CK0801 for Bone Marrow Disease

Recruiting at 1 trial location

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Cellenkos, Inc.

Disqualifiers: HIV, Uncontrolled infection, Pregnancy, others

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

Compensation: Varies

Number of Visits: 1

Duration: 1 day

18 Participants Needed

Trial Summary

What is the purpose of this trial?

The goal of this clinical research study is to determine whether it is safe and practical to give CK0801 (a Cord blood-derived T-regulatory cell product) to patients with bone marrow failure syndrome. Researchers want to determine the highest possible dose that is safe to be given. Researchers also want to learn if CK0801 may improve the symptoms of bone marrow failure syndrome. Patients enrolled in this study will all have been diagnosed with treatment refractory bone marrow failure syndrome (which includes aplastic anemia, myelodysplastic syndrome, or myelofibrosis). Participants eligible to participate in this study are unable or unwilling to be treated with standard therapy or have failed standard therapy.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. However, you cannot have received an investigational agent within 4 weeks or radiation/chemotherapy within 21 days before the CK0801 infusion.

What safety data exists for CK0801 or similar treatments for bone marrow disease?

The research does not provide specific safety data for CK0801 or similar treatments for bone marrow disease. ^{1 2 3 4 5}

How does the drug CK0801 differ from other treatments for bone marrow disease?

CK0801 is unique because it targets the FGFR1 gene, which is involved in aggressive bone marrow diseases like the 8p11 myeloproliferative syndrome. This drug may offer a more specific approach compared to the current standard, which is bone marrow transplantation. ^{6 7 8 9 10}

Research Team

Tapan M Kadia, MD

Principal Investigator

The University of Texas MD Anderson Cancer Center

Eligibility Criteria

Adults diagnosed with bone marrow failure syndrome, including aplastic anemia, myelodysplastic syndrome, or myelofibrosis, who can't be treated with standard therapy or haven't responded to it. They must have a matched cord blood unit for the treatment and their liver and kidney functions should meet specific criteria. Women of childbearing potential must use effective birth control.

Inclusion Criteria

Subject is willing and able to provide written informed consent

My kidneys are functioning well, with a creatinine clearance rate over 50 mL/min.

I can take care of myself but might not be able to do heavy physical work.

Exclusion Criteria

Subjects who are unable to provide consent or who, in the opinion of the Investigator will be unlikely to fully comply with protocol requirements

My bone marrow failure is due to a stem cell transplant.

I do not have any severe illnesses that could worsen with treatment.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive an infusion of CK0801 (Treg cells) on study Day 0

1 day

1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment, including assessment of severe cytokine release syndrome and infusion toxicity

30 days

Treatment Details

Interventions

• CK0801

Trial Overview The trial is testing CK0801, a new cell-based therapy derived from cord blood to see if it's safe at high doses and if it helps improve symptoms of bone marrow failure. All participants will receive this experimental treatment after failing or being unable to undergo standard therapies.

Participant Groups

Group I: CK0801 Experimental Treatment 1 Intervention

All subjects will receive adoptive therapy with an infusion of unrelated cord blood-derived regulatory T cells: CK0801. Subjects will receive one intravenous dose of CK0801 (Treg cells) on study Day 0.

Who Is Running the Clinical Trial?

Cellenkos, Inc.

Lead Sponsor

Trials

5

Recruited

150+

Findings from Research

Systemic Mastocytosis (SM) is characterized by abnormal mast cell proliferation in various organs, often linked to the D816V (KIT) mutation and elevated serum tryptase, which are important for diagnosis.

Treatment for SM primarily focuses on managing symptoms, with bisphosphonates recommended as a first-line therapy for bone-related issues, and denosumab showing potential effectiveness due to its action on bone metabolism.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Endocrine manifestations of systemic mastocytosis in bone. Greene, LW., Asadipooya, K., Corradi, PF., et al.[2018]

References

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

Experience with everolimus (RAD001), an oral mammalian target of rapamycin inhibitor, in patients with systemic mastocytosis. [2021]

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

Mayo alliance prognostic system for mastocytosis: clinical and hybrid clinical-molecular models. [2021]

3. England pubmed.ncbi.nlm.nih.gov

Phase II study of imatinib mesylate as therapy for patients with systemic mastocytosis. [2021]

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

Nintedanib targets KIT D816V neoplastic cells derived from induced pluripotent stem cells of systemic mastocytosis. [2021]

5. Germany pubmed.ncbi.nlm.nih.gov

Endocrine manifestations of systemic mastocytosis in bone. [2018]

6. Switzerland pubmed.ncbi.nlm.nih.gov

The 8p11 myeloproliferative syndrome: a distinct clinical entity caused by constitutive activation of FGFR1. [2017]

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

Abnormalities of chromosome band 8p11 in leukemia: two clinical syndromes can be distinguished on the basis of MOZ involvement. [2021]

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

Consistent fusion of ZNF198 to the fibroblast growth factor receptor-1 in the t(8;13)(p11;q12) myeloproliferative syndrome. [2021]

9. England pubmed.ncbi.nlm.nih.gov

A biphenotypic transformation of 8p11 myeloproliferative syndrome with CEP1/FGFR1 fusion gene. [2011]

10. England pubmed.ncbi.nlm.nih.gov

Myeloproliferative disorders with t(8;9)(p12;q33): a case report and review of the literature. [2011]