Ignite Creation Date:
2025-12-25 @ 12:33 AM
Ignite Modification Date:
2025-12-25 @ 10:41 PM
Study NCT ID:
NCT05234567
Status:
RECRUITING
Last Update Posted:
2025-08-26
First Post:
2022-02-01
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
A Prospective Sub-Study of the Global Hypophosphatasia Registry
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D007014', 'term': 'Hypophosphatasia'}], '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'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C570710', 'term': 'asfotase alfa'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 30}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2022-08-25', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-08', 'completionDateStruct': {'date': '2028-07-18', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-08-25', 'studyFirstSubmitDate': '2022-02-01', 'studyFirstSubmitQcDate': '2022-02-01', 'lastUpdatePostDateStruct': {'date': '2025-08-26', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2022-02-10', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2028-07-18', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Occurrence Of Immune-mediated Loss Of Effectiveness According To The Treating Physician', 'timeFrame': 'Up to 5 years', 'description': 'This will be based on clinical and biochemical assessments as well as positive anti-drug antibodies and positive neutralizing antibodies.'}, {'measure': 'Occurrence Of Immune-mediated Serious Adverse Events', 'timeFrame': 'Up to 5 years', 'description': 'These serious adverse events will include serious hypersensitivity reactions and anaphylaxis.'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Pediatric Onset', 'HPP', 'Asfotase alfa'], 'conditions': ['Hypophosphatasia']}, 'descriptionModule': {'briefSummary': 'In this prospective observational sub-study, participants with pediatric-onset hypophosphatasia (HPP) (perinatal/infantile- or juvenile-onset) of any age will be followed for a minimum of 5 years at sites in the United States and potentially 1 or 2 other countries.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'samplingMethod': 'PROBABILITY_SAMPLE', 'studyPopulation': 'Patients with HPP', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* Any age or sex with a confirmed diagnosis of pediatric-onset HPP (that is, first HPP sign or symptom presented at \\< 18 years of age).\n* Currently receiving asfotase alfa treatment at Enrollment (not treatment-naïve) or the Physician has decided to resume (not treatment-naïve) or start (treatment-naïve) the participant's asfotase alfa treatment within 6 months after Enrollment.\n* Participant must have documented alkaline phosphatase (ALP) activity below the lower limit of normal for age and sex, and a documented ALPL gene mutation (Note: An exception is made for infants with clinical features of HPP plus low ALP who need to start asfotase alfa treatment right away, at the Physician's discretion, but do not yet have a genetic result. In this case, ALPL gene documentation is not required at the time of sub-study enrollment but should be documented within 6 months after Enrollment).\n* Participant or participant's parent/legally authorized representative is able to read and/or understand the informed consent and study questionnaires in the local language.\n* Participant or participant's parent/legally authorized representative must be willing and able to give signed informed consent for this sub-study, and the participant must be willing to give written informed assent, if appropriate and required by local regulations.\n\nExclusion Criteria:\n\n* Currently participating in an Alexion-sponsored interventional clinical study. Participants who have concluded participation in an Alexion-sponsored asfotase alfa clinical study are eligible to enroll in this sub-study."}, 'identificationModule': {'nctId': 'NCT05234567', 'briefTitle': 'A Prospective Sub-Study of the Global Hypophosphatasia Registry', 'organization': {'class': 'INDUSTRY', 'fullName': 'Alexion Pharmaceuticals, Inc.'}, 'officialTitle': 'A Prospective Observational Sub-Study of the Global Hypophosphatasia Registry to Describe the Potential Risk of Immune-Mediated Loss of Pharmacological Effect of Asfotase Alfa in Participants With Hypophosphatasia', 'orgStudyIdInfo': {'id': 'ALX-HPP-501s'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'Participants with Pediatric-onset HPP', 'description': 'Each participant will be followed for a minimum of 5 years or, if applicable, until early withdrawal. Biochemical, clinical, imaging (if clinically indicated), and functional/quality of life outcomes relevant to HPP will be assessed.', 'interventionNames': ['Biological: Asfotase Alfa']}], 'interventions': [{'name': 'Asfotase Alfa', 'type': 'BIOLOGICAL', 'otherNames': ['Strensiq'], 'description': "All participants will receive asfotase alfa subcutaneously per standard of care. Unless otherwise specified per the Physician's standard of care, participants aged \\< 2 years are recommended for a clinic visit approximately every 3 months after Enrollment until 2 years of age, after which they should have a clinic visit approximately every 6 months. Participants should be followed for 5 years, as possible.", 'armGroupLabels': ['Participants with Pediatric-onset HPP']}]}, 'contactsLocationsModule': {'locations': [{'zip': '06106', 'city': 'Hartford', 'state': 'Connecticut', 'status': 'RECRUITING', 'country': 'United States', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 41.76371, 'lon': -72.68509}}, {'zip': '60611', 'city': 'Chicago', 'state': 'Illinois', 'status': 'RECRUITING', 'country': 'United States', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 41.85003, 'lon': -87.65005}}, {'zip': '02122', 'city': 'Boston', 'state': 'Massachusetts', 'status': 'RECRUITING', 'country': 'United States', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '64108', 'city': 'Kansas City', 'state': 'Missouri', 'status': 'RECRUITING', 'country': 'United States', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 39.09973, 'lon': -94.57857}}, {'zip': '11501', 'city': 'Mineola', 'state': 'New York', 'status': 'RECRUITING', 'country': 'United States', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 40.74927, 'lon': -73.64068}}, {'zip': '45229', 'city': 'Cincinnati', 'state': 'Ohio', 'status': 'NOT_YET_RECRUITING', 'country': 'United States', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 39.12711, 'lon': -84.51439}}, {'zip': '43203', 'city': 'Columbus', 'state': 'Ohio', 'status': 'RECRUITING', 'country': 'United States', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 39.96118, 'lon': -82.99879}}, {'zip': '15224', 'city': 'Pittsburgh', 'state': 'Pennsylvania', 'status': 'NOT_YET_RECRUITING', 'country': 'United States', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 40.44062, 'lon': -79.99589}}, {'zip': '37112', 'city': 'Nashville', 'state': 'Tennessee', 'status': 'RECRUITING', 'country': 'United States', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 36.16589, 'lon': -86.78444}}, {'zip': '84108', 'city': 'Salt Lake City', 'state': 'Utah', 'status': 'RECRUITING', 'country': 'United States', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 40.76078, 'lon': -111.89105}}, {'zip': '22903', 'city': 'Charlottesville', 'state': 'Virginia', 'status': 'RECRUITING', 'country': 'United States', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 38.02931, 'lon': -78.47668}}, {'zip': '53792', 'city': 'Madison', 'state': 'Wisconsin', 'status': 'RECRUITING', 'country': 'United States', 'facility': 'Clinical Trial Site', 'geoPoint': {'lat': 43.07305, 'lon': -89.40123}}], 'centralContacts': [{'name': 'Alexion Pharmaceuticals, Inc. (Sponsor)', 'role': 'CONTACT', 'email': 'clinicaltrials@alexion.com', 'phone': '1-855-752-2356'}]}, 'ipdSharingStatementModule': {'infoTypes': ['STUDY_PROTOCOL', 'SAP', 'CSR'], 'ipdSharing': 'YES', 'description': 'Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.', 'accessCriteria': 'Alexion will consider requests for disclosure of clinical study participant-level data provided that participant privacy is assured through methods like data de-identification, pseudonymization, or anonymization (as required by applicable law), and if such disclosure was included in the relevant study informed consent form or similar documentation. Qualified academic investigators may request participant-level clinical data and supporting documents (statistical analysis plan and protocol) pertaining to Alexion-sponsored studies. Further details regarding data availability and instructions for requesting information are available in the Alexion Clinical Trials Disclosure and Transparency Policy at https://alexion.com/our-research/research-and-development.'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Alexion Pharmaceuticals, Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}