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ClinicalTrials.gov public records Last synced Sep 6, 2026, 1:38 PM EDT

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Showing 1–24 of 116 matching trials from the live ClinicalTrials.gov search.
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Withdrawn Phase 1Phase 2 Interventional

Clinical Trial of BP1001 (Liposomal Grb2 Antisense Oligonucleotide) in Combination With Dasatinib in Patients With Ph + CML Who Have Failed TKI, Ph+ AML, Ph+ MDS

NCT02923986
View directory record Official record
Conditions
Chronic Myelogenous Leukemia, Ph1-Positive, Acute Myeloid Leukemia, Myelodysplastic Syndrome
Interventions
BP1001 (varying dose), BP1001 (fixed dose), Dasatinib
Drug
Lead sponsor
Bio-Path Holdings, Inc.
Industry
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2020
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated May 27, 2020 · Synced Sep 6, 2026, 1:38 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 6, 2026, 1:38 PM EDT
Not listed Phase 1 Interventional

A Phase I First in Human Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of WGI-0301 in Patients With Advanced Solid Tumors

NCT05267899
View directory record Official record
Conditions
Advanced Solid Tumors
Interventions
WGI-0301
Drug
Lead sponsor
Zhejiang Haichang Biotech Co., Ltd.
Industry
Eligibility
18 Years and older
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
3
States / cities
Los Angeles, California • Whittier, California • Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 25, 2023 · Synced Sep 6, 2026, 1:38 PM EDT
Terminated Phase 1 Interventional Results available

ARRx in Combination With Enzalutamide in Metastatic Castration Resistant Prostate Cancer

NCT03300505
View directory record Official record
Conditions
Prostate Cancer
Interventions
ARRx, Enzalutamide
Drug
Lead sponsor
University of Michigan Rogel Cancer Center
Other
Eligibility
18 Years and older · Male only
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
2
States / cities
Ann Arbor, Michigan • Detroit, Michigan
Source: ClinicalTrials.gov public record
Updated May 12, 2025 · Synced Sep 6, 2026, 1:38 PM EDT
Recruiting Phase 1 Interventional Accepts healthy volunteers

Danvatirsen Monotherapy Followed by Combination With Venetoclax in Relapsed/Refractory MDS & AML

NCT05986240
View directory record Official record
Conditions
AML/MDS, Acute Myeloid Leukemia, Myelodysplastic Syndromes
Interventions
Danvatirsen, Danvatirsen + Venetoclax
Drug · Combination Product
Lead sponsor
Montefiore Medical Center
Other
Eligibility
18 Years and older
Enrollment
38 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2024 – 2030
U.S. locations
2
States / cities
The Bronx, New York • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Nov 9, 2025 · Synced Sep 6, 2026, 1:38 PM EDT
Recruiting Phase 2 Interventional

Clinical Trial of BP1001 in Combination With With Venetoclax Plus Decitabine in AML

NCT02781883
View directory record Official record
Conditions
Acute Myeloid Leukemia (AML)
Interventions
BP1001 in combination with Ventoclax plus decitabine, BP1001 plus decitabine
Drug
Lead sponsor
Bio-Path Holdings, Inc.
Industry
Eligibility
18 Years and older
Enrollment
108 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2028
U.S. locations
9
States / cities
Los Angeles, California • Augusta, Georgia • Fairway, Kansas + 5 more
Source: ClinicalTrials.gov public record
Updated Mar 6, 2025 · Synced Sep 6, 2026, 1:38 PM EDT
Completed Phase 3 Interventional

Dexamethasone With or Without Oblimersen in Treating Patients With Relapsed or Refractory Multiple Myeloma

NCT00017602
View directory record Official record
Conditions
Multiple Myeloma and Plasma Cell Neoplasm
Interventions
oblimersen sodium, dexamethasone
Biological · Drug
Lead sponsor
Genta Incorporated
Industry
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2009
U.S. locations
1
States / cities
Berkeley Heights, New Jersey
Source: ClinicalTrials.gov public record
Updated Jan 5, 2014 · Synced Sep 6, 2026, 1:38 PM EDT
Terminated Phase 2Phase 3 Interventional Results available

Study to Evaluate the Efficacy Safety and Tolerability of Ultevursen in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene (Sirius)

NCT05158296
View directory record Official record
Conditions
Retinitis Pigmentosa, Usher Syndrome Type 2, Deaf Blind, Retinal Disease, Eye Diseases, Eye Diseases, Hereditary, Eye Disorders Congenital, Vision Disorders
Interventions
Ultevursen, Sham-procedure
Drug · Other
Lead sponsor
Laboratoires Thea
Industry
Eligibility
12 Years and older
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
10
States / cities
San Diego, California • San Francisco, California • Miami, Florida + 7 more
Source: ClinicalTrials.gov public record
Updated Jul 11, 2024 · Synced Sep 6, 2026, 1:38 PM EDT
Recruiting Phase 2 Interventional

Study of AHB-137 in Chronic Hepatitis B (CHB) Participants in North America and Europe Regions

NCT07654283
View directory record Official record
Conditions
Hepatitis B, Chronic
Interventions
AHB-137
Drug
Lead sponsor
AusperBio Therapeutics Inc.
Industry
Eligibility
18 Years to 65 Years
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
4
States / cities
Palo Alto, California • Baltimore, Maryland • New York, New York + 1 more
Source: ClinicalTrials.gov public record
Updated Aug 25, 2026 · Synced Sep 6, 2026, 1:38 PM EDT
Active, not recruiting Phase 2Phase 3 Interventional

A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE)

NCT03913143
View directory record Official record
Conditions
Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Vision Disorders, Sensation Disorders, Neurologic Manifestations, Eye Diseases, Eye Diseases, Hereditary, Eye Disorders Congenital, Retinal Disease
Interventions
sepofarsen, Sham
Drug · Other
Lead sponsor
ProQR Therapeutics
Industry
Eligibility
8 Years and older
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
1
States / cities
Iowa City, Iowa
Source: ClinicalTrials.gov public record
Updated Mar 16, 2022 · Synced Sep 6, 2026, 1:38 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 9, 2026 · Synced Sep 6, 2026, 1:38 PM EDT
Recruiting Phase 3 Interventional

A Study to Evaluate the Efficacy of NIO752 in Participants With Progressive Supranuclear Palsy

NCT07498426
View directory record Official record
Conditions
Progressive Supranuclear Palsy Richardson Syndrome (PSP-RS)
Interventions
NIO752, Placebo
Other · Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
41 Years to 81 Years
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2031
U.S. locations
5
States / cities
Scottsdale, Arizona • San Francisco, California • Englewood, Colorado + 2 more
Source: ClinicalTrials.gov public record
Updated Jul 28, 2026 · Synced Sep 6, 2026, 1:38 PM EDT
Active, not recruiting Phase 2 Interventional

An Open-Label Extension Study of STK-001 for Patients With Dravet Syndrome

NCT04740476
View directory record Official record
Conditions
Dravet Syndrome
Interventions
zorevunersen (STK-001)
Drug
Lead sponsor
Stoke Therapeutics, Inc
Industry
Eligibility
30 Months and older
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2029
U.S. locations
17
States / cities
San Francisco, California • Aurora, Colorado • Washington D.C., District of Columbia + 14 more
Source: ClinicalTrials.gov public record
Updated Feb 3, 2026 · Synced Sep 6, 2026, 1:38 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Autologous Dendritic Cell Therapy for Type 1 Diabetes Suppression: A Safety Study

NCT00445913
View directory record Official record
Conditions
Type 1 Diabetes
Interventions
Diabetes-suppressive dendritic cell vaccine, control dendritic cells
Biological
Lead sponsor
University of Pittsburgh
Other
Eligibility
18 Years to 60 Years
Enrollment
10 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2007 – 2016
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Feb 14, 2016 · Synced Sep 6, 2026, 1:38 PM EDT
Recruiting Phase 3 Interventional

Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)

NCT06891443
View directory record Official record
Conditions
Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Sensation Disorders, Vision Disorder, Neurological Manifestations, Eye Diseases, Hereditary, Eye Diseases, Eye Disorders Congenital, Retinal Disease
Interventions
sepofarsen, Placebo IVT
Drug · Other
Lead sponsor
Laboratoires Thea
Industry
Eligibility
6 Years and older
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
5
States / cities
San Francisco, California • Miami, Florida • Iowa City, Iowa + 2 more
Source: ClinicalTrials.gov public record
Updated Jun 24, 2026 · Synced Sep 6, 2026, 1:38 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 16, 2007 · Synced Sep 6, 2026, 1:38 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Personalized Antisense Oligonucleotide for A Single Participant With ATN1 Gene Mutation

NCT07084311
View directory record Official record
Conditions
Dentatorubral-Pallidoluysian Atrophy
Interventions
nL-ATN1-002
Drug
Lead sponsor
n-Lorem Foundation
Other
Eligibility
29 Years to 29 Years · Female only
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Honolulu, Hawaii
Source: ClinicalTrials.gov public record
Updated Jul 12, 2026 · Synced Sep 6, 2026, 1:38 PM EDT
Recruiting Phase 2 Interventional

Phase 2 Study of ADX-038 in Participants With Geographic Atrophy

NCT06990269
View directory record Official record
Conditions
Geographic Atrophy Secondary to Age-related Macular Degeneration
Interventions
ADX-038, Placebo
Drug
Lead sponsor
ADARx Pharmaceuticals, Inc.
Industry
Eligibility
60 Years and older
Enrollment
240 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
21
States / cities
Gilbert, Arizona • Phoenix, Arizona • Bakersfield, California + 18 more
Source: ClinicalTrials.gov public record
Updated Sep 2, 2026 · Synced Sep 6, 2026, 1:38 PM EDT
Completed Phase 1 Interventional

Genasense as a 2-hour Intravenous Infusion in Subjects With Solid Tumors

NCT00636545
View directory record Official record
Conditions
Solid Tumors
Interventions
Oblimersen (Genasense)
Drug
Lead sponsor
Genta Incorporated
Industry
Eligibility
18 Years and older
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2010
U.S. locations
1
States / cities
San Antonio, Texas
Source: ClinicalTrials.gov public record
Updated Apr 5, 2012 · Synced Sep 6, 2026, 1:38 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 25, 2013 · Synced Sep 6, 2026, 1:38 PM EDT
Active, not recruiting Phase 3 Interventional

Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS)

NCT06617429
View directory record Official record
Conditions
Angelman Syndrome
Interventions
GTX-102, Sham-LP
Drug · Procedure
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
4 Years to 17 Years
Enrollment
129 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
14
States / cities
Los Angeles, California • San Diego, California • San Francisco, California + 11 more
Source: ClinicalTrials.gov public record
Updated Aug 5, 2026 · Synced Sep 6, 2026, 1:38 PM EDT
Active, not recruiting Phase 2 Interventional

Study to Evaluate Ultevursen in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene

NCT06627179
View directory record Official record
Conditions
Retinitis Pigmentosa (RP), Usher Syndrome Type 2, Deaf Blind, Retinal Disease, Eye Diseases, Hereditary, Eye Disorders Congenital, Vision Disorders
Interventions
Intravitreal Injection of Ultevursen, No intervention, will not receive any active study intervention
Drug · Other
Lead sponsor
Laboratoires Thea
Industry
Eligibility
8 Years and older
Enrollment
81 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2028
U.S. locations
10
States / cities
San Francisco, California • Miami, Florida • Atlanta, Georgia + 7 more
Source: ClinicalTrials.gov public record
Updated Jul 8, 2026 · Synced Sep 6, 2026, 1:38 PM EDT
Terminated Phase 1Phase 2 Interventional

A Phase 1-2, XIAP Antisense AEG35156 With Gemcitabine in Patients With Advanced Pancreatic Cancer

NCT00557596
View directory record Official record
Conditions
Carcinoma, Pancreas
Interventions
AEG35156
Drug
Lead sponsor
Aegera Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2009
U.S. locations
4
States / cities
Scottsdale, Arizona • Tucson, Arizona • Canton, Ohio
Source: ClinicalTrials.gov public record
Updated Nov 30, 2009 · Synced Sep 6, 2026, 1:38 PM EDT
Completed Phase 1Phase 2 Interventional

Study of XIAP Antisense Given With Chemotherapy for Refractory/Relapsed AML

NCT00363974
View directory record Official record
Conditions
Leukemia, Myelomonocytic, Acute
Interventions
XIAP antisense
Drug
Lead sponsor
Aegera Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
54 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2009
U.S. locations
5
States / cities
Los Angeles, California • Chicago, Illinois • Baltimore, Maryland + 1 more
Source: ClinicalTrials.gov public record
Updated Nov 30, 2009 · Synced Sep 6, 2026, 1:38 PM EDT