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ClinicalTrials.gov public records Last synced Sep 19, 2026, 4:12 AM EDT

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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Genetic Change Clear all
Enrolling by invitation Phase 1Phase 2 Interventional

Personalized Antisense Oligonucleotide for A Single Participant With GARS1 Gene Mutation Associated With Charcot-Marie-Tooth Disease Type 2D (CMT2D)

NCT07226297
View directory record Official record
Conditions
Charcot-Marie-Tooth Disease Type 2D
Interventions
nL-GARS1-001
Drug
Lead sponsor
n-Lorem Foundation
Other
Eligibility
13 Years and older · Female only
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Nov 9, 2025 · Synced Sep 19, 2026, 4:12 AM EDT
Completed No phase listed Observational

A Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of ATTRv Amyloidosis With a V122I or T60A Mutation

NCT04201418
View directory record Official record
Conditions
Hereditary Transthyretin-mediated (ATTRv) Amyloidosis, Polyneuropathy
Interventions
Patisiran
Drug
Lead sponsor
Alnylam Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
67 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
27
States / cities
Los Angeles, California • San Diego, California • Aurora, Colorado + 22 more
Source: ClinicalTrials.gov public record
Updated Jun 5, 2022 · Synced Sep 19, 2026, 4:12 AM EDT
Recruiting Phase 1 Interventional

Open-label, Dose Escalation Study of Safety and Preliminary Efficacy of TN-401 in Adults With PKP2 Mutation-associated ARVC

NCT06228924
View directory record Official record
Conditions
Arrhythmogenic Right Ventricular Cardiomyopathy
Interventions
TN-401
Genetic
Lead sponsor
Tenaya Therapeutics
Industry
Eligibility
18 Years to 65 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2029
U.S. locations
7
States / cities
San Francisco, California • Aurora, Colorado • Baltimore, Maryland + 4 more
Source: ClinicalTrials.gov public record
Updated Feb 5, 2025 · Synced Sep 19, 2026, 4:12 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Genetic Analysis of Pheochromocytomas, Paragangliomas and Associated Conditions

NCT03160274
View directory record Official record
Conditions
Pheochromocytoma, Paraganglioma, Inherited Cancer Syndrome, Associated Conditions, Kidney Neoplasms, Bone Cancer, Thyroid Neoplasms, Other Cancer
Interventions
Genetic screening
Genetic
Lead sponsor
The University of Texas Health Science Center at San Antonio
Other
Eligibility
Not listed
Enrollment
2,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2005 – 2030
U.S. locations
1
States / cities
San Antonio, Texas
Source: ClinicalTrials.gov public record
Updated Oct 14, 2025 · Synced Sep 19, 2026, 4:12 AM EDT
Terminated Phase 1 Interventional

A Study Investigating BG-60366 in Adults With Epidermal Growth Factor Receptor (EGFR)-Mutant Non-Small Cell Lung Cancer

NCT06685718
View directory record Official record
Conditions
Non-Small Cell Lung Cancer, Lung Cancer, NSCLC, NSCLC (Non-small Cell Lung Carcinoma), EGFR Activating Mutation, EGFR Mutation-Related Tumors
Interventions
BG-60366
Drug
Lead sponsor
BeOne Medicines
Industry
Eligibility
18 Years and older
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
6
States / cities
Denver, Colorado • Boston, Massachusetts • St Louis, Missouri + 3 more
Source: ClinicalTrials.gov public record
Updated May 21, 2026 · Synced Sep 19, 2026, 4:12 AM EDT
Completed Phase 1 Interventional

A Study to Test How Well Different Doses of BI 3706674 Are Tolerated by People With Advanced Cancer in the Stomach and Oesophagus

NCT06056024
View directory record Official record
Conditions
Solid Tumor, KRAS Mutation
Interventions
BI 3706674
Drug
Lead sponsor
Boehringer Ingelheim
Industry
Eligibility
18 Years and older
Enrollment
47 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
6
States / cities
Phoenix, Arizona • New Haven, Connecticut • Boston, Massachusetts + 3 more
Source: ClinicalTrials.gov public record
Updated Mar 16, 2026 · Synced Sep 19, 2026, 4:12 AM EDT
Active, not recruiting Phase 3 Interventional Results available

AZD9291 Versus Placebo in Patients With Stage IB-IIIA Non-small Cell Lung Carcinoma, Following Complete Tumour Resection With or Without Adjuvant Chemotherapy.

NCT02511106
View directory record Official record
Conditions
Stage IB-IIIA Non-small Cell Lung Carcinoma
Interventions
AZD9291 80 mg/40 mg, Placebo AZD9291 80 mg/40 mg, Open-label AZD9291 80 mg/40 mg
Drug
Lead sponsor
AstraZeneca
Industry
Eligibility
18 Years to 130 Years
Enrollment
682 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2029
U.S. locations
23
States / cities
Los Angeles, California • Santa Monica, California • Santa Rosa, California + 20 more
Source: ClinicalTrials.gov public record
Updated Sep 10, 2026 · Synced Sep 19, 2026, 4:12 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

SX-682 and Nivolumab for the Treatment of RAS-Mutated, MSS Unresectable or Metastatic Colorectal Cancer, the STOPTRAFFIC-1 Trial

NCT04599140
View directory record Official record
Conditions
Metastatic Colon Adenocarcinoma, Metastatic Colorectal Carcinoma, Metastatic Rectal Adenocarcinoma, Stage III Colon Cancer AJCC v8, Stage III Rectal Cancer AJCC v8, Stage IIIA Colon Cancer AJCC v8, Stage IIIA Rectal Cancer AJCC v8, Stage IIIB Colon Cancer AJCC v8, Stage IIIB Rectal Cancer AJCC v8, Stage IIIC Colon Cancer AJCC v8, Stage IIIC Rectal Cancer AJCC v8, Stage IV Colon Cancer AJCC v8, Stage IV Rectal Cancer AJCC v8, Stage IVA Colon Cancer AJCC v8, Stage IVA Rectal Cancer AJCC v8, Stage IVB Colon Cancer AJCC v8, Stage IVB Rectal Cancer AJCC v8, Stage IVC Colon Cancer AJCC v8, Stage IVC Rectal Cancer AJCC v8, Unresectable Colon Adenocarcinoma, Unresectable Rectal Adenocarcinoma
Interventions
CXCR1/2 Inhibitor SX-682, Nivolumab
Drug · Biological
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
18 Years and older
Enrollment
51 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jul 19, 2026 · Synced Sep 19, 2026, 4:12 AM EDT
Recruiting Phase 2Phase 3 Interventional

A Study to Assess Adverse Events and Change in Disease Activity of Intravenous (IV) Telisotuzumab Adizutecan, Monotherapy or in Combination With Osimertinib, Compared to Standard of Care in Adult Participants With Locally Advanced or Metastatic EGFR-Mutated Non-Squamous Non-Small Cell Lung Cancer

NCT07155187
View directory record Official record
Conditions
Non-Small Cell Lung Cancer
Interventions
Telisotuzumab Adizutecan, Standard of Care, Osimertinib
Drug
Lead sponsor
AbbVie
Industry
Eligibility
18 Years and older
Enrollment
490 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
16
States / cities
Springdale, Arkansas • Duarte, California • Irvine, California + 13 more
Source: ClinicalTrials.gov public record
Updated Sep 14, 2026 · Synced Sep 19, 2026, 4:12 AM EDT
Terminated Phase 2 Interventional Results available

Bevacizumab and/or Niraparib in Patients With Recurrent Endometrial and/or Ovarian Cancer With ARID1A Mutation

NCT05523440
View directory record Official record
Conditions
Recurrent Endometrial Carcinoma, Recurrent Ovarian Carcinoma, ARID1A Gene Mutation
Interventions
Bevacizumab, Niraparib
Drug
Lead sponsor
University of Oklahoma
Other
Eligibility
18 Years and older · Female only
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
2
States / cities
Oklahoma City, Oklahoma • Charlottesville, Virginia
Source: ClinicalTrials.gov public record
Updated Jun 2, 2026 · Synced Sep 19, 2026, 4:12 AM EDT
Recruiting Phase 2 Interventional

A Study of ASTX727 in People With Malignant Peripheral Nerve Sheath Tumors (MPNST)

NCT04872543
View directory record Official record
Conditions
Malignant Peripheral Nerve Sheath Tumors (MPNST)
Interventions
ASTX727
Drug
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
16 Years and older
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
7
States / cities
Basking Ridge, New Jersey • Middletown, New Jersey • Montvale, New Jersey + 4 more
Source: ClinicalTrials.gov public record
Updated Feb 4, 2026 · Synced Sep 19, 2026, 4:12 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Study of Gene Modified Donor T-cells Following TCR Alpha Beta Positive Depleted Stem Cell Transplant

NCT03301168
View directory record Official record
Conditions
Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child, Lymphoma, Non-Hodgkin, Myelodysplastic Syndromes, Primary Immune Deficiency Disorder, Osteopetrosis, Cytopenia, Hemoglobinopathy in Children, Anemia, Aplastic
Interventions
BPX-501 T cells, Rimiducid
Biological · Drug
Lead sponsor
Bellicum Pharmaceuticals
Industry
Eligibility
1 Month to 26 Years
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2034
U.S. locations
10
States / cities
Los Angeles, California • Palo Alto, California • Washington D.C., District of Columbia + 7 more
Source: ClinicalTrials.gov public record
Updated Jul 11, 2022 · Synced Sep 19, 2026, 4:12 AM EDT
Completed Phase 2 Interventional Results available

Comparison of AZD6244 in Combination With Dacarbazine Versus (vs) Dacarbazine Alone in BRAF Mutation Positive Melanoma Patients

NCT00936221
View directory record Official record
Conditions
Melanoma
Interventions
AZD6244, Dacarbazine, Placebo
Drug
Lead sponsor
AstraZeneca
Industry
Eligibility
18 Years to 130 Years
Enrollment
385 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2014
U.S. locations
2
States / cities
Aurora, Colorado • Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 13, 2016 · Synced Sep 19, 2026, 4:12 AM EDT
Completed No phase listed Observational Results available

Safety of Pregnancy in BRCA Mutated Breast Cancer Patients

NCT03673306
View directory record Official record
Conditions
Breast Cancer
Interventions
Clinical outcomes
Other
Lead sponsor
Jules Bordet Institute
Other
Eligibility
18 Years to 40 Years · Female only
Enrollment
4,732 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2023
U.S. locations
7
States / cities
New Haven, Connecticut • Boston, Massachusetts • Lansing, Michigan + 4 more
Source: ClinicalTrials.gov public record
Updated Dec 12, 2024 · Synced Sep 19, 2026, 4:12 AM EDT
Completed Phase 1 Interventional

A Pharmacokinetics (PK) Study of the Effects Rabeprazole and Rifampin on Dabrafenib in Subjects With BRAF V600 Mutation Positive Tumors

NCT01954043
View directory record Official record
Conditions
Cancer
Interventions
Dabrafenib 150 mg twice a day (BID), Rabeprazole 40 mg once daily (OD), Rifampin 600 mg OD
Drug
Lead sponsor
GlaxoSmithKline
Industry
Eligibility
18 Years and older
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
3
States / cities
Goodyear, Arizona • Dallas, Texas • Tacoma, Washington
Source: ClinicalTrials.gov public record
Updated Nov 13, 2017 · Synced Sep 19, 2026, 4:12 AM EDT
Terminated Phase 2 Interventional Results available

Early Signs of Efficacy Study With Riociguat in Adult Homozygous Delta F508 Cystic Fibrosis Patients

NCT02170025
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Riociguat (Adempas, BAY63-2521), Placebo
Drug
Lead sponsor
Bayer
Industry
Eligibility
18 Years and older
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
3
States / cities
Birmingham, Alabama • Denver, Colorado • St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Nov 6, 2023 · Synced Sep 19, 2026, 4:12 AM EDT
Completed Phase 1 Interventional

Determination of the Absolute Bioavailability of GSK2118436 Following a Single Oral Dose Co-Administered With an Intravenous Radiolabelled Microtracer of GSK2118436 in Subjects With BRAF Mutant Solid Tumors

NCT01340833
View directory record Official record
Conditions
Cancer
Interventions
GSK2118436
Drug
Lead sponsor
GlaxoSmithKline
Industry
Eligibility
18 Years and older
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011
U.S. locations
1
States / cities
Tacoma, Washington
Source: ClinicalTrials.gov public record
Updated Nov 12, 2017 · Synced Sep 19, 2026, 4:12 AM EDT
Terminated Phase 2 Interventional Results available

Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD)

NCT00759876
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Ataluren
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
Male only
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2010
U.S. locations
3
States / cities
Cincinnati, Ohio • Philadelphia, Pennsylvania • Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Oct 28, 2020 · Synced Sep 19, 2026, 4:12 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Integrating the Genetic and Metabolic Faces of Obesity

NCT00285844
View directory record Official record
Conditions
Insulin Resistance, Obesity, Metabolic Syndrome
Interventions
weight loss, thiazolidinedione
Behavioral · Drug
Lead sponsor
Stanford University
Other
Eligibility
30 Years to 65 Years
Enrollment
88 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2005 – 2012
U.S. locations
1
States / cities
Stanford, California
Source: ClinicalTrials.gov public record
Updated Dec 3, 2024 · Synced Sep 19, 2026, 4:12 AM EDT
Recruiting Phase 3 Interventional

ROCKET-CLL Global Phase 3 Study: Rocbrutinib vs Pirtobrutinib in cBTKi-Pretreated R/R CLL/SLL

NCT07342478
View directory record Official record
Conditions
CLL / SLL, CLL (Chronic Lymphocytic Leukemia), CLL, Refractory, CLL, Relapsed, SLL (Small Lymphocytic Lymphoma), SLL, CLL, CLL Progression
Interventions
Rocbrutinib, Pirtobrutinib
Drug
Lead sponsor
Newave Pharmaceutical Inc
Industry
Eligibility
18 Years and older
Enrollment
306 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
6
States / cities
Skokie, Illinois • Bethesda, Maryland • Las Vegas, Nevada + 3 more
Source: ClinicalTrials.gov public record
Updated Aug 10, 2026 · Synced Sep 19, 2026, 4:12 AM EDT
Active, not recruiting Phase 3 Interventional Results available

Efficacy and Safety Comparison of Niraparib to Placebo in Participants With Human Epidermal Growth Factor 2 Negative (HER2-) Breast Cancer Susceptibility Gene Mutation (BRCAmut) or Triple-Negative Breast Cancer (TNBC) With Molecular Disease

NCT04915755
View directory record Official record
Conditions
Neoplasms, Breast
Interventions
Niraparib, Placebo
Drug
Lead sponsor
GlaxoSmithKline
Industry
Eligibility
18 Years and older
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
24
States / cities
Burbank, California • Duarte, California • Palo Alto, California + 20 more
Source: ClinicalTrials.gov public record
Updated Jul 15, 2025 · Synced Sep 19, 2026, 4:12 AM EDT
Recruiting Phase 1Phase 2 Interventional

First-In-Human Study of STX-721/PFL-721 in Participants With Locally Advanced or Metastatic Non-Small Cell Lung Cancer Harboring EGFR or HER2 Exon 20 Insertion Mutations

NCT06043817
View directory record Official record
Conditions
Non-Small Cell Lung Cancer, NSCLC, EGFR/HER2 Exon 20 Insertion Mutation
Interventions
STX-721/PFL-721 (Escalated), STX-721/PFL-721 (3 dose levels), STX-721/PFL-721 (RP2D)
Drug
Lead sponsor
Pierre Fabre Medicament
Industry
Eligibility
18 Years and older
Enrollment
345 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2029
U.S. locations
9
States / cities
Duarte, California • Huntington Beach, California • Irvine, California + 6 more
Source: ClinicalTrials.gov public record
Updated Sep 1, 2026 · Synced Sep 19, 2026, 4:12 AM EDT
Terminated Phase 2 Interventional Results available

The Effects of Treatment With Vemurafenib on the Immune System in Advanced Melanoma

NCT01942993
View directory record Official record
Conditions
Melanoma
Interventions
Vemurafenib
Drug
Lead sponsor
Philip Friedlander
Other
Eligibility
18 Years and older
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2015
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 29, 2020 · Synced Sep 19, 2026, 4:12 AM EDT
Recruiting No phase listed Observational

Use of Cysteamine in the Treatment of Cystinosis

NCT00359684
View directory record Official record
Conditions
Cystinosis
Interventions
Cysteamine
Drug
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
1 Week to 115 Years
Enrollment
330 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1979
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 15, 2026 · Synced Sep 19, 2026, 4:12 AM EDT