Ignite Creation Date:
2025-12-25 @ 4:04 AM
Ignite Modification Date:
2025-12-26 @ 3:00 AM
Study NCT ID:
NCT01204502
Status:
TERMINATED
Last Update Posted:
2013-09-18
First Post:
2010-09-16
Is Possible Gene Therapy:
True
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Suicide Gene Therapy Trial
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D006086', 'term': 'Graft vs Host Disease'}], 'ancestors': [{'id': 'D007154', 'term': 'Immune System Diseases'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1', 'PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 2}}, 'statusModule': {'whyStopped': 'Changes in the clinical practice', 'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2011-01'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2013-09', 'completionDateStruct': {'date': '2013-01', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2013-09-17', 'studyFirstSubmitDate': '2010-09-16', 'studyFirstSubmitQcDate': '2010-09-16', 'lastUpdatePostDateStruct': {'date': '2013-09-18', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2010-09-17', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2013-01', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'T-cell reconstitution (as defined by CD4+ cells >300/mm3 & CD3+ cells >500/mm3)', 'timeFrame': '12 months after final dose', 'description': 'T-cell reconstitution is measured until 12 months after administration of the final dose of gene modified cells'}], 'secondaryOutcomes': [{'measure': 'Incidence of GvHD', 'timeFrame': '12 months after final dose', 'description': 'Incidence of GvHD is measured until 12 months after administration of the final dose of gene modified cells'}, {'measure': 'Patient survival', 'timeFrame': '12 months after final dose', 'description': 'Patient survial is measured until 12 months after administration of the final dose of gene modified cells'}]}, 'conditionsModule': {'keywords': ['Gene therapy', 'Haploidentical', 'Bone marrow transplant', 'Graft versus host disease', 'haploidentical stem cell transplantation', 'T-cell suicide gene therapy'], 'conditions': ['Haploidentical Stem Cell Transplantation']}, 'referencesModule': {'references': [{'pmid': '15638716', 'type': 'BACKGROUND', 'citation': 'Qasim W, Gaspar HB, Thrasher AJ. T cell suicide gene therapy to aid haematopoietic stem cell transplantation. Curr Gene Ther. 2005 Feb;5(1):121-32. doi: 10.2174/1566523052997497.'}, {'pmid': '24204746', 'type': 'DERIVED', 'citation': 'Zhan H, Gilmour K, Chan L, Farzaneh F, McNicol AM, Xu JH, Adams S, Fehse B, Veys P, Thrasher A, Gaspar H, Qasim W. Production and first-in-man use of T cells engineered to express a HSVTK-CD34 sort-suicide gene. PLoS One. 2013 Oct 21;8(10):e77106. doi: 10.1371/journal.pone.0077106. eCollection 2013.'}]}, 'descriptionModule': {'briefSummary': 'Bone marrow or blood stem cell transplantation is used to treat a wide range of life-threatening conditions. T lymphocytes carried in the graft have powerful beneficial effects and play a vital role in the eradication of leukaemia and in fighting infection, but can also damage healthy tissues and cause graft-versus-host disease (GVHD).\n\nTo safeguard against GVHD, the investigators propose modifying T cells to encode a \'switch\' so that they can be eliminated if problems arise.\n\nChildren receiving half-matched (haploidentical) transplants from a parent are most likely to benefit from this strategy. At present these patients receive blood stem cells from a parent, but the T cells are removed because the risk of serious GVHD is unacceptable. This means that they are much more likely to suffer from life threatening infections or experience a relapse of leukaemia. The investigators want to use gene therapy to produce "safe" T cells which can be used to strengthen the transplant and prevent these serious complications.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD'], 'maximumAge': '16 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n1. Patients with primary immunodeficiencies, haematological malignancies or metabolic disorders at GOSH (children of both sexes, aged 0 to 16 years) undergoing haploidentical transplant\n2. Both patient and donor must give informed consent in writing.\n3. The donor must be willing, able and available for donation of T cells by collection of whole blood or leukapheresis.\n4. The patient should be free of serious intercurrent illness.\n\nExclusion Criteria:\n\n1. Donor unfit or unavailable\n2. Donor positive for Hepatitis B or C, or HTLV-1, or HIV\n3. Patient receiving Ganciclovir, Aciclovir, Cidofovir a result of active CMV, adenovirus, varicella zoster or herpes simplex infection infection\n4. GVHD ≥ grade II before infusion of gene modified T cells\n5. Serious intercurrent illness'}, 'identificationModule': {'nctId': 'NCT01204502', 'briefTitle': 'Suicide Gene Therapy Trial', 'organization': {'class': 'OTHER', 'fullName': 'Great Ormond Street Hospital for Children NHS Foundation Trust'}, 'officialTitle': 'Phase I/II Clinical Trial of T-cell Suicide Gene Therapy Following Haploidentical Stem Cell Transplantation', 'orgStudyIdInfo': {'id': '06MI04'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'HSVTK retrovirally-transduced donor T lymphocytes', 'description': 'HSVTK retrovirally-transduced donor T lymphocytes will be given at 1 month intervals, providing that there is no significant GVHD\n\n* dose 1 5x104 cells/kg\n* dose 2 5x105 cells/kg', 'interventionNames': ['Biological: HSVTK retrovirally-transduced donor T lymphocytes']}], 'interventions': [{'name': 'HSVTK retrovirally-transduced donor T lymphocytes', 'type': 'BIOLOGICAL', 'description': 'HSVTK retrovirally-transduced donor T lymphocytes will be given at 1 month intervals, providing that there is no significant GVHD\n\n* dose 1 5x104 cells/kg\n* dose 2 5x105 cells/kg', 'armGroupLabels': ['HSVTK retrovirally-transduced donor T lymphocytes']}]}, 'contactsLocationsModule': {'locations': [{'zip': 'WC1N 3JH', 'city': 'London', 'country': 'United Kingdom', 'facility': 'Great Ormond Street Hospital for Children NHS Trust', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Great Ormond Street Hospital for Children NHS Foundation Trust', 'class': 'OTHER'}, 'responsibleParty': {'oldNameTitle': 'Biren Patel', 'oldOrganization': 'Great Ormond Street Hospital for Children NHS Trust'}}}}