Ignite Creation Date:
2025-12-24 @ 2:40 PM
Ignite Modification Date:
2025-12-24 @ 2:40 PM
Study NCT ID:
NCT07264959
Status:
NOT_YET_RECRUITING
Last Update Posted:
2025-12-04
First Post:
2025-11-24
Is Possible Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
An Observational Study in Participants With Indolent Systemic Mastocytosis (ISM)
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D034721', 'term': 'Mastocytosis, Systemic'}, {'id': 'D008415', 'term': 'Mastocytosis'}], 'ancestors': [{'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': 'D000090362', 'term': 'Mast Cell Activation Disorders'}, {'id': 'D007154', 'term': 'Immune System Diseases'}]}}, 'protocolSection': {'designModule': {'bioSpec': {'retention': 'SAMPLES_WITHOUT_DNA', 'description': "With the participant's informed consent and as approved by local IRBs/IECs, de-identified biological samples may be stored at a biosample repository and may be shared with the data repository. These samples could be used to research the causes of ISM, its complications and other conditions for which individuals with ISM are at increased risk, and to improve treatment. The repository will also be provided with a code-link that will allow linking the biological specimens with the phenotypic data from each participant, maintaining the blinding of the identity of the participant."}, 'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 150}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'NOT_YET_RECRUITING', 'startDateStruct': {'date': '2026-02-01', 'type': 'ESTIMATED'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-11', 'completionDateStruct': {'date': '2032-12-01', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-11-24', 'studyFirstSubmitDate': '2025-11-24', 'studyFirstSubmitQcDate': '2025-11-24', 'lastUpdatePostDateStruct': {'date': '2025-12-04', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2025-12-04', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2032-12-01', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Baseline Demographics', 'timeFrame': 'Baseline (Month 1)'}, {'measure': 'Change From Baseline in Patient-Reported Outcomes Scores', 'timeFrame': 'Baseline up to Month 61'}, {'measure': 'Change From Baseline in Serum KIT D816V Variant Allele Frequency (VAF)', 'timeFrame': 'Baseline up to Month 61'}, {'measure': 'Change From Baseline in Serum Tryptase', 'timeFrame': 'Baseline up to Month 61'}, {'measure': 'Change From Baseline in Indolent Systemic Mastocytosis (ISM) Symptom-Directed Therapies', 'timeFrame': 'Baseline up to Month 61'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'keywords': ['ISM', 'Avapritinib', 'BLU-285', 'Selective KIT mutation-targeted tyrosine kinase inhibitor', 'Tyrosine kinase inhibitor', 'Mastocytosis', 'Neoplastic mast cells'], 'conditions': ['Indolent Systemic Mastocytosis']}, 'descriptionModule': {'briefSummary': 'This is a Phase 4, non-interventional, observational study to collect detailed data on the clinical characteristics, clinical outcomes and medical management of ISM in real-world settings. The study will describe the demographic and clinical characteristics of ISM participants, including anaphylaxis and bone manifestations in ISM. Quality of life and disease control will be assessed through participant questionnaires. The study will also evaluate real world ISM treatment management, including use of avapritinib.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Participants with ISM who are currently being treated or plan to be treated with symptom-directed therapies and/or avapritinib for ISM.', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Male or female adult participants (≥ 18 years of age) with a diagnosis of ISM according to the World Health Organization (WHO) diagnostic criteria\n* Participant is currently being treated or plans to be treated with symptom-directed therapies and/or avapritinib for ISM.\n\nExclusion Criteria:\n\n* Participants with advanced systemic mastocytosis (AdvSM) or another associated hematologic neoplasm\n* Participants with smoldering systemic mastocytosis\n* Ongoing participation in interventional studies in systemic mastocytosis (SM) at the time of enrollment\n* Participants currently receiving treatment with a KIT inhibitor other than avapritinib at the time of enrollment.'}, 'identificationModule': {'nctId': 'NCT07264959', 'briefTitle': 'An Observational Study in Participants With Indolent Systemic Mastocytosis (ISM)', 'organization': {'class': 'INDUSTRY', 'fullName': 'Blueprint Medicines Corporation'}, 'officialTitle': 'An Observational Study in Patients With Indolent Systemic Mastocytosis', 'orgStudyIdInfo': {'id': 'BLU-285-2406'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'Participants With ISM', 'description': 'Patients who are currently being treated or plan to be treated for ISM'}]}, 'contactsLocationsModule': {'centralContacts': [{'name': 'Blueprint Medicines', 'role': 'CONTACT', 'email': 'medinfo@blueprintmedicines.com', 'phone': '1-888-258-7768'}, {'name': 'Blueprint Medicines, EU Contact', 'role': 'CONTACT', 'email': 'medinfoeurope@blueprintmedicines.com', 'phone': '+31 85 064 4001'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Blueprint Medicines Corporation', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}