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ClinicalTrials.gov public records Last synced Oct 17, 2026, 5:02 PM EDT

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Showing 745–768 of 1,323 matching trials from the live ClinicalTrials.gov search.
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Terminated Phase 3 Interventional Results available

Extension Study of Ataluren in Participants With Nonsense Mutation Cystic Fibrosis

NCT02456103
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Ataluren
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
6 Years and older
Enrollment
246 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
24
States / cities
Birmingham, Alabama • Long Beach, California • Los Angeles, California + 18 more
Source: ClinicalTrials.gov public record
Updated Apr 27, 2020 · Synced Oct 17, 2026, 5:02 PM 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 17, 2026, 5:02 PM EDT
Completed Phase 1 Interventional

A Study to Assess the Effect of AZD5634 on Mucociliary Clearance, Safety, Tolerability and Pharmacokinetic Parameters in Patients With Cystic Fibrosis

NCT02950805
View directory record Official record
Conditions
Pulmonary/Respiratory Diseases
Interventions
Placebo, AZD5634
Drug
Lead sponsor
AstraZeneca
Industry
Eligibility
18 Years to 60 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2018
U.S. locations
3
States / cities
Birmingham, Alabama • Baltimore, Maryland • Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Jul 13, 2018 · Synced Oct 17, 2026, 5:02 PM EDT
Completed Not applicable Interventional Results available

Impact of Discontinuing Hypertonic Saline in People With CF on Highly Effective CFTR Modulators- A SIMPLIFY Sub-Study

NCT06350461
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Discontinuation of hypertonic saline (HS), Continuation of hypertonic saline (HS)
Other
Lead sponsor
Nicole Hamblett
Other
Eligibility
12 Years and older
Enrollment
370 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
81
States / cities
Birmingham, Alabama • Anchorage, Alaska • Tucson, Arizona + 69 more
Source: ClinicalTrials.gov public record
Updated Oct 1, 2024 · Synced Oct 17, 2026, 5:02 PM EDT
Completed Phase 1 Interventional

Effect of Simvastatin on CF Airway Inflammation

NCT00255242
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Simvastatin treatment for 28 days
Drug
Lead sponsor
Akron Children's Hospital
Other
Eligibility
10 Years and older
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2009
U.S. locations
1
States / cities
Akron, Ohio
Source: ClinicalTrials.gov public record
Updated Aug 3, 2009 · Synced Oct 17, 2026, 5:02 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Study of Vanzacaftor/Tezacaftor/Deutivacaftor (VNZ/TEZ/D-IVA) in Healthy Adult Panelists

NCT06299696
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
VNZ/TEZ/D-IVA
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
25 Years to 80 Years
Enrollment
10 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2024
U.S. locations
1
States / cities
Woburn, Massachusetts
Source: ClinicalTrials.gov public record
Updated May 16, 2024 · Synced Oct 17, 2026, 5:02 PM EDT
Completed Phase 1Phase 2 Interventional

A Study To Evaluate The Safety Of CMTX-101 In People With Cystic Fibrosis

NCT06159725
View directory record Official record
Conditions
Persistent Infection, Cystic Fibrosis
Interventions
CMTX-101, Placebo
Drug
Lead sponsor
Clarametyx Biosciences, Inc.
Industry
Eligibility
18 Years and older
Enrollment
43 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2025
U.S. locations
23
States / cities
Birmingham, Alabama • Palo Alto, California • San Franciso, California + 20 more
Source: ClinicalTrials.gov public record
Updated May 5, 2026 · Synced Oct 17, 2026, 5:02 PM EDT
Completed Phase 2 Interventional Results available

Utility of [F-18] fluoroDOPA for Neonatal Hyperinsulinism

NCT00674440
View directory record Official record
Conditions
Congenital Hyperinsulinism, Hyperinsulinism, Persistent Hyperinsulinemic Hypoglycemia of Infancy, CHI, PHHI
Interventions
F-DOPA, PET scan
Drug · Radiation
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
Up to 18 Years
Enrollment
106 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2009
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Nov 9, 2022 · Synced Oct 17, 2026, 5:02 PM EDT
Withdrawn Not applicable Interventional

Utility of Bowel Ultrasound in Diagnosing Necrotizing Eneterocolitis in Congenital Heart Disease

NCT03142685
View directory record Official record
Conditions
Enterocolitis, Necrotizing, Congenital Heart Disease
Interventions
Bowel Ultrasound
Device
Lead sponsor
Children's Mercy Hospital Kansas City
Other
Eligibility
Up to 6 Months
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2018
U.S. locations
1
States / cities
Kansas City, Missouri
Source: ClinicalTrials.gov public record
Updated Jul 18, 2018 · Synced Oct 17, 2026, 5:02 PM EDT
Completed Phase 2 Interventional

Combination Chemotherapy Followed by Bone Marrow Transplantation in Treating Patients With Rare Cancer

NCT00002515
View directory record Official record
Conditions
Childhood Germ Cell Tumor, Extragonadal Germ Cell Tumor, Head and Neck Cancer, Kidney Cancer, Liver Cancer, Lymphoma, Neuroblastoma, Ovarian Cancer, Retinoblastoma, Sarcoma, Testicular Germ Cell Tumor
Interventions
filgrastim, carboplatin, thiotepa, topotecan hydrochloride, autologous bone marrow transplantation, bone marrow ablation with stem cell support, in vitro-treated bone marrow transplantation
Biological · Drug · Procedure
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
Up to 21 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
1992 – 2005
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 24, 2013 · Synced Oct 17, 2026, 5:02 PM EDT
Completed No phase listed Observational

EXPLORE: A Natural History Study of Acute Hepatic Porphyria (AHP)

NCT02240784
View directory record Official record
Conditions
Acute Hepatic Porphyria
Interventions
Not listed
Lead sponsor
Alnylam Pharmaceuticals
Industry
Eligibility
12 Years and older
Enrollment
136 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2021
U.S. locations
8
States / cities
San Francisco, California • Miami, Florida • Ann Arbor, Michigan + 5 more
Source: ClinicalTrials.gov public record
Updated May 17, 2021 · Synced Oct 17, 2026, 5:02 PM EDT
Completed No phase listed Observational Results available

: TRANSITION: An Observational Study of Transition From Lumacaftor/Ivacaftor to Tezacaftor/Ivacaftor (Tez/Iva)

NCT03445793
View directory record Official record
Conditions
Cystic Fibrosis (CF)
Interventions
Not listed
Lead sponsor
National Jewish Health
Other
Eligibility
12 Years and older
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
1
States / cities
Denver, Colorado
Source: ClinicalTrials.gov public record
Updated Jan 23, 2024 · Synced Oct 17, 2026, 5:02 PM EDT
Withdrawn Not applicable Interventional Accepts healthy volunteers

The Sequestration of Holistic Stress Management Techniques for Adults

NCT06098755
View directory record Official record
Conditions
Stress, Job, PTSD, Hypervigilance, Hyperthyroidism, Migraine, Migraine With Aura, Immunodeficiency, Sickle Cell Trait, Sickle Cell Disease, Anemia, Sickle Cell, Anemia, Fatigue, Anxiety, Depression, Flat Feet, Lordosis, TBI (Traumatic Brain Injury), Rheumatoid Arthritis, Arthritis, Masturbation, Priapism, Erectile Dysfunction, Flight Reaction, Hypertension, Sexual Arousal Disorder, Hemorrhoids, Fibroid
Interventions
Holistic Medicine (HM)
Behavioral
Lead sponsor
Goddess Zena I. Jones
Other
Eligibility
22 Years and older
Healthy volunteers
Accepts healthy volunteers
Timeline
2026 – 2028
U.S. locations
1
States / cities
Anchorage, Alaska
Source: ClinicalTrials.gov public record
Updated Feb 4, 2026 · Synced Oct 17, 2026, 5:02 PM EDT
Not listed Not applicable Interventional Accepts healthy volunteers

Comparing the Adaptation of Commercial Milk and A2 Milk in Lactose Maldigesters

NCT05669274
View directory record Official record
Conditions
Lactose Intolerance
Interventions
Milk containing A1 and A2 beta-casein, Milk containing only A2 beta-casein
Other
Lead sponsor
Purdue University
Other
Eligibility
18 Years to 65 Years
Enrollment
35 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2025
U.S. locations
1
States / cities
West Lafayette, Indiana
Source: ClinicalTrials.gov public record
Updated Mar 30, 2023 · Synced Oct 17, 2026, 5:02 PM EDT
Active, not recruiting No phase listed Observational

Prospective Study of Pregnancy in Women With Cystic Fibrosis

NCT04828382
View directory record Official record
Conditions
Pregnancy Related, Cystic Fibrosis
Interventions
Not listed
Lead sponsor
Amalia Magaret
Other
Eligibility
16 Years and older · Female only
Enrollment
285 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
40
States / cities
Birmingham, Alabama • Little Rock, Arkansas • La Jolla, California + 35 more
Source: ClinicalTrials.gov public record
Updated Nov 3, 2025 · Synced Oct 17, 2026, 5:02 PM EDT
Recruiting No phase listed Observational

Rare CFTR Mutation Cell Collection Protocol (RARE)

NCT03161808
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Not listed
Lead sponsor
George Solomon
Other
Eligibility
17 Years and older
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2028
U.S. locations
1
States / cities
Birmingham, Alabama
Source: ClinicalTrials.gov public record
Updated May 29, 2026 · Synced Oct 17, 2026, 5:02 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers Results available

Study of ALXN1840 on the Metabolism of a CYP2B6 Substrate in Healthy Participants

NCT04526210
View directory record Official record
Conditions
Wilson Disease
Interventions
ALXN1840, Bupropion Hydrochloride
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
18 Years to 50 Years
Enrollment
54 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2021
U.S. locations
1
States / cities
Austin, Texas
Source: ClinicalTrials.gov public record
Updated Aug 31, 2023 · Synced Oct 17, 2026, 5:02 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Evaluation of GIMate Handheld Hydrogen Breath Monitor for Diagnosis of Lactose Malabsorption

NCT04754724
View directory record Official record
Conditions
Lactose Intolerance
Interventions
GIMate, H2 Check
Diagnostic Test
Lead sponsor
Vivante Health
Industry
Eligibility
18 Years to 55 Years
Enrollment
31 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Sep 28, 2021 · Synced Oct 17, 2026, 5:02 PM EDT
Withdrawn No phase listed Observational

Biomarkers in Samples From Patients With Endometrial Cancer

NCT01199250
View directory record Official record
Conditions
Lynch Syndrome, Recurrent Uterine Corpus Carcinoma, Stage I Uterine Corpus Cancer, Stage II Uterine Corpus Cancer, Stage III Uterine Corpus Cancer, Stage IV Uterine Corpus Cancer
Interventions
Laboratory Biomarker Analysis
Other
Lead sponsor
Gynecologic Oncology Group
Network
Eligibility
Female only
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2019
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 14, 2024 · Synced Oct 17, 2026, 5:02 PM EDT
Completed Phase 2 Interventional Results available

A Study to Evaluate Multiple Doses of GLPG2222 in Adult Subjects With Cystic Fibrosis

NCT03119649
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
GLPG2222 50 mg, GLPG2222 100 mg, Placebo, GLPG2222 200 mg, GLPG2222 400 mg
Drug
Lead sponsor
Lakefront Biotherapeutics NV
Industry
Eligibility
18 Years to 99 Years
Enrollment
59 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017
U.S. locations
7
States / cities
Chatom, Alabama • Little Rock, Arkansas • Orlando, Florida + 4 more
Source: ClinicalTrials.gov public record
Updated Nov 16, 2018 · Synced Oct 17, 2026, 5:02 PM EDT
Terminated Not applicable Interventional Results available

Neuromodulation for Children With Cystic Fibrosis Experiencing Chronic Abdominal Pain

NCT05515250
View directory record Official record
Conditions
Cystic Fibrosis, Irritable Bowel Syndrome, Chronic Pain
Interventions
Use of neurostimulation device
Device
Lead sponsor
St. Louis University
Other
Eligibility
11 Years to 18 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Aug 9, 2024 · Synced Oct 17, 2026, 5:02 PM EDT
Recruiting Phase 1 Interventional

A Study of KB408 for the Treatment of Alpha-1 Antitrypsin Deficiency

NCT06049082
View directory record Official record
Conditions
Alpha 1-Antitrypsin Deficiency
Interventions
KB408 (Nebulization)
Drug
Lead sponsor
Krystal Biotech, Inc.
Industry
Eligibility
18 Years to 70 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
3
States / cities
Gainesville, Florida • Charleston, South Carolina • The Woodlands, Texas
Source: ClinicalTrials.gov public record
Updated Jul 22, 2025 · Synced Oct 17, 2026, 5:02 PM EDT
Approved For Marketing No phase listed Expanded access

Aztreonam Lysine for Inhalation in Patients With Cystic Fibrosis and Pseudomonas Aeruginosa Airway Infection

NCT00499720
View directory record Official record
Conditions
Cystic Fibrosis, Pseudomonas Aeruginosa Airway Infection
Interventions
Aztreonam Lysine for Inhalation
Drug
Lead sponsor
Gilead Sciences
Industry
Eligibility
6 Years and older
U.S. locations
64
States / cities
Birmingham, Alabama • Anchorage, Alaska • Phoenix, Arizona + 53 more
Source: ClinicalTrials.gov public record
Updated Aug 2, 2011 · Synced Oct 17, 2026, 5:02 PM EDT
Recruiting Phase 2 Interventional

4D-710 in Adult Patients With Cystic Fibrosis

NCT05248230
View directory record Official record
Conditions
Cystic Fibrosis Lung
Interventions
4D-710
Biological
Lead sponsor
4D Molecular Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2031
U.S. locations
20
States / cities
Birmingham, Alabama • Tucson, Arizona • San Francisco, California + 16 more
Source: ClinicalTrials.gov public record
Updated Jul 6, 2026 · Synced Oct 17, 2026, 5:02 PM EDT