Clinical Trials on Genetic Diseases, X-Linked in Denmark

Total 436 results

Telethon Kids Institute
Rigshospitalet, Denmark; University of Haifa; Ariel University

Unknown

Telehealth Support to Increase Physical Activity in Rett Syndrome (ActivRett)

Rett Syndrome

Denmark, Israel
Rigshospitalet, Denmark

Completed

'Uptime' Participation Intervention in Girls and Women With Rett Syndrome

Rett Syndrome

Denmark
Rigshospitalet, Denmark

Completed

MRI in Patients With Kennedy Disease

Bulbo-Spinal Atrophy, X-Linked | Kennedy Disease

Denmark
NightstaRx Ltd, a Biogen Company

Enrolling by invitation

Long-term Safety and Efficacy Follow-up of BIIB111 for the Treatment of Choroideremia and BIIB112 for the Treatment of X-Linked Retinitis Pigmentosa (SOLSTICE)

Choroideremia | X-Linked Retinitis Pigmentosa

United States, United Kingdom, Netherlands, Germany, France, Brazil, Canada, Denmark, Finland
Karen Brorup Heje Pedersen

Completed

High Intensity Training in Patients With Spinal and Bulbar Muscular Atrophy (HIT in Kennedy)

Healthy Subjects | Spinal and Bulbar Muscular Atrophy

Denmark
Rigshospitalet, Denmark
Glostrup University Hospital, Copenhagen

Completed

Effect of Modulating the nNOS System on Cardiac, Muscular and Cognitive Function in Becker Muscular Dystrophy Patients

Becker Muscular Dystrophy

Denmark
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
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
Rigshospitalet, Denmark

Not yet recruiting

Follow-up Study on Female Carriers With DMD Gene Variants

Muscular Dystrophy | Duchenne Muscular Dystrophy | Becker Muscular Dystrophy

Denmark
Novartis Pharmaceuticals

Completed

Safety, Tolerability, and Efficacy of BVS857 in Patients With Spinal and Bulbar Muscular Atrophy

Spinal and Bulbar Muscular Atrophy

United States, Germany, Italy, Denmark
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
Biogen

Completed

Efficacy and Safety of BIIB111 for the Treatment of Choroideremia (STAR)

Choroideremia

United States, Finland, France, Denmark, Netherlands, Germany, United Kingdom, Canada
CSL Behring

Completed

Trial of AAV5-hFIX in Severe or Moderately Severe Hemophilia B

Hemophilia B

Germany, Denmark, Netherlands
NightstaRx Ltd, a Biogen Company

Completed

Natural History of the Progression of Choroideremia Study (NIGHT)

Choroideremia

Finland, United States, Canada, Denmark, Netherlands, Germany, United Kingdom, Brazil, France
Rigshospitalet, Denmark

Completed

Muscle MRI in Becker Muscular Dystrophy and in Limb-girdle Muscular Dystrophy Type 2I

Becker Muscular Dystrophy | Limb-Girdle Muscular Dystrophy Type 2I

Denmark
Swedish Orphan Biovitrum

Completed

Management of Health-Related QoL Impairment, Including Pain, Depression and Anxiety, in People With Haemophilia A and B (MIND)

Haemophilia A | Haemophilia B

Sweden, Finland, Denmark
Great Ormond Street Hospital for Children NHS Foundation...
Catholic University of the Sacred Heart; Università degli Studi di Ferrara; Universidad... and other collaborators

Recruiting

Brain Involvement in Dystrophinopathies Part 1

Duchenne Muscular Dystrophy | Becker Muscular Dystrophy

Denmark, France, Italy, Netherlands, Spain, United Kingdom
Great Ormond Street Hospital for Children NHS Foundation...
Catholic University of the Sacred Heart; Università degli Studi di Ferrara; Universidad... and other collaborators

Recruiting

Brain Involvement in Dystrophinopathies Part 2

Duchenne Muscular Dystrophy | Becker Muscular Dystrophy

Denmark, France, Italy, Spain, United Kingdom, Netherlands
Janssen Research & Development, LLC
Janssen Research & Development, LLC

Active, not recruiting

Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene

X-Linked Retinitis Pigmentosa

United States, Canada, Israel, United Kingdom, Spain, Denmark, France, Belgium, Italy, Netherlands, Switzerland
Novo Nordisk A/S

Enrolling by invitation

Post-marketing Surveillance (Use Result Surveillance) With Refixia®

Haemophilia B

Japan, Denmark
Caroline Michaela Kistorp
Rigshospitalet, Denmark; Amicus Therapeutics

Active, not recruiting

Real World Evidence Study of Danish Fabry Patients (RWE-FABRY)

Fabry Disease

Denmark
Janssen Research & Development, LLC
Janssen Research & Development, LLC

Active, not recruiting

Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene

X-Linked Retinitis Pigmentosa

Belgium, Canada, United States, Israel, United Kingdom, Spain, Denmark, France, Italy, Netherlands, Switzerland
Rigshospitalet, Denmark

Completed

Ophthalmic Findings During 10-year Enzyme Substitution of Danish Fabry Patients.

Fabry Disease

Denmark
Ulla Feldt-Rasmussen

Completed

Fabry: Renal Function During Long-term ERT by 51Cr-EDTA Clearance

Fabry Disease

Denmark
Danish Pain Research Center

Completed

Neuropathic Pain and Fabry Disease

Fabry Disease

Denmark
Novo Nordisk A/S

Enrolling by invitation

A Study Following Males With Haemophilia B on Prophylaxis With Refixia/REBINYN

Haemophilia B

Canada, United Kingdom, Czechia, Germany, Greece, Austria, Belgium, Croatia, Denmark, Finland, Norway, Portugal
Mads Peter Godtfeldt Stemmerik
Edgewise Therapeutics, Inc.

Completed

Fast Troponin as a Biomarker to Assess Exercise-induced Muscle Damage in Muscle Diseases

Becker Muscular Dystrophy | McArdle Disease | Limb-Girdle Muscular Dystrophy Type 2

Denmark
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
Genzyme, a Sanofi Company

Completed

A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without Inhibitors

Hemophilia A | Hemophilia B

United States, Australia, China, Denmark, France, Israel, Japan, Korea, Republic of, Malaysia, Turkey, Ukraine, United Kingdom, Germany, India, South Africa, Spain, Taiwan, Hungary, Italy
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
GlaxoSmithKline

Completed

A Clinical Study to Assess the Efficacy and Safety of GSK2402968 in Subjects With Duchenne Muscular Dystrophy (DMD114044)

Muscular Dystrophies

France, Denmark, Germany, Belgium, Spain, Turkey, Korea, Republic of, Brazil, Netherlands, Italy, Chile, Japan, Canada, Hungary, Russian Federation, Czechia, Poland, Norway, Argentina, Taiwan
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, ...
GlaxoSmithKline

Terminated

Open Label Study of GSK2402968 in Subjects With Duchenne Muscular Dystrophy

Muscular Dystrophies

France, Bulgaria, Denmark, Germany, Belgium, Canada, Spain, Turkey, United Kingdom, Korea, Republic of, Brazil, Netherlands, Italy, Japan, Czech Republic, Chile, Israel, Hungary, Russian Federation, Australia, Poland, Norway, Argentina and more
Novo Nordisk A/S

Completed

Prediction of the Efficacy of Activated Recombinant Human Factor VII in Adult Congenital Haemophilia A or B Patients With Inhibitors by Use of Thromboelastography

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

United States, Denmark, France, United Kingdom
Pfizer

Recruiting

A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B (BASIS KIDS)

Hemophilia A | Hemophilia B

Denmark, Taiwan, Canada, Turkey, Spain, China, United Kingdom, South Africa, Saudi Arabia, Israel, Korea, Republic of, Austria, Germany, Japan, Australia, Italy, India, Slovakia, France, United States, Brazil, Argentina
Novo Nordisk A/S

Completed

Safety of 40K Pegylated Recombinant Factor IX in Non-Bleeding Patients With Haemophilia B

Congenital Bleeding Disorder | Haemophilia B

United States, Sweden, Spain, Germany, United Kingdom, Denmark, France, Japan
Shire

Completed

Sophisticated Assessment of Disease Burden in Patients With Fabry Disease (SOPHIA)

Fabry Disease

Finland, Sweden, Germany, Belgium, Czechia, Denmark
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
Novo Nordisk A/S

Completed

Safety of NNC 0172-0000-2021 in Healthy Male Subjects and Subjects With Haemophilia A or B (Explorer 1)

Healthy | Congenital Bleeding Disorder | Haemophilia A | Haemophilia B

Austria, Sweden, Spain, Switzerland, United Kingdom, Germany, Malaysia, Denmark, South Africa, Thailand, Italy
Caroline Michaela Kistorp
Sanofi

Not yet recruiting

Fibrosis, Inflammation, Oxygenation of Renal Tissue In FabrY Disease (FORTIFY)

Chronic Kidney Diseases | Fabry Disease

Denmark
Novo Nordisk A/S

Active, not recruiting

Research Study to Look at How Well the Drug Concizumab Works in Your Body if You Have Haemophilia Without Inhibitors (explorer8)

Haemophilia A Without Inhibitors | Haemophilia B Without Inhibitors

Spain, United States, Korea, Republic of, United Kingdom, Australia, Lithuania, France, Turkey, India, Estonia, Russian Federation, Ukraine, Mexico, Japan, South Africa, Serbia, Canada, Croatia, Algeria, Bosnia and Herzegovina, ... and more
CENTOGENE GmbH Rostock

Completed

The Efficacy and Safety of Switch Between Agalsidase Beta to Agalsidase Alfa for Enzyme Replacement in Patients With Anderson-Fabry Disease (SWITCH)

Fabry Disease | Anderson-Fabry Disease | Fabry´s Disease

Argentina, Belgium, Croatia, Czechia, Denmark, France, Germany, United Kingdom
Bayer

Completed

A Phase 2/ 3 Trial to Evaluate the Efficacy and Safety of BAY86-6150

Hemophilia A, Hemophilia B

Italy, China, New Zealand, Russian Federation, Taiwan, Singapore, Bulgaria, United States, Denmark, Germany, Hungary, South Africa, United Kingdom, Romania, Colombia, Mexico, Turkey, France, India, Japan, Australia, Netherlands, Poland and more
Novo Nordisk A/S

Active, not recruiting

Research Study to Look at How Well the Drug Concizumab Works in Your Body if You Have Haemophilia With Inhibitors (explorer7)

Haemophilia A With Inhibitors | Haemophilia B With Inhibitors

Spain, Korea, Republic of, India, Australia, United Kingdom, Russian Federation, Poland, Ukraine, Thailand, Mexico, United States, Turkey, Japan, South Africa, Serbia, France, Croatia, Denmark, Algeria, Bulgaria, Malaysia, Portugal, Slovaki... and more
University of Aarhus

Completed

Characterising Pain, QoL, Body Composition, Arterial Stiffness, Muscles and Bones in Adult Persons With XLH and Healthy Controls

X-linked Hypophosphatemia | Hereditary Hypophosphatemia

Denmark
Chiesi Farmaceutici S.p.A.
Iqvia Pty Ltd; Protalix

Completed

Fabry Patient's Experience Of PegunigaLsidasE Alfa Monthly Infusion (PEOPLE)

Fabry Disease

United States, Belgium, Denmark, Italy, United Kingdom
Arne Astrup
University of Aarhus; University Hospital, Gentofte, Copenhagen; Technical University...

Completed

OPUS School Meal Study - Cross Sectional Analyses From Baseline Investigations

Metabolic Syndrome X

Denmark

Clinical Trials on Genetic Diseases, X-Linked in Denmark

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Denmark

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

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