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ClinicalTrials.gov public records Last synced May 21, 2026, 8:05 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 1–24 of 921 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Filters: Condition: Genetic Mutations Clear all
Completed Phase 2 Interventional Results available
View directory record Official record
Conditions
Acute Myeloid Leukemia With FLT3 Activating Mutations That Has Relapsed or Been Refractory After One or More Prior Therapies
Interventions
Crenolanib besylate
Drug
Lead sponsor
Arog Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
56 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2019
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Nov 29, 2023 · Synced May 21, 2026, 8:05 PM EDT
Terminated Phase 2 Interventional Results available
View directory record Official record
Conditions
Acute Myeloid Leukemia With FLT3/ITD Mutation, Acute Myeloid Leukemia With Gene Mutations, FLT3 Tyrosine Kinase Domain Point Mutation, Secondary Acute Myeloid Leukemia, Untreated Adult Acute Myeloid Leukemia
Interventions
Decitabine, Laboratory Biomarker Analysis, Midostaurin
Drug · Other
Lead sponsor
Mayo Clinic
Other
Eligibility
60 Years and older
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Aug 6, 2019 · Synced May 21, 2026, 8:05 PM EDT
Recruiting Phase 1 Interventional
View directory record Official record
Conditions
Hyper IgE Syndrome From STAT3 Mutation, Job s Syndrome, HIES, Lupus, Atopic Dermatitis
Interventions
baricitinib
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
12 Years to 120 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated May 19, 2026 · Synced May 21, 2026, 8:05 PM EDT
Terminated No phase listed Observational
View directory record Official record
Conditions
Partial Hearing Loss
Interventions
Not listed
Lead sponsor
National Institute on Deafness and Other Communication Disorders (NIDCD)
NIH
Eligibility
Not listed
Enrollment
404 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1997 – 2015
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 16, 2019 · Synced May 21, 2026, 8:05 PM EDT
Completed Phase 3 Interventional Results available
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
251 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
33
States / cities
Tucson, Arizona • Long Beach, California • Palo Alto, California + 28 more
Source: ClinicalTrials.gov public record
Updated Jan 15, 2024 · Synced May 21, 2026, 8:05 PM EDT
Terminated No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
Parkinson's Disease
Interventions
Not listed
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
18 Years to 80 Years
Enrollment
42 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2018
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 28, 2018 · Synced May 21, 2026, 8:05 PM EDT
Withdrawn No phase listed Observational Accepts healthy volunteers
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 16, 2014 · Synced May 21, 2026, 8:05 PM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Single Gene Disorders
Interventions
Preimplantation Genetic Diagnosis
Other
Lead sponsor
Natera, Inc.
Industry
Eligibility
Not listed
Enrollment
240 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
1
States / cities
Redwood City, California
Source: ClinicalTrials.gov public record
Updated Jul 15, 2013 · Synced May 21, 2026, 8:05 PM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Myotubular Myopathy
Interventions
Genetic Testing
Other
Lead sponsor
Cure CMD
Other
Eligibility
30 Days and older
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2017
U.S. locations
1
States / cities
Torrance, California
Source: ClinicalTrials.gov public record
Updated Mar 6, 2018 · Synced May 21, 2026, 8:05 PM EDT
Active, not recruiting No phase listed Observational
View directory record Official record
Conditions
Wilson Disease
Interventions
Not listed
Lead sponsor
Prime Medicine, Inc.
Industry
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
7
States / cities
Los Angeles, California • Sacramento, California • New Haven, Connecticut + 4 more
Source: ClinicalTrials.gov public record
Updated May 11, 2026 · Synced May 21, 2026, 8:05 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
PIEZO2-Deficiency Syndrome
Interventions
Not listed
Lead sponsor
National Center for Complementary and Integrative Health (NCCIH)
NIH
Eligibility
18 Years to 100 Years
Enrollment
16 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2026
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated May 19, 2026 · Synced May 21, 2026, 8:05 PM EDT
Completed No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
Extraocular Retinoblastoma, Intraocular Retinoblastoma, Recurrent Retinoblastoma
Interventions
Laboratory Biomarker Analysis, Questionnaire Administration
Other
Lead sponsor
Children's Oncology Group
Network
Eligibility
Not listed
Enrollment
234 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 2008
U.S. locations
49
States / cities
Los Angeles, California • Madera, California • San Francisco, California + 38 more
Source: ClinicalTrials.gov public record
Updated Oct 7, 2019 · Synced May 21, 2026, 8:05 PM EDT
Active, not recruiting Phase 2 Interventional
View directory record Official record
Conditions
X-Linked Retinitis Pigmentosa
Interventions
rAAV2tYF-GRK1-RPGR
Biological
Lead sponsor
Beacon Therapeutics
Industry
Eligibility
8 Years to 50 Years · Male only
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
6
States / cities
Jacksonville, Florida • Boston, Massachusetts • Cincinnati, Ohio + 3 more
Source: ClinicalTrials.gov public record
Updated Aug 11, 2024 · Synced May 21, 2026, 8:05 PM EDT
Completed No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
Stomach Neoplasms, Stomach Cancer, Gastric Adenocarcinoma and Proximal Polyposis of the Stomach (GAPPS), Hereditary Diffuse Gastric Cancer (HDGC), Familial Diffuse Gastric Cancer
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
2 Years and older
Enrollment
733 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2025
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Apr 30, 2026 · Synced May 21, 2026, 8:05 PM EDT
Active, not recruiting No phase listed Observational
View directory record Official record
Conditions
Methylmalonic Acidemia
Interventions
hLB-001
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2037
U.S. locations
4
States / cities
Atlanta, Georgia • Pittsburgh, Pennsylvania • Nashville, Tennessee + 1 more
Source: ClinicalTrials.gov public record
Updated Feb 4, 2026 · Synced May 21, 2026, 8:05 PM EDT
Terminated Phase 1Phase 2 Interventional Results available
View directory record Official record
Conditions
Non-Small Cell Lung Cancer
Interventions
Sotorasib and Tarloxotinib
Drug
Lead sponsor
Medical University of South Carolina
Other
Eligibility
18 Years and older
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2023
U.S. locations
1
States / cities
Charleston, South Carolina
Source: ClinicalTrials.gov public record
Updated Dec 15, 2025 · Synced May 21, 2026, 8:05 PM EDT
Terminated Phase 1 Interventional
View directory record Official record
Conditions
Solid Tumor, Tumors of Any Histology With Activating Trk (NTRK) Point or NTRK Fusion Mutations, Tenosynovial Giant Cell Tumor
Interventions
PLX7486 TsOH
Drug
Lead sponsor
Plexxikon
Industry
Eligibility
18 Years and older
Enrollment
59 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2018
U.S. locations
4
States / cities
Los Angeles, California • Baltimore, Maryland • Boston, Massachusetts + 1 more
Source: ClinicalTrials.gov public record
Updated Aug 1, 2018 · Synced May 21, 2026, 8:05 PM EDT
Recruiting No phase listed Observational
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 25, 2026 · Synced May 21, 2026, 8:05 PM EDT
Terminated No phase listed Observational
View directory record Official record
Conditions
Hormone Receptor-Positive, HER2-Negative Metastatic Breast Cancer, Metastatic Cervical Cancer
Interventions
Almac HER-Seq Assay
Diagnostic Test
Lead sponsor
Puma Biotechnology, Inc.
Industry
Eligibility
18 Years and older
Enrollment
1,583 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated May 18, 2021 · Synced May 21, 2026, 8:05 PM EDT
Active, not recruiting Phase 1 Interventional
View directory record Official record
Conditions
Non Small Cell Lung Cancer, Colorectal Cancer, Pancreatic Cancer, Solid Tumor, Cancer, Cancer of Pancreas, Cancer of Colon
Interventions
HBI-2376
Drug
Lead sponsor
HUYABIO International, LLC.
Industry
Eligibility
18 Years and older
Enrollment
42 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2026
U.S. locations
11
States / cities
Duarte, California • Encinitas, California • Fullerton, California + 7 more
Source: ClinicalTrials.gov public record
Updated Sep 23, 2025 · Synced May 21, 2026, 8:05 PM EDT
Completed Phase 2 Interventional Results available
View directory record Official record
Conditions
NF1, Neurofibromatosis, Plexiform Neurofibromas
Interventions
Cabozantinib
Drug
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
3 Years and older
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2022
U.S. locations
12
States / cities
Los Angeles, California • Washington D.C., District of Columbia • Chicago, Illinois + 8 more
Source: ClinicalTrials.gov public record
Updated May 23, 2023 · Synced May 21, 2026, 8:05 PM EDT
Completed Phase 1Phase 2 Interventional Results available
View directory record Official record
Conditions
Cancer
Interventions
Trametinib, Dabrafenib
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
1 Month to 17 Years
Enrollment
139 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2020
U.S. locations
10
States / cities
Phoenix, Arizona • San Francisco, California • Washington D.C., District of Columbia + 7 more
Source: ClinicalTrials.gov public record
Updated Jul 13, 2021 · Synced May 21, 2026, 8:05 PM EDT
Withdrawn Phase 2Phase 3 Interventional
View directory record Official record
Conditions
Retinal Disorder
Interventions
Placebos, ZA Low dose, ZA high dose
Drug
Lead sponsor
Retinagenix Holdings
Industry
Eligibility
6 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Feb 23, 2023 · Synced May 21, 2026, 8:05 PM EDT
Active, not recruiting Phase 2 Interventional
View directory record Official record
Conditions
Tumor, Tumor, Solid, Metastasis, Metastatic Cancer, Cancer, Cancer Metastatic, Tumors, Neoplasms, Neoplasm Metastasis, Solid Tumor, Advanced Solid Tumor, Advanced Cancer, Malignant Solid Tumor, Malignant Solid Neoplasm, Malignant Neoplasm, Malignant Tumor, TSC, TSC1, TSC2, Metastatic Solid Tumor, Metastatic Neoplasm
Interventions
nab-sirolimus
Drug
Lead sponsor
Aadi Bioscience, Inc.
Industry
Eligibility
12 Years and older
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
159
States / cities
Birmingham, Alabama • Mobile, Alabama • Goodyear, Arizona + 137 more
Source: ClinicalTrials.gov public record
Updated Jun 2, 2024 · Synced May 21, 2026, 8:05 PM EDT