Evaluation Of Acceptability And Satisfaction Of Hemophilia Patients Treated With FusENGO

Completed

Conditions: Hemophilia A

Interventions: Device: Refacto FusENGO

Registration Number: NCT01959919

Lead Sponsor: Pfizer

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 available

Recruitment & Eligibility

Status: COMPLETED

Sex: Male

Target Recruitment: 86

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.

Study & Design

Study Type: OBSERVATIONAL

Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Arm 1: Device	Refacto FusENGO	-

Primary Outcome Measures

Name	Time	Method
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.
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.
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.
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.
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.

Secondary Outcome Measures

Name	Time	Method
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.

Trial Locations

Locations (19): Anna Chiara Giuffrida
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Verona, Italy
Ospedale SS. Annunziata - USL 1
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Sassari, Sardegna, Italy
Azienda Ospedale Policlinico Consorziale
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Bari, Italy
Ospedale Ferrarrotto
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Catania, Italy
Azienda Ospedaliera Ciaccio - Ospedale Pugliese
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Catanzaro, Italy
Azienda Ospedaliera Careggi
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Firenze, Italy
Centro regionale di diagnosi e trattamento dell'Emofilia e delle malattie dell'emostasi e trombosi
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Macerata, Italy
Dipartimento Medicina Clinica e Sperimentale
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Napoli, Italy
Centro Emofilia e Trombosi - Centro delle Microcitemie
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Napoli, Italy
Policlinico P Giaccone
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Palermo, Italy
Arcispedale S. Maria Nuova, AO di Reggio Emilia
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Reggio Emilia, Italy
Azienda Ospedaliera di Padova
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Padova, Italy
SOS Malattie Emorragiche e Trombotiche AOU "S.M.M." Udine
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Udine, Italy
SC Servizio Immunotrasfusionale
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Torino, Italy
Ospedale Molinette
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Torino, Italy
Istituto di Ematologia
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Roma, Lazio, Italy
Struttura Complessa Di Oncoematologia Pediatrica E Patologia Della Coagulzione
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Cagliari, Italy
Ospedale Maggiore Policlinico Mangiagalli E Regina Elena
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Milano, Italy
Policlinico Agostino Gemelli
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Rome, Italy