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ClinicalTrials.gov public records Last synced Oct 21, 2026, 4:02 AM EDT

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Showing 313–336 of 8,005 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Genetics/Birth Defects Clear all
Completed Phase 3 Interventional Results available

Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A

NCT04323098
View directory record Official record
Conditions
Hemophilia A
Interventions
valoctocogene roxaparvovec
Biological
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
18 Years and older · Male only
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2025
U.S. locations
3
States / cities
Sacramento, California • St Louis, Missouri • Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Oct 8, 2025 · Synced Oct 21, 2026, 4:02 AM EDT
Completed Phase 1Phase 2 Interventional Results available

A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular BMN 190 in Patients With Late-Infantile Neuronal Ceroid Lipofuscinosis (CLN2) Disease

NCT01907087
View directory record Official record
Conditions
Jansky-Bielschowsky Disease, Batten Disease, Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2, CLN2 Disease
Interventions
BMN 190
Biological
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
3 Years to 15 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2015
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Mar 8, 2019 · Synced Oct 21, 2026, 4:02 AM EDT
Terminated Phase 2 Interventional Results available

Study of Ataluren (PTC124) in Hemophilia A and B

NCT00947193
View directory record Official record
Conditions
Hemophilia A, Hemophilia B
Interventions
Ataluren
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
18 Years and older · Male only
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2011
U.S. locations
7
States / cities
Peoria, Illinois • Indianapolis, Indiana • Worcester, Massachusetts + 4 more
Source: ClinicalTrials.gov public record
Updated Jun 16, 2020 · Synced Oct 21, 2026, 4:02 AM EDT
Completed Phase 4 Interventional Results available

A Study to Evaluate the Mode of Administration and Safety of EUR-1008 (APT-1008) in Infants 1 to 12 Months of Age

NCT01100606
View directory record Official record
Conditions
Cystic Fibrosis, Exocrine Pancreatic Insufficiency
Interventions
EUR-1008 (APT-1008)
Drug
Lead sponsor
Forest Laboratories
Industry
Eligibility
1 Month to 12 Months
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010
U.S. locations
6
States / cities
Jacksonville, Florida • Las Vegas, Nevada • Akron, Ohio + 3 more
Source: ClinicalTrials.gov public record
Updated Apr 10, 2014 · Synced Oct 21, 2026, 4:02 AM EDT
Completed Phase 2 Interventional Results available

Effect of Oral Cimetidine in the Protoporphyrias

NCT05020184
View directory record Official record
Conditions
Erythropoietic Protoporphyria, X-linked Protoporphyria
Interventions
Cimetidine, Placebo
Drug
Lead sponsor
Amy K. Dickey, M.D.
Other
Eligibility
15 Years and older
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
3
States / cities
Boston, Massachusetts • Winston-Salem, North Carolina • Galveston, Texas
Source: ClinicalTrials.gov public record
Updated Sep 4, 2026 · Synced Oct 21, 2026, 4:02 AM EDT
Recruiting No phase listed Observational

The Intersection of Oncology Care and Worker Well-Being

NCT05250284
View directory record Official record
Conditions
Solid Tumor
Interventions
WellBQ, PROMIS
Behavioral
Lead sponsor
University of Colorado, Denver
Other
Eligibility
21 Years to 70 Years · Male only
Enrollment
400 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
1
States / cities
Aurora, Colorado
Source: ClinicalTrials.gov public record
Updated Jul 13, 2026 · Synced Oct 21, 2026, 4:02 AM EDT
Completed Not applicable Interventional

Online Mindfulness-based Tic Reduction

NCT03525626
View directory record Official record
Conditions
Tourette Syndrome, Tic Disorders
Interventions
Online Mindfulness-based Tic Reduction
Behavioral
Lead sponsor
Bowdoin College
Other
Eligibility
18 Years and older
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018
U.S. locations
2
States / cities
Brunswick, Maine • Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jul 9, 2019 · Synced Oct 21, 2026, 4:02 AM EDT
Completed Phase 2Phase 3 Interventional Results available

12-Week Safety and Efficacy Study of BCX4161 as an Oral Prophylaxis Against HAE Attacks

NCT02303626
View directory record Official record
Conditions
Hereditary Angioedema, HAE
Interventions
BCX4161, Placebo
Drug
Lead sponsor
BioCryst Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
110 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
25
States / cities
Birmingham, Alabama • Scottsdale, Arizona • Granada Hills, California + 21 more
Source: ClinicalTrials.gov public record
Updated Nov 17, 2025 · Synced Oct 21, 2026, 4:02 AM EDT
Enrolling by invitation Phase 1Phase 2 Interventional

A Study of the Safety and Efficacy of Prime Editing (PM359) in Participants With p47phox Autosomal Recessive Chronic Granulomatous Disease (CGD )

NCT06559176
View directory record Official record
Conditions
Chronic Granulomatous Disease, Granulomatous Disease, Chronic
Interventions
PM359
Biological
Lead sponsor
Prime Medicine, Inc.
Industry
Eligibility
6 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2030
U.S. locations
3
States / cities
Los Angeles, California • Bethesda, Maryland • Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Apr 6, 2026 · Synced Oct 21, 2026, 4:02 AM EDT
Completed Not applicable Interventional

Computerized Cognitive Skills Training for Adolescents With Velocardiofacial Syndrome

NCT00917189
View directory record Official record
Conditions
Velocardiofacial Syndrome
Interventions
Challenging Our Minds Program
Behavioral
Lead sponsor
State University of New York - Upstate Medical University
Other
Eligibility
12 Years to 16 Years
Enrollment
37 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2015
U.S. locations
1
States / cities
Syracuse, New York
Source: ClinicalTrials.gov public record
Updated Oct 21, 2016 · Synced Oct 21, 2026, 4:02 AM EDT
Recruiting Phase 1 Interventional

Selective Antigen Specific T Cells and CAR T Cells in Subjects With Relapsed/Refractory Embryonal Tumors (SABRE)

NCT07172958
View directory record Official record
Conditions
Rhabdomyosarcoma, Ewing Sarcoma, Neuroblastoma, Wilms Tumor
Interventions
Selective Antigen Specific dTβRII-expressing T cells combined with B7-H3 CAR T cells
Biological
Lead sponsor
Children's National Research Institute
Other
Eligibility
1 Year to 23 Years
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2044
U.S. locations
2
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Aug 13, 2026 · Synced Oct 21, 2026, 4:02 AM EDT
Completed Early Phase 1 Interventional

Pimavanserin Treatment in TS

NCT04794413
View directory record Official record
Conditions
Tourette Syndrome, Tardive Dyskinesia
Interventions
Pimavanserin
Drug
Lead sponsor
Joseph Jankovic
Other
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Mar 12, 2021 · Synced Oct 21, 2026, 4:02 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Preparing for Prevention of Huntington's Disease (PREVENT-HD)

NCT04818060
View directory record Official record
Conditions
Huntington Disease
Interventions
Clinical Assessments, MRI Scan, Lumber Puncture (LP)
Other · Diagnostic Test
Lead sponsor
University of Wisconsin, Madison
Other
Eligibility
18 Years to 80 Years
Enrollment
258 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2025
U.S. locations
1
States / cities
Madison, Wisconsin
Source: ClinicalTrials.gov public record
Updated Mar 18, 2025 · Synced Oct 21, 2026, 4:02 AM EDT
Completed Not applicable Interventional

Use of Mobile Technology for Intensive Training in Medication Management

NCT02133560
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia
Interventions
Video recording, Medication Administration Log, Education
Behavioral
Lead sponsor
Duke University
Other
Eligibility
5 Years and older
Enrollment
46 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated May 25, 2017 · Synced Oct 21, 2026, 4:02 AM EDT
Completed No phase listed Observational

The Evaluation of Oral Acitretin in the Treatment of Psoriasis, Cutaneous Disorders of Keratinization, Multiple Basal Cell Carcinomas and Other Retinoid Responsive Diseases

NCT00005660
View directory record Official record
Conditions
Basal Cell Carcinoma, Keratosis Palmaris et Plantaris, Psoriasis
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
Not listed
Enrollment
130 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1977 – 2001
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 21, 2026, 4:02 AM EDT
Enrolling by invitation Not applicable Interventional

Long-term Evaluation of Nasopharyngeal Airway in Hypotonia

NCT06614582
View directory record Official record
Conditions
Hypotonia, Muscle, Obstructive Sleep Apnea, Nasal Airway Obstruction
Interventions
Self-Supporting Nasopharyngeal Airway (ssNPA)
Device
Lead sponsor
University of Michigan
Other
Eligibility
3 Years to 23 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Dec 2, 2025 · Synced Oct 21, 2026, 4:02 AM EDT
Completed Phase 1Phase 2 Interventional

Iron Balance Study of DFO and GT56-252 in Patients With Transfusional Iron Overload Secondary to Beta-Thalassemia

NCT00069862
View directory record Official record
Conditions
Beta-Thalassemia
Interventions
Deferitrin (GT56-252), desferoxamine (DFO)
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
18 Years and older
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2005
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Mar 5, 2015 · Synced Oct 21, 2026, 4:02 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Ph I/II Study of Allogeneic SCT for Clinically Aggressive Sickle Cell Disease (SCD)

NCT01499888
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Allogeneic Non-Myeloablative Stem Cell Transplantation, Alemtuzumab, Sirolimus
Procedure · Drug
Lead sponsor
University of Illinois at Chicago
Other
Eligibility
16 Years to 60 Years
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2029
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Jul 8, 2026 · Synced Oct 21, 2026, 4:02 AM EDT
Completed Phase 2 Interventional

A Randomized, Double-Blind Study to Evaluate the Safety and Efficacy of 2 Doses of S-777469 in Patients With Atopic Dermatitis

NCT00703573
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
S-777469 400 mg, S-777469 800 mg, Placebo
Drug
Lead sponsor
Shionogi
Industry
Eligibility
18 Years to 65 Years
Enrollment
209 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2009
U.S. locations
33
States / cities
Hot Springs, Arkansas • Los Angeles, California • Oceanside, California + 28 more
Source: ClinicalTrials.gov public record
Updated May 8, 2018 · Synced Oct 21, 2026, 4:02 AM EDT
Terminated Phase 2 Interventional Results available

Pilot Study of Sonidegib and Buparlisib in Treating Patients With Advanced or Metastatic Basal Cell Carcinoma

NCT02303041
View directory record Official record
Conditions
Carcinoma, Basal Cell, Recurrent Skin Cancer, Skin Neoplasms, Basal Cell Nevus Syndrome
Interventions
Buparlisib, Sonidegib
Drug
Lead sponsor
Anne Chang
Other
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
1
States / cities
Stanford, California
Source: ClinicalTrials.gov public record
Updated Jan 29, 2019 · Synced Oct 21, 2026, 4:02 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Cancer of the Pancreas Screening Study (CAPS 3)

NCT00438906
View directory record Official record
Conditions
Pancreatic Neoplasm, Peutz-Jeghers Syndrome
Interventions
Biopsy, Fine Needle Aspiration (FNA), Secretin (human synthetic) - ChiRhoClin
Procedure · Drug
Lead sponsor
Johns Hopkins University
Other
Eligibility
40 Years to 80 Years
Enrollment
200 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2006 – 2009
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 18, 2021 · Synced Oct 21, 2026, 4:02 AM EDT
Completed No phase listed Observational Accepts healthy volunteers Results available

Intervention and Outcomes in Duarte Galactosemia

NCT02519504
View directory record Official record
Conditions
Duarte Galactosemia
Interventions
Not listed
Lead sponsor
Emory University
Other
Eligibility
6 Years to 12 Years
Enrollment
566 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2017
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Sep 19, 2019 · Synced Oct 21, 2026, 4:02 AM EDT
Completed Phase 2 Interventional Results available

Tradipitant in Treatment-resistant Pruritus Associated With Atopic Dermatitis

NCT02651714
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
Tradipitant, Placebo
Drug
Lead sponsor
Vanda Pharmaceuticals
Industry
Eligibility
18 Years to 65 Years
Enrollment
168 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
28
States / cities
Encinitas, California • Encino, California • Lomita, California + 22 more
Source: ClinicalTrials.gov public record
Updated Jun 13, 2024 · Synced Oct 21, 2026, 4:02 AM EDT
Completed Phase 2 Interventional Results available

A Study to Evaluate the Safety and Efficacy of VX-121 Combination Therapy in Subjects With Cystic Fibrosis

NCT03912233
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
VX-121, TEZ, VX-561, TEZ/IVA, IVA, Placebo
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
18 Years and older
Enrollment
87 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019
U.S. locations
12
States / cities
Los Angeles, California • Oakland, California • Lexington, Kentucky + 9 more
Source: ClinicalTrials.gov public record
Updated Apr 20, 2023 · Synced Oct 21, 2026, 4:02 AM EDT