Clinical Trials on Genetic Diseases, X-Linked in Ireland

Total 395 results

IRCCS Fondazione Stella Maris
Azienda USL Toscana Nord Ovest; University of Dublin, Trinity College; Meyer...

Recruiting

Biomarkers in Rett Syndrome (BIRS)

Rett Syndrome

Italy, Ireland
Seaside Therapeutics, Inc.

Terminated

An Open Label Extension Study of STX209 in Subjects With Fragile X Syndrome

Fragile X Syndrome

United States
Seaside Therapeutics, Inc.

Completed

Safety, Tolerability and Efficacy Study of STX209 in Subjects With Fragile X Syndrome

Fragile X Syndrome

United States
Seaside Therapeutics, Inc.

Completed

Biomarker Testing and DNA Collection in Subjects Participating in Protocol 22001

Fragile X Syndrome

United States
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
Seaside Therapeutics, Inc.

Completed

Efficacy and Safety Study of STX209 (Arbaclofen) for the Treatment of Social Withdrawal in Children With Fragile X Syndrome (Harbor-C)

Fragile X Syndrome

United States
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
Seaside Therapeutics, Inc.

Completed

Efficacy and Safety Study of STX209 (Arbaclofen) for Social Withdrawal in Adolescents and Adults With Fragile X Syndrome (Harbor-A)

Fragile X Syndrome

United States
Seaside Therapeutics, Inc.

Terminated

An Open Label Extension Study in Subjects With Fragile X Syndrome (209FX303)

Fragile X Syndrome

United States
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
Rocket Pharmaceuticals Inc.

Completed

Natural History of Danon Disease

Danon Disease

Ireland, Czechia, France, Italy, Spain
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
Hoffmann-La Roche

Completed

A Study of RO4917523 in Patients With Fragile X Syndrome

Fragile X Syndrome

France, Spain, United States, Peru, Canada, United Kingdom, Chile, Argentina, Mexico, Sweden
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
University College, London

Terminated

A Factor IX Gene Therapy Study (FIX-GT) (FIX-GT)

Hemophilia B

Ireland, United Kingdom, Italy, United States
Swedish Orphan Biovitrum
Cerner Enviza

Active, not recruiting

An International Study to Evaluate the Real-world Effectiveness and Usage of Alprolix in Patients With Haemophilia B (B-MORE)

Hemophilia B

Czechia, Greece, Ireland, Italy, Norway, Saudi Arabia, Spain, Sweden, United Kingdom
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
Swedish Orphan Biovitrum
IQVIA Pvt. Ltd

Terminated

An 18-month Low-interventional Study to Assess Joint Health in Haemophilia A and B Patients on Prophylaxis With Efmoroctocog Alfa or Eftrenonacog Alfa (JOIN-us)

Hemophilia A | Hemophilia B

Bulgaria, Croatia, Czechia, France, Hungary, Ireland, Italy, Romania, Slovenia, Spain, United Kingdom
Baxalta now part of Shire

Completed

BAX 326 (rFIX) Continuation Study

Hemophilia B

Taiwan, Colombia, Argentina, Brazil, Bulgaria, Chile, Czechia, India, Ireland, Italy, Japan, Poland, Romania, Russian Federation, Sweden, Ukraine, United Kingdom
Catabasis Pharmaceuticals

Completed

Phase III Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy (PolarisDMD)

Muscular Dystrophy, Duchenne

United States, Canada, Australia, United Kingdom, Germany, Ireland, Israel, Sweden
Sarepta Therapeutics, Inc.

Active, not recruiting

A Study to Compare Safety and Efficacy of a High Dose of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) (MIS51ON)

Muscular Dystrophy, Duchenne

United States, Korea, Republic of, India, Slovenia, Turkey, United Kingdom, Germany, Switzerland, Spain, Czechia, France, Serbia, Taiwan, Netherlands, Mexico, Colombia, Denmark, New Zealand, Norway, Greece, Hungary, Ireland, Italy, Jo... and more
Bioverativ, a Sanofi company
Swedish Orphan Biovitrum

Completed

Study to Determine the Safety and Efficacy of rFIXFc in Previously Untreated Males With Severe Hemophilia B (PUPs B-LONG)

Hemophilia B

United States, Italy, Ireland, New Zealand, United Kingdom, Australia, Netherlands, Denmark, Poland, Sweden, France
CSL Behring

Active, not recruiting

HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients

Hemophilia B

United States, Belgium, Netherlands, Denmark, Germany, Ireland, Sweden, United Kingdom
PedNet Haemophilia Research Foundation
Lund University Hospital

Recruiting

The European Paediatric Network for Haemophilia Management ( PedNet Registry) (PedNet)

Factor IX Deficiency | Factor VIII Deficiency

Norway, Austria, Belgium, Canada, Czechia, Denmark, Finland, France, Germany, Greece, Ireland, Israel, Italy, Netherlands, Portugal, Spain, Sweden, Switzerland, United Kingdom
Sarepta Therapeutics, Inc.

Active, not recruiting

Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD) (ESSENCE)

Duchenne Muscular Dystrophy

United States, Korea, Republic of, Italy, United Kingdom, Canada, Poland, Spain, France, Germany, Belgium, Australia, Czechia, Serbia, Greece, Argentina, Bulgaria, Denmark, Hungary, India, Ireland, Israel, Mexico, Russian Federation, ...
Santhera Pharmaceuticals

Terminated

A Phase III Double-blind Study With Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Taking Glucocorticoid Steroids (SIDEROS)

Duchenne Muscular Dystrophy (DMD)

United States, Spain, Netherlands, Sweden, Germany, France, Belgium, United Kingdom, Italy, Ireland, Switzerland, Austria, Bulgaria, Hungary, Israel
NYU Langone Health
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Completed

Health-related Quality of Life in Rare Kidney Stone

Cystinuria | Primary Hyperoxaluria | Dent Disease | Adenine Phosphoribosyl Transferase Deficiency

United States, Iceland
Mayo Clinic
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaborators

Recruiting

Rare Kidney Stone Consortium Patient Registry (RKSC)

Cystinuria | Primary Hyperoxaluria | Dent Disease | APRT Deficiency

United States, Iceland
Freeline Therapeutics

Completed

AAV Gene Therapy Screening/Observational Protocol (ECLIPSE) (ECLIPSE)

Hemophilia B | Blood Coagulation Disorders, Inherited | Blood Coagulation Disorder | Haematologic Disease

United States, United Kingdom, Australia, Netherlands, Austria, France, Ireland, Italy, Turkey, Canada, Germany, South Africa
Kyowa Kirin Pharmaceutical Development Ltd

Active, not recruiting

Registry for Patients With X-Linked Hypophosphatemia (XLH Registry)

X-Linked Hypophosphatemia

Spain, Italy, United Kingdom, France, Belgium, Denmark, Germany, Hungary, Portugal, Norway, Switzerland, Israel, Netherlands, Czechia, Bulgaria, Sweden, Slovenia, Ireland, Latvia, Slovakia
Bioverativ Therapeutics Inc.

Completed

Long-Term Safety and Efficacy of rFIXFc in the Prevention and Treatment of Bleeding Episodes in Previously Treated Participants With Hemophilia B (B-YOND)

Severe Hemophilia B

United States, Italy, Belgium, Australia, Brazil, Hong Kong, India, Ireland, Japan, South Africa, United Kingdom, Netherlands, Sweden, Canada, Poland, China, France, Germany
National Cancer Institute (NCI)

Recruiting

Study of Voicing My CHOiCES as a Tool for Advanced Care Planning in Young Adults With Cancer

Cancer | HIV | Chronic Granulomatous Disease | Dock 8 Deficiency

United States
Novo Nordisk A/S

Completed

Observational Study on Safety of Room Temperature Stable NovoSeven® in Patients With Haemophilia A or B

Congenital Bleeding Disorder | Haemophilia A | Haemophilia B

Belgium, Germany, France, United Kingdom, Italy, Hungary, Austria, Sweden, Iran, Islamic Republic of, Argentina, Ireland, Poland, Portugal, Slovakia
Novo Nordisk A/S

Completed

PROPACT: Retrospective Prophylaxis Patient Case Collection (PROPACT)

Congenital Bleeding Disorder | Haemophilia A With Inhibitors | Haemophilia B With Inhibitors

Slovakia, Spain, Argentina, Germany, Croatia, Italy, Canada, United Kingdom, Ireland, Sweden, France, United States, Czech Republic, Switzerland
Kyowa Kirin Pharmaceutical Development Ltd

Completed

Study of the Anti-FGF23 Antibody, Burosumab, in Adults With XLH

X-linked Hypophosphatemia

United Kingdom, France, Ireland, Italy
CENTOGENE GmbH Rostock
Shire Human Genetic Therapies, Inc.

Completed

Stroke in Young Fabry Patients (sifap1): Frequency of Fabry Disease in Young Stroke Patients (sifap1)

Cerebrovascular Accident

Austria, Belgium, Croatia, Finland, France, Georgia, Germany, Ireland, Italy, Malta, Poland, Portugal, Spain, United Kingdom
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
Idorsia Pharmaceuticals Ltd.

Completed

Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease (MODIFY)

Fabry Disease

United States, Spain, Belgium, Australia, Austria, Canada, Germany, Ireland, Italy, Netherlands, Norway, Poland, Switzerland, United Kingdom
Astellas Pharma Inc

Completed

Distribution of Tacrolimus in Skin, Atopic Dermatitis

Dermatitis, Atopic

Ireland
Kyowa Kirin, Inc.
Kyowa Kirin Co., Ltd.

Completed

Study of KRN23 in Adults With X-linked Hypophosphatemia (XLH)

X-linked Hypophosphatemia

United States, Korea, Republic of, Japan, Ireland, United Kingdom, France, Italy
Mayo Clinic

Recruiting

Prospective Research Rare Kidney Stones (ProRKS) (ProRKS)

Cystinuria | Hyperoxaluria | Dent Disease | Lowe Syndrome | Adenine Phosphoribosyltransferase Deficiency

United States, Canada, Iceland, Israel
DS Biopharma

Completed

Efficacy and Steroid Sparing Potential Study of DGLA Cream in Early Childhood Patients With Moderate to Severe Atopic Dermatitis

Atopic Dermatitis

Ireland
University College Cork

Completed

Short-term Topical Application to Prevent Atopic Dermatitis (STOP AD)

Eczema | Food Allergy | Eczema, Infantile | Eczema Atopic Dermatitis

Ireland
St. James's Hospital, Ireland
University of Dublin, Trinity College; Science Foundation Ireland

Recruiting

The Role Of Neutrophil Proteases As Global Regulators Of Il-1 Family Cytokine Activity In Skin Disorders (NEUTROPRO)

Atopic Dermatitis | Psoriasis Vulgaris | Ichthyosis Vulgaris

Ireland
Genzyme, a Sanofi Company

Recruiting

Fabry Disease Registry & Pregnancy Sub-registry

Fabry Disease

Croatia, United States, Argentina, Australia, Belgium, Bulgaria, Canada, Germany, Hong Kong, India, Indonesia, Korea, Republic of, Kuwait, Malaysia, Philippines, Poland, Romania, Singapore, Slovakia, Taiwan, Thailand, United Kingdom, Vietn... and more
University College Cork
Wellcome Trust; Health Research Board, Ireland; Health Service Executive, Ireland

Recruiting

Assessing SleeP IN Infants With Atopic Dermatitis by Longitudinal Evaluation (SPINDLE)

Sleep Disturbance | Atopic Dermatitis

Ireland
Beaumont Hospital

Unknown

Randomised Controlled Trial of Needle Free Access Devices in Preventing Hemodialysis Blood Stream Infection (TEGO)

Central Line Infection | Hemodialysis Catheter Blood Stream Infection | Dialysis Line Thrombosis

Ireland
University College Dublin
National Children's Research Centre; Our Lady's Children's Hospital, Crumlin

Completed

Intranasal Fentanyl Versus Intravenous Morphine in the Treatment of Severe Painful Sickle Cell Crises in Children

Pain | Sickle Cell Disease

Ireland
Women and Infants Hospital of Rhode Island

Completed

Effect of Video Education on Patients' Knowledge and Attitudes of Privacy in Prenatal Genetics

Pregnancy Related | Aneuploidy | Genetic Disease

United States
University of Limerick

Unknown

Steps Ahead: Optimising Physical Activity and Health in Adults With Cystic Fibrosis

Cystic Fibrosis

Ireland

Clinical Trials on Genetic Diseases, X-Linked in Ireland

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Israel

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations