Orca-Q for Pediatric Leukemia

Recruiting at4 trial locations

Overseen by Jordan Milner, MD

Age: < 65

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1 & 2

Recruiting

Sponsor: University of Florida

Must not be taking: Corticosteroids, Immunosuppressants

Disqualifiers: Prior HCT, Active infections, HIV, others

No Placebo Group

Trial Summary

What is the purpose of this trial?

This is a first in children prospective study of allogeneic hematopoietic cell transplant using a centrally manufactured engineered donor graft (Orca-Q). The study will assess safety and efficacy of Orca-Q in pediatric patients with hematologic malignancies.

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.

What data supports the effectiveness of the treatment Orca-Q for pediatric leukemia?

The effectiveness of treatments for childhood leukemia has improved significantly over the years, with cure rates now ranging from 65% to 90% for certain types of leukemia. This progress is due to advancements in therapy and the development of new agents that have shown high survival rates and reduced long-term health complications. ^{1 2 3 4 5}

Research Team

Jordan Milner, MD

Principal Investigator

University of Florida

Eligibility Criteria

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.

I am between 18 and 50 years old.

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.

Trial Timeline

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

Treatment Details

Interventions

• Orca-Q (CAR T-cell Therapy)

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.

Participant Groups

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+

Dr. Stephen J. Motew

University of Florida

Chief Executive Officer since 2024

MD cum laude from the University of Illinois at Chicago School of Medicine, Master's in Healthcare Administration from the University of North Carolina at Chapel Hill

Dr. Timothy E. Morey

University of Florida

Chief Medical Officer since 2023

MD and Bachelor's from the University of Florida

Orca Biosystems, Inc.

Industry Sponsor

Trials

8

Recruited

750+

Florida Department of Health

Collaborator

Trials

30

Recruited

13,000+

Findings from Research

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]

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]

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]

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]

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]

References

1. United States pubmed.ncbi.nlm.nih.gov

Long-term survival of children with leukemia achieved by the end of the second millennium. [2019]

2. Germany pubmed.ncbi.nlm.nih.gov

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

3. United States pubmed.ncbi.nlm.nih.gov

Increased Survival for Children With Acute Myeloid Leukemia Results From Improved Postrelapse Treatment. [2019]

4. New Zealand pubmed.ncbi.nlm.nih.gov

Novel agents for the treatment of childhood leukemia: an update. [2020]

5. United States pubmed.ncbi.nlm.nih.gov

Therapies on the horizon for childhood acute lymphoblastic leukemia. [2018]