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New treatment option for Myelodysplastic Syndromes
Official title Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic/Myeloproliferative Neoplasms
ClinicalTrials.gov ID: NCT06303193
What this study is testing
What is pacritinib?
pacritinib is an investigational medicine, given as a pill taken by mouth, being studied as a potential treatment for myelodysplastic syndromes.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- Background: Myelodysplastic syndrome (MDS) and myelodysplastic/myeloproliferative neoplasm (MDS/MPN) are blood disorders that can cause serious complications in children and adults. MDS and MDS/MPN can also progress to acute myeloid leukemia.
- Phase 2: a mid-size study of how well it works
- Time commitment: about 8 years
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 12 to 120
You may be able to join if
- Participants must have histologically or cytologically confirmed MDS or MDS/MPN, including therapy-related MDS or MDS/MPN, and MDS or MDS/MPN with...
- Age 12-17 years for phase I and age \>= 18 years for phase II
- Participants \>= 18 years of age with HR-MDS must have resistance to hypomethylating agents as defined as failure to show improvement after at least...
- Participants \>= 18 years of age with LR-MDS must be refractory to or ineligible to receive standard of care therapies, i.e...
- Severe neutropenia defined by absolute neutrophils count \<=0.5(SqrRoot) 10\^9/L without the use of granulocyte colony-stimulating factors
You likely can't join if
- Participants with platelet transfusion-refractory thrombocytopenia, with inability to keep platelet threshold above 10 K/mcL with transfusions
- Participants with evidence of ongoing hemorrhage, active signs/symptoms of bleeding, or history of severe (grade \>= 3) unprovoked bleeding...
- Use of anti-platelet or anticoagulant medication other than low-dose aspirin (100 mg daily or less) in the 14 days prior to enrollment, or any...
- Participants who are unwilling to accept blood transfusions
- Participants with ANC \< 500 cells/mcL AND hospitalization for a fungal infection in the 12 months prior to enrollment
- History of allergic reactions attributed to compounds of similar chemical or biologic composition to pacritinib
See the full eligibility criteria
Who can join
- Participants must have histologically or cytologically confirmed MDS or MDS/MPN, including therapy-related MDS or MDS/MPN, and MDS or MDS/MPN with germline predisposition, by the Department of Laboratory Medicine...
- Age 12-17 years for phase I and age \>= 18 years for phase II
- Participants \>= 18 years of age with HR-MDS must have resistance to hypomethylating agents as defined as failure to show improvement after at least 4 cycles of treatment (primary resistance) or relapse in participants...
- Participants \>= 18 years of age with LR-MDS must be refractory to or ineligible to receive standard of care therapies, i.e. erythropoietin-stimulating agents, lenalidomide, luspatercept, and present with one of the...
- Severe neutropenia defined by absolute neutrophils count \<=0.5(SqrRoot) 10\^9/L without the use of granulocyte colony-stimulating factors
- Symptomatic anemia defined by hemoglobin 16-week average \<10 g/dL and symptoms that may include fatigue, weakness, reduced exercise tolerance, dyspnea on exertion, palpitations, (orthostatic) hypotension, near syncope...
- Thrombocytopenia defined as platelets \<20(SqrRoot) 10\^9/L or platelets \<50(SqrRoot) 10\^9/L and a history of clinically relevant non-major or major bleeding according to the ISTH classification
- Participants 12-17 years of age with MDS must be relapsed/refractory OR ineligible to receive immunosuppressive therapy and hematopoietic stem cell transplantation --Ineligibility to receive hematopoietic stem cell...
- Participants with MDS/MPN must be relapsed/refractory (failed a minimum of 1 standard of care therapy) OR ineligible to receive standard of care OR without known life-prolonging therapy options OR have a diagnosis for...
- Participants 12-17 years of age must weigh \>= 35 kg
- If any of the prior therapies noted below were given to the participant, they must have been completed within the following timeframes:
- 7 days from last dose of short-acting myeloid growth factors (i.e., filgrastim) and \>= 14 days for long-acting (i.e., pegfilgrastim)
- 14 days from last dose of short-acting thrombopoietic growth factors (i.e.,eltrombopag) and \>= 28 days for long-acting (i.e., romiplostim)
- 14 days or 5 pharmacokinetic half-lives from biological therapy agent
- 21 days from myelosuppressive chemotherapy
- 28 days from last dose of immunosuppressive therapy (e.g., ATG, cyclosporine, steroids greater than physiologic replacement)
- 28 days from last dose of lenalidomide
- 28 days from last dose of venetoclax
- 28 days from any other investigational agent
- 42 days from last dose of erythropoiesis stimulating agents
- 56 days from last dose of luspatercept
- 100 days from stem cell transplant with no evidence of active graft vs. host disease in participants who relapsed following transplant
- 150 days from total body irradiation
- Performance status:
- For participants \>= 16 years of age, ECOG performance status \ = 60%)
- For participants \ = 60%
- Participants must have adequate organ function as defined below:
- Total bilirubin: \<= 1.5 X institutional upper limit of normal OR \<= 3 x institutional upper limit of normal in participants with Gilbert s syndrome
- AST(SGOT)/ALT(SGPT): \<= 2.5 X institutional upper limit of normal
- Creatinine clearance: \>= 50 mL/min/1.73 m\^2 for participants with creatinine levels above institutional normal
- PT and PTT: \<= 1.5 X institutional upper limit of normal, except in the setting of PTT elevation due to lupus anticoagulant, in which case these participants would be exempt from meeting this inclusion criterion
- Individuals of child-bearing potential (IOCBP) and individuals able to father a child with a partner able to become pregnant must agree to use one (1) highly effective form of contraception (e.g., intrauterine device...
- Nursing participants must discontinue breastfeeding and/or not begin breastfeeding until 2 weeks after the last dose of study drug
- Ability of participant or parent/guardian (for participants 12-17 only) to understand and the willingness to sign a written informed consent document
What rules you out
- Participants with platelet transfusion-refractory thrombocytopenia, with inability to keep platelet threshold above 10 K/mcL with transfusions
- Participants with evidence of ongoing hemorrhage, active signs/symptoms of bleeding, or history of severe (grade \>= 3) unprovoked bleeding complications in the one year prior to enrollment, or any unprovoked grade 2...
- Use of anti-platelet or anticoagulant medication other than low-dose aspirin (100 mg daily or less) in the 14 days prior to enrollment, or any ongoing requirement for these medications
- Participants who are unwilling to accept blood transfusions
- Participants with ANC \< 500 cells/mcL AND hospitalization for a fungal infection in the 12 months prior to enrollment
- History of allergic reactions attributed to compounds of similar chemical or biologic composition to pacritinib
- Concomitant administration with sensitive substrates/narrow therapeutic index drugs of CYP3A4, CYP1A2, P-gp BCRP, and OCT1 should be avoided. Concurrent use of strong inhibitors and inducers of CYP3A4 are not allowed...
- Concomitant administration of medications with significant potential to cause QTc prolongation.
- Participants with the following cardiac conditions at screening:
- Symptomatic congestive heart failure
- Unstable angina pectoris
- Uncontrolled cardiac dysrhythmias
- QTc(F) prolongation \>450 ms, or other factors that increase the risk for QT prolongation (i.e., heart failure, or a history of long QT interval syndrome)
- Grade \>= 3 cardiac complication in the 6 months prior to enrollment
- Left ventricular ejection fraction \<= 50% by transthoracic echocardiogram (TTE) at screening
- Participants with any active, uncontrolled viral, bacterial, or fungal infection, including active HIV-1, Hepatitis B (HBV) and/or Hepatitis C (HCV) infection (positive HBV or HCV viral load in the setting of positive...
- Pregnancy
- Presence of another known cause of cytopenia or dysplastic marrow that is untreated and may limit interpretation of results
- Uncontrolled intercurrent illness or any significant disease, evaluated by history, physical exam and chemistries or social situations that may limit interpretation of results, limit compliance with study requirements...
The study team makes the final eligibility decision.
Where it's taking place
- Bethesda, Maryland, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The study runs about 8 years per participant, based on its public description. The team confirms the exact schedule and number of visits before you enroll.
Who can join this trial?
This study is enrolling all sexes, 12 years to 120 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Bethesda, Maryland, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.