Childhood Acute Myeloid Leukemia/Other Myeloid MalignanciesNCT01576185
Developing and Treating a Mouse Model of Acute Myeloid Leukemia Using Tissue Samples From Younger Patients With Acute Myeloid Leukemia
An observational study of Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies, run by Children's Oncology Group at 1 site in United States.
Trial summary
These laboratory trial studies the development and treatment of a mouse model for acute myeloid leukemia (AML) using samples from younger patients with AML. Studying tissue samples from patients with cancer in the laboratory may help doctors learn more about cancer and how well patients will respond to treatment. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies
- What is being tested?
- laboratory biomarker analysis, quizartinib and sorafenib tosylate
- How many people
- 10 participants
- Where
- 1 siteUnited States
- Study dates
- May 2012to May 2016
- Phase
- Observational
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Cryopreserved human AML samples FLT3-ITD samples with high allelic ratios
Who may be able to join
- Cryopreserved human AML samples
- FLT3-ITD samples with high allelic ratios
Who cannot join
None listed.
Trial locations and contacts
1 site in 1 country.
United States1 site
Children's Oncology Group
Monrovia, California, 91006-3776
About this study
Official title: Development of Pediatric Acute Myeloid Leukemia Xenograft Models for the Testing of Targeted Therapeutic Agents
These laboratory trial studies the development and treatment of a mouse model for acute myeloid leukemia (AML) using samples from younger patients with AML. Studying tissue samples from patients with cancer in the laboratory may help doctors learn more about cancer and how well patients will respond to treatment.
Read the detailed description
PRIMARY OBJECTIVES:
- To determine the rate of engraftment of pediatric FMS-Like Tyrosine Kinase-3 (FLT3)-internal tandem duplication (ITD) acute myeloid leukemia (AML) samples in NOD scid gamma (NSG) mice.
II. To determine the efficacy of treatment of FLT3-ITD xenografts with tyrosine kinase inhibitors.
OUTLINE:
Human acute myeloid leukemia cells are injected into NSG mice. Mice are then treated with sorafenib or quizartinib via gavage once daily for 28 days. Peripheral blood and tissue samples are collected biweekly or weekly and analyzed for the presence of human CD45+ and CD33+ cells by quantitative flow cytometry.
- Being tested
- laboratory biomarker analysis, quizartinib, sorafenib tosylate
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
laboratory biomarker analysisOther
Correlative studies
quizartinibDrug
Also called AC220, class III receptor tyrosine kinase inhibitor AC220
Via gavage
sorafenib tosylateDrug
Also called BAY 43-9006, BAY 43-9006 Tosylate Salt, BAY 54-9085, Nexavar
Via gavage
| Study group | Role | What they receive |
|---|---|---|
| Observational (xenograft models) | Human acute myeloid leukemia cells are injected into NSG mice. Mice are then treated with sorafenib or quizartinib via gavage once daily for 28 days. Peripheral blood and tissue samples are collected biweekly or weekly and analyzed for the presence of human CD45+ and CD33+ cells by quantitative flow cytometry. Receives: Other: laboratory biomarker analysis; Drug: quizartinib; Drug: sorafenib tosylate |
What is being measured
The primary outcome is the main question the study is designed to answer.
Engraftment ratio of human AML cells to murine cells
We will measure total leukemic burden from harvested femurs, tibias, and spleen by quantitative flow cytometry, estimate engraftment, and describe 95% confidence intervals. The total AML cell count of the control and treatment cohorts will be compared using an analysis of variance (ANOVA) test.
Measured: Up to 9 months
Efficacy of sorafenib or quizartinib to inhibit AML proliferation in vivo
Measured: Up to 9 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Apr 2012 (estimated)
- Study startMay 2012
- Primary completionMay 2016
- Study completionMay 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Childhood Acute Myeloid Leukemia/Other Myeloid MalignanciesStudies of the same condition, open and under way.
- More trials run by Children's Oncology GroupThe 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
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's Oncology Group
- Sponsor type
- Research network
- Collaborators
- National Cancer Institute (NCI)
- Responsible party
- Sponsor
- Investigators
- Sarah Tasian, MD, Principal investigator, Children's Oncology Group
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01576185
- Sponsor's protocol ID
- AAML12B8
- Other IDs
- NCI-2012-00724, COG-AAML12B8, CDR0000730390
- Status last verified
- May 2016
- First submitted
- 11 Apr 2012
- Last updated
- 17 May 2016
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 May 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Development of Pediatric Acute Myeloid Leukemia Xenograft Models for the Testing of Targeted Therapeutic Agents
- ClinicalTrials.gov ID
- NCT01576185
- Acronym / sponsor ID
- AAML12B8
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- Children's Oncology GroupResearch network
- Collaborators
- National Cancer Institute (NCI)
- Study start
- May 2012
- Primary completion
- May 2016 (actual)
- Study completion
- May 2016 (actual)
- First posted
- 12 Apr 2012 (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.
These laboratory trial studies the development and treatment of a mouse model for acute myeloid leukemia (AML) using samples from younger patients with AML. Studying tissue samples from patients with cancer in the laboratory may help doctors learn more about cancer and how well patients will respond to treatment.
Detailed description
PRIMARY OBJECTIVES:
- To determine the rate of engraftment of pediatric FMS-Like Tyrosine Kinase-3 (FLT3)-internal tandem duplication (ITD) acute myeloid leukemia (AML) samples in NOD scid gamma (NSG) mice.
II. To determine the efficacy of treatment of FLT3-ITD xenografts with tyrosine kinase inhibitors.
OUTLINE:
Human acute myeloid leukemia cells are injected into NSG mice. Mice are then treated with sorafenib or quizartinib via gavage once daily for 28 days. Peripheral blood and tissue samples are collected biweekly or weekly and analyzed for the presence of human CD45+ and CD33+ cells by quantitative flow cytometry.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Observational (xenograft models) | – | Other: laboratory biomarker analysis, Drug: quizartinib, Drug: sorafenib tosylateHuman acute myeloid leukemia cells are injected into NSG mice. Mice are then treated with sorafenib or quizartinib via gavage once daily for 28 days. Peripheral blood and tissue samples are collected biweekly or weekly and analyzed for the presence of human CD45+ and CD33+ cells by quantitative flow cytometry. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Cryopreserved human AML samples FLT3-ITD samples with high allelic ratios
Key inclusion criteria (2 listed)
- Cryopreserved human AML samples
Key exclusion criteria (0 listed)
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 |
|---|---|---|
| laboratory biomarker analysisCorrelative studies | Other | Observational (xenograft models) |
| quizartinibAlso: AC220, class III receptor tyrosine kinase inhibitor AC220Via gavage | Drug | Observational (xenograft models) |
| sorafenib tosylateAlso: BAY 43-9006, BAY 43-9006 Tosylate Salt, BAY 54-9085, NexavarVia gavage | Drug | Observational (xenograft models) |
Primary outcome
Engraftment ratio of human AML cells to murine cells
Time frame: Up to 9 months
We will measure total leukemic burden from harvested femurs, tibias, and spleen by quantitative flow cytometry, estimate engraftment, and describe 95% confidence intervals. The total AML cell count of the control and treatment cohorts will be compared using an analysis of variance (ANOVA) test.
Efficacy of sorafenib or quizartinib to inhibit AML proliferation in vivo
Time frame: Up to 9 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 May 2016.
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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.
Sarah Tasian, MD principal investigator
Children's Oncology Group
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies, run by Children's Oncology Group at 1 site in United States.
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 2 inclusion and 0 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: finished, as of the sponsor's last update on 17 May 2016. 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 United States. 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.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Engraftment ratio of human AML cells to murine cells (time frame: Up to 9 months). The study lists 2 primary outcomes in all. 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 17 May 2016.