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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: October 19, 2014
Last verified: October 2014

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: January 2014
Primary Completion Date: January 2014 (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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

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: October 19, 2014
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:
Abnormal Karyotype
Leukemia, Myelogenous, Chronic, BCR-ABL Positive
Leukemia, Myeloid
Philadelphia Chromosome
Bone Marrow Diseases
Chromosome Aberrations
Hematologic Diseases
Myeloproliferative Disorders
Neoplasms by Histologic Type
Pathologic Processes
Translocation, Genetic
Anti-Infective Agents
Antineoplastic Agents
Antiviral Agents
Immunologic Factors
Pharmacologic Actions
Physiological Effects of Drugs
Therapeutic Uses processed this record on November 20, 2014