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ClinicalTrials.gov public records Last synced Sep 13, 2026, 11:22 AM EDT

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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Gene Mutation Clear all
Recruiting Phase 1Phase 2 Interventional

Base Editing for Mutation Repair in Hematopoietic Stem & Progenitor Cells for X-Linked Chronic Granulomatous Disease

NCT06325709
View directory record Official record
Conditions
Chronic Granulomatous Disease (CGD), X-Linked Chronic Granulomatous Disease
Interventions
Campath, Sirolimus, Base-edited hematopoietic stem and progenitor cells, Busulfan, Palifermin, Filgrastim, Plerixafor
Drug · Biological
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
18 Years to 75 Years · Male only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2032
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 12, 2026 · Synced Sep 13, 2026, 11:22 AM EDT
Terminated Phase 3 Interventional Results available

A Study of a Single Dose of Inclacumab to Reduce Re-admission in Participants With Sickle Cell Disease and Recurrent Vaso-occlusive Crises

NCT04927247
View directory record Official record
Conditions
Sickle Cell Disease, Vaso-occlusive Crisis, Vaso-occlusive Pain Episode in Sickle Cell Disease
Interventions
Inclacumab, Placebo
Drug
Lead sponsor
Pfizer
Industry
Eligibility
12 Years and older
Enrollment
72 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
17
States / cities
Mobile, Alabama • Phoenix, Arizona • Little Rock, Arkansas + 11 more
Source: ClinicalTrials.gov public record
Updated Dec 10, 2024 · Synced Sep 13, 2026, 11:22 AM EDT
Terminated Phase 2 Interventional Results available

Study to Evaluate Adverse Events and Change in Disease Activity With Oral Capsules of Galicaftor/Navocaftor/ABBV-119 or Galicaftor/Navocaftor/ABBV-576 Combination Therapies in Adult Participants With Cystic Fibrosis

NCT04853368
View directory record Official record
Conditions
Cystic Fibrosis (CF)
Interventions
ABBV-576, Galicaftor, Placebo, Navocaftor, ABBV-119
Drug
Lead sponsor
AbbVie
Industry
Eligibility
18 Years and older
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
22
States / cities
Mobile, Alabama • Los Angeles, California • Ventura, California + 19 more
Source: ClinicalTrials.gov public record
Updated Jul 15, 2024 · Synced Sep 13, 2026, 11:22 AM EDT
Active, not recruiting Phase 2 Interventional

A Study Comparing Two Doses of AGTC-501 in Male Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (SKYLINE)

NCT06333249
View directory record Official record
Conditions
X-Linked Retinitis Pigmentosa
Interventions
rAAV2tYF-GRK1-RPGR
Biological
Lead sponsor
Beacon Therapeutics
Industry
Eligibility
8 Years to 50 Years · Male only
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
6
States / cities
Jacksonville, Florida • Boston, Massachusetts • Cincinnati, Ohio + 3 more
Source: ClinicalTrials.gov public record
Updated Aug 11, 2024 · Synced Sep 13, 2026, 11:22 AM EDT
Completed No phase listed Observational

Alternative Treatment Strategies for Stenotrophomonas Maltophilia Pneumonia

NCT06112938
View directory record Official record
Conditions
Stenotrophomonas Maltophilia Infection
Interventions
Antibiotic
Drug
Lead sponsor
Methodist Health System
Other
Eligibility
18 Years and older
Enrollment
110 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Jul 27, 2025 · Synced Sep 13, 2026, 11:22 AM EDT
Not listed Not applicable Interventional

Using Mobile Devices for Neurofeedback to Reduce Opioid Use in Chronic Pain

NCT04838925
View directory record Official record
Conditions
Chronic Pain, Opioid Use
Interventions
Mobile Neurofeedback App
Device
Lead sponsor
CrossComm, Inc.
Industry
Eligibility
18 Years and older
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Aug 23, 2023 · Synced Sep 13, 2026, 11:22 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Common Genetic Variation and Type 2 Diabetes

NCT00707616
View directory record Official record
Conditions
Pre-diabetes
Interventions
Oral glucose tolerance test
Other
Lead sponsor
Mayo Clinic
Other
Eligibility
35 Years to 80 Years
Enrollment
190 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2006 – 2012
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Jan 22, 2013 · Synced Sep 13, 2026, 11:22 AM EDT
Recruiting Phase 1Phase 2 Interventional

PRE-ISPY Phase I/II Oncology Platform Program

NCT05868226
View directory record Official record
Conditions
HER2-positive Breast Cancer, Metastatic Cancer, Metastatic Breast Cancer, Metastatic, HER2-positive Metastatic Breast Cancer, HER2 Mutation-Related Tumors, HER-2 Protein Overexpression, HER2-negative Breast Cancer, Triple Negative Breast Cancer, HR Positive, Hormone Receptor-positive Breast Cancer, Estrogen Receptor Positive Tumor, Progesterone Receptor-positive Breast Cancer, Hormone Receptor Negative Breast Carcinoma, Solid Tumor, Solid Tumor, Adult, Solid Carcinoma, HER2 Low Breast Cancer, HER2 Low Breast Carcinoma, ER Positive Breast Cancer, PR-positive Breast Cancer, Minimal Residual Disease, Colo-rectal Cancer, Colon Cancer, Rectal Cancer, ctDNA Monitoring, Colorectal Neoplasms, Colonic Neoplasms, Rectal Neoplasms, Neoplasm, Residual, Adenocarcinoma of Colon, Adenocarcinoma
Interventions
ALX148, Fam-Trastuzumab Deruxtecan-Nxki, Zanidatamab, Tucatinib, TTX-MC138
Drug
Lead sponsor
QuantumLeap Healthcare Collaborative
Other
Eligibility
18 Years and older
Enrollment
280 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2031
U.S. locations
10
States / cities
Birmingham, Alabama • Phoenix, Arizona • Aurora, Colorado + 7 more
Source: ClinicalTrials.gov public record
Updated Sep 1, 2026 · Synced Sep 13, 2026, 11:22 AM EDT
Completed Phase 1 Interventional

Enasidenib in Combination With Cobimetinib for the Treatment of Relapsed or Refractory Acute Myeloid Leukemia

NCT05441514
View directory record Official record
Conditions
Recurrent Acute Myeloid Leukemia, Refractory Acute Myeloid Leukemia
Interventions
Cobimetinib, Enasidenib Mesylate
Drug
Lead sponsor
City of Hope Medical Center
Other
Eligibility
18 Years and older
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
1
States / cities
Duarte, California
Source: ClinicalTrials.gov public record
Updated May 4, 2026 · Synced Sep 13, 2026, 11:22 AM EDT
Completed Phase 3 Interventional Results available

A Phase 3 Study of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)

NCT03447249
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
VX-659/TEZ/IVA, IVA, Placebo
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
12 Years and older
Enrollment
385 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
48
States / cities
Birmingham, Alabama • Palo Alto, California • Aurora, Colorado + 44 more
Source: ClinicalTrials.gov public record
Updated Mar 12, 2020 · Synced Sep 13, 2026, 11:22 AM EDT
Completed Phase 3 Interventional Results available

A Study of Lumacaftor in Combination With Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older Who Are Homozygous for the F508del-CFTR Mutation

NCT01807949
View directory record Official record
Conditions
Cystic Fibrosis, Homozygous for the F508del CFTR Mutation
Interventions
Placebo, Lumacaftor Plus Ivacaftor Combination, Ivacaftor
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
12 Years to 65 Years
Enrollment
563 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
46
States / cities
Oakland, California • Sacramento, California • Aurora, Colorado + 43 more
Source: ClinicalTrials.gov public record
Updated Sep 26, 2016 · Synced Sep 13, 2026, 11:22 AM EDT
Terminated Not applicable Interventional Results available

Recombinant Human Chorionic Gonadotropin in Preventing Breast Cancer in Premenopausal Women With BRCA1 Mutations

NCT00700778
View directory record Official record
Conditions
BRCA1 Mutation, Breast Cancer
Interventions
recombinant human chorionic gonadotropin, microarray analysis, immunohistochemistry staining method, laboratory biomarker analysis, needle biopsy
Biological · Genetic · Other + 1 more
Lead sponsor
Fox Chase Cancer Center
Other
Eligibility
20 Years to 40 Years · Female only
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2014
U.S. locations
2
States / cities
Rochester, Minnesota • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jan 24, 2021 · Synced Sep 13, 2026, 11:22 AM EDT
Recruiting No phase listed Observational

Non-interventional Study of Seroprevalence of Pre-existing Antibodies Against Adenovirus-associated Virus Vector (AAV9) and the Progression of Disease in Patients With Plakophilin 2 (PKP2)-Associated Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC)

NCT06311708
View directory record Official record
Conditions
Arrhythmogenic Right Ventricular Cardiomyopathy
Interventions
Not listed
Lead sponsor
Tenaya Therapeutics
Industry
Eligibility
14 Years to 65 Years
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2030
U.S. locations
8
States / cities
San Francisco, California • Aurora, Colorado • Baltimore, Maryland + 5 more
Source: ClinicalTrials.gov public record
Updated Oct 31, 2024 · Synced Sep 13, 2026, 11:22 AM EDT
Completed Phase 2 Interventional Results available

Study of VX-661 Alone and in Combination With Ivacaftor in Subjects Homozygous or Heterozygous to the F508del-Cystic Fibrosis Transmembrane Conductance Regulator(CFTR) Mutation

NCT01531673
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
VX-661, Ivacaftor, Placebo matched to VX-661, Placebo matched to ivacaftor
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
12 Years and older
Enrollment
194 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2014
U.S. locations
19
States / cities
Birmingham, Alabama • Oakland, California • Boise, Idaho + 16 more
Source: ClinicalTrials.gov public record
Updated Apr 12, 2018 · Synced Sep 13, 2026, 11:22 AM EDT
Completed Phase 1 Interventional

A Phase I Study of T-Cells Genetically Modified at the CCR5 Gene by Zinc Finger Nucleases SB-728mR in HIV-Infected Patients

NCT02388594
View directory record Official record
Conditions
Human Immunodeficiency Virus (HIV)
Interventions
ZFN Modified CD4+ T Cells, Cyclophosphamide
Drug
Lead sponsor
University of Pennsylvania
Other
Eligibility
18 Years and older
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 9, 2019 · Synced Sep 13, 2026, 11:22 AM EDT
Completed Phase 4 Interventional Accepts healthy volunteers

Effects of Kava on the Body's Elimination of Caffeine and Dextromethorphan

NCT00009542
View directory record Official record
Conditions
Healthy
Interventions
kava (Piper methysticum)
Drug
Lead sponsor
National Institutes of Health Clinical Center (CC)
NIH
Eligibility
Not listed
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2001
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced Sep 13, 2026, 11:22 AM EDT
Completed Phase 2 Interventional Results available

Study to Determine the Effectiveness of GSK1120212 in BRAF Mutation-positive Melanoma Previously Treated With or Without a BRAF Inhibitor

NCT01037127
View directory record Official record
Conditions
Cancer
Interventions
GSK1120212
Drug
Lead sponsor
GlaxoSmithKline
Industry
Eligibility
18 Years and older
Enrollment
97 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2013
U.S. locations
7
States / cities
Los Angeles, California • Aurora, Colorado • New York, New York + 3 more
Source: ClinicalTrials.gov public record
Updated Mar 30, 2014 · Synced Sep 13, 2026, 11:22 AM EDT
Not listed Not applicable Interventional Accepts healthy volunteers

Early Augmentative and Alternative Communication (AAC) Intervention Delivered Via Hybrid Telehealth

NCT05743439
View directory record Official record
Conditions
Intellectual Disability, Speech and Language Disorder
Interventions
Early Augmentative and Alternative Communication (AAC) Intervention
Behavioral
Lead sponsor
Oregon Health and Science University
Other
Eligibility
24 Months and older
Enrollment
50 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2025
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Apr 4, 2024 · Synced Sep 13, 2026, 11:22 AM EDT
Withdrawn Early Phase 1 Interventional

Multimodality MRI and Liquid Biopsy in GBM

NCT04776980
View directory record Official record
Conditions
Glioblastoma Multiforme, Brain Tumor, Adult: Glioblastoma, Brain Tumor, Recurrent, Brain Tumor, Primary
Interventions
Post Feraheme Infusion MRI
Diagnostic Test
Lead sponsor
Abramson Cancer Center at Penn Medicine
Other
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Sep 15, 2022 · Synced Sep 13, 2026, 11:22 AM EDT
Completed Phase 2 Interventional Results available

Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy

NCT02819557
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Ataluren
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
2 Years to 5 Years · Male only
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
6
States / cities
Gulf Breeze, Florida • Chicago, Illinois • Boston, Massachusetts + 3 more
Source: ClinicalTrials.gov public record
Updated Aug 27, 2020 · Synced Sep 13, 2026, 11:22 AM EDT
Active, not recruiting Phase 2 Interventional Results available

Growth Hormone in a Patient With a Dominant-Negative GHR Mutation

NCT05382637
View directory record Official record
Conditions
Growth Disorders
Interventions
Somatropin
Drug
Lead sponsor
Children's National Research Institute
Other
Eligibility
9 Years and older · Male only
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2028
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Jul 1, 2025 · Synced Sep 13, 2026, 11:22 AM EDT
Completed Phase 1Phase 2 Interventional

Safety and Efficacy Study of BMS-908662 Alone or in Combination With Cetuximab in Subjects With K-RAS or B-RAF Mutation Positive Advanced or Metastatic Colorectal Cancer

NCT01086267
View directory record Official record
Conditions
Colorectal Cancer
Interventions
BMS-908662, Cetuximab
Drug
Lead sponsor
Bristol-Myers Squibb
Industry
Eligibility
18 Years and older
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2011
U.S. locations
2
States / cities
Scottsdale, Arizona • Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Jun 26, 2016 · Synced Sep 13, 2026, 11:22 AM EDT
Completed Phase 2 Interventional

Comparison of Treatments to Maintain Disease Remission in Patients With Wegener's Granulomatosis and Related Vasculitis Syndromes

NCT00004567
View directory record Official record
Conditions
Vasculitis, Wegener's Granulomatosis
Interventions
Mycophenolate Mofetil
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
Not listed
Enrollment
75 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2004
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced Sep 13, 2026, 11:22 AM EDT
Completed Not applicable Interventional Results available

Tele-Exercise and Multiple Sclerosis

NCT03117881
View directory record Official record
Conditions
Multiple Sclerosis
Interventions
DirectCAM, TeleCAM, rDirectCAM
Behavioral
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
18 Years to 70 Years
Enrollment
911 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
40
States / cities
Athens, Alabama • Bessemer, Alabama • Birmingham, Alabama + 37 more
Source: ClinicalTrials.gov public record
Updated Dec 10, 2024 · Synced Sep 13, 2026, 11:22 AM EDT