Prophylaxis Regimen for Hemophilia A Patients (PREDICT)
Hemophilia A, Prophylaxis of Bleeding
About this trial
This is an interventional treatment trial for Hemophilia A
Eligibility Criteria
Inclusion Criteria:
- 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 dose/regimen for at least 12 months (including at the time of screening) before entering the study and documented in medical records.
- Documented bleeding rate (ABR) for at least the 6 months prior to study entry.
- No history or current evidence (≥ 0.6 BU/mL) of FVIII inhibitors.
- 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 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.
Exclusion Criteria:
- Any other inherited or acquired bleeding disorder in addition to hemophilia A.
- Platelet count < 100,000/mm^3
- 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
. - 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), restrictions, and requirements.
Sites / Locations
- Dr. Akshat Jain - Loma Linda University Medical CenterRecruiting
- Orthopaedic Institute for ChildrenRecruiting
- University of California - DavisRecruiting
- University of Colorado HospitalRecruiting
- University of Miami HospitalRecruiting
- Aflac Cancer and Blood Disorders CenterRecruiting
- Augusta University Medical CenterRecruiting
- Rush University Medical CenterRecruiting
- The University of Iowa - Div of Sponsored ProgramsRecruiting
- Johns Hopkins Univ School Med|Sidney Kimmel Comp Cancer CntrRecruiting
- Michigan State University
- University of Minnesota Medical CenterRecruiting
- Rutgers Robert Wood Johnson Medical SchoolRecruiting
- Gulf States Hemophilia & Thrombophilia Center-UT PhysiciansRecruiting
Arms of the Study
Arm 1
Experimental
Damoctocog alfa-pegol prophylaxis regimens
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).