Clinical trial NCT02546674: Study Assessing Deep Molecular Response in Adult Patients With CML in Chronic Phase Treated With Nilotinib Firstline.
Official title: A Phase IV Single Arm, Multicenter, Open-label Study Assessing Deep Molecular Response in Adult Patients With Newly Diagnosed Philadelphia Chromosome Positive CML in Chronic Phase After Two Years of Treatment With Nilotinib 300mg BID
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Chronic Myeloid Leukemia
Keywords: Chronic Myeloid Leukemia
Nilotinib
Molecular Response
MR4.5
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2015-09-11
Enrollment: 171
Sex: ALL
Minimum age (years): 18
Maximum age (years): 120
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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).

Detailed description: This was a Phase IV open-label, multi-center, single-arm study in participants with newly diagnosed Philadelphia chromosome positive (Ph+) CML in chronic phase for who nilotinib is the appropriate treatment at the discretion of the investigator.

A screening period of 2 weeks was used to assess eligibility and to taper participants off disallowed medications. Participants whose eligibility was confirmed entered a 24 months treatment phase with nilotinib 300mg BID. Nilotinib was prescribed by the investigator according to the individual needs of the participants.

Eligibility criteria: Key Inclusion Criteria:

* 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.

Key Exclusion Criteria:

* 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.

Other inclusion/exclusion criteria might apply

Primary outcomes:
measure: Percentage of Participants With Deep Molecular Response MR4.5 at 24 Months of Study Treatment
timeFrame: Month 24 and Month 21 (if assessment at Month 24 was missing)
Secondary outcomes:
measure: Percentage of Participants With MR4 at 24 Months of Study Treatment.
timeFrame: Month 24
measure: Percentage of Participants With Major Molecular Response (MMR) at 12 Months of Study Treatment
timeFrame: Month 12
measure: Percentage of Participants With Complete Cytogenetic Response (CCyR) at 6 Months of Study Treatment
timeFrame: Month 6
measure: Progression-free Survival
timeFrame: From date of start of treatment to first documented disease progression to AP/ BC or death, assessed up to 24 months
measure: Time to Progression to AP/BC
timeFrame: 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
measure: Change From Baseline in European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core-30 (EORTC QLQ-C30): Global Health Status (GHS)/Quality of Life (QoL)
timeFrame: Baseline, month 3, month 6, month 12, month 18 and month 24
measure: Change From Baseline in European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core-30 (EORTC QLQ-C30): Physical Functioning
timeFrame: Baseline, month 3, month 6, month 12, month 18 and month 24
measure: Change From Baseline in European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core-30 (EORTC QLQ-C30): Role Functioning
timeFrame: Baseline, month 3, month 6, month 12, month 18 and month 24
measure: Change From Baseline in European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core-30 (EORTC QLQ-C30): Emotional Functioning
timeFrame: Baseline, month 3, month 6, month 12, month 18 and month 24
measure: Change From Baseline in European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core-30 (EORTC QLQ-C30): Cognitive Functioning
timeFrame: Baseline, month 3, month 6, month 12, month 18 and month 24
measure: Change From Baseline in European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core-30 (EORTC QLQ-C30): Social Functioning
timeFrame: Baseline, month 3, month 6, month 12, month 18 and month 24
measure: Change From Baseline in European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Chronic Myeloid Leukemia Specific 24 (EORTC QLQ-CML 24): Symptom Burden
timeFrame: Baseline, month 3, month 6, month 12, month 18 and month 24
measure: Change From Baseline in European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Chronic Myeloid Leukemia Specific 24 (EORTC QLQ-CML 24): Impact on Worry/Mood
timeFrame: Baseline, month 3, month 6, month 12, month 18 and month 24
measure: Change From Baseline in European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Chronic Myeloid Leukemia Specific 24 (EORTC QLQ-CML 24): Impact on Daily Life
timeFrame: Baseline, month 3, month 6, month 12, month 18 and month 24
measure: Change From Baseline in European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Chronic Myeloid Leukemia Specific 24 (EORTC QLQ-CML 24): Satisfaction With Care and Information
timeFrame: Baseline, month 3, month 6, month 12, month 18 and month 24
measure: Change From Baseline in European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Chronic Myeloid Leukemia Specific 24 (EORTC QLQ-CML 24): Body Image Problems
timeFrame: Baseline, month 3, month 6, month 12, month 18 and month 24
measure: Change From Baseline in European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Chronic Myeloid Leukemia Specific 24 (EORTC QLQ-CML 24): Satisfaction With Social Life
timeFrame: Baseline, month 3, month 6, month 12, month 18 and month 24
Other outcomes:
Locations:
city: Mannheim
country: Germany
facility: Novartis Investigative Site
state: Baden-Wuerttemberg
city: Herne
country: Germany
facility: Novartis Investigative Site
state: Nordrhein-Westfalen
city: Luebeck
country: Germany
facility: Novartis Investigative Site
state: Schleswig-holstein
city: Aschaffenburg
country: Germany
facility: Novartis Investigative Site
city: Augsburg
country: Germany
facility: Novartis Investigative Site
city: Bad Mergentheim
country: Germany
facility: Novartis Investigative Site
city: Bad Reichenhall
country: Germany
facility: Novartis Investigative Site
city: Bad Saarow
country: Germany
facility: Novartis Investigative Site
city: Bad Soden
country: Germany
facility: Novartis Investigative Site
city: Bamberg
country: Germany
facility: Novartis Investigative Site
city: Bayreuth
country: Germany
facility: Novartis Investigative Site
city: Berlin
country: Germany
facility: Novartis Investigative Site
city: Berlin
country: Germany
facility: Novartis Investigative Site
city: Berlin
country: Germany
facility: Novartis Investigative Site
city: Berlin
country: Germany
facility: Novartis Investigative Site
city: Berlin
country: Germany
facility: Novartis Investigative Site
city: Biberach
country: Germany
facility: Novartis Investigative Site
city: Bremerhaven
country: Germany
facility: Novartis Investigative Site
city: Chemnitz
country: Germany
facility: Novartis Investigative Site
city: Donauwoerth
country: Germany
facility: Novartis Investigative Site
city: Dresden
country: Germany
facility: Novartis Investigative Site
city: Duesseldorf
country: Germany
facility: Novartis Investigative Site
city: Duesseldorf
country: Germany
facility: Novartis Investigative Site
city: Erfurt
country: Germany
facility: Novartis Investigative Site
city: Essen
country: Germany
facility: Novartis Investigative Site
city: Essen
country: Germany
facility: Novartis Investigative Site
city: Georgsmarienhuette
country: Germany
facility: Novartis Investigative Site
city: Giessen
country: Germany
facility: Novartis Investigative Site
city: Goslar
country: Germany
facility: Novartis Investigative Site
city: Göppingen
country: Germany
facility: Novartis Investigative Site
city: Halberstadt
country: Germany
facility: Novartis Investigative Site
city: Halle S
country: Germany
facility: Novartis Investigative Site
city: Halle
country: Germany
facility: Novartis Investigative Site
city: Hannover
country: Germany
facility: Novartis Investigative Site
city: Hannover
country: Germany
facility: Novartis Investigative Site
city: Heidelberg
country: Germany
facility: Novartis Investigative Site
city: Heidelberg
country: Germany
facility: Novartis Investigative Site
city: Jena
country: Germany
facility: Novartis Investigative Site
city: Kassel
country: Germany
facility: Novartis Investigative Site
city: Kiel
country: Germany
facility: Novartis Investigative Site
city: Koeln
country: Germany
facility: Novartis Investigative Site
city: Leipzig
country: Germany
facility: Novartis Investigative Site
city: Magdeburg
country: Germany
facility: Novartis Investigative Site
city: Memmingen
country: Germany
facility: Novartis Investigative Site
city: Minden
country: Germany
facility: Novartis Investigative Site
city: Moers
country: Germany
facility: Novartis Investigative Site
city: Muelheim
country: Germany
facility: Novartis Investigative Site
city: Muenchen
country: Germany
facility: Novartis Investigative Site
city: Muenster
country: Germany
facility: Novartis Investigative Site
city: Mutlangen
country: Germany
facility: Novartis Investigative Site
city: Nuernberg
country: Germany
facility: Novartis Investigative Site
city: Nuernberg
country: Germany
facility: Novartis Investigative Site
city: Oldenburg
country: Germany
facility: Novartis Investigative Site
city: Paderborn
country: Germany
facility: Novartis Investigative Site
city: Passau
country: Germany
facility: Novartis Investigative Site
city: Potsdam
country: Germany
facility: Novartis Investigative Site
city: Rostock
country: Germany
facility: Novartis Investigative Site
city: Rotenburg
country: Germany
facility: Novartis Investigative Site
city: Saarbruecken
country: Germany
facility: Novartis Investigative Site
city: Schorndorf
country: Germany
facility: Novartis Investigative Site
city: Schweinfurt
country: Germany
facility: Novartis Investigative Site
city: Schwäbisch-Hall
country: Germany
facility: Novartis Investigative Site
city: Tübingen
country: Germany
facility: Novartis Investigative Site
city: Velbert
country: Germany
facility: Novartis Investigative Site
city: Westerstede
country: Germany
facility: Novartis Investigative Site
city: Wiesbaden
country: Germany
facility: Novartis Investigative Site
city: Wilhelmshaven
country: Germany
facility: Novartis Investigative Site
city: Wolfsburg
country: Germany
facility: Novartis Investigative Site
city: Worms
country: Germany
facility: Novartis Investigative Site
city: Wuerzburg
country: Germany
facility: Novartis Investigative Site
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: