NCT07101926

Brief Summary

During the development of anti-TFPI antibodies, thrombin generation assay (TGA) was employed using both in vitro measurements (antibodies added to blood samples) and ex vivo approaches (blood samples from patients in phase II and III trials). While a significant improvement in thrombin generation was observed in all samples from patients with severe hemophilia, no correlation with clinical outcomes could be established. Notably, thrombin peak levels were consistently improved even in patients who experienced bleeding episodes. These measurements were conducted in platelet-poor plasma (PPP) with standard reagents, which may not adequately reflect the hemostatic efficacy of anti-TFPI antibodies given their mechanism of action. It is hypothesized that optimizing reagents and utilizing more appropriate biological materials could enhance TGA sensitivity, as previously demonstrated for monitoring emicizumab. The absence of a laboratory assay to monitor anti-TFPI (tissue factor pathway inhibitor) antibodies poses a significant challenge for managing patients in surgical settings and treating acute severe bleeding. This study aims to develop a reliable assay to evaluate the hemostatic efficacy of anti-TFPI antibodies and their combined procoagulant effect with factor concentrates (FVIII or FIX) or bypassing agents.

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
11

participants targeted

Target at below P25 for all trials

Timeline
2mo left

Started Oct 2025

Shorter than P25 for all trials

Geographic Reach
1 country

2 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Progress70%
Oct 2025Aug 2026

First Submitted

Initial submission to the registry

July 28, 2025

Completed
6 days until next milestone

First Posted

Study publicly available on registry

August 3, 2025

Completed
3 months until next milestone

Study Start

First participant enrolled

October 23, 2025

Completed
9 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 1, 2026

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

August 1, 2026

Last Updated

March 23, 2026

Status Verified

March 1, 2026

Enrollment Period

9 months

First QC Date

July 28, 2025

Last Update Submit

March 19, 2026

Conditions

Outcome Measures

Primary Outcomes (1)

  • Development of a Laboratory Assay to Assess Hemostatic Efficacy of Anti-TFPI Antibodies

    Assessment of the thrombin generation assay's ability to evaluate the procoagulant effect of anti-TFPI antibodies alone and in combination with factor concentrates (FVIII or FIX) or bypassing agents (rFVIIa, aPCC).

    At time of sample analysis (single visit; Day 0)

Secondary Outcomes (4)

  • Analytical Sensitivity of TGA to Anti-TFPI Antibodies

    Day 0

  • Identification of Optimal TGA Parameters for Monitoring Anti-TFPI Effect

    Day 0

  • Correlation Between TGA Parameters and Clinical Use of Anti-TFPI Therapies

    Day 0

  • Detection of Hypercoagulability from Combined Therapy in TGA

    Day 0

Study Arms (1)

Severe adult haemophilia A or B patient with factor levels ≤2%

* 6 on prophylaxis with FVIII or FIX concentrates after an adequate washout period of 48h for SHL FVIII molecules, at least 4 days for EHL-FVIII Fc and at least 10 days for EHL-FIX molecules * 5 receiving prophylaxis with marstacimab.

Biological: blood sampling

Interventions

blood samplingBIOLOGICAL

One-time peripheral blood draw (10.8 mL total, 4 citrated tubes) collected during a routine clinical visit for thrombin generation testing on platelet-rich and platelet-poor plasma. No additional medical procedures or treatments are involved in the study.

Severe adult haemophilia A or B patient with factor levels ≤2%

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

Potentials patients for this study will be identified within the department of clinic hemostasis at Louis Pradel Hospital and Necker Hospital during their routine visit. Hemophila patients are coming regularly to the hospital for their consultaion and a blood drawn

You may qualify if:

  • \- Male Patient
  • years old
  • severe hemophilia patients A or B (FVIII \< 1%,FIX\<=2%)
  • on prophylaxis with FVIII or IX concentrates after an adequate washout period of 48h for SHL FVIII molecules, at least 4 days for EHL-FVIII Fc and at least 10 days for EHL-FIX molecules.
  • On prophylaxis with Marstacimab
  • Willing to participate
  • Capable of following protocol procedures under investigator appreciation

You may not qualify if:

  • \- patients refusing to provide 4 additional blood tubes for research
  • Patient with an other coagulation disorder
  • patients who received an injection of FVIII or FIX during the required washout period

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

Groupement hospitalier Est Hôpital Cardilogique Service d'hémostase clinique

Bron, 69677, France

RECRUITING

Centre de Référence Hémophilie et autres déficits rares en protéine de la coagulationCentre de Traitemendes Hémophiles F. Josso

Paris, 75015, France

NOT YET RECRUITING

Biospecimen

Retention: SAMPLES WITHOUT DNA

The specific types of samples that will be retained include platelet-rich plasma (PRP) and platelet-poor plasma (PPP) derived from citrated whole blood. During a routine visit, four citrate tubes of 2.7 mL each (totaling 10.8 mL) will be collected per patient. PRP is prepared by centrifugation at 150 × g for 10 minutes at 18°C within 30 minutes of collection. The plasma is carefully transferred to avoid leukocyte contamination and adjusted to a standardized platelet count if necessary. These samples will be used for thrombin generation testing, specifically via the Calibrated Automated Thrombogram (CAT) method, and will not be stored for long-term use-no long-term storage is planned.

MeSH Terms

Conditions

Hemophilia A

Interventions

Blood Specimen Collection

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, InheritedBlood Coagulation DisordersHematologic DiseasesHemic and Lymphatic DiseasesCoagulation Protein DisordersHemorrhagic DisordersGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Specimen HandlingClinical Laboratory TechniquesDiagnostic Techniques and ProceduresDiagnosisPuncturesSurgical Procedures, OperativeInvestigative Techniques

Central Study Contacts

Study Design

Study Type
observational
Observational Model
CASE ONLY
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

July 28, 2025

First Posted

August 3, 2025

Study Start

October 23, 2025

Primary Completion (Estimated)

August 1, 2026

Study Completion (Estimated)

August 1, 2026

Last Updated

March 23, 2026

Record last verified: 2026-03

Locations