Ignite Creation Date:
2025-12-25 @ 1:33 AM
Ignite Modification Date:
2026-02-11 @ 7:44 PM
Study NCT ID:
NCT02306694
Status:
COMPLETED
Last Update Posted:
2020-04-01
First Post:
2014-11-25
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Prospective Biomarkers of Bone Metabolism in Hemophilia A
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D006467', 'term': 'Hemophilia A'}, {'id': 'D001847', 'term': 'Bone Diseases'}], 'ancestors': [{'id': 'D025861', 'term': 'Blood Coagulation Disorders, Inherited'}, {'id': 'D001778', 'term': 'Blood Coagulation Disorders'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D020147', 'term': 'Coagulation Protein Disorders'}, {'id': 'D006474', 'term': 'Hemorrhagic Disorders'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D009140', 'term': 'Musculoskeletal Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D005169', 'term': 'Factor VIII'}], 'ancestors': [{'id': 'D001779', 'term': 'Blood Coagulation Factors'}, {'id': 'D001798', 'term': 'Blood Proteins'}, {'id': 'D011506', 'term': 'Proteins'}, {'id': 'D000602', 'term': 'Amino Acids, Peptides, and Proteins'}, {'id': 'D011498', 'term': 'Protein Precursors'}, {'id': 'D001685', 'term': 'Biological Factors'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'taylojas@ohsu.edu', 'phone': '503-494-8716', 'title': 'Dr. Jayson Taylor', 'organization': 'Oregon Health & Science University'}, 'certainAgreement': {'piSponsorEmployee': True}}, 'adverseEventsModule': {'timeFrame': '1 month', 'eventGroups': [{'id': 'EG000', 'title': 'Open Label', 'description': 'Everyone receives Advate (antihemophilic factor) on Day 1 and 3.\n\nAdvate: Patients who are currently taking Advate as their factor replacement will be eligible for the 5-day study.', 'otherNumAtRisk': 16, 'deathsNumAtRisk': 16, 'otherNumAffected': 0, 'seriousNumAtRisk': 16, 'deathsNumAffected': 0, 'seriousNumAffected': 0}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Bone Biomarker Density (BMD)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Open Label', 'description': 'Everyone receives Advate (antihemophilic factor) on Day 1 and 3.\n\nAdvate: Patients who are currently taking Advate as their factor replacement will be eligible for the 5-day study.'}], 'classes': [{'title': 'Spine BMD (Spine L1-L4 Z-score)', 'categories': [{'measurements': [{'value': '-0.74', 'spread': '1.22', 'groupId': 'OG000'}]}]}, {'title': 'Hip Total BMD', 'categories': [{'measurements': [{'value': '-0.16', 'spread': '1.12', 'groupId': 'OG000'}]}]}, {'title': 'Hip Neck BMD', 'categories': [{'measurements': [{'value': '-0.16', 'spread': '1.23', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': '5 days', 'description': 'BMD was measured as Z-scores/T-scores using Dual-Energy X-ray Absorptiometry (DEXA) scanning; specifically looking at Spine, Hip/Neck, and Hip total scores. BMD Z-scores compare what would be expected in someone your age and body size. A Z-score, is a unit of standard deviation, where above 0 would indicate the bones are more dense than expected, while a Z-score below 0 would indicate the bones were less dense.', 'unitOfMeasure': 'Z-Score', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}, {'type': 'PRIMARY', 'title': 'Joint Health', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Open Label', 'description': 'Everyone receives Advate (antihemophilic factor) on Day 1 and 3.\n\nAdvate: Patients who are currently taking Advate as their factor replacement will be eligible for the 5-day study.'}], 'classes': [{'categories': [{'measurements': [{'value': '19.91', 'spread': '16.26', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': '5 days', 'description': 'Hemophilia Joint Health Score (HJHS); with a higher score representing worst outcomes, scores could range from 0 (no problems) to a max score of 120 (severe problems).', 'unitOfMeasure': 'score on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}, {'type': 'PRIMARY', 'title': 'Quality of Life Using the VAS and EQ-5D-3L', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Open Label', 'description': 'Everyone receives Advate (antihemophilic factor) on Day 1 and 3.\n\nAdvate: Patients who are currently taking Advate as their factor replacement will be eligible for the 5-day study.'}], 'classes': [{'title': 'EQ-5D-3L', 'categories': [{'measurements': [{'value': '6.92', 'spread': '1.83', 'groupId': 'OG000'}]}]}, {'title': 'VAS', 'categories': [{'measurements': [{'value': '82.42', 'spread': '15.08', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': '5 days', 'description': 'Visual Analog Scale (VAS) via the EQ-5D-3L was used to report participants self-rated health. EQ-5D-3L total score ranges from 5 (no problems) to 15 (significant problems). VAS scores could range from 0 (worst health ever) to 100 (best health ever).', 'unitOfMeasure': 'score on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}, {'type': 'PRIMARY', 'title': 'Plasma Cytokine Concentration Differences From 0-hour to 24-hour', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Open Label', 'description': 'Everyone receives Advate (antihemophilic factor) on Day 1 and 3.\n\nAdvate: Patients who are currently taking Advate as their factor replacement will be eligible for the 5-day study.'}], 'classes': [{'title': 'Basic FGF', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '2.04', 'spread': '1.78', 'groupId': 'OG000'}]}]}, {'title': 'C-Terminal telopeptide', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.03', 'spread': '0.12', 'groupId': 'OG000'}]}]}, {'title': 'DKK1', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-7.73', 'spread': '15.63', 'groupId': 'OG000'}]}]}, {'title': 'Eotaxin', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-2.84', 'spread': '14.09', 'groupId': 'OG000'}]}]}, {'title': 'fibroblast growth factor 23', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-6.72', 'spread': '9.95', 'groupId': 'OG000'}]}]}, {'title': 'interferon gamma', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.12', 'spread': '0.52', 'groupId': 'OG000'}]}]}, {'title': 'interleukin 13', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.76', 'spread': '1.52', 'groupId': 'OG000'}]}]}, {'title': 'Interleukin 1 beta', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.12', 'spread': '0.19', 'groupId': 'OG000'}]}]}, {'title': 'interleukin receptor 1 antagonist', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-131.72', 'spread': '429.93', 'groupId': 'OG000'}]}]}, {'title': 'interleukin 2', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.71', 'spread': '1.16', 'groupId': 'OG000'}]}]}, {'title': 'interleukin 4', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.28', 'spread': '0.39', 'groupId': 'OG000'}]}]}, {'title': 'interleukin 6', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.22', 'spread': '0.38', 'groupId': 'OG000'}]}]}, {'title': 'interleukin 17', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.13', 'spread': '0.80', 'groupId': 'OG000'}]}]}, {'title': 'interleukin 8', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.56', 'spread': '0.99', 'groupId': 'OG000'}]}]}, {'title': 'interleukin 9', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-14.83', 'spread': '14.03', 'groupId': 'OG000'}]}]}, {'title': 'Insulin', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-279.39', 'spread': '1518.67', 'groupId': 'OG000'}]}]}, {'title': 'IP10', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-10.06', 'spread': '36.49', 'groupId': 'OG000'}]}]}, {'title': 'Leptin', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-208.39', 'spread': '6728.25', 'groupId': 'OG000'}]}]}, {'title': 'MCP1', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-6.42', 'spread': '7.53', 'groupId': 'OG000'}]}]}, {'title': 'MIP1a', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.23', 'spread': '0.40', 'groupId': 'OG000'}]}]}, {'title': 'osteoclasts', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-2146.62', 'spread': '5921.06', 'groupId': 'OG000'}]}]}, {'title': 'osteoprotegerin', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '113.70', 'spread': '328.53', 'groupId': 'OG000'}]}]}, {'title': 'osteopontin', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-493.87', 'spread': '6795.35', 'groupId': 'OG000'}]}]}, {'title': 'platelet derived growth factors', 'denoms': [{'units': 'Participants', 'counts': [{'value': '3', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-103.83', 'spread': '156.52', 'groupId': 'OG000'}]}]}, {'title': 'parathyroid horomone', 'denoms': [{'units': 'Participants', 'counts': [{'value': '11', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-6.72', 'spread': '25.70', 'groupId': 'OG000'}]}]}, {'title': 'RANKL', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '228.10', 'spread': '277.99', 'groupId': 'OG000'}]}]}, {'title': 'chemokine ligand 5', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-414.77', 'spread': '681.82', 'groupId': 'OG000'}]}]}, {'title': 'sclerostin', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-398.13', 'spread': '838.83', 'groupId': 'OG000'}]}]}, {'title': 'transforming growth factor-beta 1', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-519.05', 'spread': '772.15', 'groupId': 'OG000'}]}]}, {'title': 'transforming growth factor beta - 2', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-10.08', 'spread': '16.43', 'groupId': 'OG000'}]}]}, {'title': 'transforming growth factor beta - 3', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.74', 'spread': '1.49', 'groupId': 'OG000'}]}]}, {'title': 'tumor necrosis factor alpha', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-1.09', 'spread': '2.76', 'groupId': 'OG000'}]}]}, {'title': 'VEGF', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-4.03', 'spread': '12.17', 'groupId': 'OG000'}]}]}, {'title': 'MIP1b', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-7.19', 'spread': '9.36', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': '24 hours', 'description': 'cytokines were measured using ELISA/magnetic bead multiplex kits. We calculated concentration change from hour 0 to hour 24. Cytokines: FGF, C-Terminal telopeptide (CTX-1), Dickkopf WNT signaling pathway inhibitor 1(DKK1), Eotaxin, fibroblast growth factor 23(FGF23), interferon gamma, interleukin 13, interleukin 1 beta, interleukin receptor 1 antagonist, interleukin 2, interleukin 4, interleukin 6, interleukin 17, interleukin 8, interleukin 9 Insulin, interferon gamma induced protein 10 (IP10), Leptin, monocyte chemoattractant protein1, monocyte chemoattractant protein1, macrophage inflammatory protein 1a (MIP1a), osteoclasts, Osteoprotegerin, osteopontin, platelet derived growth factors, parathyroid horomone, Recepter activator of nuclear factor kappa-B ligand (RANKL), chemokine ligand 5, sclerostin, transforming growth factor-beta 1, transforming growth factor beta 2, transforming growth factor beta 3, tumor necrosis factor alpha, vascular endothelial growth factor, MIP1b.', 'unitOfMeasure': 'pg/mL', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Some participant samples were unable to be analyzed at all time points due to poor sample quality or other factors.'}, {'type': 'OTHER_PRE_SPECIFIED', 'title': 'Medication Adherence', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Open Label', 'description': 'Everyone receives Advate (antihemophilic factor) on Day 1 and 3.\n\nAdvate: Patients who are currently taking Advate as their factor replacement will be eligible for the 5-day study.'}], 'classes': [{'categories': [{'measurements': [{'value': '56.25', 'spread': '5.29', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': '5 days', 'description': 'VERITAS instrument self-reported compliance with factor replacement regimen. The VERITAS is composed of 6 subsections, where scores range from 1 (best adherence) to 5 (worst adherence). A total score was calculated with the minimum score of 24 (most adherent) and a maximum score of 120 (least adherent)', 'unitOfMeasure': 'score on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}, {'type': 'OTHER_PRE_SPECIFIED', 'title': 'Hemophilia Activities List (HAL) and the International Society of Thrombosis and Hemostasis- Bleeding Assessment Tool (ISTH-BAT)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Open Label', 'description': 'Everyone receives Advate (antihemophilic factor) on Day 1 and 3.\n\nAdvate: Patients who are currently taking Advate as their factor replacement will be eligible for the 5-day study.'}], 'classes': [{'title': 'HAL', 'categories': [{'measurements': [{'value': '226', 'spread': '41.78', 'groupId': 'OG000'}]}]}, {'title': 'ISTH-BAT', 'categories': [{'measurements': [{'value': '16.92', 'spread': '6.56', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': '5 days', 'description': 'The HAL is used to assess physical activity; scores range from 42 (severe problems) to 252 (no problems). The ISTH-BAT was used to evaluate bleeding phenotype. Each section was scored using a 0 to 4 scale for each of the 12 subsections. A total score was calculated by taking the sum of each section, resulting in a range of scores from 0 (no bleeding history) to 48 (most extensive clinical intervention for bleeding)', 'unitOfMeasure': 'score on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}, {'type': 'OTHER_PRE_SPECIFIED', 'title': 'Participant Quality of Life', 'denoms': [{'units': 'Participants', 'counts': [{'value': '12', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Open Label', 'description': 'Everyone receives Advate (antihemophilic factor) on Day 1 and 3.\n\nAdvate: Patients who are currently taking Advate as their factor replacement will be eligible for the 5-day study.'}], 'classes': [{'categories': [{'measurements': [{'value': '113.83', 'spread': '58.19', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': '5 days', 'description': 'Haem-A-QoL was used to assess various components indicating participants quality of life. Haem-A-QoL is composed of 10 subsections with 3-8 questions in each. Questions are rated from 1 to 5, with 5 being the most negative influence on quality of life. Some questions are worded in the inverse, such that the 1 corresponds to the greatest negative impact on quality of life. In this case, the score is inverted to match the remainder of questions for statistical analysis. A Transformed scores is calculated for each subsection and for the total of all the subjections. The scores range from 0 (best quality of life) to 225 (worst quality of life).', 'unitOfMeasure': 'score on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Open Label', 'description': 'Everyone receives Advate (antihemophilic factor) on Day 1 and 3.\n\nAdvate: Patients who are currently taking Advate as their factor replacement will be eligible for the 5-day study.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '16'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '12'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '4'}]}], 'dropWithdraws': [{'type': 'Withdrawal by Subject', 'reasons': [{'groupId': 'FG000', 'numSubjects': '3'}]}, {'type': 'Lost to Follow-up', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}]}]}]}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '16', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'Open Label', 'description': 'Everyone receives Advate (antihemophilic factor) on Day 1 and 3.\n\nAdvate: Patients who are currently taking Advate as their factor replacement will be eligible for the 5-day study.'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '3', 'groupId': 'BG000'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '13', 'groupId': 'BG000'}]}, {'title': '>=65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '16', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race/Ethnicity, Customized', 'classes': [{'title': 'White', 'categories': [{'measurements': [{'value': '16', 'groupId': 'BG000'}]}]}, {'title': 'Hispanic', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United States', 'categories': [{'measurements': [{'value': '16', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Weight', 'classes': [{'categories': [{'measurements': [{'value': '149.3', 'spread': '192.9', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'lb', 'dispersionType': 'STANDARD_DEVIATION'}]}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2017-03-28', 'size': 478683, 'label': 'Study Protocol and Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'Prot_SAP_000.pdf', 'typeAbbrev': 'Prot_SAP', 'uploadDate': '2019-05-07T17:33', 'hasProtocol': True}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 16}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2014-12', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2020-03', 'completionDateStruct': {'date': '2018-04-16', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2020-03-23', 'studyFirstSubmitDate': '2014-11-25', 'resultsFirstSubmitDate': '2019-05-28', 'studyFirstSubmitQcDate': '2014-12-01', 'lastUpdatePostDateStruct': {'date': '2020-04-01', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2020-03-10', 'studyFirstPostDateStruct': {'date': '2014-12-03', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2020-03-24', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2018-04-16', 'type': 'ACTUAL'}}, 'outcomesModule': {'otherOutcomes': [{'measure': 'Medication Adherence', 'timeFrame': '5 days', 'description': 'VERITAS instrument self-reported compliance with factor replacement regimen. The VERITAS is composed of 6 subsections, where scores range from 1 (best adherence) to 5 (worst adherence). A total score was calculated with the minimum score of 24 (most adherent) and a maximum score of 120 (least adherent)'}, {'measure': 'Hemophilia Activities List (HAL) and the International Society of Thrombosis and Hemostasis- Bleeding Assessment Tool (ISTH-BAT)', 'timeFrame': '5 days', 'description': 'The HAL is used to assess physical activity; scores range from 42 (severe problems) to 252 (no problems). The ISTH-BAT was used to evaluate bleeding phenotype. Each section was scored using a 0 to 4 scale for each of the 12 subsections. A total score was calculated by taking the sum of each section, resulting in a range of scores from 0 (no bleeding history) to 48 (most extensive clinical intervention for bleeding)'}, {'measure': 'Participant Quality of Life', 'timeFrame': '5 days', 'description': 'Haem-A-QoL was used to assess various components indicating participants quality of life. Haem-A-QoL is composed of 10 subsections with 3-8 questions in each. Questions are rated from 1 to 5, with 5 being the most negative influence on quality of life. Some questions are worded in the inverse, such that the 1 corresponds to the greatest negative impact on quality of life. In this case, the score is inverted to match the remainder of questions for statistical analysis. A Transformed scores is calculated for each subsection and for the total of all the subjections. The scores range from 0 (best quality of life) to 225 (worst quality of life).'}], 'primaryOutcomes': [{'measure': 'Bone Biomarker Density (BMD)', 'timeFrame': '5 days', 'description': 'BMD was measured as Z-scores/T-scores using Dual-Energy X-ray Absorptiometry (DEXA) scanning; specifically looking at Spine, Hip/Neck, and Hip total scores. BMD Z-scores compare what would be expected in someone your age and body size. A Z-score, is a unit of standard deviation, where above 0 would indicate the bones are more dense than expected, while a Z-score below 0 would indicate the bones were less dense.'}, {'measure': 'Joint Health', 'timeFrame': '5 days', 'description': 'Hemophilia Joint Health Score (HJHS); with a higher score representing worst outcomes, scores could range from 0 (no problems) to a max score of 120 (severe problems).'}, {'measure': 'Quality of Life Using the VAS and EQ-5D-3L', 'timeFrame': '5 days', 'description': 'Visual Analog Scale (VAS) via the EQ-5D-3L was used to report participants self-rated health. EQ-5D-3L total score ranges from 5 (no problems) to 15 (significant problems). VAS scores could range from 0 (worst health ever) to 100 (best health ever).'}, {'measure': 'Plasma Cytokine Concentration Differences From 0-hour to 24-hour', 'timeFrame': '24 hours', 'description': 'cytokines were measured using ELISA/magnetic bead multiplex kits. We calculated concentration change from hour 0 to hour 24. Cytokines: FGF, C-Terminal telopeptide (CTX-1), Dickkopf WNT signaling pathway inhibitor 1(DKK1), Eotaxin, fibroblast growth factor 23(FGF23), interferon gamma, interleukin 13, interleukin 1 beta, interleukin receptor 1 antagonist, interleukin 2, interleukin 4, interleukin 6, interleukin 17, interleukin 8, interleukin 9 Insulin, interferon gamma induced protein 10 (IP10), Leptin, monocyte chemoattractant protein1, monocyte chemoattractant protein1, macrophage inflammatory protein 1a (MIP1a), osteoclasts, Osteoprotegerin, osteopontin, platelet derived growth factors, parathyroid horomone, Recepter activator of nuclear factor kappa-B ligand (RANKL), chemokine ligand 5, sclerostin, transforming growth factor-beta 1, transforming growth factor beta 2, transforming growth factor beta 3, tumor necrosis factor alpha, vascular endothelial growth factor, MIP1b.'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'keywords': ['hemophilia', 'bone disease', 'clinical study', 'biomarkers'], 'conditions': ['Hemophilia', 'Bone Disease']}, 'referencesModule': {'references': [{'pmid': '16460432', 'type': 'BACKGROUND', 'citation': 'Plug I, Van Der Bom JG, Peters M, Mauser-Bunschoten EP, De Goede-Bolder A, Heijnen L, Smit C, Willemse J, Rosendaal FR. Mortality and causes of death in patients with hemophilia, 1992-2001: a prospective cohort study. J Thromb Haemost. 2006 Mar;4(3):510-6. doi: 10.1111/j.1538-7836.2006.01808.x.'}, {'pmid': '19220727', 'type': 'BACKGROUND', 'citation': 'Siboni SM, Mannucci PM, Gringeri A, Franchini M, Tagliaferri A, Ferretti M, Tradati FC, Santagostino E, von Mackensen S; Italian Association of Haemophilia Centres (AICE). Health status and quality of life of elderly persons with severe hemophilia born before the advent of modern replacement therapy. J Thromb Haemost. 2009 May;7(5):780-6. doi: 10.1111/j.1538-7836.2009.03318.x. Epub 2009 Feb 12.'}, {'pmid': '19187193', 'type': 'BACKGROUND', 'citation': 'Gerstner G, Damiano ML, Tom A, Worman C, Schultz W, Recht M, Stopeck AT. Prevalence and risk factors associated with decreased bone mineral density in patients with haemophilia. Haemophilia. 2009 Mar;15(2):559-65. doi: 10.1111/j.1365-2516.2008.01963.x. Epub 2009 Feb 1.'}, {'pmid': '17212729', 'type': 'BACKGROUND', 'citation': 'Wallny TA, Scholz DT, Oldenburg J, Nicolay C, Ezziddin S, Pennekamp PH, Stoffel-Wagner B, Kraft CN. Osteoporosis in haemophilia - an underestimated comorbidity? Haemophilia. 2007 Jan;13(1):79-84. doi: 10.1111/j.1365-2516.2006.01405.x.'}, {'pmid': '15286254', 'type': 'BACKGROUND', 'citation': 'Barnes C, Wong P, Egan B, Speller T, Cameron F, Jones G, Ekert H, Monagle P. Reduced bone density among children with severe hemophilia. Pediatrics. 2004 Aug;114(2):e177-81. doi: 10.1542/peds.114.2.e177.'}, {'pmid': '8208906', 'type': 'BACKGROUND', 'citation': 'Gallacher SJ, Deighan C, Wallace AM, Cowan RA, Fraser WD, Fenner JA, Lowe GD, Boyle IT. Association of severe haemophilia A with osteoporosis: a densitometric and biochemical study. Q J Med. 1994 Mar;87(3):181-6.'}, {'pmid': '18275434', 'type': 'BACKGROUND', 'citation': 'Tlacuilo-Parra A, Morales-Zambrano R, Tostado-Rabago N, Esparza-Flores MA, Lopez-Guido B, Orozco-Alcala J. Inactivity is a risk factor for low bone mineral density among haemophilic children. Br J Haematol. 2008 Mar;140(5):562-7. doi: 10.1111/j.1365-2141.2007.06972.x.'}, {'pmid': '16323100', 'type': 'BACKGROUND', 'citation': 'Amorosa V, Tebas P. Bone disease and HIV infection. Clin Infect Dis. 2006 Jan 1;42(1):108-14. doi: 10.1086/498511. Epub 2005 Nov 30.'}, {'pmid': '24118364', 'type': 'BACKGROUND', 'citation': 'Anagnostis P, Vakalopoulou S, Vyzantiadis TA, Charizopoulou M, Karras S, Goulis DG, Karagiannis A, Gerou S, Garipidou V. The clinical utility of bone turnover markers in the evaluation of bone disease in patients with haemophilia A and B. Haemophilia. 2014 Mar;20(2):268-75. doi: 10.1111/hae.12271. Epub 2013 Oct 7.'}, {'pmid': '22469061', 'type': 'BACKGROUND', 'citation': 'Liel MS, Greenberg DL, Recht M, Vanek C, Klein RF, Taylor JA. Decreased bone density and bone strength in a mouse model of severe factor VIII deficiency. Br J Haematol. 2012 Jul;158(1):140-3. doi: 10.1111/j.1365-2141.2012.09101.x. Epub 2012 Apr 2. No abstract available.'}, {'pmid': '23902277', 'type': 'BACKGROUND', 'citation': 'Kempton CL, Antun A, Antoniucci DM, Carpenter W, Ribeiro M, Stein S, Slovensky L, Elon L. Bone density in haemophilia: a single institutional cross-sectional study. Haemophilia. 2014 Jan;20(1):121-8. doi: 10.1111/hae.12240. Epub 2013 Aug 1.'}, {'pmid': '16763463', 'type': 'BACKGROUND', 'citation': 'Lee SK, Lorenzo J. Cytokines regulating osteoclast formation and function. Curr Opin Rheumatol. 2006 Jul;18(4):411-8. doi: 10.1097/01.bor.0000231911.42666.78.'}, {'pmid': '14960673', 'type': 'BACKGROUND', 'citation': "Brinker MR, O'Connor DP. The incidence of fractures and dislocations referred for orthopaedic services in a capitated population. J Bone Joint Surg Am. 2004 Feb;86(2):290-7."}, {'pmid': '214960', 'type': 'BACKGROUND', 'citation': 'Roche AF, Roberts J, Hamill PV. Skeletal maturity of youths 12--17 years racial, geographic area, and socioeconomic differentials. United States, 1966-1970. Vital Health Stat 11. 1978 Oct;(167):1-98. No abstract available.'}]}, 'descriptionModule': {'briefSummary': 'One of the major shortcomings in studying bone disease in hemophilia is the lack of fracture outcome data demonstrating the clinical significance of decreased BMD and altered bone biomarkers in the hemophilia population. This study demonstrates that PwH have an increased risk of fracture compared to the general population and that the issue of bone health will increase in importance as the PwH population ages.', 'detailedDescription': 'This is a pilot study to determine the impact of factor replacement on bone biomarkers in up to 20 hemophilia A subjects. Subjects will be recruited over 1 year for the 5-day protocol.\n\nFollowing a 72-hour washout period, factor levels and bone biomarkers will be followed before and after 50 units/kg replacement on Day 1 and 20 units/kg replacement on Day 3. Each subject can serve as their Figure 4. Fracture rates in PwH compared to historic controls.'}, 'eligibilityModule': {'sex': 'MALE', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'maximumAge': '85 Years', 'minimumAge': '16 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n1. Males with a diagnosis of hemophilia A with a historic baseline FVIII level ≤ 2%.\n2. Age \\> 16 years old\n3. Currently using ADVATE as FVIII replacement therapy\n\nExclusion Criteria:\n\n1. Subject or guardian is unwilling or unable to give written informed consent and/or assent\n2. Joint or muscle bleeding within 2 weeks of Study Day 1\n3. Presence of a current factor inhibitor (\\>0.6 BU/mL via Nijmegan-modified Bethesda assay)\n4. Known collagen vascular bone disease.'}, 'identificationModule': {'nctId': 'NCT02306694', 'briefTitle': 'Prospective Biomarkers of Bone Metabolism in Hemophilia A', 'organization': {'class': 'OTHER', 'fullName': 'Oregon Health and Science University'}, 'officialTitle': 'Prospective Biomarkers of Bone Metabolism in Hemophilia A', 'orgStudyIdInfo': {'id': 'e11104'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Open label', 'description': 'Everyone receives Advate (antihemophilic factor) on Day 1 and 3.', 'interventionNames': ['Drug: Advate']}], 'interventions': [{'name': 'Advate', 'type': 'DRUG', 'otherNames': ['Antihemophilic Factor (Recombinant)'], 'description': 'Patients who are currently taking Advate as their factor replacement will be eligible for the 5-day study.', 'armGroupLabels': ['Open label']}]}, 'contactsLocationsModule': {'locations': [{'zip': '97239', 'city': 'Portland', 'state': 'Oregon', 'country': 'United States', 'facility': 'Oregon Health and Science University', 'geoPoint': {'lat': 45.52345, 'lon': -122.67621}}], 'overallOfficials': [{'name': 'Jason Taylor, MD, PhD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Oregon Health and Science'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Oregon Health and Science University', 'class': 'OTHER'}, 'collaborators': [{'name': 'Baxter Healthcare Corporation', 'class': 'INDUSTRY'}], 'responsibleParty': {'type': 'SPONSOR'}}}}