Clinical Trials on Genetic Diseases, X-Linked in Australia

Total 966 results

University Hospital Freiburg

Completed

Assessing Immune Function in Young Patients With Cytopenia That Did Not Respond to Treatment

Myelodysplastic Syndromes | Fanconi Anemia | Dyskeratosis Congenita | Pearson Marrow-pancreas Syndrome | Shwachman-diamond Syndrome

Spain, Germany, Switzerland, Austria, Netherlands, Italy, Czech Republic, Belgium, Denmark, Ireland
CSL Limited

Completed

Subcutaneous Ig NextGen 16% in PID Patients

Primary Immunodeficiency (PID)

Australia, New Zealand
Murdoch Childrens Research Institute
Monash University

Completed

Does Treatment With Montelukast Improve Symptoms of Atopic Dermatitis in Children?

Dermatitis, Atopic

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
RDC Clinical Pty Ltd

Completed

The Effectiveness of a Topical Palmitoylethanolamide (PEA) Formulation (Levagen+) for Reducing Symptoms of Eczema

Eczema, Atopic

Australia
Royal North Shore Hospital
University of Sydney

Recruiting

Markerless Image Guidance Using Intrafraction Kolovoltage X-ray Imaging for Lung Cancer Radiotherapy (VALKIM)

Lung Cancer | Markerless Image Guidance | Intrafraction Kolovoltage X-ray Imaging

Australia
Murdoch Childrens Research Institute

Suspended

Can Vitamin D Treatment Help Treat Moderate to Severe Atopic Dermatitis in Young Children? The D-Vex Pilot Study (D-Vex)

Atopic Dermatitis

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
Inflazome UK Ltd

Completed

Safety and Tolerability, Pharmacokinetic and Pharmacodynamic Study With IZD334

Healthy Volunteers | Cryopyrin Associated Periodic Syndrome

Australia
The Alfred
Monash University

Recruiting

The Alfred Step Test Exercise Protocol (A-STEP), for Adults With Cystic Fibrosis. (A-STEP)

Fibrosis | Lung Diseases | Pancreatic Diseases | Cystic Fibrosis | Genetic Diseases | Respiratory Diseases

Australia
Premier Specialists, Australia

Recruiting

Development and Validation of an Investigator Global Assessment Score for Keratosis Pilaris

Keratosis Pilaris

Australia
Biosion, Inc.
Southern Star Research Pty Ltd.

Completed

Safety and Tolerability of BSI-045B in Healthy Adult Subjects and Patients With Atopic Dermatitis

Atopic Dermatitis

Australia
Hoth Therapeutics, Inc.
Novotech (Australia) Pty Limited

Completed

Safety, Tolerability, Pharmacokinetics and Efficacy of Twice Daily Application of Topical BioLexa in Adult Healthy Subjects and Patients With Mild to Moderate Atopic Dermatitis

Atopic Dermatitis

Australia
Pfizer

Completed

Efficacy and Safety of AN2898 and AN2728 Topical Ointments to Treat Mild-to-Moderate Atopic Dermatitis

Dermatitis, Atopic

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
The University of Western Australia
National Health and Medical Research Council, Australia

Unknown

Postprandial Lipid Metabolism in Familial Hypercholesterolaemia:Effects of Fish Oils (FIFH)

Familial Hypercholesterolaemia | Hypertriglyceridaemia

Australia
CSL Limited

Completed

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

Primary Immune Deficiency (PID)

Australia
Shire

Completed

Short Term Observational Study in DEB Patients

Dystrophic Epidermolysis Bullosa

United States, Czechia, Italy, Austria, Australia, France, Germany
Savara Inc.
INC Research Limited

Completed

Inhaled Vancomycin Tolerability, Safety and Pharmacokinetics

Healthy | Cystic Fibrosis

Australia
Pfizer

Completed

Efficacy and Safety of AN2728 Topical Ointment to Treat Adolescents With Atopic Dermatitis

Dermatitis, Atopic

Australia, United States
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
Nestlé

Completed

Pharmacodynamics, Safety, Tolerability and Pharmacokinetics of CDX-6114 in Patients With Phenylketonuria (PKU)

Phenylketonuria

Australia, Germany
Ribon Therapeutics, Inc.

Recruiting

SAD/MAD Safety and PK Study of RBN-3143 in Healthy and Atopic Dermatitis Subjects

Atopic Dermatitis

Australia, New Zealand
ADARx Pharmaceuticals, Inc.

Recruiting

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

Hereditary Angioedema

Australia
MyoKardia, Inc.

Completed

A Single Ascending Dose Study Assessing the Safety, Tolerability, PK and PD of MYK-491

Dilated Cardiomyopathy

Australia
Ionis Pharmaceuticals, Inc.

Completed

Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS 702843 Administered Subcutaneously to Healthy Volunteers

Thalassemia

Australia
RHEACELL GmbH & Co. KG

Recruiting

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

Epidermolysis Bullosa

Austria
Otsuka Pharmaceutical Development & Commercialization...

Completed

A Study to Evaluate the Effectiveness and Safety of Topical OPA-15406 Ointment to Treat Participants With Atopic Dermatitis

Atopic Dermatitis

Poland, United States, Australia
Pfizer
Pfizer

Completed

A Study to Evaluate GBT021601 in Single and Multiple Doses in Healthy Participants

Sickle Cell Disease

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

Recruiting

The Global Angelman Syndrome Registry (GASR)

Angelman Syndrome

Australia
ASLAN Pharmaceuticals

Completed

A Multi-center, Randomized, Double-blind, Placebo-controlled, Multiple Ascending Dose Study of the Safety, Tolerability, and Pharmacokinetics of Subcutaneously Delivered ASLAN004 in Adults With Moderate-Severe Atopic Dermatitis

Atopic Dermatitis

Australia, Singapore, United States
Nacuity Pharmaceuticals, Inc.

Recruiting

PK and PD Study of NPI-001 and Cysteamine Bitartrate (INCA)

Cystinosis

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
Orphatech Pharmaceuticals, GmbH

Withdrawn

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

Molybdenum Cofactor Deficiency Type A

Australia
Flatley Discovery Lab LLC

Completed

A Study to Assess the Safety, Tolerability and PK Profile of FDL176 in Healthy and CF Participants

Cystic Fibrosis

Australia
ModernaTX, Inc.

Recruiting

A Dose-finding Study to Evaluate mRNA-3210 in Participants With Phenylketonuria

Phenylketonuria

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

Completed

Thyroid-Associated Ophthalmopathy Subtypes and Orbital Antibodies

Graves' Ophthalmopathy

Australia
LEO Pharma

Completed

A Multi-omics Disease Signature Trial in Adult Patients With Moderate to Severe AD

Atopic Dermatitis

Austria
Medical University of Vienna

Completed

Comparison of UVA1 Phototherapy Versus Photochemotherapy for Patients With Severe Generalized Atopic Dermatitis

Atopic Dermatitis

Austria
Murdoch Childrens Research Institute

Completed

Effect of Creatine Monohydrate on Functional Muscle Strength in Children With FSHD

Facio-Scapulo-Humeral Dystrophy | FSHD2 | FSHD1

Australia
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
Medical University of Vienna

Completed

FACE-Q in Blepharoplasty

Dermatochalasis of Upper Eyelid

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
Regeneron Pharmaceuticals
Sanofi

Completed

Safety and Tolerability of Dupilumab in Participants With Moderate to Severe Atopic Dermatitis

Atopic Dermatitis

Germany, New Zealand, Australia
The Alfred

Unknown

A Study Comparing Continuous Infusion Antibiotics to Standard Treatment for Lung Infections in Cystic Fibrosis (CISTIC)

Cystic Fibrosis

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
The University of Queensland
Telethon Kids Institute

Completed

Prevention of Bronchiectasis in Infants With Cystic Fibrosis (COMBATCF)

Bronchiectasis | Cystic Fibrosis

Australia, New Zealand
Bayside Health

Unknown

Modified Release Posaconazole in Patients With Cystic Fibrosis

Cystic Fibrosis

Australia

Clinical Trials on Genetic Diseases, X-Linked in Australia

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