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ClinicalTrials.gov public records Last synced Oct 28, 2026, 11:39 PM EDT

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Showing 25–48 of 941 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Genetic Mutations Clear all
Recruiting No phase listed Observational

Subclinical Transthyretin Cardiac Amyloidosis in V122I TTR Carriers

NCT05489549
View directory record Official record
Conditions
Amyloidosis, Hereditary, Amyloidosis Cardiac, Amyloidosis, Familial, Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy, Transthyretin Gene Mutation
Interventions
Not listed
Lead sponsor
University of Texas Southwestern Medical Center
Other
Eligibility
30 Years to 80 Years
Enrollment
500 participants
Timeline
2022 – 2027
U.S. locations
3
States / cities
New York, New York • Cleveland, Ohio • Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Aug 31, 2026 · Synced Oct 28, 2026, 11:39 PM EDT
Active, not recruiting Phase 1 Interventional

ELVN-002 in HER2 Mutant Non-Small Cell Lung Cancer

NCT05650879
View directory record Official record
Conditions
HER2 Mutant Non-small Cell Lung Cancer, HER2-positive Metastatic Breast Cancer, HER2 Gene Mutation, HER2 Amplification
Interventions
ELVN-002, Fam-Trastuzumab Deruxtecan-Nxki, Trastuzumab emtansine
Drug
Lead sponsor
Enliven Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
198 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
5
States / cities
Aurora, Colorado • Orlando, Florida • Plantation, Florida + 2 more
Source: ClinicalTrials.gov public record
Updated Jul 15, 2025 · Synced Oct 28, 2026, 11:39 PM EDT
Completed Phase 1Phase 2 Interventional

NAUTILUS: OKI-179 Plus Binimetinib in Patients with Advanced Solid Tumors in the RAS Pathway (Phase 1b) and NRAS-mutated Melanoma (Phase 2)

NCT05340621
View directory record Official record
Conditions
RAS Mutation, NRAS Gene Mutation, Melanoma
Interventions
OKI-179 + binimetinib
Drug
Lead sponsor
OnKure, Inc.
Industry
Eligibility
18 Years and older
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
12
States / cities
Phoenix, Arizona • San Francisco, California • Gainesville, Florida + 9 more
Source: ClinicalTrials.gov public record
Updated Mar 7, 2025 · Synced Oct 28, 2026, 11:39 PM EDT
Completed Phase 1 Interventional

Safety/Proof of Concept Study of Oral QLT091001 in Subjects With Leber Congenital Amaurosis (LCA) or Retinitis Pigmentosa (RP) Due to Retinal Pigment Epithelial 65 Protein (RPE65) or Lecithin:Retinol Acyltransferase (LRAT) Mutations

NCT01014052
View directory record Official record
Conditions
LCA (Leber Congenital Amaurosis), RP (Retinitis Pigmentosa)
Interventions
QLT091001
Drug
Lead sponsor
QLT Inc.
Industry
Eligibility
5 Years to 65 Years
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2012
U.S. locations
3
States / cities
Chicago, Illinois • Baltimore, Maryland • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated May 14, 2013 · Synced Oct 28, 2026, 11:39 PM EDT
Terminated No phase listed Observational

Studying Common Genetic Mutations Related to Mucositis in Patients With Multiple Myeloma Receiving High-Dose Melphalan

NCT00602147
View directory record Official record
Conditions
Mucositis, Multiple Myeloma and Plasma Cell Neoplasm
Interventions
laboratory biomarker analysis
Other
Lead sponsor
Vanderbilt-Ingram Cancer Center
Other
Eligibility
18 Years and older
Enrollment
95 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2010
U.S. locations
3
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Mar 14, 2013 · Synced Oct 28, 2026, 11:39 PM 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 28, 2026, 11:39 PM EDT
Suspended No phase listed Observational

CNGB1 and Allied Disorders

NCT04639635
View directory record Official record
Conditions
Retinitis Pigmentosa Associated With CNGB1 Mutations
Interventions
No intervention, this is a natural history progression study
Other
Lead sponsor
Columbia University
Other
Eligibility
Not listed
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2027
U.S. locations
2
States / cities
New York, New York • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 13, 2026 · Synced Oct 28, 2026, 11:39 PM EDT
Suspended Phase 1Phase 2 Interventional

Dose Escalation/ Expansion Study of CA-4948 as Monotherapy in Patients With Acute Myelogenous Leukemia (AML) or Myelodysplastic Syndrome (MDS)

NCT04278768
View directory record Official record
Conditions
Acute Myelogenous Leukemia, Myelodysplastic Syndrome
Interventions
Emavusertib, Venetoclax
Drug
Lead sponsor
Curis, Inc.
Industry
Eligibility
18 Years and older
Enrollment
366 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
12
States / cities
Tampa, Florida • Atlanta, Georgia • Chicago, Illinois + 8 more
Source: ClinicalTrials.gov public record
Updated Mar 19, 2026 · Synced Oct 28, 2026, 11:39 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 29, 2025 · Synced Oct 28, 2026, 11:39 PM EDT
Recruiting Phase 1Phase 2 Interventional

A Study of BH-30643 in Subjects With Locally Advanced or Metastatic NSCLC Harboring EGFR and/or HER2 Mutations

NCT06706076
View directory record Official record
Conditions
NSCLC (Advanced Non-small Cell Lung Cancer)
Interventions
BH-30643, BH-30643 combo therapy with Carboplatin/Pemetrexed
Drug
Lead sponsor
BlossomHill Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
675 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2029
U.S. locations
24
States / cities
Phoenix, Arizona • Irvine, California • La Jolla, California + 18 more
Source: ClinicalTrials.gov public record
Updated Aug 5, 2026 · Synced Oct 28, 2026, 11:39 PM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Gene Mutations and Rescue in Human Congenital Diaphragmatic Hernia

NCT01098929
View directory record Official record
Conditions
Congenital Diaphragmatic Hernia
Interventions
Not listed
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
Not listed
Enrollment
1,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2002 – 2017
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Dec 17, 2015 · Synced Oct 28, 2026, 11:39 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

A Phase I/II Study of DYP688 in Patients With Metastatic Uveal Melanoma and Other GNAQ/11 Mutant Melanomas

NCT05415072
View directory record Official record
Conditions
Metastatic Uveal Melanoma
Interventions
DYP688
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
18 Years to 100 Years
Enrollment
66 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
3
States / cities
Boston, Massachusetts • New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 28, 2026 · Synced Oct 28, 2026, 11:39 PM EDT
Completed Phase 2 Interventional Results available

A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

NCT03648827
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Ataluren
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
2 Years to 7 Years · Male only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
9
States / cities
Phoenix, Arizona • Los Angeles, California • Sacramento, California + 6 more
Source: ClinicalTrials.gov public record
Updated Apr 5, 2022 · Synced Oct 28, 2026, 11:39 PM EDT
Completed No phase listed Observational

Prospective, Longitudinal Study of the Natural History and Functional Status of Patients With Myotubular Myopathy (MTM)

NCT02057705
View directory record Official record
Conditions
Myotubular Myopathy
Interventions
Not listed
Lead sponsor
Valerion Therapeutics, LLC
Industry
Eligibility
Not listed
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jun 7, 2018 · Synced Oct 28, 2026, 11:39 PM EDT
Withdrawn Early Phase 1 Interventional

Personalized Adaptive Radiation Therapy With Individualized Systemic Targeted Therapy (PARTIST) for Locally Advanced, Non-small Cell Lung Cancer With Genomic Driver Mutations

NCT02277457
View directory record Official record
Conditions
Non-Small Cell Lung Cancer
Interventions
PET-Adaptive RT, Erlotinib, Crizotinib
Radiation · Drug
Lead sponsor
University of Michigan Rogel Cancer Center
Other
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2015
U.S. locations
1
States / cities
Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Jun 24, 2016 · Synced Oct 28, 2026, 11:39 PM 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

NCT01807923
View directory record Official record
Conditions
Cystic Fibrosis, Homozygous for the F508del CFTR Mutation
Interventions
Lumacaftor Plus Ivacaftor Combination, Ivacaftor, Placebo
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
12 Years and older
Enrollment
559 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
48
States / cities
Birmingham, Alabama • Anchorage, Alaska • Tucson, Arizona + 45 more
Source: ClinicalTrials.gov public record
Updated Aug 31, 2015 · Synced Oct 28, 2026, 11:39 PM EDT
Completed Phase 2 Interventional Results available

A Study in Subjects With Retinitis Pigmentosa

NCT05392179
View directory record Official record
Conditions
Retinitis Pigmentosa
Interventions
ADX-2191
Drug
Lead sponsor
Aldeyra Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2023
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Apr 23, 2026 · Synced Oct 28, 2026, 11:39 PM EDT
Completed Phase 2 Interventional Results available

An Open-label Study of GSK1120212 Compared With Docetaxel in Stage IV KRAS-mutant Non-small Cell Lung Cancer

NCT01362296
View directory record Official record
Conditions
Lung Cancer, Non-Small Cell
Interventions
GSK1120212, docetaxel
Drug
Lead sponsor
GlaxoSmithKline
Industry
Eligibility
18 Years and older
Enrollment
134 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
25
States / cities
Scottsdale, Arizona • Orange, California • Aurora, Colorado + 20 more
Source: ClinicalTrials.gov public record
Updated Jul 11, 2014 · Synced Oct 28, 2026, 11:39 PM EDT
Recruiting Phase 1 Interventional

A Multinational Study Assessing an Oral EGFR Inhibitor, DZD6008 in Patients Who Have Advanced NSCLC With EGFR Mutations (TIAN-SHAN1)

NCT06905197
View directory record Official record
Conditions
Non Small Cell Lung Cancer
Interventions
DZD6008, Sunvozertinib
Drug
Lead sponsor
Dizal Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
140 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
3
States / cities
New York, New York • Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Oct 23, 2026 · Synced Oct 28, 2026, 11:39 PM EDT
Terminated Phase 2 Interventional Results available

Everolimus in Treating Patients With Advanced or Metastatic Colorectal Cancer That Did Not Respond to Previous Therapy

NCT00390364
View directory record Official record
Conditions
Colorectal Cancer
Interventions
everolimus, antiangiogenesis therapy, biopsy, diagnostic procedure, gene expression analysis, immunohistochemistry staining method, laboratory biomarker analysis, mutation analysis, protein tyrosine kinase inhibitor therapy, reverse transcriptase-polymerase chain reaction
Drug · Procedure
Lead sponsor
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Other
Eligibility
18 Years and older
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2007
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 8, 2014 · Synced Oct 28, 2026, 11:39 PM 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 28, 2026, 11:39 PM EDT
Recruiting Phase 2 Interventional

Testing Osimertinib as a Treatment for Lung Cancers With an EGFR Exon 20 Change

NCT03191149
View directory record Official record
Conditions
Advanced Lung Non-Small Cell Carcinoma, Recurrent Lung Non-Small Cell Carcinoma, Stage IIIB Lung Non-Small Cell Cancer AJCC v7, Stage IV Lung Non-Small Cell Cancer AJCC v7
Interventions
Biospecimen Collection, Computed Tomography with Contrast, Echocardiography Test, Magnetic Resonance Imaging, Multigated Acquisition Scan, Osimertinib
Procedure · Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
46 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2027
U.S. locations
347
States / cities
Anchorage, Alaska • Fairbanks, Alaska • Fort Smith, Arkansas + 238 more
Source: ClinicalTrials.gov public record
Updated Oct 28, 2026 · Synced Oct 28, 2026, 11:39 PM EDT
Terminated Phase 2 Interventional Results available

An Open-label, Randomized Phase II Study to Evaluate the Efficacy of AUY922 vs Pemetrexed or Docetaxel in NSCLC Patients With EGFR Mutations

NCT01646125
View directory record Official record
Conditions
Advanced Non Small Cell Lung Cancer (NSCLC)
Interventions
AUY922, Docetaxel, Pemetrexed
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
59 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2015
U.S. locations
3
States / cities
Los Angeles, California • Rockville, Maryland • Madison, Wisconsin
Source: ClinicalTrials.gov public record
Updated Jul 24, 2019 · Synced Oct 28, 2026, 11:39 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Azacitidine and Quizartinib for the Treatment of Myelodysplastic Syndrome or Myelodysplastic/Myeloproliferative Neoplasm With FLT3 or CBL Mutations

NCT04493138
View directory record Official record
Conditions
Chronic Myelomonocytic Leukemia, Myelodysplastic Syndrome, Myeloproliferative Neoplasm, Recurrent Chronic Myelomonocytic Leukemia, Recurrent Myelodysplastic Syndrome, Recurrent Myeloproliferative Neoplasm
Interventions
Azacitidine, Quizartinib
Drug
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
18 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 Oct 5, 2026 · Synced Oct 28, 2026, 11:39 PM EDT