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ClinicalTrials.gov public records Last synced Oct 28, 2026, 5:06 AM EDT

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

Showing 1369–1392 of 8,011 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Genetics/Birth Defects Clear all
Recruiting No phase listed Observational Accepts healthy volunteers

Lung Disease and Its Affect on the Work of White Blood Cells in the Lungs

NCT01851642
View directory record Official record
Conditions
Alpha-1 Antitrypsin Deficiency, AAT Deficiency, AATD, Cystic Fibrosis (CF)
Interventions
History and physical exam., Blood draw., Pulmonary function testing., Albuterol inhaler.
Procedure · Drug
Lead sponsor
University of Florida
Other
Eligibility
18 Years and older
Enrollment
220 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2007 – 2033
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Nov 6, 2025 · Synced Oct 28, 2026, 5:06 AM EDT
Completed Phase 3 Interventional Results available

Prospective Biomarkers of Bone Metabolism in Hemophilia A

NCT02306694
View directory record Official record
Conditions
Hemophilia, Bone Disease
Interventions
Advate
Drug
Lead sponsor
Oregon Health and Science University
Other
Eligibility
16 Years to 85 Years · Male only
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Apr 1, 2020 · Synced Oct 28, 2026, 5:06 AM EDT
Completed Phase 1 Interventional

Placebo Controlled, Dose Escalation Study to Evaluate Safety, Pharmacokinetics & Efficacy of SAP-001 in Gout Patients

NCT03857165
View directory record Official record
Conditions
Gout, Hyperuricemia
Interventions
SAP-001
Drug
Lead sponsor
Shanton Pharma Co., Ltd.
Industry
Eligibility
18 Years to 75 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
3
States / cities
Anaheim, California • DeLand, Florida • High Point, North Carolina
Source: ClinicalTrials.gov public record
Updated Oct 14, 2021 · Synced Oct 28, 2026, 5:06 AM EDT
Active, not recruiting Phase 3 Interventional

Assessing Tenapanor as a Treatment of CF-related Constipation.

NCT06810167
View directory record Official record
Conditions
Cystic Fibrosis, Constipation
Interventions
Tenapanor
Drug
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years to 75 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Aug 21, 2026 · Synced Oct 28, 2026, 5:06 AM EDT
Completed Not applicable Interventional

Power Over Pain (POP) Study

NCT01800604
View directory record Official record
Conditions
Multiple Sclerosis, Spinal Cord Injury, Chronic Pain, Other and Unspecified Amputation Stump Complications, Muscular Dystrophies, Low Back Pain
Interventions
Pain Self-Management Intervention #1, Pain Self-Management Intervention #2, Pain Self-Management Intervention #3, Pain Self-Management Intervention #4
Behavioral
Lead sponsor
University of Washington
Other
Eligibility
18 Years and older
Enrollment
177 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2018
U.S. locations
1
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Oct 17, 2018 · Synced Oct 28, 2026, 5:06 AM EDT
Completed Phase 2Phase 3 Interventional Results available

Clinical Study Of caNNabidiol in childrEn and adolesCenTs With Fragile X (CONNECT-FX)

NCT03614663
View directory record Official record
Conditions
Fragile X Syndrome
Interventions
ZYN002 - Cannabidiol Transdermal Gel, Placebo Transdermal Gel
Drug · Other
Lead sponsor
Zynerba Pharmaceuticals, Inc.
Industry
Eligibility
3 Years to 17 Years
Enrollment
212 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
17
States / cities
Phoenix, Arizona • Sacramento, California • Denver, Colorado + 13 more
Source: ClinicalTrials.gov public record
Updated Jul 6, 2022 · Synced Oct 28, 2026, 5:06 AM EDT
Recruiting Phase 3 Interventional

Efficacy and Safety of CSL222 (Etranacogene Dezaparvovec) Gene Therapy in Adults With Hemophilia B With Pretreatment Adeno-associated Virus Serotype 5 (AAV5) Neutralizing Antibodies (Nabs)

NCT06003387
View directory record Official record
Conditions
Hemophilia B
Interventions
CSL222 (AAV5-hFIXco-Padua)
Genetic
Lead sponsor
CSL Behring
Industry
Eligibility
18 Years and older
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2032
U.S. locations
4
States / cities
San Diego, California • Ann Arbor, Michigan • Pittsburgh, Pennsylvania + 1 more
Source: ClinicalTrials.gov public record
Updated Aug 27, 2026 · Synced Oct 28, 2026, 5:06 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Reducing HIV Risk With High Risk HIV Negative Black MSM-Passport to Wellness

NCT02932384
View directory record Official record
Conditions
HIV, Men
Interventions
Intervention-Passport and Peer Mentor, Control-Passport Only
Behavioral
Lead sponsor
Charles Drew University of Medicine and Science
Other
Eligibility
18 Years and older · Male only
Enrollment
105 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2018
U.S. locations
2
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Aug 2, 2018 · Synced Oct 28, 2026, 5:06 AM EDT
Active, not recruiting Phase 2 Interventional

Study of UC-MSC in DMD Patients

NCT06579352
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Human Umbilical Cord Mesenchymal Stem Cells (UC-MSC)
Biological
Lead sponsor
MED Institute Inc.
Industry
Eligibility
5 Years to 10 Years · Male only
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2029
U.S. locations
2
States / cities
Dallas, Texas • Flower Mound, Texas
Source: ClinicalTrials.gov public record
Updated Aug 14, 2026 · Synced Oct 28, 2026, 5:06 AM EDT
Completed Phase 1 Interventional

A Study To Assess The Tolerability And Clinical Activity Of Gedatolisib In Combination With Palbociclib/Letrozole Or Palbociclib/Fulvestrant In Women With Metastatic Breast Cancer

NCT02684032
View directory record Official record
Conditions
Breast Cancer
Interventions
Gedatolisib, Palbociclib, Letrozole, Fulvestrant
Drug
Lead sponsor
Celcuity Inc
Industry
Eligibility
18 Years and older · Female only
Enrollment
141 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2022
U.S. locations
41
States / cities
Birmingham, Alabama • Corona, California • Fountain Valley, California + 17 more
Source: ClinicalTrials.gov public record
Updated Jul 27, 2022 · Synced Oct 28, 2026, 5:06 AM EDT
Completed No phase listed Observational

Genetic Regulation of Surfactant Deficiency

NCT00828243
View directory record Official record
Conditions
Respiratory Distress Syndrome, Newborn
Interventions
Nutrient
Drug
Lead sponsor
Washington University School of Medicine
Other
Eligibility
1 Day to 6 Months
Enrollment
525 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2013
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Jun 7, 2021 · Synced Oct 28, 2026, 5:06 AM EDT
Completed Not applicable Interventional

Auditory Neural Function in Implanted Patients With Usher Syndrome

NCT04906135
View directory record Official record
Conditions
Usher Syndrome, Cochlear Implantation
Interventions
Experimental manipulation of stimulation parameters
Other
Lead sponsor
Ohio State University
Other
Eligibility
1 Year to 85 Years
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
4
States / cities
Boston, Massachusetts • Columbus, Ohio • Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Jul 29, 2025 · Synced Oct 28, 2026, 5:06 AM EDT
Completed Phase 1Phase 2 Interventional

A Treatment Study of Mucopolysaccharidosis Type IIIB

NCT02754076
View directory record Official record
Conditions
MPS III B, Mucopolysaccharidosis Type IIIB
Interventions
AX 250
Drug
Lead sponsor
Allievex Corporation
Industry
Eligibility
1 Year to 10 Years
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2020
U.S. locations
1
States / cities
Oakland, California
Source: ClinicalTrials.gov public record
Updated Aug 6, 2020 · Synced Oct 28, 2026, 5:06 AM EDT
Completed Phase 3 Interventional

INcreased Sun Exposure Without Pain In Research Participants With EPP or XLP

NCT06144840
View directory record Official record
Conditions
Erythropoietic Protoporphyria (EPP), X-Linked Protoporphyria (XLP)
Interventions
Dersimelagon, Placebo
Drug
Lead sponsor
Tanabe Pharma America, Inc.
Industry
Eligibility
12 Years to 75 Years
Enrollment
165 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
13
States / cities
Huntington Beach, California • San Francisco, California • Miami, Florida + 10 more
Source: ClinicalTrials.gov public record
Updated Jun 4, 2026 · Synced Oct 28, 2026, 5:06 AM EDT
Completed Phase 3 Interventional

This Study is to Assess the Efficacy of Difamilast Ointment in Mild to Moderate Atopic Dermatitis(AD)

NCT05608343
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
Difamilast, Placebo
Drug
Lead sponsor
Acrotech Biopharma Inc.
Industry
Eligibility
2 Years and older
Enrollment
153 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2023
U.S. locations
39
States / cities
Birmingham, Alabama • Phoenix, Arizona • Jonesboro, Arkansas + 36 more
Source: ClinicalTrials.gov public record
Updated Aug 15, 2024 · Synced Oct 28, 2026, 5:06 AM EDT
Completed Phase 1 Interventional

Safety, Efficacy, and Tolerability of BOS172738 in Patients With Advanced Rearranged During Transfection (RET) Gene-Altered Tumors

NCT03780517
View directory record Official record
Conditions
Advanced Nonhaematologic Malignancies
Interventions
BOS172738
Drug
Lead sponsor
Boston Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
117 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
2
States / cities
Philadelphia, Pennsylvania • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Oct 30, 2023 · Synced Oct 28, 2026, 5:06 AM EDT
Completed Phase 1Phase 2 Interventional Results available

FRDA Investigator Initiated Study (IIS) With Elamipretide

NCT05168774
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Elamipretide
Drug
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
16 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Dec 12, 2025 · Synced Oct 28, 2026, 5:06 AM EDT
Completed Not applicable Interventional Results available

Rollover Study to Evaluate Histological Results of Radiofrequency Device Treatments on the Flanks

NCT04881149
View directory record Official record
Conditions
Skin Laxity
Interventions
TempSure treatment
Device
Lead sponsor
Cynosure, Inc.
Industry
Eligibility
18 Years to 55 Years
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 1, 2022 · Synced Oct 28, 2026, 5:06 AM EDT
Completed Phase 4 Interventional

Bilateral Comparison Study of Elidel 1% and Hylatopic Plus Emollient Foam for the Treatment of Subjects With Atopic Dermatitis

NCT01202149
View directory record Official record
Conditions
Eczema, Atopic Dermatitis
Interventions
Elidel (pimecrolimus cream, 1%) Hylatopic Plus Emollient Foam (non-medicated device)
Device
Lead sponsor
Frankel, Amylynne, M.D.
Other
Eligibility
2 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Apr 15, 2011 · Synced Oct 28, 2026, 5:06 AM EDT
Active, not recruiting No phase listed Observational

Non-interventional Long Term Follow-up Study of Participants Previously Enrolled in the STARLIGHT Study

NCT06048185
View directory record Official record
Conditions
Stargardt Disease
Interventions
Gene Therapy Product-MCO-010
Biological
Lead sponsor
Nanoscope Therapeutics Inc.
Industry
Eligibility
16 Years and older
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
2
States / cities
Miami, Florida • McAllen, Texas
Source: ClinicalTrials.gov public record
Updated Mar 24, 2025 · Synced Oct 28, 2026, 5:06 AM EDT
Completed Phase 3 Interventional Results available

LR769 in Congenital Hemophilia Patients With Inhibitors Undergoing Elective Surgery or Invasive Procedures

NCT02548143
View directory record Official record
Conditions
Hemophilia
Interventions
Coagulation Factor VIIa (Recombinant)
Biological
Lead sponsor
Laboratoire français de Fractionnement et de Biotechnologies
Industry
Eligibility
6 Months to 75 Years · Male only
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Feb 25, 2022 · Synced Oct 28, 2026, 5:06 AM EDT
Completed No phase listed Observational

Brain-Behavior Interactions in Tic Suppression

NCT02205918
View directory record Official record
Conditions
Tourette's Syndrome, Chronic Motor Tic Disorder, Chronic Vocal Tic Disorder
Interventions
Active TMS, Sham TMS
Other
Lead sponsor
University of Minnesota
Other
Eligibility
12 Years to 18 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jul 17, 2020 · Synced Oct 28, 2026, 5:06 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

Cancer Health Assessments Reaching Many

NCT03426878
View directory record Official record
Conditions
Hereditary Cancer Syndrome
Interventions
Modified genetic counseling, Traditional genetic counseling
Other
Lead sponsor
Kaiser Permanente
Other
Eligibility
18 Years to 49 Years
Enrollment
967 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2022
U.S. locations
2
States / cities
Denver, Colorado • Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Sep 11, 2025 · Synced Oct 28, 2026, 5:06 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Myotonic Dystrophy and Facioscapulohumeral Muscular Dystrophy Registry

NCT00082108
View directory record Official record
Conditions
Myotonic Dystrophy, Facioscapulohumeral Muscular Dystrophy, Muscular Dystrophy, Myotonic Dystrophy Type 1, Myotonic Dystrophy Type 2, Congenital Myotonic Dystrophy, PROMM (Proximal Myotonic Myopathy), Steinert's Disease, Myotonic Muscular Dystrophy
Interventions
Not listed
Lead sponsor
University of Rochester
Other
Eligibility
Not listed
Enrollment
3,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2000 – 2028
U.S. locations
1
States / cities
Rochester, New York
Source: ClinicalTrials.gov public record
Updated Oct 15, 2025 · Synced Oct 28, 2026, 5:06 AM EDT