Clinical trial NCT01826214: Study of Efficacy and Safety of LDE225 in Adult Patients With Relapsed/Refractory Acute Leukemia
Official title: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Acute Leukemias
Keywords: acute myeloid leukemia
AML
acute lymphoblastic leukemia
ALL
leukemia
acute
LDE225
adult patients
relapsed/refractory
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2013-04-08
Enrollment: 70
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief 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

Primary outcomes:
measure: Rate of Complete Remission (CR)
timeFrame: at screening, every week up to Week 9, every 2 weeks thereafter until CR, every 4 weeks after CR up to 24 months
measure: Complete Remission With Incomplete Blood Count Recovery (CRi)
timeFrame: within 3 days after clearance of blasts from peripheral blood (PB), monthly thereafter until CR or reappearance of blasts in the PB, after CR every other month until discontination up to 24 months
Secondary outcomes:
measure: Overall Response Rate (ORR)
timeFrame: Every 8 weeks for the first 6 months and every 12 weeks until 53 weeks after the last patient is enrolled or until relapse up to 24 months
measure: Parmacokintics (PK) Parameter: Cmax
timeFrame: Week 1 Day 1, Week 9 Day 1
measure: Parmacokintics (PK) Parameter: Tmax
timeFrame: Week 1 Day 1,Week 9 Day 1
measure: Parmacokintics (PK) Parameter: AUC0-8h
timeFrame: Week 1 Day 1,Week 9 Day 1
measure: Parmacokintics (PK) Parameter: AUC0-24h
timeFrame: Week 1 Day 1,Week 9 Day 1
Other outcomes:
Locations:
city: Durham
country: United States
facility: Duke University Medical Center SC-5
state: North Carolina
city: Adelaide
country: Australia
facility: Novartis Investigative Site
state: South Australia
city: Prahran
country: Australia
facility: Novartis Investigative Site
state: Victoria
city: Salzburg
country: Austria
facility: Novartis Investigative Site
city: Wien
country: Austria
facility: Novartis Investigative Site
city: Leuven
country: Belgium
facility: Novartis Investigative Site
city: Yvoir
country: Belgium
facility: Novartis Investigative Site
city: Montreal
country: Canada
facility: Novartis Investigative Site
state: Quebec
city: Montreal
country: Canada
facility: Novartis Investigative Site
state: Quebec
city: Dresden
country: Germany
facility: Novartis Investigative Site
city: Frankfurt
country: Germany
facility: Novartis Investigative Site
city: Magdeburg
country: Germany
facility: Novartis Investigative Site
city: Ulm
country: Germany
facility: Novartis Investigative Site
city: Debrecen
country: Hungary
facility: Novartis Investigative Site
city: Amsterdam
country: Netherlands
facility: Novartis Investigative Site
city: Nijmegen
country: Netherlands
facility: Novartis Investigative Site
city: Rotterdam
country: Netherlands
facility: Novartis Investigative Site
city: Rotterdam
country: Netherlands
facility: Novartis Investigative Site
city: Bergen
country: Norway
facility: Novartis Investigative Site
city: Trondheim
country: Norway
facility: Novartis Investigative Site
city: Salamanca
country: Spain
facility: Novartis Investigative Site
state: Castilla y Leon
city: Valencia
country: Spain
facility: Novartis Investigative Site
state: Comunidad Valenciana
city: London
country: United Kingdom
facility: Novartis Investigative Site
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: