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Study of the Efficacy and Safety PF-06741086 in Adult and Teenage Participants With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B | Clinical Trials | Clareo Health

COMPLETEDPHASE3

Study of the Efficacy and Safety PF-06741086 in Adult and Teenage Participants With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B

An Open-Label Study in Adolescent and Adult Severe (Coagulation Factor Activity <1%) Hemophilia A Participants With or Without Inhibitors or Moderately Severe to Severe Hemophilia B Participants (Coagulation Factor Activity ≤2%) With or Without Inhibitors Comparing Standard Treatment to PF-06741086 Prophylaxis

NCT03938792•Pfizer

View on ClinicalTrials.gov

Summary

Treatment with PF-06741086 is anticipated to demonstrate a clinically relevant advantage and/or a major contribution to patient care in comparison to current methods of treatment for hemophilia A or B because it works differently than factor replacement products and will work in the presence of inhibitors. The potential for once weekly (QW) subcutaneous (SC) administration provides for treatment options in the absence of reliable vascular access, increased convenience and may enable better compliance. Combined, these qualities should result in a reduction of bleeding episodes.

Eligibility

Age

12 - 74 years

Sex

MALE

Healthy Volunteers

Inclusion Criteria

•Participants with a diagnosis of severe hemophilia A or moderately severe to severe hemophilia B with a minimum weight of 35 kg at screening.
•Participant or legally authorized representative, or participant's caregiver capable of giving signed informed consent (or minor assent, when applicable).
•Participants who are enrolled into the Non-Inhibitor Cohort must also meet the following criteria:
•No detectable or documented history of inhibitors
•Participants on FVIII/FIX routine prophylaxis who have demonstrated at least 80% compliance with scheduled prophylaxis regimen during 6 months prior to enrollment and are willing to continue to receive routine prophylaxis treatment with FVIII/FIX replacement during the Observational Phase.
•Participants with on-demand treatment regimen with ≥6 acute bleeding episodes (spontaneous or traumatic) that required coagulation factor infusion during the 6 months period prior to enrollment and willing to continue to receive on demand treatment during the Observational Phase.
•Participants who are enrolled into the Inhibitor Cohort must also meet the following criteria:
•Documentation of current high titer inhibitor (≥5 BU/mL) or current low titer inhibitor (\<5 BU/mL) refractory to FVIII or FIX replacement and with FVIII or FIX recovery \<60% of expected within previous 6 months prior to enrolment into the Observational Phase
•Hemophilia A participants with on-demand treatment regimen with ≥6 bleeding episodes or hemophilia B participants with ≥4 bleeding episodes (spontaneous or traumatic) necessitating treatment with bypass factor during the 6 months prior to Enrollment into Observational Phase and willing to continue to receive on-demand treatment during the Observational Phase.
•Participants who have documented inhibitors while on factor-replacement therapy but who do not meet the quantitative inhibitor criteria described in the prior bullet at the time of Screening (eg, participant with a previously documented high-titer inhibitor (≥5 BU/mL) and whose condition precludes re-challenge with FVIII or FIX replacement) may be considered for eligibility on a case-by-case basis with prior agreement from the Pfizer Medical Monitor
+1 more criteria

Exclusion Criteria

•Previous or current treatment for and/or history of coronary artery diseases, venous or arterial thrombosis or ischemic disease
•Known planned surgical procedure during the planned study period.
•Known hemostatic defect other than hemophilia A or B.
•Abnormal renal or hepatic function
•Current unstable liver or biliary disease
•Abnormal hematologic parameters
•Other acute or chronic medical or psychiatric condition or laboratory abnormality that may increase the risk associated with study participation or investigational product administration or may interfere with the interpretation of study results and, in the judgment of the investigator,
•Current routine prophylaxis with bypassing agent or non-coagulation non-factor- replacement therapy, or any previous treatment with a gene therapy product for treatment of hemophilia (participants treated with prophylaxis using bypassing agents or who had prior treatment with non-factor products may be considered on a case-by-case basis).
•Regular, concomitant therapy with immunomodulatory drugs
•\- Ongoing or planned use of immune tolerance induction during the Observational Phase or Active Treatment Phase, or prophylaxis with FVIII or FIX replacement at any time after initiation of treatment with study intervention during the Active Treatment Phase
+6 more criteria

Locations (62)

USF Health Morsani Center For Advanced Healthcare

Tampa, Florida, United States

University of Iowa

Iowa City, Iowa, United States

Northwell Health HTC

New Hyde Park, New York, United States

Washington Institute for Coagulation

Seattle, Washington, United States

National Specialized Hospital for the Active Treatment of Hematological Diseases - EAD, Sofia

Sofia, Bulgaria

UMHAT "Prof.Dr. Stoyan Kirkovich"

Stara Zagora, Bulgaria

McMaster University Medical Centre - Hamilton Health Sciences

Hamilton, Ontario, Canada

McMaster Children's Hospital

Hamilton, Ontario, Canada

McMaster University

Hamilton, Ontario, Canada

The Hospital For Sick Children

Toronto, Ontario, Canada

Key Dates

Start Date

March 9, 2020

Primary Completion Date

April 29, 2025

Completion Date

April 29, 2025

Last Updated

June 10, 2025

Enrollment

189

ACTUAL participants

Conditions

Hemophilia AHemophilia B

Interventions

PF-06741086

DRUG

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

NCT06379789

Hemophilia B

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RECRUITINGPHASE1/PHASE2

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

NCT07226206

Hemophilia A

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RECRUITINGPHASE1/PHASE2

A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: June 10, 2025Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

View ClinicalTrials.gov Terms and Conditions

Study of the Efficacy and Safety PF-06741086 in Adult and Teenage Participants With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B

Inclusion Criteria

Exclusion Criteria

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

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

A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A

An Observational Cohort Study to Characterize the Effectiveness and Safety of HEMGENIX® in Patients With Hemophilia B

National Longitudinal Cohort of Hematological Diseases

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

Study of the Efficacy and Safety PF-06741086 in Adult and Teenage Participants With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B

Inclusion Criteria

Exclusion Criteria

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

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

A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A

An Observational Cohort Study to Characterize the Effectiveness and Safety of HEMGENIX® in Patients With Hemophilia B

National Longitudinal Cohort of Hematological Diseases

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

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