Neoplasms

Blood and Lymph Conditions

All Conditions

Rare Diseases

Leukemia

Leukemia, Myeloid

Leukemia, Myeloid, Acute

Neoplasms by Histologic Type

Hematologic Diseases

Myeloid Leukemia

Acute Myeloid Leukemia

Acute Non Lymphoblastic Leukemia

Acute Graft Versus Host Disease

Antineoplastic Agents

All Drugs and Chemicals

Anti-Infective Agents

Etoposide

Cytarabine

Daunorubicin

Etoposide phosphate

Asparaginase

Sorafenib

Gemtuzumab

Calicheamicins

Enzyme Inhibitors

Antimetabolites

Antiviral Agents

Immunosuppressive Agents

Immunologic Factors

Protein Kinase Inhibitors

Anti-Bacterial Agents

Antibiotics, Antitubercular

Antineoplastic Agents, Immunological

Immunotoxins

Immunoconjugates

Participants in this arm will receive the standard Aflac-AML Regimen with the following chemotherapy:

* Induction-1: patients on Induction-I will receive gemtuzumab + ADE therapy based on genotyping. Lasts a total of 28 days.
* Induction II - MA
* Intensification I - AE
* Intensification II - HD ARAC/LASP

Patients with low risk status who had low risk markers and were MRD positive at the end of Induction I and continue to be MRD positive after Induction II will come off protocol.

Participants in this arm will receive standard Aflac-AML Regimen with the following chemotherapy:

* Induction-1: patients will receive gemtuzumab in addition to ADE therapy based on genotyping. Induction I lasts a total of 28 days.
* Induction 1 for FLT3-ITD patients - ADE (10+3+5) with GO with Sorafenib
* Induction II - MA
* Induction II for FLT3-ITD patients - MA with Sorafenib
* Intensification I - AE
* Intensification I for FLT3-ITD patients - AE with sorafenib
* Intensification II - HD ARAC/LASP
* Intensification II for FLT3-ITD patients - HD ARAC/LASP with sorafenib
* Hematopoietic stem cell transplantation (HSCT)

If a patient is classified as High risk after Induction I, they may proceed to best allogenic donor SCT following Induction II. These patients may receive a third course of chemotherapy prior to HSCT. In cases where HSCT is not an option, patients can receive 4 cycles of chemotherapy. Only patients with FLT3-ITD mutation will receive sorafenib.

100 mg/m²/dose every 12 hours IV Days 1-10

25,000 International Units/m²/dose IM Days 2, 9

Erwinase

Administered as a single 3 mg/m2 dose of GO to be given between days 6-10 during Induction I for patients with CC genotype, in addition to ADE therapy.

Gemtuzumab ozogamicin

Transplantation of multipotent hematopoietic stem cells from bone marrow

Stem cell transplantation (SCT)

200 mg/m2/dose daily, rounded to accommodate tablet size. The maximum dose will be 400 mg. Days 7 through 34.

Himalee Sabnis, MD

The investigators propose to study an Aflac-AML chemotherapy backbone prospectively to validate its use in all pediatric AML and to further evaluate the cardiotoxicity with this approach for low risk AML.

Advances in risk stratification and therapy, have improved the event-free survival (EFS) and overall survival (OS) for pediatric acute myeloid leukemia (AML) to approximately 50% and 65% respectively, with current treatment strategies. Patients with good response to induction and/or those who lack high-risk cytogenetic and molecular features \[classified as low-risk AML (LR-AML)\] have even better outcomes with EFS and OS approaching 70% and 85% respectively; however, treatment-related toxicities remain a major concern. Anthracycline-based therapeutic regimens expose patients to the risk of anthracycline-induced cardiotoxicity. Therefore, strategies that reduce cardiac toxicities using tailored approaches while maintaining and/or improving outcomes are needed for all patients with AML. The Children's Oncology Group (COG) regimens AAML1031 and AAML0531 utilized an anthracycline-intensive backbone for LR-AML with cumulative anthracycline doxorubicin-equivalent doses of up to 492mg/m2. However, high-risk patients treated with chemotherapy alone received an intensified induction chemotherapy (using mitoxantrone-cytarabine) but with overall reduced doses of anthracycline-equivalent (342mg/m2). The investigators piloted an institutional practice to treat all LR-AML patients with four cycle regimen (Aflac-AML) with the goal of reducing cumulative anthracycline exposure, thereby reducing the risk of cardiotoxicity, while providing three high-dose cytarabine courses. In this pilot institutional experience with this approach, they were able to maintain excellent outcomes for this low-risk group with 3-year event-free survival (EFS) and OS of 70.0% ± 0.1% and 85.5% ± 0.08% respectively, from end of course 1. Recent evolution in cytogenetic classification has further delineated risk groups in AML. Gemtuzumab ozogamicin (GO), an antibody-drug conjugate was shown to reduce relapse risk in patients with CC genotype with de-novo AML on COG study AAML0531. The investigators propose to study an Aflac-AML chemotherapy backbone prospectively to validate its use in all pediatric AML and to further evaluate the cardiotoxicity with this approach for low risk AML.

AflacLL1901 (CHOA-AML)

CHOA-AML: A Pilot Study for Newly Diagnosed Pediatric Patients With Acute Myeloid Leukemia (AML)

Event-free survival defined as the time from on study to death, failure to achieve remission or relapse in newly diagnosed patients with pediatric acute myeloid leukemia in the Low risk stratification group and High risk stratification group

Time from study entry and from end of first course of therapy for newly diagnosed patients with pediatric acute myeloid leukemia

Number of patients that are in remission (MRD negative) after course 1 in participants receiving GO and those that did not receive GO.

Disease-free survival for patients who are MRD negative but lack high or low risk molecular and cytogenetic features, defined as time from end of first course of therapy to death or relapse

Number of patients that develop cardiac ejection fraction \<50% (CTCAE V5.0 grade 2 or greater dysfunction) either during therapy (early cardiotoxicity) or after completion of therapy (late cardiotoxicity)

Number of participants that developed infection and/or febrile neutropenia at the end of each treatment cycle.

Duration of hospital admission in patients that developed infection and febrile neutropenia at the end of each treatment cycle

Emory University

Assistant Professor

Participants in this group received AraC/Daunorubicin/Etoposide (10+3+5) +/- GO based on CC genotype\^ Patients were classified into risk groups following Induction I

* \^If has FLT3-ITD mutation, Sorafenib is added

Induction I

Participants in this group received Mitoxantrone/AraC

Low Risk - Induction II

Participants in this group received Mitoxantrone/AraC\^

* \^If has FLT3-ITD mutation, Sorafenib is added

High Risk - Induction II

Participants in this group received AraC/Etoposide

Low Risk - Intensification I

Participants in this group received HD AraC/Asparaginase

Low Risk - Intensification II

Patients were classified as low risk following Induction I. All were MRD negative without low or high risk genetic and prognostic factors.

* Induction I: AraC/Daunorubicin/Etoposide (10+3+5) +/- GO based on CC genotype
* Induction II: Mitoxantrone/AraC
* Intensification I: AraC/Etoposide
* Intensification II: HD AraC/Asparaginase

Aflac-AML Low Risk Patients

Patients were classified as high risk following Induction I. All were MRD negative with high risk genetic and prognostic factors.

Induction I: AraC/Daunorubicin/Etoposide (10+3+5) +/- GO based on CC genotype\^ Induction II: Mitoxantrone/AraC\^ Intensification:\* AraC/Etoposide\^ OR HD AraC/Asparaginase\^ Allogenic HSCT

* \^If has FLT3-ITD mutation, Sorafenib is added
* \* Depending on timing patients may proceed to HSCT following Induction II or receive Intensification. If HSCT is not an option, patients can receive 4 cycles of chemotherapy

Aflac-AML High Risk Patients

Total

Age, Continuous

Sex: Female, Male

Ethnicity (NIH/OMB)

Race (NIH/OMB)

United States

Region of Enrollment

Dr. Himalee Sabnis

Event-free Survival (EFS) in Low Risk Patients and High Risk Patients

Overall Survival (OS)

Patients with CC genotype received Induction 1 AraC/Daunorubicin/Etoposide (10+3+5) with Gemtuzumab ozogamicin (GO)

Induction I With GO

Patients without CC genotype received Induction 1 AraC/Daunorubicin/Etoposide (10+3+5)

Induction I Without GO

Minimal Residual Disease (MRD) Negative Status

Patients who are MRD negative but lack high or low risk molecular and cytogenetic features, defined as time from end of first course of therapy to death or relapse.

Patients Who Are MRD Negative

All patients were MRD negative at the end of Induction I. No patients were assigned low risk due to low risk molecular and cytogenetic features. All high risk patients had high risk molecular and cytogenetic features.

Disease-free Survival (DFS) for Patients Who Are MRD Negative

Early cardiotoxicity

Late cardiotoxicity

The definition of late cardiotoxicity is after completion of therapy. All 3 of the high risk patients came off protocol early and did not complete therapy. For the low risk patients, 2 did not complete therapy. Among the 3 that did complete therapy, 1 patient has been lost to follow-up since completion of therapy with no cardiac data available.

Cardiotoxicity in Patients With de Novo AML That Receive the Four-cycle Aflac-AML Regimen With the Inclusion of Dexrazoxane

Cycle 1 - Induction 1

Cycle 2 - Induction 2

Cycle 3 - Induction 3

Cycle 4 - Induction 4

All 3 of the high risk patients came off protocol early and did not complete therapy. For the low risk patients, 2 did not complete therapy. Among the 3 that did complete therapy, 1 patient has been lost to follow-up since completion of therapy with no cardiac data available.

Proportion of Patients Who Develop Infection and/or Febrile Neutropenia During Each Treatment Course

Duration of Hospital Admission in Patients That Developed Infection and Febrile Neutropenia at the End of Each Treatment Cycle

Overall Study

Micronutrients

Hematinics

Vitamins

Folic Acid

Vitamin B Complex

Exatecan

Folinic Acid

Folate

Vitamin B9

Dose Escalation: Escalating doses of ELU001

Folic-acid functionalized C'Dot-Drug-Conjugate (FA-CDC)

ELU001

This research study was planned to focus on a rare type of acute myeloid leukemia (with the subtype CBFA2T3::GLIS2 that overexpresses folate receptor alpha (FRα) (a protein on the surface of leukemia cells)) that has relapsed or is refractory. Relapse means the cancer has come back after treatment. Refractory means the cancer does not respond to treatment.

This study was planned as a Dose Escalation Safety Study to identify the maximum tolerated dose (MTD) and/or the recommended phase 2 dose (RP2D). This study will also evaluate the tolerability of ELU001.

ELU001 is not a drug approved by the FDA (Food and Drug Administration) yet.

ELU001 in Pediatric Subjects Who Have Relapsed and/or Refractory CBFA2T3-GLIS2-positive AML

Dose Escalation Study to Evaluate the Safety and Tolerability of ELU001 in Pediatric Patients Who Have Relapsed and/or Refractory CBFA2T3::GLIS2 Positive Acute Myeloid Leukemia

Establish the Maximum Tolerated Dose (MTD) and/or Recommended Phase 2 Dose (RP2D) of ELU001 in pediatric patients with relapsed or refractory CBFA2T3::GLIS2 positive AML.

Proportion of evaluable patients having achieved at least one of the following

* Complete Remission per IWG (CRIWG)
* Complete Remission With Partial Recovery of Platelet Count (CRp)
* Complete Remission with Incomplete Blood Count Recovery (CRi)
* Complete Remission for Minimal Residual Disease (CRm)

Duration of Complete Remission from CRIWG/CR/CRp/CRi to hematological relapse or death from any cause, whichever comes first

Measure the concentration of ELU001 in the blood.

This includes - Maximum Observed Concentration (Cmax), Time After Dosing at which Maximum Observed Concentration of Drug is Observed (tmax), Area Under the Curve to the End of the Dosing Period (AUC0-tau), and Area Under the Curve to the Last Measurable Concentration (AUC0-t), will be estimated. Other PK parameters, e.g., Terminal Elimination or Disposition Half-Life (T½), Volume of Distribution (Vd), Clearance Rate (CL), and C'Dot, payload on C'Dot

Percent incidence of Anti-Drug Antibodies (ADA) formation in the blood assessed from baseline until End-of-Treatment (EOT).

Elucida Oncology

Antibodies

Antibodies, Monoclonal

Antibodies, Bispecific

Immunoglobulins

Subcutaneous administration of CD33\*CD3 BsAb up to 12 cycles

CD33xCD3 is a bispecific monoclonal antibody, which specifically targets CD33 on the AML blasts and CD3 on the T cells

CD33*CD3 BsAb

Jessica Pollard, MD

Pediatric patients (\<21 years at study entry) with relapsed or refractory acute myeloid leukemia (AML) will be treated with CD33\*CD3 a bispecific antibody to investigate the safety and tolerability of the drug.

This is an open label, first in human dose escalation trial in pediatric patients with relapsed or refractory acute myeloid leukemia to assess the safety and tolerability of increasing doses of CD33xCD3 BsAb administered subcutaneously.

A modified Bayesian Optimal Interval Design (mBOIN) design will be applied. The trial will start with accelerated titration using single patient cohorts until one grade ≥2 AE not clearly associated to underlying disease, thereafter the trial will continue with mBOIN titration.

open-label, single-arm, dose-escalation consisting of up to 12 cycles

A Phase 1 Trial of CD33xCD3 BsAb in Pediatric Patients With Relapsed or Refractory Acute Myeloid Leukemia

A Phase 1, Open-label, Dose-escalation Trial of CD33xCD3 Bispecific Antibody in Pediatric Patients With Relapsed or Refractory Acute Myeloid Leukemia

Occurrence of Adverse Events during the trial

Children's Oncology Group

Y-mAbs Therapeutics

Immune System Diseases

Lymphoma

Precursor Cell Lymphoblastic Leukemia-Lymphoma

Leukemia, Lymphoid

Lymphoproliferative Disorders

Lymphatic Diseases

Immunoproliferative Disorders

Lymphosarcoma

Childhood Acute Lymphoblastic Leukemia

Acute Lymphoblastic Leukemia

Lymphoblastic Lymphoma

Counselling interviews and workshops will be held simultaneously with the children receiving the web-based training. There are 4 modules in total. The first module lasts 2 weeks. The counselling interview will start in the 2nd week. Implementation will be completed in 5 weeks.

It includes 4 modules that develop children's communication, coping and social life skills and psychosocial preparation for the return to school process. The modules include theoretical explanations, stories, workshops and counselling sessions.

training and counselling

Gizem Cakir, PhD(c)

This study is planned to evaluate the effect of back-to-school adaptation programme on social anxiety score, coping score and back-to-school readiness score in children aged 8-17 years who are followed up with a diagnosis of cancer.

H1: Is there a significant difference in children's social anxiety scores before and after the back-to-school adaptation programme? H2: Is there a significant difference in children's coping scores before and after the back-to-school adaptation programme? H3: Is there a significant difference in children's readiness to return to school scores before and after the back-to-school adaptation programme?

Importance of research There are disease-specific medical and psychosocial difficulties that children with a diagnosis of cancer may experience. Standard school nursing practices can be helpful in meeting the physical, emotional, psychological, social and academic needs of children. However, a standard of care that is decided to be applied on return to school has not been developed. Therefore, it is thought that standard school nursing practices are not sufficient to meet the health and educational needs of children with cancer diagnosis. In the world, plans have been made using practices/methods/models appropriate for the population in the school return process of children followed up with a diagnosis of cancer. It is recommended to develop back-to-school programmes as they can benefit the child, family, peers, teachers and school staff who are followed up with a diagnosis of cancer. In our study, we aim to manage the transition care schematically with the back-to-school protocol to be made between the school-family-hospital, and to ensure care coordination and the safety of the child. The fact that the intervention in the back-to-school adaptation programme will be made to the child, parents, teachers and peers shows a holistic approach to the needs.

Place of application:

It will be performed in the paediatric leukaemia outpatient clinic of the Children's Hospital.

Study Population The population of the study consisted of children who were followed up in the paediatric leukaemia outpatient clinic of the Children's Hospital with a diagnosis of cancer.

Sample of the study The research will be conducted with a single group. In a publication on programme evaluation studies, it is recommended that the eta square should be at least 0.06 and 85% power. Based on this information, the sample size estimated by G\*Power analysis with 85% power and 5% margin of error is 26. Although there is no similar study in the literature in terms of method and measurement tools, it is seen that case loss is between 16-32% in studies conducted with similar populations within the scope of back-to-school intervention. For this reason, the calculated sample size was increased by 32% and the sample size was determined as 35.

Data Collection Tools The following forms were planned to be used in data collection.

1. Child and Parent Descriptive Information Form
2. Data Collection Form for Children's Return to School Process
3. Social Anxiety Scale for Children-Revised Version
4. Social Anxiety Scale for Adolescents
5. Paediatric Cancer Coping Scale
6. Readiness to Return to School Scale for Children with Oncological Problems: 7-18 Age
7. Process Evaluation Forms

Statistical analysis The data obtained from the research will be evaluated in SPSS 29.0 package data programme. In the analysis of the data, independent groups t-test will be applied for sociodemographic data with normal distribution. In the comparison of the measurements before and after the implementation of the back-to-school adaptation programme to the group, t-test will be used for parametric assumptions, and Wilcoxon signed-rank test will be used when parametric assumptions cannot be met. In order to measure the statistical significance of dependent groups, one-factor analysis of variance will be used if parametric assumptions are met, Friedman analysis of variance will be used if parametric assumptions are not met. The significance level for descriptive statistics is p\<0.05. Thematic content analysis method will be used in the evaluation of qualitative data.

The research is a technical/scientific/collaborative action research design.

Return to School Adaptation Programme for Children With Cancer

Efficacy of the Web-based Return to School Adaptation Programme for Children With Cancer: a Meta-theory-based Action Research

individual participant data will not be shared with other researchers. Since the data is individual, it is not ethically appropriate.

Social anxiety scale fo children-revised. Scores that can be obtained from the scale are between 18-90 and as the scale score increases, the level of social anxiety increases.

Paediatric Cancer Coping Scale. Scores that can be obtained from the scale are between 0-99 and coping strategies increase as the scale score increases.

Back to school readiness scale for children with oncological problems: 7-18 years of age. The score that can be obtained from the scale is between 0 and 152 and a higher score indicates an increase in school readiness.

Return to School Process Data Collection Form

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
AflacLL1901 (CHOA-AML)	NCT04326439	Acute Myeloid L... AML, Childhood	Cytarabine Daunorubicin Erwinase Etoposide Gemtuzumab ozog... Stem cell trans... Sorafenib	- 21 Years	Emory University
ELU001 in Pediatric Subjects Who Have Relapsed and/or Refractory CBFA2T3-GLIS2-positive AML	NCT05622591	Acute Myeloid L... AML, Childhood Relapsed Pediat... Refractory Pedi...	ELU001	1 Month - 9 Years	Elucida Oncology
AflacLL1901 (CHOA-AML)	NCT04326439	Acute Myeloid L... AML, Childhood	Cytarabine Daunorubicin Erwinase Etoposide Gemtuzumab ozog... Stem cell trans... Sorafenib	- 21 Years	Emory University
A Phase 1 Trial of CD33xCD3 BsAb in Pediatric Patients With Relapsed or Refractory Acute Myeloid Leukemia	NCT05077423	AML, Childhood	CD33*CD3 BsAb	2 Years - 21 Years	Y-mAbs Therapeutics
Return to School Adaptation Programme for Children With Cancer	NCT06389357	Childhood Cance... ALL, Childhood AML, Childhood Lymphoma Solid Tumor, Ch...	training and co...	8 Years - 17 Years	Gazi University
Return to School Adaptation Programme for Children With Cancer	NCT06389357	Childhood Cance... ALL, Childhood AML, Childhood Lymphoma Solid Tumor, Ch...	training and co...	8 Years - 17 Years	Gazi University

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