Is participation in this Portland 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.

Familial Platelet Disorder With Associated Myeloid Malignancy Clinical Trial in Portland, OR

Q: Is NCT07844694 recruiting in Portland, OR?

Yes, NCT07844694 is currently recruiting participants at its Portland, OR location. This familial platelet disorder with associated myeloid malignancy clinical trial is actively enrolling qualified participants.

Q: How do I join the familial platelet disorder with associated myeloid malignancy clinical trial in Portland?

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

About This Familial Platelet Disorder With Associated Myeloid Malignancy Study in Portland

This phase I trial studies the safety and side effects of low-dose sirolimus in treating patients with RUNX1 familial platelet disorder (FPD). RUNX1-FPD is a rare inherited disorder with symptoms such as mild to moderately low platelet count, abnormal platelet function, and an increased risk of developing cancers like myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML). It is caused by changes (variants) in the RUNX1 gene that is passed down (inherited) from an affected parent. Sirolimus is typically given to help prevent organ rejection in patients receiving kidney transplants. However, sirolimus may also be able to prevent RUNX1-FPD from progressing to cancers like MDS and AML by targeting another protein called mTORC1. Sirolimus may be a safe treatment for patients with RUNX1-FPD.

Sponsor: OHSU Knight Cancer Institute

Who Can Participate

Inclusion Criteria

Patient has provided signed, informed consent before initiation of any study specific procedures

Aged ≥ 18 years at the time of signing the informed consent

Confirmed pathogenic/likely pathogenic (P/LP) germline RUNX1 variant per ClinGen Myeloid Malignancy Variant Curation Expert Panel (MM-VCEP) RUNX1-specific variant curation rules

Patient must be willing to provide a bone marrow sample at time of screening and at the end of treatment with sirolimus

Platelet count of ≥ 50,000/µL

Creatinine clearance (CrCl) of ≥ 60 mL/min; estimated using the Cockcroft Gault formula or measured by 24 hour urine collection. Exceptions will be allowed, at the discretion of the investigator, for cases of a concomitant medication that alters renal function

Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) \< 3 × upper limit of normal (ULN)

Total bilirubin \< 1.5 × ULN

Left ventricular ejection fraction (EF) \> 50% or at the discretion of the investigator

Exclusion Criteria

Known allergy to sirolimus

History of major bleeding events that are clinically relevant in the judgement of the investigator or non-major bleeding events that cannot be controlled using SOC practices

Any known history of lymphoma, myelodysplastic syndrome (MDS), or other hematologic malignancy using International Working Group criteria

Prior treatment with sirolimus or a rapalog, mTOR inhibitor, or B-cell-depleting therapy within 28 days before study day 1

Treatment with strong inhibitors of cytochrome P450 3A4 (CYP3A4; e.g., ketoconazole, voriconazole, itraconazole, erythromycin, telithromycin, and clarithromycin), strong inducers of CYP3A4 (e.g., rifampin and rifabutin), other drugs that could increase sirolimus blood concentrations (e.g., bromocriptine, cimetidine, cisapride, clotrimazole, danazol, diltiazem, fluconazole, letermovir, protease inhibitors \[e.g., ritonavir, indinavir, boceprevir, and telaprevir\], metoclopramide, nicardipine, troleandomycin, and verapamil), other drugs that could decrease sirolimus blood concentrations (e.g., carbamazepine, phenobarbital, phenytoin, rifapentine, St. John's Wort \[Hypericum perforatum\]), or drugs with blood concentrations that could increase (e.g., verapamil) within 7 days before study day 1, or at the discretion of the investigator

Use of cannabidiols that increase blood levels of sirolimus, within 7 days before study day 1 or at the discretion of the investigator

Myocardial infarction within 6 months before study day 1, congestive heart failure (New York Heart Association \> class II)

Total cholesterol \> 300 mg/dL or triglyceride \> 400 mg/dL

Arterial thrombosis (e.g., stroke or transient ischemic attack) within 6 months before study day 1

Infection requiring intravenous anti-infective treatment within 1 week of study day 1

Live vaccines (e.g., measles, mumps, rubella, oral polio, Bacillus Calmette-Guerin \[BCG\], yellow fever, varicella, and TY21a typhoid) within 28 days before study day 1

Known diagnosis of tuberculosis is exclusionary to provide protection against reactivation of infections by the immunosuppressive activity of the study drug sirolimus

The enrollment of a patient with a history of infection with hepatitis B virus (HBV) but with undetectable HBV deoxyribonucleic acid (DNA) by standard institutional testing can be considered after consultation with the investigator

Patients with history of chronic hepatitis C virus (HCV) must have documentation of completed curative standard of care (SOC) antiviral therapy

Patients with history of HIV infection must be well-managed with SOC antiretroviral therapy (highly active antiretroviral therapy \[HAART\])

Patients with history of Epstein-Barr may be considered after consultation with the treating physician

Participants who are pregnant may become pregnant, or who are breastfeeding

Because adequate and well-controlled studies of the impact of sirolimus on fetuses currently do not exist, study approved contraception must be used from start of study therapy to 12 weeks after the last dose of sirolimus

Adequate and well-controlled studies of infants fed breastmilk from patients undergoing sirolimus therapy do not exist and breastfeeding during study treatment is prohibited

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 Portland?

Yes, this clinical trial (NCT07844694) has an active research site in Portland, OR 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.

Familial Platelet Disorder With Associated Myeloid Malignancy Treatment Options in Portland, OR

If you're searching for familial platelet disorder with associated myeloid malignancy treatment options in Portland, OR, this clinical trial (NCT07844694) 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 Portland research site is actively enrolling participants for this phase 1 clinical trial. You'll receive care from experienced familial platelet disorder with associated myeloid malignancy 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 familial platelet disorder with associated myeloid malignancy clinical trials near you to find additional studies recruiting in your area.

Familial Platelet Disorder With Associated Myeloid Malignancy Clinical Trial in Portland, OR

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