New drug combo aims to wipe out hidden leukemia cells
NCT ID NCT04216563
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This phase II trial tested adding asciminib (ABL001) to standard tyrosine kinase inhibitor therapy in 7 people with chronic myeloid leukemia who still had traces of cancer. The goal was to see if the combination could eliminate remaining cancer cells. The study was terminated early, so we have limited data on how well it works.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- asciminib (ABL001)
- What this could lead to
- If successful, this combination could help more CML patients achieve deep remission and possibly stop treatment temporarily.
- What could go wrong
- This was a very small, early-phase trial that was terminated early, so results are limited. The approach may not work for everyone and could have side effects.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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7 people
The number who actually took part.
- Started
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Jul 2020
- Finished
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Mar 2025
- Lead sponsor
-
Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Patients with a diagnosis of Philadelphia chromosome (Ph)- or BCR-ABL1-positive CML (as determined by cytogenetics, fluorescence in situ hybridization \[FISH\], or polymerase chain reaction \[PCR\]) * Patients should be receiving therapy with nilotinib or dasatinib, whether as initial therapy or after prior TKI, at a dose equal or lower than the standard dose * Patients must have received TKI therapy for at least 24 months and not have required dose reductions, escalations, discontinuation or re-initiation after discontinuation of TKI in the last 6 months * Patients must be in CCyR (by conventional karyotype or FISH, or BCR-ABL/ABL =\< 1% IS if no cytogenetic analysis available within 3 months) * Patients must have detectable BCR-ABL1 transcript levels meeting at least one of the following criteria: a. Patient has never achieved a MMR after 18 months of therapy with their current TKI, or b. Patient has not achieved MR4.5 after 36 months of therapy with their current TKI, or c. Patient has lost MMR or MR4.5 confirmed in at least two consecutive analyses separated by at least 1 month, or d. BCR-ABL1 transcript levels have reached a plateau defined as a ratio that is stable in a molecular response category (i.e., MMR, MR4 or MR4.5) in the last at least 12 months, with at least 3 values obtained during this period * Patients must not have had a known continuous interruption of TKI therapy of greater than 14 days or for a total of 6 weeks in the 6 months prior to enrollment, unless the interruption was for an accident, unrelated hospitalization or surgical procedure, or for a treatment-free remission attempt that was unsuccessful and required re-initiation of therapy * Eastern Cooperative Oncology Group (ECOG) performance status =\< 2 * Creatinine =\<1.5 x institutional upper limit of normal * Amylase and lipase values =\< 3.0 x institutional upper limit of normal * Alkaline phosphatase =\< 2.5 x institutional upper limit of normal unless considered to be not of hepatic origin * Aspartate aminotransferase (AST) (serum glutamic-oxaloacetic transaminase \[SGOT\])/alanine aminotransferase (ALT) (serum glutamate pyruvate transaminase \[SGPT\]) =\< 3 x institutional upper limit of normal * Total bilirubin =\< 1.5 x institutional upper limit of normal (=\< 3 x upper limit of normal in patients with known Gilbert's syndrome) * The effects of ABL001 on the developing human fetus are unknown. For this reason, women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation. Women of child-bearing potential must agree to use highly effective methods of contraception during dosing and for 30 days after study treatment. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately. Allowable methods of birth control: Total abstinence (when this is in line with the preferred and usual lifestyle of the subject). Periodic abstinence (e.g., calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are not acceptable methods of contraception. Female sterilization (have had surgical bilateral oophorectomy with or without hysterectomy), total hysterectomy, or tubal ligation at least six weeks before the start of study treatment. In case of oophorectomy alone, only when the reproductive status of the woman has been confirmed by follow up hormone level assessment. Male sterilization (at least 6 months prior to screening). The vasectomized male partner should be the sole partner for that subject. Use of oral, injected or implanted hormonal methods of contraception or placement of an intrauterine device (IUD) or intrauterine system (IUS), or other forms of hormonal contraception that have comparable efficacy (failure rate \<1%), for example hormone vaginal ring or transdermal hormone contraception. Sexually active males must use a condom during intercourse while taking the drug and for 30 days after stopping treatment and should not father a child in this period. A condom is required to be used also by vasectomized men in order to prevent delivery of the drug via seminal fluid Exclusion Criteria: * Patients with New York Heart Association (NYHA) class III or IV congestive heart failure or left ventricular ejection fraction (LVEF) \< 40% by echocardiogram or multigated acquisition (MUGA) scan * Patients with a history of myocardial infarction within the last 6 months or unstable/uncontrolled angina pectoris or history of severe and/or uncontrolled ventricular arrhythmias. Clinically significant cardiac arrhythmias (e.g., ventricular tachycardia), complete left bundle branch block, high-grade atrioventricular (AV) block (e.g., bifascicular block, Mobitz type II and third degree AV block) * Corrected QT interval (QTc) of \> 480 milliseconds (ms) on baseline electrocardiogram (ECG) (using corrected QT interval per institutional standard) * Long QT syndrome, family history of idiopathic sudden death or congenital long QT syndrome, or any of the following: a. Risk factors for Torsades de Pointes (TdP) including uncorrected hypokalemia or hypomagnesemia, history of cardiac failure, or history of clinically significant/symptomatic bradycardia. b. Concomitant medication(s) with a known risk to prolong the QT interval and/or known to cause Torsades de Pointes that cannot be discontinued or replaced 7 days prior to starting study drug by safe alternative medication * Patients with known infection with human immunodeficiency virus (HIV) or active hepatitis B or C * Patients with known conditions that would significantly affect the ingestion or gastrointestinal absorption of drugs administered orally * Nursing women, women of childbearing potential (WOCBP) with positive blood or urine pregnancy test, or women of childbearing potential who are not willing to maintain adequate contraception * History of acute pancreatitis within 1 year of study entry or past medical history of chronic pancreatitis * Absolute neutrophil count (ANC) \< 500/mm\^3 * Platelet count \< 50,000 mm\^3 * History of other active malignancy within 2 years prior to study entry with the exception of previous or concomitant basal cell skin cancer and previous carcinoma in situ treated curatively * Treatment with medications that meet one of the following criteria and that cannot be discontinued at least one week prior to the start of treatment with study treatment: Moderate or strong inducers of CYP3A. Moderate or strong inhibitors of CYP3A and/or P-glycoprotein (P-gp). Substrates of CYP3A4/5, CYP2C8, or CYP2C9 with narrow therapeutic index * Previous treatment with or known/ suspected hypersensitivity to ABL001 or any of its excipients * Subject has any other significant medical or psychiatric history that in the opinion of the investigator would adversely affect participation in this study
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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M D Anderson Cancer Center
Houston, Texas, 77030, United States