Prophylaxis Regimen for Hemophilia A Patients

Phase 4

Recruiting

• Conditions: Hemophilia A; Prophylaxis of Bleeding

• Registration Number: NCT05036278
• Lead Sponsor: Bayer

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2021-9-2
• Last Update Posted: 2 September 2024

Recruitment & Eligibility

• Status: Recruiting
• Sex: All
• Target Recruitment: 25

Inclusion Criteria

Inclusion Criteria:<br><br> - Participants must be = 12 years of age inclusive, at the time of signing the<br> informed consent/assent.<br><br> - Previously treated patients (= 150 EDs) with congenital hemophilia A.<br><br> - Prophylaxis with any SHL FVIII product with a stable frequency for at least 6<br> consecutive months within the last 12 months prior to screening before entering the<br> study and documented in medical records. Stable frequency is defined as a minimum 18<br> weeks of treatment in a 6 (consecutive) calendar month period in the 12 months prior<br> to screening. Patients can be on any non-Jivi EHL between the 6-month stable SHL<br> prophylaxis period and start of study treatment.<br><br> - Documented bleeding rate (ABR) while on stable frequency SHL prophylaxis for at<br> least 6 consecutive months within the last 12 months prior to screening.<br><br> - No current evidence (= 0.6 BU/mL) of FVIII inhibitors. If a participant has had a<br> positive inhibitor titer in the past (= 0.6 BU/mL on two occasions) but has been<br> tolerized for at least 1 year since the last positive titer with at least 1 negative<br> inhibitor assay test during that period, they can be enrolled. If a participant has<br> had a positive inhibitor titer in the past (= 0.6 BU/mL) but did not require<br> tolerization and has had at least 1 negative inhibitor assay test during a minimum<br> period of at least 1 year since the last positive titer, they can be enrolled.<br><br> - If they are human immunodeficiency virus (HIV) positive, cluster of differentiation<br> 4 (CD4+) lymphocyte count should be > 200/mm^3 within 1 year before entering the<br> study and documented in medical records. -<br><br> - Participants who are willing to complete an electronic diary (eDiary).<br><br> - Contraceptive use should be consistent with local regulations regarding the methods<br> of contraception for those participating in clinical studies.<br><br> - For adolescent participants (= 12 to < 18 years), a legal guardian must be available<br> to help the study-site personnel ensure follow-up; accompany the participant to the<br> study site on each assessment day according to the Schedule of Activities (SoA)<br> (e.g. able to comply with scheduled visits, treatment plan, laboratory tests and<br> other study procedures); consistently and consecutively be available to provide<br> information on the participant using the PROs during the scheduled study visits;<br> accurately and reliably dispense study intervention as directed.<br><br> - For adolescent participants, a legal guardian must be able to accurately maintain<br> the child's take-home record, including items of general health.<br><br>Exclusion Criteria:<br><br> - Any other inherited or acquired bleeding disorder in addition to hemophilia A. Note:<br> von Willebrand disease should be diagnosed per local clinical practice. Participants<br> with a diagnosis of von Willebrand disease in medical records or diagnosed at the<br> time of screening will be excluded.<br><br> - Platelet count < 100,000/mm^3<br><br> - Evidence of inhibitor to FVIII (= 0.6 BU/mL) within the last 1 year<br><br> - The participant is currently participating in another investigational drug study or<br> has participated in a clinical study involving an investigational drug or device<br> within 30 days of signing informed consent.<br><br> - The participant has a planned major surgery.<br><br> - Documentation of missing risk score parameters other than physical activity .<br><br> - Known hypersensitivity to the drug substance, excipients, or mouse or hamster<br> protein.<br><br> - Any other significant medical condition that the investigator feels would be a risk<br> to the participant or would impede the study.<br><br> - Close affiliation with the investigational site; e.g. a close relative of the<br> investigator, dependent person (e.g. employee or student of the investigational<br> site).<br><br> - Otherwise vulnerable participants (e.g. participants who are in custody by order of<br> an authority).<br><br> - Judgment by the investigator that the participant should not participate in the<br> study if the participant is unlikely to comply with study procedures (i.e. eDiary<br> completion, clinic visits, phone updates), restrictions, and requirements.

Exclusion Criteria

Not provided

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Occurrence of favorable outcome on the score selected dosing regimen

Secondary Outcome Measures

Name	Time	Method
ABR (total, joint, spontaneous);Change in total ABR from pre-study;Change in the frequency of pre-study SHL treatment to the frequency of Jivi administration (infusions/month);Occurrence of participants with 0 and = 1 spontaneous bleeds;Change in Haemophilia Quality of Life Questionnaire (Haem-A-QoL or Haemo-QoL);Patient's Global Impression of Change (PGI-C);EuroQoL 5 Dimensions (EQ-5D-5L) questionnaire;Treatment Satisfaction Questionnaire for Medication (TSQM);Work Productivity and Activity Impairment (WPAI) questionnaire scores;Number of target joints and change in target joint status from baseline