Evaluation Of Acceptability And Satisfaction Of Hemophilia Patients Treated With FusENGO

• ClinicalTrials.gov Identifier: NCT01959919
• Recruitment Status: Completed
• First Posted: October 10, 2013
• Results First Posted: September 25, 2018
• Last Update Posted: September 25, 2018
• Sponsor: Pfizer
• Information provided by (Responsible Party): Pfizer

Tracking Information

• First Submitted Date: October 8, 2013
• First Posted Date: October 10, 2013
• Results First Submitted Date: November 13, 2017
• Results First Posted Date: September 25, 2018
• Last Update Posted Date: September 25, 2018
• Actual Study Start Date: January 27, 2014
• Actual Primary Completion Date: December 12, 2016 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: September 24, 2018)

• Ease of Using Clotting Factor Treatment Score [ Time Frame: Final Visit (Month 8) ]
  HaemoPREF is a participant rated 14-item instrument to measure experience of clotting factor treatment including ease of use, burden, impact, risk and influence of others on treatment choices. For determining score for ease of using clotting factor treatment, 4 items assessed by participants were: 1) ease to prepare treatment for injection, 2) ease to store treatment, 3) ease to dispose of containers, syringe and needle once used and 4) ease to use current treatment. Each of the 4 items was scored on a scale of 0 (not at all easy) to 10 (extremely easy), and were summed up, to give a total overall score range of 0 (no ease) to 40 (maximum ease). Higher scores indicate greater ease in using clotting factor treatment.
• Time for Reconstructing the Drug [ Time Frame: Final Visit (Month 8) ]
  In this outcome measure time consumed for performing steps to reconstitute the drug prior to infusion of drug is reported.
• Burden of Clotting Factor Treatment Score [ Time Frame: Final Visit (Month 8) ]
  HaemoPREF is a participant rated 14-item instrument to measure experience of clotting factor treatment including ease of use, burden, impact, risk and influence of others on treatment choices. For determining the score for burden of clotting factor treatment, 2 items were assessed by participants: 1) time consumption to treat with treatment and 2) difficulty in finding vein to inject treatment in to. Each of the 2 items was scored on a scale of 0 (most difficult) to 20 ( least difficult) and summed up to give a total overall score range of 0 (most burdened) to 40 (least burdened). Higher scores indicate a lower burden of treatment.
• Impact of Clotting Factor Treatment Score [ Time Frame: Final Visit (Month 8) ]
  HaemoPREF is a participant rated 14-item instrument to measure experience of clotting factor treatment including ease of use, burden, impact, risk and influence of others on treatment choices. For determining score for impact of clotting factor treatment, 3 items were assessed by participants: 1) difficulty to travel for holidays or works, 2) difficulty to perform daily activities including work or study and 3) difficulty to perform social or leisure activities. Each of the 3 items was scored on a scale of 0 (most challenging) to 10 (least challenging), and summed up to give a total overall score range of 0 (greatest negative impact) to 30 (least negative impact). Higher scores indicate less negative impact on daily life.
• Risk Associated With Clotting Factor Treatment Score [ Time Frame: Final Visit (Month 8) ]
  HaemoPREF is a participant rated 14-item instrument to measure experience of clotting factor treatment including ease of use, burden, impact, risk and influence of others on treatment choices. For determining score for risk associated with clotting factor treatment, 3 items were assessed by participants: 1) worried about getting infected with other disease while using the treatment, 2) worried to contaminate the treatment while preparing for injection and 3) worried to inject treatment by own. Each of the 3 items was scored on a scale of 0 (most concerned) to 10 (not at all concerned), and summed up to give a total overall score range of 0 (most worried) to 30 (least worried). Higher scores indicate lower levels of worry associated with treatment.
• Overall Satisfaction Score With Refacto AF FuseNGO [ Time Frame: Final Visit (Month 8) ]
  HaemoPREF: participant rated 14-item instrument to measure experience of clotting factor treatment. 14 items: 1) ease to prepare treatment for injection, 2) ease to store treatment, 3) ease to dispose container, syringe, needle once used and 4) ease to use treatment, 5) time consumed with treatment, 6) difficulty in finding a vein to inject treatment, 7) difficulty to travel, 8) difficulty to do daily activities, 9) difficulty to do social or leisure activities, 10) worried for getting infected with other disease while using the treatment, 11) worried to contaminate the treatment while preparing for injection, 12) worried to inject by own, 13) importance of family's opinion to use treatment, 14) importance what others use for their hemophilia. Each item was scaled from 0 (no satisfaction) to 10 (maximum satisfaction). Overall satisfaction score was the sum of 14 items, ranged from 0 (no satisfaction) to 140 (maximum satisfaction). Higher scores indicate greater treatment satisfaction.

Original Primary Outcome Measures (submitted: October 9, 2013)

• Ease of use, Time taken to reconstitute, Ease of storage, Rapidity to infuse, Impact on daily life of the therapy both on physical and social/ affective aspects, Perception about the therapy, Satisfaction [ Time Frame: 6 months ]
• Ease of use, Time taken to reconstitute, Ease of storage, Rapidity to infuse, Impact on daily life of the therapy both on physical and social/ affective aspects, Perception about the therapy, Satisfaction [ Time Frame: 12 months ]

Current Secondary Outcome Measures (submitted: September 24, 2018)

Number of Participants With Adverse Events and Serious Adverse Events [ Time Frame: Baseline up to 28 days after last dose of drug (up to Month 12) ]

An adverse event (AE) was any untoward medical occurrence in a participant who received treatment using Refacto AF Fusnego without regard to possibility of causal relationship. Serious adverse event 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 included both serious and non-serious AEs.

Original Secondary Outcome Measures (submitted: October 9, 2013)

• Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: 6 months ]
• Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: 12 months ]

• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Evaluation Of Acceptability And Satisfaction Of Hemophilia Patients Treated With FusENGO
• Official Title: Non Interventional Longitudinal Study To Assess The Acceptability And The Satisfaction Of Patients With Haemophilia A Treated Both On Demand And On Prophylaxis With A New Factor Viii Delivery System Fusengo
• Brief Summary: This study aims to investigate prospectively throughout a period of 3- 6 months and not to exceed 12 months (according to local therapeutic plans) patients experiences of treatment with Factor VIII in the new device named FuseNGO, a new delivery system for Factor VIII compared to their previous delivery system.
• Detailed Description: Not Provided
• Study Type: Observational
• Study Design: Observational Model: Case-Only; Time Perspective: Prospective
• Target Follow-Up Duration: Not Provided
• Biospecimen: Not Provided
• Sampling Method: Non-Probability Sample
• Study Population: Approximately 200 patients with Hemophilia A (all severity levels) will be enrolled in approximately 20 centers in Italy.
• Condition: Hemophilia A

Intervention

Device: Refacto FusENGO

Dosage will be chosen by PI based on patient condition.

Study Groups/Cohorts

Arm 1: Device

Intervention: Device: Refacto FusENGO

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: September 24, 2018): 86
• Original Estimated Enrollment (submitted: October 9, 2013): 200
• Actual Study Completion Date: December 12, 2016
• Actual Primary Completion Date: December 12, 2016 (Final data collection date for primary outcome measure)

Eligibility Criteria

Inclusion Criteria:

• Male adults (aged 18 to 65 years) with Haemophilia A (all severity levels) who currently use FVIII treatment either prophylactically or on-demand using traditional factor VIII delivery mechanisms.
• Patients advised for any reason by their physician to switch to a new factor VIII delivery device, namely FuseNGO, before receiving any details about this study or Patients who requested to be treated with a new factor VIII delivery device namely FuseNGO, before receiving any details about this study.

Exclusion Criteria:

• Patients not previously recommended by their physician to switch to this new factor VIII delivery system or Patients who did not previously ask their physician to be switched to this new factor VIII delivery system.
• Patients for which it is anticipated that 10 infusions will not occur in the 12 months following their inclusion in the study.

Sex/Gender

• Sexes Eligible for Study: Male
• Gender Based Eligibility: Yes

• Ages: 18 Years to 65 Years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Italy

Administrative Information

• NCT Number: NCT01959919
• Other Study ID Numbers: B1831081
• Has Data Monitoring Committee: No
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Responsible Party: Pfizer
• Study Sponsor: Pfizer
• Collaborators: Not Provided

Investigators

• Study Director: Pfizer CT.gov Call Center; Pfizer

• PRS Account: Pfizer
• Verification Date: September 2018