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ClinicalTrials.gov public records Last synced Oct 29, 2026, 10:58 PM EDT

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Showing 1513–1536 of 8,017 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Genetics/Birth Defects Clear all
Completed Phase 1Phase 2 Interventional Accepts healthy volunteers

A Single-ascending Dose (Part A) and Repeat-dose (Part B) Study to Investigate the Safety, Pharmacokinetics and Efficacy (Part B Only) of UCB1381 in Healthy Study Participants (Part A) and in Study Participants With Moderate to Severe Atopic Dermatitis (Part B)

NCT05277571
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
UCB1381, Placebo
Biological · Drug
Lead sponsor
UCB Biopharma SRL
Industry
Eligibility
18 Years to 65 Years
Enrollment
109 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2025
U.S. locations
16
States / cities
Beverly Hills, California • Glendale, California • Los Angeles, California + 13 more
Source: ClinicalTrials.gov public record
Updated Jul 22, 2026 · Synced Oct 29, 2026, 10:58 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Impact of the Severity of Pediatric Psoriasis on Childhood Body Mass Index

NCT00879944
View directory record Official record
Conditions
Psoriasis, Atopic Dermatitis
Interventions
Blood pressure, Height, Weight, BMI, Waist Circumference
Other
Lead sponsor
Northwestern University
Other
Eligibility
5 Years to 17 Years
Enrollment
409 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2012
U.S. locations
9
States / cities
San Diego, California • San Francisco, California • Stanford, California + 6 more
Source: ClinicalTrials.gov public record
Updated Apr 14, 2015 · Synced Oct 29, 2026, 10:58 PM EDT
Recruiting Phase 2 Interventional

Alendronate for Osteonecrosis in Adults With Sickle Cell Disease

NCT06016634
View directory record Official record
Conditions
Sickle Cell Disease, Sickle Cell Anemia, Osteonecrosis, Ischemic Necrosis, Avascular Necrosis
Interventions
Alendronate Sodium
Drug
Lead sponsor
University of California, Davis
Other
Eligibility
18 Years to 80 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
1
States / cities
Sacramento, California
Source: ClinicalTrials.gov public record
Updated Jul 16, 2026 · Synced Oct 29, 2026, 10:58 PM EDT
Completed Phase 2 Interventional Results available

Open-Label Study of ZYN002 Administered as a Transdermal Gel to Children and Adolescents With 22q11.2 Deletion Syndrome (INSPIRE)

NCT05149898
View directory record Official record
Conditions
22Q11.2 Deletion Syndrome
Interventions
ZYN002
Drug
Lead sponsor
Harmony Biosciences Management, Inc.
Industry
Eligibility
4 Years to 17 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
1
States / cities
Greenville, South Carolina
Source: ClinicalTrials.gov public record
Updated Mar 9, 2026 · Synced Oct 29, 2026, 10:58 PM EDT
Completed No phase listed Observational

Adaptation Among Adolescents and Adults With Klinefelter Syndrome

NCT00896272
View directory record Official record
Conditions
Klinefelter Syndrome
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
14 Years to 80 Years · Male only
Enrollment
302 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2015
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 17, 2019 · Synced Oct 29, 2026, 10:58 PM EDT
Completed Phase 4 Interventional Results available

Late-Onset Treatment Study Extension Protocol

NCT00455195
View directory record Official record
Conditions
Pompe Disease (Late-Onset), Glycogen Storage Disease Type II (GSD-II), Glycogenesis Type II, Acid Maltase Deficiency (AMD)
Interventions
alglucosidase alfa
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
8 Years and older
Enrollment
81 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2008
U.S. locations
26
States / cities
Anchorage, Alaska • Phoenix, Arizona • Beverly Hills, California + 23 more
Source: ClinicalTrials.gov public record
Updated Mar 7, 2014 · Synced Oct 29, 2026, 10:58 PM EDT
Completed No phase listed Observational

Study of Kidney Tumors in Younger Patients

NCT00898365
View directory record Official record
Conditions
Adult Cystic Nephroma, Anaplastic Kidney Wilms Tumor, Angiolipoma, Cellular Congenital Mesoblastic Nephroma, Classic Congenital Mesoblastic Nephroma, Clear Cell Sarcoma of the Kidney, Congenital Mesoblastic Nephroma, Cystic Partially Differentiated Kidney Nephroblastoma, Diffuse Hyperplastic Perilobar Nephroblastomatosis, Extrarenal Rhabdoid Tumor, Kidney Medullary Carcinoma, Kidney Neoplasm, Kidney Oncocytoma, Kidney Wilms Tumor, Metanephric Adenofibroma, Metanephric Adenoma, Metanephric Stromal Tumor, Metanephric Tumor, Mixed Congenital Mesoblastic Nephroma, Ossifying Renal Tumor of Infancy, Papillary Renal Cell Carcinoma, Renal Cell Carcinoma, Renal Cell Carcinoma Associated With Xp11.2 Translocations/TFE3 Gene Fusions, Rhabdoid Tumor of the Kidney, Wilms Tumor
Interventions
Cytology Specimen Collection Procedure, Laboratory Biomarker Analysis
Other
Lead sponsor
Children's Oncology Group
Network
Eligibility
Up to 29 Years
Enrollment
8,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2026
U.S. locations
214
States / cities
Birmingham, Alabama • Mobile, Alabama • Anchorage, Alaska + 153 more
Source: ClinicalTrials.gov public record
Updated May 18, 2026 · Synced Oct 29, 2026, 10:58 PM EDT
Completed Phase 1 Interventional

A Phase 1 Study of Givosiran (ALN-AS1) in Patients With Acute Intermittent Porphyria (AIP)

NCT02452372
View directory record Official record
Conditions
Acute Intermittent Porphyria
Interventions
givosiran (ALN-AS1), Sterile Normal Saline (0.9% NaCl)
Drug
Lead sponsor
Alnylam Pharmaceuticals
Industry
Eligibility
18 Years to 65 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
4
States / cities
Birmingham, Alabama • San Francisco, California • New York, New York + 1 more
Source: ClinicalTrials.gov public record
Updated Jun 14, 2018 · Synced Oct 29, 2026, 10:58 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

Omega-3 Fatty Acids in Sickle Cell Disease

NCT02947100
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
SCD-Omegatex™
Drug
Lead sponsor
Robin E. Miller
Other
Eligibility
8 Years to 25 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018
U.S. locations
1
States / cities
Wilmington, Delaware
Source: ClinicalTrials.gov public record
Updated Oct 19, 2020 · Synced Oct 29, 2026, 10:58 PM EDT
Completed Phase 1 Interventional

A Safety, Pharmacokinetic and Pharmacodynamic Study of Kevetrin in Patients With Advanced Solid Tumors

NCT01664000
View directory record Official record
Conditions
Solid Tumors
Interventions
thioureidobutyronitrile
Drug
Lead sponsor
Cellceutix Corporation
Industry
Eligibility
18 Years and older
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Feb 24, 2016 · Synced Oct 29, 2026, 10:58 PM EDT
Terminated No phase listed Observational

Evaluation of Autonomic Function in Individuals With Cornelia de Lange Syndrome (CdLS)

NCT03113877
View directory record Official record
Conditions
Cornelia de Lange Syndrome
Interventions
Autonomic Dysfunction Testing
Other
Lead sponsor
Mayo Clinic
Other
Eligibility
8 Years to 18 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 6, 2018 · Synced Oct 29, 2026, 10:58 PM EDT
Completed Not applicable Interventional

Pilot Evaluation of the Cynosure Potenza™ System for Treatment of Cosmetic Dermatologic Skin Conditions

NCT05847530
View directory record Official record
Conditions
Wrinkles, Scars, Stretch Marks, Pigmentation, Skin Laxity
Interventions
Potenza, Icon
Device
Lead sponsor
Cynosure, Inc.
Industry
Eligibility
18 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
1
States / cities
San Diego, California
Source: ClinicalTrials.gov public record
Updated Aug 28, 2025 · Synced Oct 29, 2026, 10:58 PM EDT
Active, not recruiting Phase 3 Interventional

A Study to Test a Medicine (Fitusiran) Injected Under the Skin for Preventing Bleeding Episodes in Male Adolescent or Adult Participants With Severe Hemophilia

NCT05662319
View directory record Official record
Conditions
Hemophilia
Interventions
Fitusiran, Clotting factor concentrates (CFC) or bypassing agents (BPA), Antithrombin concentrate (ATIIIC)
Drug
Lead sponsor
Sanofi
Industry
Eligibility
12 Years and older · Male only
Enrollment
91 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2029
U.S. locations
8
States / cities
Orange, California • Minneapolis, Minnesota • Hackensack, New Jersey + 5 more
Source: ClinicalTrials.gov public record
Updated Feb 4, 2026 · Synced Oct 29, 2026, 10:58 PM EDT
Recruiting No phase listed Observational

Clinical and Basic Investigations Into Congenital Disorders of Glycosylation

NCT04199000
View directory record Official record
Conditions
Congenital Disorders of Glycosylation
Interventions
Not listed
Lead sponsor
Icahn School of Medicine at Mount Sinai
Other
Eligibility
Not listed
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2030
U.S. locations
12
States / cities
San Diego, California • Aurora, Colorado • Jacksonville, Florida + 9 more
Source: ClinicalTrials.gov public record
Updated Jul 21, 2026 · Synced Oct 29, 2026, 10:58 PM EDT
Completed No phase listed Observational

Biobanking of Rett Syndrome and Related Disorders

NCT02705677
View directory record Official record
Conditions
Rett Syndrome, MECP2 Duplication, CDKL5, FOXG1 Disorders
Interventions
Not listed
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
Not listed
Enrollment
752 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
12
States / cities
Birmingham, Alabama • Oakland, California • San Diego, California + 9 more
Source: ClinicalTrials.gov public record
Updated Aug 5, 2021 · Synced Oct 29, 2026, 10:58 PM EDT
Completed Phase 3 Interventional Results available

Rollover Study of VX-770 in Cystic Fibrosis Subjects

NCT01117012
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Ivacaftor
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
6 Years and older
Enrollment
192 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2014
U.S. locations
36
States / cities
Birmingham, Alabama • Oakland, California • Palo Alto, California + 33 more
Source: ClinicalTrials.gov public record
Updated Jul 7, 2015 · Synced Oct 29, 2026, 10:58 PM EDT
Completed Not applicable Interventional Results available

Innovation in the Treatment of Persistent Pain in Adults With NF1: Implementation of the iCanCope Mobile Application- Clinical Trial

NCT04561765
View directory record Official record
Conditions
Neurofibromatosis 1, Chronic Pain
Interventions
iCanCope, iCanCope+Contingency Management
Device
Lead sponsor
Yale University
Other
Eligibility
18 Years to 72 Years
Enrollment
122 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
1
States / cities
New Haven, Connecticut
Source: ClinicalTrials.gov public record
Updated Mar 30, 2025 · Synced Oct 29, 2026, 10:58 PM EDT
Completed Not applicable Interventional Results available

Impact of Discontinuing Dornase Alfa in People With CF on Highly Effective CFTR Modulator Therapy-A SIMPLIFY Sub-Study

NCT06350474
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Discontinuation of dornase alfa (Dnase), Continuation of dornase alfa (Dnase)
Other
Lead sponsor
Nicole Hamblett
Other
Eligibility
12 Years and older
Enrollment
477 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
81
States / cities
Birmingham, Alabama • Anchorage, Alaska • Tucson, Arizona + 69 more
Source: ClinicalTrials.gov public record
Updated Nov 26, 2024 · Synced Oct 29, 2026, 10:58 PM EDT
Terminated Phase 3 Interventional Results available

An Open-Label Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of OV101 in Individuals With Angelman Syndrome

NCT03882918
View directory record Official record
Conditions
Angelman Syndrome
Interventions
OV101
Drug
Lead sponsor
Healx AI
Industry
Eligibility
13 Years to 49 Years
Enrollment
141 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
10
States / cities
Phoenix, Arizona • San Diego, California • Atlanta, Georgia + 7 more
Source: ClinicalTrials.gov public record
Updated Jul 18, 2025 · Synced Oct 29, 2026, 10:58 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

Clinical Assessment of Skin Tightening and Contour Change of Submental Tissue Using Bipolar Radiofrequency Microneedling

NCT04477187
View directory record Official record
Conditions
Skin Laxity
Interventions
Dermal Handpiece
Device
Lead sponsor
University of Texas Southwestern Medical Center
Other
Eligibility
21 Years to 70 Years
Enrollment
15 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2022
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Dec 19, 2023 · Synced Oct 29, 2026, 10:58 PM EDT
Terminated No phase listed Observational

A Multi-Center Study of the CF Quantum® Sweat Test System

NCT02404714
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Iontophoresis sweat test
Device
Lead sponsor
Polychrome Medical, Inc.
Industry
Eligibility
Not listed
Enrollment
55 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2018
U.S. locations
6
States / cities
Birmingham, Alabama • Chicago, Illinois • Ann Arbor, Michigan + 3 more
Source: ClinicalTrials.gov public record
Updated Aug 31, 2018 · Synced Oct 29, 2026, 10:58 PM EDT
Completed Phase 3 Interventional Results available

Three Drug Combination Therapy Versus Conventional Treatment of Children With Congenital Adrenal Hyperplasia

NCT00001521
View directory record Official record
Conditions
Congenital Adrenal Hyperplasia (CAH)
Interventions
Fludrocortisone, Hydrocortisone, Letrozole, Flutamide, Testolactone
Drug
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
2 Years to 18 Years
Enrollment
66 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1995 – 2024
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 12, 2025 · Synced Oct 29, 2026, 10:58 PM EDT
Completed Phase 3 Interventional Results available

Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Recombinant Coagulation Factor VIII Fc Fusion Protein (rFVIIIFc) in Previously Treated Pediatric Subjects With Hemophilia A

NCT01458106
View directory record Official record
Conditions
Hemophilia A
Interventions
BIIB031 (rFVIIIFc), FVIII (PK subgroup only)
Drug
Lead sponsor
Bioverativ Therapeutics Inc.
Industry
Eligibility
Up to 11 Years · Male only
Enrollment
71 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
12
States / cities
Los Angeles, California • Sacramento, California • San Diego, California + 9 more
Source: ClinicalTrials.gov public record
Updated Dec 19, 2020 · Synced Oct 29, 2026, 10:58 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

Open Label, Study Of Efficacy and Safety Of AVR-RD-01 for Treatment-Naive Subjects With Classic Fabry Disease

NCT03454893
View directory record Official record
Conditions
Fabry Disease
Interventions
AVR-RD-01
Drug
Lead sponsor
AVROBIO
Industry
Eligibility
16 Years to 50 Years · Male only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
2
States / cities
Hackensack, New Jersey • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jan 5, 2024 · Synced Oct 29, 2026, 10:58 PM EDT