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ClinicalTrials.gov public records Last synced Oct 30, 2026, 1:14 AM EDT

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Showing 121–144 of 941 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Genetic Mutations Clear all
Recruiting Phase 1Phase 2 Interventional

Evaluate REC-4881 in Participants With Familial Adenomatous Polyposis (FAP)

NCT05552755
View directory record Official record
Conditions
Familial Adenomatous Polyposis
Interventions
REC-4881, Placebo
Drug
Lead sponsor
Recursion Pharmaceuticals Inc.
Industry
Eligibility
18 Years and older
Enrollment
67 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
17
States / cities
Scottsdale, Arizona • Tucson, Arizona • San Diego, California + 14 more
Source: ClinicalTrials.gov public record
Updated Jun 5, 2026 · Synced Oct 30, 2026, 1:14 AM EDT
Completed Phase 2 Interventional Results available

Reduced-Intensity Conditioning Before Donor Stem Cell Transplant in With High-Risk Hematologic Malignancies

NCT01760655
View directory record Official record
Conditions
Acute Myeloid Leukemia With FLT3/ITD Mutation, Acute Myeloid Leukemia With Gene Mutations, Acute Myeloid Leukemia With Inv(3) (q21.3;q26.2); GATA2, MECOM, Acute Myeloid Leukemia With t(6;9) (p23;q34.1); DEK-NUP214, Aplastic Anemia, Chronic Lymphocytic Leukemia, Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Chronic Myelomonocytic Leukemia, Follicular Lymphoma, Hodgkin Lymphoma, Myelodysplastic Syndrome, Myelofibrosis, Myeloid Leukemia, Non-Hodgkin Lymphoma, Plasma Cell Myeloma, Polycythemia Vera, Recurrent Adult Acute Myeloid Leukemia, Recurrent Childhood Acute Myeloid Leukemia, Refractory Acute Myeloid Leukemia, Secondary Acute Myeloid Leukemia, Therapy-Related Acute Myeloid Leukemia
Interventions
Fludarabine phosphate, Busulfan, Total-Body Irradiation, Therapeutic Allogeneic Lymphocytes, Cyclophosphamide, Allogeneic Hematopoietic Stem Cell Transplantation, Peripheral Blood Stem Cell Transplantation, Tacrolimus, Mycophenolate Mofetil, Laboratory Biomarker Analysis
Drug · Radiation · Biological + 2 more
Lead sponsor
Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University
Other
Eligibility
Not listed
Enrollment
62 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2022
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 30, 2025 · Synced Oct 30, 2026, 1:14 AM EDT
Not listed Phase 1Phase 2 Interventional Results available

Liposomal Irinotecan, Fluorouracil, Leucovorin Calcium, and Rucaparib in Treating Patients With Metastatic Pancreatic, Colorectal, Gastroesophageal, or Biliary Cancer

NCT03337087
View directory record Official record
Conditions
Metastatic Biliary Tract Carcinoma, Metastatic Colorectal Carcinoma, Metastatic Gastroesophageal Junction Adenocarcinoma, Metastatic Malignant Digestive System Neoplasm, Metastatic Pancreatic Adenocarcinoma, Stage IV Colorectal Cancer AJCC v7, Stage IV Pancreatic Cancer AJCC v6 and v7, Stage IVA Colorectal Cancer AJCC v7, Stage IVB Colorectal Cancer AJCC v7
Interventions
Fluorouracil, Irinotecan Sucrosofate, Laboratory Biomarker Analysis, Leucovorin Calcium, Rucaparib
Drug · Other
Lead sponsor
Academic and Community Cancer Research United
Other
Eligibility
18 Years and older
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2025
U.S. locations
3
States / cities
Scottsdale, Arizona • Atlanta, Georgia • Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated May 17, 2024 · Synced Oct 30, 2026, 1:14 AM EDT
Recruiting No phase listed Observational

Role of Diet on the Microbiome of the Digestive System

NCT04666727
View directory record Official record
Conditions
Colorectal Carcinoma
Interventions
Not listed
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
45 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2027
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated May 26, 2026 · Synced Oct 30, 2026, 1:14 AM EDT
Not listed Phase 1Phase 2 Interventional

Optimizing IV Gentamicin in JEB

NCT04140786
View directory record Official record
Conditions
Junctional Epidermolysis Bullosa
Interventions
Gentamicin Sulfate, Injectable
Drug
Lead sponsor
University of Southern California
Other
Eligibility
30 Days and older
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Nov 3, 2022 · Synced Oct 30, 2026, 1:14 AM EDT
Completed Phase 1 Interventional

Phase I Dose-Escalation Study of AZD4785 in Patients With Advanced Solid Tumours

NCT03101839
View directory record Official record
Conditions
Non-Small Cell Lung Cancer, Advanced Solid Tumours
Interventions
AZD4785
Drug
Lead sponsor
AstraZeneca
Industry
Eligibility
18 Years to 130 Years
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
3
States / cities
Sarasota, Florida • Boston, Massachusetts • Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Oct 4, 2019 · Synced Oct 30, 2026, 1:14 AM EDT
Active, not recruiting Phase 2Phase 3 Interventional

Dominantly Inherited Alzheimer Network Trial: An Opportunity to Prevent Dementia. A Study of Potential Disease Modifying Treatments in Individuals With a Type of Early Onset Alzheimer's Disease Caused by a Genetic Mutation (DIAN-TU)

NCT05269394
View directory record Official record
Conditions
Alzheimers Disease, Dementia, Alzheimers Disease, Familial
Interventions
E2814, Lecanemab, Matching Placebo (E2814)
Drug
Lead sponsor
Washington University School of Medicine
Other
Eligibility
18 Years to 80 Years
Enrollment
197 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2028
U.S. locations
12
States / cities
Birmingham, Alabama • La Jolla, California • Los Angeles, California + 9 more
Source: ClinicalTrials.gov public record
Updated Jul 27, 2026 · Synced Oct 30, 2026, 1:14 AM EDT
Completed Phase 2 Interventional Results available

Everolimus for Cancer With TSC1 or TSC2 Mutation

NCT02201212
View directory record Official record
Conditions
TSC1, TSC2, Tuberous Sclerosis Complex, MTOR
Interventions
Everolimus
Drug
Lead sponsor
Dana-Farber Cancer Institute
Other
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2019
U.S. locations
2
States / cities
Boston, Massachusetts • New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 16, 2020 · Synced Oct 30, 2026, 1:14 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Monitoring and Early Detection of Treatable Inherited RETinal Disease in Children and Adolescents

NCT07901907
View directory record Official record
Conditions
Inherited Retinal Degeneration Associated With RPGR Mutations, Inherited Retinal Degeneration Associated With Biallelic RPE65 Mutations
Interventions
Not listed
Lead sponsor
University College, London
Other
Eligibility
Up to 50 Years
Enrollment
4,887 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2026 – 2028
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Oct 28, 2026 · Synced Oct 30, 2026, 1:14 AM EDT
Completed Phase 1Phase 2 Interventional Results available

A Phase I/II, Multicenter, Open-label Study of EGFRmut-TKI EGF816, Administered Orally in Adult Patients With EGFRmut Solid Malignancies

NCT02108964
View directory record Official record
Conditions
Advanced Non-small Cell Lung Cancer
Interventions
EGF816
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
225 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2023
U.S. locations
2
States / cities
Boston, Massachusetts • New York, New York
Source: ClinicalTrials.gov public record
Updated Dec 11, 2024 · Synced Oct 30, 2026, 1:14 AM EDT
Completed Phase 2 Interventional Results available

Alvelestat (MPH966) for the Treatment of ALpha-1 ANTitrypsin Deficiency

NCT03679598
View directory record Official record
Conditions
Alpha-1 Antitrypsin Deficiency (AATD), Pi*ZZ, Pi*SZ, Pi*Null, Another Rare Phenotype/Genotype Known to be Associated With Either Low or Functionally Impaired AAT Including F or I Mutations, Emphysema or COPD
Interventions
Alvelestat (MPH966), Placebo
Drug · Other
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
18 Years to 80 Years
Enrollment
63 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
10
States / cities
Birmingham, Alabama • Los Angeles, California • Denver, Colorado + 7 more
Source: ClinicalTrials.gov public record
Updated Aug 20, 2024 · Synced Oct 30, 2026, 1:14 AM EDT
Completed Phase 2 Interventional Results available

Dovitinib for Patients With Tumor Pathway Activations Inhibited by Dovitinib

NCT01831726
View directory record Official record
Conditions
Tumor Pathway Activations Inhibited by Dovitinib
Interventions
Dovitinib (TKI258)
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
18 Years to 100 Years
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2015
U.S. locations
49
States / cities
Birmingham, Alabama • Fayetteville, Arkansas • Pismo Beach, California + 43 more
Source: ClinicalTrials.gov public record
Updated Mar 20, 2017 · Synced Oct 30, 2026, 1:14 AM EDT
Completed Phase 2 Interventional Results available

Copanlisib in Treating Patients With Persistent or Recurrent Endometrial Cancer

NCT02728258
View directory record Official record
Conditions
Endometrial Endometrioid Adenocarcinoma, Endometrial Mixed Cell Adenocarcinoma, Endometrial Serous Adenocarcinoma, Endometrial Undifferentiated Carcinoma, Metastatic Endometrioid Adenocarcinoma, Recurrent Uterine Corpus Cancer
Interventions
Copanlisib, Laboratory Biomarker Analysis
Drug · Other
Lead sponsor
NRG Oncology
Other
Eligibility
18 Years and older · Female only
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2020
U.S. locations
43
States / cities
New Haven, Connecticut • Deerfield Beach, Florida • Miami, Florida + 35 more
Source: ClinicalTrials.gov public record
Updated Feb 15, 2022 · Synced Oct 30, 2026, 1:14 AM EDT
Withdrawn Phase 2 Interventional

Enasidenib in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia With an IDH2 Gene Mutation

NCT03881735
View directory record Official record
Conditions
Blasts Under 5 Percent of Peripheral Blood White Cells, Bone Marrow Blasts Decreased by 50 Percent or More Compared to Pretreatment Level, IDH2 Gene Mutation, Recurrent Acute Myeloid Leukemia, Refractory Acute Myeloid Leukemia
Interventions
Enasidenib, Hematopoietic Cell Transplantation
Drug · Procedure
Lead sponsor
Roswell Park Cancer Institute
Other
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
2
States / cities
Buffalo, New York • East Hills, New York
Source: ClinicalTrials.gov public record
Updated Mar 10, 2021 · Synced Oct 30, 2026, 1:14 AM EDT
Completed Phase 1 Interventional

A Study of ZN-e4 in Subjects With Epidermal Growth Factor Receptor Mutated Non-Small Cell Lung Cancer

NCT03446417
View directory record Official record
Conditions
Carcinoma, Non-Small-Cell Lung
Interventions
ZN-e4
Drug
Lead sponsor
Zeno Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
5
States / cities
Duarte, California • Detroit, Michigan • East Setauket, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Jan 25, 2024 · Synced Oct 30, 2026, 1:14 AM EDT
Completed Phase 2 Interventional Accepts healthy volunteers Results available

Tau PET Imaging With 18F-AV-1451 in Subjects With MAPT Mutations

NCT02676843
View directory record Official record
Conditions
Frontotemporal Lobar Degeneration (FTLD), Frontotemporal Dementia (FTD), Tauopathies
Interventions
18F-AV-1451
Drug
Lead sponsor
Columbia University
Other
Eligibility
18 Years and older
Enrollment
7 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2018
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Dec 6, 2019 · Synced Oct 30, 2026, 1:14 AM EDT
Active, not recruiting Phase 2Phase 3 Interventional

A Study to Evaluate the Efficacy and Safety of Multiple Targeted Therapies as Treatments for Participants With Non-Small Cell Lung Cancer (NSCLC)

NCT03178552
View directory record Official record
Conditions
Non-Small Cell Lung Cancer
Interventions
Alectinib, Atezolizumab, Pemetrexed, Cisplatin, Carboplatin, Gemcitabine, Entrectinib, Cobimetinib, Vemurafenib, Bevacizumab, Divarasib, Docetaxel
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
18 Years and older
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2027
U.S. locations
12
States / cities
Sacramento, California • Denver, Colorado • Fort Myers, Florida + 9 more
Source: ClinicalTrials.gov public record
Updated Sep 3, 2026 · Synced Oct 30, 2026, 1:14 AM EDT
Completed Phase 2 Interventional Results available

Trikafta in Cystic Fibrosis Patients

NCT03506061
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Trikafta
Drug
Lead sponsor
Emory University
Other
Eligibility
12 Years and older
Enrollment
42 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2024
U.S. locations
2
States / cities
Birmingham, Alabama • Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Aug 11, 2026 · Synced Oct 30, 2026, 1:14 AM EDT
Not yet recruiting Phase 1Phase 2 Interventional

Study of Tegavivint Plus Zanzalintinib in Patients With Advanced Hepatocellular Carcinoma

NCT07878689
View directory record Official record
Conditions
Hepatocellular Carcinoma (HCC)
Interventions
Zanzalintinib, Tegavivint
Drug
Lead sponsor
University of Texas Southwestern Medical Center
Other
Eligibility
18 Years and older
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Oct 16, 2026 · Synced Oct 30, 2026, 1:14 AM EDT
Completed Phase 3 Interventional Results available

A Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy

NCT03525574
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
ELX/TEZ/IVA, IVA
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
12 Years and older
Enrollment
507 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
52
States / cities
Tucson, Arizona • Little Rock, Arkansas • Long Beach, California + 47 more
Source: ClinicalTrials.gov public record
Updated Sep 24, 2025 · Synced Oct 30, 2026, 1:14 AM EDT
Completed No phase listed Observational

Natural History Study of SLC25A46 Mutation-related Mitochondriopathy

NCT04594590
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
9 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 8, 2024 · Synced Oct 30, 2026, 1:14 AM EDT
Recruiting No phase listed Observational

Biospecimen Collection to Identify Gene Mutations for High Risk Pancreatic Cancer in Pediatric Patients, INSPPIRE 2 Study

NCT06651580
View directory record Official record
Conditions
Chronic Pancreatitis, Exocrine Pancreas Carcinoma, Recurrent Acute Pancreatitis
Interventions
Biospecimen Collection, Quality-of-Life Assessment, Questionnaire Administration
Procedure · Other
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
Up to 17 Years
Enrollment
1,600 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
22
States / cities
Los Angeles, California • Oakland, California • Palo Alto, California + 17 more
Source: ClinicalTrials.gov public record
Updated Sep 11, 2026 · Synced Oct 30, 2026, 1:14 AM EDT
Terminated Phase 2 Interventional Results available

Merestinib In Non-Small Cell Lung Cancer And Solid Tumors

NCT02920996
View directory record Official record
Conditions
Carcinoma, Non-Small-Cell Lung, Solid Tumor
Interventions
Merestinib
Drug
Lead sponsor
Dana-Farber Cancer Institute
Other
Eligibility
18 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2023
U.S. locations
2
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Apr 11, 2024 · Synced Oct 30, 2026, 1:14 AM EDT
Not listed Phase 2 Interventional

Study of Cavosonstat (N91115) in CF Patients Who Are Heterozygous for F508del-CFTR and a Gating Mutation and Being Treated With Ivacaftor

NCT02724527
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Cavosonstat, Placebo
Drug
Lead sponsor
Nivalis Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
12
States / cities
Denver, Colorado • Baltimore, Maryland • Boston, Massachusetts + 9 more
Source: ClinicalTrials.gov public record
Updated Nov 21, 2016 · Synced Oct 30, 2026, 1:14 AM EDT