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

Post-marketing Investigation (PMI) to Assess Safety and Efficacy of Jivi (BAY 94-9027) Treatment in Participants With Hemophilia A

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.

Study Overview

• Status: Completed
• Conditions: Hemophilia A
• Intervention / Treatment: Drug: Damoctocog alfa pegol (Jivi, BAY94-9027)

• Study Type: Interventional
• Enrollment (Actual): 32
• Phase: Phase 4

Contacts and Locations

Study Locations

• Bulgaria
  • Sofia, Bulgaria, 1527
    • UMHAT Tsaritsa Joanna-ISUL EAD Sofia
  • Varna, Bulgaria, 9010
    • MHAT Sveta Marina EAD
• Denmark
  • Arhus N, Denmark, 8200
    • Aarhus Universitetshospital, Skejby
• Greece
  • Athens, Greece, 115 27
    • Laiko General Hospital of Athens
• Italy
  • Calabria
    • Catanzaro, Calabria, Italy, 88100
      • A.O. Pugliese-Ciaccio
  • Lazio
    • Roma, Lazio, Italy, 00168
      • Fondazione Policlinico Universitario Agostino Gemelli IRCCS
    • Roma, Lazio, Italy, 00161
      • A.O.U. Policlinico Umberto I
• Norway
  • Oslo, Norway, 0372
    • Oslo Universitetssykehus HF, Rikshospitalet
• Poland
  • Gdansk, Poland, 80-214
    • Uniwersyteckie Centrum Kliniczne
  • Wroclaw, Poland, 50-367
    • SP Szpital Kliniczny Nr 1
• Spain
  • Barcelona, Spain, 08035
    • Ciutat Sanitaria i Universitaria de la Vall d'Hebron
  • Madrid, Spain, 28046
    • Hospital Universitario "La Paz"
  • Valencia, Spain, 46026
    • Hospital Universitari i Politecnic La Fe | Hematologia

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Other: Severe hemophilia A patients; Prophylactic treatment regimens should be guided by clinical judgement based on individual patient characteristics and treatment response.

Drug: Damoctocog alfa pegol (Jivi, BAY94-9027); The recommended starting dose is every 5 days treatment (45 IU/kg)- An assessment of response to treatment will be performed at the next scheduled visit after 10-15 ED (8-10 weeks). Participants may be assigned to different dosing regimens (every 7 days or 2x/week) or continue with every 5 days regimen, according to individual bleeding tendency and needs at investigator's discretion.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
FVIII Inhibitor Development by the Nijmegen Bethesda Assay	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).	Observed for 100 exposure days (EDs), up to 2 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants With Treatment-emergent Adverse Events (TEAEs)	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.	Observed for 100 exposure days (EDs), up to 2 years
Development of Treatment-emergent Anti-PEG Antibodies	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.	Observed for 100 exposure days (EDs), up to 2 years
Annualized Bleeding Rate (ABR)	ABR is number of all bleeds per individual treatment period annualized to a 1-year time interval.	Observed for 100 exposure days (EDs), up to 2 years

Collaborators and Investigators

• Sponsor: Bayer

Publications and helpful links

Helpful Links

• Click here to find information about studies related to Bayer Healthcare products conducted in Europe
• Click here to find information for studies related to Bayer products. To find this study enter the ClinicalTrials.gov identifier (NCT) number or Bayer Study Identifier (ID) in the search field.

Study record dates

Study Major Dates

• Study Start (Actual): September 23, 2019
• Primary Completion (Actual): May 20, 2022
• Study Completion (Actual): August 26, 2022

Study Registration Dates

• First Submitted: September 9, 2019
• First Submitted That Met QC Criteria: September 9, 2019
• First Posted (Actual): September 11, 2019

Study Record Updates

• Last Update Posted (Actual): July 27, 2023
• Last Update Submitted That Met QC Criteria: July 26, 2023
• Last Verified: July 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Hematologic Diseases; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Blood Coagulation Disorders; Hemophilia A; Coagulants; Factor VIII
• Other Study ID Numbers: 19764; 2018-003655-37 (EudraCT Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

IPD Plan Description

Availability of this study's data will be determined according to Bayer's commitment to the EFPIA/PhRMA "Principles for responsible clinical trial data sharing". This pertains to scope, timepoint and process of data access.

As such, Bayer commits to sharing upon request from qualified researchers patient-level clinical trial data, study-level clinical trial data, and protocols from clinical trials in patients for medicines and indications approved in the US and EU as necessary for conducting legitimate research. This applies to data on new medicines and indications that have been approved by the EU and US regulatory agencies on or after January 01, 2014.

Interested researchers can use www.clinicalstudydatarequest.com to request access to anonymized patient-level data and supporting documents from clinical studies to conduct research. Information on the Bayer criteria for listing studies and other relevant information is provided in the Study sponsors section of the portal.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes