Acute myeloid leukemia with mutated NPM1
MONDO:0044923An acute myeloid leukemia with mutation of the nucleophosmin gene. It is usually associated with normal karyotype and frequently has myelomonocytic or monocytic features. It usually responds to induction therapy.
Also known as: AML with mutated NPM1, AML, Mutation of the Nucleophosmin Gene, AML, NPM1 Mutation, AML, NPM1 gene mutation, AML, Nucleophosmin Gene Mutation, NPMc+ AML, acute myeloid leukaemia with cytoplasmic nucleophosmin, acute myeloid leukemia with cytoplasmic nucleophosmin
3105 clinical trials for this condition and its sub-types, 25 tagged with Acute myeloid leukemia with mutated NPM1 itself.
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Can a targeted pill boost standard therapy for hard-to-treat leukemia?
Disease control Recruiting nowThis phase 3 trial is testing whether adding revumenib, a drug that blocks a protein called menin, to the usual two-drug combination (azacitidine and venetoclax) can improve outcomes for adults newly diagnosed with acute myeloid leukemia (AML) that has a specific genetic change (…
Phase 3 • Sponsor: Stichting Hemato-Oncologie voor Volwassenen Nederland • Aim: Disease control
Last updated Sep 04, 2026 00:00 UTC
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New pill could boost leukemia treatment for patients with specific gene changes
Disease control Recruiting nowThis phase 3 trial tests whether adding the drug bleximenib to standard chemotherapy helps adults with a certain genetic type of acute myeloid leukemia (AML). About 875 people will get either bleximenib or a placebo alongside chemo, then be followed for 4 years. The goal is to se…
Phase 3 • Sponsor: Stichting Hemato-Oncologie voor Volwassenen Nederland • Aim: Disease control
Last updated Sep 03, 2026 00:00 UTC
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Promising new combo targets hard-to-treat leukemia mutations
Disease control Recruiting nowThis study tests whether adding the experimental drug bleximenib to standard treatment (venetoclax and azacitidine) helps people with a specific genetic subtype of acute myeloid leukemia (AML) achieve remission and live longer. The trial enrolls 600 adults aged 18 and older who h…
Phase 3 • Sponsor: Janssen Research & Development, LLC • Aim: Disease control
Last updated Aug 30, 2026 00:00 UTC
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New drug combo aims to beat tough leukemia
Disease control Recruiting nowThis phase 3 trial tests whether adding the drug revumenib to standard intensive chemotherapy helps people with newly diagnosed acute myeloid leukemia (AML) that has a specific genetic change (NPM1 mutation). About 468 participants will receive either revumenib or a placebo along…
Phase 3 • Sponsor: Syndax Pharmaceuticals • Aim: Disease control
Last updated Aug 30, 2026 00:00 UTC
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Can a targeted drug combo replace harsh chemo for NPM1 leukemia?
Disease control Recruiting nowThis trial tests whether a combination of venetoclax and azacitidine can work as well as or better than standard intensive chemotherapy for adults with newly diagnosed acute myeloid leukemia that has an NPM1 mutation. Participants are aged 18 to 70 and healthy enough for intensiv…
Phase 2 • Sponsor: Technische Universität Dresden • Aim: Disease control
Last updated Aug 29, 2026 00:00 UTC
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New pill targets Leukemia's genetic weakness in patients too sick for chemo
Disease control Recruiting nowThis study tests a drug called ziftomenib in 70 adults with acute myeloid leukemia (AML) who have specific gene changes (NPM1 or KMT2A) and cannot receive standard chemotherapy. Ziftomenib blocks a protein called menin that helps cancer cells grow. The goal is to see if the drug …
Phase 2 • Sponsor: Uma Borate • Aim: Disease control
Last updated Aug 29, 2026 00:00 UTC
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New combo therapy aims to improve AML treatment for patients with specific gene changes
Disease control Recruiting nowThis early-phase trial tests whether adding a menin inhibitor (SNDX-5613) to standard chemotherapy can better control acute myeloid leukemia (AML) in people with certain gene changes (NPM1 or MLL). About 38 newly diagnosed adults will receive the combination to find the safest do…
Phase 1 • Sponsor: National Cancer Institute (NCI) • Aim: Disease control
Last updated Aug 27, 2026 00:00 UTC
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New drug cocktail takes aim at Hard-to-Treat leukemia
Disease control Recruiting nowThis early-stage trial is testing a new drug called ziftomenib combined with standard chemotherapy or another targeted drug for people with relapsed or treatment-resistant acute myeloid leukemia (AML) that has specific genetic changes. The main goal is to see if these combination…
Phase 1 • Sponsor: Kura Oncology, Inc. • Aim: Disease control
Last updated Aug 22, 2026 00:00 UTC
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New hope for AML patients: targeted drug ziftomenib enters final testing phase
Disease control Recruiting nowThis large Phase 3 trial is testing whether adding the experimental drug ziftomenib to standard chemotherapy helps people with untreated acute myeloid leukemia (AML) who have specific genetic mutations. About 1,300 participants will be randomly assigned to receive either ziftomen…
Phase 3 • Sponsor: Kura Oncology, Inc. • Aim: Disease control
Last updated Aug 22, 2026 00:00 UTC
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Experimental leukemia drug made available for patients who have no other options
Disease control Expanded accessThis program provides expanded access to the experimental drug ziftomenib for adults with acute myeloid leukemia (AML) or acute lymphoblastic leukemia (ALL) that has specific genetic changes (KMT2A rearrangements or NPM1 mutations). It is for patients who have exhausted standard …
Sponsor: Kura Oncology, Inc. • Aim: Disease control
Last updated Jul 01, 2026 00:00 UTC
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New hope for japanese AML patients: experimental drug ziftomenib enters trial
Disease control Recruiting nowThis study tests an experimental drug called ziftomenib in 6 Japanese adults with a specific type of acute myeloid leukemia (AML) that has returned or not responded to treatment. The drug is taken as a pill once daily. The goal is to see if it can help control the disease and how…
Phase 2 • Sponsor: Kyowa Kirin Co., Ltd. • Aim: Disease control
Last updated Jun 27, 2026 13:00 UTC
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New combo therapy targets Hard-to-Cure leukemia
Disease control Recruiting nowThis early-phase study tests whether combining two targeted drugs, SNDX-5613 and gilteritinib, is safe and effective for adults with a specific type of acute myeloid leukemia (AML) that has returned or not responded to treatment. The drugs work by blocking different signals that …
Phase 1 • Sponsor: Uma Borate • Aim: Disease control
Last updated Jun 27, 2026 12:32 UTC
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Engineered immune cells take aim at Hard-to-Treat leukemia
Disease control Recruiting nowThis early-stage trial tests a new treatment for adults with a specific type of acute myeloid leukemia (AML) that has come back or not responded to standard therapy. The treatment involves taking a patient's own immune cells, modifying them in a lab to better recognize and attack…
Phase 1/2 • Sponsor: Miltenyi Biomedicine GmbH • Aim: Disease control
Last updated Jun 27, 2026 12:07 UTC
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New drug aims to keep leukemia away after transplant
Disease control Recruiting nowThis early-phase study tests an investigational drug called ziftomenib in adults with acute myeloid leukemia (AML) who have had a stem cell transplant. The goal is to find a safe dose and see if the drug can help prevent the cancer from coming back. About 22 participants will tak…
Phase 1 • Sponsor: Massachusetts General Hospital • Aim: Disease control
Last updated Jun 27, 2026 11:03 UTC
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New drug cocktail aims to tackle tough leukemia
Disease control Recruiting nowThis early-stage trial is testing an experimental drug called ziftomenib alongside standard treatments for acute myeloid leukemia (AML) in people with specific gene mutations. About 420 participants with newly diagnosed or relapsed AML will be assigned to one of three treatment a…
Phase 1 • Sponsor: Kura Oncology, Inc. • Aim: Disease control
Last updated Jun 27, 2026 11:01 UTC
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New drug aims to keep leukemia away after transplant
Disease control Recruiting nowThis early-phase trial tests a drug called revumenib in people with acute leukemia who have had a stem cell transplant. The drug targets a protein that helps leukemia cells grow. The study will check safety, find the best dose, and see if it can stop the cancer from coming back. …
Phase 1 • Sponsor: City of Hope Medical Center • Aim: Disease control
Last updated Jun 27, 2026 09:10 UTC
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New hope for kids with tough leukemia: targeted drug combo enters first human trial
Disease control Recruiting nowThis early-phase trial tests a new drug called ziftomenib, taken as a capsule, together with standard chemotherapy (cytarabine and fludarabine) for children and young adults up to age 21 who have a relapsed or refractory acute leukemia with specific genetic changes (KMT2A-r, NUP9…
Phase 1 • Sponsor: PedAL BCU, LLC • Aim: Disease control
Last updated Jun 27, 2026 07:55 UTC
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New study investigates why some AML patients face early bleeding or clotting risks
Knowledge-focused Recruiting nowThis observational study examines a specific group of patients with a type of acute myeloid leukemia (AML) that has an NPM1 mutation. Researchers want to see if a particular subset, called APL-like, is linked to early blood vessel problems like bleeding or clots. The study will l…
Sponsor: Gruppo Italiano Malattie EMatologiche dell'Adulto • Aim: Knowledge-focused
Last updated Jun 27, 2026 14:01 UTC
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Scientists hunt for clues to improve leukemia treatment after transplant
Knowledge-focused Recruiting nowThis study looks at blood cells from 20 adults with acute myeloid leukemia (AML) whose cancer returned after a stem cell transplant. All participants have specific genetic changes (NPM1 mutations or KMT2A rearrangements) and are being treated with a menin inhibitor. Researchers w…
Sponsor: University of Freiburg • Aim: Knowledge-focused
Last updated Jun 27, 2026 13:07 UTC