Study to Gain More Information on How Safe and Effective Jivi Works in Patients With Severe Hemophilia A (Post-marketing Investigation)

Phase 4

Completed

• Conditions: Hemophilia A
• Interventions: Drug: Damoctocog alfa pegol (Jivi, BAY94-9027)

• Registration Number: NCT04085458
• Lead Sponsor: Bayer

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2019-09-09
• Study First Submitted QC: 2019-09-09
• Study First Posted: 2019-09-11
• Study Start: 2019-09-23
• Primary Completion: 2022-05-20
• Study Completion: 2022-08-26
• Results First Submitted: 2023-05-07
• Results First Submitted QC: 2023-06-09
• Results First Posted: 2023-06-28
• Status Verified: 2023-07
• Last Update Submitted: 2023-07-26
• Last Update Posted: 2023-07-27

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Male
• Target Recruitment: 32

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Severe hemophilia A patients	Damoctocog alfa pegol (Jivi, BAY94-9027)	Prophylactic treatment regimens should be guided by clinical judgement based on individual patient characteristics and treatment response.

Primary Outcome Measures

Name	Time	Method
FVIII Inhibitor Development by the Nijmegen Bethesda Assay	Observed for 100 exposure days (EDs), up to 2 years	FVIII inhibitor testing was performed using the Nijmegen-modified Bethesda assay. A positive inhibitor result was defined as a threshold of ≥0.6 BU/mL at the central laboratory and had to be confirmed with a second blood sample. After confirmation of the positive result, the inhibitor was to be reported as a serious adverse event (SAE).

Secondary Outcome Measures

Name	Time	Method
Annualized Bleeding Rate (ABR)	Observed for 100 exposure days (EDs), up to 2 years	ABR is number of all bleeds per individual treatment period annualized to a 1-year time interval.
Number of Participants With Treatment-emergent Adverse Events (TEAEs)	Observed for 100 exposure days (EDs), up to 2 years	Treatment-emergent AEs were defined as those that started after the first dose of study drug and up to 7 days after the last dose.
Development of Treatment-emergent Anti-PEG Antibodies	Observed for 100 exposure days (EDs), up to 2 years	Anti-PEG antibody: antibody against the PEG moiety determined by enzyme-linked immunosorbent assay (ELISA). For participants with a positive result, IgM antibodies were tested.

Trial Locations

Locations (13)

Ciutat Sanitaria i Universitaria de la Vall d'Hebron; 🇪🇸 Barcelona, Spain

Hospital Universitari i Politecnic La Fe | Hematologia; 🇪🇸 Valencia, Spain

Hospital Universitario "La Paz"; 🇪🇸 Madrid, Spain

LAIKO General Hospital of Athens; 🇬🇷 Athens, Greece

A.O.U. Policlinico Umberto I; 🇮🇹 Roma, Lazio, Italy

Aarhus Universitetshospital, Skejby; 🇩🇰 Arhus N, Denmark

UMHAT Tsaritsa Joanna-ISUL EAD Sofia; 🇧🇬 Sofia, Bulgaria

Oslo Universitetssykehus HF, Rikshospitalet; 🇳🇴 Oslo, Norway

MHAT Sveta Marina EAD; 🇧🇬 Varna, Bulgaria

A.O. Pugliese-Ciaccio; 🇮🇹 Catanzaro, Calabria, Italy

Fondazione Policlinico Universitario Agostino Gemelli IRCCS; 🇮🇹 Roma, Lazio, Italy

Uniwersyteckie Centrum Kliniczne; 🇵🇱 Gdansk, Poland

SP Szpital Kliniczny Nr 1; 🇵🇱 Wroclaw, Poland