A Phase 1 Study of Engineered Donor Grafts (Orca-Q) in Recipients Undergoing Allogeneic Transplantation for Hematologic Malignancies
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.
Conditions
- Acute Myeloid Leukemia
- Acute Lymphoblastic Leukemia
- Myelodysplastic Syndromes
- Acute Leukemia
Eligibility
- Eligible Ages
- Between 18 Years and 65 Years
- Eligible Genders
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- 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) or Myelofibrosis 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
Exclusion Criteria
- 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
Study Design
- Phase
- Phase 1
- Study Type
- Interventional
- Allocation
- Non-Randomized
- Intervention Model
- Parallel Assignment
- Primary Purpose
- Treatment
- Masking
- None (Open Label)
Arm Groups
Arm | Description | Assigned Intervention |
---|---|---|
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 |
|
Recruiting Locations
The University of Kansas Hospital
Kansas City, Kansas 66160
Kansas City, Kansas 66160
Contact:
Joseph McGuirk, MD
Joseph McGuirk, MD
More Details
- Status
- Recruiting
- Sponsor
- Orca Biosystems, Inc.