Research Institute Building

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To inquire about current studies please contact Nicklaus Children's Research Institute  at:

Ending the Diagnostic Odyssey: Whole Genome Sequencing (WGS) to Identify Genetic Determinants of Previously Undiagnosed Disease in Children

The goal of this collaborative research is to study human genomes in children with suspected congenital disease, multiple-congenital anomalies and/or multi-organ disease of unknown etiology by understanding the potential value of Whole Genome Sequencing (WGS) in establishing genetic diagnosis. The study will examine diagnosis rates, changes in clinical care as a result of a genetic diagnosis, health economics including potential cost-effectiveness of WGS and patient and provider experience with genomic medicine.

07/17/2018

Rapid Whole Genome Sequencing (rWGS): Rapid Genomic Sequencing for Acutely Ill Patients and the Collection, Storage, Analysis, and Distribution of Biological Samples, Genomic and Clinical Data

This collaborative study seeks to provide rWGS as a research test to additional pediatric hospitals nationwide to assist in the rapid diagnosis of acutely ill children suspected of a genetic condition. The study will examine diagnosis rates, changes in clinical care as a result of a genetic diagnosis, and health economics including potential cost-effectiveness of rWGS.

07/17/2018

Research Study: How your child's genetic makeup can impact the way he or she responds to certain medications

Enzymes are particularly important in the processing of medicines and drugs. They determine how our bodies are going to handle the medicines we take, and if those medicines are going to help us feel better or not. This research study includes testing the status of the CYP2D6 gene in children.

05/10/2017

List of Clinical Research Studies Nicklaus Children's Hospital is participating in:

Recruiting:

Phase II Study of Intraventricular Methotrexate in Children With Recurrent or Progressive Malignant Brain Tumors

Conditions: Recurrent Childhood Medulloblastoma;   Recurrent Childhood Ependymoma;   Childhood Atypical Teratoid/Rhabdoid Tumor;   Embryonal Tumor With Abundant Neuropil and True Rosettes;   Metastatic Malignant Neoplasm to the Leptomeninges
Interventions: Drug: Intra thecal methotrexate;   Drug: topotecan;   Drug: cyclophosphamide
Age: up to 22 Years   (Child, Adult)
Study Start: February 2016
Study Completion: February 2020
Conditions: Hypofibrinogenemia;   Afibrinogenemia;   Bleeding Disorders
Interventions: Drug: RiaStAP;   Drug: Saline
Age: up to 1 Year   (Child)
Study Start: January 2017
Study Completion: April 2019
Condition: Benign Centrally-Located Intracranial Tumors
Intervention: Device: ExAblate 4000 System
Age: 8 Years to 22 Years   (Child, Adult)
Study Start: January 12, 2017
Study Completion: January 15, 2019
Not yet recruiting:

COMPASSION XT PAS - Post-approval Study of the SAPIEN XT THV in Patients With Pulmonary Valve Dysfunction

Conditions: Pulmonary Valve Insufficiency;   Pulmonary Valve Stenosis;   Heart Defects, Congenital;   Congenital Abnormalities;   Cardiovascular Diseases;   Heart Diseases
Intervention: Device: SAPIEN XT THV
Age: 19 Years and older   (Adult, Senior)
Study Start: May 2017
Study Completion: January 2024
Condition: Epilepsy
Interventions: Drug: cannabidiol;   Other: Blood Test
Age: 2 Years to 16 Years   (Child)
Study Start:  
Study Completion:  
Condition: Epilepsy
Intervention: Drug: Ganaxolone
Age: 2 Years to 18 Years   (Child, Adult)
Study Start: March 2015
Study Completion: July 2018
Condition: Hyperphosphatemia
Interventions: Drug: PA21 (Velphoro®);   Drug: Calcium Acetate (Phoslyra®)
Age: up to 18 Years   (Child, Adult)
Study Start: June 2016
Study Completion:  
Conditions: Germ Cell Tumor;   Teratoma;   Choriocarcinoma;   Germinoma;   Mixed Germ Cell Tumor;   Yolk Sac Tumor;   Childhood Teratoma;   Malignant Germ Cell Neoplasm;   Extragonadal Seminoma;   Non-seminomatous Germ Cell Tumor;   Seminoma
Interventions: Drug: paclitaxel;   Drug: ifosfamide;   Drug: cisplatin;   Drug: pegylated G-CSF;   Drug: G-CSF;   Drug: carboplatin;   Drug: etoposide phosphate;   Procedure: stem cell reinfusion
Age: 14 Years and older   (Child, Adult, Senior)
Study Start: March 2015
Study Completion:  
Condition: Persistent Pulmonary Hypertension of the Newborn
Interventions: Drug: IV Remodulin;   Drug: Placebo
Age: up to 14 Days   (Child)
Study Start: May 2015
Study Completion: January 2018
Conditions: Ganglioneuroblastoma;   Recurrent Neuroblastoma
Interventions: Biological: Dinutuximab;   Drug: Irinotecan Hydrochloride;   Other: Laboratory Biomarker Analysis;   Biological: Sargramostim;   Drug: Temozolomide;   Drug: Temsirolimus
Age: Child, Adult, Senior
Study Start: February 2013
Study Completion:  
Conditions: PRETEXT Stage 1 Hepatoblastoma;   PRETEXT Stage 2 Hepatoblastoma;   PRETEXT Stage 3 Hepatoblastoma;   PRETEXT Stage 4 Hepatoblastoma
Interventions: Drug: Cisplatin;   Drug: Doxorubicin Hydrochloride;   Drug: Fluorouracil;   Drug: Irinotecan Hydrochloride;   Other: Laboratory Biomarker Analysis;   Procedure: Liver Transplantation;   Drug: Temsirolimus;   Procedure: Therapeutic Conventional Surgery;   Drug: Vincristine Sulfate
Age: up to 21 Years   (Child, Adult)
Study Start: September 2009
Study Completion:  
Conditions: Classical Hodgkin Lymphoma;   Stage IIB Hodgkin Lymphoma;   Stage IIIB Hodgkin Lymphoma;   Stage IVA Hodgkin Lymphoma;   Stage IVB Hodgkin Lymphoma
Interventions: Biological: Bleomycin Sulfate;   Drug: Brentuximab Vedotin;   Drug: Cyclophosphamide;   Drug: Doxorubicin Hydrochloride;   Drug: Etoposide;   Other: Laboratory Biomarker Analysis;   Drug: Prednisone;   Other: Quality-of-Life Assessment;   Other: Questionnaire Administration;   Drug: Vincristine Sulfate
Age: 2 Years to 18 Years   (Child, Adult)
Study Start: March 2015
Study Completion:  
Conditions: B Acute Lymphoblastic Leukemia;   Bone Necrosis;   Central Nervous System Leukemia;   Cognitive Side Effects of Cancer Therapy;   Neurotoxicity Syndrome;   Pain;   Testicular Leukemia;   Therapy-Related Toxicity;   Untreated Adult Acute Lymphoblastic Leukemia;   Untreated Childhood Acute Lymphoblastic Leukemia
Interventions: Drug: Clofarabine;   Drug: Cyclophosphamide;   Drug: Cytarabine;   Drug: Dasatinib;   Drug: Dexamethasone;   Drug: Doxorubicin Hydrochloride;   Drug: Etoposide;   Drug: Hydrocortisone Sodium Succinate;   Other: Laboratory Biomarker Analysis;   Drug: Leucovorin Calcium;   Drug: Mercaptopurine;   Drug: Methotrexate;   Drug: Pegaspargase;   Drug: Prednisone;   Radiation: Radiation Therapy;   Drug: Thioguanine;   Drug: Vincristine Sulfate
Age: 1 Year to 30 Years   (Child, Adult)
Study Start: February 2012
Study Completion:  
Recruiting:

Biomarkers in Tumor Tissue Samples From Patients With Newly Diagnosed Neuroblastoma or Ganglioneuroblastoma

Conditions: Disseminated Neuroblastoma;   Ganglioneuroblastoma;   Localized Resectable Neuroblastoma;   Localized Unresectable Neuroblastoma;   Regional Neuroblastoma;   Stage 4S Neuroblastoma
Interventions: Other: laboratory biomarker analysis;   Other: cytology specimen collection procedure
Age: up to 30 Years   (Child, Adult)
Study Start: November 2000
Study Completion:  
Recruiting:

Risk-Adapted Chemotherapy in Treating Younger Patients With Newly Diagnosed Standard-Risk Acute Lymphoblastic Leukemia or Localized B-Lineage Lymphoblastic Lymphoma

Conditions: Adult B Lymphoblastic Lymphoma;   Childhood B Acute Lymphoblastic Leukemia;   Childhood B Acute Lymphoblastic Leukemia With t(9;22)(q34;q11.2); BCR-ABL1;   Childhood B Lymphoblastic Lymphoma;   Down Syndrome;   Stage I B Lymphoblastic Lymphoma;   Stage II B Lymphoblastic Lymphoma;   Untreated Adult Acute Lymphoblastic Leukemia;   Untreated Childhood Acute Lymphoblastic Leukemia
Interventions: Drug: Cyclophosphamide;   Drug: Cytarabine;   Drug: Dexamethasone;   Drug: Doxorubicin Hydrochloride;   Other: Laboratory Biomarker Analysis;   Drug: Leucovorin Calcium;   Drug: Mercaptopurine;   Drug: Methotrexate;   Drug: Pegaspargase;   Other: Quality-of-Life Assessment;   Other: Questionnaire Administration;   Drug: Thioguanine;   Drug: Vincristine Sulfate
Age: 1 Year to 30 Years   (Child, Adult)
Study Start: August 2010
Study Completion:  
Conditions: Adult B Acute Lymphoblastic Leukemia;   Adult T Acute Lymphoblastic Leukemia;   Childhood B Acute Lymphoblastic Leukemia;   Childhood T Acute Lymphoblastic Leukemia;   Untreated Adult Acute Lymphoblastic Leukemia;   Untreated Childhood Acute Lymphoblastic Leukemia
Interventions: Other: Cytology Specimen Collection Procedure;   Other: Laboratory Biomarker Analysis
Age: up to 30 Years   (Child, Adult)
Study Start: August 2010
Study Completion:  
Condition: Juvenile Idiopathic Arthritis
Interventions: Drug: CP-690,550 (tofacitinib);   Other: placebo
Age: 2 Years to 17 Years   (Child)
Study Start: June 10, 2016
Study Completion: April 29, 2019
Conditions: Leukemia Cutis;   Myeloid Sarcoma;   Untreated Adult Acute Myeloid Leukemia;   Untreated Childhood Myeloid Neoplasm
Interventions: Drug: Asparaginase;   Drug: Bortezomib;   Drug: Cytarabine;   Drug: Daunorubicin Hydrochloride;   Drug: Etoposide;   Other: Laboratory Biomarker Analysis;   Drug: Mitoxantrone Hydrochloride;   Other: Pharmacological Study;   Other: Quality-of-Life Assessment;   Other: Questionnaire Administration;   Drug: Sorafenib Tosylate
Age: up to 29 Years   (Child, Adult)
Study Start: June 2011
Study Completion:  
Conditions: Metastatic Ewing Sarcoma;   Metastatic Malignant Neoplasm in the Bone;   Metastatic Malignant Neoplasm in the Bone Marrow;   Metastatic Malignant Neoplasm in the Lung;   Metastatic Peripheral Primitive Neuroectodermal Tumor of Bone;   Peripheral Primitive Neuroectodermal Tumor of Soft Tissues
Interventions: Drug: Cyclophosphamide;   Drug: Doxorubicin Hydrochloride;   Drug: Etoposide;   Radiation: External Beam Radiation Therapy;   Biological: Ganitumab;   Drug: Ifosfamide;   Other: Laboratory Biomarker Analysis;   Other: Pharmacological Study;   Radiation: Stereotactic Radiosurgery;   Procedure: Therapeutic Surgical Procedure;   Drug: Vincristine Sulfate
Age: up to 50 Years   (Child, Adult)
Study Start: December 2014
Study Completion:  
Conditions: Adult T Acute Lymphoblastic Leukemia;   Childhood T Acute Lymphoblastic Leukemia;   Stage II Childhood Lymphoblastic Lymphoma;   Stage II Contiguous Adult Lymphoblastic Lymphoma;   Stage II Non-Contiguous Adult Lymphoblastic Lymphoma;   Stage III Adult Lymphoblastic Lymphoma;   Stage III Childhood Lymphoblastic Lymphoma;   Stage IV Adult Lymphoblastic Lymphoma;   Stage IV Childhood Lymphoblastic Lymphoma;   Untreated Adult Acute Lymphoblastic Leukemia;   Untreated Childhood Acute Lymphoblastic Leukemia
Interventions: Drug: Bortezomib;   Drug: Cyclophosphamide;   Drug: Cytarabine;   Drug: Daunorubicin Hydrochloride;   Drug: Dexamethasone;   Drug: Doxorubicin Hydrochloride;   Drug: Etoposide;   Drug: Hydrocortisone Sodium Succinate;   Drug: Ifosfamide;   Other: Laboratory Biomarker Analysis;   Drug: Leucovorin Calcium;   Drug: Mercaptopurine;   Drug: Methotrexate;   Drug: Pegaspargase;   Radiation: Radiation Therapy;   Drug: Thioguanine;   Drug: Vincristine Sulfate
Age: 2 Years to 30 Years   (Child, Adult)
Study Start: September 2014
Study Completion:
 
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