Independent directory Public ClinicalTrials.gov records United States
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ClinicalTrials.gov public records Last synced Oct 30, 2026, 10:04 PM EDT

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Showing 865–888 of 2,426 matching trials from the live ClinicalTrials.gov search.
Terminated Phase 2Phase 3 Interventional Results available

Adrabetadex to Treat Niemann-Pick Type C1 (NPC1) Disease

NCT04958642
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
Glycogen Storage Disease
Interventions
Diabetes Sentry
Device
Lead sponsor
University of Florida
Other
Eligibility
Up to 70 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
1
States / cities
Gainesville, Florida
Conditions
Fanconi Anemia
Interventions
metformin HCl
Drug
Lead sponsor
Boston Children's Hospital
Other
Eligibility
6 Years to 35 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
1
States / cities
Boston, Massachusetts
Conditions
Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Diseases, Precancerous/Nonmalignant Condition, Small Intestine Cancer
Interventions
anti-thymocyte globulin, graft-versus-tumor induction therapy, cyclophosphamide, fludarabine phosphate, peripheral blood stem cell transplantation
Biological · Drug · Procedure
Lead sponsor
Case Comprehensive Cancer Center
Other
Eligibility
Up to 70 Years
Enrollment
58 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2011
U.S. locations
1
States / cities
Cleveland, Ohio
Completed Not applicable Interventional Accepts healthy volunteers

Vascular Function in Adolescent, Diabetic Children

NCT00348179
Conditions
Type 1 Diabetes
Interventions
Measurement of vascular function per SphymgnoCor equipment
Procedure
Lead sponsor
Children's Mercy Hospital Kansas City
Other
Eligibility
12 Years to 14 Years
Enrollment
85 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2004 – 2007
U.S. locations
1
States / cities
Kansas City, Missouri
Recruiting No phase listed Observational Accepts healthy volunteers

Wearable Technology to Evaluate Hyperglycemia and HRV in DMD

NCT06124196
Conditions
Duchenne Muscular Dystrophy
Interventions
wearable technology
Device
Lead sponsor
Vanderbilt University Medical Center
Other
Eligibility
10 Years and older · Male only
Enrollment
80 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2024 – 2031
U.S. locations
1
States / cities
Nashville, Tennessee
Recruiting No phase listed Observational Accepts healthy volunteers

Friedreich Ataxia Global Clinical Consortium UNIFIED Natural History Study

NCT06016946
Conditions
Friedreich Ataxia
Interventions
Not listed
Lead sponsor
Friedreich's Ataxia Research Alliance
Other
Eligibility
Not listed
Enrollment
3,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2048
U.S. locations
9
States / cities
Los Angeles, California • Denver, Colorado • Gainesville, Florida + 6 more
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
Cardiac Amyloidosis, AL Amyloidosis, ATTR Amyloidosis
Interventions
[18F]florbetaben
Drug
Lead sponsor
Lantheus Germany GmbH
Industry
Eligibility
18 Years and older
Enrollment
244 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
3
States / cities
Kansas City, Kansas • Philadelphia, Pennsylvania • Pittsburgh, Pennsylvania
Enrolling by invitation No phase listed Observational Accepts healthy volunteers

Genetic Studies of Lysosomal Storage Disorders

NCT00001215
Conditions
Lysosomal Storage Disorders, Gaucher Disease, Parkinson Disease
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
1 Week to 110 Years
Enrollment
1,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 1995
U.S. locations
1
States / cities
Bethesda, Maryland
Conditions
Gastrointestinal Stromal Tumors, Small-cell Lung Cancer, Adenoid Cystic Carcinoma, Uveal Melanoma, Neuroendocrine Tumors, Chromophobe Renal Cell Carcinoma, Clear Cell Renal Cell Carcinoma
Interventions
NN3201
Drug
Lead sponsor
Novelty Nobility, Inc.
Industry
Eligibility
18 Years and older
Enrollment
67 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
5
States / cities
Ann Arbor, Michigan • Cleveland, Ohio • Portland, Oregon + 2 more
Conditions
Pompe Disease (Late-onset)
Interventions
Cipaglucosidase Alfa, Miglustat, Alglucosidase Alfa, Placebo
Biological · Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
125 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
25
States / cities
Phoenix, Arizona • Little Rock, Arkansas • Irvine, California + 21 more
Completed Not applicable Interventional Accepts healthy volunteers

Resistance Exercise in Barth Syndrome

NCT01629459
Conditions
Barth Syndrome
Interventions
Resistance exercise and protein supplementation
Behavioral
Lead sponsor
Washington University School of Medicine
Other
Eligibility
15 Years to 35 Years · Male only
Enrollment
12 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2019
U.S. locations
1
States / cities
St Louis, Missouri
Terminated Phase 2 Interventional Results available

N-Acetyl-L-Leucine for Ataxia-Telangiectasia (A-T)

NCT03759678
Conditions
Ataxia Telangiectasia
Interventions
IB1001
Drug
Lead sponsor
IntraBio Inc
Industry
Eligibility
6 Years and older
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2025
U.S. locations
1
States / cities
Los Angeles, California
Conditions
Friedreich Ataxia
Interventions
Rosuvastatin
Drug
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
18 Years to 65 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Recruiting No phase listed Observational

Registry of Older Patients With Cancer

NCT01137825
Conditions
Chronic Myeloproliferative Disorders, Cognitive/Functional Effects, Leukemia, Lymphoma, Lymphoproliferative Disorder, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms, Unspecified Adult Solid Tumor, Protocol Specific
Interventions
Not listed
Lead sponsor
UNC Lineberger Comprehensive Cancer Center
Other
Eligibility
65 Years and older
Enrollment
3,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2030
U.S. locations
7
States / cities
Boone, North Carolina • Chapel Hill, North Carolina • Greenville, North Carolina + 4 more
Conditions
Urea Cycle Disorder, Inborn Errors of Metabolism
Interventions
UCD Anamix Infant
Other
Lead sponsor
Nutricia Research
Industry
Eligibility
Up to 30 Days
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2019
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Completed No phase listed Observational

MRI QSM Imaging for Iron Overload

NCT04631718
Conditions
Hemochromatosis, Iron Overload
Interventions
Magnetic Resonance Imaging (MRI)-based Quantitative Susceptibility Mapping (QSM)
Radiation
Lead sponsor
University of Wisconsin, Madison
Other
Eligibility
5 Years and older
Enrollment
63 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
2
States / cities
Stanford, California • Madison, Wisconsin
Conditions
Cytomegalovirus Retinitis, HIV Infections
Interventions
Sevirumab
Drug
Lead sponsor
Sandoz
Industry
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
2
States / cities
Boston, Massachusetts • Galveston, Texas
Conditions
Gout
Interventions
RDEA594, Placebo, Allopurinol
Drug
Lead sponsor
Ardea Biosciences, Inc.
Industry
Eligibility
18 Years to 80 Years
Enrollment
227 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2016
U.S. locations
25
States / cities
Phoenix, Arizona • La Jolla, California • Los Angeles, California + 22 more
Active, not recruiting Phase 3 Interventional

RZ358 Treatment for Congenital Hyperinsulinism

NCT06208215
Conditions
Congenital Hyperinsulinism
Interventions
RZ358 (5 mg/kg) + SOC (Standard-of-Care) or Placebo + SOC, RZ358 (10 mg/kg) or Placebo + SOC, RZ358 (5-10 mg/kg) + SOC
Drug
Lead sponsor
Rezolute
Other
Eligibility
3 Months to 45 Years
Enrollment
56 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
2
States / cities
Philadelphia, Pennsylvania • Fort Worth, Texas
Conditions
Mucopolysaccharidosis VII, MPS VII, MPS 7, Sly Syndrome
Interventions
No Intervention
Other
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
Not listed
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2032
U.S. locations
7
States / cities
Orange, California • Washington D.C., District of Columbia • Chicago, Illinois + 4 more
Conditions
Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Diseases, Precancerous/Nonmalignant Condition
Interventions
tetradecanoylphorbol acetate
Drug
Lead sponsor
University of Medicine and Dentistry of New Jersey
Other
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2004
U.S. locations
1
States / cities
New Brunswick, New Jersey
Conditions
Diabetes Mellitus, Type 1
Interventions
Aralast NP, Placebo
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
8 Years to 35 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2014
U.S. locations
15
States / cities
La Jolla, California • Aurora, Colorado • New Haven, Connecticut + 10 more