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ClinicalTrials.gov public records Last synced Oct 11, 2026, 5:36 AM EDT

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Showing 121–144 of 1,323 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Anomalies Gastrointestinal Clear all
Completed No phase listed Observational Results available

Erythropoietic Protoporphyrias: Studies of the Natural History, Genotype-Phenotype Correlations, and Psychosocial Impact

NCT01688895
View directory record Official record
Conditions
Erythropoietic Protoporphyria, EPP, X-Linked Protoporphyria, XLP, XLPP, X-Linked Dominant Erythropoietic Protoporphyria, XLEPP, XLDP
Interventions
Not listed
Lead sponsor
Icahn School of Medicine at Mount Sinai
Other
Eligibility
Not listed
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2019
U.S. locations
6
States / cities
Birmingham, Alabama • San Francisco, California • New York, New York + 3 more
Source: ClinicalTrials.gov public record
Updated Apr 17, 2020 · Synced Oct 11, 2026, 5:36 AM EDT
Recruiting No phase listed Observational

Hepatic Histopathology in Urea Cycle Disorders

NCT04908319
View directory record Official record
Conditions
Urea Cycle Disorder, Ornithine Transcarbamylase Deficiency, Citrullinemia 1, ARGI Deficiency, ASL Deficiency, Argininosuccinic Aciduria, ASS Deficiency, Hyperargininemia, Carbamyl Phosphate Synthetase Deficiency, NAGS Deficiency
Interventions
Not listed
Lead sponsor
Baylor College of Medicine
Other
Eligibility
Not listed
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
2
States / cities
Washington D.C., District of Columbia • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jul 20, 2025 · Synced Oct 11, 2026, 5:36 AM EDT
Completed No phase listed Observational

The PROMISE Pediatric Study 6 to 11 Years Old

NCT04613128
View directory record Official record
Conditions
Cystic Fibrosis, Cystic Fibrosis in Children
Interventions
Not listed
Lead sponsor
Nicole Hamblett
Other
Eligibility
6 Years to 11 Years
Enrollment
125 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
20
States / cities
Birmingham, Alabama • Palo Alto, California • Aurora, Colorado + 17 more
Source: ClinicalTrials.gov public record
Updated Apr 27, 2026 · Synced Oct 11, 2026, 5:36 AM EDT
Recruiting Phase 1Phase 2 Interventional

A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD)

NCT06389877
View directory record Official record
Conditions
Alpha 1-Antitrypsin Deficiency
Interventions
BEAM-302
Drug
Lead sponsor
Beam Therapeutics Inc.
Industry
Eligibility
18 Years to 75 Years
Enrollment
192 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2030
U.S. locations
3
States / cities
Birmingham, Alabama • Boston, Massachusetts • Charleston, South Carolina
Source: ClinicalTrials.gov public record
Updated Oct 5, 2026 · Synced Oct 11, 2026, 5:36 AM EDT
Completed Phase 2 Interventional Results available

A Study to Test How Well BI 1291583 is Tolerated by People With Cystic Fibrosis Bronchiectasis (Clairafly™)

NCT05865886
View directory record Official record
Conditions
Cystic Fibrosis, Bronchiectasis
Interventions
BI 1291583, Placebo to BI 1291583
Drug
Lead sponsor
Boehringer Ingelheim
Industry
Eligibility
18 Years and older
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024
U.S. locations
7
States / cities
Denver, Colorado • Altamonte Springs, Florida • South Portland, Maine + 3 more
Source: ClinicalTrials.gov public record
Updated Jun 23, 2026 · Synced Oct 11, 2026, 5:36 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Sirolimus for Autoimmune Disease of Blood Cells

NCT00392951
View directory record Official record
Conditions
Autoimmune Pancytopenia, Autoimmune Lymphoproliferative Syndrome (ALPS), Evans Syndrome, Idiopathic Thrombocytopenic Purpura, Anemia, Hemolytic, Autoimmune, Autoimmune Neutropenia, Lupus Erythematosus, Systemic, Inflammatory Bowel Disease, Rheumatoid Arthritis
Interventions
sirolimus
Drug
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
1 Year to 30 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2016
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Nov 19, 2019 · Synced Oct 11, 2026, 5:36 AM EDT
Terminated No phase listed Observational

Hirschsprung Disease Trends in the United States: Analysis of the National Inpatient Sample

NCT05038345
View directory record Official record
Conditions
Hirschsprung Disease
Interventions
Pull through surgery
Procedure
Lead sponsor
University of New Mexico
Other
Eligibility
1 Day to 18 Years
Enrollment
2,787 participants
Timeline
2019 – 2021
U.S. locations
1
States / cities
Albuquerque, New Mexico
Source: ClinicalTrials.gov public record
Updated Feb 24, 2025 · Synced Oct 11, 2026, 5:36 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Ph 1/2 Study Evaluating Safety and Tolerability of Inhaled AP-PA02 in Subjects With Chronic Pseudomonas Aeruginosa Lung Infections and Cystic Fibrosis

NCT04596319
View directory record Official record
Conditions
Cystic Fibrosis, Pseudomonas Aeruginosa, Pseudomonas, Lung Infection, Lung Infection Pseudomonal
Interventions
AP-PA02, Placebo
Biological · Other
Lead sponsor
Armata Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
20
States / cities
Los Angeles, California • Tampa, Florida • Boise, Idaho + 16 more
Source: ClinicalTrials.gov public record
Updated Jan 31, 2024 · Synced Oct 11, 2026, 5:36 AM EDT
Completed Phase 1Phase 2 Interventional

Study Assessing PTI-428 Safety, Tolerability, and Pharmacokinetics in Subjects With Cystic Fibrosis

NCT02718495
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
PTI-428, Placebo
Drug
Lead sponsor
Proteostasis Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
56 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
20
States / cities
Stanford, California • Altamonte Springs, Florida • Gainesville, Florida + 16 more
Source: ClinicalTrials.gov public record
Updated Mar 21, 2019 · Synced Oct 11, 2026, 5:36 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Neurodevelopment and Neuroimaging in Parenterally-fed Infants and Young Children

NCT00392730
View directory record Official record
Conditions
Parenteral Nutrition, Necrotizing Enterocolitis, Digestive System Abnormalities, Cholestasis
Interventions
Remove Mn from PN if evidence of increased brain Mn on MRI
Dietary Supplement
Lead sponsor
Vanderbilt University
Other
Eligibility
30 Days to 6 Years
Enrollment
122 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2006 – 2010
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Dec 19, 2013 · Synced Oct 11, 2026, 5:36 AM EDT
Completed Phase 1 Interventional

Liposomal Doxorubicin in Treating Children With Refractory Solid Tumors

NCT00019630
View directory record Official record
Conditions
Childhood Soft Tissue Sarcoma, Childhood Liver Cancer, Bone Cancer, Brain Tumor, Kidney Tumor
Interventions
doxorubicin HCl liposome
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
Up to 21 Years
Timeline
Started 1999
U.S. locations
2
States / cities
Bethesda, Maryland • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 4, 2024 · Synced Oct 11, 2026, 5:36 AM EDT
Recruiting No phase listed Observational

Long-term Follow-up (LTFU) Study of Participants in Any iECURE Protocol Using an Investigational Product (IP)

NCT06805695
View directory record Official record
Conditions
Ornithine Transcarbamylase Deficiency, Ornithine Transcarbamylase Deficiency Disease, Urea Cycle Disorders, Inborn
Interventions
No Intervention
Other
Lead sponsor
iECURE, Inc.
Industry
Eligibility
7 Months to 15 Months
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2041
U.S. locations
2
States / cities
Aurora, Colorado • New York, New York
Source: ClinicalTrials.gov public record
Updated Aug 31, 2026 · Synced Oct 11, 2026, 5:36 AM EDT
Active, not recruiting Not applicable Interventional

Weight Management and Health Behavior Intervention in Lowering Cancer Risk for BRCA Positive and Lynch Syndrome Families

NCT04125914
View directory record Official record
Conditions
Hereditary Breast Carcinoma, Hereditary Ovarian Carcinoma, Lynch Syndrome
Interventions
Behavioral Dietary Intervention, E-mail, Exercise Intervention, Health Promotion and Education, Internet-Based Intervention, Questionnaire Administration, Telephone-Based Intervention
Behavioral · Other
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
18 Years and older
Enrollment
384 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2028
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Oct 6, 2026 · Synced Oct 11, 2026, 5:36 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers Results available

Safety and Tolerability Study of Allogeneic Mesenchymal Stem Cell Infusion in Adults With Cystic Fibrosis

NCT02866721
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Mesenchymal Stem Cells
Biological
Lead sponsor
University Hospitals Cleveland Medical Center
Other
Eligibility
18 Years and older
Enrollment
14 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2020
U.S. locations
1
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Jan 30, 2023 · Synced Oct 11, 2026, 5:36 AM EDT
Withdrawn Phase 2 Interventional

Study to Determine the Efficacy&Safety of ARV-1801(ACG-701) for the Treatment of Cystic Fibrosis Pulmonary Exacerbations

NCT05641298
View directory record Official record
Conditions
Cystic Fibrosis, Cystic Fibrosis Pulmonary Exacerbation
Interventions
Sodium Fusidate, Placebo
Drug
Lead sponsor
Aceragen
Industry
Eligibility
12 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023
U.S. locations
25
States / cities
Gainesville, Florida • Orlando, Florida • Pensacola, Florida + 22 more
Source: ClinicalTrials.gov public record
Updated Jul 19, 2023 · Synced Oct 11, 2026, 5:36 AM EDT
Completed Phase 1 Interventional

A Phase 1 in Patients With HLA-A*0201+ and WT1+ Recurrent/Metastatic Cancers

NCT05360680
View directory record Official record
Conditions
Colorectal Cancer, Gastric Cancer, Pancreatic Cancer, Ovarian Cancer
Interventions
CUE-102
Drug
Lead sponsor
Cue Biopharma
Industry
Eligibility
18 Years and older
Enrollment
42 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
15
States / cities
Phoenix, Arizona • Los Angeles, California • Palo Alto, California + 12 more
Source: ClinicalTrials.gov public record
Updated Jan 22, 2026 · Synced Oct 11, 2026, 5:36 AM EDT
Terminated No phase listed Observational

Host Genetic Factors Influencing HIV1 and HCV Viral Loads and AIDS Clinical Progression in a Hemophilia Cohort (HGDS-3)

NCT00340548
View directory record Official record
Conditions
Hemophilia, AIDS
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
4 Years to 100 Years
Enrollment
333 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2020
U.S. locations
15
States / cities
La Jolla, California • Los Angeles, California • Indianapolis, Indiana + 11 more
Source: ClinicalTrials.gov public record
Updated Jan 13, 2020 · Synced Oct 11, 2026, 5:36 AM EDT
Completed No phase listed Observational

Clinical Outcomes of Triple Combination Therapy in Severe Cystic Fibrosis Disease.

NCT04038710
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Triple combination therapy
Drug
Lead sponsor
National Jewish Health
Other
Eligibility
12 Years and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
1
States / cities
Denver, Colorado
Source: ClinicalTrials.gov public record
Updated Mar 11, 2021 · Synced Oct 11, 2026, 5:36 AM EDT
Completed Phase 4 Interventional Results available

Comparison of Airway Clearance Therapy in Cystic Fibrosis Using the Same VEST Therapy Device But With Different Settings

NCT00685035
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
HFCWC with higher pressure/variable-frequency settings, HFCWC with lower pressure/mid-frequency settings
Device
Lead sponsor
University of Minnesota
Other
Eligibility
18 Years and older
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jul 25, 2016 · Synced Oct 11, 2026, 5:36 AM EDT
Not listed Phase 2Phase 3 Interventional

Cysteine Supplementation in Critically Ill Neonates

NCT00254176
View directory record Official record
Conditions
Sepsis, Bronchopulmonary Dysplasia, Necrotizing Enterocolitis, Retinopathy of Prematurity, Systemic Inflammatory Response Syndrome
Interventions
Parenteral cysteine-HCl supplementation, Placebo - added premasol
Dietary Supplement
Lead sponsor
University of California, Los Angeles
Other
Eligibility
Up to 30 Days
Enrollment
108 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2011
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Feb 26, 2010 · Synced Oct 11, 2026, 5:36 AM EDT
Recruiting Phase 2 Interventional

18FluoroLDOPA PET Imaging for the Detection and Localization of Focal Congenital Hyperinsulinism

NCT04205604
View directory record Official record
Conditions
Congenital Hyperinsulinism
Interventions
18F-Fluoro Dopa Imaging
Drug
Lead sponsor
Miguel Pampaloni
Other
Eligibility
0 Days to 18 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2026
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated May 1, 2026 · Synced Oct 11, 2026, 5:36 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Volatile Organic Compounds in Cystic Fibrosis

NCT01379040
View directory record Official record
Conditions
Cystic Fibrosis, Pseudomonas Aeruginosa
Interventions
Cystic Fibrosis patients
Other
Lead sponsor
Landon Pediatric Foundation
Other
Eligibility
1 Year to 40 Years
Enrollment
24 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2010
U.S. locations
1
States / cities
Ventura, California
Source: ClinicalTrials.gov public record
Updated Aug 13, 2014 · Synced Oct 11, 2026, 5:36 AM EDT
Withdrawn Not applicable Interventional

Relationship Between the Development of Impaired Glucose Tolerance, the Phenotype of CFLD, and the Risk of Liver Fibrosis

NCT05229640
View directory record Official record
Conditions
CF - Cystic Fibrosis, Cystic Fibrosis-related Diabetes, Cystic Fibrosis Liver Disease
Interventions
Fibroscan
Device
Lead sponsor
Dartmouth-Hitchcock Medical Center
Other
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2023
U.S. locations
1
States / cities
Lebanon, New Hampshire
Source: ClinicalTrials.gov public record
Updated Jul 13, 2023 · Synced Oct 11, 2026, 5:36 AM EDT
Completed Phase 1 Interventional Results available

Lyophilized Black Raspberries in Adults With Familial Adenomatous Polyposis (FAP)

NCT00770991
View directory record Official record
Conditions
Familial Adenomatous Polyposis
Interventions
Black raspberry (BRB) Slurry, Black Raspberry (BRB) Suppositories, Black Raspberry (BRB) Placebo Slurry
Drug
Lead sponsor
The Cleveland Clinic
Other
Eligibility
18 Years and older
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2008
U.S. locations
1
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Feb 15, 2016 · Synced Oct 11, 2026, 5:36 AM EDT