Ignite Creation Date:
2025-12-25 @ 4:23 AM
Ignite Modification Date:
2025-12-26 @ 3:25 AM
Study NCT ID:
NCT01279720
Status:
COMPLETED
Last Update Posted:
2015-09-14
First Post:
2011-01-18
Is Possible Gene Therapy:
True
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Gene Therapy ADA Deficiency
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'C531816', 'term': 'Severe combined immunodeficiency due to adenosine deaminase deficiency'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1', 'PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 8}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2003-10'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2015-09', 'completionDateStruct': {'date': '2013-11', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2015-09-11', 'studyFirstSubmitDate': '2011-01-18', 'studyFirstSubmitQcDate': '2011-01-18', 'lastUpdatePostDateStruct': {'date': '2015-09-14', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2011-01-19', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2013-11', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Immunological reconstitution', 'timeFrame': '5 years', 'description': 'Measurement of Immunological reconstitution and Metabolic Correction. 5 year follow up of the last patient enrolled into study'}], 'secondaryOutcomes': [{'measure': 'Incidence of adverse reactions', 'timeFrame': '5 years', 'description': 'Incidence of adverse reactions. 5 year follow up of the last patient enrolled into study'}, {'measure': 'Molecular characterisation of gene transfer', 'timeFrame': '5 years', 'description': 'Molecular characterisation of gene transfer. 5 year follow up of the last patient enrolled into study'}, {'measure': 'Normalisation of nutritional status, growth, and development', 'timeFrame': '5 years', 'description': 'Normalisation of nutritional status, growth, and development. 5 year follow up of the last patient enrolled into study'}]}, 'conditionsModule': {'keywords': ['Adenosine deaminase deficiency', 'Gene therapy'], 'conditions': ['Adenosine Deaminase Deficiency']}, 'referencesModule': {'references': [{'pmid': '12089448', 'type': 'BACKGROUND', 'citation': 'Aiuti A, Slavin S, Aker M, Ficara F, Deola S, Mortellaro A, Morecki S, Andolfi G, Tabucchi A, Carlucci F, Marinello E, Cattaneo F, Vai S, Servida P, Miniero R, Roncarolo MG, Bordignon C. Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning. Science. 2002 Jun 28;296(5577):2410-3. doi: 10.1126/science.1070104.'}, {'pmid': '8867866', 'type': 'BACKGROUND', 'citation': 'Hoogerbrugge PM, van Beusechem VW, Fischer A, Debree M, le Deist F, Perignon JL, Morgan G, Gaspar B, Fairbanks LD, Skeoch CH, Moseley A, Harvey M, Levinsky RJ, Valerio D. Bone marrow gene transfer in three patients with adenosine deaminase deficiency. Gene Ther. 1996 Feb;3(2):179-83.'}, {'pmid': '7749407', 'type': 'BACKGROUND', 'citation': 'Hershfield MS. PEG-ADA: an alternative to haploidentical bone marrow transplantation and an adjunct to gene therapy for adenosine deaminase deficiency. Hum Mutat. 1995;5(2):107-12. doi: 10.1002/humu.1380050202.'}]}, 'descriptionModule': {'briefSummary': 'Adenosine deaminase deficiency is an inherited disorder that results in severe abnormalities of the immune system and leaves children unable to fight infection. This trial aims to treat adenosine deaminase deficiency patients using gene therapy.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n1. Patients who lack a human leukocyte antigen (HLA)-genotypically identical bone marrow donor OR phenotypically matched family or unrelated donor AND who show incomplete immune reconstitution on Polyethylene glycol-modified adenosine deaminase (PEG-ADA) enzyme replacement therapy (defined by absolute CD4+ count \\<300 cell/mm3 and who remain on immunoglobulin replacement therapy)\n2. Diagnosis of ADA-SCID (Severe combined immunodeficiency (SCID) due to adenosine deaminase (ADA)confirmed by DNA sequencing OR by confirmed absence of \\<3% of ADA enzymatic activity in peripheral blood or (for neonates) in umbilical cord blood erythrocytes and/or leukocytes or in cultured fetal cells derived from either chorionic villus biopsy or amniocentesis, prior to institution of PEG-ADA replacement therapy\n3. Parental/guardian/patient signed informed consent'}, 'identificationModule': {'nctId': 'NCT01279720', 'briefTitle': 'Gene Therapy ADA Deficiency', 'organization': {'class': 'OTHER', 'fullName': 'Great Ormond Street Hospital for Children NHS Foundation Trust'}, 'officialTitle': 'Phase I Gene Therapy Protocol for Adenosine Deaminase Deficiency', 'orgStudyIdInfo': {'id': '03MI14'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Intravenous infusion of transduced cells', 'description': 'Intravenous infusion of transduced cells', 'interventionNames': ['Biological: Intravenous infusion of transduced cells']}], 'interventions': [{'name': 'Intravenous infusion of transduced cells', 'type': 'BIOLOGICAL', 'description': 'Intravenous infusion of transduced cells', 'armGroupLabels': ['Intravenous infusion of transduced cells']}]}, 'contactsLocationsModule': {'locations': [{'zip': 'WC1N 1EH', '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': {'type': 'SPONSOR'}}}}