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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Completed Phase 3 Interventional

Vitamin D for Enhancing the Immune System in Cystic Fibrosis (DISC Study)

NCT01426256
View directory record Official record
Conditions
Cystic Fibrosis, Respiratory Tract Infections
Interventions
Cholecalciferol
Dietary Supplement
Lead sponsor
Emory University
Other
Eligibility
16 Years and older
Enrollment
91 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2017
U.S. locations
5
States / cities
Birmingham, Alabama • Atlanta, Georgia • Iowa City, Iowa + 2 more
Source: ClinicalTrials.gov public record
Updated Jul 12, 2017 · Synced Jun 10, 2026, 7:57 PM 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 Jun 10, 2026, 7:57 PM 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 Jun 10, 2026, 7:57 PM EDT
Recruiting No phase listed Observational

Longitudinal Study of Neurodegenerative Disorders

NCT03333200
View directory record Official record
Conditions
MLD, Krabbe Disease, ALD, MPS I, MPS II, MPS III, Vanishing White Matter Disease, GM3 Gangliosidosis, PKAN, Tay-Sachs Disease, NP Deficiency, Osteopetrosis, Alpha-Mannosidosis, Sandhoff Disease, Niemann-Pick Diseases, MPS IV, Gaucher Disease, GAN, GM1 Gangliosidoses, Morquio Disease, S-Adenosylhomocysteine Hydrolase Deficiency, Batten Disease, Pelizaeus-Merzbacher Disease, Leukodystrophy, Lysosomal Storage Diseases, Purine Nucleoside Phosphorylase Deficiency, Multiple Sulfatase Deficiency Disease
Interventions
Palliative Care, Hematopoetic Stem Cell Transplantation
Other · Biological
Lead sponsor
University of Pittsburgh
Other
Eligibility
Not listed
Enrollment
1,500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2035
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Feb 8, 2026 · Synced Jun 10, 2026, 7:57 PM EDT
Completed No phase listed Observational

Mucopolysaccharidosis Type II Natural History

NCT03529786
View directory record Official record
Conditions
Mucopolysaccharidosis II
Interventions
Not listed
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
Male only
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2022
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 25, 2022 · Synced Jun 10, 2026, 7:57 PM EDT
Withdrawn Phase 1 Interventional

Safety and Efficacy of HMI-203 in ERT-Treated Adults With MPS II

NCT05238324
View directory record Official record
Conditions
Mucopolysaccharidosis II
Interventions
Genetic HMI-203
Biological
Lead sponsor
Homology Medicines, Inc
Industry
Eligibility
18 Years to 45 Years · Male only
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2029
U.S. locations
5
States / cities
Oakland, California • New Haven, Connecticut • Hackensack, New Jersey + 2 more
Source: ClinicalTrials.gov public record
Updated Aug 27, 2023 · Synced Jun 10, 2026, 7:57 PM EDT
Completed Phase 1Phase 2 Interventional

RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome)

NCT04571970
View directory record Official record
Conditions
Mucopolysaccharidosis Type II (MPS II)
Interventions
RGX-121
Genetic
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
5 Years to 17 Years · Male only
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
1
States / cities
Oakland, California
Source: ClinicalTrials.gov public record
Updated Jan 27, 2025 · Synced Jun 10, 2026, 7:57 PM EDT
Recruiting Phase 2 Interventional

Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT

NCT01962415
View directory record Official record
Conditions
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions, Systemic Juvenile Idiopathic Arthritis (sJIA), Juvenile Rheumatoid Arthritis (JRA)
Interventions
Hydroxyurea, Alemtuzumab, Fludarabine, Melphalan, Thiotepa
Drug
Lead sponsor
Paul Szabolcs
Other
Eligibility
2 Months to 55 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2027
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Dec 14, 2025 · Synced Jun 10, 2026, 7:57 PM EDT
Recruiting No phase listed Observational

Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC

NCT04528355
View directory record Official record
Conditions
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions
Interventions
data collection
Drug
Lead sponsor
Paul Szabolcs
Other
Eligibility
2 Months to 60 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2028
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jan 12, 2026 · Synced Jun 10, 2026, 7:57 PM 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 Jun 10, 2026, 7:57 PM EDT
Not yet recruiting Phase 2 Interventional

Single-dose Prophylactic INdomethacin in Extremely Preterm Infants

NCT06572917
View directory record Official record
Conditions
Extreme Prematurity, Intraventricular Hemorrhage, Morbidity;Newborn
Interventions
Indomethacin, Placebo
Drug
Lead sponsor
University of British Columbia
Other
Eligibility
0 Hours to 12 Hours
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2031
U.S. locations
5
States / cities
Roseville, California • Sacramento, California • Pittsburgh, Pennsylvania + 1 more
Source: ClinicalTrials.gov public record
Updated Sep 2, 2025 · Synced Jun 10, 2026, 7:57 PM EDT
Completed No phase listed Observational Results available

Cerebral Oxygenation and Autoregulation in Preterm Infants

NCT02147769
View directory record Official record
Conditions
Intraventricular Hemorrhage of Prematurity, Complications of Prematurity
Interventions
NIRS monitoring
Device
Lead sponsor
Stanford University
Other
Eligibility
Up to 24 Hours
Enrollment
111 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
7
States / cities
Birmingham, Alabama • Palo Alto, California • San Jose, California + 4 more
Source: ClinicalTrials.gov public record
Updated Apr 6, 2020 · Synced Jun 10, 2026, 7:57 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Improving Infant Sleep Safety With the Electronic Health Record

NCT03662048
View directory record Official record
Conditions
SIDS, Infant Development
Interventions
Safe
Behavioral
Lead sponsor
Milton S. Hershey Medical Center
Other
Eligibility
Up to 14 Days
Enrollment
130 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2019
U.S. locations
1
States / cities
Hershey, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 10, 2019 · Synced Jun 10, 2026, 7:57 PM EDT
Completed No phase listed Observational Results available

Carotid Structure and Function in MPS Syndromes: A Multicenter Study of the Lysosomal Disease Network

NCT01586871
View directory record Official record
Conditions
Mucopolysaccharidoses
Interventions
Not listed
Lead sponsor
University of Minnesota
Other
Eligibility
3 Years to 18 Years
Enrollment
1,147 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2017
U.S. locations
2
States / cities
Orange, California • Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Feb 23, 2021 · Synced Jun 10, 2026, 7:57 PM EDT
Active, not recruiting Phase 3 Interventional

RGX-121-3102 Gene Therapy in Participants With MPS II (Hunter Syndrome)

NCT07236606
View directory record Official record
Conditions
MPS II, Hunter Syndrome (MPS II)
Interventions
RGX-121-3102
Genetic
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
4 Months to 5 Years · Male only
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2031
U.S. locations
1
States / cities
New Brunswick, New Jersey
Source: ClinicalTrials.gov public record
Updated Jun 3, 2026 · Synced Jun 10, 2026, 7:57 PM EDT
Completed No phase listed Observational

A Study of Potential Treatment-Responsive Biomarkers and Clinical Outcomes in Hunter Syndrome

NCT04007536
View directory record Official record
Conditions
Mucopolysaccharidosis II
Interventions
No Intervention
Other
Lead sponsor
Denali Therapeutics Inc.
Industry
Eligibility
Up to 30 Years
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2024
U.S. locations
3
States / cities
Oakland, California • Chapel Hill, North Carolina • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 9, 2024 · Synced Jun 10, 2026, 7:57 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

The Happiest Baby, Inc. SNOO Postmarket Surveillance Study

NCT06361303
View directory record Official record
Conditions
Infant Death, Serious Injury
Interventions
SNOO Bassinet
Device
Lead sponsor
Happiest Baby, Inc.
Industry
Eligibility
18 Years and older
Enrollment
1,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2026
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Sep 4, 2025 · Synced Jun 10, 2026, 7:57 PM EDT
Completed Not applicable Interventional

Enhancing Safe Sleep Practices of Urban Low-Income Mothers

NCT03070639
View directory record Official record
Conditions
Sudden Infant Death Syndrome
Interventions
Safe Sleep Intervention, Scald Prevention Intervention
Behavioral
Lead sponsor
Johns Hopkins Bloomberg School of Public Health
Other
Eligibility
18 Years and older
Enrollment
214 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 21, 2017 · Synced Jun 10, 2026, 7:57 PM EDT
Not listed Not applicable Interventional Accepts healthy volunteers

Generations in Families Talking Safe Sleep

NCT03186469
View directory record Official record
Conditions
Sudden Infant Death
Interventions
Safety Baby shower, Standard of Care
Behavioral
Lead sponsor
Arkansas Children's Hospital Research Institute
Other
Eligibility
13 Years to 19 Years · Female only
Enrollment
146 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2020
U.S. locations
1
States / cities
Little Rock, Arkansas
Source: ClinicalTrials.gov public record
Updated Jul 17, 2019 · Synced Jun 10, 2026, 7:57 PM EDT
Completed Not applicable Interventional Results available

MOMSonLINE2 ; A Pilot Study Testing Recruitment and Retention of Women of Color to an Online Support Group for Bereaved Mothers

NCT04600076
View directory record Official record
Conditions
Stillbirth, Grief, Infant Death
Interventions
BabyCenter site and the community group
Behavioral
Lead sponsor
University of Michigan
Other
Eligibility
18 Years and older · Female only
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
1
States / cities
Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated May 31, 2022 · Synced Jun 10, 2026, 7:57 PM EDT
Recruiting Not applicable Interventional Accepts healthy volunteers

Improving Pediatrician Counseling About Infant Safe Sleep Using the Electronic Medical Record

NCT05503771
View directory record Official record
Conditions
Sudden Infant Death, Sudden Unexplained Infant Death
Interventions
ISA-MI, Standard of Care (SOC)
Behavioral · Other
Lead sponsor
Johns Hopkins University
Other
Eligibility
3 Days to 75 Years
Enrollment
350 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2027
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 8, 2025 · Synced Jun 10, 2026, 7:57 PM EDT
Completed Not applicable Interventional

Social Media and Risk Reduction Teaching-Enhanced Reach

NCT04387552
View directory record Official record
Conditions
Breastfeeding, SUID, SIDS
Interventions
Prenatal Safe Sleep Mobile Health (mHealth) Messaging, Prenatal Breastfeeding Mobile Health (mHealth) Messaging, Postnatal Safe Sleep Mobile Health (mHealth) Messaging, Postnatal Breastfeeding Mobile Health (mHealth) Messaging
Behavioral
Lead sponsor
Boston University
Other
Eligibility
14 Years and older · Female only
Enrollment
2,126 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Apr 16, 2026 · Synced Jun 10, 2026, 7:57 PM EDT
Completed Phase 3 Interventional Accepts healthy volunteers Results available

Impact of Maternal Vitamin A or Beta-Carotene Supplementation on Maternal and Infant Mortality in Bangladesh

NCT00198822
View directory record Official record
Conditions
Vitamin A Deficiency, Maternal Mortality, Infant Mortality
Interventions
Vitamin A or Beta-Carotene Supplements
Dietary Supplement
Lead sponsor
Johns Hopkins Bloomberg School of Public Health
Other
Eligibility
15 Years to 49 Years · Female only
Enrollment
59,666 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2001 – 2008
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 6, 2012 · Synced Jun 10, 2026, 7:57 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Today Not Tomorrow Pregnancy and Infant Support Program (TNT- PISP)

NCT04100577
View directory record Official record
Conditions
Infant Mortality, Infant Death, Racial Bias, Prenatal Stress, Prenatal Care, Maternal Child Health, Health Problems in Pregnancy, Adverse Childhood Experiences, Mental Health, Support Groups, Minority Health, Healthcare Disparities, Maternal-child Health Services, Social Determinants of Health, Trust, Physician-Patient Relations, Quality of Care
Interventions
Today Not Tomorrrow Pregnancy and Infant Support Program
Other
Lead sponsor
University of Wisconsin, Madison
Other
Eligibility
18 Years to 99 Years · Female only
Enrollment
24 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2022
U.S. locations
1
States / cities
Madison, Wisconsin
Source: ClinicalTrials.gov public record
Updated Sep 13, 2022 · Synced Jun 10, 2026, 7:57 PM EDT