- Conditions
- Menkes Disease
- Interventions
- Droxidopa Oral Product, Placebo Control
- Drug · Other
- Lead sponsor
- Stephen G. Kaler
- Other
- Eligibility
- 7 Years to 17 Years
- Enrollment
- 6 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2030
- U.S. locations
- 1
- States / cities
- New York, New York
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 25–48
of 1,603
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Acute Gouty Arthritis
- Interventions
- Anakinra 100 mg, Triamcinolone Acetonide 40 mg, Placebo to Anakinra 100 mg, Placebo to Triamcinolone Acetonide 40 mg
- Drug
- Lead sponsor
- Swedish Orphan Biovitrum
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 165 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2019
- U.S. locations
- 37
- States / cities
- Birmingham, Alabama • Gulf Shores, Alabama • Mobile, Alabama + 32 more
- Conditions
- Autism, Asperger Disorder, PDD-NOS
- Interventions
- Cholesterol
- Other
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- NIH
- Eligibility
- 4 Years to 12 Years
- Enrollment
- 44 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2013
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Primary Hyperoxaluria Type 3
- Interventions
- DCR-PHXC, Sterile Normal Saline (0.9% NaCl)
- Drug
- Lead sponsor
- Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
- Industry
- Eligibility
- 6 Years and older
- Enrollment
- 6 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2021
- U.S. locations
- 3
- States / cities
- Boston, Massachusetts • Rochester, Minnesota • New York, New York
- Conditions
- Fabry Disease
- Interventions
- ST-920
- Biological
- Lead sponsor
- Sangamo Therapeutics
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 36 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2025
- U.S. locations
- 9
- States / cities
- Irvine, California • Tampa, Florida • Atlanta, Georgia + 6 more
- Conditions
- Hermansky-Pudlak Syndrome (HPS), Pulmonary Fibrosis, Oculocutaneous Albinism, Platelet Storage Pool Deficiency, Metabolic Disease
- Interventions
- Losartan, Zileuton, N-Acetylcysteine, Pravastatin, Erythromycin
- Drug
- Lead sponsor
- National Human Genome Research Institute (NHGRI)
- NIH
- Eligibility
- 18 Years to 70 Years
- Enrollment
- 3 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2012
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Gaucher Disease
- Interventions
- Enzyme analysis
- Other
- Lead sponsor
- Brigham and Women's Hospital
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 2012
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- 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
- Apparent Mineralocorticoid Excess Syndrome
- Interventions
- Not listed
- Lead sponsor
- Icahn School of Medicine at Mount Sinai
- Other
- Eligibility
- Not listed
- Enrollment
- 130 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2007 – 2013
- U.S. locations
- 2
- States / cities
- New York, New York • Dallas, Texas
- Conditions
- Niemann-Pick Disease, Type C
- Interventions
- Adrabetadex
- Drug
- Lead sponsor
- Mandos LLC
- Industry
- Eligibility
- 4 Years to 21 Years
- Enrollment
- 66 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2022
- U.S. locations
- 15
- States / cities
- Birmingham, Alabama • Orange, California • San Francisco, California + 12 more
- Conditions
- Arthritis, Gouty
- Interventions
- canakinumab, dexamethasone, placebo matching canakinumab, placebo matching dexamethasone
- Biological · Drug · Other
- Lead sponsor
- Novartis
- Industry
- Eligibility
- 18 Years to 80 Years
- Enrollment
- 6 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2009
- U.S. locations
- 2
- States / cities
- Birmingham, Alabama • New Brunswick, New Jersey
- Conditions
- Hermansky-Pudlak Syndrome (HPS)
- Interventions
- Not listed
- Lead sponsor
- National Human Genome Research Institute (NHGRI)
- NIH
- Eligibility
- 1 Month to 115 Years
- Enrollment
- 600 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 1995
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Dyslipidemias, High Cholesterol, Hypercholesterolemia, Familial Hypercholesterolemia, Lipid Metabolism Disorder, Metabolic Disease, Lipid Metabolism, Inborn Errors, Genetic Disease, Inborn, Hyperlipoproteinemias
- Interventions
- Obicetrapib, Placebo
- Drug
- Lead sponsor
- NewAmsterdam Pharma
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 354 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2024
- U.S. locations
- 20
- States / cities
- Jonesboro, Arkansas • Toluca Lake, California • Sarasota, Florida + 17 more
- Conditions
- Rheumatic Diseases, Gout, Adherence, Medication, Lupus Erythematosus, Systemic
- Interventions
- Cue-Reward Intervention, Cue-Reward Intervention with possible intensification
- Behavioral
- Lead sponsor
- Brigham and Women's Hospital
- Other
- Eligibility
- 18 Years to 85 Years
- Enrollment
- 62 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2022
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Phenylketonuria
- Interventions
- SYNB1934v1, Placebo
- Drug
- Lead sponsor
- Synlogic
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 35 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2024
- U.S. locations
- 14
- States / cities
- Culver City, California • Orange, California • Palo Alto, California + 11 more
- Conditions
- Type 1 Gaucher Disease
- Interventions
- AT2101
- Drug
- Lead sponsor
- Amicus Therapeutics
- Industry
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 1 participant
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009
- U.S. locations
- 1
- States / cities
- Miramar, Florida
- Conditions
- HFpEF - Heart Failure With Preserved Ejection Fraction, HFmrEF, ATTR-CM (Transthyretin Amyloid Cardiomyopathy), Left Ventricular Hypertrophy, Cardiac Amyloidosis
- Interventions
- Not listed
- Lead sponsor
- eMyosound SAS
- Industry
- Eligibility
- 60 Years and older
- Enrollment
- 150 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2025 – 2026
- U.S. locations
- 3
- States / cities
- Boston, Massachusetts
- 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
- Nephropathic Cystinosis
- Interventions
- DFT383
- Genetic
- Lead sponsor
- Novartis Pharmaceuticals
- Industry
- Eligibility
- 2 Years to 5 Years
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2044
- U.S. locations
- 6
- States / cities
- Phoenix, Arizona • San Diego, California • Stanford, California + 3 more
- Conditions
- Late-onset Pompe Disease
- Interventions
- DNL952
- Drug
- Lead sponsor
- Denali Therapeutics Inc.
- Industry
- Eligibility
- 18 Years to 75 Years
- Enrollment
- 32 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2028
- U.S. locations
- 4
- States / cities
- Irvine, California • St Louis, Missouri • Durham, North Carolina + 1 more
- Conditions
- Hyperuricemia, Gout, Chronic Kidney Diseases
- Interventions
- ALLN-346, Placebo
- Drug
- Lead sponsor
- Allena Pharmaceuticals
- Industry
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 16 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2022
- U.S. locations
- 1
- States / cities
- Tempe, Arizona
- Conditions
- Sickle Cell Disease
- Interventions
- Liver transient elastography (Fibroscan), Ferriscan, Liver biopsy in iron overloaded subjects.
- Other · Procedure
- Lead sponsor
- University of Miami
- Other
- Eligibility
- 10 Years to 65 Years
- Enrollment
- 26 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2017
- U.S. locations
- 1
- States / cities
- Miami, Florida
- Conditions
- Transfusional Iron Overload
- Interventions
- ICL670
- Drug
- Lead sponsor
- Novartis Pharmaceuticals
- Industry
- Eligibility
- 2 Years and older
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 2003
- U.S. locations
- 8
- States / cities
- Oakland, California • Orange, California • Boston, Massachusetts + 4 more
- Conditions
- Congenital Adrenal Hyperplasia (CAH), Familial Male-Limited Precocious Puberty (FMPP)
- Interventions
- Not listed
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- NIH
- Eligibility
- 1 Day to 99 Years
- Enrollment
- 3,000 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 2006
- U.S. locations
- 2
- States / cities
- Washington D.C., District of Columbia • Bethesda, Maryland