Clinical Trials on Dehydrated Hereditary Stomatocytosis in Japan

Total 56 results

Takeda

Recruiting

A Survey of Icatibant in Pediatric Participants With Hereditary Angioedema

Hereditary Angioedema (HAE)

Japan
Takeda

Recruiting

A Survey of Lanadelumab in Participants With Hereditary Angioedema

Hereditary Angioedema (HAE)

Japan
Shire

Completed

A Study of Icatibant for Acute Attacks of Hereditary Angioedema in Japanese Participants

Hereditary Angioedema (HAE)

Japan
Takeda

Completed

A Study of Icatibant (TAK-667) in Japanese Children and Teenagers With Acute Attacks of Hereditary Angioedema

Hereditary Angioedema

Japan
BioCryst Pharmaceuticals

Completed

Study to Evaluate the Efficacy and Safety of BCX7353 as an Oral Treatment for the Prevention of HAE Attacks in Japan (APeX-J)

Hereditary Angioedema, HAE

Japan
Astellas Pharma Inc

Completed

Development of Visual Function Evaluation Method (2)

Retinitis Pigmentosa

Japan
Janssen Pharmaceutical K.K.

Completed

An Observational Study of Japanese Participants With X-linked Retinitis Pigmentosa

X-Linked Retinitis Pigmentosa

Japan
Janssen Pharmaceutical K.K.

Recruiting

A Study of AAV5-hRKp.RPGR for the Treatment of Japanese Participants With X-linked Retinitis Pigmentosa

X-Linked Retinitis Pigmentosa

Japan
Astellas Pharma Inc

Completed

Development of Visual Function Evaluation Method

Retinitis Pigmentosa

Japan
Shire

Completed

Study of C1 Inhibitor (Human) for the Prevention of Angioedema Attacks and Treatment of Breakthrough Attacks in Japanese Subjects With Hereditary Angioedema (HAE)

Hereditary Angioedema (HAE)

Japan
Takeda

Completed

Expanded Access Program With Lanadelumab for Japanese People With Hereditary Angioedema

Hereditary Angioedema

Japan
Tohoku University

Completed

Pharmacokinetic Study on N-acetylneuraminic Acid

Hereditary Inclusion Body Myopathy | Nonaka Myopathy

Japan
Nobelpharma

Completed

Efficacy Confirmation Study of NPC-09

GNE Myopathy | Nonaka Disease | Distal Myopathy With Rimmed Vacuoles (DMRV) | Hereditary Inclusion Body Myopathy (hIBM)

Japan
Shire
Takeda

Completed

FIRAZYR General Drug Use-Results Survey (Japan)

Hereditary Angioedema (HAE)

Japan
Shire

Completed

Efficacy and Safety of Lanadelumab (SHP643) in Japanese Participants With Hereditary Angioedema (HAE)

Hereditary Angioedema (HAE)

Japan
Nobelpharma

Completed

Long-term Trial of Topical Sirolimus to Angiofibroma in Patient With Tuberous Sclerosis Complex

Plaque | Tuberous Sclerosis | Angiofibroma | Hypomelanotic Macule

Japan
Nobelpharma

Completed

Phase III Trial of Topical Formulation of Sirolimus to Skin Lesions in Patients With Tuberous Sclerosis Complex (TSC)

Plaque | Tuberous Sclerosis | Angiofibroma | Hypomelanotic Macule

Japan
Mitsubishi Tanabe Pharma Corporation

Completed

Clinical Study of TA-650 in Patients With Behcet's Disease (BD) With Special Lesions

Behcet Syndrome | Behcet's Disease | Neuro-Behcet's Disease

Japan
CSL Behring

Completed

CSL312 (Garadacimab) in the Prevention of Hereditary Angioedema Attacks

Hereditary Angioedema

United States, Germany, Japan, Hungary, Canada, Netherlands, Israel
CENTOGENE GmbH Rostock

Completed

Epidemiological Analysis for Hereditary Angioedema Disease (EHA)

Abdominal Pain | Functional Abdominal Pain

United Kingdom, Germany, Turkey, Poland, Italy, Japan
AstraZeneca

Completed

Selumetinib Paediatric NF1 Japan Study

Neurofibromatosis Type 1

Japan
Nobelpharma

Terminated

A Long-term Study of NPC-12G Gel in Neurofibromatosis Type I

Neurofibromatosis

Japan
Nobelpharma

Active, not recruiting

Clinical Study of NPC-12Y Gel in Patients With Skin Lesions Associated With TSC

Tuberous Sclerosis Complex

Japan
Novartis Pharmaceuticals

Completed

An Extention Study of Safety of Canakinumab in Japanese Patients With Periodic Fever Syndromes

Periodic Fevers Syndrome

Japan
CSL Behring

Active, not recruiting

Long-term Safety and Efficacy of CSL312 (Garadacimab) in the Prophylactic Treatment of Hereditary Angioedema Attacks

Hereditary Angioedema

Australia, Canada, United States, New Zealand, Germany, Hong Kong, Netherlands, Taiwan, Czechia, Israel, Hungary, Japan, Russian Federation, Spain
KalVista Pharmaceuticals, Ltd.

Recruiting

PK Subtrial in Adolescent Patients With HAE Type I or II Participating in the KVD900-302 Trial

Hereditary Angioedema

United States, Bulgaria, France, Greece, Israel, Netherlands, Spain, United Kingdom, Australia, Germany, South Africa, Slovakia, Austria, New Zealand, Romania, Canada, Japan
KalVista Pharmaceuticals, Ltd.

Completed

A Phase III, Crossover Trial Evaluating the Efficacy and Safety of KVD900 for On-Demand Treatment of Angioedema Attacks in Adolescent and Adult Patients With Hereditary Angioedema (HAE)

Hereditary Angioedema

United States, Australia, Bulgaria, Canada, France, Germany, Greece, Hungary, Israel, Italy, Japan, Netherlands, New Zealand, North Macedonia, Poland, Portugal, Puerto Rico, Romania, Slovakia, Spain, United Kingdom
KalVista Pharmaceuticals, Ltd.

Recruiting

An Open-label Extension Trial to Evaluate the Long-term Safety of KVD900 for On-Demand Treatment of Angioedema Attacks in Adolescent and Adult Patients With Hereditary Angioedema (HAE)

Hereditary Angioedema

United States, Bulgaria, France, Greece, Hungary, Israel, Italy, Netherlands, New Zealand, North Macedonia, Poland, Spain, United Kingdom, Australia, Germany, South Africa, Austria, Portugal, Slovakia, Romania, Canada, Saudi Arabia, Japa...
Novartis Pharmaceuticals

Completed

Efficacy and Safety Study of Canakinumab Administered for 6 Months (24 Weeks) in Japanese Patients With Cryopyrin-associated Periodic Syndromes Followed by an Extension Phase

Familial Cold Autoinflammatory Syndrome | Muckle-Wells Syndrome | Neonatal Onset Multisystem Inflammatory Disease | Cryopyrin-associated Periodic Syndromes

Japan
R-Tech Ueno, Ltd.

Terminated

Phase III Efficacy and Safety Clinical Study of UF-021 for Treatment of Retinitis Pigmentosa

Retinitis Pigmentosa

Japan
Clementia Pharmaceuticals Inc.

Terminated

An Efficacy and Safety Study of Palovarotene for the Treatment of MO (MO-Ped)

Exostoses, Multiple Hereditary

United States, Spain, Canada, Japan, Italy, Portugal, United Kingdom, Turkey, Australia, Belgium, France, Netherlands
AbbVie (prior sponsor, Abbott)
Eisai Co., Ltd.

Completed

A Study of Adalimumab in Japanese Subjects With Intestinal Behçet's Disease

Intestinal Behçet's Disease

Japan
Shire
Takeda Development Center Americas, Inc.

Completed

A Study of Long-Term Safety and Efficacy of Lanadelumab for Prevention of Acute Attacks of Non-histaminergic Angioedema With Normal C1-Inhibitor

Angioedema

United States, France, Netherlands, Spain, Hungary, Italy, Canada, Poland, Japan, Germany
Baxalta now part of Shire

Completed

Phase 1 Dose Escalation, Single Dose Study to Assess Safety and Pharmacokinetics of BAX930 in Hereditary Thrombotic Thrombocytopenic Purpura (TTP)

Hereditary Thrombotic Thrombocytopenic Purpura (TTP)

United States, Germany, United Kingdom, Switzerland, Austria, Japan, Poland
Shire

Completed

Multicenter Study of HGT-1110 Administered Intrathecally in Children With Metachromatic Leukodystrophy (MLD) (IDEAMLD)

Metachromatic Leukodystrophy (MLD)

Australia, Denmark, Japan, France, Germany
Takeda
Takeda Development Center Americas, Inc.

Completed

A Study of Lanadelumab in Teenagers and Adults to Prevent Acute Attacks of Non-histaminergic Angioedema With Normal C1-Inhibitor (C1-INH)

Angioedema

United States, Spain, Netherlands, Hungary, Italy, Canada, Germany, France, Japan, Poland
Novartis Pharmaceuticals

Completed

Study of Efficacy and Safety of Canakinumab in Patients With Hereditary Periodic Fevers

Hereditary Periodic Fevers

Belgium, Italy, Japan, Russian Federation, Turkey, Spain, Germany, United Kingdom, France, United States, Ireland, Israel, Canada, Hungary, Netherlands, Switzerland
Alnylam Pharmaceuticals

Completed

APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis

Amyloid Neuropathies | Amyloid Neuropathies, Familial | TTR-mediated Amyloidosis | Amyloidosis, Hereditary | Amyloidosis, Hereditary, Transthyretin-Related | Familial Amyloid Polyneuropathies

United States, Canada, Argentina, Australia, Bulgaria, Cyprus, Italy, Japan, Malaysia, Mexico, Netherlands, Portugal, Spain, Sweden, Germany, France, Brazil, Turkey, Korea, Republic of, Taiwan, United Kingdom
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
Ionis Pharmaceuticals, Inc.

Recruiting

A Study to Evaluate the Safety and Efficacy of ION373 in Patients With Alexander Disease (AxD)

Alexander Disease

United States, United Kingdom, Australia, Canada, Israel, Italy, Japan, Netherlands
AstraZeneca
Merck Sharp & Dohme LLC

Active, not recruiting

Pharmacokinetics, Safety and Efficacy of the Selumetinib Granule Formulation in Children Aged ≥1 to <7 Years With NF1-related Symptomatic, Inoperable PN (SPRINKLE)

Neurofibromatosis Type 1

United States, Spain, Germany, Netherlands, Russian Federation, Italy, Japan
Shire
Takeda Development Center Americas, Inc.

Active, not recruiting

An Efficacy and Safety Study of HGT-1110 in Participants With Metachromatic Leukodystrophy

Metachromatic Leukodystrophy (MLD)

Denmark, Germany, Japan, Australia, France, Brazil, Czechia
Chugai Pharmaceutical

Not yet recruiting

Phase 1b Trial of RAY121 in Immunological Diseases (RAINBOW Trial)

Antiphospholipid Syndrome (APS) | Immune Thrombocytopenia (ITP) | Dermatomyositis (DM) | Bullous Pemphigoid (BP) | Behçet's Syndrome (BS) | Immune-mediated Necrotizing Myopathy (IMNM)

United States, Japan
Hoffmann-La Roche

Recruiting

A Study To Evaluate The Efficacy, Safety, Pharmacokinetics, And Pharmacodynamics Of Satralizumab In Participants With Thyroid Eye Disease

Thyroid Eye Disease | TED

Japan, Australia, Singapore, United States, Argentina, Germany, Italy, Hong Kong
Shire

Terminated

Natural History Study of Children With Metachromatic Leukodystrophy

Metabolic Diseases | Brain Diseases | Central Nervous System Diseases | Nervous System Diseases | Demyelinating Diseases | Genetic Diseases, Inborn | Metabolism, Inborn Errors | Lysosomal Storage Diseases | Lipid Metabolism Disorders | Sphingolipidoses | Hereditary Central Nervous System Demyelinating Diseases and other conditions

United States, Denmark, France, Argentina, Belgium, Brazil, Canada, Germany, Japan, Turkey
Immunovant Sciences GmbH

Recruiting

Study to Assess Batoclimab in Participants With Active Thyroid Eye Disease

Thyroid Eye Disease

United States, Italy, Poland, United Kingdom, Canada, Israel, Germany, Japan, Georgia
Alnylam Pharmaceuticals

Active, not recruiting

HELIOS-A: A Study of Vutrisiran (ALN-TTRSC02) in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)

Transthyretin Amyloidosis | Amyloidosis, Hereditary

United States, Argentina, Australia, Belgium, Brazil, Bulgaria, Canada, Cyprus, France, Germany, Greece, Italy, Japan, Korea, Republic of, Malaysia, Mexico, Netherlands, Portugal, Spain, Sweden, Taiwan, United Kingdom
AstraZeneca
Merck Sharp & Dohme LLC

Active, not recruiting

Efficacy and Safety of Selumetinib in Adults With NF1 Who Have Symptomatic, Inoperable Plexiform Neurofibromas (KOMET)

Neurofibromatosis 1 | Plexiform Neurofibroma (PN)

United States, Canada, France, Italy, Germany, China, United Kingdom, Spain, Japan, Russian Federation, Australia, Poland, Brazil
Shire
Takeda Development Center Americas, Inc.

Active, not recruiting

A Study of Intrathecal SHP611 in Children With Metachromatic Leukodystrophy (EMBOLDEN)

Metachromatic Leukodystrophy (MLD)

United States, Canada, Belgium, Israel, Netherlands, United Kingdom, Germany, Spain, Japan, Brazil, Argentina, France, Greece, Italy
Amgen

Completed

A Phase 3 Randomized, Double-blind Study to Evaluate the Efficacy and Safety of Apremilast (CC-10004) in Subjects With Active Behçet's Disease

Behçet's Syndrome

United States, Korea, Republic of, Israel, Italy, Turkey, France, Germany, Greece, Lebanon, Japan

Clinical Trials on Dehydrated Hereditary Stomatocytosis in Japan

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Kenya

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations