About Bolder Science

Our mission is to provide healthcare professionals with unbiased clinical research information, easily.

Currently, you can access the following clinical trials being conducted worldwide:

359,057 studies
in
219 countries
Clinical trial information and results are updated daily from ClinicalTrials.gov. The latest data update was conducted on 01/21/2021.
This website is for US healthcare professionals

Log In to Bolder Science

or

Don't have an account? Sign Up

Please enter your email address.

You will receive a link to create a new password via email.

Log In

Create an Account

or
(optional) ?

Welcome, !

Please complete the following 4 questions to ensure you receive the information that best suits your needs.

Clinical Trials of Interest

When I’m looking for information on clinical trials, I usually am interested in...

finding clinical trials in which to enroll my patients

Rarely Often

finding newly launched clinical trials (for all phases)

Rarely Often

updates on status changes for clinical trials

Rarely Often

pipeline molecules

Rarely Often

Drug Interventions

Enter up to 3 drug interventions you are currently interested in:

Clinical trial information and results are updated daily from ClinicalTrials.gov. The latest data update was conducted on 01/21/2021.

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

Clinicaltrials.gov identifier NCT03938792

Recruitment Status Recruiting

First Posted May 6, 2019

Last update posted October 13, 2020

Study Description

Brief 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.

  • Condition or Disease:Hemophilia A
    Hemophilia B
  • Intervention/Treatment: Drug: PF-06741086
  • Phase: Phase 3
Detailed Description

N/A

Study Design
  • Study Type: Interventional
  • Estimated Enrollment: 145 participants
  • Intervention Model: Crossover Assignment
  • Intervention Model Description: This is a one way Cross-Over Prevention study with 1 Arm that has No masking.
  • Masking: None (Open Label) ()
  • Primary Purpose: Treatment
  • Official Title: An Open-Label Study in Adolescent and Adult Severe (Coagulation Factor Activity <1%) Hemophilia A or B Patients With or Without Inhibitors Comparing Standard Treatment to PF-06741086 Prophylaxis
  • Actual Study Start Date: March 2020
  • Estimated Primary Completion Date: August 2023
  • Estimated Study Completion Date: August 2023
Arms and interventions
Arm Intervention/treatment
Experimental: PF-06741086
Participants will be assigned to treatment with PF-06741086 after a 6 month Observation Phase on their current hemophilia regimen.
Drug: PF-06741086
300 milligrams(mg) subcutaneous (sc) loading dose followed by 150 mg sq once weekly (qw). 300 mg sc qw is prescribed for participants who meet dose escalation criteria.
Outcome Measures
  • Primary Outcome Measures: 1. Incidence and severity of thromboticangiopathy [ Time Frame: Throughout Active Treatment Phase (12 months) ]
  • 2. Incidence of intravascular coagulation/consumption coagulopathy [ Time Frame: Throughout Active Treatment Phase (12 months) ]
  • 3. Annualized bleeding rate (ABR) of treated bleeding events [ Time Frame: Through Observational Phase (6months) and Active Treatment Phase (12 months) for total of approximately 18 months ]
    Derived for each subject for each treatment period by using the following formula: ABR = number of bleeds requiring treatments/ (days on treatment period / 365.25)
  • 4. Incidence and severity of thrombotic events [ Time Frame: Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months ]
  • 5. Incidence of anti drug antibody [ADA] against PF-06741086 [ Time Frame: Throughout Active Treatment Phase (12 months) ]
  • 6. Incidence of clinically significant persistent neutralizing antibody [NAb] against PF-06741086 [ Time Frame: Throughout Active Treatment Phase (12 months) ]
  • 7. Incidence and severity of injection site reaction [ Time Frame: Throughout Active Treatment Phase (12 months) ]
  • 8. Number of participants with clinically significant changes from baseline in physical exam [ Time Frame: From Baseline through Observation and Active Treatment (approximately 18 months) ]
  • 9. Incidence of clinically significant laboratory value abnormalities [ Time Frame: From Screening through Observation and Active Treatment (approximately 18 months) ]
  • 10. Incidence of severe hypersensitivity and anaphylactic reactions [ Time Frame: From Screening through Observational and Active Treatment (approximately 18 months) ]
  • 11. Incidence of adverse events and serious adverse events [ Time Frame: From screening through Observation and Active treatment (approximately 18 months) ]
  • 12. Number of participants with clinically significant changes from baseline in vital signs [ Time Frame: From Baseline through Observation and Active Treatment (approximately 18 months) ]
  • Secondary Outcome Measures: 1. Total coagulation factor and/or bypass product consumption [ Time Frame: Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months ]
    The Total Factor/bypass product consumed per participant as measured in total IU and IU/kg per month and per year
  • 2. Incidence of joint bleeds [ Time Frame: Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months ]
  • 3. Incidence of spontaneous bleeds [ Time Frame: Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months ]
  • 4. Incidence of target joint bleeds [ Time Frame: Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months ]
  • 5. Incidence of total bleeds (treated and untreated) [ Time Frame: Through Observational and Active Treatment Phases (18 Months) ]
  • 6. Percentage of participants with no bleeding episodes [ Time Frame: Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months ]
  • 7. Change from baseline in the Hemophilia Joint Health Score (HJHS) [ Time Frame: Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months ]
  • 8. Change from baseline in (Haemophilia Adult Quality of Life Questionnaire (Haem-A-QoL) [ Time Frame: Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months ]
  • 9. Change from baseline in Haemophilia Quality of Life Questionnaire for Children (Haemo-QoL) [ Time Frame: Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months ]
  • 10. Change from baseline in Hemophilia Adult Activities List (HAL) [ Time Frame: Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months ]
  • 11. Change from baseline in Hemophilia Pediatric Activities List (PedHAL) [ Time Frame: Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months ]
  • 12. Patient Global Impression of Change - Hemophilia (PGIC-H) [ Time Frame: Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months ]
  • 13. Change from baseline in EuroQol 5 Dimensions 5 Level (EQ-5D-5L) [ Time Frame: Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months ]
Eligibility Criteria
  • Ages Eligible for Study: 12 to 74 Years (Child, Adult, Older Adult)
  • Sexes Eligible for Study: Male
  • Accepts Healthy Volunteers: No
Criteria

Inclusion Criteria

- Participants with a diagnosis of severe hemophilia A or B with a minimum weight of 30
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 - Participants with on-demand treatment regimen with ≥6 bleeding episodes (spontaneous and/or traumatic) necessitating treatment with bypass factor during the 6 months prior to enrollment in the Observation Phase and willing to continue to receive on-demand treatment during this 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 approval from the Pfizer Medical Monitor 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 - Regular, concomitant therapy with immunomodulatory drugs - Ongoing or planned use of immune tolerance induction or prophylaxis with FVIII or FIX replacement during the Active Treatment Phase. - Previous exposure to PF 06741086 during to participation in studies B7841002 and B7841003. - Participation in other studies involving investigational drug(s) within 30 days (or as determined by local requirements) or 5 half-lives prior to study entry and/or during study participation. - CD4 cell count ≤200/uL if human immunodeficiency virus (HIV)-positive - Screening ECG that demonstrates clinically relevant abnormalities that may affect participant safety or interpretation of study results. - Individuals with hypersensitivity or an allergic reaction to hamster protein or other components of the study intervention. - Investigator site staff members directly involved in the conduct of the study and their family members, site staff members otherwise supervised by the investigator, or participants who are Pfizer employees, including their family members, directly involved in the conduct of the study.

Contacts and Locations
Contacts

Contact: Pfizer CT.gov Call Center 1-800-718-1021 ClinicalTrials.gov_Inquiries@pfizer.com

Locations
Show 40 Study Locations
Sponsors and Collaborators

Pfizer

Investigators

Study Director: Pfizer CT.gov Call Center Pfizer

More Information
  • Responsible Party: Pfizer
  • ClinicalTrials.gov Identifier: NCT03938792 History of Changes
  • Other Study ID Numbers: B7841005, 2018-003660-31
  • First Posted: May 6, 2019 Key Record Dates
  • Last Update Posted: October 13, 2020
  • Last Verified: October 2020
  • Individual Participant
    Data (IPD) Sharing
    Statement:
  • Plan to Share IPD: Yes
  • Plan Description: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.
  • URL: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests
  • Studies a U.S. FDA-regulated Drug Product: Yes
  • Studies a U.S. FDA-regulated Device Product: No
  • Keywords provided by Pfizer: Factor VIII
    Factor IX
    Inhibitors
    Anti-Tissue Factor Pathway Inhibitor (TFPI)
    Subcutaneous (sc)
    Prophylaxis
    On-Demand
  • Additional relevant MeSH terms: Hemophilia A Hemophilia B