Ignite Creation Date:
2025-12-25 @ 3:28 AM
Ignite Modification Date:
2026-02-25 @ 7:28 PM
Study NCT ID:
NCT02715505
Status:
WITHDRAWN
Last Update Posted:
2018-11-28
First Post:
2016-03-16
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Safety and Exploratory Efficacy of HSC835 in Patients With Inherited Metabolic Disorders (IMD)
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'removedCountries': ['United States']}, 'conditionBrowseModule': {'meshes': [{'id': 'D008059', 'term': 'Mucopolysaccharidosis I'}, {'id': 'D007966', 'term': 'Leukodystrophy, Metachromatic'}], 'ancestors': [{'id': 'D009083', 'term': 'Mucopolysaccharidoses'}, {'id': 'D002239', 'term': 'Carbohydrate Metabolism, Inborn Errors'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D016464', 'term': 'Lysosomal Storage Diseases'}, {'id': 'D017520', 'term': 'Mucinoses'}, {'id': 'D003240', 'term': 'Connective Tissue Diseases'}, {'id': 'D017437', 'term': 'Skin and Connective Tissue Diseases'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}, {'id': 'D020279', 'term': 'Hereditary Central Nervous System Demyelinating Diseases'}, {'id': 'D020739', 'term': 'Brain Diseases, Metabolic, Inborn'}, {'id': 'D001928', 'term': 'Brain Diseases, Metabolic'}, {'id': 'D001927', 'term': 'Brain Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D052516', 'term': 'Sulfatidosis'}, {'id': 'D013106', 'term': 'Sphingolipidoses'}, {'id': 'D020140', 'term': 'Lysosomal Storage Diseases, Nervous System'}, {'id': 'D056784', 'term': 'Leukoencephalopathies'}, {'id': 'D003711', 'term': 'Demyelinating Diseases'}, {'id': 'D008064', 'term': 'Lipidoses'}, {'id': 'D008052', 'term': 'Lipid Metabolism, Inborn Errors'}, {'id': 'D052439', 'term': 'Lipid Metabolism Disorders'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1', 'PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 0}}, 'statusModule': {'whyStopped': 'This study was cancelled with no patients', 'overallStatus': 'WITHDRAWN', 'startDateStruct': {'date': '2017-10-10', 'type': 'ESTIMATED'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2018-11', 'completionDateStruct': {'date': '2020-05-18', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2018-11-26', 'studyFirstSubmitDate': '2016-03-16', 'studyFirstSubmitQcDate': '2016-03-16', 'lastUpdatePostDateStruct': {'date': '2018-11-28', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2016-03-22', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2020-05-18', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Incidence of infusional toxicities', 'timeFrame': '48 hours'}, {'measure': 'Incidence of neutrophil recovery', 'timeFrame': '42 days'}, {'measure': 'Incidence of graft failure', 'timeFrame': '42 days'}], 'secondaryOutcomes': [{'measure': 'Time to neutrophil recovery', 'timeFrame': '42 days'}, {'measure': 'Time to platelet recovery', 'timeFrame': '180 days'}, {'measure': 'Number of patients with grade II-IV acute graft versus host disease (aGVHD)', 'timeFrame': '100 days'}, {'measure': 'Number of patients with chronic graft versus host disease (cGVHD)', 'timeFrame': '1 and 2 years'}, {'measure': 'Incidence of death', 'timeFrame': '100 days, 1 year and 2 years'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Hurler syndrome, MLD, Krabbe, cALD, Hematopoietic Stem Cell Transplantation (HSCT)'], 'conditions': ['Inherited Metabolic Disorders IMD']}, 'descriptionModule': {'briefSummary': 'This study is designed to assess the safety and exploratory efficacy of using HSC835 in patients with Inherited Metabolic Disorders (IMD) undergoing stem cell transplantation.', 'detailedDescription': 'This phase II study is designed to assess the safety of the Novartis product HSC835 and its ability to achieve donor blood stem cell engraftment in patients with certain Inherited Metabolic Disorders who undergo stem cell transplantation. A reduced intensity conditioning will be used prior to transplantation. Patients with Hurler syndrome, MLD, Krabbe or cALD could be eligible for this study.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '25 Years', 'minimumAge': '12 Months', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Diagnosed with Hurler syndrome, Metachromatic leukodystrophy (MLD), Globoid cell leukodystrophy (Krabbe) or Cerebral adrenoleukodystropy (cALD) -Adequate organ function -Availability of eligible donor material\n\nExclusion Criteria:\n\n* Availability of a matched-related donor who is not a carrier of the same genetic defect -Active infection at screening -Prior myeloablative transplant -Pregnant or nursing women and women of child bearing potential unless using highly effective contraception methods. For the pediatric population, female patients of child bearing potential who do not agree to abstinence or agree to use highly effective contraception methods -Sexually active male patients unless using condoms as contraception -Human Immunodeficiency virus (HIV) infection'}, 'identificationModule': {'nctId': 'NCT02715505', 'briefTitle': 'Safety and Exploratory Efficacy of HSC835 in Patients With Inherited Metabolic Disorders (IMD)', 'organization': {'class': 'INDUSTRY', 'fullName': 'Novartis'}, 'officialTitle': 'A Single-arm, Open-label, Study to Evaluate the Safety and Exploratory Efficacy of HSC835 in Patients With Inherited Metabolic Disorders (IMD) Undergoing Stem Cell Transplantation After Reduced Intensity Conditioning', 'orgStudyIdInfo': {'id': 'CHSC835X2203'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'HSC835', 'description': 'HSC835 is an expanded umbilical cord blood product used during single umbilical cord blood transplantation', 'interventionNames': ['Drug: Umbilical cord blood transplantation with HSC835']}], 'interventions': [{'name': 'Umbilical cord blood transplantation with HSC835', 'type': 'DRUG', 'otherNames': ['Spanlecortemlocel'], 'description': 'Hematopoietic Stem cell transplantation will be done with the cell therapy product HSC835', 'armGroupLabels': ['HSC835']}]}, 'ipdSharingStatementModule': {'ipdSharing': 'UNDECIDED', 'description': 'Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.\n\nThis trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Novartis Pharmaceuticals', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}