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Research and Clinical Trials

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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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Neurology, Epilepsy, Seizures

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

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Principal Investigator: Alexis Boro, MD
Gastroenterology & Liver Diseases, Inflammatory Bowel Disease, 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
Rheumatic Diseases, Pediatrics, Arthritis, Musculoskeletal Diseases, Autoimmune 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, Pediatrics, Pulmonary Hypertension, Lung Diseases

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
Pediatrics, Psychiatry - Non Cancer, Tourette Syndrome, Neurodevelopmental Disorders

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
Child Development, Endocrinology, Pediatrics

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, Pediatrics, Psychiatry - Non Cancer, Neurodevelopmental Disorders

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
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
Gastroenterology & Liver Diseases, Pediatrics, Inflammatory Bowel Disease, Colitis and Crohn's Disease

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, Hematologic Diseases, Sickle Cell, Anemia

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
Infant Care

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

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Principal Investigator: Iona Munjal, MD
Rheumatic Diseases

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

Principal Investigator: Dawn Wahezi, 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
Autism Spectrum Disorder

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

Principal Investigator: Sophie Molholm, PhD
Preterm Birth, Neonatology, 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

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

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

Childhood Asthma Perception Study (CAPS)

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Principal Investigator: Jonathan Feldman, PhD
Bone Cancer

Collecting and Storing Biological Samples From Patients With Ewing Sarcoma

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Principal Investigator: Peter Cole, 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: Lisa Gennarini, 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
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

Defensin and Innate Immunity

Principal Investigator: Gitit Tomer, MD
Bones, Muscles & Joints (Orthopaedics)

Does Pedicle Morphology Change with Age?

Principal Investigator: Vishal Sarwahi, 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
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
Anesthesia and Pain Management, Blood Disorders and Diseases

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
Blood Disorders and Diseases, Pediatric Hematology/Oncology, Leukemia, Cancer, Neoplasms

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
Rheumatic Diseases

Mood disorder and cognitive dysfunction in pediatric lupus

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

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
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 (Chronic Kidney Disease)

North American Pediatric Renal Trials and Collaborative Studies

Principal Investigator: Marcela Del Rio, MD
Heart and Vascular Diseases, Cardiology, Cardiovascular and Thoracic Surgery, 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
Kidney Diseases (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
Asthma and other Respiratory Diseases, Pediatrics, Lung 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 (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
Child Development, Genetic Disorders, Pediatrics, Psychiatry - Non Cancer, Nervous System Diseases

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
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
Heart and Vascular Diseases, Hematologic Diseases, Pediatric Hematology/Oncology, Embolism or Thrombosis

Prospective Evaluation of the Duration of Therapy for Thrombosis In Children

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Principal Investigator: Jennifer Davila, 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: 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-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
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, Musculoskeletal Diseases, Neoplasms

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: 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
Overweight, Obesity and Weight Loss, Pediatric Endocrinology, Metabolic Diseases

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
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
Heart and Vascular Diseases, Brain and Neurological, Gastroenterology & Liver Diseases, Pediatrics, Crigler-Najjar Syndrome, Metabolic Diseases

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