Study Assessing Deep Molecular Response in Adult Patients With CML in Chronic Phase Treated With Nilotinib Firstline.

NCT02546674 | Completed Phase 4 Results

• 2 other identifiers: CAMN107ADE202015-000968-34
• Study Type: interventional
• Target: 171
• Locations: 1 country
• Sites: 70

Brief Summary

The main purpose of this study was to evaluate the rate of deep molecular response (MR4.5) after 24 months of therapy with nilotinib in newly diagnosed patients with chronic phase chronic myeloid leukemia (CML) using EUTOS (European Treatment and Outcome Study for CML)-standardized laboratories. All participants received nilotinib 300 mg twice daily (BID).

Conditions

Chronic Myeloid Leukemia

Keywords

Chronic Myeloid Leukemia; Nilotinib; Molecular Response; MR4.5

Outcome Measures

Primary Outcomes (1)

• Percentage of Participants With Deep Molecular Response MR4.5 at 24 Months of Study Treatment

  Percentage of participants who were in deep molecular response MR4.5 (IS) at 24 months measured in a standardized EUTOS (European Treatment and Outcome Study for CML) MR4.5 laboratory. MR4.5 was defined as either (i) detectable disease ≤ 0.0032% BCR-ABL (fusion gene from breakpoint cluster region and Abelson genes) (IS) or (ii) undetectable disease in cDNA with 32000-99999 ABL1 transcripts or 77000-239999 glucuronidase beta (GUSB) transcripts. Responders: Participants with a MR4.5 at 24 months, or if the assessment at this time point was missing, with a MR4.5 at 21 months Non-responders: Participants dropping out early or not providing sufficient data for any other reason. Participants who achieved MR4.5 before 24 months, but was no longer in MR4.5 at 24 months or progressed (or was no longer in MR4.5 at 21 months if evaluation at 24 months was missing). Confidence intervals were calculated based on the Exact Clopper-Pearson method.

  Month 24 and Month 21 (if assessment at Month 24 was missing)

Secondary Outcomes (17)

• Percentage of Participants With MR4 at 24 Months of Study Treatment.

  Month 24

• Percentage of Participants With Major Molecular Response (MMR) at 12 Months of Study Treatment

  Month 12

• Percentage of Participants With Complete Cytogenetic Response (CCyR) at 6 Months of Study Treatment

  Month 6

• Progression-free Survival

  From date of start of treatment to first documented disease progression to AP/ BC or death, assessed up to 24 months

• Time to Progression to AP/BC

  From the date of start of study treatment to the date of earliest transformation to AP/BC or CML-related death, assessed up to 24 months

Study Arms (1)

Nilotinib

EXPERIMENTAL

Participants with newly diagnosed CML in chronic phase received nilotinib 300 mg BID for 24 months

Drug: Nilotinib

Interventions

Nilotinib DRUG

A daily dose of 300 mg was given to all participants as two 150 mg capsules BID. The prescription of study drug was not study dependent and followed medical needs of the participant only. The study treatment was administered for 24 months.

Also known as: AMN107

Nilotinib

Eligibility Criteria

• Age: 18 Years - 120 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients with newly diagnosed (within 6 months) Philadelphia chromosome positive CML in chronic phase
• Patients must be previously untreated for CML with the exception of 6 months treatment with hydroxyurea and a maximum of 6 weeks treatment with imatinib
• Adequate end organ function
• Normal serum levels ≥ lower limit of normal (LLN) of potassium, magnesium, total calcium corrected for serum albumin or phosphorus, or correctable to within normal limits with supplements, prior to the first dose of study medication.

You may not qualify if:

• Known impaired cardiac function like long QT syndrome, history of myocardial infarction or unstable angina in the past 12 months.
• Patients who are pregnant or breast feeding.

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (70)

Novartis Investigative Site

Mannheim, Baden-Wurttemberg, 68305, Germany

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Novartis Investigative Site

Herne, North Rhine-Westphalia, 44625, Germany

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Novartis Investigative Site

Lübeck, Schleswig-Holstein, 23563, Germany

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Novartis Investigative Site

Aschaffenburg, 63739, Germany

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Novartis Investigative Site

Augsburg, 86179, Germany

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Novartis Investigative Site

Bad Mergentheim, 97980, Germany

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Novartis Investigative Site

Bad Reichenhall, 83435, Germany

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Novartis Investigative Site

Bad Saarow, 15526, Germany

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Novartis Investigative Site

Bad Soden, 65812, Germany

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Novartis Investigative Site

Bamberg, 96049, Germany

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Novartis Investigative Site

Bayreuth, 95445, Germany

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Novartis Investigative Site

Berlin, 12351, Germany

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Novartis Investigative Site

Berlin, 13353, Germany

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Novartis Investigative Site

Berlin, 13357, Germany

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Novartis Investigative Site

Berlin, 13581, Germany

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Novartis Investigative Site

Berlin, 14195, Germany

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Biberach, 88400, Germany

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Novartis Investigative Site

Bremerhaven, 27568, Germany

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Chemnitz, 09113, Germany

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Cologne, 50671, Germany

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Novartis Investigative Site

Donauwörth, 86609, Germany

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Novartis Investigative Site

Dresden, 01307, Germany

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Novartis Investigative Site

Düsseldorf, 40225, Germany

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Novartis Investigative Site

Düsseldorf, 40479, Germany

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Novartis Investigative Site

Erfurt, 99085, Germany

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Novartis Investigative Site

Essen, 45136, Germany

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Novartis Investigative Site

Essen, 45147, Germany

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Novartis Investigative Site

Georgsmarienhütte, 49124, Germany

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Novartis Investigative Site

Giessen, 35392, Germany

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Novartis Investigative Site

Goslar, 38642, Germany

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Novartis Investigative Site

Göppingen, 73035, Germany

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Halberstadt, 38820, Germany

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Novartis Investigative Site

Halle, 06110, Germany

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Halle S, 06120, Germany

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Novartis Investigative Site

Hanover, 30161, Germany

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Novartis Investigative Site

Hanover, 30170, Germany

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Novartis Investigative Site

Heidelberg, 69115, Germany

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Novartis Investigative Site

Heidelberg, 69120, Germany

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Novartis Investigative Site

Jena, 07740, Germany

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Novartis Investigative Site

Kassel, 34125, Germany

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Novartis Investigative Site

Kiel, 24105, Germany

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Novartis Investigative Site

Leipzig, 04103, Germany

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Novartis Investigative Site

Magdeburg, 39104, Germany

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Memmingen, 87700, Germany

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Novartis Investigative Site

Minden, 32429, Germany

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Novartis Investigative Site

Moers, 47441, Germany

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Mutlangen, 73557, Germany

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Mülheim, 45468, Germany

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Novartis Investigative Site

München, 80335, Germany

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Novartis Investigative Site

Münster, 48149, Germany

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Novartis Investigative Site

Nuremberg, 90403, Germany

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Novartis Investigative Site

Nuremberg, 90419, Germany

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Novartis Investigative Site

Oldenburg, 26121, Germany

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Novartis Investigative Site

Paderborn, 33098, Germany

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Novartis Investigative Site

Passau, 94036, Germany

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Novartis Investigative Site

Potsdam, 14467, Germany

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Novartis Investigative Site

Rostock, 18059, Germany

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Novartis Investigative Site

Rotenburg (Wümme), 27356, Germany

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Novartis Investigative Site

Saarbrücken, 66113, Germany

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Novartis Investigative Site

Schorndorf, 73614, Germany

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Novartis Investigative Site

Schwäbisch Hall, 74523, Germany

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Novartis Investigative Site

Schweinfurt, 97422, Germany

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Novartis Investigative Site

Tübingen, 72076, Germany

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Novartis Investigative Site

Velbert, 42551, Germany

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Novartis Investigative Site

Westerstede, 26655, Germany

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Novartis Investigative Site

Wiesbaden, 65191, Germany

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Novartis Investigative Site

Wilhelmshaven, 26389, Germany

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Novartis Investigative Site

Wolfsburg, 38440, Germany

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Novartis Investigative Site

Worms, 67547, Germany

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Novartis Investigative Site

Würzburg, 97080, Germany

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Related Links

• Related Info

MeSH Terms

Conditions

Leukemia, Myelogenous, Chronic, BCR-ABL Positive

Interventions

nilotinib

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Myeloproliferative Disorders; Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Results Point of Contact

• Title: Study director
• Organization: Novartis Pharmaceuticals

Novartis.email@novartis.com

862-778-8300

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 9, 2015
• First Posted: September 11, 2015
• Study Start: February 18, 2016
• Primary Completion: February 23, 2021
• Study Completion: March 25, 2021
• Last Updated: July 22, 2022
• Results First Posted: July 22, 2022

Record last verified: 2022-03

Data Sharing

• IPD Sharing: Will share

Locations

DE (70)