A Phase II multi-center, open label, randomized study to assess safety and efficacy of two different schedules of oral LDE225 in adult patients with relapsed/refractory or untreated elderly patients with acute leukemia
- Conditions
- bloodcancertoo much cells a a certain kind in the blood10018865
- Registration Number
- NL-OMON40210
- Lead Sponsor
- ovartis
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 12
- Male or female adult patients (*18 years).;- Histological or cytological diagnosis of either relapsed or primary refractory non-M3 acute myeloid leukemia (AML); untreated AML in patients * 65 years of age, if they are not candidates for standard induction chemotherapy; or have failed alternative therapies (such as
decitabine, azacitidine, etc.) relapsed or refractory non-T-cell acute lymphoblastic leukemia (ALL);- White blood cell count (WBC) * 50 x 109/L. ;- Performance status corresponding to ECOG (WHO) score of 0, 1 or 2.;- Adequate renal function ;- Adequate liver function;- Serum CK * 1.5 x ULN.;- At least 2 weeks since end of last leukemia therapy.;- Written informed consent must be obtained prior to any screening procedures.
- Allogeneic SCT within the last 4 months and/or active GVHD which requires systemic immunosuppressant therapy, or autologous SCT within the last 4 weeks.;- Patient for which immediate allogeneic SCT is the treatment of choice.;- Patients with a life threatening or uncontrolled systemic infection.;- Active CNS leukemic involvement.;- Major surgery within 2 weeks of initiation of study medication.;- Concurrent uncontrolled medical conditions that may interfere or potentially affect the interpretation of the study.;- Unable to take oral drugs, or lack of physical integrity of the upper gastrointestinal tract, or known malabsorption syndromes.;- Patients with unresolved diarrhea > CTCAE grade 2.;- Patients who have previously been treated with systemic LDE225 or with other Hh pathway inhibitors.;- Patients who have neuromuscular disorders or are on concomitant treatment with drugs that are recognized to cause rhabdomyolysis;- patients who are planning on embarking on new physical activities, such as strenuous exercise, that can result in significant increases in plasma CK levels while on study treatment. ;- Patient has history of cardiac dysfunction ;- patient has active cardiac disease ;- Use of other investigational drugs within 30 days or 5 half-lives of initiation of study medication, whichever is longer.;- Patients who are receiving treatment with medications known to be moderate and strong inhibitors or inducers of CYP3A4/5 or drugs metabolized by CYP2B6 or CYP2C9 that have narrow therapeutic index, and that cannot be discontinued before starting treatment with LDE225. ;- Patients are excluded if the use of warfarin is necessary and cannot be substituted (Warfarin is not used in The Netherlands);- Pregnant or nursing (lactating) women;- Patients who are not willing to apply highly effective contraception during the study and the defined duration after final dose of study treatment.;- Known HIV positivity.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>To evaluate the rate of complete remission (CR) and complete remission with<br /><br>incomplete blood count recovery (CRi) on two different dosing<br /><br>schedules of LDE225 in acute leukemia</p><br>
- Secondary Outcome Measures
Name Time Method <p>-To evaluate the Overall Response Rate on two different dosing schedules of<br /><br>LDE225 in acute leukemia<br /><br>-To evaluate the safety and tolerability of two different schedules of LDE225<br /><br>in acute leukemia<br /><br>-To further characterize the pharmacokinetics of two different schedules of<br /><br>LDE225 in acute leukemia</p><br>