TG-INSIGHT With Joint POCUS, Hemostatic Potential in Patients With Severe Hemophilia A on Novel Replacement and Substitution FVIII Therapies
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Half-life factor VIII based replacement therapy, Non-FVIII based replacement therapy.
- Who it may be relevant to
- Registry conditions: Hemophilia A, Factor VIII (FVIII). Basic parameters: from 6 months · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
Hemostatic Potential in Patients With Severe Hemophilia A on Novel Replacement and Substitution FVIII Therapies (TG-INSIGHT With Joint POCUS)
Overview
This is an observational research study to find out if there is a difference in the way children with moderate or severe hemophilia A, treated on two different types of factor replacement, form a clot and also evaluate if they develop tiny bleeds within the joint and subsequently early joint changes when receiving extended half-life factor VIII.
Detailed description
This research study aims to compare the differences in the way children with moderate or severe hemophilia A respond when being treated with prophylactic extended half-life factor VIII based replacement therapy (such as Altuviiio) vs non-FVIII based replacement therapy (such as Hemlibra) as part of their hemophilia treatment to prevent spontaneous joint bleeds. This study will collect information about the patient's joint health, patient outcomes (using questionnaires), and laboratory testing at specific time points over 3 years.
The study will use an overall approach of comparative effectiveness research (CER) methodology. We plan to recruit participants with severe (FVIII \<1%) and moderate (FVIII 1-4%) HA who take extended half-life factor VIII based replacement therapy or non-FVIII based replacement therapy as their clinically prescribed treatment. Both are standard of care treatment for persons with Hemophilia A (PwHA). Using CER principals, this study will analyze differences in 1) hemostatic potential and annualized bleeding rates (Aim 1-3) and 4) joint health (structural and functional) using the Hemophilia Joint Health Score (HJHS) and imaging markers to identify subclinical bleeding and early joint tissue changes in PwHA on novel FVIII and non-FVIII therapies already in use in practice (Aim 4). The proposed study will be the first systematic observational comparative effectiveness study in PwHA on two conventional, standard-of-care novel therapies with differing mechanisms of action to evaluate the overall effectiveness of treatment.
Interventions
- Drug Half-life factor VIII based replacement therapy
Children with moderate or severe hemophilia A being treated with prophylactic extended half-life factor VIII based replacement therapy (such as Altuviiio) as part of their hemophilia treatment to prevent spontaneous joint bleeds. - Drug Non-FVIII based replacement therapy
Children with moderate or severe hemophilia A being treated with non-FVIII based replacement therapy (such as Hemlibra) as part of their hemophilia treatment to prevent spontaneous joint bleeds.
Primary outcome measures
- Thrombin Generation [Time frame: Thrombin generation assay will be measured at 5 time points within a week of receiving dose of medication]
- Hemophilia Joint Health Score [Time frame: Obtained at baseline and every 6 months]
Secondary outcome measures (5)
- Annualized bleeding rate [Time frame: From enrollment, every 6 months, until 3 years]
- Health related quality of life - CHO-KLAT [Time frame: From enrollment, every 6 months, until 3 years]
- Health related quality of life - PedHAL [Time frame: From enrollment, every 6 months, until 3 years]
- Health related quality of life - CATCH [Time frame: From enrollment, every 6 months, until 3 years]
- Joint Tissue Changes [Time frame: From enrollment, every 6 months, until 3 years]
Eligibility criteria
Inclusion criteria
- Participants with moderate or severe HA who are on prophylaxis with either weekly, biweekly, or every 4-weeks emicizumab or weekly efanesoctocog alfa for at least 2 months.
- >6 months of age
Exclusion criteria
- Participants with active FVIII inhibitor (>0.5 BU/mL)
- Presence of an additional bleeding disorder other than hemophilia A
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
United States · 1 center
- The University of Texas Southwestern Medical Center — Dallas
Identifiers
NCT: NCT07692217 · STU20260378