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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Arm 1: Device |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Refacto FusENGO | Device | Dosage will be chosen by PI based on patient condition. |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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) |
| 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) |
| 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) |
| 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. |
| Measure | Description | Time Frame |
|---|---|---|
| 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. |
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Inclusion Criteria:
Exclusion Criteria:
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Approximately 200 patients with Hemophilia A (all severity levels) will be enrolled in approximately 20 centers in Italy.
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| Name | Affiliation | Role |
|---|---|---|
| Pfizer CT.gov Call Center | Pfizer | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Istituto di Ematologia | Rome | Lazio | 00161 | Italy | ||
| Ospedale SS. Annunziata - USL 1 |
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| Label | URL |
|---|---|
| To obtain contact information for a study center near you, click here. | View source |
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| ID | Title | Description |
|---|---|---|
| FG000 | 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 summary of product characteristics (SmPC). |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
Safety population included all the participants who were enrolled and received at least 1 dose of Refacto AF FuseNGo.
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| ID | Title | Description |
|---|---|---|
| BG000 | 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. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 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. | Per-protocol population included all participants who received at least 1 treatment using the Refacto AF FuseNGo and completed the two final questionnaires. | Posted | Mean | Standard Deviation | units on a scale | Final Visit (Month 8) |
|
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Safety population included all the participants who were enrolled and received at least 1 dose of Refacto AF FuseNGo.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | 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. |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Gastroentheritis | Infections and infestations | ICD9-CM 0.0 | Non-systematic Assessment |
The outcome measure 'ease of storing clotting factor treatment score' was not analyzed as per change in planned analysis.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Pfizer, Inc. | Pfizer Clinical Trial.gov Call Center | 1-800-718-1021 | ClinicalTrails.gov_Inquiries@Pfizer.com |
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| ID | Term |
|---|---|
| D006467 | Hemophilia A |
| ID | Term |
|---|---|
| D025861 | Blood Coagulation Disorders, Inherited |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
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| 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. | 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. | Final Visit (Month 8) |
| Baseline up to 28 days after last dose of drug (up to Month 12) |
| Sassari |
| Sardinia |
| 07100 |
| Italy |
| Azienda Ospedale Policlinico Consorziale | Bari | 70121 | Italy |
| Struttura Complessa Di Oncoematologia Pediatrica E Patologia Della Coagulzione | Cagliari | 09100 | Italy |
| Ospedale Ferrarrotto | Catania | 95124 | Italy |
| Azienda Ospedaliera Ciaccio - Ospedale Pugliese | Catanzaro | 88100 | Italy |
| Azienda Ospedaliera Careggi | Florence | 50134 | Italy |
| Ospedale Maggiore Policlinico Mangiagalli E Regina Elena | Milan | Italy |
| Dipartimento Medicina Clinica e Sperimentale | Naples | 80131 | Italy |
| Centro Emofilia e Trombosi - Centro delle Microcitemie | Naples | 81044 | Italy |
| Azienda Ospedaliera di Padova | Padova | 35128 | Italy |
| Policlinico P Giaccone | Palermo | 90100 | Italy |
| Centro regionale di diagnosi e trattamento dell'Emofilia e delle malattie dell'emostasi e trombosi | Province of Macerata | 62100 | Italy |
| Arcispedale S. Maria Nuova, AO di Reggio Emilia | Reggio Emilia | 42100 | Italy |
| Policlinico Agostino Gemelli | Rome | Italy |
| Ospedale Molinette | Torino | 10126 | Italy |
| SC Servizio Immunotrasfusionale | Torino | Italy |
| SOS Malattie Emorragiche e Trombotiche AOU "S.M.M." Udine | Udine | 33100 | Italy |
| Anna Chiara Giuffrida | Verona | 37126 | Italy |
| Years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Time for Reconstructing the Drug | In this parameter time consumed for performing steps to reconstitute the drug prior to infusion of drug is reported. | Mean | Standard Deviation | minutes |
|
| Burden of Clotting Factor Treatment Score | HaemoPREF is a participant rated 14-item instrument to measure experience of clotting factor treatment 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. | Mean | Standard Deviation | units on scale |
|
| Impact of Clotting Factor Treatment Score | HaemoPREF is a 14-item instrument to measure experience of clotting factor treatment. For 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 3 items was scored on a scale of 0(most challenging) to 10(least challenging), and summed up to give total overall score range of 0(greatest negative impact) to 30(least negative impact). Higher scores indicate less negative impact on daily life. | Mean | Standard Deviation | units on scale |
|
| Risk Associated With Clotting Factor Treatment Score | HaemoPREF is 14-item instrument to measure experience of clotting factor treatment. For score for risk associated with clotting factor treatment,3 items were assessed by participants: 1)worried about getting infected with other disease while on treatment,2)worried to infect treatment while preparing for injection and 3)worried to inject treatment by own. Each of 3 items was scored on a scale of 0(most concerned) to 10(not at all concerned),summed up to give total overall score range of 0(most worried) to 30(least worried). Higher scores indicate lower levels of worry associated with treatment. | Mean | Standard Deviation | units on scale |
|
| 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. |
|
|
|
| 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. | 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. | Posted | Mean | Standard Deviation | minutes | 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. | Per-protocol population included all participants who received at least 1 treatment using the Refacto AF FuseNGo and completed the two final questionnaires. | Posted | Mean | Standard Deviation | units on scale | 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. | Per-protocol population included all participants who received at least 1 treatment using the Refacto AF FuseNGo and completed the two final questionnaires. | Posted | Mean | Standard Deviation | units on scale | 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. | Per-protocol population included all participants who received at least 1 treatment using the Refacto AF FuseNGo and completed the two final questionnaires. | Posted | Mean | Standard Deviation | units on scale | 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. | 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. | Posted | Mean | Standard Deviation | units on a scale | 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. | Safety population included all the participants who were enrolled and received at least 1 dose of Refacto AF FuseNGO. | Posted | Number | participants | Baseline up to 28 days after last dose of drug (up to Month 12) |
|
|
|
| 0 |
| 86 |
| 3 |
| 86 |
| Anemia | Blood and lymphatic system disorders | ICD9-CM 0.0 | Non-systematic Assessment |
|
| Cough | Respiratory, thoracic and mediastinal disorders | ICD9-CM 0.0 | Non-systematic Assessment |
|
| High fever (Bronchitis) | Infections and infestations | ICD9-CM 0.0 | Non-systematic Assessment |
|
| Esophageal varices | Gastrointestinal disorders | ICD9-CM 0.0 | Non-systematic Assessment |
|
| Hyperuricemia | Metabolism and nutrition disorders | ICD9-CM 0.0 | Non-systematic Assessment |
|
| Insomnia and nightmares | Psychiatric disorders | ICD9-CM 0.0 | Non-systematic Assessment |
|
| Flu | Infections and infestations | ICD9-CM 0.0 | Non-systematic Assessment |
|
| Pulpitis | Infections and infestations | ICD9-CM 0.0 | Non-systematic Assessment |
|
| Anal bleeding from hemorrhoids | Gastrointestinal disorders | ICD9-CM 0.0 | Non-systematic Assessment |
|
Pfizer has the right to review disclosures, requesting a delay of less than 60 days. Investigator will postpone single center publications until after disclosure of pooled data (all sites), less than 12 months from study completion/ termination at all participating sites. Investigator may not disclose previously undisclosed confidential information other than study results.
| D020147 | Coagulation Protein Disorders |
| D006474 | Hemorrhagic Disorders |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |