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ClinicalTrials.gov public records Last synced Nov 2, 2026, 2:08 AM EST

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

Showing 1009–1032 of 10,347 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Congenital, Hereditary, and Neonatal Diseases and Abnormalities Clear all
Terminated No phase listed Observational

Natural History Study of Patients With Methylmalonic Acidemia and Propionic Acidemia

NCT05438485
View directory record Official record
Conditions
Methylmalonic Acidemia, Propionic Acidemia
Interventions
Registry
Other
Lead sponsor
HemoShear Therapeutics
Industry
Eligibility
Not listed
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Dec 27, 2023 · Synced Nov 2, 2026, 2:08 AM EST
Recruiting No phase listed Observational

Genetic Disorders of Obesity Program Database

NCT05747976
View directory record Official record
Conditions
Obesity, Childhood, Genetic Disease
Interventions
Not listed
Lead sponsor
Baylor College of Medicine
Other
Eligibility
Not listed
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2030
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated May 12, 2026 · Synced Nov 2, 2026, 2:08 AM EST
Completed No phase listed Observational Results available

Neurology Measures in FA Children

NCT03418740
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Not listed
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
2 Years to 18 Years
Enrollment
108 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2023
U.S. locations
3
States / cities
Los Angeles, California • Gainesville, Florida • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jan 16, 2025 · Synced Nov 2, 2026, 2:08 AM EST
Completed Phase 2Phase 3 Interventional

A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia

NCT03655678
View directory record Official record
Conditions
Beta-Thalassemia, Thalassemia, Genetic Diseases, Inborn, Hematologic Diseases, Hemoglobinopathies
Interventions
CTX001
Biological
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
12 Years to 35 Years
Enrollment
59 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2025
U.S. locations
6
States / cities
Palo Alto, California • Chicago, Illinois • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Dec 17, 2025 · Synced Nov 2, 2026, 2:08 AM EST
Completed No phase listed Observational Accepts healthy volunteers

Identification of Genes Associated With Lung Disease in Patients With Rheumatoid Arthritis

NCT00001885
View directory record Official record
Conditions
Healthy, Pulmonary Fibrosis, Rheumatoid Arthritis
Interventions
Not listed
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
21 Years and older
Enrollment
400 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1999 – 2007
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Nov 2, 2026, 2:08 AM EST
Not listed Not applicable Interventional

Study of Crizanlizumab for Prevention of Silent Cerebral Infarcts in SCA

NCT05334576
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Crizanlizumab
Other
Lead sponsor
Andria Ford
Other
Eligibility
16 Years and older
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Mar 6, 2023 · Synced Nov 2, 2026, 2:08 AM EST
Terminated No phase listed Observational

Using Walk With Ease Walking Program With Men 65 and Older Being Treated for Prostate Cancer

NCT02139774
View directory record Official record
Conditions
Prostate Cancer
Interventions
Walk with Ease
Behavioral
Lead sponsor
UNC Lineberger Comprehensive Cancer Center
Other
Eligibility
65 Years and older · Male only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
1
States / cities
Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Dec 4, 2015 · Synced Nov 2, 2026, 2:08 AM EST
Withdrawn Phase 2 Interventional

Lucitanib (E3810) in Patients With Advanced Cancer and FGFR, VEGFR, or PDGFR Pathway Aberrations

NCT02747797
View directory record Official record
Conditions
Advanced Cancer
Interventions
Lucitanib
Drug
Lead sponsor
Teresa Helsten, MD
Other
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2022
U.S. locations
1
States / cities
La Jolla, California
Source: ClinicalTrials.gov public record
Updated Aug 2, 2018 · Synced Nov 2, 2026, 2:08 AM EST
Completed Not applicable Interventional Accepts healthy volunteers Results available

Microvascular Blood Flow in Sickle Cell Anemia

NCT01566890
View directory record Official record
Conditions
Sickle Cell Disease, Sickle Cell Anemia
Interventions
regadenoson infusion with contrast-enhanced ultrasound, contrast-enhanced ultrasound
Drug · Procedure
Lead sponsor
Versiti Blood Health
Other
Eligibility
18 Years to 70 Years
Enrollment
91 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2020
U.S. locations
2
States / cities
Chicago, Illinois • Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated Jun 26, 2024 · Synced Nov 2, 2026, 2:08 AM EST
Completed Early Phase 1 Interventional

Methylprednisolone Treatment of Friedreich Ataxia

NCT02424435
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Methylprednisolone
Drug
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
5 Years and older
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 25, 2021 · Synced Nov 2, 2026, 2:08 AM EST
Active, not recruiting No phase listed Observational Accepts healthy volunteers

OTIS Cimzia Pregnancy Registry

NCT01797224
View directory record Official record
Conditions
Crohn's Disease, Rheumatoid Arthritis, Ankylosing Spondylitis, Psoriatic Arthritis, Psoriasis
Interventions
Not listed
Lead sponsor
University of California, San Diego
Other
Eligibility
Female only
Enrollment
925 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2026
U.S. locations
1
States / cities
San Diego, California
Source: ClinicalTrials.gov public record
Updated Jan 28, 2025 · Synced Nov 2, 2026, 2:08 AM EST
Active, not recruiting No phase listed Observational

Study of Selected X-linked Disorders: Goltz Syndrome

NCT00691223
View directory record Official record
Conditions
Focal Dermal Hypoplasia (FDH), Goltz Syndrome
Interventions
Not listed
Lead sponsor
Baylor College of Medicine
Other
Eligibility
Not listed
Enrollment
84 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2030
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated May 1, 2026 · Synced Nov 2, 2026, 2:08 AM EST
Terminated Phase 1Phase 2 Interventional Results available

Safety and Efficacy Study of Sotatercept in Adults With Transfusion Dependent Diamond Blackfan Anemia

NCT01464164
View directory record Official record
Conditions
Diamond Blackfan Anemia
Interventions
Sotatercept, Sotatercept with prednisone boost
Drug
Lead sponsor
Northwell Health
Other
Eligibility
18 Years and older
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2021
U.S. locations
1
States / cities
Manhasset, New York
Source: ClinicalTrials.gov public record
Updated Mar 17, 2023 · Synced Nov 2, 2026, 2:08 AM EST
Not listed No phase listed Observational

PREDICT Trial: PRospective Evaluation of NTM Disease In CysTic Fibrosis

NCT02073409
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Not listed
Lead sponsor
National Jewish Health
Other
Eligibility
6 Years to 99 Years
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2025
U.S. locations
19
States / cities
Birmingham, Alabama • Los Angeles, California • San Diego, California + 15 more
Source: ClinicalTrials.gov public record
Updated Apr 2, 2024 · Synced Nov 2, 2026, 2:08 AM EST
Recruiting No phase listed Observational

WRAP North America

NCT06807099
View directory record Official record
Conditions
Venous Stenosis, Venous Occlusion
Interventions
Merit WRAPSODY® Cell Impermeable Endoprosthesis (WRAPSODY CIE)
Device
Lead sponsor
Merit Medical Systems, Inc.
Industry
Eligibility
18 Years and older
Enrollment
250 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
14
States / cities
Dothan, Alabama • Phoenix, Arizona • Riverside, California + 11 more
Source: ClinicalTrials.gov public record
Updated Aug 26, 2026 · Synced Nov 2, 2026, 2:08 AM EST
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 Nov 2, 2026, 2:08 AM EST
Completed No phase listed Observational

Alternate Measures of Glucose During OGTT Testing for CFRD

NCT05568134
View directory record Official record
Conditions
Cystic Fibrosis, Cystic Fibrosis-related Diabetes
Interventions
Oral glucose tolerance test
Diagnostic Test
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
10 Years and older
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Feb 24, 2025 · Synced Nov 2, 2026, 2:08 AM EST
Completed Phase 1Phase 2 Interventional Results available

Dose Escalation Study of QR-010 in Homozygous ΔF508 Cystic Fibrosis Patients

NCT02532764
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
QR-010, Placebo
Drug
Lead sponsor
ProQR Therapeutics
Industry
Eligibility
18 Years to 60 Years
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
12
States / cities
Los Angeles, California • Palo Alto, California • Chicago, Illinois + 8 more
Source: ClinicalTrials.gov public record
Updated Feb 6, 2019 · Synced Nov 2, 2026, 2:08 AM EST
Not listed Phase 1Phase 2 Interventional Accepts healthy volunteers

SAD and MAD of Inhaled AR-501 in Health Adults and P. Aeruginosa Infected Cystic Fibrosis Subjects

NCT03669614
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Inhaled AR-501, Inhaled Placebo
Drug
Lead sponsor
Aridis Pharmaceuticals, Inc.
Industry
Eligibility
18 Years to 49 Years
Enrollment
102 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2023
U.S. locations
25
States / cities
Tucson, Arizona • Long Beach, California • Denver, Colorado + 22 more
Source: ClinicalTrials.gov public record
Updated Mar 9, 2023 · Synced Nov 2, 2026, 2:08 AM EST
Completed Phase 2 Interventional Results available

Reduced Intensity Conditioning for Hemophagocytic Syndromes or Selected Primary Immune Deficiencies (BMT CTN 1204)

NCT01998633
View directory record Official record
Conditions
Hemophagocytic Lymphohistiocytosis, Chronic Active Epstein-Barr Virus Infection, Chronic Granulomatous Disease, HIGM-1, Leukocyte Adhesion Deficiency, IPEX
Interventions
Hematopoietic Stem Cell Transplant
Biological
Lead sponsor
Medical College of Wisconsin
Other
Eligibility
4 Months to 45 Years
Enrollment
47 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
19
States / cities
Birmingham, Alabama • Washington D.C., District of Columbia • Atlanta, Georgia + 16 more
Source: ClinicalTrials.gov public record
Updated Dec 8, 2022 · Synced Nov 2, 2026, 2:08 AM EST
Completed Phase 3 Interventional

Pharmacokinetics, Safety and Tolerability of Zavesca (Miglustat) in Patients With Infantile Onset Gangliosidosis: Single and Steady State Oral Doses

NCT00672022
View directory record Official record
Conditions
GM2 Gangliosidoses, Tay-Sachs, Sandhoff Disease
Interventions
Zavesca (Miglustat)
Drug
Lead sponsor
Children's National Research Institute
Other
Eligibility
6 Months to 5 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2007
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated May 6, 2008 · Synced Nov 2, 2026, 2:08 AM EST
Completed Not applicable Interventional Accepts healthy volunteers

Changes in Adherence After Playing "My Life With CF" Game

NCT00688051
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
My Life with CF game
Other
Lead sponsor
Children's Mercy Hospital Kansas City
Other
Eligibility
12 Years to 21 Years
Enrollment
30 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2008 – 2009
U.S. locations
1
States / cities
Kansas City, Missouri
Source: ClinicalTrials.gov public record
Updated Sep 5, 2014 · Synced Nov 2, 2026, 2:08 AM EST
Completed Phase 1 Interventional Accepts healthy volunteers

Primaquine Enantiomers in G6PD Deficient Human Volunteers

NCT04073953
View directory record Official record
Conditions
G6PD Deficiency
Interventions
RPQ, SPQ, Placebo
Drug
Lead sponsor
University of Mississippi, Oxford
Other
Eligibility
18 Years to 65 Years
Enrollment
18 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2023
U.S. locations
1
States / cities
University, Mississippi
Source: ClinicalTrials.gov public record
Updated Nov 21, 2024 · Synced Nov 2, 2026, 2:08 AM EST
Completed Phase 2 Interventional

A 6-week, Study of MG01CI Low Dose and High Dose Compared With Placebo in Adults and Adolescents With Fragile X Syndrome

NCT02126995
View directory record Official record
Conditions
Fragile X Syndrome
Interventions
MG01CI extended-release tablet
Drug
Lead sponsor
Alcobra Ltd.
Industry
Eligibility
15 Years to 55 Years
Enrollment
62 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
11
States / cities
Phoenix, Arizona • Sacramento, California • Aurora, Colorado + 8 more
Source: ClinicalTrials.gov public record
Updated Jul 6, 2016 · Synced Nov 2, 2026, 2:08 AM EST