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Nilotinib followed by treatment-free

Phase 2

Chronic Myelogenous Leukemia | Small molecule | Oncology |Novartis AG|Last Updated: Aug 13, 2026

Success Probability

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Trial Design

UNCONTROLLED
Total Trials1
Total Enrollment215

FDA Designations

No designations recorded

Clinical trial landscape

Nilotinib followed by treatment-free · 1 trial · 1 indication

Phase 2 1
NCT01784068Nilotinib Treatment-free Remission Study in CML (Chronic Myeloid Leukemia) PatientsChronic Myelogenous Leukemia
COMPLETED215 Analytics
PHASE2COMPLETED
Nilotinib Treatment-free Remission Study in CML (Chronic Myeloid Leukemia) Patients
Chronic Myelogenous LeukemiaUnlock trial analytics

Study Endpoints

Primary Endpoints

Percentage of Participants in Major Molecular Response at 48 Weeks After Start of Treatment-Free Remission (TFR) Phase
48 weeks in TFR

The primary endpoint was the percentage of participants in major molecular response (MMR) at 48 weeks after initiation of the treatment-free remission (TFR) phase. Participants who required re-initiation of treatment were considered as non-responders.

Secondary Endpoints

Percentage of Participants in MR4.5 (BCR-ABL ≤ 0.0032% IS) at 48 Weeks After Start of TFR Phase
48 weeks in TFR
Percentage of Participants in Major Molecular Response (MMR) at Multiple Time Points After Start of the Treatment-Free Remission (TFR) Phase
Weeks 96, 144, 192, and 264, and End of Years 6, 7, 8, 9, and 10
Percentage of Participants in Molecular Response 4.5 (MR4.5) at Multiple Time Points After Start of the Treatment-Free Remission (TFR) Phase
Weeks 96, 144, 192, and 264, and End of Years 6, 7, 8, 9, and 10
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeOTHER

Treatment Arms

ArmTypeDescription
Nilotinib followed by treatment-freeEXPERIMENTALPatients who received a minimum of 2 years of first line nilotinib treatment and with pre-screen PCR results in ≥ MR4.5 entered the consolidation phase of the study (52 weeks - nilotinib 300 mg BID). Patients with Minimal Residual Disease (MRD) at the end of this phase entered the Treatment-Free Remission (TFR) phase where no treatment was given. Non eligible patients will enter the continuation phase of the study. Patients with MRD at the end of the continuation phase will enter the TFR-2 phase of the study where no treatment is given. Non eligible patients will enter the prolonged continuation phase of the study. If at any time during TFR or TFR-2 the patient loses MMR, nilotinib treatment will be immediately re-initiated (nilotinib 300 mg BID).

Interventions

NameTypeDescription
Nilotinib followed by treatment-freeDRUGNilotinib is being used as commercial available capsules (except in Japan where clinical supplies is used) of 150 mg and 200 mg strength. Treatment occurs during consolidation, continuation, prolonged continuation, re-initiation and re-initiation-2 phases of the study.
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Eligibility Criteria

Age Range18 Years to 100 Years
SexALL
Healthy VolunteersNo
Study Sites113

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 persp...

Countries:United StatesArgentinaAustriaBelgiumBulgariaColombiaDenmarkFranceGermanyGreeceHungaryIrelandItalyJapanNetherlandsPolandSpainSwedenUnited Kingdom
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Frequently asked questions about Nilotinib followed by treatment-free

What is Nilotinib followed by treatment-free used for in Chronic Myelogenous Leukemia?

Nilotinib followed by treatment-free is an investigational small molecule being studied for Chronic Myelogenous Leukemia (CML). It is designed to be administered as nilotinib therapy and then followed by a treatment-free period, aiming to assess whether patients can maintain remission without continued treatment.

Who makes Nilotinib followed by treatment-free?

Nilotinib followed by treatment-free is being developed by Novartis AG, which trades under the ticker NVS. The company is conducting clinical research on this regimen for Chronic Myelogenous Leukemia.

What phase is Nilotinib followed by treatment-free in?

Nilotinib followed by treatment-free is in Phase 2 clinical development. It is an investigational regimen and has not been approved by regulatory authorities. One Phase 2 trial has been completed, with no active trials currently ongoing.

What clinical trials is Nilotinib followed by treatment-free in?

Nilotinib followed by treatment-free has one completed clinical trial, NCT01784068, titled 'Nilotinib Treatment-free Remission Study in CML (Chronic Myeloid Leukemia) Patients.' This Phase 2 study enrolled 215 participants across multiple countries including the United States, Argentina, and Japan.

Is Nilotinib followed by treatment-free the same as Nilotinib?

Nilotinib followed by treatment-free is a regimen that involves the drug nilotinib, but it is not simply the drug itself. It refers to a treatment strategy where nilotinib is given initially and then discontinued to observe treatment-free remission. This approach is being studied specifically in Chronic Myelogenous Leukemia.