Hemophilia ANCT01959919

Evaluation Of Acceptability And Satisfaction Of Hemophilia Patients Treated With FusENGO

An observational study of Hemophilia A, run by Pfizer at 19 sites in Italy.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

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. Read more about this study

Who is it for?: Ages 18 to 65 years, men only
Conditions the study is for: Hemophilia A
What is being tested?: Refacto FusENGO
Type of treatment: Medical device
How many people: 86 participants
Where: 19 sitesItaly
Study dates: 27 Jan 2014to 12 Dec 2016
Phase: Observational
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 to 65 years
Men only
No healthy volunteers
Adult, Older adult

Study population: Approximately 200 patients with Hemophilia A (all severity levels) will be enrolled in approximately 20 centers in Italy.

Who may be able to join

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.

Who cannot join

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.

Trial locations and contacts

19 sites in 1 country.

City, postcode or hospital

19 sites in 1 country

Italy19 sites

Azienda Ospedale Policlinico Consorziale
Bari, 70121
Struttura Complessa Di Oncoematologia Pediatrica E Patologia Della Coagulzione
Cagliari, 09100
Ospedale Ferrarrotto
Catania, 95124
Azienda Ospedaliera Ciaccio - Ospedale Pugliese
Catanzaro, 88100
Azienda Ospedaliera Careggi
Florence, 50134
Ospedale Maggiore Policlinico Mangiagalli E Regina Elena
Milan
Dipartimento Medicina Clinica e Sperimentale
Naples, 80131
Centro Emofilia e Trombosi - Centro delle Microcitemie
Naples, 81044
Azienda Ospedaliera di Padova
Padova, 35128
Policlinico P Giaccone
Palermo, 90100
Centro regionale di diagnosi e trattamento dell'Emofilia e delle malattie dell'emostasi e trombosi
Province of Macerata, 62100
Arcispedale S. Maria Nuova, AO di Reggio Emilia
Reggio Emilia, 42100
Istituto di Ematologia
Rome, Lazio, 00161
Policlinico Agostino Gemelli
Rome
Ospedale SS. Annunziata - USL 1
Sassari, Sardinia, 07100
Ospedale Molinette
Torino, 10126
SC Servizio Immunotrasfusionale
Torino
SOS Malattie Emorragiche e Trombotiche AOU "S.M.M." Udine
Udine, 33100
Anna Chiara Giuffrida
Verona, 37126

Other trials in Italy

About this study

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

Conditions: Hemophilia A
Being tested: Refacto FusENGO
Medical subject headings: Hemophilia A

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Refacto FusENGODevice
Dosage will be chosen by PI based on patient condition.

Study groups in NCT01959919
Study group	Role	What they receive
Arm 1: Device		Not described Receives: Device: Refacto FusENGO

What is being measured

The primary outcome is the main question the study is designed to answer.

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.
Measured: Final Visit (Month 8)
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.
Measured: Final Visit (Month 8)
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.
Measured: Final Visit (Month 8)
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.
Measured: Final Visit (Month 8)
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.
Measured: Final Visit (Month 8)
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.
Measured: Final Visit (Month 8)

Secondary outcomes (1)

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.
Measured: Baseline up to 28 days after last dose of drug (up to Month 12)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

27 Jan 201412 Dec 2016

First posted10 Oct 2013 (estimated)
Study start27 Jan 2014
Primary completion12 Dec 2016
Study completion12 Dec 2016

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Hemophilia AStudies of the same condition, open and under way.
More trials run by PfizerThe sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Observational
Observational model: Case only
Time perspective: Prospective
Enrolment: 86 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01959919
Sponsor's protocol ID: B1831081
Status last verified: Sep 2018
First submitted: 8 Oct 2013
Last updated: 25 Sep 2018
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingObservational study
The CACHE Study: Coronary Artery Care in HaEmophilia
Haemophilia, Cardiovascular Diseases
Active, not recruitingObservational study
A Study to Learn About the Study Medicine -Hympavzi in Congenital Hemophilia Patients Without Inhibitors in Japan.
Hemophilia A or B
RecruitingPhase 1/2
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A
Hemophilia A
Active, not recruitingPhase 3
A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B
Hemophilia B
Not yet recruiting
Safety of Intraosseous Anaesthesia in Children With Haemophilia
Haemophilia A, Haemophilia B

All Hemophilia A trials

Study at a glance

From the registry record, last updated by the sponsor on 25 Sep 2018. Recruitment status is as the sponsor reported it: confirm with the study team.

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
ClinicalTrials.gov ID: NCT01959919
Acronym / sponsor ID: B1831081
Conditions: Hemophilia A
Study type: Observational
Recruitment status: Completed
Enrolment: 86 participants (actual)
Lead sponsor: PfizerIndustry (pharma, biotech, devices)
Study start: 27 Jan 2014 (actual)
Primary completion: 12 Dec 2016 (actual)
Study completion: 12 Dec 2016 (actual)
First posted: 10 Oct 2013 (estimated)
Results: Posted 25 Sep 2018

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Observational model: Case only
Time perspective: Prospective
Number of arms: 1

Arm	Type	Receives
Arm 1: Device	–	Device: Refacto FusENGO

Who may qualify

Ages: 18 to 65 years
Sex: Male only
Healthy volunteers: Not accepted
Age groups: Adult, Older adult
Study population: Approximately 200 patients with Hemophilia A (all severity levels) will be enrolled in approximately 20 centers in Italy.

Key inclusion criteria (2 listed)

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.

Key exclusion criteria (2 listed)

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.

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
Refacto FusENGODosage will be chosen by PI based on patient condition.	Device	Arm 1: Device

Primary outcome

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.

1 secondary outcome

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)

Results and safety

Results posted to the registry on 25 Sep 2018, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Refacto AF FuseNGo	86	84

Primary outcome: Ease of Using Clotting Factor Treatment Score

Time frame: Final Visit (Month 8). Mean (Standard Deviation). Unit: units on a scale

Measure	Refacto AF FuseNGo n = 84
Value	31.15 (6.539)

Refacto AF FuseNGo: Mean Difference (Final Values): -4.907, p = 0.000, Wilcoxon Signed Ranks Test

Population analysed: Per-protocol population included all participants who received at least 1 treatment using the Refacto AF FuseNGo and completed the two final questionnaires.

Primary outcome: Time for Reconstructing the Drug

Time frame: Final Visit (Month 8). Mean (Standard Deviation). Unit: minutes

Measure	Refacto AF FuseNGo n = 83
Value	6.247 (4.3409)

Refacto AF FuseNGo: Mean Difference (Final Values): 4.572 (95% CI 3.411 to 5.733), p = 0.000, t-test, 2 sided

Population analysed: 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.

Primary outcome: Burden of Clotting Factor Treatment Score

Time frame: Final Visit (Month 8). Mean (Standard Deviation). Unit: units on scale

Measure	Refacto AF FuseNGo n = 84
Value	13.55 (4.275)

Refacto AF FuseNGo: Median Difference (Final Values): -3.789, p = 0.000, Wilcoxon Signed Ranks Test

Population analysed: Per-protocol population included all participants who received at least 1 treatment using the Refacto AF FuseNGo and completed the two final questionnaires.

Primary outcome: Impact of Clotting Factor Treatment Score

Time frame: Final Visit (Month 8). Mean (Standard Deviation). Unit: units on scale

Measure	Refacto AF FuseNGo n = 84
Value	20.15 (6.167)

Refacto AF FuseNGo: Median Difference (Final Values): -3.980, p = 0.000, Wilcoxon Signed Ranks Test

Population analysed: Per-protocol population included all participants who received at least 1 treatment using the Refacto AF FuseNGo and completed the two final questionnaires.

1 secondary outcome is reported in the full results.

Adverse events

Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Refacto AF FuseNGo	0 of 86 (0%)	3 of 86 (3.5%)	Not reported

Limitations and caveats (from the sponsor): The outcome measure 'ease of storing clotting factor treatment score' was not analyzed as per change in planned analysis.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 25 Sep 2018.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
To obtain contact information for a study center near you, click here.Link provided by the sponsor.

Related trials for Hemophilia A

RecruitingObservational study
The CACHE Study: Coronary Artery Care in HaEmophilia
Haemophilia, Cardiovascular Diseases
Active, not recruitingObservational study
A Study to Learn About the Study Medicine -Hympavzi in Congenital Hemophilia Patients Without Inhibitors in Japan.
Hemophilia A or B
RecruitingPhase 1/2
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A
Hemophilia A
Active, not recruitingPhase 3
A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B
Hemophilia B
Not yet recruiting
Safety of Intraosseous Anaesthesia in Children With Haemophilia
Haemophilia A, Haemophilia B

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Pfizer CT.gov Call Center study director

Pfizer

19 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

An observational study of Hemophilia A, run by Pfizer at 19 sites in Italy.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 2 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 25 Sep 2018. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 19 sites in Italy. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

Yes. Results are posted in the registry (first posted 25 Sep 2018). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Ease of Using Clotting Factor Treatment Score (time frame: Final Visit (Month 8)). The study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01959919.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Sep 2018.