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ClinicalTrials.gov public records Last synced Sep 6, 2026, 9:36 PM EDT

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Showing 1–24 of 938 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Filters: Condition: Genetic Mutations Clear all
Active, not recruiting Phase 3 Interventional

REZILIENT3 (REsearching ZIpaLertinib In Egfr Non-small Cell Lung Cancer Tumors)

NCT05973773
View directory record Official record
Conditions
Advanced or Metastatic NSCLS With Exon 20 Insertion Mutation
Interventions
TAS6417
Drug
Lead sponsor
Taiho Oncology, Inc.
Industry
Eligibility
18 Years and older
Enrollment
285 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
9
States / cities
Henderson, Nevada • Las Vegas, Nevada • Canton, Ohio + 1 more
Source: ClinicalTrials.gov public record
Updated Jul 29, 2026 · Synced Sep 6, 2026, 9:36 PM EDT
Completed Phase 2 Interventional Results available

Phase II ABT-888 With Cyclophosphamide

NCT01306032
View directory record Official record
Conditions
Ovarian Cancer, Primary Peritoneal Cancer, Serous Carcinoma Cancer, Triple-Negative Breast Cancer, Fallopian Tube Cancer
Interventions
ABT-888, Cyclophosphamide
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
124 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2016
U.S. locations
9
States / cities
Davis, California • Tampa, Florida • Chicago, Illinois + 6 more
Source: ClinicalTrials.gov public record
Updated Apr 25, 2017 · Synced Sep 6, 2026, 9:36 PM EDT
Recruiting Phase 1 Interventional

A Study to Investigate the Safety and Efficacy of KQB365 as Monotherapy and in Combination in Participants With Advanced Solid Malignancies

NCT06720987
View directory record Official record
Conditions
KRAS G12C Mutation, KRAS G12S Mutation, Solid Tumor Malignancies, CRC (Colorectal Cancer)
Interventions
KQB365, Cetuximab, KQB198
Drug
Lead sponsor
Kumquat Biosciences Inc.
Industry
Eligibility
18 Years and older
Enrollment
140 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
11
States / cities
Phoenix, Arizona • Denver, Colorado • Jacksonville, Florida + 8 more
Source: ClinicalTrials.gov public record
Updated Apr 22, 2026 · Synced Sep 6, 2026, 9:36 PM EDT
Recruiting Early Phase 1 Interventional

FES Imaging to Optimize Tamoxifen for Metastatic Breast Cancer

NCT04174352
View directory record Official record
Conditions
ERα+ Breast Cancer, ESR1 Gene Mutation
Interventions
Tamoxifen, FES PET/CT
Drug · Diagnostic Test
Lead sponsor
University of Wisconsin, Madison
Other
Eligibility
19 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2027
U.S. locations
2
States / cities
Madison, Wisconsin
Source: ClinicalTrials.gov public record
Updated Jul 20, 2026 · Synced Sep 6, 2026, 9:36 PM EDT
Terminated Phase 3 Interventional

A Study to Evaluate the Long-term Safety of Inclacumab Administered to Participants With Sickle Cell Disease

NCT05348915
View directory record Official record
Conditions
Sickle Cell Disease, Vaso-occlusive Crisis, Vaso-occlusive Pain Episode in Sickle Cell Disease
Interventions
Inclacumab
Drug
Lead sponsor
Pfizer
Industry
Eligibility
12 Years and older
Enrollment
242 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
18
States / cities
Mobile, Alabama • Little Rock, Arkansas • Orange, California + 7 more
Source: ClinicalTrials.gov public record
Updated Dec 16, 2025 · Synced Sep 6, 2026, 9:36 PM EDT
Not listed Phase 1Phase 2 Interventional

Single Ascending Dose Study in Participants With LCA10

NCT03872479
View directory record Official record
Conditions
Leber Congenital Amaurosis 10, Inherited Retinal Dystrophies, Eye Diseases, Hereditary, Retinal Disease, Retinal Degeneration, Vision Disorders, Eye Disorders Congenital
Interventions
EDIT-101
Drug
Lead sponsor
Editas Medicine, Inc.
Industry
Eligibility
3 Years and older
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2025
U.S. locations
5
States / cities
Miami, Florida • Boston, Massachusetts • Ann Arbor, Michigan + 2 more
Source: ClinicalTrials.gov public record
Updated Dec 4, 2022 · Synced Sep 6, 2026, 9:36 PM 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 6, 2026, 9:36 PM EDT
Recruiting No phase listed Observational

Genetic and Functional Analysis of Craniometaphyseal Dysplasia (CMD)

NCT01630460
View directory record Official record
Conditions
Craniometaphyseal Dysplasia
Interventions
Not listed
Lead sponsor
UConn Health
Other
Eligibility
Not listed
Enrollment
600 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2030
U.S. locations
1
States / cities
Farmington, Connecticut
Source: ClinicalTrials.gov public record
Updated Apr 16, 2026 · Synced Sep 6, 2026, 9:36 PM EDT
Completed No phase listed Observational

Natural History Study of FDXR Mutation-related Mitochondriopathy

NCT04580979
View directory record Official record
Conditions
Neurodegenerative Disease, Hereditary, Mitochondrial Diseases, Optic Atrophy
Interventions
Mutation analysis
Genetic
Lead sponsor
State University of New York at Buffalo
Other
Eligibility
0 Years to 65 Years
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
1
States / cities
Buffalo, New York
Source: ClinicalTrials.gov public record
Updated Mar 7, 2024 · Synced Sep 6, 2026, 9:36 PM EDT
Active, not recruiting Phase 1 Interventional

A Study to Learn About the Study Medicine PF-07934040 When Given Alone or With Other Anti-cancer Therapies in People With Advanced Solid Tumors That Have a Genetic Mutation.

NCT06447662
View directory record Official record
Conditions
Carcinoma, Pancreatic Ductal, Colorectal Neoplasms, Carcinoma, Non-Small-Cell Lung
Interventions
PF-07934040, Gemcitabine, Nab-paclitaxel, Cetuximab, Fluorouracil, Oxaliplatin, Leucovorin, Bevacizumab, Pembrolizumab, pemetrexed, Cisplatin, Paclitaxel, Carboplatin, Sasanlimab
Drug · Combination Product
Lead sponsor
Pfizer
Industry
Eligibility
18 Years and older
Enrollment
64 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
26
States / cities
Fayetteville, Arkansas • Rogers, Arkansas • Springdale, Arkansas + 14 more
Source: ClinicalTrials.gov public record
Updated Jul 21, 2026 · Synced Sep 6, 2026, 9:36 PM EDT
Completed Phase 2Phase 3 Interventional Accepts healthy volunteers Results available

Dominantly Inherited Alzheimer Network Trial: An Opportunity to Prevent Dementia. A Study of Potential Disease Modifying Treatments in Individuals at Risk for or With a Type of Early Onset Alzheimer's Disease Caused by a Genetic Mutation.

NCT04623242
View directory record Official record
Conditions
Alzheimers Disease, Dementia, Alzheimers Disease, Familial
Interventions
Gantenerumab, Solanezumab, Matching Placebo (Gantenerumab), Matching Placebo (Solanezumab)
Drug
Lead sponsor
Washington University School of Medicine
Other
Eligibility
18 Years to 80 Years
Enrollment
194 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2020
U.S. locations
10
States / cities
Birmingham, Alabama • La Jolla, California • New Haven, Connecticut + 7 more
Source: ClinicalTrials.gov public record
Updated Sep 21, 2022 · Synced Sep 6, 2026, 9:36 PM EDT
Completed Phase 1 Interventional

A Safety Study of SGN-LIV1A in Breast Cancer Patients

NCT01969643
View directory record Official record
Conditions
HER2 Positive Breast Neoplasms, Hormone Receptor Positive Breast Neoplasms, Triple Negative Breast Neoplasms, HER2 Mutations Breast Neoplasms
Interventions
ladiratuzumab vedotin, Trastuzumab
Drug
Lead sponsor
Seagen Inc.
Industry
Eligibility
18 Years and older · Female only
Enrollment
290 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2023
U.S. locations
38
States / cities
Birmingham, Alabama • Scottsdale, Arizona • La Jolla, California + 30 more
Source: ClinicalTrials.gov public record
Updated Mar 6, 2023 · Synced Sep 6, 2026, 9:36 PM 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 6, 2026, 9:36 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional Results available

A Study to Assess the Safety and Efficacy of Two Combinations of Isocitrate Dehydrogenase (IDH) Mutant Targeted Therapies Plus Azacitidine in Participants With Newly Diagnosed Acute Myeloid Leukemia (AML) Harboring IDH Mutations Who Are Not Candidates to Receive Intensive Induction Chemotherapy

NCT02677922
View directory record Official record
Conditions
Leukemia, Myeloid, Acute
Interventions
AG-120, Azacitidine, AG-221
Drug
Lead sponsor
Celgene
Industry
Eligibility
18 Years and older
Enrollment
130 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2026
U.S. locations
9
States / cities
Duarte, California • New Haven, Connecticut • Chicago, Illinois + 3 more
Source: ClinicalTrials.gov public record
Updated Feb 18, 2026 · Synced Sep 6, 2026, 9:36 PM EDT
Completed No phase listed Observational Results available

Evaluating the Role of Immune Responses in the Emergence of Protease Inhibitor Mutations

NCT01517529
View directory record Official record
Conditions
Hepatitis C
Interventions
Not listed
Lead sponsor
University of Cincinnati
Other
Eligibility
18 Years to 65 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2014
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Nov 19, 2015 · Synced Sep 6, 2026, 9:36 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Fabry Screening Study

NCT01019629
View directory record Official record
Conditions
Fabry Disease
Interventions
Not listed
Lead sponsor
Baylor Research Institute
Other
Eligibility
18 Years and older
Enrollment
2,724 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2017
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Feb 12, 2018 · Synced Sep 6, 2026, 9:36 PM EDT
Completed No phase listed Observational

Genetic Study in Patients Receiving Treatment for Hodgkin's Disease or Childhood Brain Tumor

NCT00006342
View directory record Official record
Conditions
Brain and Central Nervous System Tumors, Lymphoma
Interventions
chromosomal translocation analysis, gene rearrangement analysis, mutation analysis
Genetic
Lead sponsor
Duke University
Other
Eligibility
Not listed
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1997 – 2001
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Oct 14, 2015 · Synced Sep 6, 2026, 9:36 PM EDT
Recruiting Phase 1 Interventional

A Study of a Selective ERBB2 Inhibitor (CGT4255), in Patients With Advanced Solid Tumors

NCT07361562
View directory record Official record
Conditions
Advanced Solid Tumor, Adult, ERBB2 Altered Breast Cancer, ERBB2 Gene Amplification, HER2 Overexpression, Non-Small Cell Lung Cancer, HER2
Interventions
CGT4255
Drug
Lead sponsor
Cogent Biosciences, Inc.
Industry
Eligibility
18 Years and older
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
15
States / cities
Scottsdale, Arizona • New Haven, Connecticut • Jacksonville, Florida + 11 more
Source: ClinicalTrials.gov public record
Updated Sep 3, 2026 · Synced Sep 6, 2026, 9:36 PM EDT
Completed Phase 2 Interventional Results available

LEE011 for Patients With CDK4/6 Pathway Activated Tumors (SIGNATURE)

NCT02187783
View directory record Official record
Conditions
Tumors With CDK4/6 Pathway Activation
Interventions
LEE011
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
106 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
61
States / cities
Birmingham, Alabama • Anchorage, Alaska • Phoenix, Arizona + 53 more
Source: ClinicalTrials.gov public record
Updated Jul 17, 2019 · Synced Sep 6, 2026, 9:36 PM EDT
Completed Phase 2 Interventional Results available

Erlotinib Therapy and Subsequent Development of Mechanisms of Secondary Resistance in Patients With NSCLC

NCT00997334
View directory record Official record
Conditions
Non-small Cell Lung Cancer
Interventions
Erlotinib
Drug
Lead sponsor
David M. Jackman, MD
Other
Eligibility
18 Years and older
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2017
U.S. locations
2
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Feb 22, 2018 · Synced Sep 6, 2026, 9:36 PM EDT
No Longer Available No phase listed Expanded access

HBCMD01- Expanded Access for the Treatment of Congenital Muscular Dystrophy.

NCT05154851
View directory record Official record
Conditions
Congenital Muscular Dystrophy Due to Lamin A/C Mutation
Interventions
HB-adMSCs
Biological
Lead sponsor
Hope Biosciences Research Foundation
Industry
Eligibility
Not listed
U.S. locations
1
States / cities
Sugar Land, Texas
Source: ClinicalTrials.gov public record
Updated Sep 28, 2025 · Synced Sep 6, 2026, 9:36 PM EDT
Completed Phase 3 Interventional Results available

Study of Ivacaftor in Cystic Fibrosis Subjects 2 Through 5 Years of Age With a CFTR Gating Mutation

NCT01705145
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Ivacaftor
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
2 Years to 5 Years
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
16
States / cities
Birmingham, Alabama • Aurora, Colorado • Atlanta, Georgia + 13 more
Source: ClinicalTrials.gov public record
Updated Apr 4, 2016 · Synced Sep 6, 2026, 9:36 PM EDT
Completed Phase 2 Interventional Results available

A Study to Evaluate the Safety and Efficacy of VX-371 in Subjects With Cystic Fibrosis Who Are Homozygous for the F508del-CFTR Mutation

NCT02709109
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
VX-371 + HS, Hypertonic saline, Placebo, Orkambi, VX-371
Drug
Lead sponsor
Parion Sciences
Industry
Eligibility
12 Years and older
Enrollment
147 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
30
States / cities
Little Rock, Arkansas • Oakland, California • Gainesville, Florida + 27 more
Source: ClinicalTrials.gov public record
Updated Jul 28, 2021 · Synced Sep 6, 2026, 9:36 PM EDT
Completed Phase 2 Interventional

Cyclosporine A to Treat Hypertrophic Cardiomyopathy (HCM)

NCT00001965
View directory record Official record
Conditions
Cardiomyopathy, Hypertrophic, Heart Hypertrophy
Interventions
Cyclosporine A
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
Not listed
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2001
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced Sep 6, 2026, 9:36 PM EDT