- ICH GCP
- US-Register für klinische Studien
- Klinische Studie NCT02954575
Clinical Study to Investigate the PK, Efficacy, and Safety of Wilate in Patients With Severe Hemophilia A
21. Dezember 2020 aktualisiert von: Octapharma
Clinical Study to Investigate the Pharmacokinetics, Efficacy, Safety, and Immunogenicity of Wilate in Previously Treated Patients With Severe Hemophilia A
The purpose of this study is to obtain additional data on the safety and efficacy of Wilate in PTPs with hemophilia A with at least 150 previous exposure days (EDs) to a FVIII concentrate who undergo prophylactic treatment with Wilate for 6 months and at least 50 EDs, thus supplementing the existing database to obtain approval of Wilate for the indication hemophilia A in the USA.
Studienübersicht
Status
Abgeschlossen
Bedingungen
Intervention / Behandlung
Studientyp
Interventionell
Einschreibung (Tatsächlich)
57
Phase
- Phase 3
Kontakte und Standorte
Dieser Abschnitt enthält die Kontaktdaten derjenigen, die die Studie durchführen, und Informationen darüber, wo diese Studie durchgeführt wird.
Studienorte
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Sofia, Bulgarien
- Specialized Hospital for Active Treatment "Joan Pavel"
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Krakow, Polen
- Krakowskie Centrum Medyczne
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Rzeszow, Polen
- Korczowski Bartosz Gabinet Lekarski
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Bucharest, Rumänien
- Centrul Medical Unirea -Policlinica Enescu
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Barnaul, Russische Föderation
- Barnaul Branch of RAMS hematology center
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Moscow, Russische Föderation
- Federal Scientific Hematology Center
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Budapest, Ungarn
- National Haemophilia Centre
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Teilnahmekriterien
Forscher suchen nach Personen, die einer bestimmten Beschreibung entsprechen, die als Auswahlkriterien bezeichnet werden. Einige Beispiele für diese Kriterien sind der allgemeine Gesundheitszustand einer Person oder frühere Behandlungen.
Zulassungskriterien
Studienberechtigtes Alter
12 Jahre und älter (Kind, Erwachsene, Älterer Erwachsener)
Akzeptiert gesunde Freiwillige
Nein
Studienberechtigte Geschlechter
Männlich
Beschreibung
Inclusion Criteria:
- Severe hemophilia A (<1% FVIII:C) according to medical history
- Male patients aged ≥12 years
- Previous treatment with a FVIII concentrate for at least 150 exposure days (EDs)
- Immunocompetence (CD4+ count >200/µL)
- Good documentation of the historical bleeding rate (at least for the 6 months preceding study start)
- Voluntarily given, fully informed written and signed consent obtained by the patient (or parent/legal guardian in case of adolescents) before any study-related procedures are conducted
Whenever possible, the interval between the Screening Visit and the PK or Non-PK Visit should not exceed 30 days. If the 30-day interval is exceeded, determination of the CD4+ count is to be repeated and must be >200/µL for patients to be enrolled (i.e., exclusion criterion no. 4).
Exclusion Criteria:
- Any coagulation disorders other than hemophilia A
- History of FVIII inhibitor activity (≥0.6 BU) or detectable FVIII inhibitory anti-bodies (≥0.6 BU using the Nijmegen modification of the Bethesda assay) at screening, as determined by the central laboratory
- Severe liver or kidney diseases (alanine aminotransferase [ALAT] and aspartate transaminase [ASAT] levels >5 times of upper limit of normal, creatinine>120 µmol/L)
- Patients receiving or scheduled to receive immunomodulating drugs (other than anti-retroviral chemotherapy) such as alpha-interferon, prednisone (equivalent to >10 mg/day), or similar drugs
- Treatment with any investigational medicinal product in another interventional clinical study currently or within 4 weeks before enrollment
Studienplan
Dieser Abschnitt enthält Einzelheiten zum Studienplan, einschließlich des Studiendesigns und der Messung der Studieninhalte.
Wie ist die Studie aufgebaut?
Designdetails
- Hauptzweck: Behandlung
- Zuteilung: N / A
- Interventionsmodell: Einzelgruppenzuweisung
- Maskierung: Keine (Offenes Etikett)
Waffen und Interventionen
Teilnehmergruppe / Arm |
Intervention / Behandlung |
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Experimental: All patients
All patients will receive Wilate for prophylactic treatment
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Andere Namen:
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Was misst die Studie?
Primäre Ergebnismessungen
Ergebnis Maßnahme |
Maßnahmenbeschreibung |
Zeitfenster |
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Total Annualized Bleeding Rate (TABR)
Zeitfenster: 6 months
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The total number of bleeding events (BEs) was documented by patients in a patient diary (together with the investigator in case of on-site treatments), which was reviewed at each follow-up visit by site personnel.
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6 months
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Sekundäre Ergebnismessungen
Ergebnis Maßnahme |
Maßnahmenbeschreibung |
Zeitfenster |
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Spontaneous Annualized Bleeding Rate (SABR)
Zeitfenster: 6 months
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The number of spontaneous bleeding events (BEs) was documented by patients in a patient diary (together with the investigator in case of on-site treatments), which was reviewed at each follow-up visit by site personnel.
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6 months
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Efficacy of Wilate in the Treatment of Breakthrough BEs
Zeitfenster: 6 months
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The proportion of BEs successfully treated with Wilate were documented by the patient (together with the investigator in case of on-site treatments) in the patient diary for all BEs according to a 4-point hemostatic efficacy scale including the four items: 'excellent,' 'good,' moderate,' and 'none', where 'excellent' was defined as "Abrupt pain relief and/or unequivocal improvement in objective signs of bleeding within approximately 8 hours after a single injection" (best outcome) and 'none' was defined as "No improvement within 12 hours, or worsening of symptoms, requiring more than two injections for complete resolution" (worst outcome).
All efficacy ratings assessed as either 'excellent' or 'good' were considered 'successfully treated.'
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6 months
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Wilate Consumption Data (Average Total Normdose of FVIII IU/kg Per Month of Study) for Prophylaxis
Zeitfenster: 6 months
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The average consumption of Wilate per month of study (IU/kg) for all patients receiving prophylaxis.
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6 months
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Pharmacokinetic (PK) Assessment (Area Under the Curve [AUC] Norm) of FVIII:C
Zeitfenster: Initial PK visit (Day -1) and PK study completion visit (6 months); data collected 1 h prior to injection and 15 min, 1 h, 3 h, 6 h, 9 h, 24 h, 30 h and 48 h after the end of injection
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PK assessments of FVIII:C were conducted using the one-stage (OS) assay.
The value of the AUCnorm of FVIII:C was calculated based on the FVIII:C values measured in the patients participating in the PK study.
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Initial PK visit (Day -1) and PK study completion visit (6 months); data collected 1 h prior to injection and 15 min, 1 h, 3 h, 6 h, 9 h, 24 h, 30 h and 48 h after the end of injection
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Pharmacokinetic (PK) Assessment (in Vivo Half-Life (t1/2)) of FVIII:C
Zeitfenster: Initial PK assessment (Day -1) and PK study completion visit (6 months); data collected 1 h prior to infusion and 15 min, 1 h, 3 h, 6 h, 9 h, 24 h, 30 h and 48 h after the end of injection
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PK assessments of FVIII:C were conducted using the one-stage (OS) assay.
The in vivo half-life of FVIII:C was calculated based on the FVIII:C values measured in the patients participating in the PK study.
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Initial PK assessment (Day -1) and PK study completion visit (6 months); data collected 1 h prior to infusion and 15 min, 1 h, 3 h, 6 h, 9 h, 24 h, 30 h and 48 h after the end of injection
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Pharmacokinetic (PK) Assessment (Maximum Plasma Concentration [Cmax]) of FVIII:C
Zeitfenster: Initial PK assessment (Day -1) and 6 months
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PK assessments of FVIII:C were conducted using the one-stage (OS) assay.
The maximum plasma concentration of FVIII:C was calculated based on the FVIII:C values measured in the patients participating in the PK study.
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Initial PK assessment (Day -1) and 6 months
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Incremental in Vivo Recovery (IVR) of Wilate Over Time
Zeitfenster: Baseline, 3 and 6 months
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The rise in FVIII activity in IU/dl per unit dose administered in IU/kg was determined from all patients at baseline, 3 and 6 months, using the OS assay.
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Baseline, 3 and 6 months
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Association Between ABO Blood Type and the FVIII:C Half-life of Wilate (OS Assay)
Zeitfenster: 6 months
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Analysis of variance (ANOVA) was used in an exploratory sense to assess an association between ABO blood type and the FVIII:C half-life of Wilate.
This was analyzed by calculating the mean square in a one-stage assay.
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6 months
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Association Between VWF:Ag Concentration and the FVIII:C Half-life of Wilate
Zeitfenster: 6 months
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ANOVA was used in an exploratory sense to assess an association between VWF:Ag with the FVIII:C half-life of Wilate.
This was analyzed by calculating the mean square in a one-stage assay.
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6 months
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Safety and Tolerability of Wilate by Monitoring Adverse Events (AEs) Throughout the Study
Zeitfenster: 6 months
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At each (scheduled or unscheduled) study visit, AEs were documented by the investigator throughout the study.
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6 months
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Immunogenicity of Wilate by Testing for FVIII Inhibitors
Zeitfenster: 6 months
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FVIII inhibitor activity was determined at each study visit before the injection of Wilate using the modified Bethesda assay (Nijmegen modification).
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6 months
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Virus Safety Measured by the Number of Parvovirus B19 Seroconversions Between Baseline (BL) and End of Study
Zeitfenster: 6 months
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Virus safety was evaluated by taking a plasma sample for parvovirus B19 antibody testing before the first injection of Wilate.
All patients negative at screening were tested again at the study completion visit.
The number with Parvovirus B19 seroconversions between BL and end of study was recorded.
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6 months
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Andere Ergebnismessungen
Ergebnis Maßnahme |
Maßnahmenbeschreibung |
Zeitfenster |
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Efficacy of Wilate in Surgical Prophylaxis
Zeitfenster: 6 months
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Hemostatic efficacy was assessed at the end of surgery by the surgeon and at end of the postoperative period by the hematologist, using a 4-point hemostatic efficacy scale including the four items: 'excellent' (best possible outcome), 'good', 'moderate' and 'none' (worst outcome).
Overall efficacy was assessed by the investigator, taking both the intra and postoperative assessments into account, and using the 'excellent,' 'good,' moderate,' and 'none' scale.
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6 months
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Mitarbeiter und Ermittler
Hier finden Sie Personen und Organisationen, die an dieser Studie beteiligt sind.
Sponsor
Studienaufzeichnungsdaten
Diese Daten verfolgen den Fortschritt der Übermittlung von Studienaufzeichnungen und zusammenfassenden Ergebnissen an ClinicalTrials.gov. Studienaufzeichnungen und gemeldete Ergebnisse werden von der National Library of Medicine (NLM) überprüft, um sicherzustellen, dass sie bestimmten Qualitätskontrollstandards entsprechen, bevor sie auf der öffentlichen Website veröffentlicht werden.
Haupttermine studieren
Studienbeginn (Tatsächlich)
1. Dezember 2016
Primärer Abschluss (Tatsächlich)
29. März 2018
Studienabschluss (Tatsächlich)
29. März 2018
Studienanmeldedaten
Zuerst eingereicht
27. Oktober 2016
Zuerst eingereicht, das die QC-Kriterien erfüllt hat
1. November 2016
Zuerst gepostet (Schätzen)
3. November 2016
Studienaufzeichnungsaktualisierungen
Letztes Update gepostet (Tatsächlich)
19. Januar 2021
Letztes eingereichtes Update, das die QC-Kriterien erfüllt
21. Dezember 2020
Zuletzt verifiziert
1. Dezember 2020
Mehr Informationen
Begriffe im Zusammenhang mit dieser Studie
Zusätzliche relevante MeSH-Bedingungen
Andere Studien-ID-Nummern
- WIL-27
Plan für individuelle Teilnehmerdaten (IPD)
Planen Sie, individuelle Teilnehmerdaten (IPD) zu teilen?
NEIN
Arzneimittel- und Geräteinformationen, Studienunterlagen
Studiert ein von der US-amerikanischen FDA reguliertes Arzneimittelprodukt
Ja
Studiert ein von der US-amerikanischen FDA reguliertes Geräteprodukt
Nein
Diese Informationen wurden ohne Änderungen direkt von der Website clinicaltrials.gov abgerufen. Wenn Sie Ihre Studiendaten ändern, entfernen oder aktualisieren möchten, wenden Sie sich bitte an register@clinicaltrials.gov. Sobald eine Änderung auf clinicaltrials.gov implementiert wird, wird diese automatisch auch auf unserer Website aktualisiert .
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