Open Label, Phase II Study to Evaluate Efficacy and Safety of Oral Nilotinib in Philadelphia Positive (Ph+) Chronic Myelogenous Leukemia (CML) Pediatric Patients.

• Status: Completed
• Trial end date: 2020-08-28
• Target enrollment: 0
• Participant gender: All

Summary

To evaluate the safety, efficacy and pharmacokinetics of nilotinib over time in the Ph+ chronic myelogenous leukemia (CML) in pediatric patients (from 1 to <18 years).

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Novartis Pharmaceuticals
• Collaborators: Children's Oncology Group; Innovative Therapies For Children with Cancer Consortium

Criteria

Key Inclusion Criteria:

• Newly diagnosed and untreated Ph+ CML CP or Ph+ CML CP or AP resistant or intolerant to either imatinib or dasatinib

• Karnofsky ≥ 50% for patients > 10 years of age and Lansky ≥ 50 for patients ≤ 10 years of age

• Adequate renal, hepatic and pancreatic function

• Potassium, magnesium, phosphorus and total calcium values ≥ LLN (lower limit of normal)

• Written informed consent

Key Exclusion Criteria:

• Treatment with strong CYP3A4 inhibitors or inducers

• Use or planned use of any medications that have a known risk or possible risk to prolong the QT interval

• Acute or chronic liver, pancreatic or severe renal disease

• History of pancreatitis or chronic pancreatitis.

• Impaired cardiac function

• No evidence of active graft vs host and <3mo since Stem Cell Transplant

• Total body irradiation (TBI) or craniospinal radiation therapy <6months

• Hypersensitivity to the active ingredient or any of the excipients including lactose.

• the criteria regarding pregnancy and contraception

• Active or systemic bacterial, fungal, or viral infection

• known Hepatitis B, Hepatitis C, or HIV infection