A Phase 1 Study of Orca-Q in Recipients Undergoing Allogeneic Transplantation for Hematologic Malignancies

Study Purpose

This study will evaluate the safety, tolerability, and efficacy of engineered donor grafts ("OrcaGraft"/"Orca-Q") in participants undergoing myeloablative allogeneic hematopoietic cell transplant transplantation for hematologic malignancies.

Recruitment Criteria

Accepts Healthy Volunteers Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms	No
Study Type An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes. An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes. Searching Both is inclusive of interventional and observational studies.	Interventional
Eligible Ages	18 Years - 65 Years
Gender	All

More Inclusion & Exclusion Criteria

Key

Inclusion Criteria:

1. Age ≥ 18 and ≤ 65 years at the time of enrollment. 2. Diagnosed acute myeloid, lymphoid or mixed phenotype leukemia, or high or very high risk myelodysplasic syndrome (MDS), myelofibrosis, or chronic myeloid leukemia. 3. Planned to undergo myeloablative allogeneic hematopoietic stem cell transplant (HCT) 4. Matched to a 8/8 or 7/8 related or unrelated donor, or to a related haploidentical donor. 5. Estimated glomerular filtration rate (eGFR) > 50 mL/minute. 6. Cardiac ejection fraction at rest ≥ 45% or shortening fraction of ≥ 27% by echocardiogram or radionuclide scan (MUGA) 7. Diffusing capacity of the lung for carbon monoxide (DLCO) (adjusted for hemoglobin) ≥ 50% 8. Total bilirubin < 1.5 times upper limit of normal (ULN) (< 3 times if attributed to Gilbert's syndrome) and ALT/AST < 3 times ULN. Key

Exclusion Criteria:

1. Prior allogeneic HCT. 2. Currently receiving corticosteroids or other immunosuppressive therapy. Topical corticosteroids or oral systemic corticosteroid doses less than or equal to 10 mg/day are allowed. 3. Planned donor lymphocyte infusion (DLI) 4. Positive anti-donor HLA antibodies against a mismatched allele in the selected donor. 5. Karnofsky performance score < 70% (Appendix 12.7) 6. Hematopoietic cell transplantation-specific Comorbidity Index (HCT-CI) > 4 (Appendix 12.8) 7. Uncontrolled bacterial, viral or fungal infections (currently taking antimicrobial therapy and with progression or no clinical improvement) at time of enrollment. 8. Seropositive for HIV-1 or -2, HTLV-1 or -2. 9. Any uncontrolled autoimmune disease requiring active immunosuppressive treatment. 10. Concurrent malignancies or active disease within 1 year, except non-melanoma skin cancers that have been curatively resected. 11. History of idiopathic or secondary myelofibrosis. 12. Women who are pregnant or breastfeeding

Trial Details

Trial ID: This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.	NCT03802695
Phase Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans. Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data. Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs. Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.	Phase 1
Lead Sponsor The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.	Orca Biosystems, Inc.
Principal Investigator The person who is responsible for the scientific and technical direction of the entire clinical study.	James S McClellan, MD, PhD
Principal Investigator Affiliation	Orca Biosystems, Inc.
Agency Class Category of organization(s) involved as sponsor (and collaborator) supporting the trial.	Industry
Overall Status	Recruiting
Countries	United States
Conditions The disease, disorder, syndrome, illness, or injury that is being studied.	Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, Myelodysplastic Syndromes, Mixed Phenotype Acute Leukemia, Myelofibrosis, Chronic Myeloid Leukemia

Arms & Interventions

Arms

Experimental: Arm A

Recipients with HLA-identical or 1-allele mismatched (7/8 alleles) related or unrelated donor; with single-agent GVHD prophylaxis given

Experimental: Arm B

Recipients with haploidentical-related donors; with single-agent GVHD prophylaxis given

Experimental: Arm C

Recipients with an HLA-identical related or unrelated donor; no GVHD prophylaxis given

Interventions

Biological: - OrcaGraft (Orca-Q)

engineered donor allograft

Contact a Trial Team

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.

City of Hope, Duarte, California

Status

Recruiting

Address

City of Hope

Duarte, California, 91010

Site Contact

Amandeep Salhotra, MD

info@orcabiosystems.com

1-530-414-9743

UC Davis, Sacramento, California

Status