Recurrent Childhood Acute Myeloid LeukemiaPhase 1NCT05857969
Ex Vivo Drug Sensitivity Testing and Multi-Omics Profiling
A Phase 1 study testing Functional Precision Medicine in people with Recurrent Childhood Acute Myeloid Leukemia and Recurrent Childhood Acute Lymphoblastic Leukemia, run by Florida International University at 1 site in United States.
Trial summary
Functional precision medicine (FPM) is a relatively new approach to cancer therapy based on direct exposure of patient- isolated tumor cells to clinically approved drugs and integrates ex vivo drug sensitivity testing (DST) and genomic profiling to determine the optimal individualized therapy for cancer patients. Read more about this study
- Who is it for?
- Ages 1 day to 21 years
- Conditions the study is for
- Recurrent Childhood Acute Myeloid Leukemia, Recurrent Childhood Acute Lymphoblastic Leukemia, Recurrent Childhood Large Cell Lymphoma
- What is being tested?
- Functional Precision Medicine
- Type of treatment
- Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 65 participants
- Where
- 1 siteUnited States
- Study dates
- 22 Feb 2023to 31 Dec 2028
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
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 1 day to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patients aged 21 years or younger at the time of enrollment on this study of any gender, race or ethnicity.
- Subjects with suspected or confirmed diagnosis of recurrent or refractory cancer Subjects who are scheduled for or have recently had biopsy or tumor excised (solid tumors) or bone marrow aspirate (blood cancers) Subjects willing to have a blood draw or buccal swab done for the purposes of genetic testing Subjects or their parents or legal guardians willing to sign informed consent Subjects aged 7 to 17 willing to sign assent
Who cannot join
- Subjects who do not have malignant tissue available and accessible The amount of excised malignant tissue is not sufficient for the ex vivo drug testing and/or genetic profiling.
- Patients with newly diagnosed tumors and tumors that have high (>90%) cure rate with safe standard therapy.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Nicklaus Children's Hospital
Miami, Florida, 33155
Darika Sartmatova, 800-533-1792, exvivotrial@nicklaushealth.org
Lillian Garvin, 800-533-1792, exvivotrial@nicklaushealth.org
About this study
Official title: Advancing Personalized Treatment in Pediatric Oncology Through Functional Precision Medicine
Functional precision medicine (FPM) is a relatively new approach to cancer therapy based on direct exposure of patient- isolated tumor cells to clinically approved drugs and integrates ex vivo drug sensitivity testing (DST) and genomic profiling to determine the optimal individualized therapy for cancer patients. In this study, we will enroll relapsed or refractory pediatric cancer patients with tissue available for DST and genomic profiling from the South Florida area, which is 69% Hispanic and 18% Black. Tumor cells collected from tissue taken during routine biopsy or surgery will be tested.
Read the detailed description
PRIMARY OBJECTIVE: The primary objective of the study is to determine feasibility of providing pediatric cancer patients with access to personalized treatment options and clinical management recommendations based on Functional Precision Medicine (FPM), the combination of ex vivo drug sensitivity testing (DST) and genomic profiling.
SECONDARY OBJECTIVE: The secondary objective of the study is to compare individual outcomes (response and disease-free survival) in patients with pediatric cancers treated with FPM-guided therapy as compared to non-FPM guided (conventional) therapy.
EXPLORATORY OBJECTIVE: To explore associations between tumor molecular characteristics (genomic and transcriptomic variation) and ex vivo drug response with respect to patient ethnicity.
- Conditions
- Recurrent Childhood Acute Myeloid Leukemia, Recurrent Childhood Acute Lymphoblastic Leukemia, Recurrent Childhood Large Cell Lymphoma, Refractory Childhood Acute Lymphoblastic Leukemia, Refractory Childhood Hodgkin Lymphoma, Refractory Childhood Malignant Germ Cell Neoplasm
- Being tested
- Functional Precision Medicine
- Keywords
- ex vivo drug sensitivity assay, genomic profiling, Functional precision medicine, Biomarker development
- Medical subject headings
- Precursor Cell Lymphoblastic Leukemia-Lymphoma, Dendritic Cell Sarcoma, Interdigitating, Familial ependymoma, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Functional Precision MedicineDevice
Ex Vivo Drug Sensitivity Testing + Genomic Tumor Profiling
| Study group | Role | What they receive |
|---|---|---|
| Functional Precision Medicine for Chemorefractory or relapsed patients | Experimental | We intend to enroll chemorefractory or relapsed pediatric patients with all types of cancers where tumor tissue would be available for functional precision medicine that integrates ex vivo drug screening and genomic profiling. The results of the drug sensitivity assay and genetic screening will be used to inform treating physician about patient-specific drug sensitivity or resistance guiding best therapy choices. Receives: Device: Functional Precision Medicine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Patients that receive Functional Precision Medicine (FPM)-guided treatment options
This study will be considered successful (feasibility demonstrated) if it is possible to choose and initiate a monotherapy or combination drug regimen based on functional and/or genomics data within 4 weeks in at least 39 out of 65 patients (60%).
To achieve at least 90% power, the null hypothesis will be rejected when at least 39 out of 65 patients receive treatment recommendations through functional and/or genomics data within 4 weeks on the study.
With that outcome, we would have 95% confidence that the true feasibility rate is at least 40% (95% CI: 0.4905 to 1).
Measured: Up to 6 years
Secondary outcomes (3)
Assessing Progression-Free Survival (PFS) in FPM-guided therapy versus standard of care
We will assess changes in cohort PFS by comparing PFS in patients treated with FPM-guided therapy versus PFS in patients treated with non-FPM guided conventional therapy (standard of care)
Measured: Up to 6 years
Assessing Previous vs Trial PFS Ratio (PFS2/PFS1) in FPM-guided patients versus standard of care
We will assess changes in PFS from each patient's previous treatment versus their PFS from the treatment assigned during the trial. Assessments will be made both in the FPM-guided cohort and the non-FPM-guided cohort (standard of care). Analysis will include both the raw ratio as well as the number of incidences of 30% improved PFS on trial versus previous regimen (PFS2/PFS1 > 1.3x).
Measured: Up to 6 years
Assessing Overall Survival (OS) in FPM-guided patients versus standard of care patients
We will assess changes in cohort OS by comparing OS in patients treated with FPM-guided therapy versus OS in patients treated with non-FPM guided conventional therapy (standard of care)
Measured: Up to 6 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 May 2023
- Study start22 Feb 2023
- Primary completion22 Feb 2028 (estimated)
- Study completion31 Dec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Recurrent Childhood Acute Myeloid LeukemiaStudies of the same condition, open and under way.
- 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
- 65 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Florida International University
- Sponsor type
- University, hospital or other
- Collaborators
- Nicklaus Children's Hospital f/k/a Miami Children's Hospital, First Ascent Biomedical Inc., National Institute on Minority Health and Health Disparities (NIMHD)
- Responsible party
- Principal investigator, Diana Azzam, PhD, Florida International University
- Investigators
- Diana Azzam, Principal investigator, Florida International UniversityMaggie Fader, Principal investigator, Nicklaus Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05857969
- Sponsor's protocol ID
- 112215
- Other IDs
- 2U54MD012393-06
- Status last verified
- Aug 2026
- First submitted
- 5 May 2023
- Last updated
- 26 Aug 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 26 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Advancing Personalized Treatment in Pediatric Oncology Through Functional Precision Medicine
- ClinicalTrials.gov ID
- NCT05857969
- Acronym / sponsor ID
- 112215
- Conditions
- Recurrent Childhood Acute Myeloid Leukemia, Recurrent Childhood Acute Lymphoblastic Leukemia, Recurrent Childhood Large Cell Lymphoma, Refractory Childhood Acute Lymphoblastic Leukemia, Refractory Childhood Hodgkin Lymphoma, Refractory Childhood Malignant Germ Cell Neoplasm
- Keywords
- ex vivo drug sensitivity assay, genomic profiling, Functional precision medicine, Biomarker development
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 65 participants (estimated)
- Lead sponsor
- Florida International UniversityUniversity, hospital or other
- Collaborators
- Nicklaus Children's Hospital f/k/a Miami Children's Hospital, First Ascent Biomedical Inc., National Institute on Minority Health and Health Disparities (NIMHD)
- Study start
- 22 Feb 2023 (actual)
- Primary completion
- 22 Feb 2028 (estimated)
- Study completion
- 31 Dec 2028 (estimated)
- First posted
- 15 May 2023 (actual)
- 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.
Functional precision medicine (FPM) is a relatively new approach to cancer therapy based on direct exposure of patient- isolated tumor cells to clinically approved drugs and integrates ex vivo drug sensitivity testing (DST) and genomic profiling to determine the optimal individualized therapy for cancer patients. In this study, we will enroll relapsed or refractory pediatric cancer patients with tissue available for DST and genomic profiling from the South Florida area, which is 69% Hispanic and 18% Black. Tumor cells collected from tissue taken during routine biopsy or surgery will be tested.
Detailed description
PRIMARY OBJECTIVE: The primary objective of the study is to determine feasibility of providing pediatric cancer patients with access to personalized treatment options and clinical management recommendations based on Functional Precision Medicine (FPM), the combination of ex vivo drug sensitivity testing (DST) and genomic profiling.
SECONDARY OBJECTIVE: The secondary objective of the study is to compare individual outcomes (response and disease-free survival) in patients with pediatric cancers treated with FPM-guided therapy as compared to non-FPM guided (conventional) therapy.
EXPLORATORY OBJECTIVE: To explore associations between tumor molecular characteristics (genomic and transcriptomic variation) and ex vivo drug response with respect to patient ethnicity.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Functional Precision Medicine for Chemorefractory or relapsed patients | Experimental | Device: Functional Precision MedicineWe intend to enroll chemorefractory or relapsed pediatric patients with all types of cancers where tumor tissue would be available for functional precision medicine that integrates ex vivo drug screening and genomic profiling. The results of the drug sensitivity assay and genetic screening will be used to inform treating physician about patient-specific drug sensitivity or resistance guiding best therapy choices. |
Who may qualify
- Ages
- 1 day to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (2 listed)
- Patients aged 21 years or younger at the time of enrollment on this study of any gender, race or ethnicity.
- Subjects with suspected or confirmed diagnosis of recurrent or refractory cancer Subjects who are scheduled for or have recently had biopsy or tumor excised (solid tumors) or bone marrow aspirate (blood cancers) Subjects willing to have a blood draw or buccal swab done for the purposes of genetic testing Subjects or their parents or legal guardians willing to sign informed consent Subjects aged 7 to 17 willing to sign assent
Key exclusion criteria (2 listed)
- Subjects who do not have malignant tissue available and accessible The amount of excised malignant tissue is not sufficient for the ex vivo drug testing and/or genetic profiling.
- Patients with newly diagnosed tumors and tumors that have high (>90%) cure rate with safe standard therapy.
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 |
|---|---|---|
| Functional Precision MedicineEx Vivo Drug Sensitivity Testing + Genomic Tumor Profiling | Device | Functional Precision Medicine for Chemorefractory or relapsed patients |
Primary outcome
Percentage of Patients that receive Functional Precision Medicine (FPM)-guided treatment options
Time frame: Up to 6 years
This study will be considered successful (feasibility demonstrated) if it is possible to choose and initiate a monotherapy or combination drug regimen based on functional and/or genomics data within 4 weeks in at least 39 out of 65 patients (60%). To achieve at least 90% power, the null hypothesis will be rejected when at least 39 out of 65 patients receive treatment recommendations through functional and/or genomics data within 4 weeks on the study. With that outcome, we would have 95% confidence that the true feasibility rate is at least 40% (95% CI: 0.4905 to 1).
3 secondary outcomes
Assessing Progression-Free Survival (PFS) in FPM-guided therapy versus standard of care
Time frame: Up to 6 years
Assessing Previous vs Trial PFS Ratio (PFS2/PFS1) in FPM-guided patients versus standard of care
Time frame: Up to 6 years
Assessing Overall Survival (OS) in FPM-guided patients versus standard of care patients
Time frame: Up to 6 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Aug 2026.
Related trials for Recurrent Childhood Acute Myeloid Leukemia
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
A Study to Find the Highest Dose of Imetelstat in Combination With Fludarabine and Cytarabine for Patients With AML, MDS or JMML That Has Come Back or Does Not Respond to Therapy
Recurrent Childhood Acute Myeloid Leukemia, Recurrent Childhood Myelodysplastic Syndrome
RecruitingPhase 1
FH-FOLR1 Chimeric Antigen Receptor T Cell Therapy for Treating Pediatric Patients With Relapsed or Refractory Acute Myeloid Leukemia
Recurrent Childhood Acute Myeloid Leukemia, Refractory Childhood Acute Myeloid Leukemia
Active, not recruitingPhase 2
Total Marrow and Lymphoid Irradiation and Chemotherapy Before DSCT in Treating Patients With High-Risk ALL or AML
Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q)
Active, not recruitingPhase 2
Donor Peripheral Stem Cell Transplant in Treating Patients With Advanced Hematologic Cancer or Other Disorders
Chronic Myeloproliferative Disorders, Graft Versus Host Disease
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.
Diana Azzam principal investigator
Florida International University
Maggie Fader principal investigator
Nicklaus Children's Hospital
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 Functional Precision Medicine in people with Recurrent Childhood Acute Myeloid Leukemia and Recurrent Childhood Acute Lymphoblastic Leukemia, run by Florida International University 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 2 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: looking for participants now, as of the sponsor's last update on 26 Aug 2026. 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.
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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Diana Azzam, PhD (305-348-9043, fpmlab@fiu.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percentage of Patients that receive Functional Precision Medicine (FPM)-guided treatment options (time frame: Up to 6 years). 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 26 Aug 2026.