Jivi for Hemophilia A in Children

(Alfa-PROTECT Trial)

Recruiting at 39 trial locations

Overseen By Bayer Clinical Trials Contact

Age: < 18

Sex: Male

Trial Phase: Phase 3

Sponsor: Bayer

Must not be taking: Antihistamines, Chemotherapy, Corticosteroids, others

Disqualifiers: FVIII inhibitors, Other bleeding disorders, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 19

35 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

Researchers are looking for a better way to treat hemophilia A. Hemophilia A is a genetic disorder where the body does not create enough of a protein called clotting factor 8 (FVIII) present in the blood. People with hemophilia A may bleed for a long time from minor wounds, have painful bleeding into joints, or have internal bleeding. In severe hemophilia A (clotting factor 8 levels less than 1%) bleedings are more likely to happen. In this study researchers want to learn more about the treatment called BAY94-9027. BAY94-9027 is an injectable medicine used to replace missing clotting factor 8. In BAY94-9027 the clotting factor 8 has been pegylated (combined with a substance called polyethylene glycol (PEG)). This is to make the treatment last longer in the body so that less injections are required. BAY94-9027 is already available for the prevention and treatment of bleeding in adults and children who are 12 years and older. BAY 94-9027 is also called Jivi. BAY94-9027 is not yet available for children aged 7 to less than 12 years. One potential specific risk of pegylated drugs is that proteins in the blood called antibodies are built. These may attach to the pegylation part of the drug and this in turn may lead to allergic reactions and the drug not working as well as it should during first 4 infusions. In studies that have been done so far, this has been seen in some children younger than six years, but not in 29 children aged 6 to less than 12 years treated with BAY94-9027. Further safety information related to how the body reacts to BAY94-9027 is however still needed for this age group. The main purpose of this study is to learn how safe BAY94-9027 is (safety) and how it affects the body (tolerability) in previously treated children with severe hemophilia A who are between 7 to less than 12 years. To answer this question, the researchers will study information about two medical problems of special interest, if allergic reactions occur (also called hypersensitivity) and if the drug is not working as well as it should (also called loss of efficacy) during the first 4 infusions. Allergic reactions may range from mild local reactions to widespread effects such as shortness of breath, skin rashes and low blood pressure. Only allergic reactions related to the study treatment will be considered. The assessment if loss of efficacy occurred will be based on the occurrence of bleeding, the clotting factor 8 level in blood after injection called recovery, clotting factor 8 inhibitor tests and measurement of antibodies against the PEG. The study has two parts, A and B. Part A takes 6 months and part B takes 18 months. In part A the participants will receive two injections of BAY94-9027 per week. In part B, the number of injections may be decreased, with up to five days between the injections. The participants in this study will visit the study site around 14 times and will have 15 phone visits. In part A, visit 1 is for screening. Visits 2 to 5 take place twice a week for two weeks. Visit 6 two weeks after visit 5, visits 7 to 10 take place monthly with visit 11 six weeks after visit 10. In part B, site visits will occur on month 9, 12, 18 and 24 and phone calls every month between the site visits. The participants' and their caregivers will record in an electronic patient diary information about when the study treatment was given and bleeding episodes that have happened. During the study, the study doctors and their team will * take blood samples, * do physical examinations, * review the participants' electronic diary * ask questions about the participants' quality of life, * ask the participants questions about how they are feeling and what adverse events they are having An adverse event is a medical problem that happens during the study. Doctors keep track of all adverse events that happen in study, even if they do not think the adverse events might be related to the study treatments.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but you cannot participate if you are on certain treatments like chemotherapy, immune modulatory drugs, or corticosteroids. It's best to discuss your specific medications with the study team.

Is Jivi (damoctocog alfa pegol) safe for children with Hemophilia A?

Jivi (damoctocog alfa pegol) has been generally well tolerated in adults and adolescents with Hemophilia A, with most side effects not related to the treatment. In studies, no new or confirmed cases of inhibitor development (a type of immune response) were found, and any anti-PEG antibodies (immune responses to the drug's coating) were low and temporary. ^{1 2 3 4 5}

What makes the drug Jivi unique for treating Hemophilia A in children?

Jivi (damoctocog alfa pegol) is unique because it is a PEGylated recombinant factor VIII with an extended half-life, allowing for less frequent dosing, potentially as little as once a week, compared to other treatments that require more frequent administration. ^{1 2 3 5 6}

What data supports the effectiveness of the drug Jivi for treating Hemophilia A in children?

Research shows that Jivi (damoctocog alfa pegol) is effective in reducing bleeding episodes in patients with Hemophilia A. In a study, 75% of patients reported no bleeding episodes over six months, and 82% had no joint bleeds, indicating improved joint health and quality of life. ^{1 2 3 4 5}

Are You a Good Fit for This Trial?

This trial is for children aged 7 to less than 12 with severe hemophilia A, who have been previously treated with clotting factor concentrates. They must not have used certain other treatments recently and should be free of inhibitors to FVIII. Participants need the ability to use an electronic diary and consent from parents.

Inclusion Criteria

Participants with severe hemophilia A (participant's own FVIII activity [FVIII:C] <1%)

I am willing and able to learn how to use an electronic diary for the study.

I have been treated with FVIII for at least 50 days before signing the consent.

Exclusion Criteria

Any other significant medical condition that the investigator feels would be a risk to the patient or would impede the study

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 study entry or previous participation in a clinical study with BAY94-9027

I am scheduled for a major surgery during the study period.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

1 week

1 visit (in-person)

Treatment Part A

Participants receive two injections of BAY94-9027 per week to evaluate safety and tolerability

6 months

14 visits (in-person), 15 visits (phone)

Treatment Part B

Participants continue treatment with potentially reduced frequency of injections

18 months

4 visits (in-person), monthly phone calls

Follow-up

Participants are monitored for safety and effectiveness after treatment

6 months

What Are the Treatments Tested in This Trial?

Interventions

• Damoctocog alfa pegol (Jivi, BAY94-9027)

Trial Overview Researchers are testing BAY94-9027 (Jivi), a pegylated injectable treatment designed to replace missing clotting factor VIII in kids with severe hemophilia A. The study aims to determine its safety and how it affects the body over a period that spans up to two years.

How Is the Trial Designed?

Group I: Main study (Part A) and the extension study (Part B) Experimental Treatment 1 Intervention

Part A will last for 6 months. After completing Part A participants will continue in the extension study for another 18 months.

Who Is Running the Clinical Trial?

Bayer

Lead Sponsor

Trials

2,291

Recruited

25,560,000+

Founded

1863

Headquarters

Leverkusen, Germany

Known For

Pharmaceutical Innovations

Top Products

Aspirin, Aleve, Yaz, Nexavar

Bill Anderson

Bayer

Chief Executive Officer since 2023

BSc in Chemical Engineering from the University of Texas, MSc in Chemical Engineering and Management from MIT

Michael Devoy

Bayer

Chief Medical Officer since 2014

MD, PhD

Published Research Related to This Trial

BAY 94-9027 (damoctocog alfa pegol) demonstrated long-term safety in previously treated pediatric patients with severe hemophilia A, with no development of FVIII inhibitors or anti-PEG antibodies over a median treatment duration of 5 years.

The treatment significantly reduced the annualized bleeding rate (ABR), with a median ABR of 1.5 for patients under 6 years and 1.9 for those aged 6-12 years, and a spontaneous ABR of 0.0 in the last year of treatment, indicating effective bleed prevention.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

PROTECT VIII kids extension study: Long-term safety and efficacy of BAY 94-9027 (damoctocog alfa pegol) in children with severe haemophilia A. Mancuso, ME., Biss, T., Fischer, K., et al.[2021]

Citations

1. New Zealand pubmed.ncbi.nlm.nih.gov

Damoctocog Alfa Pegol: A Review in Haemophilia A. [2019]

2. England pubmed.ncbi.nlm.nih.gov

Design of the HEM-POWR study: a prospective, observational study of real-world treatment with damoctocog alfa pegol in patients with haemophilia A. [2021]

3. Switzerland pubmed.ncbi.nlm.nih.gov

Damoctocog Alfa Pegol for Hemophilia A Prophylaxis: An Italian Multicenter Survey. [2023]

4. England pubmed.ncbi.nlm.nih.gov

Interim analyses of the multinational real-world prospective cohort HEM-POWR study evaluating the effectiveness and safety of damoctocog alfa pegol in patients with hemophilia A. [2023]

5. England pubmed.ncbi.nlm.nih.gov

PROTECT VIII kids extension study: Long-term safety and efficacy of BAY 94-9027 (damoctocog alfa pegol) in children with severe haemophilia A. [2021]

6. England pubmed.ncbi.nlm.nih.gov

Confirmed long-term safety and efficacy of prophylactic treatment with BAY 94-9027 in severe haemophilia A: final results of the PROTECT VIII extension study. [2022]