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ClinicalTrials.gov public records Last synced Sep 7, 2026, 4:36 PM EDT

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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Mutations Clear all
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 7, 2026, 4:36 PM EDT
Completed Phase 1Phase 2 Interventional

Study of Avutometinib (VS-6766) + Adagrasib in KRAS G12C NSCLC Patients

NCT05375994
View directory record Official record
Conditions
Non Small Cell Lung Cancer, KRAS Activating Mutation, Advanced Cancer, Metastatic Cancer, Malignant Neoplasm of Lung, Malignant Neoplastic Disease
Interventions
avutometinib (VS-6766) and adagrasib
Drug
Lead sponsor
Verastem, Inc.
Industry
Eligibility
18 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
8
States / cities
San Francisco, California • Aurora, Colorado • Jacksonville, Florida + 5 more
Source: ClinicalTrials.gov public record
Updated Jul 1, 2026 · Synced Sep 7, 2026, 4:36 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 7, 2026, 4:36 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 7, 2026, 4:36 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 7, 2026, 4:36 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 7, 2026, 4:36 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 7, 2026, 4:36 PM EDT
Completed Phase 2 Interventional Results available

Adavosertib Plus Chemotherapy in Platinum-Resistant Epithelial Ovarian, Fallopian Tube, or Primary Peritoneal Cancer

NCT02272790
View directory record Official record
Conditions
Ovarian, Fallopian Tube, Peritoneal Cancer, P53 Mutation
Interventions
Adavosertib, Paclitaxel, Carboplatin, Gemcitabine, PLD
Drug
Lead sponsor
AstraZeneca
Industry
Eligibility
18 Years to 130 Years · Female only
Enrollment
95 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2023
U.S. locations
18
States / cities
Gilbert, Arizona • Tucson, Arizona • La Jolla, California + 14 more
Source: ClinicalTrials.gov public record
Updated Oct 2, 2023 · Synced Sep 7, 2026, 4:36 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Prevalence of a Non-Expressing 11B Mutation in Aka Peoples of the Central African Republic

NCT00340769
View directory record Official record
Conditions
Zinc Fingers Proteins
Interventions
Not listed
Lead sponsor
National Institute of Environmental Health Sciences (NIEHS)
NIH
Eligibility
Not listed
Healthy volunteers
Accepts healthy volunteers
Timeline
2001 – 2006
U.S. locations
2
States / cities
Research Triangle Park, North Carolina • Vancouver, Washington
Source: ClinicalTrials.gov public record
Updated Jul 1, 2017 · Synced Sep 7, 2026, 4:36 PM EDT
Completed Phase 2 Interventional Results available

A Study of Vemurafenib (RO5185426) in Participants With Metastatic or Unresectable Papillary Thyroid Cancer Positive for the BRAF V600 Mutation

NCT01286753
View directory record Official record
Conditions
Neoplasms
Interventions
Vemurafenib
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
18 Years and older
Enrollment
51 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2015
U.S. locations
9
States / cities
Torrance, California • New Haven, Connecticut • Chicago, Illinois + 5 more
Source: ClinicalTrials.gov public record
Updated Sep 6, 2016 · Synced Sep 7, 2026, 4:36 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 7, 2026, 4:36 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 7, 2026, 4:36 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 7, 2026, 4:36 PM EDT
Terminated Phase 4 Interventional

Testing the Safety and Efficacy of Commercial Drug Biktarvy in Subjects Known to Have the 184 Resistance Mutation to a Component in Biktarvy

NCT04040075
View directory record Official record
Conditions
HIV-1-infection
Interventions
Biktarvy Tab
Drug
Lead sponsor
Southampton Healthcare, Inc.
Other
Eligibility
18 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Jan 4, 2023 · Synced Sep 7, 2026, 4:36 PM EDT
Completed Phase 2 Interventional Results available

Ponatinib in Advanced NSCLC w/ RET Translocations

NCT01813734
View directory record Official record
Conditions
Non Small Cell Lung Cancer
Interventions
Ponatinib
Drug
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2018
U.S. locations
5
States / cities
Orange, California • Baltimore, Maryland • Boston, Massachusetts + 2 more
Source: ClinicalTrials.gov public record
Updated Dec 23, 2019 · Synced Sep 7, 2026, 4:36 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 7, 2026, 4:36 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 7, 2026, 4:36 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Personalized Antisense Oligonucleotide for A Single Participant With UBTF Gene Mutation

NCT07588581
View directory record Official record
Conditions
Childhood-Onset Neurodegeneration With Brain Atrophy (CONDBA)
Interventions
nL-UBTF-001
Drug
Lead sponsor
n-Lorem Foundation
Other
Eligibility
Not listed
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated May 14, 2026 · Synced Sep 7, 2026, 4:36 PM EDT
Recruiting No phase listed Observational

Preliminary Evaluation of Screening for Pancreatic Cancer in Patients With Inherited Genetic Risk

NCT02478892
View directory record Official record
Conditions
Pancreatic Cancer
Interventions
Collection of data from endoscopic ultrasound or MRI/MRCP of the abdomen, Blood sample collection for research
Other
Lead sponsor
Abramson Cancer Center at Penn Medicine
Other
Eligibility
18 Years and older
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2030
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Feb 4, 2026 · Synced Sep 7, 2026, 4:36 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Safety and Dose-escalation Study of AAV2-hCHM in Participants With CHM (Choroideremia) Gene Mutations

NCT02341807
View directory record Official record
Conditions
Choroideremia, CHM (Choroideremia) Gene Mutations
Interventions
AAV2-hCHM
Biological
Lead sponsor
Spark Therapeutics, Inc.
Industry
Eligibility
18 Years and older · Male only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2022
U.S. locations
3
States / cities
Boston, Massachusetts • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jan 24, 2024 · Synced Sep 7, 2026, 4:36 PM EDT
Completed No phase listed Observational

Clinical Evaluation of Patients With X-linked Retinitis Pigmentosa (XLRP)

NCT03314207
View directory record Official record
Conditions
X-Linked Retinitis Pigmentosa
Interventions
Not listed
Lead sponsor
Beacon Therapeutics
Industry
Eligibility
6 Years and older · Male only
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2022
U.S. locations
3
States / cities
Durham, North Carolina • Portland, Oregon • Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Jan 10, 2023 · Synced Sep 7, 2026, 4:36 PM EDT
Completed Phase 3 Interventional Results available

A Study to Evaluate the Efficacy and Safety of Maralixibat in Subjects With Progressive Familial Intrahepatic Cholestasis (MARCH-PFIC)

NCT03905330
View directory record Official record
Conditions
Progressive Familial Intrahepatic Cholestasis (PFIC)
Interventions
Maralixibat, Placebo
Drug · Other
Lead sponsor
Mirum Pharmaceuticals, Inc.
Industry
Eligibility
1 Year to 17 Years
Enrollment
93 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
11
States / cities
Los Angeles, California • Washington D.C., District of Columbia • Orlando, Florida + 8 more
Source: ClinicalTrials.gov public record
Updated Dec 10, 2023 · Synced Sep 7, 2026, 4:36 PM EDT
Recruiting Phase 1Phase 2 Interventional

A Beta-only IL-2 ImmunoTherapY Study

NCT05086692
View directory record Official record
Conditions
Advanced Solid Tumor, Unresectable Solid Tumor, Clear Cell Renal Cell Carcinoma, Triple Negative Breast Cancer, Non-Small Cell Lung Cancer Squamous, Non-Small Cell Lung Cancer Non-squamous, Colorectal Cancer (MSI-H), Gastric Cancer, Cervical Cancer, Basal Cell Carcinoma, Bladder Cancer, Merkel Cell Carcinoma, Squamous Cell Carcinoma of Head and Neck, Cutaneous Squamous Cell Carcinoma, Pleural Mesothelioma, Esophageal Cancer, Endometrial Carcinoma, Solid Tumor, Solid Tumor, Adult, MSI-H Solid Malignant Tumor, Cancer With A High Tumor Mutational Burden, Epithelial Ovarian Carcinoma, Primary Peritoneal Cancer, Gastroesophageal Junction (GEJ) Cancer, Acral Melanoma, Mucosal Melanoma, Cutaneous Melanoma, DMMR Solid Malignant Tumor, Fallopian Tube Cancer, Ovarian Cancer, MSI-H Cancer, DMMR Cancer, Pancreas Adenocarcinoma (MSI-H), Skin Cancer, Viral Cancer, Cervical Cancers, Endometrial Cancer
Interventions
MDNA11, Pembrolizumab (KEYTRUDA®)
Drug
Lead sponsor
Medicenna Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
115 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2026
U.S. locations
7
States / cities
San Diego, California • San Francisco, California • Santa Monica, California + 4 more
Source: ClinicalTrials.gov public record
Updated Jul 8, 2025 · Synced Sep 7, 2026, 4:36 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Quizartinib With Azacitidine or Cytarabine in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia or Myelodysplastic Syndrome

NCT01892371
View directory record Official record
Conditions
FLT3 Gene Mutation Negative, FLT3 Internal Tandem Duplication Positive, Recurrent Acute Myeloid Leukemia, Recurrent Chronic Myelomonocytic Leukemia, Recurrent Myelodysplastic Syndrome, Refractory Acute Myeloid Leukemia, Refractory Chronic Myelomonocytic Leukemia, Refractory Myelodysplastic Syndrome
Interventions
Azacitidine, Cytarabine, Quizartinib
Drug
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
18 Years and older
Enrollment
73 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2023
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Apr 5, 2025 · Synced Sep 7, 2026, 4:36 PM EDT