Clinical Trials on Birt-Hogg-Dube Syndrome in Australia

Total 258 results

Zydus Lifesciences Limited

Completed

A Clinical Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ZYIL1 in Subjects With Cryopyrin Associated Periodic Syndromes (CAPS)

Cryopyrin Associated Periodic Syndrome

Australia
Inflazome UK Ltd

Completed

Safety and Tolerability, Pharmacokinetic and Pharmacodynamic Study With IZD334

Healthy Volunteers | Cryopyrin Associated Periodic Syndrome

Australia
The University of Western Australia
Sir Charles Gairdner Hospital; Perron Institute for Neurological and Translational...

Withdrawn

Optimising Anterior Pallidal Deep Brain Stimulation for Tourette's Syndrome

Tourette's Syndrome

Australia
Peter MacCallum Cancer Centre, Australia
National Health and Medical Research Council, Australia; University of Melbourne

Recruiting

Diagnosis and Phenotype Characterisation Using Genomics in Patients With Inherited Bone Marrow Failure (IBMDx Study) (IBMDx)

Hematologic Diseases | Inherited Platelet Disorder | Inherited BMF Syndrome

Australia
Medical University of Vienna

Unknown

Effectiveness of fESWT in the Treatment of CTS

Carpal Tunnel Syndrome

Austria
Novartis

Completed

Magnetic Resonance Imaging (MRI) Assessments of the Heart and Liver Iron Load in Patients With Transfusion Induced Iron Overload

Myelodysplastic Syndromes | Hemoglobinopathies | Diamond-Blackfan Anemia | Transfusional Iron Overload | Other Inherited or Acquired Anaemia | MPD Syndrome | Other Rare Anaemias

Australia
Ascend Biopharmaceuticals Ltd

Completed

Safety and Efficacy of ASN-002 Combined With a Hedgehog Pathway Inhibitor

Basal Cell Nevus Syndrome | Basal Cell Carcinoma

Australia
CSL Limited

Completed

Subcutaneous Ig NextGen 16% in PID Patients

Primary Immunodeficiency (PID)

Australia, New Zealand
Medical University of Graz

Recruiting

The Effect of Low-Level Laser Therapy in Carpal Tunnel Syndrome (KaLi-CTS)

Carpal Tunnel Syndrome

Austria
Aqualung Therapeutics Corp.

Completed

First-In-Human Study to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ALT-100

ARDS, Human

Australia
Foundation for Angelman Syndrome Therapeutics,...
Queensland University of Technology

Recruiting

The Global Angelman Syndrome Registry (GASR)

Angelman Syndrome

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
Murdoch Childrens Research Institute

Completed

Safety of Autologous Cord Blood Cells in HLHS Patients During Norwood Heart Surgery

Heart Defects, Congenital | Hypoplastic Left Heart Syndrome | Pediatric Disorder

Australia
Medical University of Graz

Completed

Anticipative Diagnosis of Central Venous Catheter Related Bloodstream Infections

Bloodstream Infection Due to Central Venous Catheter

Austria
Nacuity Pharmaceuticals, Inc.
Foundation Fighting Blindness

Active, not recruiting

Safety and Efficacy of NPI-001 Tablets for RP Associated With Usher Syndrome (SLO RP)

Usher Syndromes

Australia
BioMarin Pharmaceutical

Completed

BMN 110 Phase 3B in Australian Patients (MOR-AUS)

Mucopolysaccharidosis IVA (Morquio A Syndrome)

Australia
Murdoch Childrens Research Institute

Completed

Is Medicinal Cannabis an Effective Treatment for Tourette Syndrome in Adolescents? A Pilot Study

Tourette Syndrome in Adolescence

Australia
Fisher and Paykel Healthcare

Not yet recruiting

Infant Respiratory Interface and Accessories Evaluation Study

Respiratory Distress Syndrome, Newborn

Australia
Rigshospitalet, Denmark

Completed

High Frequency Oscillatory Ventilation Combined With Intermittent Sigh Breaths: Effects on Lung Volume Monitored by Electric Tomography Impedance.

Bronchopulmonary Dysplasia | Ventilator-Induced Lung Injury | Respiratory Distress Syndrome In Premature Infants | Functional Residual Capacity

Australia
Medical University of Vienna
University of Vienna; Griffith University

Recruiting

Brown Adipose Tissue Activity in Gilbert's Syndrome

Gilbert Syndrome

Austria
Australian and New Zealand Intensive Care Research...
The Alfred

Not yet recruiting

Trial of ECMO to De-Sedate, Extubate Early and Mobilise in Hypoxic Respiratory Failure (REDEEM)

Pneumonia | Extracorporeal Membrane Oxygenation | Mechanical Ventilation Complication | Hypoxemia | COVID-19 Respiratory Infection | Acute Respiratory Distress Syndrome Due to COVID-19

Australia, Germany
CSL Limited

Completed

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

Primary Immune Deficiency (PID)

Australia
Leiden University Medical Center
ZonMw: The Netherlands Organisation for Health Research and Development; Horizon...

Recruiting

Phase I/II Clinical Trial Stem Cell Gene Therapy in RAG1-Deficient SCID (RAG1-SCID)

Severe Combined Immunodeficiency Due to RAG1 Deficiency

Spain, Netherlands, Italy, Poland, Australia, Turkey, United Kingdom
Shiphrah Biomedical Inc.
University of South Australia

Terminated

UniSA BackOff! Study: Adelaide PrenaBelt Trial

Pregnancy Complications | Fetal Growth Retardation | Sleep-Disordered Breathing | Infant, Very Low Birth Weight | Stillbirth | Infant, Small for Gestational Age | Fetal Hypoxia | Infant, Low Birth Weight

Australia
Bayside Health

Completed

Australia and New Zealand Adult Extracorporeal Membrane Oxygenation (ECMO) Audit 2005

Respiratory Insufficiency | Shock, Cardiogenic | Respiratory Distress Syndrome, Adult

Australia
Celgene

Completed

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

Myelodysplastic Syndromes | Beta-thalassemia

Austria
Garvan Institute of Medical Research

Completed

Gut Derived Hormones, Body Composition and Metabolism in Prader-Willi Syndrome

Prader-Willi Syndrome

Australia
Novartis Pharmaceuticals

Completed

A Trial to Evaluate the Safety, Local Tolerability, Pharmacokinetics and Pharmacodynamics of LDE225 on Skin Basal Cell Carcinomas in Gorlin Syndrome Patients

Treatment for Basal Cell Carcinomas (BCCs) in Gorlin Syndrome Patients

Austria, Switzerland
Salzburger Landeskliniken

Completed

Evaluation of Semi-rigid and Flexible Catheters for Less Invasive Surfactant Administration in Preterm Infants

Neonatology | Perinatal Distress Syndrome

Austria
Anavex Life Sciences Corp.

Completed

ANAVEX2-73 Study in Patients With Rett Syndrome (AVATAR)

Rett Syndrome

Australia, United Kingdom
Neuren Pharmaceuticals Limited

Recruiting

An Open-Label Study of the Safety, Tolerability, and Pharmacokinetics of Oral NNZ-2591 in Angelman Syndrome (AS-001)

Angelman Syndrome

Australia
Zynerba Pharmaceuticals, Inc.

Completed

Open-Label Study of ZYN002 Administered as a Transdermal Gel to Children and Adolescents With 22q11.2 Deletion Syndrome (INSPIRE)

22Q Deletion Syndrome

United States, Australia
Takeda

Active, not recruiting

A Study of Soticlestat in Adults and Children With Rare Epilepsies (Endymion 1)

Epilepsy | Dravet Syndrome (DS) | Lennox-Gastaut Syndrome (LGS)

United States, Canada, Australia, China, Israel, Poland, Spain, Portugal
Longboard Pharmaceuticals

Recruiting

Open-label, Long-term Safety Study of LP352 in Subjects With Developmental and Epileptic Encephalopathy

Dravet Syndrome | Lennox-Gastaut Syndrome | Developmental and Epileptic Encephalopathy

United States, Australia
Eloxx Pharmaceuticals, Inc.

Recruiting

A Study of ELX-02 in Patients With Alport Syndrome

Alport Syndrome

United Kingdom, Australia
Mucpharm Pty Ltd
St George Hospital, Australia; Mobius Medical Pty Ltd.

Completed

Nebulised BromAc in Healthy Volunteers

COVID-19 Pneumonia | COVID-19 Acute Respiratory Distress Syndrome | Bromelains Adverse Reaction | Acetylcysteine Adverse Reaction | Mucus; Plug

Australia
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
Medical University of Vienna

Recruiting

Infants With Severe Acute Respiratory Distress Syndrome: The Prone Trial

Infant | Acute Lung Injury/Acute Respiratory Distress Syndrome (ARDS) | Surfactant Dysfunction

Austria
Kirby Institute
National Heart, Lung, and Blood Institute (NHLBI); St Vincent's Hospital, Sydney

Completed

HIV Infection And Metabolic Abnormalities Protocol 1 (HAMA001)

Cardiovascular Disease | HIV-Associated Lipodystrophy Syndrome

Australia
Peninsula Health
Baxter Healthcare Corporation; Monash University

Recruiting

Extracorporeal Carbon Dioxide Removal Using PrismaLung in Reducing Ventilator Induced Lung Injury

ARDS, Human

Australia
ADARx Pharmaceuticals, Inc.

Recruiting

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

Hereditary Angioedema

Australia
Longboard Pharmaceuticals

Completed

Study to Investigate LP352 in Subjects With Developmental and Epileptic Encephalopathies (PACIFIC)

Dravet Syndrome | Lennox Gastaut Syndrome | Developmental and Epileptic Encephalopathy

United States, Australia
Eisai Limited

Completed

European Registry of Anti-Epileptic Drug Use in Patients With Lennox-Gastaut Syndrome (LGS)

Lennox-Gastaut Syndrome

France, United Kingdom, Germany, Spain, Denmark, Austria, Italy, Sweden
Eisai Inc.

Terminated

Study of Perampanel as Adjunctive Treatment for Inadequately Controlled Seizures Associated With Lennox-Gastaut Syndrome

Lennox-Gastaut Syndrome (LGS)

Korea, Republic of, United States, Australia, Belgium, Japan, Czechia, India
Optimed Medizinische Instrumente GmbH

Completed

Treatment of the Postthrombotic Syndrome With the Oblique Stent - TOPOS Study

Post-thrombotic Syndrome | Iliac Vein Compression Syndrome

Switzerland, Austria, Germany, Netherlands
Zogenix International Limited, Inc., a subsidiary...

Active, not recruiting

A Study to Investigate the Efficacy and Safety of ZX008 (Fenfluramine Hydrochloride) as an Adjunctive Therapy in Children and Adults With Lennox-Gastaut Syndrome

Lennox Gastaut Syndrome

United States, Australia, Belgium, Canada, Denmark, France, Germany, Italy, Japan, Mexico, Netherlands, Poland, Spain, Sweden
Thomas Staudinger

Terminated

PGE1 as Additive Anticoagulant in ECMO-Therapy (ECMO_PGE1)

Extracorporeal Membrane Oxygenation | Respiratory Distress Syndrome, Adult

Austria
Sydney Children's Hospitals Network
Children's Cancer Institute Australia

Recruiting

Cancer Predisposition Testing by Family-based Whole-genome Sequencing (WGS) in Every Child With Newly Diagnosed Cancer (PREDICT)

Cancer | Neoplastic Syndromes, Hereditary | Genetic Predisposition to Disease

Australia
Takeda
Ovid Therapeutics Inc.

Completed

A Phase 2, Multicenter, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy, Safety, and Tolerability of TAK-935 (OV935) as an Adjunctive Therapy in Pediatric Participants With Developmental and/or Epileptic Encephalopathies (ELEKTRA)

Epilepsy | Dravet Syndrome | Lennox-Gastaut Syndrome

United States, Canada, Israel, Poland, Spain, Australia, China, Portugal
Novartis Pharmaceuticals

Completed

Efficacy, Safety and Pharmacokinetics of Oral LDE225 in Treatment of Patients With Nevoid Basal Cell Carcinoma Syndrome (NBCCS)

Nevoid Basal Cell Carcinoma Syndrome | Basal Cell Carcinoma | Gorlin Syndrome

Belgium, Austria, Germany, Canada

Clinical Trials on Birt-Hogg-Dube Syndrome in Australia

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