Orca-Q for Pediatric Leukemia

Recruiting at 5 trial locations

Overseen By Alex Leggett, MS

Age: < 65

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: University of Florida

Must not be taking: Corticosteroids, Immunosuppressants

Disqualifiers: Prior HCT, Active infections, HIV, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

40 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called Orca-Q to determine its safety and effectiveness for children with certain blood cancers, such as leukemia. The focus is on children in remission who need a bone marrow transplant to maintain their health. Eligible participants must have a controlled type of leukemia and plan to undergo a bone marrow transplant with a matched donor. As a Phase 1, Phase 2 trial, this research aims to understand how Orca-Q works in people and measure its effectiveness in an initial group, offering participants a chance to contribute to groundbreaking advancements in cancer treatment.

Do I need to stop my current medications for the Orca-Q trial?

The trial requires that participants not be on corticosteroids or other immunosuppressive therapy, except for topical corticosteroids or physiologic replacement hydrocortisone. If you are currently taking these medications, you may need to stop them to participate.

Is there any evidence suggesting that Orca-Q is likely to be safe for children?

Research has shown that Orca-Q is usually well-tolerated by patients with blood cancers. In earlier studies, the treatment appeared safe. For instance, one study found that 85% of patients survived for a year without needing extra medication to prevent GvHD, a type of immune reaction. Another study found that Orca-Q was safe and effective when used with certain donor transplants. These results suggest that Orca-Q could be a safe option, but more research is needed to confirm its safety in children. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Orca-Q is unique because it introduces a novel approach to treating pediatric leukemia by targeting specific pathways involved in the progression of the disease. Unlike standard treatments like chemotherapy, which attack both cancerous and healthy cells indiscriminately, Orca-Q aims to selectively target leukemia cells, potentially reducing side effects and improving patient outcomes. Researchers are excited about Orca-Q because it could offer a more precise and less toxic treatment alternative, enhancing the quality of life for young patients battling leukemia.

What evidence suggests that Orca-Q might be an effective treatment for pediatric leukemia?

Research has shown that Orca-Q, the treatment being studied in this trial, may help treat blood cancers. In previous studies, 85% of patients who received Orca-Q were still alive one year after treatment, indicating that the treatment helped many patients live longer. The studies also showed that many patients did not experience a recurrence of their cancer. Additionally, Orca-Q worked well without causing serious side effects like GvHD, a common problem in transplants. These findings suggest Orca-Q could be effective and safe for children with leukemia. ^{1 2 4 6 7}

Who Is on the Research Team?

Jordan Milner, MD

Principal Investigator

University of Florida

Are You a Good Fit for This Trial?

This trial is for children and young adults under 22 years old diagnosed with various types of leukemia, who are in remission but need a stem cell transplant. They must have good organ function, no active infections like COVID-19 or hepatitis, and not be on immunosuppressive drugs. Donors between 18-50 years old must match the patient's criteria and agree to donate stem cells.

Inclusion Criteria

You have a donor who is willing to donate their peripheral blood stem cells.

My cancer has not spread outside the bone marrow.

Meets federal eligibility criteria for donors of viable, leukocyte-rich cells or tissues as defined by 21 CFR § 1271 2018 and all relevant FDA Guidance for Industry

Exclusion Criteria

Hematopoietic cell transplantation-specific Comorbidity Index (HCT-CI) > 4

I am pregnant or breastfeeding and not willing to stop breastfeeding.

If you were previously found ineligible due to a positive Zika virus screening result, you may still be eligible if certain conditions are met.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Transplantation

Participants undergo allogeneic hematopoietic cell transplant using the engineered donor graft (Orca-Q)

4 weeks

Post-Transplant Monitoring

Participants are monitored for primary and secondary graft failure, acute GVHD, and other treatment-emergent adverse events

6 months

Follow-up

Participants are monitored for overall survival, relapse rate, chronic GVHD, and serious infections

24 months

What Are the Treatments Tested in This Trial?

Interventions

• Orca-Q

Trial Overview The trial tests Orca-Q, an engineered donor graft for pediatric patients needing hematopoietic cell transplants. It aims to determine how safe and effective this treatment is when used in children with blood cancers that require a bone marrow transplant.

How Is the Trial Designed?

Group I: Orca-Q Experimental Treatment 1 Intervention

Who Is Running the Clinical Trial?

University of Florida

Lead Sponsor

Trials

1,428

Recruited

987,000+

Orca Biosystems, Inc.

Industry Sponsor

Trials

8

Recruited

750+

Florida Department of Health

Collaborator

Trials

30

Recruited

13,000+

Published Research Related to This Trial

Cure rates for childhood hematologic malignancies have dramatically improved from nearly zero in the 1950s to between 65% and 90% today, particularly for Acute Lymphoblastic Leukemia (ALL) and its higher-risk forms.

Advancements in treatment have been driven by the identification of specific prognostic criteria, allowing for more tailored therapies that match the needs of individual patient subgroups, highlighting the importance of pediatricians staying updated on these changes for better patient care.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Molecular diagnosis and risk-adjusted therapy in pediatric hematologic malignancies: a primer for pediatricians. Margolin, JF.[2021]

Recent advancements in understanding genetic alterations in high-risk acute lymphoblastic leukemia (ALL) have opened up new avenues for precision medicine, allowing for targeted therapies using existing FDA-approved drugs.

Innovative immunotherapeutic strategies are being utilized for relapsed ALL, and efforts to reduce the use of prophylactic central nervous system irradiation are helping to minimize long-term neurocognitive side effects in children.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Therapies on the horizon for childhood acute lymphoblastic leukemia. Carroll, WL., Hunger, SP.[2018]

A new survival analysis method called period analysis shows that the 5-year survival rate for childhood leukemia patients diagnosed by 1998 is 81%, which is significantly higher than traditional estimates suggest.

The study, which analyzed data from 8059 children diagnosed between 1981 and 1998, indicates that patients with acute lymphocytic leukemia have a 10-year survival rate of 81%, highlighting improved treatment outcomes over the years.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Long-term survival of children with leukemia achieved by the end of the second millennium. Brenner, H., Kaatsch, P., Burkhardt-Hammer, T., et al.[2019]

Childhood leukemia treatment has significantly improved, leading to lower morbidity and higher survival rates, but many survivors still face serious long-term health complications.

New treatment agents have been developed that show promise as alternatives or supplements to traditional therapies, potentially improving outcomes for patients with acute lymphoid leukemia and acute myeloid leukemia, where treatment failures remain a concern.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Novel agents for the treatment of childhood leukemia: an update. Eryılmaz, E., Canpolat, C.[2020]

A study of 276 children with acute myeloid leukemia (AML) showed that overall survival rates improved significantly from 50% in the historical cohort (1986-1999) to 75% in the contemporary cohort (2000-2012), largely due to better post-relapse treatment strategies.

The increased use of allogeneic stem cell transplantation (SCT) after relapse was a key factor in improving survival, with SCT associated with a 50% survival rate compared to only 12% for those who did not receive it.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Increased Survival for Children With Acute Myeloid Leukemia Results From Improved Postrelapse Treatment. Vedi, A., Mitchell, R., Shanmuganathan, S., et al.[2019]

Citations

1. orcabio.com orcabio.com/orca-bio-presents-clinical-results-on-use-of-orca-q-without-gvhd-prophylaxis-in-patients-with-hematological-malignancies-at-the-66th-ash-annual-meeting/

Orca Bio Presents Clinical Results on Use of Orca-Q ... Relapse-free survival (RFS) and overall survival (OS) were both 85% among all patients, and both 90% in the BFT population. GvHD-free, relapse- ...

2. onclive.com onclive.com/view/orca-q-shows-promise-in-high-risk-hematologic-cancers-and-bft-conditioning-may-synergize-with-the-agent

Orca-Q Shows Promise in High-Risk Hematologic Cancers ... The 1-year overall survival (OS) rate was 85% (95% CI, 52%-98%) among patients who received Orca-Q (n = 14) in the phase 1 trial.

3. clinicaltrials.gov clinicaltrials.gov/study/NCT05322850

Phase I/II Trial: Engineered Donor Graft (Orca Q) for ... The study will assess safety and efficacy of Orca-Q in pediatric patients with hematologic malignancies. Official Title. Phase I/II Trial: Engineered Donor ...

4. cancernetwork.com cancernetwork.com/view/orca-q-has-early-activity-tolerability-in-haplo-sct-population

Orca-Q Has Early Activity, Tolerability in Haplo-SCT ... “These findings show promising safety and efficacy outcomes using Orca-Q cell therapy for haploidentical transplant,” according to Samer A.

5. cancer.gov cancer.gov/clinicaltrials/NCI-2023-09341

Orca Q Stem Cells for the Treatment of Pediatric Patients ... This phase I/II trial tests the safety and effectiveness of Orca Q stem cells for the treatment of pediatric patients with leukemia. Orca Q are engineered ...

6. astctjournal.org astctjournal.org/article/S2666-6367(25)00197-6/fulltext

Preliminary Safety and Efficacy of Myeloablative Orca-Q ... Administration of Orca-Q without post-transplant pharmacological GvHD prophylaxis showed promising clinical outcomes at 1-year follow-up. Orca-Q offers similar ...

7. sciencedirect.com sciencedirect.com/science/article/abs/pii/S0006497123053776

Safety and Efficacy of Orca-Q with Haploidentical Donors ... Our findings reveal promising safety and efficacy outcomes using Orca-Q cell therapy for haplo-SCT despite the use of MAC with only single-agent tacrolimus, ...