Jivi for Hemophilia A in Children

(Alfa-PROTECT Trial)

No longer recruiting at 52 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to find a better way to treat severe hemophilia A in children aged 7 to less than 12 years. Researchers are testing an injectable treatment called Damoctocog alfa pegol (Jivi, BAY94-9027), which helps the blood clot by replacing a missing protein. The focus is on understanding the safety and effectiveness of this treatment, particularly regarding allergic reactions or instances where it does not stop bleeding as expected. Children with severe hemophilia A who have received clotting factor treatments at least 50 times may be suitable for this trial. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to a potentially groundbreaking treatment for severe hemophilia A.

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 there any evidence suggesting that BAY94-9027 is likely to be safe for children aged 7 to less than 12 years?

Research has shown that Jivi (also known as BAY94-9027 or damoctocog alfa pegol) is generally well-tolerated. Studies with patients aged 12 and older have found no major safety concerns, and the treatment did not cause significant immune reactions in these groups.

For children aged 6 to under 12 years, 29 children who previously used Jivi did not experience allergic reactions or issues with the treatment's effectiveness. This is promising, especially since Jivi is already approved for older children and adults. However, researchers need more information to confirm these findings for younger children, particularly those between 7 and 12 years old.

It is important to note that drugs like Jivi can sometimes cause the body to produce antibodies, which might lead to allergic reactions or reduce the drug's effectiveness. However, such problems have not been reported in this age group so far. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for hemophilia A?

Unlike the standard treatments for Hemophilia A, which often require frequent infusions, Jivi (damoctocog alfa pegol) is designed to extend the time between doses. Researchers are excited about Jivi because it uses a unique PEGylation technology that prolongs the half-life of the treatment, meaning it stays active in the body longer. This could reduce injection frequency, making life easier and more comfortable for young patients managing this condition.

What evidence suggests that Jivi might be an effective treatment for hemophilia A in children aged 7 to less than 12 years?

Research has shown that damoctocog alfa pegol (Jivi), the treatment under study in this trial, helps prevent bleeding in people with hemophilia A. In an earlier study, almost all participants with mild or moderate hemophilia A who used this treatment did not experience any bleeding. Another large study with 205 patients found it very effective for those with severe hemophilia A. This treatment replaces the missing clotting factor 8, which is essential for proper blood clotting. Real-world evidence also supports its effectiveness, as patients using it as a preventive measure generally reported fewer or no bleeding episodes. ^{2 3 4 6 7}

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. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/39558414/

Effectiveness of Damoctocog Alfa Pegol to Treat Patients ... Results: At data cutoff, 205 PLwHA were treated with damoctocog alfa pegol: 150 (73.2%) severe (1 female [0.5%]) and 55 (26.8%) mild/moderate (3 [1.5%] female).

2. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/38229269/

Safety and efficacy of damoctocog alfa pegol prophylaxis in ... We report the final results of an interventional, post-marketing study of damoctocog alfa pegol prophylaxis in patients with severe haemophilia A.

3. hematologyadvisor.com hematologyadvisor.com/news/hemophilia-damoctocog-alfa-real-world-data-efficacy-treatment-risk/

Real-World Data Supports Efficacy of Damoctocog Alfa ... According to the findings, nearly all individuals with mild or moderate hemophilia A on prophylaxis with damoctocog alfa pegol experienced no bleeds.

4. clinicaltrials.gov clinicaltrials.gov/study/NCT07088458

NCT07088458 | An Observational Study to Learn More ... In this study, the data will be collected from participants who are receiving their usual treatment with damoctocog alfa pegol as prescribed by their doctor.

5. ashpublications.org ashpublications.org/blood/article/134/Supplement_1/1117/426767/Efficacy-and-Safety-of-BAY-94-9027-Damoctocog-Alfa

Efficacy and Safety of BAY 94-9027 (Damoctocog Alfa Pegol ... Results: A total of 104 patients who received BAY 94-9027 prophylaxis during the main study and the Extension (data cut-off: Jan 2018) were ...

6. sciencedirect.com sciencedirect.com/science/article/pii/S0006497118590340

Efficacy and Safety of BAY 94-9027 (Damoctocog Alfa ... This post hoc analysis assessed bleeding rates and safety outcomes for prophylaxis patients in PROTECT VIII and its Extension, based on the ...

7. karger.com karger.com/aha/article/doi/10.1159/000538702/906506/Long-term-efficacy-and-safety-of-damoctocog-alfa

Long-Term Efficacy and Safety of Damoctocog Alfa Pegol ... We report a post hoc assessment of bleeding and safety outcomes in the subgroup of patients, aged 12–<18 years at enrolment. Method: PROTECT ...