Clinical Trials on Spastic Paraplegia, Hereditary in Australia

Total 162 results

Dr. Rebecca Schule
German Federal Ministry of Education and Research; German Center for Neurodegenerative...

Recruiting

Phenotypes, Biomarkers and Pathophysiology in Hereditary Spastic Paraplegias and Related Disorders (HSP-PBP)

Hereditary Spastic Paraplegia

Austria, Germany, Italy
Center for Eye Research Australia

Completed

Pilot Study of a Suprachoroidal Retinal Prosthesis

Retinitis Pigmentosa | Choroideremia

Australia
University of Graz
Karolinska Institutet; Medical University of Graz

Recruiting

Muscle-tendon Pathology and Metabolic Dysregulation in CP

Cerebral Palsy, Spastic

Austria
ADARx Pharmaceuticals, Inc.

Recruiting

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

Hereditary Angioedema

Australia
University of Graz
VU University of Amsterdam

Completed

Stretching in Children and Adolescents With Spastic Cerebral Palsy

Cerebral Palsy, Spastic

Austria
Medical University of Graz
University of Graz

Active, not recruiting

Influence of Immobilisation, Stretching and Activity on Morphological and Mechanical Properties of Spastic Muscle

Cerebral Palsy, Spastic

Austria
University of Sydney

Completed

Orthostatic Tolerance During FES (Functional Electrical Stimulation)-Walking in Paraplegia

Spinal Cord Injury

Australia
Peter MacCallum Cancer Centre, Australia

Recruiting

The Evaluating Multidisciplinary Bone Marrow Failure Care in Bone Marrow Failure and Related Disorders. (EMBRACE)

Aplastic Anemia | Bone Marrow Failure Syndrome

Australia
Murdoch Childrens Research Institute
Melbourne Health; Austin Health; Royal Brisbane and Women's Hospital; The University... and other collaborators

Completed

Mi-iron - Moderately Increased Iron - is Reducing Iron Overload Necessary? (Mi-iron)

Hereditary Haemochromatosis

Australia
Medical University of Vienna

Recruiting

Correlation Between In-vivo Anatomy of Corneal Dystrophies as Assessed by High- Resolution Optical Coherence Tomography (OCT) Measurement and Histological Examination

Corneal Dystrophy

Austria
Medical University of Vienna

Completed

Assessment of Corneal Graft Attachment in Patients With Fuchs Endothelial Corneal Dystrophy Following DMEK Using Ultra-high Resolution OCT

Fuchs' Endothelial Dystrophy

Austria
BioCryst Pharmaceuticals

Completed

Efficacy and Safety of BCX7353 to Prevent Angioedema Attacks in Subjects With Hereditary Angioedema (APeX-1)

Hereditary Angioedema (HAE)

Canada, United Kingdom, Germany, Spain, Australia, Hungary, Italy, Austria, North Macedonia, Denmark, Switzerland
PYC Therapeutics

Recruiting

Natural History of PRPF31 Mutation-Associated Retinal Dystrophy

Retinitis Pigmentosa | Eye Diseases, Hereditary | Retinal Dystrophies | Retinal Dystrophy Rod | Retinal Dystrophy Rod Progressive

United States, Australia
RBP4 Pty Ltd
Belite Bio, Inc

Completed

This is a Dose-finding Study Followed by 2-year Extension Study to Evaluate Safety and Tolerability of Tinlarebant in Adolescent Subjects With Stargardt Disease

Stargardt Disease

Taiwan, Australia
Kiora Pharmaceuticals, Inc.

Recruiting

A Phase I/II Dose-escalating Study of the Safety, Tolerability and Efficacy of KIO-301 Administered Intravitreally to Patients With Retinitis Pigmentosa and Choroideremia (ABACUS) (ABACUS)

Retinitis Pigmentosa | Choroideremia

Australia
PYC Therapeutics

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
Lions Eye Institute, Perth, Western Australia
Adverum Biotechnologies, Inc.

Completed

Safety and Efficacy Study of rAAV.sFlt-1 in Patients With Exudative Age-Related Macular Degeneration (AMD)

Eye Diseases | Macular Degeneration | Retinal Degeneration | Retinal Neovascularization | Age-related Maculopathies | Age-related Maculopathy | Maculopathies,Age-related | Maculopathy,Age-related

Australia
University Children's Hospital Tuebingen

Unknown

Safety Study of CD3/CD19 Depleted Haploidentical Stem Cells

Aplastic Anemia | Acute Leukemias | Childhood Solid Tumor

Germany, Austria
Pharvaris Netherlands B.V.

Active, not recruiting

Dose-ranging Study of Oral PHA-022121 for Prophylaxis Against Angioedema Attacks in Patients With Hereditary Angioedema Type I or Type II (HAE CHAPTER-1)

Hereditary Angioedema | Hereditary Angioedema Type I | Hereditary Angioedema Type II | Hereditary Angioedema Types I and II | Hereditary Angioedema Attack | Hereditary Angioedema With C1 Esterase Inhibitor Deficiency | Hereditary Angioedema - Type 1 | Hereditary Angioedema - Type 2 | C1 Esterase Inhibitor... and other conditions

United States, Poland, Germany, Austria, Bulgaria, Canada, Ireland, Italy, United Kingdom
Mobius Medical Pty Ltd.
University of Melbourne; Australian National University; Centre for Eye Research... and other collaborators

Completed

Study of a Suprachoroidal Retinal Prosthesis

Retinitis Pigmentosa | Choroideremia

Australia
Intellia Therapeutics

Active, not recruiting

NTLA-2002 in Adults With Hereditary Angioedema (HAE) (NTLA-2002)

Hereditary Angioedema

France, New Zealand, Germany, United Kingdom, Netherlands, Australia
Bond Biosciences

Terminated

A Study to Evaluate BBI-001 in Hereditary Haemochromatosis (HH) Patients and Iron Deficient Volunteers

Hereditary Hemochromatosis

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
Shire

Completed

A Pharmacokinetic, Tolerability and Safety Study of Icatibant in Children and Adolescents With Hereditary Angioedema

Hereditary Angioedema (HAE)

United States, Canada, Australia, Austria, Colombia, Germany, Hungary, Israel, Italy, Spain
Center for Eye Research Australia
Australian National University; Commonwealth Scientific and Industrial Research... and other collaborators

Enrolling by invitation

Longitudinal Study of a Bionic Eye

Retinitis Pigmentosa | Choroideremia

Australia
CSL Behring

Recruiting

CSL312_3003 Safety and Pharmacokinetic Study in Subjects 2 to 11 Years of Age With Hereditary Angioedema

Hereditary Angioedema (HAE)

United States, Germany, Israel, Australia, Canada
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
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
Barwon Health
National Health and Medical Research Council, Australia; University of Melbourne

Completed

Thyroid-Associated Ophthalmopathy Subtypes and Orbital Antibodies

Graves' Ophthalmopathy

Australia
National Eye Institute (NEI)

Recruiting

Virtual Reality Mobility Assessment of Functional Vision in Retinal Disease

Cone-Rod Degeneration | Rod-Cone Degeneration

United States, Australia
Inflazome UK Ltd

Completed

Safety and Tolerability, Pharmacokinetic and Pharmacodynamic Study With IZD334

Healthy Volunteers | Cryopyrin Associated Periodic Syndrome

Australia
Shire
Takeda Development Center Americas, Inc.

Recruiting

Firazyr® Patient Registry (Icatibant Outcome Survey - IOS)

Hereditary Angioedema (HAE)

France, Austria, Australia, Spain, Czechia, Italy, United Kingdom, Germany, Israel, Ireland, Brazil, Denmark, Greece, Sweden
Kowa Research Institute, Inc.

Completed

Study to Investigate the Safety and Efficacy of K-321 in Patients With Fuchs Endothelial Corneal Dystrophy (FECD) Following Descemetorhexis

Fuchs' Endothelial Corneal Dystrophy

United States, Australia, Germany, Spain, Denmark
Santhera Pharmaceuticals

Completed

Post Authorisation Safety Study With Raxone in LHON Patients (PAROS)

Leber's Hereditary Optic Neuropathy (LHON)

Italy, Germany, France, Austria, Greece, Netherlands
University of Leipzig
German Research Foundation; Baylor College of Medicine; University of Pennsylvania and other collaborators

Active, not recruiting

Paraplegia Prevention in Aortic Aneurysm Repair by Thoracoabdominal Staging (PAPAartis)

Aortic Aneurysm, Thoracoabdominal

Sweden, Netherlands, Germany, France, Poland, Switzerland, Austria, United Kingdom, Italy
Takeda

Completed

A Study in Teenagers and Adults With Hereditary Angioedema (HAE) Type I or Type II Who Use Lanadelumab as Long-Term Prophylaxis (INTEGRATED)

Hereditary Angioedema (HAE)

France, Germany, Austria, Greece
Santhera Pharmaceuticals

Completed

Study to Assess the Efficacy and Safety of Raxone in LHON Patients (LEROS)

Leber's Hereditary Optic Neuropathy (LHON)

United States, United Kingdom, Spain, Poland, Austria, Belgium, Bulgaria, Germany, Italy, Portugal
Medical University of Vienna

Completed

Protexa® Versus TiLoopBra® in Immediate Breast Reconstruction- A Pilot Study

Breast Cancer | Hereditary Breast and Ovarian Cancer Syndrome

Austria
KalVista Pharmaceuticals, Ltd.

Completed

A Phase II, Cross-over Clinical Trial Evaluating the Efficacy and Safety of KVD900 in the On-demand Treatment of Angioedema Attacks in Adult Subjects With Hereditary Angioedema Type I or II

Hereditary Angioedema

United States, Austria, Czechia, Germany, Hungary, Italy, Netherlands, North Macedonia, Poland, United Kingdom
CSL Behring

Completed

A Study to Investigate CSL312 in Subjects With Hereditary Angioedema (HAE)

Hereditary Angioedema

Canada, Australia, Germany, United States, Israel
Medical University of Vienna

Completed

Intranasal Submucosal Bevacizumab for Epistaxis in Hereditary Hemorrhagic Telangiectasia (HHT)

Epistaxis | HHT | Morbus Osler

Austria
Shire
Takeda Development Center Americas, Inc.

Active, not recruiting

A Study of Lanadelumab in Persons With Hereditary Angioedema (HAE) Type I or II (ENABLE)

Hereditary Angioedema (HAE)

Germany, Switzerland, Spain, Israel, Italy, Austria, Portugal, Kuwait
BioMarin Pharmaceutical

Active, not recruiting

A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema (HAErmony-1)

Hereditary Angioedema | HAE

Spain, United States, Australia
BioCryst Pharmaceuticals

Completed

Study of BCX7353 as a Treatment for Attacks of Hereditary Angioedema

Hereditary Angioedema (HAE)

Austria, Denmark, France, Germany, Hungary, Israel, Italy, North Macedonia, Poland, Romania, Switzerland, United Kingdom
ACELYRIN Inc.

Recruiting

Efficacy and Safety of Lonigutamab in Subjects With Thyroid Eye Disease (TED) (TED)

Thyroid Eye Disease

United States, Australia
Michael Frass

Completed

Homeopathic Treatment of Children Suffering From PFAPA (HOMPFAPA)

Periodic Fever Syndrome

Austria
Ascend Biopharmaceuticals Ltd

Completed

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

Basal Cell Nevus Syndrome | Basal Cell Carcinoma

Australia
Medical University of Vienna
University of Vienna; Griffith University

Recruiting

Brown Adipose Tissue Activity in Gilbert's Syndrome

Gilbert Syndrome

Austria
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
CSL Behring

Completed

A Study to Evaluate the Long-term Clinical Safety and Efficacy of Subcutaneously Administered C1-esterase Inhibitor in the Prevention of Hereditary Angioedema

Hereditary Angioedema Types I and II

United States, Spain, Germany, Australia, Canada, Czechia, Hungary, Israel, Italy, Romania, United Kingdom

Clinical Trials on Spastic Paraplegia, Hereditary in Australia

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