AzTrials
AzTrials

Myelofibrosis，MFEarly Phase 1NCT06414681

Combination of Tagraxofusp With Pacritinib in Patients With Intermediate-1 or Higher Myelofibrosis, Who Have Had Prior Therapy With the Approved JAK Inhibitors or in Which Therapy With the Approved JAK Inhibitors is Not Appropriate, Contraindicated or Declined

A Early Phase 1 study testing Tagraxofusp and Pacritinib in people with Myelofibrosis，MF, run by University of Kansas Medical Center at 1 site in United States.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
Search all trials

Trial summary

The goal of this open-label, single-center, pilot trial is to test the combination of Tagraxofusp (TAG) with Pacritinib (PAC) in patients with intermediate-II or higher myelofibrosis (MF), who have had prior therapy with the approved JAK1/2 inhibitor or in which therapy with the approved JAK1/2 inhibitors is not… Read more about this study

Who is it for?
Ages 18 years and olderHealthy volunteers can take part
Conditions the study is for
Myelofibrosis，MF
What is being tested?
Tagraxofusp and Pacritinib
Could I receive a placebo?
No, everyone receives an active treatment
How many people
20 participants
Where
1 siteUnited States
Study dates
25 Aug 2025to Dec 2026
Phase
Early Phase 1
Are there results?
Not yet: the study is under way

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
  • Healthy volunteers accepted
  • Adult, Older adult

Who may be able to join

  • Ability of participant OR Legally Authorized Representative (LAR) to understand this study, and participant or LAR willingness to sign a written informed consent.
  • The participant or LAR has signed informed consent prior to initiation of any study-specific procedures or treatment.
  • The patient is able to adhere to the study visit schedule and other protocol requirements.
  • Males and females age ≥ 18 years.
  • ECOG Performance Status 0 - 2 (Appendix A).
  • Life expectancy of > 6 months.
Show all 19Show fewer
  • Patient meets the 2016 WHO diagnostic criteria for MF and has an IPSS/DIPSS/DIPSS-plus intermediate-II or higher-risk disease.
  • Patients who have indications for therapy per investigator or patient's choice, such as Splenomegaly, >5 CM BCM or mTSS ≥ 8 or mTSS Itching, night sweats, or bone pain ≥ 5 or Significant cytopenias including Hgb <10 g/dl, Platelet count less than 75 k/UL
  • Patients treated with a JAK inhibitor for >3 months and:
  • had inadequate response to treatment, i.e., < 10% reduction of spleen by imaging, or less than 25% reduction by spleen length on physical exam or lack of control of MF symptoms that is not satisfactory to the patient. NOTE: Participants who had contraindication to therapy with the approved JAK inhibitor including subject's refusal of therapy are eligible.
  • A least 4 weeks have elapsed between the last dose of any MF-directed drug treatments, including hydroxyurea (HU), Interferon or glucocorticosteroids. NOTE: If patient is on a stable dose of glucocorticosteroids for another indication, they will be allowed into this study, AND patients on a stable dose of erythropoiesis-stimulating agents (ESA) are allowed on the study.
  • Patient is not eligible for an immediate allo-SCT.
  • Adequate baseline organ, cardiac, and renal function as defined by:
  • LVEF ≥ 50% by ECHO within 6 months of study treatment initiation No clinically significant abnormalities on a 12-lead ECG, and no QTcF ≥ 480 msec Serum creatinine ≤ 1.5 mg/dL Serum albumin ≥ 3.2 g/dL (Note: albumin infusions are not permitted to enable eligibility) INR and PTT ≤ 1.5x ULN Albumin Supplementation Prior to the first dose, participant must have serum albumin ≥ to 3.2 g/dL.
  • Note- for any participants with serum albumin ≤ to 4.0 g/dL, it will be advisable but up to physician's discretion to administer 25g increments of albumin infusion before the first dose.
  • Total bilirubin ≤ 4x ULN AST and ALT ≤ 5 x ULN ANC ≥ 0.5 x 109/L PT or INR and PTT < = 1.5 x ULN
  • If the patient is a woman of child-bearing potential (WOCBP), they should have a negative serum pregnancy test no more than 7 days prior to start of treatment.
  • (Note: WOCBP include any female who has experienced menarche and who has not undergone successful sterilization (hysterectomy, bilateral tubal ligation or bilateral oophorectomy) or is not postmenopausal (defined as amenorrhea ≥ 12 consecutive months; or women on hormone replacement therapy with documented serum follicle stimulating hormone level ≥ 35 mIU/mL).
  • Women of child-bearing potential and men with partners of child-bearing potential must agree to practice sexual abstinence or to use the forms of contraception listed in Child-Bearing Potential/Pregnancy section for the duration of study participation and for 3 months following completion of therapy.

Who cannot join

  • Simultaneously enrolled in any therapeutic clinical trial
  • Current or anticipating use of other anti-neoplastic or investigational agents while participating in this study.
  • The patient has received treatment with chemotherapy, wide-field radiation, or biologic therapy within 14 days of study entry.
  • The patient has received treatment with another investigational agent within 14 days of study entry.
  • Diagnosed with a psychiatric illness or is in a social situation that would limit compliance with study requirements.
  • Uncontrolled intercurrent illness including, but not limited to, uncontrolled infection, disseminated intravascular coagulation.
Show all 32Show fewer
  • Any condition or other contraindication to therapy as deemed by the principal investigator to place the subject at an unacceptably high risk for toxicities.
  • Is pregnant or breastfeeding.
  • Has a known allergic reaction to any excipient contained in the study drug formulation.
  • Active Grade 3 (per the NCI CTCAE, Version 5.0) or higher viral, bacterial, or fungal infection within 2 weeks prior to the first dose of study treatment.
  • Prior therapy with Tagraxofusp or Pacritinib.
  • Presence of peripheral blood or bone marrow blast count > 10%
  • Active Graft versus Host Disease (GVHD)
  • For patients who have previously had Stem Cell Therapy (SCT) - The patient is receiving immunosuppressive therapy.
  • EXCEPTION: low-dose prednisone (≤10 mg/day) - for treatment or prophylaxis of graft-versus-host disease (GVHD). If the patient has been on immunosuppressive treatment or prophylaxis for GVHD, the treatment(s) must have been discontinued at least 14 days prior to study treatment and there must be no evidence of Grade ≥ 2 GVHD.
  • Other uncontrolled active malignancy as determined by the principal investigator
  • The patient has an active malignancy and/or cancer history that may confound the assessment of the study endpoints. Patients with a past cancer history (within 2 years of entry) with substantial potential for recurrence and/or ongoing active malignancy must be discussed with the Sponsor before study entry. Patients with the following neoplastic diagnoses are eligible: non-melanoma skin cancer, carcinoma in situ, cervical intraepithelial neoplasia, organ-confined prostate cancer with no evidence of progressive disease.
  • The patient has clinically significant cardiovascular disease (e.g. uncontrolled or any New York Heart Association Class 2 or greater congestive heart failure, uncontrolled angina, history of myocardial infarction, unstable angina or stroke within 6 months prior to study entry, uncontrolled hypertension or clinically significant arrhythmias not controlled by medication).
  • Any gastrointestinal or metabolic condition that could interfere with absorption of oral medication.
  • Prior therapy with Pacritinib (PAC) or Tagraxofusp (TAG).
  • The patient has persistent clinically significant toxicities Grade ≥ 2 from previous therapies, including cytotoxic chemotherapy, targeted therapies, biological therapies, or immunotherapies, not readily controlled by supportive measures (excluding alopecia, nausea, and fatigue).
  • The patient has uncontrolled, clinically significant pulmonary disease (e.g. chronic obstructive pulmonary disease, pulmonary hypertension) that in the opinion of the Investigator would put the patient at significant risk for pulmonary complications during the study.
  • The patient has known active or suspected central nervous system (CNS) disease. If suspected, CNS disease should be ruled out with relevant imaging and/or examination of cerebrospinal fluid.
  • Current systemic treatment with strong or moderate CYP3A4 inhibitor or P450 inducer.
  • EXCEPTION: Patients who discontinue this treatment by Day 14 before start of treatment (Day -14) or 5 half-lives, whichever is shorter.
  • Use of full-dose anticoagulation and use of anti-platelet therapy other than aspirin 81mg daily within 14 days prior to D1.
  • Recent unprovoked bleeding of grade ≥ 2 within the last 3 months prior to Day 1.
  • Active uncontrolled diarrhea or inflammatory bowel disease (IBD)
  • Known sero-positivity for Hepatitis A (HAV), Hepatitis B (HBV), Hepatitis C (HCV), Human Immunodeficiency Virus (HIV)
  • Patients with history of clinically significant bleeding or on anticoagulants
  • Patients with moderate (Child-Pugh B ) and severe (Child -Pugh C) hepatic impairment will not be enrolled in the study.
  • Patients with eGFR < 30 mL/min will not be enrolled

Trial locations and contacts

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

KUCC Navigation contact

913-588-3671

kucc_navigation@kumc.edu

Victoria Guinn, B.S contact

913-574-1446

vguinn@kumc.edu

United States1 site, 1 recruiting

  • The University of Kansas Cancer Center

    Fairway, Kansas, 66204

    Dr. Abdulraheem Yacoub Professor Hematology Medicine, MD, ayacoub@kumc.edu

    Recruiting

Other trials in United States

About this study

Official title: An Open Label Pilot Trial of the Combination of Tagraxofusp With Pacritinib in Patients With Intermediate-1 or Higher Myelofibrosis, Who Have Had Prior Therapy With the Approved JAK Inhibitors or in Which Therapy With the Approved JAK Inhibitors is Not Appropriate, Contraindicated or Declined

The goal of this open-label, single-center, pilot trial is to test the combination of Tagraxofusp (TAG) with Pacritinib (PAC) in patients with intermediate-II or higher myelofibrosis (MF), who have had prior therapy with the approved JAK1/2 inhibitor or in which therapy with the approved JAK1/2 inhibitors is not appropriate, contraindicated or declined by the subjects.

The Primary Objective is to:

Read more
  1. Characterized efficacy of the combination of Tagraxofusp and Pacritinib.

The Secondary Objective is to:

  1. characterize the safety profile of the combination Tagraxofusp and Pacritinib.

2, Characterize the feasibility of the combination Tagraxofusp and Pacritinib. 3. Characterize hematologic improvement with the combination Tagraxofusp and Pacritinib.

  1. Evaluate and compare the effect of Tagraxofusp and Pacritinib on participant reports of MF symptoms.

Exploratory:

Pharmacokinetic (PK) testing of Tagraxofusp and Pacritinib to assess clinical predictors of response.

Next Generation Sequencing (NGS) Testing to define the number and the allele burden of pathological mutations, as well as the changes over the course of therapy, both in regard to progression and response.

Blood will be collected and stored at KU BRCF for future study related PK analysis

Read the detailed description

A combination of these agent provides rational scientific merit and compatible mechanisms of action by targeting myelofibrosis stem cells and BM environment, in combination with effective JAK2 and IRAK1 inhibition resulting in improvement in MPN related symptoms and splenomegaly without overlapping toxicities. Both agents have been studied in mildly depleted bone marrow phenotypes showing safety and hematological improvements

Being tested
Tagraxofusp, Pacritinib
Keywords
Myelofibrosis, MF, JAK 1/2, Tagraxofusp, TAG, Pacritinib, PAC, Tagraxofusp with Pacritinib, Intermediate II Myelofibrosis, Higher Myelofibrosis
Medical subject headings
Primary Myelofibrosis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • TagraxofuspDrug

    Tagraxofusp Patients will receive 12 micrograms/kg of Tagraxofusp (TAG) by IV infusion once daily for 3 consecutive days.

  • PacritinibDrug

    Pacritinib Pacritinib (PAC) will be given orally, 200 mg twice per day starting at C2D4 and administered continuously (Subsequent cycles start on Day 1 of the Cycle).

Study groups in NCT06414681
Study groupRoleWhat they receive
Tagrxofusp (IV) in combination with Pacritinib (Oral)ExperimentalTagraxofusp Patients will receive 12 micrograms/kg of Tagraxofusp (TAG) by IV infusion once daily for 3 consecutive days. Pacritinib Pacritinib (PAC) will be given orally, 200 mg twice per day starting at C2D4 and administered continuously (Subsequent cycles start on Day 1 of the Cycle).
Receives: Drug: Tagraxofusp; Drug: Pacritinib

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Spleen volume reduction by MRI or CT imaging, achieving ≥ 35% reduction in spleen volume imaging from baseline to week 24.

    MRI of abdomen will be performed WITHOUT contrast. If MRI is contraindicated - CT scan will be allowed (IV contrast will be used unless contraindicated). The same type of Imaging used at screening must continue to be used throughout the study.

    Measured: Baseline to up to 24 weeks

  2. Change from baseline in the Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score Version 2.0 (MPN-SAF TSS 2.0) to week 24.

    Improvement in symptoms: >/= 50% reduction in Modified Total Symptom Score (mTSS) from baseline to week 24 as measured by the Myeloproliferative Neoplasms Symptom Assessment Form Total Symptom Score Version 2.0 (MPN-SAF TSS 2.0). The MPN Symptom Assessment Form) includes the assessment of symptoms that are relevant to myelofibrosis. MPN-SAF was simplified to a concise and abbreviated tool called the MPN-SAF Total Symptom Score (MPN-SAF TSS), that is used for the assessment of the 10 most relevant symptoms in subjects with MPN (fatigue, concentration, early satiety, inactivity, night sweats, itching, bone pain, abdominal discomfort, weight loss, and fever) in both clinical practice and clinical trial settings. The symptom severity is rated by subjects on a scale of 1 to10 MPN-SAF-TSS: a total score will be calculated by the addition of every individual symptom score. The change from baseline will be statically measured and reported.

    Measured: Baseline to up to 24 weeks

Secondary outcomes (7)

  1. Number of participants with Treatment-Related Adverse Events as Assessed by CTCAE v5.0 from baseline to End of Safety Follow-up (30 days after end of treatment).

    Number of participants with treatment related adverse events a assessed by Common Terminology Criteria for Adverse Events (CTCAE) v5.0.

    Measured: Baseline to End of Safety follow up to End of Treatment (up to 1 year)

  2. Change from baseline in Anemia (hemoglobin GM/DL, iron ug/DL, and hematocrit %) improvement within or at 1 year

    From Baseline to best response within 1 year

    Measured: Baseline, Week 24 and End of Treatment (up to 1 year)

  3. Patient's Global Impression (perception) of Change

    Measured: Every cycle (each cycle is 28 days) day 1 starting at Cycle 2 to End of Treatment (up to 1 year)

  4. Improvement in Quality of Life based on Patient Impression of Global Change (PGIC). A PGIC score of 2 for "much improved" or a score of 1 for "very much improved."

    Based on the proportion of patients who report "much improved" or "very much improved" symptoms at Week 24 based on the Patient Impression of Global Change (PGIC). A PGIC score of 2 for "much improved" or a score of 1 for "very much improved."

    The PGIC uses a score between 1 and 7 (1 being "very much improved" to 7 being "very much worse)."

    Measured: Baseline to up to 24 weeks

  5. Change from baseline in platelet count in K/UL within 1 year

    From Baseline to best response within 1 year

    Measured: Baseline, up to 24 weeks and End of Treatment (up to 1 year)

  6. Change from baseline in Anemia (iron ug/DL)

    From Baseline to best response within 1 year

    Measured: Baseline, Week 24 and End of Treatment (up to 1 year)

  7. Change from baseline in Anemia (hematocrit %)

    From Baseline to best response within 1 year

    Measured: Baseline, Week 24 and End of Treatment (up to 1 year)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted16 May 2024
  2. Study start25 Aug 2025
  3. Primary completionDec 2026 (estimated)
  4. Study completionDec 2026 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Early Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
20 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06414681
Sponsor's protocol ID
STUDY00160238
Status last verified
Apr 2026
First submitted
23 Feb 2024
Last updated
6 May 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Publications

  • Mesa RA, Gotlib J, Gupta V, Catalano JV, Deininger MW, Shields AL, Miller CB, Silver RT, Talpaz M, Winton EF, Harvey JH, Hare T, Erickson-Viitanen S, Sun W, Sandor V, Levy RS, Kantarjian HM, Verstovsek S. Effect of ruxolitinib therapy on myelofibrosis-related symptoms and other patient-reported outcomes in COMFORT-I: a randomized, double-blind, placebo-controlled trial. J Clin Oncol. 2013 Apr 1;31(10):1285-92. doi: 10.1200/JCO.2012.44.4489. Epub 2013 Feb 19.

    PubMed 23423753

  • Jordan CT, Guzman ML, Noble M. Cancer stem cells. N Engl J Med. 2006 Sep 21;355(12):1253-61. doi: 10.1056/NEJMra061808. No abstract available.

    PubMed 16990388

  • Hurst H, Bolton J. Assessing the clinical significance of change scores recorded on subjective outcome measures. J Manipulative Physiol Ther. 2004 Jan;27(1):26-35. doi: 10.1016/j.jmpt.2003.11.003.

    PubMed 14739871

  • Tefferi A. Challenges facing JAK inhibitor therapy for myeloproliferative neoplasms. N Engl J Med. 2012 Mar 1;366(9):844-6. doi: 10.1056/NEJMe1115119. No abstract available.

    PubMed 22375977

  • Tefferi A. Primary myelofibrosis: 2013 update on diagnosis, risk-stratification, and management. Am J Hematol. 2013 Feb;88(2):141-50. doi: 10.1002/ajh.23384.

    PubMed 23349007

  • Barosi G, Tefferi A, Besses C, Birgegard G, Cervantes F, Finazzi G, Gisslinger H, Griesshammer M, Harrison C, Hehlmann R, Hermouet S, Kiladjian JJ, Kroger N, Mesa R, Mc Mullin MF, Pardanani A, Passamonti F, Samuelsson J, Vannucchi AM, Reiter A, Silver RT, Verstovsek S, Tognoni G, Barbui T. Clinical end points for drug treatment trials in BCR-ABL1-negative classic myeloproliferative neoplasms: consensus statements from European LeukemiaNET (ELN) and Internation Working Group-Myeloproliferative Neoplasms Research and Treatment (IWG-MRT). Leukemia. 2015 Jan;29(1):20-6. doi: 10.1038/leu.2014.250. Epub 2014 Aug 25.

    PubMed 25151955

  • Harrison 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 22375970

  • Harrison CN, Mesa RA, Kiladjian JJ, Al-Ali HK, Gisslinger H, Knoops L, Squier M, Sirulnik A, Mendelson E, Zhou X, Copley-Merriman C, Hunter DS, Levy RS, Cervantes F, Passamonti F, Barbui T, Barosi G, Vannucchi AM. Health-related quality of life and symptoms in patients with myelofibrosis treated with ruxolitinib versus best available therapy. Br J Haematol. 2013 Jul;162(2):229-39. doi: 10.1111/bjh.12375. Epub 2013 May 14.

    PubMed 23672349

  • Verstovsek S, Mesa RA, Gotlib J, Levy RS, Gupta V, DiPersio JF, Catalano JV, Deininger M, Miller C, Silver RT, Talpaz M, Winton EF, Harvey JH Jr, Arcasoy MO, Hexner E, Lyons RM, Paquette R, Raza A, Vaddi K, Erickson-Viitanen S, Koumenis IL, Sun W, Sandor V, Kantarjian HM. A double-blind, placebo-controlled trial of ruxolitinib for myelofibrosis. N Engl J Med. 2012 Mar 1;366(9):799-807. doi: 10.1056/NEJMoa1110557.

    PubMed 22375971

  • Mughal TI, Cross NC, Padron E, Tiu RV, Savona M, Malcovati L, Tibes R, Komrokji RS, Kiladjian JJ, Garcia-Manero G, Orazi A, Mesa R, Maciejewski JP, Fenaux P, Itzykson R, Mufti G, Solary E, List AF. An International MDS/MPN Working Group's perspective and recommendations on molecular pathogenesis, diagnosis and clinical characterization of myelodysplastic/myeloproliferative neoplasms. Haematologica. 2015 Sep;100(9):1117-30. doi: 10.3324/haematol.2014.114660.

    PubMed 26341525

  • Barbui T, Tefferi A, Vannucchi AM, Passamonti F, Silver RT, Hoffman R, Verstovsek S, Mesa R, Kiladjian JJ, Hehlmann R, Reiter A, Cervantes F, Harrison C, Mc Mullin MF, Hasselbalch HC, Koschmieder S, Marchetti M, Bacigalupo A, Finazzi G, Kroeger N, Griesshammer M, Birgegard G, Barosi G. Philadelphia chromosome-negative classical myeloproliferative neoplasms: revised management recommendations from European LeukemiaNet. Leukemia. 2018 May;32(5):1057-1069. doi: 10.1038/s41375-018-0077-1. Epub 2018 Feb 27.

    PubMed 29515238

  • Prager BC, Xie Q, Bao S, Rich JN. Cancer Stem Cells: The Architects of the Tumor Ecosystem. Cell Stem Cell. 2019 Jan 3;24(1):41-53. doi: 10.1016/j.stem.2018.12.009.

    PubMed 30609398

  • Desai A, Yan Y, Gerson SL. Concise Reviews: Cancer Stem Cell Targeted Therapies: Toward Clinical Success. Stem Cells Transl Med. 2019 Jan;8(1):75-81. doi: 10.1002/sctm.18-0123. Epub 2018 Oct 17.

    PubMed 30328686

  • Yan B, Chen Q, Shimada K, Tang M, Li H, Gurumurthy A, Khoury JD, Xu B, Huang S, Qiu Y. Histone deacetylase inhibitor targets CD123/CD47-positive cells and reverse chemoresistance phenotype in acute myeloid leukemia. Leukemia. 2019 Apr;33(4):931-944. doi: 10.1038/s41375-018-0279-6. Epub 2018 Oct 5.

    PubMed 30291336

  • Arai N, Homma M, Abe M, Baba Y, Murai S, Watanuki M, Kawaguchi Y, Fujiwara S, Kabasawa N, Tsukamoto H, Uto Y, Ariizumi H, Yanagisawa K, Hattori N, Saito B, Shiozawa E, Harada H, Yamochi-Onizuka T, Nakamaki T, Takimoto M. Impact of CD123 expression, analyzed by immunohistochemistry, on clinical outcomes in patients with acute myeloid leukemia. Int J Hematol. 2019 May;109(5):539-544. doi: 10.1007/s12185-019-02616-y. Epub 2019 Mar 7.

    PubMed 30847774

Questions about this trial

What is this clinical trial studying?

A Early Phase 1 study testing Tagraxofusp and Pacritinib in people with Myelofibrosis，MF, run by University of Kansas Medical Center at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 19 inclusion and 32 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 6 May 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact KUCC Navigation (913-588-3671, kucc_navigation@kumc.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Spleen volume reduction by MRI or CT imaging, achieving ≥ 35% reduction in spleen volume imaging from baseline to week 24. (time frame: Baseline to up to 24 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06414681, which also lists 39 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 May 2026.

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Log in to keep searching

You’ve used your 3 free searches. AzTrials is free: log in or create a free account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in