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Leukemia drug holiday: study tests if patients can pause nilotinib

NCT ID NCT01784068

Disease control Sponsor: Novartis Pharmaceuticals Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 24, 2026 · Last updated Aug 14, 2026 · Updated 4 times

Summary

This study looked at whether people with chronic myeloid leukemia (CML) who have done very well on the drug nilotinib can safely stop taking it. 215 patients who had been on nilotinib for at least two years and had very low levels of cancer cells were enrolled. The main goal was to see how many could stay in remission without the drug for 48 weeks. The results help doctors understand who might be able to take a break from treatment.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
nilotinib
What this could lead to
If successful, this could allow some CML patients to stop daily medication and remain in remission, reducing long-term drug side effects and improving quality of life.
What could go wrong
This is a phase 2 study with 215 patients, so results are not definitive. Not all patients maintained remission, and those who relapsed needed to restart treatment. Long-term safety of stopping therapy is still being studied.

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

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

215 people

The number who actually took part.

Started

Mar 2013

Finished

Jan 2025

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 100 years

Sex

Anyone

Healthy volunteers

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

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Male or female patients ≥ 18 years of age * Minimum of 2 calendar years of nilotinib treatment with at least the last 12 months of nilotinib treatment prior to pre-screening at approved total daily dose of 600 mg BID or at a reduced dose of 400 mg QD if required from the perspective of tolerance for BCR-ABL positive CML in documented chronic phase at the time of diagnosis * Evidence of typical BCR-ABL transcripts (b3a2 and/or b2a2) at the time of CML-CP diagnosis i.e. prior to first start of TKI treatment which are amenable to standardized RT-PCR quantification" * Patient in MR4.5 at prescreening at Novartis designated lab * ECOG performance status of 0-2 * Adequate end organ function as defined by: * Direct bilirubin ≤ 1.5 x ULN except for i) patients with documented Gilbert's syndrome for whom any bilirubin value is allowed and ii) for patients with asymptomatic hyperbilirubinemia (liver transaminases and alkaline phosphatase within normal range). * SGOT(AST) and SGPT(ALT) ≤ 3 x ULN i.e. equivalent to ≤ Grade 1 NCI-CTCAE v.4.03 * Serum lipase ≤ 2 x ULN i.e. equivalent to ≤ Grade 2 NCI-CTCAE v.4.03 * Alkaline phosphatase ≤ 2.5 x ULN * Serum creatinine \< 1.5 x ULN * Patients must have the following electrolyte values within normal limits or corrected to be within normal limits with supplements prior to first dose of study medication: * Potassium (suggested keep to prevent issues with QT and/or rhythm abnormalities) * Magnesium (suggested keep to prevent issues with QT and/or rhythm abnormalities) * Total calcium (corrected for serum albumin) * Patients must have normal marrow function as defined: * Absolute Neutrophil Count (ANC) ≥ 1.5 x 10E9/L * Hemoglobin ≥ 9.0 g/dL * Platelets ≥ 100 x 10E9/L * Documented chronic phase CML must meet all the criteria defined by: * \< 15% blasts in peripheral blood and bone marrow, * \< 30% blasts plus promyelocytes in peripheral blood and bone marrow, * \< 20% basophils in the peripheral blood, * ≥ 100 x 109/L (≥ 100,000/mm3) platelets, * No evidence of extramedullary leukemic involvement, with the exception of hepatosplenomegaly * Patients must tolerate a minimum total daily dose of nilotinib of 400 mg Exclusion Criteria: * Previous treatment with BCR-ABL inhibitors other than nilotinib for more than a total cumulative duration of 4 weeks * Previous treatment with alpha-interferon of any duration * Previous anticancer agents for CML other than nilotinib except for cytoreduction after CML diagnosis until up to 4 weeks after first dose of nilotinib * Known second chronic phase of CML after previous progression to AP/BC * Poorly controlled diabetes mellitus (defined as HbA1c \> 9%) * Impaired cardiac function including any one of the following: * LVEF \< 45% or below the institutional lower limit of the normal range (whichever is higher) * Inability to determine the QT interval on ECG, except for patients with evidence of measurable QT interval at the time of CML diagnosis (e.g. prior to first start of TKI treatment) and who have no documented clinical signs of cardiovascular disease and/or clinical signs of conduction abnormality. * Complete left bundle branch block * Right bundle branch block plus left anterior or posterior hemiblock * Use of a ventricular-paced pacemaker * Congenital long QT syndrome or a known family history of long QT syndrome * History of or presence of clinically significant ventricular or atrial tachyarrhythmias * Clinically significant resting bradycardia * QTc \> 450 msec on the average of three serial baseline ECG (using the QTcF formula). If QTcF \> 450 msec and electrolytes are not within normal ranges, electrolytes should be corrected and then the patient re-tested for QTc.This exclusion criterion is not applicable for patients with non-measurable QT interval who have evidence of measurable QT interval at the time of CML diagnosis (e.g. prior to first start of TKI treatment) and who have no documented clinical signs of cardiovascular disease and/or clinical signs of conduction abnormality. * History or clinical signs of myocardial infarction within 1 year of study entry * History of unstable angina within 1 year of study entry * Other clinically significant heart disease (e.g. congestive heart failure, cardiomyopathy or uncontrolled hypertension) * History of acute pancreatitis within 1 year of study entry or past medical history of chronic pancreatitis * Known presence of significant congenital or acquired bleeding disorder unrelated to cancer * Severe and/or uncontrolled concurrent medical disease that in the opinion of the investigator could cause unacceptable safety risks or compromise compliance with the protocol (e.g. uncontrolled diabetes, uncontrolled infection) * History of another active malignancy within 5 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 other investigational agents (defined as not used in accordance with the approved indication) within 4 weeks of Day 1 * Patients who have not recovered from prior surgery * Patients actively receiving therapy with strong CYP3A4 inhibitors and/or inducers, and the treatment cannot be either discontinued or switched to a different medication prior to starting study drug. * Patients actively receiving therapy with herbal medicines that are strong CYP3A4 inhibitors and/or inducers, and the treatment cannot be either discontinued or switched to a different medication prior to starting study drug. These herbal medicines may include Echinacea, (including E. purpurea, E. angustifolia and E. pallida), Piperine, Artemisinin, St. John's Wort, and Ginkgo. * Patients who are currently receiving treatment with any medications that have the potential to prolong the QT interval and the treatment cannot be either safely discontinued or switched to a different medication prior to starting study drug. (see http://www.torsades.org/medical-pros/drug-lists/printable-drug-list.cfm for a list of agents that prolong the QT interval) * Impairment of gastrointestinal (GI) function or GI disease that may significantly alter the absorption of study drug (e.g. ulcerative disease, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome, small bowel resection, or gastric bypass surgery) * Pregnant or nursing (lactating) women, where pregnancy is defined as the state of a female after conception and until the termination of gestation, confirmed by a positive hCG laboratory test. * Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using highly effective methods of contraception during the study and for 14 days after the final dose of nilotinib. Highly effective contraception is defined as either: * Total abstinence (when this is in line with the preferred and usual lifestyle of the patient. 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) or tubal ligation at least six weeks before taking 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 enrolling). For female patients on the study the vasectomized male partner should be the sole partner for that patient. * Use of a combination of any two of the following: 1. Use of oral, injected or implanted hormonal methods of contraception or other forms of hormonal contraception that have comparable efficacy (failure rate \<1%), for example hormone vaginal ring or transdermal hormone contraception. 2. Placement of an intrauterine device (IUD) or intrauterine system (IUS) 3. Barrier methods of contraception: Condom or Occlusive cap (diaphragm or cervical/vault caps) with spermicidal foam/gel/film/cream/vaginal suppository. In case of use of oral contraception women should have been stable on the same pill for a minimum of 3 months before taking study treatment Women are considered post-menopausal and not of child bearing potential if they have had 12 months of natural (spontaneous) amenorrhea with an appropriate clinical profile (e.g. age appropriate, history of vasomotor symptoms) or have had surgical bilateral oophorectomy (with or without hysterectomy) or tubal ligation at least six weeks prior to enrolling. In the case of oophorectomy alone, only when the reproductive status of the woman has been confirmed by follow up hormone level assessment is she considered not of child bearing potential. If a study patient becomes pregnant or suspects being pregnant during the study or within 30 days after the final dose of nilotinib, the Study Doctor needs to be informed immediately and ongoing study treatment with nilotinib has to be stopped immediately.

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Conditions

The condition(s) this trial relates to.

chronic myelogenous leukemia, BCR-ABL1 positive Leukemia, Myelogenous, Chronic, BCR-ABL Positive

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Chronic myelogenous leukemia

Contacts and locations

Locations

  • Cancer Center of Kansas

    Wichita, Kansas, 67214-3728, United States

  • Cancer Centers of the Carolinas

    Greenville, South Carolina, 29605, United States

  • Community Cancer Trials of Utah

    Ogden, Utah, 84405, United States

  • Dana Farber Cancer Institute

    Boston, Massachusetts, 02215, United States

  • Florida Cancer Specialists

    Fort Myers, Florida, 33901, United States

  • H Lee Moffitt Cancer Center and Research Institute

    Tampa, Florida, 33612, United States

  • Lakes Research

    Miami Lakes, Florida, 33014, United States

  • Memorial Sloan Kettering

    New York, New York, 10017, United States

  • Novartis Investigative Site

    Buenos Aires, C1114AAN, Argentina

  • Novartis Investigative Site

    Graz, 8036, Austria

  • Novartis Investigative Site

    Rankweil, A-6830, Austria

  • Novartis Investigative Site

    Salzburg, 5020, Austria

  • Novartis Investigative Site

    Vienna, 1140, Austria

  • Novartis Investigative Site

    Vienna, A-1130, Austria

  • Novartis Investigative Site

    Sint-Niklaas, Oost Vlaanderen, 9100, Belgium

  • Novartis Investigative Site

    Kortrijk, West-Vlaanderen, 8500, Belgium

  • Novartis Investigative Site

    Brussels, 1090, Belgium

  • Novartis Investigative Site

    Brussels, 1200, Belgium

  • Novartis Investigative Site

    Charleroi, 6000, Belgium

  • Novartis Investigative Site

    Ghent, 9000, Belgium

  • Novartis Investigative Site

    Liège, 4000, Belgium

  • Novartis Investigative Site

    Varna, 9000, Bulgaria

  • Novartis Investigative Site

    Bogota, Cundinamarca, 111411, Colombia

  • Novartis Investigative Site

    Montería, 230004, Colombia

  • Novartis Investigative Site

    Aarhus N, 8200, Denmark

  • Novartis Investigative Site

    Bayonne, Bayonne Cedex, 64109, France

  • Novartis Investigative Site

    Saint Priest Jarez, Pays de la Loire Region, 42270, France

  • Novartis Investigative Site

    Bordeaux, 33076, France

  • Novartis Investigative Site

    Brest, 29609, France

  • Novartis Investigative Site

    Corbeil-Essonnes, 91100, France

  • Novartis Investigative Site

    Dunkirk, 59240, France

  • Novartis Investigative Site

    Grenoble, 38043, France

  • Novartis Investigative Site

    Nantes, 44093, France

  • Novartis Investigative Site

    Rouen, 76038, France

  • Novartis Investigative Site

    Strasbourg, 67000, France

  • Novartis Investigative Site

    Strasbourg, 67085, France

  • Novartis Investigative Site

    Toulouse, 31059, France

  • Novartis Investigative Site

    Freiburg im Breisgau, Baden-Wurttemberg, 79106, Germany

  • Novartis Investigative Site

    Mannheim, Baden-Wurttemberg, 68305, Germany

  • Novartis Investigative Site

    Frankfurt am Main, Hesse, 60590, Germany

  • Novartis Investigative Site

    Düsseldorf, North Rhine-Westphalia, 40225, Germany

  • Novartis Investigative Site

    Leipzig, Saxony, 04103, Germany

  • Novartis Investigative Site

    Lübeck, Schleswig-Holstein, 23563, Germany

  • Novartis Investigative Site

    Jena, Thuringia, 07740, Germany

  • Novartis Investigative Site

    Aachen, 52074, Germany

  • Novartis Investigative Site

    Bayreuth, 95445, Germany

  • Novartis Investigative Site

    Berlin, 13353, Germany

  • Novartis Investigative Site

    Bonn, 53105, Germany

  • Novartis Investigative Site

    Bottrop, 46236, Germany

  • Novartis Investigative Site

    Dresden, 01307, Germany

  • Novartis Investigative Site

    Düsseldorf, 40479, Germany

  • Novartis Investigative Site

    Goslar, 38642, Germany

  • Novartis Investigative Site

    Hamburg, 20246, Germany

  • Novartis Investigative Site

    Hamburg, 22417, Germany

  • Novartis Investigative Site

    Magdeburg, 39104, Germany

  • Novartis Investigative Site

    Mainz, 55131, Germany

  • Novartis Investigative Site

    Stuttgart, 70376, Germany

  • Novartis Investigative Site

    Ulm, 89081, Germany

  • Novartis Investigative Site

    Athens, 106 76, Greece

  • Novartis Investigative Site

    Athens, 115 27, Greece

  • Novartis Investigative Site

    Budapest, H-1083, Hungary

  • Novartis Investigative Site

    Szeged, 6725, Hungary

  • Novartis Investigative Site

    Dublin, D03 VX82, Ireland

  • Novartis Investigative Site

    Galway, 12074, Ireland

  • Novartis Investigative Site

    Ancona, AN, 60126, Italy

  • Novartis Investigative Site

    Brescia, BS, 25123, Italy

  • Novartis Investigative Site

    Cona, FE, 44124, Italy

  • Novartis Investigative Site

    Florence, FI, 50134, Italy

  • Novartis Investigative Site

    Genova, GE, 16132, Italy

  • Novartis Investigative Site

    Nuoro, NU, 08100, Italy

  • Novartis Investigative Site

    Perugia, PG, 06129, Italy

  • Novartis Investigative Site

    Reggio Calabria, RC, 89124, Italy

  • Novartis Investigative Site

    Roma, RM, 00161, Italy

  • Novartis Investigative Site

    Orbassano, TO, 10043, Italy

  • Novartis Investigative Site

    Terni, TR, 05100, Italy

  • Novartis Investigative Site

    Naples, 80131, Italy

  • Novartis Investigative Site

    Novara, 28100, Italy

  • Novartis Investigative Site

    Kashiwa, Chiba, 277-8567, Japan

  • Novartis Investigative Site

    Sapporo, Hokkaido, 060-8648, Japan

  • Novartis Investigative Site

    Sagamihara, Kanagawa, 252-0375, Japan

  • Novartis Investigative Site

    Kumamoto, Kumamoto, 860-8556, Japan

  • Novartis Investigative Site

    Sakai, Osaka, 590-0197, Japan

  • Novartis Investigative Site

    Suita, Osaka, 565-0871, Japan

  • Novartis Investigative Site

    Saga, Saga-ken, 849-8501, Japan

  • Novartis Investigative Site

    Kawagoe, Saitama, 3508550, Japan

  • Novartis Investigative Site

    Shimotsuga Gun, Tochigi, 3210293, Japan

  • Novartis Investigative Site

    Bunkyo-ku, Tokyo, 1138519, Japan

  • Novartis Investigative Site

    Shinjuku Ku, Tokyo, 160-0023, Japan

  • Novartis Investigative Site

    Akita, 0108543, Japan

  • Novartis Investigative Site

    Groningen, 9713 GZ, Netherlands

  • Novartis Investigative Site

    Gdansk, 80-952, Poland

  • Novartis Investigative Site

    Warsaw, 02-172, Poland

  • Novartis Investigative Site

    Terrassa, Catalonia, 08221, Spain

  • Novartis Investigative Site

    Pamplona, Navarre, 31008, Spain

  • Novartis Investigative Site

    Oviedo, Principality of Asturias, 33011, Spain

  • Novartis Investigative Site

    San Cristóbal de La Laguna, Santa Cruz De Tenerife, 38320, Spain

  • Novartis Investigative Site

    Barakaldo, Vizcaya, 48903, Spain

  • Novartis Investigative Site

    Barcelona, 08041, Spain

  • Novartis Investigative Site

    Las Palmas de Gran Canaria, 35010, Spain

  • Novartis Investigative Site

    Madrid, 28006, Spain

  • Novartis Investigative Site

    Madrid, 28034, Spain

  • Novartis Investigative Site

    Madrid, 28040, Spain

  • Novartis Investigative Site

    Madrid, 28041, Spain

  • Novartis Investigative Site

    Madrid, 28046, Spain

  • Novartis Investigative Site

    Ourense, 32005, Spain

  • Novartis Investigative Site

    Tarragona, 43005, Spain

  • Novartis Investigative Site

    Lund, SE-221 85, Sweden

  • Novartis Investigative Site

    Stockholm, SE-171 76, Sweden

  • Novartis Investigative Site

    Uppsala, SE-751 85, Sweden

  • Novartis Investigative Site

    Cardiff, CF14 4XW, United Kingdom

  • Novartis Investigative Site

    Oxford, OX3 7LE, United Kingdom

  • Oregon Health Sciences University

    Portland, Oregon, 97239, United States

  • Tennessee Oncology PLLC

    Chattanooga, Tennessee, 37404, United States

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