Clinical Trials on Type 3 Spinal Muscular Atrophy in Australia

Total 421 results

Novartis Gene Therapies

Active, not recruiting

Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi

SMA | Spinal Muscular Atrophy Type II | Spinal Muscular Atrophy Type I | Spinal Muscular Atrophy Type III

United States, Belgium, France, Japan, United Kingdom, Italy, Taiwan, Australia, Canada
Hoffmann-La Roche

Active, not recruiting

A Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy (Rainbowfish)

Muscular Atrophy, Spinal

United States, Belgium, Brazil, Russian Federation, Australia, Taiwan, Poland
Hoffmann-La Roche

Recruiting

A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophy (MANATEE)

Spinal Muscular Atrophy (SMA)

Belgium, United States, Croatia, Japan, Netherlands, Spain, Canada, Poland, United Kingdom, Italy, Portugal, Australia
Novartis Pharmaceuticals

Recruiting

Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials (SPECTRUM)

Spinal Muscular Atrophy (SMA)

Spain, Canada, Taiwan, Belgium, Japan, Italy, Singapore, United Kingdom, France, Australia, Denmark, United States
Synchron Medical, Inc.

Withdrawn

SWITCH II Early Feasibility Study: Implantable BCI to Control a Digital Device for People With Paralysis

Paralysis | Amyotrophic Lateral Sclerosis | Muscular Dystrophies | Neurologic Disorder | Spinal Muscular Atrophy | Spinal Cord Injury | Stroke, Lacunar | Upper Limb Injury | Paralysis; Stroke | Stroke, Brainstem

Australia
Novartis Gene Therapies
PRA Health Sciences

Completed

Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2 (SPR1NT)

Spinal Muscular Atrophy

United States, United Kingdom, Australia, Belgium, Canada, Japan
Biogen

Active, not recruiting

A Study of Multiple Doses of Nusinersen (ISIS 396443) Delivered to Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy (NURTURE)

Spinal Muscular Atrophy

United States, Taiwan, Israel, Australia, Qatar, United Kingdom, Turkey, Italy, Germany, Argentina, 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
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
Novartis Pharmaceuticals

Completed

Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) (SMART)

Spinal Muscular Atrophy

Taiwan, Belgium, Canada, United States, Australia, France, Italy, Portugal, United Kingdom
Novartis Pharmaceuticals

Active, not recruiting

Phase IIIb, Open-label, Multi-center Study to Evaluate Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally to Participants With SMA Who Discontinued Treatment With Nusinersen or Risdiplam (STRENGTH)

Spinal Muscular Atrophy

Japan, Belgium, Spain, Canada, United States, Australia, Italy, France, Netherlands
Biogen

Active, not recruiting

Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy (DEVOTE)

Muscular Atrophy, Spinal

China, United States, Netherlands, Spain, Korea, Republic of, Taiwan, Australia, Canada, Chile, Saudi Arabia, Poland, Israel, Brazil, Russian Federation, Hungary, Japan, United Kingdom, Italy, Germany, Estonia, Greece, Mexico, Colombia and more
Center for Eye Research Australia
AlphaRET Pty Ltd.

Recruiting

Subthreshold Laser Treatment in Intermediate Age-related Macular Degeneration With Nascent Geographic Atrophy Study (LIANA)

Age-Related Macular Degeneration

Australia
Center for Eye Research Australia

Recruiting

Project AMD: Comprehensive Characterisation of Age-Related Macular Degeneration and Its Progression (Project AMD)

Age-related Macular Degeneration | Geographic Atrophy | Age Related Macular Degeneration | AMD

Australia
PYC Therapeutics

Not yet recruiting

Natural History of Autosomal Dominant Optic Atrophy (ADOA), Caused by OPA1 Mutation

Optic Atrophies, Hereditary | Autosomal Dominant Optic Atrophy | Optic Atrophy, Autosomal Dominant | Kjer Optic Atrophy

Australia
Medical University of Vienna
Department of Surgery, Social Medical Centre South, Vienna, Austria; Institute...

Completed

Randomized, Controlled Study to Investigate the Effect of Neuromuscular Electrical Stimulation (NMES) on Muscle Metabolism of Abdominal Surgical Patients

Muscular Atrophy

Austria
Novartis Pharmaceuticals

Completed

A Comparison of Ranibizumab and Aflibercept for the Development of Geographic Atrophy in (Wet) AMD Patients (RIVAL)

Age-Related Macular Degeneration

Australia
Allievex Corporation

Completed

A Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)

Mucopolysaccharidosis Type IIIB | Mucopolysaccharidosis Type 3 B | MPS III B | MPS 3 B

United States, Spain, Turkey, Taiwan, Australia, Colombia, Germany, United Kingdom
ONL Therapeutics

Completed

ONL1204 Ophthalmic Solution in Patients With Geographic Atrophy Associated With Age-related Macular Degeneration

Geographic Atrophy

New Zealand, Australia
Medical University of Graz

Recruiting

Microbiome and Sarcopenia in Patients With Liver Cirrhosis

Liver Cirrhosis | Sarcopenia

Austria
The Alfred

Completed

Acute Skeletal Muscle Wasting and Relation to Physical Function in Patients Requiring ECMO

Critical Illness | Extracorporeal Membrane Oxygenation | Muscle Wasting

Australia
Belite Bio, Inc

Recruiting

Phase 3, Randomized, Placebo-Controlled Study of Tinlarebant to Explore Safety and Efficacy in Geographic Atrophy (PHOENIX)

Geographic Atrophy

United States, Taiwan, Australia, China, United Kingdom
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
Allergan

Completed

Safety and Efficacy of Brimonidine Intravitreal Implant in Patients With Geographic Atrophy Due to Age-related Macular Degeneration (AMD)

Macular Degeneration

Korea, Republic of, Australia, Portugal, Germany, United States, Philippines, Italy
Christian Müller, MD
University Hospital Inselspital, Berne; Medical University Innsbruck; University... and other collaborators

Recruiting

Reliability of Cardiac Troponins for the Diagnosis of Myocardial Infarction in the Presence of Skeletal Muscle Disease (H&M)

Muscle Weakness | Muscle Atrophy | Muscle Spasticity | Muscle Cramp | Muscle Soreness | Muscle Damage | Myopathy | Muscle Injury

Switzerland, Austria
Murdoch Childrens Research Institute

Completed

Effect of Creatine Monohydrate on Functional Muscle Strength in Children With FSHD

Facio-Scapulo-Humeral Dystrophy | FSHD2 | FSHD1

Australia
Boston Image Reading Center

Active, not recruiting

Non Exudative AMD Imaged With SS-OCT (BIRC-01)

Dry Macular Degeneration

United States, Australia
Hugo W. Moser Research Institute at Kennedy Krieger...
Concord Hospital; FSHD Global Research Foundation

Completed

Bone Health in Facioscapulohumeral Muscular Dystrophy (FSHD)

Facioscapulohumeral Muscular Dystrophy

United States, Australia
Hoffmann-La Roche

Terminated

A Study of Disease Progression in Participants With Geographic Atrophy Secondary to Age-Related Macular Degeneration

Geographic Atrophy

Canada, United Kingdom, United States, Argentina, Australia, France, Germany, Hungary, Italy, Poland, Spain, Brazil
Boston Image Reading Center

Recruiting

Non Exudative AMD Imaged With SS-OCT- Extension (BIRC-02)

Dry Macular Degeneration

United States, Australia
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
Australian Catholic University
Curtin University; Pendulum Therapeutics

Active, not recruiting

OsteoPreP: Food Supplements for Postmenopausal Bone Health (OsteoPreP)

Glucose Metabolism Disorders | Postmenopausal Osteopenia | Age-related Cognitive Decline | Age-Related Sarcopenia | Bone Loss, Age Related

Australia
Hoffmann-La Roche

Terminated

A Study of Disease Progression in Participants With Geographic Atrophy Secondary to Age-Related Macular Degeneration (Proxima A)

Geographic Atrophy

United States, Israel, United Kingdom, Hungary, Germany, Argentina, Canada, France, Spain, Australia, Brazil, Italy, Austria, Poland, Greece
Annexon, Inc.

Active, not recruiting

A Study Investigating the Efficacy and Safety of Intravitreal Injections of ANX007 in Patients With Geographic Atrophy (ARCHER)

Geographic Atrophy

United States, Australia, New Zealand
Eli Lilly and Company

Completed

A Study of LY2495655 in Older Participants Undergoing Elective Total Hip Replacement

Muscular Atrophy

Spain, United States, Belgium, Canada, Austria, Sweden, Japan, Finland, Denmark, Estonia, France
Pfizer

Active, not recruiting

Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy

Muscular Dystrophy, Duchenne

United States, Australia
Sanford Health
National Ataxia Foundation; Beyond Batten Disease Foundation; Pitt Hopkins Research... and other collaborators

Recruiting

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford (CoRDS)

Mitochondrial Diseases | Retinitis Pigmentosa | Myasthenia Gravis | Eosinophilic Gastroenteritis | Multiple System Atrophy | Leiomyosarcoma | Leukodystrophy | Anal Fistula | Spinocerebellar Ataxia Type 3 | Friedreich Ataxia | Kennedy Disease | Lyme Disease | Hemophagocytic Lymphohistiocytosis | Spinocerebellar Ataxia... and other conditions

United States, Australia
Apellis Pharmaceuticals, Inc.

Completed

A Study to Compare the Efficacy and Safety of Intravitreal APL-2 Therapy With Sham Injections in Patients With Geographic Atrophy (GA) Secondary to Age-Related Macular Degeneration

Geographic Atrophy

United States, France, Italy, Netherlands, Israel, Germany, United Kingdom, Spain, Australia, New Zealand, Poland, Brazil, Canada, Czechia
Hoffmann-La Roche

Terminated

A Study Investigating the Safety and Efficacy of Lampalizumab Intravitreal Injections in Participants With Geographic Atrophy Secondary to Age-Related Macular Degeneration (SPECTRI)

Geographic Atrophy

United States, United Kingdom, Netherlands, Germany, Belgium, Poland, Spain, Argentina, Australia, Hungary, Italy, Portugal, France, Turkey, Austria, Russian Federation, Mexico, Peru, Denmark, Slovakia, Sweden, Switzerland
Apellis Pharmaceuticals, Inc.

Completed

Study of Pegcetacoplan (APL-2) Therapy in Patients With Geographic Atrophy (FILLY)

Geographic Atrophy

United States, Australia, New Zealand
Royal Brisbane and Women's Hospital

Completed

Efficacy of Therapeutic Interventions for Orthostatic Hypotension in Parkinson's Disease and Multiple System Atrophy

Parkinson's Disease | Multiple System Atrophy | Orthostatic Hypotension

Australia
Novartis Pharmaceuticals

Completed

Dose Range Finding Study of Bimagrumab in Sarcopenia

Sarcopenia

Belgium, United States, Australia, Germany, Spain, Taiwan, France, Korea, Republic of, Japan, Russian Federation, Denmark, Czechia, Switzerland
MedImmune LLC

Completed

A Study to Evaluate the Safety, Tolerability, Immunogenicity and Vaccine-like Viral Shedding of MEDI-534, Against Respiratory Syncytial Virus (RSV) and Parainfluenza Virus Type 3 (PIV3), in Healthy 6 to <24 Month-old Children and in 2 Month-old Infants

Healthy

United States, Australia, Spain, Germany, Canada, Finland, South Africa, Brazil, Israel
Astellas Pharma Global Development, Inc.

No longer available

Early Access Program (EAP) of Gilteritinib (ASP2215) in Patients With FMS-like Tyrosine Kinase 3 (FLT3) Mutated Relapsed or Refractory Acute Myeloid Leukemia (AML) or With FLT3-Mutated AML in Complete Remission (CR) With Minimal Residual Disease (MRD)

Acute Myeloid Leukemia (AML) | FMS-like Tyrosine Kinase-3 (FLT3) Mutations

Australia, Italy, United Kingdom
ML Bio Solutions, Inc.

Recruiting

Study to Evaluate the Efficacy and Safety of BBP-418 (Ribitol) in Patients With Limb Girdle Muscular Dystrophy 2I (LGMD2I) (Fortify)

Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I)

United States, Netherlands, United Kingdom, Denmark, Australia, Italy, Norway
Arrowhead Pharmaceuticals

Recruiting

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

Myotonic Dystrophy 1

New Zealand, Australia
Novartis Pharmaceuticals

Completed

A 24-week Off-drug Extension Study in Sarcopenic Elderly Who Completed Treatment in the 6-month Core Study

Sarcopenia

Taiwan, United States, Belgium, Russian Federation, Korea, Republic of, Japan, France, Denmark, Australia, Czechia, Spain, Switzerland
Hospital Rudolfstiftung
Oesterreichische Muskelforschung

Completed

Myocardial Involvement in Carriers of Duchenne Muscular Dystrophy: An MRI-study

Carrier of Duchenne Muscular Dystrophy

Austria
Seelos Therapeutics, Inc.

Terminated

STRIDES - a Clinical Research Study of an Investigational New Drug to Treat Spinocerebellar Ataxia (STRIDES)

Spinocerebellar Ataxia Type 3

United States, Spain, Korea, Republic of, Australia, United Kingdom, Brazil, Germany, Portugal
Baxalta now part of Shire

Completed

Pharmacokinetic, Safety and Tolerability Study of Recombinant Von Willebrand Factor / Recombinant Factor VIII Complex in Type 3 Von Willebrand Disease

Von Willebrand Disease

United States, Germany, United Kingdom, Italy, Austria, Canada

Clinical Trials on Type 3 Spinal Muscular Atrophy in Australia

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Mauritania

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

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