Severe Hemophilia a — Clinical Trials

MONDO:001571929 active·15 recruitingLast updated: Mar 16, 2026, 6:12 AM

Trial Landscape

Severe Hemophilia a has an active clinical trial landscape, driven by drug treatment and biological therapy.

Recruiting

Active Trials

By Approach

Drug Treatment19

Biological Therapy4

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

By Phase

Phase 1

Phase 2

Phase 3

Phase 4

9 trials are observational or have no phase designation.

Sponsor Types

Industry20Academic/Other9

Top Sponsors

ASC Therapeutics

Bayer

BioMarin Pharmaceutical

Bioverativ, a Sanofi company

Chia Tai Tianqing Pharmaceutical Group Nanjing Shunxin Pharmaceutical Co., Ltd.

Expression Therapeutics, LLC

Trial Locations

United States17 trials

Germany11 trials

France9 trials

Italy9 trials

Spain9 trials

United Kingdom8 trials

Canada6 trials

China6 trials

Taiwan6 trials

Turkey (Türkiye)6 trials

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

29 Trials

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 3Recruiting

NCT06142552

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

Sponsor:Jiangsu Gensciences lnc.

ChinaStarted: Dec 2023Est. completion: Sep 2026Updated: Feb 2024

Last updated over 18 months ago

Phase 2Recruiting

NCT06155955

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

Sponsor:Chulalongkorn University

ThailandStarted: Jun 2023Est. completion: Jun 2024Updated: Mar 2024

Last updated over 18 months ago

Phase 1RecruitingGene Therapy

NCT07226206

A Gene Therapy Study of SPK-8011QQ in Adults With Severe or Moderately Severe Hemophilia A

Sponsor:Hoffmann-La Roche

United StatesStarted: Mar 2026Est. completion: Jul 2031Updated: Mar 2026

Phase 1Recruiting

NCT06579144

Pharmacokinetic Comparison of Efanesoctocog Alfa vs Other EHL-rFVIII Products in Participants With Severe Haemophilia A

Sponsor:Swedish Orphan Biovitrum

BulgariaStarted: Feb 2025Est. completion: May 2026Updated: Feb 2026

Phase 1Recruiting

NCT06703606

A Study to Learn About How Changing Therapy From Emicizumab to Marstacimab Affects People With the Severe Hemophilia A.

Sponsor:PfizerOrphan Designation

United StatesStarted: May 2025Est. completion: May 2027Updated: Aug 2025

Phase 1RecruitingGene Therapy

NCT04676048

ASC618 Gene Therapy in Hemophilia A Patients

Sponsor:ASC Therapeutics

United StatesStarted: Aug 2022Est. completion: Dec 2026Updated: Feb 2023

Last updated over 18 months ago

Phase 4Recruiting

NCT05935358

Nuwiq for Perioperative Management Of Patients With Haemophilia A on Emicizumab Regular Prophylaxis Study

Sponsor:Octapharma

United StatesStarted: Nov 2023Est. completion: Aug 2026Updated: Oct 2025

Phase 4Recruiting

NCT06145373

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

Sponsor:Sanofi

United StatesStarted: Mar 2024Est. completion: Nov 2029Updated: Sep 2025

Phase 4Recruiting

NCT04690322

POCUS: Hemostatic Potential and Joint Health in Patients With Severe Hemophilia A on Novel Replacement Therapies

Sponsor:University of Texas Southwestern Medical Center

United StatesStarted: Apr 2021Est. completion: Jul 2027Updated: Mar 2025

Phase 3Active, Not Recruiting

NCT05662319

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

Sponsor:Sanofi

United StatesStarted: Feb 2023Est. completion: Jan 2029Updated: Feb 2026

Phase 3Active, Not Recruiting

NCT04431726

A Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Subcutaneous Emicizumab in Participants From Birth to 12 Months of Age With Hemophilia A Without Inhibitors

Sponsor:Hoffmann-La Roche

United StatesStarted: Feb 2021Est. completion: May 2030Updated: Jan 2026

Phase 3Active, Not RecruitingGene Therapy

NCT04370054

Study to Evaluate the Efficacy and Safety of PF-07055480 / Giroctocogene Fitelparvovec Gene Therapy in Moderately Severe to Severe Hemophilia A Adults

Sponsor:PfizerOrphan Designation

United StatesStarted: Aug 2020Est. completion: Oct 2028Updated: Aug 2025

Phase 3Active, Not Recruiting

NCT04644575

Long-term Safety and Efficacy of Efanesoctocog Alfa (BIVV001) in Previously Treated Patients With Hemophilia A

Sponsor:Bioverativ, a Sanofi company

United StatesStarted: Feb 2021Est. completion: Jan 2027Updated: Aug 2025

Phase 3Active, Not Recruiting

NCT06224907

Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A

Sponsor:BioMarin Pharmaceutical

JapanStarted: Dec 2023Est. completion: Mar 2029Updated: May 2024

Last updated over 18 months ago

Phase 1Active, Not RecruitingGene Therapy

NCT03588299

Study to Test the Safety and How Well Patients With Severe Hemophilia A Respond to Treatment With BAY 2599023 (DTX 201), a Drug Therapy That Delivers a Healthy Version of the Defective Factor VIII Gene Into the Nucleus of Liver Cells Using an Altered, Non-infectious Virus (AAV) as a "Shuttle"

Sponsor:Bayer

United StatesStarted: Nov 2018Est. completion: Nov 2026Updated: Mar 2026

Phase 4Active, Not Recruiting

NCT05181618

A Study to Evaluate Overall Health, Physical Activity, and Joint Outcomes in Participants With Severe or Moderate Hemophilia A Without Factor VIII Inhibitors on Emicizumab Prophylaxis

Sponsor:Hoffmann-La Roche

United StatesStarted: Jun 2022Est. completion: May 2027Updated: Jan 2026

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

Last sync: March 16, 2026 at 6:12 AM

Severe Hemophilia a — Clinical Trials

Trial Landscape

By Approach

By Phase

Sponsor Types

Top Sponsors

Trial Locations

29 Trials

Open Trials22

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

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

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

A Gene Therapy Study of SPK-8011QQ in Adults With Severe or Moderately Severe Hemophilia A

Pharmacokinetic Comparison of Efanesoctocog Alfa vs Other EHL-rFVIII Products in Participants With Severe Haemophilia A

A Study to Learn About How Changing Therapy From Emicizumab to Marstacimab Affects People With the Severe Hemophilia A.

ASC618 Gene Therapy in Hemophilia A Patients

Nuwiq for Perioperative Management Of Patients With Haemophilia A on Emicizumab Regular Prophylaxis Study

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

POCUS: Hemostatic Potential and Joint Health in Patients With Severe Hemophilia A on Novel Replacement Therapies

Active Trials7

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

A Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Subcutaneous Emicizumab in Participants From Birth to 12 Months of Age With Hemophilia A Without Inhibitors

Study to Evaluate the Efficacy and Safety of PF-07055480 / Giroctocogene Fitelparvovec Gene Therapy in Moderately Severe to Severe Hemophilia A Adults

Long-term Safety and Efficacy of Efanesoctocog Alfa (BIVV001) in Previously Treated Patients With Hemophilia A

Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A

Study to Test the Safety and How Well Patients With Severe Hemophilia A Respond to Treatment With BAY 2599023 (DTX 201), a Drug Therapy That Delivers a Healthy Version of the Defective Factor VIII Gene Into the Nucleus of Liver Cells Using an Altered, Non-infectious Virus (AAV) as a "Shuttle"

A Study to Evaluate Overall Health, Physical Activity, and Joint Outcomes in Participants With Severe or Moderate Hemophilia A Without Factor VIII Inhibitors on Emicizumab Prophylaxis