Search clinical trials for: Inherited Diseases

Total 371960 results

Washington University School of Medicine
Alzheimer's Association; Eisai Inc.

Not yet recruiting

DIAN-TU Amyloid Removal Trial (ART) in Dominantly Inherited Alzheimer's Disease (DIAN-TU)

Dementia | Alzheimer's Disease | Alzheimer's Disease, Familial

United States, Australia, United Kingdom
University of Wisconsin, Madison

Not yet recruiting

COmparison of Clarus and Optos Ultrawide Field Imaging Systems for Inherited Retinal Disease (COCO-IRD)

Retinal Disease

United States
Regeneron Pharmaceuticals
Intellia Therapeutics

Not yet recruiting

A Trial to Learn if REGV131-LNP1265 is Safe and Works to Help the Body Make Clotting Factor in Pediatric, Adolescent and Adult Patients With Hemophilia B (BEYOND-9)

Hemophilia B
Enzyre B.V.
Indiana Hemophilia & Thrombosis Center

Not yet recruiting

Case and Use Scenario Study to Gain Knowledge on the User Needs for a Medical Device for Hemophilia A Monitoring

Hemophilia A

United States
University Hospital, Bonn
Swedish Orphan Biovitrum

Recruiting

Prevalence of Synovitis in Patients With Haemophilia A (SynoPrev)

Haemophilia A | Synovitis | Hemophilia Arthropathy | Sonography

Germany
Equilibra Bioscience LLC

Recruiting

A Study of Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of SR604 in Two Participants Groups (Part A: Healthy Participants, and Part B: Participants With Hemophilia A or Hemophilia B)

Hemophilia A | Hemophilia B | Healthy Participants

United States
Washington University School of Medicine
National Cancer Institute (NCI); The Foundation for Barnes-Jewish Hospital

Recruiting

Washington University Participant Engagement and Cancer Genomic Sequencing Center (WU-PE-CGS)

Multiple Myeloma | Rectal Cancer | Cholangiocarcinoma | Colon Cancer

United States
IRCCS Fondazione Stella Maris
Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta; IRCCS Ospedale San... and other collaborators

Recruiting

The Fingerprinting of Inherited Leukoencephalopathies: A New Brain Imaging, Genetic and Clinical Assessment (FIABA)

Leukoencephalopathies

Italy
Dr. Sascha Drewlo

Not yet recruiting

Optimization of Cervical Collections in Pregnancy (OCCP)

Fetal Growth Retardation | Preeclampsia | Cervix; Pregnancy | Inherited Genetic Conditions (Diagnosis)
Kathelijn Fischer
Radboud University Medical Center; University Medical Center Groningen; Maastricht... and other collaborators

Recruiting

Pharmacokinetic-guided Dosing of Emicizumab (DosEmi)

Adolescent | Child | Hemophilia A With Inhibitor | Adult | Hemophilia A Without Inhibitor | Hemophilia A, Severe

Netherlands
Suzhou Alphamab Co., Ltd.

Recruiting

Efficacy and Safety of KN057 Prophylaxis in Patients With Haemophilia A or B With Inhibitors

Hemophilia A With Inhibitor | Hemophilia B With Inhibitor

China
Chia Tai Tianqing Pharmaceutical Group Nanjing...

Not yet recruiting

A Clinical Study of Recombinant Human Coagulation Factor VIII for Injection in Patients With Severe Hemophilia A

Hemophilia A

China
Spark Therapeutics, Inc.

Recruiting

Study of a Gene Therapy Treatment for Hemophilia A (KEYSTONE 1)

Hemophilia A

United States
Staidson (Beijing) Biopharmaceuticals Co., Ltd

Recruiting

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

Hemophilia

China
Investigación en Hemofilia y Fisioterapia

Not yet recruiting

Changes in Intra-articular Parameters by Doppler Ultrasound in Patients With Hemophilic Ankle Arthropathy

Hemophilia

Spain
Radboud University Medical Center
Enzyre B.V.

Not yet recruiting

Version Testing of EnzySystem Version A for Hemophilia A

Hemophilia A

Netherlands
Investigación en Hemofilia y Fisioterapia

Not yet recruiting

Conditioned Pain Modulation in Patients With Hemophilia

Hemophilia

Spain
Investigación en Hemofilia y Fisioterapia

Recruiting

Blood Flow Restriction in Improving Muscle Strength of Patients With Hemophilic Ankle Arthropathy

Hemophilia

Spain
Institute of Hematology & Blood Diseases Hospital...

Not yet recruiting

Safety and Efficacy Study of NGGT003 in Hemophilia A Patients

Hemophilia A

China
BioMarin Pharmaceutical

Recruiting

Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A (GENEr8-JPN)

Hemophilia A

Japan
Bayer

Recruiting

A Study to Learn More About the Safety of Damoctocog-alfa-pegol When Used in Routine Medical Care in Korean Participants With Hemophilia A

Hemophilia A | Prophylaxis of Bleeding | Treatment of Bleeding

Korea, Republic of
University College, London

Not yet recruiting

Coloured-light in Retinitis Pigmentosa. (CLIRP)

Retinitis Pigmentosa

United Kingdom
Hospices Civils de Lyon

Recruiting

EVALUATION OF THE OVERALL HAEMOSTATIC CAPACITY OF MIM8 WITH GLOBAL HAEMOSTASIS ASSAYS AND FIBRIN CLOT ULTRASTRUCTURE (Mim8-TGT)

Hemophilia A

France
Innostellar Biotherapeutics Co.,Ltd

Recruiting

Fellow-eye Study (FE) of LX101 in Subjects With Inherited Retinal Dystrophy

Inherited Retinal Dystrophy

China
Hemab ApS

Recruiting

A Phase 1/2 Study to Investigate Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of HMB-001 in Glanzmann Thrombasthenia

Glanzmann Thrombasthenia

United States, Belgium, France, Italy, Netherlands, United Kingdom
Unity Health Toronto

Not yet recruiting

A Pilot Crossover Trial of Prophylactic Wilate Compared to Placebo for Heavy Menstrual Bleeding in Patients With VWD (EMPOWER)

Von Willebrand Diseases
UMC Utrecht

Not yet recruiting

Multinational Glanzmann Study (Glanzmann-NHS)

Glanzmann Thrombasthenia
Fondazione Policlinico Universitario Campus Bio-Medico
IRCCS San Raffaele; A.O.U. Città della Salute e della Scienza - Molinette Hospital

Recruiting

A Study to Investigate the Family History of Cancer in Patients With Non-small Cell Lung Cancer (FAHIC - Lung). (FAHIC-Lung)

Non Small Cell Lung Cancer

Italy
The Hospital for Sick Children
Roche Pharma AG

Not yet recruiting

Canadian CHO-KLAT/H-FIT Study - Quality of Life of, and Burden of Caring for, Persons With Hemophilia

Hemophilia
Biocad

Recruiting

A Trial of the Safety and Efficacy of Single-Dose Administration of ANB-010 in Subjects With Hemophilia A (EDELWEISS)

Hemophilia A

Russian Federation
University Hospital, Strasbourg, France

Recruiting

Interference by Activated Protein C Resistance in the Determination of Protein S Activity (PS)

Resistance to Activated Protein C in the Determination of Protein S Activity

France
Duke University

Recruiting

SS-HH-OCT as a Novel Diagnostic Modality for Early-Onset Retinal Dystrophies (EORDs) (SS-HH-OCT)

Retinal Dystrophies

United States
Royal Marsden NHS Foundation Trust

Recruiting

Inherited Cancer Early Diagnosis (ICED) Study (ICED)

Lynch Syndrome | Familial Adenomatous Polyposis | Hereditary Diffuse Gastric Cancer | Peutz-Jeghers Syndrome | Cowden Syndrome | Li Fraumeni Syndrome | MutYH-associated Polyposis | SMAD4 Gene Mutation

United Kingdom
Hospices Civils de Lyon

Recruiting

Study of Surgical Practices in Patients With Haemophilia A or B Treated With an Extended Half-life Recombinant Factor VIII-Fc or IX-Fc (ELOCTA®, ALPROLIX®) (CHALE)

Hemophilia A | Hemophilia B

France
Chulalongkorn University

Recruiting

Comparison of Outcomes Between Low Dose Emicizumab and Factor VIII in Clinically Severe Hemophilia A

Joint Bleed | Severe Hemophilia A Without Inhibitor

Thailand
Assistance Publique - Hôpitaux de Paris

Not yet recruiting

Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders (DANNIgene)

Hemophilia A | Hemophilia B | Cystic Fibrosis | Sickle Cell Disease | Muscular Dystrophy, Duchenne | Fragile X Syndrome | Huntington Disease | Myotonic Dystrophy | Autosomal Recessive Polycystic Kidney Disease | Neurofibromatosis-Noonan Syndrome | Muscular Dystrophy, Becker | Invasive PreNatal Diagnosis in a... and other conditions

France
Sanofi

Recruiting

A Study to Test a Medicine (Fitusiran) for Preventing Bleeds in People With Severe Hemophilia Who Previously Received Preventive Treatment With Emicizumab

Hemophilia A

Taiwan
Kiminori Kimura, MD
Japan Agency for Medical Research and Development

Recruiting

A Phase 2 Trial of Foscenvivint in Liver Cirrhosis Patients Caused by HIV/HCV Co-infection With Hemophilia (OP-724-H201)

Liver Cirrhosis

Japan
Jiangsu Gensciences lnc.

Recruiting

Phase 3 Clinical Project of Pegylated Recombinant Human Coagulation Factor VIII-Fc Fusion Protein

Severe Hemophilia A

China
Jiangsu Gensciences lnc.

Not yet recruiting

Study of Efficacy and Safety of FRSW107 in Pediatric Patients With Severe Hemophilia A

Severe Hemophilia A

China
Affiliated Hospital of Guangdong Medical University

Not yet recruiting

Safety and Efficacy Clinical Study of Severe Hemophilia B Treatment With KL001

Hemophilia B Without Inhibitor
Maastricht University Medical Center

Recruiting

Exploring Patient Reported Outcomes in Inherited Ichthyosis

Congenital Ichthyosis

Netherlands
Biocad

Recruiting

Study of the Safety, Pharmacodynamics and Efficacy of ANB-002 in Patients With Hemophilia B (SAFRAN)

Hemophilia B

Russian Federation
Zhejiang University

Not yet recruiting

Safety and Efficacy Clinical Study of Severe Hemophilia B Treatment With KL001

Hemophilia B Without Inhibitor
Shanghai Belief-Delivery BioMed Co., Ltd
Shanghai MYGT Biopharmaceutical LLC

Recruiting

Evaluation of the Safety and Efficacy of Hemophilia A Gene Therapy Drugs

Hemophilia A

China
Newark Beth Israel Medical Center
Genentech, Inc.

Not yet recruiting

Health-Related Quality of Life Outcomes and Bleeding Rates Among Patients With Severe Hemophilia A on Emicizumab (ML43518)

Hemophilia A

United States
Octapharma

Not yet recruiting

Phase 3 Study on the Efficacy and Safety of Human Plasma Derived Antithrombin (Atenativ) in Heparin-resistant Patients Scheduled to Undergo Cardiac Surgery Necessitating Cardiopulmonary Bypass

Acquired Antithrombin Deficiency
University Hospital, Lille

Not yet recruiting

Severe Congenital Hemostatic Defects, Cerebral MIcrobleeds and COGnition (HEMICOG)

Cerebral Microbleeds, Congenital Haemophilia, Congenital Von Willebrand Disease
National Cancer Institute (NCI)

Recruiting

Imatinib to Increase RUNX1 Activity in Participants With Germline RUNX1 Deficiency

Inherited Bone Marrow Failure Syndrome | Familial Platelet Disorder With Predisposition to Myeloid Malignancies

United States
Xinhua Hospital, Shanghai Jiao Tong University...
HuidaGene Therapeutics Co., Ltd.

Recruiting

Leber Congenital Amaurosis Inherited Blindness of Gene Therapy Trial(LIGHT)

Leber Congenital Amaurosis

China

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