Clinical Trial Results:
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
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Summary
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EudraCT number |
2015-004625-14 |
Trial protocol |
DE BE DK CZ FR NL IT |
Global end of trial date |
28 Mar 2016
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Results information
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Results version number |
v1(current) |
This version publication date |
30 Sep 2018
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First version publication date |
30 Sep 2018
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Other versions |
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Summary report(s) |
Statement |
Trial Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
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Trial identification
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Sponsor protocol code |
01230.28
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
- | ||
WHO universal trial number (UTN) |
- | ||
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Sponsors
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Sponsor organisation name |
Boehringer Ingelheim
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Sponsor organisation address |
Binger Strasse 173, Ingelheim am Rhein, Germany, 55216
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Public contact |
QRPE Processes and Systems Coordination, Clinical Trial
Information Disclosure,, QRPE Processes and Systems Coordination, Clinical Trial
Information Disclosure,, +1 8002430127, clintriage.rdg@boehringer-ingelheim.com
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Scientific contact |
QRPE Processes and Systems Coordination, Clinical Trial
Information Disclosure,, Boehringer Ingelheim, +1 8002430127, clintriage.rdg@boehringer-ingelheim.com
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Paediatric regulatory details
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Is trial part of an agreed paediatric investigation plan (PIP) |
No
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Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Results analysis stage
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Analysis stage |
Final
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Date of interim/final analysis |
28 Mar 2016
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Is this the analysis of the primary completion data? |
No
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Global end of trial reached? |
Yes
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Global end of trial date |
28 Mar 2016
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Was the trial ended prematurely? |
No
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General information about the trial
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Main objective of the trial |
To define 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
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Protection of trial subjects |
99999 is "Not applicable" value or 0 participants, this trial was discontinued with no participants enrolled in the trial
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Background therapy |
- | ||
Evidence for comparator |
- | ||
Actual start date of recruitment |
28 Mar 2016
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Long term follow-up planned |
No
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Independent data monitoring committee (IDMC) involvement? |
No
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Population of trial subjects
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Number of subjects enrolled per country |
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Country: Number of subjects enrolled |
Netherlands: 99999
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Worldwide total number of subjects |
99999
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EEA total number of subjects |
99999
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Number of subjects enrolled per age group |
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In utero |
0
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Preterm newborn - gestational age < 37 wk |
0
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Newborns (0-27 days) |
0
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Infants and toddlers (28 days-23 months) |
0
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Children (2-11 years) |
99999
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Adolescents (12-17 years) |
0
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Adults (18-64 years) |
0
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From 65 to 84 years |
0
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85 years and over |
0
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Recruitment
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Recruitment details |
99999 is "Not applicable" value or 0 participants, this trial was discontinued with no participants enrolled in the trial | ||||||
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Pre-assignment
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Screening details |
All subjects had to be screened for eligibility to participate in the trial. Subjects had to attend specialist sites which would then ensure that they (the subjects) met all inclusion/exclusion criteria. Subjects were not to be randomised to trial treatment if any one of the specific entry criteria were violated | ||||||
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Period 1
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Period 1 title |
Overall Trial (overall period)
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Is this the baseline period? |
Yes | ||||||
Allocation method |
Not applicable
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Blinding used |
Not blinded | ||||||
Blinding implementation details |
This is an open-label, dose-escalating trial
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Arms
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Arm title
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Volasertib | ||||||
Arm description |
Patients were to be administered Volasertib when added to standard intensive salvage chemotherapy with DNX-FLA in paediatric patients with AML after failure of first-line therapy. | ||||||
Arm type |
Experimental | ||||||
Investigational medicinal product name |
Volasertib
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Investigational medicinal product code |
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Other name |
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Pharmaceutical forms |
Solution for injection/infusion
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Routes of administration |
Intravenous use
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Dosage and administration details |
Single Group Assignment Volasertib Added to the Standard Intensive Salvage Chemotherapy Regimen With Liposomal Daunorubicine, Fludarabine and Cytarabine (DNX-FLA) Followed by Fludarabine and Cytarabine (FLA)
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Baseline characteristics reporting groups
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Reporting group title |
Volasertib
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Reporting group description |
Patients were to be administered Volasertib when added to standard intensive salvage chemotherapy with DNX-FLA in paediatric patients with AML after failure of first-line therapy. | |||||||||||||||||||||||||||||||||||||||
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End points reporting groups
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Reporting group title |
Volasertib
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Reporting group description |
Patients were to be administered Volasertib when added to standard intensive salvage chemotherapy with DNX-FLA in paediatric patients with AML after failure of first-line therapy. | ||
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End point 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 [1] | ||||||||
End point description |
99999 is "Not applicable" value or 0 participants, this trial was discontinued with no participants enrolled in the trial.
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End point type |
Primary
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End point timeframe |
4 weeks
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| Notes [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Justification: No subjects were enrolled in the trial hence results are not available |
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| Notes [2] - 99999 is "Not applicable" value or 0 participants, this trial was discontinued with no participants |
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| No statistical analyses for this end point | |||||||||
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End point title |
Anti-leukaemic activity of volasertib in combination with standard salvage therapy | ||||||||
End point description |
99999 is "Not applicable" value or 0 participants, this trial was discontinued with no participants enrolled in the trial.
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End point type |
Secondary
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End point timeframe |
8 weeks
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| Notes [3] - 99999 is "Not applicable" value or 0 participants, this trial was discontinued with no participants |
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| No statistical analyses for this end point | |||||||||
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End point title |
Event-free survival (EFS) | ||||||||
End point description |
99999 is "Not applicable" value or 0 participants, this trial was discontinued with no participants enrolled in the trial.
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End point type |
Secondary
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End point timeframe |
up to 5 years
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| Notes [4] - 99999 is "Not applicable" value or 0 participants, this trial was discontinued with no participants |
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| No statistical analyses for this end point | |||||||||
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End point title |
Overall survival (OS) | ||||||||
End point description |
99999 is "Not applicable" value or 0 participants, this trial was discontinued with no participants enrolled in the trial.
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End point type |
Secondary
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End point timeframe |
up to 5 years
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| Notes [5] - 99999 is "Not applicable" value or 0 participants, this trial was discontinued with no participants |
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| No statistical analyses for this end point | |||||||||
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End point title |
Number of patients with clinically relevant lab value changes of calcium (hyperand/ or hypocalcaemia) as judged by the investigator and reported as adverse events (Common Terminology Criteria for Adverse Events (CTCAE) grade 3 or higher) | ||||||||
End point description |
99999 is "Not applicable" value or 0 participants, this trial was discontinued with no participants enrolled in the trial.
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End point type |
Secondary
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End point timeframe |
8 weeks
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| Notes [6] - 99999 is "Not applicable" value or 0 participants, this trial was discontinued with no participants |
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| No statistical analyses for this end point | |||||||||
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End point title |
Number of patients with changes in cardiac activity (prolonged QTc interval) reported as clinically relevant observations (i.e. Adverse Events) | ||||||||
End point description |
99999 is "Not applicable" value or 0 participants, this trial was discontinued with no participants enrolled in the trial.
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End point type |
Secondary
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End point timeframe |
8 weeks
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| Notes [7] - 99999 is "Not applicable" value or 0 participants, this trial was discontinued with no participants |
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| No statistical analyses for this end point | |||||||||
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End point title |
Predose concentration of volasertib before administration of second dose | ||||||||
End point description |
99999 is "Not applicable" value or 0 participants, this trial was discontinued with no participants enrolled in the trial.
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End point type |
Secondary
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End point timeframe |
8 weeks
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| Notes [8] - 99999 is "Not applicable" value or 0 participants, this trial was discontinued with no participants |
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| No statistical analyses for this end point | |||||||||
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End point title |
Area under the concentration-time curve of volasertib | ||||||||
End point description |
99999 is "Not applicable" value or 0 participants, this trial was discontinued with no participants enrolled in the trial.
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End point type |
Secondary
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End point timeframe |
8 weeks
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| Notes [9] - 99999 is "Not applicable" value or 0 participants, this trial was discontinued with no participants |
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| No statistical analyses for this end point | |||||||||
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End point title |
Terminal half-life of volasertib in plasma | ||||||||
End point description |
99999 is "Not applicable" value or 0 participants, this trial was discontinued with no participants enrolled in the trial.
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End point type |
Secondary
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End point timeframe |
8 weeks
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| Notes [10] - 99999 is "Not applicable" value or 0 participants, this trial was discontinued with no participants |
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| No statistical analyses for this end point | |||||||||
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End point title |
Maximum concentration of volasertib | ||||||||
End point description |
99999 is "Not applicable" value or 0 participants, this trial was discontinued with no participants enrolled in the trial.
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End point type |
Secondary
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End point timeframe |
8 weeks
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| Notes [11] - 99999 is "Not applicable" value or 0 participants, this trial was discontinued with no participants |
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| No statistical analyses for this end point | |||||||||
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Adverse events information [1]
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Timeframe for reporting adverse events |
All adverse events occurring during the course of the clinical trial (i.e., from signing the informed consent onwards through the observational phase) were to be collected
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Adverse event reporting additional description |
99999 is "Not applicable" value or 0 participants, this trial was discontinued with no participants enrolled in the trial.
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Assessment type |
Systematic | ||
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Dictionary used for adverse event reporting
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Dictionary name |
MedDRA | ||
Dictionary version |
0
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| Frequency threshold for reporting non-serious adverse events: 5% | |||
| Notes [1] - There are no non-serious adverse events recorded for these results. It is expected that there will be at least one non-serious adverse event reported. Justification: No subjects were enrolled in the trial hence results are not available |
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Substantial protocol amendments (globally) |
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| Were there any global substantial amendments to the protocol? No | |||
Interruptions (globally) |
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| Were there any global interruptions to the trial? No | |||
Limitations and caveats |
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| Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data. | |||
| None reported | |||
