Prophylaxis Regimen for Hemophilia A Patients

NCT05036278 | Completed Phase 4 Results FDA Drug

• 2 other identifiers: 219242021-006191-16
• Study Type: interventional
• Target: 21
• Locations: 1 country
• Sites: 12

Brief Summary

Researchers are looking for a better way to treat people who have hemophilia A. Hemophilia A is a genetic bleeding disorder that is caused by the lack of a protein in the blood called "clotting factor 8" (FVIII). FVIII is naturally found in the blood where it causes the blood to clump together to help prevent and stop bleeding. People with lower levels of FVIII or with FVIII that does not work properly may bleed for a long time from minor wounds, have painful bleeding into joints, or have internal bleeding. The study treatment, Jivi (also called damoctocog alfa pegol), is already available for doctors to prescribe to people with hemophilia A to treat and prevent bleeding. It works by replacing the missing FVIII, or the FVIII that does not work properly. People with hemophilia A need frequent injections of FVIII products into the vein. So called standard half-life (SHL) products need to be given 2 to 4 times a week for the prevention of bleeding. In recent years, new products like Jivi called extended half-life (EHL) products have become available. These products last longer in the body so that they require to be given less often with injections every 3-5 days. Thus, these treatments may be easier and more comfortable to stick to in daily life. There is no general plan concerning the best amount of treatment and the frequency of injections for the prevention of bleeding, since the severity may be different and individual risk factors have to be considered. Doctors often decide on a treatment plan based on their experience. The main purpose of this study is to learn how well a new scoring approach works to select a treatment plan for the prevention of bleeding in people with hemophilia A who switch their treatment from SHL products to Jivi. Different types of information are used to calculate the risk score like bleeding history, certain biological factors, and physical activity of the participant. All participants will receive Jivi for 6 months. In the first four weeks, all participants will receive Jivi 2 times a week at a dose level of 40 IU per kilogram body weight (also known as 40 IU/kg/dose, recommended maximum dose is 6,000 IU). Then, based on their risk score, each participant will be assigned to one of three treatment plans:

• participants with a high risk remain on Jivi administration 2 times a week at 40 IU/kg/dose
• participants with a medium risk will switch to Jivi administration every 5 days at 50 IU/kg/dose
• participants with a low risk will switch to Jivi administration every 5 days at 50 IU/kg/dose and after 4 weeks to a less frequent administration (e.g., every 7 days) at 60 IU/kg/dose To check how well the new scoring approach works for choosing the right treatment plan, researchers will look at how many participants have a favourable outcome. This means that the participant has either fewer bleeding events vs. the pre-study treatment and takes Jivi less often or as often as the previous SHL treatment but with fewer bleeding events, or that the participant has a comparable number of bleeding events but needs to take Jivi less often than the previous treatment. Each participant will be in the study for approximately 7.5 months. During this time, 4 visits to the study site and 3 phone calls are planned. During the study, the doctors and their study team will: • do physical examinations • take blood samples • ask the participants questions about how they are feeling and what adverse events they are having. In addition, participants or their guardians are required to write down the dates of Jivi treatments and bleeding events in an electronic diary and to fill in different questionnaires on their quality of life, health status, work/ school productivity, pain, and treatment satisfaction. In addition, participants are expected to keep appointments for visits and to adhere to the assigned treatment regimen. An adverse event is any medical problem that a participant has during a study. Doctors keep track of all adverse events that happen in studies, even if they do not think the adverse events might be related to the study treatments.

Conditions

Hemophilia A; Prophylaxis of Bleeding

Outcome Measures

Primary Outcomes (1)

• Overall Number of Participants With Favorable Outcome on the Score-assigned Prophylaxis Regimen

  Overall number of participants with favorable outcomes after prophylaxis regimen assignment by a risk score, based on a participant's baseline phenotypic and biologic variables, when switching from a standard half-life (SHL) product to Jivi.

  up to 6 months

Secondary Outcomes (10)

• Annualized Bleeding Rate (ABR) (Total, Joint, Spontaneous)

  up to 6 months

• Change in Total ABR From Pre-study

  up to 6 months

• Change in the Frequency of Pre-study SHL Teratment to the Frequency of Jivi Administration (Infusions/Month)

  up to 6 months

• Occurrence of Participants With 0 and ≤ 1 Spontaneous Bleeds

  up to 6 months

• Change in Haemophilia Quality of Life Questionnaire (Haem-A-QoL or Haemo-QoL)

  up to 6 months

Study Arms (1)

Damoctocog alfa-pegol prophylaxis regimens

EXPERIMENTAL

Prophylaxis regimens: All participants will begin with prophylaxis 2x/week (40 IU/kg/dose (recommended maximum dose 6,000 IU)) Participants with a high risk score (\> 4) continue on prophylaxis 2x/week (40 IU/kg/dose). Participants with a medium risk score (2 to 4) will switch after 4 weeks to prophylaxis Q5D (50 IU/kg/dose). Participants with a low risk score (\< 2) will switch after 4 weeks to prophylaxis Q5D (50 IU/kg/dose) and then after 4 weeks to a less frequent (e.g. Q7D) regimen (60 IU/kg/dose).

Biological: Damoctocog alfa-pegol is a recombinant B-domain deleted human coagulation FVIII variant site specifically conjugated with a 60 kDa, branched (30 kDa each) polyethylene glycol (PEG).

Interventions

Damoctocog alfa-pegol is a recombinant B-domain deleted human coagulation FVIII variant site specifically conjugated with a 60 kDa, branched (30 kDa each) polyethylene glycol (PEG). BIOLOGICAL

Dosage Levels: * 40 IU/kg/dose two times per week * 50 IU/kg/dose every 5 days * 60 IU/kg/dose, Less frequent dosing (e.g. every 7 days), the total recommended maximum dose/infusion is 6000 IU.

Also known as: Jivi, BAY94-9027

Damoctocog alfa-pegol prophylaxis regimens

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Participants must be ≥ 12 years of age inclusive, at the time of signing the informed consent/assent.
• Previously treated patients (≥ 150 EDs) with congenital hemophilia A.
• Prophylaxis with any SHL FVIII product with a stable frequency for at least 6 consecutive months within the last 12 months prior to screening before entering the study and documented in medical records. Stable frequency is defined as a minimum 18 weeks of treatment in a 6 (consecutive) calendar month period in the 12 months prior to screening. Patients can be on any non-Jivi EHL between the 6-month stable SHL prophylaxis period and start of study treatment.
• Documented bleeding rate (ABR) while on stable frequency SHL prophylaxis for at least 6 consecutive months within the last 12 months prior to screening.
• No current evidence (≥ 0.6 BU/mL) of FVIII inhibitors. If a participant has had a positive inhibitor titer in the past (≥ 0.6 BU/mL on two occasions) but has been tolerized for at least 1 year since the last positive titer with at least 1 negative inhibitor assay test during that period, they can be enrolled. If a participant has had a positive inhibitor titer in the past (≥ 0.6 BU/mL) but did not require tolerization and has had at least 1 negative inhibitor assay test during a minimum period of at least 1 year since the last positive titer, they can be enrolled.
• If they are human immunodeficiency virus (HIV) positive, cluster of differentiation 4 (CD4+) lymphocyte count should be \> 200/mm\^3 within 1 year before entering the study and documented in medical records. -
• Participants who are willing to complete an electronic diary (eDiary).
• Contraceptive use should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
• For adolescent participants (≥ 12 to \< 18 years), a legal guardian must be available to help the study-site personnel ensure follow-up; accompany the participant to the study site on each assessment day according to the Schedule of Activities (SoA) (e.g. able to comply with scheduled visits, treatment plan, laboratory tests and other study procedures); consistently and consecutively be available to provide information on the participant using the PROs during the scheduled study visits; accurately and reliably dispense study intervention as directed.
• For adolescent participants, a legal guardian must be able to accurately maintain the child's take-home record, including items of general health.

You may not qualify if:

• Any other inherited or acquired bleeding disorder in addition to hemophilia A. Note: von Willebrand disease should be diagnosed per local clinical practice. Participants with a diagnosis of von Willebrand disease in medical records or diagnosed at the time of screening will be excluded.
• Platelet count \< 100,000/mm\^3
• Evidence of inhibitor to FVIII (≥ 0.6 BU/mL) within the last 1 year
• The participant is currently participating in another investigational drug study or has participated in a clinical study involving an investigational drug or device within 30 days of signing informed consent.
• The participant has a planned major surgery.
• Documentation of missing risk score parameters other than physical activity .
• Known hypersensitivity to the drug substance, excipients, or mouse or hamster protein.
• Any other significant medical condition that the investigator feels would be a risk to the participant or would impede the study.
• Close affiliation with the investigational site; e.g. a close relative of the investigator, dependent person (e.g. employee or student of the investigational site).
• Otherwise vulnerable participants (e.g. participants who are in custody by order of an authority).
• Judgment by the investigator that the participant should not participate in the study if the participant is unlikely to comply with study procedures (i.e. eDiary completion, clinic visits, phone updates), restrictions, and requirements.

Sponsors & Collaborators

• Bayer: lead

Study Sites (12)

Dr. Akshat Jain - Loma Linda University Medical Center

Loma Linda, California, 92354, United States

Location

Loma Linda University Children's Hospital - Hematology / Oncology

Loma Linda, California, 92354, United States

Location

Luskin Orthopaedic Institute for Children

Los Angeles, California, 90001, United States

Location

University of Colorado | Renal Research Office

Aurora, Colorado, 80045, United States

Location

UHealth - Sylvester Comprehensive Cancer Center

Miami, Florida, 33136, United States

Location

Aflac Cancer and Blood Disorders Center - Egleston Hospital

Atlanta, Georgia, 30301, United States

Location

Wellstar MCG Health Medical Center - Gynecology

Augusta, Georgia, 30912, United States

Location

Rush University Medical Center - Oncology

Chicago, Illinois, 60612, United States

Location

Stead Family Children's Hospital - Hematology / Oncology

Iowa City, Iowa, 52240, United States

Location

M Health Fairview Masonic Cancer Clinic - Clinics and Surgery Center

Minneapolis, Minnesota, 55455, United States

Location

Rutgers Robert Wood Johnson Medical School - OBGYN

New Brunswick, New Jersey, 08901, United States

Location

Gulf States Hemophilia & Thrombophilia Center- Texas Medical Center

Houston, Texas, 77030, United States

Location

Related Links

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MeSH Terms

Conditions

Hemophilia A

Interventions

Polyethylene Glycols; 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)

Ethylene Glycols; Glycols; Alcohols; Organic Chemicals; Polymers; Macromolecular Substances; Biomedical and Dental Materials; Manufactured Materials; Technology, Industry, and Agriculture; Blood Coagulation Factors; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Protein Precursors; Biological Factors

Results Point of Contact

• Title: Bayer Clinical Trials Contact
• Organization: Bayer AG

clinical-trials-contact@bayer.com

(+) 1-888-8422937

Publication Agreements

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

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 2, 2021
• First Posted: September 5, 2021
• Study Start: July 28, 2022
• Primary Completion: September 12, 2024
• Study Completion: September 12, 2024
• Last Updated: November 12, 2025
• Results First Posted: November 12, 2025

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will not share

Locations

US (12)