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ClinicalTrials.gov public records Last synced Oct 20, 2026, 1:00 AM EDT

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

Showing 73–96 of 8,004 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Genetics/Birth Defects Clear all
Completed Not applicable Interventional

A New Diet for Patients With Autosomal Dominant Polycystic Disease (ADPKD)

NCT01810614
View directory record Official record
Conditions
Autosomal Dominant Polycystic Kidney Disease
Interventions
ADPKD Diet
Other
Lead sponsor
University of Kansas
Other
Eligibility
18 Years to 65 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
1
States / cities
Kansas City, Kansas
Source: ClinicalTrials.gov public record
Updated Aug 13, 2015 · Synced Oct 20, 2026, 1:00 AM EDT
Completed No phase listed Observational

Neonatal CMV-Ganciclovir Follow-up Study

NCT00031421
View directory record Official record
Conditions
Cytomegalovirus Infections
Interventions
Not listed
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
9 Years to 14 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2005
U.S. locations
1
States / cities
Birmingham, Alabama
Source: ClinicalTrials.gov public record
Updated Aug 12, 2011 · Synced Oct 20, 2026, 1:00 AM EDT
Completed Phase 2 Interventional Results available

Bone Marrow Transplantation in Young Adults With Severe Sickle Cell Disease

NCT01565616
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Conditioning Regimen with Bone Marrow Transplant
Drug
Lead sponsor
Emory University
Other
Eligibility
16 Years to 40 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
8
States / cities
Oakland, California • Washington D.C., District of Columbia • Miami, Florida + 4 more
Source: ClinicalTrials.gov public record
Updated Nov 21, 2017 · Synced Oct 20, 2026, 1:00 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Treatment for Improvement of Cellulite Appearance Using Form Applicator

NCT05882721
View directory record Official record
Conditions
Cellulite
Interventions
Sofwave
Device
Lead sponsor
Sofwave Medical LTD
Industry
Eligibility
18 Years to 60 Years · Female only
Enrollment
60 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023
U.S. locations
5
States / cities
Sacramento, California • Hackensack, New Jersey • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Nov 18, 2023 · Synced Oct 20, 2026, 1:00 AM EDT
Completed Phase 1 Interventional

Phase I Trial of Tariquidar (XR9576) in Combination With Doxorubicin, Vinorelbine, or Docetaxel in Pediatric Patients With Solid Tumors

NCT00011414
View directory record Official record
Conditions
Wilms' Tumor, Sarcoma, Adenaocortical Carcinoma, Refractory Cancer, Coldrhood Cancer
Interventions
Tariquidar
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
2 Years to 18 Years
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2016
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 16, 2019 · Synced Oct 20, 2026, 1:00 AM EDT
Completed Phase 2 Interventional Results available

A Vehicle Controlled Study to Evaluate Safety and Efficacy of Topical TMB-001 for Treatment of Congenital Ichthyosis

NCT04154293
View directory record Official record
Conditions
Congenital Ichthyosis
Interventions
Isotretinoin, Vehicle
Drug · Other
Lead sponsor
LEO Pharma
Industry
Eligibility
9 Years and older
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
8
States / cities
Phoenix, Arizona • Palo Alto, California • New Haven, Connecticut + 5 more
Source: ClinicalTrials.gov public record
Updated May 22, 2026 · Synced Oct 20, 2026, 1:00 AM EDT
Recruiting Not applicable Interventional

Delivering Patient-Facing Evidence-Based Guidelines Through mHealth to Adults With Sickle Cell Disease

NCT06919224
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
mHealth app + booklets
Other
Lead sponsor
Ohio State University
Other
Eligibility
18 Years and older
Enrollment
287 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2032
U.S. locations
4
States / cities
Chicago, Illinois • St Louis, Missouri • Columbus, Ohio + 1 more
Source: ClinicalTrials.gov public record
Updated Mar 23, 2026 · Synced Oct 20, 2026, 1:00 AM EDT
Completed Phase 1 Interventional

A Study Evaluating the Safety, Pharmacokinetics, Pharmacodynamics and Efficacy of Crovalimab for the Management of Acute Uncomplicated Vaso-Occlusive Episodes (VOE) in Participants With Sickle Cell Disease (SCD).

NCT04912869
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Crovalimab, Placebo
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
12 Years to 55 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
2
States / cities
Atlanta, Georgia • New York, New York
Source: ClinicalTrials.gov public record
Updated Aug 26, 2025 · Synced Oct 20, 2026, 1:00 AM EDT
Recruiting Phase 3 Interventional

Clinical Trial to Compare the Efficacy of Celiprolol to Placebo in Patients With Vascular Ehlers-Danlos Syndrome

NCT05432466
View directory record Official record
Conditions
Vascular Ehlers-Danlos Syndrome
Interventions
ACER-002 (celiprolol) 200 mg BID, Placebo BID
Drug
Lead sponsor
Acer Therapeutics Inc.
Industry
Eligibility
15 Years to 64 Years
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2029
U.S. locations
1
States / cities
Culver City, California
Source: ClinicalTrials.gov public record
Updated Aug 24, 2025 · Synced Oct 20, 2026, 1:00 AM EDT
Recruiting Phase 4 Interventional

Rollover Study for Patients With Sickle Cell Disease Who Have Completed a Prior Novartis-Sponsored Crizanlizumab Study

NCT04657822
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Crizanlizumab
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
6 Months to 100 Years
Enrollment
130 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2031
U.S. locations
7
States / cities
Birmingham, Alabama • Washington D.C., District of Columbia • Augusta, Georgia + 3 more
Source: ClinicalTrials.gov public record
Updated Jun 8, 2026 · Synced Oct 20, 2026, 1:00 AM EDT
Enrolling by invitation Phase 3 Interventional

Global Open-Label Extension Study of Del-desiran for the Treatment of DM1

NCT07008469
View directory record Official record
Conditions
Myotonic Dystrophy Type 1, DM1, Myotonic Dystrophy, Myotonia, Myotonic Dystrophy 1, Myotonic Disorders, Steinert Myotonic Dystrophy, Steinert Disease
Interventions
Del-desiran (AOC 1001)
Drug
Lead sponsor
Avidity Biosciences, Inc.
Industry
Eligibility
16 Years and older
Enrollment
199 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
19
States / cities
Los Angeles, California • Stanford, California • Denver, Colorado + 16 more
Source: ClinicalTrials.gov public record
Updated Aug 19, 2026 · Synced Oct 20, 2026, 1:00 AM EDT
Completed Phase 1 Interventional

Safety Study of Human Recombinant Tissue Non-Specific Alkaline Phosphatase Fusion Protein Asfotase Alfa in Adults With Hypophosphatasia (HPP)

NCT00739505
View directory record Official record
Conditions
Hypophosphatasia (HPP)
Interventions
Asfotase Alfa
Biological
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
18 Years to 80 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2009
U.S. locations
2
States / cities
St Louis, Missouri • Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Mar 29, 2019 · Synced Oct 20, 2026, 1:00 AM EDT
Recruiting No phase listed Observational

Clinical and Basic Investigations Into Hermansky-Pudlak Syndrome

NCT00001456
View directory record Official record
Conditions
Hermansky-Pudlak Syndrome (HPS)
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
1 Month to 115 Years
Enrollment
600 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1995
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 13, 2026 · Synced Oct 20, 2026, 1:00 AM EDT
Recruiting Not applicable Interventional Accepts healthy volunteers

Identifying and Caring for Individuals With Inherited Cancer Syndrome

NCT04494945
View directory record Official record
Conditions
BRCA1/2-Associated Hereditary Breast and Ovarian Cancer Syndrome, Breast Ductal Carcinoma In Situ, Hematopoietic and Lymphoid System Neoplasm, Hereditary Neoplastic Syndrome, Lynch Syndrome, Malignant Solid Neoplasm
Interventions
Biospecimen Collection, Genetic Counseling, Genetic Testing, Survey Administration
Procedure · Other
Lead sponsor
OHSU Knight Cancer Institute
Other
Eligibility
18 Years and older
Enrollment
27,500 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2027
U.S. locations
2
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Jul 14, 2026 · Synced Oct 20, 2026, 1:00 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 20, 2026, 1:00 AM EDT
Terminated Not applicable Interventional Accepts healthy volunteers Results available

Bone Mineral Density in Adults With Hyperphenylalaninemia on Kuvan Therapy

NCT01541397
View directory record Official record
Conditions
Hyperphenylalaninemia, Phenylketonuria
Interventions
Sapropterin
Drug
Lead sponsor
The University of Texas Health Science Center, Houston
Other
Eligibility
18 Years to 50 Years
Enrollment
6 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2012
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jul 7, 2015 · Synced Oct 20, 2026, 1:00 AM EDT
Recruiting Phase 4 Interventional

Clinical and Biomarker Effects of Depot Medroxyprogesterone Acetate in Females With Sickle Cell Disease

NCT06665997
View directory record Official record
Conditions
Sickle Cell Disease (SCD), Vaso-Occlusive Pain Episode in Sickle Cell Disease
Interventions
Depot medroxyprogesterone acetate (DMPA)
Drug
Lead sponsor
University of Pennsylvania
Other
Eligibility
18 Years to 50 Years · Female only
Enrollment
65 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
2
States / cities
Atlanta, Georgia • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated May 28, 2026 · Synced Oct 20, 2026, 1:00 AM EDT
Recruiting No phase listed Observational

FITting Non-invasive Tests in Lynch Syndrome Surveillance

NCT06898996
View directory record Official record
Conditions
Colorectal Cancer
Interventions
Fecal immunochemical test
Other
Lead sponsor
University of Chicago
Other
Eligibility
20 Years to 80 Years
Enrollment
400 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2029
U.S. locations
3
States / cities
Chicago, Illinois • Ann Arbor, Michigan • New York, New York
Source: ClinicalTrials.gov public record
Updated Apr 16, 2026 · Synced Oct 20, 2026, 1:00 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 20, 2026, 1:00 AM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Amyloid Plaque and Tangle Imaging in Alzheimer's Disease and Down Syndrome

NCT00965835
View directory record Official record
Conditions
Down Syndrome, Alzheimer's Disease
Interventions
Not listed
Lead sponsor
National Institute on Aging (NIA)
NIH
Eligibility
21 Years and older
Enrollment
40 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2011
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Dec 29, 2009 · Synced Oct 20, 2026, 1:00 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Detecting Dopaminergic Deficits in Individuals At-risk for Parkinsonism

NCT01358474
View directory record Official record
Conditions
Parkinson Disease, Gaucher Disease, Idiopathic Rapid Eye Movement Sleep Disorder
Interventions
Not listed
Lead sponsor
University of Minnesota
Other
Eligibility
18 Years to 85 Years
Enrollment
56 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2018
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Apr 18, 2019 · Synced Oct 20, 2026, 1:00 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Angelman Syndrome Video Assessment (ASVA) Source Material Study

NCT05637697
View directory record Official record
Conditions
Angelman Syndrome
Interventions
Not listed
Lead sponsor
The Emmes Company, LLC
Industry
Eligibility
1 Year and older
Enrollment
73 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2023
U.S. locations
1
States / cities
Kingston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Dec 13, 2023 · Synced Oct 20, 2026, 1:00 AM EDT
Completed Not applicable Interventional

The Impact of Exercise on Stress, Fatigue, and Quality of Life in Individuals With Primary Immunodeficiency Disease

NCT03211689
View directory record Official record
Conditions
Primary Immune Deficiency Disorder, Common Variable Immunodeficiency, Specific Antibody Deficiency, Hypergammaglobulinemia
Interventions
Exercise Program
Other
Lead sponsor
Stockton University
Other
Eligibility
18 Years and older
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017
U.S. locations
1
States / cities
Galloway, New Jersey
Source: ClinicalTrials.gov public record
Updated Sep 6, 2018 · Synced Oct 20, 2026, 1:00 AM EDT
Completed Phase 1 Interventional

Safety and Tolerability Study of rAvPAL-PEG to Treat Phenylketonuria

NCT00634660
View directory record Official record
Conditions
Phenylketonuria
Interventions
rAvPAL-PEG
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
16 Years to 50 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2009
U.S. locations
8
States / cities
Chicago, Illinois • Minneapolis, Minnesota • St Louis, Missouri + 5 more
Source: ClinicalTrials.gov public record
Updated Feb 1, 2017 · Synced Oct 20, 2026, 1:00 AM EDT