Clinical Trials on Genetic Diseases, Inborn in Denmark

Total 9308 results

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
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
Insmed Incorporated

Completed

Extension Study of Liposomal Amikacin for Inhalation in Cystic Fibrosis (CF) Patients With Chronic Pseudomonas Aeruginosa (Pa) Infection

Cystic Fibrosis

France, Poland, United Kingdom, Germany, Spain, Belgium, Bulgaria, Serbia, Denmark, Greece, Netherlands, Hungary, Ireland, Slovakia, Canada, Italy, Austria
Genzyme, a Sanofi Company

Recruiting

International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry

Gaucher Disease | Cerebroside Lipidosis Syndrome | Glucocerebrosidase Deficiency Disease | Glucosylceramide Beta-Glucosidase Deficiency Disease

Hungary, Croatia, United States, Australia, Belgium, Brazil, Bulgaria, Canada, Denmark, Germany, Greece, Hong Kong, India, Indonesia, Italy, Jordan, Korea, Republic of, Kuwait, Lebanon, Malaysia, Pakistan, Philippines, Poland, Saudi Arabia and more
Bayer

Completed

BAY81-8973 Pediatric Safety and Efficacy Trial

Haemophilia A

Spain, Russian Federation, United States, Hungary, Italy, Mexico, Norway, Romania, Bulgaria, Canada, Argentina, Denmark, Israel, Poland, Ireland, Latvia, Lithuania
Wave Life Sciences Ltd.

Terminated

Open-label Extension Study to Evaluate the Safety and Tolerability of WVE-120101 in Patients With Huntington's Disease

Huntington Disease

Denmark, Australia, Canada, France, United Kingdom, Germany, Poland
Wave Life Sciences Ltd.

Terminated

Open-label Extension Study to Evaluate the Safety and Tolerability of WVE-120102 in Patients With Huntington's Disease

Huntington Disease

Denmark, Australia, Canada, France, United Kingdom, Germany, Poland
Zogenix International Limited, Inc., a subsidiary...

Active, not recruiting

A Study to Investigate the Efficacy and Safety of ZX008 (Fenfluramine Hydrochloride) as an Adjunctive Therapy in Children and Adults With Lennox-Gastaut Syndrome

Lennox Gastaut Syndrome

United States, Australia, Belgium, Canada, Denmark, France, Germany, Italy, Japan, Mexico, Netherlands, Poland, Spain, Sweden
Wave Life Sciences Ltd.

Terminated

Safety and Tolerability of WVE-120101 in Patients With Huntington's Disease (PRECISION-HD1)

Huntington's Disease

Denmark, Canada, Australia, United Kingdom, France, Germany, Poland
Vertex Pharmaceuticals Incorporated

Completed

A Study Evaluating the Safety and Efficacy of VX-440 Combination Therapy in Subjects With Cystic Fibrosis

Cystic Fibrosis

United States, Canada, United Kingdom, Australia, Spain, Germany, Italy, Austria, Belgium, Denmark
Fulcrum Therapeutics

Active, not recruiting

Efficacy and Safety of Losmapimod in Treating Participants With Facioscapulohumeral Muscular Dystrophy (FSHD) (REACH)

Facioscapulohumeral Muscular Dystrophy (FSHD)

United States, Spain, Italy, Denmark, Canada, Germany, France, Netherlands, United Kingdom
Pfizer

Completed

Transthyretin Amyloidosis Outcome Survey (THAOS) (THAOS)

Transthyretin Amyloidosis | Transthyretin Gene Mutations

United States, France, Spain, Taiwan, Israel, Korea, Republic of, Denmark, Canada, Netherlands, Japan, Sweden, Brazil, Mexico, Argentina, Portugal, Belgium, Bulgaria, Cyprus, Germany, Italy, Malaysia, Romania, Saudi Arabia, Turkey, United...
Proteostasis Therapeutics, Inc.

Completed

Study Assessing the Safety, Tolerability, Pharmacokinetics, Food Effect, and Drug-Drug Interactions of PTI-801 in Healthy Volunteers, and Safety, Tolerability, and Pharmacokinetics of PTI-801 in Subjects With Cystic Fibrosis

Cystic Fibrosis | Healthy Volunteer

United States, Canada, Denmark, Germany, Sweden
Wave Life Sciences Ltd.

Recruiting

Study of WVE-003 in Patients With Huntington's Disease

Huntington Disease

Canada, Spain, Australia, Denmark, Netherlands, Poland, United Kingdom, France, Germany, Italy
Bayer

Completed

A Trial Investigating Safety and Efficacy of Treatment With BAY94-9027 in Severe Hemophilia A (PROTECT-VIII)

Hemophilia A

Korea, Republic of, Taiwan, Turkey, Singapore, United States, Netherlands, Austria, Denmark, United Kingdom, Belgium, Italy, France, Japan, Germany, Israel, Poland, Canada, Norway, Romania, Colombia
University Medical Center Goettingen

Completed

Comparative Effectiveness Research to Assess the Use of Primary ProphylacTic Implantable Cardioverter Defibrillators in Europe (EU-CERT-ICD)

Myocardial Infarction | Coronary Artery Disease | Dilated Cardiomyopathy | Implantable Defibrillator User

Hungary, Poland, Belgium, Bulgaria, Croatia, Czechia, Denmark, Finland, Germany, Greece, Netherlands, Slovakia, Spain, Sweden, Switzerland, United Kingdom
Pfizer

Terminated

Study Evaluating Safety Of Patients Switching To ReFacto AF In Usual Care Settings

Hemophilia A

Netherlands, Spain, Greece, Italy, France, Belgium, Germany, Austria, Sweden, Hungary, Finland, United Kingdom, Denmark, Romania
CSL Behring

Completed

Extension Study of Zemaira® i.v. Administration in Subjects With Emphysema Due to alpha1-proteinase Inhibitor Deficiency.

Emphysema | Alpha 1-proteinase Inhibitor Deficiency

Australia, Canada, Czech Republic, Denmark, Estonia, Finland, Germany, Ireland, Poland, Romania, Sweden
Vertex Pharmaceuticals Incorporated

Completed

A Study to Evaluate the Efficacy and Safety of Lumacaftor in Combination With Ivacaftor in Subjects With CF, Homozygous for the F508del-CFTR Mutation

Cystic Fibrosis

United States, France, United Kingdom, Germany, Belgium, Australia, Denmark, Canada, Sweden
Ultragenyx Pharmaceutical Inc
Mereo BioPharma

Completed

A Study in Adult Patients With Type I, III or IV Osteogenesis Imperfecta Treated With BPS804 (Asteroid)

Osteogenesis Imperfecta Type III | Osteogenesis Imperfecta Type IV | Osteogenesis Imperfecta, Type I

United States, Canada, Denmark, France, United Kingdom
Genzyme, a Sanofi Company

Active, not recruiting

Long-term Safety and Efficacy Study of Fitusiran in Patients With Hemophilia A or B, With or Without Inhibitory Antibodies to Factor VIII or IX (ATLAS-OLE)

Hemophilia

China, Hungary, Turkey, Canada, Korea, Republic of, United States, Australia, Denmark, France, Germany, India, Ireland, Israel, Italy, Japan, Malaysia, South Africa, Taiwan, Ukraine, United Kingdom
Alnylam Pharmaceuticals

Active, not recruiting

APOLLO-B: A Study to Evaluate Patisiran in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy)

Transthyretin Amyloidosis (ATTR) With Cardiomyopathy

United States, Australia, Brazil, Bulgaria, France, Italy, Japan, Mexico, Netherlands, Taiwan, United Kingdom, Belgium, Czechia, Denmark, New Zealand, Sweden, Korea, Republic of, Argentina, Chile, Hong Kong, Portugal
Baxalta now part of Shire
Takeda Development Center Americas, Inc.

Active, not recruiting

A Study of PEGylated Recombinant Factor VIII (BAX855) in Previously Untreated Young Children With Severe Hemophilia A

Hemophilia A

Spain, United States, Denmark, Italy, Taiwan, Malaysia, Belgium, Thailand, Turkey, Austria, Bulgaria, Canada, Finland, France, Germany, Hong Kong, Hungary, Korea, Republic of, Netherlands, Norway, Singapore, Ukraine, United Kingdom
Vico Therapeutics B. V.

Recruiting

A Safety and Pharmacokinetics Trial of VO659 in SCA1, SCA3 and HD

Huntington Disease | Spinocerebellar Ataxia Type 3 | Spinocerebellar Ataxia Type 1

Israel, Germany, United Kingdom, Poland, Denmark, Netherlands, France
Quince Therapeutics S.p.A.
Biotrial

Not yet recruiting

To Evaluate the Effects of EryDex in Patients With A-T (NEAT)

Ataxia Telangiectasia

Belgium, United States, Denmark, Germany, Italy, Norway, Poland, Spain, United Kingdom
Vertex Pharmaceuticals Incorporated

Completed

A Study to Evaluate the Safety and Efficacy of Long-term Treatment With TEZ/IVA in CF Participants With an F508del CFTR Mutation

Cystic Fibrosis

United States, Germany, United Kingdom, Canada, Australia, Switzerland, France, Ireland, Belgium, Denmark, Poland
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
Alnylam Pharmaceuticals

Active, not recruiting

HELIOS-B: A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy

Transthyretin Amyloidosis (ATTR) With Cardiomyopathy

United States, Poland, Argentina, Australia, Belgium, France, Germany, Japan, Netherlands, Portugal, Spain, Sweden, United Kingdom, Canada, Czechia, Denmark, Hungary, Ireland, Norway, Korea, Republic of, Austria, Croatia, Israel, Latvia, Lithuan... and more
BioCryst Pharmaceuticals

Completed

Study of BCX7353 as a Treatment for Attacks of Hereditary Angioedema

Hereditary Angioedema (HAE)

Austria, Denmark, France, Germany, Hungary, Israel, Italy, North Macedonia, Poland, Romania, Switzerland, United Kingdom
Vertex Pharmaceuticals Incorporated

Completed

Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy

Cystic Fibrosis

United States, Spain, Ireland, Belgium, Netherlands, United Kingdom, Australia, France, Denmark, Germany, Italy, Canada
Vertex Pharmaceuticals Incorporated

Completed

Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Lumacaftor in Combination With Ivacaftor

Cystic Fibrosis

United States, France, United Kingdom, Germany, Belgium, Denmark, Canada, Australia, Sweden
Baxalta now part of Shire
Baxalta Innovations GmbH, now part of Shire

Completed

ADVATE/ ADYNOVI Hemophilia A Outcome Database (AHEAD) (AHEAD)

Hemophilia A

Czechia, Switzerland, Denmark, Canada, Australia, Austria, France, United Kingdom, Italy, Sweden, Belgium, Brazil, China, Colombia, Greece, Hungary, Norway, Poland, Portugal, Russian Federation, Slovenia, Spain
CSL Behring

Completed

Zemaira in Subjects With Emphysema Due to Alpha1-Proteinase Inhibitor Deficiency

Emphysema | Alpha1-proteinase Inhibitor Deficiency

Australia, Russian Federation, United States, Denmark, Estonia, Finland, Germany, Ireland, Poland, Romania, Sweden, Canada, Czech Republic
Alnylam Pharmaceuticals

Completed

ENVISION: A Study to Evaluate the Efficacy and Safety of Givosiran (ALN-AS1) in Patients With Acute Hepatic Porphyrias (AHP)

Acute Hepatic Porphyria | Acute Intermittent Porphyria | Porphyria, Acute Intermittent | Acute Porphyria | Hereditary Coproporphyria (HCP) | Variegate Porphyria (VP) | ALA Dehydratase Deficient Porphyria (ADP)

United States, Spain, United Kingdom, Korea, Republic of, Australia, Bulgaria, Canada, Denmark, Finland, France, Germany, Italy, Japan, Mexico, Netherlands, Poland, Sweden, Taiwan
Wave Life Sciences Ltd.

Terminated

Safety and Tolerability of WVE-120102 in Patients With Huntington's Disease (PRECISION-HD2)

Huntington's Disease

United States, Denmark, Canada, Australia, United Kingdom, France, Germany, Poland
Vertex Pharmaceuticals Incorporated

Completed

A Study Evaluating the Long-term Safety of VX-445 Combination Therapy

Cystic Fibrosis

United States, Spain, Ireland, Israel, Australia, United Kingdom, Germany, Canada, Denmark, Poland, Switzerland
Bayer

Terminated

BAY79-4980 Compared to rFVIII-FS in Previously Treated Patients With Severe Hemophilia A

Hemophilia A

Belgium, Italy, Spain, United States, Canada, Switzerland, Estonia, Taiwan, France, Chile, Netherlands, United Kingdom, Austria, New Zealand, South Africa, Romania, Croatia, Israel, Germany, Poland, Australia, Denmark, Turkey, Argentina, Lithuan... and more
Vertex Pharmaceuticals Incorporated

Terminated

A Study Evaluating the Long Term Safety and Efficacy of VX-659 Combination Therapy

Cystic Fibrosis

United States, Spain, Ireland, Australia, Israel, United Kingdom, Germany, Canada, Denmark, Poland, Switzerland
Vertex Pharmaceuticals Incorporated

Completed

A Phase 3 Study of VX-445 Combination Therapy in Cystic Fibrosis (CF) Subjects Heterozygous for F508del and a Gating or Residual Function Mutation (F/G and F/RF Genotypes)

Cystic Fibrosis

United States, Spain, Ireland, Belgium, Netherlands, United Kingdom, Australia, France, Canada, Denmark, Germany, Italy, Israel
Grifols Therapeutics LLC

Enrolling by invitation

Long Term Safety of Alpha1-Proteinase Inhibitor in Subjects With Alpha1 Antitrypsin Deficiency (SPARTA-OLE)

Pulmonary Emphysema in Alpha-1 Antitrypsin Deficiency

United States, New Zealand, Denmark, Australia, Finland, Estonia, Canada, Poland, Russian Federation, Sweden, France
Spruce Biosciences

Active, not recruiting

A Ph2b to Evaluate Clinical Efficacy and Safety of Tildacerfont in Adult CAH

Congenital Adrenal Hyperplasia

United States, Australia, Brazil, Canada, Denmark, Estonia, Germany, Ireland, Italy, Korea, Republic of, Latvia, Lithuania, Netherlands, Poland, Romania, Spain, Sweden, Switzerland, Turkey, United Kingdom
Bayer

Completed

Trial to Evaluate the Efficacy and Safety of a New Full Length Recombinant Human FVIII for Hemophilia A (Leopold I)

Hemophilia A | Blood Coagulation Disorders

Spain, Taiwan, Austria, United States, Germany, Thailand, United Kingdom, Italy, Turkey, Croatia, Serbia, Poland, Israel, Sweden, Indonesia, Argentina, Norway, South Africa, India, Hong Kong, Denmark, Pakistan
Vertex Pharmaceuticals Incorporated

Completed

A Phase 3 Study of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)

Cystic Fibrosis

United States, Spain, Ireland, Australia, Israel, United Kingdom, Germany, Switzerland, Canada, Denmark, Poland
Vertex Pharmaceuticals Incorporated

Completed

A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor

Cystic Fibrosis

United States, United Kingdom, Germany, Spain, Switzerland, Canada, Netherlands, Italy, Ireland, Sweden, France, Denmark
Bayer
Novartis

Completed

Study to Evaluate the Safety and Efficacy of Ciprofloxacin (Inhaled) in Patients With Cystic Fibrosis

Cystic Fibrosis

United States, Israel, Germany, Canada, Australia, Sweden, Denmark, Norway, United Kingdom
ML Bio Solutions, Inc.

Recruiting

Study to Evaluate the Efficacy and Safety of BBP-418 (Ribitol) in Patients With Limb Girdle Muscular Dystrophy 2I (LGMD2I) (Fortify)

Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I)

United States, Netherlands, United Kingdom, Denmark, Australia, Italy, Norway
University of Leicester

Unknown

Chemotherapy Plus Surgery in Treating Children at Risk of or With Stage I Wilms' Tumor

Kidney Cancer

France, Netherlands, Sweden, Norway, Denmark, Portugal, Belgium, Croatia, Czech Republic, Greece, Italy, Poland, Slovenia, Spain, United Arab Emirates
Shire
Jerini AG

Completed

EASSI - Evaluation of the Safety of Self-Administration With Icatibant (EASSI)

Hereditary Angioedema

Spain, Argentina, Austria, Denmark, France, Germany, Israel, Italy, Switzerland, United Kingdom
Teva Branded Pharmaceutical Products R&D, Inc.
Nuvelution TS Pharma, Inc.

Completed

Alternatives for Reducing Tics in Tourette Syndrome (TS): A Study of TEV-50717 (Deutetrabenazine) for the Treatment of Tourette Syndrome in Children and Adolescents (ARTISTS1)

Tourette Syndrome

United States, Canada, Denmark, Russian Federation, Serbia, Spain
Copenhagen University Hospital, Hvidovre

Recruiting

Fatty Liver Disease in Nordic Countries (FLINC)

Non-Alcoholic Fatty Liver Disease

Denmark

Clinical Trials on Genetic Diseases, Inborn in Denmark

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Bahrain

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations