- Conditions
- Hunter Syndrome
- Interventions
- Neurobehavioral testing, Visual and auditory assessments
- Behavioral · Other
- Lead sponsor
- Shire
- Industry
- Eligibility
- 32 Months to 18 Years · Male only
- Enrollment
- 33 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2011
- U.S. locations
- 1
- States / cities
- Chapel Hill, North Carolina
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Showing 1–24
of 2,397
matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
- Conditions
- Obesity, Lifestyle-related Condition, Macrosomia, Fetal
- Interventions
- Lifestyle Intervention
- Behavioral
- Lead sponsor
- Tufts Medical Center
- Other
- Eligibility
- 18 Years to 40 Years · Female only
- Enrollment
- 99 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2017 – 2025
- U.S. locations
- 4
- States / cities
- Baton Rouge, Louisiana • Boston, Massachusetts • Cleveland, Ohio
- Conditions
- Porphyria
- Interventions
- Not listed
- Lead sponsor
- National Center for Research Resources (NCRR)
- NIH
- Eligibility
- 0 Years and older
- Enrollment
- 25 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 1992
- U.S. locations
- 1
- States / cities
- Galveston, Texas
- Conditions
- LPL Deficiency
- Interventions
- alipogene tiparvovec, Prednisolone, Cyclosporins, Mycophenolate mofetil
- Drug
- Lead sponsor
- UniQure Biopharma B.V.
- Industry
- Eligibility
- 18 Years and older
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2020
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania
- Conditions
- Mucopolysaccharidosis Type IIIA
- Interventions
- DNL126
- Drug
- Lead sponsor
- Denali Therapeutics Inc.
- Industry
- Eligibility
- 0 Years to 18 Years
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2028
- U.S. locations
- 4
- States / cities
- Oakland, California • Iowa City, Iowa • Chapel Hill, North Carolina + 1 more
- Conditions
- Stroke (CVA) or TIA, Diabetes, Asthma (Diagnosis), COPD (Chronic Obstructive Pulmonary Disease), Sepsis, Hypertension, Heart Failure, Pneumonia, Urinary Tract Infection (Diagnosis), Chest Pain, Psychiatric Disorder, Sickle Cell Disease (SCD)
- Interventions
- Not listed
- Lead sponsor
- University of Maryland, Baltimore
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 30,486 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2022 – 2025
- U.S. locations
- 1
- States / cities
- Baltimore, Maryland
- Conditions
- Cystic Fibrosis, Inflammatory Bowel Disease, Type 1 Diabetes, Chronic Disease Management
- Interventions
- MD2ME
- Behavioral
- Lead sponsor
- Huang, Jeannie, M.D.
- Individual
- Eligibility
- 14 Years to 22 Years
- Enrollment
- 81 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2012
- U.S. locations
- 1
- States / cities
- La Jolla, California
- Conditions
- Hypoxic-Ischemic Encephalopathy, Hypoglycemia, Neonatal Abstinence Syndrome
- Interventions
- nfant feeding solution
- Device
- Lead sponsor
- Boston Children's Hospital
- Other
- Eligibility
- Up to 6 Months
- Enrollment
- 16 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2017 – 2021
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Familial Hypercholesterolemia - Homozygous
- Interventions
- Inclisiran, Placebo
- Drug
- Lead sponsor
- Novartis Pharmaceuticals
- Industry
- Eligibility
- 12 Years to 17 Years
- Enrollment
- 13 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2024
- U.S. locations
- 1
- States / cities
- Boca Raton, Florida
- Conditions
- Propionic Acidemia
- Interventions
- Not listed
- Lead sponsor
- ModernaTX, Inc.
- Industry
- Eligibility
- 2 Years and older
- Enrollment
- 60 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2026
- U.S. locations
- 6
- States / cities
- Stanford, California • Baltimore, Maryland • Ann Arbor, Michigan + 3 more
- Conditions
- Cystinosis
- Interventions
- Cysteamine Bitartrate Delayed-release Capsules
- Drug
- Lead sponsor
- Amgen
- Industry
- Eligibility
- 1 Year and older
- Enrollment
- 60 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2017
- U.S. locations
- 5
- States / cities
- San Francisco, California • Stanford, California • Atlanta, Georgia + 2 more
- Conditions
- Leukodystrophy, Globoid Cell
- Interventions
- PBKR03
- Biological
- Lead sponsor
- Gemma Biotherapeutics
- Industry
- Eligibility
- 1 Month to 9 Months
- Enrollment
- 24 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2030
- U.S. locations
- 4
- States / cities
- Chicago, Illinois • New York, New York • Philadelphia, Pennsylvania + 1 more
- Conditions
- Phenylketonurias
- Interventions
- sapropterin dihydrochloride
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 8 Years and older
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2005 – 2006
- U.S. locations
- 14
- States / cities
- Los Angeles, California • Oakland, California • Sacramento, California + 11 more
- Conditions
- Urea Cycle Disorders
- Interventions
- HPN-100, Buphenyl (NaPBA)
- Drug
- Lead sponsor
- Amgen
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 46 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2010
- U.S. locations
- 21
- States / cities
- Long Beach, California • Los Angeles, California • Stanford, California + 18 more
- Conditions
- Adrenoleukodystrophy
- Interventions
- MIN-102, Placebos
- Drug
- Lead sponsor
- Minoryx Therapeutics, S.L.
- Industry
- Eligibility
- 18 Years to 65 Years · Male only
- Enrollment
- 105 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2025
- U.S. locations
- 3
- States / cities
- Stanford, California • Baltimore, Maryland • Boston, Massachusetts
- Conditions
- Recurrent Basal Cell Carcinoma
- Interventions
- Patidegib Topical Gel, 2%, Patidegib Topical Gel, Vehicle
- Drug
- Lead sponsor
- Sol-Gel Technologies, Ltd.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 50 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2021
- U.S. locations
- 14
- States / cities
- Apple Valley, California • Colton, California • Fremont, California + 11 more
- Conditions
- Inherited Metabolic Disorders, Undiagnosed Diseases, Batten's Disease, Tay Sachs, Diabetes
- Interventions
- Not listed
- Lead sponsor
- National Human Genome Research Institute (NHGRI)
- NIH
- Eligibility
- 18 Years to 100 Years
- Enrollment
- 2,800 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2022 – 2030
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Achondroplasia
- Interventions
- Infigratinib is provided as a single dose of minitablets for oral administration, Infigratinib is provided as sprinkle capsules for daily oral administration, Infigratinib or placebo comparator is provided as sprinkle capsules for daily oral administration
- Drug
- Lead sponsor
- QED Therapeutics, a BridgeBio company
- Industry
- Eligibility
- 0 Years to 32 Months
- Enrollment
- 77 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2032
- U.S. locations
- 3
- States / cities
- Oakland, California • Baltimore, Maryland • Madison, Wisconsin
- Conditions
- Adrenoleukodystrophy
- Interventions
- glyceryl trierucate/glyceryl trioleate
- Drug
- Lead sponsor
- Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
- Other
- Eligibility
- 18 Months to 8 Years · Male only
- Enrollment
- 126 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1998 – 2014
- U.S. locations
- 2
- States / cities
- Baltimore, Maryland
- Conditions
- Juvenile Neuronal Ceroid Lipofuscinosis
- Interventions
- Mycophenolate mofetil, Liquid Placebo
- Drug
- Lead sponsor
- University of Rochester
- Other
- Eligibility
- 6 Years to 25 Years
- Enrollment
- 19 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2015
- U.S. locations
- 1
- States / cities
- Rochester, New York
- Conditions
- Gout, Hyperuricemia
- Interventions
- Arhalofenate, Febuxostat, Colchicine
- Drug
- Lead sponsor
- Gilead Sciences
- Industry
- Eligibility
- 18 Years to 75 Years
- Enrollment
- 32 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014
- U.S. locations
- 1
- States / cities
- Overland Park, Kansas
- Conditions
- Gout
- Interventions
- Arhalofenate, Allopurinol, Colchicine, Placebo
- Drug
- Lead sponsor
- Gilead Sciences
- Industry
- Eligibility
- 18 Years to 75 Years
- Enrollment
- 100 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2011 – 2012
- U.S. locations
- 16
- States / cities
- Lincoln, California • Los Angeles, California • Palo Alto, California + 13 more
- Conditions
- Homozygous Familial Hypercholesterolemia
- Interventions
- Not listed
- Lead sponsor
- The Rogosin Institute
- Other
- Eligibility
- Not listed
- Enrollment
- 60 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2030
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Recurrent or Tophaceous Gout
- Interventions
- Control, Gout Disease Management Program
- Other
- Lead sponsor
- Kaiser Permanente
- Other
- Eligibility
- 21 Years to 80 Years
- Enrollment
- 104 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2014
- U.S. locations
- 1
- States / cities
- All KPNC Facilities, California