Back to resultsA Study to Find a Safe Dose of Volasertib Given in Addition to Standard Salvage Chemotherapy in Children (Age 3 Months to Less Than 18 Years) With Acute Myeloid Leukaemia, in Whom Front-line Chemotherapy Failed
Primary Purpose
Leukemia, Myeloid, Acute
Status
Withdrawn
Phase
Phase 1
Locations
Belgium
Study Type
Interventional
Intervention
Volasertib
Sponsored by
About this trial
This is an interventional treatment trial for Leukemia, Myeloid, Acute
Eligibility Criteria
Inclusion criteria:
- Patients 3 months to <18 years of age at the time of informed consent
- Patients with AML after failure of the front-line intensive AML therapy
- Lansky score at screening >=50 for patients from 3 months to <12 years
- Karnofsky score at screening >=50 for patients from 12 to <18 years
- Use of highly effective methods of birth-control, if sexually active
- Parents/legal guardians and patients have given written informed consent and informed assent suitable for the respective age group
Exclusion criteria:
- Down syndrome
- Acute promyelocytic leukaemia and treatment-related AML
- QTc prolongation
- LVSF <30%
- Cardiac disease and/or dysfunction
- Active uncontrolled infection
- HIV infection, acute or chronic hepatitis
- Inadequate lab parameters
- Impaired renal function
- Pregnancy or nursing
- Further exclusion criteria apply
Sites / Locations
- Boehringer Ingelheim Investigational Site
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Volasertib
Arm Description
Outcomes
Primary Outcome Measures
Determination of the maximal tolerated dose of volasertib or the recommended volasertib dose for further studies in combination with standard salvage therapy in paediatric patients with AML after failure of the front-line intensive chemotherapy regimen
Secondary Outcome Measures
Anti-leukaemic activity of volasertib in combination with standard salvage therapy
Event-free survival (EFS)
Overall survival (OS)
Number of patients with clinically relevant lab value changes of calcium (hyper- and/or hypocalcaemia) as judged by the investigator and reported as adverse events (Common Terminology Criteria for Adverse Events (CTCAE) grade 3 or higher)
Number of patients with changes in cardiac activity (prolonged QTc interval) reported as clinically relevant observations (i.e. Adverse Events)
Predose concentration of volasertib before administration of second dose
Area under the concentration-time curve of volasertib
Terminal half-life of volasertib in plasma
Maximum concentration of volasertib
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT02722135
Brief Title
A Study to Find a Safe Dose of Volasertib Given in Addition to Standard Salvage Chemotherapy in Children (Age 3 Months to Less Than 18 Years) With Acute Myeloid Leukaemia, in Whom Front-line Chemotherapy Failed
Official Title
Open-label, Dose-escalating Trial to Evaluate the Tolerability, Toxicity, Safety, Pharmacokinetics, Pharmacodynamics and Activity of Volasertib Added to the Standard Intensive Salvage Chemotherapy Regimen With Liposomal Daunorubicine, Fludarabine and Cytarabine (DNX-FLA) Followed by Fludarabine and Cytarabine (FLA) in Children From 3 Months to Less Than 18 Years of Age With Acute Myeloid Leukaemia After Failure of the Front-line Therapy
Study Type
Interventional
2. Study Status
Record Verification Date
January 2017
Overall Recruitment Status
Withdrawn
Study Start Date
November 2016 (undefined)
Primary Completion Date
May 2018 (Anticipated)
Study Completion Date
September 2018 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Boehringer Ingelheim
4. Oversight
5. Study Description
Brief Summary
This is an open-label, dose-escalating trial to evaluate the MTD and/or dose to be used for further development by evaluation of DLT in course 1 and the safety of volasertib when added to standard intensive salvage chemotherapy with DNX-FLA in paediatric patients with AML after failure of first-line therapy. Furthermore, data on efficacy and PK/PD of volasertib in paediatric patients with AML when added to standard intensive salvage chemotherapy will be collected.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Leukemia, Myeloid, Acute
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
0 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Volasertib
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
Volasertib
Primary Outcome Measure Information:
Title
Determination of the maximal tolerated dose of volasertib or the recommended volasertib dose for further studies in combination with standard salvage therapy in paediatric patients with AML after failure of the front-line intensive chemotherapy regimen
Time Frame
4 weeks
Secondary Outcome Measure Information:
Title
Anti-leukaemic activity of volasertib in combination with standard salvage therapy
Time Frame
8 weeks
Title
Event-free survival (EFS)
Time Frame
up to 5 years
Title
Overall survival (OS)
Time Frame
up to 5 years
Title
Number of patients with clinically relevant lab value changes of calcium (hyper- and/or hypocalcaemia) as judged by the investigator and reported as adverse events (Common Terminology Criteria for Adverse Events (CTCAE) grade 3 or higher)
Time Frame
8 weeks
Title
Number of patients with changes in cardiac activity (prolonged QTc interval) reported as clinically relevant observations (i.e. Adverse Events)
Time Frame
8 weeks
Title
Predose concentration of volasertib before administration of second dose
Time Frame
8 weeks
Title
Area under the concentration-time curve of volasertib
Time Frame
8 weeks
Title
Terminal half-life of volasertib in plasma
Time Frame
8 weeks
Title
Maximum concentration of volasertib
Time Frame
8 weeks
10. Eligibility
Sex
All
Minimum Age & Unit of Time
3 Months
Maximum Age & Unit of Time
17 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion criteria:
Patients 3 months to <18 years of age at the time of informed consent
Patients with AML after failure of the front-line intensive AML therapy
Lansky score at screening >=50 for patients from 3 months to <12 years
Karnofsky score at screening >=50 for patients from 12 to <18 years
Use of highly effective methods of birth-control, if sexually active
Parents/legal guardians and patients have given written informed consent and informed assent suitable for the respective age group
Exclusion criteria:
Down syndrome
Acute promyelocytic leukaemia and treatment-related AML
QTc prolongation
LVSF <30%
Cardiac disease and/or dysfunction
Active uncontrolled infection
HIV infection, acute or chronic hepatitis
Inadequate lab parameters
Impaired renal function
Pregnancy or nursing
Further exclusion criteria apply
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Boehringer Ingelheim
Organizational Affiliation
Boehringer Ingelheim
Official's Role
Study Chair
Facility Information:
Facility Name
Boehringer Ingelheim Investigational Site
City
Gent
Country
Belgium
12. IPD Sharing Statement
Learn more about this trial
A Study to Find a Safe Dose of Volasertib Given in Addition to Standard Salvage Chemotherapy in Children (Age 3 Months to Less Than 18 Years) With Acute Myeloid Leukaemia, in Whom Front-line Chemotherapy Failed
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