Determining the Maximum Tolerated Dose of Low Dose Interferon-alpha in Conjunction With Nilotinib in Pretreated Philadelphia Chromosome Positive (Ph+) Chronic Myeloid Leukemia Patients in Chronic Phase (CML-CP) (NICOLI)

This study has been completed.
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals ) Identifier:
First received: October 6, 2010
Last updated: December 12, 2013
Last verified: December 2013

This study will assess the maximum tolerated dose of low dose interferon in conjunction with nilotinib in pretreated Philadelphia chromosome positive (Ph+) chronic myeloid leukemia patients in chronic phase (CML-CP).

Condition Intervention Phase
Chronic Myeloid Leukemia
Drug: Nilotinib, interferon-alfa
Phase 4

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: An Open Label, Nonrandomized, Single-center, Phase I Trial of Pretreated Philadelphia Chromosome Positive (Ph+) Chronic Myeloid Leukemia Patients in Chronic Phase (CML-CP) With Nilotinib in Combination With Low Dose Interferon-alpha (IFN) - NICOLI Study -

Resource links provided by NLM:

Further study details as provided by Novartis:

Primary Outcome Measures:
  • Number of Clinically significant adverse events or abnormal laboratory values (dose-limiting toxicities) unrelated to disease progression, intercurrent illness, or concomitant medications on the combination treatment [ Time Frame: 12 months ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Rate of major cytogenetic response (MCyR) at 6 and 12 months [ Time Frame: 12 months ] [ Designated as safety issue: No ]
  • Rate of complete cytogenetic response (CCyR) at 6 and 12 months [ Time Frame: 12 months ] [ Designated as safety issue: No ]
  • Rate of major molecular response (MMR) at 12 months [ Time Frame: 12 months ] [ Designated as safety issue: No ]
  • Safety profile of nilotinib in combination with interferon alfa, i.e. the number of dose limiting toxicities (DLT) for each interferon alfa dose level [ Time Frame: 12 months ] [ Designated as safety issue: Yes ]
  • Progression-free survival (PFS) [ Time Frame: 12 months ] [ Designated as safety issue: Yes ]
  • Event-free survival [ Time Frame: 12 months ] [ Designated as safety issue: Yes ]
  • Overall survival (OS) [ Time Frame: 12 months ] [ Designated as safety issue: Yes ]

Enrollment: 4
Study Start Date: April 2012
Study Completion Date: November 2013
Primary Completion Date: November 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Nilotinib in conjunction with low dose interferon alfa Drug: Nilotinib, interferon-alfa
Other Name: AMN107


Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Patients with chronic myeloid leukemia in chronic phase (CML-CP) at screening
  • Initial diagnosis of CML cytogenetically confirmed by the presence of the Ph+ metaphases from the bone marrow
  • Patients who have been treated with nilotinib for a minimum of 6 months (1 month represents 28 days) after switch from previous CML treatments
  • Patients who have been treated with stable dosing of 2x400mg nilotinib within the last month before start of study treatment
  • No grade 3-4 CTC toxicities on nilotinib alone in the last month preceding the start of the study regimen

Exclusion Criteria:

  • Patients who are considered Ph- because they do not have a confirmed cytogenetic diagnosis of the t(9;22) translocation in their bone marrow metaphases
  • Evidence of a point mutation within the BCR-ABL gene leading to a clinically relevant amino acid exchange in the kinase domain at position T315 (gatekeeper mutation T315I)
  • Impaired cardiac function
  • Severe or uncontrolled medical conditions (i.e. uncontrolled diabetes, active or uncontrolled infection).

Other protocol-defined inclusion/exclusion criteria may apply

  Contacts and Locations
Please refer to this study by its identifier: NCT01220648

Novartis Investigative Site
Leipzig, Germany, 04103
Sponsors and Collaborators
Novartis Pharmaceuticals
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
  More Information

Responsible Party: Novartis ( Novartis Pharmaceuticals ) Identifier: NCT01220648     History of Changes
Other Study ID Numbers: CAMN107ADE10, 2010-022006-40
Study First Received: October 6, 2010
Last Updated: December 12, 2013
Health Authority: United States: Food and Drug Administration
Germany: Federal Institute for Drugs and Medical Devices

Keywords provided by Novartis:
Chronic myeloid leukemia
interferon alfa
Philadelphia chromosome positive (Ph+) chronic myeloid leukemia patients in chronic phase (CML-CP) after switch from previous CML treatment

Additional relevant MeSH terms:
Leukemia, Myeloid
Leukemia, Myelogenous, Chronic, BCR-ABL Positive
Philadelphia Chromosome
Neoplasms by Histologic Type
Myeloproliferative Disorders
Bone Marrow Diseases
Hematologic Diseases
Translocation, Genetic
Chromosome Aberrations
Pathologic Processes
Interferon Alfa-2a
Antiviral Agents
Anti-Infective Agents
Therapeutic Uses
Pharmacologic Actions
Immunologic Factors
Physiological Effects of Drugs
Angiogenesis Inhibitors
Angiogenesis Modulating Agents
Growth Substances
Growth Inhibitors
Antineoplastic Agents processed this record on April 17, 2014