Clinical trial NCT04085458: Study to Gain More Information on How Safe and Effective Jivi Works in Patients With Severe Hemophilia A (Post-marketing Investigation)
Official title: Post-marketing Investigation (PMI) to Assess Safety and Efficacy of Jivi (BAY 94-9027) Treatment in Participants With Hemophilia A
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Hemophilia A
Lead sponsor: Bayer
Sponsor class: INDUSTRY
First posted: 2019-09-11
Enrollment: 32
Sex: MALE
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The goal of this study is to give gather more information on how safe and well Jivi works in patients with severe hemophilia A. Jivi has been approved by various regulatory agencies, including the FDA, Health Canada, Japanese Health Authority and the European Medicinal Agency. 25 patients will be enrolled and will stay for 1 to 2 years in this study depending on their treatment frequency. Researcher will monitor during the course of the study whether patients are developing antibodies (a protein made by the body in response to the drug) affecting the effectiveness of Jivi. In addition information on bleedings and patient's wellbeing will be collected.


Eligibility criteria: Inclusion Criteria:

* Participants must ≥ 18 years of age inclusive, at the time of signing the informed consent.
* Participants with severe hemophilia A (FVIII: C\<1%)
* PTPs (Previously treated patients) (≥150 ED (Exposure day)) on prophylaxis treatment before enrollment
* Participants who are immunocompetent. If human immunodeficiency virus (HIV) positive, cluster of differentiation 4 (CD4)+ lymphocyte count \>200/mm\*3
* Participants who are willing to complete an eDiary
* Male participants
* Capable of giving signed informed consent

Exclusion Criteria:

* Any other inherited or acquired bleeding disorder in addition to Hemophilia A.
* Platelet count \< 100,000/mm\*3
* Creatinine \> 2x upper limit of normal
* AST or ALT \> 5x upper limit of normal (AST: aspartate aminotransferase; ALT: alanine aminotransferase)
* The participant has a planned major surgery.
* The participant is currently participating in another investigational drug study, or has participated in a clinical study involving an investigational drug within 30 days of signing informed consent or previous treatment in a clinical phase III study with BAY 94-9027 (now marketed as Jivi).
* Current evidence (by central laboratory) or history of inhibitor to FVIII with a titer ≥ 0.6 Bethesda unit (BU).
* Known hypersensitivity to the drug substance, excipients, or mouse or hamster protein.

Primary outcomes:
measure: FVIII Inhibitor Development by the Nijmegen Bethesda Assay
timeFrame: Observed for 100 exposure days (EDs), up to 2 years
Secondary outcomes:
measure: Number of Participants With Treatment-emergent Adverse Events (TEAEs)
timeFrame: Observed for 100 exposure days (EDs), up to 2 years
measure: Development of Treatment-emergent Anti-PEG Antibodies
timeFrame: Observed for 100 exposure days (EDs), up to 2 years
measure: Annualized Bleeding Rate (ABR)
timeFrame: Observed for 100 exposure days (EDs), up to 2 years
Other outcomes:
Locations:
city: Sofia
country: Bulgaria
facility: UMHAT Tsaritsa Joanna-ISUL EAD Sofia
city: Varna
country: Bulgaria
facility: MHAT Sveta Marina EAD
city: Arhus N
country: Denmark
facility: Aarhus Universitetshospital, Skejby
city: Athens
country: Greece
facility: LAIKO General Hospital of Athens
city: Catanzaro
country: Italy
facility: A.O. Pugliese-Ciaccio
state: Calabria
city: Roma
country: Italy
facility: A.O.U. Policlinico Umberto I
state: Lazio
city: Roma
country: Italy
facility: Fondazione Policlinico Universitario Agostino Gemelli IRCCS
state: Lazio
city: Oslo
country: Norway
facility: Oslo Universitetssykehus HF, Rikshospitalet
city: Gdansk
country: Poland
facility: Uniwersyteckie Centrum Kliniczne
city: Wroclaw
country: Poland
facility: SP Szpital Kliniczny Nr 1
city: Barcelona
country: Spain
facility: Ciutat Sanitaria i Universitaria de la Vall d'Hebron
city: Madrid
country: Spain
facility: Hospital Universitario "La Paz"
city: Valencia
country: Spain
facility: Hospital Universitari i Politecnic La Fe | Hematologia
Study officials:
References: