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ClinicalTrials.gov public records Last synced Oct 21, 2026, 7:23 AM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 337–360 of 8,005 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Genetics/Birth Defects Clear all
Recruiting No phase listed Observational

Avalglucosidase Alfa Pregnancy Study

NCT05734521
View directory record Official record
Conditions
Pompe Disease, Pregnancy
Interventions
avalglucosidase alfa-NGPT (GZ402666) IV, avalglucosidase alfa-NGPT (GZ402666)
Biological
Lead sponsor
Sanofi
Industry
Eligibility
Female only
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2032
U.S. locations
1
States / cities
Bridgewater, New Jersey
Source: ClinicalTrials.gov public record
Updated Jan 16, 2026 · Synced Oct 21, 2026, 7:23 AM EDT
Completed Phase 1 Interventional

Safety and Biomarker Response to (+)-Epicatechin in Becker Muscular Dystrophy

NCT04386304
View directory record Official record
Conditions
Becker Muscular Dystrophy
Interventions
(+)-Epicatechin
Drug
Lead sponsor
Epirium Bio Inc.
Industry
Eligibility
16 Years to 59 Years · Male only
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
3
States / cities
Los Angeles, California • Sacramento, California • St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Mar 23, 2022 · Synced Oct 21, 2026, 7:23 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

Lifting and Tightening of the Face and Neck Following an Increased Density Treatment

NCT01519206
View directory record Official record
Conditions
Skin Laxity
Interventions
Ulthera System Treatment
Device
Lead sponsor
Ulthera, Inc
Industry
Eligibility
30 Years to 65 Years
Enrollment
32 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2013
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Dec 11, 2017 · Synced Oct 21, 2026, 7:23 AM EDT
Completed Early Phase 1 Interventional

Angelman Syndrome (AS) Biomarker Study

NCT04103333
View directory record Official record
Conditions
Angelman Syndrome, Chromosome 15q Duplication (dup15q) Syndrome
Interventions
Lumbar Puncture, Blood Collection
Procedure
Lead sponsor
Biogen
Industry
Eligibility
Up to 50 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
5
States / cities
San Diego, California • Chicago, Illinois • Boston, Massachusetts + 2 more
Source: ClinicalTrials.gov public record
Updated Jan 28, 2022 · Synced Oct 21, 2026, 7:23 AM EDT
Withdrawn Phase 4 Interventional

Epidemiology and Treatment of Small-colony Variant Staphylococcus Aureus in Cystic Fibrosis

NCT02547116
View directory record Official record
Conditions
Cystic Fibrosis, MRSA
Interventions
Rifampin
Drug
Lead sponsor
Johns Hopkins University
Other
Eligibility
12 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jan 14, 2021 · Synced Oct 21, 2026, 7:23 AM EDT
Completed Not applicable Interventional Results available

Transcranial Magnetic Stimulation for Facial Pain

NCT02633306
View directory record Official record
Conditions
Trigeminal Nerve Injuries, Post-herpetic Neuralgia, Facial Pain, Nervus Intermedius Neuralgia
Interventions
transcranial magnetic stimulation
Device
Lead sponsor
University of Minnesota
Other
Eligibility
18 Years to 110 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Apr 8, 2021 · Synced Oct 21, 2026, 7:23 AM EDT
Terminated Phase 2 Interventional Results available

Cannabidiol Oral Solution for the Treatment of Patients With Prader-Willi Syndrome

NCT02844933
View directory record Official record
Conditions
Prader-Willi Syndrome
Interventions
Cannabidiol, Placebo
Drug
Lead sponsor
Radius Pharmaceuticals, Inc.
Industry
Eligibility
8 Years to 17 Years
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
7
States / cities
Tucson, Arizona • San Diego, California • Iowa City, Iowa + 4 more
Source: ClinicalTrials.gov public record
Updated Aug 1, 2023 · Synced Oct 21, 2026, 7:23 AM EDT
Completed Phase 3 Interventional Results available

Combination Chemotherapy With or Without Radiation Therapy in Treating Young Patients With Newly Diagnosed Stage III or Stage IV Wilms' Tumor

NCT00379340
View directory record Official record
Conditions
Stage III Kidney Wilms Tumor, Stage IV Kidney Wilms Tumor
Interventions
3-Dimensional Conformal Radiation Therapy, Conventional Surgery, Cyclophosphamide, Dactinomycin, Doxorubicin Hydrochloride, Etoposide, Vincristine Sulfate Liposome
Radiation · Procedure · Drug + 1 more
Lead sponsor
Children's Oncology Group
Network
Eligibility
Up to 29 Years
Enrollment
395 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2026
U.S. locations
199
States / cities
Birmingham, Alabama • Mobile, Alabama • Phoenix, Arizona + 143 more
Source: ClinicalTrials.gov public record
Updated Jul 24, 2026 · Synced Oct 21, 2026, 7:23 AM EDT
Not listed No phase listed Observational

Study of Systemic Amyloidosis Presentation and Prognosis

NCT00004374
View directory record Official record
Conditions
Amyloidosis
Interventions
Not listed
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
0 Years and older
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1979
U.S. locations
1
States / cities
Indianapolis, Indiana
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 21, 2026, 7:23 AM EDT
Completed Not applicable Interventional Results available

Patient-Provider Tools to Improve the Transition to Adult Care in Sickle Cell Disease

NCT02200510
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Self-management intervention for Adolescents with SCD, Patient Portal Intervention for Adolescents with SCD
Behavioral
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
13 Years to 24 Years
Enrollment
78 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2015
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Jun 15, 2018 · Synced Oct 21, 2026, 7:23 AM EDT
Recruiting Not applicable Interventional

Complex Aortic Aneurysm Repair Using Physician Modified Endografts and Custom Made Devices

NCT02050113
View directory record Official record
Conditions
Complex Aortic Aneurysms, Thoracoabdominal Aneurysms, Pararenal Aneurysms, Juxtarenal Aneurysms, Marfan Syndrome, Ehlers-Danlos Syndrome, Loeys-Dietz Syndrome, Aortic Arch Aneurysm
Interventions
Custom Made Fenestrated or Branched Stent Graft
Device
Lead sponsor
Andres Schanzer
Other
Eligibility
18 Years and older
Enrollment
600 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2030
U.S. locations
1
States / cities
Worcester, Massachusetts
Source: ClinicalTrials.gov public record
Updated Oct 22, 2025 · Synced Oct 21, 2026, 7:23 AM EDT
Completed No phase listed Observational

A Study of the Epidemiology and Hospital Management of Patients With PROS in France

NCT07222423
View directory record Official record
Conditions
PIK3CA-Related Overgrowth Spectrum
Interventions
Not listed
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years and older
Enrollment
3,605 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024
U.S. locations
1
States / cities
East Hanover, New Jersey
Source: ClinicalTrials.gov public record
Updated Oct 29, 2025 · Synced Oct 21, 2026, 7:23 AM EDT
Recruiting No phase listed Observational

Krabbe Disease Global Patient Registry

NCT02993796
View directory record Official record
Conditions
Krabbe Disease
Interventions
Not listed
Lead sponsor
State University of New York at Buffalo
Other
Eligibility
Not listed
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2026
U.S. locations
1
States / cities
Buffalo, New York
Source: ClinicalTrials.gov public record
Updated Jul 17, 2026 · Synced Oct 21, 2026, 7:23 AM EDT
Completed Phase 2 Interventional Results available

EMPIRE CF: A Phase 2 Study to Evaluate the Efficacy, Safety, and Tolerability of CTX-4430 in Adult Cystic Fibrosis (CF) Patients

NCT02443688
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
CTX-4430, Placebo
Drug
Lead sponsor
Celtaxsys, Inc.
Other
Eligibility
18 Years to 30 Years
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
39
States / cities
Birmingham, Alabama • Anchorage, Alaska • Tucson, Arizona + 34 more
Source: ClinicalTrials.gov public record
Updated Sep 4, 2019 · Synced Oct 21, 2026, 7:23 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Airway Alkalinization and Nasal Colonization

NCT03078088
View directory record Official record
Conditions
Healthy Subjects, Cystic Fibrosis
Interventions
Tham, Saline
Drug
Lead sponsor
Lakshmi Durairaj
Other
Eligibility
16 Years and older
Enrollment
32 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2019
U.S. locations
1
States / cities
Iowa City, Iowa
Source: ClinicalTrials.gov public record
Updated Mar 30, 2020 · Synced Oct 21, 2026, 7:23 AM EDT
Terminated Not applicable Interventional Results available

Designing an Implementation Strategy for Delivering Routine Mental Health Screening and Treatment

NCT04587661
View directory record Official record
Conditions
Sickle Cell Disease, Depression, Anxiety
Interventions
off-the-shelf digital CBT, adapted digital CBT
Behavioral
Lead sponsor
University of Pittsburgh
Other
Eligibility
16 Years to 35 Years
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 30, 2024 · Synced Oct 21, 2026, 7:23 AM EDT
Active, not recruiting Phase 4 Interventional

Treating Leg Symptoms in Women With X-linked Adrenoleukodystrophy

NCT05003648
View directory record Official record
Conditions
Adrenoleukodystrophy, Restless Legs Syndrome
Interventions
Pramipexole, Placebo
Drug
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years to 75 Years · Female only
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jul 1, 2026 · Synced Oct 21, 2026, 7:23 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

Dasatinib, Ifosfamide, Carboplatin, and Etoposide in Treating Young Patients With Metastatic or Recurrent Malignant Solid Tumors

NCT00788125
View directory record Official record
Conditions
Brain and Central Nervous System Tumors, Childhood Germ Cell Tumor, Extragonadal Germ Cell Tumor, Kidney Cancer, Liver Cancer, Lymphoma, Neuroblastoma, Ovarian Cancer, Sarcoma, Testicular Germ Cell Tumor, Unspecified Childhood Solid Tumor, Protocol Specific
Interventions
carboplatin, dasatinib, etoposide phosphate, ifosfamide, microarray analysis, western blotting, immunohistochemistry staining method, laboratory biomarker analysis, therapeutic conventional surgery, radiation therapy
Drug · Genetic · Other + 2 more
Lead sponsor
City of Hope Medical Center
Other
Eligibility
1 Year to 25 Years
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2022
U.S. locations
1
States / cities
Duarte, California
Source: ClinicalTrials.gov public record
Updated Jul 14, 2023 · Synced Oct 21, 2026, 7:23 AM EDT
Completed No phase listed Observational

LTFU for Gene Transfer Subjects With Hemophilia B

NCT00515710
View directory record Official record
Conditions
Hemophilia B
Interventions
Not listed
Lead sponsor
Spark Therapeutics, Inc.
Industry
Eligibility
18 Years and older · Male only
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2017
U.S. locations
2
States / cities
Philadelphia, Pennsylvania • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated May 15, 2020 · Synced Oct 21, 2026, 7:23 AM EDT
Active, not recruiting Not applicable Interventional

Promoting Goals-of-Care Discussions for Patients With Memory Problems and Their Caregivers

NCT05596760
View directory record Official record
Conditions
Dementia, Dementia, Vascular, Mixed Dementias, Alzheimer Disease, Huntington Disease, Lewy Body Disease, Creutzfeldt-Jakob Syndrome, Frontotemporal Lobar Degeneration, Cognitive Dysfunction, Cognitive Aging, Chronic Disease, Palliative Care, Patient Care, Health Care Quality, Access, and Evaluation, Patient Care, Health Communication, Patient Care Planning, Quality of Life
Interventions
Jumpstart Guide
Behavioral
Lead sponsor
University of Washington
Other
Eligibility
18 Years and older
Enrollment
1,802 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
3
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Sep 21, 2026 · Synced Oct 21, 2026, 7:23 AM EDT
Recruiting No phase listed Observational

Detection and Characterization of Infections and Infection Susceptibility

NCT00404560
View directory record Official record
Conditions
Immune Deficiencies
Interventions
Not listed
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
1 Month to 100 Years
Enrollment
2,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2007
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 13, 2026 · Synced Oct 21, 2026, 7:23 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Resistance Exercise in Barth Syndrome

NCT01629459
View directory record Official record
Conditions
Barth Syndrome
Interventions
Resistance exercise and protein supplementation
Behavioral
Lead sponsor
Washington University School of Medicine
Other
Eligibility
15 Years to 35 Years · Male only
Enrollment
12 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2019
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated May 13, 2020 · Synced Oct 21, 2026, 7:23 AM 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
253 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 Oct 14, 2026 · Synced Oct 21, 2026, 7:23 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Study to Evaluate Safety and Tolerability of BMN 111 Administered to Healthy Adult Volunteers

NCT01590446
View directory record Official record
Conditions
Achondroplasia
Interventions
BMN 111, Normal Saline
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
22 Years to 45 Years · Male only
Enrollment
74 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012
U.S. locations
1
States / cities
Evansville, Indiana
Source: ClinicalTrials.gov public record
Updated Jun 11, 2012 · Synced Oct 21, 2026, 7:23 AM EDT