Ruxolitinib in Combination With Venetoclax With and Without Azacitidine in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia
Phase I Study to Evaluate Safety of Ruxolitinib in Combination With Azacitidine + Venetoclax in Patients With Relapsed/Refractory Acute Myeloid Leukemia
Who can join
As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.
- Age
- 18 Years and older
- Sex
- All sexes
- Healthy volunteers
- Not accepted
- Compensation
- Not stated
Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.
What to expect
No one can promise a trial will help you personally — that is what the research is trying to find out.
What participation involves
- Time periods mentioned: 2 years
- Procedures mentioned: Blood draws, Biopsies
Only what the study's summary and detailed description on ClinicalTrials.gov say. Time periods may describe the whole study or a part of it — ask the study team what applies to you.
Show the study's own words
- "Patients also undergo a skin punch biopsy and echocardiography (ECHO) at screening and blood sample collection and bone marrow aspiration and biopsy throughout the study."
- "Cycles repeat every 28 days for up to 2 years in the absence of disease progression or unacceptable toxicity."
- "Patients also undergo ECHO at screening and blood sample collection and bone marrow aspiration and biopsy throughout the study."
- "After completion of study treatment, patients are followed up every 6 months for up to 2 years."
- Compensation
- Not stated
- Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
- Participants are monitored closely by the research team throughout the study.
- Some participants may receive access to an investigational treatment, which is still being studied.
- Your participation helps researchers learn whether this approach could help future patients.
About this study
In plain language
This study tests the side effects and best dose of ruxolitinib with venetoclax, and studies drug combinations that include azacitidine. It is for adults with acute myeloid leukemia (AML), a blood cancer, that came back or did not respond to treatment. Some adults with myelodysplastic syndrome (MDS), a bone marrow disease, may also qualify.
This phase I trial studies the side effects and best dose of ruxolitinib when given together with venetoclax and compares the effect of ruxolitinib in combination with venetoclax to venetoclax and azacitidine in treating patients with acute myeloid leukemia (AML) that has come back (relapsed) or has not responded to treatment (refractory). Ruxolitinib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Azacitidine stops cells from making deoxyribonucleic acid and may kill cancer cells. It is a type of antimetabolite. Venetoclax is in a class of medications called B-cell lymphoma-2 (BCL-2) inhibitors. It may stop the growth of cancer cells by blocking Bcl-2, a protein needed for cancer cell survival. Giving ruxolitinib in combination with venetoclax and azacitidine may be safe, tolerable, and/or effective compared to ruxolitinib with venetoclax in treating patients with relapsed or refractory AML.
Compensation is read from the study's own description and may be missing — always confirm with the study team.
Eligibility
Who can join
- Be age 18 or older and able to understand and sign a consent form.
- Have AML that came back or did not respond after at least one AML treatment. Prior venetoclax and azacitidine are allowed; hydroxyurea alone does not count as a prior treatment.
- People with MDS may qualify if it worsened during treatment with a hypomethylating drug and they cannot receive strong chemotherapy because of their health or ability to do daily tasks.
- Be able to care for yourself and be out of bed for at least half of your waking hours.
- Be able to swallow and absorb medicines taken by mouth.
- Meet the study’s kidney and liver blood test limits. Some liver test limits may not apply if leukemia caused the abnormal results.
- If you can become pregnant, have a negative pregnancy test within 14 days before starting study drugs.
- Agree to use birth control during treatment and afterward: for 120 days after the last ruxolitinib dose in Arm 1, or 6 months after the last azacitidine dose in Arm 2.
Who can't join
- Have acute promyelocytic leukemia, a type of AML, or active AML in the brain or spinal cord.
- Have very fast-growing disease, organ problems, or a white blood cell count above 25,000 that hydroxyurea cannot control.
- Have serious heart, breathing, blood-clotting, or liver problems; or have had a stroke, heart attack, or bleeding inside the skull within 2 months.
- Have untreated HIV, active hepatitis C, chronic hepatitis B, a history of tuberculosis, or an infection that is not well controlled. A specific hepatitis B test exception may apply.
- Have another active cancer with expected survival under 1 year, or serious graft-versus-host disease, where donated cells attack your body. Active disease needing new or stronger treatment during screening also excludes you.
- Have had recent cancer treatment or major surgery. Waiting times vary by treatment, and some exceptions may apply.
- Cannot stop or adjust medicines that interact with the study drugs, have a known sensitivity to a study drug, or are unwilling to receive blood products.
- Are breastfeeding during the study.
Simplified for readability. The official criteria below are what the study team uses.
Official eligibility criteria
Inclusion Criteria: * Ability to understand and the willingness to sign a written informed consent document * Age \>= 18 years at time of informed consent. Persons of all genders and gender identities, and members of all races and ethnic groups will be included * Morphologically documented relapsed/refractory (R/R) AML or R/R secondary AML (sAML) that has progressed after at least 1 prior therapy for AML * Prior treatment with venetoclax and azacitidine is allowed * Treatment with hydroxyurea will not be considered a line of therapy * Patients with morphologically documented myelodysplastic syndrome (MDS) that has progressed on hypomethylating agent (HMA) therapy also will be considered if the patient is ineligible for induction with intensive chemotherapy (IC), defined for this study as meeting one or more of the following criteria: * Severe cardiac disorder (e.g., congestive heart failure requiring treatment, left ventricular ejection fraction (LVEF) of ≤ 50%, or chronic stable angina) * Severe pulmonary disorder, certified by the managing physician * Creatinine clearance of \< 45 ml/min or * Hepatic disorder with total bilirubin \> 1.5 x upper limit of normal (ULN) * Eastern Cooperative Oncology Group (ECOG) equal to 2 * Other comorbidity(ies) judged to be incompatible with high dose chemotherapy by the managing physician will be considered, at the discretion of the principal investigator (PI) * ECOG performance status 0 to 2 * Persons of childbearing potential (PCBP) must have a negative serum or urine pregnancy test within 14 days prior to start of study drug administration * Patients must agree to use an adequate method of contraception while on study treatment and for 120 days after the last dose of ruxolitinib for Arm 1 and 6 months after the last dose of azacitidine for Arm 2 * Must be able to take and absorb oral medications * Creatinine clearance ≥ 30 mL/min; calculated by the Cockcroft Gault formula or measured by 24 hour urine collection * Total serum bilirubin ≤ 1.5 x ULN unless thought to be due to leukemic involvement * Serum aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) ≤ 3.0 x ULN unless thought to be due to leukemic involvement Exclusion Criteria: * Diagnosis of acute promyelocytic leukemia (APL or AML M3 subtype) * Active central nervous system involvement with AML * Chemotherapy or therapy with a non-investigational agent other than a biologic intended to within 1 week of the planned start of study therapy, with the exception of hydroxyurea for cytoreduction of proliferative disease, or at the discretion of the principal investigator (PI) * Therapy with a non-biologic investigational agent within 14 days or 5 half lives, whichever is longer, of the planned start of study therapy, or for the period recommended by the institution's research pharmacy service, or at the discretion of the PI * Therapy with a biologic investigational or non-investigational agent (e.g., monoclonal antibody) within 30 days of the planned start of study therapy, or for the period recommended by the institution's research pharmacy service, or at the discretion of the PI * Concurrent active malignancy with expected survival of less than 1 year, at the discretion of the investigator. For example, candidates with treated skin cancers, prostate cancer, breast cancer, etc. without metastatic disease are candidates for therapy since their expected survival exceeds that of relapsed or refractory AML * Clinically significant graft versus host disease (GVHD) or active GVHD requiring initiation or escalation of treatment within 28-day screening period * Participants with rapidly progressive disease (defined by blast count doubles within 48 hours) or organ dysfunction * Documented cardiac insufficiency (e.g., symptomatic heart failure, left ventricular ejection fraction of ≤ 40%) * Symptomatic shortness of breath or patient requires supplemental oxygen support * Clinically significant coagulation abnormality, such as disseminated intravascular coagulation * Known history of cerebrovascular accident, myocardial infarction, or intracranial hemorrhage within 2 months of enrollment * Known clinically significant liver disease defined as ongoing drug-induced liver injury, chronic active hepatitis C (hepatitis C virus \[HCV\]), chronic active hepatitis B (hepatitis B virus \[HBV\]), alcoholic liver disease, non-alcoholic steatohepatitis, primary biliary cirrhosis, extrahepatic obstruction caused by cholelithiasis, cirrhosis of the liver, portal hypertension, or history of autoimmune hepatitis * Untreated HIV or active hepatitis C detectable by polymerase chain reaction (PCR), or chronic hepatitis B (patients positive for hepatitis B core antibody who are receiving intravenous immunoglobulin therapy \[IVIG\] are eligible if hepatitis B \[HepB\] PCR is negative) * Per PI discretion, active infection that is not well controlled by antibacterial or antiviral therapy \*Patients with a known history of tuberculosis (TB; Mycobacterium tuberculosis) are not eligible for participation. At investigator discretion, latent TB test should be performed for individuals considered to be at high-risk (e.g., immune compromised, persons that have traveled to, or emigrated from, regions with high rates of TB) * Clinically significant surgery within 2 weeks of enrollment * Unwillingness to receive infusion of blood products * Requires use of medications interact with study drug and that cannot be terminated or adjusted. Use of the following therapies requires review by the sponsor investigator: * Strong and moderate CYP3A inhibitors * Strong and Moderate CYP3A inducers * Patients with uncontrolled white blood cell (WBC) count (defined as \> 25 K/mm\^3 and not controlled with hydroxyurea) * Patients with known sensitivity to ruxolitinib, venetoclax, or azacitidine * Since it is unknown whether ruxolitinib, venetoclax, or azacitidine (or their metabolites) are excreted in human milk and because of the potential for serious adverse reactions in the nursing infant, breastfeeding concurrent with study participation is prohibited
Study sites (3)
OH
- Ohio State University Comprehensive Cancer Center · Columbus, OH 43210Uma Borate, MD · Study contact · 614-293-9869 · uma.borate@osumc.eduUma Borate, MD · Principal investigatorMore trials in Columbus
OR
- OHSU Knight Cancer Institute · Portland, OR 97239Jennifer N. Saultz, MD · Study contact · 503-494-5566 · saultzje@ohsu.eduJennifer N. Saultz, MD · Principal investigatorMore trials in Portland
TX
- UT Southwestern/Simmons Cancer Center-Dallas · Dallas, TX 75390Meredith Pogue · Study contact · 214-648-5919 · Meredith.Pogue@UTSouthwestern.eduYazan Madanat, MD · Principal investigatorMore trials in Dallas