Hemophilia Clinical Trials: 179 Active Trials, 44 Changes This Month

25 trials changed in the last 30 days: 1 stopped, 9 with notable changes, 15 with minor changes only.

Stopped

Alphanate FVIII/VWF Concentrate in Type 3 Von Willebrand Patients

Trial terminated

Also in this update: Primary endpoint(s) modified; Results posted: Sep 30, 2026

Grifols Biologicals, LLCPhase 4 NCT00555555 Registry history of NCT00555555 on ClinicalTrials.gov (opens the registry)

Sep 30, 2026
Notable

Adverse Event Data Collection From the EUHASS Registry on Turoctocog Alfa Pegol

Primary and study completion pushed from Feb 1, 2026 to Dec 1, 2026

Novo Nordisk A/SNot specified NCT04682145 Registry history of NCT04682145 on ClinicalTrials.gov (opens the registry)

Oct 8, 2026
Notable

E-PROTECT · Efanesoctocog Prophylaxis Or The Optimization Of Treatment: Real-World Experience Of Taylorizing Clotting Therapy In Hemophilia A

Primary endpoint(s) modified

Nantes University HospitalNot specified NCT07830693 Registry history of NCT07830693 on ClinicalTrials.gov (opens the registry)

Sep 25, 2026
Notable

Substudy of PF 06838435 · Hemophilia B

Reclassified as terminated (was completed)

PfizerPhase 2 NCT03307980 Registry history of NCT03307980 on ClinicalTrials.gov (opens the registry)

Oct 5, 2026
Notable

Patient Preferences · Haemophilia A With and Without Inhibitors: a Discrete Choice Experiment in Germany

Enrolling by invitation only

Also in this update: Primary and study completion pushed from Oct 26, 2026 to Dec 26, 2026

Novo Nordisk A/SNot specified NCT07653139 Registry history of NCT07653139 on ClinicalTrials.gov (opens the registry)

Oct 6, 2026
Notable

3D Ultrasound in Hemophilic Ankles

Enrolling by invitation only

The Hospital for Sick ChildrenNot applicable NCT07452575 Registry history of NCT07452575 on ClinicalTrials.gov (opens the registry)

Oct 14, 2026
Notable

TRANSHEMO2 · Determinants and Consequences of the Transition to Adulthood · Severe Haemophilia: TRANSHEMO 2, an Ancillary Study to the TRANSHEMO Project

Recruitment opened

Assistance Publique Hopitaux De MarseilleNot specified NCT07418827 Registry history of NCT07418827 on ClinicalTrials.gov (opens the registry)

Oct 8, 2026
Notable

Bone Health · Severe Hemophilia A on Factor VIII vs Factor Mimetic Prophylaxis (Efa Emi Bone Health Study)

Recruitment opened

Also in this update: Primary completion pushed from Aug 1, 2031 to Sep 2031; Study completion pushed from Aug 1, 2032 to Sep 2032

Arkansas Children's Hospital Research InstituteNot specified NCT07582276 Registry history of NCT07582276 on ClinicalTrials.gov (opens the registry)

Oct 5, 2026
Notable

TG-INSIGHT With Joint POCUS, Hemostatic Potential · Severe Hemophilia A on Novel Replacement and Substitution FVIII Therapies

Recruitment opened

University of Texas Southwestern Medical CenterNot specified NCT07692217 Registry history of NCT07692217 on ClinicalTrials.gov (opens the registry)

Sep 29, 2026
Notable

OP-724-H202 · Foscenvivint in Liver Cirrhosis Patients Caused by HIV/HCV Co-infection With Hemophilia

Enrollment increased: 4 → 7 participants

Kiminori Kimura, MDPhase 2 NCT07681089 Registry history of NCT07681089 on ClinicalTrials.gov (opens the registry)

Oct 5, 2026

Show 15 trials with minor changes only

Minor

LIBERTY · Provide Continued Access to Treatment for Patients Completing a Previous Trial With Efanesoctocog Alfa

Enrollment closed, study ongoing

Also in this update: Final enrollment: 89 participants (planned 104)

Swedish Orphan BiovitrumPhase 3 NCT06716814 Registry history of NCT06716814 on ClinicalTrials.gov (opens the registry)

Oct 19, 2026
Minor

ATLAS-OLE · Fitusiran · Hemophilia A or B, With or Without Inhibitory Antibodies to Factor VIII or IX

Trial completed

Also in this update: Primary completion date changed from estimate of Nov 5, 2026 to Sep 14, 2026

Genzyme, a Sanofi CompanyPhase 3 NCT03754790 Registry history of NCT03754790 on ClinicalTrials.gov (opens the registry)

Sep 30, 2026
Minor

Joint Health · Haemophilia A Receiving Real-world Prophylactic Treatment With Efanesoctocog Alfa

Enrollment closed, study ongoing

Swedish Orphan BiovitrumPhase 4 NCT06940830 Registry history of NCT06940830 on ClinicalTrials.gov (opens the registry)

Sep 30, 2026
Minor

How Well · 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

Trial completed

BayerPhase 1/2 NCT03588299 Registry history of NCT03588299 on ClinicalTrials.gov (opens the registry)

Sep 23, 2026
Minor

Research Study of the Health of Joints · Haemophilia Taking the Medicine Esperoct

Trial completed

Also in this update: Primary completion date changed from estimate of Jun 30, 2026 to Apr 30, 2026; Final enrollment: 123 participants (planned 100)

Novo Nordisk A/SNot specified NCT05621746 Registry history of NCT05621746 on ClinicalTrials.gov (opens the registry)

Oct 8, 2026
Minor

PROTECT-ALT · Impact of Efanesoctocog Alfa on Long-term Joint Health · Hemophilia A in Taiwan

Enrollment closed, study ongoing

Also in this update: Final enrollment: 79 participants (planned 100)

SanofiNot specified NCT06684314 Registry history of NCT06684314 on ClinicalTrials.gov (opens the registry)

Sep 25, 2026
Minor

A-MORE · 48-Month Study to Evaluate Long-Term Effectiveness of Elocta on Joint Health

Trial completed

Also in this update: Primary completion date changed from estimate of Jul 2026 to May 4, 2026

Swedish Orphan BiovitrumNot specified NCT04293523 Registry history of NCT04293523 on ClinicalTrials.gov (opens the registry)

Sep 22, 2026
Minor

ATLAS-KIDS · Fitusiran Prophylaxis in Male Participants Aged 1 to Less Than 12 Years With Hemophilia A or B

Trial sites expanded: 30 → 41 locations

SanofiPhase 3 NCT07285460 Registry history of NCT07285460 on ClinicalTrials.gov (opens the registry)

Oct 19, 2026
Minor

Photobiomodulation in Hemophilia Patients

Trial completed

Riphah International UniversityNot applicable NCT07579585 Registry history of NCT07579585 on ClinicalTrials.gov (opens the registry)

Sep 30, 2026
Minor

Validity and Reliability of the Two-minute Step Test in Hemophilia

Completed without a posted recruiting period

Also in this update: Study completion date changed from estimate of Jun 25, 2026 to Aug 25, 2026; Final enrollment: 31 participants (planned 35)

Hasan Kalyoncu UniversityNot specified NCT07425327 Registry history of NCT07425327 on ClinicalTrials.gov (opens the registry)

Sep 23, 2026
Minor

How Changing Therapy From Emicizumab to Marstacimab Affects · the Severe Hemophilia A

Trial sites expanded: 7 → 10 locations

PfizerPhase 1 NCT06703606 Registry history of NCT06703606 on ClinicalTrials.gov (opens the registry)

Oct 15, 2026
Minor

Gene Therapy Study of SPK-8011QQ · Severe or Moderately Severe Hemophilia A

Trial sites expanded: 3 → 6 locations

Hoffmann-La RochePhase 1/2 NCT07226206 Registry history of NCT07226206 on ClinicalTrials.gov (opens the registry)

Oct 13, 2026
Minor

ZEBRHA 2 · NXT007 Compared to Emicizumab Prophylaxis · Hemophilia A

Trial sites expanded: 42 → 59 locations

Hoffmann-La RochePhase 3 NCT07416604 Registry history of NCT07416604 on ClinicalTrials.gov (opens the registry)

Oct 6, 2026
Minor

Giroctocogene Fitelparvovec or Fidanacogene Elaparvovec · Hemophilia A or B Respectively

Trial sites expanded: 23 → 39 locations

PfizerPhase 3 NCT05568719 Registry history of NCT05568719 on ClinicalTrials.gov (opens the registry)

Oct 2, 2026
Minor

ZEBRHA 1 · NXT007 Compared to Factor VIII Prophylaxis · Hemophilia A

Trial sites expanded: 27 → 32 locations

Hoffmann-La RochePhase 3 NCT07416526 Registry history of NCT07416526 on ClinicalTrials.gov (opens the registry)

Sep 23, 2026

Stopped: an ongoing trial was terminated, suspended or withdrawn.
Notable: the trial’s plan changed such as a big move in its timeline or size, a new primary endpoint, sponsor or phase, or recruiting opened or resumed.
Minor: routine progress and housekeeping, such as sites added or enrollment closed.

Hemophilia clinical trials

What changed in the last 30 days

Alphanate FVIII/VWF Concentrate in Type 3 Von Willebrand Patients

Adverse Event Data Collection From the EUHASS Registry on Turoctocog Alfa Pegol

E-PROTECT · Efanesoctocog Prophylaxis Or The Optimization Of Treatment: Real-World Experience Of Taylorizing Clotting Therapy In Hemophilia A

Substudy of PF 06838435 · Hemophilia B

Patient Preferences · Haemophilia A With and Without Inhibitors: a Discrete Choice Experiment in Germany

3D Ultrasound in Hemophilic Ankles

TRANSHEMO2 · Determinants and Consequences of the Transition to Adulthood · Severe Haemophilia: TRANSHEMO 2, an Ancillary Study to the TRANSHEMO Project

Bone Health · Severe Hemophilia A on Factor VIII vs Factor Mimetic Prophylaxis (Efa Emi Bone Health Study)

TG-INSIGHT With Joint POCUS, Hemostatic Potential · Severe Hemophilia A on Novel Replacement and Substitution FVIII Therapies

OP-724-H202 · Foscenvivint in Liver Cirrhosis Patients Caused by HIV/HCV Co-infection With Hemophilia

LIBERTY · Provide Continued Access to Treatment for Patients Completing a Previous Trial With Efanesoctocog Alfa

ATLAS-OLE · Fitusiran · Hemophilia A or B, With or Without Inhibitory Antibodies to Factor VIII or IX

Joint Health · Haemophilia A Receiving Real-world Prophylactic Treatment With Efanesoctocog Alfa

How Well · 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

Research Study of the Health of Joints · Haemophilia Taking the Medicine Esperoct

PROTECT-ALT · Impact of Efanesoctocog Alfa on Long-term Joint Health · Hemophilia A in Taiwan

A-MORE · 48-Month Study to Evaluate Long-Term Effectiveness of Elocta on Joint Health

ATLAS-KIDS · Fitusiran Prophylaxis in Male Participants Aged 1 to Less Than 12 Years With Hemophilia A or B

Photobiomodulation in Hemophilia Patients

Validity and Reliability of the Two-minute Step Test in Hemophilia

How Changing Therapy From Emicizumab to Marstacimab Affects · the Severe Hemophilia A

Gene Therapy Study of SPK-8011QQ · Severe or Moderately Severe Hemophilia A

ZEBRHA 2 · NXT007 Compared to Emicizumab Prophylaxis · Hemophilia A

Giroctocogene Fitelparvovec or Fidanacogene Elaparvovec · Hemophilia A or B Respectively

ZEBRHA 1 · NXT007 Compared to Factor VIII Prophylaxis · Hemophilia A

New sponsor entrants, last 90 days sponsors with their first trial in this area

Active trials by phase

Top sponsors by active trials in this area

Narrower conditions

Early Phase 1		2
Phase 1		9
Phase 1/2		12
Phase 2		3
Phase 2/3		4
Phase 3		36
Phase 4		15