Hemophilia A
Conditions
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.
Interventions
DEVICERefacto FusENGO
Dosage will be chosen by PI based on patient condition.
Sponsors
Pfizer
Study design
Observational model
CASE_ONLY
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
MALE
Age
18 Years to 65 Years
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Burden of Clotting Factor Treatment Score | 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. |
| Ease of Using Clotting Factor Treatment Score | 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 | Final Visit (Month 8) | In this outcome measure time consumed for performing steps to reconstitute the drug prior to infusion of drug is reported. |
| Overall Satisfaction Score With Refacto AF FuseNGO | 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. |
| Impact of Clotting Factor Treatment Score | 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 | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Adverse Events and Serious Adverse Events | 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. |
Countries
Italy
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Refacto AF FuseNGo Participants with haemophilia A, who were receiving clotting factor VIII treatment prior to enrollment in this study, used Refacto AF FuseNGo (intravenous infusion of moroctocog alfa, a human recombinant clotting factor VIII, delivered by system FuseNGo) either prophylactically or on-demand and were observed for maximum up to Week 52. Dosage and use of Refacto AF FuseNGo was based on investigator's judgement according to approved SmPC. | 86 |
| Total | 86 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Did not meet the eligibility criteria | 2 |
Baseline characteristics
| Characteristic | Refacto AF FuseNGo |
|---|---|
| Age, Continuous | 37.99 Years STANDARD_DEVIATION 11.206 |
| Burden of Clotting Factor Treatment Score | 11.88 units on scale STANDARD_DEVIATION 4.652 |
| Impact of Clotting Factor Treatment Score | 17.86 units on scale STANDARD_DEVIATION 7.117 |
| Risk Associated With Clotting Factor Treatment Score | 19.69 units on scale STANDARD_DEVIATION 7.076 |
| Sex: Female, Male Female | 0 Participants |
| Sex: Female, Male Male | 86 Participants |
| Time for Reconstructing the Drug | 10.79 minutes STANDARD_DEVIATION 7.419 |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 3 / 86 |
| serious Total, serious adverse events | 0 / 86 |
Outcome results
Primary
Burden of Clotting Factor Treatment Score
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.
Time frame: Final Visit (Month 8)
Population: Per-protocol population included all participants who received at least 1 treatment using the Refacto AF FuseNGo and completed the two final questionnaires.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Refacto AF FuseNGo | Burden of Clotting Factor Treatment Score | 13.55 units on scale | Standard Deviation 4.275 |
Comparison: Comparison between burden of clotting factor treatment score at Final Visit Month 8 and baseline visitp-value: 0Wilcoxon Signed Ranks Test
Primary
Ease of Using Clotting Factor Treatment Score
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 frame: Final Visit (Month 8)
Population: Per-protocol population included all participants who received at least 1 treatment using the Refacto AF FuseNGo and completed the two final questionnaires.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Refacto AF FuseNGo | Ease of Using Clotting Factor Treatment Score | 31.15 units on a scale | Standard Deviation 6.539 |
Comparison: Comparison between ease of using clotting factor treatment score at Final visit (Month 8) and baseline visitp-value: 0Wilcoxon Signed Ranks Test
Primary
Impact of Clotting Factor Treatment Score
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.
Time frame: Final Visit (Month 8)
Population: Per-protocol population included all participants who received at least 1 treatment using the Refacto AF FuseNGo and completed the two final questionnaires.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Refacto AF FuseNGo | Impact of Clotting Factor Treatment Score | 20.15 units on scale | Standard Deviation 6.167 |
Comparison: Comparison between impact of clotting factor treatment score at Final Visit Month 8 and baseline visitp-value: 0Wilcoxon Signed Ranks Test
Primary
Overall Satisfaction Score With Refacto AF FuseNGO
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.
Time frame: Final Visit (Month 8)
Population: Per-protocol population included all participants who received at least 1 treatment using the Refacto AF FuseNGo and completed the two final questionnaires. Here N signifies number of participants evaluable for this outcome measure.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Refacto AF FuseNGo | Overall Satisfaction Score With Refacto AF FuseNGO | 93.90 units on a scale | Standard Deviation 18.883 |
Primary
Risk Associated With Clotting Factor Treatment Score
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.
Time frame: Final Visit (Month 8)
Population: Per-protocol population included all participants who received at least 1 treatment using the Refacto AF FuseNGo and completed the two final questionnaires.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Refacto AF FuseNGo | Risk Associated With Clotting Factor Treatment Score | 21.40 units on scale | Standard Deviation 6.733 |
Comparison: Comparison between risk associated with clotting factor treatment score at Final Visit Month 8 and baseline visitp-value: 0.001Wilcoxon Signed Ranks Test
Primary
Time for Reconstructing the Drug
In this outcome measure time consumed for performing steps to reconstitute the drug prior to infusion of drug is reported.
Time frame: Final Visit (Month 8)
Population: Per-protocol population all participants who received at least 1 treatment using the Refacto AF FuseNGo and completed the two final questionnaires. Here Number of Participants Analyzed (N) signifies number of participants who were evaluable for this outcome measure.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Refacto AF FuseNGo | Time for Reconstructing the Drug | 6.247 minutes | Standard Deviation 4.3409 |
Comparison: Comparison between time for reconstructing the drug at Final visit (Month 8) and baseline visitp-value: 095% CI: [3.411, 5.733]t-test, 2 sided
Secondary
Number of Participants With Adverse Events and Serious Adverse Events
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.
Time frame: Baseline up to 28 days after last dose of drug (up to Month 12)
Population: Safety population included all the participants who were enrolled and received at least 1 dose of Refacto AF FuseNGO.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Refacto AF FuseNGo | Number of Participants With Adverse Events and Serious Adverse Events | Adverse Events | 3 participants |
| Refacto AF FuseNGo | Number of Participants With Adverse Events and Serious Adverse Events | Serious Adverse Events | 0 participants |