Ignite Creation Date:
2025-12-24 @ 7:45 PM
Ignite Modification Date:
2026-01-06 @ 11:00 PM
Study NCT ID:
NCT04959903
Status:
RECRUITING
Last Update Posted:
2023-03-06
First Post:
2021-07-01
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Safety and Efficacy of SMART101 in Pediatric and Adult Patients With Hematological Malignancies After T Cell Depleted Allo-HSCT
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D019337', 'term': 'Hematologic Neoplasms'}], 'ancestors': [{'id': 'D009371', 'term': 'Neoplasms by Site'}, {'id': 'D009369', 'term': 'Neoplasms'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1', 'PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SEQUENTIAL'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 36}}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2022-03-31', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2023-03', 'completionDateStruct': {'date': '2027-05', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2023-03-02', 'studyFirstSubmitDate': '2021-07-01', 'studyFirstSubmitQcDate': '2021-07-12', 'lastUpdatePostDateStruct': {'date': '2023-03-06', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2021-07-13', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2025-08', 'type': 'ESTIMATED'}}, 'outcomesModule': {'otherOutcomes': [{'measure': 'Overall Survival (OS)', 'timeFrame': 'Month 24 post-HSCT'}, {'measure': 'Disease-free Survival', 'timeFrame': 'Month 24 post-HSCT'}], 'primaryOutcomes': [{'measure': 'Cumulative incidence of grade III-IV GvHD', 'timeFrame': '100 days post-HSCT', 'description': 'to evaluate the safety profile of the study drug'}, {'measure': 'Occurrence of adverse events related to SMART101', 'timeFrame': '100 days post-HSCT', 'description': 'Number of adverse events and serious adverse events related to SMART101 tabulated for each dose and by age group to evaluate the safety profile of the study drug'}, {'measure': 'CD4+ T cell count', 'timeFrame': '100 days post-HSCT', 'description': 'to evaluate the efficacy of the study drug'}], 'secondaryOutcomes': [{'measure': 'T cell immune reconstitution', 'timeFrame': 'up to Month 12 post-HSCT', 'description': 'Time course of the T cell immune reconstitution, with a focus on naive CD4+ cells and total CD8+ cells'}, {'measure': 'Cumulative incidence of infections', 'timeFrame': 'Day 90, and Months 6, 12 and 24 post-HSCT'}, {'measure': 'Non-relapse mortality (NRM)', 'timeFrame': 'Day 90, and Months 6, 12 and 24 post-HSCT'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Hematological Malignancies']}, 'descriptionModule': {'briefSummary': 'The purpose of this study is to evaluate the safety and the efficacy of SMART101 (Human T Lymphoid Progenitor (HTLP)) injection to accelerate immune reconstitution after T cell depleted allogeneic hematopoietic stem cell transplantation (HSCT) in adult and pediatric patients with hematological malignancies.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\nGroup A (adults):\n\n1. Adult patients affected by:\n\n * Acute leukemia (AML, ALL) defined as:\n\n * Acute Myeloid Leukemia (AML):\n\n * High risk AML in CR1; any adverse genetic abnormality, secondary or therapy related AML excluding good risk genetic abnormalities\n * Chemo-refractory relapse (MRD+)\n * ≥ CR2\n * Acute Lymphoblastic Leukemia (ALL):\n\n * Chemo-refractory relapse (MRD+)\n * High risk ALL in CR1; Philadelphia (like) or any poor risk feature\n * ≥ CR2\n * Acute leukemia of ambiguous lineage:\n\n * ≥ CR1 with a minimal residual disease (MRD) \\<5% (flow cytometry, molecular and/or cytogenetics accepted)\n * Myelodysplastic Syndrome (MDS) with least one of the following:\n\n * Revised International Prognostic Scoring System risk score of intermediate or higher at the time of transplant evaluation.\n * Life-threatening cytopenia.\n * Karyotype or genomic changes that indicate high risk for progression to acute myelogenous leukemia, including abnormalities of chromosome 7 or 3, mutations of TP53, or complex or monosomal karyotype.\n * Therapy related disease or disease evolving from other malignant processes.\n2. Patient eligible for a T-depleted allogeneic HSCT\n3. Age ≥ 18y and clinical condition compatible with allogeneic stem cell transplantation\n4. Karnofsky index ≥ 70% prior to conditioning regimen\n5. Patients with normal organ function prior to conditioning regimen\n\nGroup B (pediatrics):\n\n1. Pediatric patients affected by acute leukemia defined as:\n\n * Acute Myeloid Leukemia (AML):\n\n * High risk AML in CR1; any adverse genetic abnormality, secondary or therapy related AML excluding good risk genetic abnormalities,\n * Chemo-refractory relapse (MRD+)\n * ≥ CR2\n * Acute Lymphoblastic Leukemia (ALL):\n\n * Chemo-refractory relapse (MRD+)\n * High risk ALL in CR1; Philadelphia (like) or any poor risk feature\n * ≥ CR2\n * Acute leukemia of ambiguous lineage:\n\n * ≥ CR1 with a minimal residual disease (MRD) \\<5% (flow cytometry, molecular and/or cytogenetics accepted)\n2. Patient eligible for a T-depleted allogeneic HSCT\n3. Age \\< 18y at the time of inclusion\n4. Absence of a matched sibling donor (MSD)\n5. Lansky ≥ 70% / Karnofsky performance status ≥ 70% prior to conditioning regimen\n6. Patients with normal organ function prior to conditioning regimen\n\nExclusion Criteria:\n\nGroups A and B:\n\n1. Use of an HLA matched Cord Blood (8/8 allele matched) or haploidentical donor\n2. Prior therapy with allogeneic stem cell transplantation\n3. Treatment with another cellular therapy within one month before inclusion'}, 'identificationModule': {'nctId': 'NCT04959903', 'briefTitle': 'Safety and Efficacy of SMART101 in Pediatric and Adult Patients With Hematological Malignancies After T Cell Depleted Allo-HSCT', 'organization': {'class': 'INDUSTRY', 'fullName': 'Smart Immune SAS'}, 'officialTitle': 'A Phase I/II Study Evaluating the Safety and the Efficacy of SMART101 Injection to Accelerate Immune Reconstitution After T Cell Depleted Allogeneic Hematopoietic Stem Cell Transplantation in Pediatric and Adult Patients With Hematological Malignancies', 'orgStudyIdInfo': {'id': 'SI101-01'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Adult patients affected by hematological malignancies', 'description': 'Adult patients affected by acute leukemia (AML, ALL or acute leukemia of ambiguous lineage) or myelodysplastic syndrome eligible for a T depleted allogeneic HSCT', 'interventionNames': ['Biological: Allogeneic T cell progenitors, cultured ex-vivo']}, {'type': 'EXPERIMENTAL', 'label': 'Pediatric patients affected by hematological malignancies', 'description': 'Pediatric patients affected by acute leukemia (AML, ALL or acute leukemia of ambiguous lineage) eligible for a T depleted allogeneic HSCT', 'interventionNames': ['Biological: Allogeneic T cell progenitors, cultured ex-vivo']}], 'interventions': [{'name': 'Allogeneic T cell progenitors, cultured ex-vivo', 'type': 'BIOLOGICAL', 'otherNames': ['SMART101'], 'description': 'Injection of T cell progenitors at \\[Day 4-Day 10\\] after T cell depleted allogeneic HSCT', 'armGroupLabels': ['Adult patients affected by hematological malignancies', 'Pediatric patients affected by hematological malignancies']}]}, 'contactsLocationsModule': {'locations': [{'zip': '10065', 'city': 'New York', 'state': 'New York', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Jaap-Jan BOELENS, MD, PhD', 'role': 'PRINCIPAL_INVESTIGATOR'}, {'name': 'Miguel-Angel PERALES, MD', 'role': 'SUB_INVESTIGATOR'}], 'facility': 'Memorial Sloan Kettering Cancer Center (MSKCC)', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}], 'centralContacts': [{'name': 'Frédéric LEHMANN, MD', 'role': 'CONTACT', 'email': 'frederic.lehmann@smart-immune.com', 'phone': '+32 (0) 492 46 23 55'}, {'name': 'Laura SIMONS, MD, PhD', 'role': 'CONTACT', 'email': 'laura.simons@smart-immune.com'}], 'overallOfficials': [{'name': 'Jaap-Jan BOELENS, MD, PhD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Memorial Sloan Kettering Cancer Center (MSKCC)'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Smart Immune SAS', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}