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NCT Number: NCT07692217

TG-INSIGHT With Joint POCUS, Hemostatic Potential in Patients With Severe Hemophilia A on Novel Replacement and Substitution FVIII Therapies

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.

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Key information

Conditions

Hemophilia A Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Coagulation Protein Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Factor VIII (FVIII) Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhagic Disorders

Age range

6 month and older

Sex eligibility

All sexes

Study type

Observational

Primary location

The University of Texas Southwestern Medical Center

Dallas, Texas, 75235, United States

Location contact

Ayesha Zia, MD

SUB_INVESTIGATOR

Guy Young, MD

SUB_INVESTIGATOR

Jessica Garcia, MD

CONTACT

[email protected]

214-456-7000

Jessica Garcia, MD

PRINCIPAL_INVESTIGATOR

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Half-life factor VIII based replacement therapy

Drug

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.

Other names: Altuviiio

Non-FVIII based replacement therapy

Drug

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.

Other names: Hemlibra

Primary outcomes

  1. Thrombin Generation

    Time frame: Thrombin generation assay will be measured at 5 time points within a week of receiving dose of medication

    To determine and compare in vivo thrombin generation parameters; endogenous thrombin potential (ETP) and peak thrombin (PT) for patients on efanesoctocog alfa or emicizumab

  2. Hemophilia Joint Health Score

    Time frame: Obtained at baseline and every 6 months

    To obtain and compare HJHS in patients on efanesoctocog alfa or emicizumab

Secondary outcomes

  1. Annualized bleeding rate

    Time frame: From enrollment, every 6 months, until 3 years

    To collect bleeding episodes in study participants and to calculate their respective total, treated, and joint annualized bleeding rates

  2. Health related quality of life - CHO-KLAT

    Time frame: From enrollment, every 6 months, until 3 years

    Canadian Hemophilia Outcomes-Kids Life Assessment Tool (CHO-KLAT) will be used to evaluate the impact of different treatment strategies. CHO-KLAT will be obtained and compared in study participants on efanesoctocog alfa or emicizumab.

  3. Health related quality of life - PedHAL

    Time frame: From enrollment, every 6 months, until 3 years

    PedHAL is a validated patient-reported outcome measure designed to assess self-perceived functional abilities in children with hemophilia. PedHAL will be obtained and compared in study participants on efanesoctocog alfa or emicizumab

  4. Health related quality of life - CATCH

    Time frame: From enrollment, every 6 months, until 3 years

    CATCH (Comprehensive Assessment Tool of Challenges in Hemophilia) is a validated, multi-version patient-reported outcome instrument designed to assess the real-world impact of hemophilia on individuals and their caregivers. CATCH will be obtained and compared in study participants on efanesoctocog alfa or emicizumab

  5. Joint Tissue Changes

    Time frame: From enrollment, every 6 months, until 3 years

    Point-of-care ultrasound using JADE protocol to evaluate joint tissue changes will be obtained and compared in study participants on efanesoctocog alfa or emicizumab

Study contacts

Contact information is provided by the study sponsor or research team.

Jessica Garcia, MD

CONTACT

[email protected]

214-456-7000

Sponsors and collaborators

Lead sponsor

University of Texas Southwestern Medical Center

Other

Collaborators

  • Sanofi

Registry information

Official study title

Hemostatic Potential in Patients With Severe Hemophilia A on Novel Replacement and Substitution FVIII Therapies (TG-INSIGHT With Joint POCUS)

Important dates

Study start
2026
Primary completion
2033
Study completion
2033
First posted
Jul 9, 2026
Registry last updated
Jul 9, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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