RecruitingPhase 3

Studies to Assess Ziftomenib in Combination With Ven+Aza or 7+3 in Patients With Untreated NPM1-m or KMT2A-r AML

Phase 3 Randomized, Double-blind, Placebo-controlled Studies Assessing Ziftomenib in Combination With Either Standard of Care Nonintensive (Venetoclax+Azacitidine) or Intensive (7+3) Therapy in Patients With Untreated NPM1 Mutated or KMT2A Rearranged 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

Not stated in the study description — ask the study team about visits and procedures.

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

These two studies test the benefits and risks of adding ziftomenib, an experimental drug, to standard treatment for acute myeloid leukemia (AML), a blood cancer. They are for adults with untreated AML and certain gene changes. A doctor chooses the study based on the patient's health, and patients receive either ziftomenib or a placebo, which has no active drug.

Ziftomenib is an investigational drug in development for the treatment of patients with acute myeloid leukemia (AML) with eligible genetic alterations. Ziftomenib is a type of therapy known to target the menin pathway in cancer cells. This protocol has 2 separate studies that will investigate the benefits and risks of adding ziftomenib to standard-of-care (SOC) AML treatments in patients with certain genetic mutations who have not received any treatment for their AML. In the first study, the Nonintensive Therapy Study, older patients or those with serious medical problems will receive the SOC therapies venetoclax (ven) and azacitidine (aza), plus either ziftomenib or a placebo. In the second study, the Intensive Therapy Study, medically fit patients will receive (a) the SOC therapies cytarabine and daunorubicin, plus either ziftomenib or a placebo during a first treatment phase called induction, (b) cytarabine plus either ziftomenib or a placebo during a second treatment phase called consolidation, and (c) ziftomenib or a placebo during a third treatment phase called maintenance. The physician will determine which study is the appropriate treatment for the patient, but neither the patient nor their physician will know whether the patient has been assigned to receive ziftomenib or a placebo. This design is called "double-blinded".

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 have AML.
  • Be able to care for yourself and be out of bed for at least half of your waking hours.
  • Have liver and kidney function that meets the study's rules.
  • If you can become pregnant, agree to use birth control through 180 days after the last study dose. Men with partners who can become pregnant must use birth control or avoid sex through 90 days after the last dose.
  • For the less intensive study: have an NPM1 gene change. Be age 75 or older, or younger than 75 with limits on daily activity or certain heart, kidney, or liver problems.
  • For the intensive study: have an NPM1 gene change or a KMT2A gene rearrangement. A KMT2A change called a partial tandem duplication does not qualify.
  • For the intensive study: have no FLT3 gene change, an FLT3-ITD ratio below 0.05, or a medical or treatment-related reason not to receive FLT3-targeted drugs. Lack of access to these drugs does not qualify.
  • For the intensive study: be healthy enough for intensive treatment, as judged by the study doctor. Your heart must pump out at least 50% of its blood with each beat.

Who can't join

  • Past AML treatment, except hydroxyurea or leukapheresis, a procedure that removes white blood cells.
  • Certain other cancers: acute promyelocytic leukemia, chronic myeloid leukemia in its blast phase, or isolated myeloid sarcoma. A known BCR-ABL gene change also rules you out.
  • Another active cancer, with exceptions for certain skin cancers, cancers treated locally with the aim of a cure, or breast or prostate cancer receiving hormone treatment after main treatment.
  • AML in the brain or spinal cord.
  • Signs that white blood cells are blocking blood flow, or a white blood cell count above 25 billion per liter before starting ziftomenib or placebo. Treatment to lower this count is allowed.
  • Uncontrolled HIV, active hepatitis B or C, or another uncontrolled infection.
  • Another uncontrolled illness, including certain heart problems.
  • Pregnancy or breastfeeding.

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

Official eligibility criteria

Key Inclusion Criteria: The following criteria apply to both the Nonintensive Therapy Study and the Intensive Therapy Study unless otherwise noted: * Age ≥18 years at time of signing the informed consent form. * Diagnosis of AML per the 2022 WHO Classification of Hematolymphoid Tumors (5th Edition). * Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2. * Adequate liver and kidney function according to protocol requirements. * A female of childbearing potential must agree to use adequate contraception from the time of screening through 180 days following the last dose of study intervention. A male with a female partner of childbearing potential must agree to use abstinence or adequate contraception from the time of screening through 90 days following the last dose of study intervention. * NONINTENSIVE THERAPY STUDY ONLY (VEN+AZA): 1. Documented NPM1-m. 2. Patients considered ineligible for Intensive Therapy defined by the following: * i. Age ≥75, OR * ii. Age \<75 with an ECOG performance status of 2 or cardiac, renal, or hepatic impairment per protocol criteria. * INTENSIVE THERAPY STUDY ONLY (7+3): 1. Documented NPM1-m or KMT2A-r (KMT2A-r patients with a partial tandem duplication are not eligible). 2. Documented FLT3 wild-type or ITD ratio \<0.05 OR ineligible to receive FLT3-targeted therapy (medically ineligible or mutation in which FLT3 inhibition is not SOC). Lack of access to an FLT3 inhibitor is not considered "ineligible" for FLT3-targeted therapy. 3. Ejection fraction of ≥50%. 4. Fit for Intensive Therapy per Investigator opinion. Key Exclusion Criteria: * Prior therapy for AML (except hydroxyurea or leukapheresis for WBC control). * Diagnosis of acute promyelocytic leukemia (APL), blast phase chronic myeloid leukemia, or isolated myeloid sarcoma. * Known history of BCR-ABL mutation. * History of other active concurrent malignancies prior to study entry except: 1. Basal cell skin cancer or localized squamous cell cancer of the skin 2. Previous malignancy confined and locally resected (or treated with other modalities) with curative intent 3. Prostate or breast cancer receiving adjuvant hormonal therapy. * Active central nervous system (CNS) involvement by AML. * Clinical signs/symptoms of leukostasis or white blood cells (WBC) \>25×10\^9/L prior to start of ziftomenib/placebo. Note: Hydroxyurea and/or leukapheresis are permitted to meet this criterion. * Known uncontrolled HIV infection or known active hepatitis B virus, hepatitis C virus infection, or other uncontrolled infection. * Uncontrolled intercurrent illness including but not limited to, cardiac illness as defined in the protocol. * Women who are pregnant or lactating.

Study sites (44)

AZ

CA

CO

CT

FL

IA

KS

KY

MA

MI

MN

NC

NJ

NM

NY

OH

OR

PA

TN

TX

VA

VT

WI

WV