Independent directory Public ClinicalTrials.gov records United States
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ClinicalTrials.gov public records Last synced Sep 6, 2026, 7:55 AM EDT

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Showing 1–24 of 1,697 matching trials from the live ClinicalTrials.gov search.
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Conditions
Sickle Cell Disease, Sickle Cell Anemia, Sickle Cell Disorders, Hemoglobin S Disease, Sickling Disorder Due to Hemoglobin S
Interventions
HQK-1001
Drug
Lead sponsor
HemaQuest Pharmaceuticals Inc.
Industry
Eligibility
12 Years to 60 Years
Enrollment
52 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2011
U.S. locations
9
States / cities
Oakland, California • Miami, Florida • Augusta, Georgia + 6 more
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
Down Syndrome (DS), Keratoconus, Pellucid Marginal Degeneration, Forme Fruste Keratoconus (FFK)
Interventions
Epi-ON corneal cross-linking (CXL)
Combination Product
Lead sponsor
Woolfson Eye Institute
Other
Eligibility
8 Years and older
Enrollment
225 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
12
States / cities
Atlanta, Georgia • Canton, Georgia • Cumming, Georgia + 8 more
Active, not recruiting Phase 1Phase 2 Interventional

NS-050/NCNP-03 in Boys With DMD (Meteor50)

NCT06053814
Conditions
Duchenne Muscular Dystrophy
Interventions
NS-050/NCNP-03, Placebo
Drug
Lead sponsor
NS Pharma, Inc.
Industry
Eligibility
4 Years to 15 Years · Male only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2028
U.S. locations
6
States / cities
Los Angeles, California • Aurora, Colorado • Chicago, Illinois + 3 more
Conditions
Retinitis Pigmentosa
Interventions
Not listed
Lead sponsor
National Eye Institute (NEI)
NIH
Eligibility
Not listed
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1993 – 2002
U.S. locations
1
States / cities
Bethesda, Maryland
Conditions
Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Interventions
VX-407, Placebo, Midazolam
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
18 Years to 55 Years
Enrollment
159 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2024 – 2025
U.S. locations
1
States / cities
Lenexa, Kansas
Completed Early Phase 1 Interventional

Burosumab for CSHS

NCT03993821
Conditions
Cutaneous Skeletal Hypophosphatemia Syndrome (CSHS), Epidermal Nevus Syndrome
Interventions
Burosumab
Drug
Lead sponsor
Laura Tosi
Other
Eligibility
Not listed
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2026
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Conditions
Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms, Nonmalignant Neoplasm
Interventions
trivalent influenza vaccine, Immunoenzyme technique, Laboratory biomarker analysis
Biological · Other
Lead sponsor
City of Hope Medical Center
Other
Eligibility
18 Years to 120 Years
Enrollment
459 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2007 – 2013
U.S. locations
2
States / cities
Duarte, California • Pasadena, California
Conditions
RAI1 Gene 17P11.2 Deletion+Duplication
Interventions
Electroencephalography/Polysomnography (EEG/PSG), Skin Biopsy, Blood draw
Diagnostic Test · Procedure
Lead sponsor
Baylor College of Medicine
Other
Eligibility
1 Month to 80 Years
Enrollment
90 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2024 – 2027
U.S. locations
1
States / cities
Houston, Texas
Conditions
Down Syndrome, Cognitive Dysfunction
Interventions
Aricept (donepezil hydrochloride), Placebo
Drug
Lead sponsor
Eisai Inc.
Industry
Eligibility
11 Years to 17 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008
U.S. locations
2
States / cities
San Antonio, Texas • Herndon, Virginia
Conditions
Tumors Characterized by Genetic Abnormalities of ALK
Interventions
LDK378
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
304 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2016
U.S. locations
6
States / cities
Aurora, Colorado • Boston, Massachusetts • New York, New York + 3 more
Conditions
Huntington's Disease
Interventions
Not listed
Lead sponsor
University College, London
Other
Eligibility
11 Years to 75 Years
Enrollment
2,500 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 2017
U.S. locations
6
States / cities
Englewood, Colorado • Washington D.C., District of Columbia • Baltimore, Maryland + 3 more
Conditions
Polycystic Kidney, Autosomal Dominant
Interventions
Tesevatinib
Drug
Lead sponsor
Kadmon, a Sanofi Company
Industry
Eligibility
18 Years to 62 Years
Enrollment
69 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2019
U.S. locations
11
States / cities
Los Angeles, California • Denver, Colorado • Kansas City, Kansas + 8 more
Conditions
Amyloid Plaque, Beta-Amyloid, DSAD, Prodromal Alzheimer's Disease, Alzheimer's Disease
Interventions
Placebo (Study Part 1a), ACI-24.060 at Dose A in Study Part 1a, ACI-24.060 at Dose B in Study Part 1a, ACI-24.060 at Dose C in Study Part 1a, ACI-24.060 with an additional adjuvant at Dose D in Study Part 1b, Placebo (Study Part 2), ACI-24.060 at Dose A in Study Part 2, ACI-24.060 at Dose B in Study Part 2, ACI-24.060 at Dose C in Study Part 2, Placebo (Study Part 1b), ACI-24.060 with an additional adjuvant at Dose E in Study Part 1b
Biological
Lead sponsor
AC Immune SA
Industry
Eligibility
35 Years to 85 Years
Enrollment
304 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2029
U.S. locations
13
States / cities
Phoenix, Arizona • Lady Lake, Florida • Orlando, Florida + 9 more
Recruiting No phase listed Observational

Complex Adult Deformity Surgery (CADS)

NCT04194138
Conditions
Adult Spinal Deformity, Scoliosis, Kyphosis, Sagittal Imbalance
Interventions
Index or spine revision surgery for complex adult spinal deformity
Procedure
Lead sponsor
International Spine Study Group Foundation
Other
Eligibility
18 Years and older
Enrollment
1,500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2032
U.S. locations
17
States / cities
La Jolla, California • Sacramento, California • San Francisco, California + 11 more
Conditions
Usher Syndrome, Cochlear Implantation
Interventions
Experimental manipulation of stimulation parameters
Other
Lead sponsor
Ohio State University
Other
Eligibility
1 Year to 85 Years
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
4
States / cities
Boston, Massachusetts • Columbus, Ohio • Nashville, Tennessee
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
Patent Ductus Arteriosus
Interventions
feeding, fasting
Other
Lead sponsor
University of California, San Francisco
Other
Eligibility
23 Weeks to 33 Weeks
Enrollment
177 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2012
U.S. locations
14
States / cities
San Francisco, California • San Jose, California • Chicago, Illinois + 11 more
Completed Not applicable Interventional

Cognitive Remediation in 22q11DS

NCT01781923
Conditions
22q11.2 Deletion Syndrome, Velo-Cardio-Facial Syndrome
Interventions
Cognitive remediation program, Small group social skills training
Behavioral
Lead sponsor
Duke University
Other
Eligibility
11 Years to 17 Years
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2015
U.S. locations
1
States / cities
Durham, North Carolina
Conditions
Cytomegalovirus Infections, HIV Infections
Interventions
Zidovudine, Didanosine, Ganciclovir
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
18 Years and older
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
3
States / cities
Irvine, California • Washington D.C., District of Columbia • Galveston, Texas
Completed Not applicable Interventional Accepts healthy volunteers

Phenotypic Specific Communication Intervention for Children With Down Syndrome

NCT02158390
Conditions
Language Impairment, Down Syndrome
Interventions
Jasper-EMT with words and AAC
Behavioral
Lead sponsor
Vanderbilt University
Other
Eligibility
30 Months to 54 Months
Enrollment
78 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2018
U.S. locations
2
States / cities
Los Angeles, California • Nashville, Tennessee
Conditions
Stage II Multiple Myeloma, Stage III Multiple Myeloma, Refractory Plasma Cell Neoplasm
Interventions
autologous tumor cell vaccine, keyhole limpet hemocyanin, melphalan, sargramostim
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Timeline
1995 – 2007
U.S. locations
3
States / cities
Little Rock, Arkansas • Bethesda, Maryland
Conditions
Congenital Hyperinsulinism
Interventions
RZ358 Sequential Group Cohort 1, RZ358 Sequential Group Cohort 2, RZ358 Sequential Group Cohort 3, RZ358 Sequential Group Cohort 4
Drug
Lead sponsor
Rezolute
Other
Eligibility
2 Years to 45 Years
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
2
States / cities
Philadelphia, Pennsylvania • Fort Worth, Texas
Conditions
Facial Angiofibroma
Interventions
Rapamune
Drug
Lead sponsor
Le Bonheur Children's Hospital
Other
Eligibility
3 Years to 45 Years
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
1
States / cities
Memphis, Tennessee