What side effects are associated with this type of intervention?

The most common treatments for Acute Myeloid Leukemia (AML) involving allogeneic hematopoietic cell transplantation (HCT) can have several side effects. Short-term side effects include infections, bleeding, and organ toxicity due to the high-dose chemotherapy or radiation used in conditioning regimens. Long-term side effects may include graft-versus-host disease (GVHD), where the donor cells attack the recipient's tissues, chronic infections, and secondary cancers. Additionally, patients may experience fatigue, infertility, and other organ-specific complications depending on the intensity of the treatment and individual patient factors.

What prior FDA approvals exist?

The FDA has approved several treatments for Acute Myeloid Leukemia (AML), including hypomethylating agents like azacitidine and decitabine, which are used for newly diagnosed AML and are well-tolerated. While azacitidine is approved by the European Medicines Agency (EMA) for AML, it is not FDA-approved for this indication. Decitabine can be administered in both outpatient and inpatient settings. Additionally, the FDA has approved an oral preparation of azacitidine (CC-486) for post-remission maintenance therapy of AML. For treatments involving transplantation, allogeneic hematopoietic cell transplantation (HCT) is a common approach, where healthy donor cells replace diseased bone marrow. This method is similar to the engineered donor grafts being studied in the trial.

What other types of research exist?

Promising novel alternatives to engineered donor grafts for AML patients include: 1) Gene therapy approaches, such as CRISPR/Cas9-mediated gene editing, which aim to correct genetic mutations in hematopoietic stem cells. 2) Ex vivo expansion of cord blood-derived CD34+ cells to enhance engraftment potential and reduce delayed engraftment issues. 3) Targeted therapies using hypomethylating agents or other small molecules that specifically target AML cells' genetic and epigenetic abnormalities. 4) Immunotherapy options, including CAR-T cell therapy, which involves engineering a patient's own T cells to target and destroy AML cells.

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CAR T-cell Therapy

OrcaGraft for Blood Cancer

Phase 1

Recruiting

Research Sponsored by Orca Biosystems, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Matched to a 8/8 or 7/8 related or unrelated donor, or to a related haploidentical donor

Planned to undergo myeloablative allogeneic hematopoietic stem cell transplant (HCT)

Must not have

Karnofsky performance score < 70%

Women who are pregnant or breastfeeding

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 28 days after administration of orca-q/orcagraft

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing the safety and effectiveness of using specially prepared donor cells for patients with blood cancers. These patients are receiving a strong type of bone marrow transplant. The goal is to replace their damaged bone marrow with healthy cells from a donor to help them recover. Bone marrow transplantation (BMT) is a powerful strategy for the treatment of leukemia, aplastic anemia, congenital immunodeficiency, and autoimmune diseases.

Who is the study for?

This trial is for adults aged 18-65 with certain blood cancers like acute leukemia or high-risk myelodysplastic syndrome. Participants must match with a donor, have good kidney and heart function, and not be pregnant or on strong immunosuppressants. Those with uncontrolled infections, other active cancers, prior transplants, or severe comorbidities cannot join.

What is being tested?

The study tests 'OrcaGraft' (Orca-Q), an engineered donor graft in patients receiving bone marrow transplants for blood cancers. It aims to assess the safety and effectiveness of this new treatment approach during the transplant process.

What are the potential side effects?

While specific side effects are not listed here, similar procedures can include risks such as immune reactions against the graft (graft-versus-host disease), infections due to weakened immunity from transplantation, organ damage related to chemotherapy used before transplanting cells.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have a donor who is a close genetic match for my transplant.

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I am scheduled for a stem cell transplant from a donor.

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I have been diagnosed with a specific type of blood cancer or a high-risk bone marrow disorder.

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I am between 18 and 65 years old.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I need assistance with my daily activities.

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I am not pregnant or breastfeeding.

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I have antibodies against my donor's tissue type.

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I have an infection that isn't getting better despite treatment.

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I am scheduled for a donor lymphocyte infusion.

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I have an autoimmune disease that is not under control and requires treatment.

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I have been diagnosed with myelofibrosis.

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I have had a stem cell transplant from a donor.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 28 days after administration of orca-q/orcagraft

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~28 days after administration of orca-q/orcagraft

This trial's timeline: 3 weeks for screening, Varies for treatment, and 28 days after administration of orca-q/orcagraft for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Number of Dose Limiting Toxicities

Primary Graft failure through Day +28 (dose expansion)

Secondary study objectives

Acute GVHD through Day +100

Chronic GVHD through Day +365

Disease-free survival (DFS) through Day +365

+10 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

3Treatment groups

Experimental Treatment

Group I: Arm CExperimental Treatment1 Intervention

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

Group II: Arm BExperimental Treatment1 Intervention

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

Group III: Arm AExperimental Treatment1 Intervention

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

0 / 12Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Acute Myeloid Leukemia (AML) include chemotherapy, targeted therapies, and allogeneic hematopoietic cell transplantation (HCT). Chemotherapy agents like clofarabine work by killing rapidly dividing cancer cells but can cause significant side effects. Targeted therapies such as gilteritinib inhibit specific mutations in cancer cells, offering a more precise approach with potentially fewer side effects. Allogeneic HCT, including the use of engineered donor grafts like Orca-Q, involves transplanting healthy donor cells to replace diseased bone marrow, which can potentially cure AML by re-establishing normal blood cell production. This is particularly important for AML patients as it offers a chance for long-term remission and survival, especially when other treatments have failed.

Immunotherapy in AML: a brief review on emerging strategies.Emerging therapies for acute myeloid leukemia: translating biology into the clinic.Development of Personalized Molecular Therapy for Acute Myeloid Leukemia.