MyelofibrosisPhase 2NCT07447817
Selinexor and Pacritinib in JAK Inhibitor-naïve MF Patients With Cytopenias
A Phase 2 study testing Pacritinib and Selinexor in people with Myelofibrosis and Post-polycythemia Vera Myelofibrosis, run by John Mascarenhas at 3 sites in United States.
Trial summary
This is a phase II, multicenter, open-label trial evaluating the safety and efficacy of pacritinib and selinexor in JAK inhibitor naïve patients with anemia and thrombocytopenia. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myelofibrosis, Post-polycythemia Vera Myelofibrosis, Post-essential Thrombocythemia Myelofibrosis
- What is being tested?
- Pacritinib and Selinexor
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 26 participants
- Where
- 3 sitesUnited States
- Study dates
- 23 Oct 2026to 24 May 2030
- Phase
- Phase 2
- 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
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Adults ≥ 18 years of age capable of providing informed consent
- Pathologically confirmed diagnosis of PMF, post-ET MF, or post-PV MF as per the World Health Organization (WHO) diagnostic criteria - Intermediate-1, Intermediate-2, or High-Risk disease by the Dynamic International Prognostic Scoring System (DIPSS)
- Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2
- Baseline splenomegaly ≥ 5cm palpable below the left costal margin and in the midclavicular line OR ≥ 450cc by imaging (i.e. ultrasound, CT, MRI)
- Baseline anemia, defined by hemoglobin < 10 g/dL within 28 days prior to Cycle 1 Day 1
- Baseline thrombocytopenia, defined by platelet count 50-150 x 109/L without platelet transfusions within 28 days prior to Cycle 1 Day 1
Show all 20Show fewer
- Adequate organ function as demonstrated by the following within 28 days prior to Cycle 1 Day 1:
- ALT (SGPT) and/or AST (SGOT) ≤ 3x the upper limit of normal (ULN), or ≤ 4 x ULN if, upon judgment of the treating physician, it is believed to be due to MF-related extramedullary hematopoiesis (EMH);
- Direct bilirubin ≤ 1.5 x ULN; or ≤ 2x ULN if, upon judgment of the treating physician, it is believed to be due to MF-related extramedullary hematopoiesis (EMH) or documented Gilbert's syndrome;
- Creatinine clearance ≥ 30 mL/min;
- Prothrombin time (PT) and activated partial thromboplastin time (aPTT) ≤ 1.5 × ULN (Exceptions to coagulation parameters may be considered for patients who are taking concomitant anticoagulation medications or have a documented anti-phospholipid antibody, after discussion with Study Chair approval)
- Bone marrow and/or peripheral blood blast count < 5%; and
- Absolute neutrophil count (ANC) ≥ 1500 mm3 without need for growth factors within 7 days prior to Cycle 1 Day 1.
- Recovery to ≤ Grade 1 or baseline of any toxicities due to prior systemic treatments, excluding alopecia
- Life expectancy of at least six months
- Patients with active hepatitis B virus are eligible if antiviral therapy for hepatitis B has been given for >8 weeks and the viral load is <100 IU/mL.
- Patients with history of hepatitis C virus (HCV) are eligible if they have received adequate curative anti-HCV treatment and HCV viral load is below the limit of quantification.
- Patients with history of human immunodeficiency virus are eligible if they have cluster of differentiation (CD)4+ T-cell counts ≥350 cells/μL, negative viral load, and no history of acquired immunodeficiency syndrome (AIDS)-defining opportunistic infections in the last year and should be on established antiretroviral therapy for at least 4 weeks.
- Women of childbearing potential (WOCBP) and men must agree to use adequate contraception prior to study entry, for the duration of study participation, and for 90 days following completion of therapy. Men must agree to use a condom and not father a child or donate sperm for the duration of the study and for 90 days after the last dose of study therapy
- Able to adhere to the study visit schedule and all protocol requirements
Who cannot join
- Prior treatment with Janus kinase (JAK) inhibitors
- Prior treatment with selinexor or other Exportin 1 (XPO1) inhibitors
- Treatment with any MF-directed therapy (including investigational therapies and excluding hydroxyurea) within 2 weeks or 5.5 half-lives, whichever is shorter, of Cycle 1 Day 1
- Completed hematopoietic cell transplant (HCT)
- Prior splenectomy, splenic irradiation, or splenic artery embolization within 6 months of Cycle 1 Day 1
- Impairment of gastrointestinal (GI) function or GI disease that could significantly alter the absorption of pacritinib or selinexor, including any unresolved nausea, vomiting, or diarrhea > National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) v6.0 Grade 1
Show all 20Show fewer
- Uncontrolled or currently progressing ocular toxicities
- Moderate or severe cardiovascular disease meeting one or both of the below criteria:
- Presence of cardiac disease, including a myocardial infarction within 6 months prior to study entry, unstable angina pectoris, New York Heart Association Class III/IV congestive heart failure, or uncontrolled hypertension
- Documented major electrocardiogram (ECG) abnormalities (not responding to medical treatments)
- Grade 2 or greater bleeding event within the past 6 months
- QT corrected by the Fridericia method (QTcF) prolongation > 480 ms or other factors that increase the risk for QT interval prolongation (eg, hypokalemia [defined as serum potassium < 3.0 mEq/L that is persistent and refractory to correction], or history of long QT interval syndrome)
- Recipient of organ transplant
- History of major surgery or any planned surgical procedures within 28 days prior to Cycle 1 Day 1
- Other malignancy within the last three years, other than curatively treated basal cell or squamous cell skin cancer, carcinoma in situ of the cervix, organ-confined or treated non-metastatic prostate cancer with normal prostate-specific antigen, in situ breast carcinoma after complete surgical resection, or superficial/non-invasive transitional cell bladder carcinoma.
- Presence of active serious infection
- Use of any prohibited medications (5.8) within two weeks or five half-lives, whichever is longer, prior to Cycle 1 Day 1
- Women who are pregnant or lactating
- Any serious, unstable medical or psychiatric condition that would prevent (as judged by the Investigator) the subject from signing the informed consent form (ICF) or any condition, including the presence of laboratory abnormalities, which places the subject at unacceptable risk if they were to participate in the study or confounds the ability to interpret data from the study
- Is or has an immediate family member (e.g., spouse, parent/legal guardian, sibling, or child) who is investigational site or sponsor staff directly involved with this trial, unless prospective IRB approval (by chair or designee) is given allowing exception to this criterion for a specific subject
Trial locations and contacts
3 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States3 sites, 3 recruiting
- Recruiting
Icahn School of Medicine at Mount Sinai
New York, New York, 10029
John Mascarenhas
- Not yet recruiting
The University of Kansas Cancer Center-Westwood
Westwood, Kansas, 66205
Abdulraheem Yacoub
- Not yet recruiting
Wake Forest Baptist Health Comprehensive Cancer Center
Winston-Salem, North Carolina, 27157
Ruben Mesa
About this study
Official title: Investigator-Initiated, Open-Label, Phase II Trial of Selinexor in Combination With Pacritinib in Patients With Myelofibrosis Who Are JAK Inhibitor-Naïve and Have Cytopenias (ILLUMINATE)
This is a phase II, multicenter, open-label trial evaluating the safety and efficacy of pacritinib and selinexor in JAK inhibitor naïve patients with anemia and thrombocytopenia.
Read the detailed description
This Phase II, multicenter, open-label will evaluate the safety and efficacy of pacritinib and selinexor in JAK inhibitor naïve patients with anemia and thrombocytopenia. Participants will receive pacritinib monotherapy for 1 cycle (4 weeks) and will be assessed for qualification to proceed to combination therapy at Cycle 2 Day 1. Participants who qualify for combination therapy will receiving pacritinib and selinexor for the remaining cycles. Participants who do not qualify at Cycle 2 Day 1 will have weekly assessments and count checks. If they meet criteria for combination therapy at any point, selinexor will be added onto pacritinib. Participants who do not qualify for combination therapy by Cycle 4 Day 1 will complete 6 cycles of pacritinib monotherapy, complete primary endpoint assessments after 24 weeks, and discontinue study participation. Participants receiving combination therapy must demonstrate clinical benefit at Cycle 7 Day 1 (24 weeks) to continue receiving therapy until loss of response, disease progression, unacceptable toxicity, participant/physician withdrawal, or the study is terminated.
- Conditions
- Myelofibrosis, Post-polycythemia Vera Myelofibrosis, Post-essential Thrombocythemia Myelofibrosis
- Being tested
- Pacritinib, Selinexor
- Keywords
- Myelofibrosis, Anemia, Thrombocytopenia, Pacritinib, Selinexor
- Medical subject headings
- Primary Myelofibrosis, Anemia, Thrombocytopenia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PacritinibDrug
Also called VONJO
Pacritinib 200mg twice a day (BID) by mouth (PO)
SelinexorDrug
Also called KPT-330; XPOVIO
Selinexor 60mg once weekly (QW) by mouth (PO)
| Study group | Role | What they receive |
|---|---|---|
| Pacritinib/Selinexor | Experimental | Study participants will receive pacritinib monotherapy (200mg BID orally) for the first cycle (28 days). If they qualify to add on selinexor at any point from Cycle 2 Day 1 to Cycle 4 Day 1, participants will have selinexor (60mg QW orally) added onto their pacritinib regimen. Receives: Drug: Pacritinib; Drug: Selinexor |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in spleen volume
Evaluate clinical efficacy of pacritinib and selinexor in myelofibrosis patients who are JAK inhibitor-naïve and have anemia and thrombocytopenia by assessing spleen volume reduction (SVR) of 35% or greater compared to baseline as measured by imaging (MRI/CT)
Measured: Baseline and 24 weeks
Secondary outcomes (10)
Spleen response rate
Spleen response rate (by palpation) after 6 and 12 cycles; spleen response is defined as baseline splenomegaly palpable at 5-10cm that becomes not palpable, or baseline splenomegaly of >10cm that decreases by ≥ 50%, as measured by palpation
Measured: At 24 weeks and 48 weeks
Change in the Myelofibrosis-Symptom Assessment Form Total Symptom Score (MF-SAF TSS)
Evaluate change in patient reported outcomes of treatment
Change in MF-SAF TSS from baseline after 6 and 12 cycles; symptom response will be determined in participants with baseline MF-SAF TSS ≥ 10.
The MF-SAF TSS comprises 7 items measuring MF-related symptoms. Each item is scored on a scale ranging from 0 (absent) to 10 (worst imaginable) and the items are summed for a total score.
The Mf-SAF TSS is the summation of all the individual scores (0-70 scale). Symptoms response requires ≥50% reduction in the MPN-SAF TSS and will be evaluable only in patients with a baseline TSS >= 10.
Measured: At 24 weeks and 48 weeks
Change in Patient Global Impression of Change (PGIC)
Patient Global Impression of Change (PGIC) response after 6 and 12 cycles; PGIC response is defined as "minimally improved" or better
The Patient Global Impression of Change (PGIC) will be used to assess self-reported belief about the efficacy of treatment. PGIC is a 7-point scale depicting a patient's rating of overall improvement. Patients rate their change as "very much improved", "much improved", "minimally improved", "no change", "minimally worse", "much worse", or "very much worse.
Total scale from 1-7, with higher score indicating poorer health outcomes.
Measured: At 24 weeks and 48 weeks
Anemia response
Anemia response after 6 and 12 cycles; anemia response is determined according to revised 2024 International Working Group-European LeukemiaNet (IWG-ELN) criteria and includes major response rate and minor response rate for transfusion-dependent anemia.
Major response: No transfusions for 12 weeks alongside a 12-week average hemoglobin increase of 150g/L
Minor response: A 50% or more reduction in transfusions and failure to meet major response thresholds.
Measured: At 24 weeks and 48 weeks
Change in hemoglobin level
Hemoglobin is the protein molecule in red blood cells that carries oxygen from the lungs to the body's tissues and returns carbon dioxide from the tissues back to the lungs.
Measured: Continuously across all cycles of treatment, up to 48 weeks
Change in platelet count
Platelets are part of the blood cells. Platelets are a part of the blood clotting system and help in preventing bleeding.
Measured: Continuously across all cycles of treatment, up to 48 weeks
Progression-free survival
Progression-free survival (PFS) is defined as time from first treatment to disease progression by IWG-ELN criteria
Measured: At time of disease progression or at 48 weeks, whichever occurs first
Overall survival
Overall survival (OS) is defined as time from first treatment to death due to any cause or censored at last follow-up
Measured: At time of death or censored at last follow-up, at 48 weeks
Number of participants achieving RBC transfusion independence)
The number of participants achieving transfusion independence will be divided by the number of participants who were transfusion-dependent at baseline
Measured: at 24 weeks and at 48 weeks
Number of participants achieving platelet transfusion independence
The number of participants achieving transfusion independence will be divided by the number of participants who were transfusion-dependent at baseline
Measured: at 24 weeks and at 48 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Mar 2026
- Study start23 Oct 2026 (estimated)
- Primary completion24 May 2030 (estimated)
- Study completion24 May 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 26 participants (estimated)
Sponsor and investigators
- Lead sponsor
- John Mascarenhas
- Sponsor type
- University, hospital or other
- Collaborators
- Sobi, Inc., Karyopharm Therapeutics Inc, National Cancer Institute (NCI)
- Responsible party
- Sponsor-investigator, John Mascarenhas, Icahn School of Medicine at Mount Sinai
- Investigators
- John Mascarenhas, MD, Study chair, Icahn School of Medicine at Mount SinaiAbdulraheem Yacoub, MD, Study chair, University of Kansas School of MedicineRuben Mesa, MD, FACP, Study chair, Atrium Health Wake Forest Baptist Comprehensive Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07447817
- Sponsor's protocol ID
- STUDY-26-00174
- Other IDs
- 5P01CA108671, MPN-RC 126
- Status last verified
- Oct 2026
- First submitted
- 25 Feb 2026
- Last updated
- 27 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: The completed dataset is the sole property of the Sponsor-Investigator's institution and should not be exported to third parties, except for authorized representatives of appropriate Health/Regulatory Authorities, without permission from the Sponsor-investigator and their institution.
Study at a glance
From the registry record, last updated by the sponsor on 27 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Investigator-Initiated, Open-Label, Phase II Trial of Selinexor in Combination With Pacritinib in Patients With Myelofibrosis Who Are JAK Inhibitor-Naïve and Have Cytopenias (ILLUMINATE)
- ClinicalTrials.gov ID
- NCT07447817
- Acronym / sponsor ID
- STUDY-26-00174
- Conditions
- Myelofibrosis, Post-polycythemia Vera Myelofibrosis, Post-essential Thrombocythemia Myelofibrosis
- Keywords
- Myelofibrosis, Anemia, Thrombocytopenia, Pacritinib, Selinexor
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 26 participants (estimated)
- Lead sponsor
- John MascarenhasUniversity, hospital or other
- Collaborators
- Sobi, Inc., Karyopharm Therapeutics Inc, National Cancer Institute (NCI)
- Study start
- 23 Oct 2026 (estimated)
- Primary completion
- 24 May 2030 (estimated)
- Study completion
- 24 May 2030 (estimated)
- First posted
- 4 Mar 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a phase II, multicenter, open-label trial evaluating the safety and efficacy of pacritinib and selinexor in JAK inhibitor naïve patients with anemia and thrombocytopenia.
Detailed description
This Phase II, multicenter, open-label will evaluate the safety and efficacy of pacritinib and selinexor in JAK inhibitor naïve patients with anemia and thrombocytopenia. Participants will receive pacritinib monotherapy for 1 cycle (4 weeks) and will be assessed for qualification to proceed to combination therapy at Cycle 2 Day 1. Participants who qualify for combination therapy will receiving pacritinib and selinexor for the remaining cycles. Participants who do not qualify at Cycle 2 Day 1 will have weekly assessments and count checks. If they meet criteria for combination therapy at any point, selinexor will be added onto pacritinib. Participants who do not qualify for combination therapy by Cycle 4 Day 1 will complete 6 cycles of pacritinib monotherapy, complete primary endpoint assessments after 24 weeks, and discontinue study participation. Participants receiving combination therapy must demonstrate clinical benefit at Cycle 7 Day 1 (24 weeks) to continue receiving therapy until loss of response, disease progression, unacceptable toxicity, participant/physician withdrawal, or the study is terminated.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Pacritinib/Selinexor | Experimental | Drug: Pacritinib, Drug: SelinexorStudy participants will receive pacritinib monotherapy (200mg BID orally) for the first cycle (28 days). If they qualify to add on selinexor at any point from Cycle 2 Day 1 to Cycle 4 Day 1, participants will have selinexor (60mg QW orally) added onto their pacritinib regimen. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (20 listed)
- Adults ≥ 18 years of age capable of providing informed consent
- Pathologically confirmed diagnosis of PMF, post-ET MF, or post-PV MF as per the World Health Organization (WHO) diagnostic criteria - Intermediate-1, Intermediate-2, or High-Risk disease by the Dynamic International Prognostic Scoring System (DIPSS)
- Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2
- Baseline splenomegaly ≥ 5cm palpable below the left costal margin and in the midclavicular line OR ≥ 450cc by imaging (i.e. ultrasound, CT, MRI)
- Baseline anemia, defined by hemoglobin < 10 g/dL within 28 days prior to Cycle 1 Day 1
Key exclusion criteria (20 listed)
- Prior treatment with Janus kinase (JAK) inhibitors
- Prior treatment with selinexor or other Exportin 1 (XPO1) inhibitors
- Treatment with any MF-directed therapy (including investigational therapies and excluding hydroxyurea) within 2 weeks or 5.5 half-lives, whichever is shorter, of Cycle 1 Day 1
- Completed hematopoietic cell transplant (HCT)
- Prior splenectomy, splenic irradiation, or splenic artery embolization within 6 months of Cycle 1 Day 1
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PacritinibAlso: VONJOPacritinib 200mg twice a day (BID) by mouth (PO) | Drug | Pacritinib/Selinexor |
| SelinexorAlso: KPT-330; XPOVIOSelinexor 60mg once weekly (QW) by mouth (PO) | Drug | Pacritinib/Selinexor |
Primary outcome
Change in spleen volume
Time frame: Baseline and 24 weeks
Evaluate clinical efficacy of pacritinib and selinexor in myelofibrosis patients who are JAK inhibitor-naïve and have anemia and thrombocytopenia by assessing spleen volume reduction (SVR) of 35% or greater compared to baseline as measured by imaging (MRI/CT)
10 secondary outcomes
Spleen response rate
Time frame: At 24 weeks and 48 weeks
Change in the Myelofibrosis-Symptom Assessment Form Total Symptom Score (MF-SAF TSS)
Time frame: At 24 weeks and 48 weeks
Change in Patient Global Impression of Change (PGIC)
Time frame: At 24 weeks and 48 weeks
Anemia response
Time frame: At 24 weeks and 48 weeks
Change in hemoglobin level
Time frame: Continuously across all cycles of treatment, up to 48 weeks
Change in platelet count
Time frame: Continuously across all cycles of treatment, up to 48 weeks
Progression-free survival
Time frame: At time of disease progression or at 48 weeks, whichever occurs first
Overall survival
Time frame: At time of death or censored at last follow-up, at 48 weeks
Number of participants achieving RBC transfusion independence)
Time frame: at 24 weeks and at 48 weeks
Number of participants achieving platelet transfusion independence
Time frame: at 24 weeks and at 48 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Oct 2026.
- Individual participant dataSharing planned: No. The completed dataset is the sole property of the Sponsor-Investigator's institution and should not be exported to third parties, except for authorized representatives of appropriate Health/Regulatory Authorities, without permission from the Sponsor-investigator and their institution.
Related trials for Myelofibrosis
RecruitingPhase 3
A Phase 3 Study of Pelabresib (DAK539) and Ruxolitinib in Myelofibrosis (MF)
Primary Myelofibrosis (PMF), Post-polycythemia Vera Myelofibrosis (PPV-MF)
RecruitingPhase 3
A Study to Compare Elritercept to Placebo in Adults With Myelofibrosis and Anemia Who Are Taking Ruxolitinib
Myelofibrosis, Anemia
RecruitingObservational study
Motor Development and Early Predictors of Psychomotor Outcomes in Preterm and Term Infants Assessed by MOS-R and Caregiver Questionnaire at 18 and 36 Months
Neurodevelopmental Disorders, Motor Skills Disorders
Active, not recruitingPhase 3
Study of Selinexor in Combination With Ruxolitinib in Myelofibrosis
Myelofibrosis
RecruitingPhase 2
Study of Momelotinib in Combination With Luspatercept in Participants With Transfusion Dependent Myelofibrosis
Primary Myelofibrosis, Myelofibrosis; Primary Myelofibrosis; Post-polycythemia Vera Myelofibrosis; Post-essential Thrombocythemia Myelofibrosis
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
John Mascarenhas, MD study chair
Icahn School of Medicine at Mount Sinai
Abdulraheem Yacoub, MD study chair
University of Kansas School of Medicine
Ruben Mesa, MD, FACP study chair
Atrium Health Wake Forest Baptist Comprehensive Cancer Center
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Pacritinib and Selinexor in people with Myelofibrosis and Post-polycythemia Vera Myelofibrosis, run by John Mascarenhas at 3 sites 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 20 inclusion and 20 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 27 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites 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?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 Gillian Sanchez ((917) 581-1774, gillian.sanchez@mssm.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in spleen volume (time frame: Baseline and 24 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 27 Oct 2026.