Evaluation Of Acceptability And Satisfaction Of Hemophilia Patients Treated With FusENGO

Hemophilia A Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01959919
• Other study ID #: B1831081
• Status: Completed
• Start date: January 27, 2014
• Est. completion date: December 12, 2016

Study information

• Verified date: September 2018
• Source: Pfizer
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 86
• Est. completion date: December 12, 2016
• Est. primary completion date: December 12, 2016
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 18 Years to 65 Years

Eligibility

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.

Related Conditions & MeSH terms

• Hemophilia A

Intervention

Device:
• Refacto FusENGO
Dosage will be chosen by PI based on patient condition.

Locations

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

Sponsors (1)

Lead Sponsor	Collaborator
Pfizer

Country where clinical trial is conducted

Italy, 

Outcome

Type	Measure	Description	Time frame	Safety issue
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.	Final Visit (Month 8)
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.	Final Visit (Month 8)
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.	Final Visit (Month 8)
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.	Final Visit (Month 8)
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.	Final Visit (Month 8)
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.	Final Visit (Month 8)
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.	Baseline up to 28 days after last dose of drug (up to Month 12)

External Links

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