Clinical trial NCT05734053: Nilotinib for Patients With Chronic Myeloid Leukemia in First Line and Any Subsequent Line
Official title: 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.
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Chronic Myeloid Leukemia
Keywords: Chronic myeloid leukemia
CML
NOFRETETE
Nilotinib
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2023-02-17
Enrollment: 222
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
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

Brief summary: 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.

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.

Eligibility criteria: 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,

Primary outcomes:
measure: Proportion of patients in Major molecular response (MMR), and Deep molecular response according to international standard (MR^4.0 and MR^4.5)
timeFrame: 24 months
measure: Time to achievement of an MR4.0 and MR4.5
timeFrame: 24 months
measure: Duration of an MR4.0 and MR4.5
timeFrame: 24 months
measure: Proportion of patients whose molecular response is routinely analyzed by a MR4.5-certified laboratory
timeFrame: 24 months
measure: Proportion of European Treatment and Outcome Study for CML (EUTOS)-qualified laboratories that perform qRT-PCR.
timeFrame: 24 months
measure: Patient-reported QoL
timeFrame: 24 months
measure: Patient adherence
timeFrame: 24 months
measure: Proportion of patients who discontinue Tyrosine kinase inhibitor (TKI) therapy
timeFrame: 24 months
Secondary outcomes:
Other outcomes:
Locations:
city: Freudenstadt
country: Germany
facility: Novartis Investigative Site
state: Baden Wuerttemberg
city: Augsburg
country: Germany
facility: Novartis Investigative Site
state: Bavaria
city: Bayreuth
country: Germany
facility: Novartis Investigative Site
state: Bavaria
city: Coburg
country: Germany
facility: Novartis Investigative Site
state: Bavaria
city: Kronach
country: Germany
facility: Novartis Investigative Site
state: Bavaria
city: Munich
country: Germany
facility: Novartis Investigative Site
state: Bavaria
city: Aschaffenburg
country: Germany
facility: Novartis Investigative Site
state: Bayern
city: Donauwoerth
country: Germany
facility: Novartis Investigative Site
state: Bayern
city: Landshut
country: Germany
facility: Novartis Investigative Site
state: Bayern
city: Frankfurt an der Oder
country: Germany
facility: Novartis Investigative Site
state: Brandenburg
city: Baden-Württemberg
country: Germany
facility: Novartis Investigative Site
state: Heidenheim a.d.B
city: Erbach
country: Germany
facility: Novartis Investigative Site
state: Hessen
city: Goettingen
country: Germany
facility: Novartis Investigative Site
state: Lower Saxony
city: Goslar
country: Germany
facility: Novartis Investigative Site
state: Lower Saxony
city: Twistringen
country: Germany
facility: Novartis Investigative Site
state: Lower Saxony
city: Hannover
country: Germany
facility: Novartis Investigative Site
state: Niedersachsen
city: Westerstede
country: Germany
facility: Novartis Investigative Site
state: Niedersachsen
city: Neuss
country: Germany
facility: Novartis Investigative Site
state: Nordrhein-Westfalen
city: Aachen
country: Germany
facility: Novartis Investigative Site
state: North Rhine-Westphalia
city: Ahaus
country: Germany
facility: Novartis Investigative Site
state: Northrhine Westfalia
city: Bad Salzuflen
country: Germany
facility: Novartis Investigative Site
state: Northrhine Westfalia
city: Hagen
country: Germany
facility: Novartis Investigative Site
state: Northrhine Westfalia
city: Iserlohn
country: Germany
facility: Novartis Investigative Site
state: Northrhine Westfalia
city: Neuwied
country: Germany
facility: Novartis Investigative Site
state: Rhineland Palatinate
city: Dresden
country: Germany
facility: Novartis Investigative Site
state: Sachsen
city: Zittau
country: Germany
facility: Novartis Investigative Site
state: Sachsen
city: Bad Schlema
country: Germany
facility: Novartis Investigative Site
state: Saxony
city: Reinbek
country: Germany
facility: Novartis Investigative Site
state: Schleswig Holstein
city: Altoetting
country: Germany
facility: Novartis Investigative Site
city: Bad Liebenwerda
country: Germany
facility: Novartis Investigative Site
city: Bad Mergentheim
country: Germany
facility: Novartis Investigative Site
city: Bamberg
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: Bielefeld
country: Germany
facility: Novartis Investigative Site
city: Erfurt
country: Germany
facility: Novartis Investigative Site
city: Erfurt
country: Germany
facility: Novartis Investigative Site
city: Frankfurt
country: Germany
facility: Novartis Investigative Site
city: Frankfurt
country: Germany
facility: Novartis Investigative Site
city: Gera
country: Germany
facility: Novartis Investigative Site
city: Halberstadt
country: Germany
facility: Novartis Investigative Site
city: Halle
country: Germany
facility: Novartis Investigative Site
city: Hamburg
country: Germany
facility: Novartis Investigative Site
city: Hameln
country: Germany
facility: Novartis Investigative Site
city: Hamm
country: Germany
facility: Novartis Investigative Site
city: Hannover
country: Germany
facility: Novartis Investigative Site
city: Heilbronn
country: Germany
facility: Novartis Investigative Site
city: Heilbronn
country: Germany
facility: Novartis Investigative Site
city: Hildesheim
country: Germany
facility: Novartis Investigative Site
city: Köln
country: Germany
facility: Novartis Investigative Site
city: Lemgo
country: Germany
facility: Novartis Investigative Site
city: Ludwigsburg
country: Germany
facility: Novartis Investigative Site
city: Memmingen
country: Germany
facility: Novartis Investigative Site
city: Merseburg
country: Germany
facility: Novartis Investigative Site
city: Moers
country: Germany
facility: Novartis Investigative Site
city: Muenchen
country: Germany
facility: Novartis Investigative Site
city: Neumuenster
country: Germany
facility: Novartis Investigative Site
city: Nordhorn
country: Germany
facility: Novartis Investigative Site
city: Nuernberg
country: Germany
facility: Novartis Investigative Site
city: Nuernberg
country: Germany
facility: Novartis Investigative Site
city: Offenburg
country: Germany
facility: Novartis Investigative Site
city: Osnabrueck
country: Germany
facility: Novartis Investigative Site
city: Porta Westfalica
country: Germany
facility: Novartis Investigative Site
city: Potsdam
country: Germany
facility: Novartis Investigative Site
city: Rotenburg
country: Germany
facility: Novartis Investigative Site
city: Ruesselsheim
country: Germany
facility: Novartis Investigative Site
city: Schorndorf
country: Germany
facility: Novartis Investigative Site
city: Singen
country: Germany
facility: Novartis Investigative Site
city: Speyer
country: Germany
facility: Novartis Investigative Site
city: Stolberg
country: Germany
facility: Novartis Investigative Site
city: Viersen
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: Wuerzburg
country: Germany
facility: Novartis Investigative Site
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: