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Post-Marketing Surveillance To Observe Safety And Efficacy Of Xyntha Solofuse Prefilled Syringe

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ClinicalTrials.gov Identifier: NCT03034044
Recruitment Status : Completed
First Posted : January 27, 2017
Results First Posted : April 5, 2019
Last Update Posted : April 5, 2019
Sponsor:
Information provided by (Responsible Party):
Pfizer

Brief Summary:

This study aims to observe the safety and efficacy of the Xyntha Solofuse prefilled syringe in the setting of routine practice. The primary objective is to detect medically significant events (factor VIII inhibitor). The secondary objective is to observe the overall efficacy and safety of the Xyntha Solofuse prefilled syringe including serious adverse events. In this open-label, non-comparative, observational, non-interventional, retrospective and multi-center study, post-marketing surveillance data will be collected retrospectively for up to 6 months from the initial administration day of the Xyntha Solofuse prefilled syringe injected into patients who have been administered the Xyntha Solofuse prefilled syringe.

As specified in the product approval issued by the Ministry of Food and Drug Safety, the study will be conducted for 4 years from the approval date. At least 600 study subjects will be enrolled in this study to meet the MFDS requirements. Although 600 is the assigned number of study subjects, the number of cases will be adjusted considering the actual number of enrolled subjects after the study start day.


Condition or disease
Factor VIII Deficiency, Congenital Factor 8 Deficiency, Congenital Autosomal Hemophilia A Classic Hemophilia Hemophilia A, Congenital

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Study Type : Observational
Actual Enrollment : 106 participants
Observational Model: Case-Only
Time Perspective: Retrospective
Official Title: POST-MARKETING SURVEILLANCE TO OBSERVE SAFETY AND EFFICACY OF XYNTHA SOLOFUSE PREFILLED SYRINGE
Actual Study Start Date : January 2017
Actual Primary Completion Date : January 17, 2018
Actual Study Completion Date : January 17, 2018

Resource links provided by the National Library of Medicine





Primary Outcome Measures :
  1. Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: From the first administration of Xyntha up to 6 months ]
    An AE was any untoward medical occurrence in participants who received Xyntha without regard to possibility of causal relationship. 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. Treatment Emergent Adverse Event (TEAE) was adverse event that started or worsened in severity after first administration of Xyntha Solofuse up to 6 months. AEs included both serious and non-serious adverse event.

  2. Number of Participants With Adverse Events (AEs) by Severity [ Time Frame: From the first administration of Xyntha up to 6 months ]
    An AE was any untoward medical occurrence in participants who received Xyntha without regard to possibility of causal relationship. 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. AEs were classified according to the severity in 3 categories a) mild - AEs does not interfere with participant's usual function b) moderate - AEs interferes to some extent with participant's usual function c) severe - AEs interferes significantly with participant's usual function, and was determined based on investigator's discretion.

  3. Number of Participants Who Discontinued Due to Adverse Events [ Time Frame: From the first administration of Xyntha up to 6 months ]
    An AE was any untoward medical occurrence in participants who received study drug without regard to possibility of causal relationship.

  4. Number of Participants With Treatment-Related Adverse Events (AEs) and Serious Adverse Events [ Time Frame: From the first administration of Xyntha up to 6 months ]
    Treatment-related AE was any untoward medical occurrence attributed to Xyntha in a participant who received study drug. 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. Relatedness to treatment was assessed by investigator. AEs included both serious and non-serious AEs.

  5. Number of Participants Who Died Due to Adverse Events [ Time Frame: From the first administration of Xyntha up to 6 months ]
    An AE was any untoward medical occurrence in participants who received Xyntha without regard to possibility of causal relationship.

  6. Number of Participants With Overall Responses on a 4-Point Scale to the Injections Used to Treat Bleeding: On-Demand Treatment According to Surgery [ Time Frame: From the first administration of Xyntha up to 6 months ]
    On-demand treatment according to surgery: treatment to increase factor in preparation for surgery. Overall responses to all injection of Xyntha Solofuse prefilled syringe used to treat bleeding in on-demand treatment (administration of an unscheduled dose of Xyntha Solofuse prefilled syringe to stop bleeding) according to surgery was assessed on 4 point scale of 1=excellent, 2=good, 3=moderate and 4=no response, higher score = better response. Excellent= Definite pain relief and/or improvement in signs of bleeding within 8 hours after an infusion, with no additional infusion, good= Definite pain relief and/or improvement in signs of bleeding within 8 hours after an infusion, with 1 additional infusion, moderate= Probable/slight improvement starting after 8 hours following infusion, with >=1 additional infusion administered for complete resolution of bleeding episode and no response= No improvement at all between infusions/during 24 hour interval following an infusion/condition worsens

  7. Number of Participants With Overall Responses on a 4-Point Scale to the Injections Used to Treat Bleeding: On-Demand Treatment According to Bleeding [ Time Frame: From the first administration of Xyntha up to 6 months ]
    On-demand treatment according to bleeding: treatment administered for spontaneous bleeding/abrasion; not requiring surgery. Overall responses to all injection of Xyntha used to treat bleeding in on-demand treatment (administration of an unscheduled dose of Xyntha Solofuse prefilled syringe to stop bleeding) according to bleeding was assessed on 4 point scale of 1=excellent, 2=good, 3=moderate,4=no response, higher score=better response. Excellent= Definite pain relief and/or improvement in signs of bleeding within 8 hours after an infusion, with no additional infusion, good= Definite pain relief and/or improvement in signs of bleeding within 8 hours after an infusion, with 1 additional infusion, moderate= Probable/slight improvement after 8 hours following infusion, with >=1 additional infusion administered for complete resolution of bleeding episode and no response= No improvement at all between infusions/during 24 hour interval following an infusion, or condition worsens.

  8. Number of Participants With Less Than Expected Therapeutic Effect (LETE): On-Demand Treatment According to Surgery [ Time Frame: Within 24 hours of on-demand treatment (anytime within the observation period of 6 months) ]
    LETE for on-demand treatment (administration of an unscheduled dose of Xyntha Solofuse prefilled syringe to stop bleeding) was defined as "no response" rated after each infusion of 2 consecutive infusions within 24 hours after on-demand treatment.

  9. Number of Participants With Less Than Expected Therapeutic Effect (LETE): On-Demand Treatment According to Bleeding [ Time Frame: Within 24 hours of on-demand treatment (anytime within the observation period of up to 6 months) ]
    LETE for on-demand treatment ((administration of an unscheduled dose of Xyntha Solofuse prefilled syringe to stop bleeding) was defined as "no response" rated after each infusion of 2 consecutive infusions within 24 hours after on-demand treatment) was defined as "no response" rated after each infusion of 2 consecutive infusions within 24 hours after on-demand treatment.

  10. Number of Infusions Required to Treat Each New Bleeding Episode [ Time Frame: From the first administration of Xyntha up to 6 months ]
    It was calculated as total number of injections given throughout the study divided by total number of bleeding events.

  11. Average Dose of Infusions Per Bleeding Event: On-Demand Treatment According to Surgery [ Time Frame: From the first administration of Xyntha up to 6 months ]
    The average dose of Xyntha per bleeding event according to surgery in on-demand treatment was calculated as total dose of Xyntha (in IU) throughout the study divided by total number of bleeding event. On-demand treatment (administration of an unscheduled dose of Xyntha Solofuse prefilled syringe to stop bleeding) according to surgery means treatment to increase factor in preparation for surgery.

  12. Average Dose of Infusions Per Bleeding Event: On-Demand Treatment According to Bleeding [ Time Frame: From the first administration of Xyntha up to 6 months ]
    The average dose of Xyntha per bleeding event was calculated as total dose of Xyntha throughout the study (in International Units [IU]) divided by total number of bleeding incidence. On-demand treatment (administration of an unscheduled dose of Xyntha Solofuse prefilled syringe to stop bleeding) according to bleeding means treatment administered due to spontaneous bleeding or abrasion.

  13. Percentage of Participants With Bleeding Event [ Time Frame: From the first administration of Xyntha up to 6 months ]
  14. Annualized Bleeding Rates (ABRs) [ Time Frame: Within 48 hours after the prophylactic administration of Xyntha (within the duration of 6 months) ]
    Annualized bleeding rate defined as number of bleeds under prophylactic setting (defined as bleeding occurred after 48 hours of prophylactic therapy [administration of Xyntha not for the treatment of a bleed but for the prevention of bleeding]) divided by (/) [(number of prophylactic therapy participants)*0.5)]

  15. Number of Participants With Less Than Expected Therapeutic Effect (LETE): Prophylactic Therapy [ Time Frame: From the first administration of Xyntha up to 6 months ]
    Less than expected therapeutic effect for prophylaxis therapy defined as breakthrough (spontaneous/non-traumatic) bleeding occurred within 48 hours of prophylaxis infusion (defined as: administration of Xyntha not for the treatment of a bleed but for the prevention of bleeding).

  16. Average Dose of Infusions Per Bleeding Event: Prophylactic Therapy [ Time Frame: From the first administration of Xyntha up to 6 months ]
    The average dose of Xyntha per bleeding event according to prophylaxis therapy treatment was calculated as total dose of Xyntha (in IU) throughout the study divided by total number of bleeding event. Prophylaxis therapy defined as breakthrough (spontaneous/non-traumatic) bleeding occurred within 48 hours of prophylaxis infusion (defined as administration of Xyntha not for the treatment of a bleed but for the prevention of bleeding).

  17. Total Factor VIII Consumption [ Time Frame: 6 months ]
    Total factor VIII consumption for each participant was calculated by sum of the total amount of Xyntha Solofuse (in IU) infused for each Xyntha Solofuse infusion.



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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
The study population is Hemophilia A (congenital factor VIII deficiency) patients who have been administered the Xyntha Solofuse prefilled syringe (as part of routine treatment at the Korean health care canter which has certified investigators).
Criteria

-. Inclusion criteria

To be eligible to enroll in this study, the study subjects will have to meet all the following inclusion criteria:

  1. Hemophilia A (congenital factor VIII deficiency) patients who have been administered according to the indication of the product 1) Control and prevention of bleeding episodes and for routine and surgical prophylaxis in patients with hemophilia A (congenital factor VIII deficiency) 2) This drug does not contain von Willebrand factor and, therefore, is not indicated in von Willebrand's disease
  2. Those who have been administered the Xyntha Solofuse prefilled syringe at least once - Exclusion criteria

Patients who satisfy the following criteria are not included in the study according to the local labeling:

  1. Patients who have a history of hypersensitivity to the Xyntha Solofuse prefilled syringe or the ingredients of this drug.
  2. Patients who have a history of hypersensitivity to hamster proteins.
  3. Patients who have bleeding disorders other than hemophilia A.
  4. Patients who have a history of FVIII inhibitors, or currently have or are suspected of having FVIII inhibitors. In case inhibitor titers quantified in Bethesda Units in the laboratory test results are within the normal laboratory range or at least 0.6 BU/mL. If laboratory tests cannot be performed, the investigator will determine whether or not inhibitors exist based on the clinical assessment results that show a decrease in efficacy of the replacement of FVIII (e.g. bleeding at least once, if the replacement of anti-bleeding agents is needed to be administered, and if frequency or dosage of replacement FVIII therapy needs to be increased).
  5. Use of immunomodulatory therapy. (e.g. intravenous injection of immunoglobulin, use of regular systemic corticosteroids, cyclosporine, and mediators of anti-TNF-α)

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03034044


Sponsors and Collaborators
Pfizer
Investigators
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Study Director: Pfizer CT.gov Call Center Pfizer
  Study Documents (Full-Text)

Documents provided by Pfizer:
Study Protocol  [PDF] May 17, 2018
Statistical Analysis Plan  [PDF] March 20, 2018


Additional Information:
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Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT03034044     History of Changes
Other Study ID Numbers: B1831086
First Posted: January 27, 2017    Key Record Dates
Results First Posted: April 5, 2019
Last Update Posted: April 5, 2019
Last Verified: January 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
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.

Additional relevant MeSH terms:
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Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn