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ClinicalTrials.gov public records Last synced Oct 22, 2026, 7:24 AM EDT

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Showing 1417–1440 of 1,711 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Central Nervous System Anomalies Clear all
Completed Phase 2 Interventional Results available

Short-Term Outcome of N-Carbamylglutamate in the Treatment of Acute Hyperammonemia

NCT01599286
View directory record Official record
Conditions
Propionic Acidemia, Type I and/or Type II, Methylmalonic Acidemia, Carbamoyl-Phosphate Synthase I Deficiency Disease, Ornithine Carbamoyltransferase Deficiency
Interventions
Carbaglu, Placebo
Drug
Lead sponsor
Mendel Tuchman
Other
Eligibility
1 Week to 99 Years
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2020
U.S. locations
9
States / cities
Los Angeles, California • Palo Alto, California • Aurora, Colorado + 6 more
Source: ClinicalTrials.gov public record
Updated Feb 15, 2021 · Synced Oct 22, 2026, 7:24 AM EDT
Not listed Phase 3 Interventional

AT-007 in Adult Subjects With Classic Galactosemia (CG)

NCT05418829
View directory record Official record
Conditions
Classic Galactosemia
Interventions
AT-007
Drug
Lead sponsor
Applied Therapeutics, Inc.
Industry
Eligibility
18 Years to 65 Years
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
1
States / cities
Berlin, New Jersey
Source: ClinicalTrials.gov public record
Updated Jun 14, 2022 · Synced Oct 22, 2026, 7:24 AM EDT
Recruiting No phase listed Observational

A Real-world Long-term Safety and Immunogenicity Study of Olipudase Alfa Therapy in Pediatric Patients Less Than 2 Years of Age With Acid Sphingomyelinase Deficiency (ASMD)

NCT06192576
View directory record Official record
Conditions
Niemann-Pick Diseases, Acid Sphingomyelinase Deficiency
Interventions
Olipudase alfa
Drug
Lead sponsor
Sanofi
Industry
Eligibility
Up to 2 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2029
U.S. locations
6
States / cities
Chicago, Illinois • Cincinnati, Ohio • Columbus, Ohio + 3 more
Source: ClinicalTrials.gov public record
Updated Sep 16, 2026 · Synced Oct 22, 2026, 7:24 AM EDT
Completed Phase 3 Interventional Results available

A Phase III Trial to Assess the Safety and Efficacy of Plant Cell Expressed GCD in Patients With Gaucher Disease

NCT00376168
View directory record Official record
Conditions
Gaucher Disease
Interventions
Plant cell expressed recombinant glucocerebrosidase (prGCD)
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years and older
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2009
U.S. locations
3
States / cities
Coral Springs, Florida • Decatur, Georgia • New York, New York
Source: ClinicalTrials.gov public record
Updated Oct 4, 2018 · Synced Oct 22, 2026, 7:24 AM EDT
Completed Phase 3 Interventional

Efficacy of [18F]Florbetaben PET for Diagnosis of Cardiac AL Amyloidosis

NCT05184088
View directory record Official record
Conditions
Cardiac Amyloidosis, AL Amyloidosis, ATTR Amyloidosis
Interventions
[18F]florbetaben
Drug
Lead sponsor
Lantheus Germany GmbH
Industry
Eligibility
18 Years and older
Enrollment
244 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
3
States / cities
Kansas City, Kansas • Philadelphia, Pennsylvania • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 3, 2026 · Synced Oct 22, 2026, 7:24 AM EDT
Active, not recruiting Phase 3 Interventional

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

NCT07082543
View directory record Official record
Conditions
Gangliosidoses, GM2, Gangliosidosis, GM1
Interventions
AZ-3102, Placebo
Drug
Lead sponsor
Azafaros B.V.
Industry
Eligibility
4 Years and older
Enrollment
75 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 Sep 22, 2026 · Synced Oct 22, 2026, 7:24 AM EDT
Terminated Phase 2 Interventional Results available

Severe Renal Disease Study in Fabry Patients Treated With Fabrazyme

NCT00837824
View directory record Official record
Conditions
Fabry Disease, Chronic Kidney Disease, Stage IV (Severe)
Interventions
Fabrazyme (agalsidase beta)
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
16 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2003
U.S. locations
9
States / cities
Farmington, Connecticut • Coral Springs, Florida • Kansas City, Kansas + 6 more
Source: ClinicalTrials.gov public record
Updated Apr 7, 2015 · Synced Oct 22, 2026, 7:24 AM EDT
Completed Phase 4 Interventional Results available

A Study Evaluating Glycosphingolipid Clearance in Patients Treated With Agalsidase Alfa Who Switch to Agalsidase Beta

NCT01650779
View directory record Official record
Conditions
Fabry Disease
Interventions
Agalsidase beta
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Male only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
6
States / cities
Coral Springs, Florida • Decatur, Georgia • Baltimore, Maryland + 3 more
Source: ClinicalTrials.gov public record
Updated Jul 10, 2014 · Synced Oct 22, 2026, 7:24 AM EDT
Enrolling by invitation Not applicable Interventional

Ready, Set, Go! A Physical Fitness Intervention for Children With Mobility Challenges

NCT04789746
View directory record Official record
Conditions
Cerebral Palsy, Myelomeningocele, Arthrogryposis, Motor Skills Disorders, Gross Motor Development Delay
Interventions
adapted Chicago Run running program
Behavioral
Lead sponsor
Northwestern University
Other
Eligibility
6 Years to 18 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2028
U.S. locations
1
States / cities
Chicago Heights, Illinois
Source: ClinicalTrials.gov public record
Updated Aug 18, 2026 · Synced Oct 22, 2026, 7:24 AM EDT
Withdrawn Phase 2 Interventional

Study of Tesomet With Open-label Extension in Subjects With Prader-Willi Syndrome

NCT05198362
View directory record Official record
Conditions
Prader-Willi Syndrome
Interventions
Placebo, Tesomet
Other · Drug
Lead sponsor
Saniona
Industry
Eligibility
13 Years to 65 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
1
States / cities
Lansing, Michigan
Source: ClinicalTrials.gov public record
Updated Dec 13, 2022 · Synced Oct 22, 2026, 7:24 AM EDT
Completed Phase 1Phase 2 Interventional

Doxorubicin Hydrochloride Liposome and Bortezomib in Treating Patients With Refractory Hematologic Cancer or Malignant Solid Tumor or Metastatic Breast Cancer

NCT00237627
View directory record Official record
Conditions
Breast Cancer, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Ovarian Cancer, Unspecified Adult Solid Tumor, Protocol Specific
Interventions
PS-341, Doxil, Velcade
Drug
Lead sponsor
UNC Lineberger Comprehensive Cancer Center
Other
Eligibility
18 Years and older
Enrollment
107 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2010
U.S. locations
1
States / cities
Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated May 18, 2012 · Synced Oct 22, 2026, 7:24 AM EDT
Completed Not applicable Interventional

Dissemination of Comprehensive Behavioral Intervention for Tics (CBIT) to Occupational Therapists: A Feasibility Study

NCT02114905
View directory record Official record
Conditions
Tourette Disorder, Chronic Tic Disorder
Interventions
OTs Trained and Deliver CBIT
Behavioral
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
7 Years to 17 Years
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2015
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 20, 2019 · Synced Oct 22, 2026, 7:24 AM EDT
Completed Phase 2 Interventional Results available

A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Female Participants With Fabry Disease

NCT00304512
View directory record Official record
Conditions
Fabry Disease
Interventions
migalastat HCl
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years to 65 Years · Female only
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2008
U.S. locations
1
States / cities
Decatur, Georgia
Source: ClinicalTrials.gov public record
Updated Oct 3, 2018 · Synced Oct 22, 2026, 7:24 AM EDT
Completed Phase 2Phase 3 Interventional

Arimidex Multicenter Trial in Growth Hormone (GH) Deficient Boys

NCT00133354
View directory record Official record
Conditions
Hypopituitarism
Interventions
Arimidex (Anastrozole), Placebo, Growth Hormone
Drug
Lead sponsor
Nemours Children's Clinic
Other
Eligibility
11 Years to 18 Years · Male only
Enrollment
53 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2010
U.S. locations
1
States / cities
Jacksonville, Florida
Source: ClinicalTrials.gov public record
Updated Oct 12, 2011 · Synced Oct 22, 2026, 7:24 AM EDT
Recruiting No phase listed Observational

Rocket Study: A Study to Characterize Biomarkers and Disease Progression in Participants With Pelizaeus-Merzbacher Disease

NCT05659901
View directory record Official record
Conditions
Pelizaeus-Merzbacher Disease
Interventions
Not listed
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
6 Months to 17 Years · Male only
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2029
U.S. locations
3
States / cities
Atlanta, Georgia • Baltimore, Maryland • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 2, 2026 · Synced Oct 22, 2026, 7:24 AM EDT
Completed Phase 3 Interventional Results available

Safety, Efficacy, & PK of PRX-102 in Patients With Fabry Disease Administered Intravenously Every 4 Weeks

NCT03180840
View directory record Official record
Conditions
Fabry Disease
Interventions
Pegunigalsidase alfa
Biological
Lead sponsor
Protalix
Industry
Eligibility
18 Years to 60 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2020
U.S. locations
7
States / cities
Birmingham, Alabama • Atlanta, Georgia • Iowa City, Iowa + 4 more
Source: ClinicalTrials.gov public record
Updated Sep 13, 2023 · Synced Oct 22, 2026, 7:24 AM EDT
Recruiting No phase listed Observational

Gonadal Tissue Freezing for Fertility Preservation in Individuals at Risk for Ovarian Dysfunction, Premature Ovarian Insufficiency and Clinically Indicated Gonadectomy

NCT04948658
View directory record Official record
Conditions
Turner Syndrome, Post-menarcheal Adolescents, Ovarian Disfunction, Galactosemia, Variations in Sex Characteristics, Differences in Sex Development
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
2 Years to 35 Years
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2030
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 5, 2026 · Synced Oct 22, 2026, 7:24 AM EDT
Terminated No phase listed Observational Accepts healthy volunteers

Neuromuscular Characteristics of Individuals With Down Syndrome (DS)

NCT00643955
View directory record Official record
Conditions
Down Syndrome
Interventions
muscle strength
Other
Lead sponsor
State University of New York - Upstate Medical University
Other
Eligibility
18 Years to 45 Years
Enrollment
15 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 2008
U.S. locations
1
States / cities
Syracuse, New York
Source: ClinicalTrials.gov public record
Updated Dec 21, 2023 · Synced Oct 22, 2026, 7:24 AM EDT
Completed No phase listed Observational

Defining the Genetic Basis for the Development of Primary Pigmented Nodular Adrenocortical Disease (PPNAD) and the Carney Complex

NCT00001452
View directory record Official record
Conditions
Cushing's Syndrome, Pituitary Adenoma, Carney Complex, Primary Pigmented Nodular Adrenocortical Disease, Peutz-Jeghers Syndrome
Interventions
oCRH
Drug
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
3 Years to 70 Years
Enrollment
1,387 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1995
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 16, 2026 · Synced Oct 22, 2026, 7:24 AM EDT
Terminated No phase listed Observational Accepts healthy volunteers

Structural Brain Abnormalities in Children Born Prematurely: New Detection Methods and Clinical-Pathological Correlates

NCT00153855
View directory record Official record
Conditions
Infant, Premature, Brain Injuries
Interventions
Not listed
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
24 Months to 27 Months
Enrollment
50 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2005 – 2006
U.S. locations
3
States / cities
Boston, Massachusetts • Kansas City, Missouri • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Sep 8, 2006 · Synced Oct 22, 2026, 7:24 AM EDT
Completed Not applicable Interventional

High Intensity Exercise and Improving Physical Activity Among People With Neurologic Dysfunction

NCT06945835
View directory record Official record
Conditions
Cerebral Palsy (CP), Stroke, Spinal Cord Injury, Traumatic Brain Injury, Charcot Marie Tooth Disease (CMT), Spina Bifida
Interventions
Participate in high intensity exercise of at least 20 minutes in 60 minutes session of RPE greater than 6.
Behavioral
Lead sponsor
University of Hartford
Other
Eligibility
8 Years to 99 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025
U.S. locations
1
States / cities
West Hartford, Connecticut
Source: ClinicalTrials.gov public record
Updated Nov 3, 2025 · Synced Oct 22, 2026, 7:24 AM EDT
Completed Phase 3 Interventional Results available

Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged 12 to <18 Years)

NCT03500094
View directory record Official record
Conditions
Fabry Disease
Interventions
Migalastat HCl 150 mg
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
12 Years to 17 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
7
States / cities
Tampa, Florida • Atlanta, Georgia • Minneapolis, Minnesota + 4 more
Source: ClinicalTrials.gov public record
Updated Nov 30, 2021 · Synced Oct 22, 2026, 7:24 AM EDT
Recruiting No phase listed Observational

A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding

NCT04252066
View directory record Official record
Conditions
Fabry Disease
Interventions
migalastat
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
Female only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 8, 2025 · Synced Oct 22, 2026, 7:24 AM EDT
Completed Phase 2 Interventional Results available

A Study to Evaluate the Effect of SAGE-718 on Cognitive Function in Participants With Huntington's Disease (HD)

NCT05107128
View directory record Official record
Conditions
Huntington's Disease
Interventions
SAGE-718, Placebo
Drug
Lead sponsor
Supernus Pharmaceuticals, Inc.
Industry
Eligibility
25 Years to 65 Years
Enrollment
189 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
34
States / cities
Little Rock, Arkansas • La Jolla, California • Los Angeles, California + 28 more
Source: ClinicalTrials.gov public record
Updated Sep 15, 2025 · Synced Oct 22, 2026, 7:24 AM EDT