Working...
ClinicalTrials.gov
ClinicalTrials.gov Menu

Evaluation Of Acceptability And Satisfaction Of Hemophilia Patients Treated With FusENGO

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
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:
Information provided by (Responsible Party):
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.

Condition or disease Intervention/treatment
Hemophilia A Device: Refacto FusENGO

Layout table for study information
Study Type : Observational
Actual Enrollment : 86 participants
Observational Model: Case-Only
Time Perspective: Prospective
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
Actual Study Start Date : January 27, 2014
Actual Primary Completion Date : December 12, 2016
Actual Study Completion Date : December 12, 2016

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Hemophilia

Group/Cohort Intervention/treatment
Arm 1: Device Device: Refacto FusENGO
Dosage will be chosen by PI based on patient condition.




Primary Outcome Measures :
  1. 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.

  2. 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.

  3. 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.

  4. 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.

  5. 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.

  6. 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.


Secondary Outcome Measures :
  1. 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.



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   Male
Gender Based Eligibility:   Yes
Accepts Healthy Volunteers:   No
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.
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01959919


Locations
Layout table for location information
Italy
Istituto di Ematologia
Roma, Lazio, Italy, 00161
Ospedale SS. Annunziata - USL 1
Sassari, Sardegna, Italy, 07100
Azienda Ospedale Policlinico Consorziale
Bari, Italy, 70121
Struttura Complessa Di Oncoematologia Pediatrica E Patologia Della Coagulzione
Cagliari, Italy, 09100
Ospedale Ferrarrotto
Catania, Italy, 95124
Azienda Ospedaliera Ciaccio - Ospedale Pugliese
Catanzaro, Italy, 88100
Azienda Ospedaliera Careggi
Firenze, Italy, 50134
Centro regionale di diagnosi e trattamento dell'Emofilia e delle malattie dell'emostasi e trombosi
Macerata, Italy, 62100
Ospedale Maggiore Policlinico Mangiagalli E Regina Elena
Milano, Italy
Dipartimento Medicina Clinica e Sperimentale
Napoli, Italy, 80131
Centro Emofilia e Trombosi - Centro delle Microcitemie
Napoli, Italy, 81044
Azienda Ospedaliera di Padova
Padova, Italy, 35128
Policlinico P Giaccone
Palermo, Italy, 90100
Arcispedale S. Maria Nuova, AO di Reggio Emilia
Reggio Emilia, Italy, 42100
Policlinico Agostino Gemelli
Rome, Italy
Ospedale Molinette
Torino, Italy, 10126
SC Servizio Immunotrasfusionale
Torino, Italy
SOS Malattie Emorragiche e Trombotiche AOU "S.M.M." Udine
Udine, Italy, 33100
Anna Chiara Giuffrida
Verona, Italy, 37126
Sponsors and Collaborators
Pfizer
Investigators
Layout table for investigator information
Study Director: Pfizer CT.gov Call Center Pfizer

Additional Information:
Layout table for additonal information
Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT01959919     History of Changes
Other Study ID Numbers: B1831081
First Posted: October 10, 2013    Key Record Dates
Results First Posted: September 25, 2018
Last Update Posted: September 25, 2018
Last Verified: September 2018

Additional relevant MeSH terms:
Layout table for MeSH terms
Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn