Trial record 1 of 2 for:
LFB USA
A Phase III Study on the Safety, Pharmacokinetics and Efficacy of Coagulation Factor VIIa (PERSEPT2)
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| ClinicalTrials.gov Identifier: NCT02448680 |
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Recruitment Status :
Recruiting
First Posted : May 19, 2015
Last Update Posted : January 25, 2017
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Sponsor:
LFB USA, Inc.
Collaborator:
Laboratoire français de Fractionnement et de Biotechnologies
Information provided by (Responsible Party):
LFB USA, Inc.
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Brief Summary:
The purpose of the study is to assess the safety, efficacy and pharmacokinetics of 2 separate dose regimens (75µg/kg and 225 µg/kg) of Coagulation Factor VIIa (Recombinant) for the treatment of bleeding episodes in hemophilia A or B patients with inhibitors to Factor VIII or IX in 12 patients ( birth to <6 years old), and 12 patients (≥6 years old to <12 years old).
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Hemophilia A With Inhibitors Hemophilia B With Inhibitors | Biological: Coagulation FVIIa (Recombinant) | Phase 3 |
A Phase III Study on the Safety, Pharmacokinetics, and Efficacy of Coagulation Factor VIIa (Recombinant) in Congenital Hemophilia A or B Pediatric Patients from birth to <12 years old with Inhibitors to Factor VIII or IX: PerSept 2
| Study Type : | Interventional (Clinical Trial) |
| Estimated Enrollment : | 24 participants |
| Allocation: | Randomized |
| Intervention Model: | Crossover Assignment |
| Masking: | None (Open Label) |
| Primary Purpose: | Treatment |
| Official Title: | A Phase III Study on the Safety, Pharmacokinetics, and Efficacy of Coagulation Factor VIIa (Recombinant) in Congenital Hemophilia A or B Pediatric Patients From Birth to <12 Years Old With Inhibitors to Factor VIII or IX: PerSept 2 |
| Study Start Date : | December 2015 |
| Estimated Primary Completion Date : | June 30, 2017 |
| Estimated Study Completion Date : | August 30, 2017 |
Resource links provided by the National Library of Medicine
Genetics Home Reference related topics:
Hemophilia
Drug Information available for:
Recombinant FVIIa
| Arm | Intervention/treatment |
|---|---|
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Active Comparator: Coagulation Factor VIIa (Recombinant): 75 µg/kg
75 µg/kg treatment regimen for 3 months
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Biological: Coagulation FVIIa (Recombinant)
A cross over design to assess the efficacy of 2 separate dose regimens (75 µg/kg and 225 µg/kg) of Coagulation Factor VIIa (Recombinant) for the treatment of bleeding episodes in hemophilia A or B patients with inhibitors to Factor VIII or Factor IX |
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Active Comparator: Coagulation Factor VIIa (Recombinant): 225 µg/kg
225 µg/kg treatment regimen for 3 months
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Biological: Coagulation FVIIa (Recombinant)
A cross over design to assess the efficacy of 2 separate dose regimens (75 µg/kg and 225 µg/kg) of Coagulation Factor VIIa (Recombinant) for the treatment of bleeding episodes in hemophilia A or B patients with inhibitors to Factor VIII or Factor IX |
Primary Outcome Measures :
- Bleeding episode treatment success [ Time Frame: 12 hours after first administration of study drug ]No additional hemostatic product required after 12 hours of first dose
Secondary Outcome Measures :
- Time to bleeding success [ Time Frame: 12 hours ]Patients shall rate the treatment of each bleeding episode. If treatment occurs under direct supervision of treating physician, the physician shall rate the response. Ratings based on a four point scale; Excellent, Good, Moderate, None
Other Outcome Measures:
- Immunogenicity assessment [ Time Frame: Pre-dose, 3 weeks post dose and then every 6 weeks until end of study ]Based on Coagulation Factor VIIa (Recombinant), binding antibody levels
- Pharmacokinetic profile assessment [ Time Frame: Half of the patients sampling: at 10±2 minutes, 1 and 4 hours (±10 minutes), and the other half of the patients sampling: at 30±5 minutes and 2 and 8 hours (±10 minutes) relative to the start of infusion of study drug ]Based on plasma concentrations of Coagulation Factor VIIa (Recombinant)
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| Ages Eligible for Study: | up to 11 Years (Child) |
| Sexes Eligible for Study: | Male |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- be male with a diagnosis of congenital hemophilia A or B of any severity
- have one of the following:
- a positive inhibitor test BU ≥5, OR
- a Bethesda Unit (BU) <5 but expected to have a high anamnestic response to FVIII or FIX, as demonstrated from the patient's medical history, precluding the use of factor VIII or IX products to treat bleeding episodes, OR
- a BU <5 but expected to be refractory to increased dosing of FVIII or FIX, as demonstrated from the patient's medical history, precluding the use of factor VIII or IX products to treat bleeding episodes
- be aged from birth to <12 years old
- have experienced at least 3 bleeding episodes of any severity in the past 6 months
- parents or legal guardians must be capable of understanding and be willing to comply with the conditions of the protocol
- parents or legal guardians must have read, understood, and provided written informed consent
Exclusion Criteria:
- have any coagulation disorder other than hemophilia A or B
- be immunosuppressed (i.e., the patient may not be receiving systemic immunosuppressive medication; cluster of differentiation 4 (CD4) counts at screening must be >200/µL)
- have a known allergy or hypersensitivity to rabbits
- have platelet count <100,000/mL
- have had a major surgical procedure (e.g. orthopedic, abdominal) within 1 month prior to first administration of study drug
- have received an investigational drug within 30 days of first study drug administration, or be expected to receive such drug during participation in this 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): NCT02448680
Contacts
| Contact: Meg Carini, LPN,BS | 508-370-5258 | meg.carini@lfb-usa.com | |
| Contact: Jeffry Lawrence, MD | 508-370-5113 | jeff.lawrence@lfb-usa.com |
Locations
| United States, Colorado | |
| University of Colorado Denver Hemophilia & Thrombosis Center | Recruiting |
| Aurora, Colorado, United States, 80045 | |
| Principal Investigator: Michael Wang, MD | |
| United States, Oklahoma | |
| Jimmy Everest Center for Cancer and Bleeding Disorders | Active, not recruiting |
| Oklahoma City, Oklahoma, United States, 73117 | |
| United States, Texas | |
| UT Southwestern Medical Center at Dallas / Children's Medical Center | Recruiting |
| Dallas, Texas, United States, 75390 | |
| Contact: Anna Winborn | |
| Principal Investigator: Janna Journeycake | |
| Bulgaria | |
| University Multiprofile Hospital for Active Treatment "Sveti Georgi" | Active, not recruiting |
| Plovdiv, Bulgaria | |
| Czech Republic | |
| University Hospital Motol | Recruiting |
| Prague, Czech Republic | |
| Contact: Veronika Cepelakova +420 (2) 2443 6537 veronika.cepelakova@fnmotol.cz | |
| Principal Investigator: Vladimir Komrska, MD | |
| Georgia | |
| Hematology of Department Hemophilia and Thromboses center | Recruiting |
| Tbilisi, Georgia | |
| Contact: Tamila Katselashvili | |
| Principal Investigator: Tamila Nadiradze | |
| South Africa | |
| Worthwhile Clinical Trials | Recruiting |
| Benoni, South Africa | |
| Contact: Fadeela Bibi Rahman fadeela@wwct.co.za | |
| Principal Investigator: Ismail Haroon Mitha | |
| Ukraine | |
| National Specialized Children's Hospital OKHMATDYT, Centre for Hemostatic Pathology (Ukraine) | Recruiting |
| Kyiv, Ukraine | |
| Contact: Kateryna Vilchevska vilchevskaya@yandex.ru | |
| Principal Investigator: Kateryna Vilchevska | |
| Institute of Blood Pathology and Transfusion Medicine | Recruiting |
| Lviv, Ukraine | |
| Contact: Oleksandra Stasyshyn | |
| Principal Investigator: Oleksandra Stasyshyn | |
Sponsors and Collaborators
LFB USA, Inc.
Laboratoire français de Fractionnement et de Biotechnologies
Investigators
| Principal Investigator: | Michael Wang, MD | University of Colorado, Denver |
| Responsible Party: | LFB USA, Inc. |
| ClinicalTrials.gov Identifier: | NCT02448680 History of Changes |
| Other Study ID Numbers: |
LFB-FVIIa-007-14 |
| First Posted: | May 19, 2015 Key Record Dates |
| Last Update Posted: | January 25, 2017 |
| Last Verified: | January 2017 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | Undecided |
Additional relevant MeSH terms:
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Hemophilia A Hemophilia B Blood Coagulation Disorders, Inherited Blood Coagulation Disorders Hematologic Diseases Coagulation Protein Disorders |
Hemorrhagic Disorders Genetic Diseases, Inborn Genetic Diseases, X-Linked Factor VIII Coagulants |