Hemophilia A Clinical Trial
Hemophilia A is a rare X chromosome-linked recessive bleeding disorder that concerns one
individual in 5000. In its severe form, hemophilia A is a life-threatening, crippling
hemorrhagic disease. The treatment of bleeding episodes in hemophilia A patients involves
the administration of exogenous human FVIII to restore normal hemostasis. The main
complication of the substitutive treatment of hemophilia A is the development, in 15 to 30%
of the cases, of anti-FVIII antibodies (FVIII inhibitors) that neutralize the pro-coagulant
activity of therapeutically administered FVIII. In 2003, the average annual cost of care for
a patient with hemophilia A was evaluated to be equal to 63,000 euros (2), which, in France
(6000 patients), represents an annual budget of 378 million euros.
In order to reduce the cost of treatment and to bypass this complication, different
therapeutic strategies (new products or adjunctive therapeutic options) have been explored,
including platelet infusion, tranexamic acid, amino caproic acid, molecules that block
tissue factor pathway inhibitor, combination of phospholipid -Factor Xa- Factor XIII and
antibodies directed to the Tissue Factor Inhibitor Pathway (TFPI). Recently, Soluble
thrombomodulin (Solulin) have been developed. This molecule may be used to partially correct
the premature lysis defect in Factor VIII deficient plasma through an activated TAFI -
dependent mechanism.
With a long half-life (15- to 30-hour) and effective dose range estimated to range from the
sub-nanomolar to approximately 40nM, Solulin could potentially be administered on a weekly
basis and provide the basis for a factor-sparing regime that would cut costs and make
therapy more widely available. However, before proceeding to advanced trials, safety
concerns stemming from the anticoagulant properties of Solulin must be addressed. The
development of Solulin mutants lacking protein C activation capacity would make this concern
redundant. At the same time, such mutant molecules are likely to possess an effective dose
range.
Our project is to compare the behavior of recombinant Solulin and mutants of Solulin lacking
protein C activation capacity with respect to their ability to stabilize fibrin clots in
whole blood of humans with different coagulation factor deficiencies (hemophilia A,
hemophilia B and rare blood coagulation deficiencies (factor X, VII, V).
n/a
Time Perspective: Prospective
| 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 |
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Study Evaluating On-Demand Treatment Of Xyntha In Chinese Subjects
|
Phase 3 | |
| Completed |
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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 |