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

NCT07692217 | Not Yet Recruiting DMC

• 1 other identifier: STU20260378
• Study Type: observational
• Target: 60
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Hemophilia A; Factor VIII (FVIII)

Outcome Measures

Primary Outcomes (2)

• Thrombin Generation

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

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

• Hemophilia Joint Health Score

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

  Obtained at baseline and every 6 months

Secondary Outcomes (5)

• Annualized bleeding rate

  From enrollment, every 6 months, until 3 years

• Health related quality of life - CHO-KLAT

  From enrollment, every 6 months, until 3 years

• Health related quality of life - PedHAL

  From enrollment, every 6 months, until 3 years

• Health related quality of life - CATCH

  From enrollment, every 6 months, until 3 years

• Joint Tissue Changes

  From enrollment, every 6 months, until 3 years

Study Arms (1)

Participants with hemophilia A

The study plans to recruit participants with severe (FVIII \<1%) and moderate (FVIII 1-4%) HA.

Drug: Half-life factor VIII based replacement therapy; Drug: Non-FVIII based replacement therapy

Interventions

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.

Also known as: Altuviiio

Participants with hemophilia A

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.

Also known as: Hemlibra

Participants with hemophilia A

Eligibility Criteria

• Age: 6 Months+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

* Children (defined as persons who have not attained the legal age for consent to treatments or procedures involved in the research, under the applicable law of the jurisdiction in which the research will be conducted. In Texas the legal age is 18.) * Non-English Speakers * Patients (defined as individuals in a clinical setting with whom there is a treatment relationship)

You may qualify if:

• 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

You may not qualify if:

• Participants with active FVIII inhibitor (\>0.5 BU/mL)
• Presence of an additional bleeding disorder other than hemophilia A

Sponsors & Collaborators

• University of Texas Southwestern Medical Center: lead
• Sanofi: collaborator

Study Sites (1)

The University of Texas Southwestern Medical Center

Dallas, Texas, 75235, United States

Location

Biospecimen

Retention: SAMPLES WITH DNA

Blood will be taken from a vein in the patient's arm for research testing.

MeSH Terms

Conditions

Hemophilia A

Interventions

emicizumab

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

Central Study Contacts

Jessica Garcia, MD

CONTACT

214-456-7000; Jessica.Garcia@UTSouthwestern.edu

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Assistant Professor of Pediatrics

Study Record Dates

• First Submitted: May 26, 2026
• First Posted: July 9, 2026
• Study Start: July 1, 2026
• Primary Completion (Estimated): May 1, 2033
• Study Completion (Estimated): May 1, 2033
• Last Updated: July 9, 2026

Record last verified: 2026-07

Locations

US (1)