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 Nov 7, 2026, 10:33 AM EST

All trial information on this site comes from the official ClinicalTrials.gov public API (clinicaltrials.gov/api/v2), run by the U.S. National Library of Medicine. We only display that public information; we do not add opinions, endorsements, or any other intent. Always review the official ClinicalTrials.gov record for the latest information.

Showing 1–24 of 7,928 matching trials from the live ClinicalTrials.gov search.

Filters: Condition: Genetic Diseases, Inborn Clear all

Conditions: Sickle Cell Disease
Interventions: ICA-17043; Drug
Lead sponsor: Icagen; Industry
Eligibility: 16 Years to 65 Years
Enrollment: 297 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2007
U.S. locations: 42
States / cities: Mobile, Alabama • Little Rock, Arkansas • Davis, California + 39 more

Conditions: Prader-Willi Syndrome, Hyperphagia
Interventions: Intranasal Oxytocin (IN-OXT), Matched Placebo; Drug · Other
Lead sponsor: Montefiore Medical Center; Other
Eligibility: 5 Years to 18 Years
Enrollment: 23 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2018
U.S. locations: 1
States / cities: The Bronx, New York

Conditions: Parkinson's Disease
Interventions: Not listed
Lead sponsor: University of Washington; Other
Eligibility: 18 Years and older
Enrollment: 5,000 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2009 – 2026
U.S. locations: 5
States / cities: Atlanta, Georgia • Cincinnati, Ohio • Portland, Oregon + 1 more

Conditions: Fabry Disease
Interventions: Not listed
Lead sponsor: Boston Children's Hospital; Other
Eligibility: 1 Year and older
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2030
U.S. locations: 1
States / cities: Boston, Massachusetts

Conditions: Hemophilia B
Interventions: Non-Interventional; Other
Lead sponsor: Regeneron Pharmaceuticals; Industry
Eligibility: 16 Years and older · Male only
Enrollment: 11 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2026
U.S. locations: 9
States / cities: Aurora, Colorado • New Haven, Connecticut • Washington D.C., District of Columbia + 6 more

Conditions: Cystic Fibrosis
Interventions: Vitamin D Powder, Vitamin D Oil; Dietary Supplement
Lead sponsor: Emory University; Other
Eligibility: 18 Years to 59 Years
Enrollment: 17 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2015
U.S. locations: 1
States / cities: Atlanta, Georgia

Conditions: Glioblastoma
Interventions: DSP-7888 Dosing Emulsion, Bevacizumab; Drug
Lead sponsor: Sumitomo Pharma America, Inc.; Industry
Eligibility: 18 Years and older
Enrollment: 221 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2021
U.S. locations: 37
States / cities: Birmingham, Alabama • Tucson, Arizona • Fayetteville, Arkansas + 30 more

Conditions: Colorectal Cancer, Precancerous Condition
Interventions: celecoxib, placebo; Drug · Other
Lead sponsor: M.D. Anderson Cancer Center; Other
Eligibility: 10 Years to 14 Years
Enrollment: 22 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2002 – 2006
U.S. locations: 4
States / cities: Cleveland, Ohio • Houston, Texas

Conditions: Sickle Cell Anemia
Interventions: Prasugrel, Placebo; Drug
Lead sponsor: Eli Lilly and Company; Industry
Eligibility: 18 Years to 55 Years
Enrollment: 62 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2011
U.S. locations: 14
States / cities: Birmingham, Alabama • Little Rock, Arkansas • Sacramento, California + 11 more

Conditions: Fragile X Syndrome, Autism Spectrum Disorder, Autistic Disorder, Asperger Syndrome
Interventions: Alpha Auditory Entrainment, Sham; Other
Lead sponsor: Children's Hospital Medical Center, Cincinnati; Other
Eligibility: 5 Years to 10 Years
Enrollment: 180 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2023 – 2028
U.S. locations: 1
States / cities: Cincinnati, Ohio

Conditions: Chronic Gout Refractory to Conventional Therapy
Interventions: pegloticase 8 mg i.v.; Drug
Lead sponsor: Savient Pharmaceuticals; Industry
Eligibility: 18 Years and older
Enrollment: 7 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2009
U.S. locations: 4
States / cities: Chicago, Illinois • Wheaton, Maryland • Durham, North Carolina + 1 more

Conditions: Hemophilia A, Bleeding Disorder
Interventions: Not listed
Lead sponsor: Oregon Health and Science University; Other
Eligibility: 10 Years to 65 Years · Female only
Enrollment: 256 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2022
U.S. locations: 1
States / cities: Portland, Oregon

Conditions: Atopic Dermatitis
Interventions: NM26-2198, Placebo; Biological · Other
Lead sponsor: Yellow Jersey Therapeutics AG; Industry
Eligibility: 18 Years and older
Enrollment: 126 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2023 – 2024
U.S. locations: 6
States / cities: Fountain Valley, California • Glendale, California • San Diego, California + 3 more

Conditions: Fabry Disease, Chronic Kidney Disease, Stage IV (Severe)
Interventions: Fabrazyme (agalsidase beta); Biological
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: 16 Years and older
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2002 – 2003
U.S. locations: 9
States / cities: Farmington, Connecticut • Coral Springs, Florida • Kansas City, Kansas + 6 more

Conditions: Acoustic Schwannoma, Adult Anaplastic Meningioma, Adult Ependymoma, Adult Grade I Meningioma, Adult Grade II Meningioma, Adult Meningeal Hemangiopericytoma, Adult Papillary Meningioma, Neurofibromatosis Type 1, Neurofibromatosis Type 2, Recurrent Adult Brain Tumor
Interventions: bevacizumab; Biological
Lead sponsor: Northwestern University; Other
Eligibility: 18 Years and older
Enrollment: 50 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2018
U.S. locations: 5
States / cities: Chicago, Illinois • Boston, Massachusetts • New York, New York + 2 more

Conditions: Sickle Cell Disease
Interventions: Demographic Questionnaire, Medical Adherence Measure Questionnaire, Acceptability Questionnaire; Behavioral
Lead sponsor: St. Jude Children's Research Hospital; Other
Eligibility: Up to 17 Years
Enrollment: 36 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2015
U.S. locations: 1
States / cities: Memphis, Tennessee

Conditions: Hypophosphatasia
Interventions: asfotase alfa; Biological
Lead sponsor: Alexion Pharmaceuticals, Inc.; Industry
Eligibility: Not listed
U.S. locations: 2
States / cities: Lakewood, Colorado • Pittsburgh, Pennsylvania

Conditions: Prader-Willi Syndrome
Interventions: Placebo, Tesomet; Other · Drug
Lead sponsor: Saniona; Industry
Eligibility: 13 Years to 65 Years
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2022
U.S. locations: 1
States / cities: Lansing, Michigan

Conditions: Urea Cycle Disorder
Interventions: Not listed
Lead sponsor: Amgen; Industry
Eligibility: Not listed
Enrollment: 203 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2020
U.S. locations: 1
States / cities: Blue Bell, Pennsylvania

Conditions: Pancreas Cancer, Pancreatitis, Chronic Pancreatitis, Pancreatic Cyst, Family History of Pancreas Cancer, Genetic Mutations
Interventions: Not listed
Lead sponsor: Icahn School of Medicine at Mount Sinai; Other
Eligibility: 18 Years and older
Enrollment: 1,368 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2033
U.S. locations: 2
States / cities: New York, New York

Conditions: Phenylketonuria (PKU)
Interventions: MZE782, Placebo; Drug · Other
Lead sponsor: Maze Therapeutics; Industry
Eligibility: 18 Years to 75 Years
Enrollment: 38 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2028
U.S. locations: 7
States / cities: Oakland, California • Palo Alto, California • Chicago, Illinois + 4 more

Conditions: Sickle Cell Disease
Interventions: Digital app and information system; Other
Lead sponsor: Makerere University; Other
Eligibility: Up to 5 Years
Enrollment: 24,000 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2024 – 2028
U.S. locations: 1
States / cities: Houston, Texas

Conditions: Hemophilia B
Interventions: rFIXFc; Biological
Lead sponsor: Bioverativ, a Sanofi company; Industry
Eligibility: Up to 17 Years · Male only
Enrollment: 33 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2019
U.S. locations: 11
States / cities: Sacramento, California • Washington D.C., District of Columbia • Atlanta, Georgia + 8 more

Conditions: GM1 Gangliosidosis, GM2 Gangliosidosis, Gaucher Disease, Type 2, Tay-Sachs Disease, AB Variant Gangliosidosis GM2, Sandhoff Disease
Interventions: Not listed
Lead sponsor: Idorsia Pharmaceuticals Ltd.; Industry
Eligibility: 0 Months and older
Enrollment: 226 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2021
U.S. locations: 3
States / cities: Chicago, Illinois • Rochester, Minnesota • Fairfax, Virginia

Search by objective public record fields.

A Stratified Sickle Event Randomized Trial (ASSERT)

Oxytocin vs. Placebo for the Treatment Hyperphagia in Children and Adolescents With Prader-Willi Syndrome

The Parkinson's Genetic Research Study

Evaluation of Phenotypic Variability in Fabry Disease

A Trial That Evaluates Disease Characteristics in Hemophilia B Adult Male Participants Receiving Prophylaxis With Standard of Care Factor IX Protein (FIX) Replacement Therapy

Comparison of Absorption of Vitamin D in Cystic Fibrosis

A Study of DSP-7888 Dosing Emulsion in Combination With Bevacizumab in Patients With Recurrent or Progressive Glioblastoma Following Initial Therapy

Celecoxib in Preventing Colorectal Cancer in Young Patients With a Genetic Predisposition for Familial Adenomatous Polyposis

Prasugrel Versus Placebo in Adult Sickle Cell Disease

Alpha Auditory Entrainment for Cognitive Enhancement and Sensory Hypersensitivity in Youth With Developmental Disorders

Re-exposure Study of Pegloticase Intravenous (i.v.) in Symptomatic Gout Patients

Characterizing the Impact and Treatment of Reproductive Tract Bleeding on Women and Post-menarchal Girls With Bleeding Disorders

Evaluation of NM26-2198 in Healthy Subjects and in Patients With Moderate-to-severe Atopic Dermatitis (AD)

Severe Renal Disease Study in Fabry Patients Treated With Fabrazyme

Bevacizumab in Treating Patients With Recurrent or Progressive Meningiomas

Medication Adherence in Youth With Sickle Cell Disease (SCD)

Expanded Access Program for Asfotase Alfa Treatment for Patients With Infantile- or Juvenile-onset Hypophosphatasia (HPP)

Study of Tesomet With Open-label Extension in Subjects With Prader-Willi Syndrome

Observational Study That Will Collect Information on Patients With Urea Cycle Disorders (UCDs)

Pancreas Registry and High Risk Registry

A Study of MZE782 in Adults With PKU

Development and Evaluation of an Information Management System and Communication System for Population-wide Point-of-care Infant Sickle Cell Disease Screening

Study to Determine the Safety and Efficacy of rFIXFc in Previously Untreated Males With Severe Hemophilia B

Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2