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ClinicalTrials.gov public records Last synced Oct 21, 2026, 10:42 PM EDT

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Showing 1–24 of 117 matching trials from the live ClinicalTrials.gov search.
Filters: Intervention: antisense oligonucleotide Clear all
Completed Phase 1 Interventional

Clinical Trial of BP1001 (L-Grb-2 Antisense Oligonucleotide) in CML, AML, ALL & MDS

NCT01159028
View directory record Official record
Conditions
Recurrent Adult Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, Myelodysplastic Syndrome, Ph1 Positive CML
Interventions
BP1001, BP1001 in combination with LDAC
Drug
Lead sponsor
Bio-Path Holdings, Inc.
Industry
Eligibility
18 Years to 70 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2017
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated May 28, 2020 · Synced Oct 21, 2026, 10:42 PM EDT
Completed Phase 2 Interventional

ISIS 104838, an Inhibitor of Tumor Necrosis Factor, for Active Rheumatoid Arthritis

NCT00048321
View directory record Official record
Conditions
Rheumatoid Arthritis
Interventions
ISIS 104838
Drug
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
160 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2003
U.S. locations
26
States / cities
Birmingham, Alabama • Paradise Valley, Arizona • Los Angeles, California + 23 more
Source: ClinicalTrials.gov public record
Updated Dec 5, 2022 · Synced Oct 21, 2026, 10:42 PM EDT
Completed Phase 2 Interventional

Oblimersen and Interferon Alfa in Treating Patients With Metastatic Renal Cell Cancer

NCT00059813
View directory record Official record
Conditions
Recurrent Renal Cell Cancer, Stage IV Renal Cell Cancer
Interventions
recombinant interferon alfa, oblimersen sodium, pharmacological study
Biological · Other
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
19 Years and older
Enrollment
41 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2003
U.S. locations
1
States / cities
Duarte, California
Source: ClinicalTrials.gov public record
Updated Aug 26, 2013 · Synced Oct 21, 2026, 10:42 PM EDT
Terminated Phase 1 Interventional

A Study of Aezea® (Cenersen) in Transfusion Dependent Anemia Associated With Myelodysplastic Syndrome (MDS)

NCT02243124
View directory record Official record
Conditions
Myelodysplastic Syndromes
Interventions
cenersen, Dexamethasone
Drug
Lead sponsor
Eleos, Inc.
Industry
Eligibility
18 Years to 95 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
1
States / cities
Tampa, Florida
Source: ClinicalTrials.gov public record
Updated Feb 21, 2021 · Synced Oct 21, 2026, 10:42 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53)

NCT04906460
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
WVE-N531
Drug
Lead sponsor
Wave Life Sciences USA, Inc.
Industry
Eligibility
4 Years to 18 Years · Male only
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
2
States / cities
Little Rock, Arkansas • Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Oct 19, 2026 · Synced Oct 21, 2026, 10:42 PM EDT
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 10, 2025 · Synced Oct 21, 2026, 10:42 PM EDT
Enrolling by invitation Phase 1Phase 2 Interventional

Personalized Antisense Oligonucleotide Therapy for a Participant With TARDBP ALS

NCT07703462
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis (ALS)
Interventions
nL-TARDB-002
Drug
Lead sponsor
n-Lorem Foundation
Other
Eligibility
Not listed
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 14, 2026 · Synced Oct 21, 2026, 10:42 PM EDT
Withdrawn Phase 2 Interventional

ION-682884 in Patients With TTR Amyloid Cardiomyopathy

NCT04843020
View directory record Official record
Conditions
Transthyretin Amyloid Cardiopathy
Interventions
ION 682884
Drug
Lead sponsor
Brigham and Women's Hospital
Other
Eligibility
65 Years to 85 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Feb 20, 2026 · Synced Oct 21, 2026, 10:42 PM EDT
Recruiting Phase 1 Interventional

BP1001-A in Patients With Advanced or Recurrent Solid Tumors

NCT04196257
View directory record Official record
Conditions
Solid Tumor, Adult, Carcinoma, Ovarian Epithelial, Fallopian Tube Neoplasms, Endometrial Cancer, Peritoneal Cancer, Solid Tumor
Interventions
BP1001-A (Liposomal Grb2 Antisense Oligonucleotide), BP1001-A (Liposomal Grb2 Antisense Oligonucleotide) with paclitaxel
Drug
Lead sponsor
Bio-Path Holdings, Inc.
Industry
Eligibility
18 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
4
States / cities
Silver Spring, Maryland • Detroit, Michigan • Dallas, Texas + 1 more
Source: ClinicalTrials.gov public record
Updated Mar 7, 2025 · Synced Oct 21, 2026, 10:42 PM EDT
Terminated Phase 3 Interventional

A Study Evaluating the Pain Palliation Benefit of Adding Custirsen to Docetaxel Retreatment or Cabazitaxel as Second Line Therapy in Men With Metastatic Castrate Resistant Prostate Cancer (mCRPC)

NCT01083615
View directory record Official record
Conditions
Castrate-Resistant Prostate Cancer, Hormone Refractory Prostate Cancer
Interventions
custirsen sodium, isotonic, 0.9% sodium chloride, docetaxel, cabazitaxel, prednisone
Drug
Lead sponsor
Achieve Life Sciences
Industry
Eligibility
18 Years to 99 Years · Male only
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
20
States / cities
La Verne, California • Fort Lauderdale, Florida • Tampa, Florida + 17 more
Source: ClinicalTrials.gov public record
Updated Oct 12, 2016 · Synced Oct 21, 2026, 10:42 PM EDT
Terminated Phase 2 Interventional

S0349 Rituximab, Cyclophosphamide, Doxorubicin, Vincristine, and Prednisone With or Without Oblimersen in Treating Patients With Advanced Diffuse Large B-Cell Non-Hodgkin's Lymphoma

NCT00080847
View directory record Official record
Conditions
Contiguous Stage II Adult Diffuse Large Cell Lymphoma, Noncontiguous Stage II Adult Diffuse Large Cell Lymphoma, Stage III Adult Diffuse Large Cell Lymphoma, Stage IV Adult Diffuse Large Cell Lymphoma
Interventions
oblimersen sodium, rituximab, cyclophosphamide, doxorubicin hydrochloride, vincristine sulfate, prednisone, laboratory biomarker analysis
Biological · Drug · Other
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
19 Years to 59 Years
Enrollment
160 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2004
U.S. locations
1
States / cities
San Antonio, Texas
Source: ClinicalTrials.gov public record
Updated Jan 7, 2013 · Synced Oct 21, 2026, 10:42 PM EDT
Completed Phase 1Phase 2 Interventional

Genasense® (Oblimersen Sodium), Fludarabine, and Rituximab in Subjects With Chronic Lymphocytic Leukemia

NCT00078234
View directory record Official record
Conditions
Chronic Lymphocytic Leukemia
Interventions
Oblimersen-rituximab-fludarabine
Drug
Lead sponsor
Genta Incorporated
Industry
Eligibility
18 Years and older
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2010
U.S. locations
3
States / cities
Washington D.C., District of Columbia • Buffalo, New York • New Hyde Park, New York
Source: ClinicalTrials.gov public record
Updated Nov 7, 2011 · Synced Oct 21, 2026, 10:42 PM EDT
Recruiting Phase 2 Interventional

A Study to Evaluate the Safety and Efficacy of NEXAGON® (Lufepirsen Ophthalmic Gel) in Subjects With PCED

NCT05966493
View directory record Official record
Conditions
Persistent Corneal Epithelial Defect
Interventions
lufepirsen high dose, Vehicle, lufepirsen low dose
Drug
Lead sponsor
Glaukos Corporation
Industry
Eligibility
2 Years and older
Enrollment
84 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
21
States / cities
Dothan, Alabama • Petaluma, California • Torrance, California + 17 more
Source: ClinicalTrials.gov public record
Updated Jul 24, 2025 · Synced Oct 21, 2026, 10:42 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Personalized Antisense Oligonucleotide Therapy for A Single Participant With CHCHD10 ALS

NCT06392126
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis
Interventions
nL-CHCHD-001
Drug
Lead sponsor
n-Lorem Foundation
Other
Eligibility
48 Years and older · Male only
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
1
States / cities
Jacksonville, Florida
Source: ClinicalTrials.gov public record
Updated Jul 13, 2026 · Synced Oct 21, 2026, 10:42 PM EDT
Active, not recruiting Phase 1 Interventional

A Clinical Trial of BP1002 in Patients With Advanced Lymphoid Malignancies

NCT04072458
View directory record Official record
Conditions
Mantle Cell Lymphoma, Peripheral T-cell Lymphoma (PTCL), Cutaneous T-cell Lymphoma (CTCL), Chronic Lymphocytic Leukemia (CLL), Small Lymphocytic Lymphoma (SLL), Follicular Lymphoma, Marginal Zone Lymphoma, Hodgkin Lymphoma, Waldenstrom Macroglobulinemia, DLBCL
Interventions
L-Bcl-2 antisense oligonucleotide
Drug
Lead sponsor
Bio-Path Holdings, Inc.
Industry
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2025
U.S. locations
5
States / cities
Augusta, Georgia • Valhalla, New York • Nashville, Tennessee + 2 more
Source: ClinicalTrials.gov public record
Updated Feb 24, 2025 · Synced Oct 21, 2026, 10:42 PM EDT
Completed Phase 2 Interventional

OGX-427 in Castration Resistant Prostate Cancer Patients

NCT01120470
View directory record Official record
Conditions
Castration Resistant Prostate Cancer
Interventions
OGX-427, Prednisone
Drug
Lead sponsor
British Columbia Cancer Agency
Other
Eligibility
18 Years and older · Male only
Enrollment
74 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2014
U.S. locations
1
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Feb 15, 2019 · Synced Oct 21, 2026, 10:42 PM EDT
Not listed Not applicable Interventional

Oblimersen and Dacarbazine in Treating Patients With Advanced Malignant Melanoma That Has Responded to Treatment on Clinical Trial GENTA-GM301

NCT00070343
View directory record Official record
Conditions
Melanoma (Skin)
Interventions
oblimersen sodium, dacarbazine
Biological · Drug
Lead sponsor
Jonsson Comprehensive Cancer Center
Other
Eligibility
Not listed
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2003
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Jan 6, 2014 · Synced Oct 21, 2026, 10:42 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Examination of Dermatologic Effects From Subcutaneous Injections of ISIS 113715

NCT00365781
View directory record Official record
Conditions
Type 2 Diabetes Mellitus
Interventions
ISIS 113715
Drug
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
18 Years to 65 Years
Enrollment
10 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2006
U.S. locations
1
States / cities
San Diego, California
Source: ClinicalTrials.gov public record
Updated Oct 17, 2007 · Synced Oct 21, 2026, 10:42 PM EDT
Recruiting Phase 1 Interventional

AMX0114 in Adult Participants With Amyotrophic Lateral Sclerosis

NCT06665165
View directory record Official record
Conditions
ALS
Interventions
AMX0114, Placebo
Drug · Other
Lead sponsor
Amylyx Pharmaceuticals Inc.
Industry
Eligibility
18 Years and older
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
10
States / cities
La Jolla, California • Washington D.C., District of Columbia • Gainesville, Florida + 7 more
Source: ClinicalTrials.gov public record
Updated Apr 14, 2026 · Synced Oct 21, 2026, 10:42 PM EDT
Recruiting Phase 2Phase 3 Interventional

A Study of OT-101 With mFOLFIRINOX in Patients With Advanced and Unresectable or Metastatic Pancreatic Cancer

NCT06079346
View directory record Official record
Conditions
Pancreatic Ductal Adenocarcinoma
Interventions
OT-101, mFOLFIRINOX
Drug
Lead sponsor
Oncotelic Inc.
Industry
Eligibility
18 Years and older
Enrollment
455 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
2
States / cities
Detroit, Michigan • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Aug 13, 2024 · Synced Oct 21, 2026, 10:42 PM EDT
Terminated Phase 1Phase 2 Interventional

A Double-blind, Randomized, Intra-subject Placebo-controlled, Multicenter, Multiple Dose Study, Evaluating Safety, Proof of Mechanism, Preliminary Efficacy and Systemic Exposure in Subjects With Confirmed DDEB or RDEB Diagnosis With One or More Pathogenic Mutations in Exon 73 in the COL7A1 Gene

NCT03605069
View directory record Official record
Conditions
Epidermolysis Bullosa Dystrophica, Recessive, Epidermolysis Bullosa Dystrophica, Dominant
Interventions
QR-313, Placebo
Drug
Lead sponsor
Phoenicis Therapeutics
Industry
Eligibility
4 Years and older
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018
U.S. locations
4
States / cities
Palo Alto, California • Aurora, Colorado • Minneapolis, Minnesota + 1 more
Source: ClinicalTrials.gov public record
Updated Aug 25, 2021 · Synced Oct 21, 2026, 10:42 PM EDT
Completed Phase 1 Interventional

Combination Chemotherapy Plus Oblimersen in Treating Patients With Previously Untreated Extensive-Stage Small Cell Lung Cancer

NCT00017251
View directory record Official record
Conditions
Extensive Stage Small Cell Lung Cancer
Interventions
oblimersen sodium, carboplatin, etoposide, pharmacological study, laboratory biomarker analysis
Biological · Drug · Other
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2001
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Jan 24, 2013 · Synced Oct 21, 2026, 10:42 PM EDT
Completed Phase 3 Interventional Results available

A Study to Evaluate the Efficacy and Safety of Intrathecally Administered RO7234292 (RG6042) in Participants With Manifest Huntington's Disease

NCT03761849
View directory record Official record
Conditions
Huntingtons Disease
Interventions
RO7234292, Placebo
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
25 Years to 65 Years
Enrollment
899 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
20
States / cities
Birmingham, Alabama • Phoenix, Arizona • La Jolla, California + 17 more
Source: ClinicalTrials.gov public record
Updated Mar 1, 2024 · Synced Oct 21, 2026, 10:42 PM EDT
Recruiting Phase 1Phase 2 Interventional

Study of ATX-01 in Participants With DM1

NCT06300307
View directory record Official record
Conditions
Myotonic Dystrophy 1
Interventions
ATX-01, Placebo
Drug
Lead sponsor
ARTHEx Biotech S.L.
Industry
Eligibility
18 Years to 64 Years
Enrollment
56 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
5
States / cities
Los Angeles, California • Gainesville, Florida • Iowa City, Iowa + 2 more
Source: ClinicalTrials.gov public record
Updated Feb 10, 2026 · Synced Oct 21, 2026, 10:42 PM EDT