search
Back to results

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

Primary Purpose

Hemophilia A

Status
Completed
Phase
Phase 4
Locations
International
Study Type
Interventional
Intervention
Damoctocog alfa pegol (Jivi, BAY94-9027)
Sponsored by
Bayer
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Hemophilia A

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)MaleDoes not accept healthy volunteers

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.

Sites / Locations

  • UMHAT Tsaritsa Joanna-ISUL EAD Sofia
  • MHAT Sveta Marina EAD
  • Aarhus Universitetshospital, Skejby
  • LAIKO General Hospital of Athens
  • A.O. Pugliese-Ciaccio
  • A.O.U. Policlinico Umberto I
  • Fondazione Policlinico Universitario Agostino Gemelli IRCCS
  • Oslo Universitetssykehus HF, Rikshospitalet
  • Uniwersyteckie Centrum Kliniczne
  • SP Szpital Kliniczny Nr 1
  • Ciutat Sanitaria i Universitaria de la Vall d'Hebron
  • Hospital Universitario "La Paz"
  • Hospital Universitari i Politecnic La Fe | Hematologia

Arms of the Study

Arm 1

Arm Type

Other

Arm Label

Severe hemophilia A patients

Arm Description

Prophylactic treatment regimens should be guided by clinical judgement based on individual patient characteristics and treatment response.

Outcomes

Primary Outcome Measures

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).

Secondary Outcome Measures

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.
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.
Annualized Bleeding Rate (ABR)
ABR is number of all bleeds per individual treatment period annualized to a 1-year time interval.

Full Information

First Posted
September 9, 2019
Last Updated
July 26, 2023
Sponsor
Bayer
search

1. Study Identification

Unique Protocol Identification Number
NCT04085458
Brief Title
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

2. Study Status

Record Verification Date
July 2023
Overall Recruitment Status
Completed
Study Start Date
September 23, 2019 (Actual)
Primary Completion Date
May 20, 2022 (Actual)
Study Completion Date
August 26, 2022 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Bayer

4. Oversight

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
Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hemophilia A

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
32 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Severe hemophilia A patients
Arm Type
Other
Arm Description
Prophylactic treatment regimens should be guided by clinical judgement based on individual patient characteristics and treatment response.
Intervention Type
Drug
Intervention Name(s)
Damoctocog alfa pegol (Jivi, BAY94-9027)
Intervention Description
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.
Primary Outcome Measure Information:
Title
FVIII Inhibitor Development by the Nijmegen Bethesda Assay
Description
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).
Time Frame
Observed for 100 exposure days (EDs), up to 2 years
Secondary Outcome Measure Information:
Title
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
Description
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.
Time Frame
Observed for 100 exposure days (EDs), up to 2 years
Title
Development of Treatment-emergent Anti-PEG Antibodies
Description
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.
Time Frame
Observed for 100 exposure days (EDs), up to 2 years
Title
Annualized Bleeding Rate (ABR)
Description
ABR is number of all bleeds per individual treatment period annualized to a 1-year time interval.
Time Frame
Observed for 100 exposure days (EDs), up to 2 years

10. Eligibility

Sex
Male
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
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.
Facility Information:
Facility Name
UMHAT Tsaritsa Joanna-ISUL EAD Sofia
City
Sofia
ZIP/Postal Code
1527
Country
Bulgaria
Facility Name
MHAT Sveta Marina EAD
City
Varna
ZIP/Postal Code
9010
Country
Bulgaria
Facility Name
Aarhus Universitetshospital, Skejby
City
Arhus N
ZIP/Postal Code
8200
Country
Denmark
Facility Name
LAIKO General Hospital of Athens
City
Athens
ZIP/Postal Code
115 27
Country
Greece
Facility Name
A.O. Pugliese-Ciaccio
City
Catanzaro
State/Province
Calabria
ZIP/Postal Code
88100
Country
Italy
Facility Name
A.O.U. Policlinico Umberto I
City
Roma
State/Province
Lazio
ZIP/Postal Code
00161
Country
Italy
Facility Name
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
City
Roma
State/Province
Lazio
ZIP/Postal Code
00168
Country
Italy
Facility Name
Oslo Universitetssykehus HF, Rikshospitalet
City
Oslo
ZIP/Postal Code
0372
Country
Norway
Facility Name
Uniwersyteckie Centrum Kliniczne
City
Gdansk
ZIP/Postal Code
80-214
Country
Poland
Facility Name
SP Szpital Kliniczny Nr 1
City
Wroclaw
ZIP/Postal Code
50-367
Country
Poland
Facility Name
Ciutat Sanitaria i Universitaria de la Vall d'Hebron
City
Barcelona
ZIP/Postal Code
08035
Country
Spain
Facility Name
Hospital Universitario "La Paz"
City
Madrid
ZIP/Postal Code
28046
Country
Spain
Facility Name
Hospital Universitari i Politecnic La Fe | Hematologia
City
Valencia
ZIP/Postal Code
46026
Country
Spain

12. IPD Sharing Statement

Plan to Share IPD
Undecided
IPD Sharing 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.
Links:
URL
http://www.clinicaltrialsregister.eu/
Description
Click here to find information about studies related to Bayer Healthcare products conducted in Europe
URL
http://clinicaltrials.bayer.com/
Description
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.

Learn more about this trial

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

We'll reach out to this number within 24 hrs