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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/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
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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
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Secondary ID [1]
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CLDE225X2203
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Acute Leukemias
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Condition category
Condition code
Cancer
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Leukaemia - Acute leukaemia
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Cancer
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Leukaemia - Chronic leukaemia
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Cancer
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Children's - Leukaemia & Lymphoma
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Other
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Research that is not of generic health relevance and not applicable to specific health categories listed above
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Intervention/exposure
Study type
Interventional
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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.
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Rate of Complete Remission (CR)
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Assessment method [1]
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Complete Response (CR) was based on the International Working Group (IWG) criteria based on weekly peripheral blood count measurements and bone marrow biopsy/aspiration collection. Efficacy assessments were performed to determine CR. A treatment cycle was defined as 4 weeks. The outcome measure for the study is based on standardized response criteria as defined by the International Working Group (IWG) for AML. The IWG was established by a group of investigators interested in the design and conduct of clinical trials in acute myeloid leukemia (AML). The criteria established by this group (a set of recommendations for response assessment) are well established, endorsed by major institutions and Health Authorities, and are widely used in clinical trials. No statistical analysis was planned for this primary outcome.
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Timepoint [1]
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at screening, every week up to Week 9, every 2 weeks thereafter until CR, every 4 weeks after CR up to 24 months
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Primary outcome [2]
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Complete Remission With Incomplete Blood Count Recovery (CRi)
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Assessment method [2]
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The other primary efficacy endpoint was CRi based on the International Working Group (IWG) criteria based on weekly peripheral blood count measurements and bone marrow biopsy/aspiration collection. Efficacy assessments were performed to determine CRi. A treatment cycle was defined as 4 weeks. No statistical analysis was planned for this primary outcome.
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Timepoint [2]
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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
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Secondary outcome [1]
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Overall Response Rate (ORR)
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Assessment method [1]
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ORR was the rate of complete remission (CR), complete remission with incomplete blood count recovery (CRi) or partial response (PR) according to IWG criteria. CR, CRi or PR will be assessed through bone marrow biopsy/aspirate and peripheral blood blasts counts.
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Timepoint [1]
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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
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Secondary outcome [2]
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Parmacokintics (PK) Parameter: Cmax
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Assessment method [2]
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Cmax is the maximum observed plasma concentration after drug administration.The PK parameters were determined in plasma using non-compartmental methods. A PK sample was excluded from analyses if the patient vomited within the first 4 hours following the last oral dose of study drug. Other PK samples were excluded as deemed appropriate by the pharmacokineticist. Cmax was derived from the PK concentrations collected at 0, 0.5, 1, 2, 4, 6, 8 and 24 hours post dose on W1D1 and W9D1. The PK concentration at each time point is not an endpoint in the protocol, but these concentrations are used to derive the PK parameters.
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Timepoint [2]
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Week 1 Day 1, Week 9 Day 1
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Secondary outcome [3]
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Parmacokintics (PK) Parameter: Tmax
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Assessment method [3]
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Tmax is the time to reach Cmax. The PK parameters were determined in plasma using non-compartmental methods. A PK sample was excluded from analyses if the patient vomited within the first 4 hours following the last oral dose of study drug. Other PK samples were excluded as deemed appropriate by the Pharmacokineticist. Tmax was derived from the PK concentrations collected at 0, 0.5, 1, 2, 4, 6, 8 and 24 hours post dose on W1D1 and W9D1. The PK concentration at each time point is not an endpoint in the protocol, but these concentrations are used to derive the PK parameters.
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Timepoint [3]
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Week 1 Day 1,Week 9 Day 1
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Secondary outcome [4]
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Parmacokintics (PK) Parameter: AUC0-8h
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Assessment method [4]
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AUC0-8h is the area under the concentration-time curve from time zero to 8 hours. The PK parameters were determined in plasma using non-compartmental methods. A PK sample was excluded from analyses if the patient vomited within the first 4 hours following the last oral dose of study drug. Other PK samples were excluded as deemed appropriate by the Pharmacokineticist. AUC0-8h was derived from the PK concentrations collected at 0, 0.5, 1, 2, 4, 6, 8 and 24 hours post dose on W1D1 and W9D1. The PK concentration at each time point is not an endpoint in the protocol, but these concentrations are used to derive the PK parameters.
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Timepoint [4]
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Week 1 Day 1,Week 9 Day 1
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Secondary outcome [5]
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Parmacokintics (PK) Parameter: AUC0-24h
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Assessment method [5]
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AUC0-24 is the area under the concentration-time curve from time zero to 24 hours done only on 800 mg once a day schedule. The PK parameters were determined in plasma using non-compartmental methods. A PK sample was excluded from analyses if the patient vomited within the first 4 hours following the last oral dose of study drug. Other PK samples were excluded as deemed appropriate by the Pharmacokineticist. AUC0-24h was derived from the PK concentrations collected at 0, 0.5, 1, 2, 4, 6, 8 and 24 hours post dose on W1D1 and W9D1. The PK concentration at each time point is not an endpoint in the protocol, but these concentrations are used to derive the PK parameters.
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Timepoint [5]
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Week 1 Day 1,Week 9 Day 1
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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)
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Minimum age
18
Years
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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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
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 2
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
1/05/2013
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
1/05/2015
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Sample size
Target
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Accrual to date
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Final
70
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Recruitment in Australia
Recruitment state(s)
SA,VIC
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Recruitment hospital [1]
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Novartis Investigative Site - Adelaide
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Recruitment hospital [2]
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Novartis Investigative Site - Prahran
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Recruitment postcode(s) [1]
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5000 - Adelaide
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Recruitment postcode(s) [2]
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3181 - Prahran
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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North Carolina
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Austria
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State/province [2]
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Salzburg
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Austria
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Wien
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Belgium
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State/province [4]
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Leuven
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Belgium
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Yvoir
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Canada
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Quebec
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Germany
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Dresden
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Germany
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Frankfurt
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Country [9]
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Germany
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State/province [9]
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Magdeburg
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Germany
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State/province [10]
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Ulm
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Hungary
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State/province [11]
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Debrecen
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Netherlands
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State/province [12]
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Amsterdam
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Netherlands
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State/province [13]
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Nijmegen
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Netherlands
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State/province [14]
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Rotterdam
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Norway
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State/province [15]
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Bergen
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Norway
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State/province [16]
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Trondheim
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Spain
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State/province [17]
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Castilla y Leon
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Spain
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State/province [18]
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Comunidad Valenciana
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Country [19]
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United Kingdom
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State/province [19]
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London
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Novartis Pharmaceuticals
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Address
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Country
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Ethics approval
Ethics application status
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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.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT01826214
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Novartis Pharmaceuticals
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Address
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Novartis Pharmaceuticals
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Address
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Fax
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Email
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT01826214
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