Hemophilia ANCT07692217
TG-INSIGHT With Joint POCUS, Hemostatic Potential in Patients With Severe Hemophilia A on Novel Replacement and Substitution FVIII Therapies
An observational study of Hemophilia A and Factor VIII (FVIII), run by University of Texas Southwestern Medical Center at 1 site in United States.
Trial 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… Read more about this study
- Who is it for?
- Ages 6 months and older
- Conditions the study is for
- Hemophilia A, Factor VIII (FVIII)
- What is being tested?
- Half-life factor VIII based replacement therapy and Non-FVIII based replacement therapy
- How many people
- 60 participants
- Where
- 1 siteUnited States
- Study dates
- 22 Jul 2026to May 2033
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 months and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
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)
Who may be able to join
- 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
Who cannot join
- Participants with active FVIII inhibitor (>0.5 BU/mL)
- Presence of an additional bleeding disorder other than hemophilia A
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
The University of Texas Southwestern Medical Center
Dallas, Texas, 75235
Jessica Garcia, MD, 214-456-7000, Jessica.Garcia@UTSouthwestern.edu
Jessica Garcia, MD
About this study
Official title: Hemostatic Potential in Patients With Severe Hemophilia A on Novel Replacement and Substitution FVIII Therapies (TG-INSIGHT With Joint POCUS)
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.
Read the 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. The investigators 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.
- Conditions
- Hemophilia A, Factor VIII (FVIII)
- Being tested
- Half-life factor VIII based replacement therapy, Non-FVIII based replacement therapy
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Half-life factor VIII based replacement therapyDrug
Also called Altuviiio
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.
Non-FVIII based replacement therapyDrug
Also called Hemlibra
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.
| Study group | Role | What they receive |
|---|---|---|
| Participants with hemophilia A | The study plans to recruit participants with severe (FVIII <1%) and moderate (FVIII 1-4%) HA. Receives: Drug: Half-life factor VIII based replacement therapy; Drug: Non-FVIII based replacement therapy |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 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
Measured: Obtained at baseline and every 6 months, until 3 years
Secondary outcomes (5)
Annualized bleeding rate
To collect bleeding episodes in study participants and to calculate their respective total, treated, and joint annualized bleeding rates
Measured: From enrollment, every 6 months, until 3 years
Health related quality of life - CHO-KLAT
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.
Measured: From enrollment, every 6 months, until 3 years
Health related quality of life - PedHAL
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
Measured: From enrollment, every 6 months, until 3 years
Health related quality of life - CATCH
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
Measured: From enrollment, every 6 months, until 3 years
Joint Tissue Changes
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
Measured: From enrollment, every 6 months, until 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Jul 2026
- Study start22 Jul 2026
- Primary completionMay 2033 (estimated)
- Study completionMay 2033 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia AStudies of the same condition, open and under way.
- More trials run by University of Texas Southwestern Medical CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Texas Southwestern Medical Center
- Sponsor type
- University, hospital or other
- Collaborators
- Sanofi
- Responsible party
- Principal investigator, Jessica Garcia, University of Texas Southwestern Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07692217
- Sponsor's protocol ID
- STU20260378
- Status last verified
- Sep 2026
- First submitted
- 26 May 2026
- Last updated
- 29 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Hemostatic Potential in Patients With Severe Hemophilia A on Novel Replacement and Substitution FVIII Therapies (TG-INSIGHT With Joint POCUS)
- ClinicalTrials.gov ID
- NCT07692217
- Acronym / sponsor ID
- STU20260378
- Conditions
- Hemophilia A, Factor VIII (FVIII)
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 60 participants (estimated)
- Lead sponsor
- University of Texas Southwestern Medical CenterUniversity, hospital or other
- Collaborators
- Sanofi
- Study start
- 22 Jul 2026 (actual)
- Primary completion
- May 2033 (estimated)
- Study completion
- May 2033 (estimated)
- First posted
- 9 Jul 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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. The investigators 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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Participants with hemophilia A | – | Drug: Half-life factor VIII based replacement therapy, Drug: Non-FVIII based replacement therapyThe study plans to recruit participants with severe (FVIII <1%) and moderate (FVIII 1-4%) HA. |
Who may qualify
- Ages
- 6 months and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- 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)
Key inclusion criteria (2 listed)
- 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
Key exclusion criteria (2 listed)
- Participants with active FVIII inhibitor (>0.5 BU/mL)
- Presence of an additional bleeding disorder other than hemophilia A
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Half-life factor VIII based replacement therapyAlso: AltuviiioChildren 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 | Participants with hemophilia A |
| Non-FVIII based replacement therapyAlso: HemlibraChildren 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. | Drug | Participants with hemophilia A |
Primary outcome
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
Hemophilia Joint Health Score
Time frame: Obtained at baseline and every 6 months, until 3 years
To obtain and compare HJHS in patients on efanesoctocog alfa or emicizumab
5 secondary outcomes
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Sep 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hemophilia A and Factor VIII (FVIII), run by University of Texas Southwestern Medical Center at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 29 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Jessica Garcia, MD (214-456-7000, Jessica.Garcia@UTSouthwestern.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Thrombin Generation (time frame: Thrombin generation assay will be measured at 5 time points within a week of receiving dose of medication). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Sep 2026.