MyelofibrosisPhase 1TREG108NCT05423691
Leading in MPNs Beyond Ruxolitinib in Combo With T-Regs
A Phase 1 study testing CK0804 in people with Myelofibrosis, run by Cellenkos, Inc. at 4 sites in United States.
Trial summary
To assess the safety and tolerability of CK0804 as add-on therapy in participants with myelofibrosis, with suboptimal response to ruxolitinib Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myelofibrosis
- What is being tested?
- CK0804
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 4 sitesUnited States
- Study dates
- 27 Dec 2022to 30 Dec 2027
- Phase
- Phase 1
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Ability to comprehend and willingness to sign a written informed consent form (ICF) for the study.
- Age above 18 years inclusive at the time of signing the ICF.
- Participants who fulfill the diagnostic criteria of myelofibrosis including primary myelofibrosis and myelofibrosis arising from polycythemia vera and essential thrombocythemia
- Life expectancy is greater than 6 months.
- Subject has been receiving ruxolitinib therapy, is unlikely to benefit from further ruxolitinib monotherapy in the opinion of the investigator; AND meeting the following criteria: receiving ruxolitinib >3 months prior to enrollment; AND stable dose for 8 weeks before starting therapy with CK0804
- Subject with evidence of evaluable residual burden of disease following ruxolitinib monotherapy treatment, consisting of:
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- presence of grade ≥2 anemia or thrombocytopenia or neutropenia, OR
- presence of disease-related symptoms, as determined by a Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN SAF TSS) score of ≥10 points, OR
- documented splenomegaly of at least 5 cm below the costal margin as measured by physical examination or splenomegaly as documented by ultrasound or MRI.
- Willingness to avoid pregnancy or fathering children based on the criteria below
- Men must agree to take appropriate precautions to avoid fathering children (with at least 99% certainty) from screening through 90 days after the last study treatment dose and must refrain from donating sperm during this period. Permitted methods that are at least 99% effective in preventing should be communicated to the participants and their understanding confirmed.
- Women of childbearing potential must have a negative serum pregnancy test at screening before the first dose (within 3 days of the first study treatment dose) and must agree to take appropriate precautions to avoid pregnancy (with at least 99% certainty) from screening through the safety follow-up visit and must not donate oocytes during this period. Permitted methods that are at least 99% effective in preventing pregnancy should be communicated to the participants and their understanding confirmed,
- Women of nonchildbearing potential (ie, surgically sterile with a hysterectomy and/or bilateral oophorectomy OR ≥ 12 months of amenorrhea and at least 50 years of age) are eligible.
- ECOG performance status of 0 to 2
Who cannot join
- Any major surgery within 28 days before the first dose of study treatment.
- Undergone any prior allogenic or autologous stem cell transplantation or a candidate for such transplantation.
- Received chemotherapy, immunomodulatory drug therapy, immunosuppressive therapy, biological therapy, endocrine therapy, targeted therapy, antibody or hypomethylating agent to treat the participant's disease, with the exception of ruxolitinib, within 5 half-lives or 28 days (whichever is shorter) before the first dose of study treatment.
- Participant has received splenic irradiation within the past 6 months.
- Significant concurrent, uncontrolled medical condition or infections, which in the opinion of the principal investigator may interfere in the study participation.
- Inability or unlikeliness of the participant to comply with the dose schedule and study evaluations, in the opinion of the investigator.
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- Women who are pregnant or breastfeeding.
- Any condition that would, in the investigator's judgment, interfere with full participation in the study, including administration of study treatment and attending required study visits; pose a significant risk to the participant; or interfere with interpretation of study data.
- Participants with laboratory values at screening as defined
- Platelets < 50 × 10^9/L without the assistance of growth factors, thrombopoietic factors, or platelet transfusions
- ANC < 0.5 × 10^9/L
- ALT ≥ 2.5 × ULN
- AST ≥ 2.5 × ULN
- Direct Bilirubin > 2.0 × ULN
- ALP ≥ 3 × ULN
- Creatinine clearance < 50 mL/min according to Cockcroft-Gault formula.
- Unwillingness to be transfused with blood components including RBC and platelet transfusions.
- Inability of the participant (or parent, guardian, or legally authorized representative) to comprehend the ICF or unwillingness to sign the ICF.
Trial locations and contacts
4 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States4 sites, 4 recruiting
- Recruiting
The University of Texas MD Anderson Cancer Center
Houston, Texas, 77030
Alleyne Genevieve, 713-792-4986, GMAlleyne@mdanderson.org
Lucia Masarova, MD
- Not yet recruiting
Columbia University
New York, New York, 10032
Beatriz Raposo Corrandini, MSc, 212-305-6679, br2469@cumc.columbia.edu
Mark Heaney, MD
- Recruiting
UC Davis Health
Sacramento, California, 95817
Trisha Yassear, CCRC, 916-718-2107, tyassear@ucdavis.edu
Mehrdad Abedi, MD
- Recruiting
Montefiore Einstein Cancer Center
The Bronx, New York, 10461
Clinical Research Coordinator
Swati Goel, MD
About this study
Official title: Phase Ib, Open-label Study of Add on Therapy With CK0804 in Participants With Myelofibrosis, With Suboptimal Response to Ruxolitinib
To assess the safety and tolerability of CK0804 as add-on therapy in participants with myelofibrosis, with suboptimal response to ruxolitinib
Read the detailed description
- Safety Run-in The study will employ a 3+3+3 design to assess the safety and tolerability of the treatment based on treatment-limiting toxicities (TLTs) occurring up to 1 Cycle (28 days) after the first infusion.
- Expansion
After a total of 9 participants completed 28 days and are evaluated for tolerability in the safety run-in phase, additional participants may be included in the expansion cohort in order to have approximately 24 evaluable myelofibrosis
- Conditions
- Myelofibrosis
- Being tested
- CK0804
- Keywords
- myelofibrosis, ruxolitinib, CK0804, T regulatory cells, cord blood unit, allogeneic, Treg Cells, Bone Marrow Diseases, Hematologic Diseases, Bone Marrow Neoplasms, Myeloproliferative Disorders, Thrombocytosis
- Medical subject headings
- Primary Myelofibrosis, Bone Marrow Diseases, Hematologic Diseases, Bone Marrow Neoplasms, Myeloproliferative Disorders, Thrombocytosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CK0804Drug
CK0804 is a cryopreserved, allogeneic T-regulatory cell product that is manipulated to traffic to the bone marrow.
| Study group | Role | What they receive |
|---|---|---|
| Arm 1 | Experimental | CK0804 will be administered intravenously (IV) 100 million Treg Cells every 28 days up to 6 infusions. Receives: Drug: CK0804 |
What is being measured
The primary outcome is the main question the study is designed to answer.
To determine Treatment limiting toxicity (TLT) as defined below
- severe (grade 3 or 4) infusion-related toxicity within 24 hours (NCI-CTCAE V5.0) of exposure that does not resolve with standard of care treatment within 72 hours.
- regimen related death within 28 days
Measured: 28 days
Secondary outcomes (4)
Assessment of overall response rate (ORR) (measured as CR or PR) and its duration, using modified International Working Group-Myeloproliferative Neoplasm Research and Treatment (IWG-MRT) and European Leukemia Net (ELN) consensus report.
To determine the efficacy of CK0804 as add-on therapy in participants with myelofibrosis, with suboptimal response to ruxolitinib
Measured: 6 months
Rate of anemia response as per modified IWG-MRT ELN response criteria.
To determine the efficacy of CK0804 as add-on therapy in participants with myelofibrosis, with suboptimal response to ruxolitinib
Measured: 6 months
Rate of spleen response by imaging at and after 24 weeks as per IWG-MRT ELN response criteria
To determine the efficacy of CK0804 as add-on therapy in participants with myelofibrosis, with suboptimal response to ruxolitinib
Measured: 6 months
Percentage of Participants who will Achieve Total Symptom Score Reduction Greater Than or Equal to (≥) 50% (TSS50) as Measured by Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN-SAF TSS)
To determine the efficacy of CK0804 as add-on therapy in participants with myelofibrosis, with suboptimal response to ruxolitinib
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Jun 2022
- Study start27 Dec 2022
- Primary completion30 Apr 2027 (estimated)
- Study completion30 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (34)Papers the sponsor has linked to this study.
- Other trials for MyelofibrosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- This is a single-arm study consisting of a safety run-in (3+3+3 design) followed by an expansion cohort after completion of safety run-in data is evaluated for tolerability in the safety.
- Masking
- None (open label)
- Enrolment
- 24 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Cellenkos, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05423691
- Sponsor's protocol ID
- CK0804.101.1
- Acronym
- TREG108
- Status last verified
- Oct 2026
- First submitted
- 10 Jun 2022
- Last updated
- 21 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Abdelouahab H, Zhang Y, Wittner M, Oishi S, Fujii N, Besancenot R, Plo I, Ribrag V, Solary E, Vainchenker W, Barosi G, Louache F. CXCL12/CXCR4 pathway is activated by oncogenic JAK2 in a PI3K-dependent manner. Oncotarget. 2016 Jul 22;8(33):54082-54095. doi: 10.18632/oncotarget.10789. eCollection 2017 Aug 15.
PubMed 28903325Barbui T, Thiele J, Gisslinger H, Finazzi G, Vannucchi AM, Tefferi A. The 2016 revision of WHO classification of myeloproliferative neoplasms: Clinical and molecular advances. Blood Rev. 2016 Nov;30(6):453-459. doi: 10.1016/j.blre.2016.06.001. Epub 2016 Jun 11.
PubMed 27341755Barbui T, Thiele J, Gisslinger H, Kvasnicka HM, Vannucchi AM, Guglielmelli P, Orazi A, Tefferi A. The 2016 WHO classification and diagnostic criteria for myeloproliferative neoplasms: document summary and in-depth discussion. Blood Cancer J. 2018 Feb 9;8(2):15. doi: 10.1038/s41408-018-0054-y.
PubMed 29426921Barosi G, Mesa RA, Thiele J, Cervantes F, Campbell PJ, Verstovsek S, Dupriez B, Levine RL, Passamonti F, Gotlib J, Reilly JT, Vannucchi AM, Hanson CA, Solberg LA, Orazi A, Tefferi A; International Working Group for Myelofibrosis Research and Treatment (IWG-MRT). Proposed criteria for the diagnosis of post-polycythemia vera and post-essential thrombocythemia myelofibrosis: a consensus statement from the International Working Group for Myelofibrosis Research and Treatment. Leukemia. 2008 Feb;22(2):437-8. doi: 10.1038/sj.leu.2404914. Epub 2007 Aug 30. No abstract available.
PubMed 17728787Bogani C, Ponziani V, Guglielmelli P, Desterke C, Rosti V, Bosi A, Le Bousse-Kerdiles MC, Barosi G, Vannucchi AM; Myeloproliferative Disorders Research Consortium. Hypermethylation of CXCR4 promoter in CD34+ cells from patients with primary myelofibrosis. Stem Cells. 2008 Aug;26(8):1920-30. doi: 10.1634/stemcells.2008-0377. Epub 2008 May 29.
PubMed 18511598Brunstein CG, Miller JS, Cao Q, McKenna DH, Hippen KL, Curtsinger J, Defor T, Levine BL, June CH, Rubinstein P, McGlave PB, Blazar BR, Wagner JE. Infusion of ex vivo expanded T regulatory cells in adults transplanted with umbilical cord blood: safety profile and detection kinetics. Blood. 2011 Jan 20;117(3):1061-70. doi: 10.1182/blood-2010-07-293795. Epub 2010 Oct 15.
PubMed 20952687Brunstein CG, Miller JS, McKenna DH, Hippen KL, DeFor TE, Sumstad D, Curtsinger J, Verneris MR, MacMillan ML, Levine BL, Riley JL, June CH, Le C, Weisdorf DJ, McGlave PB, Blazar BR, Wagner JE. Umbilical cord blood-derived T regulatory cells to prevent GVHD: kinetics, toxicity profile, and clinical effect. Blood. 2016 Feb 25;127(8):1044-51. doi: 10.1182/blood-2015-06-653667. Epub 2015 Nov 12.
PubMed 26563133Cho SY, Xu M, Roboz J, Lu M, Mascarenhas J, Hoffman R. The effect of CXCL12 processing on CD34+ cell migration in myeloproliferative neoplasms. Cancer Res. 2010 Apr 15;70(8):3402-10. doi: 10.1158/0008-5472.CAN-09-3977. Epub 2010 Apr 13.
PubMed 20388788Emanuel RM, Dueck AC, Geyer HL, Kiladjian JJ, Slot S, Zweegman S, te Boekhorst PA, Commandeur S, Schouten HC, Sackmann F, Kerguelen Fuentes A, Hernandez-Maraver D, Pahl HL, Griesshammer M, Stegelmann F, Doehner K, Lehmann T, Bonatz K, Reiter A, Boyer F, Etienne G, Ianotto JC, Ranta D, Roy L, Cahn JY, Harrison CN, Radia D, Muxi P, Maldonado N, Besses C, Cervantes F, Johansson PL, Barbui T, Barosi G, Vannucchi AM, Passamonti F, Andreasson B, Ferrari ML, Rambaldi A, Samuelsson J, Birgegard G, Tefferi A, Mesa RA. Myeloproliferative neoplasm (MPN) symptom assessment form total symptom score: prospective international assessment of an abbreviated symptom burden scoring system among patients with MPNs. J Clin Oncol. 2012 Nov 20;30(33):4098-103. doi: 10.1200/JCO.2012.42.3863. Epub 2012 Oct 15.
PubMed 23071245Gladstone DE, Kim BS, Mooney K, Karaba AH, D'Alessio FR. Regulatory T Cells for Treating Patients With COVID-19 and Acute Respiratory Distress Syndrome: Two Case Reports. Ann Intern Med. 2020 Nov 17;173(10):852-853. doi: 10.7326/L20-0681. Epub 2020 Jul 6. No abstract available.
PubMed 32628535Gleitz HFE, Dugourd AJF, Leimkuhler NB, Snoeren IAM, Fuchs SNR, Menzel S, Ziegler S, Kroger N, Triviai I, Busche G, Kreipe H, Banjanin B, Pritchard JE, Hoogenboezem R, Bindels EM, Schumacher N, Rose-John S, Elf S, Saez-Rodriguez J, Kramann R, Schneider RK. Increased CXCL4 expression in hematopoietic cells links inflammation and progression of bone marrow fibrosis in MPN. Blood. 2020 Oct 29;136(18):2051-2064. doi: 10.1182/blood.2019004095.
PubMed 32726410Harrison C, Kiladjian JJ, Al-Ali HK, Gisslinger H, Waltzman R, Stalbovskaya V, McQuitty M, Hunter DS, Levy R, Knoops L, Cervantes F, Vannucchi AM, Barbui T, Barosi G. JAK inhibition with ruxolitinib versus best available therapy for myelofibrosis. N Engl J Med. 2012 Mar 1;366(9):787-98. doi: 10.1056/NEJMoa1110556.
PubMed 22375970Hori S, Nomura T, Sakaguchi S. Control of regulatory T cell development by the transcription factor Foxp3. Science. 2003 Feb 14;299(5609):1057-61. doi: 10.1126/science.1079490. Epub 2003 Jan 9.
PubMed 12522256Kadia TM, Ma, H, Zeng K, Nishimoto M, Lyu Mi-Ae, Huang M, Yilmaz M, DiNardo DC, Issa GC, Parmar S, Iyer SP, Hari P, Daver NG, Jabbour E, Borthakur GM, and Verstovsek S. 2019. 'Phase I Clinical Trial of CK0801 (cord blood regulatory T cells) in Patients with Bone Marrow Failure Syndrome (BMF) Including Aplastic Anemia, Myelodysplasia and Myelofibrosis', Blood.
Kadia TM, Pemmaraju N, Yilmaz M, Li L, Lyu M, Huang M, Zeng K, Parmar S, DiNardo CD, Daver N, Issa GC, Jabbour E, Borthakur G, Verstovsek S. 2020. 'Adoptive Therapy with Allogeneic Cord Blood T Regulatory Cells Show Safety and Early Clinical Signal in Primary Myelofibrosis', Blood, 136: 41-42.