Search clinical trials for: IONIS-FB-LRx

Total 639 results

Ionis Pharmaceuticals, Inc.

Active, not recruiting

A Study to Evaluate the Effectiveness and Safety of IONIS-FB-LRx, an Antisense Inhibitor of Complement Factor B, in Adult Participants With Primary IgA Nephropathy

Primary IgA Nephropathy

Canada, Australia, New Zealand, Singapore
Ionis Pharmaceuticals, Inc.

Withdrawn

Safety and Efficacy of IONIS-FB-Lrx in up to 120 Patients 55 and Older With Geographic Atrophy (GA) Secondary to Age-Related Macular Degeneration (AMD)

Geographic Atrophy | Age Related Macular Degeneration

Australia, United States, New Zealand
Ionis Pharmaceuticals, Inc.

Active, not recruiting

GOLDEN STUDY: A Study to Assess Safety and Efficacy of Multiple Doses of IONIS-FB-LRx in Participants With Geographic Atrophy Secondary to Age-Related Macular Degeneration (AMD)

Macular Degeneration | Geographic Atrophy

United States, Spain, Australia, Austria, Canada, Czechia, Netherlands, New Zealand, Poland
Ionis Pharmaceuticals, Inc.

Completed

Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS 702843 Administered Subcutaneously to Healthy Volunteers

Thalassemia

Australia
Ionis Pharmaceuticals, Inc.

Completed

A Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single and Multiple Doses of IONIS FXI-LRx in up to 84 Healthy Volunteers

Healthy Participants

Canada
Ionis Pharmaceuticals, Inc.

Completed

Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Ionis AGT-LRx in Healthy Volunteers

Healthy Volunteers

Canada
Ionis Pharmaceuticals, Inc.

Recruiting

A Study to Assess the Long-Term Safety and Efficacy of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Patients With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy

Hereditary Transthyretin-Mediated Amyloid Polyneuropathy

United States, Italy, Portugal, Spain, Taiwan, Sweden, Canada, Brazil, France, New Zealand, Argentina, Cyprus, Australia, Turkey
Ionis Pharmaceuticals, Inc.

Completed

A Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single-Ascending and Multiple Doses of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Healthy Japanese Participants

Healthy Participants

United States
Akcea Therapeutics
Ionis Pharmaceuticals, Inc.

Completed

Phase 2 Study of AKCEA-ANGPTL3-LRx (ISIS 703802) in Participants With Familial Chylomicronemia Syndrome (FCS)

Familial Chylomicronemia Syndrome | Lipoprotein Lipase Deficiency | Hyperlipoproteinemia Type 1

Canada
Ionis Pharmaceuticals, Inc.

Active, not recruiting

CARDIO-TTRansform: A Study to Evaluate the Efficacy and Safety of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Participants With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)

Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)

United States, Australia, Spain, Canada, Germany, Japan, Italy, Israel, Argentina, France, Portugal, Austria, Brazil, Greece, United Kingdom, Czechia, Sweden, Belgium, Denmark, Poland, Puerto Rico
Ionis Pharmaceuticals, Inc.

Recruiting

A Study to Evaluate Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx) in Patients With Polycythemia Vera

Phlebotomy Dependent Polycythemia Vera

United States, Canada, Hungary, United Kingdom, Australia, Poland
Ionis Pharmaceuticals, Inc.

Terminated

Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx)

Beta Thalassemia Intermedia

Australia, Thailand, Greece, Lebanon, Turkey
Ionis Pharmaceuticals, Inc.

Recruiting

A Study to Assess the Long-Term Safety and Efficacy of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE)

Hereditary Angioedema

United States, Puerto Rico, Belgium, Bulgaria, Canada, France, Germany, Israel, Italy, Netherlands, Poland, Spain, Turkey, United Kingdom
Ionis Pharmaceuticals, Inc.

Active, not recruiting

An Extension Study of Donidalorsen (IONIS-PKK-LRx) in Participants With Hereditary Angioedema

Hereditary Angioedema

Netherlands, United States
Ionis Pharmaceuticals, Inc.

Completed

A Study to Assess the Safety, Tolerability, and Efficacy of IONIS-GHR-LRx Administered in Patients With Acromegaly

Acromegaly

United States, Hungary, Serbia, Estonia, Italy, Latvia, Lithuania, Poland, Romania, Russian Federation
Ionis Pharmaceuticals, Inc.

Completed

Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Donidalorsen (IONIS-PKK-LRx) Administered Subcutaneously to Healthy Volunteers

Healthy Volunteers

Canada
Ionis Pharmaceuticals, Inc.

Completed

Safety, Tolerability, and Efficacy of IONIS-GHR-LRx in Participants With Acromegaly Being Treated With Long-acting Somatostatin Receptor Ligands

Acromegaly

United States, Hungary, Serbia, Romania, Russian Federation, Lithuania, Poland
Ionis Pharmaceuticals, Inc.

Completed

NEURO-TTRansform: A Study to Evaluate the Efficacy and Safety of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Participants With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy

Hereditary Transthyretin-Mediated Amyloid Polyneuropathy

United States, Spain, Taiwan, Italy, Germany, Argentina, Australia, Brazil, Canada, Cyprus, France, Greece, New Zealand, Portugal, Sweden, Turkey
Akcea Therapeutics
Ionis Pharmaceuticals, Inc.

Completed

Study of ISIS 678354 (AKCEA-APOCIII-LRx) in Participants With Hypertriglyceridemia and Established Cardiovascular Disease (CVD)

Cardiovascular Diseases | Hypertriglyceridemia

United States, Canada
Ionis Pharmaceuticals, Inc.
Akcea Therapeutics

Completed

Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS APO(a)-LRx in Healthy Volunteers With Elevated Lipoprotein(a)

Elevated Lipoprotein(a)

Canada
Ionis Pharmaceuticals, Inc.

Completed

A Study to Assess the Safety, Tolerability and Efficacy of IONIS-AGT-LRx in Participants With Chronic Heart Failure With Reduced Ejection Fraction (ASTRAAS-HF)

Chronic Heart Failure With Reduced Ejection Fraction

United States, Hungary, Poland
Ionis Pharmaceuticals, Inc.

Completed

A Study to Assess the Safety, Tolerability and Efficacy of IONIS-AGT-LRx in Hypertensive Participants With Uncontrolled Blood Pressure (ASTRAAS)

Hypertension

United States, Canada
Ionis Pharmaceuticals, Inc.

Completed

A Study to Assess the Clinical Efficacy of Donidalorsen (Also Known as IONIS-PKK-LRx and ISIS 721744) in Participants With Hereditary Angioedema

Hereditary Angioedema

United States, Netherlands
Akcea Therapeutics
Ionis Pharmaceuticals, Inc.

Completed

Phase 2 Study of ISIS 681257 (AKCEA-APO(a)-LRx) in Participants With Hyperlipoproteinemia(a) and Cardiovascular Disease

Cardiovascular Disease | Elevated Lipoprotein(a)

United States, Canada, Denmark, Germany, Netherlands
Ionis Pharmaceuticals, Inc.

Completed

A Study to Assess the Safety, Tolerability and Efficacy of IONIS-AGT-LRx, an Antisense Inhibitor Administered Subcutaneously to Hypertensive Participants With Controlled Blood Pressure

Mild Hypertension

United States
Ionis Pharmaceuticals, Inc.
Akcea Therapeutics

Completed

Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS ANGPTL3-LRx in Healthy Volunteers With Elevated Triglycerides and Participants With Familial Hypercholesterolemia

Hypertriglyceridemia | Familial Hypercholesterolemia

Canada
Ionis Pharmaceuticals, Inc.

Completed

Evaluate the Safety and Tolerability, as Well as the Pharmacokinetic and Pharmacodynamic Profiles of Single and Multiple Doses of Eplontersen Administered Subcutaneously to Healthy Volunteers and Patients With Hereditary Transthyretin-Mediated Amyloidosis (hATTR ).

Healthy Volunteers | hATTR Amyloidosis

Canada
Ionis Pharmaceuticals, Inc.

Completed

Extension Study Assessing Long Term Safety and Efficacy of IONIS-TTR Rx in Familial Amyloid Polyneuropathy (FAP)

Amyloidosis | Familial Amyloid Polyneuropathy | FAP | TTR | Transthyretin

United States, Spain, Argentina, Brazil, France, Germany, Italy, Portugal, United Kingdom
Biogen

Completed

An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy Who Previously Participated in ISIS 396443-CS1 (NCT01494701)

Spinal Muscular Atrophy

United States
Ionis Pharmaceuticals, Inc.

Completed

Efficacy and Safety of Inotersen in Familial Amyloid Polyneuropathy

Amyloidosis | Familial Amyloid Polyneuropathy | FAP | TTR | Transthyretin

New Zealand, United States, Spain, Argentina, Brazil, France, Germany, Italy, Portugal, United Kingdom
Biogen

Completed

An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in ISIS 396443-CS2 (NCT01703988) or ISIS 396443-CS10 (NCT01780246)

Spinal Muscular Atrophy

United States
Ionis Pharmaceuticals, Inc.
Bayer

Completed

A Study of Safety, PK, & PD of ISIS 416858 Administered Subcutaneously to Patients With End-Stage Renal Disease on Hemodialysis

End-stage Renal Disease (ESRD)

Canada
Biogen

Completed

A Study for Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in Nusinersen (ISIS 396443) Investigational Studies (SHINE)

Spinal Muscular Atrophy

United States, Spain, Germany, United Kingdom, Australia, Japan, Canada, Belgium, France, Hong Kong, Italy, Korea, Republic of, Sweden, Turkey
Biogen

Completed

An Open-label Safety, Tolerability, and Dose-range Finding Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA) (SMNRx)

Spinal Muscular Atrophy

United States
Biogen

Completed

A Study to Assess the Efficacy, Safety and Pharmacokinetics of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy (SMA)

Spinal Muscular Atrophy

United States, Canada
Akcea Therapeutics
Ionis Pharmaceuticals, Inc.

Completed

Study of ISIS 703802 in Participants With Hypertriglyceridemia, Type 2 Diabetes Mellitus, and Nonalcoholic Fatty Liver Disease

Diabetes Mellitus, Type 2 | NAFLD | Hypertriglyceridemia | Fatty Liver, Nonalcoholic

United States, Canada
Biogen

Terminated

A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy (ENDEAR)

Spinal Muscular Atrophy

Korea, Republic of, United States, Spain, Canada, Germany, Australia, Japan, Sweden, Belgium, France, Italy, Turkey, United Kingdom
Forte Biosciences, Inc.

Terminated

Continued Safety Evaluation of FB-401 in Children, Adolescents and Adults (2 Years and Older) With Mild to Moderate Atopic Dermatitis Previously Enrolled in the FB401-01 Study

Atopic Dermatitis

United States
Heidi Ruotsalainen

Unknown

Promoting Overweight Adolescents Physical Activity

Obesity

Finland
Donald B. Kohn, M.D.
California Institute for Regenerative Medicine (CIRM)

Recruiting

Stem Cell Gene Therapy for Sickle Cell Disease

Sickle Cell Disease

United States
1ST Biotherapeutics, Inc.

Completed

Safety, Tolerability, and Pharmacokinetics of Oral FB-101 in Healthy Subjects

Parkinson's Disease

United States
Biogen

Completed

A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Participants With Later-onset Spinal Muscular Atrophy (SMA) (CHERISH)

Spinal Muscular Atrophy

United States, Germany, Spain, Canada, France, Hong Kong, Italy, Japan, Korea, Republic of, Sweden
University Hospital, Ghent

Recruiting

Family Involvement in CBGT of OCD: a Randomized Controlled Trial

Obsessive-Compulsive Disorder

Belgium
Forte Biosciences, Inc.

Completed

Evaluation of FB-401 in Children, Adolescents and Adults (2 Years and Older) With Mild to Moderate Atopic Dermatitis

Atopic Dermatitis

United States
University Medical Center Groningen

Active, not recruiting

IL2 Imaging in Renal Transplantation

Kidney Transplant Rejection

Netherlands
Ionis Pharmaceuticals, Inc.

Completed

A Phase 1/2a Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple Doses of IONIS-ENaCRx in Healthy Volunteers and Patients With Cystic Fibrosis

Cystic Fibrosis | Healthy Subjects

United Kingdom, Germany
Ionis Pharmaceuticals, Inc.

Completed

Safety, Tolerability, and Pharmacodynamics of IONIS-DGAT2Rx in Adult Patients With Type 2 Diabetes

Hepatic Steatosis

Canada, Hungary, Poland, United Kingdom
University of Chicago
National Institute on Minority Health and Health Disparities (NIMHD)

Completed

CommunityRx-Hunger (CRx-H)

Food Insecurity

United States
Ionis Pharmaceuticals, Inc.
AstraZeneca

Completed

Phase 1/2, Open-label, Dose-escalation Study of IONIS-STAT3Rx, Administered to Patients With Advanced Cancers

Lymphoma | DLBCL | Advanced Cancers

United States
University of Zurich

Recruiting

Comparison of Transbronchial Cryobiopsy and Forceps Biopsy in Lung Transplant Recipients (Kryo-LUTPL)

Lung Transplant Rejection | Lung Transplant Failure and Rejection

Switzerland

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