Ignite Creation Date:
2025-12-24 @ 1:29 PM
Ignite Modification Date:
2026-01-05 @ 6:41 PM
Study NCT ID:
NCT03802695
Status:
RECRUITING
Last Update Posted:
2025-12-04
First Post:
2019-01-10
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A Phase 1 Study of Orca-Q in Recipients Undergoing Allogeneic Transplantation for Hematologic Malignancies
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D015470', 'term': 'Leukemia, Myeloid, Acute'}, {'id': 'D009190', 'term': 'Myelodysplastic Syndromes'}, {'id': 'D015456', 'term': 'Leukemia, Biphenotypic, Acute'}, {'id': 'D054198', 'term': 'Precursor Cell Lymphoblastic Leukemia-Lymphoma'}], 'ancestors': [{'id': 'D007951', 'term': 'Leukemia, Myeloid'}, {'id': 'D007938', 'term': 'Leukemia'}, {'id': 'D009370', 'term': 'Neoplasms by Histologic Type'}, {'id': 'D009369', 'term': 'Neoplasms'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D001855', 'term': 'Bone Marrow Diseases'}, {'id': 'D007945', 'term': 'Leukemia, Lymphoid'}, {'id': 'D008232', 'term': 'Lymphoproliferative Disorders'}, {'id': 'D008206', 'term': 'Lymphatic Diseases'}, {'id': 'D007160', 'term': 'Immunoproliferative Disorders'}, {'id': 'D007154', 'term': 'Immune System Diseases'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 300}}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2019-04-08', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-11', 'completionDateStruct': {'date': '2027-12', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-11-26', 'studyFirstSubmitDate': '2019-01-10', 'studyFirstSubmitQcDate': '2019-01-10', 'lastUpdatePostDateStruct': {'date': '2025-12-04', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2019-01-14', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2027-12', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Dose Limiting Toxicities through Day +28 (dose escalation)', 'timeFrame': '28 Days after administration of Orca-Q/OrcaGraft', 'description': 'Safety and tolerability of Orca-Q (formerly OrcaGraft) in adults undergoing myeloablative allogeneic hematopoietic cell transplantation (MA-alloHCT) will be evaluated by identification of the following dose limiting toxicities: Grade ≥ 3 infusion-related reaction or cytokine release syndrome, Grade ≥ 3 acute GVHD, Any Grade ≥ 3 treatment-related non-hematologic event not clearly related to the underlying malignancy, intercurrent infection, the HCT conditioning regimen, or other pre-existing medical condition'}, {'measure': 'Primary Graft failure through Day +28 (dose expansion)', 'timeFrame': '28 Days after administration of Orca-Q/OrcaGraft', 'description': 'Primary graft failure in the dose expansion phase, defined as being alive without recovery of neutrophils during the evaluation period'}], 'secondaryOutcomes': [{'measure': 'Neutrophil Engraftment through Day +28', 'timeFrame': '28 days after administration of Orca-Q/OrcaGraft', 'description': 'Neutrophil engraftment defined as an absolute neutrophil count of \\>/=500/mm3 for 3 consecutive days'}, {'measure': 'Platelet Engraftment through Day +50', 'timeFrame': '50 days after administration of Orca-Q/OrcaGraft', 'description': 'Platelet engraftment is defined as achieving a platelet count \\> 20,000/mm3 for 3 consecutive days without platelet transfusion in the preceding 7 days, by Day +50'}, {'measure': 'Secondary Graft Failure through Day +100', 'timeFrame': '100 days after administration of Orca-Q/OrcaGraft', 'description': 'Secondary graft failure is defined as neutrophil engraftment followed by subsequent decline in absolute neutrophil counts \\< 500 cells/μL, unresponsive to growth factor therapy, by Day +100'}, {'measure': 'Acute GVHD through Day +100', 'timeFrame': '100 days after administration of Orca-Q/OrcaGraft', 'description': 'Acute GVHD will be staged and graded per Mount Sinai Acute GvHD International Consortium (MAGIC) Standardization criteria'}, {'measure': 'Chronic GVHD through Day +365', 'timeFrame': '365 days after administration of Orca-Q/OrcaGraft', 'description': 'Chronic GVHD will be diagnosed per 2014 International NIH Chronic GVHD Diagnosis and Staging Consensus Working Group criteria'}, {'measure': 'Incidence of Non-relapse Mortality (NRM) through Day +365', 'timeFrame': '365 days after administration of Orca-Q/OrcaGraft', 'description': 'NRM is defined as death without evidence of disease recurrence'}, {'measure': 'Incidence of Disease Relapse through Day +365', 'timeFrame': '365 days after administration of Orca-Q/OrcaGraft', 'description': 'Recurrence of primary disease for transplant'}, {'measure': 'GVHD-free and Relapse-free Survival (GRFS) through Day +365', 'timeFrame': '365 days after administration of Orca-Q/OrcaGraft', 'description': 'Survival free from GVHD and relapse'}, {'measure': 'Disease-free Survival (DFS) through Day +365', 'timeFrame': '365 days after administration of Orca-Q/OrcaGraft', 'description': 'DFS is the time from date of transplant to death or relapse, whichever comes first.'}, {'measure': 'Overall Survival through Day +365', 'timeFrame': '365 days after administration of Orca-Q/OrcaGraft', 'description': 'OS is defined as the time from the date of transplant to the date of death from any cause or, for surviving patients, to the date of last follow-up.'}]}, 'oversightModule': {'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Acute Myeloid Leukemia', 'Myelodysplastic Syndromes', 'Mixed Phenotype Acute Leukemia', 'Acute Lymphoblastic Leukemia']}, 'descriptionModule': {'briefSummary': 'This study will evaluate the safety, tolerability, and efficacy of engineered donor grafts ("OrcaGraft"/"Orca-Q") in participants undergoing allogeneic hematopoietic cell transplant (alloHCT) transplantation for hematologic malignancies.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'maximumAge': '78 Years', 'minimumAge': '12 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Key Inclusion Criteria:\n\n1. Age at the time of enrollment:\n\n 1. For MAC with fully matched donor (Arm A with 8/8 donor and Arm C) and NMA/RIC: Age ≥ 12 and ≤ 78 years\n 2. For MAC with mismatched donors (Arm A with 7/8 donor and Arm B): Age ≥ 12 and ≤ 65 years\n2. Diagnosed acute myeloid, lymphoblastic or mixed phenotype leukemia, or high or very high risk myelodysplastic syndrome (MDS) either in complete remission (CR) or with ≤ 10 percent of blast cells in bone marrow (BM)\n3. Indicated for allogeneic hematopoietic stem cell transplant (alloHCT)\n4. Matched to a 8/8 or 7/8 related or unrelated donor, or to a related haploidentical donor\n5. Estimated glomerular filtration rate (eGFR) \\> 50 mL/minute (MAC with tacrolimus) or \\> 30 mL/minute (NMA/RIC or MAC without tacrolimus)\n6. Cardiac parameters: Cardiac ejection fraction ≥ 45 percent (MAC) or ≥ 40 percent (NMA/RIC)\n7. Diffusing capacity of the lung for carbon monoxide (DLCO) (adjusted for hemoglobin) ≥ 50 percent for MAC or ≥ 40 percent for NMA/RIC\n8. Liver function: Total bilirubin \\< 1.5 times upper limit of normal (ULN) (MAC) or \\< 3 times ULN (NMA/RIC); alanine transaminase (ALT)/aspartate transaminase (AST) \\< 3 times ULN (MAC) or \\< 5 times ULN (NMA/RIC)\n9. Participants enrolling on NMA/RIC-alloHCT arms must be deemed unfit for a myeloablative alloHCT per assessment of the principal investigator (PI)\n\nKey Exclusion Criteria:\n\n1. Prior alloHCT\n2. Currently receiving corticosteroids or other immunosuppressive therapy except for approved disease-specific therapy for the patient's underlying hematologic malignancy. Topical corticosteroids or oral systemic corticosteroid doses less than or equal to 10 mg/day are allowed\n3. Planned donor lymphocyte infusion (DLI)\n4. Planned pharmaceutical in vivo or ex vivo T cell depletion, e.g., post-transplant cyclophosphamide (Cy) or alemtuzumab\n5. Positive anti-donor HLA antibodies against a mismatched allele in the selected donor\n6. Low performance score: For MAC: Karnofsky Performance Score (KPS) \\< 70 percent, For NMA/RIC: \\<60 percent\n7. High HCT-specific Comorbidity Index (HCT-CI): For MAC \\> 4, For NMA/RIC \\>6\n8. Uncontrolled bacterial, viral or fungal infections (currently taking antimicrobial therapy and with progression or no clinical improvement) at time of enrollment\n9. Seropositive for human immunodeficiency virus (HIV)-1 or -2, human T-lymphotropic virus (HTLV)-1 or -2 or Hepatitis B surface antigen (HbsAg) or anti-Hepatitis C virus (HCV) antibody (Ab)\n10. Any uncontrolled autoimmune disease requiring active immunosuppressive treatment\n11. Concurrent malignancies or active disease within 1 year, except non-melanoma skin cancers that have been curatively resected. Patients with concurrent indolent hematologic malignancies that do not require active treatment and are under active surveillance only (such as CLL, low-grade lymphomas, smoldering MM, MZL) may be included with the approval of Medical Monitor\n12. History of idiopathic or secondary myelofibrosis\n13. Women who are pregnant or breastfeeding"}, 'identificationModule': {'nctId': 'NCT03802695', 'briefTitle': 'A Phase 1 Study of Orca-Q in Recipients Undergoing Allogeneic Transplantation for Hematologic Malignancies', 'organization': {'class': 'INDUSTRY', 'fullName': 'Orca Biosystems, Inc.'}, 'officialTitle': 'A Phase 1 Dose Escalation and Expansion Study of Orca-Q, an Engineered Donor Graft Derived From Mobilized Peripheral Blood, in Recipients Undergoing Allogeneic Hematopoietic Cell Transplantation for Hematologic Malignancies', 'orgStudyIdInfo': {'id': 'OGFT001-001'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Arm A', 'description': 'Recipients with human leukocyte antigen (HLA)-identical related or unrelated or 1-allele mismatched (7/8 alleles) unrelated donor undergoing myeloablative conditioning (MAC); with single- or dual-agent graft-versus-host disease (GVHD) prophylaxis given', 'interventionNames': ['Biological: OrcaGraft (Orca-Q)']}, {'type': 'EXPERIMENTAL', 'label': 'Arm B', 'description': 'Recipients with haploidentical-related donors undergoing MAC; with single- or dual-agent GVHD prophylaxis given', 'interventionNames': ['Biological: OrcaGraft (Orca-Q)']}, {'type': 'EXPERIMENTAL', 'label': 'Arm C', 'description': 'Recipients with an HLA-identical related or unrelated donor undergoing MAC; no GVHD prophylaxis given', 'interventionNames': ['Biological: OrcaGraft (Orca-Q)']}, {'type': 'EXPERIMENTAL', 'label': 'Arm D', 'description': 'Recipients with an HLA-identical related or unrelated donor undergoing non-myeloablative (NMA)/reduced intensity conditioning (RIC); with dual agent GVHD prophylaxis given', 'interventionNames': ['Biological: OrcaGraft (Orca-Q)']}, {'type': 'EXPERIMENTAL', 'label': 'Arm E', 'description': 'Recipients with 1-allele mismatched (7/8 alleles) unrelated donor undergoing NMA/RIC; with dual-agent GVHD prophylaxis given', 'interventionNames': ['Biological: OrcaGraft (Orca-Q)']}, {'type': 'EXPERIMENTAL', 'label': 'Arm F', 'description': 'Recipients with haploidentical-related donors undergoing NMA/RIC; with dual-agent GVHD prophylaxis given', 'interventionNames': ['Biological: OrcaGraft (Orca-Q)']}], 'interventions': [{'name': 'OrcaGraft (Orca-Q)', 'type': 'BIOLOGICAL', 'description': 'engineered donor allograft', 'armGroupLabels': ['Arm A', 'Arm B', 'Arm C', 'Arm D', 'Arm E', 'Arm F']}]}, 'contactsLocationsModule': {'locations': [{'zip': '91010', 'city': 'Duarte', 'state': 'California', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Amandeep Salhotra, MD', 'role': 'CONTACT'}], 'facility': 'City of Hope', 'geoPoint': {'lat': 34.13945, 'lon': -117.97729}}, {'zip': '95817', 'city': 'Sacramento', 'state': 'California', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Mehrdad Abedi, MD', 'role': 'CONTACT'}], 'facility': 'UC Davis', 'geoPoint': {'lat': 38.58157, 'lon': -121.4944}}, {'zip': '94305', 'city': 'Stanford', 'state': 'California', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Robert Lowsky, MD', 'role': 'CONTACT'}], 'facility': 'Stanford Health Care', 'geoPoint': {'lat': 37.42411, 'lon': -122.16608}}, {'zip': '30322', 'city': 'Atlanta', 'state': 'Georgia', 'status': 'ACTIVE_NOT_RECRUITING', 'country': 'United States', 'facility': 'Emory University', 'geoPoint': {'lat': 33.749, 'lon': -84.38798}}, {'zip': '66160', 'city': 'Kansas City', 'state': 'Kansas', 'status': 'WITHDRAWN', 'country': 'United States', 'facility': 'The University of Kansas Hospital', 'geoPoint': {'lat': 39.11417, 'lon': -94.62746}}, {'zip': '43210', 'city': 'Columbus', 'state': 'Ohio', 'status': 'ACTIVE_NOT_RECRUITING', 'country': 'United States', 'facility': 'Ohio State University', 'geoPoint': {'lat': 39.96118, 'lon': -82.99879}}, {'zip': '77054', 'city': 'Houston', 'state': 'Texas', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Samer Srour, MD', 'role': 'CONTACT'}], 'facility': 'University of Texas MD Anderson Cancer Center', 'geoPoint': {'lat': 29.76328, 'lon': -95.36327}}, {'zip': '53226', 'city': 'Milwaukee', 'state': 'Wisconsin', 'status': 'WITHDRAWN', 'country': 'United States', 'facility': 'Froedtert Memorial Lutheran Hospital', 'geoPoint': {'lat': 43.0389, 'lon': -87.90647}}], 'centralContacts': [{'name': 'Tamara Zharkevich, MD, PhD', 'role': 'CONTACT', 'email': 'info@orcabiosystems.com', 'phone': '650-246-9601'}, {'name': 'James S McClellan, MD PhD', 'role': 'CONTACT', 'email': 'info@orcabiosystems.com', 'phone': '650-246-9601'}], 'overallOfficials': [{'name': 'James S McClellan, MD, PhD', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Orca Biosystems, Inc.'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Orca Biosystems, Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}