Clinical Trials on Genetic Disorders

Total 151479 results

Pfizer

Recruiting

Open-Label Extension Study of Marstacimab in Hemophilia Participants With or Without Inhibitors

Hemophilia A | Hemophilia B

Hong Kong, Spain, Serbia, Turkey, United States, China, Mexico, South Africa, India, Japan, Croatia, Korea, Republic of, Oman, Taiwan, Italy, Canada, France
McMaster University

Recruiting

Personalized Medicine for Canadians With Hemophilia (PMCH)

Hemophilia A | Hemophilia B

Canada
CSL Behring

Active, not recruiting

Study of AAV5-hFIX in Severe or Moderately Severe Haemophilia B

Hemophilia B

Netherlands, Germany
Pfizer

Active, not recruiting

A Study to Evaluate Prospective Efficacy and Safety Data of Current FIX Prophylaxis Replacement Therapy in Adult Hemophilia B Subjects (FIX:C≤2%) or Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Subjects (FVIII:C≤1%)

Hemophilia A | Hemophilia B

United States, Canada, Spain, Australia, Korea, Republic of, Taiwan, Brazil, Italy, Japan, Turkey, Germany, United Kingdom, Saudi Arabia, France, Sweden, Belgium, Greece, Israel
CSL Behring

Enrolling by invitation

Long-term Follow-up Study of Male Adults With Hemophilia B Previously Treated With Etranacogene Dezaparvovec (CSL222)

Hemophilia B

United States
Pfizer

Active, not recruiting

Long-term Safety and Efficacy Study and Dose-Escalation Substudy of PF 06838435 in Individuals With Hemophilia B

Hemophilia B

United States, Canada, Australia, Turkey
McMaster University

Enrolling by invitation

Web Accessible Population Pharmacokinetics Service - Hemophilia: Sources of Variability (WAPPS-Hemo)

Hemophilia A | Hemophilia B

Canada
Baxalta now part of Shire
Baxalta Innovations GmbH, now part of Shire

Completed

RIXUBIS PMS India (RIXUBIS PMS)

Hemophilia B

India
CSL Behring

Completed

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

Hemophilia B

United States, Australia, Japan, France, Philippines, Spain, Czechia, Israel, Canada, Germany, Austria, Bulgaria, Italy, Malaysia, South Africa
Bloodworks
CSL Behring

Completed

Joint Health Study

Hemophilia B

United States
Baxalta now part of Shire

Completed

BAX 326 Pediatric Study

Hemophilia B

Russian Federation, Ukraine, India, Poland, Romania, United Kingdom
OPKO Health, Inc.

Completed

A Study of a Long-Acting r-Factor 7a (Factor VIIa) in Adult Men With Hemophilia A or B

Hemophilia A | Hemophilia B

United States
Baxalta now part of Shire

Completed

BAX 326 (rFIX) Continuation Study

Hemophilia B

Taiwan, Colombia, Argentina, Brazil, Bulgaria, Chile, Czechia, India, Ireland, Italy, Japan, Poland, Romania, Russian Federation, Sweden, Ukraine, United Kingdom
Medexus Pharma, Inc.

Terminated

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

Hemophilia B

United States, United Kingdom, India
National Taiwan University Hospital

Unknown

The Effectiveness of Recombinant Coagulation Factor IX With Recombinant Albumin (rIX-FP) in Severe Hemophilia B Patients

Hemophilia

Taiwan
Grifols Biologicals, LLC

Completed

Efficacy and Safety of AlphaNine Versus BeneFIX in Patients With Severe Hereditary Haemophilia B

Hemophilia B

Bulgaria
Cangene Corporation

Withdrawn

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

Hemophilia B

United Kingdom, United States
Pfizer

Completed

An Open-Label, Single Dose Pharmacokinetic Study of Benefix (Recombinant Factor IX) in Male Chinese Subjects With Hemophilia B

Hemophilia B

China
CSL Behring

Completed

A Safety and Efficacy Study of a Recombinant Factor IX in Patients With Severe Hemophilia B

Hemophilia B

Bulgaria, Israel
Bayer

Completed

Study of FVIIa Variant BAY86-6150 (B0189) in Subjects With Moderate or Severe Hemophilia Types A or B With or Without Inhibitors (MATCHBOX)

Hemophilia A | Hemophilia B

South Africa, Poland, United Kingdom
Pfizer

Completed

Post Marketing Surveillance To Observe Safety and Efficacy Of BeneFIX In Patients With Hemophilia B

Hemophilia B

Korea, Republic of
Weill Medical College of Cornell University
University of Pennsylvania; CSL Behring

Completed

Females With Severe or Moderate Hemophilia A or B: A Multi-Center Study

Hemophilia A | Hemophilia B

United States, Puerto Rico
Weill Medical College of Cornell University
Bayer; University Hospital, Bonn

Completed

Females With Severe or Moderate Hemophilia A or B: an International Multi-center Study

Hemophilia A | Hemophilia B

United States, Israel, Australia, Italy, Sweden, Germany, Japan, Netherlands, Taiwan
Wyeth is now a wholly owned subsidiary of Pfizer

Completed

Study Evaluating BeneFIX in Patients With Haemophilia B, Previously Treated With Plasma Derived Factor IX

Hemophilia B

France
Wyeth is now a wholly owned subsidiary of Pfizer

Completed

Prospective Registry of European Hemophilia B Patients Receiving BeneFIX® for Usual Use

Hemophilia B

Belgium, Spain, Italy, Austria, United Kingdom, Netherlands, Sweden, Germany, Portugal, France
Wyeth is now a wholly owned subsidiary of Pfizer

Completed

Study Evaluating rFIX; BeneFIX® in Hemophilia B

Hemophilia B

United States
Wyeth is now a wholly owned subsidiary of Pfizer

Completed

Study Evaluating Approach to Treatment of Haemophilia A and B in Spain

Hemophilia A | Hemophilia B
Wyeth is now a wholly owned subsidiary of Pfizer

Completed

Study Evaluating of Recombinant Human Factor IX (BeneFIX) and a New Formulation of BeneFIX (rFIX-R) in Moderate to Severe Hemophilia B

Hemophilia B
Prothya Biosolutions

Completed

Post Marketing Study in Haemophilia B Patients Using Nonafact® (Human Coagulation Factor IX)

Hemophilia B

Netherlands
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
Pfizer

Terminated

Understanding Hemophilia A and B Drug Dosage Administration Patterns

Hemophilia A | Hemophilia B

United States

Clinical Trials on Genetic Disorders

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