Clinical Trials on Genetic Disorder

Total 151860 results

Baxalta now part of Shire

Completed

Post-Marketing Surveillance (Usage Results Study) of RIXUBIS in Adult and Pediatric Patients With Haemophilia B in South Korea

Hemophilia B

Korea, Republic of
Baxalta now part of Shire

Completed

Non-Interventional Study to Capture the Patient Experience on Gene Therapy for Hemophilia Within the AskBio009-101 Study Through Patient and Investigator Interviews

Hemophilia B

United States
Medexus Pharma, Inc.

Completed

Study of Recombinant Factor IX Product, IB1001, in Subjects With Hemophilia B

Hemophilia B

United States, France, United Kingdom, India, Israel, Italy, Poland
Baxalta now part of Shire

Completed

Study of Prophylaxis, ACtivity and Effectiveness (SPACE) in Hemophilia Patients Currently Treated With ADVATE or RIXUBIS (SPACE)

Hemophilia A | Hemophilia B

United States
ISU Abxis Co., Ltd.
Catalyst Biosciences

Completed

Dose-escalation Study to Investigate the Safety, PK, and PD of ISU304/CB2679d in Hemophilia B Patients

Hemophilia B

Korea, Republic of
Pfizer

Completed

A Gene Therapy Study for Hemophilia B

Hemophilia B

United States, Australia
Bayer

Completed

A Single Escalating Dose and Multiple Dose Study of BAY 1093884 in Subjects With Severe Hemophilia Types A or B, With or Without Inhibitors

Hemophilia A | Hemophilia B

Ukraine, United Kingdom, Bulgaria, Germany, Japan
Royal Free Hospital NHS Foundation Trust

Unknown

UK - EHL Outcomes Registry

Hemophilia A | Hemophilia B

United Kingdom
Nationwide Children's Hospital

Completed

Virtual Reality for Hemophilia

Hemophilia A | Hemophilia B

United States
CSL Behring

Completed

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

Hemophilia B

Germany, Australia, Italy, Israel, Czech Republic, Spain, France, Canada, Austria, Russian Federation
Pfizer

Completed

Study To Compare On-Demand Treatment To A Prophylaxis Regimen Of BeneFIX In Subjects With Moderately Severe to Severe Hemophilia B

Hemophilia B

Canada, Singapore, Turkey, Croatia, Korea, Republic of, Mexico, Poland, Bulgaria, Malaysia
Pfizer

Completed

Post Marketing Observational Study of Reformulated BeneFIX

Hemophilia B

France
CSL Behring

Completed

Safety and Pharmacokinetic Study of a Recombinant Coagulation Factor IX Albumin Fusion Protein in Subjects With Hemophilia B

Hemophilia B

Spain, Italy, France, Germany, Austria, Israel
Wyeth is now a wholly owned subsidiary of Pfizer

Completed

Study Evaluating On-Demand Treatment With BeneFIX In Chinese Subjects

Hemophilia B

China
Wyeth is now a wholly owned subsidiary of Pfizer

Completed

Study Evaluating BENEFIX in Previously Treated Patients With Hemophilia B

Hemophilia B
Hacettepe University
Suleyman Demirel University

Completed

Postural and Anthropometric Properties of Foot and Ankle of Patients With DMD

Duchenne Muscular Dystrophy | Ambulation Difficulty | Posture Disorders in Children | Ambulation Disorder, Neurologic

Turkey
Lithuanian University of Health Sciences

Recruiting

The Relations Among Endotoxin, Inflammatory Cytokines, Cognitive Markers and Brain MRI Changes in Subjects With Depressive Disorder

Inflammation | Cognitive Dysfunction | Genetic Predisposition to Disease | Endotoxemia | Major Depressive Disorder | Blood Brain Barrier Defect

Lithuania
Hospices Civils de Lyon

Not yet recruiting

Rehabilitation Program on Genetic and Degenerative Ataxia (RAPP)

Ataxia | Genetic Disease | Degenerative Disease

France
Essentia Health
St. Luke's Hospital of Duluth; WHITESIDE Institute for Clinical Research

Recruiting

Improved Diagnosis of Familial Hypercholesterolemia Across the Northland (ID-FH) (ID-FH)

Familial Hypercholesterolemia | Genetic Disease | Cholesterol, Elevated

United States
Massachusetts General Hospital
n-Lorem Foundation

Not yet recruiting

Single Participant Study of an Experimental ASO Treatment for TUBB4A-related Leukodystrophy

Genetic Disease
National Human Genome Research Institute (NHGRI)

Recruiting

Observational Study of Advanced Data Analytics in Genetic Conditions

Genetic Conditions

United States
Neuromed IRCCS

Recruiting

Implementation of Molecular Diagnostic Pathways

Genetic Disease

Italy
University of Pennsylvania
Fox Chase Cancer Center; Columbia University; Thomas Jefferson University

Enrolling by invitation

Penn Medicine Biobank Return of Results Program (ROR)

Cardiovascular Diseases | Cancer | Genetic Disease

United States
Robert Bosch Medical Center

Completed

Impact of Predictive Genetic Testing

Quality of Life | Physical Activity | Genetic Testing
Institut de Recerca Biomèdica de Lleida
ISCIII

Recruiting

Genetic Susceptibility to Predict Weight Loss After Bariatric Surgery (GENBASU)

Metabolic Syndrome | Weight Loss | Obesity, Morbid | Bariatric Surgery Candidate | Genetic Predisposition

Spain
Icahn School of Medicine at Mount Sinai
National Institute of Neurological Disorders and Stroke (NINDS)

Enrolling by invitation

A Controlled Trial of Growth Hormone in Phelan-McDermid Syndrome and Idiopathic Autism

Autism Spectrum Disorder (ASD) | Phelan-McDermid Syndrome

United States
Seoul National University Hospital
Ministry of Health & Welfare, Korea

Completed

Stem Cell Research on Subjects at Genetic High Risk for Schizophrenia

Genetic High Risk for Schizophrenia

Korea, Republic of
Retina Foundation of the Southwest
DSM Nutritional Products, Inc.; Foundation Fighting Blindness

Completed

DHA and X-Linked Retinitis Pigmentosa

Retinitis Pigmentosa | X-linked Genetic Diseases

United States
National Human Genome Research Institute (NHGRI)

Completed

Genetic Studies in the Amish and Mennonites

Genetic Disease

United States
Rady Pediatric Genomics & Systems Medicine Institute

Enrolling by invitation

Rapid Whole Genome Sequencing Study (rWGS)

Genetic Diseases | Genetic Syndrome

United States
CSL Behring

Not yet recruiting

Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Chinese Subjects With Hemophilia B Previously Treated With FIX Therapy

Hemophilia B
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
CSL Behring

Recruiting

An Observational Cohort Study to Characterize the Effectiveness and Safety of HEMGENIX® in Patients With Hemophilia B (IX-TEND 4001)

Hemophilia B

United States
Institute of Hematology & Blood Diseases Hospital

Not yet recruiting

The Efficacy and Safety of ZS801 in Chinese Hemophilia B Patients.

Hemophilia B

China
Chia Tai Tianqing Pharmaceutical Group Co., Ltd.

Recruiting

Clinical Study of Recombinant Human Activated Coagulation Factor VII for Injection in Patients With Hemophilia With Inhibitor

Hemophilia A | Hemophilia B

China
Shanghai Vitalgen BioPharma Co., Ltd.

Not yet recruiting

Lead-in Study of VGB-R04 Gene Therapy for Hemophilia B-- An Observational Survey Analysis Study

Hemophilia B
Shanghai Vitalgen BioPharma Co., Ltd.

Not yet recruiting

A Study to Evaluate the Safety and Efficacy of VGB-R04 in Adult Hemophilia B Patients

Hemophilia B

China
Medexus Pharma, Inc.

Completed

Evaluation of a Recombinant Factor IX Product, APVO101, in Previously-Treated Pediatric Patients With Hemophilia B

Hemophilia B

Turkey, Brazil, Georgia, Moldova, Republic of, South Africa, Ukraine
Novo Nordisk A/S

Enrolling by invitation

Adverse Event Data Collection From External Registries on Nonacog Beta Pegol

Haemophilia B

Netherlands, United Kingdom
Swedish Orphan Biovitrum
Cerner Enviza

Active, not recruiting

An International Study to Evaluate the Real-world Effectiveness and Usage of Alprolix in Patients With Haemophilia B (B-MORE)

Hemophilia B

Czechia, Greece, Ireland, Italy, Norway, Saudi Arabia, Spain, Sweden, United Kingdom
ApcinteX Ltd
Centessa Pharmaceuticals plc

Active, not recruiting

The Safety and Tolerability of SerpinPC in Healthy Men and in Men With Severe Blood Disorders (Haemophilia A and B)

Hemophilia A | Hemophilia B

Georgia, Moldova, Republic of, United Kingdom
Freeline Therapeutics

Terminated

A Long-Term Follow-Up Study of Haemophilia B Patients Who Have Undergone Gene Therapy

Hemophilia B

United Kingdom
Swedish Orphan Biovitrum
Kantar Health; Cerner Enviza (former Kantar Health)

Completed

Psychometric Validation of the Hemophilia Functional Ability Scoring Tool (Hemo-FAST) (Hemo-FAST)

Hemophilia A | Hemophilia B

France
Baxalta now part of Shire

Completed

Pivotal Study (Pharmacokinetics, Efficacy, Safety) of BAX 326 (rFIX) in Hemophilia B Patients

Hemophilia B

Poland, United Kingdom, Spain, Bulgaria, Romania, Japan, Russian Federation, Brazil, Chile, Sweden, Colombia, Ukraine, Argentina, Czechia
Baxalta now part of Shire

Completed

BAX 326 Surgery Study in Hemophilia B Patients

Hemophilia B

Colombia, Bulgaria, Chile, Poland, Romania, Argentina, Czechia, Russian Federation, Ukraine, United Kingdom
McMaster University
Canadian Hemophilia Society

Completed

Web-based Application for the Population Pharmacokinetic Service - Phase 1 (WAPPS)

Hemophilia A | Hemophilia B

Canada
Sanofi

Completed

A Phase 1 Study of an Investigational Drug, ALN-AT3SC, in Healthy Volunteers and Hemophilia A or B Patients

Hemophilia A | Hemophilia B

Bulgaria, Switzerland, United Kingdom, United States, Russian Federation
Bioverativ Therapeutics Inc.
Swedish Orphan Biovitrum

Terminated

Fc factOrs and Real-World hemophiliA Patient-ReporteD Outcomes (FORWARD)

Hemophilia A | Hemophilia B

United States
Bioverativ Therapeutics Inc.
Swedish Orphan Biovitrum; Syntonix Pharmaceuticals, Inc.

Completed

Phase I/IIa Study of FIXFc in Hemophilia B Patients

Hemophilia B

United States
Shenzhen Geno-Immune Medical Institute

Unknown

Lentiviral FIX Gene Therapy

Hemophilia B

Clinical Trials on Genetic Disorder

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