NCT01856283

Nilotinib 300 mg BID in Newly Diagnosed CP-CML Patients to Verify Disappearance of CD34+/Lin-Ph+ Cells

An Open Label, Single Arm, Phase II Study of Nilotinib 300 mg BID in Newly Diagnosed CP-CML Patients, in Order to Verify Disappearance of CD34+/Lin-Ph+ Cells From Bone Marrow During Treatment

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 87 (actual)
• Sponsor: Niguarda Hospital · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This is a multicentre, single-arm study of nilotinib 300 mg BID in newly diagnosed patients with CP-CML. This study is designed to establish the disappearance of Ph+ stem cells (CD34+/lin-) in BM during nilotinib treatment. In addition, in this study the investigators aim to perform Gene Expression Profiling (GEP) of CML enrolled patients using Affymetrix GeneChip Instruments and Software Systems, and Affymetrix GeneChip Human Genome U133 Plus 2.0 (whole human transcriptome analysis) at diagnosis and at 3 different time points during treatment with Nilotinib (after 3, 6, 12 months).

Detailed description

This study is designed to establish the disappearance of Ph+ stem cells (CD34+/lin-) in BM during nilotinib treatment. The primary efficacy endpoint is to measure the rate of CD34+/lin-Ph+ cells in the BM after 6 months of treatment. In order to obtain this result, BM blood of all enrolled patients will be stored after 6 months of treatment with nilotinib. The isolated CD34+/lin- cells will be employed for standard FISH analysis. These endpoints will be obtained at the central laboratory of Niguarda Ca' Granda Hospital, Milano, Italy. Secondary endpoints will be reached performing: * the same analyzes on CD34+/lin- cells at diagnosis, at 3 and 12 months of treatment; * cytogenetic analysis to estimate the rate of CCyR at 3, 6 and 12 months; this analysis will be performed at each local laboratory; * molecular analysis to determinate the rate of MR (≤ 10%, ≤ 1%, MMR, MR4,5 IS) at 3, 6 and 12 months in the peripheral blood; the molecular analysis will be performed using the Labnet standardized laboratories in Lombardy.

Conditions

• Leukemia, Myeloid, Chronic-Phase

Interventions

Type	Name	Description
DRUG	Nilotinib 300mg BID	Nilotinib

Timeline

• Start date: 2013-03-01
• Primary completion: 2020-07-01
• Completion: 2021-01-01
• First posted: 2013-05-17
• Last updated: 2021-01-13

Locations

17 sites across 1 country: Italy