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Trial record 11 of 159 for:    bleeding episodes | ( Map: United States )

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

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ClinicalTrials.gov Identifier: NCT03938792
Recruitment Status : Not yet recruiting
First Posted : May 6, 2019
Last Update Posted : August 7, 2019
Sponsor:
Information provided by (Responsible Party):
Pfizer

Tracking Information
First Submitted Date  ICMJE May 2, 2019
First Posted Date  ICMJE May 6, 2019
Last Update Posted Date August 7, 2019
Estimated Study Start Date  ICMJE October 2019
Estimated Primary Completion Date November 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: May 2, 2019)
  • 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)
  • 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 ]
  • Incidence of anti drug antibody [ADA] against PF-06741086 [ Time Frame: Throughout Active Treatment Phase (12 months) ]
  • Incidence of clinically significant persistent neutralizing antibody [NAb] against PF-06741086 [ Time Frame: Throughout Active Treatment Phase (12 months) ]
  • Incidence and severity of injection site reaction [ Time Frame: Throughout Active Treatment Phase (12 months) ]
  • Number of participants with clinically significant changes from baseline in physical exam [ Time Frame: From Baseline through Observation and Active Treatment (approximately 18 months) ]
  • Incidence of clinically significant laboratory value abnormalities [ Time Frame: From Screening through Observation and Active Treatment (approximately 18 months) ]
  • Incidence of severe hypersensitivity and anaphylactic reactions [ Time Frame: From Screening through Observational and Active Treatment (approximately 18 months) ]
  • Incidence of adverse events and serious adverse events [ Time Frame: From screening through Observation and Active treatment (approximately 18 months) ]
  • Number of participants with clinically significant changes from baseline in vital signs [ Time Frame: From Baseline through Observation and Active Treatment (approximately 18 months) ]
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT03938792 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: May 2, 2019)
  • 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
  • Incidence of joint bleeds [ Time Frame: Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months ]
  • Incidence of spontaneous bleeds [ Time Frame: Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months ]
  • Incidence of target joint bleeds [ Time Frame: Through Observational Phase (6 months) and Active Treatment Phase (12 months) for total of approximately 18 months ]
  • Incidence of total bleeds (treated and untreated) [ Time Frame: Through Observational and Active Treatment Phases (18 Months) ]
  • 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 ]
  • 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 ]
  • 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 ]
  • 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 ]
  • 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 ]
  • 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 ]
  • 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 ]
  • 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 ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Study of the Efficacy and Safety PF-06741086 in Adult and Teenage Patients With Severe Hemophilia A or B
Official Title  ICMJE 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
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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE 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
Condition  ICMJE
  • Hemophilia A
  • Hemophilia B
Intervention  ICMJE 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.
Study Arms  ICMJE Experimental: PF-06741086
Participants will be assigned to treatment with PF-06741086 after a 6 month Observation Phase on their current hemophilia regimen.
Intervention: Drug: PF-06741086
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Not yet recruiting
Estimated Enrollment  ICMJE
 (submitted: May 2, 2019)
145
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE November 2022
Estimated Primary Completion Date November 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria

• Participants with a diagnosis of severe hemophilia A or B

Participants who are enrolled into the Non-Inhibitor Cohort must also meet the following criteria:

  • No detectable or documented history of inhibitors
  • Participants outside the US and Canada with FVIII/FIX replacement, and willing to continue to receive routine prophylaxis treatment with FVIII/FIX replacement during the Observational Phase. Participants in the US and Canada receiving routine prophylaxis treatment not eligible to be enrolled.
  • Participants with on-demand treatment regimen with ≥6 acute bleeding episodes (spontaneous and/or traumatic) that required coagulation factor infusion during the 6 months period prior to Screening 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 30 days prior to Baseline of Observational Phase
  • Participants with on-demand treatment regimen with ≥6 bleeding episodes (spontaneous and/or traumatic) necessitating treatment with bypass factor for at least 6 months prior to screening and willing to continue to receive on-demand treatment during the Observational Phase.

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
  • Abnormal coagulation activity
  • 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
  • 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.
  • 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
  • Clinically relevant ECG abnormalities that may affect participant safety or interpretation of study results.
  • 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.
Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Gender Based Eligibility: Yes
Ages  ICMJE 12 Years to 74 Years   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Pfizer CT.gov Call Center 1-800-718-1021 ClinicalTrials.gov_Inquiries@pfizer.com
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03938792
Other Study ID Numbers  ICMJE B7841005
2018-003660-31 ( EudraCT Number )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
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
Responsible Party Pfizer
Study Sponsor  ICMJE Pfizer
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Pfizer CT.gov Call Center Pfizer
PRS Account Pfizer
Verification Date August 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP