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Conditions: Leukodystrophy, Globoid Cell
Interventions: PBKR03; Biological
Lead sponsor: Gemma Biotherapeutics; Industry
Eligibility: 1 Month to 9 Months
Enrollment: 24 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2030
U.S. locations: 4
States / cities: Chicago, Illinois • New York, New York • Philadelphia, Pennsylvania + 1 more

Conditions: Obesity, Obesity, Childhood, Weight Gain, Weight Gain Trajectory
Interventions: THRIVE 2.0; Behavioral
Lead sponsor: Children's Hospital Medical Center, Cincinnati; Other
Eligibility: 1 Day and older
Enrollment: 144 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2023 – 2026
U.S. locations: 1
States / cities: Cincinnati, Ohio

Conditions: Cognition Disorder, Healthy
Interventions: Not listed
Lead sponsor: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD); NIH
Eligibility: 1 Year to 3 Years
Enrollment: 219 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 1999 – 2012
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Infantile Hemangiomas
Interventions: Timolol 0.5% Gel Forming Solution (GFS), Mupirocin 2% Ointment; Drug
Lead sponsor: Children's Hospital of Philadelphia; Other
Eligibility: 1 Month to 8 Months
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2014
U.S. locations: 1
States / cities: Philadelphia, Pennsylvania

Conditions: Pompe Disease, Glycogen Storage Disease Type II
Interventions: Alglucosidase Alfa, Methotrexate, Rituximab; Biological · Drug
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: Not listed
Enrollment: 4 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2013
U.S. locations: 2
States / cities: Louisville, Kentucky • Durham, North Carolina

Conditions: Diet Diversity, Infant Nutrition
Interventions: SpoonfulONE; Other
Lead sponsor: Before Brands, Inc; Industry
Eligibility: 4 Months to 6 Months
Enrollment: 1,500 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2021 – 2023
U.S. locations: 1
States / cities: Durham, North Carolina

Conditions: Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets, Generalized Arterial Calcification of Infancy
Interventions: INZ-701; Drug
Lead sponsor: Inozyme Pharma; Industry
Eligibility: 18 Years to 64 Years
Enrollment: 9 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2024
U.S. locations: 2
States / cities: Rochester, Minnesota • Eatontown, New Jersey

Conditions: Cradle Cap, Infantile Seborrheic Dermatitis
Interventions: Promiseb Topical Cream, Bland emollient; Device · Other
Lead sponsor: Promius Pharma, LLC; Industry
Eligibility: 30 Days and older
Enrollment: 42 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2011
U.S. locations: 4
States / cities: Norwich, Connecticut • Louisville, Kentucky • Newark, New Jersey + 1 more

Conditions: Gaucher Disease
Interventions: Standard of Care; Other
Lead sponsor: Takeda; Industry
Eligibility: Up to 5 Years
Enrollment: 11 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2023
U.S. locations: 1
States / cities: Fairfax, Virginia

Conditions: Hemangioma Skin
Interventions: 5% simvastatin ointment; Drug
Lead sponsor: Joyce Teng; Other
Eligibility: 3 Months to 5 Years
Enrollment: 12 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2025
U.S. locations: 1
States / cities: Palo Alto, California

Conditions: Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 Deficiency, ATP-Binding Cassette Subfamily C Member 6 Deficiency
Interventions: No Intervention for this observational study; Other
Lead sponsor: Inozyme Pharma; Industry
Eligibility: Not listed
Enrollment: 54 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2026
U.S. locations: 5
States / cities: Chicago, Illinois • Boston, Massachusetts • Rochester, Minnesota + 2 more

Conditions: CLN6, Batten Disease, Batten's Disease, Neuronal Ceroid Lipofuscinosis CLN6, Neuronal Ceroid Lipofuscinosis
Interventions: scAAV9.CB.CLN6 (dose: 1.5E14 vector genomes); Drug
Lead sponsor: The Charlotte and Gwenyth Gray Foundation; Other
Eligibility: 4 Months and older
Enrollment: 12 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2028
U.S. locations: 1
States / cities: La Jolla, California

Conditions: Infantile Colic
Interventions: Probiotic, Placebo; Dietary Supplement
Lead sponsor: The Archer-Daniels-Midland Company; Industry
Eligibility: 3 Weeks to 12 Weeks
Enrollment: 80 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2026
U.S. locations: 1
States / cities: Chicago, Illinois

Conditions: Adenovirus, Anesthesia, Anxiety, Anxiolysis, Autism, Autistic Disorder, Bacterial Meningitis, Bacterial Septicemia, Benzodiazepine, Bipolar Disorder, Bone and Joint Infections, Central Nervous System Infections, Convulsions, Cytomegalovirus Retinitis, Early-onset Schizophrenia Spectrum Disorders, Epilepsy, General Anesthesia, Gynecologic Infections, Herpes Simplex Virus, Infantile Hemangioma, Infection, Inflammation, Inflammatory Conditions, Intra-abdominal Infections, Lower Respiratory Tract Infections, Migraines, Pain, Pneumonia, Schizophrenia, Sedation, Seizures, Skeletal Muscle Spasms, Skin and Skin-structure Infections, Treatment-resistant Schizophrenia, Urinary Tract Infections, Withdrawal, Sepsis, Gram-negative Infection, Bradycardia, Cardiac Arrest, Cardiac Arrhythmia, Staphylococcal Infections, Nosocomial Pneumonia, Neuromuscular Blockade, Methicillin Resistant Staphylococcus Aureus, Endocarditis, Neutropenia, Headache, Fibrinolytic Bleeding, Pulmonary Arterial Hypertension, CMV Retinitis, Hypertension, Chronic Kidney Diseases, Hyperaldosteronism, Hypokalemia, Heart Failure, Hemophilia, Heavy Menstrual Bleeding, Insomnia
Interventions: The POPS study is collecting PK data on children prescribed the following drugs of interest per standard of care:; Drug
Lead sponsor: Daniel Benjamin; Other
Eligibility: Up to 21 Years
Enrollment: 3,520 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2019
U.S. locations: 43
States / cities: Anchorage, Alaska • Little Rock, Arkansas • La Jolla, California + 37 more

Conditions: 16P11.2 Deletion Syndrome, 16p11.2 Duplications, 1Q21.1 Deletion, 1Q21.1 Microduplication Syndrome (Disorder), ACTL6B, ADNP, AHDC1, ANK2, ANKRD11, ARID1B, ASH1L, BCL11A, CHAMP1, CHD2, CHD8, CSNK2A1, CTBP1, CTNNB1 Gene Mutation, CUL3, DDX3X, DNMT3A, DSCAM, DYRK1A, FOXP1, GRIN2A, GRIN2B, HIVEP2-Related Intellectual Disability, HNRNPH2, KATNAL2, KDM5B, KDM6B, KMT2C Gene Mutation, KMT2E, KMT5B, MBD5, MED13L, PACS1, PPP2R5D-Related Intellectual Disability, PTCHD1, REST, SCN2A Encephalopathy, SETBP1 Gene Mutation, SETD5, SMARCA4 Gene Mutation, SMARCC2, STXBP1 Encephalopathy With Epilepsy, SYNGAP1-Related Intellectual Disability, TBR1, ARHGEF9, HNRNPU, PPP3CA, PPP2R1A, SLC6A1, 2p16.3 Deletions, 5q35 Deletions, 5q35 Duplications, 7q11.23 Duplications, 15Q13.3 Deletion Syndrome, 16p11.2 Triplications, 16P12.2 Microdeletion, 16P13.11 Microdeletion Syndrome (Disorder), 17Q12 Microdeletion Syndrome (Disorder), 17Q12 Duplication Syndrome, 17Q21.31 Deletion Syndrome, 17q21.3 Duplications, ACTB, ADSL, AFF2, ALDH5A1, ANK3, ARX, ATRX Gene Mutation, AUTS2 Syndrome, BCKDK, BRSK2, CACNA1C, CAPRIN1, CASK, CASZ1, CHD3, CIC, CNOT3, CREBBP Gene Mutation, CSDE1, CTCF, DEAF1, DHCR7, DLG4, EBF3, EHMT1, EP300 Gene Mutation, GIGYF1, GRIN1, GRIN2D, IQSEC2-Related Syndromic Intellectual Disability, IRF2BPL, KANSL1, KCNB1, KDM3B, NEXMIF, KMT2A, MBOAT7, MEIS2, MYT1L, NAA15, NBEA, NCKAP1, NIPBL, NLGN2, NLGN3, NLGN4X, NR4A2, NRXN1, NRXN2, NSD1 Gene Mutation, PHF21A, PHF3, PHIP, PSMD12, RELN, RERE, RFX3, RIMS1, RORB, SCN1A, SETD2 Gene Mutation, SHANK2, SIN3A, SLC9A6, SON, SOX5, SPAST, SRCAP, TAOK1, TANC2, TCF20, TLK2, TRIO, TRIP12, UPF3B, USP9X, VPS13B, WAC, WDFY3, ZBTB20, ZNF292, ZNF462, 2Q37 Deletion Syndrome, 9q34 Duplications, 15q15 Deletions, 15Q24 Deletion, NR3C2, SYNCRIP, 2q37.3 Deletion, 6q16 Deletion, 15q11.2 BP1-BP2 Deletion, 16p13.3 Deletion, 17Q11.2 Microduplication Syndrome (Disorder), 17p13.3, Xq28 Duplication, CLCN4, CSNK2B, DYNC1H1, EIF3F, GNB1, MED13, MEF2C, RALGAPB, SCN1B, YY1, Xp11.22 Duplication, PACS2, MAOA, MAOB, HNRNPC, HNRNPD, HNRNPK, HNRNPR, HNRNPUL2, 5P Deletion Syndrome, TCF7L2 Gene Mutation, HECW2, PPM1D, RNU4-2, SNAP25, FOXP2, ITSN1
Interventions: Not listed
Lead sponsor: Simons Searchlight; Other
Eligibility: Not listed
Enrollment: 100,000 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2050
U.S. locations: 2
States / cities: Boston, Massachusetts • Lewisburg, Pennsylvania

Conditions: Infantile Hemangioma
Interventions: Not listed
Lead sponsor: Columbia University; Other
Eligibility: Up to 7 Years
Enrollment: 15 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2018
U.S. locations: 1
States / cities: New York, New York

Conditions: Metabolic Bone Disease, Osteopenia, Neonatal Rickets
Interventions: Vibro-acoustic analysis (VAA), based on ultrasound radiation force; Diagnostic Test
Lead sponsor: Mayo Clinic; Other
Eligibility: 3 Days to 8 Months
Enrollment: 37 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2024
U.S. locations: 1
States / cities: Rochester, Minnesota

Conditions: Spinal Muscular Atrophy
Interventions: nusinersen, Sham procedure; Drug · Procedure
Lead sponsor: Biogen; Industry
Eligibility: Up to 210 Days
Enrollment: 122 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2016
U.S. locations: 12
States / cities: Los Angeles, California • Aurora, Colorado • Orlando, Florida + 9 more

Conditions: Chronic Atypical Neutrophilic Dermatosis With Lipodystrophy and Elevated Temperature (CANDLE), Juvenile Dermatomyositis (JDM), Stimulator of Interferon Genes (STING)-Associated Vasculopathy With Onset During Infancy (SAVI), Aicardi-Goutières Syndrome (AGS)
Interventions: Baricitinib; Drug
Lead sponsor: Eli Lilly and Company; Industry
Eligibility: 6 Months and older
Healthy volunteers: Healthy volunteers not accepted
U.S. locations: 2
States / cities: Bethesda, Maryland • Philadelphia, Pennsylvania

Conditions: Neuronal Ceroid Lipofuscinosis
Interventions: Surgery to implant human CNS stem cells (HuCNS-SC), Medication to suppress the immune system; Procedure · Drug
Lead sponsor: StemCells, Inc.; Industry
Eligibility: 18 Months to 12 Years
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2006 – 2009
U.S. locations: 1
States / cities: Portland, Oregon

Conditions: Infantile Hemangioma
Interventions: Propranolol, Placebo; Drug
Lead sponsor: Pierre Fabre Dermatology; Industry
Eligibility: 35 Days to 150 Days
Enrollment: 512 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2013
U.S. locations: 10
States / cities: Irvine, California • Redwood City, California • San Diego, California + 7 more

Conditions: Dravet Syndrome
Interventions: No Intervention; Other
Lead sponsor: Encoded Therapeutics; Industry
Eligibility: 6 Months to 60 Months
Enrollment: 58 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2023
U.S. locations: 12
States / cities: Los Angeles, California • San Francisco, California • Aurora, Colorado + 9 more

Conditions: Late Infantile Neuronal Ceroid Lipofuscinosis, Batten Disease
Interventions: AAVrh.10CUCLN2; Biological
Lead sponsor: Weill Medical College of Cornell University; Other
Eligibility: 3 Years to 18 Years
Enrollment: 8 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2017
U.S. locations: 1
States / cities: New York, New York

Conditions: Congenital Hyperinsulinism, Hyperinsulinism, Persistent Hyperinsulinemic Hypoglycemia of Infancy, CHI, PHHI
Interventions: F-DOPA, PET scan; Drug · Radiation
Lead sponsor: Children's Hospital of Philadelphia; Other
Eligibility: Up to 18 Years
Enrollment: 106 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2004 – 2009
U.S. locations: 1
States / cities: Philadelphia, Pennsylvania

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Study of Safety, Tolerability and Efficacy of PBKR03 in Pediatric Subjects With Early Infantile Krabbe Disease

A Novel Obesity Prevention Program for High-Risk Infants in Primary Care

The Development of Categorization

Timolol Option for Ulcerated Hemangiomas (TOUCH Trial)

An Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme-naive Cross-Reacting Immunologic Material (CRIM[-]) Patients With Infantile-Onset Pompe Disease

Infancy to Toddlerhood: Early Nutrition & Tolerance (INTENT)

Evaluation of Safety, Tolerability, and Efficacy of INZ-701 in Adults With ENPP1 Deficiency

Promiseb Topical Cream for Cradle Cap

A Study of Velaglucerase Alfa (VPRIV) Given as Standard Patient Care in Young Children With Gaucher Disease

Topical Simvastatin for Treating Infantile Hemangioma

PROPEL - A Prospective Observational Patient Registry to Evaluate ENPP1 and ABCC6 Deficiency

Gene Therapy Trial for CLN6 Batten Disease

Study to Evaluate the Efficacy of a Probiotic in Infantile Colic Symptoms Relief

Pharmacokinetics of Understudied Drugs Administered to Children Per Standard of Care

Online Study of People Who Have Genetic Changes and Features of Autism: Simons Searchlight

Optical Tomographic Imaging of Infantile Hemangiomas

A Study to Assess Metabolic Bone Disease of Prematurity Using an Acoustic Method

A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy

Compassionate Use Protocol for the Treatment of Autoinflammatory Syndromes

Study of HuCNS-SC Cells in Patients With Infantile or Late Infantile Neuronal Ceroid Lipofuscinosis (NCL)

Study to Demonstrate the Efficacy and Safety of Propranolol Oral Solution in Infants With Proliferating Infantile Hemangiomas Requiring Systemic Therapy

Natural History Study of Infants and Children With Developmental and Epileptic Encephalopathies

AAVRh.10 Administered to Children With Late Infantile Neuronal Ceroid Lipofuscinosis

Utility of [F-18] fluoroDOPA for Neonatal Hyperinsulinism