Is NCT06303193 recruiting in Bethesda, MD?

Yes, NCT06303193 is currently recruiting participants at its Bethesda, MD location. This myelodysplastic syndromes clinical trial is actively enrolling qualified participants.

How do I join the myelodysplastic syndromes clinical trial in Bethesda?

To join this clinical trial in Bethesda, complete the eligibility form on this page. A study coordinator will contact you to discuss next steps and determine if you qualify.

Is participation in this Bethesda clinical trial free?

Yes, all study-related care is provided at no cost to qualified participants. Many studies also offer compensation for time and travel expenses.

Myelodysplastic Syndromes Clinical Trial in Bethesda, MD

About This Myelodysplastic Syndromes Study in Bethesda

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. Treatments for these disorders are risky and not always effective. Better treatments are needed. Objective: To test a study drug (pacritinib) in adults and children with MDS or MDS/MPN. Eligibility: Children (aged 12 to 17 years) and adults (aged 18 years and older) with MDS or MDS/MPN. Design: Participants will be screened. They will have a physical exam with blood tests. They will have tests of their heart function. They may have a bone marrow biopsy: An area over the hip will be numbed; a needle will be inserted to remove a sample of soft tissue from inside the hipbone. Pacritinib is a capsule taken by mouth. All participants will take the study drug 2 times a day, every day, in 28-day cycles. They will write down the date and time they take each capsule. Doctors will assign varying dosages of the drug to different participants. Participants will have clinic visits each week during cycle 1; every 2 weeks during cycle 2; and gradually increasing to every 3 months after cycle 13. Treatment will continue for up to 8 years. Bone marrow biopsies, heart tests, and other tests will be repeated at intervals throughout the study. Participants will also fill out questionnaires about their quality of life, the symptoms of their disease, and other topics.

Sponsor: National Cancer Institute (NCI)

Who Can Participate

Inclusion Criteria

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 Hematology Laboratory, CC or by the Laboratory of Pathology, NCI as defined according to the 2016 WHO criteria, 2022 WHO criteria, or 2022 International Consensus Classification

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 with initial response to long-term treatment (secondary resistance) OR have intolerance to hypomethylating agents OR have a contraindication to hypomethylating agents

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 following characteristics:

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 and restless legs

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 transplantation will include participants who are not anticipated to be candidates to receive transplantation within the next 3 months due to medical comorbidities, lack of appropriate donor, or logistical barriers to transplant

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 which no known standard of care exists

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 \< 2 (Karnofsky \>= 60%)

For participants \< 16 years of age, Lansky \>= 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 \[IUD\], surgical) or two (2) effective forms of contraception (e.g., barrier method) while on study drug and for 30 days after the last dose of study drug

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

Exclusion Criteria

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 bleeding complications in the 3 months prior to enrollment

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. Prior use is allowed as long as medication is stopped two weeks prior to study drug initiation. Because the lists of these agents are constantly changing, it is important to regularly consult a frequently-updated medical reference. As part of the enrollment/informed consent procedures, the participant will be counseled on the risk of interactions with other agents, and what to do if new medications need to be prescribed or if the participant is considering a new over-the-counter medicine or herbal product.

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 HBV core antibody or surface antibody or HCV antibody); history of HIV, HBV, or HCV is allowed if there is no uncontrolled viral infection

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, or that could increase risk to the participant

Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility.

Frequently Asked Questions

Q:Is this study available in Bethesda?

Yes, this clinical trial (NCT06303193) has an active research site in Bethesda, MD that is currently enrolling participants.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. This study has been reviewed and approved, and participants are closely monitored by medical professionals. You can withdraw at any time.

Q:Will I be compensated?

Many clinical trials offer compensation for your time and travel expenses. Specific compensation details will be discussed during the screening process. All study-related medical care is provided at no cost.

Q:Can I leave the trial if I change my mind?

Absolutely. Participation is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty.

Still have questions? Our study coordinators are here to help.

Myelodysplastic Syndromes Treatment Options in Bethesda, MD

If you're searching for myelodysplastic syndromes treatment options in Bethesda, MD, this clinical trial (NCT06303193) may be an excellent opportunity. Clinical trials provide access to cutting-edge treatments that aren't yet available to the general public, often at no cost to participants.

Our Bethesda research site is actively enrolling participants for this phase 1/2 clinical trial. You'll receive care from experienced myelodysplastic syndromes specialists who are at the forefront of medical research. All study-related care, including examinations, treatments, and monitoring, is provided at no cost to qualified participants.

Looking for more options? Browse all myelodysplastic syndromes clinical trials near you to find additional studies recruiting in your area.

Myelodysplastic Syndromes Clinical Trial in Bethesda, MD

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