Hemophilia B — Clinical Trials

MONDO:001060447 active·25 recruitingLast updated: Mar 16, 2026, 3:02 AM

Trial Landscape

Hemophilia B has an active clinical trial landscape, driven by drug treatment and gene therapy.

Recruiting

Active Trials

By Approach

Drug Treatment14

Gene Therapy12

Biological Therapy7

Procedure2

14 additional trials involve behavioral, dietary, diagnostic, or observational approaches.

By Phase

Early Phase 1

Phase 1

Phase 2

Phase 3

Phase 4

25 trials are observational or have no phase designation.

Sponsor Types

Industry27Academic/Other17Collaborative Network3

Top Sponsors

Baxalta now part of Shire

Be Biopharma

Biocad

CSL Behring

Equilibra Bioscience LLC

Novo Nordisk A/S

Trial Locations

United States21 trials

China12 trials

Canada11 trials

United Kingdom11 trials

Germany10 trials

France8 trials

Turkey (Türkiye)7 trials

Australia7 trials

Denmark6 trials

Spain6 trials

Trials often recruit across multiple countries, so totals exceed the trial count.

47 Trials

Phase 3Recruiting

NCT05568719

Safety and Effectiveness of Giroctocogene Fitelparvovec or Fidanacogene Elaparvovec in Patients With Hemophilia A or B Respectively

Sponsor:PfizerOrphan Designation

United StatesStarted: Dec 2022Est. completion: Feb 2040Updated: Feb 2026

Phase 3Recruiting

NCT07080905

Phase 3, Open-label, Single-dose Study of CSL222 in Adolescent Male Subjects (≥ 12 to < 18 Years of Age) With Severe or Moderately Severe Hemophilia B

Sponsor:CSL BehringOrphan DesignationFDA Approved Drug

United StatesStarted: Jul 2025Est. completion: Oct 2033Updated: Feb 2026

Phase 3Recruiting

NCT05145127

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

Sponsor:PfizerOrphan Designation

United StatesStarted: Nov 2021Est. completion: Jul 2030Updated: Feb 2026

Phase 3RecruitingGene Therapy

NCT06003387

Efficacy and Safety of CSL222 (Etranacogene Dezaparvovec) Gene Therapy in Adults With Hemophilia B With Pretreatment Adeno-associated Virus Serotype 5 (AAV5) Neutralizing Antibodies (Nabs)

Sponsor:CSL BehringOrphan DesignationFDA Approved Drug

United StatesStarted: Jan 2024Est. completion: Apr 2032Updated: Dec 2025

Phase 3Recruiting

NCT06700096

An Open-Label, Comparative Study of the Efficacy, Safety and Pharmacodynamics of Single Dose of ANB-002 in Patients With Hemophilia B

Sponsor:Biocad

RussiaStarted: Nov 2024Est. completion: Mar 2032Updated: Nov 2025

Phase 3Recruiting

NCT05611801

A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B

Sponsor:PfizerOrphan Designation

United StatesStarted: Dec 2022Est. completion: Sep 2028Updated: Aug 2025

Phase 1Recruiting

NCT06349473

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 or Factor VII Deficiency)

Sponsor:Equilibra Bioscience LLC

United StatesStarted: May 2024Est. completion: Sep 2026Updated: Mar 2026

Phase 1Recruiting

NCT06379789

A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia B

Sponsor:Regeneron PharmaceuticalsOrphan Designation

United StatesStarted: Sep 2024Est. completion: Dec 2032Updated: Feb 2026

Phase 1Recruiting

NCT06611436

BeCoMe-9: A Clinical Study of BE-101 for the Treatment of Adults With Moderately Severe or Severe Hemophilia B

Sponsor:Be BiopharmaOrphan Designation

United StatesStarted: Dec 2024Est. completion: Jul 2027Updated: Oct 2025

Phase 1Recruiting

NCT06120582

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

Sponsor:Biocad

BelarusStarted: May 2023Est. completion: Nov 2029Updated: Jun 2025

Phase 3Active, Not Recruiting

NCT06399289

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

Sponsor:CSL BehringOrphan DesignationFDA Approved Drug

ChinaStarted: Jul 2024Est. completion: Dec 2026Updated: Jan 2026

Phase 3Active, Not RecruitingGene Therapy

NCT03861273

A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B

Sponsor:PfizerOrphan Designation

United StatesStarted: Jul 2019Est. completion: Feb 2031Updated: Sep 2025

Phase 3Active, Not Recruiting

NCT06312475

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

Sponsor:Suzhou Alphamab Co., Ltd.Orphan Designation

ChinaStarted: Jan 2024Est. completion: Dec 2025Updated: Sep 2025

Phase 3Active, Not Recruiting

NCT06569108

Efficacy and Safety of KN057 Prophylaxis in Patients With Haemophilia A or B Without Inhibitors

Sponsor:Suzhou Alphamab Co., Ltd.Orphan Designation

ChinaStarted: Apr 2024Est. completion: Dec 2026Updated: Sep 2025

Phase 2Active, Not Recruiting

NCT03307980

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

Sponsor:PfizerOrphan Designation

United StatesStarted: Jun 2017Est. completion: Oct 2029Updated: Feb 2026

Phase 2Active, Not RecruitingGene Therapy

NCT05203679

Evaluation of the Safety and Efficacy of Hemophilia B Gene Therapy Drug

Sponsor:Shanghai Xinzhi BioMed Co., Ltd.

ChinaStarted: Dec 2021Est. completion: Jun 2028Updated: Jul 2025

Phase 1Active, Not RecruitingGene Therapy

NCT00979238

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

Sponsor:St. Jude Children's Research Hospital

United StatesStarted: Feb 2010Est. completion: Mar 2026Updated: Feb 2026

Phase 1Active, Not RecruitingGene Therapy

NCT01687608

Open-Label Single Ascending Dose of Adeno-associated Virus Serotype 8 Factor IX Gene Therapy in Adults With Hemophilia B

Sponsor:Baxalta now part of Shire

United StatesStarted: Feb 2013Est. completion: Jan 2030Updated: Feb 2025

Active, Not Recruiting

NCT05568459

A Trial That Evaluates Disease Characteristics in Hemophilia B Adult Male Participants Receiving Prophylaxis With Standard of Care Factor IX Protein (FIX) Replacement Therapy

Sponsor:Regeneron PharmaceuticalsOrphan Designation

United StatesStarted: Jan 2024Est. completion: Jan 2026Updated: Dec 2025

Active, Not RecruitingGene Therapy

NCT05360706

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

Sponsor:CSL BehringOrphan DesignationFDA Approved Drug

GermanyStarted: Mar 2021Est. completion: May 2026Updated: Aug 2025

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Research Publications

Explore the research landscape for Hemophilia B — publications classified by type, top journals, and trends.

View Publications →

NIH Grants & Funding

Explore NIH-funded research grants for Hemophilia B — by theme, institution, and investigator.

View Grants & Funding →

Patient Advocacy Groups

Hemophilia Federation of America

hemophiliafed.org

Hemophilia Foundation of Southern California

hemosocal.org

National Bleeding Disorders Foundation

bleeding.org

World Federation of Hemophilia

wfh.org

Patient Registry

Approved Treatments

QFITLIA(FITUSIRAN)— 2025

ALHEMO(CONCIZUMAB)— 2024

HYMPAVZI(MARSTACIMAB-HNCQ)— 2024

BEQVEZ(fidanacogene elaparvovec-dzkt)— 2024

Hemgenix(etranacogene dezaparvovec-drlb)— 2022

Ixinity(Coagulation Factor IX (Recombinant))— 2015

Data sourced from ClinicalTrials.gov via Kisho's automated pipeline. Trials refreshed every 4 hours for monitored diseases.

Last sync: March 16, 2026 at 3:02 AM

Hemophilia B — Clinical Trials

Trial Landscape

By Approach

By Phase

Sponsor Types

Top Sponsors

Trial Locations

47 Trials

Open Trials35

Safety and Effectiveness of Giroctocogene Fitelparvovec or Fidanacogene Elaparvovec in Patients With Hemophilia A or B Respectively

Phase 3, Open-label, Single-dose Study of CSL222 in Adolescent Male Subjects (≥ 12 to < 18 Years of Age) With Severe or Moderately Severe Hemophilia B

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

Efficacy and Safety of CSL222 (Etranacogene Dezaparvovec) Gene Therapy in Adults With Hemophilia B With Pretreatment Adeno-associated Virus Serotype 5 (AAV5) Neutralizing Antibodies (Nabs)

An Open-Label, Comparative Study of the Efficacy, Safety and Pharmacodynamics of Single Dose of ANB-002 in Patients With Hemophilia B

A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B

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 or Factor VII Deficiency)

A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia B

BeCoMe-9: A Clinical Study of BE-101 for the Treatment of Adults With Moderately Severe or Severe Hemophilia B

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

Active Trials12

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

A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B

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

Efficacy and Safety of KN057 Prophylaxis in Patients With Haemophilia A or B Without Inhibitors

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

Evaluation of the Safety and Efficacy of Hemophilia B Gene Therapy Drug

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

Open-Label Single Ascending Dose of Adeno-associated Virus Serotype 8 Factor IX Gene Therapy in Adults With Hemophilia B

A Trial That Evaluates Disease Characteristics in Hemophilia B Adult Male Participants Receiving Prophylaxis With Standard of Care Factor IX Protein (FIX) Replacement Therapy

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

Hemophilia B — Clinical Trials

Trial Landscape

By Approach

By Phase

Sponsor Types

Top Sponsors

Trial Locations

47 Trials

Open Trials35

Safety and Effectiveness of Giroctocogene Fitelparvovec or Fidanacogene Elaparvovec in Patients With Hemophilia A or B Respectively

Phase 3, Open-label, Single-dose Study of CSL222 in Adolescent Male Subjects (≥ 12 to &lt; 18 Years of Age) With Severe or Moderately Severe Hemophilia B

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

Efficacy and Safety of CSL222 (Etranacogene Dezaparvovec) Gene Therapy in Adults With Hemophilia B With Pretreatment Adeno-associated Virus Serotype 5 (AAV5) Neutralizing Antibodies (Nabs)

An Open-Label, Comparative Study of the Efficacy, Safety and Pharmacodynamics of Single Dose of ANB-002 in Patients With Hemophilia B

A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B

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 or Factor VII Deficiency)

A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia B

BeCoMe-9: A Clinical Study of BE-101 for the Treatment of Adults With Moderately Severe or Severe Hemophilia B

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

Active Trials12

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

A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B

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

Efficacy and Safety of KN057 Prophylaxis in Patients With Haemophilia A or B Without Inhibitors

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

Evaluation of the Safety and Efficacy of Hemophilia B Gene Therapy Drug

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

Open-Label Single Ascending Dose of Adeno-associated Virus Serotype 8 Factor IX Gene Therapy in Adults With Hemophilia B

A Trial That Evaluates Disease Characteristics in Hemophilia B Adult Male Participants Receiving Prophylaxis With Standard of Care Factor IX Protein (FIX) Replacement Therapy

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

Phase 3, Open-label, Single-dose Study of CSL222 in Adolescent Male Subjects (≥ 12 to < 18 Years of Age) With Severe or Moderately Severe Hemophilia B