Ignite Creation Date:
2025-12-24 @ 5:37 PM
Ignite Modification Date:
2025-12-28 @ 5:00 AM
Study NCT ID:
NCT01451268
Status:
UNKNOWN
Last Update Posted:
2018-03-20
First Post:
2011-09-30
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Phase I/II Study With Oral Panobinostat Maintenance Therapy Following Allogeneic Stem Cell Transplantation in Patients With High Risk Myelodysplastic Syndrome (MDS) or Acute Myeloid Leukemia (AML)
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D009190', 'term': 'Myelodysplastic Syndromes'}, {'id': 'D015470', 'term': 'Leukemia, Myeloid, Acute'}], 'ancestors': [{'id': 'D001855', 'term': 'Bone Marrow Diseases'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D007951', 'term': 'Leukemia, Myeloid'}, {'id': 'D007938', 'term': 'Leukemia'}, {'id': 'D009370', 'term': 'Neoplasms by Histologic Type'}, {'id': 'D009369', 'term': 'Neoplasms'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D000077767', 'term': 'Panobinostat'}], 'ancestors': [{'id': 'D006877', 'term': 'Hydroxamic Acids'}, {'id': 'D006898', 'term': 'Hydroxylamines'}, {'id': 'D000588', 'term': 'Amines'}, {'id': 'D009930', 'term': 'Organic Chemicals'}, {'id': 'D006880', 'term': 'Hydroxy Acids'}, {'id': 'D002264', 'term': 'Carboxylic Acids'}, {'id': 'D007211', 'term': 'Indoles'}, {'id': 'D006574', 'term': 'Heterocyclic Compounds, 2-Ring'}, {'id': 'D000072471', 'term': 'Heterocyclic Compounds, Fused-Ring'}, {'id': 'D006571', 'term': 'Heterocyclic Compounds'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1', 'PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 62}}, 'statusModule': {'overallStatus': 'UNKNOWN', 'lastKnownStatus': 'ACTIVE_NOT_RECRUITING', 'startDateStruct': {'date': '2011-01', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2018-03', 'completionDateStruct': {'date': '2018-04', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2018-03-19', 'studyFirstSubmitDate': '2011-09-30', 'studyFirstSubmitQcDate': '2011-10-11', 'lastUpdatePostDateStruct': {'date': '2018-03-20', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2011-10-13', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2018-04', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Maximum tolerated dose (MTD) of panobinostat', 'timeFrame': 'after 28 days of administration'}, {'measure': 'Dose-limiting toxicity (MTD) of Panobinostat', 'timeFrame': 'after 28 days of administration'}], 'secondaryOutcomes': [{'measure': 'Cumulative incidence of hematologic relapse and death', 'timeFrame': 'one year after HSCT'}, {'measure': 'Reconstitution of the immune system as measured by changes in numbers, ratio, phenotype and activation state of peripheral blood cell populations during panobinostat therapy', 'timeFrame': 'patients will be followed for up to 2 years depending on the duration of study participation'}, {'measure': 'Time to complete donor chimerism', 'timeFrame': 'patients will be followed for up to 2 years depending on the duration of study participation'}, {'measure': 'Cumulative incidence of extensive chronic GvHD', 'timeFrame': 'one year after HSCT'}, {'measure': 'Duration of complete donor chimerism', 'timeFrame': 'patients will be followed for up to 2 years depending on the duration of study participation'}, {'measure': 'Cumulative incidence of severe acute GvHD', 'timeFrame': 'one year after HSCT'}, {'measure': 'patient-reported health-related quality of life', 'timeFrame': 'after 3 months of administration and one month after last intake of study drug'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'keywords': ['60 to 150 Days After Allogeneic Stem Cell Transplantation', 'High Risk MDS', 'MDS', 'AML'], 'conditions': ['Myelodysplastic Syndrome', 'Acute Myeloid Leukemia']}, 'referencesModule': {'seeAlsoLinks': [{'url': 'http://www.studienregister-online.de', 'label': 'Trial register of the Kompetenznetz Leukämien, additional trial information'}]}, 'descriptionModule': {'briefSummary': "The study's primary objective is to determine the maximum tolerated dose (MTD) and dose-limiting toxicity (DLT) of Panobinostat when administered within 150 days after hematopoietic stem cell transplantation (HSCT) and given in conjunction with standard immunosuppressive therapy after HSCT for patients with high-risk Myelodysplastic Syndrome (MDS) or Acute Myeloid Leukemia (AML).\n\nSecondary objectives are\n\n* To determine safety and tolerability of panobinostat\n* To determine overall and disease-free survival at 12 months after HSCT\n* To evaluate immunoregulatory properties of panobinostat\n* To evaluate patient-reported health-related quality of life (HRQL)\n\nThe hypothesis of this study is that panobinostat can be an effective drug in preventing relapse of MDS and AML patients with high-risk features after hematopoietic stem cell transplantation with reduced-intensity conditioning (RIC-HSCT) while at the same time reducing graft-versus-host disease (GvHD) with preservation of graft-versus-leukemia (GvL) effect."}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* AML (except acute promyelocytic leukemia, AML M3) with high-risk features defined as one or more of the following criteria:\n\n * refractory to or relapsed after at least one cycle of standard chemotherapy\n * \\> 10% bone marrow blasts at day 15 of the first induction cycle\n * adverse risk cytogenetics including complex karyotype (≥ 3 abnormalities or abnormalities of chromosomes 3, 5 or 7) regardless of stage\n * secondary to MDS or radio-/chemotherapy or\n* MDS RAEB according to the WHO classification or intermediate-2 or high-risk according to IPSS or\n* Chronic myelomonocytic leukemia (CMML) with ≥ 5% bone marrow blasts and\n\n * Allogeneic HSCT with reduced intensity conditioning (see Section 15.1 for definition) performed within 60 - 150 days prior to study entry\n * Complete hematologic remission documented by bone marrow aspiration within 28 days prior to study entry\n\nExclusion Criteria:\n\n* Active acute GvHD overall grade 2 - 4\n* Prior treatment with a deacetylase (DAC) inhibitor\n* Patients with impaired cardiac function or other concurrent severe and/or uncontrolled medical conditions\n* Clinical symptoms suggesting central nervous system (CNS) leukemia\n* Patient has an impairment of gastrointestinal (GI) function or GI disease that may significantly alter the absorption of oral panobinostat'}, 'identificationModule': {'nctId': 'NCT01451268', 'acronym': 'PANOBEST', 'briefTitle': 'Phase I/II Study With Oral Panobinostat Maintenance Therapy Following Allogeneic Stem Cell Transplantation in Patients With High Risk Myelodysplastic Syndrome (MDS) or Acute Myeloid Leukemia (AML)', 'organization': {'class': 'OTHER', 'fullName': 'Johann Wolfgang Goethe University Hospital'}, 'officialTitle': 'Phase I/II Study With Oral Panobinostat Maintenance Therapy Following Allogeneic Stem Cell Transplantation in Patients With High Risk MDS or AML (PANOBEST)', 'orgStudyIdInfo': {'id': 'CLBH589 BDE05T'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Panobinostat Arm A', 'interventionNames': ['Drug: Panobinostat']}, {'type': 'EXPERIMENTAL', 'label': 'Panobinostat Arm B', 'interventionNames': ['Drug: Panobinostat']}], 'interventions': [{'name': 'Panobinostat', 'type': 'DRUG', 'otherNames': ['LBH589'], 'description': '10mg upto 40mg Panobinostat dose escalation in consequent cohorts; frequency: three times a week, every week; duration: one year', 'armGroupLabels': ['Panobinostat Arm A']}, {'name': 'Panobinostat', 'type': 'DRUG', 'otherNames': ['LBH589'], 'description': 'Start of Arm B after completion of Arm A; initial dose-level: one level below MTD of Arm A; 10mg upto 60mg Panobinostat dose escalation in consequent cohorts; frequency: three times a week, every other week; duration: one year', 'armGroupLabels': ['Panobinostat Arm B']}]}, 'contactsLocationsModule': {'locations': [{'zip': '40225', 'city': 'Düsseldorf', 'country': 'Germany', 'facility': 'University Hospital Düsseldorf', 'geoPoint': {'lat': 51.22172, 'lon': 6.77616}}, {'zip': '45147', 'city': 'Essen', 'country': 'Germany', 'facility': 'University Hospital Essen', 'geoPoint': {'lat': 51.45657, 'lon': 7.01228}}, {'zip': '60590', 'city': 'Frankfurt am Main', 'country': 'Germany', 'facility': 'University Hospital Frankfurt', 'geoPoint': {'lat': 50.11552, 'lon': 8.68417}}, {'zip': '20246', 'city': 'Hamburg', 'country': 'Germany', 'facility': 'University Hospital Hamburg-Eppendorf', 'geoPoint': {'lat': 53.55073, 'lon': 9.99302}}, {'zip': '55131', 'city': 'Mainz', 'country': 'Germany', 'facility': 'University Hospital Mainz', 'geoPoint': {'lat': 49.98185, 'lon': 8.28008}}, {'zip': '35043', 'city': 'Marburg', 'country': 'Germany', 'facility': 'University Hospital Marburg', 'geoPoint': {'lat': 50.80904, 'lon': 8.77069}}], 'overallOfficials': [{'name': 'Gesine Bug, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Johann Wolfgang Goethe University Hospital'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Johann Wolfgang Goethe University Hospital', 'class': 'OTHER'}, 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'Senior physician hematology', 'investigatorFullName': 'Gesine Bug', 'investigatorAffiliation': 'Johann Wolfgang Goethe University Hospital'}}}}