RecruitingPhase 1

CC-486 and Venetoclax for 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 to 100 Years
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

  • Procedures mentioned: 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
  • "Two bone marrow biopsies will be performed during the first cycle: day 4 or 5 (+2 days), and day 28."
  • "Bone marrow biopsies will occur on day 4 or 5 (+2) and on day 28 of cycle 1."
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 early study tests CC-486 (azacitidine taken by mouth) with venetoclax to find a dose to use together. It is for people with acute myeloid leukemia (AML), a blood cancer, that has come back or has not responded to treatment. It also includes newly diagnosed people who are unlikely to handle strong chemotherapy.

This is an open label, dose escalation Phase I single institution pilot study for relapsed and refractory AML patients using CC-486 (oral azacitidine) with venetoclax. At the completion of dose escalation and after establishment of the MTD or recommended dose of CC-486 with venetoclax, an expansion phase will commence, using venetoclax with the MTD of CC-486 in relapsed/refractory patients.

Compensation is read from the study's own description and may be missing — always confirm with the study team.

Eligibility

Who can join

  • You must have confirmed AML, but not the type called APL. You must have had at least one past treatment, or join the newly diagnosed group. Prior hydroxyurea or ATRA is allowed in that group.
  • If newly diagnosed, you must be unlikely to handle strong chemotherapy. You must be 75 or older, or younger with major limits on daily activity or certain heart, lung, kidney, or other health problems.
  • You must meet limits on how much help you need with daily activities: an ECOG activity score of 0–3 if under 75, or 0–2 if 75 or older.
  • You must be expected to live at least 12 weeks and meet kidney and liver test limits. Some liver test exceptions apply.
  • People who can have a stem cell transplant may join and may have the transplant at any time during the study.
  • If you can become pregnant, you must use birth control during treatment and for 6 months after. Men must follow birth control rules and not donate sperm through 90 days after treatment; some birth control exceptions apply.
  • You must sign a consent form before any study screening or procedures.
  • You must avoid grapefruit and grapefruit products, Seville oranges (including their marmalade), and star fruit starting 3 days before treatment and throughout the study.

Who can't join

  • AML is active in your brain or spinal cord.
  • You have known HIV, or hepatitis B or C with a detectable amount of virus.
  • You have a serious health problem that the study doctor thinks would make joining unsafe, including certain recent heart problems.
  • You have a gut condition or past surgery that prevents you from taking or absorbing the study drugs safely.
  • You have an infection that is not improving despite treatment.
  • You have a history of another cancer, unless it meets an allowed exception, such as certain treated early cancers, some skin or prostate cancers, or cancer treated with the goal of a cure.
  • Your white blood cell count is above 25 billion per liter, or you can receive strong starting chemotherapy and agree to it. Medicine or a blood-filtering procedure may lower your white blood cell count so you can qualify.
  • You are pregnant or breastfeeding, or have a known or suspected allergy to azacitidine or mannitol.

Simplified for readability. The official criteria below are what the study team uses.

Official eligibility criteria

Inclusion Criteria: 1. Subject must have confirmation of non-APL AML by WHO criteria46 and have undergone at least one line of therapy (dose escalation and dose expansion R/R cohorts), or have had no prior lines of therapy (newly diagnosed cohort) Prior treatment with hydroxyurea or ATRA is allowed in the newly diagnosed cohort. 2. For the newly diagnosed cohort, subjects must be unlikely to tolerate standard intensive chemotherapy due to age, performance status, or comorbidities based on at least one of the following criteria: a. age ≥75 years old OR b. age \< 75 years old with at least one of the following: i. ECOG performance status of 3 ii. Cardiac history of CHF or documented EF ≤50% iii. pulmonary disease with DLCO ≤65% or FEV1 ≤65% iv. creatinine clearance ≥30 mL/min to \< 45 mL/min based on the CKD-EPI Creatinine Equation (2021). https://www.kidney.org/content/ckd-epi-creatinine-equation-2021 v. any other comorbidity that the investigator judges to be incompatible with intensive chemotherapy 3. Patients must have ECOG of 0 to 3 (if \< 75 years old) or 0 to 2 (if ≥75 years old) 4. Transplant eligible patients can participate in the study and they are allowed to proceed with stem cell transplantation at any time during the study. 5. Subject must have a projected life expectancy of at least 12 weeks. 6. Subject must have adequate renal function as demonstrated by a calculated creatinine clearance ≥ 30 mL/min; determined via urine collection for 24-hour creatinine clearance or by the Cockcroft Gault formula. 7. Subject must have adequate liver function as demonstrated by: 1. aspartate aminotransferase (AST) ≤ 3.0 × ULN\* 2. alanine aminotransferase (ALT) ≤ 3.0 × ULN\* 3. bilirubin ≤ 1.5 × ULN, unless due to Gilbert's syndrome\* \* Unless considered due to leukemic organ involvement 8. Non-sterile male subjects must use contraceptive methods with partner(s) at least prior to beginning study drug administration and continuing up to 90 days after the last dose of study drug. Male subjects must agree to refrain from sperm donation from initial study drug administration until 90 days after the last dose of study drug. No contraception is required if male subjects are surgically sterile (vasectomy with medical assessment confirming surgical success) or if the male subject has a female partner who is postmenopausal or permanently sterile (bilateral oophorectomy, bilateral salpingectomy or hysterectomy). 9. Female subjects must be either: 1. Postmenopausal; defined as Age \> 60 years with no menses for 12 or more months without an alternative medical cause; OR 2. Permanently surgically sterile (bilateral oophorectomy, bilateral salpingectomy or hysterectomy); OR 3. If subject is of childbearing potential, use of contraception is required while on study treatment and for 6 months after the last dose. 10. Subject must voluntarily sign and date an informed consent, approved by an Institutional Review Board (IRB), prior to the initiation of any research directed screening or procedures. 11. Subject is informed that consumption of the following fruits is prohibited 3 days prior to the initiation of study treatment and throughout participation: grapefruit, grapefruit products, Seville oranges (including marmalade containing Seville oranges) or Star fruit. Exclusion criteria: 1. Subject has known active CNS involvement from AML. 2. Subject is known to be positive for HIV. HIV testing is not required. 3. Subject is known to be positive for hepatitis B or C infection with the exception of those with an undetectable viral load. Hepatitis B or C testing is not required and subjects with serologic evidence of prior vaccination to HBV (i.e., HBs Ag, anti-HBs+ and anti-HBc-) may participate. 4. Subject has any history of clinically significant condition(s) that in the opinion of the investigator would adversely affect his/her participating in this study including, but not limited to: 1. Significant active cardiac disease within the previous 6 months including: New York Heart Association heart failure \> class 2, unstable angina, or myocardial infarction. 2. Renal, neurologic, psychiatric, endocrinologic, metabolic, immunologic, hepatic, cardiovascular disease, or bleeding disorder independent of leukemia. 5. Subject has a malabsorption syndrome or other condition that precludes enteral route of administration. This includes history of inflammatory bowel disease (e.g. Crohn's disease, ulcerative colitis), celiac disease (e.g. sprue), prior gastrectomy or upper bowel removal, or any other gastrointestinal disorder or defect that would interfere with the absorption, distribution, metabolism or excretion of the study drug and/or predispose the subject to an increased risk of gastrointestinal toxicity. 6. Subject exhibits evidence of uncontrolled systemic infection requiring therapy (viral, bacterial or fungal). Uncontrolled is defined as ongoing signs/symptoms related the infection without improvement despite appropriate antibiotics, antiviral therapy and/or other treatment. 7. Subject has a history of other malignancies prior to study entry, with the exception of: 1. Adequately treated in situ carcinoma of the breast or cervix uteri 2. Basal cell carcinoma of the skin or localized squamous cell carcinoma of the skin 3. Prostate cancer not requiring therapy beyond hormonal therapy 4. Previous malignancy confined and surgically resected (or treated with other modalities) with curative intent 8. Subject has a white blood cell count \> 25 × 109/L. Note: hydroxyurea or apheresis are permitted to meet this criterion. 9. Any subject who is a candidate for intensive induction therapy and agrees to receive this therapy. 10. Pregnant or breast-feeding females. A pregnancy test will be obtained at the time of screening. 11. Known or suspected hypersensitivity to azacitidine or mannitol.

Study sites (2)

CO