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

Novartis

Status and phase: Completed; Phase 1

Conditions: Chronic Myeloid Leukemia; Acute Lymphoblastic Leukemia

Treatments: Drug: Nilotinib

Study type: Interventional

Funder types: Industry

Identifiers

NCT01077544

CAMN107A2120

2010-018419-14 (EudraCT Number)

Details and patient eligibility

About

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.

Enrollment: 15 patients

Sex: All

Ages: 1 to 17 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• 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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

---

15 participants in 2 patient groups

Group 1

Experimental group

Description:

1 year to \< 10 years pediatric patients with newly diagnosed CP-Ph+ CML, or CP or AP-Ph+ CML resistant/intolerant to imatinib and/or dasatinib, or relapsed/refractory Ph+ ALL (acute lymphoblastic leukemia) treated at the proposed dose of 230 mg/m2 bid.

Treatment:

Drug: Nilotinib

Group 2

Experimental group

Description:

\>= 10 years to \<18 years pediatric patients with newly diagnosed CP-Ph+ CML, or CP or AP-Ph+ CML resistant/intolerant to imatinib and/or dasatinib, or relapsed/refractory Ph+ ALL (acute lymphoblastic leukemia) treated at the proposed dose of 230 mg/m2 bid.

Treatment:

Drug: Nilotinib