Nilotinib for Patients With Chronic Myeloid Leukemia in First Line and Any Subsequent Line (NOFRETETE)

Nilotinib for Patients With Chronic Myeloid Leukemia in First Line and Any Subsequent Line - a Non-interventional Study on the Assessment of Deep Molecular Response in CML Patients in Daily Routine.

This was a non-interventional observational study within the routine chronic myeloid leukemia treatment practice; no further tests were required apart from the assessments routinely performed for Chronic myeloid leukemia patients treated with nilotinib.

Study Overview

• Status: Completed
• Conditions: Chronic Myeloid Leukemia
• Intervention / Treatment: Other: Nilotinib

Detailed Description

The observation period per patient was 24 months. All patients were treated with nilotinib in accordance to the clinical routine at the respective institution and the summary of product characteristics (SmPC).

The observation intervals (documentation at baseline and at about 3, 6, 9, 12, 18 and 24 months) were not fixed and were aligned with the regular treatment schedule and the clinical symptoms of each patient. The medical decision about the schedule as well as therapeutic and diagnostic measures was made solely by the responsible physician. Patients who discontinued treatment within two years of the observation period were followed until starting a new TKI therapy line, however with a maximum time period of six months. All other patients that reached the official end of treatment after completion of the 24-month observation period were followed up for 28 days.

• Study Type: Observational
• Enrollment (Actual): 222

Contacts and Locations

Study Locations

• Germany
  • Altoetting, Germany, 84503
    • Novartis Investigative Site
  • Bad Liebenwerda, Germany, 04924
    • Novartis Investigative Site
  • Bad Mergentheim, Germany, 97980
    • Novartis Investigative Site
  • Bamberg, Germany, 96049
    • Novartis Investigative Site
  • Berlin, Germany, 13055
    • Novartis Investigative Site
  • Berlin, Germany, 10709
    • Novartis Investigative Site
  • Berlin, Germany, 12487
    • Novartis Investigative Site
  • Biberach, Germany, 88400
    • Novartis Investigative Site
  • Bielefeld, Germany, 33604
    • Novartis Investigative Site
  • Erfurt, Germany, 99085
    • Novartis Investigative Site
  • Erfurt, Germany, 99084
    • Novartis Investigative Site
  • Frankfurt, Germany, 60596
    • Novartis Investigative Site
  • Frankfurt, Germany, 60398
    • Novartis Investigative Site
  • Gera, Germany, 07548
    • Novartis Investigative Site
  • Halberstadt, Germany, 38820
    • Novartis Investigative Site
  • Halle, Germany, 06110
    • Novartis Investigative Site
  • Hamburg, Germany, 20259
    • Novartis Investigative Site
  • Hameln, Germany, 31785
    • Novartis Investigative Site
  • Hamm, Germany, 59063
    • Novartis Investigative Site
  • Hannover, Germany, 30161
    • Novartis Investigative Site
  • Heilbronn, Germany, 74072
    • Novartis Investigative Site
  • Heilbronn, Germany, 74078
    • Novartis Investigative Site
  • Hildesheim, Germany, 31135
    • Novartis Investigative Site
  • Köln, Germany, 50677
    • Novartis Investigative Site
  • Lemgo, Germany, 32657
    • Novartis Investigative Site
  • Ludwigsburg, Germany, 71636
    • Novartis Investigative Site
  • Memmingen, Germany, 87700
    • Novartis Investigative Site
  • Merseburg, Germany, 06217
    • Novartis Investigative Site
  • Moers, Germany, 47441
    • Novartis Investigative Site
  • Muenchen, Germany, 80331
    • Novartis Investigative Site
  • Neumuenster, Germany, 24534
    • Novartis Investigative Site
  • Nordhorn, Germany, 48527
    • Novartis Investigative Site
  • Nuernberg, Germany, 90403
    • Novartis Investigative Site
  • Nuernberg, Germany, 90449
    • Novartis Investigative Site
  • Offenburg, Germany, 77652
    • Novartis Investigative Site
  • Osnabrueck, Germany, 49076
    • Novartis Investigative Site
  • Porta Westfalica, Germany, 32457
    • Novartis Investigative Site
  • Potsdam, Germany, 14467
    • Novartis Investigative Site
  • Rotenburg, Germany, 27356
    • Novartis Investigative Site
  • Ruesselsheim, Germany, 65428
    • Novartis Investigative Site
  • Schorndorf, Germany, 73614
    • Novartis Investigative Site
  • Singen, Germany, 78224
    • Novartis Investigative Site
  • Speyer, Germany, 67346
    • Novartis Investigative Site
  • Stolberg, Germany, 52222
    • Novartis Investigative Site
  • Viersen, Germany, 45468
    • Novartis Investigative Site
  • Wiesbaden, Germany, 65191
    • Novartis Investigative Site
  • Wilhelmshaven, Germany, 26389
    • Novartis Investigative Site
  • Wolfsburg, Germany, 38440
    • Novartis Investigative Site
  • Wuerzburg, Germany, 97080
    • Novartis Investigative Site
  • Baden Wuerttemberg
    • Freudenstadt, Baden Wuerttemberg, Germany, 72250
      • Novartis Investigative Site
  • Bavaria
    • Augsburg, Bavaria, Germany, 86152
      • Novartis Investigative Site
    • Bayreuth, Bavaria, Germany, 95445
      • Novartis Investigative Site
    • Coburg, Bavaria, Germany, 96450
      • Novartis Investigative Site
    • Kronach, Bavaria, Germany, 96317
      • Novartis Investigative Site
    • Munich, Bavaria, Germany, 80797
      • Novartis Investigative Site
  • Bayern
    • Aschaffenburg, Bayern, Germany, 63739
      • Novartis Investigative Site
    • Donauwoerth, Bayern, Germany, 86609
      • Novartis Investigative Site
    • Landshut, Bayern, Germany, 84028
      • Novartis Investigative Site
  • Brandenburg
    • Frankfurt an der Oder, Brandenburg, Germany, 15236
      • Novartis Investigative Site
  • Heidenheim a.d.B
    • Baden-Württemberg, Heidenheim a.d.B, Germany, 89522
      • Novartis Investigative Site
  • Hessen
    • Erbach, Hessen, Germany, 64711
      • Novartis Investigative Site
  • Lower Saxony
    • Goettingen, Lower Saxony, Germany, 37073
      • Novartis Investigative Site
    • Goslar, Lower Saxony, Germany, 38642
      • Novartis Investigative Site
    • Twistringen, Lower Saxony, Germany, 27239
      • Novartis Investigative Site
  • Niedersachsen
    • Hannover, Niedersachsen, Germany, 30171
      • Novartis Investigative Site
    • Westerstede, Niedersachsen, Germany, 26655
      • Novartis Investigative Site
  • Nordrhein-Westfalen
    • Neuss, Nordrhein-Westfalen, Germany, 41464
      • Novartis Investigative Site
  • North Rhine-Westphalia
    • Aachen, North Rhine-Westphalia, Germany, 52064
      • Novartis Investigative Site
  • Northrhine Westfalia
    • Ahaus, Northrhine Westfalia, Germany, 48683
      • Novartis Investigative Site
    • Bad Salzuflen, Northrhine Westfalia, Germany, 32105
      • Novartis Investigative Site
    • Hagen, Northrhine Westfalia, Germany, 58095
      • Novartis Investigative Site
    • Iserlohn, Northrhine Westfalia, Germany, 58644
      • Novartis Investigative Site
  • Rhineland Palatinate
    • Neuwied, Rhineland Palatinate, Germany, 56564
      • Novartis Investigative Site
  • Sachsen
    • Dresden, Sachsen, Germany, 01127
      • Novartis Investigative Site
    • Zittau, Sachsen, Germany, 02763
      • Novartis Investigative Site
  • Saxony
    • Bad Schlema, Saxony, Germany, 08301
      • Novartis Investigative Site
  • Schleswig Holstein
    • Reinbek, Schleswig Holstein, Germany, 21465
      • Novartis Investigative Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Adult male and female patients with diagnosed Philadelphia chromosome positive (Ph+) and/or Breakpoint cluster region-Abelson tyrosine kinase (Abelson murine leukemia proto-oncogene)+ Chronic myeloid leukemia who were being treated with nilotinib in first or any subsequent line and for whom treatment with nilotinib was indicated according to SmPC

Description

Inclusion Criteria:

• Adult patients diagnosed with Ph+ CML (or evidence of BCR-ABL transcript) treated with nilotinib under routine medical practice and the SmPC, as amended, in first or any subsequent line, if the current therapy with nilotinib has not been in place for more than twelve months. Retrospective documentation of patients for up to one year will be permitted.
• Patients who have already had an interruption/discontinuation of nilotinib therapy.
• Patients who have been informed about this NIS and have personally dated and signed their informed consent form.

Exclusion Criteria:

• There are no exclusion criteria, apart from the contraindications mentioned in the SmPC. Participating patients are not allowed to take part in a clinical trial in parallel,

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Nilotinib; patients prescribed with nilotinib in routine medical practice	Other: Nilotinib; There was no treatment allocation. Patients administered Nilotinib by prescription could be enrolled.; Other Names: Tasigna

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Proportion of patients in Major molecular response (MMR), and Deep molecular response according to international standard (MR^4.0 and MR^4.5)	Proportion of patients in MMR, MR^4.0 and MR^4.5 was collected	24 months
Time to achievement of an MR4.0 and MR4.5	Time to achievement of an MR4.0 and MR4.5 was collected	24 months
Duration of an MR4.0 and MR4.5	Duration of an MR4.0 and MR4.5 was collected	24 months
Proportion of patients whose molecular response is routinely analyzed by a MR4.5-certified laboratory	Proportion of patients whose molecular response is routinely analyzed by a MR4.5-certified laboratory was collected	24 months
Proportion of European Treatment and Outcome Study for CML (EUTOS)-qualified laboratories that perform qRT-PCR.	Proportion of European Treatment and Outcome Study for CML (EUTOS)-qualified laboratories that perform qRT-PCR was documented	24 months
Patient-reported QoL	The EORTC QLQ-C30 questionnaire in conjunction with the EORTC QLQ-CML24 module was used to assess patient-reported QoL.	24 months
Patient adherence	Patient adherence was documented using the MMAS-8 (Morisky et al., 2008) patient questionnaire.	24 months
Proportion of patients who discontinue Tyrosine kinase inhibitor (TKI) therapy	Proportion of patients who discontinue Tyrosine kinase inhibitor (TKI) therapy was documented	24 months

Collaborators and Investigators

• Sponsor: Novartis Pharmaceuticals
• Investigators: Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Publications and helpful links

Helpful Links

• Results for Study CAMN107ADE23 that is getting linked from the Novartis Clinical Trials Website

Study record dates

Study Major Dates

• Study Start (Actual): June 28, 2016
• Primary Completion (Actual): September 8, 2022
• Study Completion (Actual): September 8, 2022

Study Registration Dates

• First Submitted: February 9, 2023
• First Submitted That Met QC Criteria: February 9, 2023
• First Posted (Actual): February 17, 2023

Study Record Updates

• Last Update Posted (Actual): August 21, 2023
• Last Update Submitted That Met QC Criteria: August 17, 2023
• Last Verified: August 1, 2023

More Information

Terms related to this study

• Keywords: Nilotinib; CML; Chronic myeloid leukemia; NOFRETETE
• Additional Relevant MeSH Terms: Pathologic Processes; Neoplasms by Histologic Type; Neoplasms; Disease Attributes; Bone Marrow Diseases; Hematologic Diseases; Myeloproliferative Disorders; Chronic Disease; Leukemia; Leukemia, Myeloid; Leukemia, Myelogenous, Chronic, BCR-ABL Positive
• Other Study ID Numbers: CAMN107ADE23

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO