Pediatric Cancer & Blood Disorders Research, Reports & Clinical Trials

Inotuzumab Ozogamicin in Treating Younger Patients With B-Lymphoblastic Lymphoma or Relapsed or Refractory CD22 Positive B Acute Lymphoblastic Leukemia

Conditions: B-cell Acute Lymphoblastic Leukemia (B-ALL)
Principal Investigator: Maggie Fader
Learn more at https://clinicaltrials.gov/study/NCT02981628.

Inotuzumab Ozogamicin and Post-Induction Chemotherapy in Treating Patients With High-Risk B-ALL, Mixed Phenotype Acute Leukemia, and B-LLy

Conditions: Leukemia
Principal Investigator: Maggie Fader
Learn more at https://clinicaltrials.gov/study/NCT03959085.

Enasidenib for the Treatment of Relapsed or Refractory Acute Myeloid Leukemia Patients With an IDH2 Mutation

Conditions: Leukemia
Principal Investigator: Maggie Fader
Learn more at https://clinicaltrials.gov/study/NCT04203316.

The Pediatric Acute Leukemia (PedAL) Screening Trial - A Study to Test Bone Marrow and Blood in Children With Leukemia That Has Come Back After Treatment or Is Difficult to Treat - A Leukemia & Lympho

Conditions: Leukemia
Principal Investigator: Maggie Fader
Learn more at https://clinicaltrials.gov/study/NCT04726241.

A Study of the Drugs Selumetinib Versus Carboplatin/​Vincristine in Patients With Neurofibromatosis and Low-Grade Glioma

Conditions: Glioma, Neurofibromatosis
Principal Investigator: Ziad Khatib
Learn more at https://clinicaltrials.gov/study/NCT03871257.

A Study of the Drugs Selumetinib vs. Carboplatin and Vincristine in Patients With Low-Grade Glioma

Conditions: Glioma, Astrocytoma
Principal Investigator: Ziad Khatib
Learn more at https://clinicaltrials.gov/study/NCT04166409.

A Study of a New Way to Treat Children and Young Adults With a Brain Tumor Called NGGCT

Conditions: Non-germinomatous germ cell tumors (NGGCT)
Principal Investigator: Ziad Khatib
Learn more at https://clinicaltrials.gov/study/NCT04684368.

Thoracotomy Versus Thoracoscopic Management of Pulmonary Metastases in Patients With Osteosarcoma

Conditions: Osteosarcoma, Metastatic Malignant Neoplasm in the Lung
Principal Investigator: Ziad Khatib
Learn more at https://clinicaltrials.gov/study/NCT05235165.

Active Surveillance, Bleomycin, Etoposide, Carboplatin or Cisplatin in Treating Pediatric and Adult Patients With Germ Cell Tumors

Conditions: Germ Cell Tumor
Principal Investigator: Maggie Fader
Learn more at https://clinicaltrials.gov/study/NCT03067181.

Accelerated v's Standard BEP Chemotherapy for Patients With Intermediate and Poor-risk Metastatic Germ Cell Tumours (P3BEP)

Conditions: Germ Cell Tumor
Principal Investigator: Maggie Fader
Learn more at https://clinicaltrials.gov/study/NCT02582697.

The EndRAD Trial: Eliminating Total Body Irradiation (TBI) for NGS-MRD Negative Children, Adolescents, and Young Adults With B-ALL

Conditions: B-cell Acute Lymphoblastic Leukemia (B-ALL)
Principal Investigator: Jorge Galvez-Silva
Learn more at https://clinicaltrials.gov/study/NCT03509961.

Pediatric Precision Laboratory Advanced Neuroblastoma Therapy (PEDS-PLAN)

Conditions: Neuroblastoma
Principal Investigator: Guillermo De Angulo
Learn more at https://clinicaltrials.gov/study/NCT02559778.

A Study With Tovorafenib (DAY101) as a Treatment Option for Progressive, Relapsed, or Refractory Langerhans Cell Histiocytosis

Conditions: Langerhans Cell Histiocytosis
Principal Investigator: Maggie Fader
Learn more at https://clinicaltrials.gov/study/NCT05828069.

A Study of Combination Chemotherapy for Patients With Newly Diagnosed DAWT and Relapsed FHWT

Conditions: Wilms Tumor
Principal Investigator: Maggie Fader
Learn more at https://clinicaltrials.gov/study/NCT04322318.

Chemotherapy for the Treatment of Patients With Newly Diagnosed Very Low-Risk and Low Risk Fusion Negative Rhabdomyosarcoma

Conditions: Sarcoma
Principal Investigator: Maggie Fader
Learn more at https://clinicaltrials.gov/study/NCT05304585.

Study to Learn More About the Safety and Effectiveness of the Drug VITRAKVI During Routine Use in Patients With TRK Fusion Cancer Which is Locally Advanced or Spread From the Place Where it Started to

Conditions: Advanced or Metastatic Solid Tumor
Principal Investigator: Ossama Maher
Learn more at https://clinicaltrials.gov/study/NCT04142437.

Studying the Effect of Levocarnitine in Protecting the Liver From Chemotherapy for Leukemia or Lymphoma

Conditions: Acute Lymphoblastic Leukemia (ALL)
Principal Investigator: Maggie Fader
Learn more at https://clinicaltrials.gov/study/NCT05602194.

A Study to Compare Standard Therapy to Treat Hodgkin Lymphoma to the Use of Two Drugs, Brentuximab Vedotin and Nivolumab

Conditions: Hodgkin's Lymphoma
Principal Investigator: Maggie Fader
Learn more at https://clinicaltrials.gov/study/NCT05675410.

A Study of Lower Radiotherapy Dose to Treat Children With CNS Germinoma

Conditions: Basal Ganglia Germinoma
Principal Investigator: Ziad Khatib
Learn more at https://clinicaltrials.gov/study/NCT06368817.

A Study Comparing Abemaciclib Plus Temozolomide to Temozolomide Monotherapy in Children and Young Adults With High-grade Glioma Following Radiotherapy

Principal Investigator: Ziad Khatib
Learn more at https://clinicaltrials.gov/study/NCT06413706.

Naxitamab Added to Induction for Newly Diagnosed High-Risk Neuroblastoma

Conditions: Neuroblastoma
Principal Investigator: Maggie Fader
Learn more at https://clinicaltrials.gov/study/NCT05489887.

A Study of Revumenib in Combination With Chemotherapy for Patients Diagnosed With Relapsed or Refractory Leukemia

Conditions: Leukemia
Principal Investigator: Maggie Fader
Learn more at https://clinicaltrials.gov/study/NCT05761171.

DFMO as Maintenance Therapy for Molecular High/​Very High Risk and Relapsed Medulloblastoma

Conditions: Medulloblastoma
Principal Investigator: Ossama Maher
Learn more at https://clinicaltrials.gov/study/NCT04696029.

Dinutuximab With Chemotherapy, Surgery and Stem Cell Transplantation for the Treatment of Children With Newly Diagnosed High Risk Neuroblastoma

Conditions: Ganglioneruoblastoma, Neuroblastoma
Principal Investigator: Maggie Fader
Learn more at https://clinicaltrials.gov/study/NCT06172296.

Mismatched Related Donor Versus Matched Unrelated Donor Stem Cell Transplantation for Children, Adolescents, and Young Adults With Acute Leukemia or Myelodysplastic Syndrome

Conditions: Leukemia, Myelodysplastic Syndrome
Principal Investigator: Jorge Galvez-Silva
Learn more at https://clinicaltrials.gov/study/NCT05457556.

• AALL1631 – International Phase 3 Trial in Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia Ph+ ALL Testing Imatinib in Combination with Two Different Cytotoxic Chemotherapy Backbones.
• AALL1732 – A Phase 3 Randomized Trial of Inotuzumab Ozogamicin (IND#:133494, NSC#: 772518) for Newly Diagnosed High-Risk B-ALL; Risk-Adapted Post-Induction Therapy for High-Risk B-ALL, Mixed Phenotype Acute Leukemia, and Disseminated B-LLy.
• AAML1531 – Risk-stratified Therapy for Acute Myeloid Leukemia in Down Syndrome.
• AAML1831 – A phase 3 randomized trial for patients with de novo AML comparing standard therapy including gemtuzumab ozogamicin GO to CPX-351 with GO, and the addition of the FLT3 inhibitor gilteritinib for patients with FLT3 mutations.
• AAML18P1 – Stopping Tyrosine Kinase Inhibitors (TKI) to Assess Treatment-Free Remission (TFR) in Pediatric Chronic Myeloid Leukemia - Chronic Phase (CML-CP).

• ACNS1422 – A Phase 2 Study of Reduced Therapy for Newly Diagnosed Average-Risk WNT-Driven Medulloblastoma Patients.
• ACNS1831 – A Phase 3 Randomized Study of Selumetinib (IND # 77782) versus Carboplatin/Vincristine in Newly Diagnosed or Previously Untreated Neurofibromatosis Type 1 (NF1) Associated Low-Grade Glioma (LGG)
• ACNS1833 – A Phase 3 Randomized Non-Inferiority Study of Carboplatin and Vincristine versus Selumetinib (NSC# 748727, IND# 77782) in Newly Diagnosed or Previously Untreated Low-Grade Glioma (LGG) not associated with BRAFV600E Mutations or Systemic Neurofibromatosis Type 1 (NF1)

• ADVL1521 – A Phase 2 Study of the MEK inhibitor Trametinib (IND#119346, NSC#763093) in Children with Relapsed or Refractory Juvenile Myelomonocytic Leukemia.
• ADVL1823 – Larotrectinib (LOXO-101, NSC# 788607, IND# 141824) for Previously Untreated TRK Fusion Pediatric Solid Tumors and TRK Fusion Relapsed Pediatric Acute Leukemias.

• AGCT1531 – A Phase 3 Study of Active Surveillance for Low Risk and a Randomized Trial of Carboplatin vs. Cisplatin for Standard Risk Pediatric and Adult Patients with Germ Cell Tumors.
• AGCT1532 – A Randomized Phase 3 Trial of Accelerated versus Standard BEP Chemotherapy for Patients with Intermediate and Poor-risk Metastatic Germ Cell Tumors.
• A031102 – A Randomized Phase III Trial Comparing Conventional-dose Chemotherapy using Paclitaxel, Ifosfamide, and Cisplatin (TIP) with High-dose Chemotherapy using Mobilizing Paclitaxel plus Ifosfamide followed by High-dose Carboplatin and Etoposide (TI-CE) as First Salvage Treatment in Relapsed or Refractory Germ Cell Tumors.

• S1826 – A Phase III, Randomized Study of Nivolumab (Opdivo) Plus AVD or Brentuximab Vedotin (Adcetris) Plus AVD in Patients (Age >/= 12 Years) with Newly Diagnosed Advanced Stage Classical Hodgkin LymphomaHodgkin Lymphoma.

• AHEP1531 – Pediatric Hepatic Malignancy International Therapeutic Trial (PHITT).

• ANBL00B1 – Neuroblastoma Classification Biology Studies.
• ANBL1232 – Utilizing Response- and Biology-Based Risk Factors to Guide Therapy in Patients with Non-High-Risk Neuroblastoma.
• ANBL1531 – A Phase 3 Study of 131I-Metaiodobenzylguanidine (131I-MIBG) or Crizotinib Added to Intensive Therapy for Children with Newly Diagnosed High-Risk Neuroblastoma (NBL) (IND# 134379) Arm C Temporarily Closed.
• ANBL1821 – A Phase 2 Randomized Study of Irinotecan/Temozolomide/Dinutuximab with or without Eflornithine (DFMO) (IND# 141913) in Children with Relapsed, Refractory or Progressive Neuroblastoma.

• APEC14B1 – Project: EveryChild A Registry, Eligibility Screening, Biology and Outcome Study.

• APEC1621A – Phase 2 subprotocol of LOXO-101 (larotrectinib) in patients with tumors harboring actionable NTRK fusions.
• APEC1621B – Phase 2 subprotocol of JNJ-42756493 (Erdafitinib) in patients with tumors harboring FGFR1/2/3/4 alterations.
• APEC1621D – Phase 2 subprotocol of LY3023414 in Solid Tumors.
• APEC1621F – Phase 2 subprotocol of Ensartinib in patients with tumors harboring ALK or ROS1 genomic alterations.
• APEC1621G – Phase 2 subprotocol of Vemurafenib in patients with tumors harboring actionable BRAF V600 mutations.
• APEC1621H – Phase 2 subprotocol of olaparib in patients with tumors harboring defects in DNA damage repair genes.
• APEC1621I – Phase 2 subprotocol of Palbociclib in Patients with Tumors Harboring Activating Alterations in Cell Cycle Genes.
• APEC1621K – Phase 2 Subprotocol of AG-120 (Ivosidenib) in Patients with Tumors Harboring IDH1 Mutations.
• APEC1621M – Phase 2 subprotocol of Tipifarnib in patients with tumors harboring HRAS genomic alterations.
• APEC1621N – Phase 2 Subprotocol of LOXO-292 in Patients with Tumors Harboring RET Gene Alterations.
• APEC1621SC – NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) Screening Protocol

• AREN03B2 – Renal Tumors Classification, Biology, and Banking Study.
• AREN1721 – A Randomized Phase 2 Trial of Axitinib/Nivolumab combination therapy vs single agent Nivolumab for the treatment of TFE/Translocation Renal Cell Carcinoma across all age groups.

• ARST1431 – A Randomized Phase 3 Study of Vincristine, Dactinomycin, Cyclophosphamide (VAC) Alternating with Vincristine and Irinotecan (VI) Versus VAC/VI Plus Temsirolimus (TORI, Torisel, NSC# 683864) in Patients with Intermediate Risk (IR) Rhabdomyosarcoma (RMS).

• ALTE07C1 – Neuropsychological, Social, Emotional and Behavioral Outcomes in Children with Cancer.

• Novartis CPKC412A2218 [Dr. De Angulo] – Phase II, Open-Label, Single Arm Study To Evaluate The Safety, Efficacy, And Pharmacokinetics Of Twice Daily Midostaurin (PKC412) Combined With Standard Chemotherapy And As A Single Agent Post-Consolidation Therapy In Children With Untreated FLT3-Mutated AML.

• PBMTC_ONC1701 - EndRad [Dr. Galvez] – A Phase II Pilot Trial To Estimate Survival After A Non-Total Body Irradiation (TBI) Based Conditioning Regimen In Patients Diagnosed With B-Acute Lymphoblastic Leukemia (ALL) Who Are Pre-Allogeneic Hematopoietic Cell Transplantation (HCT) Next-Generation-Sequence (NGS) Minimal Residual Disease (MRD) Negative.
• CIBMTR 2500 [Dr. Galvez] – Protocol for a research database for hematopoietic cell transplantation, other cellular therapies and marrow toxic injuries.
• CIBMTR 2501 [Dr. Galvez] – Protocol for a research sample repository for hematopoietic cell transplantation, other cellular therapies and marrow toxic injuries.
• 10 CBA  [Dr. Galvez] – A Multicenter Access And Distribution Protocol For unlicensed Cryopreserved Cord Blood Units (Cbus) For Transplantation In Pediatric And Adult Patients With Hematologic Malignancies And Other Indications.
• 1507 [Dr. Galvez] – Reduced Intensity Conditioning for Haploidentical Bone Marrow Transplantation in Patients with Symptomatic Sickle Cell Disease.
• WASHU 01-0923  [Dr. Galvez] – A Study Of Hematopoietic Stem Cell Transplantation (HSCT) In Pediatric Nonmalignant Disease Using A Reduced-Intensity Preparatory Regime.
• PBMTC SUP1801 17-SIBS (Donor Kids) [Dr. Galvez] – Identifying Predictors of Poor Health-Related Quality-of-Life among Pediatric Hematopoietic Stem Cell Donors.
• Astellas 9766-CL-0107 [Dr. Galvez] – A Phase 2, Open-Label, Non-Comparative, Multicenter Study To Evaluate the Safety and Tolerability, Efficacy and Pharmacokinetics of Isavuconazonium Sulfate for the Treatment of Invasive Aspergillosis (IA) or Invasive Mucormycosis (IM) in Pediatric Subjects.

• Head Start 4 [Dr. Khatib] – Newly Diagnosed Children (Less Than 10 Years Old) With Medulloblastoma And Other Central Nervous System Embryonal Tumor.

• Ex-Vivo Drug Screen [Dr. Salyakina] – Personalized Ex Vivo Drug Screening and Genomic Profiling to Guide Individualized Treatments for Children with Relapsed or Refractory Solid Tumors and Leukemias.
• CHLA Onco-Kids [Dr. Fader] – Next-Generation Sequencing of Pediatric Tumor Samples (OncoKids®) from a Multi-Institutional Consortium of Clinical Cancer Programs

• NMTRC012 [Dr. De Angulo] – A Study Using Molecular Guided Therapy with Induction Chemotherapy followed by a Randomized Controlled Trial of standard immunotherapy with or without DFMO followed by DFMO maintenance for Subjects with Newly Diagnosed High-Risk Neuroblastoma.

• Nourish-T+ [Dr. Abdella] – A Randomized Control Trial Targeting Parents in Promoting Healthy Eating and Exercise Behaviors in Pediatric Cancer Survivors with Overweight/Obesity.
• Pfizer B0661037 [Dr. Fader] – A Randomized, Open-Label, Active Controlled, Safety And Descriptive Efficacy Study In Pediatric Subjects Requiring Anticoagulation For The Treatment Of A Venous Thromboembolic Event.

• BIO-11006 [Dr. De Angulo] – A Phase 2 Study of the Safety and Efficacy of BIO-11006 in the Treatment of Recurrent Osteosarcoma and Ewing’s Sarcoma in Patients with Lung Metastases.
• Ex-Vivo Observation [Dr. Fader] – Drug Sensitivity Testing and Mutation Profiling in Childhood Sarcomas.