Clinical Trials on Pompe Disease (Late-onset) in Australia

Total 40 results

Amicus Therapeutics

Recruiting

ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD

Pompe Disease (Late-onset)

United States, Taiwan, Japan, Australia, Canada
BioMarin Pharmaceutical

Terminated

A Prospective, Noninterventional, Observational Study of Late-Onset Pompe Disease

Late-onset Pompe Patients Untreated or Treated With rhGAA

Spain, Slovenia, Serbia, Korea, Republic of, Greece, Australia, Singapore, Brazil, Canada, Taiwan, Ireland, Poland, Romania
Amicus Therapeutics

Active, not recruiting

A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With LOPD

Pompe Disease (Late-onset)

United States, Germany, Taiwan, Belgium, Australia, Slovenia, Spain, Netherlands, Hungary, Japan, Austria, United Kingdom, France, Argentina, Canada, Sweden, Denmark, New Zealand, Greece, Korea, Republic of, Bosnia and Herzegovina, Ita... and more
Amicus Therapeutics

Completed

A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease (PROPEL)

Pompe Disease (Late-onset)

United States, Australia, Korea, Republic of, Japan, Taiwan, Belgium, Slovenia, New Zealand, United Kingdom, France, Argentina, Canada, Spain, Bosnia and Herzegovina, Germany, Sweden, Hungary, Austria, Bulgaria, Denmark, Greece, I... and more
Amicus Therapeutics

Terminated

STRIDE Study - A Study in Subjects With LOPD Who Are Currently Being Treated With ERT

Late-onset Pompe Disease

United States, Canada, Australia, Belgium
Genzyme, a Sanofi Company

Completed

Late-Onset Treatment Study Extension Protocol

Glycogen Storage Disease Type II (GSD-II) | Pompe Disease (Late-Onset) | Glycogenesis Type II | Acid Maltase Deficiency (AMD)

United States, France, Canada, Netherlands, Australia
Ataxia Study Group
German Center for Neurodegenerative Diseases (DZNE)

Recruiting

Sporadic Degenerative Ataxia With Adult Onset: Natural History Study (SPORTAX-NHS)

Late Onset Sporadic Cerebellar Ataxia

Norway, Austria, Germany, Netherlands, Italy
Syneos Health
AprilBio Co., Ltd.

Completed

Assessment of Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of APB-R3

Still's Disease, Adult-Onset

Australia
Noema Pharma AG

Completed

A 10-week Efficacy Study of NOE-105 in Childhood Onset Fluency Disorder (Orpheus)

Childhood-Onset Fluency Disorder

United States, Australia
Neurocrine Biosciences

Terminated

Extension Study to Evaluate the Safety and Tolerability of NBI-921352 When Used With Anti-seizure Medications in Adults With Focal Onset Seizures

Focal Onset Seizure | Focal Onset Epilepsy

Spain, Australia, Belgium, Czechia, France, Hungary, Italy
Neurocrine Biosciences

Completed

A Study to Investigate How Effective, Safe and Tolerable the Drug NBI-921352 is When Used With Anti-seizure Medications in Adults With Focal Onset Seizures

Focal Onset Seizure | Focal Onset Epilepsy

Spain, Belgium, Czechia, France, Italy, Australia, Hungary, United Kingdom
Centrexion Therapeutics

Terminated

Study to Evaluate the Safety and Pharmacokinetics of Topically Applied 40% Lidocaine Gel Compared With Placebo in Subjects With Acute Herpes Zoster (Shingles) Pain

Acute-onset Herpes Zoster Pain

Australia
Medical University of Vienna

Terminated

Improving Secretion of Insulin in New Onset Diabetes After Renal Transplantation (ISINODAT)

New Onset Diabetes Mellitus After Renal Transplantation

Austria
Medical University of Graz

Completed

Magnetic Resonance Imaging in the Diagnosis of Pulmonary Hypertension

Pulmonary Hypertension | Ventricular Function | MR Phase Contrast Velocity Imaging | Late Enhancement Patterns

Austria
BioMarin Pharmaceutical

Terminated

Extension Study for Patients Who Have Participated in a BMN 701 Study

Pompe Disease

United States, United Kingdom, Australia, France, Germany, New Zealand
Marinus Pharmaceuticals

Completed

Phase 3 Study of Adjunctive Ganaxolone in Adults With Drug-resistant Partial Onset Seizures and Open-label Extension

Drug Resistant Partial Onset Seizure

United States, Australia, Russian Federation, Bulgaria, Germany, Poland
Medical University of Vienna

Recruiting

Novel Metabolic Muscular Biomarkers in Pompe Disease - a Non-invasive Magnetic Resonance Exploratory Pilot Study. (POMPE)

Pompe Disease | McArdle Disease

Austria
Novartis

Withdrawn

Evaluate the Efficacy of BGG492 as Adjunctive Treatment in Patients With Refractory Partial Onset Seizures

Partial Onset Seizures

United States, Austria, Spain, Estonia, Bulgaria, Latvia, Canada, India, Belgium, Lithuania, Romania
Eisai Inc.

Completed

A Post-marketing Observational Study Of The Use Of Zonisamide (ZNS) in the Adjunctive Treatment Of Adult Patients With Partial Onset Seizures (Study E2090-E044-410) (ZOOM)

Partial Onset Seizures

Germany, Sweden, Norway, Austria, Denmark
Xenon Pharmaceuticals Inc.
Worldwide Clinical Trials

Recruiting

A Randomized Study of XEN1101 Versus Placebo in Focal-Onset Seizures (X-TOLE3)

Focal Onset Seizures

United States, Spain, Italy, Croatia, Portugal, Poland, Chile, Argentina, Austria, Czechia
Eisai Inc.

No longer available

An Extended Access Program for Perampanel

Partial Onset Seizures

Italy, Spain, Belgium, Israel, Poland, Taiwan, Czechia, Finland, Thailand, Malaysia, Argentina, Chile, Canada, Hungary, Australia, Lithuania, Latvia, Estonia, Netherlands, Greece, Hong Kong
Xenon Pharmaceuticals Inc.
Worldwide Clinical Trials

Recruiting

A Randomized Study of XEN1101 Versus Placebo in Focal-Onset Seizures (X-TOLE2)

Focal Onset Seizures

United States, Spain, Australia, Poland, Canada, Bulgaria, Italy, United Kingdom
Amicus Therapeutics

Active, not recruiting

First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221

Pompe Disease

United States, Netherlands, United Kingdom, Australia, Germany, New Zealand
Eisai Co., Ltd.

Completed

A Study With an Open-label Extension Phase to Evaluate the Efficacy and Safety of Perampanel (E2007) Administered as an Adjunctive Therapy in Subjects With Refractory Partial-onset Seizures

Partial-onset Seizures

Korea, Republic of, Japan, Australia, China, Malaysia, Taiwan, Thailand
Medical University of Vienna
Vienna University of Technology

Completed

Perioperative Heart Rate Variability Analysis in Realtime (PHERVR)

Individuality | Perioperative/Postoperative Complications | Surgical Complications From General Anesthesia | Heart Rate and Rhythm Disorders

Austria
BioMarin Pharmaceutical

Completed

Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe Disease

Pompe Disease

United States, United Kingdom, France, Australia, Germany
Atrial Fibrillation Network
Daiichi Sankyo Europe, GmbH, a Daiichi Sankyo Company; Deutsches Zentrum für...

Terminated

Non-vitamin K Antagonist Oral Anticoagulants in Patients With Atrial High Rate Episodes (NOAH)

Atrial High Rate Episodes

Austria, Belgium, Bulgaria, Czechia, France, Germany, Greece, Hungary, Italy, Netherlands, Poland, Portugal, Romania, Spain, Sweden, Ukraine, United Kingdom, Denmark
Idorsia Pharmaceuticals Ltd.

Completed

Tezosentan in the Treatment of Acute Heart Failure (VERITAS 2)

Acute Heart Failure | Acute Decompensation of Chronic Heart Failure | New Onset of Heart Failure

United States, Hungary, Australia, Czechia, Germany, Italy, Norway, United Kingdom
Idorsia Pharmaceuticals Ltd.

Completed

Tezosentan in Acute Heart Failure (VERITAS 1)

Acute Heart Failure | Acute Decompensation of Chronic Heart Failure | New Onset of Heart Failure

United States, United Kingdom, Israel, Switzerland, Austria, Denmark, France, Germany, Greece, Poland
Genzyme, a Sanofi Company

Completed

High Dose or High Dose Frequency Study of Alglucosidase Alfa

Glycogen Storage Disease Type II (GSD-II) | Pompe Disease | Glycogenesis 2 Acid Maltase Deficiency

United States, Canada, Australia
Bristol-Myers Squibb

Recruiting

Study to Evaluate the Efficacy, Safety, and Tolerability of an Anti-MTBR Tau Monoclonal Antibody (BMS-986446) in Participants With Early Alzheimer's Disease (TargetTau-1)

Alzheimer Disease, Early Onset

United States, Australia, Belgium, Canada, Denmark, France, Japan, Korea, Republic of, Netherlands, Poland, Singapore, Spain, Sweden, United Kingdom
National Institute of Allergy and Infectious Diseases...
PPD; Rho Federal Systems Division, Inc.; Immune Tolerance Network (ITN)

Completed

Tocilizumab (TCZ) in New-onset Type 1 Diabetes (EXTEND)

Type 1 Diabetes Mellitus | T1DM | T1D | New-onset Type 1 Diabetes Mellitus

United States, Australia
UCB Biopharma S.P.R.L.

Completed

Study to Test the Efficacy and Safety of Padsevonil as Adjunctive Treatment of Focal-onset Seizures in Adults With Drug-resistant Epilepsy (ARISE)

Focal-Onset Seizures | Drug-resistant Epilepsy

United States, Australia, Belgium, Bulgaria, Canada, Czechia, France, Germany, Hungary, Italy, Japan, Lithuania, Mexico, Poland, Portugal, Slovakia, Spain, Turkey, United Kingdom
UCB Biopharma SRL

Terminated

A Study to Test the Efficacy and Safety of Padsevonil as Treatment of Focal-onset Seizures in Adult Subjects With Drug-resistant Epilepsy (DUET)

Drug-Resistant Epilepsy | Focal-Onset Seizures

United States, Australia, Belgium, Bosnia and Herzegovina, Bulgaria, Croatia, Czechia, Denmark, Estonia, Finland, France, Germany, Greece, Hungary, Ireland, Italy, Japan, Norway, Poland, Portugal, Romania, Serbia, Slovakia, Spain, Sweden, Switzerlan... and more
UCB Biopharma SRL

Terminated

Study to Test the Safety and Efficacy of Padsevonil as Adjunctive Treatment of Focal-onset Seizures in Adult Subjects With Drug-resistant Epilepsy

Drug-Resistant Epilepsy | Focal-Onset Seizures

United States, Australia, Belgium, Bosnia and Herzegovina, Bulgaria, Canada, Croatia, Czechia, Denmark, Estonia, Finland, France, Germany, Greece, Hungary, Ireland, Italy, Japan, Lithuania, Mexico, Poland, Romania, Serbia, Slovakia, Spain, Turk... and more
Genzyme, a Sanofi Company

Completed

Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies Avalglucosidase Alfa and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease (COMET)

Glycogen Storage Disease Type II;Pompe's Disease

Czechia, Taiwan, Belgium, Germany, Korea, Republic of, United States, Argentina, Australia, Austria, Brazil, Canada, Denmark, France, Hungary, Italy, Japan, Mexico, Netherlands, Poland, Portugal, Russian Federation, Spain, Switzerland, Turk... and more
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
Genzyme, a Sanofi Company

Recruiting

Pompe Disease Registry Protocol

Glycogen Storage Disease Type II | Pompe Disease

Hungary, Croatia, Serbia, United States, Argentina, Australia, Belgium, Brazil, Bulgaria, Canada, Chile, China, Colombia, Denmark, France, Germany, Greece, Hong Kong, India, Indonesia, Israel, Italy, Japan, Jordan, Korea, Republic of, Kuwait and more
Janssen Research & Development, LLC

Completed

A Study to Characterize the Clinical Course of Pregnant Women and Children at High Risk for Early Onset Severe Hemolytic Disease of the Fetus and Newborn

Erythroblastosis, Fetal | Early Onset Severe Hemolytic Disease of the Fetus and Newborn (EOS-HDFN)

United States, Canada, Netherlands, Belgium, Australia, Germany, Spain, Sweden, United Kingdom
Novartis Pharmaceuticals

Completed

Clinical Outcomes and Safety: A Registry Study of Ilaris (Canakinumab) Patients (B-Confident)

Cryopyrin-associated Periodic Syndromes (CAPS) | Familial Cold Autoinflam Syn (FCAS) | Muckle-wells Syn (MWS) | Neonatal Onset Multisystem Inflam Disease (NOMID)

Switzerland, United States, Germany, Norway, Austria

Clinical Trials on Pompe Disease (Late-onset) in Australia

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Mauritania

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

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