Ignite Creation Date:
2025-12-25 @ 1:22 AM
Ignite Modification Date:
2026-04-14 @ 2:10 PM
Study NCT ID:
NCT02235493
Status:
COMPLETED
Last Update Posted:
2019-03-13
First Post:
2014-09-05
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Non-interventional Substudy of ALX-HPP-502 to Assess Natural History of Patients With Juvenile-onset HPP Who Served as Historical Controls in ENB-006-09
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D007014', 'term': 'Hypophosphatasia'}, {'id': 'D001847', 'term': 'Bone Diseases'}, {'id': 'D012279', 'term': 'Rickets'}, {'id': 'D010018', 'term': 'Osteomalacia'}], 'ancestors': [{'id': 'D008664', 'term': 'Metal Metabolism, Inborn Errors'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}, {'id': 'D009140', 'term': 'Musculoskeletal Diseases'}, {'id': 'D001851', 'term': 'Bone Diseases, Metabolic'}, {'id': 'D002128', 'term': 'Calcium Metabolism Disorders'}, {'id': 'D014808', 'term': 'Vitamin D Deficiency'}, {'id': 'D001361', 'term': 'Avitaminosis'}, {'id': 'D003677', 'term': 'Deficiency Diseases'}, {'id': 'D044342', 'term': 'Malnutrition'}, {'id': 'D009748', 'term': 'Nutrition Disorders'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'clinicaltrials@alxn.com', 'phone': '203-272-2596', 'title': 'Senior Medical Director, Clinical Development', 'organization': 'Alexion Pharmaceuticals'}, 'certainAgreement': {'otherDetails': 'Participation in this single-center study involves a commitment to publish the data from the study in a cooperative publication prior to release of study results on an individual basis. Therefore, the PI is subject to certain disclosure and publication limitations.', 'restrictionType': 'OTHER', 'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': 'No adverse event data were collected during this retrospective noninterventional study.', 'eventGroups': [{'id': 'EG000', 'title': 'Retrospective Observational', 'description': 'Patients diagnosed with juvenile-onset hypophosphatasia (HPP) \\[ie, documented onset of first signs/symptoms of HPP at ≥ 6 months to \\< 18 years of age\\].', 'otherNumAtRisk': 0, 'otherNumAffected': 0, 'seriousNumAtRisk': 0, 'seriousNumAffected': 0}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Modified Performance-Oriented Mobility Assessment-Gait Subtest (MPOMA-G) - Change From Baseline to Last Overall', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Retrospective Observational', 'description': 'Patients diagnosed with juvenile-onset hypophosphatasia (HPP) \\[ie, documented onset of first signs/symptoms of HPP at ≥ 6 months to \\< 18 years of age\\].'}], 'classes': [{'categories': [{'measurements': [{'value': '1.50', 'groupId': 'OG000', 'lowerLimit': '1.00', 'upperLimit': '2.00'}]}]}], 'analyses': [{'pValue': '0.0625', 'groupIds': ['OG000'], 'ciNumSides': 'TWO_SIDED', 'pValueComment': 'The p-value is based on a nonparametric sign test to determine whether the median of change from Baseline in MPOMA-G score differs from 0. No multiple comparisons or multiplicity adjustments were conducted.', 'groupDescription': 'Videos were reviewed and scored by 3 independent raters who were masked to patient identifiers and the clinic visits and dates when the videos were recorded. Each rater scored the individual components required for the calculation of the total MPOMA-G score for each evaluable patient video. The median of the scores across the 3 raters was obtained for each individual component and summed to generate the median MPOMA-G score used in the analyses.', 'statisticalMethod': 'Sign test', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER'}], 'paramType': 'MEDIAN', 'timeFrame': "The earliest available MPOMA-G score that was assessed within the period of patients' aged 5 to 15 years, inclusive.", 'description': 'The MPOMA-G is a 5-component assessment that is used to evaluate gait performance. The first component has 4 sub-components. For 2 components and 2 sub-components, scores of 0 or 1 are assigned while scores of 0, 1 or 2 are assigned to the rest of the 2 components and 2 sub-components based on type of ambulation pattern observed. The maximum total score of 12 points = no impairment and 0 points = worst impairment.', 'unitOfMeasure': 'units on a scale', 'dispersionType': 'Inter-Quartile Range', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Performance-Oriented Mobility Assessment-Gait Subtest (POMA-G) - Change From Baseline to Last Overall', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Retrospective Observational', 'description': 'Patients diagnosed with juvenile-onset hypophosphatasia (HPP) \\[ie, documented onset of first signs/symptoms of HPP at ≥ 6 months to \\< 18 years of age\\].'}], 'classes': [{'categories': [{'measurements': [{'value': '1.50', 'groupId': 'OG000', 'lowerLimit': '0.00', 'upperLimit': '2.00'}]}]}], 'analyses': [{'pValue': '0.1250', 'groupIds': ['OG000'], 'ciNumSides': 'TWO_SIDED', 'pValueComment': 'The p-value is based on a nonparametric sign test to determine whether the median of change from Baseline in POMA-G score differs from 0. No multiple comparisons or multiplicity adjustments were conducted.', 'groupDescription': 'Videos were reviewed and scored by 3 independent raters who were masked to patient identifiers and the clinic visits and dates when the videos were recorded. Each rater scored the individual components required for the calculation of the total POMA-G score for each evaluable patient video. The median of the scores across the 3 raters was obtained for each individual component and summed to generate the median POMA-G score used in the analyses.', 'statisticalMethod': 'Sign test', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER'}], 'paramType': 'MEDIAN', 'timeFrame': "The earliest available MPOMA-G score that was assessed within the period of patients' aged 5 to 15 years, inclusive.", 'description': 'The POMA-G is a 7-component assessment that is used to evaluate gait performance. The second component has 4 sub-components. Scores of 0, 1 or 2 are assigned to 2 components while scores of 0 or 1 are assigned to the rest of the 4 components and 4 sub-components based on type of ambulation pattern observed. The maximum total score of 12 points = no impairment and 0 points = worst impairment.', 'unitOfMeasure': 'units on a scale', 'dispersionType': 'Inter-Quartile Range', 'reportingStatus': 'POSTED'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Retrospective Observational', 'description': 'Patients diagnosed with juvenile-onset hypophosphatasia (HPP) \\[ie, documented onset of first signs/symptoms of HPP at ≥ 6 months to \\< 18 years of age\\].'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '6'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '6'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}]}]}]}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'Retrospective Observational', 'description': 'Patients diagnosed with juvenile-onset hypophosphatasia (HPP) \\[ie, documented onset of first signs/symptoms of HPP at ≥ 6 months to \\< 18 years of age\\].'}], 'measures': [{'title': 'Age, Customized', 'classes': [{'categories': [{'measurements': [{'value': '28.2', 'spread': '10.6', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'description': 'Age at Diagnosis', 'unitOfMeasure': 'months', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '6', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race/Ethnicity, Customized', 'classes': [{'title': 'White, Hispanic or Latino', 'categories': [{'measurements': [{'value': '2', 'groupId': 'BG000'}]}]}, {'title': 'White, Not Hispanic or Latino', 'categories': [{'measurements': [{'value': '4', 'groupId': 'BG000'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United States', 'categories': [{'measurements': [{'value': '6', 'groupId': 'BG000'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}, {'title': 'Modified Performance-Oriented Mobility Assessment - Gait subtest (MPOMA-G)', 'classes': [{'categories': [{'measurements': [{'value': '6.00', 'groupId': 'BG000', 'lowerLimit': '4.00', 'upperLimit': '8.00'}]}]}], 'paramType': 'MEDIAN', 'description': 'The MPOMA-G is a 5-component assessment that is used to evaluate gait performance. The first component has 4 sub-components. For 2 components and 2 sub-components, scores of 0 or 1 are assigned while scores of 0, 1 or 2 are assigned to the rest of the 2 components and 2 sub-components based on type of ambulation pattern observed. The maximum total score of 12 points = no impairment and 0 points = worst impairment.', 'unitOfMeasure': 'units on a scale', 'dispersionType': 'INTER_QUARTILE_RANGE'}, {'title': 'Deceased/Alive Status - Deceased', 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}, {'title': 'Deceased/Alive Status - Alive', 'classes': [{'categories': [{'measurements': [{'value': '6', 'groupId': 'BG000'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}], 'populationDescription': 'All patients who met all of the inclusion and none of the exclusion criteria were defined as the enrolled population, which was also the analysis population.'}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'RETROSPECTIVE', 'observationalModel': 'OTHER'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 6}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2014-08'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2019-03', 'completionDateStruct': {'date': '2014-09', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2019-03-11', 'studyFirstSubmitDate': '2014-09-05', 'resultsFirstSubmitDate': '2015-08-25', 'studyFirstSubmitQcDate': '2014-09-09', 'lastUpdatePostDateStruct': {'date': '2019-03-13', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2015-08-25', 'studyFirstPostDateStruct': {'date': '2014-09-10', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2015-09-28', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2014-09', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Modified Performance-Oriented Mobility Assessment-Gait Subtest (MPOMA-G) - Change From Baseline to Last Overall', 'timeFrame': "The earliest available MPOMA-G score that was assessed within the period of patients' aged 5 to 15 years, inclusive.", 'description': 'The MPOMA-G is a 5-component assessment that is used to evaluate gait performance. The first component has 4 sub-components. For 2 components and 2 sub-components, scores of 0 or 1 are assigned while scores of 0, 1 or 2 are assigned to the rest of the 2 components and 2 sub-components based on type of ambulation pattern observed. The maximum total score of 12 points = no impairment and 0 points = worst impairment.'}], 'secondaryOutcomes': [{'measure': 'Performance-Oriented Mobility Assessment-Gait Subtest (POMA-G) - Change From Baseline to Last Overall', 'timeFrame': "The earliest available MPOMA-G score that was assessed within the period of patients' aged 5 to 15 years, inclusive.", 'description': 'The POMA-G is a 7-component assessment that is used to evaluate gait performance. The second component has 4 sub-components. Scores of 0, 1 or 2 are assigned to 2 components while scores of 0 or 1 are assigned to the rest of the 4 components and 4 sub-components based on type of ambulation pattern observed. The maximum total score of 12 points = no impairment and 0 points = worst impairment.'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'keywords': ['HPP', 'Soft Bones', 'low Alkaline Phosphatase', 'genetic metabolic disorder', 'alkaline phosphatase', 'tissue non-specific alkaline phosphatase', 'rickets', 'osteomalacia'], 'conditions': ['Hypophosphatasia', 'Bone Disease']}, 'referencesModule': {'seeAlsoLinks': [{'url': 'http://www.hypophosphatasia.com', 'label': 'Hypophosphatasia Website'}, {'url': 'http://www.hypophosphatasia.com/hcp/', 'label': 'Hypophosphatasia Website for Health Care Professionals'}, {'url': 'http://www.magicfoundation.org', 'label': 'HPP support group'}, {'url': 'http://www.softbones.org', 'label': 'US Hypophosphatasia Group (Soft Bones)'}]}, 'descriptionModule': {'briefSummary': 'The purpose of this study is to characterize the natural history of HPP in patients with Juvenile-onset HPP who served as historical controls in ENB-006-09.', 'detailedDescription': 'Hypophosphatasia (HPP) is a life-threatening, genetic, and ultra-rare metabolic disease characterized by defective bone mineralization and impaired phosphate and calcium regulation that can lead to progressive damage to multiple vital organs, including destruction and deformity of bones, profound muscle weakness, seizures, impaired renal function, and respiratory failure. There are no approved disease-modifying treatments for patients with this disease. There is also limited data available on the natural course of this disease over time, particularly in patients with the juvenile-onset form.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '5 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Patient has juvenile-onset HPP and was selected as a historical-control for Study ENB-006-09.', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Documented informed consent/assent prior to video collection and data abstraction.\n* Documented informed consent/assent in Study ALX-HPP-502.\n* Patients with juvenile-onset HPP and was selected as a historical-control for Study ENB-006-09.\n* Patients with at least 2 videos of basic mobility that were recorded at 2 different clinic visits when the patient was ≥5 to ≤15 ye\n\nExclusion Criteria:\n\n* There are no exclusion criteria for this study'}, 'identificationModule': {'nctId': 'NCT02235493', 'briefTitle': 'Non-interventional Substudy of ALX-HPP-502 to Assess Natural History of Patients With Juvenile-onset HPP Who Served as Historical Controls in ENB-006-09', 'organization': {'class': 'INDUSTRY', 'fullName': 'Alexion Pharmaceuticals, Inc.'}, 'officialTitle': 'A Single-Center, Non-interventional Substudy of ALX-HPP-502 to Assess Functional Natural History Data of Patients With Juvenile Historical Controls in ENB-006-09', 'orgStudyIdInfo': {'id': 'ALX-HPP-502s'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'Retrospective Case Only', 'interventionNames': ['Biological: Retrospective Case Only']}], 'interventions': [{'name': 'Retrospective Case Only', 'type': 'BIOLOGICAL', 'armGroupLabels': ['Retrospective Case Only']}]}, 'contactsLocationsModule': {'locations': [{'city': 'St Louis', 'state': 'Missouri', 'country': 'United States', 'geoPoint': {'lat': 38.62727, 'lon': -90.19789}}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Alexion Pharmaceuticals, Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}