Study of Efficacy and Safety of LDE225 in Adult Patients With Relapsed/Refractory Acute Leukemia

Novartis

Status and phase: Completed; Phase 2

Conditions: Acute Leukemias

Treatments: Drug: LDE225

Study type: Interventional

Funder types: Industry

Identifiers

NCT01826214

CLDE225X2203

Details and patient eligibility

About

The study will evaluate the efficacy, safety and tolerability of two dosing schedules of LDE225 in patients with relapsed/refractory acute leukemia or elderly patients with untreated acute leukemia.

Enrollment: 70 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Subjects must have relapsed or primary refractory non-M3 acute myeloid leukemia or relapsed or refractory non-T-cell acute lymphoblastic leukemia or untreated acute myeloid leukemia in elderly patients.
• Performance status of 0, 1 or 2 per WHO classification.
• Adequate renal and liver function.
• Adequate blood creatine kinase value (CK < 1.5ULN)

Exclusion criteria

• Allogeneic stem cell transplantation within the last 4 months and/or active graft versus host disease requiring systemic immunosuppressant therapy, or autologous stem cell transplantation within the last 4 weeks.
• Patient for which immediate allogeneic stem cell transplantation is the treatment of choice.
• Pregnant or nursing (lactating) women.
• Active CNS leukemic involvement

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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70 participants in 2 patient groups

LDE225-400

Experimental group

Description:

Patients who were randomized to Schedule A, and received 400 mg LDE225 twice daily for the first two weeks only and then after two weeks, received 800 mg LDE225 once daily until disease progression, toxicity, withdrawal of consent, death, discretion of the investigator or early termination of the study.

Treatment:

Drug: LDE225

LDE225-800

Experimental group

Description:

Patients who were randomized to Schedule B, received 800 mg LDE225 once daily until disease progression, toxicity, withdrawal of consent, death, discretion of the investigator or early termination of the study.

Treatment:

Drug: LDE225