CK0804 for Myelofibrosis

(TREG108 Trial)

Recruiting at 3 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

Trial Summary

What is the purpose of this trial?

To assess the safety and tolerability of CK0804 as add-on therapy in participants with myelofibrosis, with suboptimal response to ruxolitinib

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.

What safety information is available for CK0804 (Fedratinib) in treating myelofibrosis?

Fedratinib, also known as CK0804, has been approved for myelofibrosis but comes with a warning for encephalopathy (a brain disorder), which is rare. Common side effects include anemia (low red blood cells), stomach issues, and liver enzyme changes, so monitoring is recommended. ^{1 2 3 4 5}

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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+

Findings from Research

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]

References

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

Fedratinib Becomes New Option in Myelofibrosis. [2020]

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

Beyond Ruxolitinib: Fedratinib and Other Emergent Treatment Options for Myelofibrosis. [2020]

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

Phase 2 study of CEP-701, an orally available JAK2 inhibitor, in patients with primary or post-polycythemia vera/essential thrombocythemia myelofibrosis. [2023]

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

The evolving treatment paradigm in myelofibrosis. [2021]

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

The prognostic contribution of CBL, NRAS, KRAS, RUNX1, and TP53 mutations to mutation-enhanced international prognostic score systems (MIPSS70/plus/plus v2.0) for primary myelofibrosis. [2023]