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ClinicalTrials.gov public records Last synced Oct 21, 2026, 5:08 PM EDT

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Showing 1–24 of 217 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Infantile Clear all
Completed No phase listed Observational

A Multicenter Collaborative Study on the Clinical Features, Expression Profiling, and Quality of Life of Infantile Onset FSHD

NCT01437345
View directory record Official record
Conditions
Facioscapulohumeral Muscular Dystrophy
Interventions
Not listed
Lead sponsor
Cooperative International Neuromuscular Research Group
Network
Eligibility
Not listed
Enrollment
53 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2017
U.S. locations
7
States / cities
Sacramento, California • Washington D.C., District of Columbia • Minneapolis, Minnesota + 4 more
Source: ClinicalTrials.gov public record
Updated Oct 11, 2017 · Synced Oct 21, 2026, 5:08 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional Results available

A Study of Oral LOXO-292 (Selpercatinib) in Pediatric Participants With Advanced Solid or Primary Central Nervous System (CNS) Tumors

NCT03899792
View directory record Official record
Conditions
Medullary Thyroid Cancer, Infantile Myofibromatosis, Infantile Fibrosarcoma, Papillary Thyroid Cancer, Soft Tissue Sarcoma
Interventions
Selpercatinib
Drug
Lead sponsor
Eli Lilly and Company
Industry
Eligibility
6 Months to 21 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2029
U.S. locations
12
States / cities
Los Angeles, California • Aurora, Colorado • Orlando, Florida + 9 more
Source: ClinicalTrials.gov public record
Updated Jan 6, 2026 · Synced Oct 21, 2026, 5:08 PM EDT
Completed No phase listed Observational

Genotype-Phenotype Correlations of Late Infantile Neuronal Ceroid Lipofuscinosis

NCT01035424
View directory record Official record
Conditions
Batten Disease, Late Infantile Neuronal Ceroid Lipofuscinosis
Interventions
Not listed
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
2 Years to 18 Years
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2016
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 29, 2020 · Synced Oct 21, 2026, 5:08 PM EDT
Completed Phase 2Phase 3 Interventional

Extension Study of Patients With Infantile-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU01602

NCT00125879
View directory record Official record
Conditions
Glycogen Storage Disease Type II
Interventions
Myozyme
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Not listed
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2006
U.S. locations
6
States / cities
Birmingham, Alabama • Gainesville, Florida • Miami, Florida + 3 more
Source: ClinicalTrials.gov public record
Updated Feb 6, 2014 · Synced Oct 21, 2026, 5:08 PM EDT
Recruiting Phase 2 Interventional

Rifampin in CYP24A1-related Hypercalcemia and Hypercalciuria

NCT03301038
View directory record Official record
Conditions
Idiopathic Infantile Hypercalcaemia - Severe Form, Genetic Disease, Hypercalcemia, Idiopathic, of Infancy, Hypercalciuric Hypercalcemia, Idiopathic Infantile Hypercalcemia - Mild Form, Hypercalciuria
Interventions
Rifampin
Drug
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
6 Months to 70 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2030
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 13, 2026 · Synced Oct 21, 2026, 5:08 PM EDT
Recruiting No phase listed Observational

Registry of Patients Diagnosed With Lysosomal Storage Diseases

NCT05619900
View directory record Official record
Conditions
Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis IV A, Mucopolysaccharidosis VI, Mucopolysaccharidosis VII, Pompe Disease Infantile-Onset, Neuronopathic Gaucher Disease, Wolman Disease
Interventions
There is no intervention
Other
Lead sponsor
University of California, San Francisco
Other
Eligibility
Up to 64 Years
Enrollment
250 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2050
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Apr 8, 2026 · Synced Oct 21, 2026, 5:08 PM EDT
Terminated Phase 1 Interventional

Ketogenic Diet for Prevention of Epileptic Spasms in Infantile Onset Genetic Epilepsies

NCT06700811
View directory record Official record
Conditions
Developmental and Epileptic Encephalopathies, Epileptic Spasms, Genetic Epilepsy, Neonatal and Infant Epilepsy
Interventions
Ketogenic diet
Drug
Lead sponsor
Heather Olson
Other
Eligibility
0 Days to 9 Months
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Oct 14, 2026 · Synced Oct 21, 2026, 5:08 PM EDT
Completed No phase listed Observational

Pharmacokinetics of Understudied Drugs Administered to Children Per Standard of Care

NCT01431326
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Sep 6, 2023 · Synced Oct 21, 2026, 5:08 PM EDT
Withdrawn Phase 3 Interventional

A Study to Evaluate Safety and Efficacy of AMZ002 Treatment, Compared With Vigabatrin in Participants With Infantile Spasms

NCT05128344
View directory record Official record
Conditions
Spasms, Infantile
Interventions
AMZ002 injectable solution, 0.5mg/mL, Vigabatrin, oral
Drug
Lead sponsor
Amzell
Industry
Eligibility
2 Months to 24 Months
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
12
States / cities
Aurora, Colorado • Miami, Florida • Orlando, Florida + 8 more
Source: ClinicalTrials.gov public record
Updated Apr 20, 2023 · Synced Oct 21, 2026, 5:08 PM EDT
Completed No phase listed Observational

Percent Body Fat in Breast Fed Infants

NCT00616083
View directory record Official record
Conditions
Healthy
Interventions
Not listed
Lead sponsor
Arkansas Children's Hospital Research Institute
Other
Eligibility
Up to 2 Weeks
Enrollment
190 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2007
U.S. locations
3
States / cities
Little Rock, Arkansas • Cleveland, Ohio • Oklahoma City, Oklahoma
Source: ClinicalTrials.gov public record
Updated Aug 20, 2009 · Synced Oct 21, 2026, 5:08 PM EDT
Terminated No phase listed Observational Accepts healthy volunteers

Impact of Vaccination on Th2 Immunity in Infancy

NCT02213341
View directory record Official record
Conditions
Food Hypersensitivity
Interventions
Skin prick allergen test, Blood draw
Procedure
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
5 Months to 7 Months
Enrollment
77 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2016
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 23, 2019 · Synced Oct 21, 2026, 5:08 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

Effect of Infant Formula on Energy Balance

NCT01700205
View directory record Official record
Conditions
Development
Interventions
Type of Formula
Other
Lead sponsor
Monell Chemical Senses Center
Other
Eligibility
1 Week to 3 Weeks
Enrollment
113 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2016
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Nov 22, 2019 · Synced Oct 21, 2026, 5:08 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

BabyQ's: Randomized Controlled Trial of Health Messaging in Pregnancy and Infancy

NCT04238585
View directory record Official record
Conditions
Pregnancy Related, Infant Development
Interventions
Mobile messaging
Behavioral
Lead sponsor
Columbia University
Other
Eligibility
18 Years and older
Enrollment
291 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2021
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Nov 16, 2021 · Synced Oct 21, 2026, 5:08 PM EDT
Completed No phase listed Observational

Development of a Conservative Care and Bracing Registry (CCBR)

NCT07527611
View directory record Official record
Conditions
Adolescent Idiopathic Scoliosis (AIS), Ealy Onset Scoliosis, Juvenile Idiopathic Scoliosis, Infantile Idiopathic Scoliosis, Neuromuscular Scoliosis
Interventions
Not listed
Lead sponsor
National Scoliosis Center
Other
Eligibility
1 Year to 18 Years
Enrollment
280 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2025
U.S. locations
1
States / cities
Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Apr 14, 2026 · Synced Oct 21, 2026, 5:08 PM EDT
Completed Phase 3 Interventional Results available

Trial of Cannabidiol (CBD; GWP42003-P) for Infantile Spasms (GWPCARE7)

NCT02953548
View directory record Official record
Conditions
Infantile Spasms
Interventions
GWP42003-P
Drug
Lead sponsor
Jazz Pharmaceuticals
Industry
Eligibility
1 Month to 24 Months
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2018
U.S. locations
6
States / cities
Little Rock, Arkansas • Winston-Salem, North Carolina • Columbus, Ohio + 3 more
Source: ClinicalTrials.gov public record
Updated Sep 2, 2022 · Synced Oct 21, 2026, 5:08 PM EDT
Active, not recruiting No phase listed Observational

Cerliponase Alfa Observational Study in the US

NCT04476862
View directory record Official record
Conditions
Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2
Interventions
Cerliponase Alfa, Administration Kit
Drug · Device
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
Not listed
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2030
U.S. locations
16
States / cities
Little Rock, Arkansas • Orange, California • Aurora, Colorado + 12 more
Source: ClinicalTrials.gov public record
Updated Jun 30, 2026 · Synced Oct 21, 2026, 5:08 PM EDT
Completed Phase 1 Interventional Results available

A Phase I Study to Evaluate the Oral Absorption of Nepadutant in Infants

NCT00655083
View directory record Official record
Conditions
Infantile Colic, Infantile Functional Gastrointestinal Disorders
Interventions
Nepadutant
Drug
Lead sponsor
Menarini Group
Industry
Eligibility
6 Weeks to 24 Weeks
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2009
U.S. locations
3
States / cities
Little Rock, Arkansas • Louisville, Kentucky • Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Oct 17, 2011 · Synced Oct 21, 2026, 5:08 PM EDT
Completed Not applicable Interventional

Healthy Growth Abbreviated Pilot Study

NCT01977105
View directory record Official record
Conditions
Childhood Obesity
Interventions
Grow Together peer group
Behavioral
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
18 Years and older · Female only
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Feb 6, 2014 · Synced Oct 21, 2026, 5:08 PM EDT
Terminated No phase listed Observational

Natural History Study of Batten Disease

NCT04644549
View directory record Official record
Conditions
Batten Disease, Neuronal Ceroid Lipofuscinosis CLN6, Neuronal Ceroid Lipofuscinosis CLN3
Interventions
Not listed
Lead sponsor
Emily de los Reyes
Other
Eligibility
Not listed
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
1
States / cities
Rochester, New York
Source: ClinicalTrials.gov public record
Updated Jul 30, 2025 · Synced Oct 21, 2026, 5:08 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Gene Transfer Clinical Trial for Infantile and Late Infantile Krabbe Disease Treated Previously With HSCT

NCT05739643
View directory record Official record
Conditions
Krabbe Disease
Interventions
FBX-101
Biological
Lead sponsor
Forge Biologics, Inc
Industry
Eligibility
Up to 18 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
3
States / cities
Orange, California • Ann Arbor, Michigan • Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Nov 20, 2025 · Synced Oct 21, 2026, 5:08 PM EDT
Withdrawn Phase 2 Interventional

Timolol Option for Ulcerated Hemangiomas (TOUCH Trial)

NCT01408056
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jan 17, 2014 · Synced Oct 21, 2026, 5:08 PM EDT
Completed Phase 2 Interventional Results available

A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease

NCT02678689
View directory record Official record
Conditions
Jansky-Bielschowsky Disease, Batten Disease, Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2, CLN2 Disease, CLN2 Disorder
Interventions
BMN 190 recombinant human tripeptidyl peptidase-1 (rhTPP1), Intracerebroventricular access device
Biological · Device
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
Up to 17 Years
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2022
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Feb 17, 2025 · Synced Oct 21, 2026, 5:08 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Treatment Monitoring in Autism Spectrum Disorder (ASD) in Children

NCT05675371
View directory record Official record
Conditions
Autism Spectrum Disorder, Autism, Developmental Delay, Autism, Early Infantile, Autism, Infantile
Interventions
Assessment by Expert Clinician During Behavioral Intervention (ABA or similar), Assessment by the EarliPoint Assessment For Autism Spectrum Disorder
Behavioral · Device
Lead sponsor
EarliTec Diagnostics, Inc
Industry
Eligibility
15 Months to 84 Months
Enrollment
325 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2025
U.S. locations
9
States / cities
Phoenix, Arizona • Glendale, California • San Rafael, California + 6 more
Source: ClinicalTrials.gov public record
Updated Sep 15, 2025 · Synced Oct 21, 2026, 5:08 PM EDT
Completed No phase listed Observational

A Natural History Study of Participants With Potassium Sodium-Activated Channel Subfamily T Member 1 (KCNT1)-Related Epilepsy

NCT04924153
View directory record Official record
Conditions
KCNT1-Related Epilepsy
Interventions
No Intervention
Other
Lead sponsor
Biogen
Industry
Eligibility
0 Years to 50 Years
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
1
States / cities
Rochester, New York
Source: ClinicalTrials.gov public record
Updated Feb 28, 2024 · Synced Oct 21, 2026, 5:08 PM EDT