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ClinicalTrials.gov public records Last synced Oct 3, 2026, 11:45 AM EDT

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Showing 25–48 of 2,370 matching trials from the live ClinicalTrials.gov search.
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Completed Phase 2 Interventional

Hematopoietic Stem Cell Transplant for Fanconi Anemia

NCT01071239
View directory record Official record
Conditions
Fanconi Anemia
Interventions
CliniMACs device, Busulfan, Fludarabine, Cyclophosphamide, ATG
Device · Drug
Lead sponsor
Medical College of Wisconsin
Other
Eligibility
Not listed
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2016
U.S. locations
1
States / cities
Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated Sep 11, 2019 · Synced Oct 3, 2026, 11:45 AM EDT
Completed Phase 2 Interventional

An Open-Label Clinical Trial of Replagal Enzyme Therapy in Children Ages 7-17 Years With Fabry Disease

NCT00071877
View directory record Official record
Conditions
Fabry Disease
Interventions
Replagal
Drug
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2005
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 3, 2026, 11:45 AM EDT
Completed Phase 3 Interventional

Masitinib in Severe Indolent or Smoldering Systemic Mastocytosis

NCT00814073
View directory record Official record
Conditions
Indolent Systemic Mastocytosis
Interventions
Masitinib, Placebo, Best Supportive Care
Drug · Other
Lead sponsor
AB Science
Industry
Eligibility
18 Years to 75 Years
Enrollment
135 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2015
U.S. locations
2
States / cities
Sacramento, California • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Dec 3, 2019 · Synced Oct 3, 2026, 11:45 AM EDT
Completed No phase listed Observational

A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)

NCT02294877
View directory record Official record
Conditions
Mucopolysaccharidosis IV Type A, Morquio A Syndrome, MPS IVA
Interventions
Vimizim® (elosulfase alfa)
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
Not listed
Enrollment
418 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2024
U.S. locations
26
States / cities
Phoenix, Arizona • Little Rock, Arkansas • Los Angeles, California + 22 more
Source: ClinicalTrials.gov public record
Updated Mar 5, 2024 · Synced Oct 3, 2026, 11:45 AM EDT
Completed No phase listed Observational

Study of Phenotype and Genotype Correlations in Patients With Contiguous Gene Deletion Syndromes

NCT00004351
View directory record Official record
Conditions
Williams Syndrome, Angelman Syndrome, Prader-Willi Syndrome, Shprintzen Syndrome, Smith-Magenis Syndrome, DiGeorge Syndrome, Chromosome Abnormalities
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
0 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1999
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 3, 2026, 11:45 AM EDT
Completed Phase 2 Interventional Results available

Oral Pirfenidone for the Pulmonary Fibrosis of Hermansky-Pudlak Syndrome

NCT00001596
View directory record Official record
Conditions
Albinism, Inborn Errors of Metabolism, Oculocutaneous Albinism, Platelet Storage Pool Deficiency, Pulmonary Fibrosis
Interventions
Pirfenidone, Placebo
Drug
Lead sponsor
William Gahl, M.D.
NIH
Eligibility
18 Years and older
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2016
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 16, 2017 · Synced Oct 3, 2026, 11:45 AM EDT
Completed No phase listed Observational

Drug Use Study of Febuxostat After Recent Changes in the Prescribing Information

NCT04853160
View directory record Official record
Conditions
Gout, Cardiovascular Diseases
Interventions
Not listed
Lead sponsor
Takeda
Industry
Eligibility
21 Years and older
Enrollment
24,046 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
1
States / cities
Cambridge, Massachusetts
Source: ClinicalTrials.gov public record
Updated Apr 27, 2021 · Synced Oct 3, 2026, 11:45 AM EDT
Terminated Early Phase 1 Interventional

Tislelizumab for the Treatment of Recurrent Mismatch Repair Deficient Endometrial Cancer

NCT04906382
View directory record Official record
Conditions
Lynch Syndrome, Recurrent Endometrial Carcinoma, Recurrent Endometrial Clear Cell Adenocarcinoma, Recurrent Uterine Corpus Carcinosarcoma, Mismatch Repair Deficiency, Recurrent Endometrial Cancer, Metastatic Endometrial Cancer
Interventions
Biopsy, Carboplatin, Paclitaxel, Tislelizumab
Procedure · Drug · Biological
Lead sponsor
Floor Backes, MD
Other
Eligibility
18 Years and older · Female only
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Feb 13, 2025 · Synced Oct 3, 2026, 11:45 AM EDT
Recruiting No phase listed Observational

Pompe Pregnancy Sub-Registry

NCT00567073
View directory record Official record
Conditions
Glycogen Storage Disease Type II (GSD-II), Pompe Disease (Late-onset), Glycogenesis 2 Acid Maltase Deficiency
Interventions
Not listed
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Female only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2034
U.S. locations
15
States / cities
Phoenix, Arizona • Jacksonville, Florida • Atlanta, Georgia + 10 more
Source: ClinicalTrials.gov public record
Updated Apr 16, 2026 · Synced Oct 3, 2026, 11:45 AM EDT
Recruiting Not applicable Interventional

Computerized Decision Support for Identification and Management of Familial Hypercholesterolemia

NCT06743659
View directory record Official record
Conditions
Familial Hypercholesterolaemia
Interventions
Alert-based computerized decision support
Device
Lead sponsor
Brigham and Women's Hospital
Other
Eligibility
Not listed
Enrollment
450 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Sep 29, 2026 · Synced Oct 3, 2026, 11:45 AM EDT
Completed Phase 4 Interventional Results available

High Dose or High Dose Frequency Study of Alglucosidase Alfa

NCT00483379
View directory record Official record
Conditions
Pompe Disease, Glycogen Storage Disease Type II (GSD-II), Glycogenesis 2 Acid Maltase Deficiency
Interventions
alglucosidase alfa
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
6 Months and older
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2010
U.S. locations
9
States / cities
Birmingham, Alabama • Stanford, California • Washington D.C., District of Columbia + 6 more
Source: ClinicalTrials.gov public record
Updated Mar 7, 2014 · Synced Oct 3, 2026, 11:45 AM EDT
Completed Phase 1 Interventional

Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI

NCT00048620
View directory record Official record
Conditions
Mucopolysaccharidosis VI
Interventions
N-acetylgalactosamine 4-sulfatase
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
Not listed
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2005
U.S. locations
1
States / cities
Novato, California
Source: ClinicalTrials.gov public record
Updated Nov 3, 2006 · Synced Oct 3, 2026, 11:45 AM EDT
Recruiting Phase 1 Interventional

A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe Disease

NCT07354724
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Aug 17, 2026 · Synced Oct 3, 2026, 11:45 AM EDT
Recruiting Phase 3 Interventional

A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C

NCT05163288
View directory record Official record
Conditions
Niemann-Pick Disease, Type C
Interventions
N-Acetyl-L-Leucine, Placebo
Drug · Other
Lead sponsor
IntraBio Inc
Industry
Eligibility
4 Years and older
Enrollment
53 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2030
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Apr 3, 2026 · Synced Oct 3, 2026, 11:45 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Smith-Lemli-Opitz Syndrome and Cholic Acid

NCT03720990
View directory record Official record
Conditions
Smith-Lemli-Opitz Syndrome
Interventions
Cholic Acid
Drug
Lead sponsor
University of Nebraska
Other
Eligibility
2 Years to 25 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
2
States / cities
Aurora, Colorado • Omaha, Nebraska
Source: ClinicalTrials.gov public record
Updated Apr 30, 2024 · Synced Oct 3, 2026, 11:45 AM EDT
Completed Phase 1 Interventional

A Pilot Study of Vitamin D in Boys With X-linked Adrenoleukodystrophy

NCT02595489
View directory record Official record
Conditions
X-linked Adrenoleukodystrophy
Interventions
vitamin D3
Dietary Supplement
Lead sponsor
Stanford University
Other
Eligibility
18 Months to 25 Years · Male only
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2020
U.S. locations
1
States / cities
Palo Alto, California
Source: ClinicalTrials.gov public record
Updated Jun 9, 2022 · Synced Oct 3, 2026, 11:45 AM EDT
Completed No phase listed Observational

AMPED Outcomes Registry of Post-ED Pain Management

NCT01626235
View directory record Official record
Conditions
Soft Tissue Injuries, Gouty Arthritis, Fractures, Ureteral Colic, Dysmenorrhea
Interventions
Not listed
Lead sponsor
Radnor Registry Research, Inc
Industry
Eligibility
18 Years and older
Enrollment
843 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2014
U.S. locations
11
States / cities
Birmingham, Alabama • New Orleans, Louisiana • Jackson, Mississippi + 5 more
Source: ClinicalTrials.gov public record
Updated Mar 12, 2014 · Synced Oct 3, 2026, 11:45 AM EDT
Terminated No phase listed Observational

Clinical Evaluation of a Non-Invasive Hypoglycemia Detector in a Glycogen Storage Disease Population

NCT02338817
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Oct 4, 2016 · Synced Oct 3, 2026, 11:45 AM EDT
Recruiting No phase listed Observational

Clinical and Basic Investigations Into Known and Suspected Congenital Disorders of Glycosylation

NCT02089789
View directory record Official record
Conditions
Congenital Disorders of Glycosylation
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
1 Month to 80 Years
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2030
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 2, 2026 · Synced Oct 3, 2026, 11:45 AM EDT
Completed Not applicable Interventional

Prevention of Malnutrition in Children With Sickle Cell Disease

NCT06397118
View directory record Official record
Conditions
Sickle Cell Disease, Malnutrition
Interventions
Maternal Nutrition Education, Standard Care
Behavioral
Lead sponsor
Vanderbilt University Medical Center
Other
Eligibility
6 Months to 18 Months
Enrollment
146 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Jun 17, 2026 · Synced Oct 3, 2026, 11:45 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Doxycycline and TUDCA in Patients With Transthyretin Amyloid Cardiomyopathy

NCT01855360
View directory record Official record
Conditions
Amyloidosis; Heart (Manifestation), Senile Cardiac Amyloidosis
Interventions
Tauroursodeoxycholic Acid and Doxycycline
Drug
Lead sponsor
Brigham and Women's Hospital
Other
Eligibility
18 Years to 90 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2015
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Dec 4, 2020 · Synced Oct 3, 2026, 11:45 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Phase I Study of Progerinin in Healthy Volunteers

NCT04512963
View directory record Official record
Conditions
Hutchinson-Gilford Progeria Syndrome, Werner Syndrome
Interventions
Progerinin, Placebo
Drug
Lead sponsor
PRG Science & Technology Co., Ltd.
Industry
Eligibility
18 Years to 45 Years
Enrollment
64 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2021
U.S. locations
1
States / cities
Glendale, California
Source: ClinicalTrials.gov public record
Updated Sep 22, 2021 · Synced Oct 3, 2026, 11:45 AM EDT
Recruiting Phase 1Phase 2 Interventional

Phase 1/2 Study of ABO-101 in Primary Hyperoxaluria Type 1 (redePHine)

NCT06839235
View directory record Official record
Conditions
Primary Hyperoxaluria Type 1 (PH1)
Interventions
ABO-101
Drug
Lead sponsor
Arbor Biotechnologies
Industry
Eligibility
6 Years to 64 Years
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2043
U.S. locations
2
States / cities
Rochester, Minnesota • Saint Paul, Minnesota
Source: ClinicalTrials.gov public record
Updated Feb 12, 2026 · Synced Oct 3, 2026, 11:45 AM EDT
Active, not recruiting No phase listed Observational Accepts healthy volunteers

A Natural History Study to TRACK Brain and Spinal Cord Changes in Individuals with Friedreich Ataxia (TRACK-FA)

NCT04349514
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Natural history
Other
Lead sponsor
Monash University
Other
Eligibility
5 Years and older
Enrollment
300 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2025
U.S. locations
3
States / cities
Gainesville, Florida • Minneapolis, Minnesota • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Nov 27, 2024 · Synced Oct 3, 2026, 11:45 AM EDT