Clinical Trials on Inherited Ophthalmic Diseases

Total 377727 results

Bio Products Laboratory

Terminated

A Study With OPTIVATE® in People With Von Willebrand Disease

Von Willebrand Disease

Israel, United Kingdom
University Hospital, Toulouse

Not yet recruiting

Evaluate the Evolution of Body and Scalp Skin Discomfort in Patients With Hereditary Ichthyosis After Wraps (EnvelopIchtyose)

Ichthyosis Inherited

France
Centers for Disease Control and Prevention

Completed

Joint Outcome Study

Hemophilia A
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
Takeda

Completed

A Study on Estimated Risks of Certain Adverse Events in Adults With Von Willebrand Disease (VWD) Treated With Veyvondi

Von Willebrand Disease (VWD)

Germany
BioMarin Pharmaceutical

Recruiting

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

Hemophilia A

Japan
BioMarin Pharmaceutical

Recruiting

A Prospective Study Evaluating Seroprevalence and Seroconversion of Antibodies Against Adeno-associated Virus (AAV)

Hemophilia A

Argentina
Institute of Hematology & Blood Diseases Hospital

Not yet recruiting

The Efficacy and Safety of ZS802 in Chinese Hemophilia A Patients.

Hemophilia A

China
University Hospital, Bonn
Swedish Orphan Biovitrum

Recruiting

Sonography and Haemophilia

Haemophilia | Sonography

Germany
Spark Therapeutics

Completed

A Gene Transfer Study for Hemophilia A

Hemophilia A

United States, Australia, Canada, Israel, Thailand
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
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
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
Baxalta now part of Shire
Takeda Development Center Americas, Inc.; Baxalta Innovations GmbH, now part...

Active, not recruiting

A Study of BAX 888 in Male Adults With Severe Hemophilia A

Hemophilia A

United States, Spain, Hungary, Italy, Germany, Austria, France
Assistance Publique Hopitaux De Marseille

Completed

Determinants and Consequences of the Transition to Adulthood Among Adolescents With Haemophilia (TRANSHEMO)

Haemophilia

France
Bio Products Laboratory

Terminated

Post-Marketing Safety Study Following Long-Term Prophylactic OptivateTreatment in Subjects With Severe Haemophilia A

Haemophilia A

Colombia, Germany, Poland
Hoffmann-La Roche

Completed

A Study to Investigate the Pharmacokinetics, Safety, and Tolerability of Emicizumab in Healthy Chinese Volunteers

Hemophilia A | Healthy Volunteers

China
National Human Genome Research Institute (NHGRI)

Completed

Genetic Analysis of Gray Platelet Syndrome

Myelofibrosis | Genetic Linkage

United States
Bio Products Laboratory

Completed

A Study to Investigate the Safety and Efficacy of Optivate® in Children Under 6 Years of Age With Haemophilia A.

Haemophilia A

Poland
University of Pretoria
Novo Nordisk A/S; Medical Research Council, South Africa

Completed

Combining Registry Data in Haemophilia: TARGET H (TARGET-H)

Haemophilia
Stowarzyszenie Pomocy Chorym na Zakrzepicę i Skazy...

Unknown

Use of a TGA and TEM in the Assessment of the Efficacy of Treatment With APCC or rFVIIa (Thrombus)

Haemophilia

Poland
Bayer

Completed

Validation Study of a cOmputer Pharmacokinetic Tool to assIst in the Follow up Care of haeMophilia A Patients (OPTIMS)

Haemophilia A

France, Italy
Chulalongkorn University

Completed

Comparison of Prophylaxis and On-demand Treatment in Children With Moderate to Severe Hemophilia A

Hemophilia A | Prophylaxis

Thailand
Taipei Medical University Hospital
Sanofi

Enrolling by invitation

Observational Study of Long-Term Joint Health Outcome in Hemophilia Patient

Hemophilia | Arthropathy

Taiwan
Octapharma

Recruiting

Effectiveness and Tolerability of Eqwilate in Real-life Conditions

VWD - Von Willebrand's Disease

France
Takeda

Available

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

Von Willebrand Disease (VWD)
Takeda

Active, not recruiting

A Study of TAK-662 for Japanese Patients With Congenital Protein C Deficiency

Congenital Protein C Deficiency

Japan
Fondazione Policlinico Universitario Agostino Gemelli...
Antonio Maria Dell Anna; Sofia Cacciola; Claudio Sandroni; Mauro Pittiruti; Cesare... and other collaborators

Completed

Suitability of PICC for Transpulmonary Thermodilution Compared to CICC

Hemodynamic Monitoring | Peripherally Inserted Central Catheters | Transpulmonary Thermodilution | CVC

Italy
Jiangsu Gensciences lnc.

Not yet recruiting

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

Severe Hemophilia A

China
Swedish Orphan Biovitrum
Syneos Health

Recruiting

A Study Evaluating Physical Activity and Joint Health in Severe Haemophilia A Patients ≥12 Years Treated Once Weekly With Efanesoctocog Alfa (FREEDOM)

Hemophilia A, Severe

Italy, Ireland, Croatia, Greece, Spain, Sweden, Slovenia, United Kingdom, France, Germany, Norway, Austria, Czechia, Netherlands, Belgium
Laboratoire français de Fractionnement et de Biotechnologies

Recruiting

A Phase 3 Study of the Safety and Efficacy of Coagulation Factor VIIa (Recombinant) for the Prevention of Excessive Bleeding in Patients With Congenital Hemophilia A or B With Inhibitors to Factor VIII or IX Undergoing Elective Major Surgical Procedures SCOPE HIM (SCOPE HIM)

Hemophilia

United States, Thailand, Malaysia, Mexico, Turkey, South Africa
Sanofi

Recruiting

A Study to Test a Medicine (Fitusiran) Injected Under the Skin for Preventing Bleeding Episodes in Male Adolescent or Adult Participants With Severe Hemophilia (ATLAS-NEO)

Hemophilia

Greece, Korea, Republic of, France, Taiwan, Italy, Spain, United States, Canada, China, Germany, India, Japan, Mexico, South Africa, Turkey, Poland
Octapharma

Recruiting

Efficacy of Atenativ in Patients With Congenital Antithrombin Deficiency Undergoing Surgery or Delivery

Congenital Antithrombin Deficiency

Spain, Italy, United States, Serbia, France, Hungary, Austria, Germany, United Kingdom, Israel
Ascension Healthcare Development Limited

Recruiting

A Pharmacokinetic and Clotting Activity Study of FVIII-PEGLip

Hemophilia A With Inhibitor

Russian Federation
Bayer

Active, not recruiting

Study to Learn More About the Safety of Drug Jivi Over a Long Period of Time in Previously Treated Patients With Hemophilia A (Bleeding Disorder Resulting From a Lack of FVIII) Who Are Receiving Jivi Regularly at Their Treating Doctors to Prevent Bleeding (HA-SAFE)

Hemophilia A

Spain, Germany, Austria, Greece, Italy, Slovenia
Takeda

Active, not recruiting

A Study of Adynovate in Previously Treated Chinese Teenagers and Adults With Severe Hemophilia A

Hemophilia A

China
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
Jiangsu Gensciences lnc.

Completed

A Study to Evaluate the Pharmacokinetics，Safety and Tolerability of PEG Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection

Severe Hemophilia A

China
Jiangsu Gensciences lnc.

Completed

A Study of Repeat Dosing of PEG Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection

Severe Hemophilia A

China
Laboratoire français de Fractionnement et de Biotechnologies
LFB USA, Inc.

Completed

LR769 in Congenital Hemophilia Patients With Inhibitors Undergoing Elective Surgery or Invasive Procedures (PerSept3)

Hemophilia

United States, Ukraine, Mexico, Russian Federation, South Africa, Spain
Novo Nordisk A/S

Completed

A Trial Evaluating Efficacy and Safety of Prophylactic Administration of Concizumab in Patients With Severe Haemophilia A Without Inhibitors (explorer™5)

Haemophilia A | Haemostasis

United Kingdom, France, United States, Sweden, Spain, Turkey, Japan, Italy, Germany, Thailand, Ukraine
CSL Behring

Completed

An Open-label Safety and Efficacy Study of Recombinant FVIII in Patients With Severe Hemophilia A

Hemophilia A | Severe Hemophilia A

United States, Australia, Austria, Canada, Czechia, France, Georgia, Germany, Hungary, Ireland, Italy, Japan, Lebanon, Malaysia, Netherlands, Philippines, Poland, Portugal, Romania, South Africa, Spain, Switzerland, Thailand, Ukraine, United...
Baxalta now part of Shire
Baxalta Innovations GmbH, now part of Shire

Completed

BAX 855 Continuation

Hemophilia A

United States, Spain, Netherlands, Australia, Czechia, Korea, Republic of, Taiwan, Poland, Bulgaria, United Kingdom, Germany, Turkey, Israel, Malaysia, Ukraine, Russian Federation, Austria, Switzerland, Japan, Hong Kong, Lithuania, Roman... and more
Pfizer

Completed

Moroctocog Alfa (AF-CC) for Prophylaxis and Treatment of Bleeding Episodes in Previously Treated Hemophilia A Patients

Hemophilia A

India
Octapharma

Completed

Assess the Efficacy and Safety of Personalized Prophylaxis Human-cl rhFVIII in Patients With Severe Haemophilia A

Severe Haemophilia A

France, Japan, United States, Canada, Croatia, Finland, Netherlands, North Macedonia, Slovenia
Bayer

Completed

Study to Compare How the Body Distributes and Excretes the Drugs Jivi (BAY 94-9027) and Adynovi in Patients With Severe Hemophilia A (Bleeding Disorder Resulting From a Lack of Blood Clotting Factor VIII)

Hemophilia A

Bulgaria
City of Hope Medical Center
CSL Behring; Grifols Therapeutics LLC; Grifols Biologicals, LLC; Charta Foundation and other collaborators

Withdrawn

Study of First TIME Immunotolerance Induction in Severe Hemophilia A Patients With Inhibitor at High Risk of Failure: Comparison With FVIII Concentrates With or Without Von Willebrand Factor - RES.I.S.T. Naive (RESIST NAIVE)

Severe Hemophilia A
Oslo University Hospital
Norwegian School of Sport Sciences

Completed

HemFitBit, an Observational Study of Physical Activity in People With Moderate and Severe Haemophilia A

Hemophilia A Without Inhibitor

Norway
Pfizer

Withdrawn

Patient-reported Outcomes in Subjects Treated With ReFacto AF Routine Prophylaxis

Hemophilia A
Craig Seaman

Terminated

Weight-based Dosing in Hemophilia A

Hemophilia

United States

Clinical Trials on Inherited Ophthalmic Diseases

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Norway

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations