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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Completed Phase 2 Interventional Results available

A Study of the Use of Factor XIII Concentrate in Patients With Inherited FXIII Deficiency

NCT00883090
View directory record Official record
Conditions
Factor XIII Deficiency
Interventions
FXIII Concentrate (Human)
Biological
Lead sponsor
CSL Behring
Industry
Eligibility
Not listed
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2010
U.S. locations
5
States / cities
Dothan, Alabama • Orange, California • San Francisco, California + 2 more
Source: ClinicalTrials.gov public record
Updated Jan 15, 2012 · Synced Jun 7, 2026, 1:53 PM EDT
Not listed No phase listed Observational

The Natural History of Danon Disease

NCT03766386
View directory record Official record
Conditions
Danon Disease
Interventions
Not listed
Lead sponsor
University of California, San Diego
Other
Eligibility
0 Years to 100 Years
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2024
U.S. locations
1
States / cities
San Diego, California
Source: ClinicalTrials.gov public record
Updated May 11, 2022 · Synced Jun 7, 2026, 1:53 PM EDT
Terminated Phase 2 Interventional Results available

Carbamazepine in Severe Liver Disease Due to Alpha-1 Antitrypsin Deficiency

NCT01379469
View directory record Official record
Conditions
Alpha-1-antitrypsin Deficiency, Liver Cirrhosis
Interventions
Drug-Carbamazepine (Tegretol XR), Carbamazepine (Tegretol XR) Placebo
Drug
Lead sponsor
Washington University School of Medicine
Other
Eligibility
14 Years to 80 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2017
U.S. locations
3
States / cities
St Louis, Missouri • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 11, 2021 · Synced Jun 7, 2026, 1:53 PM EDT
Recruiting Phase 3 Interventional

A Study to Assess the Efficacy and Safety of Ruxolitinib Cream in Children and Adolescents (6 to <18 Years Old) With Moderate Atopic Dermatitis

NCT06832618
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
Ruxolitinib, Vehicle Cream
Drug
Lead sponsor
Incyte Corporation
Industry
Eligibility
6 Years to 17 Years
Enrollment
240 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
20
States / cities
Birmingham, Alabama • Phoenix, Arizona • Denver, Colorado + 17 more
Source: ClinicalTrials.gov public record
Updated Dec 17, 2025 · Synced Jun 7, 2026, 1:53 PM EDT
Not listed Phase 3 Interventional

RESULT: Reliable, Emergent Solution Using Liprotamase Treatment

NCT03051490
View directory record Official record
Conditions
Exocrine Pancreatic Insufficiency, Cystic Fibrosis
Interventions
Liprotamase, porcine PERT
Drug
Lead sponsor
Anthera Pharmaceuticals
Industry
Eligibility
7 Years and older
Enrollment
140 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2018
U.S. locations
28
States / cities
Little Rock, Arkansas • Long Beach, California • Los Angeles, California + 24 more
Source: ClinicalTrials.gov public record
Updated Mar 7, 2018 · Synced Jun 7, 2026, 1:53 PM EDT
Recruiting Phase 1 Interventional

The Impact of Botox on Neuroimmune Interactions in Atopic Dermatitis

NCT06928246
View directory record Official record
Conditions
Atopic Dermatitis (AD)
Interventions
Onabotulinum Toxin Type A - Phase 1b, Onabotulinum Toxin Type A - Phase 2
Drug
Lead sponsor
Daniel Kaplan
Other
Eligibility
18 Years and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
2
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated May 3, 2026 · Synced Jun 7, 2026, 1:53 PM EDT
Active, not recruiting Phase 3 Interventional Results available

Study of Two Doses of Crizanlizumab Versus Placebo in Adolescent and Adult Sickle Cell Disease Patients

NCT03814746
View directory record Official record
Conditions
Sickle Cell Disease (SCD)
Interventions
Crizanlizumab (SEG101), Placebo
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
12 Years to 100 Years
Enrollment
255 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2026
U.S. locations
5
States / cities
Atlanta, Georgia • Boston, Massachusetts • Charlotte, North Carolina + 2 more
Source: ClinicalTrials.gov public record
Updated May 25, 2026 · Synced Jun 7, 2026, 1:53 PM EDT
Not yet recruiting Phase 2 Interventional

SCI-110 in the Treatment of Tourette Syndrome

NCT05126888
View directory record Official record
Conditions
Tourette Syndrome
Interventions
SCI-110, Placebo
Drug · Other
Lead sponsor
Neurothera Labs Inc.
Industry
Eligibility
18 Years to 65 Years
Enrollment
164 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
New Haven, Connecticut
Source: ClinicalTrials.gov public record
Updated Apr 20, 2026 · Synced Jun 7, 2026, 1:53 PM EDT
Completed Phase 2 Interventional

Safety and Efficacy Study of PTC124 in Duchenne Muscular Dystrophy

NCT00264888
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
PTC124
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
5 Years and older · Male only
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2007
U.S. locations
3
States / cities
Cincinnati, Ohio • Philadelphia, Pennsylvania • Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Jan 13, 2009 · Synced Jun 7, 2026, 1:53 PM EDT
Active, not recruiting No phase listed Observational

The REPLACE Registry for Cholbam® (Cholic Acid)

NCT03115086
View directory record Official record
Conditions
Bile Acid Synthesis Disorders
Interventions
Cholbam
Drug
Lead sponsor
Mirum Pharmaceuticals, Inc.
Industry
Eligibility
Not listed
Enrollment
55 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2039
U.S. locations
23
States / cities
Birmingham, Alabama • Palo Alto, California • San Francisco, California + 18 more
Source: ClinicalTrials.gov public record
Updated Oct 1, 2023 · Synced Jun 7, 2026, 1:53 PM EDT
Completed Phase 3 Interventional Results available

Lesinurad Monotherapy in Gout Subjects Intolerant to Xanthine Oxidase Inhibitors

NCT01508702
View directory record Official record
Conditions
Gout
Interventions
lesinurad, Placebo
Drug
Lead sponsor
Ardea Biosciences, Inc.
Industry
Eligibility
18 Years to 85 Years
Enrollment
214 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
79
States / cities
Birmingham, Alabama • Glendale, Arizona • Phoenix, Arizona + 71 more
Source: ClinicalTrials.gov public record
Updated Feb 11, 2016 · Synced Jun 7, 2026, 1:53 PM EDT
Recruiting Phase 2 Interventional

Hematopoietic Stem Cell Transplantation (HSCT) for Common Variable Immunodeficiency (CVID) and Other Autoimmune Manifestations of Primary Immune Regulatory Disorders (PIRD)

NCT07284641
View directory record Official record
Conditions
Common Variable Immunodeficiency (CVID), Primary Immune Regulatory Disorder, Immune Dysregulation, DiGeorge Syndrome, STAT 1 Gain of Function, STAT 3 Gain of Function, Hypomorphic RAG1 Deficiency, CD40 Ligand Deficiency, Mendelian Susceptibility to Mycobacterial Disease, GATA2 Associated Immunodeficiency, CD40 Deficiency, Hypomorphic RAG2 Deficiency, Immune Dysregulation Polyendocrinopathy Enteropathy X-Linked Syndrome, Omenn Syndrome, Chronic Granulomatous Disease
Interventions
Hematopoietic stem cell transplant (HSCT)
Biological
Lead sponsor
Paul Szabolcs
Other
Eligibility
5 Years to 40 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2031
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated May 14, 2026 · Synced Jun 7, 2026, 1:53 PM EDT
Terminated Phase 2 Interventional Accepts healthy volunteers

E-Selectin Nasal Spray to Prevent Stroke Recurrence

NCT00012454
View directory record Official record
Conditions
Cerebrovascular Accident, Transient Ischemic Attack
Interventions
E-Selectin nasal spray
Drug
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
60 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2001 – 2004
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated May 3, 2010 · Synced Jun 7, 2026, 1:53 PM EDT
Completed Phase 3 Interventional Results available

A Study to Evaluate the Clinical Efficacy and Safety of Subcutaneously Administered C1-esterase Inhibitor in the Prevention of Hereditary Angioedema

NCT01912456
View directory record Official record
Conditions
Hereditary Angioedema Types I and II
Interventions
Low-volume C1-esterase inhibitor, Higher-volume C1-esterase inhibitor, Low-volume placebo, Higher-volume placebo
Biological
Lead sponsor
CSL Behring
Industry
Eligibility
12 Years and older
Enrollment
90 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
19
States / cities
Birmingham, Alabama • Scottsdale, Arizona • Bell Gardens, California + 16 more
Source: ClinicalTrials.gov public record
Updated Jan 28, 2021 · Synced Jun 7, 2026, 1:53 PM EDT
Completed No phase listed Observational Accepts healthy volunteers Results available

Early-onset Obesity and Cognitive Impairment in Children With Pseudohypoparathyroidism

NCT02411461
View directory record Official record
Conditions
Pseudohypoparathyroidism, Albright Hereditary Osteodystrophy
Interventions
Not listed
Lead sponsor
Vanderbilt University Medical Center
Other
Eligibility
6 Years to 18 Years
Enrollment
39 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2017
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Nov 12, 2019 · Synced Jun 7, 2026, 1:53 PM EDT
Completed Not applicable Interventional

PITA-HF: Feasibility, Safety, and Tolerability

NCT04159454
View directory record Official record
Conditions
Heart Failure, Dilated Cardiomyopathy
Interventions
PITA
Device
Lead sponsor
Johns Hopkins University
Other
Eligibility
18 Years and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Feb 13, 2023 · Synced Jun 7, 2026, 1:53 PM EDT
Completed Phase 2 Interventional Results available

A 2-Period Crossover Study of BPN14770 in Adults Males With Fragile X Syndrome

NCT03569631
View directory record Official record
Conditions
Fragile X Syndrome, FXS, Fra(X) Syndrome
Interventions
BPN14770, Placebo
Drug
Lead sponsor
Tetra Discovery Partners
Industry
Eligibility
18 Years to 45 Years · Male only
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Dec 18, 2024 · Synced Jun 7, 2026, 1:53 PM EDT
Completed Phase 1 Interventional

Therapeutic Application of Intravascular Nitrite for Sickle Cell Disease

NCT00095472
View directory record Official record
Conditions
Sickle Cell Anemia
Interventions
L-NMMA, Sodium Nitrite
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
18 Years and older
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2007
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Nov 4, 2019 · Synced Jun 7, 2026, 1:53 PM EDT
Active, not recruiting Phase 3 Interventional Accepts healthy volunteers

Improved Diagnosis of Familial Hypercholesterolemia Across the Northland (ID-FH)

NCT05238519
View directory record Official record
Conditions
Familial Hypercholesterolemia, Cholesterol, Elevated, Genetic Disease
Interventions
Motivational interview (MI)
Behavioral
Lead sponsor
Essentia Health
Other
Eligibility
2 Years to 75 Years
Enrollment
300 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2026
U.S. locations
2
States / cities
Duluth, Minnesota
Source: ClinicalTrials.gov public record
Updated Jan 15, 2026 · Synced Jun 7, 2026, 1:53 PM EDT
Completed Phase 2 Interventional Results available

Safety and Efficacy of Vicriviroc (MK-7690) in Combination With Pembrolizumab (MK-3475) in Participants With Advanced/Metastatic Microsatellite Stable (MSS) Colorectal Cancer (CRC) (MK-7690-046)

NCT03631407
View directory record Official record
Conditions
Colorectal Neoplasms
Interventions
Vicriviroc, Pembrolizumab
Drug · Biological
Lead sponsor
Merck Sharp & Dohme LLC
Industry
Eligibility
18 Years and older
Enrollment
41 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
6
States / cities
Scottsdale, Arizona • Encinitas, California • Fresno, California + 3 more
Source: ClinicalTrials.gov public record
Updated Nov 20, 2024 · Synced Jun 7, 2026, 1:53 PM EDT
Suspended Not applicable Interventional

Clinical Evaluation of the AccuCinch® Ventricular Restoration System in Patients Who Present With Symptomatic Heart Failure With Reduced Ejection Fraction (HFrEF): The CORCINCH-HF Study

NCT04331769
View directory record Official record
Conditions
Heart Failure With Reduced Ejection Fraction (HFrEF), Dilated Cardiomyopathy
Interventions
AccuCinch Ventricular Restoration System, Guideline-Directed Medical Therapy
Device · Drug
Lead sponsor
Ancora Heart, Inc.
Industry
Eligibility
18 Years and older
Enrollment
400 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2030
U.S. locations
96
States / cities
Birmingham, Alabama • Phoenix, Arizona • Tucson, Arizona + 75 more
Source: ClinicalTrials.gov public record
Updated Jun 2, 2026 · Synced Jun 7, 2026, 1:53 PM EDT
Completed Phase 1Phase 2 Interventional

Pharmacokinetics and Safety Study of Single and Multiple Oral Doses Prodarsan™ in Patients With Cockayne Syndrome

NCT01142154
View directory record Official record
Conditions
Cockayne Syndrome
Interventions
Prodarsan
Drug
Lead sponsor
DNage B.V.
Industry
Eligibility
2 Years to 10 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2011
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jun 22, 2011 · Synced Jun 7, 2026, 1:53 PM EDT
Completed Not applicable Interventional

Anger Control Training for Youth With Tourette Syndrome

NCT00486551
View directory record Official record
Conditions
Tourette Syndrome, Chronic Tic Disorder, Oppositional Defiant Disorder, ADHD
Interventions
Anger control training
Behavioral
Lead sponsor
Yale University
Other
Eligibility
11 Years to 16 Years
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2006
U.S. locations
1
States / cities
New Haven, Connecticut
Source: ClinicalTrials.gov public record
Updated Feb 10, 2022 · Synced Jun 7, 2026, 1:53 PM EDT
Completed Phase 4 Interventional Results available

Study of KRYSTEXXA® (Pegloticase) Plus Methotrexate in Participants With Uncontrolled Gout

NCT03994731
View directory record Official record
Conditions
Gout
Interventions
Pegloticase, methotrexate, placebo, folic acid, gout flare prophylaxis regimen, fexofenadine, acetaminophen, methylprednisolone
Biological · Drug · Dietary Supplement
Lead sponsor
Amgen
Industry
Eligibility
18 Years and older
Enrollment
152 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
60
States / cities
Birmingham, Alabama • Anchorage, Alaska • Flagstaff, Arizona + 54 more
Source: ClinicalTrials.gov public record
Updated Jun 25, 2024 · Synced Jun 7, 2026, 1:53 PM EDT