- 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
Search Results
Search by objective public record fields.
Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results come from ClinicalTrials.gov. When the live registry is unavailable, a clearly labeled stored copy may be shown.
These results were retrieved from the live ClinicalTrials.gov registry. Filters and sort order apply to this result list.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Showing 1–24
of 54
matching trials from the live ClinicalTrials.gov search.
- 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
- 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
- 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
- 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
- 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
- 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
- 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