Clinical Trials on Renal Tubular Transport, Inborn Errors in Sweden

Total 64 results

Kyowa Kirin Pharmaceutical Development Ltd

Completed

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
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
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
Region Örebro County

Recruiting

Genetic Causes of Familial Hypercholesterolemia

Familial Hypercholesterolemia

Sweden
University Hospital, Linkoeping

Recruiting

Clinical Decision Support for Familial Hypercholesterolemia (CDS-FH)

Hypercholesterolemia, Familial | Clinical Decision Support

Sweden
Karolinska Institutet

Enrolling by invitation

Evaluation of a Digiphysical Screening Method to Identify and Diagnose Familial Hypercholesterolemia (DigiLipids)

Hypercholesterolemia | Familial Hypercholesterolemia

Sweden
Genzyme, a Sanofi Company

Completed

A Study of the Safety and Efficacy of Patients With Familial Hypercholesterolaemia Taking Colesevelam as add-on Therapy to Their Existing Medication

Familial Hypercholesterolemia

Netherlands, France, Germany, Sweden, United Kingdom
Swedish Orphan Biovitrum

Completed

Efficacy and Safety of Once Daily Dosing Compared to Twice Daily Dosing of Nitisinone in HT-1 (HT-1)

Hereditary Tyrosinemia, Type I

Belgium, Denmark, France, Germany, Sweden
Region Stockholm

Enrolling by invitation

Effects and Importance of Epinephrine/Adrenalin Deficiency in CAH

Congenital Adrenal Hyperplasia

Sweden
Vastra Gotaland Region
Karolinska University Hospital; Göteborg University; Uppsala University Hospital

Completed

Studying Lipids as Potential Biomarkers in Patients With Fabry Disease

Fabry Disease

Sweden
Arcturus Therapeutics, Inc.

Recruiting

Study for Adolescent and Adult Participants With Ornithine Transcarbamylase Deficiency to Evaluate Safety and Tolerability of ARCT-810

OTC Deficiency | Ornithine Transcarbamylase Deficiency | OTCD

Spain, Belgium, United Kingdom, Sweden
Recordati Rare Diseases

Recruiting

Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods Approach (PROTECT)

Propionic Acidemia | Methylmalonic Acidemia

France, Spain, Italy, United Kingdom, Norway, Germany, Sweden
Sanofi
Regeneron Pharmaceuticals

Completed

An 8-Week Dose-Finding Study to Evaluate the Efficacy and Safety of Alirocumab in Children and Adolescents With Heterozygous Familial Hypercholesterolemia (ODYSSEY KIDS)

Hypercholesterolaemia

Norway, Russian Federation, Sweden, South Africa, Czechia, United States, Canada, France, Netherlands, Spain
Ionis Pharmaceuticals, Inc.

Active, not recruiting

A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRX) Administered to Adults With Familial Chylomicronemia Syndrome (FCS) Previously Treated With Volanesorsen

Familial Chylomicronemia Syndrome

United States, Canada, Sweden
Shire

Completed

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

Fabry Disease

Finland, Sweden, Germany, Belgium, Czechia, Denmark
Amgen

Completed

Reduction of LDL-C With PCSK9 Inhibition in Heterozygous Familial Hypercholesterolemia Disorder Study-2 (RUTHERFORD-2)

Hyperlipidemia

Canada, France, Netherlands, United Kingdom, New Zealand, South Africa, Spain, United States, Australia, Germany, Sweden, Hong Kong, Switzerland, Norway
Sanofi
Regeneron Pharmaceuticals

Completed

An Efficacy and Safety Study of Alirocumab in Children and Adolescents With Heterozygous Familial Hypercholesterolemia

Hypercholesterolaemia

Czechia, France, Denmark, Netherlands, Austria, Mexico, Spain, United States, Argentina, Brazil, Bulgaria, Canada, Finland, Hungary, Italy, Lebanon, Norway, Poland, Russian Federation, Slovenia, South Africa, Sweden, Taiwan, Turkey
Pfizer

Completed

Study To Evaluate The Safety And Efficacy Of Torcetrapib/Atorvastatin In Subjects With Familial Hypercholerolemia

Hyperlipidemia | Hypercholesterolemia, Familial

Australia, Canada, United States, France, South Africa, Sweden, Norway, Denmark, Iceland
University Medicine Greifswald
Wuerzburg University Hospital; University of Göttingen

Recruiting

Prednisolone Versus Colchicine for Acute Gout in Primary Care (COPAGO)

Acute Gout

Germany
The Medicines Company

Completed

Trial to Evaluate the Effect of Inclisiran Treatment on Low Density Lipoprotein Cholesterol (LDL-C) in Subjects With Heterozygous Familial Hypercholesterolemia (HeFH) (ORION-9)

Elevated Cholesterol | Heterozygous Familial Hypercholesterolemia

United States, Canada, Czechia, Denmark, Netherlands, South Africa, Spain, Sweden
Sanofi
Regeneron Pharmaceuticals

Completed

Efficacy and Safety of Alirocumab (SAR236553/REGN727) Versus Placebo on Top of Lipid-Modifying Therapy in Patients With Heterozygous Familial Hypercholesterolemia Not Adequately Controlled With Their Lipid-Modifying Therapy (ODYSSEY FH I)

Hypercholesterolemia

United States, Austria, Canada, Czech Republic, Denmark, France, Israel, Netherlands, Norway, Russian Federation, South Africa, Spain, Sweden, United Kingdom
Ionis Pharmaceuticals, Inc.

Active, not recruiting

A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) in Participants With Familial Chylomicronemia Syndrome (FCS)

Familial Chylomicronemia Syndrome

Spain, United Kingdom, France, Canada, United States, Italy, Netherlands, Norway, Portugal, Slovakia, Sweden
Novartis Pharmaceuticals

Completed

Canakinumab in the Treatment of Acute Gout Flares and Prevention of New Flares in Patients Unable to Use Non-steroidal Anti-inflammatory Drugs (NSAIDs) and/or Colchicine Including a 12 Weeks Extension and an Open-label 48 Weeks Extension Study (β-RELIEVED)

Acute Gout

Colombia, Switzerland, Germany, Russian Federation, Singapore, Australia, Belgium, Canada, Estonia, Guatemala, Latvia, Lithuania, Mexico, Norway, Poland, Sweden, Ukraine
Swedish Orphan Biovitrum

Completed

Long Term Safety Study of Orfadin Treatment in HT-1 Patients in Standard Clinical Care (OPAL)

Hereditary Tyrosinemia, Type I

Belgium, Denmark, France, Germany, Austria, Czechia, Finland, Hungary, Ireland, Italy, Netherlands, Norway, Poland, Portugal, Spain, Sweden, United Kingdom
Regeneron Pharmaceuticals

Completed

PREventative Study Against URate-Lowering Drug-Induced Gout Exacerbations (PRE-SURGE 2)

Gout

Germany, Taiwan, India, South Africa, Indonesia
Alnylam Pharmaceuticals

Completed

Patisiran in Patients With Hereditary Transthyretin-mediated Amyloidosis (hATTR Amyloidosis) Disease Progression Post-Liver Transplant

Amyloidosis, Familial | Transthyretin Amyloidosis

France, Germany, Italy, Portugal, Spain, Sweden, United Kingdom
Kastle Therapeutics, LLC

Completed

A Study of the Safety and Efficacy of Two Different Regimens of Mipomersen in Patients With Familial Hypercholesterolemia and Inadequately Controlled Low-Density Lipoprotein Cholesterol (FOCUS FH)

Hypercholesterolemia | Heterozygous Familial

United States, Italy, Poland, Ukraine, United Kingdom, Germany, Hong Kong, Taiwan, Spain, Argentina, Belgium, Israel, Malaysia, South Africa, Turkey, Netherlands, Korea, Republic of, Canada, New Zealand, Russian Federation, Brazil, Austral... and more
Ionis Pharmaceuticals, Inc.
Akcea Therapeutics

Completed

A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) Administered to Patients With Familial Chylomicronemia Syndrome (FCS) (BALANCE)

Familial Chylomicronemia Syndrome

United States, Israel, Spain, Italy, United Kingdom, Netherlands, Canada, Hungary, Sweden, France, Norway, Portugal, Slovakia
Shire

Terminated

A Study of Replagal in Treatment-naïve Adults With Fabry Disease

Fabry Disease

Spain, Greece, Germany, Portugal, Canada, Finland, Poland, Sweden
BioMarin Pharmaceutical

Completed

Observational Study on the Long Term Safety of Kuvan® Treatment in Patients With Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or BH4 Deficiency (KAMPER)

Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or Tetrahydrobiopterin (BH4) Deficiency

Sweden, France, Italy, Spain, Germany, Netherlands, Austria, Slovakia, Portugal
Genzyme, a Sanofi Company

Completed

A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease to Evaluate Once Daily Versus Twice Daily Dosing (EDGE)

Gaucher Disease

United States, Sweden, India, Netherlands, Austria, Japan, China, Serbia, France, Croatia, Australia, Portugal, Canada, Brazil, Greece, Romania, Russian Federation
Haukeland University Hospital
Karolinska Institutet; University Hospitals Bristol and Weston NHS Foundation... and other collaborators

Completed

Dynamic Hormone Diagnostics in Endocrine Disease (ultradian)

Congenital Adrenal Hyperplasia | Acromegaly | Growth Hormone Deficiency | Cushing Syndrome | Primary Hyperaldosteronism | Adrenal Insufficiency

Norway, Sweden, Greece, United Kingdom
Regeneron Pharmaceuticals

Completed

Review of Safety Using Rilonacept in Preventing Gout Exacerbations (RE-SURGE)

Gout

United States, Taiwan, South Africa, India, Germany, Indonesia
PTC Therapeutics

Completed

A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy (MIT-E)

Mitochondrial Diseases | Drug Resistant Epilepsy | Leigh Disease | Leigh Syndrome | Mitochondrial Encephalopathy (MELAS) | Pontocerebellar Hypoplasia Type 6 (PCH6) | Alpers Disease | Alpers Syndrome

United States, Spain, United Kingdom, Canada, France, Italy, Poland, Sweden, Japan
Sanofi

Active, not recruiting

Safety and Efficacy of Eliglustat With or Without Imiglucerase in Pediatric Patients With Gaucher Disease (GD) Type 1 and Type 3 (ELIKIDS)

Gaucher's Disease Type III | Gaucher's Disease Type I

Canada, Argentina, France, Italy, Japan, Russian Federation, Spain, Sweden, Turkey, United Kingdom
Enzyvant Therapeutics GmBH

Completed

Farber Disease Natural History Study

Farber Disease | Farber's Disease | Farber Lipogranulomatosis | Acid Ceramidase Deficiency | Ceramidase Deficiency | N-Laurylsphingosine Deacylase Deficiency | ASAH1 Mutation

United States, Egypt, Canada, Italy, Turkey, Argentina, Germany, India, Sweden
BioMarin Pharmaceutical

Completed

Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP)

Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy Syndrome)

United States, France, Australia, Germany, Ireland, Austria, Belgium, Italy, Lithuania, Netherlands, Portugal, Sweden, United Kingdom
Novartis Pharmaceuticals

Completed

Trial to Assess the Effect of Long Term Dosing of Inclisiran in Subjects With High CV Risk and Elevated LDL-C (ORION-8)

Elevated Cholesterol | Homozygous Familial Hypercholesterolemia | Heterozygous Familial Hypercholesterolemia | ASCVD

United States, Canada, Czechia, Denmark, Germany, Hungary, Netherlands, Poland, South Africa, Spain, Sweden, Ukraine, United Kingdom
Daiichi Sankyo, Inc.

Terminated

Efficacy and Safety of the ACAT Inhibitor CS-505 (Pactimibe) for Reducing the Progression of Carotid Artery Disease. This Study is Also Known as CAPTIVATE.

Atherosclerosis | Heterozygous Familial Hypercholesterolemia

United States, United Kingdom, Spain, South Africa, Israel, Canada, Netherlands, Norway, Sweden
Cyclo Therapeutics, Inc.

Completed

Safety and Efficacy of Intravenous Trappsol Cyclo (HPBCD) in Niemann-Pick Type C Patients

Niemann-Pick Disease, Type C1

Israel, Sweden, United Kingdom
Intellia Therapeutics

Recruiting

Long-Term Follow-Up (LTFU) of Subjects Dosed With NTLA-2001

Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy | Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy | Wild-Type Transthyretin Cardiac Amyloidosis

United Kingdom, New Zealand, Sweden
Amicus Therapeutics

Active, not recruiting

A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With LOPD

Pompe Disease (Late-onset)

United States, Germany, Taiwan, Belgium, Australia, Slovenia, Spain, Netherlands, Hungary, Japan, Austria, United Kingdom, France, Argentina, Canada, Sweden, Denmark, New Zealand, Greece, Korea, Republic of, Bosnia and Herzegovina, Ita... and more
Amicus Therapeutics

Completed

A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease (PROPEL)

Pompe Disease (Late-onset)

United States, Australia, Korea, Republic of, Japan, Taiwan, Belgium, Slovenia, New Zealand, United Kingdom, France, Argentina, Canada, Spain, Bosnia and Herzegovina, Germany, Sweden, Hungary, Austria, Bulgaria, Denmark, Greece, I... and more
Alnylam Pharmaceuticals

Completed

The Study of an Investigational Drug, Revusiran (ALN-TTRSC), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Whose Disease Has Continued to Worsen Following Liver Transplant

ATTR Amyloidosis | Transthyretin (TTR)-Mediated Amyloidosis | Familial Amyloidotic Polyneuropathy (FAP) | Familial Amyloid Neuropathies

France, Germany, Portugal, United Kingdom, Sweden, Spain
Boston University
National Institute of Neurological Disorders and Stroke (NINDS); Food and Drug...

Completed

The Effect of Diflunisal on Familial Amyloidosis

Familial Amyloid Polyneuropathy | Familial Amyloidosis

United States, Italy, Japan, Sweden, United Kingdom
Pfizer

Completed

An Extension of Study Fx-005 Evaluating Long-Term Safety And Clinical Outcomes Of Fx-1006A In Patients With Transthyretin Amyloid Polyneuropathy

Familial Amyloid Polyneuropathy | ATTR-PN

Sweden, Brazil, Germany, Argentina, France, Portugal
Intellia Therapeutics

Active, not recruiting

Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2001 in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv-PN) and Patients With Transthyretin Amyloidosis-Related Cardiomyopathy (ATTR-CM)

Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy | Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy | Wild-Type Transthyretin Cardiac Amyloidosis

France, United Kingdom, New Zealand, Sweden
Denali Therapeutics Inc.

Recruiting

A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II) (COMPASS)

Mucopolysaccharidosis II

United States, Spain, United Kingdom, Czechia, France, Canada, Italy, Belgium, Netherlands, Germany, Argentina, Sweden, Turkey, Colombia, Brazil
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
Pfizer

Completed

Safety and Efficacy Study of Fx-1006A in Patients With Familial Amyloidosis

Familial Amyloid Polyneuropathy

United Kingdom, Spain, United States, Argentina, Brazil, France, Germany, Portugal, Sweden

Clinical Trials on Renal Tubular Transport, Inborn Errors in Sweden

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