Phase 2 N=69 Randomized Treatment

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

Acute Leukemias

Bottom Line

View on ClinicalTrials.gov: NCT01826214 ↗

Enrolled (actual)

Serious AEs

72.5%

Results posted

May 2016

Primary outcome: Primary: Rate of Complete Remission (CR) — 0; 0 Participants

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: LDE225 (Drug)
Age: Adult, Older Adult · 18+ yrs
Sex: All
Sponsor: Novartis Pharmaceuticals
Primary completion: May 2015

Outcome Measures

Outcome	Result	p-value
PRIMARY Rate of Complete Remission (CR)	0; 0	—
PRIMARY Complete Remission With Incomplete Blood Count Recovery (CRi)	1; 0	—
SECONDARY Overall Response Rate (ORR)	1; 0	—
SECONDARY Parmacokintics (PK) Parameter: Cmax	237; 343; 1640; 1500	—
SECONDARY Parmacokintics (PK) Parameter: Tmax	2.13; 2.12; 1.88; 2.02	—
SECONDARY Parmacokintics (PK) Parameter: AUC0-8h	988; 1560; 9750; 7910	—
SECONDARY Parmacokintics (PK) Parameter: AUC0-24h	0; 3110; 26500; 24000	—

Summary

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.

Eligibility Criteria

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

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT01826214). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.