Clinical Trials on RYR-1 Myopathy in New Zealand

Total 763 results

Alvotech Swiss AG

Completed

PK, Safety and Tolerability Study of AVT02 (Adalimumab) Pre-filled Syringe (PFS) vs, AVT02 Autoinjector (AI)

Phase 1

New Zealand
Diatranz Otsuka Limited

Completed

Open-label Investigation of the Safety and Effectiveness of DIABECELL(R) in Patients With Type I Diabetes Mellitus

Type 1 Diabetes

New Zealand
Christchurch Clinical Studies Trust Ltd
Medtronic

Unknown

Assessment of Two Insulin Pump Insulin Delivery Systems in Type 1 Diabetes.

Type1diabetes

New Zealand
Reneo Pharma Ltd

Terminated

An Open Label, Long Term Safety Study of REN001 in Primary Mitochondrial Myopathy Patients (Stride Ahead)

Primary Mitochondrial Myopathy

Spain, Australia, Belgium, Denmark, Canada, United Kingdom, France, Germany, Hungary, Italy, Netherlands, New Zealand
Ultragenyx Pharmaceutical Inc

Terminated

A Study to Evaluate the Safety of Aceneuramic Acid Extended Release (Ace-ER; UX001) Tablets in Glucosamine (UDP-N-acetyl)-2-Epimerase (GNE) Myopathy (GNEM) (Also Known as Hereditary Inclusion Body Myopathy [HIBM]) Patients With Severe Ambulatory Impairment

Hereditary Inclusion Body Myopathy | GNE Myopathy | Inclusion Body Myopathy 2 | Distal Myopathy With Rimmed Vacuoles | Distal Myopathy, Nonaka Type | Quadriceps Sparing Myopathy

Canada, United States, Bulgaria
Arrowhead Pharmaceuticals

Completed

Study of ARO-AAT in Normal Adult Volunteers

Alpha 1-Antitrypsin Deficiency

New Zealand
Dicerna Pharmaceuticals, Inc.

Withdrawn

An Extension Study of Belcesiran in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATLD)

Alpha 1-Antitrypsin Deficiency

New Zealand
Reneo Pharma Ltd

Completed

An Efficacy and Safety Study of 24 Week Treatment With Mavodelpar (REN001) in Primary Mitochondrial Myopathy Patients (STRIDE)

Primary Mitochondrial Myopathy

United States, Spain, Australia, France, United Kingdom, New Zealand, Denmark, Norway, Czechia, Belgium, Canada, Germany, Hungary, Italy, Netherlands
Rockefeller University

Terminated

Metabolic Features of Post-Myopathy Patients Associated With Statin Treatment

Myopathy (Statin Associated)

United States
Dicerna Pharmaceuticals, Inc., a Novo Nordisk company

Completed

Study of DCR-A1AT in Healthy Adult Volunteers

Alpha 1-Antitrypsin Deficiency

New Zealand, Sweden
Ultragenyx Pharmaceutical Inc

Terminated

Study to Evaluate the Safety and Efficacy of Aceneuramic Acid Extended-Release (Ace-ER) Tablets in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)

Hereditary Inclusion Body Myopathy | GNE Myopathy | Distal Myopathy With Rimmed Vacuoles | Distal Myopathy, Nonaka Type | Quadriceps Sparing Myopathy

United States, Canada, United Kingdom, Israel, Italy, Bulgaria, France
Ultragenyx Pharmaceutical Inc

Completed

Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Sialic Acid in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM) (GNEM)

Hereditary Inclusion Body Myopathy | GNE Myopathy | Distal Myopathy With Rimmed Vacuoles | Distal Myopathy, Nonaka Type

United States, Canada, United Kingdom, Israel, Italy, Bulgaria, France
Ultragenyx Pharmaceutical Inc

Completed

An Open Label Phase 2 Extension Study of Higher Dose Sialic Acid-Extended Release (SA-ER) Tablets and Sialic Acid-Immediate Release (SA-IR) Capsules in Patients With Glucosamine (UDP-N-acetyl)-2-Epimerase (GNE) Myopathy

GNE Myopathy | Hereditary Inclusion Body Myopathy (HIBM)

United States, Israel
Ultragenyx Pharmaceutical Inc

Completed

Safety and Pharmacokinetics of Sialic Acid Tables in Patients With Hereditary Inclusion Body Myopathy (HIBM)

Hereditary Inclusion Body Myopathy (HIBM)

United States
Ra Pharmaceuticals

Terminated

Safety and Efficacy Study of Zilucoplan in Subjects With Immune-Mediated Necrotizing Myopathy

Immune Mediated Necrotizing Myopathy

United States, Netherlands, France, United Kingdom
Astellas Pharma Inc

Active, not recruiting

A Study to Evaluate ASP0367 in Participants With Primary Mitochondrial Myopathy (MOUNTAINSIDE)

Primary Mitochondrial Myopathy

United States
Ultragenyx Pharmaceutical Inc

Completed

A Phase 2 Study to Evaluate the Dose and Pharmacodynamic Efficacy of Sialic Acid-Extended Release (SA-ER) Tablets in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy (GNE Myopathy)

Hereditary Inclusion Body Myopathy | GNE Myopathy

United States, Israel
Stealth BioTherapeutics Inc.

Terminated

A Trial to Evaluate Safety and Efficacy of Elamipretide Primary Mitochondrial Myopathy Followed by Open-Label Extension (MMPOWER-3)

Primary Mitochondrial Myopathy

United States, Germany, Canada, Italy, United Kingdom, Denmark, Hungary
University of Rochester
National Institute of Neurological Disorders and Stroke (NINDS)

Recruiting

Myotonic Dystrophy and Facioscapulohumeral Muscular Dystrophy Registry

Muscular Dystrophy | Myotonic Dystrophy Type 2 | Myotonic Dystrophy Type 1 | Myotonic Dystrophy | Facioscapulohumeral Muscular Dystrophy | Steinert's Disease | Congenital Myotonic Dystrophy | PROMM (Proximal Myotonic Myopathy) | Myotonic Muscular Dystrophy

United States
Arrowhead Pharmaceuticals

Recruiting

Study of ARO-DM1 in Subjects With Type 1 Myotonic Dystrophy

Myotonic Dystrophy 1

New Zealand, Australia
Boston Scientific Corporation

Completed

Subcutaneous Implantable Defibrillator (S-ICD) System - CE Clinical Investigation

ACC/AHA/NASPE Class 1, IIa or IIb Indications

Netherlands, Italy, United Kingdom, New Zealand
Melbourne Health

Completed

Trial of Intranasal Insulin in Children and Young Adults at Risk of Type 1 Diabetes (INITII)

Type 1 Diabetes

Australia, New Zealand
Stealth BioTherapeutics Inc.

Active, not recruiting

Study to Evaluate Efficacy and Safety of Elamipretide in Subjects With Primary Mitochondrial Disease From Nuclear DNA Mutations (nPMD) (NuPower)

Mitochondrial Diseases | Mitochondrial Myopathies | Mitochondrial Complex I Deficiency | Mitochondrial Pathology | Mitochondrial DNA Depletion | Mitochondrial DNA Mutation | Mitochondrial DNA Deletion | Mitochondrial Metabolism Defect

Spain, United States, Italy, Netherlands, Australia, Germany, Hungary, New Zealand, Norway, United Kingdom
Baxalta now part of Shire

Completed

Efficacy and Safety Study of Augmentation Therapy With ARALAST Fraction IV-1 (Human Alpha 1 - Proteinase Inhibitor)

Alpha 1-Antitrypsin Deficiency

New Zealand, Australia
National Institute of Neurological Disorders and...

Completed

Treatment of AIDS Vacuolar Myelopathy With Methionine

Myelopathy | AIDS-Myelopathy | AIDS Vacuolar Myelopathy

United States
Baxalta now part of Shire
Baxter Healthcare, Ltd. (New Zealand), Baxter Healthcare Pty. Ltd. (Australia)

Completed

Pharmacokinetic Study of ARALAST (Human Alpha1- PI)

Alpha 1-Antitrypsin Deficiency

Australia, New Zealand
Yale University

Completed

Application of Electrical Impedance Myography (EIM) as a Potential Biomarker of Idiopathic Inflammatory Myopathies

Idiopathic Inflammatory Myopathies

United States
Dyne Therapeutics

Recruiting

Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1 (ACHIEVE)

Myotonic Dystrophy Type 1 (DM1)

United Kingdom, France, Netherlands, Italy, Germany, New Zealand
argenx

Recruiting

A Study to Investigate the Efficacy and Safety of Efgartigimod PH20 SC in Adult Participants With Active Idiopathic Inflammatory Myopathy. (ALKIVIA)

Myositis | Dermatomyositis | Polymyositis | Antisynthetase Syndrome | Immune-Mediated Necrotizing Myopathy | Active Idiopathic Inflammatory Myopathy

United States, Germany, Spain, Korea, Republic of, Austria, Turkey, Lithuania, Australia, Thailand, Serbia, Poland, Italy, France, Netherlands, Argentina, United Kingdom, Georgia, Belgium, Bulgaria, Canada, Cyprus, Denmark, Greece, Hu... and more
Columbia University
Eunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaborators

Completed

Oxidative Capacity and Exercise Tolerance in Ambulatory SMA

Spinal Muscular Atrophy Type 3 | Mitochondrial Myopathy

United States
Leadiant Biosciences, Inc.
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) and other collaborators

Active, not recruiting

Multi-Center Study of ManNAc for GNE Myopathy (MAGiNE)

GNE Myopathy

United States
David M. Simpson
CSL Behring

Terminated

A Double-Blind, Placebo Controlled Study of Intravenous Immunoglobulin for HIV-Associated Myelopathy

HIV-associated Myelopathy

United States
VA Office of Research and Development
Temple University

Completed

Postural Stability in Cervical Spinal Myelopathy

Cervical Spondylotic Myelopathy

United States
Genomas, Inc
University of California, San Francisco; Hartford Hospital; Cornell University

Unknown

DNA Diagnostic System for Statin Safety and Efficacy (SIM)

Hypercholesterolemia | Myopathy

United States
University of Iowa

Active, not recruiting

Critical Illness Myopathy and Trajectory of Recovery in AKI Requiring CRRT

Acute Kidney Injury | Critical Illness Myopathy | Dialysis; Complications

United States
Inhibrx, Inc.

Completed

Phase 1 Study to Assess the Safety, PK and PD of INBRX-101 in Adults With Alpha-1 Antitrypsin Deficiency (rhAAT-Fc)

Alpha-1 Antitrypsin Deficiency | AATD

United States, New Zealand, United Kingdom
Trek Therapeutics, PBC

Completed

A Study of Faldaprevir, TD-6450 and Other Antivirals in Participants With Genotype 1b Hepatitis C Virus Infection

Chronic Hepatitis C | Hepatitis C Genotype 1 | Hepatitis C (HCV) | Hepatitis C Viral Infection

United States, New Zealand
University of Sydney
National Health and Medical Research Council, Australia; Mylan Pharmaceuticals... and other collaborators

Recruiting

The Fenofibrate And Microvascular Events in Type 1 Diabetes Eye. (FAME 1 EYE)

Diabetic Retinopathy | Diabetic Nephropathies | Type 1 Diabetes Mellitus

Australia, Hong Kong, New Zealand
Murdoch Childrens Research Institute
Royal Children's Hospital

Completed

What is the Incidence of an Immune Disorder in Children With Invasive Pneumococcal Disease (IPD)?

Primary Immunodeficiency | Invasive Pneumococcal Disease, Protection Against | Invasive Pneumococcal Disease, Recurrent Isolated, 1 | Invasive Pneumococcal Disease, Recurrent Isolated, 2

Australia, New Zealand
Reproductive Medicine Associates of New Jersey

Completed

Evaluation of the Impact of Embryo Biopsy on the Reproductive Potential of Embryos Undergoing In Vitro Fertilization (IVF)

Each Patient is Both Case and Control | 1 Embryo Will be Biopsied and 1 Embryo is Not

United States
Newcastle-upon-Tyne Hospitals NHS Trust
Jain Foundation

Active, not recruiting

Clinical Outcome Study for Dysferlinopathy (Jain COS)

Dysferlinopathy | LGMD2B Now Classified as LGMDR2 | Miyoshi Myopathy

Korea, Republic of, Chile, Spain, United States, Denmark, France, Italy, Japan, United Kingdom
Bristol-Myers Squibb

Completed

Trial to Evaluate the Efficacy and Safety of Abatacept in Combination With Standard Therapy Compared to Standard Therapy Alone in Improving Disease Activity in Adults With Active Idiopathic Inflammatory Myopathy

Dermatomyositis | Polymyositis | Autoimmune Necrotizing Myopathy | Overlap Myositis | Juvenile Myositis Above the Age of 18

France, United States, Australia, Brazil, Czechia, Germany, Italy, Japan, Korea, Republic of, Mexico, Sweden
NYU Silver School of Social Work

Completed

Mobile Health Solutions for Behavioral Skill Implementation Through Homework (MHealth)

Mental Health Wellness 1

United States
International Rescue Committee
Johns Hopkins University; World Health Organization

Completed

Early Adolescent Skills for Emotions Tanzania Pilot

Mental Health Wellness 1

United States
Greenwich Hospital
Yale University

Completed

The CSM Trial: A Multicenter Study Comparing Ventral to Dorsal Surgery for Cervical Spondylotic Myelopathy (CSM)

Cervical Spondylotic Myelopathy

United States
Stony Brook University

Completed

Trial With Deltex Cardio in Elective Bowel Resection Patients

1 Fluid Management

United States
Juno Therapeutics, Inc., a Bristol-Myers Squibb...
Bristol-Myers Squibb Services Unlimited Company

Recruiting

A Study of CC-97540, CD-19-Targeted Nex-T CAR T Cells, in Participants With Severe, Refractory Autoimmune Diseases

Systemic Lupus Erythematosus | Systemic Sclerosis | Idiopathic Inflammatory Myopathy

United States, France, Germany, Italy, Belgium, Spain
Yale University

Completed

Yale Wellness Project

Mental Health Wellness 1

United States
CorStone
David and Lucile Packard Foundation

Active, not recruiting

Youth First: A Longitudinal Impact Evaluation

Mental Health Wellness 1 | Resilience, Psychological

India
Virginia Commonwealth University
Radboud University Medical Center; University of California, Los Angeles; University... and other collaborators

Recruiting

Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1)

Myotonic Dystrophy 1

United States, Netherlands, United Kingdom, New Zealand, Germany, Italy, Canada, France

Clinical Trials on RYR-1 Myopathy in New Zealand

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