Pediatric Cancer & Blood Disorders Research, Reports & Clinical Trials
The Helen & Jacob Shaham Cancer and Blood Disorders Institute at Nicklaus Children's is engaged in more than 40 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 Shaham Cancer & 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.
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.
The Children's Oncology Group (COG)
The Shaham Cancer & 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.
Ongoing Clinical Trials
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A Study Comparing Abemaciclib Plus Temozolomide to Temozolomide Monotherapy in Children and Young Adults With High-grade Glioma Following RadiotherapyStudy ID:Principal Investigator:
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A Study of Combination Chemotherapy for Patients With Newly Diagnosed DAWT and Relapsed FHWTStudy ID:Principal Investigator:
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A Study of Lower Radiotherapy Dose to Treat Children With CNS GerminomaStudy ID:Principal Investigator:
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A Study of the Drugs Selumetinib vs. Carboplatin and Vincristine in Patients With Low-Grade GliomaStudy ID:Principal Investigator:
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A Study of Treatment for Medulloblastoma Using Sodium Thiosulfate to Reduce Hearing LossStudy ID:Principal Investigator:
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A Study to Compare Standard Chemotherapy to Therapy With CPX-351 and/or Gilteritinib for Patients With Newly Diagnosed AML With or Without FLT3 MutationsStudy ID:Principal Investigator:
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A Study to Compare Standard Therapy to Treat Hodgkin Lymphoma to the Use of Two Drugs, Brentuximab Vedotin and NivolumabStudy ID:Principal Investigator:
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A Study to Test the Addition of the Drug Cabozantinib to Chemotherapy in Patients With Newly Diagnosed OsteosarcomaStudy ID:Principal Investigator:
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A Study Using Nivolumab, in Combination With Chemotherapy Drugs to Treat Nasopharyngeal Carcinoma (NPC)Study ID:Principal Investigator:
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A Study With Tovorafenib (DAY101) as a Treatment Option for Progressive, Relapsed, or Refractory Langerhans Cell HistiocytosisStudy ID:Principal Investigator:
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A Trial Comparing Unrelated Donor BMT With IST for Pediatric and Young Adult Patients With Severe Aplastic Anemia (TransIT, BMT CTN 2202) (TransIT)Study ID:Principal Investigator:
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Accelerated v's Standard BEP Chemotherapy for Patients With Intermediate and Poor-risk Metastatic Germ Cell Tumours (P3BEP)Study ID:Principal Investigator:
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ACTEMRA® for the Treatment of Pediatric Adamantinomatous CraniopharyngiomaStudy ID:Principal Investigator:
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Active Surveillance, Bleomycin, Etoposide, Carboplatin or Cisplatin in Treating Pediatric and Adult Patients With Germ Cell TumorsStudy ID:Principal Investigator:
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Chemotherapy for the Treatment of Patients With Newly Diagnosed Very Low-Risk and Low Risk Fusion Negative RhabdomyosarcomaStudy ID:Principal Investigator:
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DAY101 vs. Standard of Care Chemotherapy in Pediatric Participants With Low-Grade Glioma Requiring First-Line Systemic Therapy (LOGGIC/FIREFLY-2)Study ID:Principal Investigator:
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DFMO as Maintenance Therapy for Molecular High/Very High Risk and Relapsed MedulloblastomaStudy ID:Principal Investigator:
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Dinutuximab With Chemotherapy, Surgery and Stem Cell Transplantation for the Treatment of Children With Newly Diagnosed High Risk NeuroblastomaStudy ID:Principal Investigator:
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Inotuzumab Ozogamicin and Post-Induction Chemotherapy in Treating Patients With High-Risk B-ALL, Mixed Phenotype Acute Leukemia, and B-LLyStudy ID:Principal Investigator:
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Inotuzumab Ozogamicin in Treating Younger Patients With B-Lymphoblastic Lymphoma or Relapsed or Refractory CD22 Positive B Acute Lymphoblastic LeukemiaStudy ID:Principal Investigator:
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MEKTOVI® for the Treatment of Pediatric Adamantinomatous CraniopharyngiomaStudy ID:Principal Investigator:
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Naxitamab Added to Induction for Newly Diagnosed High-Risk NeuroblastomaStudy ID:Principal Investigator:
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Pediatric Precision Laboratory Advanced Neuroblastoma Therapy (PEDS-PLAN)Study ID:Principal Investigator:
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PEP-CMV Vaccine Targeting CMV Antigen to Treat Newly Diagnosed Pediatric HGG and DIPG and Recurrent MedulloblastomaStudy ID:Principal Investigator:
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Phase I/II Trial: Engineered Donor Graft (Orca Q) for Pediatric Hematopoietic Cell Transplant (HCT)Study ID:Principal Investigator:
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Safety and Efficacy of Oral Belumosudil in Black or African American, American Indian or Alaska Native, and Native Hawaiian or Other Pacific Islander Male and Female Participants Aged 12 Years and Above With Chronic Graft Versus Host Disease (cGVHD) After At Least 2 Prior Lines of Systemic TherapyStudy ID:Principal Investigator:
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Silmitasertib (CX-4945) in Combination With Chemotherapy for Relapsed Refractory Solid TumorsStudy ID:Principal Investigator:
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Studying the Effect of Levocarnitine in Protecting the Liver From Chemotherapy for Leukemia or LymphomaStudy ID:Principal Investigator:
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The EndRAD Trial: Eliminating Total Body Irradiation (TBI) for NGS-MRD Negative Children, Adolescents, and Young Adults With B-ALLStudy ID:Principal Investigator:
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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 & Lymphoma Society and Children's Oncology Group StudyStudy ID:Principal Investigator:
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Thoracotomy Versus Thoracoscopic Management of Pulmonary Metastases in Patients With OsteosarcomaStudy ID:Principal Investigator:



