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Showing 1–24 of 88 matching trials from the live ClinicalTrials.gov search.
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Terminated Phase 3 Interventional

ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases

NCT00654433
View directory record Official record
Conditions
Inherited Metabolic Diseases, Lysosomal Storage Disorders, Peroxisomal Storage Diseases, Inborn Errors of Metabolism, Mucopolysaccharidosis
Interventions
ALD-101
Biological
Lead sponsor
Aldagen
Industry
Eligibility
Up to 16 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2011
U.S. locations
3
States / cities
Los Angeles, California • New York, New York • Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Jul 7, 2014 · Synced Jul 21, 2026, 9:58 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II

NCT03041324
View directory record Official record
Conditions
Mucopolysaccharidosis II, MPS II
Interventions
SB-913
Biological
Lead sponsor
Sangamo Therapeutics
Industry
Eligibility
5 Years to 65 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
5
States / cities
Oakland, California • Chicago, Illinois • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Oct 24, 2022 · Synced Jul 21, 2026, 9:58 AM EDT
Terminated No phase listed Observational

Mucopolysaccharidosis (MPS) I, II, and VI Screening in a High-Risk Population With Previous Surgical Repair or Presence of Inguinal and/or Umbilical Hernia in Combination With Pediatric ENT Surgery (The HATT Project)

NCT02095015
View directory record Official record
Conditions
Mucopolysaccharidosis (MPS)
Interventions
Not listed
Lead sponsor
Shire
Industry
Eligibility
Up to 7 Years · Male only
Enrollment
159 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
7
States / cities
Los Angeles, California • Jacksonville, Florida • Minneapolis, Minnesota + 4 more
Source: ClinicalTrials.gov public record
Updated Mar 16, 2021 · Synced Jul 21, 2026, 9:58 AM EDT
Active, not recruiting Phase 2Phase 3 Interventional

CAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome)

NCT03566043
View directory record Official record
Conditions
Mucopolysaccharidosis Type II (MPS II)
Interventions
RGX-121
Genetic
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
4 Months to 5 Years · Male only
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2025
U.S. locations
4
States / cities
Oakland, California • New Brunswick, New Jersey • Philadelphia, Pennsylvania + 1 more
Source: ClinicalTrials.gov public record
Updated Jan 27, 2025 · Synced Jul 21, 2026, 9:58 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Gene Therapy for ACM Due to a PKP2 Pathogenic Variant

NCT06109181
View directory record Official record
Conditions
Arrhythmogenic Cardiomyopathy, PKP2-ACM, PKP2-ARVC
Interventions
LX2020
Genetic
Lead sponsor
Lexeo Therapeutics
Industry
Eligibility
18 Years to 65 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
5
States / cities
Stanford, California • Baltimore, Maryland • Ann Arbor, Michigan + 2 more
Source: ClinicalTrials.gov public record
Updated Nov 11, 2025 · Synced Jul 21, 2026, 9:58 AM EDT
Terminated Phase 2Phase 3 Interventional Results available

Clinical Trial of Growth Hormone in MPS I, II, and VI

NCT00748969
View directory record Official record
Conditions
Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis VI
Interventions
Somatropin (DNA origin)
Drug
Lead sponsor
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
Other
Eligibility
5 Years to 17 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2013
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Sep 4, 2018 · Synced Jul 21, 2026, 9:58 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Mayo AVC Registry and Biobank

NCT03049254
View directory record Official record
Conditions
Arrhythmogenic Right Ventricular Cardiomyopathy, Cardiomyopathies, Heart Diseases, Cardiovascular Diseases, Sudden Cardiac Arrest, Sudden Cardiac Death, Arrhythmogenic Right Ventricular Dysplasia, Arrhythmogenic Ventricular Cardiomyopathy, Familial Dilated Cardiomyopathy, Cardiovascular Abnormalities, Sarcoidosis, Cardiac Arrhythmia, Cardiac Sarcoidosis, Myocarditis, Inflammatory Cardiomyopathy, Ventricular Tachycardia, Right Ventricular Outflow Tract Ventricular Tachycardia
Interventions
Not listed
Lead sponsor
Mayo Clinic
Other
Eligibility
Not listed
Enrollment
1,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2027
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Apr 26, 2026 · Synced Jul 21, 2026, 9:58 AM EDT
Terminated Phase 2 Interventional Results available

Fetal Tracheal Balloon Study in Diaphragmatic Hernia

NCT00966823
View directory record Official record
Conditions
Diaphragmatic Hernia, Lung Disease
Interventions
Fetal tracheal obstruction with detachable balloon (device)
Device
Lead sponsor
Rhode Island Hospital
Other
Eligibility
18 Years and older · Female only
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2015
U.S. locations
1
States / cities
Providence, Rhode Island
Source: ClinicalTrials.gov public record
Updated May 29, 2017 · Synced Jul 21, 2026, 9:58 AM EDT
Active, not recruiting No phase listed Observational

Longitudinal Evaluation of Active-Duty Personnel with Accessory Pathways (LEAD-AP)

NCT06798961
View directory record Official record
Conditions
Cardiac Death, Cardiac Death, Sudden, Wolf Parkinson White Syndrome
Interventions
Ablation, Standard of care without ablation
Procedure
Lead sponsor
United States Naval Medical Center, San Diego
Federal
Eligibility
18 Years to 40 Years
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2025
U.S. locations
1
States / cities
San Diego, California
Source: ClinicalTrials.gov public record
Updated Mar 13, 2025 · Synced Jul 21, 2026, 9:58 AM EDT
No Longer Available No phase listed Expanded access

EPI-743 for Mitochondrial Respiratory Chain Diseases

NCT01370447
View directory record Official record
Conditions
Mitochondrial Diseases
Interventions
EPI-743
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
1 Year and older
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
16
States / cities
Los Angeles, California • Orange, California • Palo Alto, California + 12 more
Source: ClinicalTrials.gov public record
Updated Jan 22, 2024 · Synced Jul 21, 2026, 9:58 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Inflammatory Cytokines Associated With Perinatal Brain Injury

NCT01035697
View directory record Official record
Conditions
Infant, Newborn, Infant, Low Birth Weight, Infant, Small for Gestational Age, Infant, Premature, Cerebral Palsy
Interventions
Not listed
Lead sponsor
NICHD Neonatal Research Network
Network
Eligibility
Up to 72 Hours
Enrollment
1,067 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1999 – 2004
U.S. locations
18
States / cities
Birmingham, Alabama • Palo Alto, California • San Diego, California + 14 more
Source: ClinicalTrials.gov public record
Updated Mar 21, 2019 · Synced Jul 21, 2026, 9:58 AM EDT
Completed Phase 2Phase 3 Interventional Results available

Study of Intrathecal Idursulfase-IT Administered in Conjunction With Elaprase® in Pediatric Patients With Hunter Syndrome and Early Cognitive Impairment

NCT02055118
View directory record Official record
Conditions
Hunter Syndrome
Interventions
idursulfase-IT, No IT treatment
Biological · Other
Lead sponsor
Shire
Industry
Eligibility
Up to 18 Years · Male only
Enrollment
58 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
3
States / cities
Oakland, California • Chicago, Illinois • Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Jun 10, 2021 · Synced Jul 21, 2026, 9:58 AM EDT
Not listed No phase listed Observational

South Asian Arrhythmogenic Cardiomyopathy Registry

NCT04895540
View directory record Official record
Conditions
Arrhythmogenic Right Ventricular Cardiomyopathy, Arrhythmogenic Right Ventricular Dysplasia, Arrhythmogenic Ventricular Cardiomyopathy, Arrhythmogenic Cardiomyopathy, Ventricular Tachycardia, Sudden Cardiac Death
Interventions
ECG, Echocardiography, Cardiac MRI, Ambulatory ECG recording, Cardiac EP study, All study subjects will undergo sample collection for genetic testing.
Diagnostic Test · Genetic
Lead sponsor
Sheri Kashmir Institute of Medical Sciences
Other
Eligibility
Not listed
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 1, 2021 · Synced Jul 21, 2026, 9:58 AM EDT
Completed Phase 2Phase 3 Interventional Results available

Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Idursulfase

NCT00630747
View directory record Official record
Conditions
Hunter Syndrome, Mucopolysaccharidosis II (MPS II)
Interventions
Idursulfase
Biological
Lead sponsor
Shire
Industry
Eligibility
5 Years and older · Male only
Enrollment
94 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2008
U.S. locations
16
States / cities
Phoenix, Arizona • Oakland, California • Denver, Colorado + 13 more
Source: ClinicalTrials.gov public record
Updated Jun 9, 2021 · Synced Jul 21, 2026, 9:58 AM EDT
Completed Phase 1Phase 2 Interventional Results available

A Safety and Dose Ranging Study of Idursulfase (Intrathecal) Administration Via an Intrathecal Drug Delivery Device in Pediatric Patients With Hunter Syndrome Who Have Central Nervous System Involvement and Are Receiving Treatment With Elaprase®

NCT00920647
View directory record Official record
Conditions
Hunter Syndrome
Interventions
Control, Idursulfase IT (1 mg), Idursulfase IT (10 mg), Idursulfase IT (30 mg)
Other · Drug
Lead sponsor
Shire
Industry
Eligibility
3 Years to 18 Years · Male only
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2012
U.S. locations
1
States / cities
Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Jun 27, 2021 · Synced Jul 21, 2026, 9:58 AM EDT
Completed Phase 1Phase 2 Interventional Results available

An Extension Study of HGT-HIT-045 Evaluating Long-Term Safety and Clinical Outcomes of Idursulfase-IT in Conjunction With Elaprase in Pediatric Participants With Hunter Syndrome and Cognitive Impairment

NCT01506141
View directory record Official record
Conditions
Hunter Syndrome
Interventions
Idursulfase-IT, Elaprase
Drug
Lead sponsor
Takeda
Industry
Eligibility
3 Years to 18 Years · Male only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2024
U.S. locations
7
States / cities
Chicago, Illinois • Chapel Hill, North Carolina • Portland, Oregon + 4 more
Source: ClinicalTrials.gov public record
Updated Aug 5, 2025 · Synced Jul 21, 2026, 9:58 AM EDT
Recruiting Phase 3 Interventional

InfasurfAero™ Versus Sham Treatment in Preterm Newborns With RDS

NCT05960929
View directory record Official record
Conditions
Respiratory Distress Syndrome (Neonatal), Intubation Complication, Death; Neonatal
Interventions
Infasurf Aero™
Combination Product
Lead sponsor
ONY
Industry
Eligibility
29 Weeks to 36 Weeks
Enrollment
220 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
10
States / cities
Phoenix, Arizona • San Diego, California • Jacksonville, Florida + 6 more
Source: ClinicalTrials.gov public record
Updated Mar 29, 2025 · Synced Jul 21, 2026, 9:58 AM EDT
Completed No phase listed Observational

Lysosomal Storage Disease: Health, Development, and Functional Outcome Surveillance in Preschool Children

NCT01938014
View directory record Official record
Conditions
Mucopolysaccharidosis Type I (MPS I), Mucopolysaccharidosis Type II (MPS II), Mucopolysaccharidosis Type III (MPS III), Mucopolysaccharidosis Type VI (MPS VI), Krabbe Disease
Interventions
Not listed
Lead sponsor
University of Chicago
Other
Eligibility
1 Day to 18 Years
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2016
U.S. locations
3
States / cities
Chicago, Illinois • Minneapolis, Minnesota • Buffalo, New York
Source: ClinicalTrials.gov public record
Updated Oct 3, 2019 · Synced Jul 21, 2026, 9:58 AM EDT
Recruiting Not applicable Interventional

Placental Imaging Techniques

NCT06861309
View directory record Official record
Conditions
Fetal Growth Restriction (FGR), Placental Insufficiency, Preeclampsia, Still Births, Pregnancy, Pregnancy Complications, Pregnancy Outcomes, Ultrasound
Interventions
ultrafast power Doppler imaging (uPDI) / quantitative ultrasound (QUS)
Device
Lead sponsor
Carilion Clinic
Other
Eligibility
18 Years to 45 Years · Female only
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
1
States / cities
Roanoke, Virginia
Source: ClinicalTrials.gov public record
Updated May 21, 2026 · Synced Jul 21, 2026, 9:58 AM EDT
Recruiting Phase 1 Interventional

A Phase 1, Dose Escalation Trial of RP-A601 in Subjects With PKP2 Variant-Mediated Arrhythmogenic Cardiomyopathy (PKP2-ACM)

NCT05885412
View directory record Official record
Conditions
PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM)
Interventions
RP-A601
Genetic
Lead sponsor
Rocket Pharmaceuticals Inc.
Industry
Eligibility
18 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2029
U.S. locations
3
States / cities
La Jolla, California • Durham, North Carolina • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 15, 2026 · Synced Jul 21, 2026, 9:58 AM EDT
Terminated Not applicable Interventional Results available

Pediatric Lead Extractability and Survival Evaluation (PLEASE)

NCT00335036
View directory record Official record
Conditions
Ventricular Tachycardia, Ventricular Fibrillation, Cardiomyopathy, Long QT Syndrome, Congenital Heart Disease
Interventions
ICD lead implant, Thin leads
Device
Lead sponsor
Charles Berul
Other
Eligibility
1 Year to 55 Years
Enrollment
748 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2009
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 18, 2026 · Synced Jul 21, 2026, 9:58 AM EDT
Completed Phase 4 Interventional Results available

Pharmacokinetics of Retapamulin in Pediatric Subjects With Uncomplicated Skin Infections.

NCT00555061
View directory record Official record
Conditions
Skin Infections, Bacterial
Interventions
Retapamulin Ointment, 1%
Drug
Lead sponsor
GlaxoSmithKline
Industry
Eligibility
2 Months to 24 Months
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2008
U.S. locations
6
States / cities
Tuscaloosa, Alabama • Bentonville, Arkansas • Orange, California + 3 more
Source: ClinicalTrials.gov public record
Updated Dec 27, 2016 · Synced Jul 21, 2026, 9:58 AM EDT
Recruiting Phase 1 Interventional

Clinical Trial Evaluating the Safety, Tolerability and Preliminary Efficacy of BNT116 Alone and in Combinations in Patients With Advanced Non-small Cell Lung Cancer

NCT05142189
View directory record Official record
Conditions
Non-Small Cell Lung Cancer
Interventions
BNT116, Cemiplimab, Docetaxel, Carboplatin, Paclitaxel, BNT316, anti-B7-H3 antibody conjugated to topoisomerase I inhibitor, anti-HER3 antibody conjugated to topoisomerase I inhibitor, Bispecific antibody for PD-L1 and VEGF-A, Osimertinib, ALK-inhibitor or RET-inhibitor
Biological · Drug
Lead sponsor
BioNTech SE
Industry
Eligibility
18 Years and older
Enrollment
320 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2031
U.S. locations
5
States / cities
Lexington, Kentucky • Louisville, Kentucky • Baltimore, Maryland + 2 more
Source: ClinicalTrials.gov public record
Updated Apr 13, 2026 · Synced Jul 21, 2026, 9:58 AM EDT
Completed Not applicable Interventional

Premature Infants Receiving Milking or Delayed Cord Clamping: PREMOD2

NCT03019367
View directory record Official record
Conditions
Intraventricular Haemorrhage Neonatal, Death; Neonatal
Interventions
Umbilical cord milking UCM, Delayed cord clamping DCC
Procedure
Lead sponsor
Sharp HealthCare
Other
Eligibility
23 Weeks to 33 Weeks
Enrollment
1,201 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2022
U.S. locations
15
States / cities
Birmingham, Alabama • Loma Linda, California • Los Angeles, California + 10 more
Source: ClinicalTrials.gov public record
Updated Nov 22, 2022 · Synced Jul 21, 2026, 9:58 AM EDT