Clinical trial NCT01844765
Conditions: Philadelphia Positive (Ph+) Chronic Myelogenous Leukemia
Keywords: Tasigna nilotinib treatment chronic phase Ph+ CML accelerated phase newly diagnosed Ph+ CML pediatric leukemia leukemia, pediatric leukemia, myleiod leukemia, mylegenous, chronic leukemia, mylegenous, accelerated BCR-ABL positive myeloproliferative disorder bone marrow disease hematologic diseases neoplastic processes imatinib dasatinib enzyme inhibitor protein kinase inhibitor
Sponsor: Novartis Pharmaceuticals
Start date: 2013-08-20; Completion date: 2020-08-28
Enrollment: 59 participants; Allocation: NON_RANDOMIZED; Intervention model: SINGLE_GROUP

Brief 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).

Detailed description:
The study was designed as a multi-center, open-label, non-controlled phase II study to assess efficacy, safety and PK parameters of 230 mg/m2 twice daily nilotinib in pediatric patients (1 to \<18 years old). The study population consisted of three cohorts of Ph+ CML pediatric patients:

* Cohort 1: Ph+ CML-CP patients resistant or intolerant to either imatinib or dasatinib
* Cohort 2: Ph+ CML-AP patients resistant or intolerant to either imatinib or dasatinib
* Cohort 3: Newly-diagnosed Ph+ CML-CP patients in first chronic phase A minimum number of 50 pediatric patients (from 1 to \<18 years) were enrolled in the study. Of them, at least 15 patients were Ph+ CML-CP patients resistant or intolerant to either imatinib or dasatinib, and at least 15 were newly-diagnosed Ph+ CML-CP patients in first chronic phase patients. There was no minimum number of patients required for Ph+ CML-AP patients resistant or intolerant to either imatinib or dasatinib.

Based on enrollment forecasts as of Jan 2015, and to reflect the agreements with the US FDA and the PDCO, the study remained open for enrollment until the targeted number of 50 patients with at least 15 newly diagnosed Ph+CML patients was achieved or until 31May2015, whichever was later.

Patients who completed the study were treated with nilotinib for a total of 66 cycles of 28 days unless the patient prematurely discontinued study treatment.

The primary analysis cut-off date was the date when all patients enrolled in the trial either completed their visit for treatment cycle 12 or had discontinued study treatment early (EoT/early discontinuation visit). These analyses were reported in the 12-cycle clinical study report (CSR). A 24-cycle analysis was done when all patients had either completed their 24-cycle treatment visit or had discontinued study treatment early.

At trial end, a final comprehensive CSR of all data collected during the trial was produced.

Arms: arm 1: Diagnosis within 6 months of date of first cytogenetic analysis confirming Philadelphia chromosome with (9;22) translocation by standard conventional cytogenetic analysis. arm 2: Resistant or Intolerant to either imatinib or dasatinib arm 3: Resistant or intolerant to either imatinib or dasatinib - at the end no patients were enrolled in this arm.
Interventions: intervention 1: nilotinib
Intervention descriptions: intervention 1: Nilotinib supplied in 50mg, 150mg, and 200mg capsules. It was administered orally at 230mg/m2, twice daily for up to 66 cycles (1 cycle = 28 days). Dose administration was rounded to the nearest 50mg dose (to a maximum dose of 400mg).

Protocol documents: