Factor VIII Deficiency, CongenitalNCT03034044
Post-Marketing Surveillance To Observe Safety And Efficacy Of Xyntha Solofuse Prefilled Syringe
An observational study of Factor VIII Deficiency, Congenital and Factor 8 Deficiency, Congenital, run by Pfizer.
Trial 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). Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Factor VIII Deficiency, Congenital, Factor 8 Deficiency, Congenital, Autosomal Hemophilia A
- How many people
- 106 participants
- Where
- No sites listed yet
- Study dates
- Jan 2017to 17 Jan 2018
- Phase
- Observational
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
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).
-. Inclusion criteria
To be eligible to enroll in this study, the study subjects will have to meet all the following inclusion criteria:
- 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
- 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:
- Patients who have a history of hypersensitivity to the Xyntha Solofuse prefilled syringe or the ingredients of this drug.
- Patients who have a history of hypersensitivity to hamster proteins.
- Patients who have bleeding disorders other than hemophilia A.
- 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).
- Use of immunomodulatory therapy. (e.g. intravenous injection of immunoglobulin, use of regular systemic corticosteroids, cyclosporine, and mediators of anti-TNF-α)
About this study
Official title: POST-MARKETING SURVEILLANCE TO OBSERVE SAFETY AND EFFICACY OF XYNTHA SOLOFUSE PREFILLED SYRINGE
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.
- Conditions
- Factor VIII Deficiency, Congenital, Factor 8 Deficiency, Congenital, Autosomal Hemophilia A, Classic Hemophilia, Hemophilia A, Congenital
- Medical subject headings
- Hemophilia A
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
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.
Measured: From the first administration of Xyntha up to 6 months
Number of Participants With Adverse Events (AEs) by Severity
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.
Measured: From the first administration of Xyntha up to 6 months
Number of Participants Who Discontinued Due to Adverse Events
An AE was any untoward medical occurrence in participants who received study drug without regard to possibility of causal relationship.
Measured: From the first administration of Xyntha up to 6 months
Number of Participants With Treatment-Related Adverse Events (AEs) and Serious Adverse Events
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.
Measured: From the first administration of Xyntha up to 6 months
Number of Participants Who Died Due to Adverse Events
An AE was any untoward medical occurrence in participants who received Xyntha without regard to possibility of causal relationship.
Measured: From the first administration of Xyntha up to 6 months
Number of Participants With Overall Responses on a 4-Point Scale to the Injections Used to Treat Bleeding: On-Demand Treatment According to Surgery
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
Measured: From the first administration of Xyntha up to 6 months
Number of Participants With Overall Responses on a 4-Point Scale to the Injections Used to Treat Bleeding: On-Demand Treatment According to Bleeding
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.
Measured: From the first administration of Xyntha up to 6 months
Number of Participants With Less Than Expected Therapeutic Effect (LETE): On-Demand Treatment According to Surgery
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.
Measured: Within 24 hours of on-demand treatment (anytime within the observation period of 6 months)
Number of Participants With Less Than Expected Therapeutic Effect (LETE): On-Demand Treatment According to Bleeding
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.
Measured: Within 24 hours of on-demand treatment (anytime within the observation period of up to 6 months)
Number of Infusions Required to Treat Each New Bleeding Episode
It was calculated as total number of injections given throughout the study divided by total number of bleeding events.
Measured: From the first administration of Xyntha up to 6 months
Average Dose of Infusions Per Bleeding Event: On-Demand Treatment According to Surgery
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.
Measured: From the first administration of Xyntha up to 6 months
Average Dose of Infusions Per Bleeding Event: On-Demand Treatment According to Bleeding
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.
Measured: From the first administration of Xyntha up to 6 months
Percentage of Participants With Bleeding Event
Measured: From the first administration of Xyntha up to 6 months
Annualized Bleeding Rates (ABRs)
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)]
Measured: Within 48 hours after the prophylactic administration of Xyntha (within the duration of 6 months)
Number of Participants With Less Than Expected Therapeutic Effect (LETE): Prophylactic Therapy
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).
Measured: From the first administration of Xyntha up to 6 months
Average Dose of Infusions Per Bleeding Event: Prophylactic Therapy
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).
Measured: From the first administration of Xyntha up to 6 months
Total Factor VIII Consumption
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.
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Jan 2017 (estimated)
- Study startJan 2017
- Primary completion17 Jan 2018
- Study completion17 Jan 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Factor VIII Deficiency, CongenitalStudies of the same condition, open and under way.
- More trials run by PfizerThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Retrospective
- Enrolment
- 106 participants (actual)
Sponsor and investigators
- Lead sponsor
- Pfizer
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Pfizer CT.gov Call Center, Study director, Pfizer
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03034044
- Sponsor's protocol ID
- B1831086
- Status last verified
- Jan 2019
- First submitted
- 30 Nov 2016
- Last updated
- 13 Nov 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Individual data sharing
- No: 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.
Study at a glance
From the registry record, last updated by the sponsor on 13 Nov 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- POST-MARKETING SURVEILLANCE TO OBSERVE SAFETY AND EFFICACY OF XYNTHA SOLOFUSE PREFILLED SYRINGE
- ClinicalTrials.gov ID
- NCT03034044
- Acronym / sponsor ID
- B1831086
- Conditions
- Factor VIII Deficiency, Congenital, Factor 8 Deficiency, Congenital, Autosomal Hemophilia A, Classic Hemophilia, Hemophilia A, Congenital
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 106 participants (actual)
- Lead sponsor
- PfizerIndustry (pharma, biotech, devices)
- Study start
- Jan 2017 (actual)
- Primary completion
- 17 Jan 2018 (actual)
- Study completion
- 17 Jan 2018 (actual)
- First posted
- 27 Jan 2017 (estimated)
- Results
- Posted 5 Apr 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Case only
- Time perspective
- Retrospective
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- 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).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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.
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.
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.
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.
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.
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
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.
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.
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.
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.
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.
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.
Percentage of Participants With Bleeding Event
Time frame: From the first administration of Xyntha up to 6 months
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)]
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).
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).
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.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Nov 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- To obtain contact information for a study center near you, click here.Link provided by the sponsor.
- To obtain contact information for a study center near you, click here.Link provided by the sponsor.
- Individual participant dataSharing planned: No. 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Pfizer CT.gov Call Center study director
Pfizer
Questions about this trial
What is this clinical trial studying?
An observational study of Factor VIII Deficiency, Congenital and Factor 8 Deficiency, Congenital, run by Pfizer.
The full registered description is in the About this study section and the official record.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 13 Nov 2023. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Yes. Results are posted in the registry (first posted 5 Apr 2019). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). The study lists 17 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Nov 2023.