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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT01826214




Registration number
NCT01826214
Ethics application status
Date submitted
25/03/2013
Date registered
8/04/2013
Date last updated
30/08/2016

Titles & IDs
Public title
Study of Efficacy and Safety of LDE225 in Adult Patients With Relapsed/Refractory Acute Leukemia
Scientific 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
Secondary ID [1] 0 0
CLDE225X2203
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Acute Leukemias 0 0
Condition category
Condition code
Cancer 0 0 0 0
Leukaemia - Acute leukaemia
Cancer 0 0 0 0
Leukaemia - Chronic leukaemia
Cancer 0 0 0 0
Children's - Leukaemia & Lymphoma
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - LDE225

Experimental: LDE225-400 - 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.

Experimental: LDE225-800 - 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: Drugs: LDE225
LDE225 will be supplied as 200 mg capsules by Novartis. Patients will receive study treatment on an outpatient basis. LDE225 will be dispensed every two weeks for the first four weeks and at the start of every four weeks thereafter, as needed.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Rate of Complete Remission (CR)
Timepoint [1] 0 0
at screening, every week up to Week 9, every 2 weeks thereafter until CR, every 4 weeks after CR up to 24 months
Primary outcome [2] 0 0
Complete Remission With Incomplete Blood Count Recovery (CRi)
Timepoint [2] 0 0
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 outcome [1] 0 0
Overall Response Rate (ORR)
Timepoint [1] 0 0
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
Secondary outcome [2] 0 0
Parmacokintics (PK) Parameter: Cmax
Timepoint [2] 0 0
Week 1 Day 1, Week 9 Day 1
Secondary outcome [3] 0 0
Parmacokintics (PK) Parameter: Tmax
Timepoint [3] 0 0
Week 1 Day 1,Week 9 Day 1
Secondary outcome [4] 0 0
Parmacokintics (PK) Parameter: AUC0-8h
Timepoint [4] 0 0
Week 1 Day 1,Week 9 Day 1
Secondary outcome [5] 0 0
Parmacokintics (PK) Parameter: AUC0-24h
Timepoint [5] 0 0
Week 1 Day 1,Week 9 Day 1

Eligibility
Key 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)
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/05/2013
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/05/2015
Sample size
Target
Accrual to date
Final
70
Recruitment in Australia
Recruitment state(s)
SA,VIC
Recruitment hospital [1] 0 0
Novartis Investigative Site - Adelaide
Recruitment hospital [2] 0 0
Novartis Investigative Site - Prahran
Recruitment postcode(s) [1] 0 0
5000 - Adelaide
Recruitment postcode(s) [2] 0 0
3181 - Prahran
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
North Carolina
Country [2] 0 0
Austria
State/province [2] 0 0
Salzburg
Country [3] 0 0
Austria
State/province [3] 0 0
Wien
Country [4] 0 0
Belgium
State/province [4] 0 0
Leuven
Country [5] 0 0
Belgium
State/province [5] 0 0
Yvoir
Country [6] 0 0
Canada
State/province [6] 0 0
Quebec
Country [7] 0 0
Germany
State/province [7] 0 0
Dresden
Country [8] 0 0
Germany
State/province [8] 0 0
Frankfurt
Country [9] 0 0
Germany
State/province [9] 0 0
Magdeburg
Country [10] 0 0
Germany
State/province [10] 0 0
Ulm
Country [11] 0 0
Hungary
State/province [11] 0 0
Debrecen
Country [12] 0 0
Netherlands
State/province [12] 0 0
Amsterdam
Country [13] 0 0
Netherlands
State/province [13] 0 0
Nijmegen
Country [14] 0 0
Netherlands
State/province [14] 0 0
Rotterdam
Country [15] 0 0
Norway
State/province [15] 0 0
Bergen
Country [16] 0 0
Norway
State/province [16] 0 0
Trondheim
Country [17] 0 0
Spain
State/province [17] 0 0
Castilla y Leon
Country [18] 0 0
Spain
State/province [18] 0 0
Comunidad Valenciana
Country [19] 0 0
United Kingdom
State/province [19] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Novartis Pharmaceuticals
Address
Country

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/study/NCT01826214
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Novartis Pharmaceuticals
Address 0 0
Novartis Pharmaceuticals
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT01826214