Ignite Creation Date:
2025-12-25 @ 12:05 AM
Ignite Modification Date:
2026-02-23 @ 1:12 AM
Study NCT ID:
NCT02745158
Status:
RECRUITING
Last Update Posted:
2017-08-17
First Post:
2016-03-06
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
The Fibrodysplasia Ossificans Progressiva (FOP) Connection Registry
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D009221', 'term': 'Myositis Ossificans'}, {'id': 'D009999', 'term': 'Ossification, Heterotopic'}, {'id': 'D015831', 'term': 'Osteochondroma'}, {'id': 'D006215', 'term': 'Hallux Valgus'}], 'ancestors': [{'id': 'D009220', 'term': 'Myositis'}, {'id': 'D009135', 'term': 'Muscular Diseases'}, {'id': 'D009140', 'term': 'Musculoskeletal Diseases'}, {'id': 'D010335', 'term': 'Pathologic Processes'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}, {'id': 'D018213', 'term': 'Neoplasms, Bone Tissue'}, {'id': 'D009372', 'term': 'Neoplasms, Connective Tissue'}, {'id': 'D018204', 'term': 'Neoplasms, Connective and Soft Tissue'}, {'id': 'D009370', 'term': 'Neoplasms by Histologic Type'}, {'id': 'D009369', 'term': 'Neoplasms'}, {'id': 'D010009', 'term': 'Osteochondrodysplasias'}, {'id': 'D001848', 'term': 'Bone Diseases, Developmental'}, {'id': 'D001847', 'term': 'Bone Diseases'}, {'id': 'D005530', 'term': 'Foot Deformities'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 800}, 'targetDuration': '10 Years', 'patientRegistry': True}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2015-07', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2016-03', 'completionDateStruct': {'date': '2025-12', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2017-08-15', 'studyFirstSubmitDate': '2016-03-06', 'studyFirstSubmitQcDate': '2016-04-15', 'lastUpdatePostDateStruct': {'date': '2017-08-17', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2016-04-20', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2025-07', 'type': 'ESTIMATED'}}, 'outcomesModule': {'otherOutcomes': [{'measure': 'Patient Reported Changes in Activities of Daily Living Using the FOP-Physical Functioning Questionnaire (PFQ)', 'timeFrame': 'Baseline, then every six months for up to 10 years'}, {'measure': 'Patient Reported Changes in Overall Health Using the PROMIS Global Health Scale', 'timeFrame': 'Baseline, then every six months for up to 10 years'}, {'measure': 'Patient Reported Changes in the Need for Assistive Devices, Aids, Attendants, and Adaptations Using a Survey Developed by the IFOPA', 'timeFrame': 'Baseline, then every six months for up to 10 years', 'description': 'The patient is able to respond Yes or No to questions related to his/her need for Assistive Devices, Aids, Attendants, and Adaptations as a result of his/her FOP disease.'}], 'primaryOutcomes': [{'measure': 'Patient Reported Changes in New Bone Growth Using a Patient-Directed Survey Developed by the IFOPA', 'timeFrame': 'Baseline, then every six months for up to 10 years', 'description': 'The patient is able to report changes in new bone growth among 27 joints and body locations.'}, {'measure': 'Patient Reported Changes in Episodic Flare-Ups Using a Patient-Directed Survey Developed by the IFOPA', 'timeFrame': 'Baseline, then every six months for up to 10 years', 'description': 'The patient is able to report changes in episodic flare-ups among 27 joints and body locations.'}, {'measure': 'Patient Reported Changes in Mobility Using a Patient-Directed Survey Developed by the IFOPA', 'timeFrame': 'Baseline, then every six months for up to 10 years', 'description': 'The patient is able to report changes in mobility among 27 joints and body locations.'}], 'secondaryOutcomes': [{'measure': 'Patient Reported Changes in Health Resource Utilization Using a Patient-Directed Survey Developed by the IFOPA', 'timeFrame': 'Baseline, then every six months for up to 10 years', 'description': 'The number of visits to a Physician and a Dentist, as well as the number of hospitalizations and reasons for the hospitalizations, are used to assess health resource utilization.'}, {'measure': 'Patient Reported Changes in Signs Related to the Ear Using a Patient-Directed Survey Developed by the IFOPA', 'timeFrame': 'Baseline, then every six months for up to 10 years', 'description': 'The patient is able to report new or continuing health issues related to the ears.'}, {'measure': 'Patient Reported Changes in Signs Related to the Skin Using a Patient-Directed Survey Developed by the IFOPA', 'timeFrame': 'Baseline, then every six months for up to 10 years', 'description': 'The patient is able to report new or continuing health issues related to the skin.'}, {'measure': 'Patient Reported Changes in Signs Related to the Central Nervous System Using a Patient-Directed Survey Developed by the IFOPA', 'timeFrame': 'Baseline, then every six months for up to 10 years', 'description': 'The patient is able to report new or continuing health issues related to the central nervous system.'}, {'measure': 'Patient Reported Changes in Signs Related to the Pulmonary System Using a Patient-Directed Survey Developed by the IFOPA', 'timeFrame': 'Baseline, then every six months for up to 10 years', 'description': 'The patient is able to report new or continuing health issues related to the pulmonary system.'}, {'measure': 'Patient Reported Changes in Signs Related to the Gastrointestinal System Using a Patient-Directed Survey Developed by the IFOPA', 'timeFrame': 'Baseline, then every six months for up to 10 years', 'description': 'The patient is able to report new or continuing health issues related to the gastrointestinal system.'}, {'measure': 'Patient Reported Changes in Signs Related to the Cardiovascular System Using a Patient-Directed Survey Developed by the IFOPA', 'timeFrame': 'Baseline, then every six months for up to 10 years', 'description': 'The patient is able to report new or continuing health issues related to the cardiovascular system.'}, {'measure': 'Patient Reported Changes in Signs Related to the Renal System Using a Patient-Directed Survey Developed by the IFOPA', 'timeFrame': 'Baseline, then every six months for up to 10 years', 'description': 'The patient is able to report new or continuing health issues related to the renal system.'}, {'measure': 'Patient Reported Changes in Signs Related to the Endocrine System Using a Patient-Directed Survey Developed by the IFOPA', 'timeFrame': 'Baseline, then every six months for up to 10 years', 'description': 'The patient is able to report new or continuing health issues related to the endocrine system.'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'keywords': ['Fibrodysplasia ossificans progressiva', 'FOP', 'International FOP Association', 'Patient Advocacy Group', 'Patient Registry', 'Natural History', 'Patient-Reported Outcomes', 'Observational Database', 'Bone Morphogenetic Protein', 'BMP', 'Bone Growth', 'Joint', 'Mobility', 'Flare-Up', 'Heterotopic Ossification', 'Ectopic Bone', 'Osteochondroma', 'Hallux Valgus', 'ALK2', 'ACVR1', 'Activin A', 'IFOPA', 'Activin A Receptor Type I', 'Activin Receptor-like Kinase-2'], 'conditions': ['Fibrodysplasia Ossificans Progressiva (FOP)']}, 'referencesModule': {'references': [{'pmid': '36526263', 'type': 'DERIVED', 'citation': 'Pignolo RJ, Kimel M, Whalen J, Kawata AK, Artyomenko A, Kaplan FS. The Fibrodysplasia Ossificans Progressiva Physical Function Questionnaire (FOP-PFQ): A patient-reported, disease-specific measure. Bone. 2023 Mar;168:116642. doi: 10.1016/j.bone.2022.116642. Epub 2022 Dec 13.'}], 'seeAlsoLinks': [{'url': 'http://www.fopconnection.org', 'label': 'Click here for more information about this study: The FOP Connection Registry'}, {'url': 'http://www.ifopa.org', 'label': 'The International FOP Association is a 501(c)(3) non-profit organization supporting medical research, education and communication for those afflicted by the rare genetic condition Fibrodysplasia Ossificans Progressiva (FOP).'}]}, 'descriptionModule': {'briefSummary': 'The Fibrodysplasia Ossificans Progressiva (FOP) Connection Registry is a global, non-interventional, voluntary database that captures demographic and disease data directly from FOP patients and their caregivers via a secure, web-based patient portal. A physician portal (in development) will allow physicians to enter clinical data about their patients. The objectives are to organize the international FOP community for participation in clinical trials; to enable FOP patients worldwide to report data in a shared forum; to improve the collective understanding of FOP natural history; and to advance the understanding of FOP treatment outcomes.', 'detailedDescription': 'The FOP Connection Registry is a global, non-interventional, voluntary database that captures demographic and disease data directly from FOP patients and their caregivers via a secure, web-based patient portal. No experimental intervention is involved. The objectives are to organize the international FOP community for participation in clinical trials; to enable FOP patients worldwide to report data in a shared forum; to improve the collective understanding of FOP natural history; and to advance the understanding of FOP treatment outcomes.\n\nThe Registry collects data through two sources. First, the Patient Portal (launched in July 2015) allows FOP patients and caregivers to enter information about their experiences living with FOP. Second, the Physician Portal (in development) will allow physicians to enter clinical data about patients under their care. The Registry will be capable of including data on specific marketed therapies under the direction and control of a sponsoring pharmaceutical company. Key identifiers will link the physician-reported data with the Patient Portal data.\n\nParticipants must have a confirmed diagnosis of FOP and the participant (or a parent or legal guardian) must be willing and able to provide written informed consent. There are no exclusion criteria. Data collected in the Patient Portal include: patient demographics and diagnosis pathway; medical and dental care; clinical research participation and biospecimen donation; heterotopic ossification (bone growth and episodic flare-ups); other signs and symptoms by body system; patient-reported outcomes (physical functioning, pain, fatigue, and general health); and assistive devices, aids, attendants, and adaptations. Because the Registry is designed to accommodate participants along a broad spectrum of FOP disease severity, most of the data fields are optional, allowing participants a high degree of flexibility in how much information they contribute, which also minimizes participant burden.\n\nAfter completing the informed consent, participants enter their baseline (historical) data. Participants will be encouraged to update their information at least twice per year. Participants may withdraw their consent at any time without prejudice or providing an explanation. The Registry has no pre-specified end date and will continue for as long as it is sustainable and useful to the FOP community.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'samplingMethod': 'PROBABILITY_SAMPLE', 'studyPopulation': 'Individuals with Fibrodysplasia Ossificans Progressive (FOP) from the international FOP community will be invited to join the Registry.', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Participants must have a confirmed diagnosis of FOP.\n* Participants (or a parent or legal guardian) must be willing and able to provide written informed consent.\n\nExclusion Criteria:\n\n* There are no exclusion criteria.'}, 'identificationModule': {'nctId': 'NCT02745158', 'briefTitle': 'The Fibrodysplasia Ossificans Progressiva (FOP) Connection Registry', 'organization': {'class': 'OTHER', 'fullName': 'The International FOP Association'}, 'officialTitle': 'FOP Connection: A Global Registry for the Fibrodysplasia Ossificans Progressiva Community', 'orgStudyIdInfo': {'id': 'IFOPA-REG-001'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'FOP Patients'}]}, 'contactsLocationsModule': {'locations': [{'zip': '32707', 'city': 'Casselberry', 'state': 'Florida', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Neal S Mantick, BS, MS', 'role': 'CONTACT', 'email': 'registry@fopconnection.org', 'phone': '617-910-8508'}], 'facility': 'The International FOP Association', 'geoPoint': {'lat': 28.67778, 'lon': -81.32785}}], 'centralContacts': [{'name': 'Neal S Mantick, BS, MS', 'role': 'CONTACT', 'email': 'registry@fopconnection.org', 'phone': '1-617-910-8508'}, {'name': 'Betsy Bogard, BS, MS', 'role': 'CONTACT', 'email': 'registry@fopconnection.org', 'phone': '1-407-365-4194'}], 'overallOfficials': [{'name': 'Betsy Bogard, BS, MS', 'role': 'STUDY_DIRECTOR', 'affiliation': 'The International FOP Association'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'YES', 'description': 'Formal data sharing agreements are planned between the International FOP Association and biopharmaceutical companies doing research in Fibrodysplasia Ossificans Progressiva (FOP). Publications are also planned with biopharmaceutical companies and physician researchers.'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'The International FOP Association', 'class': 'OTHER'}, 'responsibleParty': {'type': 'SPONSOR'}}}}