Treatment-free Remission After Achieving Sustained MR4.5 on Nilotinib (ENESTop)

Completed · Phase 2

Conditions studied: Chronic Myeloid Leukemia

In brief

A clinical research study to find out if it is safe to stop the drug nilotinib (Tasigna) in chronic myeloid leukemia (CML) patients. Patients who started treatment with imatinib (Gleevec) when they were first diagnosed with CML, then switched to nilotinib (Tasigna) for at least 2 years with the combined time on imatinib (Gleevec) and nilotinib (Tasigna) for at least 3 years and have very small amount of leukemia cells remaining after the nilotinib (Tasigna) treatment will qualify for the study.

Key facts

Study ID

NCT01698905

Run by

Novartis Pharmaceuticals

People needed

163

Starts

2012-12-20

Expected to finish

2025-01-29

Last updated by the study team

2025-09-02

Who can join

Age: 18 and older, up to 100. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Male or female patients >= 18 years of age
• ECOG Performance Status of 0, 1, or 2
• Patient with diagnosis of BCR-ABL positive CML CP
• Patient has received a minimum of 3 years of tyrosine kinase inhibitor treatment (first with imatinib (> 4 weeks) and then switched to nilotinib) since initial diagnosis
• Patient has at least 2 years of nilotinib treatment prior to study entry.
• Patient has achieved MR4.5 (local laboratory assessment) during nilotinib treatment, and determined by a Novartis designated central PCR lab assessment at screening
• Adequate end organ function as defined by:
• Direct bilirubin ≤ 1.5 x ULN except for i) patient 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 (upper limit of normal)
• Serum lipase ≤ 2 x ULN
• Alkaline phosphatase ≤ 2.5 x ULN
• Serum creatinine < 1.5 x ULN
• Patients must have the following electrolyte values ≥ LLN (lower limit of normal) limits or corrected to within normal limits with supplements prior to the first dose of study medication:
• Potassium
• Magnesium
• Total calcium (corrected for serum albumin)
• Patients must have normal marrow function as defined below:
• Absolute Neutrophil Count (ANC) ≥ 1.5 x 109/L
• Platelets ≥ 100 x 109/L
• Hemoglobin ≥ 9.0 g/dL
• Written informed consent obtained prior to any screening procedures

You may not qualify if…

• Prior AP, BC or allo-transplant
• Patient has documented MR4.5 at the time when switched from imatinib to nilotinib
• Patients with known atypical transcript
• CML treatment resistant mutation(s) (T315I, E255K/V, Y253H, F359C/V) detected if a testing was done in the past (there is no requirement to perform mutation testing at study entry if it was not done in the past)
• Dose reductions due to neutropenia or thrombocytopenia in the past 6 months
• Patient ever attempted to permanently discontinue imatinib or nilotinib treatment
• Known impaired cardiac function including any one of the following:
• Inability to determine the QT interval on ECG
• Complete left bundle branch block
• 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
• QTcF > 480 msec
• History or clinical signs of myocardial infarction within 1 year prior to study entry
• History of unstable angina within 1 year prior to study entry
• Other clinically significant heart disease (e.g. uncontrolled congestive heart failure or uncontrolled hypertension)
• 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 (defined as HbA1c > 9%), uncontrolled infection)
• History of acute pancreatitis within 1 year prior to study entry or past medical history of chronic pancreatitis
• Known presence of a significant congenital or acquired bleeding disorder unrelated to cancer
• History of other active malignancy within 5 years prior to study entry with the exception of previous or concomitant basal cell skin cancer, previous cervical carcinoma in situ treated curatively
• Patients who have not recovered from prior surgery
• Treatment with other investigational agents (defined as not used in accordance with the approved indication) within 4 weeks of Day 1
• 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 study entry. See Appendix 14.1 for a list of these medications. This list may not be comprehensive.
• 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 study entry. 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 study entry. (Please see www.azcert.org/medical-pros/drug-lists/printable-drug-list.cfm for a list of agents that prolong the QT interval.)

Where it is running

• Indiana Blood and Marrow Institute — Beech Grove, Indiana, United States
• St Agnes Hospital — Baltimore, Maryland, United States
• University of Texas Medical Branch — Galveston, Texas, United States
• Compass Oncology — Vancouver, Washington, United States
• Novartis Investigative Site — CABA, Buenos Aires, Argentina
• Novartis Investigative Site — Buenos Aires, Argentina
• Novartis Investigative Site — Adelaide, South Australia, Australia
• Novartis Investigative Site — Box Hill, Victoria, Australia
• Novartis Investigative Site — Antwerp, Belgium
• Novartis Investigative Site — Goiânia, Goiás, Brazil
• Novartis Investigative Site — Belo Horizonte, Minas Gerais, Brazil
• Novartis Investigative Site — Rio de Janeiro, Rio de Janeiro, Brazil
• Novartis Investigative Site — Rio de Janiero, Rio de Janeiro, Brazil
• Novartis Investigative Site — Porto Alegre, Rio Grande do Sul, Brazil
• Novartis Investigative Site — Campinas, São Paulo, Brazil
• Novartis Investigative Site — Hamilton, Ontario, Canada
• Novartis Investigative Site — Toronto, Ontario, Canada
• Novartis Investigative Site — Montreal, Quebec, Canada
• Novartis Investigative Site — Québec, Quebec, Canada
• Novartis Investigative Site — Bordeaux, France
• Novartis Investigative Site — Grenoble, France
• Novartis Investigative Site — Lyon, France
• Novartis Investigative Site — Strasbourg, France
• Novartis Investigative Site — Vandœuvre-lès-Nancy, France
• USC Kenneth Norris Comprehensive Cancer Center — Los Angeles, California, United States

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.