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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT02147873
Registration number
NCT02147873
Ethics application status
Date submitted
13/05/2014
Date registered
28/05/2014
Date last updated
20/10/2016
Titles & IDs
Public title
Study of Azacitidine With or Without Birinapant in Subjects With MDS or CMMoL
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Scientific title
A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study of Azacitidine With or Without Birinapant With a Single Arm Open-Label Run-In Phase in Subjects With Higher Risk Myelodysplastic Syndrome or Chronic Myelomonocytic Leukemia
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Secondary ID [1]
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2014-001719-37
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Secondary ID [2]
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TL32711-RAN-0094-PTL
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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:
Myelodysplastic Syndrome (MDS)
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Chronic Myelomonocytic Leukemia (CMML)
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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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Blood
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Haematological diseases
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Blood
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Other blood disorders
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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
Active comparator: azacitidine with birinapant - Azacitidine 75 mg/m2 IV on days 1-5, 8 \& 9 OR days 1-7 and birinapant 13 mg/m2 IV twice a week (days 1 \& 4) for 3 out of 4 weeks
Placebo comparator: Azacitidine and placebo - Azacitidine 75mg/m2 IV days 1-5, 8 \& 9 OR days 1-7 and placebo IV twice a week (days 1 \& 4) for 3 out of 4 weeks
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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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Response Rate
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Assessment method [1]
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Timepoint [1]
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participants will be followed for until disease progression an expected average of 1 year
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Secondary outcome [1]
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Hematologic improvement
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Assessment method [1]
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Timepoint [1]
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participants will be followed for until disease progression an expected average of 1 year
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Secondary outcome [2]
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Relapse free survival
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Assessment method [2]
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According to modified IWG 2006 criteria
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Timepoint [2]
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An expected average of 2 year post last study dose
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Secondary outcome [3]
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Time to respond
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Assessment method [3]
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Timepoint [3]
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participants will be followed for until disease progression an expected average of 1 year
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Secondary outcome [4]
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Change in transfusion requirements
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Assessment method [4]
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Timepoint [4]
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participants will be followed for until disease progression an expected average of 1 year
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Secondary outcome [5]
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duration of response
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Assessment method [5]
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According to modified IWG 2006 criteria
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Timepoint [5]
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participants will be followed for until disease progression an expected average of 1 year
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Secondary outcome [6]
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overall survival
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Assessment method [6]
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Timepoint [6]
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An expected average of 2 year post last study dose
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Secondary outcome [7]
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Adverse events profile
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Assessment method [7]
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Timepoint [7]
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participants will be monitored for adverse events throughout the treatment period and during follow up period
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Eligibility
Key inclusion criteria
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* Morphologically confirmed diagnosis of MDS/CMMoL according to FAB or WHO classification, including RAEB-t and MDS/MPN
* International prognostic score-revised (IPSS-R) of >3.5 (Intermediate, High or Very High)
* Previously untreated with hypomethylating agents for MDS/CMMoL
* Performance status of 0, 1 or 2 by the ECOG scale
* Adequate renal and liver function
* Female subjects of childbearing potential must have a negative serum pregnancy test at screening within 96 hours prior to the first study dose.
* Female subjects of childbearing potential and male subjects with partners of childbearing potential should ensure use of a highly effective method of birth control as defined by the investigator, for example, those which result in a low failure rate (i.e., less than 1% per year) when used consistently and correctly during the study and for a period of 3 months following the last dose of any drug administered during the study.
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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
* Relapsed or refractory to hypomethylating agents
* Acute myeloid leukemia (AML), except those patients with RAEB-t who are not candidates for intensive AML therapy.
* Participated in any interventional study within 4 weeks of randomization or 5 half lives (whichever is longer).
* Received any hematopoietic growth factors within 14 days prior to screening.
* Prior malignancy or secondary malignancy within the prior 2 years (except in situ cervical cancer, squamous cell carcinoma or basal cell carcinoma of the skin).
* known diagnosis of human immunodeficiency virus or chronic active Hep B or C.
* Uncontrolled hypertension
* Impaired cardiac function, uncontrolled cardiac arrhythmias despite medications, or clinically significant cardiac disease
* Lack of recovery of prior adverse events to Grade =1 severity (National Cancer Institute Common Terminology Criteria for Adverse Events version 4) (except alopecia) due to therapy administered prior to the initiation of study drug dosing.
* Nursing or pregnant.
* Known allergy or hypersensitivity to any of the formulation components
* Any concurrent disease and/or medical condition that, in the opinion of the Investigator, would prevent the subject's participation.
* History of cranial nerve palsy.
* Being treated with anti-TNF therapies or has been treated with an anti-TNF therapy within 5 half-lives of randomization.
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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
Blinded (masking used)
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Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
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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
Stopped early
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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
1/06/2014
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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
1/06/2016
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Sample size
Target
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Accrual to date
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Final
118
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Recruitment in Australia
Recruitment state(s)
NSW,QLD,SA,TAS,VIC,WA
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Recruitment hospital [1]
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Royal Prince Alfred Hospital - Camperdown
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Westmead Hospital - Westmead
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Metro South Health, Princess Alexandra Hospital - Woolloongabba
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Recruitment hospital [4]
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Royal Adelaide Hospital - Adelaide
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Recruitment hospital [5]
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Royal Hobart Hospital - Hobart
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Recruitment hospital [6]
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Austin Health - Heidelberg
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Recruitment hospital [7]
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Cabrini Hospital - Malvern
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Recruitment hospital [8]
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The Alfred - Melbourne
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Recruitment hospital [9]
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Border Medical Oncology - Wodonga
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Recruitment hospital [10]
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Perth Blood Institute - Nedlands
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Recruitment postcode(s) [1]
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2050 - Camperdown
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Recruitment postcode(s) [2]
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2145 - Westmead
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Recruitment postcode(s) [3]
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4102 - Woolloongabba
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Recruitment postcode(s) [4]
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5000 - Adelaide
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Recruitment postcode(s) [5]
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7000 - Hobart
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Recruitment postcode(s) [6]
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3084 - Heidelberg
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Recruitment postcode(s) [7]
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3144 - Malvern
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Recruitment postcode(s) [8]
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3004 - Melbourne
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Recruitment postcode(s) [9]
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3690 - Wodonga
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Recruitment postcode(s) [10]
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6009 - Nedlands
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Recruitment outside Australia
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United States of America
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Arizona
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United States of America
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California
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Colorado
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Florida
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Illinois
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Iowa
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Kentucky
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Louisiana
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Maryland
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Massachusetts
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Michigan
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New York
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North Carolina
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Ohio
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Pennsylvania
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South Carolina
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Washington
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Germany
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Bayern
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Germany
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Nordrhein-Westfalen
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Germany
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North Rhine Westphalia
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Germany
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Saxony-Anhalt
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Germany
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Essen
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Germany
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Hannover
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Germany
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Heidelberg
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Germany
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Wurzburg
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Madrid
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Murcia
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Navarra
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Badalona
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Cordoba
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Salamanca
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Toledo
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Spain
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Valencia
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
TetraLogic Pharmaceuticals
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Address
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Ethics approval
Ethics application status
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Summary
Brief summary
This is a randomized double blind placebo controlled study of azacitidine with or without birinapant in subjects with higher risk Myelodysplastic syndrome, secondary MDS or myelomonocytic leukemia (CMMoL) who are naïve, to azacitidine therapy. Pre-clinical and mechanistic studies support that azacitidine may modulate pathways that enable birinapant-mediated anti-tumor activity.
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Trial website
https://clinicaltrials.gov/study/NCT02147873
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
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Contact person for scientific queries
No information has been provided regarding IPD availability
What supporting documents are/will be available?
No Supporting Document Provided
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results not provided in
https://clinicaltrials.gov/study/NCT02147873
Download to PDF