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ClinicalTrials.gov public records Last synced Oct 30, 2026, 4:54 AM EDT

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Showing 145–168 of 941 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Genetic Mutations Clear all
Active, not recruiting Phase 3 Interventional

DIAN-TU Amyloid Removal Trial (ART) in Dominantly Inherited Alzheimer's Disease

NCT06384573
View directory record Official record
Conditions
Alzheimer's Disease, Dementia, Alzheimer's Disease, Familial
Interventions
lecanemab
Drug
Lead sponsor
Washington University School of Medicine
Other
Eligibility
18 Years and older
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2030
U.S. locations
4
States / cities
Birmingham, Alabama • Indianapolis, Indiana • St Louis, Missouri + 1 more
Source: ClinicalTrials.gov public record
Updated Jul 27, 2026 · Synced Oct 30, 2026, 4:54 AM EDT
Terminated Phase 2 Interventional Results available

A Study to Evaluate Rucaparib in Participants With Solid Tumors and With Deleterious Mutations in HRR Genes

NCT04171700
View directory record Official record
Conditions
Solid Tumor
Interventions
Rucaparib
Drug
Lead sponsor
pharmaand GmbH
Industry
Eligibility
18 Years and older
Enrollment
83 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
19
States / cities
Los Angeles, California • San Francisco, California • Fort Myers, Florida + 15 more
Source: ClinicalTrials.gov public record
Updated Oct 2, 2023 · Synced Oct 30, 2026, 4:54 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

A Study of E6201 for the Treatment of Advanced Hematologic Malignancies With FLT3 and/or Ras Mutations

NCT02418000
View directory record Official record
Conditions
AML, MDS, CMML
Interventions
E6201
Drug
Lead sponsor
Spirita Oncology, LLC
Industry
Eligibility
18 Years and older
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
4
States / cities
Denver, Colorado • Tampa, Florida • Houston, Texas + 1 more
Source: ClinicalTrials.gov public record
Updated Mar 20, 2019 · Synced Oct 30, 2026, 4:54 AM EDT
Completed Phase 2 Interventional Results available

Pilot and Feasibility Study of Reduced-Intensity Hematopoietic Stem Cell Transplant for MonoMAC

NCT00923364
View directory record Official record
Conditions
MDS, Immunodeficiency, GATA2
Interventions
Cyclophosphamide (CTX, Cytoxan), Fludarabine(Fludara,Berlex Laboratories), Total Body Irradiation (TBI), Allogeneic Hematopoietic Stem Cell (HSC), Equine Anti-Thymocyte Globulin
Drug · Procedure
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
12 Years to 60 Years
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2017
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 12, 2020 · Synced Oct 30, 2026, 4:54 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Natural History Study of and Genetic Modifiers in Spinocerebellar Ataxias

NCT01060371
View directory record Official record
Conditions
Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia Type 2, Spinocerebellar Ataxia Type 3, Spinocerebellar Ataxia Type 6, Spinocerebellar Ataxia Type 7, Spinocerebellar Ataxia Type 8, Spinocerebellar Ataxia Type 10, RFC1 Gene Mutation, Spinocerebellar Ataxia Type 27b, Healthy Participants
Interventions
Genetic Testing, Blood Collection, Magnetic Resonance Imaging (MRI) Scan, Assessments and Questionnaires, Cerebrospinal Fluid Collection
Genetic · Other
Lead sponsor
Lauren Moore
Other
Eligibility
6 Years and older
Enrollment
1,400 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2010 – 2030
U.S. locations
16
States / cities
Los Angeles, California • San Francisco, California • Gainesville, Florida + 12 more
Source: ClinicalTrials.gov public record
Updated Aug 3, 2026 · Synced Oct 30, 2026, 4:54 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 13, 2018 · Synced Oct 30, 2026, 4:54 AM EDT
Active, not recruiting Phase 1 Interventional

Study of TPX-0022 in Patients With Advanced NSCLC, Gastric Cancer or Solid Tumors Harboring Genetic Alterations in MET

NCT03993873
View directory record Official record
Conditions
Advanced Solid Tumor, Metastatic Solid Tumors, MET Gene Alterations
Interventions
elzovantinib (TPX-0022)
Drug
Lead sponsor
Turning Point Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
95 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2027
U.S. locations
12
States / cities
La Jolla, California • Orange, California • Denver, Colorado + 8 more
Source: ClinicalTrials.gov public record
Updated Oct 1, 2025 · Synced Oct 30, 2026, 4:54 AM EDT
Recruiting No phase listed Observational

Studies of the Variable Phenotypic Presentations of Rapid-Onset Dystonia Parkinsonism and Other Movement Disorders

NCT00682513
View directory record Official record
Conditions
Dystonia, Parkinsonism
Interventions
Not listed
Lead sponsor
State University of New York at Buffalo
Other
Eligibility
Not listed
Enrollment
198 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2027
U.S. locations
2
States / cities
Miami, Florida • Buffalo, New York
Source: ClinicalTrials.gov public record
Updated Oct 28, 2026 · Synced Oct 30, 2026, 4:54 AM EDT
Recruiting No phase listed Observational

Effects of Anti-PD1 Adjuvant Checkpoint Blockade Immunotherapy on Atypical/Dysplastic Nevi

NCT06599619
View directory record Official record
Conditions
Melanoma
Interventions
Single agent, adjuvant anti-PD1 therapy
Drug
Lead sponsor
John Kirkwood
Other
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2029
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jan 26, 2026 · Synced Oct 30, 2026, 4:54 AM EDT
Active, not recruiting Phase 2 Interventional Results available

A Study of T-DXd for the Treatment of Solid Tumors Harboring HER2 Activating Mutations

NCT04639219
View directory record Official record
Conditions
Advanced Solid Tumors With HER2 Mutation,eg:Colorectal,Urothelial,Gastric, Hepatobiliary,Endometrial,Melanoma,Ovarian,Cervical,Salivary Gland,Pancreatic,Breast
Interventions
Trastuzumab deruxtecan
Drug
Lead sponsor
AstraZeneca
Industry
Eligibility
18 Years to 120 Years
Enrollment
102 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
10
States / cities
Santa Rosa, California • Muncie, Indiana • Boston, Massachusetts + 6 more
Source: ClinicalTrials.gov public record
Updated Mar 23, 2026 · Synced Oct 30, 2026, 4:54 AM EDT
Completed Phase 2 Interventional Results available

Vemurafenib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With BRAF V600 Mutations (A Pediatric MATCH Treatment Trial)

NCT03220035
View directory record Official record
Conditions
Advanced Malignant Solid Neoplasm, Ann Arbor Stage III Childhood Non-Hodgkin Lymphoma, Ann Arbor Stage IV Childhood Non-Hodgkin Lymphoma, Ependymoma, Ewing Sarcoma, Hepatoblastoma, Langerhans Cell Histiocytosis, Malignant Germ Cell Tumor, Malignant Glioma, Osteosarcoma, Peripheral Primitive Neuroectodermal Tumor, Recurrent Childhood Central Nervous System Neoplasm, Recurrent Childhood Non-Hodgkin Lymphoma, Recurrent Malignant Solid Neoplasm, Recurrent Neuroblastoma, Refractory Malignant Solid Neoplasm, Refractory Neuroblastoma, Refractory Non-Hodgkin Lymphoma, Refractory Primary Central Nervous System Neoplasm, Rhabdoid Tumor, Rhabdomyosarcoma, Soft Tissue Sarcoma, Wilms Tumor
Interventions
Laboratory Biomarker Analysis, Vemurafenib
Other · Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
12 Months to 21 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2024
U.S. locations
114
States / cities
Birmingham, Alabama • Anchorage, Alaska • Mesa, Arizona + 90 more
Source: ClinicalTrials.gov public record
Updated Jan 27, 2025 · Synced Oct 30, 2026, 4:54 AM EDT
Active, not recruiting Phase 2 Interventional

Ph2 Open-label Study of AOC 1044 in Duchenne Muscular Dystrophy Participants With Mutations Amenable to Exon44 Skipping

NCT06244082
View directory record Official record
Conditions
DMD, Duchenne Muscular Dystrophy, Duchenne, Exon 44
Interventions
AOC 1044
Drug
Lead sponsor
Avidity Biosciences, Inc.
Industry
Eligibility
7 Years to 27 Years · Male only
Enrollment
39 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
10
States / cities
Little Rock, Arkansas • La Jolla, California • Sacramento, California + 7 more
Source: ClinicalTrials.gov public record
Updated Jul 30, 2026 · Synced Oct 30, 2026, 4:54 AM EDT
Recruiting Phase 1Phase 2 Interventional

FOG-001 in Locally Advanced or Metastatic Solid Tumors

NCT05919264
View directory record Official record
Conditions
Cancer, Colorectal Cancer, Solid Tumor, Locally Advanced Solid Tumor, Metastatic Cancer, WNT Pathway, HCC, Desmoid, Microsatellite Stable Colorectal Cancer, Metastatic Castration-resistant Prostate Cancer, Familial Adenomatous Polyposis (FAP), Endometrial Carcinoma, Prostate Cancer, Microsatellite Instability-High Colorectal Cancer, Adamantinomatous Craniopharyngioma
Interventions
FOG-001, mFOLFOX-6, Nivolumab, Trifluridine/tipiracil, Bevacizumab
Drug
Lead sponsor
Parabilis Medicines, Inc.
Industry
Eligibility
18 Years and older
Enrollment
619 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
31
States / cities
Phoenix, Arizona • Scottsdale, Arizona • Tucson, Arizona + 25 more
Source: ClinicalTrials.gov public record
Updated Oct 7, 2026 · Synced Oct 30, 2026, 4:54 AM EDT
Terminated Phase 2 Interventional Results available

Basket Study of Neratinib in Participants With Solid Tumors Harboring Somatic HER2 or EGFR Exon 18 Mutations

NCT01953926
View directory record Official record
Conditions
Solid Tumors Harboring Somatic HER2 or EGFR Exon 18 Mutations
Interventions
Neratinib, Fulvestrant, Trastuzumab, Paclitaxel
Drug
Lead sponsor
Puma Biotechnology, Inc.
Industry
Eligibility
18 Years and older
Enrollment
582 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2023
U.S. locations
30
States / cities
Birmingham, Alabama • Phoenix, Arizona • Duarte, California + 26 more
Source: ClinicalTrials.gov public record
Updated Mar 12, 2024 · Synced Oct 30, 2026, 4:54 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Phase 1/2 Study of Silevertinib (BDTX-1535) in Patients With Glioblastoma or Non-Small Cell Lung Cancer With EGFR Mutations

NCT05256290
View directory record Official record
Conditions
Non-Small Cell Lung Cancer, Advanced Non-Small Cell Squamous Lung Cancer, Metastatic Lung Non-Small Cell Carcinoma, Metastatic Lung Cancer, NSCLC, Advanced Lung Carcinoma, Epidermal Growth Factor Receptor C797S, Epidermal Growth Factor Receptor G719X, EGF-R Positive Non-Small Cell Lung Cancer, EGFR-TKI Resistant Mutation
Interventions
silevertinib (BDTX-1535) monotherapy
Drug
Lead sponsor
Black Diamond Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
25
States / cities
Birmingham, Alabama • Gilbert, Arizona • Huntington Beach, California + 20 more
Source: ClinicalTrials.gov public record
Updated Jul 22, 2026 · Synced Oct 30, 2026, 4:54 AM EDT
Active, not recruiting Phase 3 Interventional

A Study of Ripretinib vs Sunitinib in Patients With Advanced GIST With Specific KIT Exon Mutations Who Were Previously Treated With Imatinib

NCT05734105
View directory record Official record
Conditions
GIST
Interventions
Ripretinib, Sunitinib
Drug
Lead sponsor
Deciphera Pharmaceuticals, LLC
Industry
Eligibility
18 Years and older
Enrollment
54 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2028
U.S. locations
20
States / cities
La Jolla, California • New Haven, Connecticut • Jacksonville, Florida + 15 more
Source: ClinicalTrials.gov public record
Updated Dec 17, 2025 · Synced Oct 30, 2026, 4:54 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Vascular Disease Discovery Protocol

NCT03538639
View directory record Official record
Conditions
Vascular Dysfunction, Genetic Mutations, Genetic Predisposition
Interventions
Not listed
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
2 Years to 100 Years
Enrollment
1,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2037
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 26, 2026 · Synced Oct 30, 2026, 4:54 AM EDT
Active, not recruiting Phase 1 Interventional

A Study of IDH305 in Patients With Advanced Malignancies That Harbor IDH1R132 Mutations

NCT02381886
View directory record Official record
Conditions
Advanced Malignancies That Harbor IDHR132 Mutations
Interventions
IDH305
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
18 Years to 100 Years
Enrollment
166 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2030
U.S. locations
5
States / cities
Boston, Massachusetts • New York, New York • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Apr 13, 2026 · Synced Oct 30, 2026, 4:54 AM EDT
Terminated Phase 4 Interventional Results available

A Pilot Study to Evaluate the Use of Smart Adherence Technology to Measure Lumacaftor/Ivacaftor Adherence in CF Subjects Homozygous for the F508del CFTR Mutation

NCT02823470
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
LUM/IVA, activated smart device, de-activated smart device
Drug · Device
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
16 Years and older
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
7
States / cities
Hartford, Connecticut • Washington D.C., District of Columbia • Orlando, Florida + 4 more
Source: ClinicalTrials.gov public record
Updated Sep 27, 2018 · Synced Oct 30, 2026, 4:54 AM EDT
Completed Phase 2 Interventional Results available

Study of VX-809 in Cystic Fibrosis Subjects With the ∆F508-CFTR Gene Mutation

NCT00865904
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
VX-809, Placebo
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
18 Years and older
Enrollment
93 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009
U.S. locations
18
States / cities
Birmingham, Alabama • Palo Alto, California • San Diego, California + 15 more
Source: ClinicalTrials.gov public record
Updated Aug 28, 2015 · Synced Oct 30, 2026, 4:54 AM EDT
Withdrawn No phase listed Observational Accepts healthy volunteers

Genes Mutation Pentalogy of Cantrell

NCT00477932
View directory record Official record
Conditions
Pentalogy of Cantrell, Mutations in Non-muscle Genes
Interventions
Not listed
Lead sponsor
Children's Healthcare of Atlanta
Other
Eligibility
2 Years and older
Healthy volunteers
Accepts healthy volunteers
Timeline
2006 – 2012
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Dec 17, 2014 · Synced Oct 30, 2026, 4:54 AM EDT
Terminated Phase 2 Interventional Results available

Cabozantinib in Patients With Metastatic Castrate Resistant Prostate Cancer (mCRPC)

NCT04631744
View directory record Official record
Conditions
Prostate Cancer
Interventions
Cabozantinib
Drug
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
18 Years to 99 Years · Male only
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated May 1, 2026 · Synced Oct 30, 2026, 4:54 AM EDT
Completed Phase 1 Interventional Results available

(NAVIGATOR) Study of BLU-285 in Patients With Gastrointestinal Stromal Tumors (GIST) and Other Relapsed and Refractory Solid Tumors

NCT02508532
View directory record Official record
Conditions
Gastrointestinal Stromal Tumors (GIST), Other Relapsed or Refractory Solid Tumors
Interventions
Avapritinib
Drug
Lead sponsor
Blueprint Medicines Corporation
Industry
Eligibility
18 Years and older
Enrollment
250 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2021
U.S. locations
9
States / cities
Scottsdale, Arizona • Santa Monica, California • Miami, Florida + 6 more
Source: ClinicalTrials.gov public record
Updated Jun 21, 2022 · Synced Oct 30, 2026, 4:54 AM EDT
Recruiting Phase 1Phase 2 Interventional

Lurbinectedin With Osimertinib in Transformed Small Cell Lung Cancer

NCT07153055
View directory record Official record
Conditions
Small Cell Lung Cancer ( SCLC ), Transformed Small Cell Lung Cancer
Interventions
Lurbinectedin, Osimertinib
Drug
Lead sponsor
Misty Shields
Other
Eligibility
18 Years and older
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2027 – 2029
U.S. locations
1
States / cities
Indianapolis, Indiana
Source: ClinicalTrials.gov public record
Updated Oct 15, 2026 · Synced Oct 30, 2026, 4:54 AM EDT