Clinical Trials on X-linked Lymphoproliferative Syndrome in Australia

Total 194 results

Anavex Life Sciences Corp.

Completed

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

Rett Syndrome

Australia, United Kingdom
Medical University of Vienna

Completed

Effects of Creatine Supplementation in Rett Syndrome

Rett Syndrome

Austria
Murdoch Childrens Research Institute
University of Sydney; Sydney Children's Hospitals Network; Women's and Children... and other collaborators

Active, not recruiting

Mackenzie's Mission: The Australian Reproductive Carrier Screening Project

Autosomal Recessive Disorder | X-Linked Genetic Diseases

Australia
Anavex Life Sciences Corp.
Anavex Australia Pty Ltd.; Anavex Germany GmbH

Completed

ANAVEX2-73 Study in Pediatric Patients With Rett Syndrome (EXCELLENCE)

Rett Syndrome

Canada, Australia, United Kingdom
Newron Pharmaceuticals SPA

Terminated

Evaluation of the Efficacy, Safety, and Tolerability of Sarizotan in Rett Syndrome With Respiratory Symptoms (STARS)

Rett Syndrome

United States, India, United Kingdom, Australia, Italy
Healx Limited

Withdrawn

Investigation of Sulindac (HLX-0201) and Gaboxadol (HLX-0206) in Male Fragile X Syndrome Patients Aged 13-40 (IMPACT-FXS)

Fragile X Syndrome

United States, Australia
Zynerba Pharmaceuticals, Inc.

Recruiting

Clinical Study of Cannabidiol in Children, Adolescents, and Young Adults With Fragile X Syndrome (RECONNECT)

Fragile X Syndrome

United States, Australia, United Kingdom, Ireland
Zynerba Pharmaceuticals, Inc.

Completed

Clinical Study Of caNNabidiol in childrEn and adolesCenTs With Fragile X (CONNECT-FX) (CONNECT-FX)

Fragile X Syndrome

United States, New Zealand, Australia
Center for Eye Research Australia

Completed

Pilot Study of a Suprachoroidal Retinal Prosthesis

Retinitis Pigmentosa | Choroideremia

Australia
Novartis Pharmaceuticals

Terminated

Long-term, Safety, Tolerability and Efficacy Study of AFQ056 in Adult Patients With Fragile X Syndrome

Fragile X Syndrome

Germany, United States, Switzerland, Australia, Canada, Denmark, France, Italy, United Kingdom, Spain
Zynerba Pharmaceuticals, Inc.

Enrolling by invitation

Open Label Extension to Assess the Long-Term Safety and Tolerability of ZYN002 in Children and Adolescents With FXS

Fragile X Syndrome

United States, United Kingdom, New Zealand, Australia
Novartis Pharmaceuticals

Completed

Safety and Efficacy of AFQ056 in Adult Patients With Fragile X Syndrome

Fragile X Syndrome

United States, Switzerland, Germany, Australia, Canada, Denmark, France, Italy, Spain, United Kingdom
Novartis Pharmaceuticals

Terminated

Long-term, Safety and Tolerability Study of AFQ056 in Adolescent Patients With Fragile X Syndrome (Open-label)

Fragile X Syndrome

United States, Belgium, Australia, Israel, Switzerland, Germany, Denmark, Italy, Netherlands, Spain, Sweden, United Kingdom, France
Jazz Pharmaceuticals
GW Pharmaceuticals

Terminated

A Long-term Safety Study of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Rett Syndrome

Rett Syndrome | RTT

United States, Canada, Italy, Spain, United Kingdom, Australia
Novartis Pharmaceuticals

Completed

Safety and Efficacy of AFQ056 in Adolescent Patients With Fragile X Syndrome

Fragile X Syndrome

United States, Belgium, Australia, Israel, Switzerland, Germany, France, Canada, Denmark, Indonesia, Italy, Netherlands, Spain, Sweden, Turkey, United Kingdom
Mitsubishi Tanabe Pharma America Inc.

Recruiting

INcreased Sun Exposure Without Pain In Research Participants With EPP or XLP (INSPIRE)

Erythropoietic Protoporphyria (EPP) | X-Linked Protoporphyria (XLP)

United States, Spain, France, United Kingdom, Japan, Italy, Australia, Bulgaria, Czechia, Netherlands, Poland
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
The University of Western Australia

Unknown

High Density Lipoprotein (HDL) Functionality in Metabolic Syndrome

Metabolic Syndrome X

Australia
Shire

Active, not recruiting

Study of Long Term Safety and Clinical Outcomes of Idursulfase IT and Elaprase Treatment in Pediatric Participants Who Have Completed Study HGT-HIT-094

Hunter Syndrome

United States, Spain, Canada, United Kingdom, Mexico, Australia, France
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
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
NeuroVia, Inc.

Withdrawn

Safety, Pharmacokinetics and Pharmacodynamics of NV1205 in Pediatric Male Subjects With Adrenoleukodystrophy

X-Linked Adrenoleukodystrophy

Australia, Argentina, United Kingdom, Colombia, Chile, France, Russian Federation, Ukraine
bluebird bio

Active, not recruiting

Long-term Follow-up of Participants With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product

Cerebral Adrenoleukodystrophy (CALD) | Adrenoleukodystrophy (ALD) | X-Linked Adrenoleukodystrophy (X-ALD)

United States, Argentina, Australia, Brazil, France, Germany, Netherlands, United Kingdom, Italy
Takeda

Available

Post-trial Access Program of Idursulfase-IT Along With Elaprase in Children With Hunter Syndrome

Hunter Syndrome

United States, Australia, Mexico, Spain, United Kingdom
Shire

Completed

Study of Intrathecal Idursulfase-IT Administered in Conjunction With Elaprase® in Pediatric Patients With Hunter Syndrome and Early Cognitive Impairment (AIM-IT)

Hunter Syndrome

Spain, United States, Canada, United Kingdom, France, Australia, Mexico
Bayside Health

Unknown

Sympathetic Activity in Individuals With the Metabolic Syndrome: Benefits of Lifestyle Interventions

Metabolic Syndrome X

Australia
Medical University of Graz

Completed

Evaluation of an App for Smartphones for People With a Bipolar Affective Disorder

Bipolar Affective Disorder

Austria
bluebird bio

Completed

A Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy (CALD)

Cerebral Adrenoleukodystrophy (CALD)

United States, Germany, Argentina, Australia, France, United Kingdom
The University of Queensland

Recruiting

The TELO-SCOPE Study: Attenuating Telomere Attrition With Danazol. Is There Scope to Dramatically Improve Health Outcomes for Adults and Children With Pulmonary Fibrosis

Pulmonary Fibrosis | Dyskeratosis Congenita | Telomere Shortening | Telomere Disease

Australia
Pfizer

Active, not recruiting

Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy

Muscular Dystrophy, Duchenne

United States, Australia
Spark Therapeutics
Children's Hospital of Philadelphia; University of Pittsburgh; St. James's Hospital... and other collaborators

Terminated

Hemophilia B Gene Therapy With AAV8 Vector

Hemophilia B

United States, Australia
CSL Behring

Completed

A Safety, Efficacy and Pharmacokinetics Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Children With Hemophilia B

Hemophilia B

Germany, Australia, Italy, Israel, Czech Republic, Spain, France, Canada, Austria, Russian Federation
Spark Therapeutics

Completed

Lead-in Study to Collect Prospective Efficacy and Safety Data of Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Participants

Hematologic Diseases | Hemophilia A | Blood Coagulation Disorders, Inherited | Coagulation Protein Disorders | Hemorrhagic Disorders | Genetic Diseases, Inborn | Genetic Diseases, X-Linked | Blood Coagulation Disorder | Factor VIII Deficiency

United States, Australia, Thailand, Canada
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
Hospital Rudolfstiftung
Oesterreichische Muskelforschung

Completed

Myocardial Involvement in Carriers of Duchenne Muscular Dystrophy: An MRI-study

Carrier of Duchenne Muscular Dystrophy

Austria
Pfizer

Completed

A Gene Therapy Study for Hemophilia B

Hemophilia B

United States, Australia
Cooperative International Neuromuscular Research...
Washington University School of Medicine; Children's National Research Institute and other collaborators

Completed

Comparative Study of Clinical Endpoint in DMD: Handheld Myometry (HHM) Versus CINRG Quantitative Measurement System (CQMS)

Duchenne Muscular Dystrophy

United States, Australia, Italy
Kyowa Kirin, Inc.
Kyowa Kirin Co., Ltd.

Completed

Efficacy and Safety of Burosumab (KRN23) Versus Oral Phosphate and Active Vitamin D Treatment in Pediatric Patients With X Linked Hypophosphatemia (XLH)

X-Linked Hypophosphatemia

Korea, Republic of, Canada, United States, Australia, Japan, United Kingdom, Sweden
Bioverativ Therapeutics Inc.
Swedish Orphan Biovitrum

Completed

Study of Recombinant Coagulation Factor IX Fc Fusion Protein, BIIB029, in Previously Treated Pediatric Participants With Hemophilia B (Kids B-LONG)

Hemophilia B

United States, United Kingdom, South Africa, Netherlands, Ireland, 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
Kyowa Kirin Pharmaceutical Development Ltd

Active, not recruiting

Study to Assess the Safety, Tolerability, Pharmacokinetics and Efficacy of Burosumab in Patients Less Than 1 Year of Age

X-linked Hypophosphatemia (XLH)

Sweden, Spain, United Kingdom, Italy, Austria, France
Percheron Therapeutics

Recruiting

A Study of ATL1102 or Placebo in Participants With Non-ambulatory Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy

Turkey, United Kingdom, Australia, Serbia, Bulgaria
GlaxoSmithKline

Completed

Study In Patients With Dyslipidaemia

Metabolic Syndrome X

Spain, Argentina, Australia, Latvia, India, Mexico, Pakistan, Slovakia, Costa Rica, New Zealand, Romania, Russian Federation, Chile
Peter MacCallum Cancer Centre, Australia

Unknown

Safety Study of Anti LewisY Chimeric Antigen Receptor in Myeloma, Acute Myeloid Leukemia or Myelodysplastic Syndrome

Multiple Myeloma | Myelodysplastic Syndrome | Acute Myeloid Leukaemia

Australia
Pixium Vision SA

Completed

Restoring Vision With the Intelligent Retinal Implant System (IRIS V1)in Patients With Retinal Dystrophy (IRIS-1)

Retinitis Pigmentosa | Choroideremia | Cone Rod Dystrophy

Austria, France, Germany
Weill Medical College of Cornell University
Bayer; University Hospital, Bonn

Completed

Females With Severe or Moderate Hemophilia A or B: an International Multi-center Study

Hemophilia A | Hemophilia B

United States, Israel, Australia, Italy, Sweden, Germany, Japan, Netherlands, Taiwan
Cooperative International Neuromuscular Research...

Completed

Study of Daily Pentoxifylline as a Rescue Treatment in Duchenne Muscular Dystrophy

Muscular Dystrophy, Duchenne

United States, Canada, Argentina, Australia, Israel, Italy
ReveraGen BioPharma, Inc.
University of Pittsburgh; Cooperative International Neuromuscular Research...

Completed

Long-term Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)

Duchenne Muscular Dystrophy

United States, Canada, United Kingdom, Sweden, Australia, Israel
Pfizer

Recruiting

Safety and Effectiveness of Giroctocogene Fitelparvovec or Fidanacogene Elaparvovec in Patients With Hemophilia A or B Respectively

Hemophilia A | Hemophilia B

Australia, United States, Turkey
Amicus Therapeutics

Completed

A Phase I, Randomized, Single-Blind, Four-Period Cross-Over, Placebo-Controlled, Dose-Escalation Study to Evaluate the Safety and Pharmacokinetics of Single Oral Doses of GR181413A/AT1001 in Healthy Japanese Subjects

Fabry Disease

Australia

Clinical Trials on X-linked Lymphoproliferative Syndrome in Australia

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in United States

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Rare Diseases

Drug Interventions

Dietary Supplements

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