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Showing 1–24 of 54 matching trials from the live ClinicalTrials.gov search.
Conditions
Mucopolysaccharidosis II
Interventions
tividenofusp alfa
Drug
Lead sponsor
Denali Therapeutics Inc.
Industry
Eligibility
Up to 18 Years
Enrollment
99 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
8
States / cities
Oakland, California • Chicago, Illinois • Hackensack, New Jersey + 5 more
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
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
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
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
Completed Not applicable Interventional Accepts healthy volunteers Results available

Study of the Impact of Parental Note Taking on the Effectiveness of Anticipatory Guidance

NCT01014169
Conditions
Health Education, Sudden Infant Death
Interventions
Note taking
Behavioral
Lead sponsor
Boston Medical Center
Other
Eligibility
15 Years and older · Female only
Enrollment
126 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2010
U.S. locations
1
States / cities
Boston, Massachusetts
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
Completed Not applicable Interventional Accepts healthy volunteers

Improving the Self-Efficacy of African American Parents in Infant Supine Sleep

NCT01707173
Conditions
African American Infant Sleep Beliefs and Practices
Interventions
Tailored education, Standard Education
Behavioral
Lead sponsor
University of Michigan
Other
Eligibility
18 Years and older
Enrollment
735 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2016
U.S. locations
1
States / cities
Ann Arbor, Michigan
Conditions
Sudden Unexplained Infant Death, Child Rearing, Literacy
Interventions
Sleep Baby, Safe and Snug, Read Baby, Every Day
Behavioral
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
16 Years and older · Female only
Enrollment
144 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2020
U.S. locations
1
States / cities
Cincinnati, Ohio
Conditions
Breastfeeding, Infant Death, Sudden Infant Death Syndrome Without Mention of Autopsy
Interventions
Safe Sleep, Breastfeeding, Early Brain Development and Parent-Child Relationships
Behavioral
Lead sponsor
University of Virginia
Other
Eligibility
Female only
Enrollment
3,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Boston, Massachusetts
Conditions
Hurler Syndrome (MPS I), Hurler-Scheie Syndrome, Hunter Syndrome (MPS II), Sanfilippo Syndrome (MPS III), Krabbe Disease (Globoid Leukodystrophy), Metachromatic Leukodystrophy (MLD), Adrenoleukodystrophy (ALD and AMN), Sandhoff Disease, Tay Sachs Disease, Pelizaeus Merzbacher (PMD), Niemann-Pick Disease, Alpha-mannosidosis
Interventions
hematopoietic stem cell infusion
Biological
Lead sponsor
Talaris Therapeutics Inc.
Industry
Eligibility
Not listed
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2016
U.S. locations
1
States / cities
Durham, North Carolina
Not yet recruiting Not applicable Interventional Accepts healthy volunteers

Social Media and Risk-reduction Training for Preterm Infant Care Practices

NCT07420686
Conditions
Safe Sleep Education, SUID, Sudden Infant Death Syndrome (SIDS)
Interventions
Safe Sleep NICU Quality Improvement Campaign, Shared Reading NICU Quality Improvement Campaign, Safe Sleep TodaysNICUBaby videos and messages, Shared Reading TodaysNICUBaby videos and messages
Behavioral
Lead sponsor
University of Massachusetts, Worcester
Other
Eligibility
13 Years and older · Female only
Enrollment
1,600 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2027 – 2028
U.S. locations
1
States / cities
Worcester, Massachusetts
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
Not listed Not applicable Interventional Accepts healthy volunteers

Generations in Families Talking Safe Sleep

NCT03186469
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
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
Conditions
Hunter Syndrome
Interventions
Idursulfase
Biological
Lead sponsor
Shire
Industry
Eligibility
5 Years and older · Male only
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2013
U.S. locations
2
States / cities
Oakland, California • Minneapolis, Minnesota
Active, not recruiting No phase listed Observational

Long-term Follow-Up for RGX-121

NCT04597385
Conditions
Mucopolysaccharidosis II
Interventions
Long-term Follow-Up
Other
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
Male only
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2028
U.S. locations
3
States / cities
San Francisco, California • New Brunswich, New Jersey • Philadelphia, Pennsylvania
Completed Not applicable Interventional Accepts healthy volunteers

Improving Infant Sleep Safety With the Electronic Health Record

NCT03662048
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
Conditions
Prevention of Sudden Death
Interventions
CPR Anytime
Other
Lead sponsor
Lehigh Valley Hospital
Other
Eligibility
18 Years and older · Female only
Enrollment
126 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008
U.S. locations
1
States / cities
Allentown, Pennsylvania
Conditions
Mucopolysaccharidosis Type I, Mucopolysaccharidosis Type II, Mucopolysaccharidosis Type VI, Mucopolysaccharidosis Type IV, Mucopolysaccharidosis Type VII
Interventions
Not listed
Lead sponsor
University of Minnesota
Other
Eligibility
6 Years and older
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2019
U.S. locations
3
States / cities
Oakland, California • Minneapolis, Minnesota • New York, New York
Conditions
Sudden Infant Death Syndrome
Interventions
Lifestyle Counseling
Other
Lead sponsor
University of Virginia
Other
Eligibility
18 Years and older · Female only
Enrollment
616 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2004 – 2015
U.S. locations
1
States / cities
Washington D.C., District of Columbia
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
Conditions
Mucopolysaccharidosis II
Interventions
tividenofusp alfa, idursulfase
Drug
Lead sponsor
Denali Therapeutics Inc.
Industry
Eligibility
2 Years to 25 Years
Enrollment
65 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
8
States / cities
Oakland, California • Chicago, Illinois • Hackensack, New Jersey + 5 more
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