Search clinical trials for: N-acetyl galactosamine-conjugated antisense oligonucleotide

Total 655 results

AstraZeneca

Completed

A Study to Investigate the Safety, Tolerability, and Pharmacokinetics of AZD7503 in Japanese Healthy Participants.

Heathy Participants

Japan
Algemeen Ziekenhuis Maria Middelares

Not yet recruiting

Interpectoral-pectoserratus Plane Block for Minimally Invasive Aortic Valve Replacement Via Right Anterior Minithoracotomy (PAINCARE)

Analgesic Effect

Belgium
Massachusetts General Hospital
n-Lorem Foundation

Not yet recruiting

Single Participant Study of an Experimental ASO Treatment for TUBB4A-related Leukodystrophy

Genetic Disease
CAMP4 Therapeutics Corporation

Recruiting

Study of CMP-CPS-001 in Healthy Volunteers

Healthy Volunteers

Australia
QurAlis Corporation

Recruiting

A Study Evaluating the Safety and Tolerability of QRL-201 in ALS

Amyotrophic Lateral Sclerosis

Canada, Germany, Netherlands, Ireland, United Kingdom, Belgium
First Affiliated Hospital of Fujian Medical University
Peking Union Medical College Hospital; Fujian Medical University Union Hospital

Recruiting

Antisense Oligonucleotide for Spinal Muscular Atrophy

Spinal Muscular Atrophy

China
University Hospital, Montpellier
Hôpital Cochin; Hôpital Necker-Enfants Malades; Foch Hospital, Suresnes, FRANCE

Recruiting

Validation of Therapeutic Efficacy Targeting the Splicing Variants in Cystic Fibrosis and CFTR Pathologies (ONB-CFTR)

Cystic Fibrosis | CFTR Gene Mutation

France
ProQR Therapeutics

Recruiting

An Open-label, Dose Escalation and Double-masked, Randomized, Controlled Trial Evaluating Safety and Tolerability of Sepofarsen in Children (<8 Years of Age) With LCA10 Caused by Mutations in the CEP290 Gene. (BRIGHTEN)

Eye Diseases | Neurologic Manifestations | Retinal Degeneration | Retinal Dystrophies | Sensation Disorders | Vision Disorders | Blindness | Leber Congenital Amaurosis | Leber Congenital Amaurosis 10 | Retinal Disease | Eye Disorders Congenital

Belgium, Brazil, Canada, Germany, Italy, Netherlands, United Kingdom
Brigham and Women's Hospital

Not yet recruiting

ION-682884 in Patients With TTR Amyloid Cardiomyopathy

Transthyretin Amyloid Cardiopathy

United States
Bio-Path Holdings, Inc.

Recruiting

BP1001-A in Patients With Advanced or Recurrent Solid Tumors

Fallopian Tube Neoplasms | Carcinoma, Ovarian Epithelial | Solid Tumor | Endometrial Cancer | Peritoneal Cancer | Solid Tumor, Adult

United States
Bio-Path Holdings, Inc.

Recruiting

A Clinical Trial of BP1002 in Patients With Advanced Lymphoid Malignancies

Follicular Lymphoma | Hodgkin Lymphoma | Waldenstrom Macroglobulinemia | Mantle Cell Lymphoma | Marginal Zone Lymphoma | Chronic Lymphocytic Leukemia (CLL) | Small Lymphocytic Lymphoma (SLL) | DLBCL | Cutaneous T-cell Lymphoma (CTCL) | Peripheral T-cell Lymphoma (PTCL)

United States
Assistance Publique - Hôpitaux de Paris

Recruiting

Natural History of Pompe Disease (POMPE)

Glycogen Storage Disease Type II, Adult

France
GlaxoSmithKline

Active, not recruiting

Study of Bepirovirsen in Nucleos(t)Ide Analogue-treated Participants With Chronic Hepatitis B (B-Well 1) (B-Well 1)

Chronic Hepatitis B | Hepatitis B, Chronic

France, Spain, Taiwan, Bulgaria, Poland, United Kingdom, United States, Mexico, Singapore, Canada, Korea, Republic of, India, China, Thailand, Greece, Hong Kong, Italy, Romania, Brazil, Germany, Hungary, Japan, Malaysia, Turkey, Argentina, Vietn... and more
GlaxoSmithKline

Active, not recruiting

Study of Bepirovirsen in Nucleos(t)Ide Analogue-treated Participants With Chronic Hepatitis B (B-Well 2) (B-Well 2)

Chronic Hepatitis B | Hepatitis B, Chronic

United States, Australia, Germany, Spain, Taiwan, Bulgaria, France, Thailand, Canada, Italy, Malaysia, Philippines, China, Hungary, Korea, Republic of, Romania, Greece, India, Mexico, United Kingdom, Japan, Panama, Turkey, Poland, Brazil, Argentin... and more
Novartis Pharmaceuticals

Active, not recruiting

Safety, Tolerability and Pharmacokinetics of Multiple Ascending Doses of NIO752 in Progressive Supranuclear Palsy

Progressive Supranuclear Palsy (PSP)

Germany, United Kingdom, Canada, United States
n-Lorem Foundation
Mayo Clinic

Active, not recruiting

Personalized Antisense Oligonucleotide Therapy for A Single Participant With CHCHD10 ALS

Amyotrophic Lateral Sclerosis

United States
University of California, San Diego
California Institute for Regenerative Medicine (CIRM); n-Lorem Foundation

Active, not recruiting

Personalized Antisense Oligonucleotide Therapy for Rare Pediatric Genetic Disease: SCN2A

Genetic Disease

United States
Daiichi Sankyo Co., Ltd.
Orphan Disease Treatment Institute Co., Ltd.

Completed

Study of DS-5141b in Patients With Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy

Japan
Hoffmann-La Roche

Completed

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

Huntingtons Disease

United States, United Kingdom, Australia, Canada, Japan, Poland, Netherlands, Germany, Russian Federation, France, Spain, Argentina, New Zealand, Austria, Denmark, Italy, Switzerland, Chile
Autotelicbio

Completed

TASO-001 in Combination With Recombinant Interleukin-2(Aldesleukin) in Advanced or Metastatic Solid Tumor

Solid Tumor

Korea, Republic of
Praxis Precision Medicines

Active, not recruiting

A Clinical Trial of PRAX-222 in Pediatric Participants With Early Onset SCN2A Developmental and Epileptic Encephalopathy

Epilepsy | SCN2A-DEE

United States
Daiichi Sankyo Co., Ltd.

Active, not recruiting

Long-term, Extension Study of DS-5141b in Patients With Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy

Japan
Institut National de la Santé Et de la Recherche...
Biogen; Ionis Pharmaceuticals, Inc.

Completed

Integrated Functional Evaluation of the Cerebellum (CERMOI)

Spinocerebellar Ataxia Type 2 | Spinocerebellar Ataxia Type 7

France
ProQR Therapeutics

Terminated

Study to Evaluate the Efficacy Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene With Early to Moderate Vision Loss (Celeste)

Eye Diseases | Retinitis Pigmentosa | Eye Diseases, Hereditary | Vision Disorders | Retinal Disease | Eye Disorders Congenital | Usher Syndrome Type 2 | Deaf Blind

United States, United Kingdom
ProQR Therapeutics

Terminated

Extension Study to Study PQ-110-001 (NCT03140969) (INSIGHT)

Eye Diseases | Neurologic Manifestations | Eye Diseases, Hereditary | Sensation Disorders | Vision Disorders | Blindness | Leber Congenital Amaurosis | Leber Congenital Amaurosis 10 | Retinal Disease | Eye Disorders Congenital

United States, Belgium
ProQR Therapeutics

Terminated

An Open-Label Extension Study to Evaluate Safety & Tolerability of QR-421a in Subjects With Retinitis Pigmentosa (HELIA)

Retinitis Pigmentosa | Usher Syndrome Type 2

United States, Canada, France
ProQR Therapeutics

Completed

Study to Evaluate QR-110 in Leber's Congenital Amaurosis (LCA) Due to the c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene

Leber's Congenital Amaurosis

United States, Belgium
ProQR Therapeutics

Active, not recruiting

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)

Eye Diseases | Retinitis Pigmentosa | Eye Diseases, Hereditary | Vision Disorders | Retinal Disease | Eye Disorders Congenital | Usher Syndrome Type 2 | Deaf Blind

United States, Netherlands, United Kingdom, Germany
TAK-Circulator Co.

Completed

The Safety and Pharmacokinetics Study of TAKC-02 Inhalation Solution in Healthy Adult Males.

Healthy Volunteer

Japan
ProQR Therapeutics

Active, not recruiting

A Study to Evaluate the Safety and Tolerability of QR-1123 in Subjects With Autosomal Dominant Retinitis Pigmentosa Due to the P23H Mutation in the RHO Gene (AURORA)

Eye Diseases | Retinitis | Eye Diseases, Hereditary | Retinal Dystrophies | Vision Disorders | Retinal Disease | Autosomal Dominant Retinitis Pigmentosa | Vision Tunnel

United States
ProQR Therapeutics

Completed

Study to Evaluate Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene (Stellar)

Eye Diseases | Retinitis Pigmentosa | Eye Diseases, Hereditary | Vision Disorders | Retinal Disease | Eye Disorders Congenital | Usher Syndrome Type 2 | Deaf Blind

United States, Canada, France
ProQR Therapeutics

Active, not recruiting

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

Eye Diseases | Neurologic Manifestations | Eye Diseases, Hereditary | Sensation Disorders | Vision Disorders | Blindness | Leber Congenital Amaurosis | Leber Congenital Amaurosis 10 | Retinal Disease | Eye Disorders Congenital

United States, Belgium, Brazil, Canada, France, Germany, Italy, Netherlands, United Kingdom
Phoenicis Therapeutics

Terminated

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

Epidermolysis Bullosa Dystrophica, Recessive | Epidermolysis Bullosa Dystrophica, Dominant

United States, Spain, France
Brigham and Women's Hospital

Unknown

24 Month Open Label Study of the Tolerability and Efficacy of Inotersen in TTR Amyloid Cardiomyopathy Patients

Amyloidosis

United States
ProQR Therapeutics
European Commission

Completed

Exploratory Study to Evaluate QR-010 in Subjects With Cystic Fibrosis ΔF508 CFTR Mutation

Cystic Fibrosis

United States, France, Belgium
Bio-Path Holdings, Inc.

Withdrawn

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

Acute Myeloid Leukemia | Myelodysplastic Syndrome | Chronic Myelogenous Leukemia, Ph1-Positive

United States
Bio-Path Holdings, Inc.

Completed

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

Recurrent Adult Acute Myeloid Leukemia | Acute Lymphoblastic Leukemia | Myelodysplastic Syndrome | Ph1 Positive CML

United States
Imperial College London
Sarepta Therapeutics, Inc.; Department of Health, United Kingdom

Completed

Safety and Efficacy Study of Antisense Oligonucleotides in Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy

United Kingdom
ProQR Therapeutics
European Commission

Completed

Dose Escalation Study of QR-010 in Homozygous ΔF508 Cystic Fibrosis Patients

Cystic Fibrosis

United States, Spain, United Kingdom, Germany, Italy, Belgium, Canada, Czechia, Denmark, France
National Cancer Institute (NCI)

Completed

A Pilot Study of EZN-2968, an Antisense Oligonucleotide Inhibitor of HIF-1alpha, in Adults With Advanced Solid Tumors With Liver Metastases

Neoplasms | Liver Metastases

United States
Sterna Biologicals GmbH & Co. KG

Completed

Efficacy, Pharmacokinetics, Tolerability, Safety of SB012 Intrarectally Applied in Active Ulcerative Colitis Patients (SECURE)

Colitis, Ulcerative

Germany
Isarna Therapeutics GmbH

Completed

Phase I Dose Escalation Study to Investigate the Safety of ISTH0036 in Subjects With Glaucoma Undergoing Trabeculectomy

Primary Open Angle Glaucoma

Germany
National Cancer Institute (NCI)

Terminated

AZD9150, a STAT3 Antisense Oligonucleotide, in People With Malignant Ascites

Gastrointestinal Neoplasms | Ovarian Neoplasms | Ovarian Cancer | Ascites | Gastrointestinal Cancer

United States
AstraZeneca
Ionis Pharmaceuticals, Inc.

Completed

A Phase I/Ib Study of AZD9150 (ISIS-STAT3Rx) in Patients With Advanced/Metastatic Hepatocellular Carcinoma

Advanced Adult Hepatocellular Carcinoma | Hepatocellular Carcinoma Metastatic

Korea, Republic of, Taiwan, Hong Kong, Japan
Sterna Biologicals GmbH & Co. KG

Completed

Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of the Topical Formulation SB011 Applied to Lesional Skin in Patients With Atopic Eczema

Mild to Moderate Atopic Dermatitis

Germany
Brigham and Women's Hospital
GlaxoSmithKline; Ionis Pharmaceuticals, Inc.

Withdrawn

The Effect of an Antisense Oligonucleotide to Lower Transthyretin (TTR) Levels on the Progression of -Wild-type TTR Involving the Heart

Amyloidosis
Sterna Biologicals GmbH & Co. KG

Completed

Safety, Efficacy, PK, and PD Characteristics of Orally Inhaled SB010 in Male Patients With Mild Asthma (Multiple Dose)

Asthma

Germany
Abramson Cancer Center of the University of Pennsylvania
National Cancer Institute (NCI)

Completed

Chemotherapy and Bone Marrow Transplantation in Treating Patients With Chronic Myelogenous Leukemia

Leukemia

United States
Eleos, Inc.

Withdrawn

Aezea® (Cenersen) and Chemotherapy for AML Subjects ≥ 55 Years of Age With No Response to Frontline Induction Course

Acute Myelogenous Leukemia
University of Southern California
Sponsor Name Pending

Withdrawn

A Study of VEGF-Antisense Oligonucleotide in Combination With Pemetrexed and Cisplatin for the Treatment of Advanced Malignant Mesothelioma

Mesothelioma

United States

Search clinical trials for: N-acetyl galactosamine-conjugated antisense oligonucleotide

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