Hemophilia APhase 4NCT04085458
Study to Gain More Information on How Safe and Effective Jivi Works in Patients With Severe Hemophilia A (Post-marketing Investigation)
A Phase 4 study testing Damoctocog alfa pegol (Jivi, BAY94-9027) in people with Hemophilia A, run by Bayer at 13 sites in 7 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older, men only
- Conditions the study is for
- Hemophilia A
- What is being tested?
- Damoctocog alfa pegol (Jivi, BAY94-9027)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 32 participants
- Where
- 13 sitesin 7 countries
- Study dates
- 23 Sep 2019to 26 Aug 2022
- Phase
- Phase 4
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- Men only
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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.
Trial locations and contacts
13 sites in 7 countries.
13 sites in 7 countries
Italy3 sites
A.O. Pugliese-Ciaccio
Catanzaro, Calabria, 88100
A.O.U. Policlinico Umberto I
Rome, Lazio, 00161
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Rome, Lazio, 00168
Spain3 sites
Ciutat Sanitaria i Universitaria de la Vall d'Hebron
Barcelona, 08035
Hospital Universitario "La Paz"
Madrid, 28046
Hospital Universitari i Politecnic La Fe | Hematologia
Valencia, 46026
Bulgaria2 sites
UMHAT Tsaritsa Joanna-ISUL EAD Sofia
Sofia, 1527
MHAT Sveta Marina EAD
Varna, 9010
Poland2 sites
Uniwersyteckie Centrum Kliniczne
Gdansk, 80-214
SP Szpital Kliniczny Nr 1
Wroclaw, 50-367
About this study
Official title: 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.
- Conditions
- Hemophilia A
- Being tested
- Damoctocog alfa pegol (Jivi, BAY94-9027)
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Damoctocog alfa pegol (Jivi, BAY94-9027)Drug
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.
| Study group | Role | What they receive |
|---|---|---|
| Severe hemophilia A patients | Other | Prophylactic treatment regimens should be guided by clinical judgement based on individual patient characteristics and treatment response. Receives: Drug: Damoctocog alfa pegol (Jivi, BAY94-9027) |
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: Observed for 100 exposure days (EDs), up to 2 years
Secondary outcomes (3)
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.
Measured: 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.
Measured: 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.
Measured: Observed for 100 exposure days (EDs), up to 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Sep 2019
- Study start23 Sep 2019
- Primary completion20 May 2022
- Study completion26 Aug 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Hemophilia AStudies of the same condition, open and under way.
- More trials run by BayerThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 32 participants (actual)
Sponsor and investigators
- Lead sponsor
- Bayer
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04085458
- Sponsor's protocol ID
- 19764
- Other IDs
- 2018-003655-37, 2018
- Status last verified
- Jul 2023
- First submitted
- 9 Sep 2019
- Last updated
- 27 Jul 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided: 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.
Study at a glance
From the registry record, last updated by the sponsor on 27 Jul 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Post-marketing Investigation (PMI) to Assess Safety and Efficacy of Jivi (BAY 94-9027) Treatment in Participants With Hemophilia A
- ClinicalTrials.gov ID
- NCT04085458
- Acronym / sponsor ID
- 19764
- Conditions
- Hemophilia A
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 32 participants (actual)
- Lead sponsor
- BayerIndustry (pharma, biotech, devices)
- Study start
- 23 Sep 2019 (actual)
- Primary completion
- 20 May 2022 (actual)
- Study completion
- 26 Aug 2022 (actual)
- First posted
- 11 Sep 2019 (actual)
- Results
- Posted 28 Jun 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Severe hemophilia A patients | Other | Drug: Damoctocog alfa pegol (Jivi, BAY94-9027)Prophylactic treatment regimens should be guided by clinical judgement based on individual patient characteristics and treatment response. |
Who may qualify
- Ages
- 18 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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
Key exclusion criteria (8 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 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. | Drug | Severe hemophilia A patients |
Primary outcome
FVIII Inhibitor Development by the Nijmegen Bethesda Assay
Time frame: 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).
3 secondary outcomes
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
Time frame: Observed for 100 exposure days (EDs), up to 2 years
Development of Treatment-emergent Anti-PEG Antibodies
Time frame: Observed for 100 exposure days (EDs), up to 2 years
Annualized Bleeding Rate (ABR)
Time frame: Observed for 100 exposure days (EDs), up to 2 years
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Jul 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Click here to find information about studies related to Bayer Healthcare products conducted in EuropeLink provided by the sponsor.
- Individual participant dataSharing planned: Undecided. 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
13 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Damoctocog alfa pegol (Jivi, BAY94-9027) in people with Hemophilia A, run by Bayer at 13 sites in 7 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 27 Jul 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 13 sites in Italy, Spain, Bulgaria, Poland, Denmark and 2 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 28 Jun 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
FVIII Inhibitor Development by the Nijmegen Bethesda Assay (time frame: Observed for 100 exposure days (EDs), up to 2 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Jul 2023.