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ClinicalTrials.gov public records Last synced Oct 23, 2026, 3:48 PM EDT

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Showing 1033–1056 of 8,007 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Genetics/Birth Defects Clear all
Completed Phase 3 Interventional Results available

An Open-label Safety and Efficacy Study of Recombinant FVIII in Patients With Severe Hemophilia A

NCT02172950
View directory record Official record
Conditions
Hemophilia A, Severe Hemophilia A
Interventions
rVIII-SingleChain
Biological
Lead sponsor
CSL Behring
Industry
Eligibility
Male only
Enrollment
246 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2021
U.S. locations
9
States / cities
San Diego, California • Aurora, Colorado • Hartford, Connecticut + 6 more
Source: ClinicalTrials.gov public record
Updated Oct 27, 2021 · Synced Oct 23, 2026, 3:48 PM EDT
Recruiting No phase listed Observational

Clinical Registry Investigating Bardet-Biedl Syndrome

NCT02329210
View directory record Official record
Conditions
Bardet-Biedl Syndrome
Interventions
Not listed
Lead sponsor
Marshfield Clinic Research Foundation
Other
Eligibility
Not listed
Enrollment
1,200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2030
U.S. locations
1
States / cities
Marshfield, Wisconsin
Source: ClinicalTrials.gov public record
Updated Jul 19, 2022 · Synced Oct 23, 2026, 3:48 PM EDT
Completed Phase 4 Interventional Results available

Vitamin D and Prebiotics for Intestinal Health in Cystic Fibrosis

NCT04118010
View directory record Official record
Conditions
Cystic Fibrosis, Dysbiosis
Interventions
Vitamin D3, Placebo vitamin D3, Inulin, Placebo Inulin
Drug
Lead sponsor
Emory University
Other
Eligibility
18 Years and older
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Feb 7, 2024 · Synced Oct 23, 2026, 3:48 PM EDT
Recruiting No phase listed Observational

Rhizomelic Chondrodysplasia Punctata Registry

NCT04569162
View directory record Official record
Conditions
RCDP - Rhizomelic Chondrodysplasia Punctata, RCDP1, RCDP2, RCDP3, RCDP4, RCDP5
Interventions
Not listed
Lead sponsor
Nemours Children's Clinic
Other
Eligibility
Not listed
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2030
U.S. locations
1
States / cities
Wilmington, Delaware
Source: ClinicalTrials.gov public record
Updated Aug 7, 2026 · Synced Oct 23, 2026, 3:48 PM EDT
Active, not recruiting Phase 2 Interventional Results available

Selpercatinib for the Treatment of Advanced Solid Tumors, Lymphomas, or Histiocytic Disorders With Activating RET Gene Alterations, a Pediatric MATCH Treatment Trial

NCT04320888
View directory record Official record
Conditions
Hematopoietic and Lymphatic System Neoplasm, Recurrent Ependymoma, Recurrent Ewing Sarcoma, Recurrent Hepatoblastoma, Recurrent Histiocytic and Dendritic Cell Neoplasm, Recurrent Langerhans Cell Histiocytosis, Recurrent Lymphoma, Recurrent Malignant Germ Cell Tumor, Recurrent Malignant Glioma, Recurrent Malignant Solid Neoplasm, Recurrent Medulloblastoma, Recurrent Neuroblastoma, Recurrent Non-Hodgkin Lymphoma, Recurrent Osteosarcoma, Recurrent Peripheral Primitive Neuroectodermal Tumor, Recurrent Rhabdoid Tumor, Recurrent Rhabdomyosarcoma, Recurrent Soft Tissue Sarcoma, Recurrent WHO Grade 2 Glioma, Refractory Ependymoma, Refractory Ewing Sarcoma, Refractory Hepatoblastoma, Refractory Histiocytic and Dendritic Cell Neoplasm, Refractory Langerhans Cell Histiocytosis, Refractory Lymphoma, Refractory Malignant Germ Cell Tumor, Refractory Malignant Glioma, Refractory Malignant Solid Neoplasm, Refractory Medulloblastoma, Refractory Neuroblastoma, Refractory Non-Hodgkin Lymphoma, Refractory Osteosarcoma, Refractory Peripheral Primitive Neuroectodermal Tumor, Refractory Rhabdoid Tumor, Refractory Rhabdomyosarcoma, Refractory Soft Tissue Sarcoma, Refractory WHO Grade 2 Glioma, Wilms Tumor
Interventions
Computed Tomography, Magnetic Resonance Imaging, Positron Emission Tomography, Radionuclide Imaging, Selpercatinib, X-Ray Imaging
Procedure · Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
12 Months to 21 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
172
States / cities
Birmingham, Alabama • Anchorage, Alaska • Mesa, Arizona + 129 more
Source: ClinicalTrials.gov public record
Updated Oct 9, 2026 · Synced Oct 23, 2026, 3:48 PM EDT
Recruiting Phase 1Phase 2 Interventional

A Study of the Safety and Efficacy of Prime Editing (PM577) in Participants With Wilson Disease (WD)

NCT07748403
View directory record Official record
Conditions
Wilson Disease, Wilson's Disease, Wilsons Disease
Interventions
PM577a
Drug
Lead sponsor
Prime Medicine, Inc.
Industry
Eligibility
12 Years and older
Enrollment
42 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
3
States / cities
New Haven, Connecticut • Chicago, Illinois • San Antonio, Texas
Source: ClinicalTrials.gov public record
Updated Oct 1, 2026 · Synced Oct 23, 2026, 3:48 PM EDT
Completed Phase 3 Interventional

A Study to Evaluate the Efficacy and Safety of Subcutaneous Amlitelimab Monotherapy Compared With Placebo in Participants Aged 12 Years and Older With Moderate-to-severe Atopic Dermatitis

NCT06130566
View directory record Official record
Conditions
Dermatitis Atopic
Interventions
Amlitelimab, Placebo
Drug
Lead sponsor
Sanofi
Industry
Eligibility
12 Years and older
Enrollment
601 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
42
States / cities
Scottsdale, Arizona • Fountain Valley, California • Fremont, California + 36 more
Source: ClinicalTrials.gov public record
Updated Jul 22, 2026 · Synced Oct 23, 2026, 3:48 PM EDT
Completed Not applicable Interventional

Early Intervention in Pulmonary Exacerbation in Cystic Fibrosis

NCT00850551
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Early Intervention
Other
Lead sponsor
University of Washington
Other
Eligibility
18 Years and older
Enrollment
42 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2011
U.S. locations
1
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Dec 1, 2016 · Synced Oct 23, 2026, 3:48 PM EDT
Enrolling by invitation Phase 3 Interventional

A Long-term Follow-up Study in Participants Who Received CTX001

NCT04208529
View directory record Official record
Conditions
Beta-Thalassemia, Thalassemia, Sickle Cell Disease, Hematologic Diseases, Hemoglobinopathies, Genetic Diseases, Inborn, Sickle Cell Anemia
Interventions
CTX001
Biological
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
2 Years and older
Enrollment
160 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2039
U.S. locations
9
States / cities
Palo Alto, California • Chicago, Illinois • New York, New York + 5 more
Source: ClinicalTrials.gov public record
Updated Aug 11, 2026 · Synced Oct 23, 2026, 3:48 PM EDT
Terminated Phase 2Phase 3 Interventional Results available

Gemcitabine Plus Cisplatin With or Without Bintrafusp Alfa (M7824) in Participants With 1L BTC

NCT04066491
View directory record Official record
Conditions
Biliary Tract Cancer, Cholangiocarcinoma, Gallbladder Cancer
Interventions
M7824, Placebo, Gemcitabine, Cisplatin
Drug
Lead sponsor
EMD Serono Research & Development Institute, Inc.
Industry
Eligibility
18 Years and older
Enrollment
309 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
12
States / cities
Chandler, Arizona • Gilbert, Arizona • Phoenix, Arizona + 9 more
Source: ClinicalTrials.gov public record
Updated Nov 14, 2023 · Synced Oct 23, 2026, 3:48 PM EDT
Completed Phase 3 Interventional Results available

Study of Patidegib Topical Gel, 2%, for the Reduction of Disease Burden of Persistently Developing Basal Cell Carcinomas (BCCs) in Subjects With Basal Cell Nevus Syndrome (Gorlin Syndrome)

NCT03703310
View directory record Official record
Conditions
Basal Cell Nevus Syndrome
Interventions
Patidegib Topical Gel, 2%, Patidegib Topical Gel, Vehicle
Drug
Lead sponsor
Sol-Gel Technologies, Ltd.
Industry
Eligibility
18 Years and older
Enrollment
174 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2020
U.S. locations
22
States / cities
Phoenix, Arizona • Newport Beach, California • Redwood City, California + 19 more
Source: ClinicalTrials.gov public record
Updated Jul 3, 2024 · Synced Oct 23, 2026, 3:48 PM EDT
Recruiting No phase listed Observational

Clinical Course Of Disease In Participants With FA-CM

NCT06865482
View directory record Official record
Conditions
Friedreich Ataxia, Cardiomyopathy
Interventions
Not listed
Lead sponsor
Lexeo Therapeutics
Industry
Eligibility
6 Years and older
Enrollment
65 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
8
States / cities
La Jolla, California • Tampa, Florida • Indianapolis, Indiana + 5 more
Source: ClinicalTrials.gov public record
Updated Oct 19, 2026 · Synced Oct 23, 2026, 3:48 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Zinc and Diabetes in Patients With Thalassemia: a Pilot Study

NCT01772680
View directory record Official record
Conditions
Thalassemia
Interventions
Zinc Supplementation
Dietary Supplement
Lead sponsor
UCSF Benioff Children's Hospital Oakland
Other
Eligibility
12 Years and older
Enrollment
40 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2015
U.S. locations
1
States / cities
Oakland, California
Source: ClinicalTrials.gov public record
Updated Nov 24, 2020 · Synced Oct 23, 2026, 3:48 PM EDT
Completed Phase 3 Interventional Results available

Study of Ivacaftor in Cystic Fibrosis Subjects Aged 6 to 11 Years With the G551D Mutation

NCT00909727
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Ivacaftor, Placebo
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
6 Years to 11 Years
Enrollment
52 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2011
U.S. locations
17
States / cities
Birmingham, Alabama • Atlanta, Georgia • Chicago, Illinois + 13 more
Source: ClinicalTrials.gov public record
Updated Aug 21, 2012 · Synced Oct 23, 2026, 3:48 PM EDT
Completed Phase 3 Interventional Results available

Recombinant Factor VIIa BI (rFVIIa BI) Treatment of Acute Bleeding Episodes Per an On-demand Regimen

NCT01757405
View directory record Official record
Conditions
Hemophilia A, Hemophilia B
Interventions
Recombinant Factor VIIa BI (rFVIIa BI)
Biological
Lead sponsor
Baxalta now part of Shire
Industry
Eligibility
12 Years to 65 Years · Male only
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
1
States / cities
Tampa, Florida
Source: ClinicalTrials.gov public record
Updated May 11, 2021 · Synced Oct 23, 2026, 3:48 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

A Study to Assess Plasma Ammonia Time-Normalized Area Under the Curve and Rate of Ureagenesis in Healthy Adult Subjects

NCT04269122
View directory record Official record
Conditions
Ornithine Transcarbamylase Deficiency
Interventions
No Intervention
Other
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
18 Years to 55 Years
Enrollment
120 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2020
U.S. locations
1
States / cities
Austin, Texas
Source: ClinicalTrials.gov public record
Updated Mar 30, 2020 · Synced Oct 23, 2026, 3:48 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Efficacy and Safety Study of Genetically Targeted Enzyme Replacement Therapy for Advanced Heart Failure

NCT00454818
View directory record Official record
Conditions
Heart Failure, Congestive, Dilated Cardiomyopathy
Interventions
MYDICAR Phase 1 (Open-label, Serial Dose-Escalation Study), Placebo Infusion, MYDICAR Phase 2 (Placebo-controlled, Randomized Study)
Genetic · Procedure
Lead sponsor
Celladon Corporation
Industry
Eligibility
18 Years to 75 Years
Enrollment
51 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2012
U.S. locations
22
States / cities
San Diego, California • Gainesville, Florida • Chicago, Illinois + 15 more
Source: ClinicalTrials.gov public record
Updated Aug 20, 2014 · Synced Oct 23, 2026, 3:48 PM EDT
Completed Phase 1 Interventional

Safety and Pharmacokinetics of NNC 0129-0000-1003 in Subjects With Haemophilia A

NCT01205724
View directory record Official record
Conditions
Congenital Bleeding Disorder, Haemophilia A
Interventions
turoctocog alfa pegol
Drug
Lead sponsor
Novo Nordisk A/S
Industry
Eligibility
18 Years to 65 Years · Male only
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2011
U.S. locations
5
States / cities
Iowa City, Iowa • Lexington, Kentucky • Baltimore, Maryland + 2 more
Source: ClinicalTrials.gov public record
Updated Feb 10, 2017 · Synced Oct 23, 2026, 3:48 PM EDT
Terminated Phase 2 Interventional Results available

Study of ACE-083 in Patients With Charcot-Marie-Tooth Disease

NCT03124459
View directory record Official record
Conditions
Charcot-Marie-Tooth Disease
Interventions
ACE-083, Placebo
Drug
Lead sponsor
Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
Industry
Eligibility
18 Years and older
Enrollment
63 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2020
U.S. locations
16
States / cities
Orange, California • Aurora, Colorado • Gainesville, Florida + 13 more
Source: ClinicalTrials.gov public record
Updated Sep 26, 2022 · Synced Oct 23, 2026, 3:48 PM EDT
Completed Phase 2 Interventional Results available

Trial to Evaluate the Effect of ALN-PCSSC Treatment on Low Density Lipoprotein Cholesterol (LDL-C)

NCT02597127
View directory record Official record
Conditions
Atherosclerotic Cardiovascular Disease, Familial Hypercholesterolemia, Diabetes
Interventions
ALN-PCSSC, Normal Saline
Drug
Lead sponsor
The Medicines Company
Industry
Eligibility
18 Years to 99 Years
Enrollment
501 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
8
States / cities
Jacksonville, Florida • Indianapolis, Indiana • New York, New York + 4 more
Source: ClinicalTrials.gov public record
Updated May 17, 2019 · Synced Oct 23, 2026, 3:48 PM EDT
Completed Not applicable Interventional Results available

Behavioral & Nutritional Treatment to Help CF Preschoolers Grow

NCT00241969
View directory record Official record
Conditions
Cystic Fibrosis, Pancreatic Cystic Fibrosis
Interventions
Behavioral and Nutrition Treatment, Education and Attention Control
Behavioral
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
2 Years to 6 Years
Enrollment
78 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2015
U.S. locations
5
States / cities
Tucson, Arizona • Ann Arbor, Michigan • Cincinnati, Ohio + 2 more
Source: ClinicalTrials.gov public record
Updated May 22, 2018 · Synced Oct 23, 2026, 3:48 PM EDT
Recruiting Phase 3 Interventional

A Study to Assess the Efficacy and Safety of Emicizumab in Participants With Type 3 Von Willebrand Disease

NCT06998524
View directory record Official record
Conditions
Von Willebrand Disease, Type 3
Interventions
Emicizumab, von Willebrand Factor (VWF) Concentrates, Factor VIII (FVIII) Concentrates, von Willebrand Factor (VWF) and Factor VIII (FVIII) Concentrates, Bypassing Agents
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
1 Month and older
Enrollment
75 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2029
U.S. locations
6
States / cities
Sacramento, California • Gainesville, Florida • Lexington, Kentucky + 3 more
Source: ClinicalTrials.gov public record
Updated Oct 9, 2026 · Synced Oct 23, 2026, 3:48 PM EDT
Active, not recruiting No phase listed Observational Accepts healthy volunteers

Defining the Skin and Blood Biomarkers of Pediatric Atopic Dermatitis

NCT01782703
View directory record Official record
Conditions
Atopic Dermatitis, Eczema
Interventions
Not listed
Lead sponsor
Northwestern University
Other
Eligibility
0 Months to 17 Years
Enrollment
505 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2027
U.S. locations
4
States / cities
Chicago, Illinois • Northbrook, Illinois • New York, New York
Source: ClinicalTrials.gov public record
Updated Apr 27, 2026 · Synced Oct 23, 2026, 3:48 PM EDT
Completed Phase 1 Interventional

A Phase I/II Trial of Recombinant-Methionyl Human Stem Cell Factor (SCF) in Adult Patients With Sickling Disorders

NCT00005783
View directory record Official record
Conditions
Hemoglobin SC Disease, Sickle Cell Anemia
Interventions
Recombinant-methionyl human stem cell factor
Drug
Lead sponsor
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
NIH
Eligibility
Not listed
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 23, 2026, 3:48 PM EDT