Trial Outcomes & Findings for Study to Gain More Information on How Safe and Effective Jivi Works in Patients With Severe Hemophilia A (Post-marketing Investigation) (NCT NCT04085458)

Last Updated: 2023-07-27

Results Overview

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).

Recruitment status

COMPLETED

Study phase

PHASE4

Target enrollment

32 participants

Primary outcome timeframe

Observed for 100 exposure days (EDs), up to 2 years

Results posted on

2023-07-27

Participant Flow

The study was conducted at 13 centers in 7 countries between 23 SEP 2019 (First participant first visit) and 26 Aug 2022 (last participant's data from the last visit were received). Bulgaria (2 centers), Denmark (1 centers), Spain (3 centers), Greece (1 center), Italy (3 centers), Norway (1 center), Poland (2 centers).

36 participants were screened into the study (signed informed consent form (ICF)). 4 participants were screening failed.

Participant milestones

Participant milestones
Measure	Severe Hemophilia A Patients With Damoctocog Alfa Pegol (Jivi, BAY94-9027) Treatment Prophylaxis regimens with every 5 days treatment (45-60 IU/kg), or every 7 days (60 IU/kg) or twice per week (40 IU/kg).
Overall Study STARTED	32
Overall Study COMPLETED	27
Overall Study NOT COMPLETED	5

Reasons for withdrawal

Reasons for withdrawal
Measure	Severe Hemophilia A Patients With Damoctocog Alfa Pegol (Jivi, BAY94-9027) Treatment Prophylaxis regimens with every 5 days treatment (45-60 IU/kg), or every 7 days (60 IU/kg) or twice per week (40 IU/kg).
Overall Study Other	1
Overall Study Withdrawal by Subject	2
Overall Study Adverse Event	2

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Severe Hemophilia A Patients With Damoctocog Alfa Pegol (Jivi, BAY94-9027) Treatment n=32 Participants Prophylaxis regimens with every 5 days treatment (45-60 IU/kg), or every 7 days (60 IU/kg) or twice per week (40 IU/kg).
Age, Continuous	42.8 years STANDARD_DEVIATION 15.1 • n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants
Sex: Female, Male Male	32 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	2 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	30 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	32 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants

PRIMARY outcome

Timeframe: Observed for 100 exposure days (EDs), up to 2 years

Population: Safety analysis set (SAF): A participant was included in the SAF if he received at least 1 infusion of study drug.

Outcome measures

Outcome measures
Measure	Severe Hemophilia A Patients With Damoctocog Alfa Pegol (Jivi, BAY94-9027) Treatment n=32 Participants Prophylaxis regimens with every 5 days treatment (45-60 IU/kg), or every 7 days (60 IU/kg) or twice per week (40 IU/kg).
FVIII Inhibitor Development by the Nijmegen Bethesda Assay Any positive FVIII inhibitor	0 Participants
FVIII Inhibitor Development by the Nijmegen Bethesda Assay High titer FVIII inhibitor	0 Participants
FVIII Inhibitor Development by the Nijmegen Bethesda Assay Low titer FVIII inhibitor	0 Participants

SECONDARY outcome

Timeframe: Observed for 100 exposure days (EDs), up to 2 years

Population: Safety analysis set (SAF): A participant was included in the SAF if he received at least 1 infusion of study drug.

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.

Outcome measures

Outcome measures
Measure	Severe Hemophilia A Patients With Damoctocog Alfa Pegol (Jivi, BAY94-9027) Treatment n=32 Participants Prophylaxis regimens with every 5 days treatment (45-60 IU/kg), or every 7 days (60 IU/kg) or twice per week (40 IU/kg).
Number of Participants With Treatment-emergent Adverse Events (TEAEs) Any AE	21 participants
Number of Participants With Treatment-emergent Adverse Events (TEAEs) Any study drug-related AE	3 participants
Number of Participants With Treatment-emergent Adverse Events (TEAEs) Any AE related to procedures required by the protocol	0 participants
Number of Participants With Treatment-emergent Adverse Events (TEAEs) Any AE leading to discontinuation of study drug	2 participants
Number of Participants With Treatment-emergent Adverse Events (TEAEs) Any SAE	2 participants
Number of Participants With Treatment-emergent Adverse Events (TEAEs) Any study drug-related SAE	0 participants
Number of Participants With Treatment-emergent Adverse Events (TEAEs) Any SAE related to procedures required by the protocol	0 participants
Number of Participants With Treatment-emergent Adverse Events (TEAEs) Any SAE leading to discontinuation of study drug	0 participants
Number of Participants With Treatment-emergent Adverse Events (TEAEs) AE with outcome death	0 participants

SECONDARY outcome

Timeframe: Observed for 100 exposure days (EDs), up to 2 years

Population: Safety analysis set (SAF): A participant was included in the SAF if he received at least 1 infusion of study drug.

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.

Outcome measures

Outcome measures
Measure	Severe Hemophilia A Patients With Damoctocog Alfa Pegol (Jivi, BAY94-9027) Treatment n=32 Participants Prophylaxis regimens with every 5 days treatment (45-60 IU/kg), or every 7 days (60 IU/kg) or twice per week (40 IU/kg).
Development of Treatment-emergent Anti-PEG Antibodies	3 participants

SECONDARY outcome

Timeframe: Observed for 100 exposure days (EDs), up to 2 years

Population: Modified intent-to-treat set (mITT): A participant was included in the mITT if he received at least 1 infusion of study drug and had injection/bleeding data available for at least 3 months. This time period is considered the minimum observation time for a reliable annualization of the observed bleed rate.

ABR is number of all bleeds per individual treatment period annualized to a 1-year time interval.

Outcome measures

Outcome measures
Measure	Severe Hemophilia A Patients With Damoctocog Alfa Pegol (Jivi, BAY94-9027) Treatment n=30 Participants Prophylaxis regimens with every 5 days treatment (45-60 IU/kg), or every 7 days (60 IU/kg) or twice per week (40 IU/kg).
Annualized Bleeding Rate (ABR)	1.8 Bleeds per year Interval 0.7 to 5.9

Adverse Events

Severe Hemophilia A Patients With Damoctocog Alfa Pegol (Jivi, BAY94-9027) Treatment

Serious events: 2 serious events

Other events: 15 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Severe Hemophilia A Patients With Damoctocog Alfa Pegol (Jivi, BAY94-9027) Treatment n=32 participants at risk Prophylaxis regimens with every 5 days treatment (45-60 IU/kg), or every 7 days (60 IU/kg) or twice per week (40 IU/kg).
Injury, poisoning and procedural complications Fall	3.1% 1/32 • Number of events 1 • Timeframe for TEAEs: After the first study intervention up to 7 days after the end of study intervention, with an average of 475 days. Timeframe for the all-cause mortality: All deaths that occurred at any time during the study before the last contact, with an average of 476 days.
Musculoskeletal and connective tissue disorders Osteoarthritis	3.1% 1/32 • Number of events 1 • Timeframe for TEAEs: After the first study intervention up to 7 days after the end of study intervention, with an average of 475 days. Timeframe for the all-cause mortality: All deaths that occurred at any time during the study before the last contact, with an average of 476 days.

Other adverse events

Other adverse events
Measure	Severe Hemophilia A Patients With Damoctocog Alfa Pegol (Jivi, BAY94-9027) Treatment n=32 participants at risk Prophylaxis regimens with every 5 days treatment (45-60 IU/kg), or every 7 days (60 IU/kg) or twice per week (40 IU/kg).
General disorders Pyrexia	6.2% 2/32 • Number of events 2 • Timeframe for TEAEs: After the first study intervention up to 7 days after the end of study intervention, with an average of 475 days. Timeframe for the all-cause mortality: All deaths that occurred at any time during the study before the last contact, with an average of 476 days.
Infections and infestations Upper respiratory tract infection	15.6% 5/32 • Number of events 5 • Timeframe for TEAEs: After the first study intervention up to 7 days after the end of study intervention, with an average of 475 days. Timeframe for the all-cause mortality: All deaths that occurred at any time during the study before the last contact, with an average of 476 days.
Injury, poisoning and procedural complications Limb injury	9.4% 3/32 • Number of events 3 • Timeframe for TEAEs: After the first study intervention up to 7 days after the end of study intervention, with an average of 475 days. Timeframe for the all-cause mortality: All deaths that occurred at any time during the study before the last contact, with an average of 476 days.
Musculoskeletal and connective tissue disorders Arthralgia	9.4% 3/32 • Number of events 3 • Timeframe for TEAEs: After the first study intervention up to 7 days after the end of study intervention, with an average of 475 days. Timeframe for the all-cause mortality: All deaths that occurred at any time during the study before the last contact, with an average of 476 days.
Musculoskeletal and connective tissue disorders Back pain	6.2% 2/32 • Number of events 2 • Timeframe for TEAEs: After the first study intervention up to 7 days after the end of study intervention, with an average of 475 days. Timeframe for the all-cause mortality: All deaths that occurred at any time during the study before the last contact, with an average of 476 days.
Respiratory, thoracic and mediastinal disorders Cough	6.2% 2/32 • Number of events 2 • Timeframe for TEAEs: After the first study intervention up to 7 days after the end of study intervention, with an average of 475 days. Timeframe for the all-cause mortality: All deaths that occurred at any time during the study before the last contact, with an average of 476 days.
Respiratory, thoracic and mediastinal disorders Epistaxis	6.2% 2/32 • Number of events 4 • Timeframe for TEAEs: After the first study intervention up to 7 days after the end of study intervention, with an average of 475 days. Timeframe for the all-cause mortality: All deaths that occurred at any time during the study before the last contact, with an average of 476 days.
Surgical and medical procedures Tooth extraction	6.2% 2/32 • Number of events 2 • Timeframe for TEAEs: After the first study intervention up to 7 days after the end of study intervention, with an average of 475 days. Timeframe for the all-cause mortality: All deaths that occurred at any time during the study before the last contact, with an average of 476 days.

Additional Information

Therapeutic Area Head

Bayer

Phone: 1-888-8422937

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: GT60