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ClinicalTrials.gov public records Last synced Jul 22, 2026, 9:02 PM EDT

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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Infantile Clear all
Active, not recruiting Phase 4 Interventional

Pediatric Long-Term Follow-up and Rollover Study

NCT03975829
View directory record Official record
Conditions
Diffuse Astrocytoma, Anaplastic Astrocytoma, Astrocytoma, Oligodendroglioma, Childhood, Anaplastic Oligodendroglioma, Glioblastoma, Pilocytic Astrocytoma, Giant Cell Astrocytoma, Pleomorphic Xanthoastrocytoma, Anaplastic Pleomorphic Xanthoastrocytoma, Angiocentric Glioma, Chordoid Glioma of Third Ventricle, Gangliocytoma, Ganglioglioma, Anaplastic Ganglioglioma, Dysplastic Gangliocytoma of Cerebrellum, Desmoplastic Infantile Astrocytoma and Ganglioglioma, Papillary Glioneuronal Tumor, Rosette-forming Glioneuronal Tumor, Central Neurocytoma, Extraventricular Neurocytoma, Cerebellar Liponeurocytoma, Neurofibromatosis Type 1
Interventions
dabrafenib, trametinib
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
1 Year to 99 Years
Enrollment
163 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2026
U.S. locations
12
States / cities
Phoenix, Arizona • Washington D.C., District of Columbia • Miami, Florida + 8 more
Source: ClinicalTrials.gov public record
Updated Jul 6, 2026 · Synced Jul 22, 2026, 9:02 PM EDT
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 10, 2017 · Synced Jul 22, 2026, 9:02 PM EDT
Completed Not applicable Interventional

Nutritional Intervention to Prevent Diabetes

NCT00333554
View directory record Official record
Conditions
Type 1 Diabetes Mellitus
Interventions
DHA Treatment, Placebo for DHA
Dietary Supplement
Lead sponsor
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
NIH
Eligibility
Up to 5 Months
Enrollment
99 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2013
U.S. locations
9
States / cities
Los Angeles, California • Orange, California • San Francisco, California + 6 more
Source: ClinicalTrials.gov public record
Updated Apr 28, 2020 · Synced Jul 22, 2026, 9:02 PM EDT
Completed No phase listed Observational

Real-time State of Vigilance Monitor for the Neonatal Intensive Care Unit

NCT04920175
View directory record Official record
Conditions
Sleep Disturbance in Infancy (Disorder), Neonatal Disease
Interventions
Novel Real-time Neonatal Sleep Stage Detection Algorithm
Other
Lead sponsor
University of Michigan
Other
Eligibility
1 Day to 1 Year
Enrollment
39 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
1
States / cities
Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Jan 23, 2025 · Synced Jul 22, 2026, 9:02 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Hemangioma Growth During the First 6 Months of Life

NCT00911781
View directory record Official record
Conditions
Hemangiomas of Infancy
Interventions
No intervention
Other
Lead sponsor
University of California, San Francisco
Other
Eligibility
3 Months to 5 Years
Enrollment
36 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2011
U.S. locations
2
States / cities
San Francisco, California • Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Feb 4, 2013 · Synced Jul 22, 2026, 9:02 PM EDT
Completed No phase listed Observational Results available

A Retrospective Study of the Natural History of Patients With Severe Perinatal and Infantile Hypophosphatasia (HPP)

NCT01419028
View directory record Official record
Conditions
Hypophosphatasia (HPP)
Interventions
Not listed
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
Not listed
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2014
U.S. locations
5
States / cities
Los Angeles, California • Indianapolis, Indiana • St Louis, Missouri + 2 more
Source: ClinicalTrials.gov public record
Updated Mar 31, 2019 · Synced Jul 22, 2026, 9:02 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Study of IV VTS-270 for Infantile Liver Disease Associated With Niemann-Pick Disease, Type C

NCT03471143
View directory record Official record
Conditions
Niemann-Pick Disease, Type C
Interventions
VTS-270
Drug
Lead sponsor
Washington University School of Medicine
Other
Eligibility
Up to 6 Months
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2024
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Jan 4, 2026 · Synced Jul 22, 2026, 9:02 PM EDT
Recruiting Not applicable Interventional

Let's E.A.T.! (Eating With Assistive Technology): An Intervention to Support Children With Feeding Tubes and Tracheostomies

NCT06525818
View directory record Official record
Conditions
Feeding Tube, Children With Medical Complexity, Tracheostomy, Feeding Disorder, Infancy or Early Childhood
Interventions
Tube weaning therapy intervention, Feeding Group
Other
Lead sponsor
University of Chicago
Other
Eligibility
Up to 3 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Jul 9, 2026 · Synced Jul 22, 2026, 9:02 PM EDT
Completed Phase 2 Interventional Accepts healthy volunteers

Prevention of Overfeeding During Infancy

NCT01043978
View directory record Official record
Conditions
Obesity
Interventions
Use of baby bottle nipple
Behavioral
Lead sponsor
University of California, Davis
Other
Eligibility
Up to 3 Months
Enrollment
231 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2010 – 2012
U.S. locations
1
States / cities
Sacramento, California
Source: ClinicalTrials.gov public record
Updated Aug 23, 2012 · Synced Jul 22, 2026, 9:02 PM EDT
Recruiting Phase 3 Interventional

A Double-blind Study Evaluating the Efficacy, Safety, and Tolerability of Zorevunersen in Patients With Dravet Syndrome

NCT06872125
View directory record Official record
Conditions
Dravet Syndrome
Interventions
zorevunersen, Sham Comparator
Drug · Other
Lead sponsor
Stoke Therapeutics, Inc
Industry
Eligibility
2 Years to 17 Years
Enrollment
170 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
28
States / cities
Phoenix, Arizona • Little Rock, Arkansas • Los Angeles, California + 23 more
Source: ClinicalTrials.gov public record
Updated Jun 30, 2026 · Synced Jul 22, 2026, 9:02 PM EDT
Active, not recruiting Phase 4 Interventional Accepts healthy volunteers

Infant Mortality Reduction by the Mass Administration of Azithromycin

NCT04716712
View directory record Official record
Conditions
Child Mortality
Interventions
Azithromycin, Placebo
Drug
Lead sponsor
University of California, San Francisco
Other
Eligibility
1 Month to 11 Months
Enrollment
694,400 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2026
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Mar 12, 2025 · Synced Jul 22, 2026, 9:02 PM EDT
Approved For Marketing No phase listed Expanded access

Expanded Access Program for Asfotase Alfa Treatment for Patients With Infantile- or Juvenile-onset Hypophosphatasia (HPP)

NCT02496689
View directory record Official record
Conditions
Hypophosphatasia
Interventions
asfotase alfa
Biological
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
Not listed
U.S. locations
2
States / cities
Lakewood, Colorado • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 28, 2019 · Synced Jul 22, 2026, 9:02 PM EDT
Terminated Phase 3 Interventional Results available

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

NCT02193074
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Feb 16, 2021 · Synced Jul 22, 2026, 9:02 PM EDT
Recruiting No phase listed Observational

Congenital Muscle Disease Study of Patient and Family Reported Medical Information

NCT01403402
View directory record Official record
Conditions
Congenital Muscular Dystrophy With ITGA7 (Integrin Alpha-7) Deficiency, Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy and Abnormal Glycosylation of Dystroglycan With Severe Epilepsy), Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy With Fatty Liver and Infantile-onset Cataract Caused by TRAPPC11 Mutations), Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy With Hypoglycosylation of Dystroglycan), Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy With Hypoglycosylation of Dystroglycan and Epilepsy), Alpha-Dystroglycanopathy (Dystroglycanopathy, Congenital With or Without Mental Retardation (Formerly MDC1C)), Alpha-Dystroglycanopathy (Fukuyama CMD), Alpha-Dystroglycanopathy (LGMDR09 FKRP Related (Formerly LGMD2I)), Alpha-Dystroglycanopathy (LGMDR11 POMT1 Related (Formerly LGMD2K)), Alpha-Dystroglycanopathy (LGMDR13 FKTN Related (Formerly LGMD2M)), Alpha-Dystroglycanopathy (LGMDR14 POMT2 Related (Formerly LGMD2N)), Alpha-Dystroglycanopathy (LGMDR15 POMGnT1 Related (Formerly LGMD2O)), Alpha-Dystroglycanopathy (LGMDR19 GMPPB Related (Formerly LGMD2T)), Alpha-Dystroglycanopathy (LGMDR20 ISPD Related (Formerly LGMD2U)), Alpha-Dystroglycanopathy (LGMDR24 POMGnT2 Related), Alpha-Dystroglycanopathy (Muscle Eye Brain Disease (MEB)), Alpha-Dystroglycanopathy (Walker Warburg Syndrome (WWS)), Choline Kinase B Receptor - CHKB, Collagen VI Related Disorders, Collagen XII Related Disorders, Congenital Muscular Dystrophy Not Otherwise Specified (Including Merosin Positive), Congenital Muscular Dystrophy With Cataracts and Intellectual Disability (MDCCAID), Congenital Muscular Dystrophy With Joint Hyperlaxity, Congenital Muscular Dystrophy With Rigid Spine Related to ACTA1, Emery-Dreifuss Muscular Dystrophy, GOLGA2-related Congenital Muscle Dystrophy With Brain Involvement, LMNA Related Disorders, Merosin Deficient CMD (Full or Partial), Nesprin Related MD (SYNE1), SELENON Related Disorders (Previously Known as SEPN1), SELENON Related Myopathy (Aka SEPN1), Telethonin CMD, Congenital Myasthenic Syndrome, Limb-Girdle Muscular Dystrophy, LGMDD01 - DNAJB6 (Formerly LGMD1D), LGMDD05 - Collagen VI Related Bethlem Myopathy (Dominant), LGMDR07 - Telethonin (TCAP) Related (Formerly LGMD2G), LGMDR08 - TRIM Related (Formerly LGMD2H), LGMDR09 - FKRP Related (Formerly LGMD2I), LGMDR10 - Titin (TTN) Related (Formerly LGMD2J), LGMDR11 - POMT1 Related (Formerly LGMD2K), LGMDR13 - Fukutin (FKTN) Related (Formerly LGMD2M), LGMDR14 - POMT2 Related (Formerly LGMD2N), LGMDR15 - POMGnT1 Related (Formerly LGMD2O), LGMDR16 - DAG1 Related Dystroglycanopathy (Formerly LGMD2P), LGMDR17 - Plectin (PLEC) Related (Formerly LGMD2Q), LGMDR18 - TRAPPC11 Related (Formerly LGMD2S), LGMDR19 - GMPPB Related (Formerly LGMD2T), LGMDR20 - ISPD Related (Formerly LGMD2U), LGMDR22 - Collagen VI Related Bethlem Myopathy (Recessive), LGMDR23 - LAMA2 Related, LGMDR24 - POMGnT2 Related
Interventions
Not listed
Lead sponsor
Cure CMD
Other
Eligibility
Not listed
Enrollment
4,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2029
U.S. locations
1
States / cities
Lakewood, California
Source: ClinicalTrials.gov public record
Updated Aug 8, 2021 · Synced Jul 22, 2026, 9:02 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Longitudinal Follow Up of Subjects Enrolled in Randomized Trial of Prenatal and Infancy Home Visitation

NCT00443638
View directory record Official record
Conditions
Risk Reduction Behavior, Reproductive Behavior
Interventions
nurse home visitation
Behavioral
Lead sponsor
University of Colorado, Denver
Other
Eligibility
Female only
Enrollment
320 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1998 – 2000
U.S. locations
1
States / cities
Elmira, New York
Source: ClinicalTrials.gov public record
Updated Jan 30, 2013 · Synced Jul 22, 2026, 9:02 PM EDT
Recruiting Phase 3 Interventional

A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis

NCT07054515
View directory record Official record
Conditions
Niemann-Pick Type C Disease, GM1 Gangliosidosis, GM2 Gangliosidosis
Interventions
AZ-3102, Placebo
Drug
Lead sponsor
Azafaros B.V.
Industry
Eligibility
4 Years and older
Enrollment
147 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
5
States / cities
Oakland, California • Minneapolis, Minnesota • Rochester, Minnesota + 2 more
Source: ClinicalTrials.gov public record
Updated Jul 20, 2026 · Synced Jul 22, 2026, 9:02 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

The Role of Motion in Infants' Ability to Categorize

NCT00362076
View directory record Official record
Conditions
Child Development
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
3 Months to 6 Years
Enrollment
941 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1998 – 2012
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 1, 2017 · Synced Jul 22, 2026, 9:02 PM EDT
Completed No phase listed Observational

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

NCT04721366
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 28, 2024 · Synced Jul 22, 2026, 9:02 PM EDT
Completed Phase 1Phase 2 Interventional

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

NCT01414985
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Aug 30, 2020 · Synced Jul 22, 2026, 9:02 PM EDT
Completed No phase listed Observational

GM1 and GM2 Gangliosidosis PROspective Neurological Disease TrajectOry Study (PRONTO)

NCT05109793
View directory record Official record
Conditions
GM1 Gangliosidosis, Sandhoff Disease, Tay-Sachs Disease
Interventions
Not listed
Lead sponsor
Azafaros B.V.
Industry
Eligibility
2 Years to 20 Years
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
2
States / cities
Oakland, California • Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Sep 28, 2025 · Synced Jul 22, 2026, 9:02 PM EDT
Completed No phase listed Observational

Interviews and Video Capture in Patients With GM1 Gangliosidosis

NCT04310163
View directory record Official record
Conditions
GM1 Gangliosidosis
Interventions
Natural history
Other
Lead sponsor
LYSOGENE
Industry
Eligibility
Not listed
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
1
States / cities
Plymouth, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jun 7, 2023 · Synced Jul 22, 2026, 9:02 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 29, 2026 · Synced Jul 22, 2026, 9:02 PM EDT
Completed No phase listed Observational

Artificial Intelligence-based Video Analysis to Detect Infantile Spasms

NCT06315829
View directory record Official record
Conditions
Infantile Spasms
Interventions
Spasm Vision
Device
Lead sponsor
Johns Hopkins University
Other
Eligibility
Up to 2 Years
Enrollment
61 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated May 6, 2026 · Synced Jul 22, 2026, 9:02 PM EDT
Completed Phase 4 Interventional Results available

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

NCT00701129
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated May 12, 2014 · Synced Jul 22, 2026, 9:02 PM EDT