Study of Safety And Efficacy Of ReFacto AF In Previously Untreated Hemophilia A Patients In The Usual Care Setting
10. maj 2019 opdateret af: Pfizer
AN OPEN-LABEL STUDY OF THE SAFETY AND EFFICACY OF REFACTO AF IN PREVIOUSLY UNTREATED PATIENTS IN USUAL CARE SETTINGS
Study to evaluate the safety and effectiveness of ReFacto AF for the treatment of severe hemophilia A in patients who have not yet received treatment for their hemophilia.
Study subjects will be males less than 6 years old who have not taken any clotting factor or other blood products before the study.
The safety and effectiveness of ReFacto AF will be determined in this study by tests and procedures done at the doctor's office.
Studieoversigt
Detaljeret beskrivelse
Regulatory Commitment
Undersøgelsestype
Interventionel
Tilmelding (Faktiske)
23
Fase
- Fase 4
Kontakter og lokationer
Dette afsnit indeholder kontaktoplysninger for dem, der udfører undersøgelsen, og oplysninger om, hvor denne undersøgelse udføres.
Studiesteder
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Nantes, Frankrig, 44093
- CHU de Nantes
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Groningen, Holland, 9713 GZ
- University Medical Center Groningen
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Firenze, Italien, 50134
- Agenzia per L'emofilia e Centro Regionale Riferimento Coagulopatie Congenite
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Adana, Kalkun, 01330
- Cukurova Universitesi Tip Fakultesi
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Istanbul, Kalkun, 34098
- Istanbul Universitesi Cerrahpasa Tip Fakultesi
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Izmir
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Bornova, Izmir, Kalkun, 35100
- Ege Üniversitesi Tip Fakültesi
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Madrid, Spanien, 28046
- Hospital Universitario La Paz
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Valencia, Spanien, 46009
- Hospital Universitario La Fe
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Bremen, Tyskland, 28177
- Klinikum Bremen-Mitte gGmbH, Professor Hess Kinderklinik
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Niedersachsen
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Hannover, Niedersachsen, Tyskland, 30625
- Medizinische Hochschule, Hannover
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Lviv, Ukraine, 79044
- Derzhavna ustanova "Instytut patolohii krovi ta transfuziinoi medytsyny Natsionalnoi akademii
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Deltagelseskriterier
Forskere leder efter personer, der passer til en bestemt beskrivelse, kaldet berettigelseskriterier. Nogle eksempler på disse kriterier er en persons generelle helbredstilstand eller tidligere behandlinger.
Berettigelseskriterier
Aldre berettiget til at studere
Ikke ældre end 6 år (Barn)
Tager imod sunde frivillige
Ingen
Køn, der er berettiget til at studere
Han
Beskrivelse
Inclusion Criteria:
- Male subjects <6 years of age with severe hemophilia A (FVIII:C <1%) based on clinical records, including newborns.
- No prior exposure to factor products or any blood products.
Exclusion Criteria:
- Presence of any bleeding disorder in addition to hemophilia A.
- Treatment with any investigational agent or device within the past 30 days.
- Any condition(s) that compromises the ability to collect study-related observations, or that poses a contraindication to study participation (these conditions include, but are not limited to, inadequate medical history to assure study eligibility; and expectation of poor adherence to study requirements).
Studieplan
Dette afsnit indeholder detaljer om studieplanen, herunder hvordan undersøgelsen er designet, og hvad undersøgelsen måler.
Hvordan er undersøgelsen tilrettelagt?
Design detaljer
- Primært formål: Andet
- Tildeling: N/A
- Interventionel model: Enkelt gruppeopgave
- Maskning: Ingen (Åben etiket)
Våben og indgreb
Deltagergruppe / Arm |
Intervention / Behandling |
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Eksperimentel: 1
The investigator treats subjects with ReFacto AF in the usual care setting.
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Blood draws to determine the level of factor VIII activity before and after dosing at appropriate visits, and blood draws to determine levels of factor VIII inhibitor (antibody to factor VIII).
Andre navne:
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Hvad måler undersøgelsen?
Primære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
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Percentage of Participants Who Developed Clinically Significant Factor VIII (FVIII) Inhibitors During the Course of the Study
Tidsramme: 2 years
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Percentage of participants who developed clinically significant FVIII inhibitors: those persistent over a defined period with clinically impactful effects like breakthrough bleed, low recovery, etc., during the course of the study.
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2 years
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Sekundære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
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Annualized Bleeding Rate (ABR)
Tidsramme: 2 years
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Annualized bleeding rate was calculated as the number of bleeds divided by the treatment interval duration (enrollment visit to final visit) and then multiplied by 365.25.
If there was more than 1 bleed location (like ankle and joint) with identical bleed start date and time, it was treated as 1 bleed occurrence.
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2 years
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Total Number of Infusions to Treat a New Bleed Classified on Basis of Response to First On-Demand Treatment With Refacto AF
Tidsramme: 2 years
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Number of infusions of Refacto AF required to treat a new bleed were classified on basis of the response to at 4-point response scale of assessment (excellent, good, moderate and no response).
Assessment was completed each time a participant experienced a new bleed requiring an 'on-demand' IV infusion.
Excellent: definite pain relief and/or improvement in bleeding signs within 8 hours (hr) after infusion, no additional infusion administered; Good: definite pain relief and/or improvement in bleeding signs within 8 hr after infusion, at least 1 additional infusion administered for complete resolution or with no additional infusion administered; Moderate: probable or slight improvement starting after 8 hr following infusion, at least 1 additional infusion administered for complete resolution; No Response: no improvement at all between infusions or during 24 hr interval following infusion or condition worsen.
Bleeds for which response not recorded, reported as: Data Not Recorded.
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2 years
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Total Number of Infusions Needed for Resolution of Bleeding Episodes Classified on Basis of Response to Study Drug Infusion
Tidsramme: Within 48 hours after infusion, up to 2 years treatment duration
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Number of infusions of Refacto AF required for resolution of a bleeding episodes were classified on basis of the response at 4-point response scale of assessment (excellent, good, moderate and no response).
Excellent: definite pain relief and/or improvement in bleeding signs within 8 hours (hr) after infusion, no additional infusion administered; Good: definite pain relief and/or improvement in bleeding signs within 8 hr after infusion, at least 1 additional infusion administered for complete resolution or with no additional infusion administered; Moderate: probable or slight improvement starting after 8 hr following infusion, at least 1 additional infusion administered for complete resolution; No Response: no improvement at all between infusions or during 24 hr interval following infusion or condition worsen.
Bleeds for which response not recorded, reported as: Data Not Recorded.
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Within 48 hours after infusion, up to 2 years treatment duration
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Total Number of Breakthrough Bleeding Episodes Occurring Within 48 Hours After a Prophylaxis Infusion of ReFacto AF
Tidsramme: 2 years
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The number of breakthrough bleeds (spontaneous or traumatic) within 48 hours following a prophylaxis dose of ReFacto AF are summarized.
If there was more than 1 bleed location (like ankle and joint) with identical bleed start date and time, it was treated as 1 bleed occurrence.
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2 years
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Consumption of Total International Units of Factor VIII
Tidsramme: 2 years
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2 years
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Consumption of Total International Units of Factor VIII Per Year
Tidsramme: 2 years
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Consumption of total international units of Factor VIII per year was calculated for a participant: dividing the total consumption of factor VIII by participant's treatment interval duration (in days), then multiplying by 365.25.
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2 years
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Mean Dose (IU) of Study Drug Consumed Per Infusion
Tidsramme: 2 years
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Mean dose for each participant was calculated as participant's total factor VIII consumption (in IU) divided by the number of infusions administered.
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2 years
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Consumption of Total International Units of Factor VIII by Weight
Tidsramme: 2 years
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Consumption of total international units of Factor VIII by weight was calculated for a participant: dividing the total consumption of factor VIII by participant's weight (the most recently recorded).
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2 years
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Consumption of Total International Units of Factor VIII Per Year by Weight
Tidsramme: 2 years
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Consumption of total international units of Factor VIII per year by weight was calculated for a participant: the total consumption of factor VIII divided by participant's treatment interval duration (in days), then multiplying by 365.25 and then dividing by participant's weight (the most recently recorded).
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2 years
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Mean Dose (IU) of Study Drug Consumed Per Infusion by Weight
Tidsramme: 2 years
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Mean dose for each participant was calculated as participant's total factor consumption (in IU) divided by the number of infusions administered and then dividing by participant's weight (the most recently recorded).
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2 years
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Mean of Total Number of Infusions of Study Drug Received
Tidsramme: 2 years
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2 years
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Mean of Total Number of Days Participants Exposed to Study Drug
Tidsramme: 2 years
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2 years
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Number of Participants Who Required Dose Escalation of Their Prescribed Prophylaxis Regimen During Their Participation in This Study
Tidsramme: 2 years
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The number of participants who met the dose escalation criteria were prescribed a higher dose and/or were prescribed more frequent doses.
When dose escalation was required, the specific dose and dosing schedule was at the investigator's discretion.
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2 years
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Percentage of Bleeding Episodes With Less-Than-Expected Therapeutic Effect (LETE) in On-Demand (OD) Setting
Tidsramme: 2 years
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LETE occurs in OD setting if participant recorded 2 successive "No Response" (no improvement at all between infusions, or condition worsens) ratings after 2 successive infusions of study drug.
Infusions must have been given within 24 hours (hr) of each other for treatment of same bleeding event in absence of confounding factors (known presence or subsequent identification of a FVIII inhibitor, known inadequate dose for type and/or severity of bleed in opinion of investigator, delay of greater than (>) 4 hr between onset of bleed to infusion, delay of >24 hr before administration of a follow-up infusion, known compromised study drug, faulty administration of study drug, participant had an underlying, predisposing condition responsible for bleed in opinion of investigator, ongoing trauma responsible for continued bleeding.
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2 years
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Percentage of Bleeding Episodes With Less-Than-Expected Therapeutic Effect (LETE) in the Prophylaxis Setting
Tidsramme: 2 years
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LETE in prophylaxis setting if there was a spontaneous bleed within 48 hours after a regularly scheduled prophylactic dose of study drug (which was not used to treat a bleed) in the absence of confounding factors (known presence or subsequent identification of a FVIII inhibitor, known inadequate prophylactic dose [a dose less than that prescribed in participant's regimen], known lack of adherence to the prescribed prophylaxis regimen, known compromised study drug, faulty administration of study drug, participant had an underlying, predisposing condition responsible for the bleed in the opinion of the investigator, traumatic injury responsible for bleeding.
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2 years
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Total Number of Events of Potential Less-Than-Expected Therapeutic Effect (LETE) in the Low Recovery Setting
Tidsramme: 2 years
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LETE was lower than expected recovery of FVIII in the opinion of the investigator following infusion of study drug in the absence of confounding factors (known presence or subsequent identification of a FVIII inhibitor, known compromised study drug, faulty administration of study drug including inadequate dosing).
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2 years
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Samarbejdspartnere og efterforskere
Det er her, du vil finde personer og organisationer, der er involveret i denne undersøgelse.
Sponsor
Publikationer og nyttige links
Den person, der er ansvarlig for at indtaste oplysninger om undersøgelsen, leverer frivilligt disse publikationer. Disse kan handle om alt relateret til undersøgelsen.
Datoer for undersøgelser
Disse datoer sporer fremskridtene for indsendelser af undersøgelsesrekord og resumeresultater til ClinicalTrials.gov. Studieregistreringer og rapporterede resultater gennemgås af National Library of Medicine (NLM) for at sikre, at de opfylder specifikke kvalitetskontrolstandarder, før de offentliggøres på den offentlige hjemmeside.
Studer store datoer
Studiestart (Faktiske)
10. februar 2010
Primær færdiggørelse (Faktiske)
24. november 2016
Studieafslutning (Faktiske)
24. november 2016
Datoer for studieregistrering
Først indsendt
29. juli 2009
Først indsendt, der opfyldte QC-kriterier
30. juli 2009
Først opslået (Skøn)
31. juli 2009
Opdateringer af undersøgelsesjournaler
Sidste opdatering sendt (Faktiske)
19. juli 2019
Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier
10. maj 2019
Sidst verificeret
1. maj 2019
Mere information
Begreber relateret til denne undersøgelse
Nøgleord
Yderligere relevante MeSH-vilkår
Andre undersøgelses-id-numre
- 3082B2-4434
- B1831006 (Anden identifikator: Alias Study Number)
- 2008-008436-93 (EudraCT nummer)
Plan for individuelle deltagerdata (IPD)
Planlægger du at dele individuelle deltagerdata (IPD)?
JA
IPD-planbeskrivelse
Pfizer will provide access to individual de-identified participant data and related study documents (e.g.
protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions.
Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.
Disse oplysninger blev hentet direkte fra webstedet clinicaltrials.gov uden ændringer. Hvis du har nogen anmodninger om at ændre, fjerne eller opdatere dine undersøgelsesoplysninger, bedes du kontakte register@clinicaltrials.gov. Så snart en ændring er implementeret på clinicaltrials.gov, vil denne også blive opdateret automatisk på vores hjemmeside .