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ClinicalTrials.gov public records Last synced Oct 14, 2026, 9:52 AM EDT

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Showing 169–192 of 1,323 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Gastrointestinal Anomalies Clear all
Active, not recruiting Phase 1Phase 2 Interventional Results available

A Phase I/II Study of VTX-801 in Adult Patients With Wilson's Disease

NCT04537377
View directory record Official record
Conditions
Wilson's Disease
Interventions
VTX-801
Genetic
Lead sponsor
Vivet Therapeutics SAS
Industry
Eligibility
18 Years to 65 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2029
U.S. locations
6
States / cities
Sacramento, California • New Haven, Connecticut • Orlando, Florida + 3 more
Source: ClinicalTrials.gov public record
Updated Jan 30, 2026 · Synced Oct 14, 2026, 9:52 AM EDT
Recruiting No phase listed Observational

Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC

NCT04528355
View directory record Official record
Conditions
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions
Interventions
data collection
Drug
Lead sponsor
Paul Szabolcs
Other
Eligibility
2 Months to 60 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2028
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jan 13, 2026 · Synced Oct 14, 2026, 9:52 AM EDT
Completed No phase listed Observational

Real World Evidence Study in Subjects With Wilson's Disease

NCT05783687
View directory record Official record
Conditions
Wilson Disease
Interventions
NCC-Sp Assay
Diagnostic Test
Lead sponsor
Orphalan
Industry
Eligibility
18 Years and older
Enrollment
50 participants
Timeline
2023 – 2025
U.S. locations
9
States / cities
Sacramento, California • New Haven, Connecticut • Miami, Florida + 6 more
Source: ClinicalTrials.gov public record
Updated Apr 6, 2026 · Synced Oct 14, 2026, 9:52 AM EDT
Recruiting Phase 3 Interventional

Phase 3 Trial of eRapa in Patients With Familial Adenomatous Polyposis

NCT06950385
View directory record Official record
Conditions
Familial Adenomatous Polyposis (FAP)
Interventions
eRapa (encapsulated rapamycin), Placebo
Drug
Lead sponsor
Rapamycin Holdings Inc.
Industry
Eligibility
18 Years and older
Enrollment
168 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2031
U.S. locations
18
States / cities
Arcadia, California • New Haven, Connecticut • Washington D.C., District of Columbia + 14 more
Source: ClinicalTrials.gov public record
Updated Jun 2, 2026 · Synced Oct 14, 2026, 9:52 AM EDT
Active, not recruiting Phase 3 Interventional

RZ358 Treatment for Congenital Hyperinsulinism

NCT06208215
View directory record Official record
Conditions
Congenital Hyperinsulinism
Interventions
RZ358 (5 mg/kg) + SOC (Standard-of-Care) or Placebo + SOC, RZ358 (10 mg/kg) or Placebo + SOC, RZ358 (5-10 mg/kg) + SOC
Drug
Lead sponsor
Rezolute
Other
Eligibility
3 Months to 45 Years
Enrollment
56 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
2
States / cities
Philadelphia, Pennsylvania • Fort Worth, Texas
Source: ClinicalTrials.gov public record
Updated Jan 8, 2026 · Synced Oct 14, 2026, 9:52 AM EDT
Completed Not applicable Interventional

Building Adherence to Live With And Navigate my Cystic Fibrosis (CF) Experience

NCT01013896
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Motivational Interviewing, Cystic Fibrosis Education
Behavioral · Other
Lead sponsor
Johns Hopkins University
Other
Eligibility
16 Years and older
Enrollment
128 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2014
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 31, 2015 · Synced Oct 14, 2026, 9:52 AM EDT
Enrolling by invitation Not applicable Interventional Accepts healthy volunteers

Palliative Care Needs of Children With Rare Diseases and Their Families

NCT06938542
View directory record Official record
Conditions
Trisomy 13 Syndrome, Arthrogryposis Congenita Multiplex With Intestinal Atresia, Asparagine Synthetase Deficiency, CHARGE Syndrome, Early Infantile Epileptic Encephalopathy, FOXG1 Syndrome, KBG Syndrome, Noonan Syndrome, Severe Hemophilia A, Short Bowel Syndrome, Beta-Propeller Protein-Associated Neurodegeneration, Brain Injury of Prematurity With Periventricular Leukomalacia, Chromosome 17p13.3 Microdeletion Syndrome, Chromosome 1q43-1q44 Deletion, Cockayne Syndrome, Congenital Diaphragmatic Hernia, End-Stage Renal Disease With Cloacal Anomaly, Mitochondrial Depletion Disorder, Severe Factor VII Deficiency
Interventions
Family Centered pediatric palliative care for family caregivers of children with rare diseases.
Behavioral
Lead sponsor
Children's National Research Institute
Other
Eligibility
12 Months to 99 Years
Enrollment
480 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2029
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Jan 28, 2026 · Synced Oct 14, 2026, 9:52 AM EDT
Completed No phase listed Observational Results available

Alpha-1 Carrier Genomics Study

NCT02810327
View directory record Official record
Conditions
Alpha-1 Antitrypsin Deficiency, Emphysema, COPD, Smoking
Interventions
Genetic Sequencing
Genetic
Lead sponsor
Medical University of South Carolina
Other
Eligibility
18 Years and older
Enrollment
117 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
1
States / cities
Charleston, South Carolina
Source: ClinicalTrials.gov public record
Updated Jul 22, 2019 · Synced Oct 14, 2026, 9:52 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Uncertain Genetic Test Results for Lynch Syndrome

NCT01646112
View directory record Official record
Conditions
HNPCC
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
18 Years to 100 Years
Enrollment
27 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2016
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 17, 2019 · Synced Oct 14, 2026, 9:52 AM EDT
Recruiting Not applicable Interventional Accepts healthy volunteers

Skin-interfaced Colorimetric Bifluidic Sweat Sensor Device for the Diagnosis of Cystic Fibrosis (CF)

NCT05998629
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
a skin-interfaced colorimetric bifluidic sweat device with two synchronous channels, Standard of Care laboratory procedure for measurement of sweat
Device · Other
Lead sponsor
Milton S. Hershey Medical Center
Other
Eligibility
18 Years to 80 Years
Enrollment
30 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2024 – 2026
U.S. locations
1
States / cities
Hershey, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jan 9, 2026 · Synced Oct 14, 2026, 9:52 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

In-Utero Vascular Accidents in Neonates From COVID-19 Infected Mothers

NCT04431869
View directory record Official record
Conditions
Pregnancy Related, COVID, Intestinal Atresia, Limb Anomaly
Interventions
Not listed
Lead sponsor
University of Colorado, Denver
Other
Eligibility
18 Years and older
Enrollment
215 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2021
U.S. locations
1
States / cities
Aurora, Colorado
Source: ClinicalTrials.gov public record
Updated Nov 29, 2023 · Synced Oct 14, 2026, 9:52 AM EDT
Terminated Phase 2 Interventional Results available

Carbamazepine in Severe Liver Disease Due to Alpha-1 Antitrypsin Deficiency

NCT01379469
View directory record Official record
Conditions
Alpha-1-antitrypsin Deficiency, Liver Cirrhosis
Interventions
Drug-Carbamazepine (Tegretol XR), Carbamazepine (Tegretol XR) Placebo
Drug
Lead sponsor
Washington University School of Medicine
Other
Eligibility
14 Years to 80 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2017
U.S. locations
3
States / cities
St Louis, Missouri • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 12, 2021 · Synced Oct 14, 2026, 9:52 AM EDT
Completed No phase listed Observational

Prevalence of Antibodies to Selected Porcine Viruses in Patients With Cystic Fibrosis Receiving Porcine-derived Pancreatic Enzyme Replacement Therapy

NCT01858519
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Not listed
Lead sponsor
AbbVie
Industry
Eligibility
2 Years to 99 Years
Enrollment
1,310 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
47
States / cities
Anchorage, Alaska • Little Rock, Arkansas • Long Beach, California + 41 more
Source: ClinicalTrials.gov public record
Updated Dec 9, 2020 · Synced Oct 14, 2026, 9:52 AM EDT
Completed No phase listed Observational

Gastrointestinal Microbiome Influence on the Development of Bronchopulmonary Dysplasia

NCT03229967
View directory record Official record
Conditions
Bronchopulmonary Dysplasia
Interventions
16S rRNA stool microbiome sequencing.
Diagnostic Test
Lead sponsor
University of Tennessee
Other
Eligibility
0 Days to 7 Days
Enrollment
197 participants
Timeline
2017 – 2020
U.S. locations
2
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated May 10, 2023 · Synced Oct 14, 2026, 9:52 AM EDT
Not listed No phase listed Observational

Simultaneous quanTification of Elexacaftor/tezAcaftor/Ivacaftor Via Reverse Phase High Performance liquiD chromatographY

NCT05822102
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Not listed
Lead sponsor
National Jewish Health
Other
Eligibility
18 Years and older
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023
U.S. locations
2
States / cities
Denver, Colorado
Source: ClinicalTrials.gov public record
Updated Jul 24, 2023 · Synced Oct 14, 2026, 9:52 AM EDT
Recruiting Phase 1 Interventional

A Study Assessing KB407 for the Treatment of Cystic Fibrosis

NCT05504837
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
KB407 (Nebulization)
Biological
Lead sponsor
Krystal Biotech, Inc.
Industry
Eligibility
18 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
6
States / cities
Gainesville, Florida • Boise, Idaho • Northfield, Illinois + 3 more
Source: ClinicalTrials.gov public record
Updated May 7, 2026 · Synced Oct 14, 2026, 9:52 AM EDT
Active, not recruiting Phase 1 Interventional

Interleukin-15 Armored Glypican 3-specific Chimeric Antigen Receptor Expressed in Autologous T Cells for Solid Tumors

NCT05103631
View directory record Official record
Conditions
Liver Cell Carcinoma, Solid Tumor, Wilms Tumor, Malignant Rhabdoid Tumor, Yolk Sac Tumor, Rhabdomyosarcoma, Liposarcoma, Embryonal Sarcoma of the Liver
Interventions
CATCH T cells
Genetic
Lead sponsor
Baylor College of Medicine
Other
Eligibility
18 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2040
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Mar 6, 2026 · Synced Oct 14, 2026, 9:52 AM EDT
Terminated Phase 2 Interventional Results available

Evaluation of the Efficacy and Safety of VX-814 in Subjects With the PiZZ Genotype

NCT04167345
View directory record Official record
Conditions
Alpha 1-Antitrypsin Deficiency
Interventions
VX-814, Placebo
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
18 Years to 80 Years
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020
U.S. locations
22
States / cities
Birmingham, Alabama • Sacramento, California • Denver, Colorado + 19 more
Source: ClinicalTrials.gov public record
Updated Feb 2, 2022 · Synced Oct 14, 2026, 9:52 AM EDT
Completed Phase 2 Interventional Results available

Safety, Tolerability and Efficacy of MP-376 Given for 28 Days to Cystic Fibrosis (CF) Patients

NCT00677365
View directory record Official record
Conditions
Cystic Fibrosis (CF)
Interventions
MP-376, Placebo
Drug
Lead sponsor
Amgen
Industry
Eligibility
16 Years and older
Enrollment
151 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2009
U.S. locations
39
States / cities
Mobile, Alabama • Phoenix, Arizona • Tucson, Arizona + 34 more
Source: ClinicalTrials.gov public record
Updated Dec 27, 2024 · Synced Oct 14, 2026, 9:52 AM EDT
Completed Phase 2 Interventional

Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of QAU145 in Patients With Cystic Fibrosis

NCT00506792
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
QAU145, Placebo
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
18 Years to 50 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2009
U.S. locations
1
States / cities
Birmingham, Alabama
Source: ClinicalTrials.gov public record
Updated Dec 19, 2020 · Synced Oct 14, 2026, 9:52 AM EDT
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 Oct 14, 2026, 9:52 AM EDT
Completed Phase 2 Interventional

Aztreonam for Inhalation (AI) in Patients With Cystic Fibrosis & P. Aeruginosa Infection

NCT01055847
View directory record Official record
Conditions
Cystic Fibrosis, CF, Lung Infection, Pseudomonas Aeruginosa
Interventions
Aztreonam for Inhalation (AI), Placebo
Drug
Lead sponsor
Gilead Sciences
Industry
Eligibility
13 Years and older
Enrollment
105 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2004
U.S. locations
20
States / cities
Los Angeles, California • Orange, California • San Diego, California + 17 more
Source: ClinicalTrials.gov public record
Updated Jan 26, 2010 · Synced Oct 14, 2026, 9:52 AM EDT
Completed Phase 2 Interventional Results available

Aztreonam Lysine for Pseudomonas Infection Eradication Study

NCT01375049
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Aztreonam for Inhalation Solution (AZLI)
Drug
Lead sponsor
Gilead Sciences
Industry
Eligibility
3 Months to 17 Years
Enrollment
105 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
23
States / cities
Phoenix, Arizona • Jacksonville, Florida • Orlando, Florida + 20 more
Source: ClinicalTrials.gov public record
Updated Jul 17, 2014 · Synced Oct 14, 2026, 9:52 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

(Study: Vertex IIS) Does Ivacaftor Alter Wild Type CFTR-Open Probability In The Sweat Gland Secretory Coil?

NCT02310789
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Ivacaftor, β-Adrenergic cocktail, Pilocarpine Nitrate 5%, Macroduct sweat stimulator
Drug · Device
Lead sponsor
Richard Barry Moss
Other
Eligibility
18 Years and older
Enrollment
8 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2017
U.S. locations
1
States / cities
Stanford, California
Source: ClinicalTrials.gov public record
Updated Jan 9, 2019 · Synced Oct 14, 2026, 9:52 AM EDT