Search | Clinical Trials Finder

These results were retrieved from the live ClinicalTrials.gov registry. Filters and sort order apply to this result list.

ClinicalTrials.gov public records Last synced Oct 21, 2026, 3:37 PM EDT

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

Showing 433–456 of 8,007 matching trials from the live ClinicalTrials.gov search.

Filters: Condition: Genetics/Birth Defects Clear all

Conditions: Congenital Myotonic Dystrophy
Interventions: Natural history; Other
Lead sponsor: Virginia Commonwealth University; Other
Eligibility: 0 Years to 15 Years
Enrollment: 100 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2016 – 2025
U.S. locations: 1
States / cities: Richmond, Virginia

Conditions: COPD, Cystic Fibrosis, Pulmonary Fibrosis
Interventions: Plerixafor mobilization of autologous CD117 stem cells; Drug
Lead sponsor: University of Colorado, Denver; Other
Eligibility: 18 Years to 70 Years
Enrollment: 15 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2013 – 2017
U.S. locations: 1
States / cities: Aurora, Colorado

Conditions: Rett Syndrome
Interventions: Dextromethorphan; Drug
Lead sponsor: Hugo W. Moser Research Institute at Kennedy Krieger, Inc.; Other
Eligibility: 2 Years to 15 Years
Enrollment: 38 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2004 – 2010
U.S. locations: 1
States / cities: Baltimore, Maryland

Conditions: Atopic Dermatitis
Interventions: Not listed
Lead sponsor: CorEvitas; Network
Eligibility: 10 Years to 15 Years
Enrollment: 1,500 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2023 – 2099
U.S. locations: 1
States / cities: Waltham, Massachusetts

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

Conditions: Advanced Unresectable Melanoma, Metastatic Melanoma
Interventions: Mirdametinib; Drug
Lead sponsor: Kevin Kim, MD; Other
Eligibility: 18 Years and older
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2030
U.S. locations: 1
States / cities: San Francisco, California

Conditions: Fabry Disease
Interventions: Fabrazyme (agalsidase beta); Drug
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: 18 Years to 65 Years
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 2006
U.S. locations: 1
States / cities: North Charleston, South Carolina

Conditions: Hurler Syndrome, Inherited Metabolic Disorder, Lysosomal Storage Disorder, Metachromatic Leukodystrophy, Inborn Errors of Metabolism
Interventions: rivogenlecleucel, rimiducid; Biological · Drug
Lead sponsor: Bellicum Pharmaceuticals; Industry
Eligibility: 3 Months to 21 Years
U.S. locations: 2
States / cities: Los Angeles, California • Palo Alto, California

Conditions: Gout
Interventions: Not listed
Lead sponsor: University of Massachusetts, Worcester; Other
Eligibility: 18 Years and older
Enrollment: 838 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2010 – 2012
U.S. locations: 1
States / cities: Worcester, Massachusetts

Conditions: Cervical Cancer, Endometrial Cancer, Fallopian Tube Cancer, Hereditary Breast/Ovarian Cancer (brca1, brca2), Ovarian Cancer, Sarcoma, Uterine Leiomyomata, Vaginal Cancer, Vulvar Cancer
Interventions: Ca-125, screening questionnaire administration, Surgery; Other · Procedure
Lead sponsor: Northwestern University; Other
Eligibility: 18 Years to 80 Years · Female only
Enrollment: 6,000 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2000 – 2040
U.S. locations: 1
States / cities: Chicago, Illinois

Conditions: Cystic Fibrosis
Interventions: Pioglitazone; Drug
Lead sponsor: Paul Beringer; Other
Eligibility: 18 Years and older
Enrollment: 24 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2009
U.S. locations: 1
States / cities: Los Angeles, California

Conditions: Tourette Syndrome, Chronic Tic Disorder
Interventions: Active Oral Orthotic Treatment, Placebo Oral Orthotic Treatment; Device
Lead sponsor: Weill Medical College of Cornell University; Other
Eligibility: 7 Years to 25 Years
Enrollment: 21 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2021
U.S. locations: 1
States / cities: New York, New York

Conditions: Congenital Disorders of Glycosylation
Interventions: Not listed
Lead sponsor: Mayo Clinic; Other
Eligibility: Not listed
Enrollment: 240 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2019 – 2025
U.S. locations: 1
States / cities: Rochester, Minnesota

Conditions: Hereditary Angioedema
Interventions: C1 esterase inhibitor [human] (C1INH-nf), Placebo (saline); Biological · Drug
Lead sponsor: Shire; Industry
Eligibility: 6 Years and older
Enrollment: 83 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2007
U.S. locations: 37
States / cities: Hoover, Alabama • Scottsdale, Arizona • Los Angeles, California + 34 more

Conditions: Duodenal Adenomas, Familial Adenomatous Polyposis
Interventions: CryoBalloon ablation; Device
Lead sponsor: Johns Hopkins University; Other
Eligibility: 18 Years and older
Enrollment: 33 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2026
U.S. locations: 4
States / cities: Baltimore, Maryland • Manhasset, New York • Danville, Pennsylvania + 1 more

Conditions: Severe Combined Immunodeficiency
Interventions: Gene transfer; Biological
Lead sponsor: David Williams; Other
Eligibility: Male only
Enrollment: 8 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2023
U.S. locations: 3
States / cities: Los Angeles, California • Boston, Massachusetts • Cincinnati, Ohio

Conditions: Neurofibromatosis 1
Interventions: Lovastatin, placebo pill; Drug
Lead sponsor: University of California, Los Angeles; Other
Eligibility: 10 Years to 50 Years
Enrollment: 44 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2014
U.S. locations: 1
States / cities: Los Angeles, California

Conditions: Sickle Cell Disease, Vaso-occlusive Crisis
Interventions: Nitrous oxide 50%; Drug
Lead sponsor: Columbia University; Other
Eligibility: 8 Years to 18 Years
Enrollment: 5 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2018
U.S. locations: 1
States / cities: New York, New York

Conditions: Ehlers-Danlos Syndrome, Multiple Sclerosis
Interventions: Not listed
Lead sponsor: Genetic Disease Investigators; Other
Eligibility: Not listed
Enrollment: 60 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2011 – 2015
U.S. locations: 1
States / cities: Colleyville, Texas

Conditions: Von Hippel-Lindau Syndrome, Renal Cell Carcinoma, Hemangioblastoma
Interventions: SU011248; Drug
Lead sponsor: M.D. Anderson Cancer Center; Other
Eligibility: 18 Years and older
Enrollment: 15 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2006 – 2011
U.S. locations: 1
States / cities: Houston, Texas

Conditions: Recessive Dystrophic Epidermolysis Bullosa
Interventions: Gentamicin; Drug
Lead sponsor: University of Southern California; Other
Eligibility: 7 Years and older
Enrollment: 9 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2023
U.S. locations: 1
States / cities: Los Angeles, California

Conditions: Mucopolysaccharidosis I, Cognitive Decline
Interventions: Intrathecal recombinant human alpha iduronidase; Drug
Lead sponsor: agnes chen; Other
Eligibility: 6 Years and older
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2020
U.S. locations: 1
States / cities: Torrance, California

Conditions: Duchenne's Muscular Dystrophy, Scoliosis
Interventions: Not listed
Lead sponsor: Children's Healthcare of Atlanta; Other
Eligibility: 6 Years to 21 Years · Male only
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2004 – 2005
U.S. locations: 1
States / cities: Atlanta, Georgia

Conditions: Fabry Disease
Interventions: Not listed
Lead sponsor: Shire; Industry
Eligibility: Not listed
Enrollment: 4,000 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2001 – 2021
U.S. locations: 1
States / cities: Lexington, Massachusetts

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Trial Readiness and Endpoint Assessment in Congenital Myotonic Dystrophy

Autologous CD117+ Progenitor Cell Mobilization for Lung Transplantation

Trial of Dextromethorphan in Rett Syndrome

CorEvitas International Adolescent Atopic Dermatitis (AD) Drug Safety and Effectiveness Registry

Assessing Tenapanor as a Treatment of CF-related Constipation.

Mirdametinib in Patients With Advanced NF1-mutant Melanoma

A Study in Patients With Fabry Disease Who Are on Chronic Hemodialysis Therapy for Treatment of End-stage Renal Insufficiency.

BPX-501 T Cells Infused Post Stem Cell Transplant in Pediatrics With Non-Malignant Disorders Ineligible for BPU004 Study

Assessment of Primary Care Providers' Knowledge and Beliefs Regarding Gout Management

Specimen and Data Study for Ovarian Cancer Early Detection and Prevention

Effect of Pioglitazone on Inflammation in Cystic Fibrosis

Proof of Concept Study of an Oral Orthotic to Reduce Tic Severity in Chronic Tic Disorder and Tourette Syndrome

Large-Scale Metabolomic Profiling for the Diagnosis of Inborn Errors of Metabolism

C1 Esterase Inhibitor (C1INH-nf) for the Treatment of Acute Hereditary Angioedema (HAE) Attacks

CryoBalloon Ablation for Treatment of Duodenal Adenomas

Gene Transfer for Severe Combined Immunodeficiency, X-linked (SCID-X1) Using a Self-inactivating (SIN) Gammaretroviral Vector

Trial to Evaluate the Safety of Lovastatin in Individuals With Neurofibromatosis Type I (NF1)

Nitrous Oxide Analgesia Vaso-occlusive Crisis

Vascular Fundus Changes in Patients With High Probability of Chronic Cerebrospinal Venous Insufficiency (CCSVI)

Evaluation of Sunitinib Malate in Patients With Von Hippel-Lindau Syndrome (VHL) Who Have VHL Lesions to Follow

Intravenous Gentamicin Therapy for Recessive Dystrophic Epidermolysis Bullosa (RDEB)

Extension Study of Intrathecal Enzyme Replacement for Cognitive Decline in MPS I

Test-Retest Reliability of Pulmonary Function Tests in Patients With Duchenne's Muscular Dystrophy

Fabry Outcome Survey (FOS)