Ignite Creation Date:
2025-12-25 @ 2:13 AM
Ignite Modification Date:
2026-02-21 @ 9:25 PM
Study NCT ID:
NCT02333760
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2021-06-03
First Post:
2014-10-28
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Long Term Safety Follow up of Haematopoietic Stem Cell Gene Therapy for the Wiskott Aldrich Syndrome
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D014923', 'term': 'Wiskott-Aldrich Syndrome'}], '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': 'D008231', 'term': 'Lymphopenia'}, {'id': 'D007970', 'term': 'Leukopenia'}, {'id': 'D000095542', 'term': 'Cytopenia'}, {'id': 'D006474', 'term': 'Hemorrhagic Disorders'}, {'id': 'D007960', 'term': 'Leukocyte Disorders'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D040181', 'term': 'Genetic Diseases, X-Linked'}, {'id': 'D000081207', 'term': 'Primary Immunodeficiency Diseases'}, {'id': 'D007153', 'term': 'Immunologic Deficiency Syndromes'}, {'id': 'D007154', 'term': 'Immune System Diseases'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1', 'PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'OTHER', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 10}}, 'statusModule': {'overallStatus': 'ACTIVE_NOT_RECRUITING', 'startDateStruct': {'date': '2014-09'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2021-05', 'completionDateStruct': {'date': '2032-10', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2021-05-31', 'studyFirstSubmitDate': '2014-10-28', 'studyFirstSubmitQcDate': '2015-01-05', 'lastUpdatePostDateStruct': {'date': '2021-06-03', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2015-01-07', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2032-10', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Incidence and type of SAEs', 'timeFrame': 'yearly from 3 years to 15 years', 'description': 'Incidence and nature of delayed events such as malignancies, hematologic, autoimmune events, mortality'}, {'measure': 'Lentiviral integration sites', 'timeFrame': 'yearly from 3 years to 15 years (from 11 to 15 yearly time points, only in case of Advers Events of Special Interest)', 'description': 'Presence of lentiviral integration sites in different cells sub-populations'}, {'measure': 'Vector copy numbers', 'timeFrame': 'yearly from 3 years to 15 years (from 11 to 15 yearly time points, only in case of Advers Events of Special Interest)', 'description': 'Quantification of vector copy numbers on sorted cells population by q-PCR'}, {'measure': 'Replication competent lentivirus (RCL)', 'timeFrame': 'yearly from 3 years to 15 years (from 11 to 15 yearly time points, only in case of Advers Events of Special Interest)', 'description': 'Presence of RCL'}, {'measure': 'Change in medical conditions', 'timeFrame': 'yearly from 3 years to 10 years', 'description': 'Weight and complete clinical exam'}, {'measure': 'Key medical events related to WAS', 'timeFrame': 'yearly from 3 years to 10 years', 'description': 'Eczema status, infections, bleeding symptoms, autoimmune manifestation'}, {'measure': 'Hematological reconstitution', 'timeFrame': 'yearly from 3 years to 10 years', 'description': 'CBC including platelets count and size'}, {'measure': 'Reconstitution of cell mediated and humoral immunity', 'timeFrame': 'yearly from 3 years to 10 years (from 3 years to 5 years for PHA and candida )', 'description': 'Immunophenotyping panel, whole blood lymphocytes proliferation assays, restoration of antibody production, humoral response to antigene'}], 'secondaryOutcomes': [{'measure': 'Need for associated treatments', 'timeFrame': 'yearly from 3 years to 15 years', 'description': 'Immunoglobulins, antibacterial, antifungal, antiviral drugs, transfusions'}, {'measure': 'Representation of TCR families', 'timeFrame': 'yearly from 3 years to 5 years', 'description': 'Representation of TCR families by PCR TREC (TCR excision circle) and TCR V beta panel'}, {'measure': 'Bone marrow content', 'timeFrame': 'yearly from 3 years to 5 years (optional)', 'description': 'Numbers and type of cells in bone marrow'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'conditions': ['Wiskott-Aldrich Syndrome']}, 'referencesModule': {'references': [{'pmid': '35075289', 'type': 'DERIVED', 'citation': 'Magnani A, Semeraro M, Adam F, Booth C, Dupre L, Morris EC, Gabrion A, Roudaut C, Borgel D, Toubert A, Clave E, Abdo C, Gorochov G, Petermann R, Guiot M, Miyara M, Moshous D, Magrin E, Denis A, Suarez F, Lagresle C, Roche AM, Everett J, Trinquand A, Guisset M, Bayford JX, Hacein-Bey-Abina S, Kauskot A, Elfeky R, Rivat C, Abbas S, Gaspar HB, Macintyre E, Picard C, Bushman FD, Galy A, Fischer A, Six E, Thrasher AJ, Cavazzana M. Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott-Aldrich syndrome. Nat Med. 2022 Jan;28(1):71-80. doi: 10.1038/s41591-021-01641-x. Epub 2022 Jan 24.'}]}, 'descriptionModule': {'briefSummary': 'An open follow up study of patients enrolled in the Phase 1/2 clinical trial of haematopoietic stem cell gene therapy for the Wiskott-Aldrich Syndrome and treated with autologous CD34+ cells transduced with the w1.6\\_hWASP\\_WPRE (VSVg) lentiviral vector.'}, 'eligibilityModule': {'sex': 'MALE', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Patients enrolled in the initial phase I/II WAS conducted in France and United Kingdom (GTG002.07 and GTG003.08).\n* Parents, guardians or patient signed informed consent, guardians or patient signed informed consent\n\nExclusion Criteria:\n\n• Parents, guardians, patients unwilling to return for the follow up study period.'}, 'identificationModule': {'nctId': 'NCT02333760', 'acronym': 'WASFUP', 'briefTitle': 'Long Term Safety Follow up of Haematopoietic Stem Cell Gene Therapy for the Wiskott Aldrich Syndrome', 'organization': {'class': 'OTHER', 'fullName': 'Genethon'}, 'officialTitle': 'Long Term Safety Follow up of Patients Enrolled in the Phase I/II Clinical Trial of Haematopoietic Stem Cell Gene Therapy for the Wiskott Aldrich Syndrome (GTG002-07 and GTG003-08).', 'orgStudyIdInfo': {'id': 'GNT-WAS-03'}}, 'armsInterventionsModule': {'interventions': [{'name': 'Autologous CD34+ cells transduced with WASP lentiviral vector', 'type': 'GENETIC', 'description': "Follow up of ex vivo gene therapy transplantation of patient's autologous CD34+ cells transduced with lentiviral vector containing human WASP gene"}]}, 'contactsLocationsModule': {'locations': [{'zip': '75743', 'city': 'Paris', 'country': 'France', 'facility': 'Hopital Necker - Enfants Malades', 'geoPoint': {'lat': 48.85341, 'lon': 2.3488}}, {'zip': 'WC1N 1EH', 'city': 'London', 'country': 'United Kingdom', 'facility': 'UCL Institute of Child Health', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Genethon', 'class': 'OTHER'}, 'responsibleParty': {'type': 'SPONSOR'}}}}