Clinical Trials on Glucosylceramide Beta-Glucosidase Deficiency Disease in Australia

Total 302 results

Genzyme, a Sanofi Company

Recruiting

International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry

Gaucher Disease | Cerebroside Lipidosis Syndrome | Glucocerebrosidase Deficiency Disease | Glucosylceramide Beta-Glucosidase Deficiency Disease

Hungary, Croatia, United States, Australia, Belgium, Brazil, Bulgaria, Canada, Denmark, Germany, Greece, Hong Kong, India, Indonesia, Italy, Jordan, Korea, Republic of, Kuwait, Lebanon, Malaysia, Pakistan, Philippines, Poland, Saudi Arabia and more
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
Celgene

Completed

Study to Evaluate Additional Risk Minimisation Measures (aRMMs) for REBLOZYL Among Healthcare Professionals (HCPs)

Myelodysplastic Syndromes | Beta-thalassemia

Austria
FerroKin BioSciences, Inc.

Completed

Safety and Pharmacokinetic Study of Escalating Multiple Doses of an Iron Chelator in Patients With Iron Overload

Beta-thalassemia | Transfusional Iron Overload

United States, Australia, Thailand
bluebird bio

Active, not recruiting

Long-term Follow-up of Subjects With Transfusion-Dependent β-Thalassemia (TDT) Treated With Ex Vivo Gene Therapy

Transfusion-dependent Beta-Thalassemia

United States, France, United Kingdom, Australia, Thailand, Italy, Germany, Greece
CSL Limited

Completed

Ig NextGen 10% in Patients With Primary Immune Deficiency (PID)

Primary Immune Deficiency (PID)

Australia
Celgene
Acceleron Pharma Inc. (a wholly owned subsidiary of Merck Sharp and Dohme...

Completed

An Efficacy and Safety Study of Luspatercept (ACE-536) Versus Placebo in Adults Who Require Regular Red Blood Cell Transfusions Due to Beta (β) Thalassemia (BELIEVE)

Beta-Thalassemia | Erythrocyte Transfusion

United States, Israel, Australia, Turkey, Taiwan, Malaysia, United Kingdom, Canada, Thailand, France, Lebanon, Italy, Bulgaria, Greece, Tunisia
La Jolla Pharmaceutical Company

Terminated

A Study With LJPC-401 for the Treatment of Myocardial Iron Overload in Patients With Transfusion-Dependent Beta Thalassemia

Beta-Thalassemia

Australia, Thailand, United States, Greece, Italy, Lebanon, Turkey, United Kingdom
Nemysis Ltd
RDC Clinical Pty Ltd

Recruiting

Safety and Effectiveness of Iron Hydroxide Adipate Tartrate (IHAT) in Iron Deficient Premenopausal Women

Iron-deficiency

Australia
The Queen Elizabeth Hospital

Completed

Safety of Continuous Potassium Chloride Infusion in Critical Care (ASPIC)

Arrhythmias, Cardiac | Hypokalemia

Australia
Keros Therapeutics, Inc.

Recruiting

A Study to Evaluate the Safety and Preliminary Efficacy of a Response-guided Dose Titration of KER-047 in the Treatment of Functional IDA (Iron Deficiency Anemia).

Iron Deficiency Anemia

Australia, Israel, Romania
CSL Behring

Recruiting

First-In-Human Study To Evaluate The Safety, Tolerability, Pharmacokinetics, And Pharmacodynamics Of Escalating Single And Multiple Doses Of CSL040 In Healthy Subjects

Complement Deficiencies

Australia
Life Molecular Imaging SA

Completed

Florbetaben (BAY94-9172) PET (Positron Emission Tomography) Imaging in MCI (Mild Cognitive Impairment) Patients

Alzheimer Disease | Amyloid Beta-Protein

Australia
Aeglea Biotherapeutics

Terminated

A Multiple Ascending Dose Study of ACN00177 (Pegtarviliase) in Subjects With CBS Deficiency

Homocystinuria Due to Cystathionine Beta-Synthase Deficiency

United States, United Kingdom, Australia
RDC Clinical Pty Ltd

Not yet recruiting

Safety and Effectiveness of IHAT in Iron Deficient Pre-menopausal Woman

Iron-deficiency

Australia
Medical University of Graz

Completed

Natural Versus Synthetic Vitamin B Complexes in Human

Healthy | Oxidative Stress | Vitamin B 12 Deficiency | Polyphenols | Thiamine and Niacin Deficiency States | Pyridoxine Deficiency | Folic Acid Deficiency Anemia, Dietary | Peroxidase; Defect | Homocystine; Metabolic Disorder

Austria
Medical University of Vienna

Completed

Homocysteine After Laparoscopic Roux-enY Gastric Bypass

Obesity, Morbid | Bariatric Surgery Candidate | Vitamin B 12 Deficiency | Folate Deficiency | Homocystine; Metabolic Disorder

Austria
University Children's Hospital, Zurich

Active, not recruiting

Cobalamin Supply and Metabolism in Healthy Children From Birth to the Age of 12 Months and in Their Mothers (Cbl_Neo) (Cbl_Neo)

Vitamin B 12 Deficiency

Austria, Germany
CSL Limited

Completed

Safety Study of Subcutaneous Ig NextGen 16% in Patients With Primary Immunodeficiency

Primary Immune Deficiency

Australia, New Zealand
South Australian Health and Medical Research Institute
Flinders University

Completed

Do Iron Supplements Impact the Gut Microbiome of Women of Reproductive Age?

Iron-deficiency | Anemia | Microbial Colonization

Australia
Orphatech Pharmaceuticals, GmbH

Withdrawn

Study of cPMP (Precusor Z) to Treat Molybdenum Cofactor Deficiency (MoCD) Type A

Molybdenum Cofactor Deficiency Type A

Australia
Kirby Institute
Bristol-Myers Squibb; GlaxoSmithKline; Merck Sharp & Dohme LLC; Roche Pharma AG; Boehringer... and other collaborators

Completed

Can Resistance Enhance Selection of Treatment? (CREST)

HIV Infection

Australia
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
Menzies Institute for Medical Research
Royal Hobart Hospital Research Foundation (funding source)

Completed

Cholecalciferol Intervention to Prevent Respiratory Infections Study (CIPRIS)

Vitamin D Deficiency | Respiratory Tract Infection

Australia
CSL Limited

Completed

Subcutaneous Ig NextGen 16% in PID Patients

Primary Immunodeficiency (PID)

Australia, New Zealand
ADARx Pharmaceuticals, Inc.

Recruiting

Safety, Tolerability, PK, PD of ADX-324 in Healthy Volunteers and Hereditary Angioedema Patients

Hereditary Angioedema

Australia
Medical University of Graz
European Union

Completed

Vitamin D and Mortality: an Individual Participant Data Meta-analysis of Standardized 25-hydroxyvitamin D

Vitamin D Deficiency

Austria
Vifor Pharma
Parexel; ClinStar, LLC

Completed

FERINJECT for Correction of Anaemia in IBD Patients, FER-IBD-COR (FER-IBD-COR)

Anemia | Crohn's Disease | Ulcerative Colitis | Iron Deficiency | Inflammatory Bowel Disease | Iron-Deficiency Anemia

Austria
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
Medical University of Graz
Johannes Kepler University of Linz; Fresenius Kabi Austria GmbH, Graz

Completed

Sucrosomal Iron Supplementation in Blood Donors

Iron-deficiency

Austria
Lumos Pharma

Enrolling by invitation

A Long-Term Safety Trial of LUM-201 in Children With Idiopathic Growth Hormone Deficiency Who Have Previously Completed a LUM-201 Clinical Trial (OraGrowtH211) (OraGrowtH211)

Growth Hormone Deficiency

United States, Australia
Medical University of Vienna

Completed

Preoperative Parenteral Thiamine Supplementation in Patients Undergoing Heart Surgery

Heart Failure | Acidosis, Lactic | Thiamine Deficiency

Austria
Merck KGaA, Darmstadt, Germany

Completed

An Open-label Phase 4 Study to Explore Immunogenicity of the Liquid Formulation of Saizen® in Subjects With Adult Growth Hormone Deficiency (AGHD)

Adult Growth Hormone Deficiency

United Kingdom, Germany, Australia, Sweden
Chelsea Therapeutics
Chiltern International Inc.

Completed

Clinical Study of Droxidopa in Patients With Neurogenic Orthostatic Hypotension (NOH) (NOH302)

Primary Autonomic Failure | Dopamine Beta Hydroxylase Deficiency | Symptomatic Neurogenic Orthostatic Hypotension (NOH) | Non-diabetic Neuropathy

United States, Australia, Canada, New Zealand
Menzies School of Health Research

Completed

Optimum Thiamine Intervention (OpTIn) Trial (OpTIn)

Wernicke-Korsakoff Syndrome

Australia
Harald Dobnig, MD

Completed

VITdAL@ICU - Correction of Vitamin D Deficiency in Critically Ill Patients (VITdAL@ICU)

Critical Illness | Vitamin D Deficiency

Austria
Celgene

Recruiting

A Study to Evaluate Long-term Safety in Participants Who Have Participated in Other Luspatercept (ACE-536) Clinical Trials

Beta-thalassemia | Myelodysplastic Syndromes (MDS) | Myeloproliferative Neoplasm(MPN)-Associated Myelofibrosis

United States, Japan, Israel, United Kingdom, Turkey, Greece, Netherlands, France, Australia, Belgium, Bulgaria, Canada, Germany, Italy, Malaysia, Spain, Sweden, Taiwan, Thailand, Tunisia, China, Lebanon
bluebird bio

Completed

A Study Evaluating the Safety and Efficacy of the LentiGlobin BB305 Drug Product in β-Thalassemia Major Participants

β-thalassemia Major

United States, Australia, Thailand
Vifor Pharma
ICON Clinical Research; American Regent, Inc.

Completed

Ferric Carboxymaltose (FCM) Assessment in Subjects With Iron Deficiency Anaemia and Non-dialysis-dependent Chronic Kidney Disease (NDD-CKD) (FIND-CKD)

Chronic Kidney Disease | Iron Deficiency Anaemia

United Kingdom, United States, Australia, Austria, Belgium, Czech Republic, Denmark, France, Germany, Greece, Italy, Netherlands, Norway, Poland, Portugal, Romania, Spain, Sweden, Turkey
Medicure

Recruiting

Oral Pyridoxal 5'-Phosphate for the Treatment of Patients With PNPO Deficiency (MEND-PNPO)

Pyridox(am)Ine 5'-Phosphate Oxidase Deficiency

United States, Australia
Medical University of Graz

Completed

Vitamin D Supplementation in Chronic Stable Heart Failure (VITD-HI)

Heart Failure | Vitamin D Deficiency

Austria
Kepler University Hospital

Terminated

Oral Iron Substitution for Orthopedic Surgery

Iron Deficiency Anemia

Austria
Kirby Institute
Gilead Sciences; Melanoma Institute Australia; St Vincent's Hospital, Sydney; Royal... and other collaborators

Active, not recruiting

Coronavirus (COVID-19) Outcomes Registries in Immunocompromised Individuals Australia (CORIA) (CORIA)

COVID-19 | Cancer | Primary Immune Deficiency Disorder | HIV-1-infection | Immunosuppression Disorders

Australia
University of Colorado, Denver
National Institute of Neurological Disorders and Stroke (NINDS); National Institutes... and other collaborators

Recruiting

International CDKL5 Clinical Research Network (ICCRN)

CDKL5 | CDKL5 Deficiency Disorder | CDD

United States, Australia
Medical University of Graz

Completed

Vitamin D Supplementation in Cirrhotic Patients

Vitamin D Deficiency | Cirrhosis

Austria
Monash University

Recruiting

Role of Antibiotic Therapy or Immunoglobulin On iNfections in hAematoLogy: Immunoglobulin Stopping or Extension (RATIONALISE)

Hypogammaglobulinemia | Haematological Malignancy

Australia
Medical University of Graz

Completed

SpHb Perioperatively

Anemia | Anemia, Iron Deficiency

Austria
Mein Hanusch-Krankenhaus

Unknown

Preoperative Supplementation of Sucrosomal Iron as Hematopoietic Support. (Preop Iron)

Anemia, Iron-Deficiency

Austria
Medical University of Graz

Completed

Styrian Vitamin D Hypertension Trial

Vitamin D Deficiency | Arterial Hypertension

Austria
Vifor Pharma

Terminated

Ferinject® in Patient With Thrombocytosis Secondary to Inflammatory Bowel Disease (IBD)

Thrombocytosis | Iron-Deficiency Anemia

Austria

Clinical Trials on Glucosylceramide Beta-Glucosidase Deficiency Disease in Australia

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Congo, DR of

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations