Leukemia, Myeloid, AcutePhase 1NCT02722135
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
A Phase 1 study testing Volasertib in people with Leukemia, Myeloid, Acute, run by Boehringer Ingelheim at 1 site in Belgium.
Trial 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. Read more about this study
- Who is it for?
- Ages 3 months to 17 years
- Conditions the study is for
- Leukemia, Myeloid, Acute
- What is being tested?
- Volasertib
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteBelgium
- Study dates
- Nov 2016to Sep 2018
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 months to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
Who cannot join
- Down syndrome
- Acute promyelocytic leukaemia and treatment-related AML
- QTc prolongation
- LVSF <30%
- Cardiac disease and/or dysfunction
- Active uncontrolled infection
Show all 11Show fewer
- HIV infection, acute or chronic hepatitis
- Inadequate lab parameters
- Impaired renal function
- Pregnancy or nursing
- Further exclusion criteria apply
Trial locations and contacts
1 site in 1 country.
About this study
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
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.
- Conditions
- Leukemia, Myeloid, Acute
- Being tested
- Volasertib
- Medical subject headings
- Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VolasertibDrug
| Study group | Role | What they receive |
|---|---|---|
| Volasertib | Experimental | Not described Receives: Drug: Volasertib |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 4 weeks
Secondary outcomes (9)
Anti-leukaemic activity of volasertib in combination with standard salvage therapy
Measured: 8 weeks
Event-free survival (EFS)
Measured: up to 5 years
Overall survival (OS)
Measured: up to 5 years
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)
Measured: 8 weeks
Number of patients with changes in cardiac activity (prolonged QTc interval) reported as clinically relevant observations (i.e. Adverse Events)
Measured: 8 weeks
Predose concentration of volasertib before administration of second dose
Measured: 8 weeks
Area under the concentration-time curve of volasertib
Measured: 8 weeks
Terminal half-life of volasertib in plasma
Measured: 8 weeks
Maximum concentration of volasertib
Measured: 8 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Mar 2016 (estimated)
- Study startNov 2016
- Primary completionMay 2018 (estimated)
- Study completionSep 2018 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Leukemia, Myeloid, AcuteStudies of the same condition, open and under way.
- More trials run by Boehringer IngelheimThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Boehringer Ingelheim
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Boehringer Ingelheim, Study chair, Boehringer Ingelheim
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02722135
- Sponsor's protocol ID
- 1230.28
- Other IDs
- 2015-004625-14, 2015
- Status last verified
- Jan 2017
- First submitted
- 23 Mar 2016
- Last updated
- 27 Jan 2017
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Jan 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT02722135
- Acronym / sponsor ID
- 1230.28
- Conditions
- Leukemia, Myeloid, Acute
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Withdrawn
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Boehringer IngelheimIndustry (pharma, biotech, devices)
- Study start
- Nov 2016
- Primary completion
- May 2018 (estimated)
- Study completion
- Sep 2018 (estimated)
- First posted
- 29 Mar 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Volasertib | Experimental | Drug: Volasertib |
Who may qualify
- Ages
- 3 months to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (11 listed)
- Down syndrome
- Acute promyelocytic leukaemia and treatment-related AML
- QTc prolongation
- LVSF <30%
- Cardiac disease and/or dysfunction
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Volasertib | Drug | Volasertib |
Primary outcome
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
9 secondary outcomes
Anti-leukaemic activity of volasertib in combination with standard salvage therapy
Time frame: 8 weeks
Event-free survival (EFS)
Time frame: up to 5 years
Overall survival (OS)
Time frame: up to 5 years
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
Number of patients with changes in cardiac activity (prolonged QTc interval) reported as clinically relevant observations (i.e. Adverse Events)
Time frame: 8 weeks
Predose concentration of volasertib before administration of second dose
Time frame: 8 weeks
Area under the concentration-time curve of volasertib
Time frame: 8 weeks
Terminal half-life of volasertib in plasma
Time frame: 8 weeks
Maximum concentration of volasertib
Time frame: 8 weeks
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Jan 2017.
Related trials for Leukemia, Myeloid, Acute
Not yet recruitingPhase 1/2
A Study of Sonrotoclax (BGB-11417) in Children With Relapsed or Refractory Acute Myeloid Leukemia and B-cell Acute Lymphoblastic Leukemia
Relapsed Acute Myeloid Leukemia, Refractory Acute Myeloid Leukemia
RecruitingPhase 1
Autologous T Cells Transduced With Retroviral Vectors Expressing TCRs for Participant-specific Neoantigens in Patients With Hematologic Malignancies
Malignancy, Hematologic, Neoplasms, Hematologic
RecruitingPhase 2
MYELOMATCH: A Screening Study to Assign People With Myeloid Cancer to a Treatment Study or Standard of Care Treatment Within myeloMATCH (MyeloMATCH Screening Trial)
Acute Myeloid Leukemia, Acute Myeloid Leukemia Arising From Previous Myelodysplastic/Myeloproliferative Neoplasm
RecruitingPhase 2
Comparing Cytarabine + Daunorubicin Therapy Versus Cytarabine + Daunorubicin + Venetoclax Versus Venetoclax + Azacitidine in Younger Patients With Intermediate Risk AML (A MyeloMATCH Treatment Trial)
Acute Myeloid Leukemia
RecruitingPhase 2
Cord Blood Transplant, Cyclophosphamide, Fludarabine, and Total-Body Irradiation in Treating Patients With High-Risk Hematologic Diseases
Acute Leukemia of Ambiguous Lineage, Acute Lymphoblastic Leukemia
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Boehringer Ingelheim study chair
Boehringer Ingelheim
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Volasertib in people with Leukemia, Myeloid, Acute, run by Boehringer Ingelheim at 1 site in Belgium.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 27 Jan 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Belgium. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Jan 2017.