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Research

Clinical Trials

CHAM researchers are involved in literally hundreds of clinical trials and research studies to evaluate the effectiveness and safety of innovative treatments and diagnostics. Search and refine for clinical trials and research studies at CHAM by keyword or disease/condition.

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Epilepsy, Seizures, Neurology, Brain and Neurological

A 12-Month, Open-Label, Repeat-Dose Safety Study of NRL-1 in Epilepsy Subjects

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Principal Investigator: Alexis Boro, MD
Inflammatory Bowel Disease, Gastroenterology & Liver Diseases, Colitis and Crohn's Disease

A 5-year Longitudinal Observational Study of Patients Undergoing Therapy for Inflammatory Bowel Disease (TARGET-IBD)

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Principal Investigator: Gitit Tomer, MD
Liver Diseases, Gastroenterology & Liver Diseases

A 5-year Longitudinal Observational Study of Patients With Nonalcoholic Fatty Liver (NAFL) or Nonalcoholic Steatohepatitis (NASH)

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Principal Investigator: Bryan Rudolph, MD
Arthritis, Autoimmune Diseases, Musculoskeletal Diseases, Pediatrics, Rheumatic Diseases

A Long-Term, Open-Label Follow-up Study of Tofacitinib for Treatment of Juvenile Idiopathic Arthritis (JIA)

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Principal Investigator: Dawn Wahezi, MD
Anemia, Hematologic Diseases, Immune System Diseases, Leukemia, Lymphoma, Lymphoproliferative Disorders, Neoplasms, Non-Hodgkins Lymphoma, Oncology (Medical/Hematologic), Pediatric Hematology/Oncology, Blood Disorders and Diseases

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

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Principal Investigator: David Loeb, MD
Blood and Bone Marrow Cancer, Blood Disorders and Diseases

A Multicenter Safety Study of Unlicensed, Investigational Cryopreserved Cord Blood Units Manufactured by the National Cord Blood Program and Provided for Unrelated Hematopoietic Stem Cell Transplantation of Pediatric and Adult Patients

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Principal Investigator: David Loeb, MD
Heart and Vascular Diseases, Lung Diseases, Pediatrics, Pulmonary Hypertension

A Multicenter, Open-label, Randomized, Event-driven Study to Assess Efficacy, Safety and Pharmacokinetics of Macitentan Versus Standard of Care in Children With Pulmonary Arterial Hypertension (PAH) (TOMORROW)

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Principal Investigator: Nicole Sutton, MD
Autoimmune Diseases, Dermatology, Lupus, Pediatrics, Rheumatic Diseases, Geriatrics

A Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group Study of Ustekinumab in Subjects With Active Systemic Lupus Erythematosus

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Principal Investigator: Chaim Putterman, MD
Colitis and Crohn's Disease, Gastroenterology & Liver Diseases, Pediatrics, Inflammatory Bowel Disease

A Phase 2, Randomized, Double-Blind, Dose-Ranging Study to Determine the Pharmacokinetics, Safety and Tolerability of Vedolizumab IV in Pediatric Subjects With Ulcerative Colitis or Crohn's Disease

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Principal Investigator: John Thompson, MD
Geriatrics, Infectious Diseases, Influenza, Pediatrics

A Phase 3 Randomized, Double-blind, Placebo-controlled, Multicenter Study to Evaluate the Efficacy and Safety of Pimodivir in Combination With the Standard-of-Care Treatment in Adolescent, Adult, and Elderly Hospitalized Patients w/Influenza A Infection

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Principal Investigator: Paul Riska, MD
Dermatology, Pediatrics, Skin Disorders, Geriatrics, Immune System Diseases

A Phase 3 Randomized, Placebo-Controlled, Double-Blind Study to Evaluate Upadacitinib in Adolescent and Adult Subjects With Moderate to Severe Atopic Dermatitis

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Principal Investigator: Holly Kanavy, MD
Hematologic Diseases, Kidney Diseases (incl Chronic Kidney Disease), Metabolic Diseases, Nephrology, Urologic Diseases, Pediatrics

A Phase 3, Randomized, Open-Label, Multicenter Study to Evaluate the Safety (Compared to Iron Sucrose), Efficacy and Pharmacokinetics of Ferumoxytol for the Treatment of Iron Deficiency Anemia (IDA) in Pediatric Subjects With Chronic Kidney Disease (CKD)

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Principal Investigator: Anna Zolotnitskaya, MD
Autism Spectrum Disorder, Neurodevelopmental Disorders, Pediatrics, Psychiatry - Non Cancer, Mental Health and Disorders

A Phase II Multi-Center, Randomized, Double-Blind, 24-Week, Parallel Group, Placebo-Controlled Study to Investigate the Efficacy and Safety of Balovaptan (RO5285119) in Children and Adolescents Age 5-17 With Autism Spectrum Disorder (ASD)

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Principal Investigator: Eric Hollander, MD
Leukemia, Oncology (Medical/Hematologic), Pancreatic Cancer, Pediatric Hematology/Oncology, Neoplasms

A Phase III Study for Patients With Newly Diagnosed Acute Promyelocytic Leukemia (APL) Using Arsenic Trioxide and All-Trans Retinoic Acid

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Principal Investigator: Lisa Gennarini, MD
Gastroenterology & Liver Diseases, Pediatrics, Liver Diseases

A Prospective, Observational, Non-Interventional, Post-Marketing, Patient Registry to Collect Data on Routine Clinical Care in Patients Treated With Cholbam®/Kolbam® (Cholic Acid)

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Principal Investigator: Nadia Ovchinsky, MD
Colitis and Crohn's Disease, Gastroenterology & Liver Diseases, Inflammatory Bowel Disease, Pediatrics

A Randomized, Double-Blind, Placebo-Controlled, Multi-Center Pragmatic Clinical Trial To Evaluate The Effectiveness Of Low Dose Oral Methotrexate In Patients With Pediatric Crohn's Disease Initiating Anti-TNF Therapy (COMBINE)

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Principal Investigator: Gitit Tomer, MD
Anemia, Blood Disorders and Diseases, Sickle Cell, Pediatrics, Genetic Disorders, Hematologic Diseases

A Study to Compare Bone Marrow Transplantation to Standard Care in Adolescents and Young Adults With Severe Sickle Cell Disease (BMT CTN #1503)

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Principal Investigator: Caterina Minniti, MD
Cardiology, Oncology (Medical/Hematologic), Urogenital Neoplasms, Urologic Oncology (Med Onc), Prostate Cancer

A Trial Comparing Cardiovascular Safety of Degarelix Versus Leuprolide in Patients With Advanced Prostate Cancer and Cardiovascular Disease

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Principal Investigator: Benjamin Gartrell, MD
Breast Cancer, Neoplasms, Oncology (Medical/Hematologic), Women`s Health

A Trial of Endocrine Response in Women With Invasive Lobular Breast Cancer

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Principal Investigator: Joseph Sparano, MD
Genetic Disorders, Ophthalmic Diseases and Disorders, Pediatrics

An Observational, Multicenter Study of the Prevalence of Cerebrotendinous Xanthomatosis (CTX) in Patient Populations Diagnosed With Early-Onset Idiopathic Bilateral Cataracts

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Principal Investigator: Norman Medow, MD
Heart and Vascular Diseases, Pediatrics, Rheumatic Diseases, Autoimmune Diseases

ARCHIVE: A registry for children with vasculitis: e-entry

Principal Investigator: Maria Yee, MD
Anesthesia and Pain Management, Pediatrics, Rehabilitation Medicine

Assessing the Efficacy of Virtual Reality Analgesia (VRA) in Pediatric Patients for Pain Control During Botox Injections for Spasticity Management: A Randomized Control Trial

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Principal Investigator: Yuxi Chen, MD
Autism Spectrum Disorder, Neurology, Pediatrics, Genetic Disorders

Autism Genetics Network, Phase II: Increasing Representation of Human Diversity

Principal Investigator: Sophie Molholm, PhD
Immune System Diseases, Infectious Diseases, Pediatrics

Bezlotoxumab (MK-6072) Versus Placebo in Children With Clostridium Difficile Infection (CDI) (MK-6072-001) (MODIFY III)

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Principal Investigator: David Goldman, MD
Lymphoproliferative Disorders, Neoplasms, Pediatric Hematology/Oncology, Blood and Bone Marrow Cancer, Immune System Diseases, Lymphatic and Spleen Diseases, Lymphoma

Brentuximab Vedotin and Combination Chemotherapy in Treating Younger Patients With Newly Diagnosed Hodgkin Lymphoma

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Principal Investigator: Lisa Gennarini, MD
Child Development, Neurodevelopmental Disorders, Autism Spectrum Disorder

Cannabidivarin (CBDV) vs. Placebo in Children With Autism Spectrum Disorder (ASD)

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Principal Investigator: Eric Hollander, MD
Allergy & Immunology, Allergy and Immune Disorders, Asthma and other Respiratory Diseases, Pediatrics

Childhood Asthma Perception Study (CAPS)

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Principal Investigator: Jonathan Feldman, PhD
Medical and Hematologic Oncology, Neuroblastoma, Neoplasms

COG-ANBL00B1 Neuroblastoma Biology Studies

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Principal Investigator: Lisa Gennarini, MD
Blood and Bone Marrow Cancer, Immune System Diseases, Leukemia, Lymphatic and Spleen Diseases, Lymphoma, Lymphoproliferative Disorders, Neoplasms, Pediatric Hematology/Oncology

Combination Chemotherapy in Treating Young Patients With Newly Diagnosed High-Risk B Acute Lymphoblastic Leukemia and Ph-Like TKI Sensitive Mutations

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Principal Investigator: Lisa Gennarini, MD
Bones, Muscles & Joints (Orthopaedics)

Consideration of Pedicle Screw Misplacement on a Per-Patient Basis: Can we Better Delineate Surgical Risk?

Principal Investigator: Vishal Sarwahi, MD
Gastroenterology & Liver Diseases, Inflammatory Bowel Disease, Pediatrics

Defensin and Innate Mucosal Immune Mediator Expression in Pediatric Inflammatory Bowel Disease

Principal Investigator: Gitit Tomer, MD
Diabetes and Endocrine, Metabolic Diseases, Pediatric Endocrinology

Diabetes Study of Linagliptin and Empagliflozin in Children and Adolescents (DINAMO)TM

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Principal Investigator: Lisa Underland, MD
Bones, Muscles & Joints (Orthopaedics)

Does Pedicle Morphology Change with Age?

Principal Investigator: Vishal Sarwahi, MD
Kidney Diseases (incl Chronic Kidney Disease), Nephrology, Pediatrics, Urologic Diseases

Efficacy of a Gluten-free Diet in Difficult to Manage Nephrotic Syndrome: Utility of Plasma Zonulin Levels as a Predictive Biomarker

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Principal Investigator: Frederick Kaskel, MD
Autism Spectrum Disorder

Follow-up study of children with early diagnosis of autism spectrum disorder

Principal Investigator: Lisa Shulman, MD

Gene Discoveries in Subjects with Crohns Disease of African Descent

Principal Investigator: Gitit Tomer, MD
Anemia, Hematologic Diseases, Pediatric Hematology/Oncology, Pediatrics, Genetic Disorders

Hydroxyurea Adherence for Personal Best in Sickle Cell Disease (HABIT): Efficacy Trial

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Principal Investigator: Deepa Manwani, MD
Graft Versus Host Disease (GVHD)

Ibrutinib in Combination With Corticosteroids Versus Placebo in Combination With Corticosteroids in Subjects With New Onset Chronic Graft Versus Host Disease (cGVHD) (INTEGRATE)

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Principal Investigator: Olga Derman, MD
Brain and Neurological

Infantile Spasms Multicenter Database

Principal Investigator: Elissa Yozawitz, MD
Blood Disorders and Diseases

International Registry for Severe Chronic Neutropenia

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Principal Investigator: Kerry Morrone, MD
Blood Disorders and Diseases, Anesthesia and Pain Management

Intravenous Gammaglobulin for Sickle Cell Pain Crises

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Principal Investigator: Deepa Manwani, MD
Brain and Neurological

Maintenance Chemotherapy or Observation Following Induction Chemotherapy and Radiation Therapy in Treating Younger Patients With Newly Diagnosed Ependymoma

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Principal Investigator: Lisa Gennarini, MD
Pediatric Hematology/Oncology, Leukemia, Neoplasms, Blood Disorders and Diseases, Cancer

Matched Targeted Therapy (MTT) Recommendation for Patients with Recurrent, Refractory, or High Risk Leukemias

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Principal Investigator: Lisa Gennarini, MD
Solid Tumor

Metformin in Children With Relapsed or Refractory Solid Tumors

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Principal Investigator: Daniel Weiser, MD
Neonatology, Pediatrics, Overweight, Obesity and Weight Loss

Molecular Basis of Early Childhood Obesity Programming by Intrauterine Growth Restriction

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Principal Investigator: Mamta Fuloria, MD
Rheumatic Diseases

Mood disorder and cognitive dysfunction in pediatric lupus

Principal Investigator: Chaim Putterman, MD
Cancer, Hematologic Diseases, Pediatric Hematology/Oncology, Solid Tumor

Multicenter Cohort Study To Evaluate Outcomes After Receipt of Targeted Therapy Matched to an Individualized Cancer Therapy (iCat) Recommendations in Children and Young Adults With Solid Tumors: The iCat2, GAIN Consortium Study

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Principal Investigator: Daniel Weiser, MD
Bones, Muscles & Joints (Orthopaedics)

Multicenter Prospective Cohort Study on Current Treatments of Legg-Calvé-Perthes Disease (IPSG1)

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Principal Investigator: Eric Fornari, MD
Epstein-Barr Virus (EBV), Lymphoproliferative Disorders, Pediatric Hematology/Oncology, Pediatrics, Solid Organ Transplant (SOT) Complications, Immune System Diseases

Multicenter, Open Label, Phase 3 Study of Tabelecleucel for Solid Organ Transplant Subjects With Epstein-Barr Virus-Associated Post-Transplant Lymphoproliferative Disease After Failure of Rituximab or Rituximab and Chemotherapy (ALLELE)

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Principal Investigator: David Loeb, MD
Epstein-Barr Virus (EBV), Immune System Diseases, Lymphatic and Spleen Diseases, Lymphoma, Lymphoproliferative Disorders, Pediatric Hematology/Oncology, Cancer

Multicenter, Open-Label, Phase 3 Study of Tabelecleucel for Allogeneic Hematopoietic Cell Transplant Subjects With Epstein-Barr Virus-Associated Post-Transplant Lymphoproliferative Disease After Failure of Rituximab (MATCH Study)

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Principal Investigator: David Loeb, MD
Diabetes and Endocrine, Endocrinology, Metabolic Diseases, Autoimmune Diseases

Natural History Study of the Development of Type 1 Diabetes

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Principal Investigator: Lisa Underland, MD
Geriatrics, Kidney Diseases (incl Chronic Kidney Disease), Nephrotic Syndrome, Pediatrics, Urologic Diseases, Immune System Diseases

Nephrotic Syndrome Study Network Under the Rare Diseases Clinical Research Network (NEPTUNE)

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Principal Investigator: Frederick Kaskel, MD
Supportive Cancer Care

Neuropsychological and Behavioral Testing in Younger Patients With Cancer

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Principal Investigator: Lisa Gennarini, MD
Allergy and Immune Disorders

Non-Interventional Post-Marketing Safety Study on the Long-Term Safety of HYQVIA (Global)

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Principal Investigator: Arye Rubinstein, MD
Kidney Diseases (incl Chronic Kidney Disease)

North American Pediatric Renal Trials and Collaborative Studies

Principal Investigator: Marcela Del Rio, MD
Rheumatic Diseases

Observational Study of Pediatric Rheumatic Diseases: The CARRA Registry

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Principal Investigator: Yonit Sterba Ruas, MD
Immune System Diseases, Lymphatic and Spleen Diseases, Lymphoma, Lymphoproliferative Disorders, Neoplasms, Pediatric Hematology/Oncology, Solid Tumor

Open-label, Phase I/II Study to Evaluate Pharmacokinetics, Pharmacodynamics, Safety, and Anticancer Activity of Avelumab in Pediatric Subjects From Birth to Less Than 18 Years of Age With Refractory or Relapsed Solid Tumors and Lymphoma

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Principal Investigator: David Loeb, MD
Kidney Diseases (incl Chronic Kidney Disease)

Outcomes of Neonatal Acute Kidney Injury In Premature Infants

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Principal Investigator: Mamta Fuloria, MD
Heart and Vascular Diseases

Ped Cardiac Quality of LIfe

Principal Investigator: Jacqueline Lamour, MD
Heart and Vascular Diseases

Pediatric Heart Transplant Study Group

Principal Investigator: Jacqueline Lamour, MD
Lung Diseases, Pediatrics, Asthma and other Respiratory Diseases

Peer Mentor Training to Reduce Hospitalizations for Children With Asthma

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Principal Investigator: Deepa Rastogi, MD
Supportive Cancer Care

Personalized Medicine: Pediatric Total Cancer Care™ Protocol

Principal Investigator: Daniel Weiser, MD
Kidney Diseases (incl Chronic Kidney Disease)

Pharmacodynamic & Safety of Patiromer in Children & Adolescents (2-<18 Yrs) With Chronic Kidney Disease and Hyperkalemia (EMERALD)

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Principal Investigator: Anna Zolotnitskaya, MD
Psychiatry - Non Cancer, Nervous System Diseases, Child Development, Pediatrics, Genetic Disorders

Phase 2 Study: Intranasal Oxytocin vs. Placebo for the Treatment of Hyperphagia in Children and Adolescents With Prader-Willi Syndrome

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Principal Investigator: Eric Hollander, MD
Heart and Vascular Diseases, Kidney Diseases (incl Chronic Kidney Disease), Pediatrics, Urologic Diseases, Cardiovascular and Thoracic Surgery

Phase III Multicenter Open-label Randomized Clinical Trial Comparing Everolimus and Low Dose Tacrolimus to Tacrolimus and Mycophenolate Mofetil at 6 mo Post-Transplant to Prevent Long-term Complications After Pediatric Heart Transplantation

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Principal Investigator: Jacqueline Lamour, MD
Cancer and Genetics

Project: Every Child for Younger Patients With Cancer

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Principal Investigator: Lisa Gennarini, MD
Asthma and other Respiratory Diseases, Pediatrics, Lung Diseases

Promoting Asthma Guidelines and Management through Technology-Based Intervention and Care Coordination (PRAGMATIC)

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Principal Investigator: Marina Reznik, MD
Bone Cancer, Cancer, Infectious Diseases, Musculoskeletal Diseases, Neoplasms, Surgery

Prophylactic Antibiotic Regimens in Tumor Surgery (PARITY): A Multi-Center International Randomized Controlled Trial Comparing Alternative Antibiotic Regimens in Patients Undergoing Tumor Resections With Endoprosthetic Replacements

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Principal Investigator: David Geller, MD
Neoplasms, Oncology (Medical/Hematologic), Pediatric Hematology/Oncology, Blood and Bone Marrow Cancer, Blood Disorders and Diseases, Hematologic Diseases

Protocol For A Research Database For Hematopoietic Stem Cell Transplantation, Other Cellular Therapies and Marrow Toxic Injuries

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Principal Investigator: Ira Braunschweig, MD
Hematologic Diseases, Neoplasms, Pediatric Hematology/Oncology, Pediatrics, Oncology (Medical/Hematologic)

Protocol for a Research Sample Repository and for a Research Database for Hematopoietic Cell Transplantation, Other Cellular Therapies, and Marrow Toxic Injuries

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Principal Investigator: David Loeb, MD
Autoimmune Diseases, Blood and Bone Marrow Cancer, Blood Disorders and Diseases

Protocol for a Research Sample Repository for Allogeneic Hematopoietic Stem Cell Transplantation

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Principal Investigator: Ira Braunschweig, MD
Infant Care

Quarenta Semanas: Innovative Intervention in Prenatal Care for Reduction of Prematurity (quarenta)

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Principal Investigator: Judy Aschner, MD
Soft Tissue Sarcoma/Cancer

Radiation Therapy With or Without Combination Chemotherapy or Pazopanib Hydrochloride Before Surgery in Treating Patients With Newly Diagnosed Non-Rhabdomyosarcoma Soft Tissue Sarcomas That Can be Removed by Surgery (PAZNTIS)

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Principal Investigator: Lisa Gennarini, MD
Neuroblastoma

Response and Biology-Based Risk Factor-Guided Therapy in Treating Younger Patients With Non-high Risk Neuroblastoma

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Principal Investigator: Peter Cole, MD
Blood and Bone Marrow Cancer

Risk-Based Classification System of Patients With Newly Diagnosed Acute Lymphoblastic Leukemia

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Principal Investigator: Peter Cole, MD
Epstein-Barr Virus (EBV), Leukemia, Lymphoma, Lymphoproliferative Disorders, Oncology (Medical/Hematologic), Neoplasms, Non-Hodgkins Lymphoma

Risk-Stratified Randomized Phase III Testing of Blinatumomab (NSC#765986) in First Relapse of Childhood B-Lymphoblastic Leukemia (B-ALL)

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Principal Investigator: Lisa Gennarini, MD
Blood and Bone Marrow Cancer

Safety Study of Gene Modified Donor T-cells Following TCR Alpha Beta Depleted Stem Cell Transplant

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Principal Investigator: Kris Mahadeo, MD
Child Development

Self regulation in children with learning disabilities and inattention/hyperactivity

Principal Investigator: Katherine Lawson, PhD
Autism Spectrum Disorder

Sensory Integration Therapy in Autism: Mechanisms and Effectiveness

Principal Investigator: Sophie Molholm, PhD
Bones, Muscles & Joints (Orthopaedics), Orthopaedic Surgery, Pediatrics, Neoplasms, Musculoskeletal Diseases

Simple Bone Cysts in Kids (SBoCK)

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Principal Investigator: Jacob Schulz, MD
Hematologic Diseases, Pediatrics

Study of Allogeneic EBV-CTLs in EBV-Associated Viremia or Malignancies

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Principal Investigator: David Loeb, MD
Kidney Cancer

Study of Kidney Tumors in Young Patients

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Principal Investigator: Lisa Gennarini, MD
Heart and Vascular Diseases

Study of the Safety and Effectiveness of SAMSCA® (Tolvaptan) in Children and Adolescents With Euvolemic or Hypervolemic Hyponatremia

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Principal Investigator: Scott Aydin,MD
Bones, Muscles & Joints (Orthopaedics)

The Children’s Spine Registry

Principal Investigator: Jaime Gomez, MD
Blood and Bone Marrow Cancer

The Chimerix CMX001 Registry

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Principal Investigator: Olga Derman, MD
Metabolic Diseases, Pediatric Endocrinology, Overweight, Obesity and Weight Loss

The Influence of Breakfast on Hormone Responses and Cognitive Performance, as Assessed by CTET in Lean and Obese Adolescent Males

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Principal Investigator: Lisa Underland, MD
Asthma and other Respiratory Diseases

The National Emergency Airway Registry for Children

Principal Investigator: Michael Miksa, MD
Bones, Muscles & Joints (Orthopaedics)

The Reliability of X-Ray Based Evaluation of Pedicle Screw Misplacement in Adolescent Spinal Deformity

Principal Investigator: Vishal Sarwahi, MD
Autism Spectrum Disorder

The use of Complementary and Alternative Medicine in Autism

Principal Investigator: Maria Valicenti-McDermott, MD
Brain and Neurological

The use of Virtual Reality Games to Enhance Therapy for Children with Cerebral Palsy. A pilot Study

Principal Investigator: Maria Valicenti-McDermott, MD
Leukemia, Lymphatic and Spleen Diseases, Neoplasms, Pediatric Hematology/Oncology, Lymphoproliferative Disorders

Treatment of Newly Diagnosed Acute Lymphoblastic Leukemia in Children and Adolescents

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Principal Investigator: Lisa Gennarini, MD
Heart and Vascular Diseases

US-based, Observational, Drug Registry of Opsumit® (Macitentan) New Users in Clinical Practice

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Principal Investigator: Sandhya Murthy, MD
Brain and Neurological, Crigler-Najjar Syndrome, Gastroenterology & Liver Diseases, Heart and Vascular Diseases, Metabolic Diseases, Pediatrics

VALENS: A Ph 1/2, Random'd, Open-Label, Ascending-Dose, Delayed-Treatment Concurrent Control Clinical Study to Evaluate Safety/Preliminary Efficacy of AT342, an AAV8-Delivered Gene Transfer Therapy in Crigler-Najjar Syndrome Subjects Aged 1 Yr and Older

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Principal Investigator: Nadia Ovchinsky, MD
Asthma and other Respiratory Diseases, Pediatrics, Lung Diseases

Web-based Peer Mentor Training to Reduce Hospitalizations for Children with Asthma

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Principal Investigator: Deepa Rastogi, MD