Clinical Trials on Hereditary Epidermolysis Bullosa in Australia

Total 169 results

Holostem Terapie Avanzate s.r.l.
Paracelsus Medical University

Terminated

Clinical Trial to Assess Safety and Efficacy of Autologous Cultured Epidermal Grafts Containing Epidermal Stem Cells Genetically Modified in Patients With RDEB. (HOLOGENE7)

Recessive Dystrophic Epidermolysis Bullosa

Austria
Holostem Terapie Avanzate s.r.l.
Paracelsus Medical University

Terminated

Clinical Trial to Assess Safety and Efficacy of Autologous Cultured Epidermal Grafts Containing Epidermal Stem Cells Genetically Modified in Patients With JEB (HOLOGENE17) (HOLOGENE17)

Junctional Epidermolysis Bullosa

Austria
Shire

Completed

Short Term Observational Study in DEB Patients

Dystrophic Epidermolysis Bullosa

United States, Czechia, Italy, Austria, Australia, France, Germany
RHEACELL GmbH & Co. KG
FGK Clinical Research GmbH; Ticeba GmbH; Granzer Regulatory Consulting & Services

Completed

Allogeneic ABCB5-positive Stem Cells for Treatment of Epidermolysis Bullosa

Recessive Dystrophic Epidermolysis Bullosa

United States, Austria, France, Germany, United Kingdom
TWi Biotechnology, Inc.

Recruiting

An International, Multicenter, Randomized, Double-Blind, Parallel Group, Vehicle-Controlled, Phase 2/3 Study With Open-Label Extension Evaluating the Efficacy and Safety of Diacerein 1% Ointment for the Treatment of Generalized Epidermolysis Bullosa Simplex (EBS)

Generalized Epidermolysis Bullosa Simplex

United States, Taiwan, Australia, Philippines, Austria, Italy, Poland
Castle Creek Pharmaceuticals, LLC

Terminated

Safety and Efficacy of Diacerein 1% Ointment for Subjects With Epidermolysis Bullosa Simplex (EBS)

Epidermolysis Bullosa Simplex

United States, Israel, Australia, Austria, France, Germany, Netherlands, United Kingdom
Premier Specialists, Australia

Unknown

Using Topical Sirolimus 2% for Patients With Epidermolysis Bullous Simplex (EBS) Study

Epidermolysis Bullosa Simplex | Epidermolysis Bullosa Simplex Kobner | Weber-Cockayne Syndrome

Australia
Prof. Johann Bauer

Recruiting

Rigosertib for RDEB-SCC

Epidermolysis Bullosa Dystrophica | Squamous Cell Carcinoma

Austria
RHEACELL GmbH & Co. KG

Recruiting

Allogeneic ABCB5-positive Dermal Mesenchymal Stromal Cells for Treatment of Epidermolysis Bullosa (Phase III, Cross-over)

Epidermolysis Bullosa

Austria
Scioderm, Inc.
Amicus Therapeutics

Terminated

Open Label Extension Study to Evaluate the Long-term Safety of Zorblisa (SD-101-6.0) in Patients With Epidermolysis Bullosa

Epidermolysis Bullosa

Poland, Spain, Serbia, Netherlands, United States, Austria, Australia, Israel, Germany, France, United Kingdom, Lithuania
Amryt Research Limited

Completed

Phase III Efficacy and Safety Study of Oleogel-S10 in Epidermolysis Bullosa (EASE)

Epidermolysis Bullosa

United States, Spain, Israel, Australia, France, Denmark, Switzerland, Italy, Hong Kong, Argentina, Austria, Brazil, Chile, Colombia, Czechia, Georgia, Germany, Greece, Hungary, Ireland, Romania, Russian Federation, Serbia, Singapore, U... and more
Scioderm, Inc.
Amicus Therapeutics

Completed

ESSENCE Study: Efficacy and Safety of SD-101 Cream in Participants With Epidermolysis Bullosa

Epidermolysis Bullosa

Italy, Spain, Serbia, United States, Australia, Netherlands, Austria, Germany, Israel, Lithuania, France, United Kingdom, Poland
Shire Regenerative Medicine, Inc.

Terminated

A Study of the Efficacy and Safety of ABH001 in the Treatment of Patients With Epidermolysis Bullosa Who Have Wounds That Are Not Healing

Epidermolysis Bullosa

United States, Spain, Austria, Canada, France, Germany, Poland, Portugal
Premier Specialists, Australia
Steritas

Active, not recruiting

Investigating the Morbidity of Glucocorticoid Use in Patients With Autoimmune Bullous Diseases (AIBDs)

Glucocorticoids Toxicity | Autoimmune Bullous Dermatosis

Australia
Center for Eye Research Australia

Completed

Pilot Study of a Suprachoroidal Retinal Prosthesis

Retinitis Pigmentosa | Choroideremia

Australia
ADARx Pharmaceuticals, Inc.

Recruiting

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

Hereditary Angioedema

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
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
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

Completed

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

Fuchs' Endothelial Dystrophy

Austria
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
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
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
Novartis Pharmaceuticals

Terminated

Safety, Efficacy and PK/PD of QGE031 vs. Placebo in Patients With Active Bullous Pemphigoid Despite Oral Steroid Treatment

Bullous Pemphigoid

Taiwan, Germany, United States, Japan, Austria, France
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
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
argenx

Recruiting

A Phase 3 Study to Evaluate the Long-term Safety, Tolerability and Efficacy of Efgartigimod PH20 SC in Adult Participants With Bullous Pemphigoid (BALLAD+)

Bullous Pemphigoid

Netherlands, Spain, Germany, United Kingdom, China, Italy, Hungary, United States, Croatia, Japan, Serbia, Bulgaria, Israel, Australia, Greece, Slovakia
Bioverativ, a Sanofi company
Assign Data Management and Biostatistics GmbH; Quest Diagnostics-Nichols Insitute and other collaborators

Completed

Safety, Tolerability and Activity of BIVV009 in Healthy Volunteers and Patients With Complement Mediated Disorders (BIVV009-01)

End-stage Renal Disease (ESRD) | Cold Agglutinin Disease (CAD) | Bullous Pemphigoid (BP) | Warm Autoimmune Hemolytic Anemia (WAIHA)

Austria
Bond Biosciences

Terminated

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

Hereditary Hemochromatosis

Australia
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
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
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
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
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
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
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
Regeneron Pharmaceuticals
Sanofi

Active, not recruiting

A Study to Evaluate the Efficacy and Safety of Dupilumab in Adult Patients With Bullous Pemphigoid (LIBERTY-BP)

Bullous Pemphigoid

United States, Japan, Germany, Australia, France, Israel, Poland, Spain, Taiwan
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
AstraZeneca

Terminated

A Study to Investigate the Use of Benralizumab in Patients With Bullous Pemphigoid. (FJORD)

Bullous Pemphigoid

France, Italy, Spain, Germany, United States, Australia, China, Bulgaria, Israel, Japan, Greece
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
argenx

Active, not recruiting

A Phase 2/3 Study of Efgartigimod PH20 SC in Adult Participants With Bullous Pemphigoid (BALLAD)

Bullous Pemphigoid

United States, Australia, Bulgaria, China, Croatia, Czechia, France, Germany, Greece, Hungary, Israel, Italy, Japan, Latvia, Netherlands, Poland, Romania, Serbia, Slovakia, Spain, United Kingdom
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
Medical University of Vienna

Completed

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

Epistaxis | HHT | Morbus Osler

Austria

Clinical Trials on Hereditary Epidermolysis Bullosa in Australia

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