Individualizing Hemophilia Prophylaxis Using Thromboelastography

Status Completed

Clinical Trial Summary

Currently dosing for prophylaxis is not individualized, and the general approach is to use a dose of 25-40 units/kg given 3 times per week or every other day. One of the issues with weight-based dosing is the possible over-treatment. This is likely due to the fact that laboratory tests are not sensitive enough at the low levels to support decision-making. The Thromboelastograph (TEG®) and Thromboelastometry (ROTEM®) are coagulation devices, which assess the dynamics of clot formation over time and have several characteristics which suggest they may provide important information for individualized prophylaxis treatment for our patients.

Clinical Trial Description

This is a pilot study in children and adults with severe hemophilia A that utilizes the TEG/ROTEM to personalize their prophylaxis treatment.

Primary objectives:

• Assess the feasibility of TEG/ROTEM-guided modification of the current prophylaxis regimen for individuals with severe hemophilia A. Specifically, to:

- Test and refine the operational protocol for using TEG/ROTEM to guide factor dosing

- Estimate the proportion of hemophilia A patients whose dose will be modified based on TEG/ROTEM results

- Monitor short term safety of patients whose dose has been modified.

Secondary objectives

- Perform thrombin generation assays to provide additional evidence supporting the TEG/ROTEM-guided dosing.

- Obtain preliminary data on the economic impact of TEG/ROTEM-guided hemophilia A dosing regimens.

- Obtain preliminary data on the potential improvement of subject/family burden resulting from TEG/ROTEM-guided regimen using generic and specific quality of life tools.

The study period will consist of screening, pharmacokinetic (PK) study and follow-up. For subjects whose prophylaxis treatment regimen is modified, the follow-up period will include 3 follow-up clinic visits and 4 follow-up phone calls; these patients will remain on study for approximately 6 months following completion of the PK-study then will have an end of the study visit (visit 9). Subjects whose treatment regimen is not altered will end their study participation after Visit 2 and continuing with their current prophylaxis regimen.

Study endpoints: A safety review will be conducted after the first 6 subjects complete 30 days with the extended prophylaxis treatment. If no safety issues are identified, the study will proceed, and second safety review will be after an additional 8 subjects complete 30 days of extended treatment. If a subject has two or more spontaneous bleeds in 30 days, the subject will be removed from the study. For subjects who did not have bleeds and completed at least 6 months of the study, ongoing prophylactic treatment will be decided by their treating physician. ;

Study Design

Related Conditions & MeSH terms

Hemophilia A

Clinical Trial Details

NCT number	NCT02582060
Study type	Interventional
Source	Children's Hospital Los Angeles
Contact
Status	Completed
Phase	N/A
Start date	May 20, 2014
Completion date	December 31, 2017

View Details

See also
Status	Clinical Trial	Phase
Completed	`NCT03834727` - Characterizing the Impact and Treatment of Reproductive Tract Bleeding on Women and Post-menarchal Girls With Bleeding Disorders
Completed	`NCT03191799` - A Study to Evaluate the Safety and Tolerability of Prophylactic Emicizumab in Hemophilia A Patients With Inhibitors	Phase 3
Completed	`NCT01599819` - BAX 855 Dose-Escalation Safety Study	Phase 1
Terminated	`NCT04541628` - Safety & Efficacy of Encapsulated Allogeneic FVIII Cell Therapy in Haemophilia A	Phase 1/Phase 2
Completed	`NCT02847637` - A Clinical Trial to Evaluate Prophylactic Emicizumab Versus no Prophylaxis in Hemophilia A Participants Without Inhibitors	Phase 3
Completed	`NCT04072237` - Study of Coagulation Faction VIIa Variant Marzeptacog Alfa (Activated) in Adult Subjects With Hemophilia	Phase 1
Completed	`NCT04085458` - Study to Gain More Information on How Safe and Effective Jivi Works in Patients With Severe Hemophilia A (Post-marketing Investigation)	Phase 4
Completed	`NCT04565236` - A Post Approval Commitment Study to Gain More Information on How Safe and Effective KOVALTRY is in Chinese Children, Adolescents /Adults With Severe Hemophilia A	Phase 4
Recruiting	`NCT05987449` - A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A	Phase 1/Phase 2
Active, not recruiting	`NCT04621916` - Preventing Inhibitor Recurrence Indefinitely	Phase 4
Not yet recruiting	`NCT02888223` - Pharmacokinetic Study of SCT800 in Previously Treated Patients With Hemophilia A	Phase 1
Completed	`NCT02528968` - National Study of a Pharmacokinetic-Focused Educational Package for Patients With Severe Haemophilia A	N/A
Completed	`NCT02225483` - Phenotypic Heterogeneity in Hemophilia A: An Investigation of the Role of Platelet Function	N/A
Completed	`NCT02199717` - An Institutional Pilot Study to Investigate Physical Activity Patterns in Boys With Hemophilia	N/A
Completed	`NCT01217255` - Comparing the Burden of Illness of Hemophilia in the Developing and the Developed World
Terminated	`NCT00995046` - Individually Tailored Prophylaxis in Patients With Severe Hemophilia A	N/A
Completed	`NCT00969319` - Effekt-2 - Efficacy and Safety of Long-term Treatment With KOGENATE® FS in Latin America	N/A
Completed	`NCT00868530` - Study Evaluating On-Demand Treatment Of Xyntha In Chinese Subjects	Phase 3
Completed	`NCT00839202` - Activity and Content of Factor VIII (FVIII) in Human Plasma: The Assessment of a Novel Immunoassay	N/A
Completed	`NCT00629837` - Pharmacokinetics and Safety of a Single Intravenous Infusion of BAY 79-4980	Phase 1