Hemophilia a with InhibitorsNCT03002480
Individualizing Hemophilia Bypassing Agent Therapy Utilizing Thromboelastography
A study testing Thromboelastography-Guided Treatment Regimen in people with Hemophilia a with Inhibitors, run by Children's Hospital Los Angeles.
Trial summary
This pilot study will investigate the use of thromboelastograph (TEG) to determine the primary bypassing agent for the management of bleeding in children and adults severe hemophilia A patients with inhibitors. Read more about this study
- Who is it for?
- Ages 4 to 70 years, men only
- Conditions the study is for
- Hemophilia a with Inhibitors
- What is being tested?
- Thromboelastography-Guided Treatment Regimen
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- Jan 2017to Jan 2019
- Phase
- Interventional
- Are there results?
- None posted in the registry
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 4 to 70 years
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Males, 4-60 years old, inclusive on Prophylaxis or On-Demand treatment
- Diagnosis of Hemophilia with active titer inhibitors (> 0.6 BU)
- Willing to alter their treatment regimen per study protocol
Who cannot join
- Bleeding disorder(s) other than hemophilia A with inhibitors
- Thrombocytopenia (platelet count <100,000K/µL)
- Any concurrent clinically significant major disease that, in the opinion of the investigator, would make the subject unsuitable for enrollment
- Participation within the past 30 days in a clinical study involving investigational drugs
- Planned major surgery within 30 days prior to screening or during the study period
About this study
Official title: Individualizing Bypassing Agent Therapy Utilizing TEG in Hemophilia Patients with Inhibitors
This pilot study will investigate the use of thromboelastograph (TEG) to determine the primary bypassing agent for the management of bleeding in children and adults severe hemophilia A patients with inhibitors.
The study will evaluate the TEG profile for each of the available bypassing agents (FEIBA and rFVIIa) in each participating patient, which will then determine which agent provides the most robust clot formation as measured by the TEG. This study will consist of screening visit and 2-4 pharmacokinetic studies to determine the "best" bypassing agent based on the TEG results. Patients will then be assigned that bypassing agent and dose for the treatment of their bleeding episodes (Prophylaxis or On-demand). Each patient will be then followed for a period of 6 months to monitor short-term safety of those patients whose bypassing agent was modified.
Read the detailed description
Currently, there are no laboratory assays that can assess the pharmaceutical activity of the available agents known collectively as bypassing agents, which negatively impacts the treating physician's ability to manage these patients, leaving decisions on the dosing regimen to trial and error. Both agents, FEIBA and recombinant activated factor VII (rFVIIa), are approved for the treatment of bleeding in inhibitor patients with recommended dosing regimens that are based on clinical trials. However, a significant amount of "experimenting" with other dosing regimens is used by physicians and patients.
The goal of this project is to demonstrate that TEG is an excellent biomarker for predicting, managing and individualizing the treatment with FEIBA and rFVIIa of this rare and difficult to treat patient population.
- Conditions
- Hemophilia a with Inhibitors
- Being tested
- Thromboelastography-Guided Treatment Regimen
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Thromboelastography-Guided Treatment RegimenOther
The thromboelastograph (TEG) will be used to determine the "best" bypassing agent for the management of subjects with severe hemophilia A with inhibitors. Patients will be assigned that bypassing agent and dose for the treatment of their bleeding episodes and subsequently will be followed for a period of 6 months to determine their annual bleeding rate.
| Study group | Role | What they receive |
|---|---|---|
| Severe Hemophilia A subjects w/ inhibitors | Other | Males age 4-70 years diagnosed with severe hemophilia A with inhibitors who are currently treated with prophylaxis or on-demand treatment will be enrolled. Receives: Other: Thromboelastography-Guided Treatment Regimen |
What is being measured
The primary outcome is the main question the study is designed to answer.
Elastic properties of a forming clot using thromboelastograph to determine best bypassing agent
This study will assess the feasibility of TEG-guided individualization of bypassing agent treatment of bleeding for severe hemophilia A patients with inhibitors.
Measured: 6 months
Secondary outcomes (2)
Thrombin generation assay measuring the formation of thrombin during clot formation
Thrombin generation assays will be performed to provide additional evidence supporting the TEG-guided individualization of bypassing agent treatment.
Measured: 6 months
Number of participants with treatment-related adverse events
The study will monitor short-term safety of patients whose bypassing agent treatment has been modified.
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Dec 2016 (estimated)
- Study startJan 2017
- Primary completionJan 2019 (estimated)
- Study completionJan 2019 (estimated)
Why it stopped: Study never opened.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia a with InhibitorsStudies of the same condition, open and under way.
- 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
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's Hospital Los Angeles
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Guy Young, Children's Hospital Los Angeles
- Investigators
- Guy Young, MD, Principal investigator, Children's Hospital Los Angeles
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03002480
- Sponsor's protocol ID
- CHLA-16-00501
- Status last verified
- Sep 2024
- First submitted
- 19 Dec 2016
- Last updated
- 26 Sep 2024
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 26 Sep 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Individualizing Bypassing Agent Therapy Utilizing TEG in Hemophilia Patients with Inhibitors
- ClinicalTrials.gov ID
- NCT03002480
- Acronym / sponsor ID
- CHLA-16-00501
- Conditions
- Hemophilia a with Inhibitors
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Study never opened.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Children's Hospital Los AngelesUniversity, hospital or other
- Study start
- Jan 2017 (actual)
- Primary completion
- Jan 2019 (estimated)
- Study completion
- Jan 2019 (estimated)
- First posted
- 23 Dec 2016 (estimated)
- 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 pilot study will investigate the use of thromboelastograph (TEG) to determine the primary bypassing agent for the management of bleeding in children and adults severe hemophilia A patients with inhibitors.
The study will evaluate the TEG profile for each of the available bypassing agents (FEIBA and rFVIIa) in each participating patient, which will then determine which agent provides the most robust clot formation as measured by the TEG. This study will consist of screening visit and 2-4 pharmacokinetic studies to determine the "best" bypassing agent based on the TEG results. Patients will then be assigned that bypassing agent and dose for the treatment of their bleeding episodes (Prophylaxis or On-demand). Each patient will be then followed for a period of 6 months to monitor short-term safety of those patients whose bypassing agent was modified.
Detailed description
Currently, there are no laboratory assays that can assess the pharmaceutical activity of the available agents known collectively as bypassing agents, which negatively impacts the treating physician's ability to manage these patients, leaving decisions on the dosing regimen to trial and error. Both agents, FEIBA and recombinant activated factor VII (rFVIIa), are approved for the treatment of bleeding in inhibitor patients with recommended dosing regimens that are based on clinical trials. However, a significant amount of "experimenting" with other dosing regimens is used by physicians and patients.
The goal of this project is to demonstrate that TEG is an excellent biomarker for predicting, managing and individualizing the treatment with FEIBA and rFVIIa of this rare and difficult to treat patient population.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Severe Hemophilia A subjects w/ inhibitors | Other | Other: Thromboelastography-Guided Treatment RegimenMales age 4-70 years diagnosed with severe hemophilia A with inhibitors who are currently treated with prophylaxis or on-demand treatment will be enrolled. |
Who may qualify
- Ages
- 4 to 70 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- Males, 4-60 years old, inclusive on Prophylaxis or On-Demand treatment
- Diagnosis of Hemophilia with active titer inhibitors (> 0.6 BU)
- Willing to alter their treatment regimen per study protocol
Key exclusion criteria (5 listed)
- Bleeding disorder(s) other than hemophilia A with inhibitors
- Thrombocytopenia (platelet count <100,000K/µL)
- Any concurrent clinically significant major disease that, in the opinion of the investigator, would make the subject unsuitable for enrollment
- Participation within the past 30 days in a clinical study involving investigational drugs
- Planned major surgery within 30 days prior to screening or during the study period
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 |
|---|---|---|
| Thromboelastography-Guided Treatment RegimenThe thromboelastograph (TEG) will be used to determine the "best" bypassing agent for the management of subjects with severe hemophilia A with inhibitors. Patients will be assigned that bypassing agent and dose for the treatment of their bleeding episodes and subsequently will be followed for a period of 6 months to determine their annual bleeding rate. | Other | Severe Hemophilia A subjects w/ inhibitors |
Primary outcome
Elastic properties of a forming clot using thromboelastograph to determine best bypassing agent
Time frame: 6 months
This study will assess the feasibility of TEG-guided individualization of bypassing agent treatment of bleeding for severe hemophilia A patients with inhibitors.
2 secondary outcomes
Thrombin generation assay measuring the formation of thrombin during clot formation
Time frame: 6 months
Number of participants with treatment-related adverse events
Time frame: 1 year
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Sep 2024.
- Individual participant dataSharing planned: No
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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. This study is not recruiting at the moment.
Guy Young, MD principal investigator
Children's Hospital Los Angeles
Questions about this trial
What is this clinical trial studying?
A study testing Thromboelastography-Guided Treatment Regimen in people with Hemophilia a with Inhibitors, run by Children's Hospital Los Angeles.
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 3 inclusion and 5 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: withdrawn, as of the sponsor's last update on 26 Sep 2024. Status can change between updates, so confirm with the study contact before making plans.
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?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Elastic properties of a forming clot using thromboelastograph to determine best bypassing agent (time frame: 6 months). 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 26 Sep 2024.