CK0801 for Bone Marrow Disease

Not currently 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to test the safety and practicality of a treatment called CK0801, derived from special cells in umbilical cord blood, for individuals with bone marrow failure syndrome. The study seeks to determine the highest safe dose and assess whether CK0801 can improve symptoms. Individuals diagnosed with bone marrow failure conditions such as aplastic anemia, myelodysplastic syndrome, or myelofibrosis, who have not responded to standard treatments, might be suitable candidates for this trial. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this new treatment.

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.

Is there any evidence suggesting that CK0801 is likely to be safe for humans?

Research has shown that CK0801, a treatment derived from cord blood, has been tested in patients before. In these studies, CK0801 did not cause any serious side effects or problems at the doses used, indicating that patients tolerated the treatment well. Additionally, CK0801 has demonstrated positive results in treating conditions like aplastic anemia, a type of bone marrow failure. Specifically, the treatment helped some patients avoid needing blood transfusions for up to 3.5 years. This evidence suggests that CK0801 could be a safe and promising option for patients with bone marrow diseases considering participation in a clinical trial. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for bone marrow diseases, which often involve chemotherapy or bone marrow transplants, CK0801 introduces a new approach by using cord blood-derived regulatory T cells. This treatment harnesses the body's immune system by infusing these specific T cells to potentially regulate immune responses more effectively. Researchers are excited about CK0801 because it targets the condition in a way that could offer more precise and possibly safer outcomes, reducing the risks associated with traditional therapies.

What evidence suggests that CK0801 might be an effective treatment for bone marrow failure syndrome?

Research has shown that CK0801, a treatment using special cells from umbilical cord blood, holds potential for treating bone marrow failure conditions. Previous studies found no serious side effects, indicating the treatment is generally safe. Early evidence suggests CK0801 may help reduce inflammation, a key factor in bone marrow diseases. Specifically, patients receiving CK0801 in this trial demonstrated some health improvements, offering hope for those with conditions like aplastic anemia, myelodysplastic syndrome, or myelofibrosis. ^{1 2 5 6 7}

Who Is on the Research Team?

Tapan M Kadia, MD

Principal Investigator

The University of Texas MD Anderson Cancer Center

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Published Research Related to This Trial

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]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT03773393

Study Details | NCT03773393 | A Clinical Trial of CK0801 ... 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 ...

2. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/38804782/

Phase 1 Study of CK0801 in Treatment of Bone Marrow ... CK0801 demonstrated no dose-limiting toxicity and showed evidence of efficacy, providing proof of concept for targeting inflammation as a therapy for bone ...

3. pharmacytimes.com pharmacytimes.com/view/allogenic-t-regulatory-cell-product-ck0801-granted-fda-orphan-drug-designation-for-aplastic-anemia-treatmen

Allogeneic T Regulatory Cell Product CK0801 Granted ... Phase 1 trial results showed CK0801's efficacy in bone marrow failure syndromes, with no severe adverse events or dose-limiting toxicity. CK0801 ...

4. sciencedirect.com sciencedirect.com/science/article/pii/S000649712105792X

Adoptive Therapy with Allogeneic Cord Blood T Regulatory ... A single infusion of allogeneic CB derived Treg cells (CK0801) is well tolerated, feasible, and may be associated with clinical improvement in patients with ...

5. prnewswire.com prnewswire.com/news-releases/cellenkos-inc-initiates-phase-1-clinical-trial-of-ck0801-for-treatment-of-bone-marrow-failure-syndromes-300861217.html

Cellenkos, Inc. Initiates Phase 1 Clinical Trial of CK0801 ... "This Phase 1 trial of CK0801 in bone marrow failure syndromes represents an important milestone for Cellenkos," said Elizabeth J. Read, MD, ...

6. withpower.com withpower.com/trial/phase-1-bone-marrow-failure-disorders-4-2019-41204

CK0801 for Bone Marrow Disease · Info for Participants The research does not provide specific safety data for CK0801 or similar treatments for bone marrow disease. How does the drug CK0801 differ from ...

7. prnewswire.com prnewswire.com/news-releases/cellenkos-announces-us-fda-orphan-drug-designation-granted-to-ck0801-allogeneic-cord-blood-derived-t-regulatory-cell-product-for-treatment-of-aplastic-anemia-302427571.html

Cellenkos Announces US FDA Orphan Drug Designation ... CK0801, an allogeneic T regulatory (Treg) cell therapy, leads to durable transfusion independence up to 3.5 years in patients with Aplastic Anemia.