CK0804 for Myelofibrosis

(TREG108 Trial)

Recruiting at 4 trial locations

Overseen By Tara Sadeghi

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Cellenkos, Inc.

Must be taking: Ruxolitinib

Must not be taking: Chemotherapy, Immunosuppressants, others

Disqualifiers: Major surgery, Stem cell transplant, others

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

Compensation: Varies

Number of Visits: 2

24 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called CK0804 for individuals with myelofibrosis who aren't responding well to their current treatment with ruxolitinib. The goal is to determine if CK0804 is safe and tolerable as an additional treatment. Participants will receive CK0804 through an IV every 28 days, up to six times. Suitable candidates for this trial include those with myelofibrosis who continue to experience symptoms or issues like anemia (low red blood cells) or splenomegaly (enlarged spleen) despite prolonged use of ruxolitinib. As a Phase 1 trial, this research aims to understand how CK0804 works in people, offering participants the chance to be among the first to receive this new treatment.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but you must have been on a stable dose of ruxolitinib for at least 8 weeks before starting the trial. Other treatments, except ruxolitinib, should not have been taken within 28 days or 5 half-lives before the trial.

Is there any evidence suggesting that CK0804 is likely to be safe for humans?

Research has shown that CK0804 is safe for people with bone marrow issues. Studies found that patients tolerated the treatment well. In these studies, CK0804 was used alongside other treatments.

The trials used Treg cells, a type of immune cell that helps reduce inflammation. This method appears safe, even if the cells aren't a perfect match for the patient.

While more research is needed, the current results are promising. So far, no major safety issues have been reported with CK0804, which is encouraging for those considering joining a trial. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Researchers are excited about CK0804 for treating myelofibrosis because it introduces a novel approach by using Treg cells, which are a type of regulatory T cell. Unlike standard treatments that often focus on symptom management or slowing disease progression, CK0804 specifically aims to modulate the immune system by delivering 100 million Treg cells intravenously every 28 days. This innovative mechanism could potentially address the underlying issues of inflammation and fibrosis in a more targeted way, offering hope for improved outcomes for patients.

What evidence suggests that CK0804 might be an effective treatment for myelofibrosis?

Research has shown that CK0804, the investigational treatment in this trial, may serve as a helpful additional treatment for myelofibrosis, particularly for patients who don't fully benefit from ruxolitinib. Studies have found that CK0804 can lower inflammation, enhancing the treatment's effectiveness. In one study, patients experienced a median symptom score drop of 38%, with some reductions reaching up to 71%. Additionally, patients reported feeling less tired and more comfortable after eating. These early results suggest CK0804 could be a promising option for people with myelofibrosis. ^{1 3 5 6 7}

Are You a Good Fit for This Trial?

Adults over 18 with myelofibrosis not responding well to ruxolitinib can join this trial. They should have a life expectancy over 6 months, some remaining disease symptoms or spleen enlargement, and be able to follow the study plan. Women must test negative for pregnancy and avoid becoming pregnant; men must prevent fathering children during the study.

Inclusion Criteria

I still have signs of my disease after treatment with ruxolitinib alone.

I am over 18 years old.

I can take care of myself and am up and about more than half of my waking hours.

Exclusion Criteria

I refuse to receive blood transfusions.

Any condition that would interfere with full participation in the study

I have had radiation treatment to my spleen in the last 6 months.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Safety Run-in

The study employs a 3+3+3 design to assess safety and tolerability based on treatment-limiting toxicities occurring up to 1 Cycle (28 days) after the first infusion.

4 weeks

1 visit (in-person) every 28 days

Expansion

Additional participants are included in the expansion cohort to have approximately 24 evaluable myelofibrosis participants.

6 months

1 visit (in-person) every 28 days

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• CK0804

Trial Overview The trial is testing CK0804 as an additional treatment for those with myelofibrosis who haven't had enough improvement from ruxolitinib alone. The goal is to see if it's safe and how well patients tolerate it when added to their current therapy.

How Is the Trial Designed?

Group I: Arm 1 Experimental Treatment 1 Intervention

CK0804 will be administered intravenously (IV) 100 million Treg Cells every 28 days up to 6 infusions.

Who Is Running the Clinical Trial?

Cellenkos, Inc.

Lead Sponsor

Trials

5

Recruited

150+

Published Research Related to This Trial

Fedratinib has been approved by the FDA as a treatment for myelofibrosis, marking it as the second drug and second JAK inhibitor approved for this condition.

In the phase III JAKARTA trial, fedratinib significantly reduced symptoms of myelofibrosis compared to a placebo, but it carries a Boxed Warning for the risk of encephalopathy, highlighting the need for careful monitoring.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Fedratinib Becomes New Option in Myelofibrosis. [2020]

Fedratinib, a selective JAK2 inhibitor, was approved by the FDA for treating intermediate-2 or high-risk myelofibrosis (MF) based on phase II and phase III trials, showing significant reductions in spleen size and symptom burden compared to placebo.

While fedratinib is effective, it has notable side effects including anemia and gastrointestinal issues, and carries a black box warning for encephalopathy, which occurred in about 1% of patients, necessitating careful monitoring of thiamine levels.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Beyond Ruxolitinib: Fedratinib and Other Emergent Treatment Options for Myelofibrosis. Bewersdorf, JP., Jaszczur, SM., Afifi, S., et al.[2020]

Citations

1. ashpublications.org ashpublications.org/blood/article/144/Supplement%201/999/530416/A-Phase-Ib-Open-Label-Study-of-Add-on-Therapy-with

A Phase Ib, Open-Label Study of Add on Therapy with ... The median best decline in MPN-SAF TSS was -38% (range, -20% to -71%); with ³50% reduction in 4 patients. Four out of 6 evaluable patients had ...

2. healthtree.org healthtree.org/myelofibrosis/community/articles/ash-24-ck0804-ruxolitinib-myelofibrosis-phase-1

Ruxolitinib + CK0804 Clinical Trial for MF This study found that reducing inflammation with CK0804 enhanced the effectiveness of treatment for individuals with myelofibrosis who did not respond well to ...

3. ascopubs.org ascopubs.org/doi/10.1200/JCO.2024.42.16_suppl.6580

A phase Ib, open-label study of add-on therapy with ... Results: Nine patients of median age 68 years (range, 55-84) were enrolled, 44% were males. Median blood counts [range] showed while blood cells ...

4. pharmatimes.com pharmatimes.com/news/cellenkos-presents-promising-phase-1b-data-for-ck0804-in-myelofibrosis/

Cellenkos presents promising phase 1b data for CK0804 in ... The study demonstrated reductions in spleen volume and symptom burden, with patients showing improvement in fatigue and early satiety. “We are ...

5. oncodaily.com oncodaily.com/insight/206721

Cellenkos Presented Results of Phase 1b Data for ... Cellenkos Presented Results of Phase 1b Data for CK0804 T-Regulatory Cell Therapy in Myelofibrosis at ASH 2024. Innovative Cellular Therapy ...

6. ascopubs.org ascopubs.org/doi/10.1200/JCO.2023.41.16_suppl.TPS7087

Phase Ib, open-label study of add on therapy with CK0804 ... Adoptive therapy with allogeneic, cord blood (CB) derived Regulatory T cells (Tregs) have shown to be safe in bone marrow failure patients.

7. clinicaltrials.gov clinicaltrials.gov/study/NCT05423691

Study Details | NCT05423691 | Leading in MPNs Beyond ... Study Overview. Brief Summary. To assess the safety and tolerability of CK0804 as add-on therapy in participants with myelofibrosis, with suboptimal ...