The Cancer and Blood Disorders Institute at Nicklaus Children's is engaged in more than 80 clinical cancer research studies, all of which are focused on finding new cures, treatments and diagnostic procedures for pediatric malignancies. No hospital in the region features such a wide array of clinical trials for childhood cancer research. As such, the Cancer and Blood Disorders Institute offers its patients unparalleled hope, offering the latest drug treatments, medical devices, biotechnology and other technological innovations in the fight against pediatric cancer.
The Children's Oncology Group (COG)
The Cancer and Blood Disorders Institute at Nicklaus Children's has been recognized as a full member of the prestigious Children’s Oncology Group (COG), a nationally funded group of pioneering physicians and institutions. To maintain this standing, full compliance with COG requirements is mandated. Compliance in this group involves not only participation in and adherence to multi-center research protocols, but also submission of accurate and timely data. The institute has also been awarded a grant by the National Cancer Institute to advance childhood cancer research. In addition, members of the team share their knowledge through publication of clinical research study findings and presentations to national societies of physician scientists.
Laboratory Cancer Research
Significant progress has been made in the early development of new treatments and diagnosis through childhood cancer research in laboratories at Nicklaus Children's. Molecular and genetic diagnosis and classification of pediatric brain tumors is being developed by micro-array technology. This leading-edge approach is being used for diagnostic and prognostic evaluation of malignant solid tumors. Also underway is work focused on the roles of cell growth in the development of drug resistance in pediatric cancers. In an exciting new effort, Nicklaus Children's has made progress in evaluating alternative medicines that are used to treat cancer in other cultures.
Brain Cancer Research Program
Nicklaus Children's Hospital and Research Institute has initiated pediatric neuro/oncology research as a collaborative effort between the Divisions of Pathology, Hematology/Oncology, Blood and Marrow Transplantation and Neurological Surgery. Several investigators from these departments are involved in brain cancer research to improve the brain cancer treatment and disease free survival.
The major focus of the brain cancer research program is to understand the mechanisms behind the failure of chemotherapy and radiation therapy, and the reasons for the aggressiveness and invasiveness of brain tumors. The program is investigating the prognostic significance several genetic markers associated with cellular oncogenesis, drug resistance, cell proliferation, angiogenesis, differentiation and programmed cell death (apoptosis). The role of telomerase enzyme in the oncogenesis and aggressive behavior of certain brain tumors is also investigated in this research program. Alternative Medicine Projects include evaluation of the anticancer effects of curcumin and other "natural" compounds.
Through brain tumor research, our aim is to understand the origin, diagnosis, development and differentiation of brain tumor cells during the process of carcinogenesis. Simultaneously, we plan to translate the brain tumor research findings from evaluation of prognostic factors into the treatment of brain cancer patients.
Clinical trials including stem cell transplantation and anti-angiogenesis protocols are being used for patients with brain tumors. Young children are treated without radiation therapy to avoid late effects.
Active COG Trials
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- 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)
Various Tumors Trials
- 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.
Germ Cell Trials
- 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.
Registry and Biology
- 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.
Active Non-COG Trials
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- 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.
Various Tumor Trials
- 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.
Published reports are in compliance with the ACoS, CoC and are standard specific.