A Study to Learn How Safe the Study Treatment BAY94-9027 is and How it Affects the Body in Previously Treated Children Aged 7 to Less Than 12 Years With Severe Hemophilia A, a Genetic Bleeding Disorder That is Caused by the Lack of a Protein Called Clotting Factor 8 (FVIII) in the Blood

NCT05147662 | Completed Phase 3 Results FDA Drug

• 3 other identifiers: 218242023-504388-18-002021-004858-30
• Study Type: interventional
• Target: 36
• Locations: 7 countries
• Sites: 17

Brief Summary

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.

Conditions

Treatment of Bleeding; Prophylaxis of Bleeding; Hemophilia A; Children

Outcome Measures

Primary Outcomes (1)

• Adverse Events of Special Interest (AESI) Hypersensitivity and Loss of Efficacy Associated With the First 4 Exposure Days (EDs) Leading to Discontinuation

  The occurrence of the defined adverse events of special interest was documented during the first 4 days that a participant was exposed to the study intervention.

  Individual first 4 exposure days

Secondary Outcomes (6)

• Adverse Drug Reactions (ADRs)

  Up to 24 months

• Anti-drug Antibody (ADA) Development

  Up to 24 months

• The Number of Participants With Confirmed Factor VIII Inhibitors

  Up to 24 months

• Annualized Bleeding Rate (ABR)

  Up to 24 months

• BAY94-9027 Consumption

  Up to 24 months

Study Arms (1)

Main study (Part A) and the extension study (Part B)

EXPERIMENTAL

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

Biological: Damoctocog alfa pegol (Jivi, BAY94-9027)

Interventions

Damoctocog alfa pegol (Jivi, BAY94-9027) BIOLOGICAL

Part A: 40 IU/kg (up to 60 IU/kg at the investigator's discretion), two times per week (2x/week) with the first 4 infusions under medical supervision. Thereafter, participants will continue their treatment as home treatment. Dose may be increased up to 60 IU/kg if needed at any time during the study at the investigator's discretion. Part B: Each participant may continue on prophylaxis dose regimen as prescribed in part A (40 - 60 IU/kg, 2x per week) or adjustments to prophylaxis dose / dose frequency can be made at the investigator's discretion (based on the bleeding events and individual needs): Dose frequency may be decreased to every 5 days with a prophylaxis dose of 60 IU/kg.

Main study (Part A) and the extension study (Part B)

Eligibility Criteria

• Age: 7 Years - 11 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Participants with severe hemophilia A (participant's own FVIII activity \[FVIII:C\] \<1%)
• Participants must be previously treated with FVIII concentrate(s) (plasma derived or recombinant) for a minimum of 50 exposure days (EDs) at the time of signing the informed consent
• Participant has understood the study if appropriate for his age, informed consent must be signed by the parent, the participant can only sign the assent
• Willingness and ability of participants and/or parents /caregivers to complete training in the use of the electronic patient diary (EPD) and to document infusions during the study

You may not qualify if:

• History of FVIII inhibitors
• Current evidence of inhibitor to FVIII measured using the Nijmegen-modified Bethesda assay (≥0.6 BU/mL) at the time of screening (central laboratory)
• Any other inherited or acquired bleeding disorder in addition to hemophilia A (e.g. von Willebrand disease, hemophilia B)
• Known hypersensitivity or allergic reaction to drug substance, excipients or mouse or hamster protein
• Any other significant medical condition that the investigator feels would be a risk to the patient or would impede the study
• Requires any pre-medication to tolerate FVIII treatment (e.g. antihistamines)
• Planned major surgery during the study
• Any individual who is receiving chemotherapy, immune modulatory drugs other than anti-retroviral chemotherapy, or chronic use of oral or intravenous (IV) corticosteroids (\> 14 days) within the last 3 months
• Any individual who received commercially available subcutaneous factor substitution therapy (emicizumab) within the last 6 months
• 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

Sponsors & Collaborators

• Bayer: lead

Study Sites (17)

Arnold Palmer Hospital for Children

Orlando, Florida, 32806, United States

Location

Hospital de Niños Sor María Ludovica

La Plata, Buenos Aires, 1900, Argentina

Location

Instituo Hematología Arbesú

Godoy Cruz, Mendoza Province, 5501, Argentina

Location

Instituto de Hematología Dr. Rubén Dávoli

Rosario, Santa Fe Province, S2000CKF, Argentina

Location

HEMORIO

Rio de Janeiro, Rio de Janeiro, 20211-030, Brazil

Location

Hospital das Clínicas de Campinas - UNICAMP

Campinas, São Paulo, 13083-878, Brazil

Location

Hosp Clínicas Facult. Med. de Ribeirão Preto / USP

Ribeirão Preto, São Paulo, 14051-140, Brazil

Location

Irmandadade da Santa Casa de Misericordia de Sao Paulo (iSANTACASA)

São Paulo, São Paulo, 01223-001, Brazil

Location

McMaster Children's Hospital

Hamilton, Ontario, L8N 3Z5, Canada

Location

Ospedale Pediatrico Bambino Gesù - Oncoematologia, Trapianto Emopoietico e Terapie Cellulari

Roma (RM), Lazio, 00165, Italy

Location

OUS Rikshospitalet Klinisk Forskningspost Barn

Oslo, 372, Norway

Location

Acibadem Adana Hastanesi, Çocuk Sagligi ve Hastaliklari, Çocuk Hematoloji-Onkoloji Bölümü

Adana, 01130, Turkey (Türkiye)

Location

Hacettepe Üniversitesi Tip Fakültesi, Hacettepe Ihsan Dogramaci Çocuk Hastanesi,

Ankara, 06230, Turkey (Türkiye)

Location

Akdeniz Üniversitesi Tip Fakültesi Çocuk Sagligi ve Hastaliklari Anabilim Dali Çocuk Hematoloji ve Onkoloji Bilim Dali

Antalya, 07059, Turkey (Türkiye)

Location

Gaziantep Üniversitesi Tip Fakültesi, Sahinbey Arastirma Ve Uygulama Hastanesi, Çocuk Hematoloji ve Onkoloji Bilim Dali

Gaziantep, 27100, Turkey (Türkiye)

Location

Ege Üniversitesi Tip Fakültesi, Çocuk Sagligi ve Hastaliklari Anabilim Dali, Çocuk Hematoloji Bilim Dali

Izmir, 35100, Turkey (Türkiye)

Location

Ondokuz Mayis Üniversitesi Tip Fakültesi, Saglik Uygulama ve Arastirma Merkezi, Çocuk Sagligi ve Hastaliklari Anabilim Dali

Samsun, 55200, Turkey (Türkiye)

Location

Related Publications (2)

• Ozelo MC, Luciani M, Glosli H, Kavakli K, Samji N, Makris GC, Tueckmantel C, Enriquez MM, Oliveira LC, Gupta S, Arbesu MG, Davoli M, Chan AKC, Mancuso ME. Plain Language Summary on Safety and Efficacy of Damoctocog Alfa Pegol in Previously Treated Children Aged 7 to < 12 Years With Severe Haemophilia A in the Phase 3, Open Label Alfa-PROTECT Main Study. Eur J Haematol. 2026 Apr;116(4):471-472. doi: 10.1111/ejh.70078. Epub 2026 Jan 14.

  PMID: 41531337 DERIVED

• Ozelo MC, Luciani M, Glosli H, Kavakli K, Samji N, Makris GC, Tueckmantel C, Maas Enriquez M, Oliveira LC, Gupta S, Arbesu MG, Davoli M, Chan AKC, Mancuso ME. Safety and Efficacy of Damoctocog Alfa Pegol in Previously Treated Children Aged 7 to < 12 Years With Severe Haemophilia A in the Phase 3, Open Label Alfa-PROTECT Main Study. Eur J Haematol. 2026 Apr;116(4):435-442. doi: 10.1111/ejh.70059. Epub 2026 Jan 4.

  PMID: 41486550 DERIVED

MeSH Terms

Conditions

Hemophilia A

Interventions

Factor VIII

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Blood Coagulation Factors; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Protein Precursors; Biological Factors

Limitations and Caveats

Small sample size is a limitation in this study.

Results Point of Contact

• Title: Therapeutic Area Head
• Organization: Bayer

clinical-trials-contact@bayer.com

(+)1-888-84 22937

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 24, 2021
• First Posted: December 7, 2021
• Study Start: March 23, 2022
• Primary Completion: January 4, 2024
• Study Completion: June 25, 2025
• Last Updated: January 21, 2026
• Results First Posted: July 24, 2025

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will not share

Locations

BR (4); IT (1); CA (1); US (1); NO (1); TU (6); AR (3)