Study to Assess Efficacy and Safety of Nilotinib 300mg Twice Daily in Patients With Philadelphia Positive Chronic Myeloid Leukaemia (CML) in Chronic Phase Who Are Intolerant to Prior Tyrosine Kinase Inhibitors.

NCT ID: NCT02108951

Last Updated: 2017-10-27

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE3
• Total Enrollment: 20 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2014-07-07
• Study Completion Date: 2016-08-10

Brief Summary

The purpose of this Australian study was to assess the efficacy and safety of nilotinib 300mg twice daily in patients with chronic myeloid leukemia chronic phase who were intolerant but responsive to 1st line treatment with imatinib or dasatinib. Eligible patients have been previously treated with imatinib or dasatinib for at least 3 months and are experiencing non-hematologic toxicity whilst having documented responses that meet PBS authority for 1st line treatment of CML without current MR4.5.

Detailed Description

The study was planned to enroll 130 patients to achieve the sample size requirement for a meaning full conclusion. Study got terminated with 20 patients because of slow recruitment. Therefore, due to small sample size, the results cannot be considered clinical significant.

Conditions

Philidelphia Positive Chronic Myeloid Leukaemia

Keywords

CML; Ph-CML; leukaemia; leukemia; chronic myeloid leukemia; chronic myeloid leukaemia; TKI intolerance; tyrosine kinase inhibitor; tyrosine kinase inhibitor intolerance

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Nilotinib

Nilotinib was administered orally at 300 mg BD at approximately 12 hour intervals which had to be taken without food. The capsules were to be swallowed whole with water and no food should have been consumed for at least 2 hours before and at least 1 hour after the dose was taken. Prior to the first dose of nilotinib, patients were required to have an imatinib or dasatinib washout period of at least 3 days. Therapy with nilotinib was to be continued for up to 24 months while on study.

Group Type: EXPERIMENTAL

Nilotinib

Intervention Type: DRUG

Nilotinib 150mg hard gelatin capsules taken orally

Interventions

Nilotinib

Nilotinib 150mg hard gelatin capsules taken orally

Intervention Type: DRUG

Other Intervention Names

AMN107

Eligibility Criteria

Inclusion Criteria

1. Written informed consent prior to screening procedures

2. Eastern Cooperative Oncology Grou (ECOG) Performance Status of 0, 1, or 2.

3. Patient with diagnosis of Ph+ CML-CP associated with BCR-ABL quantifiable by RQ-PCR (IS).

4. Patient has received a minimum of 3 months of imatinib or dasatinib treatment (any dose) since initial diagnosis with a documented response.

5. Patient is eligible for Pharmaceutical Benefits Scheme (PBS) reimbursed 1st line TKI treatment.

6. Patient has experienced non-hematological Adverse Events (AE(s)) of any grade, which persisted for at least 1 month despite supportive care or recurred at any grade at least once. Patients who, at the Investigator's discretion, require immediate discontinuation due to the severity of the adverse event are also eligible.

7. No other current or planned anti-leukemia therapies.

8. Adequate organ function.

9. Potassium, Magnesium and Total Calcium above Lower limit of normal.

10. life expectancy of more than 12 months in the absence of any intervention

Exclusion Criteria

1. Prior treatment with nilotinib.

2. Prior Accelerated Phase (AP), Blast Crisis (BC) or allogeneic-transplant (unless the patient received an autologous transplant and was in Chrionic Phase (CP) prior to transplant and never in AP or BC).

3. Patient has documented Molecular Response (MR) 4.5 at the time of study entry

4. Patients with atypical BCR-ABL transcript not quantifiable by standard RQ-PCR.

5. Known impaired cardiac function.

6. Impairment of gastrointestinal (GI) function or GI disease that may significantly alter the absorption of study drug.

7. Pregnant or breast feeding (lactating) women.

8. Women of child-bearing potential unwilling or unable to use highly effective contraception.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Novartis Pharmaceuticals

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Novartis Pharmaceuticals

Role: STUDY_DIRECTOR

Novartis Pharmaceuticals

Locations

Novartis Investigative Site

Canberra, Australian Capital Territory, Australia

Novartis Investigative Site

Kingswood, New South Wales, Australia

Novartis Investigative Site

Kogarah, New South Wales, Australia

Novartis Investigative Site

Liverpool, New South Wales, Australia

Novartis Investigative Site

St Leonards, New South Wales, Australia

Novartis Investigative Site

Douglas, Queensland, Australia

Novartis Investigative Site

Nambour, Queensland, Australia

Novartis Investigative Site

South Brisbane, Queensland, Australia

Novartis Investigative Site

Adelaide, South Australia, Australia

Novartis Investigative Site

Fitzroy, Victoria, Australia

Novartis Investigative Site

Geelong, Victoria, Australia

Novartis Investigative Site

Melbourne, Victoria, Australia

Novartis Investigative Site

Murdoch, Western Australia, Australia

Novartis Investigative Site

Nedlands, Western Australia, Australia

Countries

Australia

Other Identifiers

CAMN107AAU04

Identifier Type: -

Identifier Source: org_study_id