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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

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

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
Oncology (Medical/Hematologic), Pediatric Hematology/Oncology, Pediatrics

A Childrens Oncology Group Protocol for Collecting and Banking Ewing Sarcoma Specimens

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Principal Investigator: Lisa Gennarini, MD
Genetic Disorders, Genetics, Heart and Vascular Diseases, Liver Diseases, Pediatrics, Metabolic Diseases

A Clinical Assessment Study in Crigler-Najjar Syndrome (LUSTRO)

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Principal Investigator: Nadia Ovchinsky, MD
Autism Spectrum Disorder

A Double Blind, Randomized, Placebo-Controlled Study of CM-AT for the Treatment of Autism in Children With All Levels of Fecal Chymotrypsin (FCT)

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Principal Investigator: Eric Hollander, MD
Autism Spectrum Disorder, Neurodevelopmental Disorders

A Long Term Extension Study of CM-AT for the Treatment of Children with Autism

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Principal Investigator: Eric Hollander, 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
Blood and Bone Marrow Cancer

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: Kris Mahadeo, MD
Blood and Bone Marrow Cancer

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: Kris Mahadeo, MD
Diabetes and Endocrine, Kidney Diseases (Chronic Kidney Disease)

A Multicenter Single-arm Extension Study to Characterize the Long-term Safety of Cinacalcet Hydrochloride in the Treatment of Secondary Hyperparathyroidism in Pediatric Subjects With Chronic Kidney Disease on Dialysis

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Principal Investigator: Anna Zolotnitskaya, MD

A Multicenter Study to Obtain Retrospective Data for Subjects Previously Diagnosed With Adenovirus Infection to Serve as Matched Historical Controls for Study CMX001-304

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Principal Investigator: Kris Mahadeo, 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

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Principal Investigator: Nicole Sutton, MD
Neurodevelopmental Disorders, Pediatrics, Psychiatry - Non Cancer, Tourette Syndrome

A Phase 2b, Randomized, Double-Blind, Placebo-Controlled, Dose Optimization Study to Assess the Safety, Tolerability, and Efficacy of NBI-98854 for the Treatment of Pediatric Subjects With Tourette Syndrome

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Principal Investigator: Eric Hollander, MD
Hematologic Diseases, Pediatrics, Sickle Cell

A Phase 3, Double-blind, Randomized, Placebo-controlled, Multicenter Study of GBT440 Administered Orally to Patients With Sickle Cell Disease

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Principal Investigator: Caterina Minniti, MD
Pediatrics, Child Development, Endocrinology

A PHASE 3, OPEN-LABEL, RANDOMIZED, MULTICENTER, 12 MONTHS, EFFICACY AND SAFETY STUDY OF WEEKLY MOD-4023 COMPARED TO DAILY GENOTROPIN- THERAPY IN PRE-PUBERTAL CHILDREN WITH GROWTH HORMONE DEFICIENCY

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Principal Investigator: Ping Zhou, MD
Autism Spectrum Disorder, Neurodevelopmental Disorders, Pediatrics, Psychiatry - Non Cancer

A Phase II Multi-Center, Randomized, Double-Blind, 24-Week, 3-Arm, Parallel-Group, Placebo-Controlled Study to Investigate the Efficacy and Safety of RO5285119 in Children and Adolescents Age 5-17 with Autism Spectrum Disorder (ASD)

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Principal Investigator: Eric Hollander, MD
Women`s Health

A Phase III Efficacy and Safety Study of Intravenous Retosiban Versus Placebo for Women in Spontaneous Preterm Labor (NEWBORN-1)

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Principal Investigator: Ashlesha Dayal, MD

A Prospective, Multi-center Registry for Patients With Short Bowel Syndrome

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Principal Investigator: John Thompson, MD
Gastroenterology & Liver Diseases, Pediatrics

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
Infectious Diseases, Pediatrics

A Rando'd, Double-Blind, Placebo-Cont'd Trial to Evaluate Safety, Tolerability, PK and Efficacy of Single Infusion of Bezlotoxumab (MK-6072, Human Monoclonal Antibody to C.Diff Toxin B) in Children 1 to <18 Yrs Receiving Antibacterial Drug Tx for C.Diff

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Principal Investigator: David Goldman, 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
Asthma and other Respiratory Diseases

A Safety Extension Study to Evaluate the Safety and Tolerability of Benralizumab (MEDI-563) in Asthmatic Adults and Adolescents on Inhaled Corticosteroid Plus LABA (BORA)

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Principal Investigator: Golda Hudes, MD
Blood and Bone Marrow Cancer, Sickle Cell, Anemia, 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
Child Development

Active commuting to school and behavior problems among school-aged children

Principal Investigator: Ruth Stein, MD
Kidney Diseases (Chronic Kidney Disease), Asthma and other Respiratory Diseases

Acute Kidney Injury in Children with Acute Respiratory Failure

Principal Investigator: Scott Aydin, MD
Infant Care

An Observational Study of Respiratory Syncytial Virus (RSV) Hospitalizations in Preterm Infants (SENTINEL1)

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Principal Investigator: Iona Munjal, MD
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
Autism Spectrum Disorder, Psychiatry - Non Cancer, Pediatrics

An Open Label Study of CM-AT for the Treatment of Children With Autism

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Principal Investigator: Eric Hollander, MD

Analysis of Clinical and Laboratory Measures in Children with Intestinal Failure at the Children's Hospital at Montefiore

Principal Investigator: John Thompson, MD
Rheumatic Diseases

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

Principal Investigator: Dawn Wahezi, MD
Autism Spectrum Disorder

Are children with Autistic Spectrum Disorder diagnosed before 2 years of age different clinically from older children diagnosed after 2 years of age?

Principal Investigator: Maria Valicenti-McDermott, MD
Blood and Bone Marrow Cancer

Assessing Compliance With Mercaptopurine Treatment in Younger Patients With Acute Lymphoblastic Leukemia in First Remission

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Principal Investigator: Peter Cole, MD
Kidney Diseases (Chronic Kidney Disease)

Atypcial Hemolytic-Uremic Syndrome (aHUS) Registry

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Principal Investigator: Frederick Kaskel, MD
Autism Spectrum Disorder

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

Principal Investigator: Sophie Molholm, PhD
Neuroblastoma

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

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Principal Investigator: Jonathan Gill, MD
Neonatology, Preterm Birth, Neurodevelopmental Disorders

Biomarkers to Predict Neurodevelopmental Outcomes in Very Preterm Infants

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Principal Investigator: Mamta Fuloria, MD
Blood and Bone Marrow Cancer

Blinatumomab in Treating Younger Patients With Relapsed B-cell Acute Lymphoblastic Leukemia

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Principal Investigator: Jonathan Gill, MD
Lymphoma

Blood Sample Markers of Reproductive Hormones in Assessing Ovarian Reserve in Younger Patients With Newly Diagnosed Lymphomas

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

Bortezomib and Sorafenib Tosylate in Treating Patients With Newly Diagnosed Acute Myeloid Leukemia

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

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

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Principal Investigator: Jonathan Gill, MD
Blood and Bone Marrow Cancer

Brentuximab Vedotin and Gemcitabine Hydrochloride in Treating Younger Patients With Relapsed or Refractory Hodgkin Lymphoma

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

Brentuximab Vedotin or Crizotinib and Combination Chemotherapy in Treating Patients With Newly Diagnosed Stage II-IV Anaplastic Large Cell Lymphoma

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

Caspofungin Acetate or Fluconazole in Preventing Invasive Fungal Infections in Patients With Acute Myeloid Leukemia Who Are Undergoing Chemotherapy

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

Caspofungin Acetate, Fluconazole, or Voriconazole in Preventing Fungal Infections in Patients Following Donor Stem Cell Transplant

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Principal Investigator: Jonathan Gill, MD

Cerebrotendinous Xanthomatosis (CTX) Prevalence Study

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Principal Investigator: Norman Medow, MD
Brain Cancer

Chemotherapy and Radiation Therapy in Treating Young Patients With Newly Diagnosed, Previously Untreated, High-Risk Medulloblastoma

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Principal Investigator: Jonathan Gill, MD
Brain Cancer

Chemotherapy Followed by Radiation Therapy in Treating Younger Patients With Newly Diagnosed Localized Central Nervous System Germ Cell Tumors

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Principal Investigator: Jonathan Gill, 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
Brain and Neurological

Clinical characterization of the pre-ictal state in the pediatric population: A caretaker's perspective on seizure prediction

Principal Investigator: Sheryl Haut, MD
Leukemia, Non-Hodgkins Lymphoma

COG-AALL05B1 A Childrens Oncology Group Protocol for Collecting and Banking Relapsed Acute Lymphoblastic Leukemia Research Specimens

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Principal Investigator: Lisa Gennarini, MD
Bone Cancer

Collecting and Storing Biological Samples From Patients With Ewing Sarcoma

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Principal Investigator: Peter Cole, MD
Bone Cancer

Collecting and Storing Samples of Blood and Tumor Tissue From Patients With Osteosarcoma

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Principal Investigator: Jonathan Gill, MD
Blood and Bone Marrow Cancer

Collecting and Storing Samples of Bone Marrow and Blood From Patients With Relapsed Acute Lymphoblastic Leukemia or Non-Hodgkin Lymphoma

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Principal Investigator: Peter Cole, MD
Cancer and Genetics

Collecting and Storing Tissue From Young Patients With Cancer

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

Collecting and Storing Tissue Samples From Patients With Rare or Cutaneous Non-Hodgkin Lymphoma

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Principal Investigator: Peter Cole, MD
Soft Tissue Sarcoma/Cancer

Collecting and Storing Tissue, Blood, and Bone Marrow Samples From Patients With Rhabdomyosarcoma or Other Soft Tissue Sarcoma

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Principal Investigator: Jonathan Gill, MD
Blood and Bone Marrow Cancer

Combination Chemotherapy in Treating Young Patients With Newly Diagnosed High-Risk Acute Lymphoblastic Leukemia

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

Combination Chemotherapy With or Without Bortezomib in Treating Younger Patients With Newly Diagnosed T-Cell Acute Lymphoblastic Leukemia or Stage II-IV T-Cell Lymphoblastic Lymphoma

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Principal Investigator: Jonathan Gill, MD
Bones, Muscles & Joints (Orthopaedics)

Compared Outcomes of Closed Reduction in the Emergency Room Versus the Operating Room in the Treatment of Pediatric Supracondylar Humerus Fractures

Principal Investigator: Jacob Schulz, MD
Rheumatic Diseases

Comparison of menstrual patterns and ovarian reserve in adolescent girls with systemic lupus erythematosus, juvenile idiopathic arthritis, and girls without chronic illness

Principal Investigator: Dawn Wahezi, 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
Kidney Diseases (Chronic Kidney Disease)

CureGN: Cure Glomerulonephropathy Network

Principal Investigator: Frederick Kaskel, MD

Defensin and Innate Immunity

Principal Investigator: Gitit Tomer, MD
Bone Cancer

Denosumab in Treating Patients With Recurrent or Refractory Osteosarcoma

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Principal Investigator: Jonathan Gill, MD
Pediatrics

Developmental Impact of NICU Exposures (DINE)

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Principal Investigator: Judy Aschner, MD
Bone Cancer

Dinutuximab in Combination With Sargramostim in Treating Patients With Recurrent Osteosarcoma

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Principal Investigator: Jonathan Gill, MD
Bones, Muscles & Joints (Orthopaedics)

Does Pedicle Morphology Change with Age?

Principal Investigator: Vishal Sarwahi, MD
Infant Care

Effect of Early L-Carnitine Supplementation on Neurodevelopmental Outcomes in Very Preterm Infants

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Principal Investigator: Mamta Fuloria, MD
Rehabilitation Medicine, Pediatrics

Effectiveness and Safety of Dysport® (abobotulinumtoxinA) in Paediatric Lower Limb Spasticity

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Principal Investigator: Yuxi Chen, MD
Rheumatic Diseases

Efficacy, Safety And Tolerability Of Tofacitinib For Treatment Of Polyarticular Course Juvenile Idiopathic Arthritis (JIA) In Children And Adolescent Subjects

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Principal Investigator: Norman Ilowite, MD
Blood and Bone Marrow Cancer

Enrollment on the Childhood Cancer Research Network (CCRN) of the Children s Oncology Group

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Principal Investigator: Jonathan Gill, MD
Ears, Nose and Throat

Evaluation of CYP2D6 genetic polymorphisms in a diverse urban population

Principal Investigator: TJ Ow, MD

Evaluation of the Rate of Discordance between Thiopurine Methyltransferase Phenotype and Genotype in Pediatric Patients with Gastrointestinal Disease

Principal Investigator: Inna Novak, MD
Child Development

Exploring Brain Responses to Affective Images in Mothers with High or Low Adverse Childhood Experiences (Aces)

Principal Investigator: Anne Murphy, PhD
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
Bone Cancer

Glembatumumab Vedotin in Treating Patients With Recurrent or Refractory Osteosarcoma

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Principal Investigator: Jonathan Gill, MD
Pediatric Critical Care

Heart And Lung Failure - Pediatric INsulin Titration Trial (HALF-PINT)

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Principal Investigator: Shivanand Medar, 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
Skin Disorders

Long Term Safety and Efficacy Study of Calcitriol 3 mcg/g Ointment in Pediatric Subjects With Plaque Psoriasis

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Principal Investigator: Holly Kanavy, DO
Bones, Muscles & Joints (Orthopaedics)

Long-Term Clinical and Radiologic Outcomes of Patients Treated At a Single Institution For Slipped Capital Femoral Epiphysis

Principal Investigator: Jacob Schulz, MD
Supportive Cancer Care

Long-Term Follow-Up of Patients Who Have Participated in Children's Oncology Group Studies

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Principal Investigator: Jonathan Gill, 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: Jonathan Gill, 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
Leukemia, Pediatrics

Matched Targeted Therapy For High-Risk Leukemias

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Principal Investigator: Peter Cole, MD
Solid Tumor

Metformin in Children With Relapsed or Refractory Solid Tumors

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Principal Investigator: Jonathan Gill, MD
Women`s Health

Mifepristone Induction for Fetal Demise (MIFD)

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Principal Investigator: Jessica Atrio, 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
Diabetes and Endocrine

Natural History Study of the Development of Type 1 Diabetes

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Principal Investigator: Rubina Heptulla, MD
Kidney Diseases (Chronic Kidney Disease)

Nephrotic Syndrome Study 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: Jonathan Gill, 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 (Chronic Kidney Disease)

North American Pediatric Renal Trials and Collaborative Studies

Principal Investigator: Marcela Del Rio, MD
Cardiology, Cardiovascular and Thoracic Surgery, Heart and Vascular Diseases, Pediatrics

Novel Mobile Device Application to Improve Adherence

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Principal Investigator: Jacqueline Lamour, MD
Rheumatic Diseases

Observational Study of Pediatric Rheumatic Diseases: The CARRA Registry

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Principal Investigator: Yonit Sterba Ruas, MD
Heart and Vascular Diseases

Outcomes of coronary artery aneurysms after Kawasaki disease:An international multicentre registry

Principal Investigator: Nadine Choueiter, MD
Kidney Diseases (Chronic Kidney Disease)

Outcomes of Neonatal Acute Kidney Injury In Premature Infants

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Principal Investigator: Mamta Fuloria, MD
Bones, Muscles & Joints (Orthopaedics)

Parental Understanding of an In-Toeing Gait

Principal Investigator: Jacob Schulz, MD
Heart and Vascular Diseases

Ped Cardiac Quality of LIfe

Principal Investigator: Jacqueline Lamour, MD
Ears, Nose and Throat

Pediatric Airway Database: An Observational Trial

Principal Investigator: John Bent, MD
Heart and Vascular Diseases

Pediatric Appropriate Use Criteria Implementation Project: An Outpatient Echocardiography Quality Initiative

Principal Investigator: Kenan Stern, 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: Jonathan Gill, MD
Kidney Diseases (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
Infant Care

Prematurity and Respiratory Outcomes Program (PROP)

Principal Investigator: Judy Aschner, MD
Cancer and Genetics

Profiling Actionable Genetic Alterations in Pediatric Tumors: The Children's Hospital at Montefiore Protocol for Screening Pediatric Tumors to Define Genetic Alterations

Principal Investigator: Adam Levy, MD
Cancer and Genetics

Project: Every Child for Younger Patients With Cancer

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Principal Investigator: Jonathan Gill, 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
Embolism or Thrombosis, Heart and Vascular Diseases, Hematologic Diseases, Pediatric Hematology/Oncology

Prospective Evaluation of the Duration of Therapy for Thrombosis In Children

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Principal Investigator: Jennifer Davila, MD
Bones, Muscles & Joints (Orthopaedics)

Prospective Scoliosis Outcomes Database

Principal Investigator: Jacob Schulz, MD
Hematologic Diseases, Pediatrics, Neoplasms

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
Blood and Bone Marrow Cancer

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

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: Jonathan Gill, MD
Heart and Vascular Diseases

Renal Biomarkers in Pediatric Acute Decompensated Heart Failure

Principal Investigator: Scott Aydin, 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
Anesthesia and Pain Management

Retrospective Evaluation of Sedation Efficacy for Magnetic Resonance Imaging in Pediatric Patients: Dexmedetomidine versus Propofol.

Principal Investigator: Chhavi Katyal, MD
Blood and Bone Marrow Cancer

Risk-Adapted Chemotherapy in Younger Patients With Newly Diagnosed Standard-Risk Acute Lymphoblastic Leukemia

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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
Liver Cancer

Risk-Based Therapy in Treating Younger Patients With Newly Diagnosed Liver Cancer

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Principal Investigator: Jonathan Gill, MD
Blood and Bone Marrow Cancer

Safety Follow-up of Treatment With Remestemcel-L in Pediatric Patients Who Have Failed to Respond to Steroid Treatment for Acute GVHD

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Principal Investigator: Kris Mahadeo, 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

Short Bowel Syndrome Research Study for Children Up To 17 Years of Age on Parenteral Nutrition

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Principal Investigator: John Thompson, MD
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
Sleep-Wake Disorders

Sodium Oxybate for Treatment of Excessive Daytime Sleepiness and Cataplexy in Narcolepsy

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Principal Investigator: Michael Thorpy, 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: Jonathan Gill, MD
Heart and Vascular Diseases

Study of Tadalafil in Pediatric Participants With Pulmonary Arterial Hypertension (PAH)

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Principal Investigator: Nicole Sutton, 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
Brain and Neurological

Study to Evaluate the Long-term Safety and Tolerability of USL261 in Patients With Seizure Clusters

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Principal Investigator: Alexis Boro, MD
Brain and Neurological

Study to Evaluate the Safety and Efficacy of USL261 (Intranasal Midazolam) in Patients With Seizure Clusters (ARTEMIS1)

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Principal Investigator: Alexis Boro, MD
Blood Disorders and Diseases

Study to Evaluate Treatment Compliance, Efficacy and Safety of an Improved Deferasirox Formulation (Granules) in Pediatric Patients (2-<18 Years Old) With Iron Overload

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Principal Investigator: Deepa Manwani, 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
Pediatrics

The Influence of Breakfast on Hormone Responses and Cognitive Performance

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Principal Investigator: Rubina Heptulla, 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
Nephrotic Syndrome, Kidney Diseases (Chronic Kidney Disease)

The Nephrotic Syndrome Study Network (NEPTUNE)

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Principal Investigator: Frederick Kaskel, MD
Nephrology, Kidney Diseases (Chronic Kidney Disease)

The Nephrotic Syndrome Study Network (NEPTUNE)

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Principal Investigator: Frederick Kaskel, MD
Diabetes and Endocrine

The Pediatric Diabetes Consortium (PDC): Type 2 Diabetes in Youth Registry

Principal Investigator: Rubina Heptulla, 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
Heart and Vascular Diseases

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

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Principal Investigator: Ali Zaidi, 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
Bones, Muscles & Joints (Orthopaedics)

Vertical Expandable Prosthetic Titanium Rib (VEPTR) - (Humanitarian Project)

Principal Investigator: Vishal Sarwahi, 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