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PF-06741086 Long-term Treatment in Severe Hemophilia

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03363321
Recruitment Status : Completed
First Posted : December 6, 2017
Last Update Posted : October 20, 2020
Sponsor:
Information provided by (Responsible Party):
Pfizer

Tracking Information
First Submitted Date  ICMJE November 30, 2017
First Posted Date  ICMJE December 6, 2017
Last Update Posted Date October 20, 2020
Actual Study Start Date  ICMJE May 30, 2018
Actual Primary Completion Date August 5, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: December 17, 2018)
  • Frequency, severity and causal relationship of treatment emergent adverse events (TEAEs) and withdrawals due to TEAEs [ Time Frame: Day 1 up to Day 393 ]
    Treatment-related AE was any untoward medical occurrence attributed to study drug in a participant who received study drug. Serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly.
  • Percentage of subjects with laboratory abnormalities [ Time Frame: Day 1 to Day 393 ]
    Following parameters were analyzed for laboratory examination: hematology, serum chemistry, and urinalysis.
  • Number of subjects with changes from baseline in vital signs [ Time Frame: Day 1 to Day 393 ]
    Following parameters were analyzed for vital sign examination: blood pressure, pulse rate, temperature, respiration rate.
  • Number of subjects with change from baseline in electrocardiogram (ECG) parameters [ Time Frame: Day 1 to Day 29 ]
    Following parameters were analyzed for ECG examinations: QT interval (QTc), QTcF interval (Fridericia's Correction), QRS complex, PR interval, and RR interval.
  • Percentage of subjects with injection site reactions [ Time Frame: Day 1 to Day 393 ]
    Following parameters were analyzed for injection site reaction examinations: induration, erythema, edema, rash, pruritus.
  • Percentage of subejct with infusion site reactions [ Time Frame: Day 1 to Day 393 ]
    Following parameters were analyzed for infusion site reactions: induration, erythema, edema, rash, pruritus.
Original Primary Outcome Measures  ICMJE
 (submitted: November 30, 2017)
  • Frequency, severity and causal relationship of treatment emergent adverse events (TEAEs) and withdrawals due to TEAEs [ Time Frame: Day 1 up to Day 197 ]
    Treatment-related AE was any untoward medical occurrence attributed to study drug in a participant who received study drug. Serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly.
  • Percentage of subjects with laboratory abnormalities [ Time Frame: Day 1 to Day 197 ]
    Following parameters were analyzed for laboratory examination: hematology, serum chemistry, and urinalysis.
  • Number of subjects with changes from baseline in vital signs [ Time Frame: Day 1 to Day 197 ]
    Following parameters were analyzed for vital sign examination: blood pressure, pulse rate, temperature, respiration rate.
  • Number of subjects with change from baseline in electrocardiogram (ECG) parameters [ Time Frame: Day 1 to Day 29 ]
    Following parameters were analyzed for ECG examinations: QT interval (QTc), QTcF interval (Fridericia's Correction), QRS complex, PR interval, and RR interval.
  • Percentage of subjects with injection site reactions [ Time Frame: Day 1 to Day 197 ]
    Following parameters were analyzed for injection site reaction examinations: induration, erythema, edema, rash, pruritus.
  • Percentage of subjects with infusion site reactions [ Time Frame: Day 1 to Day 197 ]
    Following parameters were analyzed for infusion site reactions: induration, erythema, edema, rash, pruritus.
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: December 17, 2018)
  • Number of bleeding episodes [ Time Frame: Day 1 to Day 393 ]
    Bleeding episodes requiring treatment with factor replacement.
  • Number of treatments for breakthrough bleeding episodes [ Time Frame: Day 1 to Day 393 ]
    Treatments with factor replacement.
Original Secondary Outcome Measures  ICMJE
 (submitted: November 30, 2017)
  • Number of bleeding episodes [ Time Frame: Day 1 to Day 197 ]
    Bleeding episodes requiring treatment with factor replacement.
  • Number of treatments for breakthrough bleeding episodes [ Time Frame: Day 1 to Day 197 ]
    Treatments with factor replacement.
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE PF-06741086 Long-term Treatment in Severe Hemophilia
Official Title  ICMJE A MULTICENTER, OPEN-LABEL STUDY TO EVALUATE THE LONG-TERM SAFETY, TOLERABILITY AND EFFICACY OF SUBCUTANEOUS PF-06741086 IN SUBJECTS WITH SEVERE HEMOPHILIA
Brief Summary This study is designed to evaluate the safety, tolerability and efficacy of long-term treatment with PF-06741086 in subjects with severe hemophilia who participated in the 3-month Phase 1b/2 B7841002 study. Additionally, de novo subjects will be recruited into this study.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Prevention
Condition  ICMJE Hemophilia A or B
Intervention  ICMJE Biological: PF-06741086
PF-06741086 subcutaneous injection
Study Arms  ICMJE
  • Experimental: PF-06741086 (Cohort 1)
    Intervention: Biological: PF-06741086
  • Experimental: PF-06741086 (Cohort 2)
    Intervention: Biological: PF-06741086
  • Experimental: PF-06741086 (Cohort 3)
    Intervention: Biological: PF-06741086
  • Experimental: PF-06741086 (Cohort 4)
    Intervention: Biological: PF-06741086
  • Experimental: PF-06741086 (Cohort 5)
    Intervention: Biological: PF-06741086
  • Experimental: PF-06741086 (Cohort 6)
    Intervention: Biological: 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 Completed
Actual Enrollment  ICMJE
 (submitted: September 8, 2020)
20
Original Estimated Enrollment  ICMJE
 (submitted: November 30, 2017)
36
Actual Study Completion Date  ICMJE August 5, 2020
Actual Primary Completion Date August 5, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Severe hemophilia A or B (Factor VIII or Factor IX activity ≤ 1%)
  • Subjects enrolled as Factor VIII or Factor IX inhibitor patients must have a positive inhibitor test result (above the upper limit of normal) at the local laboratory and must receive a bypass agent as primary treatment for bleeding episodes.
  • Episodic (on-demand) treatment regimen prior to screening
  • At least 6 acute bleeding episodes during the 6-month period prior to screening

Exclusion Criteria:

  • Known coronary artery, thrombotic, or ischemic disease
  • Concomitant treatment with activated prothrombin complex concentrate
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 information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Brazil,   Chile,   Croatia,   Poland,   South Africa,   Switzerland,   United States
Removed Location Countries France,   Spain
 
Administrative Information
NCT Number  ICMJE NCT03363321
Other Study ID Numbers  ICMJE B7841003
2017-001255-31 ( EudraCT Number )
Has Data Monitoring Committee No
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 October 2020

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