Treatment of BleedingPhase 3Alfa-PROTECTNCT05147662
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
A Phase 3 study testing Damoctocog alfa pegol (Jivi, BAY94-9027) in people with Treatment of Bleeding and Prophylaxis of Bleeding, run by Bayer at 18 sites in 7 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 7 to 11 years, men only
- Conditions the study is for
- Treatment of Bleeding, Prophylaxis of Bleeding, Hemophilia A
- What is being tested?
- Damoctocog alfa pegol (Jivi, BAY94-9027)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 36 participants
- Where
- 18 sitesin 7 countries
- Study dates
- 23 Mar 2022to 25 Jun 2025
- Phase
- Phase 3
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 7 to 11 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- 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
Who cannot join
- 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)
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- 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
Trial locations and contacts
18 sites in 7 countries.
18 sites in 7 countries
Turkey (Türkiye)6 sites
Acibadem Adana Hastanesi, Çocuk Sagligi ve Hastaliklari, Çocuk Hematoloji-Onkoloji Bölümü
Adana, 01130
Hacettepe Üniversitesi Tip Fakültesi, Hacettepe Ihsan Dogramaci Çocuk Hastanesi,
Ankara, 06230
Akdeniz Üniversitesi Tip Fakültesi Çocuk Sagligi ve Hastaliklari Anabilim Dali Çocuk Hematoloji ve Onkoloji Bilim Dali
Antalya, 07059
Gaziantep Üniversitesi Tip Fakültesi, Sahinbey Arastirma Ve Uygulama Hastanesi, Çocuk Hematoloji ve Onkoloji Bilim Dali
Gaziantep, 27100
Ege Üniversitesi Tip Fakültesi, Çocuk Sagligi ve Hastaliklari Anabilim Dali, Çocuk Hematoloji Bilim Dali
Izmir, 35100
Ondokuz Mayis Üniversitesi Tip Fakültesi, Saglik Uygulama ve Arastirma Merkezi, Çocuk Sagligi ve Hastaliklari Anabilim Dali
Samsun, 55200
Brazil4 sites
Hosp Clínicas Facult. Med. de Ribeirão Preto / USP
Ribeirão Preto, São Paulo, 14051-140
HEMORIO
Rio de Janeiro, Rio de Janeiro, 20211-030
Irmandadade da Santa Casa de Misericordia de Sao Paulo (iSANTACASA)
São Paulo, São Paulo, 01223-001
Hospital das Clínicas de Campinas - UNICAMP
Uberlândia, Minas Gerais, 13083-878
Argentina3 sites
Instituo Hematología Arbesú
Godoy Cruz, Mendoza Province, 5501
Hospital de Niños Sor María Ludovica
La Plata, Buenos Aires, 1900
Instituto de Hematología Dr. Rubén Dávoli
Rosario, Santa Fe Province, S2000CKF
Canada2 sites
Alberta Children's Hospital | Hematology / Oncology / Blood and Marrow Transplant Clinic
Calgary, Alberta, T3B 6A8
McMaster University Medical Centre | McMaster Children's Hospital | Hematology
Hamilton, Ontario, L8N 3Z5
United States1 site
Arnold Palmer Hospital for Children
Orlando, Florida, 32806
Italy1 site
Ospedale Pediatrico Bambino Gesù - Oncoematologia, Trapianto Emopoietico e Terapie Cellulari
Roma (RM), Lazio, 00165
About this study
Official title: A Phase 3, Single Group Treatment, Open-label, Study to Evaluate the Safety of BAY 94-9027 Infusions for Prophylaxis and Treatment of Bleeding in Previously Treated Children Aged 7 to <12 Years With Severe Hemophilia A
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.
Read more
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
- 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)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.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Biological: Damoctocog alfa pegol (Jivi, BAY94-9027) |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Individual first 4 exposure days
Secondary outcomes (8)
Adverse Drug Reactions (ADRs)
Adverse drug reactions were defined as any adverse events where a causal relationship of at least possibly related with the use of BAY 94-9027 had been ascribed by the investigator. In this study, treatment emergent study drug-related adverse events were used in defining ADRs.
Measured: From start of treatment until completion of study part A, up to 26 weeks, with a mean and median duration of study intervention of 182 days in part A
Number of Participants With Anti-drug Antibody (ADA) Development
All participants were tested for the development of anti-drug antibodies (ADAs) (anti-polyethylene glycol [PEG] and anti-PEG immunoglobulin M [IgM]).
Measured: 2nd exposure day in week 1 (visit 3), 3rd exposure day in week 2 (visit 4), 4th exposure day in week 2 (visit 5), after 8 weeks (visit 7), individual last visit in part A (visit 11, after 26 weeks, up to week 28)
The Number of Participants With Confirmed Factor VIII Inhibitors
A positive FVIII inhibitor test was defined with a threshold of = 0.6 BU/mL at the central laboratory. The first positive measurement was confirmed by a second, different sample.
Measured: From start of treatment until completion of study part A, up to 26 weeks, with a mean and median duration of study intervention of 182 days in part A
Annualized Bleeding Rate (ABR)
For each participant, the number of bleeds was related to the individual observation period to assess bleeding rates. For descriptive analyses, bleeding rates were annualized at the individual participant level using the formula: ABR = (number of bleeds x 365.25 x 24 x 60)/(Period). Period was defined as the number of minutes calculated from the date and time of the beginning of the treatment period and the date and time of the end of the treatment period of interest.
Measured: From start of treatment in study part A until the end of the study (after part B), planned total of 24 months, with a mean and median duration on study intervention of 182/182 days in part A, and of 503/508 days in part B
BAY94-9027 Consumption Per Infusion
Summary statistics for BAY 94-9027 consumption are provided per infusion, as dose per body weight (IU/kg).
Measured: From start of treatment in study part A until the end of the study (after part B), planned total of 24 months, with a mean and median duration on study intervention of 182/182 days in part A, and of 503/508 days in part B
BAY94-9027 Consumption Per Year
Summary statistics for BAY 94-9027 consumption are provided per year, as annual dose per body weight (IU/kg/year).
Measured: From start of treatment in study part A until the end of the study (after part B), planned total of 24 months, with a mean and median duration on study intervention of 182/182 days in part A, and of 503/508 days in part B
Number of Infusions Per Month
Number of infusions per month
Measured: From start of treatment in study part A until the end of the study (after part B), planned total of 24 months, with a mean and median duration on study intervention of 182/182 days in part A, and of 503/508 days in part B
Number of Infusions Per Year
Number of infusions per year (Annualized Infusion Rate)
Measured: From start of treatment in study part A until the end of the study (after part B), planned total of 24 months, with a mean and median duration on study intervention of 182/182 days in part A, and of 503/508 days in part B
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Dec 2021
- Study start23 Mar 2022
- Primary completion4 Jan 2024
- Study completion25 Jun 2025
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.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Treatment of BleedingStudies 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 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 36 participants (actual)
Sponsor and investigators
- Lead sponsor
- Bayer
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05147662
- Sponsor's protocol ID
- 21824
- Other IDs
- 2023-504388-18-00, 2021-004858-30, 2023
- Acronym
- Alfa-PROTECT
- Status last verified
- Jun 2026
- First submitted
- 24 Nov 2021
- Last updated
- 2 Jul 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: There are no current plans to share data. Interested researchers can use www.vivli.org 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 member section of the portal.
Publications
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.
PubMed 41531337Ozelo 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.
PubMed 41486550