Clinical Trials on X-linked Creatine Deficiency in Japan

Total 90 results

CSL Behring

Completed

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

Hemophilia B

United States, Australia, Japan, France, Philippines, Spain, Czechia, Israel, Canada, Germany, Austria, Bulgaria, Italy, Malaysia, South Africa
Genzyme, a Sanofi Company

Completed

A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients With Inhibitors (ATLAS-INH)

Hemophilia A | Hemophilia B

United States, Australia, Canada, China, France, Korea, Republic of, Malaysia, Turkey, Ukraine, United Kingdom, Germany, India, Italy, Japan, South Africa, Spain, Taiwan
Sarepta Therapeutics, Inc.
Hoffmann-La Roche

Active, not recruiting

A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) (EMBARK)

Duchenne Muscular Dystrophy

United States, Japan, Taiwan, Belgium, Spain, Germany, Hong Kong, Italy, United Kingdom
Pfizer

Active, not recruiting

Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy

United States, Spain, Belgium, Korea, Republic of, Taiwan, Canada, Australia, Germany, Japan, France, Italy, United Kingdom, Russian Federation, Israel, Switzerland
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
Pfizer

Recruiting

Open-Label Extension Study of Marstacimab in Hemophilia Participants With or Without Inhibitors

Hemophilia A | Hemophilia B

Hong Kong, Spain, Serbia, Turkey, United States, China, Mexico, South Africa, India, Japan, Croatia, Korea, Republic of, Oman, Taiwan, Italy, Canada, France
Hoffmann-La Roche

Completed

Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy

United States, Belgium, Canada, Australia, Japan, France, Netherlands, Spain, Argentina, Germany, Italy, United Kingdom, Sweden
Chiesi Farmaceutici S.p.A.
ICON plc

Recruiting

Study to Evaluate the Safety, PK, PD, and Efficacy of PRX-102 in Japanese Patients With Fabry Disease (RISE)

Fabry Disease

Japan
Eli Lilly and Company

Terminated

A Study of Tadalafil for Duchenne Muscular Dystrophy

Muscular Dystrophy, Duchenne

Germany, United States, Spain, Belgium, France, Italy, Taiwan, Netherlands, Turkey, Korea, Republic of, Argentina, Canada, Japan, Puerto Rico, Russian Federation, United Kingdom
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
Amicus Therapeutics

Completed

A Study to Describe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease With Enzyme Replacement Therapy.

Fabry Disease

Japan, Taiwan, Turkey, United States, Brazil
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
Pfizer

Active, not recruiting

A Study to Evaluate Prospective Efficacy and Safety Data of Current FIX Prophylaxis Replacement Therapy in Adult Hemophilia B Subjects (FIX:C≤2%) or Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Subjects (FVIII:C≤1%)

Hemophilia A | Hemophilia B

United States, Canada, Spain, Australia, Korea, Republic of, Taiwan, Brazil, Italy, Japan, Turkey, Germany, United Kingdom, Saudi Arabia, France, Sweden, Belgium, Greece, Israel
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
Pfizer

Active, not recruiting

A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B (BENEGENE-2)

Hemophilia B

Germany, United Kingdom, Canada, Australia, Taiwan, Japan, France, Turkey, Korea, Republic of, United States, Brazil, Greece, Saudi Arabia, Sweden
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, Italy, Saudi Arabia, Israel, Korea, Republic of, Austria, Germany, Japan, Australia, India, Slovakia, France, United States, Brazil, Argentina
Pfizer

Active, not recruiting

Study of the Efficacy and Safety PF-06741086 in Adult and Teenage Participants With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B

Hemophilia A | Hemophilia B

United States, Spain, Canada, China, Korea, Republic of, Hong Kong, Serbia, Taiwan, Turkey, France, Oman, Saudi Arabia, Japan, India, South Africa, Mexico, Croatia, Italy, Bulgaria, Russian Federation
Bioverativ Therapeutics Inc.
Swedish Orphan Biovitrum

Completed

Study of Recombinant Factor IX Fc Fusion Protein (rFIXFc) in Participants With Hemophilia B

Severe Hemophilia B

Sweden, United States, France, Italy, Russian Federation, United Kingdom, Germany, China, Poland, Japan, Australia, Brazil, Canada, India, South Africa, Hong Kong, Belgium
Novo Nordisk A/S

Completed

Safety, Efficacy and Pharmacokinetics of NNC-0156-0000-0009 in Previously Treated Children With Haemophilia B. (paradigm™5)

Congenital Bleeding Disorder | Haemophilia B

United States, Canada, United Kingdom, France, Malaysia, Brazil, Croatia, Germany, Italy, Japan, Taiwan
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
Novo Nordisk A/S

Completed

Pharmacogenetic Testing of Saliva Samples From Patients With Five or More Exposure Days to rFVIIa Analogue in the Adept™2 Trial

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

United States, Serbia, Malaysia, Thailand, Greece, Japan, Romania
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 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, Serbia, Canada, Croatia, Algeria, Bosnia and Herzegovina, Bulgaria, Denmark 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
Novo Nordisk A/S

Completed

A Trial Evaluating the Efficacy and Safety of Prophylactic Administration of Concizumab in Haemophilia A and B Patients With Inhibitors (explorer™4)

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

United States, Austria, Spain, Japan, Croatia, Italy, United Kingdom, Israel, Sweden, Ukraine, Malaysia, Greece, Denmark, Canada
Novo Nordisk A/S

Completed

Efficacy and Safety of NNC-0156-0000-0009 During Surgical Procedures in Subjects With Haemophilia B (paradigm™ 3)

Congenital Bleeding Disorder | Haemophilia B

United States, Netherlands, Austria, Spain, Germany, Macedonia, The Former Yugoslav Republic of, Taiwan, United Kingdom, Italy, Malaysia, Thailand, France, Greece, Turkey, Japan, South Africa, Romania
PTC Therapeutics

Completed

Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy

Nervous System Diseases | Genetic Diseases, Inborn | Genetic Diseases, X-Linked | Muscular Diseases | Neuromuscular Diseases | Muscular Dystrophies | Muscular Disorders, Atrophic | Muscular Dystrophy, Duchenne | Musculoskeletal Disease

United States, Taiwan, India, Korea, Republic of, Thailand, Australia, Brazil, Bulgaria, Canada, China, Hong Kong, Malaysia, Mexico, Poland, Puerto Rico, Russian Federation, Turkey, Japan
Novo Nordisk A/S

Completed

Safety and Efficacy of NNC-0156-0000-0009 After Long-Term Exposure in Patients With Haemophilia B: An Extension to Trials NN7999-3747 and NN7999-3773 (paradigm™ 4)

Congenital Bleeding Disorder | Haemophilia B

United States, Netherlands, Austria, Spain, Germany, Macedonia, The Former Yugoslav Republic of, Taiwan, United Kingdom, Italy, Malaysia, Thailand, France, Greece, Russian Federation, Turkey, Japan, South Africa, Romania
Novo Nordisk A/S

Completed

Safety and Efficacy of 3 Different Doses of Long Acting Factor VII in Haemophilia A or B Patients With Inhibitors

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

France, United States, Sweden, Serbia, Turkey, United Kingdom, Malaysia, Brazil, Former Serbia and Montenegro, Japan, South Africa
Amicus Therapeutics

Completed

Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry Disease

Fabry Disease

France, Austria, United States, Belgium, Japan, United Kingdom, Denmark, Brazil, Australia, Italy
Novo Nordisk A/S

Completed

Safety and Efficacy of Nonacog Beta Pegol (N9-GP) in Previously Untreated Patients With Haemophilia B (paradigm™6)

Congenital Bleeding Disorder | Haemophilia B

Spain, United Kingdom, Australia, Taiwan, France, Malaysia, United States, Austria, Canada, Israel, Japan, Thailand, Algeria, Argentina
Novo Nordisk A/S

Completed

Safety and Efficacy of NNC-0156-0000-0009 in Haemophilia B Patients (paradigm™ 2)

Congenital Bleeding Disorder | Haemophilia B

United States, Netherlands, Germany, Macedonia, The Former Yugoslav Republic of, United Kingdom, Italy, Malaysia, Hungary, Thailand, France, Russian Federation, Turkey, Japan, South Africa, Canada
Novo Nordisk A/S

Completed

A Study Following People With Haemophilia A and B, With or Without Inhibitors, When on Usual Treatment (Explorer™6) (explorer™6)

Haemophilia A With Inhibitors | Haemophilia B With Inhibitors | Haemophilia A | Haemophilia B

France, United States, India, Mexico, United Kingdom, Netherlands, Sweden, Korea, Republic of, Spain, Switzerland, Serbia, Turkey, Bulgaria, Italy, Japan, Poland, South Africa, Croatia, Algeria, Australia, Bosnia and Herzegovina, ... and more
Novo Nordisk A/S

Completed

Haemophilia Patients With Inhibitors Being Treated for Acute Joint Bleeds

Congenital Bleeding Disorder | Haemophilia A | Haemophilia B

United States, Spain, Taiwan, Turkey, Poland, Croatia, Italy, Malaysia, Brazil, United Kingdom, Hungary, Israel, Thailand, Japan, South Africa, Canada, France, Argentina
Amicus Therapeutics

Completed

Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease

Fabry Disease

United States, Austria, United Kingdom, Argentina, Australia, Belgium, Brazil, Canada, Denmark, Egypt, France, Italy, Japan, Spain, Turkey
Amicus Therapeutics

Recruiting

A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease

Fabry Disease

Spain, United States, Japan, Australia, France, Portugal
Novo Nordisk A/S

Completed

Efficacy and Safety of NNC 0078-0000-0007 in Patients With Congenital Haemophilia and Inhibitors (adept™2)

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

Taiwan, United Kingdom, Thailand, Serbia, Croatia, Italy, Poland, Romania, Hungary, Malaysia, United States, Austria, Brazil, Greece, Japan, Puerto Rico, Russian Federation, South Africa, Turkey
Sanofi

Recruiting

A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease (CARAT)

Fabry Disease

Italy, United States, Austria, China, Czechia, Denmark, France, Germany, Greece, Japan, Korea, Republic of, Netherlands, Norway, Spain, Taiwan, Turkey, United Kingdom, Canada, Poland
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
Sanofi

Recruiting

A Study to Evaluate the Effect of Venglustat Tablets on Neuropathic and Abdominal Pain in Male and Female Participants ≥16 Years of Age With Fabry Disease (PERIDOT)

Fabry Disease

China, Finland, Germany, Norway, United Kingdom, United States, Denmark, Austria, Greece, Romania, Argentina, Brazil, Canada, Japan, Mexico, Poland, Italy, France, Turkey, Australia, Switzerland

Clinical Trials on X-linked Creatine Deficiency in Japan

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