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

  • A Study of Combination Chemotherapy for Patients With Newly Diagnosed DAWT and Relapsed FHWT
    Study ID: ONC99274 (AREN1921)
    PI: Maggie Fader. View Study Details

  • 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 Other Places in the Body (ON-TRK)
    Study ID: ONC99126 (ON-TRK)
    PI: Ossama Maher. View Study Details

  • DFMO as Maintenance Therapy for Molecular High/​Very High Risk and Relapsed Medulloblastoma
    Study ID: ONC24008 (BCC016)
    PI: Ossama Maher. View Study Details

  • Accelerated v's Standard BEP Chemotherapy for Patients With Intermediate and Poor-risk Metastatic Germ Cell Tumours (P3BEP)
    Study ID: ONC99078 (AGCT1532)
    PI: Maggie Fader. View Study Details

  • PEP-CMV Vaccine Targeting CMV Antigen to Treat Newly Diagnosed Pediatric HGG and DIPG and Recurrent Medulloblastoma
    Study ID: ONC23174 (CONNECT1906)
    PI: Ziad Khatib. View Study Details

  • A Study With Tovorafenib (DAY101) as a Treatment Option for Progressive, Relapsed, or Refractory Langerhans Cell Histiocytosis
    Study ID: ONC23006 (ANHL2121)
    PI: Maggie Fader. View Study Details

  • MEKTOVI® for the Treatment of Pediatric Adamantinomatous Craniopharyngioma
    Study ID: ONC23127 (CONNECT2108)
    View Study Details

  • ACTEMRA® for the Treatment of Pediatric Adamantinomatous Craniopharyngioma
    Study ID: ONC23173 (CONNECT1905)
    PI: Ziad Khatib. View Study Details

  • 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 Study
    Study ID: ONC99101 (APAL2020SC)
    PI: Maggie Fader. View Study Details

  • Pediatric Precision Laboratory Advanced Neuroblastoma Therapy (PEDS-PLAN)
    Study ID: ONC99113 (NMTRC012)
    PI: Guillermo De Angulo. View Study Details

  • A Study Comparing Abemaciclib Plus Temozolomide to Temozolomide Monotherapy in Children and Young Adults With High-grade Glioma Following Radiotherapy
    Study ID: ONC23162 (I3Y-MC-JPEH)
    PI: Ziad Khatib. View Study Details

  • Studying the Effect of Levocarnitine in Protecting the Liver From Chemotherapy for Leukemia or Lymphoma
    Study ID: ONC23150 (ACCL1931)
    PI: Maggie Fader. View Study Details

  • A Study to Compare Standard Therapy to Treat Hodgkin Lymphoma to the Use of Two Drugs, Brentuximab Vedotin and Nivolumab
    Study ID: ONC99271 (AHOD2131)
    PI: Maggie Fader. View Study Details

  • Chemotherapy for the Treatment of Patients With Newly Diagnosed Very Low-Risk and Low Risk Fusion Negative Rhabdomyosarcoma
    Study ID: ONC99276 (ARST2032)
    PI: Maggie Fader. View Study Details

  • A Study of Lower Radiotherapy Dose to Treat Children With CNS Germinoma
    Study ID: ONC25006 (ACNS2321)
    PI: Ziad Khatib. View Study Details

  • Naxitamab Added to Induction for Newly Diagnosed High-Risk Neuroblastoma
    Study ID: ONC23118 (BCC018)
    PI: Maggie Fader. View Study Details

  • Dinutuximab With Chemotherapy, Surgery and Stem Cell Transplantation for the Treatment of Children With Newly Diagnosed High Risk Neuroblastoma
    Study ID: ONC24030 (ANBL2131)
    PI: Maggie Fader. View Study Details

  • Active Surveillance, Bleomycin, Etoposide, Carboplatin or Cisplatin in Treating Pediatric and Adult Patients With Germ Cell Tumors
    Study ID: ONC99077 (AGCT1531)
    PI: Maggie Fader. View Study Details

  • Inotuzumab Ozogamicin and Post-Induction Chemotherapy in Treating Patients With High-Risk B-ALL, Mixed Phenotype Acute Leukemia, and B-LLy
    Study ID: ONC99062 (AALL1732)
    PI: Maggie Fader. View Study Details

  • A Study of the Drugs Selumetinib Versus Carboplatin/​Vincristine in Patients With Neurofibromatosis and Low-Grade Glioma
    Study ID: ONC99073 (ACNS1831)
    PI: Ziad Khatib. View Study Details

  • A Study of the Drugs Selumetinib vs. Carboplatin and Vincristine in Patients With Low-Grade Glioma
    Study ID: ONC99074 (ACNS1833)
    PI: Ziad Khatib. View Study Details

  • Thoracotomy Versus Thoracoscopic Management of Pulmonary Metastases in Patients With Osteosarcoma
    Study ID: ONC99100 (AOST2031)
    PI: Ziad Khatib. View Study Details

  • A Study of a New Way to Treat Children and Young Adults With a Brain Tumor Called NGGCT
    Study ID: ONC99099 (ACNS2021)
    PI: Ziad Khatib. View Study Details

  • A Study Using Nivolumab, in Combination With Chemotherapy Drugs to Treat Nasopharyngeal Carcinoma (NPC)
    Study ID: ONC24032 (ARAR2221)
    PI: Maggie Fader. View Study Details

  • A Study of Revumenib in Combination With Chemotherapy for Patients Diagnosed With Relapsed or Refractory Leukemia
    Study ID: ONC24004 (AALL2121)
    PI: Maggie Fader. View Study Details

  • Inotuzumab Ozogamicin in Treating Younger Patients With B-Lymphoblastic Lymphoma or Relapsed or Refractory CD22 Positive B Acute Lymphoblastic Leukemia
    Study ID: ONC99060 (AALL1621)
    PI: Maggie Fader. View Study Details