Search clinical trials for: Inherited Diseases

Total 373022 results

Idorsia Pharmaceuticals Ltd.

Active, not recruiting

A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease

Fabry Disease

United States, United Kingdom, Norway, Germany, Australia, Canada, Spain, Switzerland, Austria, Belgium, France, Netherlands, Poland
PTC Therapeutics

Completed

A Study of PTC923 in Participants With Phenylketonuria

Phenylketonuria

Spain, United States, Australia, Germany, United Kingdom, Brazil, Denmark, Canada, France, Georgia, Italy, Mexico, Netherlands, Portugal, Turkey
Academisch Medisch Centrum - Universiteit van Amsterdam...
Stichting Metakids

Completed

Zoektocht Naar Erfelijke MetaBole Aandoening (Dutch)/ Solve The Unsolved (English) (ZOEMBA/STU)

Inherited Metabolic Disorders

Netherlands
Spark Therapeutics

Completed

A Gene Transfer Study for Hemophilia A

Hemophilia A

United States, Australia, Canada, Israel, Thailand
Innostellar Biotherapeutics Co.,Ltd

Active, not recruiting

Safety and Tolerability of LX101 for Inherited Retinal Dystrophy Associated With RPE65 Mutations

Inherited Retinal Dystrophy Associated With RPE65 Mutations

China
Johns Hopkins University
National Heart, Lung, and Blood Institute (NHLBI)

Enrolling by invitation

Derivation of Human Induced Pluripotent Stem (iPS) Cells to Heritable Cardiac Arrhythmias

Hypertrophic Cardiomyopathy (HCM) | Long QT Syndrome (LQTS) | Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT) | Inherited Cardiac Arrythmias | Brugada Syndrome (BrS) | Early Repolarization Syndrome (ERS) | Arrhythmogenic Cardiomyopathy (AC, ARVD/C) | Dilated Cardiomyopathy (DCM) | Muscular... and other conditions

United States
Radboud University Medical Center

Active, not recruiting

He-move-philia, Lifestyle Intervention for Patients With Hemophilia

Hemophilia

Netherlands
GenSight Biologics

No longer available

EAP_GS010_single Patient

Leber Hereditary Optic Neuropathy (Optic, Atrophy, Hereditary, Leber)
Emory University
Genentech, Inc.

Withdrawn

Emicizumab PUPs and Nuwiq ITI Study

Hemophilia A

United States
Takeda

Available

Post Trial Access Program of TAK-577 for Von Willebrand Disease (VWD)

Von Willebrand Disease (VWD)
Nanoscope Therapeutics Inc.

Completed

Observational Study to Assess the Reliability and Validity of the MLYMT and MLSDT

Retinitis Pigmentosa | Retinal Dystrophy

United States, Puerto Rico
Staidson (Beijing) Biopharmaceuticals Co., Ltd
Beijing Novikang Medical Technology Co., LTD

Completed

Safety and Efficacy of STSP-0601 in Adult Patients With Hemophilia A or B Without Inhibitor

Hemophilia

China
Novo Nordisk A/S

Completed

Safety and Efficacy of Activated Recombinant Human Factor VII in Haemophilia Patients With Inhibitors During and After Major Surgery

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

United States
Johns Hopkins University
National Human Genome Research Institute (NHGRI)

Enrolling by invitation

Clinical Trial of the Sequence of Cardiovascular Genetic Counseling and Testing (RESEQUENCEGC)

Genetic Counseling | Inherited Cardiac Disease

United States
Novo Nordisk A/S

Completed

A Long-term Assessment of Physical Activity, Range of Motion, and Functional Status Following Elective Orthopedic Surgery in Hemophilia Patients With Inhibitors

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

United States
Sun Yat-sen University

Completed

Minocycline Treatment in Retinitis Pigmentosa

Retinitis Pigmentosa | Inherited Retinal Dystrophy | Retina Disorder

China
Novo Nordisk A/S

Enrolling by invitation

A Study Following Males With Haemophilia A on Prophylaxis With Esperoct® (pathfinder9)

Haemophilia A

Austria, Spain, Bulgaria, Croatia, Estonia, Hungary, Italy, Lithuania, Portugal, Greece, Germany, Czechia, Slovenia, Switzerland, Slovakia
Swedish Orphan Biovitrum
IQVIA Pvt. Ltd

Terminated

An 18-month Low-interventional Study to Assess Joint Health in Haemophilia A and B Patients on Prophylaxis With Efmoroctocog Alfa or Eftrenonacog Alfa (JOIN-us)

Hemophilia A | Hemophilia B

Bulgaria, Croatia, Czechia, France, Hungary, Ireland, Italy, Romania, Slovenia, Spain, United Kingdom
Takeda

Completed

A Survey to Describe the Experience and Unmet Needs of Persons Living With Von Willebrand Disease (VWD) and Their Caregivers

Von Willebrand Disease (VWD)

Canada
BioMarin Pharmaceutical

Active, not recruiting

Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301) (BMN 270-301)

Hemophilia A

United States, Taiwan, United Kingdom, Australia, Israel, Belgium, Brazil, France, Germany, Italy, Korea, Republic of, South Africa, Spain
Takeda
Takeda

Completed

ADYNOVATE Drug Use-Results Survey

Hemophilia A

Japan
BioMarin Pharmaceutical

Active, not recruiting

Gene Therapy Study in Severe Haemophilia A Patients (270-201)

Severe Haemophilia A

United Kingdom
Zhengzhou Gensciences Inc
Haimen Gensciences lnc

Completed

An Open Label Study to Determine the Safety and Efficacy of Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection in Adolescent and Adult Patients With Hemophilia A.

Hemophilia A

China
The League of Clinical Research, Russia

Completed

Health Related Quality of Life of Youth and Young Adults With Haemophilia A (RELOQ)

Hemophilia A

Russian Federation
Rigel Pharmaceuticals

Completed

Open Label Study of R788 in the Treatment of Persistent/Chronic Immune Thrombocytopenic Purpura (ITP)

Immune Thrombocytopenic Purpura

Spain, United States, Australia, Poland, United Kingdom, Canada, Czechia, Bulgaria, Romania, Austria, Denmark, Hungary, Italy, Netherlands, Norway
Octapharma

Completed

Bleeding Incidence in VWD Patients Undergoing On-Demand Treatment

Von Willebrand Diseases

Lebanon, Croatia, United States, Hungary, Russian Federation, Ukraine, Belarus, Bulgaria
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
University of Campania "Luigi Vanvitelli"
Retina Italia Onlus

Completed

Natural History of Patients With Inherited Retinal Diseases Due to Mutations in RPE65 Gene (RPE65-NHS)

Leber Congenital Amaurosis 2 | Retinitis Pigmentosa 20

Italy
Bayer

Terminated

A Study to Learn More About Treatment With Damoctocog Alfa Pegol, How it is Used in Every Day Practice ("Real-World"), and How Satisfied People Who Receive Damoctocog Alfa Pegol Are in United States (US) Hemophilia Treatment Centers (REAL)

Hemophilia A

United States
Novo Nordisk A/S

Completed

Turoctocog Alfa in Haemophilic Italian Patients: Protection and Engagement in Recreational Activities (THIRd)

Haemophilia A

Italy
Novo Nordisk A/S

Enrolling by invitation

Post-Marketing Surveillance (Use-results Surveillance) With Esperoct®

Haemophilia A

Japan
Paul Szabolcs

Enrolling by invitation

Sequential Cadaveric Lung and Bone Marrow Transplant for Immune Deficiency Diseases (BOLT+BMT)

Chronic Granulomatous Disease (CGD) | Wiskott-Aldrich Syndrome | Severe Chronic Neutropenia | Severe Combined Immunodeficiency (SCID) | Immunodeficiency With Predominant T-cell Defect, Unspecified | Hyper IgE Syndromes | Hyper IgM Deficiencies | Mendelian Susceptibility to Mycobacterial Disease | Common...

United States
AryoGen Pharmed Co.

Completed

rFVIII-Fc (Produced by AryoGen Pharmed Co.) Pharmacokinetic Study

Severe Hemophilia A

Iran, Islamic Republic of
CSL Behring

Completed

Dose Confirmation Trial of AAV5-hFIXco-Padua

Hemophilia B

United States
St. Jude Children's Research Hospital
Children's Hospital of Philadelphia; National Heart, Lung, and Blood Institute... and other collaborators

Active, not recruiting

Dose-Escalation Study Of A Self Complementary Adeno-Associated Viral Vector For Gene Transfer in Hemophilia B

Hemophilia B

United States, United Kingdom
Staidson (Beijing) Biopharmaceuticals Co., Ltd

Completed

A Safety and Efficacy Study of STSP-0601 in Adult Patients With Hemophilia A or B With Inhibitor

Hemophilia

China
Novo Nordisk A/S

Completed

A Study to Compare the Blood Levels of Mim8 in Healthy Men When Administered With the DV3407-C1 Pen Injector or a Syringe and Cartridge

Haemophilia A

Germany
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
Novo Nordisk A/S

Completed

A Research Study Investigating Mim8 in People With Haemophilia A (FRONTIER1)

Healthy Volunteers | Haemophilia A With or Without Inhibitors

United States, Germany, Spain, Austria, Bulgaria, Italy, Japan, South Africa, Switzerland, Turkey, United Kingdom, Poland
Bayer

Completed

Study Evaluating "Real World" Treatment Pattern in Previously Treated Hemophilia A Patients Receiving KOVALTRY (Octocog Alfa) for Routine Prophylaxis (TAURUS)

Hemophilia A, Congenital

Slovenia, Italy, Germany, France, United States, Belgium, Canada, Colombia, Greece, Luxembourg, Netherlands, Spain, Taiwan
Novo Nordisk A/S

Completed

A Research Study of How a New Medicine NNC0365-3769 (Mim8) Works in the Body of Healthy People

Healthy Volunteers - Haemophilia A

Germany
Octapharma

Completed

Clinical Study to Investigate the Efficacy and Safety of Wilate During Prophylaxis in Previously Treated Patients With VWD

Von Willebrand Diseases

Lebanon, Croatia, United States, Belarus, Bulgaria, Hungary, Russian Federation, Ukraine
Instituto Grifols, S.A.

Withdrawn

FIB Grifols Congenital Deficiency for On-demand Treatment and Surgical Prophylaxis

Hypofibrinogenemia | Congenital Afibrinogenemia

India, Turkey, Lebanon, United States, Bulgaria
Imara, Inc.

Terminated

A Study of IMR-687 in Subjects With Sickle Cell Disease

Sickle Cell Disease

United States, Oman, Italy, United Kingdom, Lebanon, Greece, Morocco, Tunisia, Ghana, Kenya, Netherlands, Senegal, Uganda
Children's Hospital Los Angeles
Grifols Biologicals, LLC

Active, not recruiting

Preventing Inhibitor Recurrence Indefinitely (PRIORITY)

Hemophilia A | Immune Tolerance

United States
BioMarin Pharmaceutical

Completed

Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg (GENEr8-2)

Hemophilia A

United States
M.D. Anderson Cancer Center

Withdrawn

Pharmacologic Pretransplant Immunosuppression (PTIS) + Reduced Toxicity Conditioning (RTC) Allogeneic Stem Cell Transplantation in Inherited Hematologic Disorders

Stem Cell Transplantation

United States
University of Florida
Live Like Bella Pediatric Cancer Research

Completed

Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation

Common Variable Immunodeficiency | Severe Combined Immunodeficiency | Sickle Cell Disease | Hurler Syndrome | Chronic Granulomatous Disease | Thalassemia | Wiskott-Aldrich Syndrome | Adrenoleukodystrophy | X-linked Lymphoproliferative Disease | Diamond Blackfan Anemia | Bone Marrow Failure Syndrome | Hemophagocytic... and other conditions

United States
SwanBio Therapeutics, Inc.

Active, not recruiting

Study of Disease Progression in Adults With Inherited Forms of Spastic Paraplegia (CYGNET)

AMN | AMN Gene Mutation | X-ALD

United States, Netherlands, Germany
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

Search clinical trials for: Inherited Diseases

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