'id': 'OG000', 'title': 'Arm A: Dyskeratosis Congenita, Non-Haploidentical Donor', 'description': 'Arm A Dyskeratosis Congenita (DKC) (non-haploidentical donor)'}, {'id': 'OG001', 'title': 'Arm B: Severe Aplastic Anemia, Non-Matched Related, Non-Haploidentical Donor', 'description': 'Arm B: Severe Aplastic Anemia (SAA ) (non-matched related, non-haploidentical donor)'}, {'id': 'OG002', 'title': 'Arm C: Severe Aplastic Anemia, Matched Related Donor', 'description': 'Arm C: Severe Aplastic Anemia (matched related donor)'}, {'id': 'OG003', 'title': 'Arm E: Severe Aplastic Anemia, PTCy Platform', 'description': 'Arm E: Severe Aplastic Anemia (SAA), PTCy platform'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000', 'lowerLimit': '0', 'upperLimit': '0'}, {'value': '6', 'groupId': 'OG001', 'lowerLimit': '0', 'upperLimit': '16'}, {'value': '0', 'groupId': 'OG002', 'lowerLimit': '0', 'upperLimit': '0'}, {'value': '0', 'groupId': 'OG003', 'lowerLimit': '0', 'upperLimit': '0'}]}]}], 'paramType': 'NUMBER', 'timeFrame': '6 Months', 'description': 'Incidence of chronic graft-versus-host disease by 6 months', 'unitOfMeasure': 'Percentage of participants', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Incidence of Chronic Graft-versus-host Disease', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}, {'value': '18', 'groupId': 'OG001'}, {'value': '23', 'groupId': 'OG002'}, {'value': '8', 'groupId': 'OG003'}]}], 'groups': [{'id': 'OG000', 'title': 'Arm A: Dyskeratosis Congenita, Non-Haploidentical Donor', 'description': 'Arm A Dyskeratosis Congenita (DKC) (non-haploidentical donor)'}, {'id': 'OG001', 'title': 'Arm B: Severe Aplastic Anemia, Non-Matched Related, Non-Haploidentical Donor', 'description': 'Arm B: Severe Aplastic Anemia (SAA ) (non-matched related, non-haploidentical donor)'}, {'id': 'OG002', 'title': 'Arm C: Severe Aplastic Anemia, Matched Related Donor', 'description': 'Arm C: Severe Aplastic Anemia (matched related donor)'}, {'id': 'OG003', 'title': 'Arm E: Severe Aplastic Anemia, PTCy Platform', 'description': 'Arm E: Severe Aplastic Anemia (SAA), PTCy platform'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000', 'lowerLimit': '0', 'upperLimit': '0'}, {'value': '6', 'groupId': 'OG001', 'lowerLimit': '0', 'upperLimit': '16'}, {'value': '0', 'groupId': 'OG002', 'lowerLimit': '0', 'upperLimit': '0'}, {'value': '0', 'groupId': 'OG003', 'lowerLimit': '0', 'upperLimit': '0'}]}]}], 'paramType': 'NUMBER', 'timeFrame': '1 Year', 'description': 'Incidence of chronic graft-versus-host disease by 1 year', 'unitOfMeasure': 'Percentage of participants', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Incidence of Secondary Malignancies', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}, {'value': '18', 'groupId': 'OG001'}, {'value': '23', 'groupId': 'OG002'}, {'value': '8', 'groupId': 'OG003'}]}], 'groups': [{'id': 'OG000', 'title': 'Arm A: Dyskeratosis Congenita, Non-Haploidentical Donor', 'description': 'Arm A Dyskeratosis Congenita (DKC) (non-haploidentical donor)'}, {'id': 'OG001', 'title': 'Arm B: Severe Aplastic Anemia, Non-Matched Related, Non-Haploidentical Donor', 'description': 'Arm B: Severe Aplastic Anemia (SAA ) (non-matched related, non-haploidentical donor)'}, {'id': 'OG002', 'title': 'Arm C: Severe Aplastic Anemia, Matched Related Donor', 'description': 'Arm C: Severe Aplastic Anemia (matched related donor)'}, {'id': 'OG003', 'title': 'Arm E: Severe Aplastic Anemia, PTCy Platform', 'description': 'Arm E: Severe Aplastic Anemia (SAA), PTCy platform'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000', 'lowerLimit': '0', 'upperLimit': '0'}, {'value': '0', 'groupId': 'OG001', 'lowerLimit': '0', 'upperLimit': '0'}, {'value': '0', 'groupId': 'OG002', 'lowerLimit': '0', 'upperLimit': '0'}, {'value': '0', 'groupId': 'OG003', 'lowerLimit': '0', 'upperLimit': '0'}]}]}], 'paramType': 'NUMBER', 'timeFrame': '1 Year', 'description': 'Incidence of secondary malingancies', 'unitOfMeasure': 'Percentage of participants', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Arm A: Dyskeratosis Congenita, Non-Haploidentical Donor', 'description': 'Arm A Dyskeratosis Congenita (DKC) (non-haploidentical donor)'}, {'id': 'FG001', 'title': 'Arm B: Severe Aplastic Anemia, Non-Matched Related, Non-Haploidentical Donor', 'description': 'Arm B: Severe Aplastic Anemia (SAA ) (non-matched related, non-haploidentical donor)'}, {'id': 'FG002', 'title': 'Arm C: Severe Aplastic Anemia, Matched Related Donor', 'description': 'Arm C: Severe Aplastic Anemia (matched related donor)'}, {'id': 'FG003', 'title': 'Arm D: Dyskeratosis Congenita, PTCy Platform', 'description': 'Arm D: Dyskeratosis Congenita (DKC), PTCy platform'}, {'id': 'FG004', 'title': 'Arm E: Severe Aplastic Anemia, PTCy Platform', 'description': 'Arm E: Severe Aplastic Anemia (SAA), PTCy platform'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '12'}, {'groupId': 'FG001', 'numSubjects': '18'}, {'groupId': 'FG002', 'numSubjects': '23'}, {'groupId': 'FG003', 'numSubjects': '0'}, {'groupId': 'FG004', 'numSubjects': '8'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '11'}, {'groupId': 'FG001', 'numSubjects': '15'}, {'groupId': 'FG002', 'numSubjects': '22'}, {'groupId': 'FG003', 'numSubjects': '0'}, {'groupId': 'FG004', 'numSubjects': '8'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '1'}, {'groupId': 'FG001', 'numSubjects': '3'}, {'groupId': 'FG002', 'numSubjects': '1'}, {'groupId': 'FG003', 'numSubjects': '0'}, {'groupId': 'FG004', 'numSubjects': '0'}]}]}]}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'BG000'}, {'value': '18', 'groupId': 'BG001'}, {'value': '23', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}, {'value': '8', 'groupId': 'BG004'}, {'value': '61', 'groupId': 'BG005'}]}], 'groups': [{'id': 'BG000', 'title': 'Arm A: Dyskeratosis Congenita, Non-Haploidentical Donor', 'description': 'Arm A Dyskeratosis Congenita (DKC) (non-haploidentical donor)'}, {'id': 'BG001', 'title': 'Arm B: Severe Aplastic Anemia, Non-Matched Related, Non-Haploidentical Donor', 'description': 'Arm B: Severe Aplastic Anemia (SAA ) (non-matched related, non-haploidentical donor)'}, {'id': 'BG002', 'title': 'Arm C: Severe Aplastic Anemia, Matched Related Donor', 'description': 'Arm C: Severe Aplastic Anemia (matched related donor)'}, {'id': 'BG003', 'title': 'Arm D: Dyskeratosis Congenita, PTCy Platform', 'description': 'Arm D: Dyskeratosis Congenita (DKC), PTCy platform'}, {'id': 'BG004', 'title': 'Arm E: Severe Aplastic Anemia, PTCy Platform', 'description': 'Arm E: Severe Aplastic Anemia (SAA), PTCy platform'}, {'id': 'BG005', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '9', 'groupId': 'BG000'}, {'value': '15', 'groupId': 'BG001'}, {'value': '13', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}, {'value': '4', 'groupId': 'BG004'}, {'value': '41', 'groupId': 'BG005'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '9', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}, {'value': '4', 'groupId': 'BG004'}, {'value': '17', 'groupId': 'BG005'}]}, {'title': '>=65 years', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}, {'value': '0', 'groupId': 'BG004'}, {'value': '3', 'groupId': 'BG005'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '6', 'groupId': 'BG000'}, {'value': '11', 'groupId': 'BG001'}, {'value': '10', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}, {'value': '2', 'groupId': 'BG004'}, {'value': '29', 'groupId': 'BG005'}]}, {'title': 'Male', 'measurements': [{'value': '6', 'groupId': 'BG000'}, {'value': '7', 'groupId': 'BG001'}, {'value': '13', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}, {'value': '6', 'groupId': 'BG004'}, {'value': '32', 'groupId': 'BG005'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Ethnicity (NIH/OMB)', 'classes': [{'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}, {'value': '0', 'groupId': 'BG004'}, {'value': '1', 'groupId': 'BG005'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '11', 'groupId': 'BG000'}, {'value': '17', 'groupId': 'BG001'}, {'value': '21', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}, {'value': '7', 'groupId': 'BG004'}, {'value': '56', 'groupId': 'BG005'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}, {'value': '1', 'groupId': 'BG004'}, {'value': '4', 'groupId': 'BG005'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race (NIH/OMB)', 'classes': [{'categories': [{'title': 'American Indian or Alaska Native', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}, {'value': '0', 'groupId': 'BG004'}, {'value': '3', 'groupId': 'BG005'}]}, {'title': 'Asian', 'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '3', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}, {'value': '0', 'groupId': 'BG004'}, {'value': '6', 'groupId': 'BG005'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}, {'value': '0', 'groupId': 'BG004'}, {'value': '0', 'groupId': 'BG005'}]}, {'title': 'Black or African American', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}, {'value': '3', 'groupId': 'BG004'}, {'value': '3', 'groupId': 'BG005'}]}, {'title': 'White', 'measurements': [{'value': '8', 'groupId': 'BG000'}, {'value': '13', 'groupId': 'BG001'}, {'value': '14', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}, {'value': '1', 'groupId': 'BG004'}, {'value': '36', 'groupId': 'BG005'}]}, {'title': 'More than one race', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '3', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}, {'value': '2', 'groupId': 'BG004'}, {'value': '5', 'groupId': 'BG005'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '3', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}, {'value': '2', 'groupId': 'BG004'}, {'value': '8', 'groupId': 'BG005'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United States', 'categories': [{'measurements': [{'value': '12', 'groupId': 'BG000'}, {'value': '18', 'groupId': 'BG001'}, {'value': '23', 'groupId': 'BG002'}, {'value': '8', 'groupId': 'BG004'}, {'value': '61', 'groupId': 'BG005'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}], 'populationDescription': 'No participants accrued to Arm D: Dyskeratosis Congenita, Haploidentical Donor.'}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2021-02-15', 'size': 852555, 'label': 'Study Protocol and Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'Prot_SAP_000.pdf', 'typeAbbrev': 'Prot_SAP', 'uploadDate': '2025-03-05T11:19', 'hasProtocol': True}, {'date': '2020-03-20', 'size': 4178811, 'label': 'Informed Consent Form', 'hasIcf': True, 'hasSap': False, 'filename': 'ICF_001.pdf', 'typeAbbrev': 'ICF', 'uploadDate': '2025-03-05T11:21', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'phases': ['NA'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 61}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2015-01-10', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-05', 'completionDateStruct': {'date': '2025-03-11', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2025-05-07', 'studyFirstSubmitDate': '2014-06-10', 'resultsFirstSubmitDate': '2025-04-07', 'studyFirstSubmitQcDate': '2014-06-10', 'lastUpdatePostDateStruct': {'date': '2025-05-08', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2025-05-07', 'studyFirstPostDateStruct': {'date': '2014-06-12', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2025-05-08', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2024-08-08', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Incidence of Neutrophil Engraftment', 'timeFrame': 'Day 42', 'description': 'Incidence of neutrophil engraftment by day 42.'}, {'measure': 'Incidence of Platelet Engraftment', 'timeFrame': '1 year', 'description': 'Incidence of platelet engraftment at 1 year'}], 'secondaryOutcomes': [{'measure': 'Incidence of Regimen Related Mortality', 'timeFrame': 'Day 100', 'description': 'Incidence of regimen related mortality by day 100.'}, {'measure': 'Incidence of Acute Graft-versus-host Disease', 'timeFrame': 'Day 100', 'description': 'Incidence of acute graft-versus-host disease by day 100.'}, {'measure': 'Incidence of Chronic Graft-versus-host Disease', 'timeFrame': '6 Months', 'description': 'Incidence of chronic graft-versus-host disease by 6 months'}, {'measure': 'Incidence of Chronic Graft-versus-host Disease', 'timeFrame': '1 Year', 'description': 'Incidence of chronic graft-versus-host disease by 1 year'}, {'measure': 'Incidence of Secondary Malignancies', 'timeFrame': '1 Year', 'description': 'Incidence of secondary malingancies'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['severe aplastic anemia', 'Hematopoietic Stem Cell Transplant'], 'conditions': ['Dyskeratosis Congenita', 'Aplastic Anemia']}, 'descriptionModule': {'briefSummary': 'Fludarabine-based preparative regimen followed by an allogeneic hematopoietic stem cell transplant using related or unrelated donor in persons 0-70 years of age diagnosed with dyskeratosis congenita or severe aplastic anemia who have bone marrow failure characterized by a requirement for red blood cell and platelet transfusions. Three different preparative regimens are included based on disease and donor type.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'maximumAge': '70 Years', 'minimumAge': '0 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Aged 0 - 70 years\n* Acceptable hematopoeitic stem cell donor\n* Dyskeratosis Congenita (DC) with evidence of BM failure defined as:\n\n * requirement for red blood cell and/or platelet transfusions or\n * requirement for G-CSF or GM-CSF or erythropoietin or\n * refractory cytopenias having one of the following three\n\n * platelets \\<50,000/uL or transfusion dependent\n * absolute neutrophil count \\<500/uL without hematopoietic growth factor support\n * hemoglobin \\<9g/uL or transfusion dependent\n* Diagnosis of DC with a triad of mucocutaneous features:\n\n * oral leukoplakia\n * nail dystrophy\n * abnormal reticular skin hyperpigmentation, or\n* Diagnosis of DC with one of the following:\n\n * short telomeres (under a research study)\n * mutation in telomerase holoenzyme (DKC1, TERT, TERC, NOP10, NHP2, TCAB1)\n * mutation in shelterin complex (TINF2)\n * mutation in telomere-capping complex (CTC1)\n* Severe Aplastic Anemia (SAA) primary transplant with evidence of BM failure:\n\n * Refractory cytopenia defined by bone marrow cellularity \\<50% (with \\< 30% residual hematopoietic cells)\n* Diagnosis of SAA with refractory cytopenias having one of the following three:\n\n * platelets \\<20,000/uL or transfusion dependent\n * absolute neutrophil count \\<500/uL without hematopoietic growth factor support\n * absolute reticulocyte count \\<20,000/uL\n* Severe Aplastic Anemia (SAA) requiring a 2nd transplant\n\n * Graft failure as defined by blood/marrow chimerism of \\< 5%\n* Early myelodysplastic features\n* With or without clonal cytogenetic abnormalities\n* Adequate organ function defined as:\n\n * cardiac: left ventricular ejection fraction ≥ 35% with no evidence of decompensated heart failure\n * pulmonary: DLCO ≥30% predicted, no supplemental oxygen requirement\n * renal: Glomerular filtration rate (GFR) ≥30% predicted\n* Voluntary written consent\n\nExclusion Criteria:\n\n* Acute hepatitis or evidence of moderate or severe portal fibrosis or cirrhosis on biopsy\n* Pregnant or lactating\n* Uncontrolled infection\n* Prior radiation therapy (applies to SAA patients only)\n* Diagnosis of Fanconi anemia based on DEB\n* Diagnosis of dyskeratosis congenita with advanced MDS or acute myeloid leukemia with \\>30% blasts'}, 'identificationModule': {'nctId': 'NCT02162420', 'briefTitle': 'Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic Anemia', 'organization': {'class': 'OTHER', 'fullName': 'Masonic Cancer Center, University of Minnesota'}, 'officialTitle': 'Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic Anemia', 'orgStudyIdInfo': {'id': '2013OC127'}, 'secondaryIdInfos': [{'id': 'MT2013-34C', 'type': 'OTHER', 'domain': 'University of Minnesota Blood and Marrow Transplant Program'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Arm A Dyskeratosis Congenita (DKC) (non-haploidentical donor)', 'description': 'Fludarabine based preparative regimen, including alemtuzumab, cyclophosphamide, fludarabine, followed by stem cell transplant for the treatment of dyskeratosis congenita.', 'interventionNames': ['Drug: Alemtuzumab', 'Drug: Fludarabine', 'Drug: Cyclophosphamide', 'Biological: Stem Cell Transplant']}, {'type': 'EXPERIMENTAL', 'label': 'Arm B: Severe Aplastic Anemia (SAA ) (non-matched related, non-haploidentical donor)', 'description': 'Fludarabine based preparative regimen which includes: cyclophosphamide, fludarabine, rabbit ATG and total body irradiation. Followed by stem cell transplant.', 'interventionNames': ['Drug: Alemtuzumab', 'Drug: Fludarabine', 'Drug: Cyclophosphamide', 'Radiation: Total Body Irradiation', 'Biological: Stem Cell Transplant', 'Drug: Anti-thymocyte globulin']}, {'type': 'EXPERIMENTAL', 'label': 'Arm C: Severe Aplastic Anemia (matched related donor)', 'description': 'Fludarabine based preparative regimen which includes: cyclophosphamide, fludarabine, rabbit ATG and total body irradiation. Followed by stem cell transplant.', 'interventionNames': ['Drug: Alemtuzumab', 'Drug: Fludarabine', 'Drug: Cyclophosphamide', 'Radiation: Total Body Irradiation', 'Biological: Stem Cell Transplant', 'Drug: Anti-thymocyte globulin']}, {'type': 'EXPERIMENTAL', 'label': 'Arm D: Dyskeratosis Congenita (DKC), PTCy platform', 'description': 'Fludarabine based preparative regimen, including alemtuzumab, cyclophosphamide, fludarabine, followed by stem cell transplant for the treatment of dyskeratosis congenita.', 'interventionNames': ['Drug: Alemtuzumab', 'Drug: Fludarabine', 'Drug: Cyclophosphamide', 'Biological: Stem Cell Transplant']}, {'type': 'EXPERIMENTAL', 'label': 'Arm E: Severe Aplastic Anemia (SAA), PTCy platform', 'description': 'Fludarabine based preparative regimen which includes: cyclophosphamide, fludarabine, rabbit ATG and total body irradiation. Followed by stem cell transplant.', 'interventionNames': ['Drug: Alemtuzumab', 'Drug: Fludarabine', 'Drug: Cyclophosphamide', 'Radiation: Total Body Irradiation', 'Biological: Stem Cell Transplant', 'Drug: Anti-thymocyte globulin']}], 'interventions': [{'name': 'Alemtuzumab', 'type': 'DRUG', 'description': 'Alemtuzumab 0.2 mg/kg IV over 2 hours on days -10 to -6 from transplant.', 'armGroupLabels': ['Arm A Dyskeratosis Congenita (DKC) (non-haploidentical donor)', 'Arm B: Severe Aplastic Anemia (SAA ) (non-matched related, non-haploidentical donor)', 'Arm C: Severe Aplastic Anemia (matched related donor)', 'Arm D: Dyskeratosis Congenita (DKC), PTCy platform', 'Arm E: Severe Aplastic Anemia (SAA), PTCy platform']}, {'name': 'Fludarabine', 'type': 'DRUG', 'otherNames': ['Fludara'], 'description': 'Fludarabine 40 mg/m2 IV over 1 hour on days -6 to -2 from transplant.', 'armGroupLabels': ['Arm A Dyskeratosis Congenita (DKC) (non-haploidentical donor)', 'Arm B: Severe Aplastic Anemia (SAA ) (non-matched related, non-haploidentical donor)', 'Arm C: Severe Aplastic Anemia (matched related donor)', 'Arm D: Dyskeratosis Congenita (DKC), PTCy platform', 'Arm E: Severe Aplastic Anemia (SAA), PTCy platform']}, {'name': 'Cyclophosphamide', 'type': 'DRUG', 'description': 'Cyclophosphamide 50 mg/kg IV over 2 hours on day -7 from transplant.', 'armGroupLabels': ['Arm A Dyskeratosis Congenita (DKC) (non-haploidentical donor)', 'Arm B: Severe Aplastic Anemia (SAA ) (non-matched related, non-haploidentical donor)', 'Arm C: Severe Aplastic Anemia (matched related donor)', 'Arm D: Dyskeratosis Congenita (DKC), PTCy platform', 'Arm E: Severe Aplastic Anemia (SAA), PTCy platform']}, {'name': 'Total Body Irradiation', 'type': 'RADIATION', 'otherNames': ['TBI'], 'description': 'TBI 200 cGy as a single fraction on day -1 from transplant.', 'armGroupLabels': ['Arm B: Severe Aplastic Anemia (SAA ) (non-matched related, non-haploidentical donor)', 'Arm C: Severe Aplastic Anemia (matched related donor)', 'Arm E: Severe Aplastic Anemia (SAA), PTCy platform']}, {'name': 'Stem Cell Transplant', 'type': 'BIOLOGICAL', 'otherNames': ['HSCT'], 'description': 'Stem cell transplant on day 0.', 'armGroupLabels': ['Arm A Dyskeratosis Congenita (DKC) (non-haploidentical donor)', 'Arm B: Severe Aplastic Anemia (SAA ) (non-matched related, non-haploidentical donor)', 'Arm C: Severe Aplastic Anemia (matched related donor)', 'Arm D: Dyskeratosis Congenita (DKC), PTCy platform', 'Arm E: Severe Aplastic Anemia (SAA), PTCy platform']}, {'name': 'Anti-thymocyte globulin', 'type': 'DRUG', 'otherNames': ['Rabbit ATG'], 'description': 'ATG (Thymoglobulin - Rabbit ) 3 mg/kg IV on days -5 to -3 from stem cell transplant.', 'armGroupLabels': ['Arm B: Severe Aplastic Anemia (SAA ) (non-matched related, non-haploidentical donor)', 'Arm C: Severe Aplastic Anemia (matched related donor)', 'Arm E: Severe Aplastic Anemia (SAA), PTCy platform']}]}, 'contactsLocationsModule': {'locations': [{'zip': '55455', 'city': 'Minneapolis', 'state': 'Minnesota', 'country': 'United States', 'facility': 'University of Minnesota Medical Center, Fairview', 'geoPoint': {'lat': 44.97997, 'lon': -93.26384}}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Masonic Cancer Center, University of Minnesota', 'class': 'OTHER'}, 'responsibleParty': {'type': 'SPONSOR'}}}}