A Pharmacokinetic (PK) Study of Nilotinib in Pediatric Patients With Philadelphia Chromosome-positive (Ph+) Chronic Myelogenous Leukemia (CML) or Acute Lymphoblastic Leukemia (ALL)

This study has been completed.
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
First received: February 26, 2010
Last updated: August 3, 2015
Last verified: August 2015
This study will assess the pharmacokinetics of nilotinib in Ph+ CML pediatric patients that are newly diagnosed or resistant or intolerant to imatinib or dasatinib or refractory or relapsed Ph+ ALL compared to the adult populations. It will also evaluate safety and activity of nilotinib as secondary objectives.

Condition Intervention Phase
Chronic Myeloid Leukemia
Acute Lymphoblastic Leukemia
Drug: Nilotinib
Phase 1

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Pharmacokinetics/Dynamics Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Multi-center, Open-label, Pharmacokinetic Study of Oral Nilotinib in Pediatric Patients With Newly Diagnosed Chronic Phase (CP) Ph+ CML, With CP or Accelerated Phase (AP) Ph+ CML Resistant/Intolerant to Imatinib and/or Dasatinib, or With Refractory/Relapsed Ph+ ALL

Resource links provided by NLM:

Further study details as provided by Novartis:

Primary Outcome Measures:
  • Pharmacokinetic parameters of nilotinib in pediatric patients [ Time Frame: 28 days ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Safety and tolerability of nilotinib (AEs, laboratory assessments) [ Time Frame: 12 months ] [ Designated as safety issue: Yes ]
  • PK of nilotinib by activity (hematologic, cytogenetic and molecular responses) [ Time Frame: 12 months ] [ Designated as safety issue: Yes ]
  • Mutational assessment of BCR-ABL [ Time Frame: 12 months ] [ Designated as safety issue: Yes ]

Enrollment: 16
Study Start Date: April 2011
Study Completion Date: July 2015
Primary Completion Date: July 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Newly diagnosed
Newly diagnosed and untreated Ph+ CML in first chronic phase Diagnosis within 6 months of date of first cytogenetic analysis confirming Philadelphia chromosome with (9;22) translocation by standard conventional cytogenetic analysis.
Drug: Nilotinib
Nilotinib will be administered at 230mg/m2 ,twice daily for up to 24 cycles (1 cycle = 28 days). Drug will be supplied in 50mg, 150mg,and 200mg capsules. Dose administration will be rounded to the nearest 50mg dose (to a maximum dose of 400mg).
Other Name: Tasigna, AMN107
Experimental: Resistant/intolerant Ph+CML
Resistant/intolerant Ph+ CML in chronic or accelerated phase Resistant or Intolerant to either imatinib or dasatinib
Drug: Nilotinib
Nilotinib will be administered at 230mg/m2 ,twice daily for up to 24 cycles (1 cycle = 28 days). Drug will be supplied in 50mg, 150mg,and 200mg capsules. Dose administration will be rounded to the nearest 50mg dose (to a maximum dose of 400mg).
Other Name: Tasigna
Experimental: Ph+ ALL
Ph+ ALL refractory/relapsed to standard therapy
Drug: Nilotinib
Nilotinib will be administered at 230mg/m2 ,twice daily for up to 24 cycles (1 cycle = 28 days). Drug will be supplied in 50mg, 150mg,and 200mg capsules. Dose administration will be rounded to the nearest 50mg dose (to a maximum dose of 400mg).
Other Name: Tasigna


Ages Eligible for Study:   1 Year to 18 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • enrollment open only patients 1 < 10 years
  • Must have one of the following: newly diagnosed CP Ph+CML, CP or AP resistant/ intolerant to imatinib and/or dasatinib, or Ph+ ALL either relapsed after or refractory to standard therapy
  • adequate renal, hepatic and pancreatic function

Exclusion Criteria:

  • patients receiving therapy with strong CYP3A4 inhibitors and/or inducers and treatments cannot be stopped or changed to a different medication at least 14 days prior to starting study drug
  • patients receiving therapy with any medications with a known risk or possible risk to prolong the QT interval and the treatment cannot be either discontinued or switched to a different medication prior to starting study drug.
  • gastrointestinal impairment or disease that may interfere with drug absorption
  • liver, pancreatic or severe renal disease unrelated to disease under study
  • impaired cardiac function
  • patients who received dasatinib within 3 days of starting study drug
  • patients who received imatinib within 5 days of starting study drug
  • patients receiving hydroxyurea or corticosteroids that has not been discontinued at least 1 week after initiation of nilotinib
  • patients who received hematopoietic growth factors within 7 days of starting study drug or Pegfilgrastim (Neulasta®) within 14 days of starting study drug
  • patients with Stem Cell Transplant (SCT) or Rescue without TBI: Evidence of active graft vs. host disease and < 3 months since SCT

Other protocol-defined inclusion/exclusion criteria may apply

  Contacts and Locations
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Please refer to this study by its ClinicalTrials.gov identifier: NCT01077544

United States, Illinois
Ann & Robert H. Lurie Children's Hospital of Chicago Ann&Robert Lurie
Chicago, Illinois, United States, 60611
Novartis Investigative Site
Barretos, SP, Brazil, 14784-400
Novartis Investigative Site
Sao Paulo, SP, Brazil
Novartis Investigative Site
São Paulo, SP, Brazil, 08270-070
Novartis Investigative Site
Bordeaux, Aquitaine, France, 33076
Novartis Investigative Site
Lille cedex, France, 59037
Novartis Investigative Site
Paris, France, 75571
Novartis Investigative Site
Poitiers, France, 86021
Novartis Investigative Site
Monza, MB, Italy, 20900
Novartis Investigative Site
Padova, PD, Italy, 35128
Novartis Investigative Site
Roma, RM, Italy, 00161
Korea, Republic of
Novartis Investigative Site
Seoul, Korea, Korea, Republic of, 110 744
Novartis Investigative Site
Amsterdam, Netherlands, 1081 HV
Novartis Investigative Site
Rotterdam, Netherlands, 3015 GJ
Russian Federation
Novartis Investigative Site
Moscow, Russian Federation, 117997
United Kingdom
Novartis Investigative Site
Sutton, Surrey, United Kingdom, SM2 5PT
Novartis Investigative Site
Birmingham, United Kingdom, B4 6NH
Novartis Investigative Site
Bristol, United Kingdom, BS2 8BJ
Sponsors and Collaborators
Novartis Pharmaceuticals
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
  More Information

No publications provided

Responsible Party: Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier: NCT01077544     History of Changes
Other Study ID Numbers: CAMN107A2120, 2010-018419-14
Study First Received: February 26, 2010
Last Updated: August 3, 2015
Health Authority: United States: Food and Drug Administration
European Union: European Medicines Agency
Italy: Italian Medicines Agency (AIFA)
United Kingdom: Medicines and Healthcare Products Regulatory Agency
Netherlands: Centrale Commissie Mensgebonden Onderzoek (CCMO)
Russia: Ministry of Health of the Russian Federation
France: ANSM
Thailand: Food and Drug Administration
Brazil: ANVISA - Agência Nacional de Vigilância Sanitária
Germany: Federal Institute for Drugs & Medical Devices
Korea: MFDS Ministry of Food & Drug Safety

Keywords provided by Novartis:
chronic phase
accelerated phase
newly diagnosed Ph+ CML

Additional relevant MeSH terms:
Leukemia, Lymphoid
Leukemia, Myelogenous, Chronic, BCR-ABL Positive
Philadelphia Chromosome
Precursor Cell Lymphoblastic Leukemia-Lymphoma
Bone Marrow Diseases
Chromosome Aberrations
Hematologic Diseases
Immune System Diseases
Immunoproliferative Disorders
Leukemia, Myeloid
Lymphatic Diseases
Lymphoproliferative Disorders
Myeloproliferative Disorders
Neoplasms by Histologic Type
Pathologic Processes
Translocation, Genetic

ClinicalTrials.gov processed this record on December 01, 2015